Ocugen Says FDA Cleared IND Amendment To Initiate Phase 2/3 Study Of OCU410ST In Stargardt Disease

(RTTNews) - Ocugen, Inc. (OCGN), Monday said 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 study of OCU410ST, the company's gene therapy candidate being developed for the treatment of Stargardt disease.

Stargardt disease is a genetic eye condition that causes progressive vision loss.

In the Phase 2/3 study, 34 out 51 participants will receive a one-time subretinal injection of OCU410ST, and the rest will be assigned to an untreated control group. The primary goal of the study is to evaluate the reduction in atrophic lesion size.

Ocugen plans to submit a Biologics License Application for OCU410ST in 2027.

The FDA had 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.