Opko Health, Inc. · NASDAQ:OPK

Opko Health — Jefferies 2013 Global Healthcare Conference

26 slides from Opko Health's conference presentation, Jun 5, 2013

  1. Slide 1: Jefferies 2013 Global Healthcare Conference

    Exhibit 99.1 Jefferies June 4, 2013 2013 Global Healthcare Conference

  2. Slide 2: Forward-looking statements

    Thisprocesses “expects,”“plans,”“projects,”“will,” financial results long-acting drugs validation potential markers, candidates, statements. including product technologies, research and successfully 2filings studies autoimmune presentation and with will availability, programs, and projections, benef

  3. Slide 3: OPKO: Diversified Company with Growth Pipeline and Markets

    OPKO IP –– Markets 3ƒ–O nternational harmaceuticals Anti-virals pportunistic Vitamin Vascular Microfluidics CLIA-certified Platform Israel —ƒ A D Chile disease toHigh therapeutics ƒdevelop ƒMexico system Antibodies ƒcentral Growth Brazil diagnostic ƒfor lab Spain for Medical rapid, with SHPT*tests l

  4. Slide 4: Key Milestones

    Key 4•–top Acquired Top-line Phase Issued Entered Readying Netline Milestones cash 3results $175 trials into Phase position interest 4KScore™ million definitive strategic expected for in 3 data H1 RayaldyTM in at growing aggregate $181.6 2013 pooling commercial ata agreement mid-2014 fo million orRu

  5. Slide 5: OPKO Pharmaceuticals Pipeline

    OPKO3Stage Product (CKD RayaldyTM Fermagate Phase Rolapitant Pending hGH-CTP 5CTA018Pharmaceuticals results Indication Acquisition (Topical) CINV expected 3-4 Patients) and SCH Preclinical H1 –PONV H12017 900978 —expected 2018 Clinical Outlicensed Phase CTAP201 H1 Pipeline 1 Phase 2014 to TESARO 2 P

  6. Slide 6: Pending Acquisition of PROLOR Biotech

    ĥPending 6o8.1 OPKO Pre-revenue Late-stage Preclinical 0.9951 Closing Complementary million Acquisition to shares expected acquire options product products biopharmaceutical of drug OPKO all +inof targeting warrants) H2 targeting outstanding PROLOR development 2013, stockgrowth based hemophilia, su

  7. Slide 7: hGH-CTP Effects in Growth Hormone Deficient Adults

    Effectivelean hGH-CTP Increase 7Improve bone physical hGH is designed body density Therapy performance massDecrease to:in Growth Improve fat Hormone masscardiac Deficient function Adults

  8. Slide 8: PROLOR Pipeline and Indication Market Size

    PROLOR Indication Market hGH Growth $3.5B Hormone Disorders $1.3B Factor Hemophilia Type 8Diabetes Obesity –CTP II1-2/week VIIa- IX—CTP & (Adults) hGH Oxyntomodulin Pipeline Product –CTP injection 1/week $0.7B Preclin. 1/week injection CTP ?Ph 1/week 1injection Ph 2injection Ph 3 Size

  9. Slide 9: PROLOR: Weekly hGH-CTP Clinical Program

    9•PROLOR: Weekly 189centers Primary Ongoing 40-56 12 Key Dose 35 Expected Orphan months patients, outcome: finding naïve drug injection endpoint: Pediatric hGH-CTP to(EU, duration efficacy be growth designation >40 study Height first US, compared vs. centers Change Phase –& (6 long-acting hormone Cl

  10. Slide 10: Cytochroma Acquisition: Rayaldy for SHPT

    *10 •–Cytochroma **Unmet Two First-in-class Compelling USPTO NDA Early Cytochroma’s 25DPhase filing stage =SHPT Medical andAcquistion 2pipeline expected Phase EPO and = modified-release product Secondary Need: two have 2bincludes in 25-hydroxyvitaminPhase data No –candidates issued first Product cur

  11. Slide 11: Rayaldy: Stage 3-4 CKD Opportunity

    •ƒ?Rayaldytm: Stage Safety Hormone 1,25D Nutritional RayaldyTM BioTrends 11 8.0M RayaldyTM’s 4.0M Low320% ~1.0M Existing concerns serum CKD patients CKD patients vitamin treatments Research Nutritional insufficiency Siggnificant Capsules Stage 25D Treatment efficacy with and seen D and 3-4 1,25D Unt

  12. Slide 12: Rayaldy: Phase 2b Results

    Total± RayaldyTM: 730 H 60 25D SE) *40 0 0n=16 n=14 80 20 Serum50 Cohort10*2n=12 (ng/mL; Change Placebo Treatment ** Corrects 12 m *n=17 ean Placebo 30in Phase n=14 (mea30 µg/day 30-10 Significantly Cohort Group Significantly Vitamin SHPT 2b 1Change -50 Results Placebo µg/day 90 (Elevated different

  13. Slide 13: Fermagate: A Phosphate Binder Overview

    *13 Fermagate: A ĥOverview Clinical Two Partial Fermagate 90% Claims Higher Smaller No Potentially A Versus Low phosphate non-calcium, Phase risk ofCOGS FosrenolPhase for key Status Renagel potency/gram U.S. EU competitive of pills base use 3Product sales lanthanum protective binder and studies 3ma

  14. Slide 14: Rayaldy and Fermagate Clinical Timelines

    214 RayaldyTM 2012 Q1 EndQ2 of 2013 3 Week SPA Phase Q3 MeetingsMeeting Q4 2014 Data Fermagate 52 Two &Q1 Safety Phase with API Fermagate: FDA & 2015 Final Q2&(US 3Trial Q3 2016 trials Tablet Phase EU Q4 &Clinical NDA Q1 w/ FDA Q2 Timelines Q3Studies Manufacturing EU) NDADDI Q4 Filing Q1 Q2 Q3 Appro

  15. Slide 15: Rolapitant: Three Phase 3 Trials

    •– Three 15Rolapitant Potentially Single Rapid Long Phase Five-day Well Schering-Plough Payments Double-digit Potent Proof Over accepted acting 1,000 global 3of onset dose neurokinin-1(NK-1) program concept activity of out-licensed best-in-class patients Phase up tiered regulatory toinitiated follow

  16. Slide 16: OPKO Diagnostics: Our Vision

    3//444 Significant 3 Low OPKO 3 Easy-to-use Cost Diagnostics / Highly Quantitative Inexpensive — Our Vision economic Point-of-Care benefit to physicians through in-sourcing of basic panels 3 3333//444 3 CLIA Urology, 4 Profitable Lab 15+ About Ob/Gyn, CLIA personlabHIV, with Hepatitis nationalurolog

  17. Slide 17: 4KScore and Point-of-Care Market Opportunity

    Market •OPKO –Developed biopsy Next /Markers CombinesWW: 33Total-PSA, //44444 Costs 70 >1Generation and million in million Diagnostics Opportunity tested Test reduce PSA, Testosterone (LDT) infree Prostate prostate excess PSA unnecessary over – Initial PSA, with 10,000 Cancer tests ofPoint-of-Care A

  18. Slide 18: Projected Key Requirements: Microfluidics Platform

    Projected –•Key 18Injection-molded On-board Multiplex Sensitive Reliable No User-friendly Biohazard Requirements sample Launch standard proprietary delivery platform containment prep sample –microfluidics optics of High amplification multiple introduction Quantitative reagents chemistry Performance

  19. Slide 19: OPKO Diagnostic Technology Capabilities and Applications

    3•//444• Whole 3 Liquid OPKO Women’s Urology Urologist •PCa 3 19 •Panel: Serum Urine 3Blood • Sample Dx Cardiology Infectious Ob/Gyn Card Panel 3/Cardio /34/Capabilities 4Technology Tests: Emergency GPs Saliva Amniotic Semen 4Health 3/Target 43Clinic /Panel: Disease Tears /STD 4 Spinal 3and Applicat

  20. Slide 20: Product Launch Timelines

    Diagnostic 2013 Q1 Urologic 4K Point RayaldyTM MOD4023- 20 Q22014 Score OfQ3 of Care Care Pathology asQ4 2015 &Endo Laboratory Capsules 4KScore Pharmaceutical Testosterone PSA 25D Q12016 Q2 Test Test—Urology Laboratory Q3 –2017 Test- –Neph, Developed Q4 Neph, Test—Urology Urology Q1 Product Endo, –E

  21. Slide 21: OPKO Farmadiet Access to European Union

    OPKO –•Farmadiet 21 Access Established Fully Broad Veterinary Basedintegrated 42-person Production/research Pharmaceuticals: Nutritionals: Citicoline 25-Hydroxyvitamin European pharmaceutical, in toGroup Barcelona, promising (supports sales : 20-year 50 70Products Union force specialty Products 45pr

  22. Slide 22: OPKO Emerging Markets

    –•OPKO 22 Acquired ANVISA Rapidly 25 Primarily products Emerging growing ALS February licensed branded across Distribution Markets sales pharmaceutical ophthalmics, a2013 range fromof Limited, >100 therapeutic with products company the expanding exclusive indications in most proprietary product impo

  23. Slide 23: OPKO Emerging Markets (cont'd)

    OPKO –••OPKO 23 Immunogenic Faster 98% Higher Acquired Develops FDA Sci-B-Vac™ protection registered Emerging onset Israel levels in controlling andof December is of regions produces action in astate highly currently Markets anti-HBV third-generation vaccinated of interest (earlier of immunogenic 20

  24. Slide 24: Strategic Investments

    Strategic Proprietary *–•surgery 24 Therapeutics .Discovery, ,(~10% Development RNA RXI-109 New Next Manufactures Develop Oncology-focused Developing Dietary OPKO (~21% LLC Inc. (~ (~1% approach technology gen interference (~13% As equity distribution Investments supplement 4% equity and in technolo

  25. Slide 25: Additional Information and Where to Find It

    Additional and that PROLOR. DOCUMENTS security through also Shlosberger, This offer, requirements OPKO, OPKO’s statement interests, 25 does will be communication solicitation obtained PROLOR the holders include directors not by for STOCKHOLDERS Information website security PROLOR constitute of itsFI

  26. Slide 26: Jefferies 2013 Global Healthcare Conference

    Jefferies 2013 Global Healthcare Conference June 4, 2013

OPKO Health — Jefferies 2013 Global Healthcare Conference on 6ix