MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has announced the completion of dosing in the Phase 2 portion of its ArMaDa clinical trial for OCU410. This novel multifunctional modifier gene therapy is being developed as a one-time treatment for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). The achievement comes ahead of schedule, marking a major milestone in Ocugen’s quest to address an area of high unmet medical need.
Geographic atrophy, which affects 2-3 million people in the U.S. and Europe, is a progressive and incurable condition that causes central vision impairment. Current treatments, including FDA-approved therapies, primarily target the complement pathway and require frequent injections, which come with significant side effects. OCU410 stands apart as a gene therapy designed to target multiple pathways associated with GA, potentially delivering long-term benefits through a single subretinal injection.
Dr. Shankar Musunuri, Ocugen’s Chairman, CEO, and Co-founder, underscored the program’s importance, stating, “The successful completion of Phase 2 dosing is a pivotal step in advancing OCU410, which we hope will become a gold-standard treatment for GA worldwide. With its unique multifunctional mechanism, OCU410 has the potential to profoundly impact millions of patients who lack effective treatment options today.”
Promising Results from Phase 1/2 Study
The ongoing ArMaDa clinical trial aims to evaluate the safety and efficacy of OCU410 in patients with GA secondary to dAMD. The Phase 1 portion demonstrated favorable safety and tolerability, with no serious adverse events such as intraocular inflammation or vasculitis. Encouragingly, treated eyes exhibited a 44% reduction in lesion growth compared to untreated eyes at nine months. Improvements in visual function, including a clinically meaningful two-line (10-letter) gain in low-luminance visual acuity, were also observed.
Notably, the ability of OCU410 to preserve retinal tissue around GA lesions compares favorably to data for leading FDA-approved complement inhibitors, which require monthly or bi-monthly injections.
According to Dr. Lejla Vajzovic, a distinguished ophthalmology expert from Duke University Eye Center involved in the trial, “A one-time gene therapy like OCU410 could transform the treatment landscape for GA, providing both structural and functional benefits while alleviating the burden of frequent injections.”
Phase 2 Expands Insights into Safety and Efficacy
During the Phase 2 portion, 51 patients were randomized into medium-dose, high-dose, and control groups, receiving a single subretinal injection of OCU410. Conducted across 14 leading retinal surgery centers in the U.S., this phase aims to build on Phase 1 findings by further assessing the therapy’s safety profile and functional outcomes.
Dr. Huma Qamar, Ocugen’s Chief Medical Officer, emphasized the therapy’s potential, stating, “OCU410’s promising safety and tolerability profile sets it apart from existing options. By reducing the need for frequent interventions, it addresses both patient compliance and quality of life for those living with this debilitating condition.”
Transforming the Future of GA Treatment
Age-related macular degeneration impacts an estimated 266 million people globally, with dAMD accounting for 85-90% of all cases. Compounded by genetic and environmental factors, GA represents the most severe stage of this disease, progressively eroding patients’ ability to see clearly.
With no approved therapies for GA in Europe and limited options in the U.S., OCU410 offers a groundbreaking approach by targeting multiple pathways in the disease’s complex etiology. Ocugen’s innovative gene therapy could expand treatment accessibility and redefine standards for managing the condition.
Next Steps and Outlook
The completion of Phase 2 dosing accelerates OCU410 toward its next milestone—a pivotal Phase 3 trial, scheduled to begin in 2026. Data from the current trial will inform the design of the next phase and support future regulatory filings, including a Biologics License Application (BLA) and Marketing Authorization Application (MAA), anticipated by 2028.
Ocugen’s work exemplifies its effort to advance breakthrough therapies for serious and underserved conditions. If successful, OCU410 could offer a life-changing alternative for millions of patients with geographic atrophy, setting new benchmarks in vision health and quality of life worldwide.
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