Full Press Release Details
Diagnostics & Pharmaceuticals for Large Markets with Unmet Needs
January 2017 NASDAQ: OPK
FORWARD-LOOKING STATEMENTS
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, expected milestones and royalties from the outlicense of our products, our ability to achieve high levels of growth, the potential for our products under development, the potential of the
4Kscore to influence 89% of biopsy decisions 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 marketing approval or commercialization, the expected market penetration and size of the market for our products, including without
limitation, Rolapitant, Rayaldee , hGH-CTP, the 4Kscore, Factor VIIa-CTP, oxyntomodulin the SARM candidate, 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 oxyntomodulin 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, our ability to double our sales force
and the timeline for doing so, our ability to obtain commercial and Part D coverage for 70-80% of U.S. covered lives by mid-2017, our ability to develop Rayaldee for new indications including stage 5 CKD and the timeline for doing so, expectations
surrounding the sensitivity analysis for primary and secondary endpoints for the adult hGH-CTP study, whether the results of the analysis and any post-hoc efficacy analysis will be positive, whether the FDA would consider the analysis and whether
the drug will be approvable, whether we will be required to make any changes to our development plans for hGH-CTP, expectations regarding patent coverage, the expected timing for commencing, completing and announcing 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 for our products, our ability to obtain Medicare coverage for the 4Kscore and whether we have enough scientific
and clinical data to justify a positive coverage determination, expectations about our animal health business and the introduction of several OTC and prescription products for the animal market, and the timing of commercial launch of our product
candidates. 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 and other acquired businesses, 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, 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.
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
BioReference Rayaldee
SARM CKD BPH Fermagate Vitamin D Phosphate binder Stress Incontinence 4Kscore Tests
NK-1 Claros Claros NK-1
PSA Vitamin D Pruritus
Overactive Bladder Testosterone Factor VIIa
Growth hormone deficiency SARM
Increase muscle mass LA hGH Anolog GeneDx Decrease fat mass Agromegaly Increase bone mass IGF-1 CTP
receptor deficiency Oxyntomodulin
LA GLP-2 Type 2 diabetes
Short bowel syndrome Overweight
LA Parathyroid Hormone
Endocrinology Aging Metabolic Syndrome
DIVERSIFIED INVESTMENT HIGHLIGHTS
Bio-Reference Laboratories 3Q 2015, revenue of approximately $260 million in 3Q 2016 420-person salesforce drives industry-leading esoteric testing, ~70% of revenues Facilitates uptake of 4Kscore prostate cancer test and Claros 1 in office
platform Initiated clinical study on Claros 1 for PSA
Rayaldee addresses unmet need in ~$12 billion CKD
market, ~9 million patients Rayaldee license (VFMCRP); Up to $837 million in milestones, double digit royalties
VARUBI partnered with Tesaro; Up to $95 million in milestones, double digit royalties, ~$1 billion market Acquired
Transition Therapeutics SARM Phase 2b initiating in 2H17
Platform technologies for extending circulating time
of biologics 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 Long acting Factor VIIa-CTP Phase 2a ongoing, ~$1.7 billion market
Oxyntomodulin Phase 2b initiating in 2H17
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.
~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.
Over 12 million patients served during 2016
9M16 revenue ? $778 million; 3Q16 revenue ? $260 million
GeneDx is a genomics leader known for its expertise in rare disease and whole exome testing? Utilizing BRL commercial
infrastructure to drive 4Kscore and Claros 1 adoption
4KSCORE TEST: BLOOD TEST 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 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 and 2016 NCCN and 2016 EAU Prostate Cancer Guidelines Category I CPT published and effective January 1, 2017 >5,000 physicians have used
the 4Kscore in practice; > 16,000 tests performed during Q32016 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 TEST: REIMBURSEMENT UPDATE
PROGRESS WITH COMMERICIAL PAYORS; MEDICARE LOCAL COVERAGE DECISIONS EXPECTED
Obtained positive coverage
decision from one national payer? Obtained pricing agreements from several regional payers? CMS national rate for 2017 $602.10
Novitas Solutions (Medicare Administrative Contractor for OPKO Elmwood Park, NJ facility)
Initial draft positive coverage determination (LDC) tentatively retired due to a potential conflict with another MAC
Novitas has been and continues to pay for 4Kscore Medicare submissions
Expect Novitas to include 4Kscore in the February review cycle for a draft local coverage determination.
Palmetto GBA and CGS Administrators
Issued negative coverage determination
addressing concerns largely around clinical study protocols and clinical utility
OPKO confident it has now
supplied sufficient scientific and clinical data to justify positive LCD by any MAC
CLAROS 1 PLATFORM ADDRESSES LARGE POINT OF CARE TEST MARKET
25M PSA TESTS IN THE US ANNUALLY; $625M MARKET OPPORTUNITY
Initiated clinical study for PSA test in January 2017
Filing modular PMA with FDA for PSA test expected in 1H2017 and expect testosterone 510(k) filing 2H2017 Claros 1 point of care platform will leverage BioReference Labs distribution and
marketing Menu expansion following initial FDA filings
ROBUST & LATE-STAGE DRUG PIPELINE
INDICATION PRECLINICAL PHASE 1 PHASE 2 PHASE 3 MARKETED MILESTONE
VARUBI (Rolapitant) CINV Out-licensed to
Tesaro Launched Nov 2015
Partnered with Vifor Fresenius Ex U.S. Launched Nov 2016
(CTAP101) (CKD stage 3-4)
Rayaldee SHPT Ph 3 in 1H 2017
Partnered with Vifor Fresenius in U.S.
Adult Ph 3 completed YE16
hGH-CTP hGH deficiency Collaboration with Pfizer
Pediatric Ph 3 Initiated YE16
Alpharen Hyperphosphatemia