Ocugen gets EMA backing for single trial of Stargardt disease therapy

MALVERN, Pa. - Ocugen Inc. (NASDAQ:OCGN), a clinical-stage biopharmaceutical company with a market capitalization of $321.67 million and current trading price of $1.03, announced Wednesday that the European Medicines Agency’s Committee for Medicinal Products for Human Use has endorsed the design of its pivotal OCU410ST Phase 2/3 clinical trial for Stargardt disease, confirming that a single U.S.-based study would be sufficient for a Marketing Authorization Application in Europe. According to InvestingPro analysis, the company’s stock has shown strong momentum with a 46.58% return over the past six months, though current valuations suggest the stock is slightly overvalued.

The EMA’s decision was based on safety and efficacy data from the Phase 1 GARDian trial, which showed OCU410ST demonstrated 48% slower lesion growth and a statistically significant improvement in visual acuity compared to untreated eyes at the 12-month follow-up. While these clinical developments are promising, InvestingPro data reveals the company faces financial challenges with an EBITDA of -$52.34 million in the last twelve months, highlighting the capital-intensive nature of drug development.

The Phase 2/3 study will enroll 51 participants with Stargardt disease, with 34 receiving a one-time subretinal injection of OCU410ST in the eye with poorer vision and 17 assigned to an untreated control group. The trial includes a masked interim analysis of 24 subjects at 8 months.

"This positive opinion endorses a single trial as the basis for both BLA and MAA submissions and brings us closer to providing a one-time, modifier gene therapy to approximately 100,000 Stargardt patients in the U.S. and Europe combined," said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen, according to the press release.

The company expects to complete enrollment in the first quarter of 2026 and remains on track for a Biologics License Application filing in the first half of 2027.

OCU410ST uses an AAV delivery platform for the retinal delivery of the RORA gene, targeting pathways linked to Stargardt disease, the most common form of inherited macular degeneration that typically develops during childhood or adolescence.

Recent milestones for the OCU410ST program include Rare Pediatric Disease Designation in May, IND clearance in June, and first patient dosing in July.

In other recent news, Ocugen, Inc. reported its Q2 2025 earnings with a slight improvement over expectations. The company posted an earnings per share (EPS) of -$0.05, surpassing the forecasted -$0.06, which represents a 16.67% surprise. Revenue for the quarter reached $1.37 million. Additionally, Ocugen successfully raised $20 million through a registered direct offering with Janus Henderson Investors. This transaction involved the sale of 20 million shares of common stock, priced at $1.00 per share, along with warrants to purchase an additional 20 million shares at an exercise price of $1.50 per share. The warrants are immediately exercisable and will expire in two years. These recent developments highlight Ocugen’s ongoing financial activities and investor engagement.

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