MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has announced a positive 2-year safety and efficacy update from its Phase 1/2 clinical trial for OCU400, a groundbreaking gene therapy targeting retinitis pigmentosa (RP). The data highlights sustained improvements in visual function and positions OCU400 as a potential one-time therapy for a condition that affects approximately 2 million people worldwide, including 300,000 in the U.S. and Europe. Without intervention, many patients with RP face the risk of legal blindness.
Results from the trial demonstrate that 100% of evaluable treated participants (9 out of 9) showed improvement or preservation in visual function compared to untreated eyes over two years. Additionally, all treated subjects exhibited improvement or stabilization in mobility testing, conducted up to one year. Notably, treated eyes achieved a statistically significant two-line gain (10 letters) in low-luminance visual acuity (LLVA) on the ETDRS chart, regardless of genetic mutation, with a p-value of 0.01.
“These findings represent a significant step forward in our mission to bring life-changing therapies to patients with inherited retinal disorders and confirm the transformative potential of our modifier gene therapy platform,” said Shankar Musunuri, PhD, MBA, Chairman, CEO, and Co-Founder of Ocugen.
RP presents a significant clinical challenge due to the wide variety of genetic mutations involved. However, OCU400’s mutation-agnostic mechanism offers renewed hope for patients.
“The broad spectrum of genes and mutations causing RP presents a unique challenge in developing treatments for this unmet need. This is where the promise of mutation-agnostic therapies becomes particularly compelling,” said Dr. Syed M. Shah, Vice Chair for Research and Digital Medicine, Gundersen Health System. “OCU400’s demonstrated effectiveness across multiple mutations not only offers hope to RP patients but also opens new possibilities for treating other retinal diseases.”
The trial results also underscore OCU400’s safety profile, with no serious adverse events related to the therapy.
“Establishing the long-term safety and efficacy of OCU400 demonstrates the durability of this novel gene therapy,” added Huma Qamar, MD, MPH, Chief Medical Officer at Ocugen. “These 2-year LLVA findings, which are the most sensitive measure of visual function, are consistent with the results observed at one year.”
Encouraged by the results, Ocugen is advancing the Phase 3 liMeliGhT clinical trial for OCU400, with a Biologics License Application (BLA) submission expected in the first half of 2026. If proven successful, OCU400 could become a game-changing treatment for millions suffering from progressive vision loss.
For the latest news on everything happening in Chester County and the surrounding area, be sure to follow MyChesCo on Google News and MSN.