Ocugen (OCGN) Piper Sandler Virtual Ophthalmology Day summary

Pipeline overview and strategic goals

• Three late-stage gene therapy programs target retinitis pigmentosa, Stargardt disease, and geographic atrophy, addressing significant unmet needs in inherited retinal diseases and AMD.

• Plans to file two BLAs in the next year for retinitis pigmentosa and Stargardt, with a third BLA for geographic atrophy expected in 2028, aiming for global approvals and launches.

• Programs are designed as one-time treatments, with broad patient coverage from pediatric to adult and early to advanced stages.

• All programs utilize adaptive trial designs and robust safety monitoring, with no serious adverse events related to the drugs reported to date.

OCU410 for geographic atrophy (GA)

• OCU410 uses a modified RORA gene to regulate multiple disease pathways, aiming to reset cellular homeostasis and potentially reverse disease progression.

• Phase II showed a 31–33% reduction in lesion size at one year with the medium dose; phase III will use this dose and focus on lesion size as the primary endpoint.

• Phase III will enroll fewer than 300 patients, with adaptive design and interim analysis at 50% enrollment to optimize trial power.

• Secondary endpoints include ellipsoid zone preservation and low luminance visual acuity, with long-term follow-up mandated for safety.

• EMA and FDA alignment is ongoing, with additional visual function data to be provided at filing.

OCU410ST for Stargardt disease

• Phase I data showed a 54% reduction in lesion growth at one year compared to untreated eyes, with strong safety and tolerability.

• Phase II/III pivotal trial uses adaptive sample size re-estimation, with over-recruitment already achieved to minimize risk.

• The therapy targets a broad patient population and aims for a one-time treatment effect, with potential to stall or reverse disease progression.

• Top-line results are expected in the second quarter of next year, with BLA filing anticipated soon after.