Opthea (OPT) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Key product and clinical insights

• Sozinibercept is a first-in-class anti-VEGF-C/D molecule developed for combination with standard anti-VEGF-A therapies to improve vision outcomes in retinal diseases.

• Phase II-B trial showed statistically significant superiority in vision benefit at week 24, with a 3.4-letter gain overall and 5.7-letter gain in harder-to-treat subgroups.

• High-dose sozinibercept is advancing to phase III, targeting minimally classic and occult lesion patients, who represent about 75% of real-world cases.

• Combination therapy led to a 42% increase in patients reaching the 20/40 vision threshold, critical for daily activities like driving.

• Safety profile was consistent with standard anti-VEGF-A treatments, with no new safety signals observed.

Market opportunity and business strategy

• Wet AMD market is estimated at $10 billion annually, with additional indications like diabetic macular edema under development.

• 1% market penetration equates to $100 million in annual revenue; survey data suggests strong adoption if vision gains are demonstrated.

• Business model leverages concentrated retina clinics in the U.S. for efficient commercial launch.

• Co-formulation feasibility trials are ongoing to combine sozinibercept with anti-VEGF-A in a single vial.

• Recent financing provides over $200 million in cash, supporting operations through key phase III readouts.

Clinical development and regulatory pathway

• Two global phase III trials (COAST and ShORe) are fully enrolled, with readouts expected in early and mid-2025.

• Trials compare sozinibercept plus standard of care (Lucentis or Eylea) versus monotherapy, with primary endpoint at week 52.

• Design is classical, using well-established endpoints and comparators as per label, aligning with FDA expectations.

• Fast-track designation from FDA may expedite review; manufacturing scale-up and BLA preparation are underway.

• No defined FDA superiority threshold, but focus is on vision improvement and totality of data for approval and reimbursement.