Opko Health, Inc. · NASDAQ:OPK
Opko Health — Jefferies 2014 Global Healthcare Conference
24 slides from Opko Health's conference presentation, Jun 3, 2014
Slide 1: Jefferies 2014 Global Healthcare Conference
Exhibit 99.1 Jefferies 2014 Global Healthcare Conference June 3, 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: Important Products Available or Coming to Market Near Term
OPKO Important Products Available or Coming to Market Near Term – 4Kscore™ Test blood test for personalized risk of high-grade prostate cancer Diagnostics – CLIA-certified urological specialty laboratory – Claros ® 1 immunoassay system for rapid, lab quality in-office testing (PSA, Testosterone, Vit
Slide 4: OPKO Diagnostics – Addressing Large Dx Markets
OPKO Diagnostics – Addressing Large Dx Markets 4Kscore™ Test Initially targeting pre-prostate biopsy market PSA market 60 million tests globally Recently launched in US at $395 Highly significant clinical data on 1,012 patients presented at AUA plenary session May 18, 2014 Launch in Europe: Septembe
Slide 5: Convenient, Lab Quality, Accuracy
CONVENIENT LAB QUALITY ACCURACY DELIVERING BETTER HEALTHCARE 5
Slide 6: Convenience: Finger stick blood sample
Convenience 1-2 10 mins mins Finger stick blood sample 6
Slide 7: Claros 1 Update
Claros 1 Update • Testosterone – FDA: Pre-submission comments received from FDA – On track to file 510(k) in 2014 – CE Mark: 4Q2014 • PSA – FDA: Pre-submission response expected in August – Timing of 510k submission based on longitudinal trial requirements – CE Mark Update (Formulation and Chemistry
Slide 8: Challenges in Prostate Cancer Screening
Challenges in Prostate Cancer Screening High false positive rate of PSA Patient Anxiety 1M Prostate biopsies in US 75% Negative or Low-Grade Pain, bleeding, infection, hospitalization Recommendations to stop PSA screening 8
Slide 9: 4Kscore Test – Avoiding Unnecessary Prostate Biopsies
4Kscore Test – Avoiding Unnecessary Prostate Biopsies The only test to identify men with high-grade prostate cancer from a blood sample Combines results of multiple biomarkers to create a patient’s personal risk score Based on 10 years of clinical research by scientists at Memorial Sloan-Kettering C
Slide 10: 4Kscore Test US Clinical Study
4Kscore Test US Clinical Study 1,012 Patients Enrolled – Prospective Clinical Trial 10
Slide 11: 4Kscore Test Clinical Study Results in 1,012 Men
4Kscore Test Clinical Study Results in 1,012 Men • Discrimination: AUC = 0.82 • Risk Calibration • Decision Curve Analysis • Biopsy reduction of 30% to 58% 11
Slide 12: The 4Kscore Patient Counseling Report
The 4Kscore Patient Counseling Report for a Result of 5% 12
Slide 13: The 4Kscore Test Conclusions
The 4Kscore Test Conclusions Validated test based on a decade of clinical research and a prospective, multi-institutional, contemporary US clinical trial Convenient blood test, cost $395 Excellent discrimination for high-grade cancer (AUC = 0.82) and high net benefit for clinical use Reduces 30 – 58
Slide 14: 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 expected mid- $12.0 BN (CTAP101) (CKD Stage 3-4 Patients) 2014 hGH-CTP hGH deficiency $3.5 BN Alpharen™ Hyperphosphatemia $1.2 BN (Fermagate) (CK
Slide 15: Rayaldee (CTAP101) – A Late-Stage Investigational Drug
Rayaldee (CTAP101) – A Late-Stage Investigational Drug Product Overview Clinical Status • Oral formulation of 25D3* addresses significant unmet need • Clinical development guided by prominent Scientific Advisory • Safe and effective treatment for Board elevated PTH (SHPT) associated with low 25D lev
Slide 16: Rayaldee - Commercial Opportunity
Rayaldee - Commercial Opportunity • Low serum 25D and elevated plasma PTH are prevalent in CKD Stage 3-4 patients – 8 million CKD Stage 3-4 patients in the US – 4 million patients with low serum 25D and high plasma PTH Stage 3 & 4 CKD Treatment Vitamin D Hormone Safety concerns; 20-36% exacerbates v
Slide 17: Comparison of Vitamin D Therapies for Stage 3-4 CKD
Comparison of Vitamin D Therapies for Stage 3-4 CKD Rayaldee is expected to raise serum total 25-hydroxyvitamin D (25D) and lower plasma iPTH more effectively than any currently marketed over-the- counter (OTC) or prescription (Rx) product without the risk of hypercalcemia. *And generics **25-hydrox
Slide 18: CTP Technology: Clinically Validated Proprietary Platform
CTP Technology: Clinically Validated Proprietary Platform • CTP increases protein circulation time CTP – a natural sequence created during evolution to enhance the longevity of peptides and proteins Any Short Acting CTP Long Acting without increasing toxicity Protein Protein • Merck’s long acting FS
Slide 19: 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 20: hGH-CTP Clinical Development
hGH-CTP Clinical Development • Adult Pivotal Phase 3 trial (ongoing) – 189 patients – 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
Slide 21: 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 22: MOD-6031: Long Acting Oxyntomodulin for Obesity
MOD-6031: Long Acting Oxyntomodulin for Obesity • >$15 billion market • Growing rapidly • Oxyntomodulin – Nature’s Appetite Control Mechanism • Natural appetite suppressor • Secreted by the digestive system following food intake and induces satiety in the brain • Increases glucose tolerance • Short
Slide 23: 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 24: Strategic Investments
Strategic Investments Proprietary Technologies with Significant Upside Potential • ARNO Therapeutics, Inc. (OTC: ARNI) (~5% equity interest) – Anti-progestins for breast (phase 2) , endometrial and prostate cancers • Zebra Biologics, Inc. (~19% equity interest) – Combinatorial antibody libraries bas
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