Ocugen reports FDA alignment on Phase 2/3 OCU410ST trial in Stargardt disease

Ocugen (OCGN) announced that alignment has been reached with the FDA to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which, if positive, can be the basis of a biologics license application submission. The GARDian trial for OCU410ST demonstrated: a favorable safety and tolerability profile with no serious adverse events related to OCU410ST, including no cases of ischemic optic neuropathy, vasculitis, intraocular inflammation, endophthalmitis or choroidal neovascularization and no adverse events of special interest; considerably slower lesion growth from baseline in treated eyes versus untreated fellow eyes at 6-month follow-up from the Phase 1 study; clinically meaningful 2-line improvement in visual function at 6-month follow-up from the Phase 1 study, which is statistically significant in treated eyes. Stargardt disease affects 100,000 people in the U.S. and Europe combined, and there is no treatment. OCU410ST received orphan drug designations from the FDA and the EMA in 2023 and 2024, respectively.