Ocular Therapeutix (OCUL) 49th Annual Macula Society Meeting presentation summary

Unmet needs in neovascular AMD (nAMD) management

• Current anti-VEGF therapies require frequent injections, leading to high treatment burden and poor long-term adherence, with up to 40% discontinuation in the first year and declining visual outcomes over time.

• Real-world data show fewer injections and worse vision outcomes compared to clinical trials, with attrition rates underestimated in literature and significant drop-off after year 1.

• Durable, low-burden therapies are needed to improve persistence and maintain vision.

OTX-TKI: Mechanism and delivery platform

• OTX-TKI uses axitinib, a highly selective and potent pan-VEGFR tyrosine kinase inhibitor, delivered via a bioresorbable hydrogel for sustained intraocular release.

• The hydrogel platform enables continuous drug delivery for up to 12 months, with complete bioresorption and no implant remnants.

• Axitinib demonstrates superior potency and selectivity for VEGFR1/2/3 and PDGFR compared to other TKIs.

SOL-1 Phase 3 trial design and regulatory alignment

• SOL-1 is a multicenter, double-masked, randomized superiority trial comparing a single OTX-TKI (0.45 mg) dose to a single aflibercept (2 mg) dose in treatment-naïve nAMD patients.

• The trial design aligns with FDA guidance, using identical dosing schedules in both arms and no sham injections, conducted under a Special Protocol Assessment (SPA) with the FDA.

• The primary endpoint is the proportion of subjects maintaining visual acuity (<15 ETDRS letter loss) at week 36.