Ocugen announces U.S. FDA cleared the IND amendment to initiate Phase 2/3 trial

Ocugen (OCGN) announced that the U.S. Food and Drug Administration, FDA, has cleared the Investigational New Drug, IND, amendment to initiate a Phase 2/3 pivotal confirmatory trial of OCU410ST, a modifier gene therapy candidate being developed for all Stargardt disease. The FDA previously granted Rare Pediatric Disease Designation, RPDD, and Orphan Drug Designation for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3. Positive data from the Phase 1 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-48% at 12-month follow up in evaluable treated eyes when compared to untreated eyes; Statistically significant improvement with clinically meaningful, nearly 2-line gain in visual function at 12-month follow-up in evaluable treated eyes when compared to untreated eyes