Key Takeaways

• OCU410 gene therapy showed significant lesion growth reduction in phase 2 trial for geographic atrophy secondary to dry AMD.
• The trial demonstrated a 46% lesion growth reduction at 12 months, with medium and high doses showing 54% and 36% reductions, respectively.
• Phase 1 data indicated a 60% slower ellipsoid zone loss in treated eyes, suggesting photoreceptor and RPE preservation.
• No drug-related serious adverse events or injection complications were reported, supporting a favorable risk-benefit profile.
• Ocugen aims to submit a biologics license application for OCU410 in 2028, with full phase 2 data expected in the first quarter of 2026.

The trial is evaluating OCU410 (AAV5-RORA) for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration.

Ocugen has announced positive preliminary 12-month data from its phase 2 ArMaDa trial evaluating OCU410 (AAV5-RORA) for the treatment of geographic atrophy secondary to dry age-related macular degeneration.

OCU410 is an investigational, intravitreally administered, AAV5-based gene therapy that delivers RORA, a nuclear receptor that regulates key pathways involved in retinal homeostasis, including oxidative stress response, complement regulation, inflammation, and lipid metabolism.¹

The phase 2 ArMaDa trial enrolled 51 patients who were randomly assigned 1:1:1 to a high-dose, medium-dose, or control group. Patients received a single subretinal 200-µL administration of 5 x 10¹⁰ vector genomes (vg)/mL (medium dose) or 1.5 x 10¹¹ vg/mL (high dose), while the control group remained untreated.

Approximately 50% of patients in the phase 2 trial have been evaluated in the 12-month data to date. According to the results, a 46% lesion growth reduction was seen at 12 months (medium + high dose vs control; P = .015; n=23). Those treated with a medium dose (P = .02; n=10) achieved 54% lesion reduction compared with the control group, while those in the higher dose (P = .05; n=8) achieved 36% lesion reduction compared with the control group. Additionally, a subgroup of patients with ≥7.5 mm² at baseline (n=14) showed a 57% greater reduction in lesion size when compared with the control group.

Additional findings from the phase 1 trial were also released, showing that in assessable patients (n=7) ellipsoid zone (EZ) loss was 60% slower in OCU410-treated eyes when compared with untreated fellow eyes at 12 months. Additionally, EZ-retinal pigment epithelium (RPE) complex loss was reduced in treated eyes vs fellow eyes, which demonstrates photoreceptor and RPE preservation, according to the company.

Shankar Musunuri, PhD, chairman, CEO, and co-founder of Ocugen, and Huma Qamar, MD, MPH, CMI, chief medical officer at Ocugen, both commented on the results in a news release.

“Delivering 60% slower EZ loss in phase 1 and 46% lesion growth reduction in the phase 2 preliminary analysis demonstrates the capability of our multi-pathway RORA approach,” said Musunuri.

“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,” added Qamar.

The company noted it is on track to submit a biologics license application filing for OCU410 in 2028 and plans to release full data from the phase 2 ArMaDa trial in the first quarter of 2026, while initiating phase 3 sometime the same year.

Ocugen also recently announced the publication of positive results from its phase 1 GARDian1 trial of OCU410ST, a gene therapy 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. News release. Ocugen. 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