Opko Health, Inc. · NASDAQ:OPK
Opko Health — UBS Life Sciences Meeting 2009
30 slides from Opko Health's conference presentation, Sep 24, 2009
Slide 1: UBS Life Sciences Meeting
Jamie Freedman M.D., Ph.D.Executive Vice President, Research & Development OPKO Health, Inc.Miami, Florida The UBS Life Sciences MeetingNew York, Sept. 22, 2009
Slide 2: Cautionary Statement
Cautionary Statement 2 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," "s
Slide 3: OPKO Health, Inc.
OPKO Health, Inc. A specialty healthcare company building a diverse portfolio of important medical products with significant commercial value. 3
Slide 4: Board of Directors
Board of Directors Phillip Frost, Chairman of the Board & CEOJane Hsiao, Vice-Chairman & CTOSteve Rubin, Director and Exec. VP Administration Richard Lerner, President, The Scripps Research InstitutePascal Goldschmidt, Dean, University of Miami School of MedicineAlice Yu, Professor, National Taiwan
Slide 5: Management Team
Management Team Dr. Phillip Frost, CEO & Chairman of the BoardDr. Jane Hsiao, Vice-Chairman & Chief Technical OfficerMr. Steve Rubin, Exec. VP, AdministrationDr. Rao Uppaluri, Sr. VP & Chief Financial OfficerDr. Jamie Freedman, Exec. VP R&D 5
Slide 6: Financial History
Financial History Capital InvestmentAcuity Pharmaceuticals & Froptix (Pre-acquisition) $20.0 MillioneXegenics (Public Company) $16.0 MillionThe Frost Group (December, 2007) $20.0 MillionThe Frost Group (September, 2008) $15.0 MillionThe Frost Group (April 2009) $20.0 MillionOutside investors (May 20
Slide 7: OPKO Portfolio
OPKO Portfolio Ophthalmics ^ General Medicine 7 OPHTHALMICS GENERAL MEDICINE Drugs Drugs Vaccines Diagnostics Devices Diagnostic Instruments Bevasiranib & other siRNAs (AMD) Civamide (Dry Eye)Doxovir (Viral Conjunctivitis) Aquashunt (Glaucoma) UltrasoundOCT/SLOMicroperimetry Molecular Diagnostic Arr
Slide 8: OPKO Instruments Ultrasound
Wide range of ultrasound productsA-Scan, B-Scan, 3D-Scan, UBMDistributed in 45 countriesUltrasound market ~$60M OPKO Instruments Ultrasound 8
Slide 9: Integrated OCT and SLO
OCT and SLO from single sourcePrecise pixel-to-pixel correlation of coronal OCT slice to SLO surface imageUltra-high resolution imaging of eyeDiagnosis of retinal and choroidal diseases 9 OPKO Instruments Integrated OCT and SLO OCT SLO OCT=Ocular coherence tomography SLO=Scanning laser ophthalmoscop
Slide 10: Microperimetry Add-On to OCT-SLO
OPKO Instruments Microperimetry Add-On to OCT-SLO Tests retinal function at level of photoreceptors in maculaFunctional-anatomical correlationTrack visual function response to treatment Functional Sensitivity Map ~$250M market potential
Slide 11: Molecular Diagnostics Overview
Molecular Diagnostics Overview Global molecular diagnostic market projected to reach ~$4B by 2010Largest growth segments:Early detectionCompanion diagnostics 11
Slide 12: Molecular Diagnostics Small Molecule Microarray
Molecular Diagnostics Small Molecule Microarray Molecular microarray technology acquired June 17, 2009"Peptoids" as first example of small molecule array to detect disease- associated antibodiesBroad application for diagnosing diseases by a simple blood testNeurological disorders (Alzheimers Disease
Slide 13: Small Molecule Microarray Peptoid Methodology
Small Molecule Microarray Peptoid Methodology 13 HEALTHY DISEASE
Slide 14: Diagnosis of Alzheimers Disease
Small Molecule Microarray Diagnosis of Alzheimers Disease AD most common form of dementiaDiagnosis relies on cognition tests and expensive imagingMolecular test would be useful in drug development 14 NC=Normal ControlAD=Alzheimers DiseasePD=Parkinson's Disease
Slide 15: Multiple Peptoid Applications
Multiple Peptoid Applications 15 Application Example Diagnostic Detection of disease and subpopulationsTissue-specific imaging agentsBiomarkers to follow disease and response to therapy Therapeutic Identification of therapeutic targetsModulation of therapeutic targets (agonist or antagonist) Vaccine
Slide 16: OPKO R&D Pipeline
OPKO R&D Pipeline 16 Products Mode of Action Indication Pre-Clinical Phase I Phase II Phase III Bevasiranib Gene SilencingAnti-VEGF-A Wet AMD Doxovir Anti-Viral Epidemic viral conjunctivitis Civamide Vanilloid Rec Inhibitor Dry Eye Aquashunt Drainage Shunt Refractory Glaucoma siRNA Gene Silencing Mu
Slide 17: Bevasiranib VEGF Gene Silencing
Bevasiranib VEGF Gene Silencing Target vascular endothelial growth factor (VEGF)Treatment for wet Age-Related Macular Degeneration (AMD)Most advanced siRNA in clinical development 17 New Blood Vessel Growth & Leakage RNA, e.g VEGF message Protein, e.g. VEGF Lucentis / AvastinNeutralized secreted VEG
Slide 18: Phase 3 Trial Bevasiranib vs. Lucentis
Phase 3 Trial Bevasiranib vs. Lucentis Bevasiranib every 8 or 12 weeks versus Lucentis every 4 weeksLucentis/Bevasiranib run-in for Bevasiranib arms 18 Rx Arm Day 0 Week 2 Week 4 Week 6 Week 8 Week 12-Week 60 Lucentis q4wk(N=113) Lucentis Sham Lucentis Sham Lucentis Lucentis Beva q8wk(N=112) Lucenti
Slide 19: Bevasiranib Phase 2 Trial Early Gain in Vision?
Bevasiranib Phase 3 Trial Early Gain in Vision? Trend for 15-letter gain in vision at 8 & 12 weeks (alternating Lucentis & Bevasiranib) Percent 15-letter gain
Slide 20: Bevasiranib Next Steps
Bevasiranib Next Steps Consider more frequent dosing Every 4 weeks like LucentisCombining with LucentisBased on promising activity during Lucentis run-in (up to 12 weeks)Reformulation with novel drug delivery vehicles (undisclosed)To enhance cellular penetration
Slide 21: OPKO siRNA Discovery Programs
OPKO siRNA Discovery Programs Target Biological Mechanism Indications VEGF165b-sparing Angiogenesis AMDCancer Hypoxia-inducible Factor (HIF-1alpha) AngiogenesisErythropoiesis AMDCancer Intercellular Adhesion Molecule (ICAM) Inflammation AMDAtherosclerosisRespiratory viruses Complement C3 / C5 Immuni
Slide 22: Doxovir Viral Conjunctivitis
Doxovir Viral Conjunctivitis Common eye infection, no effective treatmentDoxovir is a broad-spectrum anti-viral & anti-inflammatory agent with excellent preclinical efficacyEvaluated and shown to be safe in Phase I clinical trialsPhase 2 clinical trial to be initiated by OPKO in 3Q09 22
Slide 23: Civamide Dry Eye
Civamide Dry Eye Common eye illness with no effective treatmentCivamide is a villanoid receptor antagonist administered intranasallyInduces high frequency tearing in clinical trialsPhase 2 trial for dry eye to be initiated by OPKO in 3Q09 23
Slide 24: Glaucoma
Glaucoma Glaucoma is a leading cause of blindnessElevated intraocular pressureDamage of optic nerve 24 Treatment options Limitations Potential market* Medications~840,000 patients($2B sales/year) Poor compliance Unpleasant/dangerous side effects High costs Lack of efficacy 35%(296K patients) Laser~1
Slide 25: Aquashunt Glaucoma
Aquashunt(tm) Glaucoma 25 "Tube vs Trab" study show shunts better than surgery Higher success rate Lower complication rateCurrent shunts can be improvedBetter efficacyLower complicationsLess complicated surgeryAquashunt(tm) is a novel shunt deviceSuprachoroidalSimple surgeryStrong IP
Slide 26: Aquashunt Phase 1 Study
Aquashunt(tm) Phase 1 Study initiated in 2009 Aquashunt implanted in 15 glaucoma patients refractory to medical therapy and failed surgeryGenerally well-tolerated with few adverse events related to surgery or deviceIOP lowering by 1 month in ~80% patients>20% reduction in IOP in 3 of 4 patients at 6
Slide 27: Influenza
Influenza 200,000 hospitalizations and 46,000 deaths in U.S. each yearEconomic costs in U.S. ~$80 billionAnnual "flu shot" against common strains Flu vaccine grown in chicken eggsLimitations:New vaccine each yearOnly partially protectiveManufacturing capacity limited 27
Slide 28: Universal Flu Vaccine Preclinical Development
Universal Flu Vaccine Preclinical Development Universal flu vaccine licensed from Academia Sinica July 20, 2009Vaccine based on hemagglutinin to block viral entry Terminal sugars removed from hemagglutininCore common to all strains, including mutantsProtein-based vaccine more immunogenicPreclinical
Slide 29: OPKO Future
OPKO Future Expand applications and increase sales of ophthalmic instruments worldwideExplore innovative drug delivery systems for RNA interferenceDemonstrate clinical proof-of-concept with early clinical productsStrategic acquisitions of late stage clinical productsOpportunistic acquisitions of mat
Slide 30: Thank You
THANK YOU