Ocugen Inc. has announced positive preliminary efficacy and safety data from the Phase 1 dose-escalation segment of its Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). The data suggest that OCU410, a novel modifier gene therapy, could offer a significant advancement in treating this progressive retinal disease.
Promising Phase 1 Results
The Phase 1 trial enrolled nine patients across three dose cohorts (low, medium, and high). Key findings from the low-dose cohort (three patients) at six months include:
- A 21.4% slower lesion growth rate in treated eyes compared to untreated fellow eyes, following the natural disease progression.
- Increased preservation of retinal tissue surrounding GA lesions in treated eyes over six months.
- Stabilization of visual function in all treated eyes, as measured by low luminance visual acuity (LLVA), suggesting treatment benefit.
Notably, no drug-related serious adverse events were reported during the trial.
Addressing Unmet Needs in GA Treatment
Geographic atrophy affects an estimated 2-3 million people in the United States and Europe. Current treatment options in the U.S. are limited to anti-complement therapies requiring frequent injections, which address only one aspect of the disease. There is currently no approved treatment for GA in Europe.
Syed M. Shah, MD, FACS, director of Retina Service and Vice Chair for Research & Digital Health at Emplify Health, noted that currently approved treatments for GA have not shown significant improvements in visual function and that OCU410 has the potential to improve both structural and functional outcomes with its “one-and-done” approach.
OCU410: A Multifunctional Approach
OCU410 utilizes an adeno-associated virus (AAV) platform to deliver the RORA (ROR Related Orphan Receptor A) gene. The RORA protein plays a crucial role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and exhibits anti-inflammatory properties while inhibiting the complement system. This multifunctional approach targets multiple pathways associated with dAMD pathophysiology.
Huma Qamar, MD, chief medical officer at Ocugen, stated that OCU410 targets multiple disease pathways beyond complement inhibition, highlighting the promise of their modifier gene therapy platform as a one-time treatment for dAMD.
Phase 2 Trial and Future Plans
Ocugen has completed dosing in the Phase 2 portion of the ArMaDa clinical trial, randomizing 51 subjects into treatment and control arms. Preliminary data from the Phase 1/2 study demonstrated a favorable safety and tolerability profile with no serious adverse events related to OCU410. Subjects in the Phase 1 study showed a considerably slower lesion growth (44%) from baseline in treated eyes versus untreated fellow eyes at 9 months follow-up. A clinically meaningful 2-line (10-letter) improvement in visual function (LLVA) in treated eyes compared to untreated eyes was also noted in the Phase 1 portion of the trial.
Ocugen plans to use the data from this trial to design a future pivotal Phase 3 study planned for 2026 and enable their commercial strategy for Biologics License Application (BLA) and Marketing Authorization Application (MAA) filings as soon as 2028.
