Ocugen, Inc, a biotechnology company specializing in gene and cell therapies, and vaccines, has announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has reviewed and accepted the study design, endpoints, and planned statistical analysis of the Phase 3 clinical trial for OCU400, named liMeliGhT, targeting retinitis pigmentosa (RP). The EMA has deemed the U.S.-based trial suitable for submission of a Marketing Authorization Application (MAA).

The decision from EMA was influenced by the safety and tolerability demonstrated in the Phase 1/2 study of OCU400. The Phase 3 liMeliGhT study is expected to enroll 150 participants mainly from the U.S., split into two arms: one with 75 participants having RHO gene mutations and the other with 75 participants representing various gene mutations associated with RP. In each arm, participants will be randomized in a 2:1 ratio to either the treatment group (receiving 2.5 x 1010 vg/eye of OCU400) or the untreated control group.

This positive feedback from EMA aligns with the U.S. FDA's clearance of the IND amendment to initiate the Phase 3 liMeliGhT clinical trial for OCU400. Notably, OCU400 is the first gene therapy to advance to Phase 3 with such a broad RP indication. It had previously been granted broad Orphan Drug Designation for RP and Leber congenital amaurosis in the EU.

Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen, expressed gratitude towards EMA for their collaborative efforts and support in offering a gene-agnostic therapeutic solution to RP patients facing severe unmet medical needs. He emphasized that this positive opinion marks a crucial advancement in making their transformative modifier gene therapies available to patients worldwide.

EMA's opinion represents a significant milestone, potentially streamlining the process and reducing costs associated with obtaining marketing authorization in the EU. With this development, OCU400 remains on track for its 2026 BLA and MAA approval targets.

OCU400 is Ocugen's gene-agnostic modifier gene therapy product centered on the NHR gene, NR2E3. NR2E3 plays a pivotal role in regulating various physiological functions within the retina, including photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival networks. By resetting altered cellular gene-networks and establishing homeostasis, OCU400 holds promise in enhancing retinal health and function for patients with inherited retinal diseases.

RP encompasses a group of rare, genetic disorders characterized by the deterioration and loss of cells in the retina, ultimately leading to vision impairment and blindness. Currently, there are no approved treatments capable of slowing or halting the progression of multiple forms of RP. While gene-replacement therapies show promise, they are typically limited to addressing a single mutation. The development of gene-specific replacement therapy is particularly challenging when multiple and unknown genes are involved. Therefore, novel therapeutic approaches targeting broader RP disease in a gene-agnostic manner offer a beacon of hope for patients.

 

Source: globenewswire.com

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