Ocugen releases positive preliminary 12-month data from phase 2 ArMaDa trial

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

OCU410 is an investigational, intravitreally administered, AAV5-based gene therapy that delivers RORA (retinoid-related orphan receptor alpha), 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 that were randomized 1:1:1 into either 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=0.015; n=23). Those treated with a medium dose (p=0.02; n=10) achieved 54% lesion reduction compared to control, while those in the higher dose (p=0.05; n=8) achieved 36% lesion reduction compared to control. Additionally, a subgroup of patients with ≥7.5 mm² at baseline (n=14) showed a 57% greater reduction in lesion size when compared to the control.

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 to untreated fellow eyes at 12 months. Additionally, EZ-RPE complex loss was reduced in treated eyes versus 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 of Ocugen, both commented on the results in a press release from the company.

"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 (BLA) filing for OCU410 in 2028 and plans to release full data from the phase 2 ArMaDa trial in Q1 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.

References:

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