FDA Approves Ocugen's OCU400 for Phase 3 Retinitis Pigmentosa Trial

Ocugen, Inc., a biotechnology firm engaged in the development of innovative gene and cell therapies and vaccines, has received approval from the U.S. Food and Drug Administration (FDA) to commence a Phase 3 clinical trial for OCU400, a gene therapy candidate designed for treating retinitis pigmentosa (RP). The trial marks a significant advancement for individuals suffering from RP and represents a crucial step for Ocugen.

Dr. Shankar Musunuri, the Chairman, CEO, and Co-Founder of Ocugen, emphasized the importance of this trial, highlighting that OCU400 is the first gene therapy to reach Phase 3 with a broad indication for RP. Previously, only one product was available in the market to address a single gene mutation out of the 100 associated with RP, leaving a significant patient population without treatment options.

The Phase 3 study is set to include 150 participants, divided into two groups: one with the RHO gene mutation and the other without genetic specificity. The trial will allocate participants to a treatment group, receiving OCU400, and a control group, in a 2:1 ratio.

In the prior Phase 1/2 clinical trial, the Multi-Luminance Mobility Testing (MLMT) scale was used as the primary endpoint. For the upcoming Phase 3 trial, an enhanced mobility course, the Luminance Dependent Navigation Assessment (LDNA), will be implemented. This new assessment covers a broader range of light intensity and levels, providing a more comprehensive evaluation.

Dr. Arun Upadhyay, Ocugen's Chief Scientific Officer, explained that the LDNA was developed in collaboration with the FDA to facilitate the inclusion of patients at various stages of the disease. The company is optimistic that the Phase 3 study's design will enable more than half of the RHO patients to meet the responder criteria, which is defined as a two or more Lux level improvement after one year of treatment.

RP affects approximately 110,000 people in the United States and 1.6 million globally, with over 10% of these individuals having the RHO genetic mutation. Dr. Huma Qamar, Ocugen's Chief Medical Officer, stated that the gene-agnostic design of the clinical trial aims to provide a therapeutic option for a broader range of patients who could benefit from the treatment.

Ocugen has announced that OCU400 has received orphan drug and RMAT designations from the FDA. The company is targeting a 2026 approval for the Biologics License Application (BLA) for OCU400.

OCU400 is based on the NHR gene, NR2E3, which plays a role in various physiological functions within the retina, including photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival networks. The therapy aims to reset altered cellular gene-networks and establish a state of balance, potentially improving retinal health and function in patients with inherited retinal diseases.

RP is a group of rare genetic disorders characterized by the degeneration of cells in the retina, leading to vision loss and potentially blindness. There are currently no approved treatments that can slow or halt the progression of multiple forms of RP. While gene-replacement therapies show promise, they are limited to addressing a single mutation and do not eliminate the underlying genetic defect, which can still lead to retinal degeneration.

Ocugen is dedicated to discovering, developing, and commercializing novel therapies that can improve health and offer hope to patients worldwide. The company's breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product and is also advancing research in infectious and orthopedic diseases to address unmet medical needs.