Opko Health, Inc. · NASDAQ:OPK
Opko Health — Corporate Update (June 2014)
17 slides from Opko Health's corporate update, Jun 17, 2014
Slide 1: Update on Phase 3 Development of Rayaldee Capsules
Exhibit 99.1 Update on Phase 3 Development of Rayaldee ™ Capsules June 12, 2014
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: Mission Statement (for Renal Division)
To Improve People’s Lives by Treating and Preventing the Clinical Consequences of Vitamin D Insufficiency and Secondary Hyperparathyroidism Mission Statement (for Renal Division)
Slide 4: Vitamin D Insufficiency
Vitamin D Insufficiency Obesity Bone Diseases Infection Diabetes Critical Illness Cancer Psoriasis Skeletal-related Events (SRE’s) CKD Cystic Fibrosis Diseases Associated with Vitamin D Insufficiency SHPT Autism SHPT = Secondary Hyperparathyroidism
Slide 5: What is Vitamin D Insufficiency?
Vitamin D 25 hydroxy- vitamin D 1,25 dihydroxy- vitamin D Ergocalciferol Cholecalciferol Vitamin D insufficiency is serum 25-hydroxyvitamin D 3 below 30 ng/mL Liver Enzyme Kidney Enzyme Limited and unreliable efficacy What is Vitamin D Insufficiency?
Slide 6: Vitamin D Insufficiency: Increases Mortality in Breast Cancer
Vitamin D Insufficiency: Increases Mortality in Breast Cancer Mohr et al 2014 Anticancer Res
Slide 7: Vitamin D Insufficiency: Increases Mortality in Colon Cancer
Garland CF et al. Ann Epidemiol 2009 Vitamin D Insufficiency: Increases Mortality in Colon Cancer ~32 ng/mL
Slide 8: Vitamin D Insufficiency: Increases Mortality in CKD
Vitamin D Insufficiency: Increases Mortality in CKD Ravani et al Kidney Int 2008
Slide 9: Rayaldee (CTAP101) – A Late-Stage Investigational Drug
Product Overview Clinical Status Intellectual Property Rayaldee (CTAP101) – A Late-Stage Investigational Drug • Modified-release (MR) oral formulation of 25D * addresses significant unmet market need • Safe and effective treatment for elevated PTH (SHPT) associated with low 25D levels in Stages 3-4
Slide 10: Market Opportunity: Chronic Kidney Disease (U.S.)
CKD Prevalence rates are similarly high outside of the U.S., and the population base is much larger. Market Opportunity: Chronic Kidney Disease (U.S.) *National Kidney Foundation 2002 **US Renal Data Service 2011 Annual Data Report Sources: Levin, A et al., Kidney International 2007; 71: pp.31-38. G
Slide 11: Comparison of Vitamin D Therapies for Stage 3-4 CKD
Effect on Blood Levels of: Rayaldee raises serum total 25-hydroxyvitamin D (25D) and lowers plasma iPTH more effectively than any over-the-counter (OTC) or prescription (Rx) product currently marketed without the risk of hypercalcemia. Comparison of Vitamin D Therapies for Stage 3-4 CKD *And generic
Slide 12: Rayaldee (CTAP101) - Commercial Opportunity
Untreated 26-44% Vitamin D Hormone 20-36% Untreated 26-44% Stage 3 & 4 CKD Treatment Rayaldee is expected to take significant market share in Stage 3 and 4 CKD patients suffering from SHPT – a potential $12 billion revenue opportunity Rayaldee (CTAP101) - Commercial Opportunity Source: BioTrends Res
Slide 13: Rayaldee Capsules: Positioning vs. Competitive Products
Vitamin D 25 hydroxy- vitamin D 1,25 dihydroxy - vitamin D Inactivated Vitamin D Ergocalciferol Cholecalciferol Calcitriol Paricalcitol Doxercalciferol Liver Enzyme Kidney Enzyme CYP24 Enzyme Parathyroid Hormone (iPTH) Feedback Loop Target Tissues (Parathyroid gland) Rayaldee TM Capsules Limited and
Slide 14: Rayaldee: Phase 3 and NDA Timelines
2012 2013 2014 SPA Open Label Extension (OLE) study 2015 SPA Stability studies on 3 registration lots Rayaldee: Phase 3 and NDA Timelines Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 NDA Filing CL-3002 Data CL-3001 trial Q1 Q2 Q3 Q4 NDA Safety Update NDA Approval TG Mouse carcinogenicity study Dose ranging s
Slide 15: CL-3001 & CL-3002 Combined Site Map
CL-3001 & CL-3002 Combined Site Map = Sites for CL-3001 (n=45 ) = Sites for CL-3002 (n=45)
Slide 16: Status Update: Phase 3 Trials (as of June 5, 2014)
Status Update: Phase Trials (as of June 5, 2014) CL-3001: • 637subjects entered screening • 213 subjects were enrolled (~210 needed) • Enrollment is 100% complete • 109 subjects had stage 3 CKD (target = ~105) • 104 subjects had stage 4 CKD (target = ~105) CL-3002: • 582 subjects entered screening •
Slide 17: OPKO
OPKO: c ic 2 (2) a c o ac