Opthea Limited · ASX:OPT
Opthea — Investor Presentation (January 2025)
33 slides from Opthea's investor presentation, Jan 14, 2025
Slide 1: Transforming Patient Outcomes with Superior Vision Gains
Transforming Patient Outcomes with Superior Vision Gains Corporate Overview | January 2025 NASDAQ (OPT); ASX (OPT.AX)
Slide 2: Disclaimer and Forward-looking Statements
Disclaimer and Forward-looking Statements This presentation includes general background information about the activities of Opthea Limited (ABN 32 006 340 567) (“Opthea” or “Company”) and its affiliates and subsidiaries (together, the “Opthea Group”). The information contained in this presentation i
Slide 3: Sozinibercept Has the Potential to Be the First Product in 20 Years to Deliver Superior Visual Outcomes
Sozinibercept Has the Potential to Be the First Product in 20 Years to Deliver Superior Visual Outcomes Addressing High • Despite wide use of anti-VEGF-A therapy, wet AMD patients still experience loss in vision long term1 Unmet Need • Every letter of vision counts to improve quality of life and red
Slide 4: Sozinibercept Designed to Deliver Superior Visual Outcomes in Combo with VEGF-A Inhibitors
Sozinibercept Designed to Deliver Superior Visual Outcomes in Combo with VEGF-A Inhibitors; Potential to Create New Multi-Billion Dollar Medicine Global Marketed VEGF-A Inhibitors Sozinibercept is a VEGF-C/D “Trap” Inhibitor Potential value proposition: Targeting Improved Visual Function ~$15bn Glob
Slide 5: Experienced Leadership Team
Experienced Leadership Team Expertise and Track Record to Make a Positive Impact on the Retinal Community Management Team Chief Medical Advisor Arshad M. Khanani, MD, MA, FASRS Managing Partner, Director of Clinical Research Fred Guerard, PharmD, MS and Director of Fellowship at Sierra Eye Chief Exe
Slide 6: Improving Vision Now the Largest Unmet Need in Wet AMD for Retina Specialists
Improving Vision Now the Largest Unmet Need in Wet AMD for Retina Specialists ASRS PAT Survey 2023 ASRS PAT Survey 2024 What are the greatest unmet needs in treating wet AMD and DME? Which factors are more important to you when selecting an anti-VEGF agent? n=1,012 n=1,021 US 78.6% Abilitytotoimprov
Slide 7: Despite Treatment with Standard of Care Anti-VEGF-A Therapies, the Majority of Patients Achieve Suboptimal Vision Outcomes
Despite Treatment with Standard of Care Anti-VEGF-A Therapies, the Majority of Patients Achieve Suboptimal Vision Outcomes Despite treatment with anti-VEGF-A therapy* The majority of patients fail to achieve 20/40 vision1 >45% do not achieve significant vision gains >60% will have persisting macular
Slide 8: Every Letter Counts; Vision Loss Associated with Increased Mortality Risk
Every Letter Counts; Vision Loss Associated with Increased Mortality Risk Hazard for All-cause Mortality1 Higher in People with Vision Impairment 89% Decrease of 1 ETDRS letter per year 43% expected to increase mortality risk by 16%2 29% associated exclusively with IADL levels < 20/40 vs. ≥ 20/40 <
Slide 9: U.S. Retina Specialists Willing to Administer Second Injection to at Least 24% of Their Patients
U.S. Retina Specialists Willing to Administer Second Injection to at Least 24% of Their Patients for Additional BCVA Improvement BCVA BCVA BCVA BCVA improvement improvement improvement improvement over 5 of 4.1 - 5 of 3.0 - 4 under 3 Average Percentage of Patients Treated 41% 32% 25% 24% with Second
Slide 10: Sozinibercept, a First-In-Class VEGF-C/D Trap Inhibitor, Has the Potential to Address the Limitations of Anti-VEGF-A Therapies
Sozinibercept, a First-In-Class VEGF-C/D “Trap” Inhibitor, Has the Potential to Address the Limitations of Anti-VEGF-A Therapies Faricimab (Vabysmo®)1 The Problem The Solution Wet AMD is a multi-factorial When used in combination disease. Treatment with VEGF-A with any VEGF-A inhibitor, inhibitors u
Slide 11: Published Evidence Supports Broader VEGF Pathway Inhibition with Sozinibercept
Published Evidence Supports Broader VEGF Pathway Inhibition with Sozinibercept Circulating VEGF-C Levels Significantly VEGF-C Stimulates RetinalAngiogenesis^ Elevated in AMD Patients ⍭ 300 Plasma concentrations (pg/ml) Control ** AMD Additive Benefit of VEGF-A and 200 VEGF-C/D Inhibition in Mouse We
Slide 12: Sozinibercept Has Demonstrated Improvement in Vision Gains and Reduction in Vision Loss
Sozinibercept Has Demonstrated Improvement in Vision Gains and Reduction in Vision Loss Opportunity in Wet AMD Market for an Overall Shift Towards Superior Visual Outcomes High QoL SOZINIBERCEPT Lucentis remains the standard of care for visual improvements Visual Acuity 20/40 AVASTIN (OFF LABEL) VAB
Slide 13: Long-term Value Opportunities for Sozinibercept
Long-term Value Opportunities for Sozinibercept Main Patent Family Extends through 2034, with Expansion Opportunities Beyond 2034* DEVELOPMENT PHASE ANTICIPATED PROGRAM RESEARCH / MILESTONES PHASE 1 PHASE 2 PHASE 3 PRECLINICAL Wet Age-Related Macular Degeneration (Wet AMD) Sozinibercept Topline data
Slide 14: Next Steps
Advancing Therapeutic Innovations to Transform Patient Outcomes with Superior Vision Gains We are dedicated to advancing sozinibercept to improve patients’ visual outcomes • Phase 3 program enrolled 1,984 patients across COAST and ShORe Clinical Milestones • Topline data anticipated for COAST in ear
Slide 15: Robust Phase 2b Trial in Wet AMD Demonstrated Superiority in Visual Outcome
Robust Phase 2b Trial in Wet AMD Demonstrated Superiority in Visual Outcome Treatment naïve patients with neovascular AMD Key Inclusion Criteria Key Exclusion Criteria ✓ Active CNV >50% lesion, classic × Subfoveal fibrosis or >25% of total lesion Screening / minimally classic / occult × Hemorrhage >
Slide 16: Phase 2b Primary and Secondary Endpoints
Phase 2b Primary and Secondary Endpoints Pre-Specified Anatomical Sub-Groups Informed Enrichment of Phase 3 Program Primary Endpoint Select Pre-specified Subgroups Mean change from baseline in BCVA at week 24 Predominantly classic, minimally classic, & occult lesions (Stratification Factor) Key Seco
Slide 17: Well-Balanced Phase 2b Trial Demographics and Baseline Characteristics
Well-Balanced Phase 2b Trial Demographics and Baseline Characteristics Sham + 0.5 mg sozinibercept + 2 mg sozinibercept + Demographic/Baseline Disease Characteristic ranibizumab ranibizumab ranibizumab n=121 n=122 n=123 Mean Age – years ± SD 76.1 ± 9.48 78.8 ± 8.16 77.8 ± 8.82 Male 48 (39.7%) 49 (40
Slide 18: Sozinibercept Combination Therapy Demonstrated Over 30% Improvement in Visual Acuity over Ranibizumab Monotherapy
Sozinibercept 2 mg Combination Therapy Demonstrated Over 30% Improvement in Visual Acuity over Ranibizumab Monotherapy Phase 2b Primary Endpoint Achieved Mean Change in BCVA (SEM) (Letters) 20 Total Patient Population 15 +14.2 Δ = +3.4 +10.8 (p=0.0107) 10 5 0 0 4 8 12 16 20 24 Weeks Sham + 0.5 mg ra
Slide 19: Phase 2b Superiority Data Informed Enrichment of Phase 3
Phase 2b Superiority Data Informed Enrichment of Phase 3 Occult & Minimally Classic Lesions (RAP Absent) Total Patient Population (RAP Absent) Represents ~75%1 of wet AMD patients Represents ~90%2 of wet AMD patients 20 20 Mean Change in BCVA in BCVA(SEM) Mena Change in BCVA (SEM) (Letters) +16.1 (S
Slide 20: Greater Proportion of Sozinibercept Patients Gained Substantial Vision and Fewer Experienced Vision Loss
Greater Proportion of Sozinibercept Patients Gained Substantial Vision and Fewer Experienced Vision Loss Change from Baseline to Week 24 (ETDRS Letters, Individual Participants) Occult and Minimally Classic Lesions (RAP Absent) Vision Loss >30 Letter Gain 2.3% 9.2% 2 mg sozinibercept + ranibizumab N
Slide 21: Sozinibercept Reduced Retinal Thickness and Dried the Retina Better
Sozinibercept Reduced Retinal Thickness and Dried the Retina Better Mean Change in CST % of Participants with SRF % of Participants with IR Cysts Baseline to Week 24 at Week 24 at Week 24 -60 40 40 85 86 Mean Change in CST (SEM) (µm) 28.2 % Participants -100 % Participants 20 20 -126.8 19.8 -133.5 1
Slide 22: Sozinibercept Demonstrated Greater CNV and Lesion Regression
Sozinibercept Demonstrated Greater CNV and Lesion Regression Mean Change in Total Lesion Area Mean Change in CNV Area at Week 24 at Week 24 Occult & Minimally Classic (RAP Absent) Patients Sham + 0.5 mg ranibizumab 2 mg sozinibercept + 0.5 mg ranibizumab (n=80) (n=81) 22 mITT; as observed; top of ba
Slide 23: Superior Vision Gains in Hard-To-Treat PCV Lesion Patients
Superior Vision Gains in Hard-To-Treat PCV Lesion Patients PCV Lesions 20 Polypoidal Choroidal Vasculopathy (PCV) is a Δ +6.7 difficult-to-treat wet AMD subtype; it is often described as the most prevalent form of Mean Change in BCVA at Week 24 p = 0.0253* wet AMD worldwide 15 (SEM) (Letters) 13.5 1
Slide 24: Sozinibercept Was Well Tolerated
Sozinibercept Was Well Tolerated Safety of Combination Therapy Comparable to Standard of Care Monotherapy Sozinibercept Sozinibercept Sham + anti-VEGF-A Any dose* 2 mg control N Participants (%) N=399 N=263 N=170 (N=1,842 injections) (N=1,121 injections) (N=854 injections) Ocular TEAEs - Study Eye –
Slide 25: Similar Rate of Intraocular Inflammation Between Standard Of Care and Sozinibercept in Combination Therapy
Similar Rate of Intraocular Inflammation Between Standard Of Care and Sozinibercept in Combination Therapy Sozinibercept Sozinibercept Sham + anti-VEGF-A Any dose* 2 mg control N Participants (%) N=399 N=263 N=170 (N=1,842 injections) (N=1,121 injections) (N=854 injections) Intraocular Inflammation1
Slide 26: Phase 3 Clinical Program Is Informed by Phase 2b Results and Optimized for Success
Phase 3 Clinical Program Is Informed by Phase 2b Results and Optimized for Success Enriched patient population by excluding RAP lesions (+4.4 letters in Phase 2b); key inclusion and exclusion criteria otherwise unchanged Hierarchical primary analysis first conducted in the high-responding occult and
Slide 27: Global Pivotal Program Involves 33 Countries and ~400 Sites
Global Pivotal Program Involves 33 Countries and ~400 Sites Multi-center, sham controlled, double-masked trials in treatment Combination with Aflibercept Combination with Ranibizumab naïve wet AMD patients Enrolled (N=998) Enrolled (N=986) Randomized (1:1:1) Randomized (1:1:1) Key Inclusion Criteria
Slide 28: Pivotal Trial Design Supports Potential Broad Label for Use With Any Anti-VEGF-A Therapy
Pivotal Trial Design Supports Potential Broad Label for Use With Any Anti-VEGF-A Therapy Key Secondary Endpoints Primary Endpoint Topline Data (Baseline to Week 52) • Proportion of participants • Change in CNV area gaining ≥15 letters COAST in early 2Q CY 25 • Mean change in BCVA from baseline to we
Slide 29: Phase 3 Enrolled a Higher Proportion of Patients With Best Responding Lesion Types Compared to Phase 2b
Phase 3 Enrolled a Higher Proportion of Patients With Best Responding Lesion Types Compared to Phase 2b Phase 2b Phase 3 Sham + 2 mg sozinibercept + ranibizumab ranibizumab COAST ShORe Demographic/Baseline Disease Characteristic N=997* N=985* n=121 n=123 Mean Age – years ± SD 76.1 ± 9.48 77.8 ± 8.82
Slide 30: Higher Proportion of Patients With Best Responding Lesion Types
Higher Proportion of Patients With Best Responding Lesion Types Occult (RAP Absent) Lesion Types PCV Lesion Types Phase 2b Proportion of Phase 2b Proportion of Δ +6.5 Population at Baseline Δ +6.7 Population at Baseline p = 0.0005* p = 0.0253* Phase 2b Phase 2b 43.9% 17.2% COAST COAST 55.7% 26.2% Sh
Slide 31: Cash Runway Through Both Pivotal Topline Data Readouts
Cash Runway Through Both Pivotal Topline Data Readouts Financial Overview Development Funding Agreement (DFA) Ticker OPT (ASX/NASDAQ) • Total funding drawn under DFA: US$170M • Provides non-dilutive funding for development of Ordinary Shares: 1,231.1M sozinibercept Shares Outstanding1 ADS equivalent
Slide 32: Anticipated Clinical and Manufacturing Timelines Support BLA Submission in 1H26 and Potential Approval by End of CY2026
Anticipated Clinical and Manufacturing Timelines Support BLA Submission in 1H26 and Potential Approval by End of CY2026 Drug product PPQ ShORe topline data release in early CY25 in mid-CY25 Potential BLA approval as early as end of CY26 COAST topline data BLA submission in early 2Q CY25 in 1H CY26 F
Slide 33: Thank You.
Thank You. UPCOMING EVENT: Opthea Investor Days Discuss commercial insights and readiness plans Location Time Format New York City Jan 28 1:30 pm EST In-person & virtual Sydney Feb 3 4:30 pm AEDT In-person Melbourne Feb 5 4:30 pm AEDT In-person Details under “Events & Presentations” of the Investor