Ocugen announces positive preliminary data from OCU410 Phase 1 clinical trial for GA

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Ocugen Inc. announced positive preliminary efficacy and safety data from the Phase 1 dose-escalation segment of the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD).

Key findings include:

• No drug-related serious adverse events
• Reduced lesion growth
• Preservation of retinal tissue
• Improvement in functional vision as measured by low luminance visual acuity (LLVA)

An estimated 3 million individuals in the United States and Europe are affected by GA. In the US, treatment options are limited to anti-complement therapies requiring multiple injections, which address only one aspect of the disease. There is currently no approved treatment for GA in Europe.¹

Syed M. Shah, MD, FACS, director of Retina Service and Vice Chair for Research & Digital Health at Emplify Health, La Crosse, Wisconsin, opined that currently approved treatments for GA have not shown significant improvements in visual function.

“Moreover, the logistical and emotional burdens of monthly or bi-monthly treatments can be substantial for both patients and caregivers,” Shah said. “Based on the science and early data, OCU410 has the potential to improve both structural and functional outcomes. Its ‘one-and-done’ approach could redefine how we treat GA.”

The Phase 1 trial enrolled 9 patients across 3 dose cohorts (low, medium, and high). At 6 months, data from the low-dose cohort (three patients) demonstrated:

• A 21.4% slower lesion growth rate in treated eyes compared to untreated fellow eyes, following the natural disease progression. This result favorably compares to published data on pegcetacoplan administered monthly or every other month.
• Increased preservation of retinal tissue surrounding GA lesions in treated eyes over 6 months, again outperforming pegcetacoplan data.
• Stabilization of visual function in all treated eyes, as measured by LLVA, suggesting treatment benefit.

“OCU410 targets multiple disease pathways beyond complement inhibition,” said Huma Qamar, MD, chief medical officer at Ocugen. “These preliminary data highlight the promise of our modifier gene therapy platform as a one-time treatment for dAMD. The observed safety and efficacy profiles, coupled with meaningful patient outcomes, are encouraging.”

In July 2024, Majda Hadziahmetovic, MD, an associate professor of Ophthalmology, Vitreoretinal Diseases and Surgery at Duke University Eye Center, and the lead investigator for the OCU410 Phase 1/2 trial, pointed out in a news release there currently are 2 FDA approved anti-complement therapies for GA, which targets only one pathway for a disease that has a multifactorial and complex etiology.²

“Though these treatments are a significant milestone, they provide limited benefits and involve a continuous regimen of multiple intravitreal injections over several years. This modifier gene therapy could potentially transform the landscape of GA treatment,” Hadziahmetovic said.

Ocugen also reported promising data from the Phase 1/2 OCU410ST GARDian trial for Stargardt disease and the Phase 1/2 OCU400 trial for Leber congenital amaurosis (LCA). These findings, alongside insights from study investigators and patient testimonials, were presented at the Company’s recent Clinical Showcase. The data affirm the potential of modifier gene therapy to address both rare inherited retinal disorders and common causes of blindness affecting millions.

References:

1. Ocugen. Ocugen Announces Compelling Preliminary Data for OCU410—a Single Dose Novel Modifier Gene Therapy to Treat Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration. GlobeNewswire News Room. Published November 19, 2024. Accessed November 19, 2024. https://www.globenewswire.com/news-release/2024/11/19/2983456/0/en/Ocugen-Announces-Compelling-Preliminary-Data-for-OCU410-a-Single-Dose-Novel-Modifier-Gene-Therapy-to-Treat-Geographic-Atrophy-Secondary-to-Dry-Age-Related-Macular-Degeneration.html

2. Ocugen. Ocugen, Inc. Announces Completion of Dosing in Subjects with Geographic Atrophy Secondary to dAMD in High-Dose Cohort of Phase 1/2 ArMaDa Clinical Trial of OCU410—A Novel Modifier Gene Therapy. GlobeNewswire News Room. Published July 25, 2024. Accessed July 25, 2024. https://www.globenewswire.com/news-release/2024/07/25/2918663/0/en/Ocugen-Inc-Announces-Completion-of-Dosing-in-Subjects-with-Geographic-Atrophy-Secondary-to-dAMD-in-High-Dose-Cohort-of-Phase-1-2-ArMaDa-Clinical-Trial-of-OCU410-A-Novel-Modifier-Ge.html