Ocugen (OCGN) H.C. Wainwright 28th Annual Global Investment Conference summary

Pipeline and regulatory strategy

• Three late-stage gene therapy programs target major inherited retinal diseases and dry AMD, aiming for broad patient coverage with a gene-agnostic approach.

• OCU400 for retinitis pigmentosa expects top-line phase III data in Q1 2027, with a rolling BLA submission planned.

• OCU410ST for Stargardt disease targets top-line pivotal data in Q2 2027, followed by BLA submission; EMA has waived additional clinical trials for both RP and Stargardt.

• OCU410 for geographic atrophy began phase III in 2023, with BLA submission targeted for late 2028; global trial expansion includes Europe.

• All programs are designed as one-time treatments, with significant unmet medical need and potential for priority review due to RMAT designation.

Operational execution and commercialization

• CMC and non-clinical modules are complete for OCU400, minimizing regulatory risk and enabling rapid BLA filing upon clinical data readout.

• Commercialization strategy focuses on U.S. launch with preparation for payer engagement, centers of excellence, and patient registries ahead of top-line data.

• Ex-U.S. commercialization will rely on strategic partnerships to maximize reach and manage capital efficiently.

• More than 300 patients have been dosed across trials with no product-related SAEs; surgical delivery is standardized to support broad adoption.

• Capital-intensive activities will be timed to coincide with positive data readouts to preserve cash runway.

Clinical trial updates and data monitoring

• OCU410ST for Stargardt disease is in a one-year phase II/III trial; a recent DMC review found no safety concerns and recommended continuation.

• Lesion size heterogeneity in interim data was noted, but full data at 12 months is expected to provide clearer efficacy signals.

• Early-stage data for Stargardt and RP show potential for not only slowing but also reversing disease progression in some patients.

• OCU410 for geographic atrophy showed a 31% lesion reduction in phase II; primary endpoint for phase III is lesion size at one year, with 237 patients targeted for robust statistical power.

• FDA and EMA have shown strong support, with RMAT designation and rapid regulatory alignment.