Opthea (OPT) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Key product and clinical development

• Sozinibercept is a first-in-class VEGF-C and -D trap developed for use with standard anti-VEGF-A therapies to improve visual outcomes in wet AMD patients.

• Phase II-B trial showed superior efficacy and safety for sozinibercept plus standard of care versus standard of care alone, with a 3.4-letter gain overall and 5.7-letter gain in biomarker-positive subgroups.

• Two pivotal phase III trials, COAST and SHORE, are fully enrolled with about 1,000 patients each; top-line data expected in early to mid-2025.

• The drug is being developed for use with any anti-VEGF-A therapy, offering flexibility and potential fixed-dose combinations.

• Plans are underway to expand into diabetic macular edema after wet AMD data readout.

Market opportunity and positioning

• Wet AMD is a $15 billion annual market, dominated by a few players, with Eylea and Vabysmo as leading drugs.

• Sozinibercept targets improved vision outcomes, unlike competitors focused on injection durability.

• No competitors are at a similar stage in clinical development for this approach.

• The market is highly concentrated, with a few hundred retina centers in the U.S. administering most injections.

• Commercial and market access teams are being built in preparation for launch.

Clinical rationale and trial design

• Current anti-VEGF-A therapies leave VEGF-C and -D pathways unblocked, which can limit vision gains.

• Sozinibercept blocks these additional pathways, aiming for better disease control and vision improvement.

• Phase II-B and III trials include treatment-naive patients, with primary endpoints focused on best-corrected visual acuity at one year.

• Combination therapy showed anatomical and functional benefits, especially in hard-to-treat subgroups like polypoidal choroidal vasculopathy.

• Safety profile is comparable to standard anti-VEGF-A therapies, with no increased risk of inflammation.