Board Approves Cohort 3 Enrollment and Phase 2 Start in OCU410 Study for Geographic Atrophy

Ocugen, Inc., a biotechnology company specializing in gene and cell therapies and vaccines, has announced a favorable review from the Data and Safety Monitoring Board (DSMB) for its Phase 1/2 ArMaDa clinical trial involving OCU410, a new gene therapy candidate for treating geographic atrophy (GA). GA is an advanced form of dry age-related macular degeneration (dAMD), affecting approximately 2-3 million individuals in the U.S. and Europe.

In the ongoing clinical trial, six GA patients have received OCU410, with three patients administered a low dose and three a medium dose. The next phase will include an additional three patients who will receive a high dose of OCU410.

Dr. Peter Chang, Chair of the DSMB for the OCU410 clinical trial, stated, “The DSMB recommends proceeding with the high-dose cohort and simultaneously initiating Phase 2 dosing. No serious adverse events (SAEs) have been reported in both low- and medium-dose groups. This is a key milestone in determining the maximum tolerated dose for OCU410.”

Huma Qamar, Chief Medical Officer of Ocugen, expressed optimism about the second positive DSMB recommendation. She commented, “We are excited to see OCU410 showing a favorable safety and tolerability profile. We believe OCU410 has the potential to be a one-time treatment for GA, offering a single sub-retinal injection. Current treatments for GA require 6-12 intravitreal injections annually, targeting only the complement pathway. OCU410, however, addresses multiple pathways, including complement, lipid metabolism, inflammation, and oxidative stress, promising long-term benefits for patients.”

The ArMaDa clinical trial aims to evaluate the safety and efficacy of OCU410 administered unilaterally under the retina in GA patients. The trial is structured into two phases. Phase 1 is a multicenter, open-label, dose-ranging study with three dose levels: low (2.5×10^10 vg/mL), medium (5×10^10 vg/mL), and high (1.5×10^11 vg/mL). Phase 2 will be a randomized, outcome assessor-blinded, dose-expansion study, where subjects will be allocated in a 1:1:1 ratio to one of two OCU410 treatment groups or an untreated control group.

Geographic atrophy is a condition where the macula, the central part of the retina responsible for clear vision, progressively thins. Affecting about 10 million Americans and more than 266 million people globally, GA is characterized by retinal deterioration, submacular drusen, atrophy, and central vision impairment. dAMD, comprising 85-90% of the total AMD population, involves slow retinal degeneration, leading to significant vision loss over time.

OCU410 employs an AAV delivery system to administer the RORA gene to the retina. The RORA protein is vital in lipid metabolism, reducing oxidative stress and lipofuscin deposits, and has demonstrated anti-inflammatory properties and complement system inhibition in preclinical studies. This multifaceted approach allows OCU410 to target various pathways involved in dAMD pathophysiology. Ocugen is advancing the development of this gene therapy to offer a one-time treatment for GA.

Ocugen, Inc. is committed to discovering and developing novel gene and cell therapies and vaccines. The company's efforts aim to improve global health and provide patients with new treatment options. Their innovative gene therapy platform shows promise in treating multiple retinal diseases with a single product. Additionally, Ocugen is engaged in infectious disease research to enhance public health and address unmet needs in orthopedic diseases.

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