FDA Clears Ocugen’s OCU410ST To Begin Confirmatory Stargardt Study

Ocugen, Inc. has received FDA clearance to initiate a Phase 2/3 pivotal confirmatory trial of its gene therapy candidate OCU410ST for Stargardt disease, a rare inherited retinal disorder. The therapy targets ABCA4-associated retinopathies and has already earned Rare Pediatric Disease and Orphan Drug Designations from the FDA. The upcoming trial will be Ocugen’s second late-stage clinical program and a key component of its strategic plan to file three Biologics License Applications (BLAs) by 2027.

“Stargardt disease represents a significant unmet medical need, particularly among children and young adults. The Phase 2/3 study of OCU410ST is thoughtfully designed with scientific rigor and a patient-centered focus to evaluate both structural and functional outcomes. We are optimistic that this approach will move us closer to a meaningful therapeutic solution for affected families,” said Lejla Vajzovic, MD, FASRS, Director of the Duke Surgical Vitreoretinal Fellowship Program and Professor of Ophthalmology, Pediatrics, and Biomedical Engineering with Tenure at Duke University Eye Center. 

The IND clearance follows positive results from the Phase 1 GARDian trial, where OCU410ST demonstrated a favorable safety profile with no serious adverse events and showed a 48% reduction in lesion growth after 12 months. Moreover, treated eyes experienced a statistically significant improvement in visual function, including a nearly 2-line gain in best corrected visual acuity (BCVA) compared to untreated eyes.

“The initiation of this pivotal Phase 2/3 study represents a significant milestone in our commitment to bringing transformative genetic therapies to individuals affected by Stargardt disease, a progressive and debilitating condition. The recent RPDD granted by the FDA for this program further underscores the urgent need for innovative treatment options for children living with Stargardt disease. OCU410ST, developed through our proprietary modifier gene therapy platform, is designed to target the underlying biological mechanisms of the disease,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “

The Phase 2/3 trial will enroll 51 patients with Stargardt disease, with 34 receiving a one-time subretinal injection of OCU410ST and 17 serving as controls. The primary endpoint will measure reduction in atrophic lesion size, while key secondary endpoints include improvements in BCVA and low luminance visual acuity (LLVA). With an estimated 1 million patients affected globally and over 1,200 known ABCA4 mutations, this trial marks a significant step toward addressing a complex genetic disease with high unmet need.

1Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen, said, “We have had a highly productive and collaborative engagement with the FDA’s Center for Biologics Evaluation and Research (CBER) in establishing the pivotal confirmatory trial for OCU410ST. It’s evident that there is a real sense of urgency by the agency in providing treatment options for patients who currently have nothing available to them. As we initiate the Phase 2/3 registration trial, we are expediting the clinical development of OCU410ST by two to three years and potentially providing an innovative gene therapy to patients desperate for a treatment option.”