GERON CORP - Annual Report 2025 | FinancialReports.eu

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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 10-K

(Mark One)

x ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the Fiscal Year Ended December 31 , 2025

o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from __ to ____.

Commission File Number: 000-20859

GERON CORP ORATION

(Exact name of registrant as specified in its charter)

Delaware (State or other jurisdiction of incorporation or organization)	75-2287752 (I.R.S. Employer Identification No.)
919 East Hillsdale Blvd. , Suite 250 , Foster City , CA (Address of principal executive offices)	94404 (Zip Code)

Registrant’s telephone number, including area code: ( 650 ) 473-7700

Securities registered pursuant to Section 12(b) of the Act: — Title of each class:	Trading symbol(s):	Name of each exchange on which registered:
Common Stock, $0.001 par value	GERN	The Nasdaq Stock Market LLC

Securities registered pursuant to Section 12(g) of the Act: None

Indicate by check mark if the registrant is a well‑known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes x No o

Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Act. Yes o No

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities

Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2)

has been subject to such filing requirements for the past 90 days. Yes x No o

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted

pursuant to Rule 405 of Regulation S‑T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant

was required to submit such files). Yes x No o

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non‑accelerated filer, a smaller

reporting company, or emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting

company,” and “emerging growth company” in Rule 12b‑2 of the Exchange Act.

Large accelerated filer	x	Accelerated filer	o
Non-accelerated filer	o	Smaller reporting company	o
		Emerging growth company	o

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for

complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. o

Indicate by check mark whether the registrant has filed a report on and attestation to its management’s assessment of the

effectiveness of its internal control over financial reporting under Section 404(b) of the Sarbanes-Oxley Act (15 U.S.C. 7262(b)) by the

registered public accounting firm that prepared or issued its audit report. x

If securities are registered pursuant to Section 12(b) of the Act, indicate by check mark whether the financial statements of the

registrant included in the filing reflect the correction of an error to previously issued financial statements. o

Indicate by check mark whether any of those error corrections are restatements that required a recovery analysis of incentive-based

compensation received by any of the registrant’s executive officers during the relevant recovery period pursuant to §240.10D-1(b). o

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b‑2 of the Act). Yes o No x

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The aggregate market value of voting and non‑voting common equity held by non‑affiliates of the registrant was approximately

$ 860,500,000 based upon the closing price of the registrant’s common stock on June 30, 2025 on the Nasdaq Global Select Market. The

calculation of the aggregate market value of voting and non‑voting common equity held by non‑affiliates of the registrant excludes shares of

common stock held by each officer, director and stockholder that the registrant concluded were affiliates on that date. This determination of

affiliate status is not necessarily a conclusive determination for other purposes.

As of February 20, 2026 , there were 640,544,661 s hares of common stock outstanding.

DOCUMENTS INCORPORATED BY REFERENCE:

Document	Form 10‑K Parts
Portions of the Registrant’s definitive proxy statement for the 2026 annual meeting of stockholders to be filed pursuant to Regulation 14A within 120 days of the Registrant’s fiscal year ended December 31, 2025.	III

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TABLE OF CONTENTS

		Page
PART I
Item 1.	Business	5
Item 1A.	Risk Factors	26
Item 1B.	Unresolved Staff Comments	77
Item 1C.	Cybersecurity	77
Item 2.	Properties	78
Item 3.	Legal Proceedings	79
Item 4.	Mine Safety Disclosures	79
PART II
Item 5.	Market for the Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities	80
Item 6.	[Reserved]	80
Item 7.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	81
Item 7A.	Quantitative and Qualitative Disclosures About Market Risk	96
Item 8.	Financial Statements and Supplementary Data	97
Item 9.	Changes in and Disagreements with Accountants on Accounting and Financial Disclosure	139
Item 9A.	Controls and Procedures	139
Item 9B.	Other Information	140
Item 9C.	Disclosure Regarding Foreign Jurisdictions that Prevent Inspections	140
PART III
Item 10.	Directors, Executive Officers and Corporate Governance	141
Item 11.	Executive Compensation	141
Item 12.	Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters	141
Item 13.	Certain Relationships and Related Transactions, and Director Independence	141
Item 14.	Principal Accountant Fees and Services	142
PART IV
Item 15.	Exhibits and Financial Statement Schedules	143
Item 16.	Form 10‑K Summary	146
	SIGNATURES	147

RYTELO ® and other trademarks or service marks of Geron Corporation appearing in this Annual Report on Form 10-K

(this "Report") are the property of Geron Corporation. This Report contains additional trade names, trademarks and service

marks of others, which are the property of their respective owners. We do not intend our use or display of other companies’

trade names, trademarks or service marks to imply a relationship with, or endorsement or sponsorship of us by, these other

companies.

In this Report, unless otherwise indicated or the context otherwise requires, “Geron,” “the registrant,” “we,” “us,” and

“our” refer to Geron Corporation, a Delaware corporation, and its wholly owned subsidiaries, Geron UK Limited, a United

Kingdom company, and Geron Netherlands, B.V., a Dutch company.

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Forward‑Looking Statements

This Report, including “Business” in Part I, Item 1 of this Report and “Management’s Discussion and

Analysis of Financial Condition and Results of Operations” in Part II, Item 7 of this Report, contains forward‑looking

statements that involve risks and uncertainties, as well as assumptions that, if they never materialize or prove incorrect,

could cause the results of Geron Corporation, or Geron or the Company, to differ materially from those expressed or

implied by such forward‑looking statements. All statements other than statements of historical fact are statements that

could be deemed forward‑looking statements. In some cases, forward‑looking statements can be identified by the use of

terminology such as “may,” “expects,” “plans,” “intends,” “will,” “should,” “could,” “projects,” “believes,” “predicts,”

“anticipates,” “estimates,” “potential,” “seek,” or “continue” or the negative thereof or other comparable terminology. The

risks and uncertainties referred to above include, without limitation, risks and uncertainties related to: (a) whether we are

successful in commercializing RYTELO (imetelstat) for the treatment of certain patients with lower-risk myelodysplastic

syndromes, or lower-risk MDS, with transfusion dependent anemia; (b) whether the U.S. Food and Drug Administration, or

FDA, or European Commission, or EC, will approve RYTELO for other indications on the timelines that may be expected,

or at all; (c) our pursuit of paths to make RYTELO available to eligible patients with lower-risk MDS outside of the U.S.,

including in the European Union, or EU; (d) whether we overcome potential delays and other adverse impacts caused by

enrollment, clinical, safety, efficacy, technical, scientific, intellectual property, manufacturing and regulatory challenges in

order to have the financial resources for and meet expected timelines and planned milestones; (e) whether regulatory

authorities permit the further development of imetelstat on a timely basis, or at all, without any clinical holds; (f) whether

RYTELO may cause, or have attributed to it, adverse events that could delay or prevent the commencement and/or

completion of clinical trials, impact its regulatory approval, or limit its commercial potential; (g) whether the IMpactMF

Phase 3 trial for relapsed/refractory myelofibrosis, or R/R MF, has a positive outcome and demonstrates safety and

effectiveness to the satisfaction of the FDA and international regulatory authorities, and whether our projected rates for

death events differ from actual rates, which may cause the planned interim and final analyses to occur later than

anticipated; (h) whether any future safety or efficacy results of RYTELO treatment cause its benefit-risk profile to become

unacceptable; (i) whether imetelstat actually demonstrates disease-modifying activity in patients and the ability to target the

malignant stem and progenitor cells of the underlying disease; (j) whether we meet our post-marketing requirements and

commitments for RYTELO; (k) whether there are failures or delays in manufacturing or supplying sufficient quantities of

RYTELO or other clinical trial materials that impact commercialization of RYTELO or the continuation of the IMpactMF

trial and other clinical trials; (l) whether we are able to establish and maintain effective sales, marketing and distribution

capabilities, obtain adequate coverage and third-party payor reimbursement, and achieve adequate acceptance in the

marketplace; (m) whether we are able to obtain and maintain the exclusivity terms and scopes provided by patent and

patent term extensions, regulatory exclusivity, and have freedom to operate; (n) that we may be unable to successfully

commercialize RYTELO due to competitive products, or otherwise; (o) that we may not be able to establish partnerships to

commercialize RYTELO in the international markets where RYTELO may be approved for marketing; (p) whether we

stay in compliance with and satisfy our obligations under our debt and synthetic royalty agreements; and (q) the impact of

general economic, industry or political climate in the U.S. or internationally and the effects of macroeconomic conditions

on our business and business prospects, financial condition and results of operations; as well as other risks that are

described herein and that are otherwise described from time to time in our Securities and Exchange Commission reports

including, but not limited to, the factors described in “Risk Factors,” in Part I, Item 1A of this Report. Geron assumes no

obligation for and except as required by law, disclaims any obligation to update these forward‑looking statements to reflect

future information, events or circumstances.

Risk Factor Summary

Below is a summary of material factors that make an investment in our common stock speculative or risky.

Importantly, this summary does not address all of the risks and uncertainties that we face. You should understand that it is

not possible to predict or identify all such factors. Consequently, you should not consider this summary to be a complete

discussion of all potential risks or uncertainties that may substantially impact our business. Additional discussion of the

risks and uncertainties summarized in this risk factor summary, as well as other risks and uncertainties that we face, can be

found under “Risk Factors” in Part I, Item 1A of this Report. The summary below is qualified in its entirety by that more

complete discussion of such risks and uncertainties. Moreover, we operate in a competitive and rapidly changing

environment. New factors emerge from time to time and it is not possible to predict the impact of all of these factors on our

business, financial condition or results of operations. You should consider carefully the risks and uncertainties described

under “Risk Factors” in Part I, Item 1A of this Report as part of your evaluation of an investment in our common stock.

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Risks Related to the Commercialization of RYTELO® (Imetelstat)

• Our near-term prospects are wholly dependent on RYTELO. We have limited experience

with the commercialization of RYTELO, and if we are unable to successfully

commercialize RYTELO in the U.S. for lower-risk MDS, or to expand its indication of use,

our ability to generate meaningful revenue or achieve profitability will be materially and

adversely affected.

• Although we have established commercial operations and sales, marketing and distribution

infrastructure for RYTELO, we have limited experience in sustaining and scaling these

operations and our commercialization efforts may be unsuccessful or less successful than

anticipated.

• If we are unable to continue to execute on our sales, marketing and distribution plans to

commercialize RYTELO, we may be unable to generate meaningful product revenue.

• We face competition from existing products, product candidates and technologies, and

competitors may develop new products and technologies. If these products, product

candidates or technologies are deemed by the healthcare community to be superior to or

more cost-effective than RYTELO, it would significantly impact the development and

commercial viability of RYTELO, which would severely and adversely affect our financial

results, business and business prospects, and the future of RYTELO, and might cause us to

cease operations.

• We will be subject to pricing and reimbursement regulations in the European Union, or EU,

which may materially affect our ability to commercialize and receive reimbursement

coverage for RYTELO in the EU.

Risks Related to Regulatory Approval of RYTELO

• We may be unable to maintain regulatory approvals for RYTELO in the U.S. and the EU

for lower-risk MDS, which would severely and adversely affect our business and business

prospects, and might cause us to cease operations.

• Our regulatory approval for RYTELO in the U.S. and in the EU for certain patients with

lower-risk MDS is subject to post-marketing requirements and commitments, and we may

be subject to penalties or product withdrawal if we fail to comply with these regulatory

requirements and commitments, or if we experience unanticipated problems with RYTELO.

Risks Related to Compliance with Healthcare Laws

• Our relationships with healthcare providers, including physicians and third-party payors,

the methods by which we promote RYTELO, and the content of our promotional materials

and programs, are subject to applicable promotional, anti-kickback, fraud and abuse, and

other healthcare laws and regulations, and our failure to comply with these laws could

expose us to criminal sanctions, civil penalties, exclusion from federal health care

programs, contractual damages, reputational harm and may adversely affect our business

and financial results.

Risks Related to the Further Development of Imetelstat

• We cannot be certain that we will be able to continue to develop imetelstat or advance it in

clinical trials, or that we will be able to receive regulatory approval for imetelstat in any

other indications in the U.S., the EU or any other region, on a timely basis or at all.

• RYTELO may cause, or have attributed to it, undesirable or unintended side effects or other

adverse events that could halt or limit its further commercialization, delay or prevent its

regulatory approval in any other jurisdiction or indication, or cause us to delay or terminate

our clinical trials.

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• Results and data we disclosed from prior non-clinical studies and clinical trials, as well as

any data disclosed as a result of an interim analysis, may not predict success in later clinical

trials or the final analysis, and we cannot assure you that any ongoing or future clinical

trials of imetelstat, including IMpactMF, will lead to similar results and data that could

potentially enable us to obtain any further regulatory approvals.

Risks Related to Manufacturing RYTELO (Imetelstat)

• Failure by us to maintain a manufacturing supply chain to appropriately and adequately

supply RYTELO for commercial and future clinical uses would adversely affect our ability

to commercialize RYTELO and result in a further delay in or cessation of clinical trials, and

our business and business prospects could be severely harmed, and we could cease

operations.

Risks Related to Our Operating Results, Financial Position and Need for Additional Capital

• We have a history of net losses and may not achieve consistent future profitability for some

time, if ever.

• Our failure to obtain additional capital, if and when needed, would force us to further delay,

reduce or eliminate the further development of RYTELO, or to halt the commercialization

of RYTELO, any of which would severely and adversely affect our financial results,

business and business prospects, and might cause us to cease operations.

Risks Related to Our Indebtedness and Royalty Payment Obligations

• Our level of indebtedness and debt service obligations could adversely affect our financial

condition and may make it more difficult for us to fund our operations, including by

limiting our operating and financial flexibility.

Risks Related to Protecting Our Intellectual Property

• If we are unable to obtain and maintain sufficient intellectual property protection and

relevant regulatory exclusivities for RYTELO, both in the U.S. and in other countries, our

competitors could develop and commercialize products similar or identical to RYTELO,

and our ability to successfully commercialize RYTELO may be adversely affected.

Risks Related to Managing Our Growth and Other Business Operations

• Our strategic restructuring plan and the associated workforce reduction implemented in

December 2025 may not result in anticipated savings and long-term value creation, could

result in total costs and operating expenses that are greater than expected and could disrupt

our business.

• We and certain of our current and former officers and directors have been named as

defendants in securities class action lawsuits and derivative lawsuits. These lawsuits, and

potential similar or related lawsuits, are costly to defend, could result in substantial

damages, divert management’s time and attention from our business, and have a material

adverse effect on our results of operations.

Calculation of Aggregate Market Value of Non‑Affiliate Shares

For purposes of calculating the aggregate market value of shares of our common stock held by

non‑affiliates as set forth on the cover page of this Report, we have assumed that all outstanding shares are held by

non‑affiliates, except for shares held directly or indirectly by each of our executive officers and directors. In the case of 5%

or greater stockholders, we have not deemed any such stockholders to be affiliates given the lack of facts and

circumstances that would indicate that any such stockholders exercise, or have the ability to exercise, any control over

Geron. These assumptions should not be deemed to constitute an admission that all executive officers and directors are, in

fact, affiliates of Geron, or that there are no other persons who may be deemed to be affiliates of Geron. Further

information concerning shareholdings of our executive officers, directors and principal stockholders is incorporated by

reference in Part III, Item 12 of this Report.

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PART I

ITEM 1. BUSINESS

Company Overview

We are a commercial-stage biopharmaceutical company aiming to change lives by changing the course of

blood cancer. Our first-in-class telomerase inhibitor, RYTELO® (imetelstat), harnesses Nobel Prize winning science in a

treatment that scientific evidence suggests reduces proliferation of malignant cells, allowing production of new healthy

cells, which we believe drives differentiated clinical benefits, potentially altering the underlying course and modifying the

disease of these hematologic malignancies.

We commercially launched RYTELO in the U.S. in June 2024 following its approval by the U.S. Food and

Drug Administration, or FDA, on June 6, 2024 for the treatment of adult patients with low- to intermediate-1 risk

myelodysplastic syndromes, or lower-risk MDS, with transfusion-dependent, or TD, anemia requiring four or more red

blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-

stimulating agents, or ESAs. Lower-risk MDS is a progressive blood cancer with high unmet need, where many patients

with anemia become dependent on red blood cell transfusions, which can be associated with clinical consequences and

decreased quality of life. We believe that the results of our Phase 3 clinical trial, IMerge, in LR-MDS, favorable FDA label

and National Comprehensive Cancer Network, or NCCN®, Clinical Practice Guidelines in Oncology, or NCCN

Guidelines®, position RYTELO as a potential treatment that can compete for significant market segments in lower-risk

MDS, including second-line ESA ineligible patients regardless of prior treatment or RS status and first-line ESA ineligible

patients.

In March 2025, we received European Commission (EC) approval of RYTELO for the treatment of adults

with TD anemia due to lower-risk MDS without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had

an unsatisfactory response to or are ineligible for ESAs. RYTELO is the first and only telomerase inhibitor approved by the

EC, and the marketing authorization applies to all 27 European Union member states, and Iceland, Norway and

Liechtenstein. We are pursuing paths to make RYTELO available to eligible LR-MDS patients outside of the U.S.,

including in the EU. To enable paid access to patients outside the United States through approved Named Patient Programs

(NPPs), we have partnered with Tanner Pharma, a distributor with broad global reach to support patient access. To date,

our revenues pursuant to NPPs have been minimal.

In addition to lower-risk MDS, we are developing imetelstat for the treatment of other myeloid hematologic

malignancies. Our Phase 3 IMpactMF clinical trial is evaluating imetelstat in patients with intermediate-2 or high-risk

myelofibrosis, or MF, who have relapsed after or are refractory to treatment with a janus associate kinase inhibitor, or JAK

inhibitor, or relapsed/refractory MF, or R/R MF, with overall survival, or OS, as the primary endpoint. As of September

2025, the trial completed enrollment. Based on our current assumptions for event (death) rates in the trial, we expect the

interim analysis for OS in IMpactMF may occur in the second half of 2026 and the final analysis may occur in the second

half of 2028.

We believe that telomerase inhibition with imetelstat represents a novel mechanism of action with unique

benefits in hematologic malignancies and potentially in other tumor types.

Our Strategy

Our strategy is to maximize the value of our first-in-class telomerase inhibitor, RYTELO (imetelstat). This

includes maximizing the commercial opportunity for RYTELO in lower-risk MDS by investing in and executing on our

U.S. commercial efforts. We expect to deliver steady growth by executing across several key imperatives, including driving

new patient starts across all eligible lower-risk MDS population segments, particularly in second-line lower-risk MDS;

reinforcing with health care providers, or HCPs, the value of duration of treatment we have observed with RYTELO;

educating HCPs on appropriate management of patient safety with RYTELO; and leveraging strong payor access for

RYTELO. We are also pursing paths to bring RYTELO to eligible LR-MDS patients outside the United States including

the EU.

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We also plan to progress our development programs that could help identify potential additional indications

for imetelstat. This includes the Phase 3 IMpactMF trial, which, if positive and approved in label expansion, could

significantly increase the RYTELO commercial opportunity. Additionally, we plan to execute on our pipeline programs

and assess the data to understand the potential to develop imetelstat in additional hematologic malignancies and as a

potential combination therapy.

U.S. Commercialization of RYTELO

RYTELO is the first and only FDA approved telomerase inhibitor. The FDA label indicates that RYTELO

is approved for certain ESA ineligible or ESA relapsed/refractory lower-risk MDS patients, regardless of RS status. The

MDS NCCN Guidelines® include imetelstat as a Category 1 preferred option treatment in second-line RS+/RS- ESA-

eligible patients regardless of prior treatment options and as a Category 2A treatment for first-line ESA-ineligible RS+/RS-

patients. In October 2025, the MDS NCCN Guidelines® were updated to change from azacitadine to imetelstat as the

preferred option in first-line RS- ESA-ineligible patients.

Our commercial strategy is designed to ensure that RYTELO reaches eligible patients when they are most

likely to benefit.

Our commercial execution is focused on three core initiatives.

• First, targeted engagement with high-volume accounts that treat earlier-line patients.

• Second, we are investing in what we believe to be the most effective marketing channels.

This includes a strong emphasis on digital, non-personal promotion, and third-party

educational platforms designed to ensure consistent, high-quality messaging across multiple

touchpoints.

• Third, we are executing cross-functionally through effective account management

initiatives.

Our cross-functional customer-facing teams include over 60 key account managers, regional business

directors, regional marketers, regional access directors , and regional medical scientific directors. We offer a wide range of

resources to support access and affordability for eligible RYTELO patients, including our REACH4RYTELO ® patient

support program, which provides a range of resources which are designed to support access and affordability to eligible

patients prescribed RYTELO.

Commercialization Plans for RYTELO Outside of the U.S.

In March 2025, we received European Commission approval, of RYTELO for the treatment of adults with

TD anemia due to lower-risk MDS. We are pursuing paths to make RYTELO available to eligible LR-MDS patients

outside of the U.S., including in the EU. We are preparing for the planned commercialization of RYTELO in select EU

markets in 2026. At this time, we do not plan to commercialize RYTELO independently in the EU (or in any other regions

outside of the U.S. where RYTELO may be approved for marketing in the future). Accordingly, we plan to work with

experienced third parties for the commercialization and marketing of RYTELO in the EU, including on critical path

activities for the planned launch of RYTELO in the EU, such as reimbursement, Health Technology Assessment, or HTA,

submissions, market access and distribution.To enable paid access to patients outside the United States through approved

Named Patient Programs (NPPs), we partnered with Tanner Pharma, a distributor with broad global reach to support

patient access. To date, revenues pursuant to NPPs have been minimal.

Background of Telomerase Inhibition in Hematologic Malignancies and Imetelstat Development

In the human body, normal growth and maintenance of tissues occurs by cell division. However, most cells

are only able to divide a limited number of times, and this number of divisions is regulated by telomere length. Telomeres

are repetitions of a deoxyribonucleic acid, or DNA, sequence located at the ends of chromosomes. They act as protective

caps to maintain stability and integrity of the chromosomes, which contain the cell’s genetic material. Normally, every time

a cell divides, the telomeres shorten. Eventually, they shrink to a critically short length, and as a result, the cell either dies

by apoptosis or stops dividing and senesces.

Telomerase is a naturally occurring enzyme that maintains telomeres and prevents them from shortening

during cell division, such as stem cells that must remain immortalized to support normal health. Telomerase consists of at

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least two essential components: a ribonucleic acid, or RNA, template, which binds to the telomere, and a catalytic subunit

with reverse transcriptase activity, which adds a specific DNA sequence to the chromosome ends. The 2009 Nobel Prize

for Physiology or Medicine was awarded to Drs. Elizabeth H. Blackburn, Carol W. Greider and Jack Szostak, former

Geron collaborators, for the discovery of how chromosomes are protected by both telomeres and telomerase.

Telomerase is upregulated in many tumor cells and malignant stem and progenitor cells, enabling the

continued and uncontrolled proliferation of the malignant cells that drive tumor growth and progression. We believe that

inhibiting telomerase may be an attractive approach to treating cancer because it may limit the proliferative capacity of

malignant stem and progenitor cells, which are believed to be important drivers of tumor growth and progression. We and

others have observed in various in vitro, ex vivo and rodent tumor models that inhibiting telomerase: (a) results in telomere

shortening and (b) arrests uncontrolled malignant cell proliferation and tumor growth.

Many myeloid hematologic malignancies, such as essential thrombocythemia, or ET, MF and MDS, have

been shown to arise from malignant stem and progenitor cells that express higher telomerase activity and have shorter

telomeres when compared to normal healthy cells. In vitro studies have suggested that tumor cells with short telomeres

may be especially sensitive to the anti‑proliferative effects of inhibiting telomerase.

Imetelstat, our proprietary telomerase inhibitor which was discovered and developed at Geron, was

designed to inhibit telomerase in malignant cells with continuously upregulated telomerase.

Imetelstat is a lipid conjugated 13‑mer oligonucleotide that we designed to be complementary to and bind

with high affinity to the RNA template of telomerase, thereby directly inhibiting telomerase activity. Imetelstat does not act

as an antisense inhibitor of protein translation. The compound has a proprietary thio‑phosphoramidate backbone, which is

designed to provide resistance to the effect of cellular nucleases, thus conferring improved stability in plasma and tissues,

as well as improved binding affinity to its target. To improve the ability of imetelstat to penetrate cellular membranes, we

conjugated the oligonucleotide to a lipid group. Imetelstat’s IC50, or half maximal inhibitory concentration, is 3 – 9 nM in

cell free assays.

We believe that imetelstat may have the potential to suppress the proliferation of malignant stem and

progenitor cells while transiently affecting normal cells. Early clinical data from a Phase 2 trial of imetelstat in patients

with ET, or the ET Trial, and a pilot study of imetelstat in patients with MF conducted at Mayo Clinic, or the Pilot Study,

suggested that imetelstat inhibits the progenitor cells of the malignant clones believed to be responsible for the underlying

diseases in a relatively select manner, indicating potential disease-modifying activity. These data were published in two

separate articles in a September 2015 issue of The New England Journal of Medicine . In the Phase 2 IMbark study, an

association of survival improvement and reduction in variant allele frequency, or VAF, was observed for high-risk

imetelstat-treated MF patients, results which were published in Journal of Clinical Oncology in 2021. Additionally, in the

Phase 2/3 IMerge study, SF3B1 VAF reduction was associated with longest transfusion independence, or TI, and with 8-

week, 24-week and 1-year TI duration in imetelstat-treated lower-risk MDS patients. These results were published in The

Lancet and at the European Hematology Association, or EHA, annual meeting in 2023.

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Pi peline Ch art

Lower-Risk Myelodysplastic Syndromes (MDS)

MDS is a group of blood disorders in which the proliferation of malignant progenitor cells produces

multiple malignant cell clones in the bone marrow resulting in disordered and ineffective production of the myeloid

lineage, which includes red blood cells, white blood cells and platelets. In MDS, bone marrow and peripheral blood cells

may have abnormal, or dysplastic, cell morphology. MDS is frequently characterized clinically by severe anemia, or low

red blood cell counts, and low hemoglobin. In addition, other peripheral cytopenias, or low numbers of white blood cells

and platelets, may cause life‑threatening infections and bleeding. Transformation to acute myeloid leukemia, or AML, is

reported to occur in up to 30% of MDS cases and results in poorer overall survival.

MDS is the most common of the myeloid malignancies. There are approximately 64,000 people in the U.S.

living with the disease and approximately 21,000 reported new cases of MDS in the U.S. every year, according to

Clarivate/DRG MDS Syndicated Report 2025. MDS is primarily a disease of the elderly, with median age at diagnosis

around 70 years. The majority of patients, approximately 70%, fall into what are considered to be the lower-risk groups at

diagnosis, according to the International Prognostic Scoring System, or IPSS, which assigns relative risk of progression to

AML and overall survival by taking into account the presence of a number of disease factors, such as cytopenias and

cytogenetics.

Chronic anemia is the predominant clinical problem in patients who have lower-risk MDS. The current

standard of care for the treatment of lower-risk MDS is the use of ESAs as supportive care, and more recently luspatercept

to improve upon disease-associated chronic anemia. The majority of patients who no longer respond to ESAs or other

available drug therapies become dependent on red blood cell transfusions due to low hemoglobin. Serial red blood cell

transfusions can lead to elevated levels of iron in the blood and other tissues, which the body has no normal way to

eliminate. Iron overload is a potentially dangerous condition. Published studies in patients with MDS have shown that iron

overload resulting from regular red blood cell transfusions is associated with a poorer overall survival and a higher risk of

developing AML.

Phase 3 IMerge Trial in Lower-Risk MDS

Our regulatory approval in the U.S. for certain patients with lower-risk MDS and our EMA submission are

each based on positive data from the IMerge Phase 3 clinical trial. The trial met its primary endpoint of ≥ 8-week red blood

cell transfusion independence rate and a key secondary endpoint of ≥ 24-week red blood cell transfusion independence rate,

demonstrating highly statistically significant (i.e., p<0.001 for both) and clinically meaningful benefits with imetelstat

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treatment versus placebo. Furthermore, statistically significant and clinically meaningful efficacy results were observed in

the trial across key MDS patient subtypes, including patients who were ringed sideroblast positive, or RS positive, and

ringed sideroblast negative, or RS negative; patients with high (4-6 RBC units/8 weeks) and very high baseline transfusion

burden (>6 RBC units/8 weeks); and patients classified as Low or Intermediate-1 risk according to the IPSS. The most

common Grade 3/4 adverse reactions were neutropenia (72%) and thrombocytopenia (65%), which lasted a median

duration of less than two weeks, and in more than 80% of patients were resolved to Grade <2 in under four weeks.

Myelofibrosis (MF)

MF, a type of myeloproliferative neoplasm, is a chronic blood cancer in which abnormal or malignant

precursor cells in the bone marrow proliferate rapidly, causing scar tissue, or fibrosis, to form. As a result, normal blood

production in the bone marrow is impaired and may shift to other organs, such as the spleen and liver, which can cause

them to enlarge substantially. People with MF may have abnormally low or high numbers of circulating RBCs, white blood

cells or platelets, and abnormally high numbers of immature cells in the blood or bone marrow. MF patients can also suffer

from debilitating constitutional symptoms, such as drenching night sweats, fatigue, severe itching, or pruritus, abdominal

pain, fever and bone pain.

Approximately 70% of MF patients are classified as having Intermediate‑2 or High-risk disease, as defined

by the Dynamic International Prognostic Scoring System Plus described in a 2011 Journal of Clinical Oncology article.

Drug therapies currently approved by the FDA and other regulatory authorities for treating these MF patients include JAK

inhibitors, ruxolitinib, fedratinib and momelotinib, as well as pacritinib, a kinase inhibitor. Currently, no drug therapy is

approved for those patients who fail or no longer respond to JAK inhibitor treatment. A variety of best available therapies

are used in absence of an approved treatment for this patient population, and median survival is limited, representing a

significant unmet medical need.

Ongoing Phase 3 IMpactMF Trial in Relapsed/Refractory MF

Trial Design

IMpactMF, our Phase 3 clinical trial in relapsed/refractory MF, is an open label, 2:1 randomized, controlled

clinical trial designed to evaluate imetelstat (9.4 mg/kg administered by intravenous infusion over two hours every three

weeks) in approximately 320 patients. Patients relapsed after or refractory to a JAK inhibitor are defined as having an

inadequate spleen response or symptom response after treatment with a JAK inhibitor for at least six months, including an

optimal dose of a JAK inhibitor for at least two months. The best available therapy, or BAT, control arm of IMpactMF

excludes the use of JAK inhibitors. With respect to the trial design for IMpactMF, the FDA urged us to consider adding a

third dosing arm to assess a lower dose and/or a more frequent dosing schedule that might improve the planned trial’s

chance of success by identifying a less toxic regimen and/or more effective spleen response, one of the trial’s secondary

endpoints. Based on data from IMbark, which evaluated a low and high doses, we believe that testing a lower dose regimen

would likely result in a lower median OS, which is the trial’s primary endpoint, in the imetelstat treatment arm. We believe

existing data also suggest that lowering the dose would not result in a clinically meaningful reduction in toxicity. For these

reasons, we therefore determined not to add a third dosing arm to the trial design, and the FDA did not object to our

proposed imetelstat dose and schedule of 9.4 mg/kg every three weeks. Our belief may ultimately be incorrect. Therefore,

our failure to add a third dosing arm could result in a failure to maintain regulatory clearance from the FDA and similar

international regulatory authorities, could result in the trial’s failure, or could otherwise delay, limit or prevent marketing

approval of imetelstat for relapsed/refractory MF by the FDA or similar international regulatory authorities.

The primary efficacy endpoint for IMpactMF is OS. Key secondary endpoints include symptom response;

spleen response; progression free survival; complete remission, partial remission or clinical improvement, as defined by the

International Working Group for Myeloproliferative Neoplasms Research and Treatment criteria; duration of response;

safety; pharmacokinetics; and patient reported outcomes. There are IMpactMF sites across North America, South America,

Europe, Australia and Asia.

Current Status of IMpactMF

IMpactMF opened for patient screening and enrollment in December 2020. In September 2025, the trial

completed enrollment. Based on our current assumptions for event (death) rates in the trial, we expect the interim analysis

for OS in IMpactMF may occur in the second half of 2026 and the final analysis may occur in the second half of 2028.

Because these analyses are event-driven and it is uncertain whether actual rates for events will reflect current planning

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assumptions, the results may be available at different times than currently expected. At the interim analysis, if the pre-

specified statistical OS criterion is met, then we expect such data may potentially support the registration of imetelstat in

relapsed/refractory MF. Subject to protocol-specified stopping rules for futility, if the pre-specified OS criterion is not met

at the interim analysis, the trial will continue to the final analysis.

The timing and achievement of either or both of the planned analyses depend on numerous factors,

including blinded death rates, which have in the past been, and may continue to be, lower than our projections. In addition,

our ability to conduct and complete IMpactMF depends on whether we can obtain and maintain the relevant clearances

from regulatory authorities and other institutions to continue to conduct and complete the trial.

Improvement in Overall Survival and Potential Disease-Modifying Activity Observed in IMbark Phase 2

The IMbark Phase 2 clinical trial was designed to evaluate two dosing regimens of imetelstat (either 4.7

mg/kg or 9.4 mg/kg administered by intravenous infusion every three weeks) in patients with relapsed/refractory MF.

We previously reported efficacy and safety results from the IMbark Phase 2 clinical trial, including median

OS of 28.1 months for patients on the high dose arm of the study, which is almost twice the reported median OS of 14–16

months in medical literature. To evaluate this potential benefit, we conducted a post-hoc analysis of OS for patients treated

with imetelstat 9.4 mg/kg in IMbark compared to OS calculated from real world data, or RWD, collected at the Moffitt

Cancer Center for patients who had discontinued treatment with ruxolitinib, a JAK inhibitor, and who were subsequently

treated with BAT. To make a comparison between the IMbark data and RWD, a cohort from the real-world dataset was

identified that closely matched the IMbark patients, using guidelines for inclusion and exclusion criteria as defined in the

IMbark clinical protocol, such as platelet count and spleen size. Calculations from two propensity score analysis

approaches resulted in a median OS of 30.7 months for the imetelstat-treated patients from IMbark, which is more than

double the median OS of 12.0 months using RWD for patients treated with BAT. These analyses also showed a 65% – 67%

lower risk of death for the imetelstat-treated patients vs. BAT-treated patients. We believe these analyses suggest

potentially longer OS for imetelstat-treated relapsed/refractory MF patients in IMbark, compared to BAT in closely-

matched patients from RWD. However, comparative analyses between RWD and our clinical trial data have several

limitations. For instance, the analyses create a balance between treatment groups with respect to commonly available

covariates, but do not take into account the unmeasured and unknown covariates that may affect the outcomes of the

analyses. Potential biases are introduced by factors which include, for example, the selection of the patients included in the

analyses, misclassification in the matching process, the small sample size, and estimates that may not represent the

outcomes for the true treated patient population. For these and other reasons, such comparative analyses and any

conclusions from such analyses should be considered carefully and with caution, and should not be relied upon as

demonstrative or otherwise predictive or indicative of any current or potential future clinical trial results of imetelstat in

relapsed/refractory MF, including IMpactMF.

In IMbark, patients also experienced other positive clinical outcomes, including symptom improvement,

spleen reduction and bone marrow fibrosis improvement. In June 2020, we reported correlation analyses from IMbark that

showed a trend of longer OS in patients who achieved symptom response, spleen volume reductions and improved bone

marrow fibrosis, in a dose-dependent manner. Furthermore, the reductions in the variant allele frequency of key driver

mutations in MF and the improvement in bone marrow fibrosis observed in IMbark have also been correlated to the

improvement in OS. We believe the improvement in bone marrow fibrosis, potential survival benefit, molecular data and

correlations from IMbark provide strong evidence of the potential for disease modification with imetelstat, which we

believe would differentiate imetelstat from currently approved treatments for MF, if approved.

The safety results observed in IMbark were consistent with prior clinical trials of imetelstat in hematologic

malignancies, and no new safety signals were identified. In the 9.4 mg/kg arm, reversible and manageable Grade 3/4

thrombocytopenia and neutropenia were reported in 24/59 patients (41%) and 19/59 patients (32%), respectively, without

significant clinical consequences. 1/59 patients (2%) had Grade 3 febrile neutropenia. 3/59 patients (5%) had Grade 3/4

bleeding. 6/59 patients (10%) had Grade 3/4 infections. Furthermore, more than 70% of the observed Grade 3/4 cytopenias

resolved to Grade 2 or lower by laboratory assessment within four weeks.

FDA Fast Track Designation

Fast Track designation provides opportunities for frequent interactions with FDA review staff, as well as

eligibility for priority review, if relevant criteria are met, and rolling review. Fast Track designation is intended to facilitate

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and expedite development and review of an NDA to address unmet medical needs in the treatment of serious or life-

threatening conditions. However, Fast Track designation does not accelerate conduct of clinical trials or mean that the

regulatory requirements are less stringent, nor does it ensure that imetelstat will receive marketing approval or that

approval will be granted within any particular timeframe. In addition, the FDA may withdraw Fast Track designation if it

believes that the designation is no longer supported by data emerging from the imetelstat clinical development program.

In October 2017, the FDA granted Fast Track designation to imetelstat for the treatment of adult patients

with TD anemia due to lower-risk MDS who do not have a non-del 5q abnormality and who are refractory or resistant to

treatment with an ESA (i.e., the treatment population in IMerge Phase 3).

In September 2019, the FDA granted Fast Track designation to imetelstat for the treatment of adult patients

with Intermediate-2 or High-Risk MF whose disease has relapsed after or is refractory to JAK inhibitor treatment (i.e., the

treatment population in IMpactMF).

Potential Additional Indications

IMproveMF: Phase 1 Combination Clinical Trial in Frontline Myelofibrosis (Frontline MF)

We are also evaluating imetelstat as a combination therapy in the Phase 1 IMproveMF clinical trial as a

first-line treatment for patients with Intermediate-1, Intermediate-2 or High-Risk myelofibrosis. Based on the dose

escalation findings in Part 1 of the study, presented at the American Society of Hematology, or ASH, annual meeting in

December 2024, imetelstat 9.4 mg/kg dosed every four weeks with ruxolitinib was the selected dose for the dose expansion

Part 2 of the study, which is currently enrolling patients.

IMpress: Investigator-Led Phase 2 Clinical Trial in Higher Risk Myelodysplastic Syndromes (Higher Risk MDS) and

Acute Myeloid Leukemia (AML)

Imetelstat is also being studied in an investigator-led IMpress Phase 2 clinical trial in Intermediate-2 or

High-Risk myelodysplastic syndromes, or higher-risk MDS, and acute myeloid leukemia, or AML, patients that are

relapsed or refractory to hypomethylating agent, or HMA, treatment. Based on observations from an interim analysis from

the first cohort, presented at ASH in December 2024, the protocol was amended to a more frequent dosing schedule for a

second cohort of patients being enrolled and treated with this modified schedule as of August 2024. These data were

presented at ASH in December 2025.

IMAGINE: Investigator-Led Phase 1/2 Clinical Trial in Relapsed/RefractoryAML

We are enrolling a Phase 1/2 investigator-led study, called IMAGINE, in relapsed/refractory AML, using a

combination approach of imetelstat and azacitidine with or without venetoclax.

Research Programs

Next Generation Telomerase Inhibitor Discovery

We have initiated a discovery program to identify lead compounds as a potential next generation oral

telomerase inhibitor. If the leads we have identified are optimized, we may conduct preclinical experiments that may serve

as a basis for potential future clinical testing. Discovery research is an uncertain and unpredictable process. As such, the

timing and nature of any results from this discovery effort are difficult to forecast. If we optimize lead compounds from

this discovery program, we expect to provide an update on our efforts at that time.

Preclinical Lymphoid Hematologic Malignancies

Academic research data suggests that certain lymphoid hematologic malignancies have higher telomerase

activity and shorter telomeres when compared to normal healthy cells. Based on this scientific hypothesis, we conducted a

preclinical research project with MD Anderson Cancer Center to determine the potential application of imetelstat in

lymphoid hematologic malignancies. The project was completed, and preliminary results of the research project were

published in Blood in November 2022. Exploring the utility of imetelstat in lymphoid hematologic malignancies remains

an area of interest for us.

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Intellectual Property and Regulatory Exclusivity

Intellectual property, including patent protection, is very important to our business. We file patent

applications in the U.S. and other jurisdictions, and we also rely on trade secret protection and contractual arrangements to

protect aspects of our business. An enforceable patent with appropriate claim coverage can provide an advantage over

competitors who may seek to employ similar approaches to develop therapeutics, and so the future commercial success of

RYTELO (imetelstat), and therefore our future success, will be in part dependent on our intellectual property strategy.

Our intellectual property strategy includes the early development of a technology, such as imetelstat,

followed by rounds of increasingly focused innovation around a product opportunity, including identification and definition

of a specific product candidate and uses thereof, manufacturing processes, product formulation and methods of treatment

and administration. The result of this process is that products in development are often protected by several families of

patent filings that are filed at different times during the development process and cover different aspects of the product.

Consequently, earlier filed, broad technology patents will usually expire ahead of patents covering later developments, such

as product formulations and methods of treatment and administration, so that patent expirations on a product may span

several years. Patent coverage may also vary from country to country based on the scope of available patent protection.

There are also opportunities to obtain an extension of patent coverage for a product in certain jurisdictions, which adds

further complexity to the determination of patent life.

From time to time, we may endeavor to monitor worldwide patent filings by third parties that are relevant

to our business. Based on this monitoring, we may determine that an action is appropriate to protect our business interests.

Such actions may include negotiating patent licenses where appropriate, filing oppositions against a patent, filing a request

for post grant review against a patent or filing a request for the declaration of an interference with a patent application or

issued patent.

The information provided in this section should be reviewed in the context of the section entitled “Risks

Related to Protecting Our Intellectual Property” described in “Risk Factors” in Part I, Item 1A of this Report.

RYTELO (imetelstat)

Summary

RYTELO was developed internally by us, and we hold global commercial rights to it. We own issued

patents related to RYTELO in the U.S., Europe and other jurisdictions. Although composition of matter patents generally

provide the most comprehensive coverage of a therapeutic product such as RYTELO, subsequent patent filings directed to

other aspects of RYTELO may also provide additional patent coverage with later expiration dates. In addition, it may be

possible to obtain patent term extensions of some patents in some jurisdictions for claims covering RYTELO or relating to

RYTELO, such as methods of treatment with RYTELO, which could further extend the patent term.

We have issued patents in the U.S., Europe and other jurisdictions that provide patent coverage into 2033

(not including any patent term extension) pertaining to the treatment of MDS and MF with RYTELO.

In the U.S., our method of treatment patent rights for MDS and MF expire in March 2033 (not including

any patent term extension). We also hold an issued patent in the U.S. covering the composition of matter of RYTELO

(imetelstat) that that was set to expire in December 2025 according to its original patent term, including patent term

adjustment for delays at the USPTO. Now that we have received approval for RYTELO in the U.S., we have applied for

patent term extensions under the provisions of the Drug Price Competition and Patent Term Restoration Act of 1984 (as

amended), or the Hatch-Waxman Act, which, if granted, could extend the patent term of either our method of treatment

patent for MDS and MF or our composition of matter patent by up to five years (but not beyond 14 years from the date of

approval). Our composition of matter patent was granted interim patent term extension under the Hatch-Waxman Act,

which extends its expiration date to December 2026, while a decision is being rendered on the patient term extension

application.

In Europe and other countries, our patent rights for use in MDS and MF expire in November 2033 (not

including any patent term extension). Our composition of matter patent coverage expired in September 2024. We plan to

seek patent term extension under a Supplementary Protection Certificate, or SPC, as permitted under European Council, or

EC, Regulation No 469/2009 of the European Parliament and of the Council of 6 May 2009 concerning the supplementary

protection certificate of medicinal products (the Medicinal SPC Regulation), of one of our use patents, such as our patent

for use in MDS, in the European Economic Area, or the EEA, which could extend the patent term by up to five years.

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In the U.S., Europe, and other jurisdictions, we are also pursuing other patent rights relating to RYTELO

(imetelstat), such as methods of treatment of MDS in specific patient subpopulations, reagents useful in the manufacturing

processes for the drug, and other methods of treatment and kit claims, certain of which are co‑owned with other entities.

Patent Term Extension

Although we are in the process of seeking patent term extension for some of our issued patents covering

RYTELO, it is not possible to obtain patent term extension of any patents that expired prior to or are issued following

regulatory approval. For the patents for which we are seeking a patent term extension, we may not be granted any such

patent term extension and/or the applicable time period of such patent term extension could be less than we have projected.

In the U.S., patent term extension is available for a product that contains an active ingredient that has not previously been

approved. The extension, which compensates for patent term lost during product development and FDA regulatory review

process, is generally equal to the sum of one-half the time between the effective date of an IND application and the

submission date of an NDA, and all of the time between the submission date of an NDA and the approval of that

application. Moreover, in some jurisdictions, including the U.S., such patent term extensions, if any, are available for only

one patent for a given product and the rights derived from such extension are limited to those claims which encompass the

product composition and treatment indications as approved by the relevant healthcare regulatory authority on or after the

regulatory review period on which the extension is based. During the life of the patent term extension, however, its scope

of protection may expand to include any additional indications subsequently approved for the product and claimed by the

patent. Furthermore, some jurisdictions, including the U.S., allow the filing of patent term extension applications on

multiple patents, but ultimately the patent owner must select one patent to which the extension is applied.

In the U.S., now that we have received approval for RYTELO in certain patients with lower-risk MDS, we

may potentially extend the term of our composition of matter patent in the U.S. for a maximum of five years until

December 2030, subject to U.S. Patent and Trademark Office, or USPTO, approval. Alternatively, we may potentially

extend the term of our method of treatment for MDS claims in the U.S. until August 2037, subject to USPTO approval. As

we have previously disclosed, we expect to apply patent term extension, if granted, to our method of treatment patent, since

doing so extends the expiration date of the method of treatment patent to a later date than the composition of matter patent.

However, if we do not receive a patent term extension for our U.S. method of treatment patent for MDS, it will expire in

March 2033. Once our composition of matter patent expires in the U.S., we must rely on our method of treatment patent

and other patents and regulatory exclusivity for RYTELO in the U.S.

Similarly, in Europe, we are in the process of seeking to potentially extend the term of our patents in the

EEA for the use of RYTELO in MDS for a maximum of five years, from November 2033 until November 2038, subject to

the approval of the national patent offices, in accordance with the Medicinal SPC Regulation. Since our European

composition of matter patents expired in September 2024, we must rely on our use and other patents and, subject to

receiving approval from the EC, regulatory exclusivity for RYTELO in the EEA.

If we do not have sufficient patent life and regulatory exclusivity to protect RYTELO in the U.S. and

Europe, our financial results, business and business prospects, and future development of imetelstat could be materially and

adversely affected, which might cause us to cease operations.

Orphan Drug Designation and Market Exclusivity

United States

Some jurisdictions, including the U.S., may designate drugs or biologics for relatively small patient

populations as orphan drugs. For a drug to qualify for orphan drug designation by the FDA, both the drug and the disease

or condition must meet certain criteria specified in the Orphan Drug Act, or ODA, and FDA’s implementing regulations.

Orphan drug designation is granted by the FDA’s Office of Orphan Products Development in order to support

development of medicines for rare diseases or conditions, which generally are those that affect fewer than 200,000 people

in the U.S. or, if the disease or condition affects more than 200,000 individuals annually in the U.S., if there is no

reasonable expectation that the cost of developing and making the drug available in the U.S. would be recovered from sales

of such drug in the U.S. Orphan drug designation does not shorten the duration of the regulatory review process or lower

the approval standards, but can provide important benefits, including consultation with FDA. Orphan drug designation

qualifies the sponsor of the drug for various development incentives under the ODA, including certain tax credits for

qualified clinical testing and exemption from user fees.

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A drug granted approval for an orphan designated indication generally receives seven years of market

exclusivity, during which time the FDA generally may not approve any other application for the same drug for the same

use, with certain limited exceptions, including when the later product is shown to be clinically superior to the product with

exclusivity (and thus not the same). If there is a previously-approved product that is the same drug for the same indication,

orphan drug designation requires the sponsor to provide a plausible hypothesis of clinical superiority over the approved

product, whereas ODE requires the sponsor to actually demonstrate clinical superiority. Clinical superiority can be

established by way of greater efficacy, greater safety, or making a major contribution to patient care. Orphan drug

exclusivity does not prevent the FDA from approving a different drug for the same disease or condition, or the same drug

for a different use. The FDA can revoke a product’s orphan drug exclusivity under certain circumstances, including when

the product sponsor is unable to assure the availability of sufficient quantities of the product to meet patient needs. A later

product may also be approved during the exclusivity period if the orphan drug exclusivity holder consents to the approval

of that product.

A marketing application for a prescription drug product that has received orphan drug designation is not

subject to a prescription drug user fee unless the application includes an indication for a disease or condition other than the

rare disease or condition for which the drug was granted orphan drug designation. The granting of orphan drug designation

does not alter the standard regulatory requirements and process for obtaining marketing approval, including the

requirement that a drug’s effectiveness be established by substantial evidence, as demonstrated through adequate and

well‑controlled studies (and, as appropriate, confirmatory evidence).

In June 2015 and December 2015, the FDA granted orphan drug designation to imetelstat for the treatment

of MF and MDS, respectively, and following approval of RYTELO in June 2024, the FDA listed in its Approved Drug

Products with Therapeutic Equivalence Evaluations, commonly known as the Orange Book, and its database for orphan-

drug designations and approvals, that RYTELO has orphan drug exclusivity, which is expected to provide orphan drug

exclusivity until June 2031 for its MDS indication, subject to our continuing compliance with the requirements to maintain

such orphan drug exclusivity.

In addition to orphan drug exclusivity, under the Hatch-Waxman Act, if a product is a “new chemical

entity” or NCE, which generally means that the active moiety has never before been previously approved by the FDA,

there is a period of five years from approval of the first indication during which the FDA may not accept for filing any

abbreviated new drug application, or ANDA, under section 505(j) of the Federal Food, Drug, and Cosmetic Act, or an

application under section 505(b)(2) of the statute for a drug with the same active moiety. An ANDA or 505(b)(2)

application may be submitted after four years, however, if the sponsor of the application makes a Paragraph IV

certification, which may trigger litigation and a 30-month stay of approval, as further described in the section titled " The

validity, scope and enforceability of any patents listed in the Orange Book that cover RYTELO or its methods of use can be

challenged by third parties and may not protect us from generic or innovator competition " under “Risk Factors” in Part I,

Item 1A of this Report. Our request for NCE exclusivity for RYTELO is pending review by the FDA. If the FDA were to

grant NCE exclusivity for RYTELO, NCE exclusivity would continue until June 2029.

The Hatch-Waxman Act also provides three years of marketing exclusivity for a product that is not an NCE

but that incorporates a change(such as a new indication, dosage form or strength) from the approved product with the same

moiety. Such product may qualify for a three-year period of exclusivity if the NDA contains reports of new clinical

investigations (other than bioavailability studies) conducted or sponsored by the sponsor that were necessary for approval

of the NDA. In that instance, the exclusivity period does not preclude filing or review of an ANDA or 505(b)(2)

application; rather, the FDA is precluded from granting final approval to the ANDA or 505(b)(2) application until three

years after approval of the Reference Listed Drug, or RLD. Additionally, the exclusivity applies only to the extent that any

subsequent ANDA or Section 505(b)(2) product that shares the conditions of approval that required submission of the

clinical data.

In the US, the exclusivity periods and patent-related protections described above also may be eligible for a

six-month extension of regulatory exclusivity, or pediatric exclusivity, pursuant to section 505A of the Federal Food, Drug,

and Cosmetic Act, if the sponsor submits pediatric data that “fairly respond” to a written request from FDA for such data;

however, we do not expect to receive pediatric exclusivity for RYTELO in the U.S.

Europe

In the EEA, a medicinal product containing a new active substance generally receives eight years of data

exclusivity, which prevents competitors from relying on the results of pre-clinical tests and of clinical trials when applying

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for a marketing authorization for a generic or biosimilar product, and an additional two years of market exclusivity, during

which competitors can apply for a marketing authorization by referencing the marketing authorization holder's data but are

not allowed to place their product on the market until the end of this period, conferring a total of ten years of exclusivity for

the medicinal product. The ten years of exclusivity can be extended to a maximum of eleven years if, during the first eight

years of those ten years, the marketing authorization holder obtains an authorization for one or more new therapeutic

indications which, during the scientific evaluation prior to their authorization, are held to bring a significant clinical benefit

in comparison with existing therapies. When RYTELO was granted marketing authorization by the EC for the treatment of

certain patients with lower-risk MDS on March 7, 2025, it was confirmed to contain a new active substance and so

RYTELO is entitled to ten years of data exclusivity in the EEA from the time of approval.

In addition to data exclusivity, orphan drug designation by the EC provides regulatory and financial

incentives for companies to develop and market therapies that treat a life‑threatening or chronically debilitating condition

affecting no more than five in 10,000 persons in the EU (or if it is unlikely that marketing of the medicinal product would

generate sufficient returns to justify the investment needed for its development), and where no satisfactory treatment is

available, or, if such method exists, the medicine must be of significant benefit to those affected by the condition in

accordance with Regulation (EC) No 141/2000 (the EU Orphan Regulation). Orphan drug designation also entitles a party

to financial incentives such as reduction of fees or fee waivers, as well as protocol assistance from the EMA during the

product development phase, and direct access to the centralized authorization procedure. In addition, ten years of market

exclusivity is granted following receipt of marketing authorization as an orphan medicinal product, meaning that a

competing similar medicinal product for the same therapeutic indication in principle may not enter the market for a period

of 10 years following marketing authorization, subject to specific conditions. To benefit from market exclusivity, a

medicine must maintain its orphan designation at the time of marketing authorization. Each indication with an orphan

designation confers ten years' market exclusivity for the particular indication. This period may be reduced to six years if the

orphan drug designation criteria are no longer met, including where it is shown that the product is sufficiently profitable to

not justify maintenance of market exclusivity.

In December 2015 and July 2020, the EC granted orphan drug designation to imetelstat for the treatment of

MF and MDS, respectively. On March 7, 2025, the EC granted marketing authorization for RYTELO (imetelstat sodium)

as an orphan medicinal product and considered that imetelstat is a new active substance. RYTELO is indicated as

monotherapy for the treatment of adult patients with TD anemia due to very low, low or intermediate risk myelodysplastic

syndromes (MDS) without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory

response to or are ineligible for erythropoietin-based therapy. With the grant of the marketing authorization, RYTELO in

principle is currently entitled to maintain orphan market exclusivity in the EEA in the approved indication for ten years

post-approval.

In addition, under the European Pediatric Regulation, if we fulfill our pediatric investigation plan agreed

upon with the EMA, we would be eligible to receive an additional two years of market exclusivity

under the European Pediatrics Regulation (Regulation (EC) No 1901/2006).

Prior Collaboration with Janssen Biotech, Inc.

Upon the effective date of termination of the license and collaboration agreement, or the Prior

Collaboration Agreement, with Janssen Biotech, Inc., or Janssen, on September 28, 2018, we regained global rights to

imetelstat and are continuing the development, commercialization and marketing of imetelstat on our own. In accordance

with the termination provisions of the Prior Collaboration Agreement, we have an exclusive worldwide license for

intellectual property developed under the Prior Collaboration Agreement for the further development, commercialization

and marketing of imetelstat, without any economic obligations to Janssen with respect to such license. Janssen has assigned

to us certain intellectual property developed by it under the Prior Collaboration Agreement. We now are responsible for the

costs of maintaining, prosecuting and litigating all imetelstat intellectual property that we own.

Licensing

We have no material license agreements. We have global rights to imetelstat, which was discovered and

developed at Geron.

Manufacturing

A typical sequence of steps in the manufacture of imetelstat drug product includes the following key

components:

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• starting materials, which are well‑defined raw materials that are used to make bulk drug

substance;

• bulk drug substance, which is the active pharmaceutical ingredient in a drug product that

provides pharmacological activity or other direct effect in the treatment of disease; and

• final drug product, which is the finished dosage form that contains the drug substance and is

supplied for patient treatment.

Since September 2018, we have engaged third‑party contract manufacturers and have established a supply

chain to manufacture and supply imetelstat that meets applicable regulatory standards for current and potential commercial

uses and current and potential future clinical trials.

We do not have direct control over third‑party personnel or operations. These third‑party contract

manufacturers, and/or any other third parties that we may rely upon for the manufacture and/or supply of imetelstat,

typically complete their services on a proposal by proposal basis under master supply agreements and may need to make

substantial investments to enable sufficient capacity increases and cost reductions, and to implement those regulatory and

compliance standards necessary for commercial production and successful Phase 3 clinical trials. These third‑party contract

manufacturers, and/or any other third parties that we may rely upon for the manufacture and/or supply of imetelstat, may

not be able to achieve such capacity increases, cost reductions, or regulatory and compliance standards, and even if they do,

such achievements may not be at a commercially reasonable cost. We are responsible for establishing any long‑term

commitments or commercial supply agreements with any of the third‑party contract manufacturers for imetelstat. The

information provided in this section should be reviewed in the context of the section entitled “Risks Related to

Manufacturing RYTELO (Imetelstat)” under Part I, Item 1A, “Risk Factors” of this Report.

Competition

The pharmaceutical and biotechnology industries are characterized by intense and dynamic competition

with rapidly advancing technologies and a strong emphasis on proprietary products. While we believe our proprietary

oligonucleotide chemistry; experience with the biological mechanisms related to RYTELO, telomeres and telomerase;

clinical data to date indicating potential disease-modifying activity with RYTELO treatment; and knowledge and expertise

around the development of potential treatments for myeloid hematologic malignancies provide us with competitive

advantages, we face competition from many different sources, including major pharmaceutical, specialty pharmaceutical

and biotechnology companies, academic institutions, governmental agencies, and public and private research institutions.

RYTELO is competing with other products and therapies that currently exist, are being developed or will in the future be

developed, some of which we may not currently be aware of.

Competition in Lower-Risk MDS

The current standard of care for the treatment of lower-risk MDS is the use of ESAs as supportive care, and more

recently luspatercept to improve upon disease-associated chronic anemia. Historically, ESAs have been used as a mainstay

of treatment; once no longer effective, serial blood transfusions are often administered, which can cause organ damage due

to iron overload and result in poor outcomes and shorter survival. In recent years, considerable advancements have been

made in the treatment of LR-MDS with the approval of new therapies, expanding indications, and ongoing clinical

investigation of novel ag ents.

In lower-r isk MDS, data from the IMerge Phase 3 clinical trial resulted in FDA approval of RYTELO in June

2024 and EC approval in March 2025 for the treatment of certain patients with lower-risk MDS. IMerge showed

meaningful and durable transfusion independence, activity across MDS patient subtypes, and potential disease-modifying

activity achievable with RYTELO treatment. We believe that these key features are differentiators compared to currently

approved products as well as investigational drugs currently in clinical development.

In lower-risk MDS, RYTELO competes against a number of currently existing therapies, including ESAs (Epoetin

alfa, Procrit; Darbepoetin alfa, Aranesp-Amgen) that are indicated for anemia; immunomodulators, such as Revlimid

(lenalidomide) by Celgene Corporation, or Celgene, a Bristol Myers Squibb, or BMS, company; hypomethylating agents,

such as Vidaza (azacitidine) by BMS and Dacogen (decitabine) by Otsuka America Pharmaceutical, Inc. and other

manufacturers in the U.S. and Janssen in the EU; Inqovi (oral combination of decitabine and cedazuridine) by Astex

Pharmaceuticals, Inc., or Astex, and Taiho Oncology ; Tibsovo (ivosidenib), an IDH1 inhibitor, by Servier Pharmaceuticals,

LLC; and Reblozyl (luspatercept), a TGF-beta ligand trap , by BMS. In August 2023, luspatercept was approved for the

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treatment of anemia in ESA-naive adult patients with very low-to intermediate-risk MDS who may require regular RBC

transfusions.

Other therapies currently in Phase 3 development in lower-risk MDS include elritercept (KER-050), a

TGF-beta inhibitor, by Keros Therapeutics, Inc.; and Reblozyl (luspatercept) in non-transfusion-dependent lower-risk MDS

patients, by BMS.

In addition, there are multiple Phase 1 and Phase 2 clinical trials of other agents being developed for lower-

risk MDS, including but not limited to: LB‐100, a PP2A inhibitor by Lixte Biotechnology Holdings, Inc., and bemcentinib,

an AXL inhibitor by BerGenBio. Also, a lower dose of ASTX727, an oral formulation of decitabine and cedazuridine,

referred to as ASTX727 LD, by Astex; ASTX030, an oral formulation of azacitidine and cedazuridine, by Astex; R289, a

dual oral inhibitor of interleukin receptor-associated kinases 1 and 4, or IRAK1/4, by Rigel Pharmaceuticals, Inc.; and a

combination regimen of luspatercept and ESA.

Competition in Relapsed/Refractory MF

The current standard of care for the treatment of Intermediate-2 or High-risk MF is the use of JAK

inhibitors to address the patient’s symptoms. Once JAK inhibitors fail or are no longer effective, a variety of best available

therapies are used since there are no approved treatments for this patient population and median OS is limited.

In Intermediate-2 or High-risk relapsed/refractory MF, data from IMbark suggest potential disease-

modifying activity with RYTELO treatment and a potential meaningful improvement in OS, which is supported in a

comparison to real-world data.

If approved for commercial sale for the treatment of relapsed/refractory MF, RYTELO would compete

against currently approved JAK inhibitors: Jakafi (ruxolitinib) by Incyte Corporation, or Incyte, Inrebic (fedratinib) by

BMS, and OJJAARA (momelotinib) by GlaxoSmithKline plc, or GSK, which was approved in September 2023 for the

treatment of intermediate or high-risk MF, including primary MF or secondary MF (postpolycythemia vera and post-

essential thrombocytopenia), in adults with anemia; and Vonjo (pacritinib), by Sobi, Inc., which was approved in February

2022 for the treatment of adults with intermediate or high-risk primary or secondary MF with a platelet count below 50 ×

109/L. Other treatment modalities for MF include hydroxyurea for the management of splenomegaly, leukocytosis,

thrombocytosis and constitutional symptoms; splenectomy and splenic irradiation for the management of splenomegaly and

co-existing cytopenias; chemotherapy; and pegylated interferon. Drugs for the treatment of MF-associated anemia include

ESAs, androgens, danazol, corticosteroids, thalidomide and lenalidomide. In addition, luspatercept has been used to treat

MF-associated anemia, although the Phase 3 trial did not meet its primary endpoint for RBC-TI.

Other therapies currently in Phase 3 development in MF, some of which may obtain regulatory approval earlier

than RYTELO for MF, include pelabresib (CPI-0610), a BET inhibitor, by Novartis AG; and navtemadlin, an MDM2-

inhibitor, by Kartos Therapeutics, Inc. Other approaches for MF currently under investigation that could compete with

RYTELO in the future include luspatercept; zinpentraxin alfa (RG6354, formerly PRM-151), an anti-fibrosis antibody, by

F. Hoffmann-La Roche, Ltd.; INCB160058, a JAK2 inhibitor, by Incyte; AJ1-11095, a JAK2 inhibitor, by Ajax

Therapeutics, Inc.; SLT-5505, a pan-LOX inhibitor, by Syntara Limited; tasquinimod, an S100A9 inhibitor, by Active

Biotech AB; XPOVIO (selinexor), a nuclear export inhibitor, by Karyopharm Therapeutics, Inc.; TL-895, an oral tyrosine

kinase inhibitor, by Telios Pharma, Inc.; pelcitoclax (APG-1252), a dual BCL-2/BCL-XL inhibitor, by Ascentage Pharma;

DISC-0974, a monoclonal antibody against hemojuvelin (HJV) by DISC Management Inc.; elritercept ( KER-050) in

combination with ruxolitinib, by Keros Therapeutics; CK0804, an allogeneic T-regulatory cell agent, by Cellenkos, Inc. in

collaboration with Incyte; nuvisertib (TP-3654), an oral PIM kinase inhibitor by Sumitomo Pharma Co., Ltd.; and a

mutated-CALR peptide vaccine, from the Icahn School of Medicine at Mount Sinai.

Government Regulation

Regulation by governmental authorities in the U.S. and other countries is a significant factor in the

development, manufacture, distribution and marketing of RYTELO (imetelstat). Imetelstat will require regulatory approval

by regulatory authorities prior to commercialization in any jurisdictions where it is not yet approved. In particular, potential

human therapeutic products, such as imetelstat, are subject to rigorous preclinical testing, clinical testing and quality

standards by the FDA and similar regulatory authorities in European and other countries. Various statutes and regulations

at the federal and state level—as well as regulations and guidance at the international level—also govern or influence the

testing, manufacturing, safety reporting, labeling, storage, import, export, distribution, sale and recordkeeping related to

such products and their marketing. The process of obtaining these approvals and the subsequent compliance with

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appropriate statutes and regulations require the expenditure of substantial time and money, and there can be no guarantee

that approvals will be granted. Moreover, compliance with government regulations governing personal data and

information security requires the expenditure of substantial time and financial resources. The information provided in this

section should be reviewed in the context of the sections entitled “Risks Related to the Further Development of RYTELO

(Imetelstat)” and “Risks Related to Regulatory Approval of RYTELO” under Part I, Item 1A, “Risk Factors” of this

Report.

United States Food and Drug Administration Regulatory Approval Process

Prior to commencement of clinical trials of new pharmaceutical products in humans, government

authorities generally require the completion of laboratory studies as well as certain preclinical testing in animals to evaluate

the potential efficacy and safety of a product candidate, though, under the 2022 FDA Modernization Act 2.0, FDA has

begun to phase out the requirement for animal testing by incorporating new approval methodologies such as AI-based

computational models, organ-on-a-chip systems, and advanced in vitro assays. The results of animal testing or alternative

models are submitted to the FDA as part of an Investigational New Drug Application, or IND, which must become

effective before clinical testing in humans can begin. The FDA can place an IND on clinical hold at any time, which can

prevent the conduct of a clinical trial or all trials under an IND or it may be a partial hold (e.g., a hold on further

enrollment) until safety concerns or questions are addressed by the IND sponsor to the FDA’s satisfaction.

Typically, a clinical development program is lengthy and involves three phases. In Phase 1, clinical trials

are conducted with a small number of healthy volunteers or patients afflicted with a specific disease to assess safety and to

evaluate the metabolism, pharmacokinetics, and pharmacologic action of the drug in humans, and, if possible, to gain early

evidence on effectiveness. In Phase 2, clinical trials are typically well controlled, closely monitored, and conducted in a

relatively small number of patients, usually involving no more than several hundred subjects to evaluate the effectiveness

of the drug in patients to determine the common short-term side effects and risks associated with the drug. Phase 2 trials

can be conducted comparing the investigational treatment to a comparator arm, or not. If used, a comparator arm often

includes standard of care therapy. Safety and efficacy data from Phase 2 clinical trials, even if favorable, may not provide

sufficient rationale for proceeding to a Phase 3 clinical trial. In Phase 3, clinical trials are typically large scale, multi‑center,

comparative trials conducted with patients with the disease or condition under study and are intended to gather sufficient

data to demonstrate the efficacy and safety required by the FDA. The FDA closely monitors the progress of each of the

three phases of clinical testing and may, at its discretion, re‑evaluate, alter, suspend, or terminate the trials. Human clinical

trials must be conducted in compliance with Good Clinical Practice, or GCP, regulations and applicable laws, with

informed consent from patients and the oversight of Institutional Review Boards for the protection of human subjects.

Drugs used in clinical trials must be manufactured, packaged and labeled in conformity with current Good Manufacturing

Practices, or cGMP, and applicable laws.

The results of the preclinical and clinical testing of drugs and complete manufacturing information are

submitted to the FDA in the form of an NDA for review and approval prior to commencement of commercial sales.

Submission of an NDA requires the payment of a substantial user fee to the FDA, which may be waived in certain cases. In

responding to an NDA submission, the FDA may approve the drug for commercialization, impose limitations on its

indications for use and labeling, including in the form of Risk Evaluation and Mitigation Strategies or may issue a complete

response letter explaining the reason the NDA cannot be approved in its present form. Even if an NDA is approved, its

sponsor and drug will continue to be subject to ongoing and pervasive regulatory compliance requirements.

European Union and Other Regulatory Approval Process

Prior to initiating clinical trials in a region outside of the U.S., a clinical trial application must be submitted

and reviewed by the appropriate regulatory authority governing clinical trials in the country in which the trial will be

conducted. Whether or not FDA clearance or approval has been obtained, approval or authorization of a product by

comparable regulatory authorities in the EU and other countries is necessary prior to marketing the product in such

countries. The competent regulatory authorities may impose their own requirements and may refuse to grant an approval,

or may require additional data before granting it, even though the relevant product has been cleared or approved by the

FDA or another authority. As with the FDA, the regulatory authorities in the EU and other developed countries have

lengthy approval processes for pharmaceutical products. The process for gaining approval in particular countries varies, but

it generally follows a similar sequence to that described for FDA approval. In Europe, the EMA and the CHMP provide a

mechanism for EU member states to exchange information on all aspects of product licensing. The EU has established the

EMA for the evaluation of medical products, with a centralized procedure which is mandatory for orphan and oncology

products and which grants a single marketing authorization valid in all EU member states.

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Fraud and Abuse, and Transparency Laws and Regulations

We may also be subject to additional regulation and enforcement by the federal government and by

authorities in the states and foreign jurisdictions in which we conduct our business. These additional regulations could

affect our current and future arrangements with healthcare professionals, principal investigators, consultants, customers and

third‑party payors. Such laws include, without limitation, state and federal bribery/anti‑kickback, the False Claims Act,

privacy and data security laws, and healthcare professionals payment transparency laws.

The federal Anti‑Kickback Statute makes it illegal for any person or entity, including a prescription drug

manufacturer (or a party acting on its behalf) to knowingly and willfully, directly or indirectly, solicit, receive, offer, or pay

any remuneration that is intended to induce the referral of business, including the purchase, order, or lease of any good,

facility, item or service for which payment may be made under a federal healthcare program, such as Medicare, Medicaid

TRICARE, and the Veterans Health Care Program. The term “remuneration” has been broadly interpreted to include

anything of value. Several courts have interpreted the statute’s intent requirement to mean that if any one purpose of an

arrangement involving remuneration is to induce referrals, the Anti‑Kickback Statute has been violated. In addition, a

person or entity does not need to have actual knowledge of the statute or specific intent to violate, in order to commit a

violation.

Federal civil and criminal false claims and false statement laws, including the federal civil False Claims Act

and its whistleblower or qui tam provisions (which permit private individuals to bring an action on behalf of the

government to enforce the civil False Claims Act), prohibit, among other things, any person or entity from knowingly

presenting, or causing to be presented, for payment to, or approval by, federal programs, including Medicare and Medicaid,

claims for items or services, including drugs, that are false or fraudulent or not provided as claimed. Entities can be held

liable under these laws if they are deemed to “cause” the submission of false or fraudulent claims by, for example,

providing inaccurate billing or coding information to customers, promoting a product off‑label, or for providing medically

unnecessary services or items. In addition, a claim including items or services resulting from a violation of the federal Anti-

Kickback Statute constitutes a false or fraudulent claim for purposes of the federal civil False Claims Act. Criminal

prosecution is also possible for making or presenting a false, fictitious or fraudulent claim to the federal government.

The federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, created criminal and

civil liability for, among other things, knowingly and willfully executing, or attempting to execute, a scheme to defraud any

healthcare benefit program, including private third‑party payors, knowingly and willfully embezzling or stealing from a

healthcare benefit program, willfully obstructing a criminal investigation of a healthcare offense, and knowingly and

willfully falsifying, concealing or covering up a material fact or making any materially false, fictitious or fraudulent

statement in connection with the delivery of or payment for healthcare benefits, items or services.

HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act, or

HITECH, and their implementing regulations, impose obligations, including mandatory contractual terms, with respect to

safeguarding the privacy, security, transmission and breach reporting of individually identifiable health information, upon

entities subject to the law, such as health plans, healthcare clearinghouses and certain healthcare providers and their

respective business associates and their subcontractors that perform services for them that involve individually identifiable

health information.

The federal Physician Payments Sunshine Act requires certain manufacturers of drugs, devices, biologics

and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance

Program to report annually to the Centers for Medicare & Medicaid Services, or CMS, information related to payments or

other transfers of value made to physicians (defined to include doctors, dentists, optometrists, podiatrists, and

chiropractors), other healthcare professionals (such as physicians assistants and nurse practitioners), and teaching hospitals,

and information related to ownership and investment interests held by physicians and their immediate family members.

Analogous state and foreign laws and regulations, such as state anti‑kickback and false claims laws, may

apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by

non‑governmental third-party payors, including private insurers. Additionally, we may be subject to state and foreign laws

that require pharmaceutical companies to comply with the pharmaceutical industry’s otherwise voluntary compliance

guidelines and certain industry compliance guidance documents. Further, we may be subject to state and foreign laws that

require drug manufacturers or other pharmaceutical companies to report information related to payments and other

transfers of value to physicians, other healthcare providers and healthcare entities, or marketing expenditures, as well as

state, foreign and local laws that require the registration of pharmaceutical sales representatives; state and foreign laws that

require the reporting of information related to drug pricing; and state, federal and foreign laws governing the privacy and

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security of personal data (including key-coded data and health information), including the European Union’s General Data

Protection Regulation, or EU GDPR, many of which differ from each other in significant ways, thus complicating

compliance efforts.

If our operations are found to be in violation of any of these or any other healthcare regulatory laws that

may apply to us, we may be subject to significant penalties, including the imposition of significant civil, criminal and

administrative penalties, damages, monetary fines, disgorgement, imprisonment, possible exclusion from participation in

Medicare, Medicaid and other federal healthcare programs, reputational harm, diminished profits and future earnings,

additional reporting requirements and oversight if we become subject to a corporate integrity agreement or similar

agreement to resolve allegations of non-compliance with these laws, and curtailment of our operations, any of which could

adversely affect our ability to operate our business and our results of operations. Defending against any such actions can be

costly, time-consuming and may require significant financial and personnel resources. Therefore, even if we are successful

in defending against any such actions that may be brought against us, our business may be impaired.

Data Privacy and Security

In the ordinary course of our business, we process personal or sensitive data. Accordingly, we are, or may

become, subject to numerous data privacy and security obligations, including federal, state, local, and foreign laws,

regulations, guidance, and industry standards related to data privacy and security. Efforts to ensure that our current and

future business arrangements will comply with applicable data privacy and data security laws and regulations will involve

substantial costs. For example, foreign data privacy and security laws (including but not limited to the EU GDPR and UK

GDPR) impose strict significant and complex compliance obligations on entities that are subject to those laws. As one

example, the EU GDPR applies to any company established in the EEA and to companies established outside the EEA that

process personal data in connection with the offering of goods or services to data subjects in the EEA or the monitoring of

the behavior of data subjects in the EEA. These obligations in the EU and UK include limiting the collection and

processing of personal data to only what is necessary for specified, explicit, and legitimate purposes; requiring a legal

basis for personal data processing; requiring the appointment of a data protection officer in certain circumstances;

increasing transparency obligations to data subjects; requiring data protection impact assessments in certain circumstances;

limiting the duration for which personal data may be retained; increasing rights for data subjects formalizing a heightened

and codified standard for data subject consent, requiring the implementation and maintenance of technical and

organizational safeguards for personal data, mandating data breach notifications to relevant supervisory authority(ies), and

mandating the appointment of representatives in the UK and/or the EU in certain circumstances. Moreover, there have

recently been, and we expect that there will continue to be, new data privacy and security laws, regulations and industry

standards in the U.S. As one example, the California Consumer Privacy Act of 2018, or CCPA, imposes numerous

obligations on covered business. Although the CCPA exempts certain data (such as some data processed in the context of

clinical trials), the CCPA, to the extent applicable to our business and operations, may increase our compliance costs and

potential liability with respect to the personal data we maintain about California residents. The CCPA provides for civil

penalties and a private right of action for data breaches which may include an award of statutory damages. Failure, or

perceived failure, to comply with all applicable obligations could result in enforcement actions, fines, litigation, and other

consequences. See the section titled “We and third parties with whom we work are subject to stringent and changing U.S.

and foreign laws, regulations, rules, contractual obligations, industry standards, policies and other obligations related to

data privacy and security. Our (or the third parties with whom we work) actual or perceived failure to comply with such

obligations could lead to regulatory investigations or actions; litigation; fines and penalties; disruptions of our business

operations; reputational harm; loss of revenue and profits; and other adverse business impacts,” under “Risk Factors” in

Part I, Item 1A of this Report for additional information about the laws and regulations to which we may become subject

and about the risks to our business associated with such laws and regulations.

Coverage and Reimbursement

Significant uncertainty exists as to the coverage and reimbursement status of any product candidate that

receives regulatory approval. In the U.S. and markets in other countries, sales of RYTELO will depend, in part, on the

extent to which third‑party payors provide coverage and establish adequate reimbursement levels.

In the U.S., third‑party payors include federal and state healthcare programs, government authorities,

private managed care providers, private health insurers, employer health plans, and other organizations. Decisions

regarding the extent of coverage and amount of reimbursement to be provided are made on a plan-by-plan basis.

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Reimbursement by a third-party payor may depend upon a number of factors, including the third-party payor’s

determination that a product is safe, effective and medically necessary; appropriate for the specific patient; cost-effective;

supported by peer-reviewed medical journals; included in clinical practice guidelines; and neither cosmetic, experimental,

nor investigational. A third-party payor could also require that certain lines of therapy be completed or failed prior to

reimbursing our therapy. The principal decisions about reimbursement for new medicines are typically made by CMS.

CMS decides whether and to what extent products will be covered and reimbursed under Medicare and private payors tend

to follow CMS to a substantial degree. Third-party payors determine which products and procedures they will cover and

establish reimbursement levels. Third‑party payors are increasingly challenging the price, examining the medical necessity

and reviewing the cost‑effectiveness of medical drug products and medical services, in addition to questioning their safety

and efficacy. Such payors may limit coverage to specific drug products on an approved list, also known as a formulary,

which might not include all of the FDA‑approved drugs for a particular indication. We may need to conduct expensive

pharmacoeconomic studies in order to demonstrate the medical necessity and cost‑effectiveness of RYTELO, in addition to

the costs required to obtain the FDA approvals. Nonetheless, RYTELO may not be considered medically necessary or

cost‑effective. Moreover, the process for determining whether a third‑party payor will provide coverage for a drug product

may be separate from the process for setting the price of a drug product or for establishing the reimbursement rate that such

a payor will pay for the drug product. A payor’s decision to provide coverage for a drug product does not imply that an

adequate reimbursement rate will be approved. Further, one payor’s determination to provide coverage for a drug product

does not assure that other payors will also provide coverage for the drug product, as there is no uniform coverage and

reimbursement policy among third-party payors in the U.S. Adequate third‑party reimbursement may not be available to

enable us to maintain price levels sufficient to realize an appropriate return on our investment in RYTELO. Even if a third-

party payor covers a particular product or procedure, the resulting reimbursement payment rates may not be adequate.

Coverage policies and third-party payor reimbursement rates may change. Thus, even if favorable coverage and

reimbursement status is attained, less favorable coverage policies and reimbursement rates may be implemented in the

future. These third-party payors are increasingly reducing coverage and reimbursement for medical products, drugs and

services. In addition, the U.S. government, state legislatures and foreign governments have continued implementing cost-

containment programs, including price controls, restrictions on coverage and reimbursement and requirements for

substitution of generic products. Adoption of price controls and cost-containment measures, and adoption of more

restrictive policies in jurisdictions with existing controls and measures, could further limit sales of any product. Decreases

in third-party reimbursement for any product or a decision by a third-party payor not to cover a product could reduce

demand for the product and also have a material adverse effect on future sales.

Healthcare Reform

There has been increasing legislative and enforcement interest in the U.S. with respect to specialty drug

pricing practices. Specifically, there have been several recent U.S. Congressional inquiries, Presidential executive orders,

and federal and state legislative activity designed to, among other things, bring more transparency to drug pricing, review

the relationship between pricing and manufacturer patient programs, reduce the cost of drugs under Medicare, and reform

government program reimbursement methodologies for drugs. For example, the Inflation Reduction Act of 2022, or IRA,

which, among other things, (i) directs the Department of Health and Human Services, or HHS, to negotiate the price of

certain high-expenditure, single-source drugs and biologics covered under Medicare that have been on the market for at

least seven years (the “Medicare Drug Price Negotiation Program”), and subject drug manufacturers to civil monetary

penalties and a potential excise tax by offering a price that is not equal to or less than the negotiated “maximum fair price”

for such drugs and biologics under the law, and (ii) imposes rebates with respect to certain drugs and biologics covered

under Medicare Part B or Medicare Part D to penalize price increases that outpace inflation. The IRA permits HHS to

implement many of these provisions through guidance, as opposed to regulation, for the initial years. These provisions

began to take effect progressively in fiscal year 2023. The agreed upon prices of the first ten drugs that were subject to

price negotiations took effect on January 1, 2026, although the Medicare Drug Price Negotiation Program is currently

subject to legal challenges. In2025, HHS selected fifteen additional products covered under Part D for price negotiation and

announced “maximum fair prices” that will go into effect on January 1, 2027. Each subsequent year, more Part B and Part

D products will become subject to the Medicare Drug Price Negotiation Program. Further, on December 7, 2023, an

initiative was announced to evaluate whether the use of march-in authorities under the Bayh-Dole Act could impact the

price of or promote equitable access to prescription drugs. On December 8, 2023, the National Institute of Standards and

Technology published for comment a Draft Interagency Guidance Framework for Considering the Exercise of March-In

Rights, or the Draft Framework, which contemplated the price of a product as one of several factors an agency could use

when deciding to exercise march-in rights. The Bayh-Dole Act does not include product pricing as an express basis for

exercising march-in rights, and prior rulemaking initiatives have not adopted proposed regulatory revisions that would have

permitted the exercise of march-in rights on the basis of product pricing. While march-in rights have not previously been

exercised, it is uncertain if that will continue if the Draft Framework ultimately is adopted. Additionally, at the state level,

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legislatures have increasingly passed legislation and implemented regulations designed to control pharmaceutical and

biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product

access and marketing cost disclosure and transparency measures, and, in some cases, to encourage importation from other

countries and bulk purchasing.

The U.S. and some foreign jurisdictions are considering or have enacted legislative and regulatory

proposals to contain healthcare costs, as well as to improve quality and expand access. For example, the IRA also

eliminated the “donut hole” under the Medicare Part D program by significantly lowering the beneficiary maximum out-of-

pocket cost and creating a new manufacturer discount program. It is possible that the IRA will be subject to additional

judicial or Congressional challenges in the future. We expect that other healthcare reform measures that may be adopted in

the future may result in more rigorous coverage criteria and lower reimbursement, and additional downward pressure on

the price that may be charged for RYTELO. It is unclear how any such healthcare reform measures will impact the

pharmaceutical industry.

In addition, aggregate reductions to Medicare payments to providers of up to 2% per fiscal year, which

went into effect beginning on April 1, 2013 under provisions of the Budget Control Act of 2011 will stay in effect through

2032 unless additional Congressional action is taken.

Information About Our Executive Officers

The following table sets forth certain information with respect to our executive officers and other members

of management as of January 31, 2026:

Name	Age	Position
Executive Officers
Harout Semerjian	55	President, Chief Executive Officer and Board member
Michelle Robertson	59	Executive Vice President, Finance, Chief Financial Officer and Treasurer
Joseph Eid, M.D.	58	Executive Vice President, Research and Development
Ahmed ElNawawi	45	Executive Vice President, Chief Commercial Officer

Harout Semerjian has served as our President and Chief Executive Officer and member of our Board

since August, 2025. Prior to joining the Company, Mr. Semerjian served as President and Chief Executive Officer of

GlycoMimetics, Inc., a late-stage clinical biotechnology company subsequently combined with Crescent Biopharma, Inc.,

from August 2021 until February 2025. He also previously served on the board of directors of GlycoMimetics until

February 2025 . Since October 2023, Mr. Semerjian has also served as a member of the board of directors at the

Biotechnology Innovation Organization. From June 2020 to July 2021, Mr. Semerjian served as an independent healthcare

consultant at Emerge Bio Consulting, advising private equity firms on healthcare investment projects. Mr. Semerjian

served as President and Chief Executive Officer of Immunomedics Inc., a biotechnology company specializing in antibody-

drug conjugates for cancer treatment, from April 2020 to May 2020, prior to its acquisition by Gilead Sciences, Inc. From

March 2018 to April 2020, Mr. Semerjian served as Executive Vice President, Chief Commercial Officer of Ipsen Pharma,

a global, pharmaceutical company focusing on areas of high unmet medical need, leading and executing Ipsen’s global

commercial strategy and functions across oncology, neurosciences and rare diseases. From February 2017 to February

2018, he served as President and Head of Ipsen’s Specialty Care International Region & Global Franchises. From 1994 to

January 2017, Mr. Semerjian held several commercial, marketing and sales positions of increasing responsibility within

Novartis Pharmaceuticals, a global pharmaceutical company, including serving as Senior Vice President and Global

Launch Leader for KISQALI®, in regional vice president hematology and oncology roles in the U.S., MENA and the

Nordics, and as global brand director for Gleevec®, as well as for Merck, a global pharmaceutical company, and Solvay, a

multinational chemical and materials company. Mr. Semerjian holds an MBA from Cornell University and Queen's

University in Canada, and a B.S. in Biology from Lebanese American University.

Michelle Robertson has served as our Executive Vice President, Chief Financial Officer and Treasurer

since September 2023. Prior to joining Geron, she served as the Chief Financial Officer and Treasurer of Editas Medicine,

Inc., a CRISPR genome editing company, from January 2020 to May 2023. Before that, she served as Chief Financial

Officer of Momenta Pharmaceuticals, Inc. from 2018 until 2020, when Momenta was acquired by Johnson & Johnson.

Prior to joining Momenta, Ms. Robertson held multiple commercial finance roles of increasing responsibility, including

Vice President, Oncology Finance for Baxalta Incorporated following its spin-off from Baxter International Inc., from 2015

to 2016; Head of Financial Planning and Analysis and Operations Excellence at Ironwood Pharmaceuticals, Inc. from 2012

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to 2015; and various finance and commercial operations roles at Genzyme Corporation (acquired by Sanofi). She also

currently serves as a member of the board of directors and as the chair of the audit committee for Verastem, Inc., a

publicly-traded biopharmaceutical company. Ms. Robertson received her B.S. in Finance and A.S. in Accounting and

Management from Bentley University.

Joseph Eid, M.D., has served as our Executive Vice President Research and Development since November

Prior to joining Geron, Dr. Eid served as President, Research and Development for Dragonfly Therapeutics, Inc., a

clinical stage biopharmaceutical company, with overall responsibilities for Dragonfly's discovery and clinical research

strategy and execution, from February 2023 to March 2024. Before joining Dragonfly Dr. Eid served as Executive Vice

President, Chief Medical Officer at Luzsana Biotechnology, Inc., a pharmaceutical company and a subsidiary of Hengrui

Pharmaceuticals, from October 2021 to September 2022. Prior to his biotechnology leadership roles, Dr. Eid served as

Senior Vice President and Head, Global Medical Affairs for Bristol Myers Squibb, or BMS, from 2017 to 2021, where he

led global medical affairs across four therapeutic franchises. Prior to BMS, Dr. Eid spent nine years at Merck, first at

Merck Research Labs, where he led the first-in-human strategy of their global KEYTRUDA® program, and then at Merck

Global Human Health, where he built Merck's global oncology medical affairs team. Dr. Eid started his pharmaceutical

career at Hoffmann La Roche, where he was responsible for both early- and late-stage assets and led several clinical teams.

Prior to entering the biopharmaceutical industry, Dr. Eid was an Assistant Professor in the hematology department of

Robert Wood Johnson Medical School in New Jersey from 1999 to 2004 and as a volunteer, through 2019. Dr. Eid

received his M.D. from Saint Joseph University, Faculty of Medicine, and serves on ALSAC/St Jude Children’s Research

Hospital board, and on the board of Angle PLC, a liquid biopsy company.

Ahmed ElNawawi has served as our Executive Vice President, Chief Commercial Officer since October

Prior to joining the Company, Mr. ElNawawi most recently served as Senior Vice President and U.S. Commercial

Head at Stemline Therapeutics, a wholly-owned subsidiary of the Menarini Group, from April 2022 until October 2025,

where he led the U.S. commercial organization spanning the sales, marketing, market access, commercial excellence, and

data analytics functions. Before joining Stemline, Mr. ElNawawi spent nearly two decades at Novartis Oncology, from

April 2004 until April 2022, in roles of increasing responsibility, including Oncology General Manager for Romania and

the Gulf region, Executive Director for U.S. Melanoma, and Global Indication Lead for both breast and lung cancer.

Earlier in his career, he held commercial and marketing positions in Egypt, the UAE, and Saudi Arabia with Merck and

Schering-Plough. He received an MBA from the University of Leicester and a B.S. in Clinical Pharmacy from Ain Shams

University in Cairo, Egypt.

Human Capital

Corporate Values

Fostering and maintaining a strong, healthy culture is a key strategic focus. We recognize and value the

unique strengths of each of our team members, and the impact and contributions of every employee.

Our core values are the foundational principles of our organization. These values reflect who we are, how

we work and the way our employees interact with one another, our partners, our communities, and our shareholders. They

are the essential tenets that guide our business decisions, govern our relationships, both internally and externally, and

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articulate what we stand for and who we are. These values dictate the ways in which we interact, work and communicate,

how we resolve conflicts and ultimately, how we strive to make Geron successful.

Our team of talented professionals is the foundation of our company and fuels our historical and

prospective achievements for patients. We consider the intellectual capital of our employees to be an essential driver of our

business and key to our future opportunities. As of December 31, 2025 , we had 258 full‑time employees. However, in

December 2025, we announced a strategic restructuring plan that is intended to position us for long-term value creation and

improve our financial discipline. The restructuring plan will result in a reduction in headcount of approximately one-third

of our workforce. We anticipate that the restructuring plan will be substantially completed in the first quarter of 2026. See

Note 16 on Restructuring in Notes to Consolidated Financial Statements of this Report for additional information . Every

employee plays a vital role in furthering our business goals and advancing the development and delivery of our novel

medicine to patients.

In addition to our employee base, we have established, and expect to continue to establish, consulting

agreements with drug development professionals, clinicians, attorneys and regulatory experts with experience in numerous

fields, including clinical science, biostatistics, clinical operations, pharmacovigilance, quality, manufacturing and

regulatory affairs.

To succeed in our mission, we must attract, recruit, retain, develop and motivate qualified clinical,

nonclinical, commercial, scientific, manufacturing, regulatory, management and other personnel needed to support our

business and operations. As a biotechnology company with office locations in the San Francisco Bay Area and northern

New Jersey, and with remote employees throughout the U.S., we operate in a highly competitive industry and geographies

for employee talent. We maintain a comprehensive dashboard of measurements, including recruitment productivity,

employee engagement scores, total rewards benchmarking, participation rates and satisfaction scores for internal training,

turnover rates and exit interview results, to guide our human capital management efforts.

We believe that our ability to attract highly skilled and talented employees in a competitive labor market is

enhanced by nurturing our workplace culture, providing competitive compensation and benefits programs and supporting

employee career development and related management training. To that end, we continue to invest resources and energy

into being an employer of choice – attracting and engaging individuals who are innovative, curious, driven, diligent,

collaborative and of the highest integrity and ethics. Some of our key efforts in this area and management of our human

capital assets generally are described here.

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Compensation and Benefits

Our compensation philosophy is to provide pay and benefits that are competitive in the biotechnology and

pharmaceutical industry where we compete for talent. We monitor our compensation programs closely and review them

annually to provide what we consider a competitive mix of compensation and health, welfare and retirement benefits for all

our employees. Our compensation package for all employees includes market-competitive base salaries, eligibility for

annual performance bonuses or incentive compensation for field- based roles, and equity grants. Annual cash bonus

opportunity and equity compensation increase as a percentage of total compensation based on level of responsibility.

Actual bonus payouts for annual performance bonus are generally based on a combination of achievement of our annual

corporate goals and individual performance, with our CEO’s annual performance bonus being based entirely on the level of

achievement of our annual corporate goals . All regular-status, full-time employees are eligible to participate in our

comprehensive benefit program, pursuant to plan terms and conditions. Plan choices include medical, dental, vision, life

insurance, flexible spending accounts, short and long-term disability insurance, a 401(k) retirement savings plan with a

discretionary matching employer contribution, and an employee stock purchase plan. We also provide regular-status, full-

time employees with a generous time off program that includes vacation, sick, holiday, and paid leave for certain life

events.

Every year, we undertake a detailed review of our compensation by position and level and make

adjustments necessary to ensure that we continue to provide competitive compensation. We publish pay ranges in all job

postings for jobs as required by various states’ pay disclosure requirements.

Corporate Culture

We pride ourselves on an open culture that respects co-workers, values employees’ health and well-being

and fosters professional development. We support employee growth and development in a variety of ways, including with

group training, individual mentoring and coaching, conference attendance and tuition reimbursement. Our management

conducts annual employee engagement surveys and reports to our board of directors on human capital management topics,

including corporate culture, employee development and retention, and compensation and benefits. Similarly, our board of

directors regularly provides input on important decisions relating to these matters, including with respect to employee

compensation and benefits, talent retention and development.

During 2025, we furthered the development of our hybrid workforce program that provides a variety of

virtual and in-person collaboration opportunities, such as leadership training and coaching resources. Since 2021, we have

utilized a peer-centric employee recognition program to empower employees to champion our workplace culture and

values, and promote direct praise to peers. In addition, we have implemented a reward program that enables managers to

recognize employees who have demonstrated exceptional performance.

Corporate Responsibility Efforts

Our commitment to corporate responsibility is integrated throughout our business and informed by our

values and ambition to change lives by changing the course of blood cancer. To support lower-risk MDS patients eligible

for RYTELO, we have a patient support program called REACH4RYTELO that can help patients navigate access and

coverage. Our corporate responsibility initiatives reflect our commitment to making a difference for blood cancer patients

and health care providers who care for them through RYTELO. Our corporate responsibility priorities also reflect our

commitment to fostering a strong culture for employees and governing with integrity to advance our mission and create

value for stockholders. We review our corporate responsibility practices and disclosures on an ongoing basis.

Communication and Engagement

We believe that part of what sets us apart from other companies is our culture and, in particular, our focus

on providing timely and transparent communications and creating a strong sense of belonging and inclusiveness. We

engage in periodic in-office and in-person meetings and interactions, as well as in-office and in-person training and

development opportunities, to encourage cross-functional team-building and collaboration, in conjunction with which

many of our teams engage in group lunches and dinners. We held a summer contest that encouraged our employees to

share summer travel experiences and special events, building rapport and strengthening employee relationships, and we

conduct organizational and team-specific holiday events to promote connectivity among our employees. We share

information and news with employees through quarterly all-hands meetings, monthly newsletters to employees, social

media posts on our intranet and outward facing social media sites, such as LinkedIn, and regular employee chats with our

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Chief Executive Officer and other members of senior management. We survey our employees each year to measure their

level of engagement at the Company. Our employee engagement scores have remained relatively steady over the past three

years. These surveys provide rich feedback each year that helps us to continue to grow our culture and make Geron a great

place to work.

Health, Wellness and Safety

We offer benefits that promote our employees’ whole health and wellness, including reimbursement for

certain wellness costs, external support from our employee assistance programs and mental wellness services, which covers

therapy and/or coaching for our employees and their dependents, including high school and college-aged children.

None of our employees is subject to a collective bargaining agreement or represented by a trade or labor

union. We consider our relations with our employees to be good.

Corporate and Available Information

Geron Corporation was incorporated in the State of Delaware on November 28, 1990. Geron UK Limited

was incorporated in the United Kingdom on September 29, 2021. Geron Netherlands B.V. was incorporated in the

Netherlands on February 17, 2023. Our principal executive offices are located at 919 E. Hillsdale Blvd., Suite 250, Foster

City, CA 94404, and our telephone number is 650-473-7700. Our website address is http://www.geron.com.

We file or furnish electronically with the U.S. Securities and Exchange Commission, or the SEC, annual

reports on Form 10-K, quarterly reports on Form 10-Q, current reports on Form 8-K and amendments to those reports filed

or furnished pursuant to Section 13(a) or 15(d) of the Securities Exchange Act of 1934, as amended, or the Exchange Act.

We make copies of these reports available free of charge through the “SEC Filings” tab on the “Investors &

Media” page of our website as soon as reasonably practicable after we file or furnish them with the SEC.

Information contained on or accessible through our website is not incorporated into, and does not form a

part of, this Report or any other report or document we file with the SEC, and any references to our website are intended to

be inactive textual references only.

ITEM 1A. RISK FACTORS

We operate in a dynamic and rapidly changing environment involving numerous risks and uncertainties

that may have a material adverse effect on our business, financial condition or results of operations. You should carefully

consider the risks and uncertainties described below, together with all of the other information included in this Report. Our

business faces significant risks and uncertainties, and those described below may not be the only risks and uncertainties we

face. Additional risks and uncertainties not presently known to us or that we currently believe are immaterial may also

significantly impair our business, financial condition or results of operations. If any of these risks or uncertainties occur,

our business, financial condition or results of operations could suffer, the market price of our common stock could decline

and you could lose all or part of your investment in our common stock.

RISKS RELATED TO THE COMMERCIALIZATION OF RYTELO

Our near-term prospects are wholly dependent on RYTELO. We have limited experience with the commercialization of

RYTELO, and if we are unable to successfully commercialize RYTELO in the U.S. for lower-risk MDS, or to expand its

indication of use, our ability to generate meaningful revenue or achieve profitability will be materially and adversely

affected.

In June 2024, we received FDA approval to commercialize RYTELO in the U.S. for certain patients with

lower-risk MDS, and we initiated a commercial launch of RYTELO in the U.S. in that indication. While we have generated

revenue from U.S. sales of RYTELO since mid-2024, the commercialization of RYTELO marks our first effort to

commercially launch a product candidate, and our ability to grow revenue on a sustained basis has not yet been proven.

RYTELO is our only product approved for marketing by the FDA, and our ability to generate meaningful revenue from

product sales and achieve profitability is wholly dependent on our ability to successfully commercialize RYTELO in the

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U.S. for lower-risk MDS or to expand its indications of use. We may not be able to successfully commercialize RYTELO

for a number of reasons, including:

• we may not be able to establish or demonstrate in the medical community the safety and

efficacy of RYTELO and its potential advantages over, and side effects compared to,

existing treatments;

• physicians may be reluctant to prescribe RYTELO until longer-term efficacy and safety

data exists;

• our limited historical experience in marketing, selling and distributing RYTELO;

• we may not be able to increase physician adoption of RYTELO through our

commercialization strategy and plans, including any potential amendments or adjustments

to those plans;

• our inability to achieve and maintain revenue growth in the near future under our current

commercialization strategy and plans or any potential further amendments or adjustments

thereto;

• potential future changes in the reimbursement and coverage policies of government and

private payors such as Medicare, Medicaid, insurance companies, health maintenance

organizations and other plan administrators;

• the relative position of RYTELO as compared to alternative treatment options in healthcare

guidelines and recommendations, such as those developed the National Comprehensive

Cancer Network, or NCCN, and similar guidelines and recommendations outside the U.S.;

• the relative price of RYTELO as compared to alternative treatment options;

• the relatively low incidence and prevalence of patients in RYTELO’s approved indication,

including the reliability of our market and sales estimates;

• the market penetration rate of RYTELO across the breadth of eligible patient segments in

its approved indication may continue to be lower than our expectations;

• our projections regarding the market opportunities for RYTELO may not be accurate, and

the actual market for RYTELO may be smaller than we estimate;

• future competitive or other market factors may adversely affect the commercial potential of

RYTELO;

• we may not be able to obtain and maintain regulatory approvals for RYTELO in any other

jurisdictions for lower-risk MDS or for any other indications, including relapsed/refractory

MF;

• changed or increased regulatory restrictions;

• changes to the label for RYTELO that further restrict how we market and sell RYTELO,

including adverse events observed in ongoing and future studies of imetelstat, such as our

Phase 3 IMpactMF clinical trial;

• the capabilities of third party manufacturers may adversely affect the success of our

commercialization of RYTELO;

• we may need additional financial or other resources that might not be available to us to

successfully commercialize RYTELO; and

• we may not be able to maintain adequate commercial supplies of RYTELO to meet demand

or at an acceptable cost or at all.

Moreover, commercialization of RYTELO may not generate sufficient revenue from product sales, and we

may not become profitable in the near term, or at all. In any event, if we are unable to successfully commercialize

RYTELO in the U.S. for lower-risk MDS, or to expand its indications of use, our ability to generate meaningful revenue

from product sales and achieve profitability will be materially and adversely affected, which in turn would severely and

adversely affect our financial results, business and business prospects, and might cause us to cease operations.

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Although we have established commercial operations and sales, marketing and distribution infrastructure for RYTELO

in the U.S., our commercialization strategy may ultimately be unsuccessful or less successful than anticipated

As a company, we have limited prior experience in selling and marketing or commercializing an approved

drug product in the U.S., and we have no experience selling, marketing or commercializing an approved drug outside of the

U.S. The success of our commercialization efforts is subject to, among other things, managing our internal sales,

marketing, and distribution capabilities and our ability to navigate the significant expenses and risks involved with the

management of such capabilities. Our initial commercialization efforts in the U.S. have not to date achieved meaningful

sales growth. Our RYTELO sales trends and resulting revenue have been and may continue to be variable. Net product

revenue was approximately $ 48.0 million in the fourth quarter of 2025, $47.2 million in the third quarter of 2025, and

$49.0 million in the second quarter of 2025. In an effort to better execute on our plans to commercialize RYTELO and

generate more meaningful product revenue, we decided to focus our commercial execution on targeted engagement with

high-volume accounts that treat earlier-line patients, and invest in non-personal promotion and third-party education to

reach the full breath of the eligible patient population, while cross-functionally collaborating for effective account

management. However, our revised strategy to drive RYTELO commercial growth may still not achieve meaningful sales

growth, which may require us to, among other things, further adjust or amend our commercialization strategy and plans and

incur significant expenses, and there can be no assurance that we will be able to meet or grow projected RYTELO net

product revenue in future periods. In particular, our strategy may not drive new patient starts in lower-risk MDS U.S.

patients, particularly in second-line lower-risk MDS, in a timely manner or at all, or the duration of therapy could be

shorter than we expect, each of which would limit RYTELO's growth potential and could preclude or delay our ability to

generate meaningful revenue from product sales and to achieve profitability. If we are unsuccessful in accomplishing our

objectives from our commercialization efforts and strategy, we may not be able to generate meaningful revenue from the

commercialization of RYTELO in lower-risk MDS, or may require significant additional capital and financial resources to

do make further investment in our commercial operations. Any of these outcomes would severely and adversely affect our

financial results, business and business prospects, and might cause us to cease operations.

In December 2025, we announced a strategic restructuring plan designed to position us for long-term value

creation and improve our financial discipline. However, there can be no assurance that our recent restructuring will result in

long-term value creation or that our commercialization strategy will result in meaningful improvement in our sales

performance. Additionally, we may determine that we need further changes in the future that require additional hiring or

other organizational changes to adequately support our strategy, which could further increase our costs. As such, we

continue to compete with many companies that currently have extensive, experienced and well-funded sales, distribution

and marketing operations to recruit, hire, train and retain marketing and sales personnel. If we are unable to recruit, retain

and effectively train marketing, sales and medical personnel as needed and equip them with compliant and effective

materials, our efforts to successfully commercialize RYTELO could be adversely affected.

Further, although we received marketing authorization for RYTELO in the EU for the treatment of certain

adult patients with TD anemia due to lower-risk MDS in March 2025, we currently have no marketing or sales organization

outside of the U.S., and as a company, we have no experience selling and marketing approved drugs outside of the U.S. To

successfully commercialize RYTELO in the EU or in any other regions outside the U.S. where we might seek marketing

authorization in the future, we will need to develop these capabilities, which we plan to do at this time by working with

experienced third-party contractors or commercialization partners. Doing so will require additional investment of capital

and time. We currently intend to seek contractual arrangements, strategic partnerships, collaborations, alliances or licensing

arrangements with third parties to assist us in the commercialization of RYTELO in the EU and in any other regions

outside of the U.S. Any failure or delay in entering into and conducting such contractual arrangements, strategic

partnerships, collaborations, alliances or licensing arrangements with third parties would adversely impact the

commercialization of RYTELO in the EU or in any other regions outside the U.S. where RYTELO may be approved for

marketing in the future.

If we do not maintain acceptable prices or adequate reimbursement for RYTELO, the use of RYTELO could be severely

limited.

Our ability to successfully commercialize RYTELO will depend significantly on maintaining acceptable

prices and the availability of coverage and adequate reimbursement to patients from third-party payors. Government

payors, such as the Medicare and Medicaid programs, and other third-party payors, such as private health insurers and

health maintenance organizations, determine which medications they will cover and the reimbursement levels. The

resulting reimbursement payment rates may not be adequate or may require significant restrictions on use or increased co-

payments from commercially insured patients that patients may find unacceptably high. Patients are unlikely to use

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RYTELO unless coverage is provided, and reimbursement is adequate to cover all or a significant portion of its cost.

Therefore, coverage and adequate reimbursement are critical to market acceptance of RYTELO.

In the U.S. and some jurisdictions outside the U.S., there have been a number of legislative and regulatory

changes and proposed changes regarding the healthcare system that could impact our business. Generally, there has been

increasing legislative and enforcement interest in the U.S. with respect to drug pricing, including specialty drug pricing

practices, in light of the rising cost of prescription drugs and biologics. Specifically, there have been U.S. Congressional

inquiries and federal and state legislative activity designed to, among other things, bring more transparency to drug pricing,

review the relationship between pricing and manufacturer patient programs, reduce the price of drugs under Medicare, and

reform government program reimbursement methodologies for drugs and biologics. For details regarding these legislative

and regulatory changes and proposed changes regarding the healthcare system that may affect our ability to operate, see

Item 1 “Business - Healthcare Reform” in this Report.

In addition, government authorities and other third-party payors in the U.S. and other jurisdictions are

developing increasingly sophisticated methods of controlling healthcare costs, such as by limiting coverage and the amount

of reimbursement for particular medications. Increasingly, third-party payors are requiring that drug companies provide

them with predetermined discounts from list prices as a condition of coverage, are using restrictive formularies and

preferred drug lists to leverage greater discounts in competitive classes, and are challenging the prices charged for medical

products. The Inflation Reduction Act of 2022 includes several provisions to lower prescription drug costs for people with

Medicare and reduce drug spending by the federal government, including the Medicare Drug Price Negotiation Program,

which may ultimately have a negative effect on the pricing for RYTELO. However, the Medicare Drug Pricing Negotiation

Program provisions of the law are currently subject to legal challenges. Further, the final CY 2026 Medicare Physician Fee

Schedule rule, issued by the Centers for Medicare & Medicaid Services, among other things, increases bona fide service

fee documentation requirements, defines “bundled arrangement,” to require “unbundling” of both contingent and non-

contingent discounts and includes sales of Part B units at the Maximum Fair Price in average sales price calculations. These

changes could lower reimbursement for Medicare Part B utilization and require manufacturers to comply with new,

uncertain or complex reporting obligations and drug pricing documentation practices .

Further, no uniform policy requirement for coverage and reimbursement for drug products exists among

third-party payors in the U.S. As a result, the coverage determination process is often time-consuming and costly, and it

will require us to provide scientific and clinical support for the use of RYTELO to each payor separately, with no assurance

that coverage and adequate reimbursement will be applied consistently or obtained in the first instance.

Although we have received a permanent and product-specific J-Code (J0870) for RYTELO which became

effective on January 1, 2025, coverage may significantly change or may be more limited than the indications for which the

drug is approved by the FDA or similar international regulatory authorities. Coverage and reimbursement may impact the

demand for, or the price of RYTELO, and reimbursement policies in the U.S., the EU, and other jurisdictions may evolve

which may adversely impact our ability to successfully commercialize RYTELO. Even if favorable coverage and

reimbursement status is attained for one or more products for which we receive regulatory approval, less favorable

coverage policies and reimbursement rates may be implemented in the future. If coverage and reimbursement are not

available or the reimbursement amount is inadequate, we may not be able to successfully commercialize RYTELO, which

would negatively impact our business and business prospects.

If future legislation were to impose direct governmental price controls and access restrictions, it could have

a significant adverse impact on our business and financial results. Managed care organizations, as well as Medicaid and

other government authorities, continue to seek price discounts. At the state level, legislatures have increasingly passed

legislation and implemented regulations designed to control pharmaceutical and biologic product pricing, including price or

patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and

transparency measures, and, in some cases, to encourage importation from other countries and bulk purchasing. Due to the

volatility in the current economic and market dynamics, we are unable to predict the impact of any unforeseen or unknown

legislative, regulatory, payor or policy actions, which may include cost containment and healthcare reform measures. Such

policy actions could have a material adverse impact on future sales of RYTELO in the U.S., the EU, and in any other

jurisdictions where we may seek approval in the future.

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To be commercially successful, RYTELO must be accepted by the healthcare community, which can be slow to adopt or

unreceptive to new technologies and products.

Our strategy to drive sales growth and our ongoing commercialization efforts has not to date achieved and

may not in the future achieve the rate of market acceptance by the healthcare community or breadth of eligible patient

segments that we expect, which may require us to, among others, further adjust or amend our commercialization strategy

and plans and incur significant expenses, and there can be no assurance that we will be able to grow RYTELO net product

revenue in future periods. While our current priority is to drive new patient starts across appropriate second-line patients in

RYTELO's approved indication, we may be unable to do so in a timely manner or at all, which would limit RYTELO's

growth potential and which could delay or preclude our ability to generate meaningful revenue from product sales and to

achieve profitability. Furthermore, RYTELO competes with a number of conventional and widely accepted drugs and

therapies manufactured and marketed by major pharmaceutical companies. The degree of market acceptance of RYTELO

depends on a number of factors, including:

• the clinical indications for which RYTELO is or may in the future be approved;

• the establishment and demonstration to the medical community of the clinical efficacy and

safety of RYTELO;

• the ability to demonstrate that RYTELO is superior to alternatives on the market at the time,

including with respect to efficacy, safety, cost or route of administration;

• the willingness of medical professionals to prescribe, and patients to use, RYTELO, or to

continue to use RYTELO;

• the publication of favorable safety or efficacy data concerning RYTELO by third parties or

us;

• restrictions on use of RYTELO alone or in combination with other products;

• the label and promotional claims allowed by the FDA for RYTELO, as well as any such

claims allowed by similar international regulatory authorities for RYTELO, including usage

for only certain indications and any limitations or warnings about the prevalence or severity

of any side effects;

• the timing of market introduction of RYTELO for new indications;

• the effectiveness of sales, marketing and distribution infrastructure for RYTELO;

• the ability of the third-party distributors and specialty pharmacies we contract with to

process prescriptions and dispense RYTELO and the processes required to place orders

with such distributors and specialty pharmacies;

• the extent to which RYTELO is approved for inclusion on formularies in hospitals and

managed care organizations;

• the pricing of RYTELO, both in absolute terms and relative to alternative treatments;

• the availability of coverage and adequate reimbursement by government and third-party

payors; and

• the willingness of patients to pay out-of-pocket in the absence of coverage by third-party

payors, including governmental authorities.

We may be unable to demonstrate any therapeutic or economic advantage for RYTELO compared to

established or standard-of-care therapies, or newly developed therapies, for myeloid hematologic malignancies. National

health insurance and/or third-party payors may decide that any potential benefit that RYTELO may provide to clinical

outcomes in myeloid hematologic malignancies is not adequate to justify the potential adverse effects or the costs of

treatment with RYTELO. If the healthcare community does not accept RYTELO for any of the foregoing reasons, or for

any other reasons, our ability to commercialize RYTELO in the U.S. or the EU for lower-risk MDS or for any other

indications for which RYTELO may be approved, may be negatively impacted or precluded altogether, which would

seriously and adversely affect our business and business prospects.

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If the market opportunities for RYTELO are smaller than we believe, our revenue may be adversely affected, and our

business may suffer.

We are commercializing RYTELO in lower-risk MDS, and the addressable patient population in lower-risk

MDS is based on our estimates. These estimates, which have been derived from a variety of sources, including scientific

literature, surveys of clinics, patient foundations and market research, may prove to be incorrect. Further, new information

from us or others may change the estimated incidence or prevalence of patients with lower-risk MDS in the U.S. or the EU.

Additionally, the potentially addressable patient population for RYTELO may not ultimately be amenable to treatment with

RYTELO, we may be unable to successfully identify patients and achieve a significant market share in all eligible patient

segments in RYTELO’s approved indication, or the duration of therapy for patients receiving RYTELO could be shorter

than we expect, each of which would have a negative impac t on sales of RYTELO in the future and may limit its growth

potential. Our commercialization of RYTELO in the U.S. and our planned commercialization in the EU is limited to certain

patients with lower-risk MDS, and any future potential commercialization will be limited to the therapeutic indications

examined in our clinical trials and approved by the FDA and similar international regulatory authorities, which would

preclude us from marketing RYTELO for any other indications not expressly approved by those regulatory authorities.

Future regulatory approvals for RYTELO, if any, could be conditioned upon label restrictions that materially limit the

addressable patient population.

Our market opportunity may also be limited by the pricing, reimbursement and access we are able to

achieve for RYTELO, the quality and expiration of our intellectual property rights and regulatory exclusivity, duration of

RYTELO treatment in lower-risk MDS and future competitor treatments that enter the market. If any of our estimates

prove to be inaccurate, the market opportunities for RYTELO that we or any potential future collaborative partners develop

could be significantly diminished, which would have a material adverse impact on our business and business prospects, and

would adversely affect our ability to achieve profitability.

We face competition from existing products, product candidates and technologies, and competitors may develop new

products and technologies. If these products, product candidates or technologies are deemed by the healthcare

community to be superior to or more cost-effective than RYTELO, it would significantly impact the development and

commercial viability of RYTELO, which would severely and adversely affect our financial results, business and business

prospects, and the future of RYTELO, and might cause us to cease operations.

The pharmaceutical and biotechnology industries are characterized by intense and dynamic competition

with rapidly advancing technologies and a strong emphasis on proprietary products. While we believe our proprietary

oligonucleotide chemistry; experience with the biological mechanisms related to RYTELO, telomeres and telomerase;

clinical data to date indicating potential disease-modifying activity with RYTELO treatment; and knowledge and expertise

around the development of potential treatments for myeloid hematologic malignancies may provide us with certain

competitive advantages, we face competition from many different sources, including major pharmaceutical, specialty

pharmaceutical and biotechnology companies, academic institutions, governmental agencies, and public and private

research institutions. RYTELO competes with other products and therapies that currently exist, are being developed or will

in the future be developed, some of which we may not currently be aware of. A discussion of current and potential future

competitors of RYTELO can be found in the sub‑section titled “Competition” in Part I, Item 1, titled “Business” in this

Report.

Many of our competitors, either alone or with their strategic partners, have substantially greater financial,

technical and human resources than we do and significantly greater experience in obtaining FDA and other regulatory

approvals of treatments and commercializing those treatments. Smaller companies may also prove to be significant

competitors, particularly through collaborative arrangements with large and established companies. We anticipate increased

competition in the future as new companies explore treatments for myeloid hematologic malignancies, which may

significantly impact the commercial viability of RYTELO. Academic institutions, government agencies and other public

and private research organizations may also conduct research, seek patent protection and establish collaborative

arrangements for research, clinical development and marketing of products similar to RYTELO. These companies and

institutions compete with us in recruiting and retaining qualified development and management personnel as well as in

acquiring technologies complementary to the RYTELO program.

As a result of the foregoing, competitors may develop more commercially desirable or affordable products

than RYTELO. Competitors have developed, or are in the process of developing, technologies that are, or in the future may

be, competitive to RYTELO. Some of these products may have an entirely different approach or means of accomplishing

therapeutic effects similar, or superior, to those that may be demonstrated by RYTELO. Competitors may develop products

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that are safer, more effective, more convenient to administer to patients, or less costly than RYTELO, and, therefore,

present a serious competitive threat to RYTELO. In addition, competitors may price their products below what we may

determine to be an acceptable price for RYTELO, may receive better third-party payor coverage and/or reimbursement, or

may be more cost effective than RYTELO. Such competitive products or activities by competitors may render RYTELO

obsolete, which may cause us to cease any further development or future commercialization of RYTELO, which would

severely and adversely affect our financial results, business and business prospects, and the future of RYTELO, and might

cause us to cease operations.

We rely on a select network of third party distributors, specialty pharmacies and other vendors to distribute RYTELO in

the U.S., and any failure by such distributors, specialty pharmacies and vendors could adversely affect our revenues,

financial condition, or results of operations.

We rely on a select network of third party distributors, specialty pharmacies and other vendors to distribute

RYTELO in the U.S., and the financial failure of any of these parties could adversely affect our revenues, financial

condition or results of operations. We rely on such distributors and specialty pharmacies to effectively distribute RYTELO

in a timely manner, provide certain patient support services, manage prescription intake, collect accurate patient and

inventory data and collect payments from payors. While we have entered into agreements with each of these parties, they

may not perform as agreed, our strategic priorities may change or they may terminate their agreements with us. Further, an

inability by our distributors or specialty pharmacies to meet our patients’ needs may lead to reputational harm or patient

loss. In the event that such network fails to properly meet our or our patients’ needs, we may need to partner with other

distributors, specialty pharmacies or vendors to replace or supplement our current network and there is no guarantee that

we will be able to do so on commercially reasonable terms or at all.

We will be subject to pricing and reimbursement regulations in the EU, which may materially affect our ability to

commercialize and receive reimbursement coverage for RYTELO in the EU.

In March 2025, we received marketing authorization for RYTELO in the EU for the treatment of adult

patients with TD anemia due to lower-risk MDS without an isolated deletion 5q cytogenic abnormality and who had an

unsatisfactory response to or are ineligible for erythropoietin-based therapy. The pricing of RYTELO will be subject to

governmental control and other market regulations which could put pressure on the pricing and usage of RYTELO. In the

EU, pricing negotiations with governmental authorities can take considerable time after obtaining marketing approval of a

product candidate and market acceptance and sales of RYTELO will depend significantly on the availability of adequate

coverage and reimbursement from third-party payors for RYTELO and may be affected by existing and future healthcare

reform measures.

The requirements governing drug pricing and reimbursement vary widely from country to country. For

example, within the EU member states may restrict the range of medicinal products for which their national health

insurance systems provide reimbursement and may control the prices of medicinal products for human use. Reference

pricing used by various EU Member States and parallel distribution, or arbitrage between low-priced and high-priced EU

Member States, can further reduce prices. An EU Member State may approve a specific price for the medicinal product or

it may instead adopt a system of direct or indirect controls on the profitability of the company placing the medicinal

product on the market. In some countries in the EU, we may be required to conduct a clinical study or other studies that

compare the cost-effectiveness of RYTELO to other available therapies in order to obtain or maintain reimbursement or

pricing approval. There can be no assurance that any country that has price controls or reimbursement limitations for

biopharmaceutical products will allow favorable reimbursement and pricing arrangements for RYTELO. Historically,

products launched in the EU do not follow price structures of the U.S. and generally prices tend to be significantly lower.

Publication of discounts by third-party payors or authorities may lead to further pressure on the prices or reimbursement

levels within the country of publication and other countries. If pricing is set at unsatisfactory levels or if reimbursement of

RYTELO is unavailable or limited in scope or amount, our revenues from sales and the potential profitability of RYTELO

in those countries would be negatively affected.

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RISKS RELATED TO REGULATORY APPROVAL OF RYTELO

We may be unable to maintain regulatory approvals for RYTELO in the U.S. and the EU for lower-risk MDS, which

would severely and adversely affect our business and business prospects, and might cause us to cease operations.

In June 2024, we received regulatory approval from the FDA to commercialize RYTELO in the U.S. in

certain patients with lower-risk MDS, and in March 2025, we received marketing authorization from the EC to

commercialize RYTELO in the EU for certain adult patients with TD anemia due to lower-risk MDS. Federal, state and

local governments in the U.S., and regulatory authorities in the EU, have significant regulations in place that may limit or

prevent us from successfully commercializing RYTELO for lower-risk MDS. We do not currently have regulatory

approval for RYTELO in any other jurisdictions or for any other indication, and governments in other jurisdictions have

significant regulations that may limit or prevent us from successfully commercializing RYTELO in other jurisdictions.

The regulatory frameworks and practices of the FDA, EMA/EC and comparable authorities in other

countries may change over time, and we cannot predict the nature, timing, or impact of any such developments. We cannot

project with certainty if or when we might submit or gain regulatory approval for RYTELO for other indications or in other

jurisdictions. The FDA, EMA/EC and other regulatory authorities exercise broad discretion throughout the product review

and approval process, including in determining the specific conditions for submission, including the requirement for a pre-

approval inspection of RYTELO’s listed manufacturing facility. As a result, the FDA, EMA/EC and other authorities may

delay or extend application review, may decline to accept an application for substantive review, or may conclude, after

review, that the information provided is inadequate to obtain or maintain approval of RYTELO. Any such decision could

materially and adversely affect the timing of potential approval and our business prospects. If the FDA, for instance,

determines that an NDA is not sufficiently complete for filing or issues a complete response letter, or CRL, requiring

additional data or clarification, we may be required to resubmit the application and any such resubmission or CRL‑driven

delays could significantly postpone review and potential approval RYTELO for other indications. Even if we resubmit an

NDA, it remains uncertain whether the FDA will ultimately accept the completed application or additional data, and any

such outcome could delay or prevent approval of RYTELO for other indications.

Failure to maintain regulatory approval for RYTELO from the FDA in the U.S. and from the EC in the EU

for lower-risk MDS, or delays in obtaining, failing to obtain, or limitations in the scope of such approvals in any other

jurisdictions or for any other indications, could:

• result in a withdrawal of RYTELO from the market or could otherwise delay, limit or

preclude any revenue we may receive from the commercialization of RYTELO for lower-

risk MDS;

• significantly harm the commercial potential of RYTELO;

• impede, halt or increase the costs of our activities and plans for clinical development;

• diminish any competitive advantages that may have been available to us; or

• delay or preclude any revenue we may receive from the future commercialization of

RYTELO in any other jurisdictions or for any other indications, if any.

In addition, approved products and their manufacturers, together with other vendors involved in the

commercialization process, are subject to continual review, and discovery of previously unknown problems with a product

or its manufacturer, including if regulatory inspectors identify regulatory noncompliance by third-party manufacturers

requiring remediation, may result in restrictions on the product or manufacturer, including import restrictions, seizure, and

withdrawal of the product from the market, or may otherwise cause manufacturing delays and supply disruptions.

Further, if RYTELO causes serious or unexpected side effects, or if other safety risks are observed as a

result of our commercialization efforts for RYTELO in the U.S. or the EU in lower-risk MDS or in current or potential

future clinical trials, a number of potential significant negative consequences could result, including:

• regulatory authorities may withdraw approval of RYTELO;

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• we may be required to recall RYTELO, seek to change the way it is administered, conduct

additional costly, time-consuming and burdensome clinical trials or change the labeling of

the product;

• regulatory authorities may require revisions to the labeling of RYTELO, including

limitations on approved uses or the addition of further warnings, contraindications or other

safety information, or may impose restrictions on distribution in the form of additional

requirements in a risk evaluation and management plan or risk management plan;

• RYTELO may be rendered less competitive and sales, if any, may decrease;

• our reputation may suffer generally among clinicians and patients;

• we may be exposed to potential lawsuits and associated legal expenses, including costs of

resolving claims;

• regulatory authorities may refuse to approve supplements to approved applications filed by

us, or may suspend or revoke license approvals; or

• we may be required to change or stop ongoing clinical trials of RYTELO, which would

negatively impact the development of RYTELO for other potential indications.

Any of these events could prevent us from achieving or maintaining market acceptance for RYTELO, could

substantially increase the costs and expenses of commercializing RYTELO, or could limit its commercial potential, which

in turn could delay or prevent us from generating any meaningful revenues from the sale of the RYTELO. If RYTELO is

approved outside the U.S. and EU, we will be subject to similar requirements, considerations and risks in other regions.

Our regulatory approval for RYTELO in the U.S. and in the EU for certain patients with lower-risk MDS is subject to

post-marketing requirements and commitments, and we may be subject to penalties or product withdrawal if we fail to

comply with these regulatory requirements and commitments, or if we experience unanticipated problems with

RYTELO.

Our regulatory approval for RYTELO in lower-risk MDS in the U.S. and EU are subject to non-clinical,

clinical and manufacturing post-marketing requirements and/or commitments, including the requirement to assess the long-

term safety of RYTELO (imetelstat) in the Phase 3 IMerge trial and a clinical trial to evaluate alternative dosing regimens

in lower-risk MDS, with timelines for completion and reporting established by the FDA. In the EU, our regulatory approval

for RYTELO in certain patients with TD anemia due to lower-risk MDS is subject to our commitment to submit the results

from certain ongoing non-clinical and clinical studies required by the FDA, including the assessment of the long-term

safety of RYTELO in the extension to the Phase 3 IMerge trial. In addition, RYTELO and the manufacturing processes and

facilities, post-approval clinical data, labeling, advertising and promotional activities related to RYTELO will be subject to

continual requirements of, and review by, the FDA and comparable regulatory authorities. These requirements include

submissions of safety and other post-marketing information and reports, compliance with good pharmacovigilance

practices, registration requirements, current Good Manufacturing Practice, or cGMP, requirements relating to quality

control, quality assurance and corresponding maintenance of records and documents, and requirements regarding

promotional interactions with healthcare professionals.

Failure to comply with these post-marketing requirements and commitments on the timeline required or at

all, or any other regulatory requirements, including the FDA’s regulation of promotional claims, or later discovery of

previously unknown problems with RYTELO, or our manufacturers, or manufacturing processes for RYTELO, may result

in actions such as:

• adverse regulatory inspection findings;

• restrictions on RYTELO manufacturing, distribution or use;

• restrictions on, or prohibitions against, marketing, importing or exporting RYTELO;

• additional post-marketing requirements or commitments;

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• fines, untitled letters, warning letters or the withdrawal of RYTELO from the market;

• voluntary or mandatory product recalls or public notification or medical product safety

alerts to healthcare professionals;

• suspension or termination of ongoing clinical trials of imetelstat in other indications;

• suspension of review or refusal to approve pending applications or supplements to approved

applications;

• exclusion from participation in government-funded healthcare programs and/or eligibility

for the award of government contracts for RYTELO; suspension or withdrawal of

regulatory approval for RYTELO;

• significant civil, criminal and administrative penalties, including fines, restitutions or

disgorgement of profits or revenues;

• product seizure or detentions;

• injunctions or the imposition of civil or criminal penalties; and

• adverse publicity.

The imposition of any of these penalties or other commercial limitations, including equivalent penalties or

commercial limitations imposed by foreign regulatory authorities, could severely and adversely affect our financial results,

business and business prospects, including the commercialization of RYTELO, and might cause us to cease operations.

Similar requirements and related consequences apply outside the U.S.

Any government investigation of alleged violations of law could require us to spend significant time and

resources in response and could generate negative publicity. In addition, the regulations, policies or guidance of the FDA,

EC or any other regulatory authority may change and new or additional statutes or government regulations may be enacted

that could prevent or delay regulatory approval of our product candidates or further restrict or regulate post-approval

activities. We also cannot predict the likelihood, nature, or extent of adverse government regulation that may arise from

pending or future legislation or administrative action, either in the U.S. or abroad.

If we are unable to fulfill the post-marketing requirements and commitments established by the FDA, or

that may be or are applied to the approval and commercialization of RYTELO by any regulatory authority, or are unable to

adapt to changes in existing regulatory requirements or adoption of new regulatory requirements or policies, there may be a

negative impact to our business and continued regulatory approval of RYTELO. Under such circumstances, we or our

respective service providers may be subject to the actions listed above, including losing marketing approval for RYTELO,

which would severely and adversely affect our business and business prospects, and might cause us to cease operations.

Furthermore, in connection with our marketing authorization for RYTELO in the EU for certain patients

with TD anemia due to lower-risk MDS, we are subject to post-marketing requirements to submit final results from certain

ongoing non-clinical and clinical studies and the completion of certain quality-related activities and study. We are also

subject to rules and regulations in the EU applicable to the manufacturing, marketing, promotion, and sale of medicinal

products. If we, or a regulatory authority, discover previously unknown problems with RYTELO, such as adverse events of

unanticipated severity or frequency, or problems with a facility where RYTELO is manufactured, a regulatory authority

may impose restrictions relative to RYTELO or the manufacturing facility, including requiring recall or withdrawal of

RYTELO from the market or suspension of manufacturing. Moreover, product labeling, advertising and promotion for

RYTELO will be subject to regulatory requirements and continuing regulatory review.

Failure to comply with EU and EU Member State laws that apply to the conduct of clinical trials,

manufacturing approval, marketing authorization of medicinal products and marketing of such products, both before and

after grant of the marketing authorization, or with other applicable regulatory requirements, or failure to complete post-

marketing requirements on the timeline required, may result in administrative, civil or criminal penalties. This could also

result in delays or refusal to authorize the conduct of clinical trials, or to grant marketing authorization, product

withdrawals and recalls, product seizures, suspension, withdrawal or variation of the marketing authorization, total or

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partial suspension of production, distribution, manufacturing or clinical trials, operating restrictions, injunctions,

suspension of licenses, fines and criminal penalties, among others. If RYTELO is approved outside the U.S. and the EU,

we will be subject to similar requirements, considerations and risks in other regions.

We may be unable to obtain regulatory approval to commercialize RYTELO in any other jurisdictions or for any new

indications, or may experience significant delays in doing so, any of which could severely and adversely affect our

business and business prospects, and might cause us to cease operations.

We may never receive regulatory approval for RYTELO in any other jurisdictions or for any new

indications. It can take many years to obtain approval, if approval is obtained at all. Of the large number of drugs in

development, only a small percentage complete the development and regulatory approval process and are successfully

commercialized. In addition, the lengthy review process and the unpredictability of ongoing or future clinical trials may

result in a delay in obtaining, or our failure to obtain, regulatory approval for RYTELO in lower-risk MDS in any

jurisdictions other than the U.S. and the EU, or for other indications, such as relapsed/refractory MF, in any jurisdictions.

Failure to obtain approval could significantly harm our business and business prospects, render our significant clinical

development expenditures unrecoverable, and might cause us to cease operations.

Securing marketing approval requires the submission of extensive non-clinical and clinical data and

supporting information to regulatory authorities for each therapeutic indication to establish the product candidate’s safety

and efficacy to the satisfaction of such regulatory authorities, as well as information about the product manufacturing

process and any inspections of manufacturing facilities conducted by regulatory authorities through the filing of a New

Drug Application, or NDA, in the U.S. and a Marketing Authorization Application, or MAA, in the EU. Although

RYTELO is approved in the U.S. and the EU in lower-risk MDS, there can be no assurance that we will receive regulatory

approval for the commercialization of RYTELO for lower-risk MDS in any other jurisdiction or for any new indications,

including relapsed/refractory MF or any other indications.

Any marketing approval that we may receive for RYTELO in any other jurisdiction or for any other

indication may also be limited or subject to restrictions or post-approval commitments and requirements that increase our

costs or render RYTELO not commercially viable, which would harm our business and business prospects.

Regulatory authorities may also not approve the labeling claims that are necessary or desirable for the

successful commercialization of a drug, such as RYTELO. For example, although we received regulatory approval from

the FDA in June 2024, and from the EC in March 2025, to commercialize RYTELO in lower-risk MDS, any future

regulatory clearances that we might obtain for RYTELO may be limited to fewer or narrower indications than we might

request, or may be granted subject to the performance of post-marketing studies, which may impose further requirements or

restrictions on the distribution or use of RYTELO, such as limiting prescribing to certain physicians or medical centers that

have undergone specialized training, limiting treatment to patients who meet certain safe-use criteria, and requiring treated

patients to enroll in a registry. These limitations and restrictions may limit the size of the market for RYTELO and affect

reimbursement by third-party payors. Future regulatory clearances, if any, may be limited to a smaller patient population,

or may require a different drug formulation or a different manufacturing process, than we might in the future decide to

seek.

Any delay in obtaining or failure to obtain required approvals of RYTELO in any other jurisdictions or for

any other indications, or limitations on any regulatory approval that we might receive in the future, if any, could reduce the

potential commercial use of RYTELO, and potential market demand for RYTELO and therefore result in decreased

revenue for us from any commercialization of RYTELO in any other jurisdictions or for any other indications, any of

which could severely and adversely affect our financial results and ability to raise additional capital, if needed, the price of

our common stock, our business and business prospects, and might cause us to cease operations.

Although orphan drug designation has been granted to RYTELO for the treatment of MDS and MF in the U.S. and in

the EU, these designations may not be maintained, which would eliminate the benefits associated with orphan drug

designation, including market exclusivity, which could limit the period of exclusivity we are able to maintain for the

commercialization of RYTELO, and would likely harm our business and business prospects.

The FDA granted orphan drug designation to RYTELO in June 2015 for the treatment of MF and for the

treatment of MDS in December 2015, and the EC granted orphan drug designation in December 2015 to RYTELO for the

treatment of MF and in July 2020 for the treatment of MDS. Orphan drug exclusivity confers seven and ten years of

exclusivity in the U.S. and EU, respectively, following approval, subject to satisfying regulatory requirements. The FDA

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has confirmed seven years of orphan drug exclusivity for RYTELO following its approval on June 6, 2024 for its approved

indication in lower-risk MDS. On March 7, 2025, the EC granted marketing authorization for RYTELO as an orphan

medicinal product indicated for the treatment of adult patients with TD anemia due to very low, low or intermediate risk

MDS without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or

are ineligible for erythropoietin-based therapy.

Designation as an orphan drug does n ot guarantee that any regulatory authority will accelerate regulatory

review of, or ultimately approve, RYTELO for any indication, or at all, in the U.S., EU or any other country, nor does it

limit the ability of any regulatory authority to grant orphan drug designation to product candidates of other companies that

treat the same indications as RYTELO if such products are able to demonstrate superiority to RYTELO.

We may lose orphan drug exclusivity in the U.S. for certain reasons, including if the FDA determines that

the request for orphan drug designation was materially defective or if we cannot ensure sufficient quantities of RYTELO to

meet the needs of patients. In the EU, orphan designation may be lost before marketing authorization if it is established that

the criteria for orphan designation are no longer met. Failure to maintain orphan designation status would lead to the

inability to obtain or the loss of associated regulatory exclusivity.

Even if we maintain orphan drug exclusivity for RYTELO in the U.S., the exclusivity may not effectively

protect RYTELO from all competition because different drugs with different active moieties can be approved for the same

condition or a drug containing the same active moiety or principal molecular structure can be approved for a different

indication. Even after an orphan drug product is approved, such as the approval of RYTELO in the U.S. in June 2024 for

certain patients with lower-risk MDS, the FDA can subsequently grant orphan designation to a different drug with the same

active moiety for the same condition, if the FDA concludes that the later drug is safer, more effective, or makes a major

contribution to patient care. The occurrence of any of these events could limit the period of exclusivity we are able to

maintain for RYTELO, allow competitive products to enter the market, and harm our business and business prospects. In

addition, for any other indication that we are currently or may in the future seek to develop or obtain regulatory approval

for RYTELO, orphan drug designation will neither shorten the development time nor regulatory review time for RYTELO

in the US and other comparable jurisdictions, and it does not give RYTELO advantages in the regulatory review or

approval process. Notably, there have been legal challenges to aspects of FDA’s regulations and policies concerning the

exclusivity provisions of the Orphan Drug Act, including whether two drugs are the same drug product. Future challenges

could lead to changes that affect the protections potentially afforded to our products in ways that are difficult to predict.

Similarly, in the EU, orphan market exclusivity may not effectively protect RYTELO from all competition.

Following the authorization of RYTELO as an orphan medicinal product by the EC in March 2025 for certain patients with

lower-risk MDS, the EC can subsequently approve similar orphan medicinal products for the same therapeutic indication or

if we are unable to supply RYTELO in sufficient quantities or if a similar medicinal product with the same orphan

indication is shown to be safer, more effective or otherwise clinically superior to the original orphan medicinal product. If

it is established that the criteria for orphan designation are no longer met inter alia where it is shown that the product is

sufficiently profitable, EU orphan market exclusivity may be reduced to six instead of 10 years, allowing similar medicinal

products for the same therapeutic indication to the market sooner. In the EU, orphan drug designation for any additional

indication we may pursue for RYTELO does confer certain regulatory advantages—such as protocol assistance—but it

does not shorten development timelines or accelerate the EMA’s overall review process.

In April 2023, the EC published a proposal to reform the current pharmaceutical framework, including

revision of orphan market exclusivity. The new legislation, if adopted, is expected to start to apply from mid-2028. The

proposal to reform the current pharmaceutical framework intends to revise the orphan drug designation and exclusivity

regime. In the latest version of the proposal, orphan market exclusivity will be reduced from the current 10 years to 9 years.

Extension by another two years will be possible for so-called “breakthrough orphan medicinal products”. Previous drafts

also included the concept of “global orphan marketing authorization”, which would no longer grant additional separate

orphan market exclusivity for second or further orphan therapeutic indications. Although the final text has not yet been

published and RYTELO for the treatment of MDS is and remains regulated under the current regulatory framework,

authorization of future indications may be affected by the new legislation.

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Even though we reported positive top-line results from IMerge Phase 3 in January 2023 and received regulatory

approval from the FDA in June 2024 to commercialize RYTELO in the U.S. for lower-risk MDS, the top-line results

from IMerge Phase 3 are not necessarily predictive of RYTELO’s activity in other indications, such as in relapsed/

refractory MF.

Even though we reported positive top-line results from IMerge Phase 3 in January 2023 and received

regulatory approval from the FDA in June 2024 and the EC in March 2025 to commercialize RYTELO in lower-risk MDS,

the top-line results from IMerge Phase 3 are not necessarily predictive of RYTELO’s activity in other indications and for

other pivotal trials that may be needed to support any application to the FDA or similar international regulatory authorities

for such other indications, such as in relapsed/refractory MF.

In addition, with respect to the trial design for IMpactMF, our Phase 3 trial in relapsed/refractory MF, the

FDA urged us to consider adding a third dosing arm to the trial to assess a lower dose and/or a more frequent dosing

schedule that might improve the trial’s chance of success by identifying a less toxic regimen and/or more effective spleen

response, one of the trial’s secondary endpoints. Based on data from IMbark, our Phase 2 clinical trial that evaluated two

doses of imetelstat in relapsed/refractory MF and the results of which our IMpactMF trial is based on, we believe that

testing a lower dose regimen would likely result in a lower median OS, which is the trial’s primary endpoint, in the

imetelstat treatment arm. Existing data also suggest that lowering the dose would not result in a clinically meaningful

reduction in toxicity, and for these reasons we determined not to add a third dosing arm to the trial design and the FDA did

not object to our proposed imetelstat sodium dose and schedule of 9.4 mg/kg every three weeks. Our belief may ultimately

be incorrect. Therefore, our failure to add a third dosing arm could result in a failure to maintain regulatory clearance from

the FDA and similar international regulatory authorities for relapsed/refractory MF, could result in the trial’s failure, or

could otherwise delay, limit or prevent marketing approval of imetelstat for relapsed/refractory MF by the FDA or similar

international regulatory authorities.

Regulatory authorities have substantial discretion in the approval process and can delay, limit or deny

approval of RYTELO in other jurisdictions or indications, or require us to conduct additional non-clinical or clinical testing

or abandon a program for many reasons, including:

• disagreement with the design or implementation of our clinical trials, including our

statistical analysis of trial results;

• failure to demonstrate that RYTELO’s efficacy results provide sufficient evidence of

overall clinical benefit;

• unfavorable benefit-to-risk assessment, in the case of marginal efficacy and/or clinically

relevant safety concerns, for any proposed indication;

• serious and unexpected drug-related side effects experienced by participants in our clinical

trials or by individuals using RYTELO or drugs similar to RYTELO;

• disagreement with our interpretation of data from non-clinical studies or clinical trials;

• rejection by the FDA of foreign data included in any future supplemental NDA, or sNDA,

submissions for any future indications and the non-applicability of this data to the U.S.

population and U.S. medical practice;

• identification of critical issues as a result of a pre-approval health authority inspection that

could negatively impact the integrity of data in the MAA and any future sNDA and lead to

a rejection by the FDA, EMA, or similar international regulatory authorities;

• a determination by international regulatory authorities that regulatory approval for

RYTELO should be narrowed or made more restrictive than our current approvals in the

U.S. and the EU for lower-risk MDS or any future indication for which approval is sought,

if any;

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• disagreement regarding the formulation, labeling and/or the specifications for RYTELO;

• the failure of the quality or stability of RYTELO to meet acceptable regulatory standards;

• the EMA or the competent authorities of the individual EU Member States or similar

international regulatory authorities may lack resources or be delayed in conducting pre-

approval inspections due to lack of resources or other reasons;

• we or any third-party service providers may be unable to demonstrate compliance with

GMP, good clinical practices, or GCP, or other applicable regulatory and other

requirements to the satisfaction of the FDA, the EMA, the competent authorities of the

individual EU Member States or similar international regulatory authorities; or

• changes in regulatory policies or approval processes, or potential reduction of unmet

medical need with the entry of competitive therapies to the market, could render our clinical

efficacy or safety data insufficient for approval.

Any of these events may result in a failure to further develop, obtain regulatory approval for or

commercialize RYTELO in any jurisdiction or in any indication other than lower-risk MDS in the U.S. and the EU, which

could severely and adversely affect our business and business prospects.

Furthermore, in recent years, there has been increased public and political scrutiny on the FDA and similar

international regulatory authorities with respect to the approval process for new drugs, and as a result regulatory authorities

may apply more stringent regulatory standards, especially regarding drug safety, when reviewing regulatory submissions.

RISKS RELATED TO COMPLIANCE WITH HEALTHCARE LAWS

Our relationships with healthcare providers, including physicians and third-party payors, the methods by which we

promote RYTELO, and the content of our promotional materials and programs, are subject to applicable promotional,

anti-kickback, fraud and abuse, and other healthcare laws and regulations, and our failure to comply with these laws

could expose us to criminal sanctions, civil penalties, exclusion from federal health care programs, contractual

damages, reputational harm and may adversely affect our business and financial results.

The FDA strictly regulates the promotional claims that may be made about drug products. In particular,

FDA asserts that a product may not be promoted for uses that are not approved by the FDA as reflected in the product’s

approved labeling. The FDA, DOJ and other agencies actively enforce regulations related to the promotion and

advertisement of pharmaceutical products. If our promotional materials or methods were found to have violated the Food,

Drug, and Cosmetic Act, or False Claims Act, we could be subject to Warning or Untitled Letters, or to significant civil,

criminal, or administrative penalties, which could inhibit our ability to commercialize RYTELO and generate revenue,

require us to expend significant time and resources in response, and generate negative publicity.

Healthcare professionals, including but not limited to physicians, nurses, medical directors, hospitals,

pharmacies, pharmacy benefit managers, group purchasing organizations, wholesalers, insurers, and all individuals

employed by such entities (collectively, HCPs), and patients, caregivers, patient advocacy organizations, or medical

societies, may influence the recommendation and prescription of RYTELO. There is ongoing government focus on the

relationships between the pharmaceutical industry and HCPs or others who can influence the prescription or

recommendation of products, and common industry activities that we may engage in such as speaker programs, advisory

boards, consulting agreements with HCPs, relationships with charitable foundations providing copayment assistance, and

relationships with patient organizations and patients continue to receive increased governmental attention.

Our arrangements with HCPs and others who have the ability to influence the recommendation and

prescription of RYTELO may expose us to broadly applicable federal and state fraud and abuse and other healthcare laws

and regulations, including anti-kickback and false claims laws; data privacy and security laws, including the Health

Insurance Portability and Accountability Act, or HIPAA, as amended by the Health Information Technology for Economic

and Clinical Health Act, or HITECH; and transparency laws related to payments and/or other transfers of value made to

physicians, other healthcare professionals and teaching hospitals. These laws may constrain the business or financial

arrangements and relationships through which we conduct our operations, including how we market, sell and distribute

RYTELO. For details regarding the restrictions under applicable federal and state healthcare laws and regulations that may

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affect our ability to operate, see Item 1 “Business-Government Regulation- Fraud and Abuse, and Transparency Laws and

Regulations” of this Report.

Because of the breadth of these laws and the narrowness of the statutory exceptions and safe harbors

available, it is possible that some of our business activities could be subject to challenge under one or more of such laws.

For example, to help patients afford our products, we have a patient assistance program and also occasionally make

donations to independent charitable foundations that help financially needy patients. These types of programs designed to

assist patients in affording pharmaceuticals have become the subject of scrutiny under the AKS and other federal and state

laws in recent years. If we or our vendors or donation recipients are deemed to fail to comply with laws or regulations in

the operation of these programs, we could be subject to damages, fines, penalties or other criminal, civil or administrative

sanctions or enforcement actions. We cannot ensure that our compliance controls, policies and procedures will be sufficient

to protect against acts of our employees, business partners or vendors that may violate the laws or regulations of the

jurisdictions in which we operate. A government investigation could negatively impact our business practices, harm our

reputation, divert the attention of management and increase our expenses.

While we maintain a comprehensive compliance program designed with the goal of ensuring that our

practices and the activities of our third party contractors and employees fall within the scope of available statutory

exceptions and regulatory safe harbors whenever possible, and otherwise comply with applicable laws, it is possible that

government authorities may disagree with our assessment, find fault with the conduct of our employees or contractors or

conclude that our business practices do not comply with current or future statutes, regulations or case law involving

applicable fraud and abuse or other healthcare laws and regulations. If our operations are found to be in violation of any of

these laws or any other governmental regulations that may apply to us, we may be subject to significant civil, criminal and

administrative penalties, damages, fines, exclusion from government funded healthcare programs, such as Medicare and

Medicaid, and the curtailment or restructuring of our operations. Even if we are not determined to have violated these laws,

government investigations into these issues typically require the expenditure of significant resources and generate negative

publicity, or could result in related shareholder lawsuits, any of which would adversely affect our business, financial

condition, results of operations and prospects.

Foreign, federal and state enforcement bodies have increased their scrutiny of interactions between

healthcare companies and healthcare providers, which has led to a number of investigations, prosecutions, convictions and

settlements in the healthcare industry. If our operations are found to be in violation of any of these or any other healthcare

and privacy-related regulatory laws that may apply to us, our ability to operate our business and our results of operations

could be adversely affected by:

• the imposition of significant civil, criminal and administrative penalties, damages, monetary

fines, disgorgement and imprisonment;

• possible exclusion from participation in Medicare, Medicaid and other federal healthcare

programs or comparable foreign programs;

• reputational harm;

• diminished profits and future earnings;

• additional reporting requirements and oversight if we become subject to a corporate

integrity agreement or similar agreement to resolve allegations of non-compliance with

these laws; and

• curtailment of our operations.

Defending against any such actions can be costly, time-consuming and may require significant financial

and personnel resources. Therefore, even if we are successful in defending against any such actions that may be brought

against us, our business may be impaired.

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RISKS RELATED TO THE FURTHER DEVELOPMENT OF IMETELSTAT

We cannot be certain that we will be able to continue to develop imetelstat or advance it in clinical trials, or that we will

be able to receive regulatory approval for imetelstat in any other indications in the U.S., the EU or any other region, on

a timely basis or at all.

We are wholly dependent on the success of RYTELO (imetelstat), which is our only approved product, and

our ability to generate revenue from product sales and achieve profitability is wholly dependent on our ability to

successfully commercialize RYTELO for lower-risk MDS or to expand its indications of use. In this regard, in addition to

lower-risk MDS, which is the only indication for which RYTELO has received marketing approval in the U.S. and the EU,

we are developing imetelstat for the treatment of several myeloid hematologic malignancies. Our ability to further develop

imetelstat and to expand its indications of use to other myeloid hematologic malignancies is subject to significant risks and

uncertainties, including, among other things, our ability to:

• generate sufficient safety and efficacy data from the IMpactMF, IMproveMF, IMpress and

IMAGINE clinical trials, as well as our studies in frontline MF and acute myeloid leukemia

or high-risk MDS, to support any application for regulatory approval, without clinically

meaningful safety issues, side effects or dose-limiting toxicities related to imetelstat that

may negatively impact its benefit-risk profile;

• ascertain that the use of imetelstat does not result in significant systemic or organ toxicities,

including hepatotoxicity, or other safety issues resulting in an unacceptable benefit-risk

profile;

• obtain additional capital, if and when needed, in order to enable us to further advance

imetelstat clinical trials in other myeloid hematologic malignancies;

• obtain and maintain required regulatory clearances and approvals to enable continued

clinical development of imetelstat;

• enter into and maintain commercially reasonable arrangements with third parties to provide

services needed to further research, develop and commercialize RYTELO, including

maintaining the agreements with our contract research organizations, or CROs, and third-

party manufacturers and for territories outside of the U.S. in compliance with applicable

laws;

• recruit and retain sufficient qualified and experienced personnel to support the development

and commercialization of RYTELO in potential other approved indications and other

jurisdictions outside of the U.S. and the EU;

• achieve acceptance of RYTELO treatment by patients and the relevant medical

communities;

• compete effectively with other approved treatments in lower-risk MDS, and relapsed/

refractory MF, if imetelstat is approved in relapsed/refractory MF, and potentially other

myeloid hematologic malignancies;

• obtain appropriate coverage and reimbursement levels for the cost of RYTELO from

governmental authorities, private health insurers and other third-party payors; and

• obtain, maintain and enforce adequate intellectual property and regulatory exclusivity for

RYTELO in the U.S., EU and globally.

If we are not able to successfully achieve these goals and overcome other challenges that we may

encounter in the research, development, manufacturing and commercialization of RYTELO in indications other than lower-

risk MDS, we may be forced to abandon our development and/or commercialization of RYTELO in indications other than

lower-risk MDS, which could severely harm our business and business prospects.

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Our clinical trials of imetelstat could be interrupted, delayed, terminated or abandoned for a variety of reasons which

could severely and adversely affect our financial results, business and business prospects.

The conduct and completion of our clinical trials could be interrupted, delayed or abandoned for a variety

of reasons, including as a result of clinical trial failures, suspensions, terminations or delays related to:

• patient recruitment, enrollment and retention challenges and operational delays, including in

connection with opening new clinical trial sites, while also competing with clinical trials for

other investigational drugs in the same patient population;

• use of OS as a trial endpoint, which inherently requires prolonged periods of clinical

observation and follow-up, including the need for a certain number of events, or deaths, to

occur in IMpactMF prior to the interim or final analysis in that trial of OS;

• use of trial endpoints, including patient-reported outcomes, that necessitate a

comprehensive analysis of the resulting data to determine trial outcomes;

• obtaining and/or maintaining regulatory clearances in the U.S. or other jurisdictions to

commence, conduct or modify current or potential future clinical trials of imetelstat, in a

timely manner, or at all;

• investigational new drug applications, or INDs, and equivalent submissions in other

countries for imetelstat being placed on full or partial clinical hold, suspended or subject to

other requirements by the FDA or other similar international regulatory authorities;

• contracting with a sufficient number of clinical trial sites to conduct current and potential

future clinical trials, and ensuring that such contracts contain all necessary terms and

conditions required by applicable laws, including providing for valid mechanisms to engage

in cross-border data transfers, as well as identifying, recruiting and training suitable clinical

investigators;

• obtaining or accessing necessary clinical data in accordance with appropriate clinical or

quality practices and regulatory requirements, in a timely and accurate manner to ensure

complete data sets;

• responding to safety findings, recommendations or conclusions by the data safety review

committees, independent data monitoring committees and/or expert committees of current

and potential future clinical trials of imetelstat based on emerging data occurring during

such clinical trials;

• manufacturing sufficient quantities that meet our specifications, cost and quality

requirements, and timelines for imetelstat, or for other clinical trial materials, in a manner

that meets the quality standards of the FDA and other similar international regulatory

authorities, and responding to any disruptions to drug supply, clinical trial materials or

quality issues that may arise;

• the effects of macroeconomic or other global conditions, such as inflation, fluctuations in

interest rates, prospects of a recession, government shutdowns, changes in tariffs or other

trade restrictions, bank failures and other disruptions to financial systems, civil or political

unrest, military conflicts, pandemics or other health crises and supply chain and resource

issues;

• complying with current and future regulatory requirements, policies or guidelines, including

domestic and international laws and regulations pertaining to fraud and abuse, transparency,

and the privacy and security of health information;

• reaching agreement on acceptable terms and on a timely basis, if at all, with collaborators,

physician investigators, vendors and other third parties located in the U.S. or other

countries, including our CROs, laboratory service providers and clinical trial sites, on all

aspects of clinical development and collaborating with them successfully; and

• third-party clinical contractors, including investigators or our CROs not performing our

clinical trials according to our anticipated schedule or consistent with the clinical trial

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protocol, GCP, or other regulatory requirements, or not performing data collection or

analyses in a timely or accurate manner.

In addition, recent actions by the current administration in the U.S. to limit federal agency budgets and

personnel have resulted in reductions in the FDA's budget and employees, which may lead to slower response times, longer

review periods, delayed inspections or other disruptions that we cannot currently predict. Failures or delays with respect to

any of the foregoing events and such disruptions in the timely review and processing of our regulatory submissions and

inspections could adversely affect our ability to conduct or complete the clinical trials being conducted by us or our

investigators, or to commence, conduct and complete potential future clinical trials of imetelstat, which could increase

development costs or interrupt, further delay or halt our development of imetelstat, any of which could severely and

adversely affect our financial results, business and business prospects.

RYTELO may cause, or have attributed to it, undesirable or unintended side effects or other adverse events that could

halt or limit its further commercialization, delay or prevent its regulatory approval in any other jurisdiction or

indication, or cause us to delay or terminate our clinical trials.

While the FDA and the EC granted approval of RYTELO based on the data included in our NDA and

MAA, respectively, including data from the Phase 3 IMerge trial, we cannot be certain whether the results as a larger

number of patients receive RYTELO from commercial use, including results related to safety, will be consistent with the

results from earlier clinical trials that served as the basis for its approval.

In addition, because remaining patients in ongoing clinical trials continue to receive imetelstat, additional

or more severe toxicities or safety issues may be observed, and the benefit-risk profile of imetelstat will continue to be

assessed, including the risk of hepatotoxicity, severe cytopenias, fatal bleeding with or without any associated

thrombocytopenia, patient injury or death. New data relating to imetelstat, including from adverse event reports and our

post-marketing requirements in the U.S., and from ongoing clinical trials of imetelstat, may result in changes to the product

label and may adversely affect sales, or result in withdrawal of imetelstat from the market. The FDA and regulatory

authorities in other jurisdictions may also consider the new data in reviewing our marketing applications for additional

indications and/or in other jurisdictions, or impose post-approval requirements. If any of these actions were to occur, it

could result in significant expense and delay or limit our ability to generate sales revenues.

Further, as a result of commercialization of RYTELO, or in current or potential future clinical trials,

RYTELO may cause, or have attributed to it, undesirable or unintended side effects or other adverse events affecting its

safety or efficacy that could interrupt, further delay or halt its commercialization or current or potential future clinical trials.

In this regard, adverse events and dose-limiting toxicities observed in previous and ongoing clinical trials include:

• hematologic toxicities, such as profound and/or prolonged thrombocytopenia or

neutropenia;

• bleeding events, with or without thrombocytopenia, including Grade 3/4 bleeding events;

• febrile neutropenia;

• hepatotoxicity and liver function test abnormalities, as well as hepatic failure;

• gastrointestinal events;

• infection events, with or without neutropenia, including Grade 3/4 infection events;

• muscular and joint pain;

• fatigue;

• headache; and

• infusion-related reactions.

If patients experience similar or more severe adverse events, or new or unusual adverse events, or if the

FDA or other similar international regulatory authorities determine that efficacy and safety data from our

commercialization efforts or in clinical trials do not support an adequate benefit-risk profile to justify continued treatment

of patients, then the FDA or other similar international regulatory authorities may halt or restrict the commercialization of

RYTELO or place one or more of our INDs on clinical hold, as occurred in March 2014. If this were to occur, there could

be a significant delay in, or possible termination of, one or more of our clinical trials, and our commercialization efforts

could be halted, which might cause us to cease operations. If such toxicities or other safety issues identified as a result of

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our commercialization of RYTELO or in any clinical trial are determined by us, the FDA or similar international regulatory

authorities to result in an unacceptable benefit-risk profile, then:

• the FDA and EC could withdraw or restrict regulatory approval for RYTELO in the U.S.

and EU, respectively, for lower-risk MDS;

• additional information supporting the benefit-risk profile of RYTELO may be requested by

the FDA or similar international regulatory authorities and if any such information is not

available or, if available, not deemed acceptable, regulatory approval could be withdrawn

by the FDA in the U.S. and the EC in the EU, and/or current clinical trials could be

suspended, terminated, or placed on clinical hold by the FDA or similar international

regulatory authorities;

• the ability to retain enrolled patients in our current clinical trials may be negatively affected,

resulting in incomplete data sets and the inability to adequately assess the benefit-risk

profile of RYTELO in a specific patient population;

• additional, unexpected clinical trials or non-clinical studies may be required to be

conducted; or

• RYTELO may not receive or maintain regulatory clearances and approvals required to

enable its continued development.

The occurrence of any of these events could interrupt, further delay, or halt our commercialization of

RYTELO or its further development, and as a result, could preclude the commercialization of RYTELO in any additional

indications, as well as increase costs for continued development in additional indications, which would have a severe

adverse effect on our results of operations, financial condition and ability to raise additional capital, business and business

prospects, any of which might cause us to cease operations.

Results and data we disclosed from prior non-clinical studies and clinical trials, as well as any data disclosed as a result

of an interim analysis, may not predict success in later clinical trials or the final analysis, and we cannot assure you that

any ongoing or future clinical trials of imetelstat, including IMpactMF, will lead to similar results and data that could

potentially enable us to obtain any further regulatory approvals.

The design of a clinical trial can determine whether its results will support regulatory approval of a product,

and flaws in the trial design may not become apparent until the clinical trial is well advanced or during the approval

process after the trial is completed. A clinical trial design that is considered appropriate for regulatory approval includes a

sufficiently large sample size with appropriate statistical power, as well as proper control of bias, to allow a meaningful

interpretation of the results. The preliminary results of imetelstat clinical trials with smaller sample sizes can be

disproportionately influenced by the impact the treatment had on a few individuals, which limits the ability to generalize

the results across a broader community, making the trial results of clinical trials with smaller sample sizes less reliable than

trials with a larger number of patients. As a result, there may be less certainty that imetelstat will achieve a statistically

significant effect in any future clinical trials.

Further, success in non-clinical testing and early clinical trials, including Phase 2 clinical trials, such as

IMbark, does not ensure that later clinical trials will be successful, nor does it predict final clinical trial results. In addition,

even though we reported positive top-line results from IMerge Phase 3 in January 2023, this does not ensure that any other

clinical trials of imetelstat will be successful. Later stage clinical trials of imetelstat may fail to show an acceptable benefit-

risk profile despite having progressed through non-clinical studies and initial clinical trials. Many companies in the

biopharmaceutical industry have frequently suffered significant setbacks in later clinical trials, even after achieving

promising results in earlier non-clinical studies or clinical trials.

In general, Phase 3 clinical trials with larger numbers of patients or longer durations of therapy may fail to

replicate efficacy and safety results observed in earlier clinical trials, such as the results observed in IMbark, and if this

were to occur with IMpactMF, this would adversely affect future development prospects of imetelstat, and as a result,

impact the potential commercialization of imetelstat in relapsed/refractory MF.

Furthermore, non-clinical and clinical data are often susceptible to varying interpretations and analyses. In

some instances, there can be significant variability between different clinical trials of imetelstat due to numerous factors,

including changes in trial procedures set forth in trial protocols, differences in the size and type of patient populations, and

changes in and adherence to the dosing regimens. For example, although the statistical analyses comparing IMbark data to

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closely matched real world data, or RWD, published in the September 2021 issue of the Annals of Hematology, suggest

potentially favorable OS in relapsed/refractory MF patients treated with imetelstat, compared to bipolar androgen therapy

using closely matched patients’ RWD, such comparative analyses between RWD and our clinical trial data have several

limitations. For instance, the analyses create a balance between treatment groups with respect to commonly available

covariates, but do not take into account the unmeasured and unknown covariates that may affect the outcomes of the

analyses. Potential biases are introduced by factors which include, for example, the selection of the patients included in the

analyses, misclassification in the matching process, the small sample size, and estimates that may not represent the

outcomes for the true treated patient population. Failure to achieve results supporting a positive benefit-risk profile in

current or potential future imetelstat clinical trials would interrupt, further delay, or halt, any development of imetelstat,

which would have a severe adverse effect on our results of operations, financial condition and ability to raise additional

capital, if needed, business and business prospects.

Further, preliminary data are based on a preliminary analysis of then-available data, and the results and

related findings and conclusions are subject to change following a more comprehensive review of the data related to the

particular study or trial. We also make assumptions, estimations, calculations and conclusions as part of our analyses of

data, and we may not have received or had the opportunity to fully and carefully evaluate all data. Additional or updated

safety and efficacy data from current or potential future clinical trials of imetelstat may result in a benefit-risk profile that

does not justify the continued development and/or potential regulatory approval of imetelstat in a particular patient

population, or at all. Any data reported from IMpactMF may materially differ from and be less positive than data

previously reported from IMbark. Thus, reported data should be considered carefully and with caution, and not relied upon

as indicative of future clinical results. Such additional data could result in a lower benefit-risk profile than initially

expected, which could halt the commercialization of RYTELO, hinder the potential success of IMpactMF, IMproveMF,

IMpress or IMAGINE, or cause us to abandon further development of imetelstat entirely.

Top-line or interim results and data may differ from future results of the same study, or different

conclusions or considerations may qualify such results, once additional data have been received and fully evaluated.

Moreover, as remaining patients in IMerge Phase 3 continue to be treated and followed under the extension phase of the

trial and longer-term outcomes are assessed, these additional and more mature data may alter the benefit-risk profile of

imetelstat in an adverse manner, including with respect to OS. Material adverse differences in future results, compared to

preliminary, interim or top-line data, could severely and adversely affect our financial results, business and business

prospects, including the commercialization of RYTELO, and might cause us to cease operations.

We rely on third parties to conduct our current and potential future clinical trials of imetelstat. If these third parties do

not successfully carry out their contractual duties or meet expected deadlines, we may be unable to continue the

development of imetelstat.

We do not have the ability to independently conduct clinical trials. Therefore, we rely on medical

institutions, clinical investigators, contract laboratories and other third parties, such as CROs, service providers, vendors,

suppliers and consultants, to conduct clinical trials of imetelstat. The third parties we contract with for execution of our

current and potential future clinical or investigator-sponsored trials of imetelstat play a critical role in the conduct of these

trials and the subsequent collection and analysis of data. However, these third parties are not our employees, and except for

contractual duties and obligations, we have limited ability to control their performance, or the amount or timing of

resources that they devote to imetelstat. For example, we have retained CROs to support our clinical development

activities, and any failure by our CROs to perform their contractual obligations, or disputes with our CROs about the

quality of their performance or other matters, could further delay or halt our clinical development activities. These third

parties may also have relationships with other commercial entities, some of which may compete with us. Under certain

circumstances, these third parties may terminate their agreements with us without cause and upon immediate written notice.

Although we rely on third parties to conduct our clinical trials, we remain responsible for ensuring that each

clinical trial is conducted in accordance with its investigational plan and protocol, and applicable laws. Moreover, the FDA

and similar international regulatory authorities require us to comply with GCP regulations and standards for conducting,

monitoring, recording and reporting the results of clinical trials to ensure that the data and results are scientifically credible

and accurate, and that the rights, integrity and confidentiality of patients participating in clinical trials are protected,

including being adequately informed of the potential risks. Regulatory authorities enforce these GCP requirements through

periodic inspections of trial sponsors, principal investigators and trial sites. If we or any of our CROs fail to comply with

applicable GCP requirements, the clinical data generated in our clinical trials may be deemed unreliable and the FDA, or

similar international regulatory authorities, may require us to perform additional clinical trials. We cannot assure you that

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upon inspection by a given regulatory authority, such regulatory authority will determine whether any of our clinical trials

comply with GCP or other applicable regulations. In addition, our clinical trials must be conducted with imetelstat

produced under applicable GMP regulations. Our failure to comply with these regulations may require us to repeat clinical

trials. Our ability to comply with these regulations and standards may be contingent upon activities conducted by third

parties, and if they fail to perform in accordance with contractual obligations and legal requirements, our development of

imetelstat may be interrupted, further delayed or halted. Any failures by us or third parties noted above would have a severe

adverse effect on our results of operations, financial condition and ability to raise additional capital, if needed, business and

business prospects, including the commercialization of RYTELO, any of which might cause us to cease operations.

Furthermore, the execution of clinical trials and the subsequent compilation and analysis of the data

produced, including the interim and final analyses for IMpactMF, requires coordination among various parties. In order for

these functions to be carried out effectively and efficiently, it is imperative that these parties communicate and coordinate

with one another. If the quality or accuracy of the clinical data obtained, compiled or analyzed by third parties is

compromised due to their failure to adhere to our clinical trial protocols, GCP or GMP requirements, or for any other

reason, we may need to enter into new arrangements with alternative third parties, which would cause delay, and could be

difficult, costly or impossible.

Switching or adding CROs, investigators, vendors and other third parties involves additional costs and

delays because of the time it takes to finalize a contract with a new CRO and for their commencement of work. Although

we carefully manage our relationships with our CROs, investigators, vendors and other third parties, we and any of these

third parties may nonetheless encounter challenges or delays in the future, which could have a material and adverse impact

on our business and business prospects.

In addition, certain principal investigators for our clinical trials serve as scientific advisors or consultants to

us from time to time and receive compensation in connection with such services. Under certain circumstances, we may be

required to report some of these relationships to the FDA or comparable foreign regulatory authorities. The FDA or

comparable foreign regulatory authorities may conclude that a financial relationship between us and a principal investigator

has created a conflict of interest or otherwise affected conduct of the trial. The FDA or comparable foreign regulatory

authorities may therefore question the integrity of the data generated at the applicable clinical trial site and the utility of the

clinical trial itself may be jeopardized. This could result in a delay in approval, or rejection, of any future applications for

regulatory approval of imetelstat, including in any additional indications by the FDA.

We do not control the conduct of current or any potential future investigator-led clinical trials, and data from such

trials could show marginal efficacy and/or clinically relevant safety concerns related to imetelstat resulting in an

unfavorable benefit-risk assessment that could materially and adversely impact our ongoing clinical trials, or our

development program as a whole.

We do not control the design or administration of investigator-led clinical trials, nor the submission,

approval or maintenance of any IND or international equivalent filings required to conduct these clinical trials. In addition,

we do not have control over the timing and reporting of the data from any such investigator-led clinical trials. A delay in

the timely completion of or reporting of data from any current or potential future investigator-led clinical trial could have a

material adverse effect on our ability to maintain regulatory approval for RYTELO in lower-risk MDS, or to further

develop or advance it in clinical trials, as such delays may impede our ability to generate necessary supplemental clinical

evidence, fulfill post-marketing commitments or requirements, or provide regulatory authorities with timely safety and

efficacy data needed to support ongoing approval or future development.

Investigator-led clinical trials may be conducted under less rigorous clinical standards than those used in

company-sponsored clinical trials. Accordingly, regulatory authorities may closely scrutinize the data collected from these

investigator-led clinical trials. In addition, any investigator-led clinical trials could show marginal efficacy and/or clinically

relevant safety concerns that could delay, limit or preclude the further clinical development or marketing approval of

RYTELO in any indication. To the extent that the results of any investigator-led clinical trials raise safety or other

concerns, regulatory authorities may withdraw or restrict approval for RYTELO, question the results of such investigator-

led clinical trials, or question the results of any of our clinical trials. Safety concerns arising from future investigator-led

clinical trials could result in withdrawal of approval of RYTELO, partial or full clinical holds being placed on our INDs by

the FDA or other similar international regulatory authorities, as occurred in March 2014, which would further delay or

prevent us from commercializing RYTELO or advancing it into further clinical development. Any of the foregoing would

delay or preclude any future marketing approvals for RYTELO and could cause us to discontinue our development, which

would severely harm our business and prospects and could potentially cause us to cease operations.

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RISKS RELATED TO MANUFACTURING RYTELO (IMETELSTAT)

Failure by us to maintain a manufacturing supply chain to appropriately and adequately supply RYTELO for

commercial and future clinical uses would adversely affect our ability to commercialize RYTELO and result in a further

delay in or cessation of clinical trials, and our business and business prospects could be severely harmed, and we could

cease operations.

The manufacture of RYTELO (imetelstat) must comply with applicable regulatory standards for

commercial uses and current and potential future clinical trials. The process of manufacturing RYTELO is complex and

subject to several risks, including:

• the ability to consistently manufacture and attain sufficient production yields with

acceptable quality control and quality assurance to meet market demand for our

commercialization of RYTELO, as well as the needs for continuing clinical trials;

• our ability to maintain existing commercial supply agreements and to establish additional or

alternative supply agreements if necessary, including our ability to successfully transfer

manufacturing technology and attain regulatory approval at any such additional or

alternative suppliers;

• reliance on third-party manufacturers and suppliers, whose efforts we do not control;

• supply chain issues, including the timely availability of product and management of shelf-

life, including raw materials, active pharmaceutical ingredient, or API, and drug product

and other supplies, and the cost of procuring the foregoing, any of which may be impacted

by a number of factors, including the effects of macroeconomic or other global conditions,

such as increased tariffs, renegotiation of existing international trade agreements, escalating

trade tensions and other trade restrictions;

• shortage of qualified personnel at any of our third party suppliers; and

• regulatory acceptance and compliance with regulatory requirements, which are less well-

defined for oligonucleotide products than for small molecule drugs and vary in each

country.

As a result of these and other risks, we may be unable to maintain a manufacturing infrastructure and

supply chain capable of providing RYTELO for clinical and commercial use, which would delay or adversely affect our

RYTELO commercialization efforts; result in lost sales; delay or result in a cessation of our current or potential future

clinical trials; delay or preclude potential future regulatory approvals of RYTELO in other jurisdictions or indications; and

could cause financial and reputational harm.

If third parties that manufacture RYTELO fail to perform as needed, the commercial and clinical supply of RYTELO

could be interrupted or limited, and we may be unable to successfully commercialize RYTELO or conduct or complete

current or potential future clinical trials.

Our RYTELO manufacturing supply chain relies, and will continue to rely, solely upon third-party

manufacturers to perform certain manufacturing, quality control, and other technical and scientific work with respect to

RYTELO, as well as to supply starting materials and manufacture the API and drug product for our commercialization of

RYTELO, as well as current and potential future clinical trials. While we have established arrangements with third parties

for the manufacture of RYTELO, our manufacturing supply chain is highly specialized, and as such we are reliant upon a

small group of third-party manufacturers to supply starting materials and drug product, and we rely on a single source to

supply the API for RYTELO. Failure by such third-party manufacturers to perform in a timely manner and in compliance

with all regulatory requirements, or at all, or the termination of one of our supply agreements before we have retained and

established an acceptable alternative supplier, could lead to delays or shortages in drug supply, perhaps substantially, that

are necessary for our clinical activities and commercialization of RYTELO. For example, one of our third-party

manufacturers received a warning letter from the FDA due to certain deficiencies in the manufacturer’s process and

facilities that were not in compliance with FDA requirements and regulations governing the manufacturing, processing,

packing and holding of drug product. While not related to the production of RYTELO, if the warning letter is not timely

remediated by the manufacturer, it could lead to delays or shortages in drug supply, or the inability to manufacture or ship

drug supply necessary for non-clinical and clinical activities, and commercialization.

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In addition, we plan to retain additional third-party manufacturers to provide redundancy in our supply

chain; however, we may be unable to do so on a timely basis on terms that are acceptable to us, or at all. We expect to rely

on third-party manufacturers to produce and deliver sufficient quantities of RYTELO and other materials to support our

commercialization of RYTELO and clinical trials on a timely basis and to comply with applicable regulatory requirements.

We do not have direct control over these third-party personnel or operations. Reliance on these third-party manufacturers is

subject to numerous risks, including:

• the inability to execute timely contracts or production orders with any additional third-party

manufacturers and suppliers that we may identify on acceptable terms, or at all;

• delays and disruptions experienced by third-party manufacturers that adversely impact the

ability of such parties to fulfill their contractual obligations to us, including providing the

quantities of RYTELO required to meet commercial and clinical needs;

• capacity limitations and scheduling constraints experienced by third-party manufacturers

due to scheduling, maintenance and other commitments, and queued manufacturing

activities in contracted facilities;

• requirements by regulatory authorities to validate and qualify significant activities for any

current or additional manufacturer, which could involve technology transfer, new testing,

compliance inspections, and would likely require FDA or comparable foreign regulatory

authority approval;

• the inability of third-party manufacturers to timely formulate and manufacture RYTELO or

to produce or ship RYTELO in the quantities or of the quality required to meet commercial

and clinical needs;

• the possible mislabeling by third-party manufacturers of finished drug product for both

commercial and clinical use, potentially resulting in product recall and harm to our

business;

• decisions by third-party manufacturers to exit the contract manufacturing business during

the time required to supply clinical trials or to successfully produce, store and distribute

RYTELO to meet our commercial needs or before we establish an acceptable alternative

supplier;

• compliance by third-party manufacturers with GMP standards mandated by the FDA and

state agencies and other government regulations, including foreign governing regulations,

corresponding to similar international regulatory authorities, including any deficiencies

identified during regulatory inspections, such as those identified in the warning letter issued

to one of our third-party manufacturers;

• breach or termination of manufacturing or supply contracts;

• inadequate storage or maintenance at contracted facilities resulting in theft or spoilage; and

• natural disasters that affect contracted facilities, including manufacturing, warehousing, and

distribution facilities.

Each of these risks could lead to delays or shortages in drug supply, or the inability to manufacture or ship

drug supply necessary for commercialization, and non-clinical and clinical activities, which could severely and adversely

affect our financial results, business and business prospects.

In addition, third-party manufacturers and/or any other manufacturers may need to make substantial

investments to enable sufficient capacity increases and cost reductions, and to implement those regulatory and compliance

standards necessary for successful commercialization of RYTELO. These third-party manufacturers may not be willing or

able to achieve such capacity increases, cost reductions, or regulatory and compliance standards, and even if they do, such

achievements may not be at commercially reasonable costs. Changing or adding alternative manufacturers may be

prolonged, difficult and expensive, due to inherent technical complexities, regulatory risks, and because the number of

potential manufacturers for oligonucleotide products is limited. It may be difficult or impossible for us to find a

replacement or alternative manufacturer on acceptable terms, or at all.

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RISKS RELATED TO OUR OPERATING RESULTS AND FINANCIAL POSITION

We have a history of net losses and may not achieve consistent future profitability for some time, if ever.

We are incurring and have incurred net losses every year since our operations began in 1990, except for

one. As of December 31, 2025, our accumulated deficit was approximately $1.9 billion. Losses have resulted principally

from costs incurred in connection with our research and development activities and from general and administrative costs

associated with our operations. Although we began commercializing RYTELO in June 2024, the commercial potential of

and our ability to successfully commercialize RYTELO remains unproven, and our limited operating history as a

commercial company makes our future operating results difficult to predict. We expect that our sales revenue may continue

to vary from period to period as a result of the evolving effects of our commercialization strategy and as our

commercialization efforts otherwise progress. If we do not generate sufficient revenue from commercial sales of RYTELO,

or if we experience unforeseen events or choose to make other investments in our business, we may continue to experience

negative cash flow as we fund our operations and imetelstat clinical development activities and research programs, and

continue with the commercialization of RYTELO, including as a result of our obligation to pay royalty payments under the

Royalty Pharma Agreement and service our debt obligations. We will need to generate significant revenues to achieve

consistent future profitability, and we may never achieve consistent future profitability. Even if we do become profitable in

the future, we may not be able to sustain or increase profitability on a quarterly or annual basis. Our failure to achieve

consistent future profitability could negatively impact the market price of our common stock and our ability to sustain

operations.

Our operating results are unpredictable and may fluctuate. If our operating results are below the expectations of

securities analysts or investors, the trading price of our common stock could decline.

Our operating results are difficult to predict and will likely fluctuate from quarter to quarter and year to

year. Due to the limited historical sales data of RYTELO in lower-risk MDS since its approval by the FDA in June 2024,

RYTELO sales will be difficult to predict from period to period and as a result, you should not rely on RYTELO sales

results in any period as being indicative of future performance. Sales of RYTELO have in the past been below the

expectations of securities analysts and investors, and sales of RYTELO have been and may in the future be below prior

sequential or prior period sales, our own guidance and/or the expectations of securities analysts and investors. To the extent

that we do not meet our guidance, our financial projections or estimates, or the expectations of analysts or investors, our

stock price may be adversely impacted, perhaps significantly. For example, following releases of earnings for both of the

quarters and years ended December 31, 2025 and 2024, our stock price declined significantly both times. As a result of this

volatility, our stockholders may not be able to sell their common stock at or above the price they paid for their common

stock. We believe that our quarterly and annual results of operations may be affected by a variety of factors, including:

• the overall level of demand for RYTELO in its approved indication, including across the breadth of the eligible

patient segments;

• the extent to which coverage and reimbursement for RYTELO is available from government and health

administration authorities, private health insurers, managed care programs and other third-party payors;

• changes in the amount of deductions from gross sales, including government-mandated rebates, chargebacks and

discounts that can vary because of changes to the government discount percentage, including increases in the

government discount percentage resulting from price increases we may take in the future, or due to different levels

of utilization by entities entitled to government rebates and discounts and changes in patient demographics;

• increases in the scope of eligibility for customers to purchase RYTELO at the discounted government price or to

obtain government-mandated rebates on purchases of RYTELO;

• changes in our cost of sales;

• the timing and level of royalty payments under the Royalty Pharma Agreement;

• the timing, cost and level of investment in our sales and marketing efforts to support RYTELO sales;

• the timing, cost and level of investment in our research and development activities involving imetelstat and

potential future product candidates; and

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• expenditures we may incur to develop and/or commercialize any additional products, product candidates, or

technologies that we may develop, in-license, or acquire.

Further, changes in our operations, such as increased development, manufacturing and clinical trial

expenses, or our undertaking of additional programs, business activities, or entry into strategic transactions, including

potential future acquisitions of products, technologies or businesses may also cause significant fluctuations in our expenses.

In addition, we measure compensation cost for stock-based awards made to employees at the grant date of the award, based

on the fair value of the award, and recognize the cost as an expense over the employee’s requisite service period. As the

variables that we use as a basis for valuing these awards change over time, including our underlying stock price, the

magnitude of the expense that we must recognize may vary significantly.

For these and other reasons, it is difficult for us to accurately forecast future sales of RYTELO, operating

expenses or future profits or losses. As a result, our operating results in future periods could be below our guidance or the

expectations of securities analysts or investors, which could cause the trading price of our common stock to further decline,

perhaps significantly.

Our financial projections and estimates are subject to significant risks, assumptions, and uncertainties, and our actual

results may differ materially.

Our financial projections and estimates are subject to significant risks, assumptions, and uncertainties, and

our actual results may differ materially. These projections and estimates include estimates of the total addressable market

for RYTELO, assumptions regarding patient market share and duration of therapy for patients receiving RYTELO, as well

as assumptions regarding our ability to meet demand and assumptions regarding our future costs of goods. These

projections and estimates are subject to various factors beyond our control, including, for example, the level of demand for

RYTELO, the extent to which coverage and reimbursement for RYTELO is available from government and health

administration authorities, private health insurers, managed care programs and other third-party payors, increased costs in

the supply chain, including as a result of increased tariffs, renegotiation of existing international trade agreements,

escalating trade tensions and other trade restrictions, increased labor costs, changes in the regulatory environment, the

impact of global health crises or macroeconomic or other global conditions, and changes in our senior management team.

Our financial projections and estimates constitute forward-looking statements, are for illustrative purposes only and should

not be relied upon as necessarily being indicative of future results. The assumptions and estimates underlying such

financial projections and estimates are inherently uncertain and are subject to a wide variety of significant business,

economic, competitive and other risks and uncertainties. Actual results may differ materially from the results contemplated

by the financial projections. Our independent auditors have not studied, reviewed, compiled or performed any procedures

with respect to the projections, and accordingly, they did not express an opinion or provide any other form of assurance

with respect thereto. While all financial projections, estimates and targets are necessarily speculative, we believe that the

preparation of financial projections involves increasingly higher levels of uncertainty the further out the projection,

estimate or target extends from the date of preparation. Accordingly, there can be no assurance that the prospective results

are indicative of our future performance or that actual results will not differ materially from those presented in the financial

projections or estimates .

Our failure to obtain additional capital, if and when needed, would force us to further delay, reduce or eliminate the

further development of RYTELO, or to halt the commercialization of RYTELO, any of which would severely and

adversely affect our financial results, business and business prospects, and might cause us to cease operations.

Successful drug development and commercialization requires significant amounts of capital. As of

December 31, 2025, we had approximately $401.1 million in cash, cash equivalents, restricted cash and marketable

securities. While we believe that, based on our current operating plans and assumptions, our existing cash, cash

equivalents, and marketable securities, together with anticipated net revenues from sales of RYTELO, will be sufficient to

fund our projected operating requirements for the foreseeable future, if we do not generate net revenues from commercial

sales of RYTELO at the levels we anticipate, if we experience unforeseen events or choose to make other investments in

our business, or our assumptions regarding our projected operating expenses are otherwise incorrect, we may require

additional funding, which could include a combination of public or private equity offerings, debt financings (including

additional tranches, if available, under the Pharmakon Loan Agreement (as defined below) , collaborations, strategic

alliances, licensing arrangements or marketing and distribution arrangements, which may not be possible. For example,

changes in our operations, such as increased development, manufacturing and clinical trial expenses, or our undertaking of

additional programs, business activities, or entry into strategic transactions, including potential future acquisitions of

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products, technologies or businesses, may cause our operating expenses to increase, perhaps significantly, which could

require us to raise additional funding. If adequate funds are not available to us when we need them, our RYTELO

commercialization efforts may be adversely affected and we may be unable to pursue further development of imetelstat,

which would severely harm our business and we might cease operations.

Despite approval of RYTELO in the U.S. in June 2024 and in the EU in March 2025, the outcome of any

clinical activities and/or regulatory approval process is highly uncertain, and we cannot reasonably estimate whether our

future development activities may succeed, whether we will obtain regulatory approval for RYTELO in any other

jurisdictions or indications we are pursuing or may in the future pursue, or whether we will be able to effectively

commercialize RYTELO for lower-risk MDS in the U.S., the EU or other potential jurisdictions or indications, if at all. We

may never recoup our investment in any RYTELO development, which would adversely affect our financial condition and

our business and business prospects, and might cause us to cease operations. In addition, our plans and timing expectations

could be further delayed or interrupted by the effects of macroeconomic or other global conditions, including those

resulting from further changes in tariffs and other trade restrictions, renegotiation of existing international trade agreements

or further escalation of trade tensions, inflation, fluctuations in interest rates, prospects of a recession, government

shutdowns, bank failures and other disruptions to financial systems, civil or political unrest, military conflicts, pandemics

or other health crises and supply chain and resource issues. Further, our future capital requirements are difficult to forecast

and will depend on many factors, including:

• the accuracy of the assumptions underlying our estimates for our capital needs;

• the overall level of sales and market acceptance of RYTELO;

• the scope, progress, timing, magnitude and costs of non-clinical and clinical development,

manufacturing and commercialization of RYTELO, including commercialization in the

U.S. and any commercialization in the EU for lower-risk MDS, or in any other jurisdictions

or other indication we may pursue, subject to clearances and approvals by the FDA and

similar international regulatory authorities;

• delays or disruptions in opening sites, screening and enrolling patients or treating and

following patients, in our current or any potential future clinical trials of RYTELO;

• the costs, timing and outcomes of regulatory reviews or other regulatory actions related to

RYTELO;

• the costs of preparing, filing and prosecuting patent applications and maintaining, enforcing

and defending intellectual property-related claims;

• the costs of manufacturing, developing, commercializing and marketing RYTELO,

including with respect to third-party vendors and service providers and our ability to

achieve any meaningful reduction in manufacturing costs;

• the sales price for RYTELO, including the availability of coverage and adequate third-party

reimbursement for RYTELO;

• the extent to which we acquire or in-license other drugs and technologies, or invest in

businesses, products or technologies, although we currently have no commitments or

agreements relating to any of these types of transactions, or to which we out-license

RYTELO;

• the extent to which we are able to enter into and conduct successful commercialization

arrangements with third parties, including for the commercialization and marketing of

RYTELO in the EU and in any other regions outside of the U.S., if approved for

commercialization in such other regions;

• expenses associated with the pending putative securities class action and shareholder

derivative lawsuits and potential additional related lawsuits, as well as any other litigation;

• the extent and scope of our selling, general and administrative expenses, including expenses

associated with pending and potential future litigation;

• our level of indebtedness and associated debt service obligations;

• the costs of maintaining and operating facilities in California and New Jersey, as well as

higher expenses for travel;

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• macroeconomic or other global conditions that may reduce our ability to access equity or

debt capital or other financing on preferable terms, which may adversely affect future

capital requirements and forecasts; and

• the costs of enabling our personnel to work remotely, including providing supplies,

equipment and technology necessary for them to perform their responsibilities.

In the event we need to raise additional capital to fund our business, including pursuant to the 2026 Sales

Agreement with TD Cowen (as defined below), the Tranche B Loan and the Tranche C Loan under the Pharmakon Loan

Agreement , which are subject to certain funding conditions; capital lease transactions or other financing sources, such

additional capital may not be available on acceptable terms, or at all. We may be unable to raise equity capital, or may be

forced to do so at a stock price or on other terms that could result in substantial dilution of ownership for our stockholders.

The receptivity of the public and private debt and equity markets to proposed financings has been substantially affected by

uncertainty in the general economic, market and political climate due to the effects of macroeconomic or other global

conditions, such as inflation, fluctuations in interest rates, prospects of a recession, government shutdowns, further changes

in tariffs and other trade restrictions, bank failures and other disruptions to financial systems, civil or political unrest,

military conflicts, pandemics or other health crises and supply chain and resource issues, and may in the future be affected

by other factors which are unpredictable and over which we have no control. These effects have increased market volatility

and could result in a significant long-term disruption of global financial markets, which could reduce or eliminate our

ability to raise additional funds through financings, and could negatively impact the terms upon which we may raise those

funds. Similarly, these macroeconomic conditions have created extreme volatility and disruption in the capital markets and

are expected to have further global economic consequences. If the equity and credit markets deteriorate, it may make any

necessary debt or equity financing more difficult to obtain in a timely manner or on favorable terms, more costly or more

dilutive. If we are unable to effectively commercialize RYTELO, or raise additional capital, if needed, or establish

alternative collaborative arrangements with third-party collaborative partners for RYTELO when needed, the development

and commercialization of RYTELO may be further delayed, altered or abandoned, which might cause us to cease

operations.

In addition, we may seek additional capital due to market conditions or strategic considerations even if we

believe we have sufficient funds for our current or future operating plans. Due to uncertainty in the general economic,

market and political climate, we may determine that it is necessary or appropriate to raise additional funds proactively to

meet longer-term anticipated operating plans. To the extent that we raise additional capital through the sale of equity or

convertible debt securities, including pursuant to the 2026 Sales Agreement, our stockholders may be diluted, and the terms

may include liquidation or other preferences that could materially and adversely affect the rights of our existing

stockholders. In addition, we have borrowed, and in the future may borrow, additional capital from institutional and

commercial banking sources to fund development and our future growth, including pursuant to our Pharmakon Loan

Agreement or potentially pursuant to new arrangements with different lenders. We may borrow funds on terms under

agreements, such as our Pharmakon Loan Agreement, that include restrictive covenants, including covenants limiting or

restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring

dividends. Moreover, if we raise additional funds through alliance, collaborative or licensing arrangements with third

parties, we may have to relinquish valuable rights to RYTELO or our technologies or grant licenses on terms that are not

favorable to us.

RISKS RELATED TO OUR INDEBTEDNESS AND ROYALTY PAYMENT OBLIGATIONS

Our level of indebtedness and debt service obligations could adversely affect our financial condition and may make it

more difficult for us to fund our operations, including by limiting our operating and financial flexibility.

On November 1, 2024, we entered into the Pharmakon Loan Agreement , which we amended on January 5,

2026 . We drew the Tranche A Loan of $125.0 million on November 1, 2024 and as of November 1, 2024, the total

outstanding principal amount under the Pharmakon Loan Agreement was $125.0 million. The tranches for the remaining

$125.0 million available to us under the Pharmakon Loan Agreement are as follows: (a) a Tranche B Loan of $75.0

million, which is available for us to request at our option until July 30, 2026, subject to certain customary and limited

conditions; and (b) a Tranche C Loan of $50.0 million, which is available for us to request until July 30, 2026, subject to

certain conditions, including us reaching a specified trailing twelve-month RYTELO revenue milestone on or prior to June

30, 2026 . If we do not achieve such revenue milestone within the required timeline, we will not be eligible to draw down

the Tranche C Loan. In addition, before we would consider drawing down any of the remaining tranches under the

Pharmakon Loan Agreement, if available, we must first satisfy ourselves that we will have access to future alternate

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sources of capital, such as from commercial revenues or the equity capital markets or debt capital markets, in order to repay

any additional principal borrowed, which we may be unable to do, in which case, our liquidity and ability to fund our

operations may be substantially impaired.

All obligations under the Pharmakon Loan Agreement are secured by substantially all of our assets,

including our intellectual property. Further, the terms of the Pharmakon Loan Agreement place restrictions on our

operating and financial flexibility, and limit or prohibit our ability to dispose of certain assets, change our line of business,

and engage in other significant transactions. Such restrictions will affect, and in many respects limit or prohibit, our ability

and the ability of our subsidiaries to, among other things:

• dispose of certain assets;

• change our line of business;

• engage in mergers, acquisitions or consolidations;

• incur additional indebtedness;

• create liens on assets;

• pay dividends and make contributions or repurchase our capital stock; and

• engage in certain transactions with affiliates.

This indebtedness may create additional financing risk for us, particularly if our business or prevailing

financial market conditions are not conducive to paying off or refinancing the outstanding debt obligations at maturity. If

we draw down any of the remaining tranches under the Pharmakon Loan Agreement, our indebtedness will increase, which

would further increase our risk of being unable to pay off or refinance our outstanding debt obligations at maturity.

Our indebtedness could also have important negative consequences, including:

• we will need to repay the indebtedness by making payments of interest and principal, which

will reduce the amount of cash available to finance our operations, our research and

development efforts and other general corporate activities; and

• our failure to comply with the obligations of our affirmative and restrictive covenants in the

Pharmakon Loan Agreement could result in an event of default that, if not cured or waived,

would permit the Lenders to accelerate our obligation to repay this indebtedness, and the

Lenders could seek to enforce their security interest in the assets securing such

indebtedness.

In addition, we may borrow additional capital in the future to fund clinical development and our future

growth, including pursuant to the Pharmakon Loan Agreement or potentially pursuant to new arrangements with different

lenders. To the extent additional debt is added to our current debt levels, the risks described above could increase.

We may not have cash available in an amount sufficient to enable us to make interest or principal payments on our

indebtedness when due.

Our ability to make scheduled interest payments on or to refinance our indebtedness depends on our future

performance and ability to raise additional sources of cash, which is subject to economic, financial, competitive and other

factors beyond our control. If we are unable to generate sufficient cash to service our debt, we may be required to adopt one

or more alternatives, such as selling assets, restructuring our debt or obtaining additional equity capital on terms that may

be onerous or highly dilutive. If we desire to refinance our indebtedness, our ability to do so will depend on the state of the

capital and lending markets and our financial condition at such time. We may not be able to engage in any of these

activities or engage in these activities on desirable terms, which could result in a default on our debt obligations.

Failure to satisfy our current and future debt obligations under the Pharmakon Loan Agreement or to

comply with certain covenants in the Pharmakon Loan Agreement could result in an event of default, the occurrence and

continuance of which provides the lenders with the right to demand immediate repayment of all outstanding obligations

under the Pharmakon Loan Agreement (and in the case of certain insolvency, liquidation, bankruptcy or similar events,

automatically requires immediate repayment of all outstanding obligations under the Pharmakon Loan Agreement), and to

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exercise remedies against us and the collateral securing the Pharmakon Loan Agreement. These events of default include,

among other things:

• insolvency, liquidation, bankruptcy or similar events;

• failure to observe covenants under the Pharmakon Loan Agreement and ancillary collateral

documents, which failure, in certain limited cases, is not cured within 10 or 20 days;

• the occurrence of a withdrawal event in respect to RYTELO;

• the occurrence of a material adverse change;

• material misrepresentations;

• certain cross-default of third-party indebtedness or certain default or termination events of

hedging assessments;

• certain money judgments being entered against us which are not timely paid, discharged or

stayed; and

• our assets are attached or seized.

In the event of default, the lenders could accelerate all of the amounts due under the Pharmakon Loan

Agreement. Under such circumstances, we may not have enough available cash or be able to raise additional funds through

equity or debt financings to repay such indebtedness at the time of such acceleration. We may be required to delay, limit,

reduce or terminate our RYTELO development or commercialization efforts or grant to others rights to develop and market

RYTELO. The lenders could also exercise their rights to take possession and dispose of the collateral securing the

Pharmakon Loan Agreement, which collateral includes substantially all of our property including, without limitation, our

intellectual property, subject to certain exceptions. Our business, financial condition and results of operations could be

materially adversely affected as a result of any of these events.

The Royalty Pharma Agreement places certain restrictions on our operational flexibility.

The Royalty Pharma Agreement contains covenants that impose on us certain obligations with respect to

royalty payments, diligence, reporting, indemnification and includes restrictions on intellectual property transfers and out-

licenses, and certain other actions. For example, we are obligated to make royalty payments each quarter based on U.S. net

sales of RYTELO at the royalty rates set forth in the Royalty Pharma Agreement, until the date when the aggregate Royalty

Payments equal or exceed 1.65 times the Purchase Price, if this occurs by June 30, 2031. However, in the event we are

unable to repay our obligation to Royalty Pharma before June 30, 2031, we will be required to make royalty payments

equal to or exceeding 2.0 times the Purchase Price thereafter, which may negatively impact our business, financial

condition and results of operations. The Royalty Pharma Agreement also limits our ability to create or incur liens or

dispose of certain assets related to imetelstat. We have no rights to repurchase the revenue interests in RYTELO sold to

Royalty Pharma (other than in connection with a change of control event), thereby limiting our ability to eliminate future

applicability of the covenants contained in the Royalty Pharma Agreement. Compliance with these covenants may limit our

flexibility in operating our business and our ability to take actions that might otherwise be advantageous to us and our

stockholders.

RISKS RELATED TO PROTECTING OUR INTELLECTUAL PROPERTY

If we are unable to obtain and maintain sufficient intellectual property protection and relevant regulatory exclusivities

for RYTELO, both in the U.S. and in other countries, our competitors could develop and commercialize products similar

or identical to RYTELO, and our ability to successfully commercialize RYTELO may be adversely affected.

Protection of our proprietary technology is critically important to our business. Our success and the success

of our commercialization and planned future development of RYTELO will depend on our ability to protect our

technologies and RYTELO through patents, regulatory exclusivity, and other intellectual property rights. Our success will

depend in part on our ability to obtain, maintain, enforce, and extend our patents and maintain trade secrets, both in the

U.S. and in other countries.

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As a result of the Leahy-Smith America Invents Act, or the AIA, in March 2013, the U.S. transitioned to a

first-inventor-to-file system under which, assuming the other requirements for patentability are met, the first inventor to file

a patent application is entitled to the patent. However, since the publication of discoveries in scientific or patent literature

tends to lag behind actual discoveries by at least several months and sometimes several years, we are not able to be certain

upon filing a patent application that the persons or entities that we name as inventors or applicants in our patent

applications were the first to invent the inventions disclosed therein, or the first to file patent applications for these

inventions. Thus, our ability to protect our patentable intellectual property depends, in part, on our ability to be the first to

file patent applications with respect to our inventions, or inventions that were developed by our former collaboration

partner and assigned to us, for the future development, commercialization and manufacture of RYTELO. As a result, if we

are not the first inventor-to-file, we may not be able to obtain patents for discoveries that we otherwise would consider

patentable and that we consider to be significant to the future success of RYTELO. Delay in the filing of a patent

application for any purpose, including further development or refinement of an invention, may result in the risk of loss of

patent rights.

The issuance of a patent is not conclusive as to its inventorship, scope, validity or enforceability, and our

patents may be challenged in the courts or patent offices in the U.S. and in other countries. Such challenges may result in

loss of exclusivity or in patent claims being narrowed, invalidated, or held unenforceable, which could limit our ability to

stop others from using or commercializing RYTELO or our technology and/or limit the duration of the patent protection

for RYTELO and our technology.

While we have method-of-use patents that protect the use of RYTELO for the treatment of certain diseases,

this type of patent does not prevent a generic competitor from making and marketing a product that is identical to RYTELO

for an indication that is outside the scope of our approved use after our composition-of-matter patents or their patent term

extensions, and any regulatory exclusivities have expired. Moreover, even if competitors do not actively promote their

product for our approved indications, physicians may prescribe or use these generic products “off-label,” which would

result in decreased sales for us.

In addition to our patents covering RYTELO, we also expect to rely on regulatory exclusivity, including

orphan drug exclusivity of up to seven years in the U.S. and ten years in the EU following approval, to protect our rights to

commercialize RYTELO for its approved uses, but such regulatory exclusivity may be limited or withdrawn. See “ Risks

Related to Regulatory Approval of RYTELO -- Although orphan drug designation has been granted to RYTELO for the

treatment of MDS and MF in the U.S. and in the EU, these designations may not be maintained, which would eliminate the

benefits associated with orphan drug designation, including market exclusivity, which could limit the period of exclusivity

we are able to maintain for the commercialization of RYTELO, and would likely harm our business and business

prospects. ”

In addition to orphan drug exclusivity, we expect to rely on other forms of regulatory exclusivity to protect

our ability to commercialize RYTELO. In the U.S., New Chemical Entity, or NCE, exclusivity would entitle us to four

years of data exclusivity and one year of market exclusivity, for a total of five years of NCE exclusivity from the date of

approval of the first-approved indication. Our request for NCE exclusivity is still pending with FDA, and might not be

awarded or could be awarded and then later withdrawn. In the EU, imetelstat is designated as a New Active Substance, or

NAS. For this NAS, regulatory data protection entitles us to eight years of data exclusivity and two years of market

exclusivity, for a total of ten years in parallel to orphan market exclusivity.

In the event that we are unsuccessful in obtaining, maintaining, enforcing and extending our patents and

other intellectual property rights or having our licensors maintain the intellectual property rights we have licensed, the

value of RYTELO and/or our technologies will be adversely affected, and we may not be able to further develop or

commercialize RYTELO. Furthermore, such loss of intellectual property rights could impair our ability to exclude others

from commercializing products similar or identical to RYTELO and therefore result in decreased sales for us. Occurrence

of any of these events would materially and adversely affect our financial results, business and business prospects, and

might cause us to cease operations.

Obtaining and maintaining our patent rights depends on compliance with various procedural, document submission, fee

payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced

or eliminated for noncompliance with these requirements.

The U.S. Patent and Trademark Office, or USPTO, and various governmental patent agencies in other

countries require compliance with a number of procedural, documentary, fee payment, periodic maintenance fees, renewal

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fees, annuity fees and various other government fees on patents and/or patent applications. Failure to respond to official

actions within prescribed time limits, and nonpayment of fees, for example, maintenance fees, renewal fees, and annuity

fees, could result in abandonment or lapse of a patent or patent application, resulting in partial or complete loss of patent

rights in the jurisdiction. In such an event, potential competitors might be able to enter the market with the same or similar

products to RYTELO, and this circumstance could harm our financial condition, business and business prospects. In

addition, if we are responsible for patent prosecution and maintenance of patent rights in-licensed to us or jointly owned

with us, any of the foregoing could expose us to liability to the applicable patent owner or patent co-owner.

Patent terms may be inadequate to protect our competitive position on RYTELO for an adequate amount of time.

Patents have a limited lifespan. In the U.S., the natural expiration of a patent is generally 20 years after its

first effective nonprovisional filing date. As a result, our intellectual property may not provide us with sufficient patent

rights to exclude others from commercializing products similar or identical to RYTELO.

In the U.S., the Hatch-Waxman Act permits one patent per approved product to receive one patent term

extension of up to five years beyond its normal expiration. The USPTO, in consultation with the FDA, reviews and

approves the application for any patent term extension or restoration. The length of the patent term extension is typically

calculated as one half of period between the effective date of an IND and the submission date of an NDA less any time the

sponsor did not act with due diligence during the period, plus the time between the submission date of an NDA and the

approval of that application less any time the sponsor did not act with due diligence during the period . There is also a limit

on the patent term extension to a term that is no greater than fourteen years from drug approval. We have applied to the

USPTO for patent term extension of some of our patents. Currently, communication of patent term extension approval and

the length of the granted extension period by the USPTO may occur up to several years from filing of an application for

patent term extension. If the USPTO and the FDA determine the extension period for each proposed eligible patent, we will

select the one patent to be extended. We expect to apply any patent term extension that is granted in the U.S. to our method

of treatment patent for MDS and MF that expires on March 15, 2033. If such patent term extension is granted, we expect

the term of the patent to extend through August 2037, although such timing is subject to approval by the USPTO and could

differ from our calculation.

Similar extensions are also available in certain countries and territories outside the U.S., such as in Japan,

and in Europe as Supplementary Protection Certificates, or SPCs. However, we might not be granted a patent term

extension at all because of failure to satisfy any of the numerous applicable requirements, including:

• failure to adhere to the required filing deadlines;

• submission of a patent term extension application after the underlying patents have expired;

• failure to exercise due diligence during clinical development or regulatory review; or

• failure to otherwise meet the applicable criteria.

Moreover, the applicable authorities, including the FDA and the USPTO in the U.S., and any equivalent

regulatory authorities and patent offices in other countries, may not agree with our assessment of whether such extensions

are available, may refuse to grant extensions to our patents, or may grant more limited extensions than we request and

could be less than five years. If we select and are granted a patent term extension on a recently filed and issued patent, we

may not receive the full benefit of a possible patent term extension, if at all. If we do not have sufficient patent life and

regulatory exclusivity to protect RYTELO, our financial results, business and business prospects would be materially and

adversely affected, which might cause us to cease operations.

In Europe and other jurisdictions, our composition of matter patent coverage expired in September 2024,

and our method of treatment patent rights for MDS and MF expire in November 2033. Our method of treatment patents

may be eligible for patent term extension of up to five years under an SPC, permitted under European Council (EC)

Regulation No. 469/2009, or the European SPC Regulation, upon receipt of marketing authorization, such as, for example,

our method of treatment patent for MDS. In Europe, we have separate method of treatment patents covering MDS and MF,

and an SPC may only be applied for once with respect to a product. Accordingly, in countries of the European Economic

Area, or EEA, we must rely on regulatory exclusivity and our method of treatment patents.

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If regulatory approval of RYTELO occurs after a patent has expired in a country that does not allow interim

patent term extensions, as is the case in many jurisdictions and territories including in Europe, we will be unable to obtain

any patent term extension of that expired patent, and the duration of our patent rights may be limited. Accordingly, in

Europe and such other similar jurisdictions and territories, we will not be able to seek patent term extension of our

composition of matter patent, as it expired in September 2024. If we do not have sufficient patent life and regulatory

exclusivity to protect RYTELO, our financial results, business and business prospects would be materially and adversely

affected, which might cause us to cease operations.

Additionally, there are regulations for the listing of patents in the Approved Drug Products with

Therapeutic Equivalence Evaluations, or the Orange Book. Some of our patents have been listed in the Orange Book.

Manufacturers of generic drugs may challenge the listing. If an appropriate patent covering RYTELO is not listed in the

Orange Book or is subsequently removed from the Orange Book, a manufacturer of generic drugs would not be required to

provide advance notice to us of any abbreviated NDA filed with the FDA to obtain permission to sell a generic version of

RYTELO. Any of the foregoing could harm our competitive position, business, financial condition, results of operations

and prospects.

The validity, scope and enforceability of any patents listed in the Orange Book that cover RYTELO or its methods of use

can be challenged by third parties and may not protect us from generic or innovator competition.

If a third party files an application under Section 505(b)(2) of the FDCA or an abbreviated new drug

application, or ANDA, under Section 505(j) to obtain permission to sell a generic or follow-on version of RYTELO, and

relies in whole or in part on studies conducted by or for us, the third-party will be required to certify to the FDA that either:

(1) there is no patent information listed in the Orange Book with respect to our NDA for RYTELO; (2) the patents listed in

the Orange Book have expired; (3) the listed patents have not expired, but will expire on a particular date and approval is

sought after patent expiration; or (4) the listed patents are invalid or will not be infringed by the manufacture, use or sale of

the third-party’s generic product. A certification that the new product will not infringe the Orange Book-listed patents for

RYTELO, or that such patents are invalid, is called a paragraph IV certification. If the third-party submits a paragraph IV

certification to the FDA, a notice of the paragraph IV certification must also be sent to us within 20 days after the third-

party’s ANDA is accepted for filing by the FDA. We may then initiate a lawsuit to defend the patents identified in the

notice. The filing of a patent infringement lawsuit within 45 days of receipt of the notice automatically prevents the FDA

from approving the third-party’s ANDA until the earliest of 30 months after the NDA holder’s receipt of the notice of

Paragraph IV Certification, the expiration of the patent, certain settlements of the lawsuit, or the court reaches a decision in

the infringement lawsuit in favor of the third-party. If the product has NCE exclusivity and the notice is given and the suit

filed in the fifth year of exclusivity, the regulatory stay extends until 7.5 years after approval of the reference product. If

we do not file a patent infringement lawsuit within the required 45-day period, the third-party’s ANDA will not be subject

to the 30-month stay of FDA approval. However, if an appropriate patent covering RYTELO is not listed in the Orange

Book or is subsequently removed from the Orange Book, a manufacturer of generic drugs would not be required to provide

advance notice to us of any abbreviated NDA filed with the FDA to obtain permission to sell a generic version of

RYTELO. Any of the foregoing could harm our competitive position, business, financial condition, results of operations

and prospects.

Our issued U.S. patents covering RYTELO or its methods of use may not provide adequate protection from

competitive products if competitors receive approval of an ANDA application or are able to design around the patents. One

or more competitors may circumvent these patents by filing a marketing application with the FDA for a competitive

product containing the active moiety in RYTELO and successfully challenging the validity of the patents or successfully

designing around the patents. Any successful challenge and/or designing around one or more of the patents could result in a

generic version of RYTELO being commercialized before the expiration of the patents.

If the patents covering RYTELO or its methods of use are successfully challenged or designed around, or if

we are unsuccessful in enforcing our patents against generics, we could face competition prior to the expiration of these

patents, which could have a material adverse effect on our business, financial condition, results of operations and prospects.

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Changes in U.S. or international patent law or interpretations of such patent laws could diminish the value of our

patents in general, thereby impairing our ability to protect our technologies and RYTELO.

The patent positions of pharmaceutical and biopharmaceutical companies, including ours, are highly

uncertain and involve complex legal and technical questions. In particular, legal principles for biotechnology and

pharmaceutical patents in the U.S. and in other countries are evolving, and the extent to which we will be able to obtain

patent coverage to protect our technologies and RYTELO, or enforce or defend issued patents, is uncertain.

For instance, the U.S. has enacted and implemented wide-ranging patent reform legislation, including AIA,

signed into law on September 16, 2011. The U.S. Supreme Court has ruled on several patent cases in recent years, either

narrowing the scope of patent protection available in certain circumstances or weakening the rights of patent owners in

certain situations. Depending on actions by Congress, the federal courts, and the USPTO, the laws and regulations

governing patents could change in unpredictable ways that would weaken our ability to obtain new patents or to enforce

our existing patents or patents that we might obtain in the future. Similarly, changes in patent law, regulations in other

jurisdictions or the governmental bodies that enforce them or changes in how the relevant governmental authority enforces

patent laws or regulations may weaken our ability to obtain new patents or to enforce our existing patents or patents that we

may obtain in the future. Occurrence of these events and/or significant impairment of our RYTELO patent rights could

severely and adversely affect our financial results, business and business prospects, which might cause us to cease

operations.

In 2012, the European Patent Package, or EU Patent Package, was approved and included regulations with

the goal of providing for the possibility of a single Unitary Patent covering the Contracting Member States, and a new

Unified Patent Court, or UPC, for litigation of Unitary Patents and as well as European patents not opted out from the UPC

system. The EU Patent Package entered into force fully in June 2023 and currently covers 18 EU Member States. As of

June 1, 2023, all European patents, including those issued prior to June 1, 2023 in principle fall under the jurisdiction of the

UPC and allow for the possibility of obtaining injunctions for the UPC Member States and are at risk of central revocation

at the UPC in participating UPC Member States. Under the EU Patent Package, patent holders are permitted to “opt out”

their European patents (but not their Unitary Patents) from the UPC on a patent-by-patent basis during an initial seven year

transitional period after June 1, 2023. Owners of European patent applications who receive notice of grant after the EU

Patent Package came into effect could, for the UPC Member States, either obtain a Unitary Patent or validate the European

patent nationally and optionally file an opt-out demand. The EU Patent Package may increase the uncertainties and costs

surrounding the enforcement or defense of our issued European patents and pending applications. The full impact on future

European patent filing strategy and the enforcement or defense of our issued European patents in European jurisdictions

and/or the UPC is not fully known.

Filing, prosecuting, maintaining, defending and enforcing patents for RYTELO and our technologies in all

countries throughout the world would be prohibitively expensive, and our intellectual property rights in some countries

outside the U.S. are less extensive than those in the U.S. The requirements for patentability may differ in certain countries,

particularly in developing countries; thus, even in countries where we do pursue patent protection, there can be no

assurance that any patents will be issued with claims that cover RYTELO and our technologies.

We may be involved in lawsuits to protect or enforce our patents or other intellectual property rights, which could be

expensive, time consuming and unsuccessful, and which could result in the invalidity or unenforceability of our patents

covering RYTELO or its methods of use.

Competitors may infringe, misappropriate or otherwise violate our patents or other intellectual property

rights. To counter infringement or unauthorized use, we may be required to file and prosecute legal claims against one or

more third parties, which can be expensive and time-consuming, even if ultimately successful.

The initiation of a claim against a third party by us may also cause the third party to bring counter claims

against us, such as claims asserting that our patents are invalid or unenforceable. Grounds for a validity challenge could be

an alleged failure to meet any of several statutory requirements, including lack of novelty, obviousness, non-enablement or

lack of written description or non-statutory subject matter. Grounds for an unenforceability assertion could be an allegation

that someone connected with prosecution of the patent withheld relevant material information from the USPTO, or made a

materially misleading statement, during prosecution. Third parties may also raise similar validity claims before the USPTO

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in post-grant proceedings such as ex parte reexaminations, inter partes review, or IPR, or post-grant review, or oppositions

or similar proceedings outside the U.S., in parallel with litigation or even outside the context of litigation.

In an infringement proceeding, a court may decide that a patent of ours is not valid or is unenforceable, or

may refuse to stop the other party from using the technology at issue on the grounds that our patents do not cover the

technology in question. The standards that courts use to interpret patents are not always applied predictably or uniformly

and can change, particularly as new technologies develop. As a result, we cannot predict with certainty how much

protection, if any, will be given to our patents if we attempt to enforce them and they are challenged in court and if any

such lawsuits will ultimately be resolved successfully. Further, even if we prevail, the infringer may file an appeal and the

court judgment may be overturned and/or that an adverse decision may be issued by an appeals court relating to the validity

or enforceability of our patents. An adverse result in any litigation or defense proceedings could put one or more of our

patents at risk of being invalidated or interpreted narrowly in a manner insufficient to achieve our business objectives. Even

if we establish infringement, we may not seek, or the court may decide not to grant, an injunction against further infringing

activity and instead award only monetary damages, which may or may not be an adequate remedy.

If a defendant were to prevail on a legal assertion of invalidity or unenforceability, we would lose at least

part, and perhaps all, of any future patent protection for RYTELO, which could have a material adverse effect on our

business, financial condition, results of operations and prospects. Additionally, any adverse outcome could allow third

parties to commercialize RYTELO and compete directly with us, without payment to us.

Furthermore, if we are engaged in intellectual property litigation, there would be public announcements of

filings, briefings, hearings, motions or other interim proceedings or developments. If securities analysts or investors

perceive these events to be negative, it could have an adverse effect on the price of our common stock.

Many companies have encountered significant problems in protecting and defending intellectual property

rights in jurisdictions outside the U.S. The legal systems of certain countries, particularly certain developing countries, do

not favor the enforcement of patents, trade secrets and other intellectual property protection, particularly those relating to

biotechnology and pharmaceutical products, which could make it difficult for us to stop the infringement of our patents or

marketing of competing products in violation of our proprietary rights generally. For example, many countries outside the

U.S. have compulsory licensing laws under which a patent owner must grant licenses to third parties. Proceedings to

enforce our patent rights in jurisdictions outside the U.S. could result in substantial costs and divert our efforts and

attention from other aspects of our business, and could put our patents at risk of being invalidated or interpreted narrowly.

We may not be able to protect our intellectual property rights in the U.S or worldwide and challenges to our owned or

licensed patent rights would result in costly and time-consuming legal proceedings that could prevent or limit

development or commercialization of RYTELO.

Our patents or those patent rights we have licensed, including patent rights that we may seek with respect to

inventions made by past or future collaborators, may be challenged through administrative or judicial proceedings, which

could result in the loss of important patent rights. For example, where more than one party seeks U.S. patent protection for

the same technology in patent applications that are subject to the law before the implementation of the AIA, the USPTO

may declare an interference proceeding in order to ascertain the party to which the patent should be issued. Patent

interferences are typically complex, highly contested legal proceedings, subject to appeal. They are usually expensive and

prolonged and can cause significant delays in the issuance of patents. Our pending patent applications or our issued patents,

or those we have licensed and may license from others, may be drawn into interference proceedings or be challenged

through post-grant review procedures or litigation, any of which could delay or prevent the issuance of patents, or result in

the loss of issued patent rights. We may not be able to obtain from our past or future collaborators the information needed

to support our patent rights which could result in the loss of important patent rights.

Under the AIA, interference proceedings between patent applications filed on or after March 16, 2013, have

been replaced with other types of proceedings, including derivation proceedings. The AIA also includes post-grant review

procedures subjecting U.S. patents to post-grant review procedures similar to European oppositions, such as IPR, covered

business method post-grant reviews and other post-grant reviews. This applies to all our U.S. patents and those we have

licensed and may license from others, even those issued before March 16, 2013. A third party could attempt to use the

USPTO procedures to invalidate patent claims that would not have been invalidated if first challenged by the third party as

a defendant in a district court action. U.S. patents owned or licensed by us may therefore be subject to post-grant review

procedures, as well as other forms of review and re-examination. In addition, the IPR process under the AIA permits any

person, whether they are accused of infringing the patent at issue or not, such as entities associated with hedge funds, to

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challenge the validity of certain patents. Significant impairment of our RYTELO patent rights could severely and adversely

affect our financial results, business and business prospects, which might cause us to cease operations.

Certain jurisdictions, such as Europe, China, Japan, New Zealand and Australia, permit third parties to file

third party observations, oppositions or invalidation trials against granted patents and/or patent applications. Because we

seek to enable potential global commercialization of RYTELO, securing both proprietary protection and freedom to operate

outside of the U.S. is important to our business.

Third party proceedings such as oppositions and invalidation trials require significant time and costs, and if

we are unsuccessful or are unable to commit these types of resources to protect our RYTELO patent rights, we could lose

our patent rights and we could be prevented or limited in the development and commercialization of RYTELO.

As more groups become engaged in scientific research and product development in the areas of telomerase

biology and hematologic malignancies, the risk of our patents, or patents that we have in-licensed, being challenged

through patent interferences, derivation proceedings, IPRs, post-grant proceedings, oppositions, invalidation trials, re-

examinations, litigation or other means will likely increase. Challenges to our patents through these procedures would be

extremely expensive and time-consuming, even if the outcome was favorable to us. An adverse outcome in a patent dispute

could severely harm our ability to further develop or commercialize RYTELO, or could otherwise have a material adverse

effect on our business, and might cause us to cease operations, by:

• causing us to lose patent rights in the relevant jurisdiction(s);

• subjecting us to litigation, or other types of proceedings aimed at preventing us from

commercializing RYTELO in the relevant jurisdiction(s);

• requiring us to obtain licenses to certain patents and patent applications;

• forcing us to cease using the disputed technology; or

• requiring us to develop or obtain alternative technologies.

We may be subject to infringement claims that are costly to defend, and such claims may limit our ability to use disputed

technologies and prevent us from pursuing research, development, manufacturing or commercialization of RYTELO.

The commercial success of RYTELO will depend upon our ability to research, develop, manufacture,

market and sell RYTELO without infringing or otherwise violating the intellectual property and other proprietary rights of

third parties. There is considerable intellectual property litigation in the biotechnology and pharmaceutical industries, and

many pharmaceutical companies, including potential competitors, have substantial patent portfolios. Since we cannot be

aware of all intellectual property rights potentially relating to RYTELO and its uses, we do not know with certainty that

RYTELO, or the commercialization thereof, does not and will not infringe or otherwise violate any third party’s

intellectual property. For example, we are aware that certain third parties have or may be prosecuting patents and patent

estates that may relate to RYTELO, and while these patents have expired, or we believe that a reasonable court should find

they are invalid and/or would not be infringed by the manufacture, use or sale of RYTELO, it is possible that the owner(s)

of these patents will assert claims against us in the future.

In the event our technologies infringe the rights of others or require the use of discoveries and technologies

controlled by third parties, we may be prevented from pursuing research, development, manufacturing or

commercialization of RYTELO, or may be required to obtain unblocking licenses from such third parties, develop

alternative non-infringing technologies, which we may not be able to do at an acceptable cost or on acceptable terms, or at

all, or cease the commercialization and continued development of RYTELO. If we are unable to resolve an infringement

claim successfully, we could be subject to an injunction that would prevent us from commercializing RYTELO and could

also require us to pay substantial damages.

In addition, while our past collaboration agreements have terminated, we are still subject to indemnification

obligations to certain collaborators, including with respect to claims of third-party patent infringement. In addition to

infringement claims, in the future we may also be subject to other claims relating to intellectual property, such as claims

that we have misappropriated the trade secrets of third parties. Our success therefore depends significantly on our ability to

operate without infringing patents and the proprietary rights of others.

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We may become aware of discoveries and technologies controlled by third parties that are advantageous or

necessary to further develop or manufacture RYTELO. Under such circumstances, we may initiate negotiations for licenses

to other technologies as the need or opportunity arises. We may not be able to obtain a license to a technology required to

pursue the research, development, manufacturing or commercialization of RYTELO on commercially favorable terms, or

at all, or such licenses may be terminated on certain grounds, including as a result of our failure to comply with any

material obligations under such licenses. If we do not obtain a necessary license or if such a license is terminated, we may

need to redesign such technologies or obtain rights to alternative technologies, which may not be possible, and even if

possible, could cause further delays in the development efforts for RYTELO and could increase the development and/or

production costs of RYTELO. In cases where we are unable to license necessary technologies, we could be subject to

litigation and prevented from pursuing research, development, manufacturing or commercialization of RYTELO, which

would materially and adversely impact our business. Failure by us to obtain rights to alternative technologies or a license to

any technology that may be required to pursue research, development, manufacturing or commercialization of RYTELO

would further delay current and potential future clinical trials of RYTELO and any applications for regulatory approval,

impair our ability to sell RYTELO, and therefore result in decreased sales of RYTELO for us. Occurrence of any of these

events could materially and adversely affect our business and might cause us to cease operations.

We have a registered trademark, RYTELO, for our product and failure to maintain such trademark could adversely

affect our business.

We have a registered trademark, RYTELO, which is the commercial trade name for imetelstat, in a number

of countries and regions, including in the U.S. and Europe. Our product trademark, RYTELO, is approved for use as name

of the imetelstat medicinal product by the FDA and the EC. Opposition or cancellation proceedings, however, may be filed

against our trademarks, and our trademarks may not survive such proceedings. If our U.S. trademark application which

forms the basis for our international registration, or IR, for our commercial trade name is withdrawn or abandoned within

the first five years of our IR, we will lose our IR registrations which could adversely affect our business. We may be unable

to maintain or enforce our current and future trademarks, and if we fail to satisfy the applicable regulatory requirements,

we may not have enforceable trademark rights or registrations in such jurisdictions.

We may become involved in disputes with past or future collaborator(s) over intellectual property inventorship,

ownership or use, and publications by us, or by investigators, scientific consultants, research collaborators or others.

Such disputes could impair our ability to obtain patent protection or protect our proprietary information, which, in

either case, could have a significant impact on our business.

Inventions discovered under research, material transfer or other collaboration agreements may become

jointly owned by us and the other party to such agreements in some cases and may be the exclusive property of either party

in other cases. Under some circumstances, it may be difficult to determine who invents and owns a particular invention, or

whether it is jointly owned, and disputes can arise regarding inventorship, ownership and use of those inventions. These

disputes could be costly and time-consuming, and an unfavorable outcome could have a significant adverse effect on our

business if we are not able to protect or license rights to these inventions. In addition, clinical trial investigators, scientific

consultants and research collaborators generally have contractual rights to publish data and other proprietary information,

subject to review by the trial sponsor. Publications by us, or by investigators, scientific consultants, previous employees,

research collaborators or others, either with permission or in contravention of the terms of their agreements with us or with

our past or future collaborators, may impair our ability to obtain patent protection or protect proprietary information, which

could have a material adverse effect on our business and might cause us to cease operations.

Much of the information and know-how that is critical to our business is not patentable, and we may not be able to

prevent others from obtaining this information and establishing competitive enterprises.

In addition to patent and trademark protection, we rely on trade secrets to protect our proprietary

technology, especially in circumstances in which we believe patent protection is not appropriate or available. We attempt to

protect our proprietary technology in part by entering into confidentiality agreements with our employees, consultants,

collaborators and contractors. However, certain consultants and third parties with whom we have business relationships,

and to whom in some cases we have disclosed trade secrets and other proprietary knowledge, may also provide services to

other parties in the medical device/pharmaceutical industry, including companies, universities and research organizations

that are developing competing products. In addition, some of our former employees who were exposed to certain of our

trade secrets and other proprietary knowledge in the course of their employment may seek employment with, and become

employed by, our competitors. We cannot provide assurance that these agreements will not be breached, that we would

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have adequate remedies for any breach, or that our trade secrets will not otherwise become known or be independently

discovered by competitors, any of which would harm our business significantly.

Trade secret protection does not prevent independent discovery of the technology or proprietary

information or use of the same. Competitors may independently duplicate or exceed our technology in whole or in part. If

any of our trade secrets were to be lawfully obtained or independently developed by a competitor, we would have no right

to prevent them, or those to whom they communicate it, from using that technology or information to compete with us in

countries where we do not have patent protection.

RISKS RELATED TO MANAGING OUR GROWTH AND OTHER BUSINESS OPERATIONS

Our strategic restructuring plan and the associated workforce reduction implemented in December 2025 may not result

in anticipated savings and long-term value creation, could result in total costs and operating expenses that are greater

than expected and could disrupt our business.

In connection with our strategic restructuring plan announced in December 2025, we implemented a

workforce reduction, representing approximately one-third of our workforce prior to the reduction in headcount. We may

not realize, in full or in part, the anticipated benefits on our 2026 operating expenses from our restructuring efforts due to

unforeseen difficulties, delays or unexpected costs. We incurred approximately $17.0 million in restructuring and

restructuring-related charges for the year ended December 31, 2025, primarily consisting of one-time employee severance

payments, healthcare and related benefits, and other employee-related costs, and we estimate the workforce reduction will

be substantially completed in the first quarter of 2026. If we are unable to realize the expected operational efficiencies and

cost savings from the restructuring, our operating results and financial condition could be adversely affected. We also

cannot guarantee that we will not have to undertake additional workforce reductions or restructuring activities in the future.

Furthermore, our workforce reduction may be disruptive to our operations, or could yield unanticipated consequences, such

as attrition beyond planned staff reductions, or disruptions in our day-to-day operations. Our workforce reductions could

also harm our ability to attract and retain qualified management, scientific, clinical, and manufacturing personnel who are

critical to our business. Any failure to attract or retain qualified personnel could prevent us from successfully developing

and commercializing our product candidates in the future, if approved.

We may be unable to successfully retain or recruit key personnel to support the commercialization and further

development of RYTELO or to otherwise successfully manage our growth.

Our ability to successfully commercialize RYTELO in the U.S. and in the EU for lower-risk MDS, and to

continue to develop RYTELO in other myeloid hematologic malignancies depends to a significant extent on the skills,

experience and efforts of our executive officers and key members of our staff. In addition, we need to recruit, maintain,

motivate and integrate additional personnel with expertise and experience in sales, marketing, market access, commercial

operations, pricing, clinical science, biostatistics, clinical operations, pharmacovigilance, quality, manufacturing, regulatory

affairs, medical affairs, legal affairs, and compliance to enable us to further commercialize and further develop RYTELO.

We face intense competition for qualified individuals from numerous pharmaceutical, biopharmaceutical

and biotechnology companies, as well as academic and other research institutions, and competition in our geographic

regions is particularly intense. The substantial risks and uncertainties related to our commercialization and further

development of RYTELO, and the risks and uncertainties regarding our future business viability could have an adverse

impact on our ability to retain and recruit qualified personnel. We may also face higher than expected personnel costs in

order to attract new personnel due to shortages in qualified applicants, or to maintain our current management and

personnel due to the increased number of opportunities in the biotechnology sector. If we are unable to successfully retain,

motivate and incentivize our existing personnel, or to attract, assimilate and retain other highly qualified personnel in the

future on acceptable terms, our ability to commercialize and further develop RYTELO will be impaired, and our business

and the price of our common stock would be adversely impacted.

In addition, our personnel are currently performing their duties in multiple jurisdictions, and if we are

unable or fail to comply with employment, tax, benefits and other laws in such jurisdictions, we may face penalties, fines or

litigation.

Our future financial performance and our ability to develop, manufacture and commercialize RYTELO

depends, in part, on our ability to effectively manage any future growth. Our management may have to divert financial and

other resources, as well as devote a substantial amount of time, to managing growth activities, such as enhancing

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operational, financial and management processes and systems. If we do not effectively manage the expansion of our

operations, we could experience weaknesses in our infrastructure and ability to comply with applicable legal and regulatory

requirements and regulations, operational mistakes or shortcomings, loss of business opportunities, loss of employees and

reduced productivity among remaining employees.

Management transition creates uncertainties and could harm our business.

Over the past few years, we have experienced significant changes in executive leadership, and more could

occur. For example, on August 6, 2025, we announced the appointment of Harout Semerjian as our President and Chief

Executive Officer and a member of our board of directors, effective August 7, 2025. In addition, in October 2025, we

appointed a new EVP, Chief Commercial Officer and in November 2024, we appointed a new EVP of Research and

Development. Additionally, in connection with our strategic restructuring plan announced in December 2025, we

implemented a workforce reduction, representing approximately one-third of our workforce prior to the reduction in

headcount. Changes to company strategy, which can often times occur with the appointment of new executives and

departure of prior executives, can create uncertainty, may negatively impact our ability to execute quickly and effectively,

and may ultimately be unsuccessful. In addition, executive leadership transition periods are often difficult as the new

executives gain detailed knowledge of our operations, and friction can result from changes in strategy and management

style. Management transition inherently causes some loss of institutional knowledge, which may have a disruptive impact

on our ability to implement our strategy and could have a material adverse effect on our financial condition and our

business and business prospects can negatively affect strategy and execution. Until we integrate new personnel, and unless

they are able to succeed in their positions, we may be unable to successfully manage and grow our business, and our results

of operations and financial condition could suffer as a result.

Although we intend to establish potential future collaborative arrangements for RYTELO, we may be unable to

establish such collaborative arrangements on acceptable terms, or at all, and may have to delay, alter or abandon

commercialization or further development of RYTELO.

We intend to develop RYTELO broadly for hematologic malignancies, and to continue to commercialize,

market and sell RYTELO in the U.S. for certain patients with lower-risk MDS ourselves. At this time, we do not plan to

commercialize RYTELO independently in the EU (or in any other regions outside of the U.S. where RYTELO may be

approved for marketing in the future). Accordingly, we plan to work with experienced third parties for the

commercialization and marketing of RYTELO in the EU, including on critical path activities for the planned launch of

RYTELO in the EU, such as reimbursement, Health Technology Assessment, or HTA, submissions, market access and

distribution, and w e may otherwise seek collaborative partners, at an appropriate time, to assist us in the potential

development and commercialization of RYTELO outside the U.S., and to provide funding for such activities. We face

significant competition in seeking appropriate collaborative partners, and these potential collaborative arrangements are

complex and time consuming to negotiate, document and implement. In addition, the terms of our Pharmakon Loan

Agreement may limit our ability to enter into certain collaborative arrangements and any future debt agreements may

continue or further limit our ability to enter into such agreements. We may not be able to establish collaborative

arrangements on acceptable terms, or at all. In this regard, collaborative arrangements with third parties may require us to

relinquish material rights, including revenue from commercialization, or assume material ongoing development obligations

that we would have to fund or otherwise support.

If we are unable to negotiate collaborative arrangements, we may have to:

• delay, curtail or abandon the additional development of RYTELO;

• delay, curtail or abandon the commercialization of RYTELO in jurisdictions where it is

approved;

• reduce the scope of potential future sales or marketing activities; or

• increase our expenditures and undertake development or commercialization activities at our

own expense, which will require additional capital than our current resources.

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We have established subsidiaries in the United Kingdom and the Netherlands, which exposes us to additional costs and

risks.

The wholly-owned subsidiaries we have established in the U.K. and the Netherlands subject us to certain

additional costs and risks associated with doing business outside the U.S., including:

• the increased complexity and costs inherent in managing international operations in

geographically disparate locations;

• challenges and costs of complying with diverse regulatory, financial and legal requirements,

which are subject to change at any time;

• potentially adverse tax consequences, including changes in applicable tax laws and

regulations;

• potentially costly trade laws, tariffs, export quotas, custom duties or other trade restrictions,

and any changes to them, including in connection with new Trump administration changes;

• compliance with tax, employment, immigration and labor laws for employees living or

traveling abroad;

• challenges inherent in efficiently managing employees in diverse geographies, including the

need to adapt systems, policies, benefits and compliance programs to differing labor and

other regulations;

• natural disasters, political and economic instability, including terrorism and civil and

political unrest, outbreak of health epidemics, and the resulting global economic and social

impacts; and

• workforce uncertainty in countries where labor unrest is more common than in the U.S.

We may experience additional risks related to operating outside of the U.S. that could materially adversely affect our

business.

We have employees located outside of the U.S., conduct clinical trials outside of the U.S., and are pursuing

paths to make RYTELO available to LR-MDS patients outside of the U.S., including in the EU, which may subject us to

additional risks, including risks related to operating outside of the U.S., such as:

• we may experience unexpected changes in tariffs, trade barriers, price and exchange

controls and other regulatory requirements;

• risks of potential noncompliance by us or by any third parties we engage with legal

requirements applicable to privacy, data protection, information security and other matters;

• risks of potential noncompliance with tax, employment, immigration and labor laws for

employees living or traveling abroad;

• increased taxes outside of the U.S., including withholding and payroll taxes;

• significant foreign currency fluctuations, which could result in increased operating expenses

and reduced revenue, and other obligations incident to doing business in another country;

• difficulties staffing and managing operations outside of the U.S.;

• complexities associated with managing multiple payor reimbursement regimes and

government payors in foreign countries;

• workforce uncertainty in countries where labor unrest is more common than in the U.S.;

• potential liability under the Foreign Corrupt Practices Act of 1977 or comparable

regulations outside of the U.S.; and

• business interruptions resulting from geopolitical actions, including war and terrorism.

For example, the current administration in the U.S. has called for substantial changes to foreign trade policy

and has recently imposed significant increases in tariffs on international trade and the renegotiation of international trade

agreements. We cannot predict what effects these and potential additional tariffs or renegotiation of existing international

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trade agreements, scope and nature of tariffs in the future, including as a result of litigation or or other challenges, will have

on our business, including in the context of escalating global trade and political tensions. However, such tariffs and other

trade restrictions could increase our cost of doing business, reduce our gross margins or otherwise negatively impact our

financial results. See the risk factor titled, “ Global trade issues and changes in and uncertainties with respect to trade

policies and export regulations, including import and export license requirements, trade sanctions, tariffs and international

trade disputes, could increase our costs and negatively impact net revenues from sales of RYTELO .”

Uncertainty in the regulatory framework and future legislation could lead to disruption in the execution of

international multi-center clinical trials, the monitoring of adverse events through pharmacovigilance programs, the

evaluation of the benefit-risk profiles of new medicinal products, and determination of marketing authorization across

different jurisdictions. Changes to existing regulations may add considerably to the time from clinical development to

marketing authorization and commercialization of products in foreign jurisdictions and increase our costs. We cannot

predict the impact of such changes and future regulation on our business or the results of our operations. These and other

risks associated with international operations may materially adversely affect our ability to attain or maintain profitable

operations.

Global trade issues and changes in and uncertainties with respect to trade policies and export regulations, including

import and export license requirements, trade sanctions, tariffs and international trade disputes, could increase our

costs and negatively impact net revenues from sales of RYTELO.

There is inherent risk, based on the complex relationships among the U.S. and the countries in which we

now conduct or may in the future conduct our business, that political, diplomatic, and national security factors can lead to

global trade restrictions and changes in trade policies and export regulations that may adversely affect our business and

operations. Compliance with applicable regulatory requirements regarding the export of products may create delays in the

introduction of RYTELO in international markets, including in the EU, or, in some cases, prevent the export of RYTELO

to some countries altogether. Furthermore, U.S. export control laws and economic sanctions prohibit the provision of

certain products and services to countries, governments and persons targeted by U.S. sanctions. The U.S. and other

countries have imposed and may continue to impose new trade restrictions and export regulations, have levied tariffs and

taxes on certain goods, and could continue to significantly increase tariffs on a broad array of goods, including

pharmaceutical and biological products. Notwithstanding the U.S. Supreme Court’s recent decision invalidating tariffs

imposed under the International Emergency Economic Powers Act, the magnitude and impact of tariffs are uncertain and

are subject to a variety of factors, including the effective date and duration of additional tariffs, changes in the amount,

scope and nature of tariffs in the future, including as a result of litigation or other challenges, any retaliatory tariffs that

other countries may impose in response to tariffs levied by the United States and any mitigating actions that may become

available.

The ongoing trade tensions between the U.S. and other jurisdictions have resulted in multiple rounds of

tariffs and potential tariffs affecting pharmaceuticals and pharmaceutical ingredients, including finished drug products,

manufacturing equipment, and related supplies. In April 2025, the U.S. government imposed a 10% baseline global tariff

and in August 2025, the U.S. imposed higher “reciprocal” tariffs on numerous other territories, including EU Member

States and South Korea. While the U.S. Supreme Court recently issued a ruling invalidating tariffs imposed by the Trump

administration under the International Emergency Economic Powers Act, other tariffs imposed by the U.S. government

remain in place, including the 10% global tariff imposed by the Trump administration under Section 122 of the Trade Act

of 1974 following the U.S. Supreme Court decision. Moreover, the Bureau of Industry and Security, U.S. Department of

Commerce, has initiated an investigation to determine whether pharmaceutical ingredients, including finished drug product,

manufactured outside the U.S. pose a national security risk and should be subject to additional tariffs. Unlike consumer

goods, pharmaceuticals face unique regulatory constraints that make rapid supply chain adjustments particularly difficult

and costly . Although a significant portion of our supply chain for imetelstat is currently based in the U.S., the active

pharmaceutical ingredient, or API, for imetelstat is manufactured in South Korea and our 47mg vial of RYTELO drug

product is currently manufactured in Italy. In addition, in the future we may choose to utilize other contract manufacturers

for different presentations of imetelstat and source materials for our supply chain from other international jurisdictions.

Accordingly, such global or industry-specific tariffs, or the renegotiation of current international trade agreements, or other

trade restrictions could result in additional costs on our business, including generally increasing our manufacturing costs,

and may decrease our gross margins and increase our supply chain complexity. Moreover, other governments have

imposed and may continue to impose retaliatory tariffs, trade restrictions or trade barriers impacting RYTELO, which

could impose additional costs and complexity on our business, including with respect to our planned commercialization of

RYTELO in select EU markets in 2026 or restrict our ability to sell RYTELO in the EU or in other international markets

where we may obtain approval of RYTELO.

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Further, the continued threats of new or increased tariffs, sanctions, trade restrictions and trade barriers as

well as ongoing changes in U.S. and foreign government trade policies, including potential modifications to existing

international trade agreements, have had and may continue to have a generally disruptive impact on the global economy

and, therefore, could negatively impact revenues from sales of RYTELO. Given the significant volatility and uncertainty

regarding the scope and duration of such tariffs and other aspects of U.S. and foreign government trade policies, the

ultimate impact on our operations and financial results is uncertain and could be significant in the future.

We may not be able to obtain or maintain sufficient insurance on commercially reasonable terms or with adequate

coverage against potential liabilities in order to protect ourselves against claims such as product liability or personal

injury claims arising from our commercialization of RYTELO, claims related to clinical trial conduct, or claims related

to data protection.

Our business exposes us to potential product liability and other risks that are inherent in the testing,

manufacturing and marketing of human therapeutic products. We may become subject to product liability or personal

injury claims related to the commercialization of RYTELO, or claims related to clinical trial conduct, including if the use

of RYTELO is alleged to have injured patients, such as injuries alleged to arise from any hepatotoxicity or hemorrhagic

event associated with the use of RYTELO. We currently have product liability and clinical trial liability insurance that we

believe is adequate, but we may experience losses in excess of our coverage or that are not covered by our insurance, and

we may not be able to maintain this type of insurance for the commercialization of RYTELO, or any of our current or

potential future clinical trials of RYTELO. In addition, this type of insurance may become too expensive for us to afford

because of the highly risky and uncertain nature of commercialization of RYTELO, clinical trials generally and the high

cost of insurance for our business activities. We may be unable to obtain or maintain clinical trial insurance in all of the

jurisdictions where we conduct current or potential future clinical trials. In addition, business liability, product liability and

cybersecurity insurance are becoming increasingly expensive, particularly for biotechnology and pharmaceutical

companies, and the pool of insurers offering insurance coverage to biotechnology and pharmaceutical companies generally

is becoming smaller, making it more difficult to obtain insurance for our business activities at a reasonable price, or at all.

Being unable to obtain or maintain product liability, clinical trial liability, cybersecurity or other insurance for our business

activities in the future on acceptable terms or with adequate coverage against potential liabilities would have a material

adverse effect on our business, and could cause us to limit or cease our commercialization and further development of

RYTELO.

We and certain of our current and former officers and directors have been named as defendants in securities class

action lawsuits and derivative lawsuits. These lawsuits, and potential similar or related lawsuits, could result in

substantial damages, divert management’s time and attention from our business, and have a material adverse effect on

our results of operations. These lawsuits, and any other lawsuits to which we are subject, will be costly to defend or

pursue and are uncertain in their outcome.

Securities class action lawsuits and/or derivative lawsuits have often been brought against companies,

including biotechnology and biopharmaceutical companies, that experience volatility in the market price of their securities.

This risk is especially relevant for us because we often experience significant stock price volatility in connection with our

activities. On March 13 and March 14, 2025, we and certain of our current and former officers were named as defendants

in two putative securities class action lawsuits filed in the United States District Court for the Northern District of

California captioned Dabestani v. Geron Corporation, et al., No. 3:25-cv-02507 and Potvin v. Geron Corporation, et al.,

No. 3:25-cv-02563, respectively. Both lawsuits allege violation of Sections 10(b) and 20(a) of the Securities Exchange Act

of 1934, as amended, and Rule 10b-5 promulgated thereunder in connection with allegedly false and misleading statements

concerning the commercial potential of RYTELO. The plaintiffs allege, among other things, that we overstated RYTELO's

commercial potential by making materially false and misleading statements and/or concealing material adverse facts

concerning RYTELO's commercial potential, including the lack of awareness among healthcare providers for RYTELO,

the burden of monitoring requirements in administering the drug, and the impacts of seasonality and existing competition

on RYTELO's sales, and that our stock price dropped when we disclosed in our earnings call on February 26, 2025 that we

had observed flat revenue trends over the prior few months. The plaintiffs seek damages and interest, and an award of

reasonable costs, including attorneys' and experts' fees. On May 29, 2025, the Court consolidated the Dabestani and Potvin

cases into one consolidated action captioned In re Geron Corporation Securities Litigation, or the Securities Class Action,

and appointed lead plaintiffs and counsel for lead plaintiffs. On August 8, 2025, lead plaintiffs filed a consolidated

amended complaint. On October 7, 2025, we filed our motion to dismiss the consolidated amended complaint. A hearing

on the motion to dismiss is scheduled for March 19, 2026.

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In addition, on April 15, 2025 and April 16, 2025, three purported stockholders filed derivative complaints,

each filed in the United States District Court for the Northern District of California, captioned Bishop v. Scarlett, et al., No.

3:25-cv-03356, Lerner v. Scarlett, et al. , No. 3:25-cv-03401, and Willis v. Scarlett, et al., No: 3:25-cv-03396, respectively.

The three lawsuits name certain of our current and former directors and officers and allege that they breached their

fiduciary duties and violated federal securities laws by issuing allegedly false and misleading statements concerning the

commercial potential of RYTELO. The allegations in each of the three derivative complaints are substantially similar to the

two aforementioned securities class action lawsuits, which these lawsuits are premised on. The plaintiff seeks damages and

interest, and an award of reasonable costs, including attorneys' and experts' fees. The plaintiffs in Bishop v. Scarlett, et al.

and Willis v. Scarlett, et al. also seek punitive damages. On May 16, 2025, the Court consolidated the three derivative

complaints into one consolidated action captioned In re Geron Corporation Derivative Litigation , or the Consolidated

Derivative Action. On June 17, 2025, the Court stayed the Consolidated Derivative Action pending a final ruling on the

anticipated motion to dismiss in the Securities Class Action.

On August 29, 2025, a purported stockholder made a demand on our board of directors to commence a civil

action against certain of our current and former directors for breaching their fiduciary duties and violating the securities

laws by issuing allegedly false and misleading statements concerning the commercial potential of RYTELO. On

September 19, 2025, the board of directors responded that it would defer a final decision on the demand given the other

pending derivative lawsuits and the Securities Class Action. On October 7, 2025, the purported stockholder filed a suit in

the United States District Court for the Northern District of California, captioned Jae Hyung v. Bir, et al., No. 3:25-

cv-08575. The derivative lawsuit names certain of our current and former directors and officers. The allegations in the

derivative lawsuit are substantially similar to the Securities Class Action and the aforementioned derivative lawsuits. The

plaintiffs seek damages and an award of reasonable costs, including attorneys’ and experts’ fees. On January 23 and

February 19, 2026, respectively, two additional purported stockholders each made a similar demand on our board of

directors.

We have also been subject to securities class action lawsuits in the past. In 2020, three securities class

action lawsuits were filed against us and certain of our officers. One of the lawsuits was voluntarily dismissed, and we

settled the other two lawsuits and a final judgment was entered in October 2023. In 2020 and 2021, seven shareholder

derivative actions were filed in a number of courts, naming as defendants certain of our then current officers and certain of

our then current and former members of our board. All seven of the shareholder derivative actions were dismissed with

prejudice.

It is possible that additional lawsuits might be filed, or allegations might be received from stockholders,

with respect to these same matters as alleged in the pending lawsuits or other matters and also naming us and/or our

officers and directors as defendants. Such lawsuits and any other related lawsuits are subject to inherent uncertainties, and

the actual defense and disposition costs will depend upon many unknown factors. The outcome of the pending lawsuits and

any other related lawsuits is necessarily uncertain. We could be forced to expend significant resources and may incur

substantial legal fees and costs in the defense of the pending lawsuits and any related or additional lawsuits, and we may

not prevail. Monitoring, initiating and defending against legal actions is also time-consuming for our management, is likely

to be expensive and may detract from our ability to fully focus our internal resources on our business activities. Given the

early stage of these lawsuits and the inherent uncertainly of litigation, we cannot predict how long it may take to resolve the

pending lawsuits or the amount of costs we may incur, or the potential outcome or the possible amount of any damages we

may be required to pay. A decision adverse to our interests in the pending lawsuits or in similar or related litigation, could

result in the payment of substantial damages or settlements, or possibly fines, and, although we maintain liability insurance,

we may not be successful in having any such lawsuits dismissed or settled within the limits of our insurance coverage. If

any judgment or settlement against us and costs or expenses associated with the pending litigation exceed our insurance

coverage or insurance coverage is denied, we may be forced to bear some or all of these costs and expenses directly, which

could be substantial and could have a material adverse effect on our business, our stock price, cash flow, results of

operations and financial condition.

We may be subject to third-party litigation, and such litigation would be costly to defend or pursue and uncertain in its

outcome .

Our business may bring us into conflict with our licensees, licensors, or others with whom we have

contractual or other business relationships, or with our competitors or others whose interests differ from ours. Our

commercialization of RYTELO may result in product or personal injury disputes, or other disputes with health care

providers, patients or other third parties as a result of our commercialization efforts. We may experience employment-

related disputes. We may become involved in performance or other disputes with the CROs we have retained to support

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our clinical development activities, or with other third parties such as service providers, vendors, manufacturers, suppliers

or consultants. If we are unable to resolve those conflicts on terms that are satisfactory to all parties, we may become

involved in litigation brought by or against us.

Lawsuits are subject to inherent uncertainties, and defense and disposition costs depend upon many

unknown factors. Despite the availability of insurance, we may incur substantial legal fees and costs in connection with

litigation. Lawsuits could result in judgments against us that require us to pay damages, enjoin us from certain activities, or

otherwise negatively affect our legal or contractual rights, which could have a significant adverse effect on our business. In

addition, the inherent uncertainty of such litigation could lead to increased volatility in our stock price and a decrease in the

value of our stockholders’ investment in our securities.

We are subject to U.S. and certain foreign export and import controls, sanctions, embargoes, anti-corruption laws, and

anti-money laundering laws and regulations. Compliance with these legal standards could impair our ability to compete

in domestic and international markets. We can face criminal liability and other serious consequences for violations,

which can harm our business.

We are subject to export control and import laws and regulations, and other state and national anti-bribery

and anti-money laundering laws in the countries in which we conduct activities. Anti-corruption laws are interpreted

broadly and prohibit companies and their employees, agents, contractors, and other collaborators from authorizing,

promising, offering, or providing, directly or indirectly, improper payments or anything else of value to recipients in the

public or private sector. We may engage third parties to sell our products outside the U.S., to conduct clinical trials, and/or

to obtain necessary permits, licenses, patent registrations, and other regulatory approvals. We have direct or indirect

interactions with officials and employees of government agencies or government-affiliated hospitals, universities, and other

organizations. We can be held liable for the corrupt or other illegal activities of our employees, agents, contractors, and

other collaborators, even if we do not explicitly authorize or have actual knowledge of such activities. Any violations of the

laws and regulations described above may result in substantial civil and criminal fines and penalties, imprisonment, the loss

of export or import privileges, debarment, tax reassessments, breach of contract and fraud litigation, reputational harm, and

other consequences.

RISKS RELATED TO INFORMATION TECHNOLOGY SYSTEMS, DATA SECURITY AND DATA PRIVACY

If our information technology systems or data, or those of third parties with whom we work, are or were compromised,

we could experience adverse consequences resulting from such compromise, including regulatory investigations or

actions; litigation; fines and penalties; a disruption of our business operations, including our clinical trials;

reputational harm; loss of revenue and profits; and other adverse consequences.

In the ordinary course of our business, we and third parties with whom we work collect, receive, store, use,

transfer, make accessible, protect, secure, dispose of, transmit, disclose, or otherwise process proprietary, confidential, and

sensitive data, including personal data such as health-related data and participant study related data, intellectual property,

and trade secrets (collectively, sensitive information). In addition, we rely on third-party service providers to establish and

maintain appropriate information technology and data security protections, including disaster recovery and business

continuity procedures, over the information technology systems they provide us to operate our critical business systems,

including cloud-based infrastructure and systems, employee email, and data storage and management systems. However,

except for the initial cyber security assessments that we conduct for critical service providers and contractual duties and

obligations, we have limited ability to control or monitor third parties’ safeguards and actions related to such matters.

Furthermore, while we may be entitled to damages if our third-party service providers fail to satisfy their privacy or

security-related obligations to us, any award may be insufficient to cover our damages, or we may be unable to recover

such award. Most of our employees work remotely, resulting in increased risks of loss or theft of company devices as well

as increased risks to our information technology systems and data, as employees utilize network connections, computers,

and devices outside our premises and networks, including working at home and while in transit and in public locations.

Future or past business transactions, such as acquisitions or integrations, could expose us to additional

cybersecurity risks and vulnerabilities, as our systems could be negatively affected by vulnerabilities present in acquired or

integrated entities’ systems and technologies. Furthermore, we may discover security issues that were not found during due

diligence of such acquired or integrated entities, and it may be difficult to integrate companies into our information

technology environment and security program.

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Our information technology systems, including in our remote work environment, and those of the third

parties with whom we work, have been in the past and may continue to be vulnerable to evolving threats. These threats are

prevalent, continue to increase, and come from a variety of sources such as traditional “hackers,” threat actors,

“hacktivists,” organized criminal threats actors, or internal bad actors, personnel, sophisticated nation states and nation-

state-supported actors. These threats include, but are not limited to, social-engineering attacks, targeted phishing

campaigns, malicious code or malware, unauthorized intrusions, denial-of-service attacks, personnel misconduct or errors,

ransomware attacks, supply-chain attacks, software bugs, computer viruses, server malfunctions, software, hardware or

data center failures, loss of data or other information technology assets, natural disasters, terrorism, war,

telecommunication and electrical failures and attacks enhanced or facilitated by artificial intelligence, or AI, and other

similar threats. In particular, ransomware attacks are becoming increasingly prevalent and severe and can lead to significant

interruptions in operations, loss of sensitive data and income, reputational harm, and diversion of funds.

If we were to experience such an attack, extortion payments might alleviate the negative impact of a

ransomware attack, but we might be unwilling or unable to make such payments due to, for example, applicable laws or

regulations prohibiting such payments. Similarly, supply-chain attacks and attacks on clinical trial sites as well as

regulatory and health authorities have increased in frequency and severity, and we cannot guarantee that third parties and

infrastructure in our supply chain or our third-party partners’ supply chains, or of clinical trial sites and regulatory and

health authorities, have not been compromised or that they do not contain exploitable defects or bugs that could result in a

breach of or disruption to our information technology systems or the third-party information technology systems that

support us and the services provided to us, or remediate and recover compromised systems in a timely manner.

Such incidents or threats may result in unauthorized, unlawful or accidental loss, corruption, access,

modification, destruction, alteration, acquisition or disclosure of sensitive information. The costs to us to attempt to protect

against such security incidents could be significant, including potentially requiring us to modify our business, and while we

have implemented security measures, policies and procedures designed to protect our information technology systems from

cybersecurity threats and to identify and remediate vulnerabilities, such measures may not be fully implemented, complied

with or successful in protecting our systems and information. We may expend significant resources or modify our business

activities (including our clinical trial activities) to try to protect against security incidents. We may be unable in the future

to detect cybersecurity threats or vulnerabilities in our information technology systems because such threats and techniques

change frequently, are sophisticated in nature, and may not be detected until after a security incident has occurred. We may

also experience security breaches that may remain undetected for an extended period. Even if identified, we may be unable

to adequately investigate or remediate incidents or breaches due to attackers increasingly using tools and techniques that

are designed to circumvent controls, to avoid detection, and to remove or obfuscate forensic evidence. Unremediated high

risk or critical vulnerabilities pose material risks to our business, particularly due to the reliance on software vendors to

adequately patch and implement fixes to address critical or high-risk vulnerabilities in a timely manner. Further, we may be

materially impacted by software updates applied by our software vendors if such updates cause significant downtime to our

systems.

If we or third parties with whom we work experience or are perceived to have experienced a breach, we

may experience material adverse consequences. These consequences may include: government enforcement actions (for

example, investigations, fines, penalties, audits, and inspections), interruptions in our operations, including disruption of

our commercialization and development efforts, interruptions or restrictions on processing sensitive data (which could

result in delays in obtaining, or our inability to obtain, regulatory approvals and significantly increase our costs to recover

or reproduce the data), reputational harm, litigation (including class action claims), indemnification obligations, negative

publicity, financial loss, and other harms. In addition, such a breach may require public notification of the breach, or we

may choose to voluntarily notify relevant stakeholders, or take other actions, such as providing credit monitoring and

identity theft protection services, and we have done so in the past. Such disclosures are costly, and the disclosure or the

failure to comply with such requirements could lead to adverse consequences.

In addition to experiencing a security incident, third parties may gather, collect, or infer sensitive

information about us from public sources, data brokers, or other means that reveals competitively sensitive details about

our organization and could be used to undermine our competitive advantage or market position. Additionally, sensitive

information of the Company could be leaked, disclosed, or revealed as a result of or in connection with our employees’,

personnel’s, or vendors’ use of generative AI technologies.

Many of our contracts with relevant stakeholders include obligations relating to the safeguard of sensitive

information, and a breach could lead to claims against us by such stakeholders. There can be no assurance that the

limitations of liability in our contracts would be enforceable or adequate or would otherwise protect us from liabilities,

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damages, or claims relating to our data privacy and security obligations. In addition, failure to maintain effective internal

accounting controls related to data security breaches and cybersecurity in general could impact our ability to produce

timely and accurate financial statements and could subject us to regulatory scrutiny. While we do maintain cyber liability

insurance, our insurance coverages may not be sufficient in type or amount to cover us against any such losses, claims, or

liabilities related to security breaches, cyber-attacks, cyber intrusion, or other related breaches or disruptions.

We and third parties with whom we work are subject to stringent and changing U.S. and foreign laws, regulations,

rules, contractual obligations, industry standards, policies and other obligations related to data privacy and security.

Our (or the third parties with whom we work) actual or perceived failure to comply with such obligations could lead to

regulatory investigations or actions; litigation; fines and penalties; disruptions of our business operations; reputational

harm; loss of revenue and profits; and other adverse business impacts.

In the ordinary course of business, we process personal data and other sensitive data, including proprietary

and confidential business data, trade secrets, intellectual property, clinical trial participant data, and other sensitive third-

party data. We are therefore subject to or affected by numerous data privacy and security obligations, such as federal, state,

local and foreign laws, regulations, guidance, industry standards, external and internal privacy and security policies,

contracts, and other obligations governing the processing of personal data. These obligations may change, are subject to

differing interpretations and may be inconsistent or conflict among jurisdictions. The global data protection landscape is

rapidly evolving, and implementation standards and enforcement practices are likely to remain uncertain for the foreseeable

future. This evolution may create uncertainty in our business; affect us or our collaborators’, service providers’ and

contractors’ ability to operate in certain jurisdictions or to collect, store, transfer, use and share personal data; necessitate

the acceptance of more onerous obligations in our contracts; result in liability; or impose additional costs on us. These

obligations may necessitate changes to our information technologies, systems, and practices and to those of any third

parties that process personal data on our behalf. In addition, these obligations may require us to change our business model.

Outside the U.S., an increasing number of laws, regulations, and industry standards apply to data privacy

and security. For example, the European Union’s General Data Protection Regulation, or the EU GDPR, and the United

Kingdom’s GDPR, or the UK GDPR (collectively, the “GDPR”), impose strict requirements on the processing of personal

data. For example, under GDPR, government regulators may impose temporary or definitive bans on data processing, fines

of up to 20 million Euros under the EU GDPR, 17.5 million pounds sterling under the UK GDPR or, in each case, 4% of

annual global revenue, whichever is greater; or private litigation related to processing of personal data brought by classes

of data subjects or consumer protection organizations authorized at law to represent their interests.

In addition, we may be unable to transfer personal data from the EEA, the UK and other jurisdictions to the

U.S. or other countries due to data localization requirements or limitations on cross-border data flows. The EEA and other

jurisdictions have enacted laws requiring data to be localized or limiting the transfer of personal data to other countries. In

particular, the EEA and the UK have significantly restricted the transfer of personal data to the U.S. and other countries

whose privacy laws it believes are inadequate. Other jurisdictions may adopt similarly stringent interpretations of their data

localization and cross-border data transfer laws. Although there are currently various mechanisms that may be used to

transfer personal data from the EEA and UK to the U.S. in compliance with law, such as the EEA and UK’s standard

contractual clauses, the UK’s International Data Transfer Agreement / Addendum, and the EU-U.S. Data Privacy

Framework and the UK extension thereto, these mechanisms are subject to legal challenges, and there is no assurance that

we can satisfy or rely on these measures to lawfully transfer personal data to the U.S. If there is no lawful manner for us to

transfer personal data from the EEA, the UK, or other jurisdictions to the U.S., or if the requirements for a legally-

compliant transfer are too onerous, we could face significant adverse consequences, including the interruption or

degradation of our operations, the need to relocate part of or all of our business or data processing activities to other

jurisdictions (such as Europe) at significant expense, increased exposure to regulatory actions, substantial fines and

penalties, the inability to transfer data and work with partners, vendors and other third parties, and injunctions against our

processing or transferring of personal data necessary to operate our business. Additionally, companies that transfer personal

data out of the EEA and UK to other jurisdictions, particularly to the U.S., are subject to increased scrutiny from regulators,

individual litigants, and activist groups, and some EEA regulators have prevented companies from transferring personal

data out of the EEA for allegedly violating the EU GDPR’s cross-border data transfer limitations.

Likewise, we expect that there will continue to be new proposed laws, regulations and industry standards

relating to data privacy and security in the U.S. For example, HIPAA, as amended by HITECH, imposes specific

requirements relating to the privacy, security, and transmission of individually identifiable health data. Additionally, the

California Consumer Privacy Act of 2018, as amended by the California Privacy Rights Act of 2020, or CPRA, collectively

CCPA, imposes obligations on businesses to which it applies. These obligations include, but are not limited to, providing

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specific disclosures in privacy notices and affording California residents certain rights related to their personal data. The

CCPA allows for statutory fines for noncompliance. While the CCPA contains limited exceptions for clinical trial data, the

CCPA’s implementation standards and enforcement practices are likely to remain uncertain for the foreseeable future. In

addition, the CPRA establishes a California Privacy Protection Agency to implement and enforce the CPRA, which could

increase the risk of an enforcement action, and applies to personal data of business representatives and employees. Other

states have also enacted data privacy and security laws. For example, Virginia passed the Consumer Data Protection Act,

and Colorado passed the Colorado Privacy Act, both of which differ from the CPRA and became effective in 2023. If we

become subject to new data privacy and security laws, at the state level or otherwise, the risk of enforcement action against

us could increase because we may become subject to additional obligations, and the number of individuals or entities that

can initiate actions against us may increase.

In Europe, the Network and Information Security Directive (“NIS2”) regulates resilience and incident

response capabilities of entities operating in a number of sectors, including the health sector. Non-compliance with NIS2

may lead up to administrative fines of a maximum of 10 million Euros or up to 2% of the total worldwide revenue of the

preceding fiscal year.

Our employees and personnel use generative AI technologies to perform their work, and the disclosure and

use of personal data in generative AI technologies is subject to various privacy laws and other privacy obligations.

Governments have passed and are likely to pass additional laws regulating generative AI. Our use of this technology could

result in additional compliance costs, regulatory investigations and actions, and lawsuits. If we are unable to use generative

AI, it could make our business less efficient and result in competitive disadvantages.

In addition to data privacy and security laws, we are contractually subject to industry standards adopted by

industry groups and we are, and may become in the future, subject to such obligations. Moreover, clinical trial participants

or research subjects about whom we or our vendors obtain information, as well as the providers who share this information

with us, may contractually limit our ability to use and disclose the information. We are also bound by other contractual

obligations related to data privacy and security, and our efforts to comply with such obligations may not be successful. We

may publish privacy policies, marketing materials, white papers, and other statements, such as statements relating to

compliance with certain certifications or self-regulatory principles concerning data privacy and security. Regulators in the

U.S. are increasingly scrutinizing these statements, and if these policies, materials or statements are found to be deficient,

lacking in transparency, deceptive, unfair, misleading or misrepresentative of our practices, we may be subject to

investigation, enforcement actions by regulators, or other adverse consequences.

It is possible that, in the future, we may fail or be perceived to have failed to comply with applicable data

privacy and security obligations. Moreover, despite our best compliance efforts, we may not be successful in achieving

compliance if our personnel or third parties with whom we work fail to comply with such obligations, which could

negatively impact our business operations and compliance posture. If we or the third parties with whom we work fail, or

are perceived to have failed, to address or comply with data privacy and security obligations, we could face significant

consequences. These consequences may include, but are not limited to, government enforcement actions; litigation;

additional reporting requirements and/or oversight; bans on processing personal data; orders to destroy or not use personal

data; and imprisonment of company officials. Any of these events could have a material adverse effect on our reputation,

business, or financial condition, including: interruptions or stoppages in our business operations including, as relevant,

clinical trials; inability to process personal data or to operate in certain jurisdictions; limited ability to continue to develop

or commercialize RYTELO; expenditure of time and resources to defend any claim or inquiry; adverse publicity; or

revision or restructuring of our operations.

RISKS RELATED TO OUR COMMON STOCK AND FINANCIAL REPORTING

Historically, our stock price has been extremely volatile and your investment may suffer a decline in value.

Historically, our stock price has been extremely volatile. Between January 1, 2015 and December 31, 2025,

our stock has traded as high as $6.38 per share and as low as $0.89 per share. Between January 1, 2025 and December 31,

2025, the price has ranged between a high of $3.60 per share and a low of $1.07 per share. The significant market price

fluctuations of our common stock have been due to and may in the future be influenced by a variety of factors, including:

• the level of and market opportunity for future sales of RYTELO;

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• announcements regarding regulatory approval or non-approval of RYTELO in any other

jurisdictions or indications, or specific label indications for RYTELO, or restrictions,

warnings or limitations in its use;

• announcements regarding the research and development of imetelstat, or adverse efficacy or

safety results of, further delays in the commencement, enrollment or conduct of,

discontinuation of, or further modifications or refinements to any current or potential future

clinical trials, or our inability to successfully continue the development of imetelstat;

• our ability to obtain additional capital, if and when needed, to further advance our

development program;

• changes in laws or regulations applicable to RYTELO, including laws or regulations

concerning the commercialization of RYTELO or clinical trial requirements for approval or

other regulatory developments related to RYTELO;

• adverse developments concerning our manufacturers, including our inability to obtain

adequate product supply for RYTELO or inability to do so at acceptable prices;

• the size and growth of the market opportunity for RYTELO in its currently approved and

any potential future approved indications;

• disputes or other developments relating to RYTELO proprietary rights, including patents,

litigation matters and our ability to obtain, enforce and defend patent protection and

maintain regulatory exclusivity for RYTELO and our technologies;

• the terms and timing of any future collaboration agreements for the further development

and commercialization of RYTELO that we may establish;

• announcements of significant acquisitions, strategic partnerships, collaborations, joint

ventures or capital commitments by us or our competitors;

• increased or continuing operating losses;

• general domestic and international market conditions or market conditions relating to the

biopharmaceutical and pharmaceutical industries, especially given the volatility caused by

macroeconomic or other global conditions,

• perceptions of the biotechnology and pharmaceutical industry by the public, legislature,

regulators and the investment community;

• our failure to meet the estimates and projections of the investment community or general

public;

• publication of commentary, articles or research reports about us or our industry, or positive

or negative recommendations or withdrawal of research coverage by securities analysts,

bloggers, news media or other third parties;

• actual or expected sales of common stock by stockholders;

• announcements of or developments concerning pending and potential future litigation;

• actions instituted by activist shareholders or others;

• other events or factors that are beyond our control; and

• the occurrence of any other risks and uncertainties discussed under the heading “Risk

Factors.”

In addition, as further discussed in the Risk Factor above titled “ We and certain of our current and former

officers and directors have been named as defendants in securities class action lawsuits and derivative lawsuits. These

lawsuits, and potential similar or related lawsuits, could result in substantial damages, divert management’s time and

attention from our business, and have a material adverse effect on our results of operations. These lawsuits, and any other

lawsuits to which we are subject, may be costly to defend or pursue and are uncertain in their outcome ,” we and certain of

our current and former officers and directors have been named as defendants in securities class action and derivative

lawsuits. Such lawsuits have often been instituted against companies, including us, whose securities have experienced

periods of volatility in market price. The pending lawsuits and any lawsuits brought against us in the future could result in

substantial costs and divert our management’s attention and resources, which could have a material adverse effect on our

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financial condition and business operations and lead to increased volatility in our stock price and a decrease in the value of

our stockholders’ investment in our securities.

Our failure to maintain compliance with the continued listing requirements of the Nasdaq Global Select Market may

result in our common stock being delisted from the Nasdaq Global Select Market, which could negatively impact the

price of our common stock, liquidity, our ability to access the capital markets and our stockholders’ ability to sell their

shares.

Our common stock is currently listed on the Nasdaq Global Select Market, or Nasdaq, under the symbol

“GERN.” The listing standards of Nasdaq provide that a company, in order to qualify for continued listing, must maintain a

minimum stock price of $1.00 and satisfy standards relative to minimum stockholders’ equity, minimum market value of

publicly held shares and various additional requirements. Historically, our stock price has been extremely volatile and

recently, our stock has traded as low as $0.89 per share through February 20, 2026. While our common stock is currently

listed on Nasdaq, we can give no assurance that we will be able to maintain compliance with the continued listing

requirements for Nasdaq. If we fail to maintain compliance with any such continued listing requirement, there can also be

no assurance that we will be able to regain compliance with any such continued listing requirement in the future or that our

common stock will not be delisted in the future. If Nasdaq delists our securities from trading on its exchange for failure to

meet the listing standards, we and our stockholders could face significant negative consequences including:

• limited availability of market quotations for our securities;

• a determination that the common stock is a “penny stock”, which would require brokers

trading in the common stock to adhere to more stringent rules, possibly resulting in a

reduced level of trading activity in the secondary trading market for shares of common

stock;

• a limited amount of analyst coverage, if any; and

• a decreased ability to issue additional securities or obtain additional financing in the future.

Delisting from Nasdaq could also result in other negative consequences, including the potential loss of

confidence by suppliers, customers and employees, the loss of institutional investor interest and fewer business

development opportunities.

Provisions in our charter, bylaws and Delaware law may inhibit potential acquisition bids for us, which may adversely

affect the market price of our common stock and/or prevent holders of our common stock from benefiting from what

they believe may be the positive aspects of acquisitions and takeovers.

Provisions of our charter documents and bylaws may make it substantially more difficult for a third party to

acquire control of us and may prevent changes in our management, including provisions that prevent stockholders from

taking actions by written consent, divide the board of directors into separate classes with terms of office that are structured

to prevent all of the directors from being elected in any one year and set forth procedures for nominating directors and

submitting proposals for consideration at stockholders’ meetings.

In addition, our certificate of incorporation provides our board of directors with the authority to issue up to

3,000,000 shares of undesignated preferred stock and to determine or alter the rights, preferences, privileges and

restrictions granted to or imported upon these shares without further vote or action by our stockholders. The issuance of

shares of preferred stock may delay or prevent a change in control transaction without further action by our stockholders.

As a result, the market price of our common stock may be adversely affected.

If in the future, we issue preferred stock that has preference over our common stock with respect to the

payment of dividends or upon our liquidation, dissolution or winding up, or if we issue preferred stock with voting rights

that dilute the voting power of our common stock, the rights of holders of our common stock or the market price of our

common stock could be adversely affected.

Provisions of Delaware law may also inhibit potential acquisition bids for us or prevent us from engaging

in business combinations. In addition, we have individual severance agreements with our executive officers and a

company-wide severance plan, either of which could require a potential acquirer to pay a higher price. Either collectively

or individually, these provisions may prevent holders of our common stock from benefiting from what they may believe are

the positive aspects of acquisitions and takeovers, including the potential realization of a higher rate of return on their

investment from these types of transactions.

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The exclusive forum provisions in our amended and restated bylaws could limit our stockholders’ ability to obtain a

favorable judicial forum for disputes with us or any of our directors, officers, or employees, or the underwriters of any

offering giving rise to such claim, which may discourage lawsuits with respect to such claims.

Our amended and restated bylaws provide that, unless we consent to the selection of an alternative forum,

the Court of Chancery of the State of Delaware (or, if and only if the Court of Chancery of the State of Delaware lacks

subject matter jurisdiction, any state court located within the State of Delaware or, if and only if all such state courts lack

subject matter jurisdiction, the federal district court for the District of Delaware) will be the sole and exclusive forum for:

• any derivative claim or cause of action or proceeding brought on our behalf;

• any claim or cause of action for breach of a fiduciary duty owed by any of our current or

former directors, officers or other employees, or our stockholders, to us or to our

stockholders;

• any claim or cause of action against us or any of our current or former directors, officers or

other employees, or our stockholders, arising pursuant to any provision of the General

Corporation Law of the State of Delaware, our certificate of incorporation, or our bylaws;

• any claim or cause of action seeking to interpret, apply, enforce or determine the validity of

our certificate of incorporation or bylaws;

• any claim or cause of action as to which the General Corporation Law of the State of

Delaware confers jurisdiction on the Court of Chancery of the State of Delaware; or

• any claim or cause of action against us or any of our current or former directors, officers or

other employees, or our stockholders, governed by the internal affairs doctrine or otherwise

related to our internal affairs.

In addition, Section 22 of the Securities Act creates concurrent jurisdiction for federal and state courts over

all claims brought to enforce any duty or liability created by the Securities Act of 1933, as amended, or the Securities Act,

or the rules and regulations thereunder. Our amended and restated bylaws provide that the federal district courts of the

United States of America will, to the fullest extent permitted by law, be the exclusive forum for resolving any complaint

asserting a cause of action arising under the Securities Act, or the Federal Forum Provision, including for all causes of

action asserted against any defendant named in such complaint. For the avoidance of doubt, this provision is intended to

benefit and may be enforced by us, our officers and directors, the underwriters to any offering giving rise to such

complaint, and any other professional entity whose profession gives authority to a statement made by that person or entity

and who has prepared or certified any part of the documents underlying the offering. The application of the Federal Forum

Provision means that suits brought by our stockholders to enforce any duty or liability created by the Securities Act must

be brought in federal court and cannot be brought in state court, and our stockholders cannot waive compliance with the

federal securities laws and the rules and regulations thereunder.

While the Delaware courts have determined that such choice of forum provisions are facially valid and

several state trial courts have enforced such provisions and required that suits asserting Securities Act claims be filed in

federal court, there is no guarantee that courts of appeal will affirm the enforceability of such provisions, and a stockholder

may nevertheless seek to bring a claim in a venue other than those designated in the exclusive forum provisions. In such an

instance, we would expect to vigorously assert the validity and enforceability of the exclusive forum provisions of our

amended and restated bylaws. This may require significant additional costs associated with resolving such action in other

jurisdictions, which costs could be borne by stockholders, and there can be no assurance that the provisions will be

enforced by a court in those other jurisdictions.

Any person or entity purchasing or otherwise acquiring or holding any interest in any of our securities shall

be deemed to have notice of and consented to the exclusive forum provisions in our amended and restated bylaws,

including the Federal Forum Provision. These provisions could limit a stockholder’s ability to bring a claim in a judicial

forum that it finds favorable for disputes with us or any of our directors, officers, or other employees, or our stockholders

or the underwriters of any offering giving rise to such claims, which may discourage lawsuits with respect to such claims.

Furthermore, if a court were to find the exclusive forum provisions contained in our bylaws to be inapplicable or

unenforceable in an action, we may incur additional costs associated with resolving such action in other jurisdictions,

which could have a material and adverse impact on our business and our financial condition.

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We do not intend to pay cash dividends on our common stock in the foreseeable future.

We do not anticipate paying cash dividends on our common stock in the foreseeable future. Any payment

of cash dividends will depend upon our financial condition, results of operations, capital requirements and other factors,

and will be at the discretion of our board of directors. In addition, the terms of our Pharmakon Loan Agreement restrict our

ability to pay dividends and any future debt agreements may continue to or further restrict our ability to pay dividends. As

a result, capital appreciation, if any, of our common stock will be the sole source of gain for our stockholders for the

foreseeable future.

Our employees, independent contractors, principal investigators, clinical trial sites, CROs, consultants or vendors may

engage in misconduct or other improper activities, including noncompliance with regulatory standards and

requirements.

We are exposed to the risk that our employees, independent contractors, principal investigators, clinical

trial sites, CROs, consultants or vendors may engage in fraudulent or other illegal activity. Misconduct by these parties

could include intentional, reckless and/or negligent conduct or disclosure of unauthorized activities to us that violate the

FDA’s or similar international regulatory authorities’ regulations, including those laws requiring the reporting of true,

complete and accurate information; manufacturing standards; healthcare fraud and abuse laws and regulations; or laws that

require the true, complete and accurate reporting of financial information or data. Specifically, sales, marketing and

business arrangements in the healthcare industry are subject to extensive laws and regulations intended to prevent fraud,

kickbacks, self-dealing and other abusive practices. These laws and regulations may restrict or prohibit a wide range of

pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business

arrangements.

Activities subject to these laws also involve the improper use or misrepresentation of information obtained

in the course of clinical trials or creating fraudulent data in our non-clinical studies or clinical trials, which could result in

regulatory sanctions and serious harm to our reputation. It is not always possible to identify and deter misconduct by our

employees and third parties, and the precautions we take to detect and prevent this activity may not be effective in

controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or

lawsuits stemming from a failure to be in compliance with such laws or regulations. Additionally, we are subject to the risk

that a person could allege such fraud or other misconduct, even if none occurred. If any such actions are instituted against

us, and we are not successful in defending ourselves or asserting our rights, those actions could adversely affect our

business, financial condition, results of operations or prospects through:

• t he imposition of civil, criminal and administrative penalties, damages and monetary fines;

• possible exclusion from participation in Medicare, Medicaid and other federal healthcare

programs;

• contractual damages;

• reputational harm;

• diminished potential profits and future earnings; and

• curtailment of our operations.

Failure to maintain effective internal controls in accordance with Section 404 of the Sarbanes-Oxley Act of 2002 could

have a material adverse effect on our business and stock price.

Section 404 of the Sarbanes-Oxley Act of 2002, or Section 404, requires that we establish and maintain an

adequate internal control structure and procedures for financial reporting. Our Annual Reports on Form 10-K must contain

an annual assessment by management of the effectiveness of our internal control over financial reporting and must include

disclosure of any material weaknesses in internal control over financial reporting that we have identified. In addition, our

independent registered public accounting firm must provide an opinion annually on the effectiveness of our internal control

over financial reporting. As described further below, we identified a material weakness in our internal control over

financial reporting that existed as of September 30, 2025, which we remediated as of December 31, 2025. However, in the

future, any testing by us conducted in connection with Section 404, or any testing by our independent registered public

accounting firm, may reveal additional deficiencies in our internal control over financial reporting that are deemed to be

material weaknesses or that may require prospective or retroactive changes to our financial statements or identify other

areas for further attention or improvement.

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The requirements of Section 404 are ongoing and also apply to future years. We expect that our internal

control over financial reporting will continue to evolve as our business develops, including in connection with our

commercialization of RYTELO. Although we are committed to continue to improve our internal control processes and we

will continue to review our internal control over financial reporting, any control system, regardless of how well designed,

operated and evaluated, can provide only reasonable, not absolute, assurance that its objectives will be met. .

Therefore, we cannot assure you that additional material weaknesses or significant deficiencies will not

exist or otherwise be discovered in the future, particularly in light of our increased reliance on personnel working remotely.

If material weaknesses or other significant deficiencies continue to occur, such weaknesses or deficiencies could result in

misstatements of our results of operations, restatements of our financial statements, a decline in our stock price, or other

material adverse effects on our business, reputation, results of operations, financial condition or liquidity.

We have previously identified a material weakness in our internal control over financial reporting and may identify

additional material weaknesses in the future or otherwise fail to maintain an effective system of internal controls, which

may result in material misstatements of our consolidated financial statements or cause us to fail to meet our periodic

reporting obligations.

A material weakness is a deficiency, or a combination of deficiencies, in internal control over financial

reporting, such that there is a reasonable possibility that a material misstatement of a company’s annual or interim financial

statements will not be prevented or detected on a timely basis. We previously identified a material weakness in internal

control related to the design and operation of certain Information Technology General Controls, or ITGCs, related to user

access and program change management for our Enterprise Resource Planning and payment processing systems.

Consequently, IT application controls and IT dependent manual business process controls that rely upon information from

these systems, were also deemed ineffective. Although the material weakness identified did not result in any material

misstatements in our consolidated financial statements for the periods presented in this Report and there were otherwise no

changes to our previously issued financial statements, our management concluded that these control deficiencies existed as

of September 30, 2025 and constitute a material weakness. Accordingly, our internal control over financial reporting and

our disclosure controls and procedures were not effective as of September 30, 2025. While we subsequently remediated

this material weakness as of December 31, 2025, we cannot assure you that there will not be material weaknesses or

significant deficiencies in our internal control over financial reporting in the future. Our failure to implement and maintain

effective internal control over financial reporting could result in errors in our consolidated financial statements that could

result in a restatement of our previously issued financial statements and could cause us to fail to meet our periodic reporting

obligations, any of which could diminish investor confidence in us and cause a decline in the price of our common stock

Changes in tax laws or regulations that are applied adversely to us or our customers may have a material adverse effect

on our business, cash flow, financial condition or results of operations.

New income, sales, use, excise or other tax laws, statutes, rules, regulations or ordinances could be enacted

at any time, which could affect the tax treatment of our domestic and foreign sales and earnings. Any new taxes could

adversely affect our domestic and international business operations and our business and financial condition. Further,

existing tax laws, statutes, rules, regulations or ordinances could be interpreted, changed, modified or applied adversely to

us. For example, the Inflation Reduction Act of 2022 included provisions that impacted the U.S. federal income taxation of

corporations, including imposing a minimum tax on the book income of certain large corporations and an excise tax on

certain corporate stock repurchases that is imposed on the corporation repurchasing such stock. In addition, the U.S.

government recently enacted legislation commonly referred to as the One Big Beautiful Bill Act, that (along with other

recent U.S. federal tax reform) has resulted in significant changes to the taxation of business entities including, among

other changes, changes to the taxation of income derived from international operations, changes in the deduction and

amortization of research and development expenditures, and limitations on the deductibility of business interest. Future

guidance from the Internal Revenue Service and other tax authorities with respect to any legislation may affect us, and

certain aspects of such legislation could be repealed or modified or sunset in future years. Changes in tax reform legislation

could have a material impact on the value of our deferred tax assets, could result in significant one-time charges, and could

increase our future United States tax expense.

Our ability to use our net operating loss carryforwards and certain other tax attributes may be limited.

Our net operating loss carryforwards attributable to tax years beginning before January 1, 2018 could

expire unused and be unavailable to offset future income tax liabilities. In addition, federal net operating losses incurred in

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taxable years beginning after December 31, 2017, can be carried forward indefinitely, but the deductibility of such federal

net operating losses in a taxable year is limited to 80% of taxable income in such year. Under Sections 382 and 383 of the

Internal Revenue Code of 1986, as amended, and corresponding provisions of state law, if a corporation undergoes an

“ownership change,” generally defined as a greater than 50 percentage point cumulative change (by value) in its equity

ownership over a three-year period, the corporation’s ability to use its pre-change net operating loss carryforwards and

other pre-change tax attributes (such as research and development tax credits) to offset its post-change taxable income or

taxes may be limited. Changes in our stock ownership have occurred in the past, and future ownership changes, some of

which may be outside our control, could occur in the future, as a result of shifts in our stock ownership. If a limitation were

to apply, utilization of a portion of our domestic net operating loss and tax credit carryforwards could be limited in future

periods, and a portion of the carryforwards may expire before being available to reduce future income tax liabilities, which

could adversely impact our financial position. At the state level, there may be periods during which the use of net operating

loss carryforwards is suspended or otherwise limited, which could accelerate or permanently increase state taxes owed. For

example, in June 2024, California enacted legislation that, with certain exceptions, suspends the use of California net

operating losses to offset California income and limits the use of California business tax credits to offset California taxes,

for taxable years beginning after 2023 and before 2027.

ITEM 1B. UNRESOLVED STAFF COMMENTS

None.

ITEM 1C. CYBERSECURITY

Risk management and strategy

We operate in the biopharmaceutical sector, which is a highly regulated sector subject to various

cybersecurity risks that could adversely affect our business, financial condition, and results of operations, including

intellectual property theft; fraud; extortion; harm to employees or customers; disruption of our clinical trials, manufacturing

or supply chain; violation of privacy laws and other litigation and legal risk; and reputational risk. We rely primarily on

industry-leading third parties and a cloud-based infrastructure for our information technology systems, and accordingly are

dependent on these third parties’ own cybersecurity risk management practices and strategy. We have implemented and

maintain various information security processes designed to identify, assess and manage material risks from cybersecurity

threats to our critical computer networks, third party hosted services, communications systems, hardware and software, and

our critical data, including clinical trial data, intellectual property, confidential information that is proprietary, strategic,

financial or competitive in nature, and personal data (“Information Systems and Data”).

We take a risk-based approach to identify and assess the cybersecurity threats and risks that could affect our

business and Information Systems and Data. Our Information Technology personnel help identify, assess and manage our

cybersecurity threats and risks, and support our efforts to identify and assess risks from cybersecurity threats by monitoring

and evaluating our threat environment. We use various methods and tools to identify, assess and manage cybersecurity

threats and risks, including, for example, automated tools, a third-party managed detection and response firm to monitor

security alerts on a 24-hour basis, industry reports, engaging a virtual Chief Information Security Officer, third party threat

assessments and penetration testing. In addition, we encrypt data at rest and maintain network security controls, such as

firewalls and virtual private networks. We also conduct computerized system monitoring and access control, including

asset management, tracking and disposal associated with onboarding and offboarding of personnel. We maintain

cybersecurity insurance.

Depending on the environment, we implement and maintain various technical, physical, and organizational

measures, processes, standards and policies designed to manage and mitigate material risks from cybersecurity threats to

our Information Systems and Data. For example, in 2025, we expanded our monitoring capabilities by engaging with a

managed detection and response firm to monitor and respond to security alerts on a 24-hour, seven-day-a-week basis. We

have implemented and maintain an incident response plan, and we utilize automated tools designed to maintain email

security. We have also implemented a computerized system security and password policy that defines security for access to

computer systems managed and controlled by us, and a procedure for computerized system incident management to

address any unplanned issues in regulated computerized systems that could impact subject safety, product quality, and data

integrity. We further protect access by monitoring risk-based sign-in attempts, and require stronger authentication (e.g.,

multi-factor authentication) based on defined policies. We periodically conduct cybersecurity incident tabletop training

exercises involving our personnel and plan to continue conducting similar training exercises in 2026.

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Our assessment and management of material risks from cybersecurity threats are integrated into our overall

risk management processes. For example, our head of Information Technology evaluates material risks from cybersecurity

threats and reports periodically to the Audit Committee of our Board, which evaluates our overall enterprise risk. We use

third-party service providers to assist us from time to time to identify, assess, and manage material risks from cybersecurity

threats, including, for example, cybersecurity software providers such as our managed detection and response firm,

cybersecurity service providers, penetration testing firms, auditors, and professional services firms, such as our virtual

Chief Information Security Officer, including legal counsel. These relationships enable us to leverage specialized

knowledge and insights, enabling our cybersecurity strategies and processes to remain consistent with industry best

practices.

We rely on third-party service providers to perform a variety of functions throughout our business, such as

contract manufacturing organizations, contract research organizations, suppliers and consultants, and third party logistics

organizations and distributors to distribute RYTELO. We conduct quality and cyber audits of regulated vendors, which

typically include an assessment of such vendor’s information technology systems, checks for recent cybersecurity

incidents, analyzing potential vulnerabilities associated with such vendor's externally facing infrastructure, and the

cybersecurity controls they have in place to protect the Company's data, and we impose appropriate contractual obligations

on vendors pertaining to information security. Depending on the nature of the services provided, the sensitivity of the

Information Systems and Data at issue, and the identity of the provider, our efforts may involve different levels of

assessment designed to help identify cybersecurity risks associated with a provider and impose contractual obligations

related to cybersecurity on the provider.

For a description of the risks from cybersecurity threats that may materially affect us and how they may do

so, see our risk factors under Part 1. Item 1A. Risk Factors in this Report, including “ Risks Related to Information

Technology Systems, Data Security and Data Privacy .”

Governance

Our Board of Directors addresses our cybersecurity risk management as part of its general oversight

function. The Audit Committee of our Board is responsible for overseeing our cybersecurity risk management processes,

including oversight and mitigation of risks from cybersecurity threats.

Our Audit Committee, as well as our Chief Financial Officer and other members of our executive

management as appropriate, receives periodic reports from our head of Information Technology concerning our significant

cybersecurity threats and risks and the processes we have implemented to address them. The Audit Committee also

receives various periodic presentations related to cybersecurity threats, risk and mitigation.

Risk Management Personnel

Our Information Technology personnel responsible for cybersecurity risk assessment and management

processes are managed by certain members of our executive management, including our Chief Financial Officer. Together

with our executive management, our Information Technology personnel are responsible for helping to integrate

cybersecurity risk considerations into our overall risk management strategy, and communicating key priorities to relevant

personnel. We believe these Information Technology personnel have the skills appropriate to help us prepare for

cybersecurity incidents, approve cybersecurity processes, and review security assessments and other security-related

reports.

Our cybersecurity incident response plan is designed to escalate certain cybersecurity incidents to members

of management depending on the circumstances, including executive management. When appropriate given the nature of

any potential cybersecurity incident, our executive management works with our incident response team to help us mitigate

and remediate cybersecurity incidents of which they are notified, and to make any legally required notifications to

individuals or regulatory agencies, including making any required disclosures under the Exchange Act.

ITEM 2. PROPERTIES

In April 2019, we entered into an operating lease agreement for office space located at 3 Sylvan Way,

Parsippany, New Jersey, or the New Jersey Lease. The initial term of the New Jersey Lease is 11 years with an option to

extend for an additional five years and a one-time option to terminate the New Jersey Lease without cause as of the 103 rd

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month anniversary of the commencement date of the lease. The New Jersey Lease commenced on October 1, 2019, upon

our control of the office space on that date.

In October 2019, we entered into an operating lease agreement for office space located at 919 East

Hillsdale Boulevard, Foster City, California, or the Foster City Lease. The initial term of the Foster City Lease is 87

months with an option to extend for an additional five years. The Foster City Lease commenced on March 10, 2020, upon

our control of the office space on that date.

ITEM 3. LEGAL PROCEEDINGS

The information required to be set forth under this Item 3 is incorporated by reference to Note 8,

Commitments and Contingencies, of the Notes to Consolidated Financial Statements, included in Part II, Item 8 of this

Report.

ITEM 4. MINE SAFETY DISCLOSURES

Not applicable.

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PART II

ITEM 5. MARKET FOR THE REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS

AND ISSUER PURCHASES OF EQUITY SECURITIES

Market Information

Our common stock is listed on the Nasdaq Global Select Market under the symbol GERN. As of

February 20, 2026 , there were approximately 411 stockholders of record of our common stock. This number does not

include “street name” or beneficial holders, whose shares are held of record by banks, brokers and other financial

institutions.

Stock Performance Graph

The following graph shows a comparison from December 31, 2020 through December 31, 2025 , of the

cumulative total return on an assumed investment of $100.00 in cash in our common stock as compared to the same

investment in the Nasdaq Composite Index and the Nasdaq Biotechnology Index. Such returns are based on historical

results and are not intended to suggest future performance. Data for the Nasdaq Composite Index and Nasdaq

Biotechnology Index assume reinvestment of dividends.

This performance graph shall not be deemed “soliciting material” or to be “filed” with the SEC for

purposes of Section 18 of the Exchange Act or incorporated by reference into any filing of Geron Corporation under the

Securities Act or the Exchange Act, except to the extent we specifically incorporate it by reference into such filing.

Dividend Policy

We have never paid cash dividends on our capital stock and do not anticipate paying cash dividends in the

foreseeable future, but intend to retain our capital resources for reinvestment in our business. In addition, the terms of our

Pharmakon Loan Agreement restrict our ability to pay dividends and any future debt agreements may continue to or further

restrict our ability to pay dividends. Any future determination to pay cash dividends will be at the discretion of the board of

directors and will be dependent upon our financial condition, results of operations, capital requirements, compliance with

the terms of our Pharmakon Loan Agreement or other future debt agreements, and other factors our board of directors

deems relevant.

Recent Sales of Unregistered Securities

During the year ended December 31, 2025 , there were no unregistered sales of equity securities by us.

ITEM 6. [RESERVED]

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ITEM 7. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF

OPERATIONS

The following discussion should be read in conjunction with the section entitled “Business” in Part I, Item

1 and the audited financial statements and notes thereto included in Part II, Item 8 of this Report. The information provided

should be reviewed in the context of the sections entitled “Risks Related to the Further Development of RYTELO

(Imetelstat),” “Risks Related to the Commercialization of RYTELO” and “Risks Related to Regulatory Approval of

RYTELO” in Part II, Item 1A entitled “Risk Factors” and elsewhere in this Report.

Company Overview

Summary

We are a commercial-stage biopharmaceutical company aiming to change lives by changing the course of

blood cancer. Our first-in-class telomerase inhibitor, RYTELO ® (imetelstat), harnesses Nobel Prize winning science in a

treatment that scientific evidence suggests reduces proliferation of malignant cells, allowing production of new healthy

cells, which we believe drives differentiated clinical benefits, potentially altering the underlying course and modifying the

disease of these hematologic malignancies.

We commercially launched RYTELO in the U.S. in June 2024 following its approval by the U.S. Food and

Drug Administration, or FDA on June 6, 2024 for the treatment of adult patients with low- to intermediate-1 risk

myelodysplastic syndromes, or lower-risk MDS, with transfusion-dependent, or TD, anemia requiring four or more red

blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-

stimulating agents, or ESAs. Lower-risk MDS is a progressive blood cancer with high unmet need, where many patients

with anemia become dependent on red blood cell transfusions, which can be associated with clinical consequences and

decreased quality of life. We believe that the high unmet need in lower-risk MDS and significant product differentiation,

including observed benefit of RYTELO in difficult-to-treat sub-populations such as patients with high transfusion burden

and ring sideroblast negative, or RS- patients, as well as the favorable FDA label and the National Comprehensive Cancer

Network, or NCCN ® , Clinical Practice Guidelines in Oncology, or NCCN Guidelines ® , position RYTELO to potentially

compete for significant market segments in lower-risk MDS.

In March 2025, we were granted marketing authorization by the European Commission, or EC, for

RYTELO as a monotherapy for the treatment of adult patients with TD anemia due to very low, low or intermediate risk

myelodysplastic syndromes without an isolated deletion 5q cytogenetic, or non-del 5q, abnormality and who had an

unsatisfactory response to or are ineligible for erythropoietin-based therapy . We are preparing for the planned

commercialization of RYTELO in select EU markets in 2026. At this time, we do not plan to commercialize RYTELO

independently in the EU (or in any other regions outside of the U.S. where RYTELO may be approved for marketing in the

future). Accordingly, we plan to work with experienced third parties for the commercialization and marketing of RYTELO

in the EU, including on critical path activities for the planned launch of RYTELO in the EU, such as reimbursement,

Health Technology Assessment, or HTA, submissions, market access and distribution. To enable paid access to patients

outside the U.S. through approved Named Patient Programs, or NPPs, in 2025 we partnered with Tanner Pharma, a

distributor with broad global reach to support patient access. To date, product revenue pursuant to NPPs have been

minimal.

In addition to lower-risk MDS, we are developing imetelstat for the treatment of other myeloid hematologic

malignancies. Our Phase 3 IMpactMF clinical trial is evaluating imetelstat in patients with intermediate-2 or high-risk

myelofibrosis, or MF, who have relapsed after or are refractory to treatment with a janus associate kinase inhibitor, or JAK

inhibitor, or relapsed/refractory MF, or R/R MF, with overall survival, or OS, as the primary endpoint. As of September

2025, the trial was fully enrolled. Based on our current planning assumptions for event (death) rates in the trial, we expect

the interim analysis for OS in IMpactMF may occur in the second half of 2026 and the final analysis may occur in the

second half of 2028.

We believe that telomerase inhibition with imetelstat represents a novel mechanism of action with unique

benefits in hematologic malignancies and potentially in other tumor types.

Financial Overview

Since our inception, we have financed our operations primarily through the sale of equity securities, draw

downs on our debt facilities, cash generated from sales of RYTELO, interest income on our marketable securities,

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payments we received under the Royalty Pharma Agreement and our prior collaborative and licensing arrangements. As of

December 31, 2025 , we had approximately $401.1 million in cash, cash equivalents, restricted cash and marketable

securities.

We began commercializing RYTELO in June 2024, and the commercial potential of and our ability to

successfully commercialize RYTELO remains unproven. Our success in commercializing RYTELO will require, among

other things, effective sales, marketing, manufacturing, distribution, information systems and pricing strategies, as well as

compliance with applicable laws and regulations. Prior to our commercialization of RYTELO, substantially all of our

revenues were generated from payments under prior collaboration agreements, and milestones, royalties and other revenues

from our licensing arrangements. We reported a small profit for the year ended December 31, 2015, and we have not

reported any profit since. We have incurred significant net losses since our inception in 1990, resulting principally from

costs incurred in connection with our research and development activities and from general and administrative costs

associated with our operations. As of December 31, 2025 , we had an accumulated deficit of approximately $1.9 billion.

On November 1, 2024, we entered into a loan agreement, or the Pharmakon Loan Agreement, with

BioPharma Credit Investments V (Master) LP and BPCR Limited Partnership, each, a Lender, which are investment funds

managed by Pharmakon Advisors, LP, and BioPharma Credit PLC, as collateral agent, that provides for a 5-year senior

secured term loan facility of up to $250.0 million, divided into three committed tranches : (i) a Tranche A Loan in an

aggregate principal amount of $125.0 million, or the Tranche A Loan, which was funded on November 1, 2024, or the

Tranche A Closing Date; (ii) a Tranche B Loan in an aggregate principal amount of $75.0 million, or the Tranche B Loan,

which is available, subject to certain limited conditions, at our option; and (iii) a Tranche C Loan in an aggregate principal

amount of $50.0 million, or the Tranche C Loan, and together with the Tranche A Loan and the Tranche B Loan,

collectively, the Term Loans, which is available to us upon reaching a specified trailing twelve-month RYTELO revenue

milestone. The Tranche B Loan and the Tranche C Loan, once available, could have been requested on or prior to

December 31, 2025. A portion of the proceeds from the Tranche A Loan were used to repay, in full, all amounts owed

($86.5 million) under the Hercules Loan Agreement, which was terminated effective November 1, 2024. The Term Loans

mature on November 1, 2029. The Term Loans bear interest at a variable rate per annum equal to 5.75% plus the three-

month Secured Overnight Financing Rate, or SOFR, with a SOFR floor of 3.00%. See Note 10 on Debt in Notes to

Consolidated Financial Statements of this Report for additional information on the Pharmakon Loan Agreement.

On January 5, 2026, the Pharmakon Loan Agreement was amended to extend the date for requesting the

Tranche B Loan and Tranche C Loan from December 31, 2025 to July 30, 2026. We may elect to prepay the Term Loans

in part or in whole prior to the Maturity Date with such prepayments being subject to a prepayment premium equal to the

principal amount so prepaid multiplied by 3% if made prior to the 3rd anniversary of the funding date of the applicable

Term Loan, 2% if made on or after the third anniversary of the funding date of the applicable Term Loan but prior to the

fourth anniversary of the funding date of the applicable Term Loan, and 1% if made on or after the fourth anniversary of

the funding date of the applicable Term Loan but prior to the Maturity Date. In addition to the prepayment premium,

prepayments of any Term Loan prior to a specified date, or the Makewhole Date, are subject to a makewhole amount equal

to the sum of all interest that would have accrued from the date of such payment through such Makewhole Date. The First

Amendment Agreement also extended the Makewhole Date from November 1, 2026 to May 1, 2027.

On November 1, 2024, we entered into a revenue participation right purchase and sale agreement, or the

Royalty Pharma Agreement, with Royalty Pharma Development Funding, LLC, or Royalty Pharma. Pursuant to the

Royalty Pharma Agreement, we received an upfront payment of $125.0 million, or the Purchase Price, in exchange for

which Royalty Pharma obtained the right to receive tiered royalty payments with respect to annual U.S. net sales, or

Annual Net Sales, of RYTELO beginning on July 1, 2024, ranging from: (i) 7.75% of Annual Net Sales up to $500.0

million; (ii) 3.0% of Annual Net Sales in excess of $500.0 million but less than or equal to $1.0 billion; and (iii) 1.0% in

respect of Annual Net Sales in excess of $1.0 billion, or the Royalty Payments. The Royalty Payments to Royalty Pharma

are capped, such that they will cease upon reaching a multiple of 1.65 times the Purchase Price if Royalty Pharma receives

Royalty Payments in that amount in respect of net sales occurring on or before June 30, 2031, or upon reaching a multiple

of 2.0 times the Purchase Price thereafter. Our Royalty Payment obligations under the Royalty Pharma Agreement may be

discharged in connection with a change of control of Geron in an amount equal to 1.65 times the Purchase Price minus the

aggregate Royalty Payments received by Royalty Pharma as of the date of the closing of the change of control, if the

closing of the change of control occurs on or prior to December 31, 2027, or in an amount equal to 2.0 times the Purchase

Price minus the aggregate Royalty Payments received by Royalty Pharma as of the date of the closing of the change of

control, if the closing of the change of control occurs after December 31, 2027. There are no other royalties payable on

RYTELO, which was developed internally and is exclusively owned by Geron.

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In December 2025, we implemented a workforce reduction, representing approximately one-third of our

workforce prior to the reduction in headcount. We may not realize, in full or in part, the anticipated benefits on our 2026

operating expenses from our restructuring efforts due to unforeseen difficulties, delays or unexpected costs. We incurred

approximately $17.0 million in restructuring and restructuring-related charges in the fourth quarter of 2025, primarily

consisting of one-time employee severance payments, healthcare and related benefits, and other employee-related costs,

and we estimate the workforce reduction will be substantially completed in the first quarter of 2026. If we are unable to

realize the expected operational efficiencies and cost savings from the restructuring, our operating results and financial

condition could be adversely affected.

The significance of future losses, future revenues and any potential future profitability will depend

primarily on the clinical and commercial success of RYTELO, our sole product. In this regard, our ability to generate

meaningful revenue from product sales and achieve profitability is wholly dependent on our ability to successfully

commercialize RYTELO in the U.S. for lower-risk MDS or to expand its indications of use. We have seen and may

continue to see variability in RYTELO sales trends.

Our commercial strategy is designed to ensure that RYTELO reaches eligible patients when they are most

likely to benefit. Our commercial execution is focused on targeted engagement with high-volume accounts that treat

earlier-line patients, investment in non-personal promotion and third-party education to further consistent, high-quality

messaging across multiple touchpoints, and cross-functional execution of effective account management. However, our

strategy to drive sales growth and our ongoing commercialization efforts has not to date achieved and may not in the future

achieve meaningful sales growth, which may require us to, among others, further adjust or amend our commercialization

strategy and plans and incur significant expenses, and there can be no assurance that we will be able to grow RYTELO net

product revenue in future periods. In particular, our strategy may not drive new patient starts across the breadth of the

eligible patient population in RYTELO's approved indication in a timely manner or at all, or the duration of therapy could

be shorter than we expect, each of which would limit RYTELO’s growth potential and could preclude or delay our ability

to generate meaningful revenue from product sales and to achieve profitability.

In addition, in an effort to expand its indications of use, we are also developing RYTELO for the treatment

of several myeloid hematologic malignancies that will continue to require additional time and significant investment in

clinical trials to complete. We also expect to continue to seek regulatory approvals of RYTELO in jurisdictions outside of

the U.S., such as our recent marketing authorization in the EU, and to establish arrangements with third parties to assist us

in the commercialization of RYTELO in such jurisdictions. As a result, we expect research and development expenses and

selling, general and administrative expenses to continue to be substantial as we continue to support the commercialization

of RYTELO in the U.S. and further development of RYTELO, including the conduct and completion of our IMpactMF

Phase 3 clinical trial, as well as our ongoing Phase 1 IMproveMF combination clinical trial in frontline MF, our Phase 2

investigator-led IMpress clinical trial in higher-risk MDS and acute myeloid leukemia, and our Phase 1/2 investigator-led

IMAGINE clinical trial in relapsed/refractory acute myeloid leukemia, and as we pursue paths to make RYTELO available

to eligible LR-MDS patients outside of the U.S., including in the EU. In addition, we expect our interest expense to

increase due to the draw down of the Tranche A Loan and potential future draw downs of the other Term Loans under the

Pharmakon Loan Agreement, if available, as well as the non-cash interest expense related to the Royalty Pharma

Agreement.

Critical Accounting Policies and Estimates

Our consolidated financial statements have been prepared in accordance with accounting principles

generally accepted in the United States of America. The preparation of our consolidated financial statements requires

management to make estimates and assumptions that affect the reported amounts of assets, liabilities, revenues and

expenses. Our estimates are based on our historical experience and on various other factors that we believe are reasonable

under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and

liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different

assumptions and conditions.

A critical accounting policy is one that is both important to the portrayal of our financial condition and

results of operations and requires management’s most difficult, subjective or complex judgments, often as a result of the

need to make estimates about the effect of matters that are inherently uncertain. While Note 1 of Notes to Consolidated

Financial Statements of this Report describes the significant accounting policies used in the preparation of our consolidated

financial statements, we believe the following accounting estimates and policies to be critical.

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Revenue Recognition

We recognize revenue when our customer obtains control of promised goods or services, in an amount that

reflects the consideration which we expect to receive in exchange for those goods or services. We recognize revenue

following the five-step model prescribed under Accounting Standards Codification Topic 606, Revenue from Contracts

with Customers: (i) identify contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii)

determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v)

recognize revenue when (or as) we satisfy the performance obligations.

Product Revenue

We sell our product s primarily to third party distributors and specialty pharmacies. These customers subsequently resell our

products to health care providers and patients. In addition, we enter into arrangements with health care providers and

payors that provide for government-mandated or privately-negotiated discounts and allowances related to our RYTELO.

Product revenue is recognized when the customer obtains control of our product, which occurs at a point in

time, typically upon delivery to the customer.

Reserves for Discounts and Allowances

Product revenue is recorded net of reserves established for applicable discounts and allowances that are

offered within contracts with our customers, health care providers or payors, Product revenue reserves, which are classified

as a reduction in product revenue, are generally characterized in the following categories: discounts, contractual

adjustments and returns.

These reserves are based on estimates of the amounts earned or to be claimed on the related sales and are

classified as reductions of accounts receivable (if the amount is payable to our customer) or a liability (if the amount is

payable to a party other than our customer). Our estimates of reserves established for variable consideration are calculated

based upon a consistent application of our methodology utilizing the expected value method. These estimates reflect our

historical experience, current contractual and statutory requirements, specific known market events and trends, industry

data and forecasted customer buying and payment patterns. The transaction price, which includes variable consideration

reflecting the impact of discounts and allowances, may be subject to constraint and is included in the net sales price only to

the extent that it is probable that a significant reversal of the amount of the cumulative revenue recognized will not occur in

a future period. Actual amounts may ultimately differ from our estimates. If actual results vary, we adjust these estimates,

which could have an effect on earnings in the period of adjustment.

For additional information on our revenue, please read Note 2 on Product Revenue to our consolidated

financial statements included in this report.

Inventory

Inventory is recorded at the lower of cost or net realizable value, with cost determined under the weighted

average method. Inventory costs include third-party contract manufacturing, third-party packaging services, freight,

salaries, wages and stock-based compensation for personnel involved in the manufacturing process, and indirect overhead

costs. We periodically review our inventories to identify excess, obsolete, or slow moving items. If excess, obsolete, or

slow moving inventory without an alternate use is identified, we adjust the recorded amount to its net realizable value. The

determination of net realizable value requires judgment including consideration of many factors, such as estimates of future

product demand, product net selling prices, current and future market conditions and potential product obsolescence,

among others. Additionally, our products are subject to strict quality control and monitoring that we perform throughout

the manufacturing process. In the event that certain batches or units of product no longer meet quality specifications, we

will record a charge to cost of sales to write-down any unmarketable inventory to its estimated net realizable value. In all

cases, product inventory is carried at the lower of cost or its estimated net realizable value.

Although we believe that the assumptions we use in estimating inventory write-downs are reasonable, no

assurance can be given that significant future changes in these assumptions or changes in future events and market

conditions could result in different estimates.

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Royalty Pharma Agreement Interest Expense

The liability related to the Royalty Payments under the Royalty Pharma Agreement and the related revenue

interest expense are measured based on our current estimate of the timing and amount of expected future Royalty Payments

expected to be paid over the estimated term of the Royalty Pharma Agreement using a discounted cash flow model. The

liability is amortized using the effective interest rate method, resulting in recognition of non-cash interest expense over the

estimated term of the agreement. Each reporting period, we assess the estimated timing and amount of future expected

Royalty Payments over the estimated term. If there are changes to the estimate, we recognize the impact to the liability’s

amortization schedule and the related non-cash interest expense prospectively. Additionally, the transaction costs

associated with the liability is amortized to non-cash interest expense over the estimated term of the Royalty Pharma

Agreement.

Results of Operations

Our results of operations have fluctuated from period to period and may continue to fluctuate in the future.

Results of operations for any period may be unrelated to results of operations for any other period. Thus, historical results

should not be viewed as indicative of future operating results. In this regard, although we have begun to recognize revenue

from RYTELO product sales in the U.S., we are early in our commercialization efforts and our related strategy to drive

sales growth in the U.S.. We expect that our sales revenue may continue to vary from period to period as a result of the

evolving effects of our commercialization strategy and as our commercialization efforts otherwise progress.

RYTELO is our only product approved for marketing in the U.S. and in the EU for certain patients with

lower-risk MDS. Revenue based on sales of RYTELO is dependent on our ability to successfully commercialize RYTELO

in the U.S. and in the EU and to obtain regulatory approvals to commercialize RYTELO in other jurisdictions and in other

indications. We are subject to risks common to companies in our industry and at our stage of development, including, but

not limited to, risks inherent in the development, manufacture, regulatory approval for and commercialization of RYTELO;

uncertainty of non-clinical and clinical trial results or regulatory approvals or clearances; the future development of

imetelstat by us and its use by patients generally, including any future efficacy or safety results from clinical or commercial

use that may cause the benefit-risk profile of imetelstat to become unacceptable; the uncertain and unpredictable drug

research and development process; our ability to obtain and maintain contractual arrangements, strategic partnerships,

collaborations, alliances or licensing arrangements with third parties to assist us with the commercialization of RYTELO in

jurisdictions outside of the U.S.; overcoming disruptions and/or delays due to macroeconomic or other global conditions,

such as further changes in tariffs and other trade restrictions, renegotiation of existing international trade agreements or

further escalation of trade tensions, inflation, fluctuations in interest rates, prospects of a recession, government shutdowns,

bank failures and other disruptions to financial systems, civil or political unrest, military conflicts, pandemics or other

health crises and supply chain and resource issues; our ability to obtain additional capital if and when needed; enforcement

of our patent and proprietary rights; reliance upon our CROs, contract manufacturing organizations, or CMOs, consultants,

licensees, investigators and other third parties; and potential competition.

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Comparison of the Years Ended December 31, 2025 , 2024 , and 2023

The following table sets forth our results of operations for the years ended December 31:

	2025	2024	Change $	Change %	2023	Change $	Change %
	(in thousands, except for percentage data)
Revenues:
Product revenue, net	$ 183,623	$ 76,495	$ 107,128	58%	$ —	$ 76,495	100%
Royalties	258	499	(241)	(48%)	237	262	111%
Total revenues	183,881	76,994	106,887	139%	237	76,757	324%
Costs and operating expenses:
Cost of goods sold	4,745	1,256	3,489	74%	—	1,256	100%
Research and development	71,433	103,738	(32,305)	(31%)	125,046	(21,308)	(17%)
Selling, general and administrative	159,256	145,732	13,524	9%	69,135	76,597	111%
Restructuring charges	17,032	—	—	100%	—	—	0%
Total costs and operating expenses	252,466	250,726	1,740	1%	194,181	56,545	29%
Interest income	18,117	19,607	(1,490)	(8%)	18,152	1,455	8%
Interest expense	(32,657)	(18,504)	(14,153)	76%	(8,312)	(10,192)	123%
Other income (expense), net	(375)	(236)	(139)	59%	(23)	(213)	926%
Loss on extinguishment of debt	—	(1,707)	1,707	(100%)	—	8,485	100%
Net income (loss)	$ (83,500)	$ (174,572)	$ 91,072	(52%)	$ (184,127)	$ 9,555	(5%)

Revenues

Product Revenue, Net

On June 6, 2024, we announced that the FDA approved RYTELO for the treatment of adult patients with

lower-risk MDS, with TD anemia requiring four or more red blood cell units over eight weeks who have not responded to

or have lost response to or are ineligible for ESA. To date, our only source of product revenue has been from the sales of

RYTELO, which we began shipping to our customers in June 2024. We did not generate any revenue from product sales

prior to June 2024. Total product revenue, net for the twelve months ended December 31, 2025 and 2024 was

approximately $183.6 million and $76.5 million, respectively.

Total gross-to-net adjustments for the twelve months ended December 31, 2025 and 2024 were 17.7% and

14.5% of gross product revenue, respectively. We expect total gross-to-net adjustments to be in the percentage range of

high-teen to low-twenties of gross product revenue in 2026.

The reconciliation of gross product revenue to product revenue, net by each significant category of gross-

to-net adjustments is as set forth below.

(in thousands)	Year Ended December 31, — 2025	2024
Gross product revenue	$ 223,112	$ 89,418
Gross-to-net adjustments:
Chargebacks	(25,525)	(8,724)
Distributor service fees	(7,512)	(3,048)
Government rebates	(2,603)	(557)
Sales returns and allowances	(3,849)	(594)
Total gross-to-net adjustments	$ (39,489)	$ (12,923)
Product revenue, net	$ 183,623	$ 76,495

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Royalties

In connection with the divestiture of our human embryonic stem cell assets, including intellectual property

and proprietary technology, to Lineage Cell Therapeutics, Inc. (formerly BioTime, Inc. which acquired Asterias

Biotherapeutics, Inc.), or Lineage, in 2013, we are entitled to receive royalties on sales from certain research or commercial

products utilizing our divested intellectual property.

We recognized royalty revenues of $0.3 million , $0.5 million and $0.2 million during the years ended

December 31, 2025 , 2024 and 2023 , respectively. Royalty revenues reflect estimated royalties from sales of cell-based

research products from our divested stem cell assets.

Future license fee and royalty revenues are dependent on additional agreements being signed, if any, our

current license agreement with Lineage being maintained, and the underlying patent rights for the license remaining active.

Costs and Operating Expenses

The following table summarizes our costs and operating expenses for the years ended December 31:

(In thousands)	2025	2024	Change $	Change %	2023	Change $	Change %
Cost of goods sold	$ 4,745	$ 1,256	$ 3,489	278%	$ —	1,256	100%
Research and development	71,433	103,738	(32,305)	(31%)	125,046	(21,308)	(17%)
Selling, general and administrative	159,256	145,732	13,524	9%	69,135	76,597	111%
Restructuring charges	$ 17,032	$ —	17,032	100%	$ —	—	0%
Total costs and operating expenses	$ 252,466	$ 250,726	$ 1,740	1 %	$ 194,181	$ 56,545	29%

Cost of Goods Sold

Cost of goods sold was approximately $4.7 million for the year ended December 31, 2025 , compared to

$1.3 million for the year ended December 31, 2024, which consists of raw materials, third-party manufacturing costs to

manufacture the raw materials into finished product, freight, and indirect overhead costs associated with the sale of

RYTELO .

Prior to receiving FDA approval for RYTELO in June 2024, we manufactured inventory to be sold upon

commercialization and recorded the costs as research and development expense. As a result, a significant portion of the

manufacturing costs related to the inventory manufactured prior to receiving FDA approval was partially expensed in a

prior periods and are therefore excluded from the cost of goods sold for the twelve months ended December 31, 2025 and

We estimate our cost of sales as a percentage of product revenue, net will continue to be positively impacted for at

least the next 12 months as we sell through certain inventory that was partially expensed prior to FDA approval.

Research and Development Expenses

During the year ended December 31, 2025 , our RYTELO (imetelstat) program and our research discovery

program related to potential next generation telomerase inhibitors were the only research and development programs we

supported. For these research and development programs, we incur direct external, personnel-related and other research and

development costs. For the years ended December 31, 2025 , 2024 and 2023 , research and development expenses consist of

expenses incurred in developing and testing imetelstat and research related to potential next generation telomerase

inhibitors. These expenses include, but are not limited to, payroll and personnel expense, lab supplies, non-clinical studies,

clinical trials, including support for investigator-led clinical trials, raw materials to manufacture clinical trial supply,

manufacturing costs for research and clinical trial materials, sponsored research at other labs, consulting expenses, costs to

maintain technology licenses and research-related overhead.

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Research and development expenses for the years ended December 31, 2025 , 2024 and 2023 were as

follows:

(In thousands)	Year Ended December 31, — 2025	2024	2023
Direct external research and development expenses:
Clinical program: Imetelstat	$ 39,732	$ 68,424	$ 86,914
Personnel related expenses	29,054	33,411	31,595
All other research and development expenses	2,647	1,903	6,537
Total	$ 71,433	$ 103,738	$ 125,046

The decrease in research and development expenses in 2025 , as compared to 2024 , was primarily due to

lower manufacturing and quality costs that were capitalized in the current period now that RYTELO is approved, versus

being partially expensed in 2024, and lower clinical trial costs associated with a decrease of activity in our Phase 3 IMerge

MDS study after FDA approval of RYTELO in 2024. We expect our research and development expenses to decrease

slightly in 2026, primarily due to lower labor costs driven by a decrease in headcount as a result of the workforce reduction

in December 2025, partially offset by higher clinical trial costs.

The decrease in research and development expenses in 2024, as compared to 2023, was primarily due to

manufacturing and quality costs that were capitalized beginning with the third quarter of 2024, due to FDA approval of

RYTELO in June 2024, versus being expensed as research and development expenses in 2023. The decrease is partially

offset by an increase in labor costs due to higher headcount and incentive and stock-based compensation expense

recognized due to the vesting of performance-based stock options upon FDA approval.

A discussion of the risks and uncertainties associated with the development of imetelstat can be found in

the sub‑sections entitled “Risks Related to the Further Development of RYTELO (Imetelstat),” “Risks Related to the

Commercialization of RYTELO” and “Risks Related to Regulatory Approval of RYTELO” in Part II, Item 1A entitled

“Risk Factors” and elsewhere in this Report. As a result of these risks and uncertainties, we are unable to determine with

any degree of certainty the duration and completion costs of ongoing and potential future imetelstat research and

development projects, anticipated completion dates, or when and to what extent we will receive cash inflows from the

commercialization and sale of RYTELO in any other jurisdictions or indications we are pursuing or may in the future

pursue, if at all.

Selling, General and Administrative Expenses

Selling, general and administrative expenses w ere $159.3 million , $145.7 million, and $69.1 million for the

years ended December 31, 2025 , 2024 and 2023 , respectively.

The increase in selling, general and administrative expenses in 2025 as compared to 2024 is primarily due

to an increase in sales and marketing full-time employees of approximately $3.7 million, additional investment in

marketing programs of approximately $2.6 million and approximately $1.3 million higher legal expenses. W e expect our

selling, general and administrative expenses to decrease in 2026, primarily due to lower labor costs driven by a decrease in

headcount as a result of the workforce reduction in December 2025, partially offset by higher marketing costs due to

continued investment in our RYTELO commercialization strategy.

The increase in selling, general and administrative expenses in 2024 as compared to 2023 primarily reflects

the net result of higher personnel-related expenses of approximately $40.0 million related to increased headcount to support

commercial launch of RYTELO in the U.S. and stock-based compensation recognized upon FDA approval of RYTELO

due to the vesting of performance-based stock options, as well as increased costs for commercial preparatory activities and

launch support of approximately $33.0 million.

Restructuring Charges

In December 2025, we implemented a workforce reduction, representing approximately one-third of our

workforce prior to the reduction in headcount. Restructuring charges consist of termination benefits such as one-time

employee severance payments, healthcare and related benefits, and other employee-related costs . In 2025, restructuring

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charges were $ 17.0 million. There were no restructuring charges in 2024 or 2023. These costs were recorded in the

consolidated statement of operations as restructuring charges.

Interest Income

Interest income was $18.1 million, $19.6 million, and $18.2 million for the years ended December 31,

2025 , 2024 and 2023 , respectively. The decrease in interest income in 2025 compared to 2024 primarily reflects a smaller

marketable securities portfolio. Interest earned in future periods will depend on the size of our marketable securities

portfolio and prevailing interest rates.

The increase in interest income in 2024 compared to 2023 primarily reflects a larger marketable securities

portfolio due to the receipt of net cash proceeds from the underwritten offering completed in March 2024, as well as higher

yields from marketable securities purchases.

Interest Expense

Interest expense was $32.7 million, $18.5 million, and $8.3 million for the years ended December 31, 2025 ,

2024 and 2023 , respectively.

The increase in interest expense in 2025 as compared to 2024 primarily reflects $18.9 million in non-cash

interest expense related to the Royalty Pharma Agreement and $13.7 million related to the Pharmakon Loan Agreement,

which were entered into in November 2024.

The increase in interest expense in 2024 compared to 2023 primarily reflects $5.3 million in non-cash

interest expense related to the Royalty Pharma Agreement, $2.3 million in Pharmakon Loan Agreement and $2.6 million

increase related to the Hercules agreement in comparison to the prior year.

On November 1, 2024, we entered into the Pharmakon Loan Agreement, and in connection with this

transaction, all obligations outstanding under the Hercules Loan Agreement were repaid in full on November 1, 2024, upon

which the Hercules Loan Agreement was terminated. On January 5, 2026, the Pharmakon Loan Agreement was amended

to extend the date for requesting the Tranche B Loan and Tranche C Loan, once available, from December 31, 2025 to July

30, 2026. The amendment also extended the Makewhole Date from November 1, 2026 to May 1, 2027. See Note 10 on

Debt in Notes to Consolidated Financial Statements of this Report for additional information.

Other Income (Expense), Net

Other income (expense), net was a loss of $ 0.4 million for the year ended December 31, 2025 , and a loss of

$ 0.2 million and less than $ 0.1 million for the years ended December 31, 2024 and 2023 , respectively. Other income

(expense), net primarily reflects bank charges related to our cash operating accounts and marketable securities portfolio as

well as foreign currency transaction adjustments.

Loss on Extinguishment of Debt

We recorded a loss on the extinguishment of debt of $1.7 million for the year ended December 31, 2024 .

This loss is related to the settlement of debt outstanding under the Hercules Loan Agreement. No debt extinguishment

occurred during the years ended December 31, 2025 or 2023. See Note 10 on Debt in Notes to Consolidated Financial

Statements of this Report for additional information.

Liquidity and Capital Resources

As of December 31, 2025 , we had cash, restricted cash, cash equivalents and marketable securities of

$401.1 million, compared to $502.9 million at December 31, 2024 . The decrease in cash, restricted cash, cash equivalents,

and marketable securities from December 31, 2025 was primarily the net result of cash used in operations, partially offset

by the receipt of net cash proceeds from accounts receivable.

On March 21, 2024, we completed an underwritten public offering consisting of 41,999,998 shares of our

common stock and a pre-funded warrant to purchase 8,002,668 shares of our common stock. All of the securities were

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issued separately. The offering price of the common stock was $3.00 per share. The offering price of the 2024 pre-funded

warrant was $2.99 per share. The 2024 pre-funded warrant has an exercise price of $0.001 per share and may be exercised

at any time until it is exercised in full. The net cash proceeds from this offering were approximately $141.0 million, after

deducting the underwriting discount and other offering expenses paid by us, and excluding any proceeds from the exercise

of the 2024 pre-funded warrant. See Note 11 on Stockholders’ Equity in Notes to Consolidated Financial Statements of this

Report for additional information about the underwritten offering completed in March 2024. During the year ended

December 31, 2025 , 5.4 million of the 2024 pre-funded warrants were exercised.

On November 1, 2024, we entered into the Pharmakon Loan Agreement. We drew the Tranche A Loan of

$125.0 million on November 1, 2024, a portion of which was utilized to repay all outstanding indebtedness associated with

the Hercules Loan Agreement. The Pharmakon Loan Agreement provides two additional committed term loan tranches, the

Tranche B Loan and the Tranche C Loan, in principal amounts of $75.0 million and $50.0 million, respectively, subject to

customary conditions to fund and, in the case of the Tranche C Loan, achieving certain minimum net sales milestone. The

Tranche B Loan and the Tranche C Loan could have been requested on or prior to December 31, 2025. The Term Loans

mature on November 1, 2029. The Term Loans bear interest at a variable rate per annum equal to 5.75% plus three-month

SOFR with a SOFR floor of 3.00%. On January 5, 2026, the Pharmakon Loan Agreement was amended to extend the date

for requesting the Tranche B Loan and Tranche C Loan, once available, from December 31, 2025 to July 30, 2026. The

amendment also extended the Makewhole Date from November 1, 2026 to May 1, 2027. See Note 10 on Debt in Notes to

Consolidated Financial Statements of this Report for additional information on the Pharmakon Loan Agreement.

On November 1, 2024, we entered into the Royalty Pharma Agreement with Royalty Pharma. Pursuant to

the Royalty Pharma Agreement, we received $125.0 million, or the Purchase Price, in exchange for which Royalty Pharma

obtained the right to receive the Royalty Payments. The Royalty Payments to Royalty Pharma are capped, such that they

will cease upon reaching a multiple of 1.65 times the Purchase Price if Royalty Pharma receives Royalty Payments in that

amount in respect of net sales occurring on or before June 30, 2031, or upon reaching a multiple of 2.0 times the Purchase

Price thereafter. There are no other royalties payable on RYTELO, which was developed internally and is exclusively

owned by Geron. See Note 10 on Debt in Notes to Consolidated Financial Statements of this Report for additional

information on the Royalty Pharma Agreement.

In 2024, warrants to purchase 1,071,981 shares of our common stock were exercised for net cash proceeds

of approximately $1.4 million . In 2025, warrants to purchase 1,162,376 shares of our common stock were exercised,

generating cash proceeds of $1.5 million , which proceeds were received in January 2026. These warrants were issued in

connection with underwritten public offerings of our common stock in 2020. The remaining warrants to purchase 240,146

shares of our common stock expired on December 31, 2025 and are no longer outstanding.

On January 10, 2023, we completed an underwritten public offering of 68,007,741 shares of our common

stock and a pre-funded warrant to purchase 25,000,000 shares of our common stock, or the 2023 pre-funded warrant. The

net cash proceeds from this offering were approximately $213.3 million, after deducting the underwriting discount and

other offering expenses paid by us.

On November 1, 2023, we entered into an At Market Issuance Sales Agreement, or the 2023 Sales

Agreement, with B. Riley Securities, pursuant to which we could elect to issue and sell shares of our common stock having

an aggregate offering price of up to $100.0 million in such quantities and on such minimum price terms as we set from time

to time through B. Riley Securities as our sales agent. We have agreed to pay B. Riley Securities an aggregate commission

equal to up to 3.0% of the gross proceeds of the sales under the agreement. No sales of common stock occurred under the

2023 Sales Agreement, which was terminated in January 2026 .

On February 27, 2026, we entered into a sales agreement, or the 2026 Sales Agreement, with TD Securities

(USA) LLC, or TD Cowen, pursuant to which we may issue and sell shares of our common stock having an aggregate

offering price of up to $150 million from time to time through TD Cowen as the sales agent. We will pay TD Cowen an

aggregate commission rate equal to up to 2.5% of the gross proceeds of the sales price per share for common stock sold

through TD Cowen under the 2026 Sales Agreement. We are not obligated to make any sales of common stock under the

2026 Sales Agreement.

The issuance and sale of our common stock under the 2026 Sales Agreement will be made pursuant to an

automatically effective registration statement on Form S-3 and the related prospectus, in each case to be filed with the

United States Securities and Exchange Commission on or about March 2, 2026.

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We have an investment policy to invest our cash in liquid, investment-grade securities, such as interest-

bearing money market funds, certificates of deposit, U.S. Treasury securities, municipal securities, government and agency

securities, corporate notes and commercial paper. Our investment portfolio does not contain securities with exposure to

sub-prime mortgages, collateralized debt obligations, asset-backed securities or auction rate securities and, to date, we have

not recognized any other-than-temporary impairment charges on our marketable securities or any significant changes in

aggregate fair value that would impact our cash resources or liquidity. To date, we have not experienced lack of access to

our invested cash and cash equivalents; however, access to our invested cash and cash equivalents may be impacted by

adverse conditions in the financial and credit markets.

Financing Strategy

We may, from time to time, consider additional funding through a combination of new collaborative

arrangements, strategic alliances, and additional equity and debt financings or from other sources. We will continue to

manage our capital structure and consider all financing opportunities, whenever they may occur, that could strengthen our

long-term liquidity profile. Any such capital transactions may or may not be similar to transactions in which we have

engaged in the past. There can be no assurance that any such financing opportunities will be available on acceptable terms,

if at all.

Future Funding Requirements

Successful drug development and commercialization requires significant amounts of capital. As of

December 31, 2025 , we had approximately $401.1 million in cash, cash equivalents, restricted cash and marketable

securities. Based on our current operating plans and assumptions, we believe that our existing cash, cash equivalents, and

marketable securities, together with anticipated net revenues from sales of RYTELO, will be sufficient to fund our

projected operating requirements for the foreseeable future. However, if we do not generate net revenues from commercial

sales of RYTELO at the levels we anticipate, if we experience unforeseen events or choose to make other investments in

our business, or our assumptions regarding our projected operating expenses are otherwise incorrect, we may require

additional funding, which could include a combination of public or private equity offerings, debt financings (including

additional tranches under the Pharmakon Loan Agreement, if available), collaborations, strategic alliances, licensing

arrangements or marketing and distribution arrangements, which may not be possible. For example, changes in our

operations, such as increased development, manufacturing and clinical trial expenses, or our undertaking of additional

programs, business activities, or entry into strategic transactions, including potential future acquisitions of products,

technologies or businesses, may cause our operating expenses to increase, perhaps significantly, which could require us to

raise additional funding. If adequate funds are not available to us when we need them, our RYTELO commercialization

efforts may be adversely affected and we may be unable to pursue further development of imetelstat, which would severely

harm our business and we might cease operations.

Despite receiving FDA approval of RYTELO in the U.S. in June 2024 and marketing authorization in the

EU in March 2025, the outcome of any clinical activities and/or regulatory approval process is highly uncertain, and we

cannot reasonably estimate whether our future development activities may succeed, whether we will obtain regulatory

approval for RYTELO in any other jurisdictions or indications we are pursuing or may in the future pursue, or whether we

will be able to effectively commercialize RYTELO in the U.S. or in the EU for lower-risk MDS or other potential

indications, if at all. We may never recoup our investment in any RYTELO development, which would adversely affect our

financial condition and our business and business prospects, and might cause us to cease operations. In addition, our plans

and timing expectations could be further delayed or interrupted by the effects of macroeconomic or other global conditions,

including those resulting from further changes in tariffs and other trade restrictions, renegotiation of existing international

trade agreements or further escalation of trade tensions, inflation, fluctuations in interest rates, prospects of a recession,

government shutdowns, bank failures and other disruptions to financial systems, civil or political unrest, military conflicts,

pandemics or other health crises and supply chain and resource issues. Further, our future capital requirements are difficult

to forecast and will depend on many factors, including:

• the accuracy of the assumptions underlying our estimates for our capital needs;

• the overall level of sales and market acceptance of RYTELO;

• the scope, progress, timing, magnitude and costs of non-clinical and clinical development, manufacturing and

commercialization of RYTELO, including commercialization in the U.S. and any commercialization in the EU for

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lower-risk MDS, or in any other jurisdictions or other indication we may pursue, subject to clearances and

approvals by the FDA and similar international regulatory authorities;

• delays or disruptions in opening sites, screening and enrolling patients or treating and following patients, in our

current or any potential future clinical trials of RYTELO;

• the costs, timing and outcomes of regulatory reviews or other regulatory actions related to RYTELO,

• the costs of preparing, filing and prosecuting patent applications and maintaining, enforcing and defending

intellectual property-related claims;

• the costs of manufacturing, developing, commercializing and marketing RYTELO, including with respect to third-

party vendors and service providers and our ability to achieve any meaningful reduction in manufacturing costs;

• the sales price for RYTELO;

• the availability of coverage and adequate third-party reimbursement for RYTELO;

• the extent to which we acquire or in-license other drugs and technologies, or invest in businesses, products or

technologies, although we currently have no commitments or agreements relating to any of these types of

transactions, or to which we out-license RYTELO;

• the extent to which we are able to enter into and conduct successful commercial arrangements with third parties,

including for the commercialization and marketing of RYTELO in the EU and in any other regions outside of the

U.S., if approved for commercialization in such other regions;

• expenses associated with the pending putative securities class action and shareholder derivative lawsuits and

potential additional related lawsuits, as well as any other litigation;

• the extent and scope of our selling, general and administrative expenses, including expenses associated with

pending and potential future litigation;

• our level of indebtedness and associated debt service obligations;

• the costs of maintaining and operating facilities in California and New Jersey, as well as higher expenses for

travel;

• macroeconomic or other global conditions that may reduce our ability to access equity or debt capital or other

financing on preferable terms, which may adversely affect future capital requirements and forecasts; and

• the costs of enabling our personnel to work remotely, including providing supplies, equipment and technology

necessary for them to perform their responsibilities.

In the event we need to raise additional capital to fund our business, including pursuant to the 2026 Sales

Agreement with TD Cowen, the Tranche B Loan and the Tranche C Loan under the Pharmakon Loan Agreement, which

are subject to certain funding conditions, capital lease transactions or other financing sources, such additional capital may

not be available on acceptable terms, or at all. We may be unable to raise equity capital, or may be forced to do so at a

stock price or on other terms that could result in substantial dilution of ownership for our stockholders. The receptivity of

the public and private debt and equity markets to proposed financings has been substantially affected by uncertainty in the

general economic, market and political climate due to the effects of macroeconomic or other global conditions, such as

further changes in tariffs and other trade restrictions and uncertainty around further escalation of trade tensions and

renegotiation of existing international trade agreements, inflation, fluctuations in interest rates, prospects of a recession,

government shutdowns, bank failures and other disruptions to financial systems, civil or political unrest, military conflicts,

pandemics or other health crises and supply chain and resource issues, and may in the future be affected by other factors

which are unpredictable and over which we have no control. These effects have increased market volatility and could result

in a significant long-term disruption of global financial markets, which could reduce or eliminate our ability to raise

additional funds through financings, and could negatively impact the terms upon which we may raise those funds.

Similarly, these macroeconomic conditions have created extreme volatility and disruption in the capital markets and is

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expected to have further global economic consequences. If the equity and credit markets deteriorate, including as a result of

macroeconomic or other global conditions, such as inflation, changes in interest rates, prospects of a recession, government

shutdowns, further changes in tariffs and other trade restrictions, bank failures and other disruptions to financial systems,

civil or political unrest, military conflicts, pandemics or other health crises and supply chain and resource issues, it may

make any necessary debt or equity financing more difficult to obtain in a timely manner or on favorable terms, more costly

or more dilutive. If we are unable to effectively commercialize RYTELO, or raise additional capital, if needed, or establish

alternative collaborative arrangements with third-party collaborative partners for RYTELO, when needed, the development