Ocugen (OCGN) Stifel 2026 Virtual Ophthalmology Forum summary

Pipeline and clinical development

• Advancing three gene therapy programs for retinal diseases: retinitis pigmentosa (RP), Stargardt disease, and geographic atrophy (GA), targeting large patient populations with significant unmet needs.

• OCU400 for RP completed phase III enrollment; top-line results expected in Q1 next year, with rolling BLA submission planned soon after.

• OCU410ST for Stargardt is in a phase II/III registration trial, with interim analysis in Q3 and top-line results expected in Q2 next year.

• OCU410 for GA showed promising phase II data; phase III initiation targeted for later this year, aiming for BLA approval in 2028.

• All three programs aim for BLA filings by 2028, with regulatory alignment in both the US and EU.

Scientific approach and differentiation

• Uses master regulator modifier genes (NR2E3 for RP, RORA for Stargardt and GA) to reset retinal cell homeostasis and address disease networks, not just single mutations.

• Approach enables treatment of broad patient populations, including those with diverse or unknown genetic mutations.

• Demonstrated ability to slow, stall, or even reverse disease progression in some patients, with effects observed in both early and advanced disease stages.

• Modifier gene therapy creates a healthy environment for non-dividing retinal cells, potentially offering lifelong benefit from a single treatment.

• Endpoints for trials are based on FDA-approved measures, such as mobility tests for RP and lesion growth for Stargardt and GA.

Clinical trial design and regulatory strategy

• RP phase III trial enrolled 140 patients in a 2:1 treated-to-control ratio; expanded access program allows up to 75 additional patients.

• Rolling BLA submission enabled by RMAT designation, with non-clinical and CMC modules to be submitted ahead of clinical data.

• Adaptive trial designs and interim analyses used to minimize risk and optimize trial size and duration.

• Stargardt and GA trials use lesion growth as primary endpoint, with potential for earlier filing if interim results are strong.

• All clinical data, including long-term safety and expanded access, will be included in regulatory submissions.