Opko Health, Inc. · NASDAQ:OPK
Opko Health — Investor Presentation (September 2015)
36 slides from Opko Health's investor presentation, Sep 1, 2015
Slide 1: September 2015
Exhibit 99.1 September 2015 1
Slide 2: Cautionary Statement
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,” “sho
Slide 3: What's New?
What’s New? • Completed Acquisition of Bio-Reference Laboratories August 20, 2015 • Acquired EirGen Pharma, a Growing, Profitable Specialty Pharmaceutical Developer and Manufacturer • Rayaldee™ PDUFA Date – March 29, 2016 • 4Kscore® Included in NCCN Guidelines - CPT Code Became Active on July 1, 201
Slide 4: A High-Growth Diversified Medical Products Company
A High-Growth Diversified Medical Products Company - Bio Reference Laboratories – a unique clinical lab - 4Kscore® blood test for aggressive prostate cancer risk Diagnostics - Claros 1® immunoassay system for rapid, lab quality in-office testing - Vitamin D therapeutics for SHPT - Platform technolog
Slide 5: Acquired EirGen Pharma, Ltd.
Acquired EirGen Pharma, Ltd. • Developer of High Potency Specialty Pharmaceutical Products – Growing, profitable and cash flow positive – Cancer chemotherapy and other niche, high potency drugs require special expertise and infrastructure - significant barriers to entry • Rich Pipeline of Products –
Slide 6: Bio-Reference is a Unique Clinical Lab Asset
Bio-Reference is a Unique Clinical Lab Asset – Acquisition closed August 20, 2015 1 Demonstrated strong, consistent organic growth - 21 years of ~20% compound annual revenue growth 2 Created and expanded franchises in multiple specialty markets including oncology, women’s health and genetics in lab
Slide 7: Transaction Rationale: Near-term
1 Transaction Rationale: Near-term Leverage BRLI’s channels to accelerate the adoption of OPKO’s diagnostic products Extensive phlebotomy draw stations offer synergistic opportunity for efficient commercialization of 4Kscore test for high-grade prostate cancer ~175 BRLI patient service centers locat
Slide 8: Transaction Rationale: Longer-term
2 Transaction Rationale: Longer-term Utilization of genomic data for personalized therapy BRLI’s vast array of genetics and genomics data should benefit OPKO in its drug discovery and clinical trial programs GeneDx was the first commercial laboratory to offer next generation sequencing for panels Of
Slide 9: Bio-Reference has Developed Capability in Specialty Areas
Bio-Reference has Developed Capability in Specialty Areas Routine Clinical Laboratorio Buena Oncology Women’s Health Genetics Testing Salud Target Markets: Target Markets: Target Markets: Target Markets: Target Markets: • Physician Offices • Medical Clinics • Hematologists • Obstetricians •Geneticis
Slide 10: The 4Kscore Test as a Minimally Invasive Alternative to Prostate Biopsy
The 4Kscore Test as a Minimally Invasive Alternative to Prostate Biopsy Clinical utility is based on three decades of clinical biomarker research and over 20,000 men tested in Europe and the US Identifies the actual risk of aggressive prostate cancer for the individual patient with: High grade prost
Slide 11: What is the 4Kscore Test?
What is the 4Kscore Test? Components OPKO ALGORITHM Results 4 kallikrein levels: Total PSA % risk of having Free PSA aggressive prostate cancer OPKO { Intact PSA hK2 for an individual patient + Age, DRE, and prior biopsy status 11
Slide 12: PSA Screenings and Biopsy Pathway
700,000 PSA Screenings 4,000 Serious 100,000 140,000 Infections Standard TRUS or Abnormal PSA or DRE mp MRI-Guided Biopsy Biopsy Result 55,000 20,000 25,000 Negative Biopsy Gleason score 7 Histopathology Histopathology Gleason score 6 OncotypeDx Histopathology or Prolaris 7,500 17,500 Monitor w PSA
Slide 13: $143 M net savings / $1,400 per patient
700,000 PSA Screenings 100,000 Prostate Biopsy Patients 36% Biopsy Reduction $78M Savings Standard TRUS or 51% Reduction$22 mp MRI-Guided Biopsy M Savings 29% Reduction$16 M Savings Biopsy Result Negative Biopsy Gleason score 7 Histopathology Histopathology Gleason score 6 OncotypeDx Histopathology
Slide 14: The 4Kscore Test Predicts Metastases Within 20 years
The 4Kscore Test Predicts Metastases Within 20 years¹ 16% 4Kscore Test 7.5% 4Kscore Test 7.5% < 2% ¹ Improving the Specificity of Screening for Lethal Prostate Cancer Using Prostate-specific Antigen and a Panel of Kallikrein Markers: A Nested Case–Control Study European Urology (in press) 14
Slide 15: 4Kscore Commercial Update
4Kscore Commercial Update • 4Kscore ProtecT study published – over 6,000 subjects – Total now 22,000 subjects studied with 4K panel • Over 1,000 US urologists have used the 4Kscore test in routine practice • American Urological Society Meeting in New Orleans May, 2015 – six podium or poster presenta
Slide 16: Claros 1: Rapid Testing in the Physician Office
Claros ®1: Rapid Testing in the Physician Office 1-2 10 mins mins Snap Insert cassette Finger stick Quantitative cartridge into into Claros®1 blood sample Results Sangia™ analyzer cassette 16
Slide 17: Claros 1 Platform Addresses Large Testing Markets
Claros 1 Platform Addresses Large Testing Markets • PSA – US test volume: 30 million tests, $750 M – Intended use to focus on detection claim – Modular PMA filing with FDA in 2016 • Other Test Menus in Development – Aligned with BRL specialty sales focus • Sectors – Oncology – Women’s Health – Healt
Slide 18: OPKO Pharmaceuticals–Advanced, Deep Pipeline
OPKO Pharmaceuticals–Advanced, Deep Pipeline Product Indication Preclinical Phase 1 Phase 2 Phase 3 Milestone Market Size SHPT PDUFA date Rayaldee™ (CKD Stage 3-4 March 29, $12.0 BN (CTAP101) Patients) 2016 hGH-CTP hGH deficiency Collaboration with Pfizer $3.0 BN Alpharen™ Hyperphosphatemia $1.2 BN
Slide 19: Rayaldee – A Late-Stage Investigational Drug
Rayaldee – A Late-Stage Investigational Drug Product Overview • Modified-release (MR) oral formulation of 25D 3 * addresses significant unmet market need • Safe and effective treatment for elevated PTH (SHPT) associated with low 25D levels in Stages 3–4 CKD • Achieves more reliable increases in seru
Slide 20: Market Opportunity: Chronic Kidney Disease (US)
Market Opportunity: Chronic Kidney Disease (US) • The CKD patient population is large and growing as a result of: – Obesity – Hypertension – Diabetes % of CKD Patients with Vitamin D SHPT Hyperphosphatemia Stage Kidney Function CKD Prevalence Insufficiency ( 25D) ( PTH) ( Phosphorus) Moderate 3 impa
Slide 21: Comparison of Vitamin D Therapies for Stage 3-4 CKD
Comparison of Vitamin D Therapies for Stage 3-4 CKD Effect on Blood Levels of: Drug Active Type 25D ** Ca iPTH Rayaldee Calcifediol Rx (25-hydroxyvitamin D3) Cholecalciferol/Ergocalciferol OTC Vitamin D (vitamin D3/vitamin D2) Drisdol ™* Ergocalciferol Rx (vitamin D2) Rocaltrol ™* Calcitriol Rx (1a,
Slide 22: Rayaldee - Commercial Opportunity
Rayaldee - Commercial Opportunity Stage 3 & 4 CKD Treatment for SHPT Vitamin D Hormone 14-25% Untreated Safety concerns; exacerbates vitamin D 31-47% insufficiency Untreated 26-44% Nutritional Vitamin D 39-44% Efficacy Concerns Rayaldee is expected to take significant market share in Stage 3 and 4 C
Slide 23: Rayaldee Top-Line Phase 3 Data: Plasma iPTH
Rayaldee Top-Line Phase 3 Data: Plasma iPTH 23
Slide 24: Rayaldee Top-Line Phase 3 Data: Serum 25D
Rayaldee Top-Line Phase 3 Data: Serum 25D 24
Slide 25: Rayaldee Top-Line Phase 3 Data: Serum Ca & P
Rayaldee Top-Line Phase 3 Data: Serum Ca &P Placebo Placebo to Modified-Release Calcifediol Modified-Release Calcifediol Modified-Release Calcifediol Continuation 10.0 Placebo-Controlled Extension * * * * * 9.0 Serum Calcium 8.0 7.0 6.0 5.0 Serum Phosphorus 4.0 * * 3.0 0 4 8 12 16 20 24 28 32 36 40
Slide 26: Rayaldee – Steps to Commercialization
Rayaldee – Steps to Commercialization • NDA filing (accepted for full FDA review) in late July • Marketing & sales management team to be hired in Q3-Q4 2015 • PDUFA date: March 29, 2016 • Launch expected within 3 months of PDUFA date • Initial line extension plans: – Additional phase 3 clinical tria
Slide 27: OPKO Biologics: Extends the Half-Life of Protein Drugs
OPKO Biologics: Extends the Half-Life of Protein Drugs Developing biobetter long acting proteins and peptides Reversible Pegylation CTP Technology Technology • Significant reduction in injection frequency • Safety profile comparable to non-modified active therapeutic agents • Potential expanded prop
Slide 28: CTP Increases Protein Circulation Time
CTP Increases Protein Circulation Time CTP – A Natural Chemical Entity Created During Evolution to Enhance Longevity of the Hormone hCG + = Any Short-Lasting CTP Long-Lasting Protein Protein 28
Slide 29: Global Collaboration with Pfizer for OPKO's Long-Acting hGH
Global Collaboration with Pfizer for OPKO’s Long- Acting Human Growth Hormone (hGH-CTP) Collaboration Terms: – $295 M up-front payment Financial – $275 M for achievement of regulatory based milestones – OPKO responsible for funding development program for the key Development indications: • Adult and
Slide 30: hGH-CTP Competitive Advantages
hGH-CTP Competitive Advantages • New molecular entity and maintain natural native sequence of GH • Human growth hormone is used for: – Growth hormone deficient children – Growth hormone deficient adults – SGA, PWS, ISS • Once-a-week injection (current products require daily injections) • Final Prese
Slide 31: Daily hGH Market is $3B, Undifferentiated and Growing
Daily hGH Market is $3B, Undifferentiated and Growing Established and growing Fragmented Others Roche 6% (Nutropin) 12% Novo Nordisk ~$4B (Norditropin) 31% Merck KGaA (Saizen, $3B+ Serostim) Other 8% Adult GHD Sandoz ISS, Turner (Omnitrope) Syndrome 8% 80% Pediatric Pediatric Eli Lilly GHD GHD (Huma
Slide 32: MOD-5014: Long-acting Factor VIIa for Hemophilia A & B
MOD-5014: Long-acting Factor VIIa for Hemophilia A & B • $1.7 billion market – Growing 7% annually – 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 • Pharmacological st
Slide 33: Oxyntomodulin – Nature's Appetite Control Mechanism
Oxyntomodulin – Nature’s Appetite Control Mechanism • Natural appetite suppressor • Oxyntomodulin is 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 to
Slide 34: MOD 6031: Meets Unmet Market Need
MOD 6031: Meets Unmet Market Need • MOD-6031 significantly inhibits food intake and reduces body weight by reduction in fat • MOD-6031 improves glycemic control by inducing glucose dependent insulin secretion (direct mechanism) and by reducing fat (indirect mechanism) • MOD-6031 improves lipid profi
Slide 35: Rolapitant – Potential Near-Term Revenue Driver
Rolapitant – Potential Near-Term Revenue Driver • Rolapitant out-licensed to Tesaro in December 2010 – Payments of up to $121 million – Double-digit tiered royalties • Differentiated cancer supportive care product with $1.5B US Market Opportunity – Potent neurokinin-1(NK-1) receptor antagonist for c
Slide 36: Strategic Investments
Strategic Investments Proprietary Technologies with Significant Upside Potential • ARNO Therapeutics, Inc. (OTC: ARNI) (~4% equity interest) – Anti-progestin therapy for breast (phase 2) , endometrial and prostate cancers • Zebra Biologics, Inc. (~27% equity interest) – Combinatorial antibody librar