Diagnostics & Pharmaceuticals for Large Markets with Unmet Needs

Transcription

1 FEBRUARY 2016 NYSE: OPK Diagnostics & Pharmaceuticals for Large Markets with Unmet Needs

2 FORWARD-LOOKING STATEMENTS This presentation contains forward-looking statements, as that term is defined under the Private Securities Litigation Reform Act of 1995 (PSLRA), which statements may be identified by words such as expects, plans, projects, will, may, anticipates, believes, should, intends, estimates, potential, and other words of similar meaning, including statements regarding our estimated revenues and financial projections, our ability to achieve high levels of growth, the potential for our products under development, the potential of the 4Kscore to reduce prostate biopsies by 60% and predict the risk of aggressive prostate cancer within twenty years, our ability to develop, test and launch new products, the expected timing of the clinical studies and regulatory approval for our products under development, the outcome of our clinical trials and validation studies and that such outcomes will support commercialization, the expected market penetration and size of the market for our products under development, including without limitation, Rolapitant, Rayaldee, hgh-ctp, the 4Kscore, Factor VIIa-CTP, oxyntomodulin, and our point-of-care diagnostic products, the potential benefits of our products under development, including whether the 4Kscore will predict the risk of distant metastases and result in 40-55% cost savings, the expected submission dates for the PMA for PSA and 510k for testosterone and expected launch date for each, that MOD-6031 will provide superior long-term therapy for obesity and Type II diabetes patients, our ability to successfully commercialize our product candidates such as Rolapitant, the 4Kscore, hgh-ctp and Rayaldee and whether Rayaldee will take significant market share in stage 3 and 4 CKD patients with SHPT, whether Rayaldee will raise serum total 25-hydroxyvitamin D (25D) more effectively than any over-the-counter (OTC) or prescription (Rx) products currently marketed without the risk of hypercalcemia, whether we can reach more than half of the CKD population with a small sales force, our ability to establish a sales and marketing and clinical support infrastructure for Rayaldee and the timeline for doing so, the expected PDUFA date and launch date for Rayaldee, our ability to develop Rayaldee for new indications including stage 5 CKD, institutionalized elderly, osteoporosis, and cancer, our ability to execute an ex-u.s. licensing agreement during 2016 for Rayaldee, expectations regarding patent coverage, the expected timing for commencing, completing and obtaining results for our clinical trials, the timing for release of trial data and seeking and obtaining FDA and European regulatory approvals as well as reimbursement coverage, the timing of commercial launch of our product candidates, and expectations about near term profitability. These forward-looking statements are only predictions and reflect our views as of the date they were made, and we undertake no obligation to update such statements. Such statements are subject to many risks and uncertainties that could cause our activities or actual results to differ materially from the activities and results anticipated in forward looking statements, including integration challenges with Bio-Reference, risks inherent in funding, developing and obtaining regulatory approvals of new, commercially viable and competitive products and treatments, the success of our collaboration with Pfizer, general market factors, competitive product development, product availability, federal and state regulations and legislation, and integration issues arising from the transactions, delays associated with development of novel technologies, unexpected difficulties and delays in validating and testing product candidates, the regulatory process for new products and indications, manufacturing issues that may arise, the cost of funding lengthy research programs, the need for and availability of additional capital, the possibility of infringing a third party s patents or other intellectual property rights, the uncertainty of obtaining patents covering our products and processes and in successfully enforcing them against third parties, and the possibility of litigation, among other factors, including all of the risks identified under the heading Risk Factors in our Annual Report on Form 10-K and other filings with the Securities and Exchange Commission. 2

3 OPKO Health is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large growth markets by leveraging its discovery, development and commercialization expertise. 3

4 INVESTMENT HIGHLIGHTS Diagnostics Pharmaceuticals Biologics Acquired Bio-Reference Laboratories 3Q 2015, >$800 million fiscal 2014 revenues 420-person salesforce drives higher-margin esoteric testing, ~70% of revenues Facilitates uptake of 4Kscore prostate cancer test and Claros 1 in-office platform Rayaldee addresses unmet need in ~$12 billion CKD market, ~9 million patients VARUBI (Tesaro) $95 million in milestones, double-digit royalty, ~$1 billion market Long-term focus on high-value products including orphan diseases hgh-ctp (Pfizer) 1x/week hgh, $570 million total pre-commercialization milestones; double-digit royalties on adult sales, profit-sharing on pediatric sales, ~$3 billion growing market Once-daily Factor VIIa-CTP Phase 2a currently enrolling, ~$1.7 billion market Platform technologies for extending circulating time of biologics Near-term Milestones Execution Strategy and Execution Rayaldee PDUFA, Launch March 29, 2016, 2H Kscore reimbursement On-going Factor VIIa Phase 2a 1Q 2016 Oxyntomodulin Phase 1 initiation 1Q 2016 hgh-ctp topline Phase 3 data 2H 2016 (adults) Management team with a track record of success and access to capital Commitment to opportunistic business development Production and distribution assets worldwide, multiple strategic investments 4

5 BIO-REFERENCE LABS: NEAR-TERM OPPORTUNITIES LEVERAGING ESTABLISHED CHANNELS TO ACCELERATE ADOPTION OF NEW TESTS AND PRODUCTS Bio-Reference Labs is the third largest full-service reference laboratory in the U.S. Strategic rationale: Leverage the national marketing, sales and distribution resources to drive rapid and widespread uptake of OPKO s diagnostic platforms ~420 sales and marketing personnel ~5,000 people working together to support the needs of clients and patients ~180+ patient service centers located throughout the U.S. Leveraging Bio-Reference Commercial Infrastructure for 4Kscore Validated the 4Kscore test in blood serum at Bio-Reference s Elmwood Park, NJ facility BioReference sales training completed for ~200 sales representatives 4Kscore test reimbursement continues to expand 5

6 4KSCORE TEST: MINIMALLY INVASIVE ALTERNATIVE TO BIOPSY MORE THAN 2 MILLION PROSTATE BIOPSIES PER YEAR WORLDWIDE 4Kscore is the only blood test that accurately identifies risk for aggressive prostate cancer Clinical utility is based on decades of biomarker research and more than 20,000 men tested in Europe and the U.S. Long term outcomes data shows 4Kscore predicts 20 year metastasis-free survival for the individual patient Inclusion in NCCN Guidelines and Category I CPT published and effective January 1, 2017 More than 1,500 urologists have used the 4Kscore in routine practice Primary care physicians beginning to order test Health economics study shows a 40 55% cost savings to avoid unnecessary MRI, prostate biopsies, and add treatment or monitoring of indolent cancer Clinical utility study completed 6

7 Clinical Utility Study was designed to assess the influence of the 4Kscore Test on the decision to perform prostate biopsy at 35 urology clinics with a total of 611 men referred due to abnormal PSA and/or DRE Reduces prostate biopsy procedures by over 60% Influences 89% of decisions about performing a prostate biopsy Konety B. et al. Rev. Urol (4)

8 4KSCORE INFLUENCES CLINICAL DECISIONS REDUCES BIOPSIES Before you ordered the 4Kscore Test, were you considering a prostate biopsy for this patient? After you received the 4Kscore Test results, did the patient either a) have performed by you, b) get scheduled for, or c) get referred by you, to have a prostate biopsy within 90 days of the 4Kscore Test result? Do you feel the 4Kscore Test influenced the decision for the patient to have (or not have) a prostate biopsy? Konety B. et al. Rev. Urol (4)

9 CLAROS 1 PLATFORM ADDRESSES LARGE TESTING MARKETS 25M PSA TESTS IN THE US ANNUALLY; $625M MARKET FOR A SINGLE TEST Filing modular PMA with the FDA for rapid PSA test expected in 2016 and expect testosterone 510(k) filing 2H launch expected for both Claros 1 point-of-care platform will leverage Bio-Reference Labs distribution and marketing Menu expansion following initial FDA filings 9

10 ROBUST LATE-STAGE DRUG PIPELINE PRODUCT INDICATION PRECLINICAL PHASE 1 PHASE 2 PHASE 3 MILESTONE MARKET SIZE VARUBI (Rolapitant) CINV Out-licensed to Tesaro Launched 11/16/2015 $1.0 BN Rayaldee (CTAP101) SHPT (CKD stage 3-4 patients) PDUFA date 3/29/2016 $12.0 BN hgh-ctp hgh deficiency Collaboration with Pfizer Phase 3 data (adults) 2H 2016 $3.0 BN Alpharen (Fermagate) Hyperphosphatemia (CKD stage 5 patients) $1.2 BN CTAP201 Mild-to-moderate SHPT (CKD stage 5 patients) $1.1 BN Factor VIIa-CTP Hemophilia Phase 2a enrolling subjects $1.7 BN Oxyntomodulin Diabetes, obesity Phase I trial targeted for 1Q 2016 $15 BN AntagoNAT Platform Cancer, CV, metabolic and orphan disease $1.0 BN 10

11 RAYALDEE OVERVIEW PDUFA DATE MARCH 29, 2016; DRUG MET ALL PHASE 3 PRIMARY ENDPOINTS Product launch estimated 2H 2016 into a ~$12 billion market Modified-release oral formulation of 25D 3 * addresses significant unmet market need Indication: Treatment for elevated PTH (SHPT) associated with Vitamin D Insufficency (VDI) (low 25D levels) in stage 3-4 CKD Increases serum 25D and reduces plasma PTH No adverse effect on serum calcium or phosphorus (key drivers of vascular calcification) Existing therapies have efficacy and safety concerns Expected to take significant market share in stage 3-4 CKD patients with SHPT & (VDI) 9 million patients in the U.S. Potential for new indications including stage 5 CKD, institutionalized elderly, osteoporosis and cancer * 25-Hydroxyvitamin D 3 or Calcifediol ** 1,25-Dihydroxyvitamin D 3 or Calcitriol 11

12 RAYALDEE MARKET OPPORTUNITY The CKD patient population is large and growing as a result of: Obesity Hypertension Diabetes % of CKD Patients with Stage Kidney Function CKD Prevalence Vitamin D Insufficiency ( 25D) SHPT ( PTH) Hyperphosphatemia ( Phosphorus) 3 4 Moderate impairment Severe impairment 18.7 million* 71% 40% 37% 1.4 million* 83% 82% 50% 5 Failure 0.5 million* 97% 95% 70% *US Renal Data Service 2013 Annual Data Report Sources: Levin, A et al., Kidney International 2007; 71: pp.31-38; Gonzalez, E et al. Am J Nephrol 2004;24: ; LaClair, R et al. Am J Kidney Dis 2005;45: ; Tentori, F et al., Clin J Am Soc Nephrol 2015; 10:

13 RAYALDEE VS. ALTERNATIVE THERAPIES Effect on blood levels of: Drug Active Type 25D ** Ca ipth Rayaldee Vitamin D Drisdol * Rocaltrol * Hectorol * Zemplar * *And generics **25-hydroxyvitamin D Calcifediol (25-hydroxyvitamin D 3 ) Cholecalciferol/Ergocalciferol (vitamin D 3 /vitamin D 2 ) Ergocalciferol (vitamin D 2 ) Calcitriol (1α,25-dihydroxyvitamin D 3 ) Doxercalciferol (1α-hydroxyvitamin D 2 ) Paricalcitol (19-nor-1α,25-dihydroxyvitamin D 2 ) Rx OTC Rx Significant Increase Minor Increase Minor Increase No Change No Change No Change Rx Decrease Increase Rx Decrease Increase Rx Decrease Increase Significant Decrease Minor Decrease Minor Decrease Significant Decrease Significant Decrease Significant Decrease 13

14 RAYALDEE COMMERCIALIZATION NDA accepted for full review by FDA July 29, 2015 Build marketing and sales capabilities 1Q-2Q 2016 PDUFA date: March 29, 2016 Launch expected 2H 2016 Execute an ex-u.s. licensing agreement during 2016 Initial line-extension plans: Clinical trial planned in stage 5 CKD Initial clinical trial ongoing for new oncology indication Other indications being evaluated 14

15 VARUBI: NEAR-TERM REVENUE DRIVER COMMERCIAL LAUNCH - November 16, 2015 VARUBI (rolapitant) out-licensed to Tesaro in December 2010 Additional milestone payments of up to $95 million Double-digit tiered royalties Differentiated cancer supportive care product with >$1 billion U.S. market opportunity Potent neurokinin-1(nk-1) receptor antagonist for chemotherapy-induced nausea and vomiting (CINV) Opportunity to differentiate on convenience, market access and safety Long-acting, sustained effect Lack of CYP 3A4 drug-drug interactions Oral and IV formulations allow full market access 15

16 OPKO BIOLOGICS: EXTENDING THE HALF-LIFE OF PROTEINS Developing improved long-acting proteins and peptides Carboxyl Terminal Peptide (CTP) Technology Reversible Pegylation Technology Significant reduction in injection frequency CTP technology safety profile is comparable to non-modified active therapeutic agents Potential expanded prophylactic uses with longer-half-life agents, in addition to short-term treatment Maintain drug bioactivity 16

17 LONG-ACTING HUMAN GROWTH HORMONE (hgh-ctp) Terms of global collaboration with Pfizer: FINANCIAL $295 million in upfront payments to OPKO $275 million to OPKO upon achievement of regulatory milestones DEVELOPMENT COMMERCIAL OPKO responsible for funding development program for the key indications: Growth hormone deficiency (GHD) adult & pediatric Short for gestational age - pediatric Pfizer responsible for funding: Development programs for additional indications All post-marketing studies All commercialization activities Initial double-digit tiered royalties on sales of adult GHD Profit sharing commencing upon launch for pediatric GHD encompassing combined sales for all indications of hgh-ctp and Pfizer s Genotropin Genotropin represents about 23% of the global market with 2013 annual sales exceeding $700 million; total global market is ~$3 billion and growing 17

18 hgh-ctp COMPETITIVE ADVANTAGES PHASE 3 TOP LINE DATA IN ADULTS AVAILABLE 2H 2016 New molecular entity (NME) that maintains natural native sequence of growth hormone A key differentiator is once-weekly injection (current products require 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 a thin needle and low injection volume Phase 2 study completed in naive growth hormone deficiency pediatric population Phase 3 study to be initiated 2H 2016 in naive growth hormone deficiency pediatric population Orphan drug designation in the U.S. and the EU for children and adults 18

19 hgh MARKET IS ~$3 BILLION, UNDIFFERENTIATED, GROWING ESTABLISHED AND GROWING FRAGMENTED 80% $3B+ Other Adult GHD ISS, Turner syndrome Pediatric GHD ($1.5B) ~$4B Pediatric GHD Daily 2013 Daily 2018 Long-acting potential market Merck KGaA (Saizen, Serostim) 8% Sandoz (Omnitrope) 8% Roche (Nutropin) 12% Eli Lilly (Humatrope) 12% Others 6% Novo Nordisk (Norditropin) 31% Pfizer (Genotropin) 23% Projected market growth Competition based on history, service and device innovation 19

20 LONG-ACTING FACTOR VIIA-CTP FOR HEMOPHILIA A & B PHASE 2a STUDY CURRENTLY ENROLLING SUBJECTS - $1.7 BILLION MARKET Market is 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 Orphan drug designation in the U.S. and the EU 20

21 THE MOD-6031 SOLUTION Oxyntomodulin is a natural appetite suppressor and a dual GLP-1/ glucagon receptor agonist Secreted by the digestive system following food intake and induces satiety in the brain Crosses blood-brain barrier to induce satiety Increases glucose tolerance in insulin resistant pre-diabetic state associated with obesity DEVELOPMENT CHALLENGE Oxyntomodulin has short half-life that necessitates multiple daily injections OPKO SOLUTION: MOD-6031, a reversible PEG 30 formulation, provides for onceweekly dosing 21

22 MOD-6031 ADDRESSES SIGNIFICANT UNMET MARKET NEED MARKET SIZE FOR DIABETES AND OBESITY IS ~$15 BILLION MOD-6031 (in animal studies) Significantly inhibits food intake and reduces body weight by fat reduction Improves glycemic control by inducing glucose-dependent insulin secretion (direct mechanism) and by reducing fat (indirect mechanism) Phase 1 study to be initiated in 1Q 2016 Phase 1 study will evaluate the safety and PK/PD profile of MOD-6301 in overweight or obese healthy volunteers Improves lipid profile Expected to provide effective long-term therapy for obese people and for people with type 2 diabetes A battery of comprehensive toxicological studies has been completed confirming the safety of MOD-6031 following a single injection 22

23 SELECT FINANCIAL INFORMATION Balance sheet at 9/30/15 Cash, cash equivalents & marketable securities: $212.1 million Net investments: $34.7 million Current portion of credit and notes payable: $78.5 million Senior notes (net of embedded derivatives): $44.2 million Capital structure at 9/30/15 Common shares outstanding: 545,951,707 Potentially dilutive shares: 29,874,112 Revenue* 2015 vs Three months ended September 30: $143.0 million vs. $19.8 million Nine months ended September 30: $215.0 million vs. $65.6 million * 2015 revenue reflects acquisition of Bio-Reference Laboratories on August 20,

24 VALUE-CREATING MILESTONES Diagnostics Pharmaceuticals Biologics Granted Category 1 CPT code for 4Kscore test from AMA Obtain broad reimbursement for the 4Kscore test Leverage BRL sales force to drive 4Kscore test revenue (2016) Modular PMA for PSA with FDA for Claros 1 point-of-care diagnostic platform (2016) VARUBI oral formulation launched by partner Tesaro (November 2015) Rayaldee PDUFA date (1Q 16) Rayaldee launch (2H 16) Rayaldee ex-u.s. licensing (2016) Phase 2a study of Factor VIIa-CTP (1Q 16) Start Phase 1 trial of oxynotomodulin for treatment of obesity and diabetes (1Q 16) Phase 3 clinical trial results for hgh-ctp for adults (2H 16) Initiate pediatric Phase 3 clinical trial (2H 16) Strategy and Execution Execution Closed on a new 5-year $175.million secured revolving credit facility with JPMorgan Chase that replaced and improved upon BRL s previous facility Build Sales and Marketing infrastructure for Renal Division (1Q 16) 24

25 KEY TAKEAWAYS Core Base Business with Nearly $1 Billion in Revenue Advanced Deep Pipeline in Multibillion-Dollar Markets Solid Financial Position Execution Experienced Leadership with Strategic Vision and Ability to Execute 25

26 Diagnostics & Pharmaceuticals for Large Markets with Unmet Needs