Opthea Limited · ASX:OPT
Opthea — Corporate Update (April 2017)
21 slides from Opthea's corporate update, Apr 3, 2017
Slide 1: OPT-302: Phase 1/2A wet AMD Trial Update
OPT-302: Phase 1/2A wet AMD Trial Update Corporate Update, April 3 2017 Megan Baldwin, CEO & Managing Director
Slide 2: Disclaimer
Disclaimer Investment in Opthea Limited (‘Opthea’) is subject to investment risk, including possible loss of income and capital invested. Neither Opthea nor any subsidiary of Opthea guarantees any particular rate of return or performance, nor do they guarantee the repayment of capital. This presenta
Slide 3: Corporate Summary
Corporate Summary • OPT-302 is in development for the treatment of wet Age-related Macular Degeneration (‘wet AMD’), a progressive eye disease that is the leading cause of blindness in people over the age of 50 years • OPT-302 blocks VEGF-C and VEGF-D that cause vessels to grow and leak and are hall
Slide 4: Trial Results
A Phase 1/2A dose escalation study evaluating the safety, pharmacokinetics and pharmacodynamics of OPT-302 in combination with ranibizumab (Lucentis®) in subjects with wet AMD Trial Results 4
Slide 5: Phase 1/2A trial design: dose escalation and expansion
OPT-302 Phase 1/2A ClinTrials.gov ID#: NCT02543229 Dose-escalation & dose-expansion of repeated IVT injections Phase 1: Dose-escalation Phase 2A: Dose-expansion (Open-label) (Randomised) OPT-302 (2.0 mg) OPT-302 (2.0 mg) Monotherapy* Monotherapy* 24 IVT Q4W x 3, n=5 IVT Q4W x 3, n=8 pts at Week Foll
Slide 6: OPT-302 Phase 1/2A: Patient Cohorts
OPT-302 Phase 1/2A: Patient Cohorts Phase 1/2A • 51 patients, 32 (63%) females, 19 (37%) males, mean age 77 years • 37 /51 (73%) occult, 12/51 (23%) min classic, 2/51 (4%) predominantly classic • Mean min classic component 5.9% • 49% treatment-naïve • 51% difficult to treat patients sub-responsive t
Slide 7: Phase 1/2A Visual Acuity Outcomes
Phase 1/2A Visual Acuity Outcomes In MARINA trial (Phase 3 registrational): ~6 letters gain in vision compared to baseline at Week 12 in patients with minimally classic/occult wet AMD lesions treated with Lucentis® (ranibizumab) Opthea’s Phase 1/2A trial recruited patients with wet AMD lesion types
Slide 8: Phase 1/2A: Patients Gaining or Maintaining Vision at Week 12
Phase 1/2A: Patients Gaining or Maintaining Vision @ Wk12 • 51 patients enrolled • 49 patients evaluable at the week 12 endpoint Cohort Treatment # # pts Gained # pts # pts # pts lost less # pts lost b/w patients or Maintained Gained Maintained than 5 letters >5 = <10 letters ALL: OPT-302 (0.3/1.0/2
Slide 9: Naïve Patients OPT-302 + Lucentis
Phase 1/2A: Naïve Patients OPT-302 (0.3 & 2mg) + Lucentis (0.5mg) ® Mean Change in Visual Acuity from Baseline (letters) Mean Sub-Retinal Fluid (SRF) (uM) Mean Central Subfield Thickness (CST) (uM) Naïve patients: • Mean gain in visual acuity at week 12 from baseline was +10.8 letters • In the MARIN
Slide 10: Naïve Patients: Improved Visual Outcome through week 12
Phase 1/2A: Naïve Patients OPT-302 (0.3 & 2mg) + Lucentis (0.5mg) ® Improved Visual Outcome through week 12 ≥ 2 line gain ≥ 3 line gain 60 35 50 30 25 40 % Patients % Patients 20 30 15 20 10 10 5 0 0 Week 4 Week 8 Week 12 Week 4 Week 8 Week 12 Week 12 OPT-302 + Lucentis® OPT-302 + Lucentis® Lucentis
Slide 11: Prior-Treated Patients: Visual Acuity
Prior-Treated Patients: Visual Acuity • Majority of vision gain in Lucentis® treated patients occurs within 3 months • Plateau “ceiling effect” of response with no other treatment options • Difficult to treat patient population, very large market opportunity Plateau of Response after 3 mos MARINA Ph
Slide 12: Prior-Treated Patients OPT-302 + Lucentis
Phase 1/2A: Prior-Treated Patients OPT-302 (0.3, 1 & 2mg) + Lucentis (0.5mg) ® Mean Change in Visual Acuity from Baseline (letters) Mean Sub-Retinal Fluid (SRF) (uM) Mean Central Subfield Thickness (CST) (uM) Prior-Treated patients: • Mean number prior anti-VEGF-A injections per patient: 17 • Mean g
Slide 13: OPT-302 Monotherapy
OPT-302 Monotherapy • OPT-302 monotherapy included to identify clinical activity without a background standard of care • Natural history of wet AMD: without treatment, often chronic, rapid decline in visual acuity and increase in retinal fluid • a-VEGF-A rescue therapy offered to patients at physici
Slide 14: Monotherapy Patients OPT-302 (2mg) Naïve & Prior-Treated
Phase 1/2A: Monotherapy Patients OPT-302 (2mg) (Naïve & Prior-Treated) Mean Change in Visual Acuity in Non- Visual Acuity in Non-Rescue Patients Rescue Patients at Each Time Point at Each Time Point Naïve Patients Prior Tx Patients 25 25 20 20 ( M e a n C h a n g e fr o m B a s e lin e ) ( M e a n C
Slide 15: Monotherapy Patients OPT-302 (2mg) Naïve & Prior-Treated
Phase 1/2A: Monotherapy Patients OPT-302 (2mg) (Naïve & Prior-Treated) • 7/13 (54%) patients did not require ‘rescue’ therapy Visual Acuity in Patients who were Sub-Retinal Fluid in Patients who not rescued by Week 12 were not rescued by Week 12 ( M e a n C h a n g e fr o m B a s e lin e ) 200 192 1
Slide 16: Phase 1/2A Safety Summary
Phase 1/2A Safety Summary • OPT-302 Lucentis® administered by repeat IVT injection (Baseline, Week 4, Week 8) • No missed doses, safety experience with ~150 intravitreal (ocular) injections of OPT-302 • OPT-302 at ocular doses up to 2 mg Lucentis® (0.5 mg): • No dose limiting toxicities (MTD was
Slide 17: OPT-302 Phase 1/2A: Key Take-Aways
OPT-302 Phase 1/2A: Key Take-Aways • OPT-302 met primary safety objective of Phase 1/2A study (well tolerated) • Evidence of clinical activity of OPT-302 (anti-VEGF-C/D) in patients including heavily pre-treated patients (51%) and a high proportion of occult (73%) wet AMD lesions: • Naïve Patients:
Slide 18: OPT-302 Proposed Phase 2B in Wet AMD
OPT-302 Proposed Phase 2B in Wet AMD Combination OPT-302 + Lucentis® vs Lucentis® OPT-302 (Dose 1) + Lucentis® (0.5 mg) Primary Endpoint: Mean Change in Visual Acuity from Baseline at Week 24 Wet AMD Secondary OPT-302 (Dose 2) + Lucentis® (0.5 mg) Endpoints: Naïve Pts Retinal Thickness (SD-OCT, wk 1
Slide 19: OPT-302 Proposed Phase 2A in Diabetic Macular Edema
OPT-302 Proposed Phase 2A in Diabetic Macular Edema Combination OPT-302 + Lucentis® vs Lucentis® Dose-escalation Phase 2A: Randomized (1:1) Dose-expansion Long term follow-up at Week 24 Lucentis® (0.3 mg) Std.of Care week1212 IVT Q4W x 3,~n=40 pts Follow-uptotoweek OPT-302 (TBD) 28 Day DLT window +
Slide 20: Opthea – Developing OPT-302 for Eye Diseases
Opthea – Developing OPT-302 for Eye Diseases • OPT-302 has broad development potential in a range of eye diseases, including wet AMD and DME • Targets validated pathway involved in wet AMD progression and mechanism of escape from existing therapies that is differentiated to a-VEGF-A therapies • Larg
Slide 21: Opthea contact details
Suite 0403, Level 4, 650 Chapel Street, South Yarra 3141 Victoria Australia T +61 (3) 9826 0399 E [email protected] www.opthea.com