Ocugen has secured a win for its one-time gene therapy, OCU410, in geographic atrophy (GA), though the recent data is clouded by previous studies.
In the ongoing Phase II ArMaDa study (NCT06018558), Ocugen is assessing the potential of its subretinal therapy in patients with GA secondary to dry age-related macular degeneration (dAMD).
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Analysis of the 12-month data from this study found that patients treated with OCU410 experienced a 31% reduction in macular lesion growth compared with the control group at 12 months. While this result was statistically significant, it does not match the efficacy observed in the preliminary 12-month analysis, where OCU410 reduced lesion growth by 46%.
The results also revealed that 55% of patients on the OCU410 arm experienced a lesion size reduction of 30% or more versus control. It is currently unclear exactly how this directly compares with the preliminary analysis, where half of the treated patients achieved a ≥50% drop in lesion size.
This study also demonstrated OCU410’s safety and tolerability, with no serious treatment-emergent adverse events (TEAEs) seen throughout the trial as of yet.
OCU410’s potential in the GA market
Despite the muted investor response to the ArMaDa readout, Ocugen’s chair and CEO, Shankar Musunuri, noted that he has a “high degree of confidence” in OCU410’s treatment effect. “This moves us one step closer to bringing a transformative one-time treatment to GA patients globally,” Musunuri stated.
To take a step closer to this reality, Ocugen is looking to initiate a pivotal Phase III study on OCU410 in Q3 2026, which the biotech hopes will enrol up to 300 patients with GA.
If OCU410 were to gain approval in GA, it would join Apellis Pharmaceuticals’ Syfovre (pegcetacoplan) and Astellas Pharma’s Iservay (avacincaptad pegol) on the market, which are currently the only two medications which have secured the regulatory greenlight in this indication. Both drugs are available in the US, though European regulators declined to approve both drugs due to its concern with each’s benefit-risk profile.
According to Sara Reci, managing analyst at GlobalData, the results of the ArMaDa study are “notably lower in magnitude” from the readout in January, OCU410’s 31% lesion reduction from a single subretinal administration compares favourably to approved complement inhibitors like Izervay (avacincaptad pegol) and Empaveli (pegcetacoplan).
“The core market proposition is a potential one-time surgical alternative to the 6-12 chronic intravitreal injections per year currently required, which is particularly compelling given real-world drop-out rates reported at over 40% with approved agents by 18 months,” Reci stated.
If the Phase III trial reproduces this effect, Reci notes that OCU410 could carve a “meaningful niche” within the GA indication.
GA currently impacts an estimated eight million people worldwide, and is one of the leading causes of legal blindness.
A recent report from GlobalData, parent company of Clinical Trials Arena, identified the availability of GA therapies as a key growth driver for the AMD market, as this will enhance the number of treatable cases within this segment.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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