Ocugen (OCGN) Status Update summary

Corporate and clinical program update

• Three gene therapy programs (OCU400, OCU410, OCU410-ST) are advancing for inherited retinal diseases and age-related macular degeneration, focusing on global patient access and regulatory alignment in the US, EU, and Canada.

• OCU400 phase III trial for RP is underway, with enrollment on track for completion in the first half of 2026 and regulatory filings planned for late 2026.

• OCU410 is in phase II for geographic atrophy, showing promising safety and efficacy, with a single subretinal injection approach and no serious adverse events reported; dosing completion is planned by the second half of 2025.

• OCU410-ST for Stargardt disease has completed phase I dosing, with favorable safety and early efficacy signals; phase II enrollment is approved and design is being aligned with FDA for potential pivotal trial.

• Expanded access program for OCU400 is available for patients not meeting trial criteria, and Health Canada approval enables broader North American recruitment.

Scientific and clinical insights / Innovation and therapeutic approach

• Modifier gene therapy platform is gene-agnostic, targeting multiple pathways and genotypes, offering potential for stabilization or reversal of disease in inherited and multifactorial retinal conditions.

• OCU400 demonstrated stabilization or improvement in visual function in 67% of treated eyes in LCA and RP, with 100% stabilization or improvement in pediatric FST at 9 months.

• OCU410 showed a 21.4% reduction in geographic atrophy lesion growth at 6 months, outperforming current anti-complement therapies, and 100% of patients had stabilization of visual function.

• OCU410-ST early data showed 84% reduction in atrophic lesion growth and 80% preservation of macular volume at 6 months, with 60% of treated eyes showing stabilization or improvement in visual function.

• All three programs report excellent safety and tolerability, with no serious adverse events related to the investigational products.

Patient experience and commercial outlook / Market opportunity and commercial outlook

• Patients reported positive experiences with the gene therapy trials, highlighting ease of procedure, minimal pain, and hope for improved vision and quality of life.

• Projected market opportunities: $61B for RP (OCU400), $115B for GA (OCU410), and $23B for Stargardt (OCU410ST), with anticipated launches between 2025-2027.

• The company aims to provide one-time, durable treatments that reduce patient burden compared to current therapies requiring frequent injections.

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

• High demand and interest from patients and clinicians reflect the significant unmet need and enthusiasm for these novel therapies.