Ocugen (OCGN) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Gene therapy platform and strategy

• Focus remains on gene therapies, particularly modifier gene technology, to address multiple blindness diseases.

• Modifier gene therapies target master regulator genes, aiming to restore retinal network function and cell survival.

• Lead candidate OCU400 is in phase III for retinitis pigmentosa (RP), with gene-agnostic and rhodopsin-specific trial arms.

• OCU410 is in phase I/II for geographic atrophy (dry AMD) and Stargardt disease, leveraging RORA as a master regulator.

• Platform aims to provide broad solutions for diseases with high unmet needs and complex genetic backgrounds.

Clinical trial progress and design

• OCU400 phase III trial includes both gene-agnostic and rhodopsin-specific cohorts, targeting all RP mutations.

• Phase I/II data showed a 62.5% responder rate in mobility tests, surpassing the approved benchmark.

• Expanded access program for OCU400 allows patients outside strict trial criteria to receive treatment before approval.

• Health Canada approved trial sites, supporting faster recruitment and parallel regulatory submissions in US, EU, and Canada.

• Data lock for OCU400 phase III is expected in early 2026, with potential approvals by end of 2026.

Market opportunities and regulatory pathways

• RP affects 1.6 million globally; OCU400 aims to address all genetic forms, unlike single-gene therapies.

• Dry AMD has 260 million global cases; OCU410 targets multiple disease pathways for broader efficacy.

• Stargardt disease has 44,000 US patients with no approved therapies; OCU410ST is in phase I/II.

• OCU410 phase II is ongoing, with plans to complete recruitment by end of next year and seek a partner before phase III.

• Regulatory designations (RMAT, orphan drug) facilitate accelerated review and rolling submissions.