Ocugen Reports Positive Preliminary Data from OCU410 Trial for Geographic Atrophy

Ocugen announced encouraging preliminary results from the phase 1 portion of its phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD).

The trial, which includes nine patients across three dose cohorts, revealed promising efficacy and safety outcomes for the low-dose group after 6 months. The findings highlight potential improvements over existing treatment options:

• No drug-related serious adverse events were reported, affirming the treatment's safety
• Treated eyes experienced 21.4% slower lesion growth compared to untreated eyes, outperforming published data on monthly and bi-monthly pegcetacoplan injections
• Retinal tissue preservation around GA lesions increased, suggesting structural benefits
• Functional visual outcomes showed improvement, with 100% of treated eyes stabilizing low luminance visual acuity (LLVA), a critical measure of visual function

“These results demonstrate the potential for OCU410 to address both structural and functional aspects of geographic atrophy,” said Dr. Huma Qamar, Ocugen’s Chief Medical Officer. “As a one-time treatment, it could revolutionize the management of dry AMD.”

Treatment options for GA are limited, particularly in Europe, where no approved therapies exist. In the US, the only available treatment is anti-complement therapy, requiring frequent injections and addressing only part of the disease process.

“Currently approved treatments for GA have not shown significant benefit in visual function. More importantly, we often do not realize the logistical challenge and emotional burden both patients and their caregivers must endure for every month or every other month visits,” Syed M. Shah, MD, FACS, Director of Retina Service, Vice Chair for Research & Digital Health at Emplify Health – La Crosse, Wisconsin, said in a company news release. “Based on the science and preliminary data, OCU410 has the potential to improve structural as well as functional outcomes. This ‘one-and-done’ treatment paradigm can be a gamechanger for how we treat patients with GA.”  

Ocugen also reported progress in OCU410ST GARDian trial for Stargardt disease; and OCU400 trial targeting Leber congenital amaurosis (LCA).