Ocugen (OCGN) Canaccord Genuity's 46th Annual Growth Conference summary

Strategic vision and pipeline overview

• Leading late-stage gene therapy programs for blindness diseases, targeting retinitis pigmentosa, Stargardt, and dry AMD, with two BLAs planned for next year and AMD in 2028.

• Aiming for global approvals and launches in the U.S., EU, and Japan within three years to address significant unmet needs in ophthalmology.

• Modifier gene therapy platform leverages nuclear hormone receptors (Nr2e3 and RORA) to address broad disease pathways, not just single mutations.

• Technology originated from Harvard, with IP covering both ophthalmology and neurospace applications.

Clinical program highlights

• OCU410 for Stargardt and GA uses RORA to regulate oxidative stress, lipid metabolism, and inflammation, aiming to reset cellular homeostasis and potentially reverse disease progression.

• Stargardt phase II/III trial includes patients from age 3+, with rare pediatric disease designation and EMA endorsement; top-line data expected Q2 next year, BLA filing to follow.

• OCU410 for GA received RMAT designation, enabling accelerated approval and close FDA collaboration; phase III global trial starting this quarter.

• OCU400 for RP uses Nr2e3, targeting phototransduction pathways; phase III pivotal readout expected Q1 next year, with robust design and broad mutation coverage.

• Over 325 patients treated across trials, with strong safety data and no serious adverse events related to gene therapy.

Regulatory and commercialization plans

• Rolling BLA submissions planned under RMAT, with commercial-scale lots and CMC sections ready; approval clock starts with final clinical module.

• If timelines hold, potential approvals and launches for RP and Stargardt in 2028.

• $130 million convertible note financing extends runway into 2028; spend expected to remain stable as recruitment completes and new trials begin.

• Actively pursuing regional partnerships for ex-U.S. commercialization and evaluating non-dilutive funding opportunities.