Opthea (OPT) Jefferies 2024 Global Healthcare Conference summary

Strategic overview and market context

• Sozinibercept aims to be the first drug in nearly 20 years to show superior vision outcomes for wet AMD patients, complementing existing anti-VEGF-A therapies.

• Wet AMD remains the leading cause of vision loss in Western countries, with the market now valued at $14–15 billion annually.

• The drug is designed for use alongside any anti-VEGF-A, targeting broad adoption without disrupting current clinical practices or reimbursement models.

• Recent market changes include biosimilar launches and new drugs like Eylea high dose and Vabysmo, which are shifting treatment patterns.

• Individualized treatment regimens are standard, and sozinibercept is positioned to integrate seamlessly with these evolving protocols.

Clinical development and trial results

• Sozinibercept has composition of matter IP until at least 2034 and demonstrated superiority in a phase IIb trial when combined with Lucentis.

• Two pivotal phase 3 trials, COAST and SHORE, are fully enrolled, with top-line data expected mid-next year.

• The phase IIb trial showed a 3.4-letter vision improvement overall and a 5.7-letter gain in harder-to-treat subgroups at 24 weeks.

• Anatomical benefits included reduced retinal thickness, less fluid, and smaller lesion areas, indicating lower disease activity.

• Safety profile was similar to anti-VEGF-A therapies, with no significant inflammation or adverse signals.

Regulatory and commercialization plans

• Fast track designation from the FDA allows for modular BLA submission and potentially faster review.

• Manufacturing at commercial scale is underway, with three consecutive batches required for regulatory submission.

• The company is preparing for BLA filing and commercial readiness, supported by a team of regulatory and clinical experts.

• Reimbursement is expected to align with vision gains, and payers will have comparative data with both Lucentis and Eylea.

• The drug is also being studied for diabetic macular edema and as a fixed-dose co-formulation with anti-VEGF-A.