MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) announced the first patient has been dosed in its Phase 3 liMeliGhT clinical trial for OCU400, a gene therapy candidate for retinitis pigmentosa (RP). This marks a significant milestone in the quest to offer a one-time treatment for this progressive eye disease.
“Each clinical milestone brings us closer to providing a one-time treatment for patients living with RP,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. With 300,000 RP patients in the U.S. and Europe, and 1.6 million worldwide, this development could mean a substantial improvement in quality of life for many.
The Phase 3 trial is based on promising results from earlier studies. Phase 1/2 data showed positive trends in visual acuity and mobility tests. Specifically, 89% of subjects showed preservation or improvement in visual function. These results suggest that OCU400 could be effective across various genetic mutations associated with RP.
The current study will run for one year and include 150 participants. One arm will focus on patients with RHO gene mutations, while the other will be gene-agnostic. Participants are randomized 2:1 to receive OCU400 or no treatment. Researchers aim to measure improvements using the Luminance Dependent Navigation Assessment (LDNA), a sensitive test of visual function.
“Patients with RP associated with multiple genes currently have no therapeutic options,” said Dr. Lejla Vajzovic from Duke University Eye Center. The potential for OCU400 to provide long-term benefits could shift the treatment paradigm for RP.
Dr. Benjamin Bakall from Associated Retina Consultants also expressed optimism. “The current Phase 3 study gives hope to thousands of individuals with RP.”
Ocugen’s OCU400 has received orphan drug and RMAT designations from the FDA. The European Medicines Agency (EMA) has also accepted the U.S.-based trial for Marketing Authorization Application (MAA) submission. With the first patient dosed, Ocugen remains on track for 2026 approval targets.
The implications of this trial are significant. If successful, OCU400 could transform the lives of RP patients by offering a long-lasting solution to a presently incurable condition. This advancement underscores the critical need for innovative therapies in ophthalmology and highlights the potential for gene therapy to address complex genetic disorders.
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