Opko Health, Inc. · NASDAQ:OPK
Opko Health — 33rd Annual J.P. Morgan Healthcare Conference 2015
22 slides from Opko Health's conference presentation, Jan 14, 2015
Slide 1: 33rd Annual J.P. Morgan Healthcare Conference, January 2015
Exhibit 99.1 rd 33 Annual J.P. Morgan Healthcare Conference January 2015
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: OPKO Health: Focus on Large Market Potential Products
OPKO Health Focus on Large Market Potential Products Diagnostics – Claros ®1 immunoassay system for rapid, lab quality in-office testing – 4Kscore™ blood test for aggressive prostate cancer risk – New Vitamin D products for SHPT – Platform technologies to make peptides and proteins long-acting Pharm
Slide 4: 4Kscore Test to Identify Risk for Aggressive Prostate Cancer
4Kscore Test to Identify Risk for Aggressive Prostate Cancer • Two types of prostate cancer: – Low grade, non-lethal – Aggressive, lethal • 4Kscore: ONLY blood test to predict risk of aggressive form • Backed by 10 years of clinical research at Memorial Sloan- Kettering Cancer Center • Validated by
Slide 5: 4Kscore Commercial Update
4Kscore Commercial Update • 4Kscore US clinical trial manuscript in press (Eur. Urol.) • 4Kscore CPT code application accepted by AMA CPT Editorial Board (MAAA Category 3) • 4Kscore was discussed at NCCN Guidelines meeting Nov. 2014 for potential inclusion in 2015 guidelines • Over 465 US urologists
Slide 6: Claros 1: Rapid Testing in the Physician Office
Claros ®1: Rapid Testing in the Physician Office 1-2 10 mins mins Finger stick blood sample 6
Slide 7: Claros 1 Platform Addresses Large Dx Markets
Claros 1 Platform Addresses Large Dx Markets • Testosterone – 15 million US tests: $525 M – 510(k) filing in 2Q2015 • PSA – 30 million US tests: $750 M – Intended use to focus on screening (early detection) claim – PMA filing in 1Q2016 • Vitamin D – 70 million US tests: $3.5 B – On track to support
Slide 8: OPKO Pharmaceuticals — Advanced, Deep Pipeline
OPKO Pharmaceuticals — Advanced, Deep Pipeline Product Indication Preclinical Phase 1 Phase 2 Phase 3 Milestone Market Size Phase 3 results Rayaldee™ SHPT reported $12.0 BN (CTAP101) (CKD Stage 3-4 Patients) 3Q 2014 hGH-CTP hGH deficiency $3.5 BN Collaboration with Pfizer Alpharen™ Hyperphosphatemia
Slide 9: Rayaldee (CTAP101) – A Late-Stage Investigational Drug
Rayaldee (CTAP101) – A Late-Stage Investigational Drug Product Overview Clinical Status • Oral formulation of 25D 3* addresses significant unmet • Clinical development guided need by prominent Scientific Advisory Board • Achieves reliable increases in serum 25D and reductions in • Positive Phase 3 d
Slide 10: Market Opportunity: Chronic Kidney Disease (U.S.)
Market Opportunity: Chronic Kidney Disease (U.S.) 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) 3 Moderate impa
Slide 11: Rayaldee - Commercial Opportunity
Rayaldee - Commercial Opportunity Low serum 25D and elevated plasma PTH are prevalent in CKD Stage 3-4 patients – 20 million CKD Stage 3-4 patients in the US – 8 million patients with low serum 25D and high plasma PTH Stage 3 & 4 CKD Treatment Vitamin D Hormone Safety concerns; 14-25% exacerbates vi
Slide 12: 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 D 3 ) Vitamin D Cholecalciferol/Ergocalciferol OTC (vitamin D 3 /vitamin D 2 ) Drisdol ™* Ergocalciferol Rx (vitamin D 2 ) Rocaltrol ™* Calcitriol
Slide 13: OPKO Biologics: Leader in BioBetter Drugs
OPKO Biologics: Leader in BioBetter Drugs Developing biobetter long acting proteins and peptides Reversible Pegylation Technology CTP Technology (Peptides and small molecules) (Recombinant Proteins) • Dramatically reduce injection frequency • Reduce drug load • Reduce potential side-effects for most
Slide 14: hGH-CTP – The Long Acting hGH
hGH-CTP – The Long Acting hGH + = Any Short-Lasting CTP Long-Lasting Protein Protein Natural sequence No need for linker 14
Slide 15: Global Collaboration with Pfizer for hGH-CTP
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 16: hGH-CTP Opportunity
hGH-CTP Opportunity • $3.5 billion market, growing 5% annually • Once-a-week injection (current products require daily injections) • Small needle size (31 gauge) due to low viscosity – Competitive long acting formulations have high viscosity • Superb clinical, safety and immunogenicity profile • Hum
Slide 17: hGH-CTP Clinical Development
hGH-CTP Clinical Development Adult Pivotal Phase 3 trial (ongoing) • Primary efficacy endpoint: reduction in truncal fat mass after 6 months vs. placebo • Secondary efficacy endpoints include: – Reduction in total body fat – Increase in lean body mass • Single pivotal trial required by FDA for BLA s
Slide 18: hGH-CTP Clinical Development
hGH-CTP Clinical Development Pediatric GHD Phase 2 trial (advanced stage) • 4 Cohorts: – 3 dose levels of once-weekly hGH-CTP – Commercially available standard daily rhGH treatment • Key outcome: height velocity • Positive clinical data presented at ENDO meeting June 2014 – All doses provided excell
Slide 19: FVIIa–CTP: Long Acting for Treating Hemophilic Patients
FVIIa –CTP: Long Acting for Treating Hemophilic Patients • $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
Slide 20: MOD-6031: Long Acting Oxyntomodulin for Obesity
MOD-6031: Long Acting Oxyntomodulin for Obesity • WHO estimates 500 million severely overweight or obese people • >$15 billion market - growing rapidly • Oxyntomodulin: Nature’s Appetite Control Mechanism – Natural appetite suppressor – Secreted by the digestive system following food intake and indu
Slide 21: 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 22: Strategic Investments
Strategic Investments Proprietary Technologies with Significant Upside Potential • ARNO Therapeutics, Inc. (OTC: ARNI) (~4% equity interest) – Anti-progestins for breast (phase 2), endometrial and prostate cancers • Zebra Biologics, Inc. (~19% equity interest) – Combinatorial antibody libraries base
OPKO Health — 33rd Annual J.P. Morgan Healthcare Conference 2015 on 6ix