Ocugen, Inc. Q3 FY2025 earnings call

• Modifier gene therapy platform progress: OCU400 nears Phase 3 enrollment completion, OCU410ST Phase 2/3 trial on track for Q1 2026 enrollment completion and H1 2027 BLA submission. OCU410 shows potential for dry AMD with positive Phase 1 data.
• Partnerships: Exclusive licensing agreement with Kwangdong Pharmaceutical for OCU400 in South Korea, with upfront and milestone payments.
• Financials: Cash position as of September 30, 2025, is $32.9 million, with operating expenses for Q3 2025 at $19.4 million including R&D and G&A expenses.

Ocugen's segment performance is centered around its modifier gene therapy platform and drug candidates. The lead candidate OCU400 is nearing Phase 3 enrollment completion, with the OCU410ST Phase 2/3 pivotal confirmatory trial on track to complete enrollment in Q1 2026 and planned BLA submission in H1 2027. OCU410 shows promise for dry age-related macular degeneration. Financially, cash, cash equivalents, and restricted cash totaled $32.9 million as of September 30, 2025, with operating expenses for the quarter at $19.4 million.

• OCU400 Phase 3 liMeLiGhT trial on track for BLA and MAA submissions in 2026.
• OCU410ST Phase 2/3 trial to complete enrollment in Q1 2026, BLA submission in H1 2027.
• $20 million financing in Q3 extends runway through Q2 2026, with potential $30 million if warrants exercised extending runway into 2027.
• OCU410 to provide full 12-month Phase 2 data in Q1 2026 and initiate Phase 3 study next year.

• Forward-looking statements subject to numerous risks and uncertainties, detailed in SEC filings, including risks that may cause actual events or results to differ materially from expectations.

Q: About OCU400's clinical trial design and FDA discussions A: Shankar Musunuri and Huma Qamar discussed that OCU400 trials have control arms within the study, differentiating from uniQure's situation, and detailed the trial design with 150 subjects, 2:1 randomization, and 97% power.

Q: On OCU410ST enrollment A: Huma Qamar stated OCU410ST's Phase 2/3 GARDian3 trial is almost 50% enrolled, with 51 subjects (34 treatment, 17 control) and no geographic restrictions Q: On OCU400 commercial infrastructure A: Shankar Musunuri said they are ramping up slowly for U.S. commercialization, looking for partnerships like the one in South Korea, and will allocate a limited budget starting next year Q: On OCU410 endpoints A: Huma Qamar discussed endpoints in the Phase 2 study including lesion growth reduction and visual equity compared to untreated controls Q: On manufacturing capacity A: Shankar Musunuri talked about having ex-U.S. manufacturing capacity and plans to have a U.S. manufacturing site ready by 2027 for producing U.S. supply Q: On OCU400 BLA prep A: Shankar Musunuri said they will initiate rolling submission in H1 2026, submitting nonclinical and CMC sections first, followed by clinical sections later Q: On LCA A: Shankar Musunuri stated LCA is not in current plan as the market is small, but could be considered for Phase 4 after RP commercialization