Ocugen has announced the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) amendment to begin a phase 2/3 pivotal confirmatory trial of OCU410ST.
OCU410ST uses an adeno-associated virus delivery platform for the retinal delivery of the RORA gene. RORA regulates pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.1-2
Previously, the FDA granted a rare pediatric disease designation (RPDD) and orphan drug designations to OCU410ST for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.2
Shankar Musunuri, PhD, MBA, chairman, CEO, and cofounder of Ocugen, commented on the FDA decision in a press release from the company.1
“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,” said Musunuri. “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 2-3 years and potentially providing an innovative gene therapy to patients desperate for a treatment option.”
Positive data from the phase 1 GARDian trial for OCU410ST
- 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 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 the 51, 34 will receive a 1-time subretinal injection of OCU410ST (200 μL at a concentration of 1.5 × 10¹¹ vector genomes/mL) in the eye with poorer visual acuity. The remaining 17 will be assigned to an untreated control group. The company states the primary objective as the reduction in atrophic lesion size. Key secondary endpoints include improvements in best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA).
Lejla Vajzovic, MD, FASRS, director of the Duke Surgical Vitreoretinal Fellowship Program stated, “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.”
The company stated it plans on submitting a Biologics License Application (BLA) for OCU410ST in 2027 with data from the 1-year follow-up of the phase 2/3 trial to be used as support.
Recently, Ocugen announced the signing of a binding term sheet to negotiate and enter into a licensing agreement with a “well-established leader in the pharmaceutical and healthcare sector in Korea” for exclusive Korean rights to OCU400.
References:
Ocugen, Inc. announces US FDA clearance of Investigational New Drug Amendment to initiate phase 2/3 pivotal confirmatory clinical trial of OCU410ST—modifier gene therapy candidate for stargardt disease. Published June 16, 2025. Accessed June 16, 2025. https://ir.ocugen.com/news-releases/news-release-details/ocugen-inc-announces-us-fda-clearance-investigational-new-drug
