Ocugen (OCGN) Study result summary

Clinical context and unmet need

• Geographic atrophy (GA) affects 2–3 million patients in the US and EU, with limited treatment options and significant disease burden.

• GA is a late-stage, vision-threatening form of dry AMD affecting millions globally.

• Recently approved therapies require frequent injections and only target one disease pathway, leaving unmet needs for efficacy and convenience.

Therapy profile and mechanism of action

• OCU410 is a one-time subretinal gene therapy using AAV5 to deliver RORA, targeting anti-drusen, anti-oxidative, anti-inflammatory, and anti-complement pathways.

• OCU410 aims to address all four major GA pathways for durable benefit and reduced treatment burden.

• OCU410 has received Advanced Therapy Medicinal Product classification from the European Medicines Agency.

• Preclinical studies show RORA activation reduces drusen, protects against oxidative stress, suppresses inflammation, and upregulates complement regulatory proteins.

• The multi-pathway approach is expected to provide more robust and durable therapeutic benefit than single-pathway therapies.

Phase I study design and results

• Phase I was an open-label, dose-escalation study in nine elderly AMD patients, focusing on safety and tolerability.

• No serious adverse events or inflammatory complications were observed, supporting advancement to phase II.

• OCU410 showed a 20.2% reduction in GA lesion growth at 12 months versus untreated fellow eyes.

• Treated eyes had 60% slower loss of the ellipsoid zone, indicating preserved photoreceptor structure.

• Reduced loss of the EZ-RPE complex suggests protection of both photoreceptors and RPE.