Ocugen Begins Phase 3 Trial for OCU400 in Retinitis Pigmentosa

Ocugen, Inc., a biotechnology company headquartered in Malvern, Pennsylvania, has announced a significant milestone in its ongoing efforts to combat retinitis pigmentosa (RP). On June 20, 2024, the company revealed that the first patient has been dosed in the Phase 3 liMeliGhT clinical trial for their gene therapy product, OCU400.

Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen, expressed enthusiasm about this development, highlighting that each milestone achieved brings the company closer to offering a one-time treatment for RP. He emphasized the importance of this first patient dosing, noting the potential impact on the 300,000 RP patients in the U.S. and Europe, and the 1.6 million affected worldwide.

The Phase 3 liMeliGhT trial is built on the promising results from the Phase 1/2 OCU400 studies, which indicated positive trends in Best-Corrected Visual Acuity (BCVA), Multi-Luminance Mobility Testing (MLMT), and Low-Luminance Visual Acuity (LLVA) among treated eyes. Specifically, 89% of RP subjects showed either preservation or improvement in treated eyes across these metrics. Additionally, 80% of subjects with RHO mutations saw preservation or improvement in MLMT scores, and 78% of the overall subjects demonstrated similar benefits in MLMT scores.

The Phase 3 study will span one year and include 150 participants. This group will be divided into two arms: one consisting of 75 participants with RHO gene mutations and the other with 75 gene-agnostic participants. Each arm will have participants randomized in a 2:1 ratio to receive either the treatment (2.5 x 10^10 vg/eye of OCU400) or be part of an untreated control group. The study will include patients aged eight and older with varying stages of RP.

The primary endpoint of the study is the Luminance Dependent Navigation Assessment (LDNA), a more sensitive measurement than the MLMT used in previous trials. The focus will be on the proportion of responders in both the treated and untreated groups, specifically those achieving an improvement of at least 2 Lux levels from baseline in the study eyes.

Dr. Lejla Vajzovic, a retinal surgeon and Director of the Duke Surgical Vitreoretinal Fellowship Program, expressed optimism about OCU400's potential to benefit RP patients. She highlighted the innovative nature of this gene therapy, which aims to reset cellular gene networks and restore balance within the retina.

Dr. Benjamin Bakall, Director of Clinical Research at Associated Retina Consultants (ARC) and Clinical Assistant Professor at the University of Arizona, expressed excitement about the ongoing study, noting the potential for a treatment option that could preserve or improve vision in RP patients, regardless of their specific gene mutations. He also acknowledged the significance of the first patient dosing conducted at ARC.

Dr. Huma Qamar, Chief Medical Officer of Ocugen, expressed gratitude for the continued collaboration with Dr. Bakall and ARC. She emphasized the importance of expanding enrollment to include a diverse range of RP gene mutations, which will further validate this novel gene therapy platform.

OCU400 has already received orphan drug designation and RMAT designations from the FDA, and the EMA has accepted the U.S.-based trial for a future Marketing Authorization Application (MAA). Ocugen aims to meet its BLA and MAA approval targets by 2026.

OCU400 is based on the nuclear hormone receptor (NHR) gene, NR2E3, which regulates several critical functions within the retina. By reestablishing homeostasis within affected cells, OCU400 aims to improve retinal health and function in RP patients. This gene-agnostic approach sets it apart from single-gene replacement therapies, offering potential benefits across multiple retinal diseases.

Ocugen is also conducting trials for other treatments, including OCU410 for geographic atrophy secondary to dry age-related macular degeneration and OCU410ST for Stargardt disease. These trials reflect Ocugen's commitment to addressing unmet medical needs in retinal diseases.