Ocugen, Inc. Q4 FY2024 earnings call

• Ocugen is advancing a novel modifier gene therapy platform across three clinical programs. Reached alignment with FDA for Phase 2/3 pivotal confirmatory trial for OCU410ST BLA targeting Stargardt disease. Goal of three potential BLAs in next three years: OCU400 in 2026, OCU410ST in 2027, OCU410 in 2028.
• OCU400: Retinitis pigmentosa affects many globally. European Commission gave positive opinion for ATMP classification. Positive two-year safety and efficacy data from Phase 1/2 trial. Phase 3 liMeliGhT trial enrolling patients, intends to complete enrollment in first half of 2025.
• OCU410ST: No approved treatments for Stargardt disease. Received orphan medicinal product designation from EMA and ATMP classification. Six-month data from Phase 1 showed clinically meaningful improvement. Phase 2/3 trial to enroll 51 subjects, primary endpoint change in atrophic lesion size, plan to initiate mid-2025, target BLA submission by 2027.
• OCU410: Targets dry age-related macular degeneration. Preliminary nine-month data showed improvement. Phase 2 study ongoing, interim data in second half of 2025, potential BLA and MAA filings by 2028.
• Biologic candidate Inhalation Vaccines Platform: OCU200 in Phase 1 clinical trial for diabetic macular edema. OCU500's IND application cleared by FDA, NIAID expected to sponsor Phase 1 trial.

For the quarter ended December 31, 2024, research and development expenses were $8.3 million compared to $7.8 million in the fourth quarter of 2023. Full year 2024 research and development expenses were $32.1 million versus $39.6 million in 2023. General and administrative expenses for the fourth quarter of 2024 were $6.3 million compared to $5.2 million in the fourth quarter of 2023. Full year 2024 general and administrative expenses were $26.7 million versus $32.0 million in 2023. Net loss for the quarter ended December 31, 2024 was approximately $13.9 million or $0.05 net loss per share, compared to a net loss of approximately $11 million or $0.04 per share in the fourth quarter of 2023. Full year net loss in 2024 was $54.1 million or $0.20 net loss per share versus $63.1 million or $0.26 net loss per share in 2023. Cash and restricted cash totaled $58.8 million as of December 31, 2024, compared to $39.5 million as of December 31, 2023, and is expected to fund operations into the first quarter of 2026.

• Cash and restricted cash as of December 31, 2024, is expected to fund operations into the first quarter of 2026. - Proactively exploring shareholder-friendly opportunities to increase working capital, including partnerships.

• Forward-looking statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from current expectations, as described in Ocugen's periodic filings with the SEC, including risk factors in Quarterly and Annual Reports.

Q: When thinking about runway, does it factor in for potentially newly launching studies like the OCU410ST Phase 2/3?

A: Yes, it's already budgeted.

Q: When could we expect to see data start to emerge from the OCU200 Phase 1, and what efficacy endpoints are being evaluated?

A: We are looking at the safety and efficacy report of OCU200 towards the end of this year. Evaluating safety of unilateral intravitreal administration, exploratory endpoints of BCVA and dose response, and secondary endpoints of antibody formation and PK of OCU200.

Q: For OCU500, will there be any grant revenue to the company associated with the Phase 1 trial?

A: NIAID is sponsoring the program, we have done our part, they will fund the Phase 1 clinical program and we'll turn it over to them to run the trial.

Q: What is your manufacturing strategy for 410ST and do you plan on using the commercial-grade product for the Phase 2/3 study?

A: We already made at a commercial scale for Phase 1 and will use the same scale for Phase 2/3, following the similar pattern as with RP and FDA agreed with the strategy.

Q: What proportion of US and ex-US sites are you looking at for the 410ST Phase 2/3 study?

A: There are no ex-US sites except Canada, and we think we can get the 51 patients needed in the US quickly