Ocugen (OCGN) Status Update summary

Corporate and clinical program updates

• Modifier gene therapy platform targets inherited and multifactorial retinal diseases, including RP, GA, and Stargardt disease, with gene-agnostic, one-time treatments addressing significant unmet needs.

• OCU400 phase III trial for RP is underway, with enrollment completion targeted for 1H2025–1H2026 and regulatory filings and potential approvals expected in 2026.

• OCU410 for GA is in phase II, showing promising safety and efficacy, with a single subretinal injection approach and no serious adverse events reported.

• OCU410-ST for Stargardt disease has completed phase I, with phase II enrollment approved and a confirmatory pivotal trial pending FDA alignment.

• Expanded access program for OCU400 is available for patients not meeting trial criteria, with Health Canada and FDA approvals enabling broader North American trial recruitment.

Clinical trial data and patient outcomes

• OCU400 demonstrated stabilization or improvement of visual function in RP and LCA, with 67% of treated eyes for LCA and 100% stabilization or improvement in pediatric patients at nine months.

• OCU410 showed a 21.4% reduction in GA lesion progression at six months, outperforming current anti-complement therapies, and 100% of patients had stabilization of visual function.

• OCU410-ST early data revealed an 84% reduction in atrophic lesion growth and up to 80% preservation of retinal structure at six months, with 60% of treated eyes showing stabilization or improvement in visual function.

• All gene therapy programs reported excellent safety and tolerability, with no serious adverse events related to the investigational products.

Patient and market perspectives

• Patient testimonials highlighted life-changing improvements in vision and quality of life, with minimal pain or recovery time from the procedure.

• Projected U.S. and EU market opportunities: $61B for RP, $115B for GA, and $23B for Stargardt disease, with significant patient populations and anticipated launches between 2025-2027.

• Gene therapy's one-time treatment model is positioned to reduce patient burden compared to current therapies requiring frequent injections.

• The company emphasizes global access and affordability, aiming to serve patients regardless of socioeconomic status while delivering shareholder value.

• OCU400 and OCU410 are positioned as first-in-class, broad-spectrum therapies with potential to transform current treatment paradigms.