OPKO Health Inc.

Earnings Release • Jun 7, 2018

UNITED STATES SECURITIES AND EXCHANGE COMMISSION

Washington, DC 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): June 6, 2018

OPKO Health, Inc.

(Exact name of registrant as specified in charter)

Delaware 001-33528 75-2402409 (State or other jurisdiction of incorporation)

(Commission File Number)

(IRS Employer Identification No.)

4400 Biscayne Blvd. Miami, Florida 33137 (Address of principal executive offices) (Zip Code)

Registrant's telephone number, including area code: (305) 575-4100

Not Applicable Former name or former address, if changed since last report

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

☐ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company ☐

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. ☐

ITEM 7.01. Regulation FD Disclosure.

On June 6, 2018, OPKO Health, Inc. (the "Company") will participate in meetings with investors at the Jefferies 2018 Global Healthcare Conference. A copy of the Company's presentation materials is furnished as Exhibit 99.1 to this Current Report on Form 8-K and incorporated herein by reference. The presentation materials are also available on the Company's website at www.opko.com under Investor Relations. The information contained on the Company's website shall not be deemed part of this report.

The information contained in Item 7.01 to this Current Report on Form 8-K and Exhibit 99.1 attached hereto shall not be deemed "filed" for purposes of Section 18 of the Securities Exchange Act of 1934 as amended ("Exchange Act"), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing by the Company under the Securities Act of 1933 as amended ("Securities Act") or the Exchange Act, except as expressly set forth by specific reference in such a filing.

ITEM 9.01. Financial Statements and Exhibits.

(d) Exhibits

Exhibit No.	Description
99.1	OPKO Health, Inc. Presentation Materials

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

OPKO Health, Inc.

By: /s/ Adam Logal

Date: June 6, 2018 Name: Adam Logal

Title: Senior Vice President, Chief Financial Officer

ОРКО

June 2018

Forward Looking Statements

This presentation contains "oward-ooking statemise the Private Securities Liligation Reform Act of 195 (PS.RA), wich statents nay be identified by words such as "expects," will," may, "anticipates," believes," should," and other words of similar nearing, including statements regarding our esimaled revenues and ryallies from the oulicense of our products, our ability to ability to ability to ever of growth, the potential for our products under the 44score® to influence 80% of biopsy decisions and predict the risk of aggressive prostale cancer, the expected iming of the clinical studies and regulate under development, the outcome of our cinical irials and hall such outcomes will supor marketing approval or commercialin and size of the market for our products including williout limitation. Rolarian, Rayadee® had-CTP, the 4Kscore, Factor VIlla-CTP, Alphare, oxytomodule, and our point-of-care diagnosic products, the potential benefits of our products under development, including whether the 46sore will preached in 40-55% oast swings, the expected response date from he mo approval for the PMA for PSA and submission and expected launch date for each, hat oxyntomobilin will provide superior long-erm theray for obesiy and Type I diabeles paients, our ability o successfully commendiates such as Rolapiant, the 4Kscore, NEA-CTP, Rayaldee, Alpharen, the SARM, and oxyntomodulin, and whether Rayalde in stage in stage 3 and 4 CXD patients with SHPT, whether Rayaldee will raise serum total 25-hydroxyvianin D (25D) more effectively than any over-he-counter (R) products currently marketed without the risk of hypercaleer on new indications including sage 5 CX2 and the lincal dala and post hoc sensibily anaysis for hGH-CTP will support submission of a Biologics License Application (BLA) and aproval for hGH-CTP in adults, whether we will be required to make any changes to or development plans for hGH-CTP, expectations regarding gatent timing for commending, completing and annuncing results for our clinical the liming to release of thi cala and seeking and obtaining FDA and European regulatory approved on our products, our ability to obtain a positive coverage deemination for the 4Kscore and whether we have enough sierities and justify a positive coverage determination, whether lovitis and the payors will reimburse us for the 4Ksore test, and be iming of commercial launch of our producting statements are only predictions and effect our views as of the date hey were mate, and we underlake no obligation to update such statements are subject o many risks and cause ou activites or activites or actual prom the advilies and results and including statements, including integration challenges with Bio-Reference and cher acquired business and risks inherent in funding, developing and obtaining regulative products and treatments, the success of our collaboration with Plizer, general market factors, compellive product availability, federal and state regulations and legislation, delays associated with development of rovel lectiminges, unexpected difficulies and delays in validates, the regulator process for new products and indicaling ssues that may arise, the oos of funding lenghy research programs, the new loved, the possibility of infinging a third party's patents or the intellectual propery rights, the uncertainly of obtaining patents overing our processfuly enforcing them against third parties, and the potential for litgation or government investigations, anony other factor, including all of the heading Risk Factors in our Annual Report on Form 10-1 and other filings with the Securites and Exchange Commission.

June 2018 | 2

Multi-Faceted Growth Strategy

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UROLOGY

SARM: BPH, Stress Incontinence

Fa he

ENDOCRINOLOGY HEMATOLOGY

B

ARM: BPH, Stress Incontinence K-1: Overactive Bladder		DIAGNOSTICS	RAYALDEE™: CKD Alpharen 100: Phosphate Binder NK-1 Antagonist: Uremic Pruritus	NEPHROLOGY
ctor VIIa: Hemophilia H: Growth hormone deficiency	4Kscore Claros: PSA, Testosterone	Claros: Vitamin D
		>2000 Tests GeneDx
A hGH Analog: Agromegaly F-1 CTP: GH receptor deficiency			SARM: Increase muscle mass, Decrease fat mass, Increase bone mass	METABOLIC
A GLP-2: Short bowel syndrome A Parathyroid Hormone A Insulin		AntagoNAT: Dravet Syndrome	Oxyntomodulin: Type 2 diabetes, Overweight, Obesity	AGING
		GENETIC DISEASES		lun

June 2018 | 4

SYNDROME

Investment Highlights

Mature Diagnostic Business with ~900MM in revenue & growing

Marketed Pharmaceuticals

in early life-cycle with multi \$B market opportunities in large underserved markets

Robust Development Pipeline

Multiple late-stage clinical trials addressing indications with large unmet medical needs

Strategy & Execution

Management team with a track record of success and commitment to opportunistic development

Marketed Pharmaceuticals

• · RAYALDEE addresses unmet need in CKD market, ~9 million patients
• · RAYALDEE partnered with Vifor Fresenius; Up to \$837 million in milestones, doubledigit royalties
• · RAYALDEE partnered in Japan with Japan Tobacco; up to \$112 milestones with tiered double-digit royalties

· hGH-CTP partnered with Pfizer, \$570 million pre-commercial milestones; double digit

• royalties and profit-sharing,
  • ~\$3 billion growing market for hGH
  • · Pivotal Phase 3 pediatric hGH-CTP study to complete enrollment in 2018
• · Initiating multiple Phase 2 clinical studies in various areas of unmet need
• · Bio-Reference Laboratories revenue of ~900MM in 2017
• · 400-person sales and marketing team drives industry-leading esoteric testing, ~70% of revenues
• Facilitates uptake of 4Kscore® prostate cancer test and Claros® 1 point-of-care platform

Diversified assets across business units

Diversified Assets Early Life Cycle Pharmaceuticals

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RAYALDEE®

Chronic Kidney Disease (CKD) - The Silent Killer

• · 9th Leading cause of death, ahead of breast and prostate cancer
• · Prevalence expected to increase due to obesity, diabetes and hypertension
• · Most patients die from cardiovascular disease, precipitated by secondary hyperparathyroidism (SHPT)
• · SHPT is driven by vitamin D insufficiency (VDI) and characterized by elevated blood levels of parathyroid hormone (PTH)
• · High PTH levels promote calcification (hardening) of vascular and renal tissues, the major cause of CKD mortality
• · New KDIGO Clinical Practice Guidelines recommend against routine use of VDRAs in CKD and highlight the unproven effectiveness of vitamin D supplementation

Healthcare providers have no good options to treat SHPT in stage 3-4 CKD except for RAYALDEE

DPKC

RAYALDEE® Overview

Product Launched November 30, 2016

• = Extended-Release (1x daily) oral formulation of 25D3* addresses significant unmet need
• ။ FDA-approved for SHPT (elevated PTH) in patients with stage 3-4 CKD and VDI
• Reduces plasma PTH and increases serum 25D with a safety profile similar to placebo
• = Minimal effects on serum calcium or phosphorus (key drivers of vascular calcification)
• · Expected to take significant market share in stage 3-4 CKD patients with SHPT & VDI (~9M patients in US)
• · Potential for new indications including stage 5 CKD, institutionalized elderly, osteoporosis and cancer

RAYALDEE® Sends Clear Message

RAYALDEE signals changes in treatment of SHPT

• First and only extended-release prohormone of active form of vitamin D3
• · Brings 25-hydroxyvitamin D levels up
• Brings iPTH levels down

June 2018 | 10

opko

RAYALDEE® Commercialization

• 50-Person sales and marketing team launched RAYALDEE on November 30, 2016
  • Increased field sales force from 35 to 64 reps (October 2017)
  • Commercial insurance under contract for >83% of U.S. covered lives
  • Medicare Part D insurance under contract for >53% of U.S. covered lives
• · Total RAYALDEE prescriptions increased approximately 730% in 1Q18 compared to 1Q17, and 38% compared to 4Q17
• = 1Q18 RAYALDEE revenue of \$3.7MM
• International partnership with Vifor Fresenius; up to \$837 MM in milestones, double digit royalties
• Japanese partnership with Japan Tobacco; up to \$112 MM in milestone payments, tiered doubledigit royalties
• Initial line extension plans
  • Phase 2 clinical trials for higher-dose RAYALDEE to treat stage 5 CKD to begin 2H18
  • Phase 2 clinical trial of NK-1 antagonist to treat pruritus in CKD patients to begin 2H18

June 2018 | 11

Diversified Assets Broad Development Pipeline

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hGH-CTP (Somatrogon) hGH deficiency Adult Ph 3 completed YE16 Pediatric Ph 3 Initiated YE16 OPK88005 (Factor VIIa-CTP) Hemophilia A & B Ph 2a ongoing (Seeking Partner) OPK88003 (Oxyntomodulin) Diabetes, obesity Ph 2 initiated in 1Q 2018 OPK88004 (SARM) BPH Ph 2 initiated in 4Q 2017 OPK88001 (AntagoNAT) Dravet Syndrome Ph 2a in 2H 2018 Robust Late-Stage Pipeline Rayaldee® (CTAP101) SHPT (CKD stage 5) Ph 2 in 2H 2018 Alpharen™ (Fermagate) Hyperphosphatemia (CKD stage 5 patients) Seeking Partner OPK88002 (NK1) Pruritus (itching) Ph 2a in 2H 2018 CTP Biopharma Preclinical Phase 1 Phase 2 Phase 3 Partnered with Vifor Fresenius Collaboration with Pfizer Renal June 2018 |

hGH-CTP Competitive Advantages

New molecular entity (NME) that maintains native sequence of growth hormone

• · Once weekly injection vs. current products requiring daily injections
• · Phase 3 study in growth hormone deficient adults completed at the end of 2016
• · Phase 3 study in naive growth hormone deficiency pediatric population underway
• Final presentation:
  • Refrigerated, liquid, non viscous formulation
  • Disposable easy-to-handle pen injection device with thin needle and small injection volume
• · Orphan drug designation in the U.S. and the EU for children and adults

1 National Center for Biotechnology Information: 2 http://www.childgrowthfoundation.org/

al-K

Partnered with Pfizer \$570M Pre-commercial milestones Double digit royalties

1 in 10K

Adults diagnosed with GHD1

1 in 3.8K

Children in U.S. have growth failure due to growth hormone deficiency2

June 2018 | 14

hGH-CTP (Somatrogon) Program Status

Initiated Phase 3 pediatric hGH-CTP study in December 2016

• · 220 patients, non-inferiority comparison of weekly hGH-CTP to daily growth hormone
• · Global study sites initiated in December 2016
• · Easy-to-use, disposable, refrigerated pen device

Phase 3 adult hGH-CTP

• · In December 2016 reported that printery end rial mass from baseline to 26 weeks did not demonstrate a statistical significance between the IGH-CTP treated group and placebo
• · Completed post hoc outlier analysis in June 2017 to eliminate the influence of outliers on the primary endpoint results
• · Analyses, which excluded outlies, showed a statifies and placed on the change in tunk fat mas; additional analyses that did not exclude outliers showed mixed results
• No safety concerns 피
• Correspondence and communication with FDA indicated that:
  • · There is a path for submission whereby FDA would assess the totally of the data all relevant efficacy and salents patients
  • " The number of patients to be included in the safety database seems sufficient
  • " The design of the bioequivalence study for the change from vial to pen formulation is generally acceptable
• Next Steps 배
  • · Assess regulatory strategy for adult indication based on response from FDA

hGH-CTP pediatric registration study in Japan underway

• 44 patients, comparison of weekly hGH-CTP to daily growth hormone
• · Same pen device, dosage and formulation used in global Phase 3 pediatric study

June 2018 | 15

Selective Androgen Receptor Modulator (SARM)

OPK-88004 Once Daily Oral Tablet

• · Phase 2 study of 350 male subjects for another indication showed significantly increased lean body mass and muscle strength and significant fat mass reduction with no change or lower prostate specific antigen (PSA) levels
• Animal studies resulted in decreased size of prostate

Clinical Plan

• Initiated 125-patient Phase 2 trial in benign prostatic hypertrophy (BPH) or enlarged prostate to determine optimal dose
  • Complete enrollment by YE18
  • Top-line data mid-2019

1 Deters LA. Benign prostatic hypertrophy. Emedicine http://emedicine.medscape.com/

adkt

50 Million

men in the U.S. are affected by BPH

90%

Men 80 years old and older are affected by enlarged prostate1

14M

Men in the U.S. with lower urinary tract symptoms suggestive of BPH1

June 2018 | 16

NK 1 Antagonist

Pruritus

• · Acute and chronic pruritus ("itching") occurs in 10% to 15% of the population
  • Most prevalent in skin, kidney and liver diseases
• · Substance P is implicated in pruritus
• NK-1 antagonists block substance P activity and have been shown to reduce itching in human trials

Pruritus in Dialysis Patients

· Major medical need and requires management

OPK-88002 Clinical Development

· Phase 2a Clinical Study expected to begin in 2H 2018

Uremic pruritus associated with CKD in adults1

52%

70-90%

Of kidney dialysis patients suffer from Pruritus

40%

Patients undergoing hemodialysis suffer chronic pruritus1

1 Medscape.com, International Society of Nephrology (ISN)/

June 2018 | 17

a -K

Oxyntomodulin Analog

for Treatment of Type 2 Diabetes and Obesity

OPK-88003

• · Drug development focused on: blood glucose control and reducing body weight
• · Once-weekly analog with potentially first to market dual GLP-1/glucagon agonist
• · Data support that combining GLP-1 and glucagon activity provides superior weight loss

Clinical Development

• " Phase 2b dose-escalation study initiated in March 2018
• · Expect to complete enrollment in June 2018
• · Preparation of pen and formulation for phase 3 is ongoing

Phase 2 study in 420 type 2 diabetes patients (week 12)

% Body weight change from baseline % Patients with ≥ 5% weight loss

1 http://www.diabetes.org; 2 https://www.cdc.gov/diabetes

OPKO

93%

Of the American population suffers from diabetes1

\$176B

Direct medical costs related to Diabetes in U.S.1

17.7 M

Adults diagnosed with diabetes reported taking any medication2

June 2018 | 18

Diversified Assets Core \$900 MM Business And Growing

June 2018 | 19

OPKO Diagnostics

Near-term Opportunities

Leveraging National Marketing, Sales and Distribution Resources to Drive Uptake of Diagnostic Platforms

• · Over 12 million patients served during 2017
• = Revenue of-\$900MM in 2017; 4Q17 revenue of~\$150MM
• " GeneDx is a genomics leader known for its expertise in rare disease and whole exome testing
• · Utilizing BRL commercial infrastructure to drive 4Kscore adoption and will use for Claros 1 launch and adoption upon approval

BioReference Labs is third largest full service reference laboratory in the U.S.

• · ~ 400 Person sales and marketing team
• ~ ~ 5,000+ People working together to support the needs of clients and patients
• · ~ 200+ Patient service centers located throughout the U.S

June 2018 | 20

OPKC

4Kscore® Test Blood Test Alternative To Biopsy

More Than 2 Million Prostate Biopsies/Year WW

• · Clinical utility based on decades of biomarker research and >20,000 men tested in Europe and U.S.
• " In long-term outcome data, 4Kscore test can predict 20 year metastasis free survival for individual patient
• " Included in the 2015-2017 NCCN and 2016-2017 EAU Prostate Cancer Guidelines
• · Health economics study shows 40-55% cost savings by avoiding unnecessary MRI, prostate biopsy, and additional treatment or monitoring of indolent cancer
  • 80% of men undergoing prostate biopsy based on PSA are found to have no cancer or indolent cancer
• " Clinical utility study shows 4Kscore influences 89% of decisions about performing prostate biopsy

4Kscore®

Only blood test that accurately identifies risk for aggressive prostate cancer

>5K

Physicians have used 4Kscore in practice

21K

Tests performed during 1Q18, a 13% increase compared with 1Q17

June 2018 | 21

DEKO

4Kscore Clinical and Commercial Update

• · Category | CPT published and effective January 1, 2017; CMS national rate for 2018 increased to \$760 vs \$600 in 2017
• · Novitas Solutions (Medicare Administrative Contractor for OPKO Elmwood Park, NJ facility)
  • Novitas issued a draft non-coverage determination in May 2018 subject to a public comment period ending July 5, 2018
  • Novitas has been and continues to pay for 4Kscore Medicare submissions of the draft noncoverage decision.
  • OPKO is taking a multiple pronged approach to address the concerns raised in the Novitas draft LCD
• · Data from study at VA hospitals confirming the 4Kscore's ability to accurately predict aggressive prostate cancer presented
  • Demonstrated equally effective and vital clinical test for African men, who have the highest rates of prostate cancer mortality
• · Radical prostatectomy study demonstrated 4Kscore can effectively differentiate biopsy Gleason 6 cancer from those likely to harbor adverse pathology

June 2018

Claros 1 Platform Addresses Large Point of Care Test Market

25M PSA Tests in U.S. Annually;

• · Novel diagnostic system can provide rapid, quantitative blood test results in 10 minutes - right in the physician's office
• · Modular PMA with FDA for PSA test filed in 4Q17; expect response 2H18
• Expect testosterone 510(k) filing YE18
• · Claros 1 point of care platform will leverage BioReference Labs distribution and marketing
• Menu expansion following initial FDA filings

opko

Select Financial Information March 31, 2018

Balance Sheet	" Cash, cash equivalents & marketable securities: \$99.9 million = Net investments: \$297.5 million " Current portion of line of credit and notes payable: \$11.9 million · Senior notes: \$31.8 million · \$55 million convertible note issued February 2018
Capital Structure	" Common shares outstanding: 559.47 million
Revenue	· Consolidated revenues for the three months ended March 31, 2018 were \$254.9 million compared with \$266.4 million for the comparable period of 2017 - Revenue from services were \$211.3 million for the three months ended March 31, 2018 compared with \$228.6 million for the comparable 2017 period > While not directly comparable, this represents a marked improvement from the most recently completed quarter ended December 31, 2017

June 2018 | 24

орко

Upcoming milestones

Progress Across Multiple Business Areas

V 4Kscore	Launched TV ads in FL 1Q18
✓ Oxyntomodulin Phase 2b	Initiated March 2018
RAYALDEE Stage 5 CKD Phase 2	Initiate 2H18
NK-1 Antagonist Pruritus Phase 2a	Initiate 2H18
Claros 1 PSA approval	3Q18
SARM Phase 2 complete enrollment	YE18
Claros 1 Testosterone 510(k) submission	YE18
Phase 3 Pediatric hgh-CTP study complete enrollment YE18

Thank You