Ocugen receives unanimous FDA approval on key aspects of pivotal Phase 3 study design for OCU400 modified gene therapy

MALVERN, Pa., Dec. 21, 2023 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN) is a biotechnology company focused on the discovery, development and commercialization of novel gene and cell therapies and vaccines Company, announced today that it has received FDA coordination on key aspects of the Phase 3 clinical trial design to evaluate the safety and efficacy of OCU400 in patients with: right chamber and other genetic mutations associated with retinitis pigmentosa (RP).

“This news brings us one step closer to realizing our mission to bring best-in-class, gene-agnostic therapies to market and serve patients around the world,” said Shankar Musunuri, Ph.D., chairman, CEO and co-founder of Ocugen. . We look forward to commencing Phase 3 clinical trials, scheduled to begin in early 2024.

In a multidisciplinary meeting with the FDA, based on preliminary results from the ongoing Phase 1/2 study, Ocugen coordinated key aspects of the Phase 3 study design, including study endpoints, patient enrollment strategy and one-year study duration. time. Phase 3 clinical trial will recruit wider range of RP patients, including those with most common diseases right chamber Gene mutations, based on the underlying gene-agnostic mechanism of action of OCU400.

With OCU400 receiving Orphan Drug and RMAT designations, the FDA has agreed on key aspects of the Phase 3 study design, allowing Ocugen to confidently advance product development and licensing of OCU400.

Currently, there are approximately 110,000 RP patients in the United States and approximately 1.6 million patients worldwide.More than 10% of these patients have right chamber Gene mutation. Advancing OCU400 into Phase 3 clinical development will be an important step in addressing the unmet needs of the RP patient population.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing and commercializing novel gene and cell therapies and vaccines to improve health and bring hope to patients around the world. Through bold innovation, we leverage our unique intellectual and human capital to pioneer new scientific pathways and impact patient lives. Our breakthrough modified gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing infectious disease research to support public health and orthopedic diseases to address unmet medical needs. For more information, visit www.ocugen.com and follow us on X and LinkedIn.

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touch:
Tiffany Hamilton
communications director
IR@ocugen.com