Opko Health, Inc. · NASDAQ:OPK
Opko Health — Jefferies 2012 Global Healthcare Conference
23 slides from Opko Health's conference presentation, Jun 7, 2012
Slide 1: Jefferies 2012 Global Healthcare Conference
Jefferies 2012 Global Healthcare Conference June 5, 2012 Exhibit 99.1
Slide 2: Company Overview
Discovery Earlier Clinical Commercial Mid / Late Clinical Company Overview Opportunistic pharmaceutical and diagnostics company focused on large, high growth markets: – New PLATFORM to develop diagnostic tests for cancer and neurodegenerative diseases – New microfluidics SYSTEM for rapid, lab-qualit
Slide 3: Rolapitant Overview
Rolapitant Overview Rolapitant out-licensed to Tesaro in December 2010 Schering-Plough (S-P) divested to OPKO as FTC condition for S-P merger with Merck Payments of up to $121MM Double digit tiered royalties Potentially best-in-class cancer supportive care product Potent neurokinin-1(NK-1) receptor
Slide 4: Rolapitant Market Opportunity
Rolapitant Market Opportunity U.S. market opportunity of ~ $1.25 billion 6.6 million annual CINV patient treatments in 2011 NCCN and ASCO guideline recommendations could lead to 70% penetration by the NK-1 class Merck - EMEND® currently only NK-1 receptor antagonist on market EMEND® 2011 global sale
Slide 5: Therapeutic Potential of JNK Inhibitors
Therapeutic Potential of JNK Inhibitors Orally bioavailable, brain penetrant JNK inhibitors have shown efficacy in two animal models of Parkinson’s disease and represent first in class neuroprotective agents for the treatment of PD These JNK inhibitors have potential in all neurodegenerative disease
Slide 6: Antigen Surrogate Technology
Antigen Surrogate Technology Foreign Antigen B Cell Re ee 7 . SYA Y N 4 A VV¥AY, wnpiieaton B Cells wane ae 7A AS Sas aon O?-10° Synthetic SAS KS 2. / Molecules
Slide 7: Second Generation Screening System For Larger Libraries
Second Generation Screening System For Larger Libraries ¢ @ Qo dln Bead Library (= one million compounds) N , H H 8 IgG ligands 4 Healthy serum sample Denuded Library (still almost one million compounds) Disease-specific IgG ligands | Disease serum sample Cleave, spot A Denuded Library (still almost
Slide 8: Neuromyelitis Optica (NMO)
Neuromyelitis Optica (NMO) + Anautoimmune disorder of the CNS where autoantibodies destroy cells in the spinal cord and/or optic nerve. * Often mis-diagnosed as multiple sclerosis, with serious adverse consequences. + Autoantibodies recognize AQP4 on the surface of target cells in = 70% of NMO patie
Slide 9: Discovery of Peptoid Surrogate of AQP4
Discovery of Peptoid Surrogate of AQP4 7 @ 100K Qo hy NL 4 Molecules 4H 5 1. Control Serum 2. NMO Serum SH106 i SH109 fj sH105 Spot Onto Slides In Triplicate 1. Probe With Monoclonal Antibody 2. Detect With Red-Labeled Anti-Human Ab SH109 SH108 SH106 SH105 Anti-AQP4 Monoclonal Antibody SH109 SH108 S
Slide 10: Sensitive and Specific Detection of NMO
Sensitive and Specific Detection of NMO SH109 = SH108 Normal Serum #6 SH106 SH105 Blinded Study of SH109 SH108 30 Samples NMO Serum #30 10/10 NMO Anti-AQP4 Ab Positive Anti-AQP4+ SH106 $H105 SH109 =©SH108 8/10 NMO NMO Serum #74 =| Anti-AQP4 Ab Negative Anti-Aqp4- SH106 SH105 OPKO
Slide 11: Development of A Commercially Viable ELISA-like Assay
Development of A Commercially Viable ELISA-like Assay Blue Product 4 Colorless substrate S HRP C Peptoid - Linker ——N oO 3 SH 106 aS 2 —NMO 15 NC e ~teMS 0.5 oO Yo, ¥, ¥. v. OPKO
Slide 12: Plans - NMO
Plans - NMO Identify higher affinity derivatives of SH106 and other peptoids. Akin to movement of a screening hit to a drug lead. Complete a blinded study of ~ 300 patients using the ELISA test to determine diagnostic sensitivity and specificity. Optimize test on both Claros point of care platform a
Slide 13: Lung Cancer Screening Protocol
Lung Cancer Screening Protocol ° Q om, H 8 Non-smoker (2 million beads) (~ 300 beads Removed) { Pool of 3 non-smoker serum samples Pool of 5 Healthy Healthy (~ 150 beads Smoker serum somnotes Smoker ’ Removed) ¥ Pool of 3 Stage | 70 beads ( _— “| sae serum samples Removed) Pool of 3 Stage | Adenocar
Slide 14: Microarray Validation of LCP6
Microarray Validation of LCP6 ©) Non-Smokers @ Healthy Smokers @® NSCLC @B Adenocarcinoma G Squamous Cell © Small Cell Carcinoma Intensity (Disease Stage) Serum Samples OPKO
Slide 15: Progress On Alzheimer's Disease (AD)
Progress On Alzheimer’s Disease (AD) Jan. 2011: Reported discovery of three peptoids (ADP1, ADP2, ADP3) that bind two different AD-specific antibody. (Reddy, et al. (2011) Cell 144, 132-142. Mid-2011: Completed validation study of 105 patients by microarray for ADPs 1-3. Mid-2011: Began working on E
Slide 16: Blinded ELISA Study of Samples
Blinded ELISA Study of 80 Samples 2 e 200000: 8 = 100000- M 3 v 3 ey v 8 o. z rv 2 AD NC ° FS 5 rd JC3BR8 z. g @ s E s 2 Clinical AD: 72% Agreement with OPKO test Age-Matched Controls: 86% Agreement with OPKO test OPKO
Slide 17: Status Of Biomarker Discovery Projects
Status Of Biomarker Discovery Projects Samples Acquired/Acquiring: Discovery (Library Screening): Initial Validation (Microarray): First-Generation ELISA Assay: Extensive Validation By ELISA: Peptoid Optimization: Extensive Validation of Optimized Peptoids by ELISA: Ovarian Cancer, Prostate Cancer,
Slide 18: OPKO Diagnostics Platform
OPKO Diagnostics Platform Product: Immunoassay System Yielding Rapid Lab-Quality Quantitative Test Results with Finger-Stick Drop of Blood (OPKO Dx Card) Technology Acquired Claros Diagnostics in October 2011 Proprietary Microfluidics and Amplification Chemistry Pre-loaded Reagents on a Disposable C
Slide 19: OPKO Dx Card Technology Applications
OPKO Dx Card Technology Applications Urology Women’s Health Cardiology Infectious Disease Other Urologist Office Ob/Gyn Office Emergency Room Clinic and Blood Bank GP Office PCa Panel: • total-PSA • free-PSA • Testosterone • Novel Markers Panel Tests: • Pre-term bleed • Fertility • Menopause Cardio
Slide 20: Key Attributes – High Quantitative Performance / Low Cost
Key Attributes – High Quantitative Performance / Low Cost Overview of OPKO Diagnostics Technology Inexpensive Disposable Injection-Molded Microfluidics On-Board Delivery of Multiple Reagents Multiplex Platform Robust Signal Detection Sensitive Proprietary Amplification Chemistry for Microfluidics In
Slide 21: Easy to Use
Easy to Use 1. Collect Blood 3. Insert into OPKO Dx Reader 2. Snap into Cassette 4. Read Results in less than 10 minutes
Slide 22: OPKO Diagnostics 2012 Plans
OPKO Diagnostics 2012 Plans Urology U.S. FDA Trial Completion / Filing Proprietary Next Generation Prostate Markers Signed license agreement in May, 2012 for launch of OPKO’s novel panel of Kallikrein markers in UK, Ireland, Sweden and Denmark 70 Million WW Tests Per Year $2 Billion Market Vitamin D
Slide 23: Proprietary technologies with significant upside potential
Proprietary technologies with significant upside potential Strategic Investments CoCrystal Discovery, Inc. (~16% equity interest*) – Founded by Nobel Laureate, Roger Kornberg, Ph.D. – New approach to develop broad spectrum anti-viral drugs – Development program with Teva for Hepatitis C drug Sorrent
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