Ocugen (OCGN) Study update summary

Study design and patient population

• Phase II ArMaDa trial randomized 51 adults aged 50+ with GA secondary to dry AMD to receive a single subretinal injection of OCU410 at medium or high dose, or no treatment (control).

• Baseline characteristics were balanced, with mean ages around 75–78 and mean baseline lesion sizes of 8–9 mm².

• 45 subjects were included in the safety population and 42 in the main efficacy analysis set.

• Inclusion allowed prior exposure to complement inhibitors or other GA therapies after a three-month washout; about 10–12% had such history.

• Primary endpoint was change in GA lesion size at 12 months, measured by fundus autofluorescence; exploratory endpoints included ellipsoid zone preservation on OCT.

Safety and tolerability

• No serious adverse events or adverse events of special interest were deemed related to OCU410 across both dose groups.

• One mild intraocular inflammation case in the high dose group resolved with standard care and was attributed to the procedure.

• CNV events were related to disease natural history or procedure, not the therapy.

• Safety profile was favorable even in older patients with comorbidities.

• Standardized surgical procedures contributed to consistent safety outcomes.

Efficacy results

• OCU410-treated eyes showed a statistically significant 31% reduction in lesion growth at 12 months with the optimal dose versus control (p<0.05).

• In the 2.5–17.5 mm² lesion subgroup, medium dose achieved a 31% reduction and high dose a 16% reduction in lesion growth at 12 months.

• For lesions 5–17.5 mm², medium and high doses showed 33% and 31% reductions, respectively.

• Treated eyes had a 27% slower rate of ellipsoid zone loss, indicating photoreceptor preservation and potential visual function benefit.

• 55% of treated patients achieved at least 30% lesion size reduction compared to control.