Ocugen reports positive 12-month Phase 2 ArMaDa trial results for OCU410 gene therapy in geographic atrophy secondary to dry AMD, showing significant reduction in lesion growth
Written By: Anand Sathappan, BPharm
Reviewed By: Pharmacally Editorial Team
Ocugen, Inc. announced positive 12-month results from the Phase 2 ArMaDa clinical trial (NCT06018558) evaluating OCU410 (AAV5-RORA), an investigational modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). The results showed significant reduction in lesion growth along with preservation of retinal structure, supporting advancement of the therapy into a Phase 3 registrational trial.
OCU410 is designed as a one-time gene therapy delivered through a subretinal injection. The therapy delivers the RORA (retinoid-related orphan receptor alpha) gene and is intended to regulate multiple pathways involved in retinal degeneration, including oxidative stress, inflammation, complement activation, and lipid metabolism.
In the Phase 2 study, the optimal medium dose of OCU410 demonstrated a 31% reduction in GA lesion growth compared with control (p<0.05) at 12 months. Investigators also reported a 27% slower rate of ellipsoid zone loss, suggesting preservation of photoreceptor structure associated with visual function. Additionally, 55% of treated patients achieved at least a 30% reduction in lesion size versus control. Subgroup analysis of patients with baseline lesions between ≥5 mm² and ≤17.5 mm² showed a 33% reduction in lesion growth, with similar effects observed in the high-dose cohort.
The therapy demonstrated a favorable safety profile with no OCU410-related serious adverse events reported. Investigators also observed no cases of endophthalmitis, retinal detachment, vasculitis, choroidal neovascularization, or ischemic optic neuropathy during the study.
Ocugen leadership said the results provide strong evidence of treatment benefit and support progression to a Phase 3 study. The company plans to launch a pivotal Phase 3 trial enrolling up to 300 patients using an adaptive design.
The Phase 2 trial enrolled 51 patients aged 50 years or older with GA lesions in the foveal or non-foveal region. Participants were randomized to receive a single subretinal injection of OCU410 at either a medium or high dose, or to a control group. The primary endpoint evaluated change in GA lesion size at 12 months using fundus autofluorescence imaging.
Ocugen expects to initiate the Phase 3 registrational trial in the third quarter of 2026 as part of its broader strategy to advance multiple gene therapy programs toward regulatory submissions.
Geographic atrophy is an advanced form of dry age-related macular degeneration that leads to progressive loss of central vision. Dry AMD affects more than 266 million people worldwide, and GA remains an area of significant unmet medical need despite recent treatment advances.
Reference
Ocugen Announces Topline 12-month Data from Phase 2 ArMaDa Clinical Trial Evaluating OCU410 Modifier Gene Therapy for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration, 24 March 2026, https://ir.ocugen.com/news-releases/news-release-details/ocugen-announces-topline-12-month-data-phase-2-armada-clinical
Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy (ArMaDa), ClinicalTrials.gov ID NCT06018558, https://clinicaltrials.gov/study/NCT06018558
About the Writer
Anand Sathappan is a pharmacy graduate with interests in pharmaceutical sciences, pharmacology, and medical writing. He also holds certification in medical coding and has familiarity with pharmacology and healthcare documentation. He has completed the Medical Writing Skills for Beginners program offered by the International Medical Writers Association (IMWA). His work focuses on exploring developments in pharmaceuticals and presenting scientific information in a clear and structured manner.