Ocugen has posted positive results for its lead investigational gene therapy, OCU410, in geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD).
In a preliminary 12-month data readout from the ArMaDa study (NCT06018558), the single-dose gene therapy demonstrated a 46% reduction in macular lesion growth across the medium and high dose groups versus control at 12 months.
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A 54% reduction in lesions was seen across the medium dose cohort, while patients allocated to the high dose had 36% less lesions compared with placebo after 12 months of treatment.
Of those given OCU401, 50% achieved a greater than 50% lesion size reduction versus the control group, which Ocugen’s CMO, Huma Qamar, said demonstrated “robust anatomic efficacy”.
While details on OCU410’s safety profile remain scarce, Ocugen noted that no serious treatment-emergent adverse events (TEAEs) have been reported thus far in the Phase II study.
Following the positive outcome of the ArMaDa trial, Ocugen will progress OCU410 to Phase III, with the trial beginning in 2026. The biotech will also report full data from the Phase II trial later in Q1 2026.
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By GlobalDataExpanding options in GA
According to the US-based biotech, the results of the ArMaDa trial could be significant for patients in Europe, as there are currently no approved medicines for dAMD or GA.
In the US, there are currently two treatments indicated for these two ophthalmic indications, including Apellis Pharmaceuticals’ Syfovre (pegcetacoplan) and Astellas Pharma’s Iservay (avacincaptad pegol). While both companies filed for European regulatory approval, the European Medicines Agency (EMA) rejected their applications based on concerns around each medicine’s risk-benefit profile.
There are other candidates in trials, which companies hope will garner better sentiments from global regulators, including several disease-modifying therapies (DMTs).
One example is Belite Bio’s retinol-binding protein 4 (RBP4) inhibitor tinlarebant, which is currently being evaluated in the global, pivotal Phase III PHOENIX trial (NCT05949593) in GA. Enrolment in this study concluded in July 2025.
Aviceda Therapeutics is also looking to throw its hat into the GA ring with AVD-104, which is set to enter a Phase III study in the indication despite missing the trial’s primary endpoint in the Phase IIb SIGLEC study (NCT05839041).
German pharma Boehringer Ingelheim has set its sights on the GA market with BI 1584862, which the company is assessing as a vision preservation agent in the US-only Phase II JADE study (NCT06769048).
According to the Age-Related Macular Degeneration: Seven-Market Drug Forecast and Market Analysis report from GlobalData, the AMD market is set to reach $20.5bn across the seven major markets (7MM: the US, France, Germany, Italy, Spain, the UK, and Japan) by 2034.
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