Opko Health, Inc. · NASDAQ:OPK
Opko Health — ENDO 2014
23 slides from Opko Health's conference presentation, Jun 24, 2014
Slide 1: June 2014
Exhibit 99.4 June 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: Ron G. Rosenfeld, MD
Ron G. Rosenfeld, MD Professor of Pediatrics (emeritus), Stanford University Professor of Pediatrics, Oregon Health & Science University (emeritus) President, STAT5 Consulting, LLC
Slide 4: The Long Acting Human Growth Hormone ENDO 2014
Lagova™ The Long Acting Human Growth Hormone ENDO 2014 OPKO Biologics Ltd. Nes-Ziona, Israel
Slide 5: C-Terminal Peptide (CTP)
C-Terminal Peptide (CTP) Created By Nature During Evolution To Extend Circulation Time of Functional Proteins
Slide 6: CTP: Clinically Validated Technology
CTP: Clinically Validated Technology CTP technology has been used in an approved product (Elonva®, Once weekly FSH-CTP, Merck)
Slide 7: The Long Acting Growth Hormone
Lagova The Long Acting Growth Hormone + = Any Short-Lasting CTP Long-Lasting Protein Protein Natural sequence No need for linker
Slide 8: Competitive Advantages
Lagova Competitive Advantages Increase size PEGylation Slow enzymatic cleavage DNA Add sugar chains Mutations Increase negative charge Protein Increase size Fusion Slow enzymatic cleavage
Slide 9: Competitive Advantages
Lagova Competitive Advantages Lagova Potentially non-immunogenic Lagova ~75% native hGH Lagova Ease of administration
Slide 10: The Long Acting hGH
Lagova The Long Acting hGH Global multi-center Phase 3 study in growth hormone deficient adults is ongoing
Slide 11: Compliance in Children Receiving Nutropin tend to reduce over time
Compliance in Children Receiving Nutropin tend to reduce over time Daily dosing is consistently found to have a baseline of non-compliance – Most pronounced in the pediatric population 100% 100% 92% 91% 89% 90% 84% 82% e 80% 79% 75% 80% 73% Persistenc 70% 67% 60% Percent 50% 40% 30% 20% 1M 2M 3M 4M
Slide 12: Persistence Rates Among Nutropin Patients Have Been Declining Steadily Over Time
Persistence Rates Among Nutropin Patients Have Been Declining Steadily Over Time Persistence Rates From NCGS (Mainly Pediatric) 100% 95% 92% 92% 92% 92% 93% 93% 92% 92% 91% 90% 91% 91% 91% 91% 87% 89% 88% 81% 81% 81% 85% 80% 80% 80% 81% 80% 81% 81% 81% 81% 79% 81% 79% GH 80% 76% 77% 74% n 70% 1y 68%
Slide 13: Poor Compliance Leads to Slower Growth
Poor Compliance Leads to Slower Growth Cutfield et al 2011 HVSDS Low Medium High 0 1 2 3 4
Slide 14: Pediatric Phase Study Design
Pediatric Phase 2 Study Design C1= 0.25 mg/kg/week (0.18 mg hGH/kg/week C2 = 0.48 mg/kg/week (0.35 mg hGH/kg/week) C3 = 0.66 mg/kg/week (0.48 mg hGH/kg/week) C4 (hGH)= 0.24 mg/kg/week SCREENING 4 weeks MOD-4023 Genotropin* C1 C2 C3 C4 2 WEEKS 2 WEEKS 2 WEEKS DOSE1 PK/PD PERIOD DOSE 1 DOSE 2 DOSE 1 D
Slide 15: Baseline Characteristics – Patients Completing Months Treatment
Baseline Characteristics – Patients Completing 6 Months Treatment Cohort 1 Cohort 2 Cohort 3 Cohort 4 Dose 0.25 mg/kg/week 0.48 mg/kg/week 0.66 mg/kg/week 0.034 mg/kg/day Lagova Lagova Lagova Genotropin hGH content 0.18 mg/kg/week 0.35 mg/kg/week 0.66mg/kg/week 0.24 mg/kg/week N 9 9 10 7 Mean SD Mea
Slide 16: Increases Height Velocity SDS Following 6m Treatment
Lagova Increases Height Velocity SDS Following 6m Treatment Pre-Study HV SDS 6m HV SDS Cohort Dose N Mean Std Dev Cohort Dose N Mean Std Dev 0.25 mg/kg/w 0.25 mg/kg/w Cohort 1 8 -3.21 2.05 Cohort 1 8 5.03 1.23 Lagova™ Lagova™ 0.48 mg/kg/w 0.48 mg/kg/w Cohort 2 8 -2.94 1.14 Cohort 2 8 3.23 1.88 Lagov
Slide 17: Increases Height Velocity in All Doses
Lagova Increases Height Velocity in All Doses Mean Cohort Dose hGH Content N Std Dev (cm/year) Cohort 1 0.25 mg/kg/w Lagova™ 0.18 mg/kg/week 9 13.48 2.71 Cohort 2 0.48 mg/kg/w Lagova™ 0.35 mg/kg/week 9 12.25 2.64 Cohort 3 0.66 mg/kg/w Lagova™ 0.48 mg/kg/week 10 14.37 5.26 Cohort 4 0.034 mg/kg/d Geno
Slide 18: Pharmacokinetic profile
Lagova Pharmacokinetic profile Average Lagova Weekly PK Profile (Cohort 1-3) 1000.0 ml) Cohort 1 Cohort 2 (ng/ 100.0 Cohort 3 Level Serum va™ 10.0 Lago 1.0 0 24 48 72 96 120 144 168 Hours PK profile supports once-weekly regimen
Slide 19: Normalizes IGF-1 During the First Months of Treatment
Lagova Normalizes IGF-1 During the First 6 Months of Treatment 3.0 Cohort 1 Cohort 2 2.0 Cohort 3 Cohort 4 SDS 1.0 1 IGF - 0.0 5 10 15 20 25 -1.0 -2.0 -3.0 Week post dosing IGF-1 SDS of Cohort 2 and 3 are comparable to daily hGH Gradual increase in IGF-1 SDS values reaching steady state within the n
Slide 20: Promising Safety Profile
Promising Safety Profile No serious adverse events No lipoatrophy No clinically significant local tolerability issues were identified. Comparable rate of AEs between Lagova groups and control group
Slide 21: The Long Acting hGH
Lagova The Long Acting hGH Study supports safety and efficacy and allows selection of a dose for a Phase 3 study which is expected to start in 2015
Slide 22: The Long Acting hGH
LagovaThe Long Acting hGH hGH content is ~75% CTP is a naturally occurring peptide Once a week injection Non Viscous-30 -31G needle High concentrations-single weekly injection Address $3.5B market at an annual growth of 6%
Slide 23: Thank You
Thank You OPKO