Ocugen Posts Positive Preliminary Phase 2 ArMaDa Trial Data of OCU410

This article originally appeared on our sister site, Ophthalmology Times.

Ocugen has reported encouraging preliminary 12‑month findings from its phase 2 ArMaDa clinical trial evaluating OCU410 (AAV5‑RORA) as a potential treatment for geographic atrophy (GA) associated with dry age-related macular degeneration (dAMD).

OCU410 is an investigational gene therapy administered via intravitreal injection. It utilizes an adeno-associated virus serotype 5 (AAV5) vector to deliver retinoid-related orphan receptor alpha (RORA), a nuclear receptor involved in regulating multiple biological pathways essential to retinal health, including oxidative stress, complement activity, inflammation, and lipid metabolism.

"The clinical development journey of OCU410 has been remarkable," Huma Qamar, MD, MPH, CMI, the chief medical officer of Ocugen, said in a statement.¹ "Our phase 2 randomized trial delivered robust anatomic efficacy that was statistically significant across multiple analyses. Critically, our safety data across 60 patients has shown no drug-related serious adverse events, no inflammation signals, and no injection complications to date, supporting a favorable risk-benefit profile."

The randomized phase 2 ArMaDa study enrolled 51 patients, who were assigned in a 1:1:1 ratio to receive either a high-dose treatment, a medium-dose treatment, or no treatment. Participants received a single 200‑µL subretinal administration of OCU410 at either 5 × 10¹⁰ vector genomes (vg)/mL for the medium-dose group or 1.5 × 10¹¹ vg/mL for the high-dose group, while patients in the control arm did not receive treatment.

At the time of this analysis, approximately half of enrolled participants had reached the 12‑month evaluation point. Among these patients, lesion growth was reduced by 46% at 12 months in the combined medium- and high-dose groups compared with the untreated control group (P = 0.015; n = 23). When analyzed separately, patients receiving the medium dose experienced a 54% reduction in lesion growth versus control (P = 0.02; n = 10), while those in the high-dose cohort demonstrated a 36% reduction (P = 0.05; n = 8). In addition, a subgroup of patients with baseline lesion areas of at least 7.5 mm² (n = 14) showed a 57% greater reduction in lesion growth compared with controls.

"As a practicing retinal specialist, OCU410's clinical profile is genuinely exciting for geographic atrophy patients—including a reduction in ellipsoid zone loss observed in Phase 1, which may serve as a potential marker of retinal health, and a reduction in lesion growth seen in phase 2.”

"With these promising results, I believe OCU410 has the potential to set a new standard of care with a single treatment for life,” Vajzovic, who is also the retina scientific advisory board chair for Ocugen, said.

Ocugen also shared additional data from its phase 1 study, which indicated that among assessable patients (n = 7), loss of the ellipsoid zone progressed 60% more slowly in OCU410-treated eyes compared with untreated fellow eyes at 12 months. Reductions in ellipsoid zone–retinal pigment epithelium (EZ‑RPE) complex loss were also observed, suggesting preservation of photoreceptors and retinal pigment epithelium in treated eyes.

The company stated that it remains on schedule to submit a biologics license application for OCU410 in 2028. Full results from the phase 2 ArMaDa trial are expected in the first quarter of 2026, with initiation of a phase 3 study planned for later that year.

Separately, Ocugen recently announced the publication of positive phase 1 results from its GARDian1 trial evaluating OCU410ST, a gene therapy candidate for Stargardt disease.

REFERENCE

1. Ocugen Announces Positive Preliminary Phase 2 Data from OCU410 Modifier Gene Therapy for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration. Published January 15, 2026. Accessed January 15, 2026. https://www.globenewswire.com/news-release/2026/01/15/3219537/0/en/Ocugen-Announces-Positive-Preliminary-Phase-2-Data-from-OCU410-Modifier-Gene-Therapy-for-Geographic-Atrophy-Secondary-to-Dry-Age-Related-Macular-Degeneration.html