Ocugen, Inc. 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 (ABCA4-associated retinopathies). 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 (p=0.031) improvement with clinically meaningful, nearly 2-line gain in visual function (BCVA) at 12-month follow-up in evaluable treated eyes when Compared to untreated eyes. The Phase 2/3 clinical trial for OCU410ST will enroll 51 participants diagnosed with Stargardt disease.
Of these, 34 will receive a one-time subretinal injection of OCU410ST (200 mL at a concentration of 1.5 x 1011 vector genomes/mL) in the eye with poorer visual acuity, while 17 will be assigned to an untreated control group. The OCU410ST Phase 2/3 pivotal confirm confirmatory trial represents a major advancement as Ocugen's second late-stage clinical program. Ocugen plans to submit a BLA for OCU410ST in 2027 in alignment with its strategic goal of filing three BLAs over the next three years.
