Ocugen Eyes 3 Retinal Gene Therapy Filings as Late-Stage Pipeline Advances

Ocugen plans three late-stage retinal gene therapy filings, targeting BLAs for retinitis pigmentosa and Stargardt disease next year and for geographic atrophy in 2028. The company says it aims to deliver two top-line readouts in 2025 and a third in geographic atrophy later on. The geographic atrophy program (OCU410) is moving into Phase 3 with a medium-dose selection based on Phase 2 data showing about a 33% reduction in lesion growth. Ocugen expects to enroll fewer than 300 patients in an adaptive trial designed with regulators in the U.S. and Europe. OCU410ST for Stargardt disease and OCU400 for retinitis pigmentosa are also advancing, with Stargardt’s pivotal study potentially supporting a BLA filing around mid-next year and retinitis pigmentosa top-line data expected in the first half of next year. Ocugen says its therapies are intended as one-time treatments across large patient populations.

Ocugen NASDAQ: OCGN outlined plans to advance three late-stage retinal disease programs, with company speaker Shankar saying the company is targeting biologics license applications for retinitis pigmentosa and Stargardt disease next year and a third BLA for geographic atrophy in 2028. Speaking during a Piper Sandler discussion with analyst Biren Amin, Shankar said Ocugen is focused on inherited retinal diseases and dry age-related macular degeneration, including retinitis pigmentosa, Stargardt disease and geographic atrophy, the late form of dry AMD. He cited more than 100,000 retinitis pigmentosa patients in the U.S., about 50,000 Stargardt disease patients in the U.S. and 2 million to 3 million geographic atrophy patients in the U.S. and EU in the late stage of disease.