Wednesday, December 20, 2023
Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
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OCU400 Received RMAT Designation. The FDA has designated OCU400 as a Regenerative Medicine Advanced Therapy (RMAT) for treating retinitis pigmentosa associated with RHO mutations. RMAT designation is intended to speed development of regenerative medicines, and is awarded to therapies that have potential treat, reverse, or modify a life-threatening disease. We see this as a validation of Ocugen’s clinical trial data. Additional data submissions to expand the designation to other gene mutations in RP and Leber congenital amaurosis (LCA) are planned.
Phase 3 Trial Should Benefit From RMAT Designation. RMAT designation includes the benefits of Fast Track and Breakthrough designations, with increased guidance from the FDA. It also allows the use of surrogate markers as endpoints in clinical trials, and accelerated review after the application is filed. Ocugen is currently finalizing the design of the Phase 3 trial for OCU400. We expect the trial to begin in early 2024 with data available in 2H24.
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