OPKO Health, Inc. · NASDAQ:OPK
OPKO Health — Conference Presentation (January 2017)
27 slides from OPKO Health's conference presentation, Jan 10, 2017
Slide 1: OPKO: Diagnostics and Pharmaceuticals for Large Markets with Unmet Needs
Exhibit 99.1 Diagnostics January 2017&NASDAQ: Pharmaceuticals OPK for Large Markets with Unmet Needs
Slide 2: Forward-Looking Statements
FORWARD-LOOKING This “plans,” projections, influence products market benefits such D ability surrounding consider commencing, products, the views materially obtaining availability, for or 2factors, (25D) other testosterone new introduction presentation asas to for Rolapitant, including products inte
Slide 3: Multinational biopharmaceutical and diagnostics company
3A multinational biopharmaceutical and diagnostics company establishing important positions in large markets by leveraging its extensive health care industry, expertise and experience.
Slide 4: Multi-faceted growth strategy
MULTI-FACETED Urology SARM Nephrology NK-1 PSA hGH Hemophilia Growth GH Short Hematology LA 4Endocrinology GLP-2 Parathyroid Insulin receptor Vitamin >2000 stature bowel Claros CKD hormone BioReference Type Tests syndrome Obesity deficiency Increase Claros BPH DAging Hormone Pruritus 2deficiency dia
Slide 5: Diversified investment highlights
DIVERSIFIED Acquired prostate Rayaldee VARUBI™ Platform growing Management 5Production cancer market technologies addresses Bio-Reference and partnered team test distribution INVESTMENT Long with and unmet with acting for Claros® aLaboratories track extending need Tesaro; assets Factor record in HIG
Slide 6: Leveraging BioReference
OPKO12 LEVERAGING BioReference ~420 ~5,000+ ~180+ Over 9M16 6GeneDx sales revenue patient DIAGNOSTICS: million is people and a genomics Labs service ?marketing working patients NATIONAL $778 is the centers million; leader served together NEAR-TERM third personnel MARKETING, known located 3Q16 larges
Slide 7: 4Kscore test
4KSCORE MORE 4Kscore Clinical 2015 Q32016 780% of and THAN men utility Health is2016 the TEST: undergoing only based 2NCCN economics MILLION BLOOD blood onand decades prostate test study 2016 TEST PROSTATE thatEAU of shows biopsy ALTERNATIVE accurately biomarker Prostate 40–55% based BIOPSIES identi
Slide 8: 4Kscore positive reimbursement progress
4KSCORE POSITIVE Obtained Initial Novitas Expect Palmetto Issued 8OPKO draft negative addressing confident Novitas Solutions has GBA positive TEST: PROGRESS positive been and to coverage itand coverage (Medicare include concerns REIMBURSEMENT has CGS coverage continues now Administrators WITH determ
Slide 9: Claros 1 point of care platform
CLAROS 25M Initiated Filing 9expansion PSA modular clinical TESTS 1following PLATFORM PMA study IN initial with THE forADDRESSES PSA FDA US FDAANNUALLY; test for filings PSA in January test LARGEexpected $625M 2017 POINT MARKET in 1H2017 OF CARE OPPORTUNITY andTEST expectMARKET testosterone 510(k) f
Slide 10: Robust product pipeline
ROBUST PRODUCT VARUBI Partnered Rayaldee® (CKD Adult (CTAP101) hGH-CTP Pediatric Alpharen™ Ph (Fermagate) OXYNTOMODULIN Diabetes, SARM Factor BPH Hemophilia AntagoNAT 10 3 inPh stage VIIa-CTP 2H PhPh 3(Rolapitant) obesity with & hGH 2b 2017 completed SHPT (CKD INDICATION Hyperphosphatemia Ph 5) 3Pla
Slide 11: Chronic kidney disease: the silent killer
CHRONIC RAYALDEE CKD Most parathyroid but The Healthcare 11 isforthcoming CKD is thethe current providers patients 9th KIDNEY hormone leading KDIGO “standard die have (PTH) DISEASE cause from Clinical no ofHigh cardiovascular of good care” death, – PTH Practice THE options Vitamin ahead levels SILEN
Slide 12: Rayaldee product overview
RAYALDEE PRODUCT Extended-Release 3F phosphorus Expected *Potential 12 DA-approved 25-Hydroxyvitamin for to (key LAUNCHED take new OVERVIEW for drivers significant indications (1x SHPT D3 daily) of(elevated or NOVEMBER vascular market Calcifediol oral including formulation PTH) share calcification)
Slide 13: Rayaldee: Simple Positioning
RAYALDEE: SIMPLE POSITIONING 13
Slide 14: Rayaldee commercialization
RAYALDEE 70-person Plans Comprehensive Leveraging Commercial Growing Initial Clinical CKD 14 to line begin trials nearly tosales extension KOL 70-80% and COMMERCIALIZATION for 1H17 double and ongoing Part advocates stage marketing by plans Dthe 5mid-2017 insurance market CKD sales in community team
Slide 15: SARM – Selective Androgen Receptor Modulator
SARM–SELECTIVE ONCE Phase levels NEXT Begin 15 Animal 2Ph STEP: DAILY study 2b trial of studies ORAL 350 to determine male resulted ANDROGEN TABLET subjects in optimal decreased for RECEPTOR dose another size to treat indication ofMODULATOR prostate BPH showed Currently significantly TT701 in Phase
Slide 16: Extending the half-life of proteins with CTP technology
OPKO Developing Carboxyl Reversible Prolongs Significant CTP 16 technology BIOLOGICS: drug Terminal Pegylation reduction improved bioactivity safety Peptide in EXTENDING long Technology profile injection acting (CTP) is comparable frequency proteins Technology THE HALF and topeptides non LIFE modifi
Slide 17: hGH-CTP partnered with Pfizer
hGH-CTP PARTNERED New Once Human Growth SGA, Final Refrigerated, Disposable Phase children 17 molecular presentation: weekly PWS, 3 growth hormone study and COMPETITIVE easy ISS adults liquid, injection in WITH hormone entity to growth deficient handle non PFIZER (NME) vs. hormone viscous iscurrent
Slide 18: Adult Phase 3 study topline data
ADULT Efficacy A Primary Topline GH-CTP Does 97% Safety Found 18global of not profile an GH-CTP data showed: endpoint GHD endpoints multicenter group meet exceptional means isPHASE statistical has consistent vs isof mean first 6% trunk study treating value 3of look significance STUDY change fat plac
Slide 19: Next steps for adult GHD study
NEXT Phase The Developed Proceed Communicated 19 exceptional 3STEP adult with a statistical analysis; GHD todata allstudy point investigators discuss planwarrants forwith dataand regulatory an sensitivity CROs outlierinvolved sensitivity authorities analysisintoanalysis all identify on-going ofany t
Slide 20: GH-CTP global Phase 3 program status
GH-CTP Initiated 220 Global Easy Phase No Switching Initiating 44 Same Plan 20 patients, safety patients, to to pen 3study commence use, adult phase PROGRAM Pediatric concerns device, tocomparison disposable, pen non-inferiority CROs GHD 3 pediatric device dosage global GHD and selected; STATUS phas
Slide 21: Long-acting Factor VIIa-CTP for hemophilia
LONG-ACTING PHASE Market Current 3-4 1-2 In Demonstrated Reduced Enabled Orphan 21 pharmacological times growing drug product 2a prophylactic frequency a STUDY day designation potential during for (NovoSeven®) 7% FACTOR prophylactic of studies UNDERWAY- annually treatment injection bleeding for in s
Slide 22: TT401 oxyntomodulin for diabetes and obesity
TT401—OXYNTOMODULIN—TYPE POTENTIAL Once Superior Reduction NEXT Dose 22 optimization weekly STEP: weight in HbA1c, administered FIRST loss phase compared TO marker 2b MARKET oxyntomodulin trial ofwith sugarGLP-1/GLUCAGON currently metabolism, 2 DIABETES/OBESITY for approved type similar 2 diabetes e
Slide 23: Uniquely positioned to offer animal health products
OPKO UNIQUELY Minimal Plans >6 Developed experience 23 million toANIMAL introduce investment dogs and pet POSITIONED friendly success and HEALTH several >6 by formulations in utilizing million developing OTC TOcats products OFFER existing inand and thePRODUCTS 1Q17 packaging product marketing U.S. a
Slide 24: Select financial information
SELECT Cash, Balance Current Capital Common Three 9/30/2016 Revenue* *Nine 242016 months cash months revenue structure portion sheet FINANCIAL shares 2016 equivalents ended at ended reflects of vs. outstanding: 9/30/2016 atline 2015 September September of & INFORMATION acquisition credit marketable
Slide 25: Upcoming progress milestones across multiple business areas
UPCOMING PROGRESS 4Kscore *clinical 2525-Hydroxyvitamin study reimbursement ACROSS MILESTONES PMA filing D3MULTIPLE Coverage 1H17 or Calcifediol Claros decisions BUSINESS 1 testosterone and AREAS pricing clinical negotiations study Start underway 1H17 Oxyntomodulin Factor VIIa Phase Phase 2a Top-lin
Slide 26: Investment highlights
INVESTMENT Core Pharmaceutical growth platform 26 business strategy Biologics with pipeline HIGHLIGHTS with nearlyGrowth with $1 billion Executive expansion in revenue team and significant large market near-with througha history opportunities of termacquisition success Multi-faceted opportunities co
Slide 27: Thank you
OPKO THANK YOU