EX-99.1
Published on September 24, 2015
Exhibit 99.1
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Exhibit 99.1
SEPTEMBER 2015
Advancing Our Deep Pipeline of Pharmaceuticals and Diagnostics
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CAUTIONARY STATEMENT
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 and predict the risk of aggressive prostate cancer, 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 product for PSA, the potential benefits of our products under development, including whether the 4Kscore will improve selection of candidates for prostate biopsy, predict the risk of distant metastases, and result in 40-55% cost savings, the expected PMA submission date for PSA, expected per patient savings, 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) product 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, 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, and the timing of commercial launch of our product candidates, expectations about near term profitability, that EirGen will manufacture our current and future products resulting in higher margins, that Bio-References vast array of genetics and genomics data will benefit OPKO, as well as other non-historical statements. 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 partys 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.
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OPKO 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
OPKO is positioned for strength and growth in large target markets with significant unmet needs
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VALUE DRIVERS
GLOBAL AGREEMENT WITH PFIZER
OPKO and Pfizer entered Global Agreement for long acting human growth hormone (hGH-CTP) in December 2014
$295 million up front payments to OPKO;
Up to an additional $275 million in milestones prior to profit sharing
Pfizers Genotropin represents about 23% of the world market and has annual revenues exceeding $700 million; worldwide market is $3+ billion and growing
Profit sharing shall commence upon commercial launch for Pediatric
GHD encompassing combined sales for all indications of OPKOs hGH-CTP and Pfizers Genotropin
TRANSFORMATIVE DIAGNOSTICS
Acquisition of Bio Reference Labs creates near term opportunities for vertical integration with OPKOs diagnostic pipeline
Bio Reference Labs reaches 10 million patients annually and has grown over the past 20 years at a CAGR over 20%
Generated $832 million in profitable revenue last year as the third largest full service reference laboratory in U.S.
Will provide national marketing and distribution for OPKOs diagnostic pipeline with an initial focus on launching the 4Kscore and Claros 1® platform
RAYALDEES PDUFA DATE SET
OPKOs Rayaldee PDUFA date set for March 29, 2016
Addresses significant unmet market need in a potential $12 billion market
Rayaldee targets Stages 3-4 CKD, which has an estimated patient population of 20 million in the U.S.
TESARO RECEIVES FDA APPROVAL
OPKO licensee Tesaro receives FDA approval for VARUBI (rolapitant)
OPKO to receive up to $110 million of additional milestone payments
Double digit tiered royalties with a product launch targeted for Q4 2015
U.S. market opportunity exceeds $1 billion annually
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NEAR TERM CATALYSTS
SIZE OF
ADDRESSABLE COMPETITIVE
DATE POTENTIAL CATALYST MARKET ADVANTAGE
Coverage decisions on > $1 billion Greater accuracy;
2015 / 2016 Reimbursement for annual sales Only predictor of aggressive prostate cancer;
4Kscore test Simple non-invasive blood test
Commercial launch of > $1 billion Longer acting; sustained coverage; and IV;
Q4 2015 VARUBI (rolapitant) annual sales Allow full market access
Begin patient dosing in Only 25% of patients are currently treated;
Q4 2015 Phase 2a study of Factor $1.7 billion Orphan Drug designation in U.S. and EU;
VII-CTP reduced frequency of injection
Significant unmet market need for Stages 3-
March 29, 2016 FDA Approval of $12 billion 4 CKD (20 million patients)
Rayaldee
Phase 3 clinical trial Once a week injection versus daily
2H 2016 results for hGH-CTP for $3 billion injections; orphan drug designation in the
adults U.S. and EU
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OPKO PHARMACEUTICALS ADVANCED, DEEP PIPELINE
PRODUCT INDICATION PRECLINICAL PHASE 1 PHASE 2 PHASE 3 MILESTONE MARKET SIZE
PDUFA date
Rayaldee SHPT March 29, $12.0 BN
(CTAP101) (CKD Stage 3-4 Patients) 2016
hGH-CTP hGH deficiency Collaboration with Pfizer $3.0 BN
Alpharen Hyperphosphatemia $1.2 BN
(Fermagate) (CKD Stage 5 Patients)
VARUBI Outlicensed to TESARO Product
CINV Launch Q4 $1.0 BN
(rolapitant) 2015
CTAP201 Mild to moderate SHPT $1.1 BN
(CKD Stage 5 Patients)
Phase 2a
Factor VIIa-CTP Hemophilia trial in 4Q $1.7 BN
2015
Phase I trial
Oxyntomodulin Diabetes, Obesity targeted for $15 BN
Q1 2016
AntagoNAT Cancer, CV, metabolic and $1.0 BN
Platform orphan disease
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RAYALDEE
PDUFA DATE MARCH 29, 2016; MET ALL PHASE 3 ENDPOINTS
Product launch estimated 2H 2016 in a $12 billion market
Rayaldee is expected to take significant market share in Stage 3 and 4 CKD patients suffering from SHPT
20 million patients in the U.S.
Modified-release (MR) oral formulation of 25D3* addresses significant unmet market need
Safe and effective treatment for elevated PTH (SHPT) associated with low 25D levels in Stages 34 CKD
Existing therapies have efficacy and safety concerns
Achieves more reliable increases in serum 25D and reductions in plasma PTH than nutritional vitamin D
Lower risk of side effects compared to active 1,25D** products
Additional potential for new indications including stage 5 CKD, institutionalized elderly, osteoporosis & cancer
25-Hydroxyvitamin D3 or Calcifediol
1,25-Dihydroxyvitamin D3 or Calcitriol
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RAYALDEE STEPS TO COMMERCIALIZATION
NDA accepted for SHPT in Marketing & Sales
Stage 3 or 4 CKD and PDUFA Date:
Vitamin D Insufficency management team
being hired in Q4 2015 March 29, 2016
July 29, 2015
Initial line extension plans:
Additional phase 3 clinical trial(s)
Launch Expected planned in stage 5 CKD
Initial clinical trial ongoing for
2H 2016 new oncology indication
Other indications being evaluated
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RAYALDEEMARKET OPPORTUNITY
The Chronic Kidney Disease (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 SHPT Hyperphosphatemia
Insufficiency (?25D) (? PTH) (? Phosphorus)
Moderate
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18.7 Million* 71% 40% 37% |
impairment
Severe
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1.4 Million* 83% 82% 50% |
impairment
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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;24:503-510; LaClair, R et al. Am J Kidney Dis 2005;45:1026-1033; Tentori, F et al., Clin J Am Soc Nephrol 2015; 10:98-109
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RAYALDEE DIFFERENTIATED COMPARISON OF ALTERNATIVE THERAPIES
Effect on Blood Levels of:
Drug Active Type 25D** Ca iPTH
Rayaldee Calcifediol Rx Significant No Significant
(25-hydroxyvitamin D3 ) Increase Change Decrease
Vitamin D Cholecalciferol/Ergocalciferol OTC Minor No Minor
(vitamin D3 /vitamin D2 ) Increase Change Decrease
Drisdol* Ergocalciferol Rx Minor No Minor
(vitamin D2 ) Increase Change Decrease
Rocaltrol* Calcitriol Rx Decrease Increase Significant
(1á,25-dihydroxyvitamin D3 ) Decrease
Hectorol* Doxercalciferol Rx Decrease Increase Significant
(1á-hydroxyvitamin D2 ) Decrease
Zemplar* Paricalcitol Rx Decrease Increase Significant
(19-nor-1á,25-dihydroxyvitamin D2 ) Decrease
*And generics
**25-hydroxyvitamin D 10
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LONG-ACTING HUMAN GROWTH HORMONE (hGH-CTP)
Terms of Global Collaboration with Pfizer:
FINANCIAL $295 million up-front payments to OPKO
$275 million to OPKO upon achievement of regulatory based milestones
OPKO responsible for funding development program for the key indications:
DEVELOPMENT Adult and Pediatric Growth Hormone Deficiency (GHD)
Pediatric Short for Gestational Age
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
COMMERCIAL Profit sharing commencing upon launch for Pediatric GHD encompassing
combined sales for all indications of OPKOs hGH-CTP and Pfizers
Genotropin
Pfizer Genotropin represents about 23% of the world market with annual sales
exceeding $700 million; total worldwide market is $3+ billion and growing
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hGH-CTPCOMPETITIVE ADVANTAGES
PHASE 3 ENROLLMENT COMPLETED FOR ADULTS IN JULY 2015
New molecular entity that maintains natural native sequence of Growth Hormone
A key differentiator is a once-a-week injection (current products require daily injections)
Human growth hormone is used for:
Growth hormone deficient children and adults
SGA, PWS, ISS
Final Presentation:
The drug product will be a refrigerated, liquid non viscous formulation
Injected using a disposable easy to handle pen device with a thin needle and low injection volume Phase 2 study in naive growth hormone deficiency pediatric population has been completed Orphan drug designation in the U.S. & EU for the treatment of children and adults with GHD
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DAILY hGH MARKET IS $3B, UNDIFFERENTIATED, AND GROWING
ESTABLISHED AND GROWING
~$4B
$3B+
Other
Adult GHD
ISS, Turner
Syndrome
80% Pediatric Pediatric
GHD GHD
($1.5B)
Daily 2013 Daily 2018 Long-acting potential
market
Projected market growth
FRAGMENTED
Others
Roche 6%
(Nutropin)
12% Novo Nordisk
(Norditropin)
31%
Merck KGaA
(Saizen,
Serostim)
8%
Sandoz
(Omnitrope)
8%
Eli Lilly
(Humatrope) Pfizer
12% (Genotropin)
23%
Competition based on history, service and device innovation
Source: Market Research 2013 & 2014
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VARUBI (ROLAPITANT): NEAR-TERM REVENUE DRIVER
COMMERCIAL LAUNCH ANTICIPATED Q4 2015; FDA APPROVED ON SEPTEMBER 2, 2015
Rolapitant out-licensed to Tesaro in December 2010
Additional milestone payments of up to $110 million to OPKO
Double-digit tiered royalties for OPKO
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 coverage
Lack of CYP 3A4 drug-drug interactions
Oral and IV formulations allow full market access
¹ Moderately emetogenic chemotherapy
² Highly emetogenic chemotherapy
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BIO REFERENCE LABS ACQUISITION: NEAR TERM OPPORTUNITY
LEVERAGE BIO REFERENCES CHANNELS TO ACCELERATE ADOPTION OF OPKOS DIAGNOSTIC PRODUCTS
Bio Reference Labs is the third largest full service reference laboratory in the U.S.
Leverage the national marketing, sales, and distribution resources of Bio Reference Labs to enhance sales of OPKOs diagnostic platforms
~420 sales and marketing personnel
~5,000 people working together to support the needs of clients and patients ~175 BRLI patient service centers located throughout the U.S.
Bio Reference Labs generated $832 million in revenue at 44% gross margins in FY 2014 Has grown at a 20% CAGR over the past 20 years Will serve as a launching pad for the 4Kscore as reimbursement for the 4Kscore continues to be expanded
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4KSCORE TEST: MINIMALLY INVASIVE ALTERNATIVE TO PROSTATE BIOPSY
MORE THAN 2 MILLION BIOPSIES PER YEAR WORLDWIDE
4Kscore is the only blood test that accurately identifies risk for aggressive prostate cancer
Reimbursement to be expanded throughout the remainder of 2015 and 2016
Clinical utility is based on three decades of clinical biomarker research and over 20,000 men tested in Europe and the U.S.
Over 1,000 urologists have used the 4Kscore in routine practice
Identifies the actual risk of aggressive prostate cancer for the individual patient with high grade prostate cancer pathology and poor prostate cancer clinical outcomes within 20 years
4055% cost savings to potentially avoid unnecessary MRI and prostate biopsies
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THE 4KSCORE TEST PREDICTS METASTASES WITHIN 20 YEARS1
4Kscore Test > 7.5%
4Kscore Test 7.5%
Improving the Specificity of Screening for Lethal Prostate Cancer Using Prostate- specific Antigen and a Panel of Kallikrein Markers: A Nested CaseControl Study European Urology (in press)
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CLAROS 1 PLATFORM ADDRESSES LARGE TESTING MARKETS
30 MILLION PSA TESTS IN THE U.S. GENERATING $750 MILLION PER YEAR
Modular PMA filing with the FDA expected in 2016
Claros 1 Platform will leverage Bio Reference Labs distribution and marketing
Rapid Testing in the Physician Office with simple user steps:
Finger stick blood Snap cartridge into Insert cassette into Quantitative
sample Sangia cassette Claros®1 analyzer Results
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OPKO BIOLOGICS: EXTENDS THE HALF-LIFE OF PROTEIN DRUGS
Developing biobetter long acting
proteins and peptides
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 bio-activity
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MOD-5014: LONG-ACTING FACTOR VIIA FOR HEMOPHILIA A & B
PHASE 2a STUDY TO COMMENCE Q4 2015 IN $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, FVIIa-CTP demonstrated:
Potential for subcutaneous administration
Reduced frequency of injection during on-demand therapy
Enable prophylactic treatment while reducing the frequency of injections to 2-3 times a week
Orphan drug designation in the U.S. and EU
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MOD-6031MEETS 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 REDUCTION IN FAT
IMPROVES GLYCEMIC CONTROL BY INDUCING GLUCOSE DEPENDENT INSULIN SECRETION (DIRECT MECHANISM) AND BY REDUCING FAT (INDIRECT MECHANISM)
IMPROVES LIPID PROFILE
IS EXPECTED TO PROVIDE EFFECTIVE LONG-TERM THERAPY FOR OBESE AND TYPE II DIABETES PATIENTS
Phase 1 study to be initiated in Q1 2016
Phase 1 study will evaluate the safety and pk-pd profile of MOD-6301 in overweight or obese healthy volunteers
A battery of comprehensive toxicological studies have been completed confirming the safety of MOD-6031 following a single injection
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MOD-6031OPKOS 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 resistance pre-diabetic state associated with obesity
DEVELOPMENT CHALLENGE:
Oxyntomodulin has short half-life which requires multiple daily injections
OPKO SOLUTION:
MOD-6031, a reversible PEG30 formulation which provides for once-a-week dosing frequency
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