Ocugen (OCGN) investor relations material

Strategic pipeline and clinical development

• Three gene therapy BLAs are planned within three years, starting in 2026, targeting retinitis pigmentosa, Stargardt disease, and geographic atrophy.

• OCU400, the lead candidate for retinitis pigmentosa, is in phase 3 with a broad indication covering most mutations and aims for BLA submission in 2026.

• OCU410ST for Stargardt disease and OCU410 for geographic atrophy are advancing, with adaptive trial designs and promising early data.

• EMA has agreed to accept US clinical data for European submission, expediting potential market access.

• The company’s modifier gene therapy platform targets cellular homeostasis, offering broad applicability across genetic mutations.

Clinical data and endpoints

• OCU400 phase 3 trial uses a sensitive luminance-dependent navigation assessment as the primary endpoint, with secondary endpoints including low luminance visual acuity.

• Prior trials show 24-month durability, safety, and statistically significant efficacy with no serious adverse events.

• OCU410ST for Stargardt disease demonstrated a 48% lesion size reduction at 12 months in early to advanced cases, with a single subretinal injection.

• OCU410 for geographic atrophy showed a 27% lesion reduction at six months, outperforming existing therapies in early data.

• Both structural and functional endpoints are being pursued for regulatory approval.

Financial strategy and balance sheet

• Divestment of the neocart asset through a reverse merger valued at $150 million allows focus on gene therapies and provides a significant balance sheet asset.

• The company maintains a quarterly burn rate of $12 million, with a current cash runway of $50 million, recently extended by a $20 million investment and a potential $30 million option.

• Runway is projected to extend into 2027, supporting ongoing clinical programs.