OPKO Health Inc.

Earnings Release • Sep 7, 2017

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): September 7, 2017

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)

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

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 (see General Instruction A.2. below):

☐ 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 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.

The information set forth under Item 8.01 of this Current Report on Form 8-K is incorporated herein by reference to this Item 7.01.

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 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 Act, unless expressly stated otherwise.

Item 8.01 Other Events.

Members of management for OPKO Health, Inc. (the "Company"), a Delaware corporation, prepared a slide deck to be presented at the 2017 Wells Fargo Healthcare Conference in Boston on September 7, 2017. Due to the possible effects of Hurricane Irma on South Florida, the Company's management will no longer be presenting at the conference and is instead filing an updated management presentation with this Form 8-K. The Company's management intends to use a similar presentation at the Cantor Fitzgerald Global Healthcare Conference on Monday, September 25, 2017, the Ladenburg Thalmann 2017 Healthcare Conference on Tuesday, September 26, 2017, and the 10th Annual Barrington Fall Investment Conference on Wednesday, September 27, 2017.

A copy of the Company's updated corporate presentation is furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference. The corporate presentation is also available on the OPKO website at www.opko.com under Investor Relations. The information contained on OPKO's website shall not be deemed part of this report.

Item 9.01 Financial Statements and Exhibits.

(d) Exhibits

Exhibit No. Description 99.1 Corporate Presentation of OPKO Health, Inc.

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.

Date: September 7, 2017 By /s/ Adam Logal

Name: Adam Logal Title: Senior Vice President, Chief Financial Officer

Exhibit Index

Exhibit No.	Description
99.1	Corporate Presentation of OPKO Health, Inc.

Exhibit 99.1

opko

Diagnostics & Pharmaceuticals for Large Markets with Unmet Needs

September 2017

NASDAQ: OPK

FORWARD-LOOKING STATEMENTS

OPKI

This presentation contains "oward-looking statem is defined under the Private Securities Litigation Referrn Act of 1955 (PSLRA), which statements may be identified by words such as "expects," will," may," "anticipates," believes," potential," and other words of similar meaning, including statements regarding our esimated projections, expected milestines and royalites from the outlicense of our products, our ability to achieve high levels of grown, the potential for or products unite the 4Kscore® to influence 89% of biopsy decisions and predict the risk of aggressive proslate cancer, the expected iming of the clinical studies ander development, the outcome of our clinical trials and valiation studies and that such outcomes will support narketing the expected market peneration and size of the market for our products, indusing without imitation, Rolapitant, Rayaldee®, hGH-CTP, the 4Ksore, Factor VII-CTP, Alpharen, oxyntomodulin, the SAM candidate, our porint heath, products, the potential benefits of our products whether the 4 (socre will prodict the nist of 20 year metestasis free survival and result in 40-55% cost savings, the expected submission dates for the PM for testesterne and expected aumbr and or each, that oxyritomobilin will provide superior ongtheray for obesty and Type I dabels patients, our annoncialize our product candidates such as Rolapitant, the 4Kscore, (GH-CTP, Rayadee, Alphaen, the SARM, and whether Rayaldee will take significant market share in stage 3 and 4 CKD patients with SHPT, whehr Rayadee will raise serun total 25hydroxyitamin D (25D) nore effectively than any over-then (Rx) products currently narketed without the risk of hypercalemia, our ability to obtain commercial and Part D covered lives by end of 2017, our ability to develop Rayaldee for new indications including stage 5 CK and the timeline for doing so, whether data from adult clinical studies of approval of a biologics Liense Application, whether we will be required to nale any changes to our development plans for hGH-CTP, expected timing for commensing, compeling and announcing results for our clinical trials, the timing for release of trial data and seeking approvals as vell as reimbursement coverage for our products, expectations regarding growth in sales of our 4Kscore and Rayality to obtain a postive coverage determination for the 4Kscore and vineli data b justly a postive coverations about our animal health business, and the liming of commercial launch of our product candidates. These or warlooking statements are only predictions and the date they were nade, and we undertake no obligation to update such statements are subject to many isks and uncertainles that cause our assults to differ materialy from the activities and results anticipated in forward looking statements, including interation challenges with Bio-Refered businesses, risks interent in tinding, developing and other commercially viable and competitive products and treatment with Pfitzer, general harket factors, competitive product development, product availability, federal and state regulations and legistation with development of novel technologies, in validating and testing nod testing noduct candidates, the regulatory process for new products and including issues that may arise, the cost of funding lengthy research programs, the need for and availability of additional capilal, the possibility of intellectual propery rights, the uncerainly of obtaining patents covering our products and processes and in successfully enfroing them against for ligation or government investigations, anong other factors, including all of the risks dentified under the heading Risk Factors in our Annual Report on Form 10-K and other filings with the Securities and Exchange Commission

A multinational biopharmaceutical and diagnostics company establishing important positions in large markets by leveraging its extensive health care industry expertise and experience.

MULTI-FACETED GROWTH STRATEGY

র্ব

DIVERSIFIED INVESTMENT HIGHLIGHTS

Diagnostics	· Bio-Reference Laboratories revenue of more than \$1Billion in 2016 · 400-person sales and marketing team drives industry-leading esoteric testing, ~70% of revenues · Facilitates uptake of 4Kscore® prostate cancer test and Claros® 1 in office platform · Completed clinical study on Claros® 1 for PSA- Preparing PMA submission to FDA
	· Rayaldee addresses unmet need in ~\$12 billion CKD market, ~9 million patients · Rayaldee license (VFMCRP); Up to \$837 million in milestones, double digit royalties · Phase 2 for higher dosage Rayaldee in Stage 5 CKD patients initiating 4Q17 · VARUBI™ partnered with Tesaro; Up to \$85 million in milestones, double digit royalties, \$1 billion market
Pharmaceuticals	· hGH-CTP is a 1x/week hGH, Partnered with Pfizer, \$570 million pre-commercial milestones; double digit royalties and profit-sharing, ~\$3 billion growing market · Pediatric Phase 3 clinical trial underway and completed adult Phase 3 study in hGH deficiency · Initiating a multitude of Phase 2 clinical studies in various areas of unmet need in: Hemophilia I Benign Prostatic Hypertrophy Obesity/Diabetes Pruritus Dravet Syndrome
Strategy & Execution	· Management team with a track record of success and access to capital " Commitment to opportunistic business development · Production and distribution assets expanding worldwide, multiple strategic investments

OPKO DIAGNOSTICS: NEAR-TERM OPPORTUNITIES

LEVERAGING NATIONAL MARKETING, SALES AND DISTRIBUTION RESOURCES TO DRIVE RAPID AND WIDESPREAD UPTAKE OF OPKO DX PLATFORMS

• BioReference Labs is the 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.
• · Over 12 million patients served during 2016
• · Continued investment in new systems provides better financial data and more information about customers, products and sales
• = New leadership team introducing new programs that are expected to benefit all aspects of the business
• · Revenue of more than \$1Billion in 2016; 2Q17 revenue of \$256.7 million
• · GeneDx is a genomics leader known for its expertise in rare disease and whole exome testing
  • · Continues to further develop it relationship with health care providers and systems
  • · Continues to actively expand its innovative tests and service offerings

019

4KSCORE TEST: BLOOD TEST ALTERNATIVE TO BIOPSY

7

MORE THAN 2 MILLON PROSITE BOOSSES PER VEAR WORLDWIDE

• = 4Kscore is the only blood test that accurately identifies risk for aggressive prostate cancer
• · 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 predicts 20 year metastasis free survival for individual patient
• · Included in the 2015-2017 NCCN and 2016-2017 EAU Prostate Cancer Guidelines
• · Category I CPT published and effective January 1, 2017
• = >5,000 physicians have used the 4Kscore in practice; > 18,700 tests performed during 2Q17
• · 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

CLINICAL AND COMMERCIAL UPDATE

• Significant YOY growth ■
• · Novitas Solutions (Medicare Administrative Contractor for OPKO Elmwood Park, NJ facility)
  • · CMS national rate for 2017 \$602.10
  • · Novitas has been and continues to pay for 4Kscore Medicare submissions
• · Strong showing at the recent AUA meeting
• · 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 American men, who have the highest rates of prostate cancer mortality
  • · Preparing manuscript for publication in major urology journal
• · Deploying specialized sales force for Urology
• Initiating new sales incentive plan in U.S.
• Initiating TV advertisement in Northeast

4Kscore

OK

CLAROS 1 PLATFORM ADDRESSES LARGE POINT OF CARE TEST MARKET

25M PSA TESTS IN THE US ANNUALLY; \$625M MARKET OPPORTUNITY

• · Claros 1 can run immunoassay tests in the physician's office or hospital nurse's station using a single draw of blood from a finger stick
• · Negates the need for a full blood draw or a centralized reference lab for many common tests
• · Completed clinical study for PSA test in August 2017
• = Filing modular PMA with FDA for PSA test expected in 4Q17 and expect testosterone 510(k) filing in 2018
• · Claros 1 point of care platform will leverage BioReference Labs distribution and marketing
• · Menu expansion following initial FDA filings

ROBUST & LATE-STAGE DRUG PIPELINE

PRODUCT	INDICATION	PRECLINICAL	PHASE 1	PHASE 2	PHASE 3	MARKETED	MILESTONE
VARUBI (Rolapitant)	CINV	Out-licensed to Tesaro					IV Launch 4Q 2017 EU Oral Launch 2H 2017
Rayaldee® (CTAP101)	SHPT (CKD stage 3-4)	Partnered with Vifor Fresenius Ex U.S.					68% of U.S. potential lives have access; anticipate 70% by year end
Rayaldee® (CTAP101)	SHPT (CKD stage 5)	Partnered with Vifor Fresenius					Ph 2 in 4Q 2017
hGH-CTP (Somatrogon)	hGH deficiency	Collaboration with Pfizer					Adult Ph 3 completed YE16 Pediatric Ph 3 Initiated YE16
Alpharen ™ (Fermagate)	Hyperphosphatemia (CKD stage 5 patients)						Ph 3 in 1H 2018
OPK88003 (Oxyntomodulin)	Diabetes, obesity						Ph 2 in 1H 2018
OPK88004 (SARM)	BPH						Ph 2 in 4Q 2017
OPK88002 (NK1)	Pruritus (itching)						Ph 2a 4Q 2017
OPK88005 (Factor Vila-CTP)	Hemophilia A & B						Ph 2a ongoing
OPK88001 (AntagoNAT)	Dravet Syndrome						Ph 2a in 4Q 2017

10

RAYALDEE

CHRONIC KIDNEY DISEASE -THE SILENT KILLER

• · CKD is the 9th leading cause of death, ahead of breast and prostate cancer
• · CKD prevalence is expected to increase due to obesity, diabetes and hypertension
• · Most CKD patients die from cardiovascular disease (CVD), 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
• · Nutritional vitamin D is ineffective for treating VDI and SHPT in CKD, but is the current "standard of care"
• · Vitamin D receptor activators (VDRAs) are approved for SHPT in CKD but drive vascular calcification.
• · The 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

RAY ALDEE OVERVIEW

PRODUCT LAUNCHED NOVEMBER 29, 2016

• · Extended-Release (1x daily) oral formulation of 25D2 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 (~12M patients in US)
• · Potential for new indications including stage 5 CKD, institutionalized elderly, osteoporosis and cancer

* 25-Hydroxyvitamin D3 or Calcifediol

RAYALDEE SENDS A CLEAR MESSAGE

RAYALDEE COMMERCIALIZATION

• · Total prescriptions in Q2 increased by 140% compared to 1Q 2017
• · Number of nephrologists prescribing Rayladee in Q2 has almost doubled compared to Q1
• = 50-person sales and marketing team launched Rayaldee on November 30, 2016
  • · Increasing field sales force from 35 to 70 reps roll out expected October 2017
• · Comprehensive ongoing market education campaign highlighting the unmet need re: SHPT
• " Leveraging KOL advocates in community outreach (i.e., Speaker Bureaus and Patient Advocacy)
• · Commercial and Part D insurance under contract for >68% of U.S. covered lives
  • · Growing to more than 70% by end of 2017
• · Initial line extension plans
  • · Clinical trials for stage 5 CKD to begin 4Q17

SARM-SELECTIVE ANDROGEN RECEPTOR MODULATOR OPK-88004 -BENIGN PROSTATIC HYPERTROPHY (BPH)

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
• · Currently in Phase 2 study in prostate cancer patients who have undergone radical prostatectorny
• · BPH affects approximately 50 million men in the U.S.

NEXT STEP:

• · Begin Phase 2 trial in 4Q2017 to determine optimal dose to treat BPH
• · Study will be a 4 month treatment period involving 80 to 120 BPH patients
• · Will also examine for improvement in secondary endpoints:
  • PSA .
  • · Lean body mass
  • Fat mass
  • · Physical function

hGH-CTP COMPETITIVE ADVANTAGES

PARTNERED WITH PFIZER

• = New molecular entity (NME) that maintains natural native sequence of growth hormone
• = Once weekly injection vs. current products requiring daily injections
• · Human growth hormone is used for:
  • · Growth hormone deficient children and adults
  • · SGA, PWS, ISS
• · Final presentation:
  • · Refrigerated, liquid, non viscous formulation
  • · Disposable easy to handle pen injection device with thin needle and small injection volume
• 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
• Orphan drug designation in the U.S. and the EU for children and adults

hGH-CTP 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 CROs selected; sites initiated in December
  • · Easy-to-use, disposable, refrigerated pen device

· Phase 3 adult hGH-CTP

• · In December 2016 reported that primary endpoint of change in trunk fat mass from baseline to 26 weeks did not demonstrate a statistical significance between the hGH-CTP treated group and placebo
• · Completed post hoc outlier analysis in June 2017 to assess the influence of outliers on the primary endpoint results
• · Analyses which excluded outliers showed a statistically significant difference between hGH-CTP and placebo on the change in trunk fat mass; additional analyses that did not exclude outliers showed mixed results
• · No safety concerns
• · OPKO and Pfizer have agreed that OPKO may proceed with a pre-BLA meeting with FDA to discuss a submission plan
• · OPKO plans to carry out an additional study in adults using a pen device
• " Initiated pediatric hGH-CTP registration study in Japan
  • · 44 patients, comparison of weekly hGH-CTP to daily growth hormone
  • · Same pen device, dosage and formulation used in global study

LONG-ACTING FACTOR VIIA-CTP FOR HEMOPHILIA A & B

\$1.7 billion market growing 7% annually and only 25% of patients are treated

• · Current product (NovoSeven®) requires frequent IV doses
  • · 3-4 times a day during bleeding episodes
  • 1-2 times a day for prophylactic treatment
• · In pharmacological studies in hemophilic mice and dogs, Factor VIIa-CTP:
  • · Demonstrated potential for subcutaneous administration
  • · Reduced frequency of injection during on-demand therapy
  • · Enabled prophylactic treatment while reducing the injection frequency to 2-3 times a week
• · Commenced a Phase 2a single intravenous injection (1/) dose escalating study in January 2016 to evaluate the safety, PK and PD properties of Factor VIIa
• · Commenced a Phase 1 single dose subcutaneous (SC) administered, dose escalating study in December 2016 to evaluate the safety, PK and PD properties of Factor VIIa when administered by SC injection
• · Top line data from both studies as well as presented at the International Society on Thrombosis and Haemostasis in June
  • · Factor VIIA-CTP in both IV and SC administration demonstrated a favorable safety profile and local tolerance following single administration.
  • No unexpected adverse events related to the drug
• · Orphan drug designation in the U.S. and the EU

18

OPK-88003 OXYNTOMODULIN ANALOG

FOR THE TREATMENT OF TYPE 2 DIABETES AND OBESITY

• = 26 million diabetics in US: drug development focused on: blood glucose control and reducing body weight
• · OPK-88003 is an once-weekly analog with both GLP-1 and glucagon activity
• · Data support that combining GLP-1 and glucagon activity provides superior weight loss
• " Data from phase 2 study in 420 type 2 diabetes patients (week 12):

OPK-88003 CLINICAL DEVELOPMENT

• · Phase 2b dose-escalation study expected to start 1H18
• · Preparation of pen and formulation for phase 3 is ongoing

OPK

FOR THE TREATMENT OF PRURITUS IN DIALYSIS PATIENTS

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
• · 70 to 90% of patients Kidney dialysis patients suffer from Pruritus

PHASE 2a CLNICAL STUDY

Expected to begin in 4Q2017 트

SELECT FINANCIAL INFORMATION

Balance sheet at 6/30/2017	· Cash, cash equivalents & marketable securities: \$130.5 million Net investments: \$34.5 million Current portion of line of credit and notes payable: \$14.5 million · Senior notes (net of embedded derivatives): \$34.8 million
Capital structure at 6/30/2107	· Common shares outstanding: 559,995,118
Revenues	" Three months ended June 30, 2017 were \$314.2 million compared to \$357.1 million for the comparable period of 2016. · 2016 period includes a \$50 million upfront payment for Rayaldee from Vifor Fresenius

opko

UPCOMING MILESTONES

OPKC

PROGRESS ACROSS MULTIPLE BUSINESS AREAS

• · Expand Rayaldee field sales force
• · SARM Phase 2
• · Claros 1 PSA clinical study
• · Claros 1 testosterone clinical study
• · Oxyntomodulin Phase 2b
• ✓ hGH-CTP Phase 3 Pediatric
• · Rayaldee Stage 5 CKD Phase 2
• · AntagoNAT Dravet Phase 2b
• = NK-1 Antagonist Pruritus Phase 2a
• V Pediatric hgh-CTP registration study in Japan

October 2017 Initiate in 4017 PMA filing 4Q17 Initiate 1H18 Initiate 1H18 Enrollment ongoing Initiate 4Q17 Initiate 4Q17 Initiate 4Q17 Initiated 3Q17

OPKO THANK YOU