Ocugen’s OCU410ST gene therapy gets another green light. Next stop: a Phase II/III trial.
The U.S. Food and Drug Administration (FDA) has cleared Ocugen, Inc.’s (Pennsylvania, United States) Investigational New Drug (IND) amendment to initiate a Phase II/III pivotal confirmatory clinical trial of OCU410ST, a modifier gene therapy candidate for Stargardt disease.
Ocugen announces the news on its LinkedIn page.
The therapy is being developed to treat all ABCA4-associated retinopathies, including Stargardt disease, a genetic eye disorder that causes retinal degeneration and vision loss primarily affecting children and young adults. The FDA had previously granted Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation for OCU410ST.
READ MORE: FDA Grants Rare Pediatric Disease Designation to Ocugen’s Gene Therapy for Stargardt Disease
“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 Dr. Shankar Musunuri, chairman, CEO and co-founder of Ocugen, in a news release.
“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.”
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Trial design and early data
The Phase II/III clinical trial will enroll 51 participants diagnosed with Stargardt disease. Of these, 34 will receive a one-time subretinal injection of OCU410ST in the eye with poorer visual acuity, while 17 will serve as untreated controls. The primary objective is to evaluate reduction in atrophic lesion size, with secondary endpoints including improvements in best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA).
Positive data from the earlier Phase I GARDian trial demonstrated a favorable safety profile with no serious adverse events related to the therapy. The study also showed 48% slower lesion growth at 12-month follow-up in treated eyes compared to untreated eyes, and a statistically significant improvement in visual function (2-line BCVA gain).
“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,” said Dr. Huma Qamar, Ocugen’s chief medical officer.
Dr. Huma Qamar discusses the promise OCU410ST holds for Stargardt patients.
About Stargardt disease
According to the company, Stargardt disease affects approximately 100,000 patients in the U.S. and Europe combined, and one million patients globally. The condition involves more than 1,200 known mutations, making it challenging to address with traditional gene therapy approaches.
READ MORE: FDA Grants Breakthrough Therapy Designation to Belite Bio’s Tinlarebant for Stargardt Disease
Dr. Lejla Vajzovic, professor of Ophthalmology, Pediatrics and Biomedical Engineering at Duke University Eye Center, noted, “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.”
Ocugen plans to submit a Biologics License Application (BLA) for OCU410ST in 2027.
Editor’s Note: This content is intended exclusively for healthcare professionals. It is not intended for the general public. Products or therapies discussed may not be registered or approved in all jurisdictions, including Singapore.