Ocugen, Inc. Q2 FY2025 earnings call

• Ocugen's novel modified gene therapy platform has all 3 candidates advancing, with 2 in late stage, aiming for 3 biological licensing applications (BLA) and market authorization application (MAA) filings in the next 3 years.
• OCU400 Phase III liMeliGhT clinical trial for retinitis pigmentosa (RP) has patients recruited in the US and Canada, on track for BLA and MAA filings in 2026; social media collaboration with an RP patient advocate to raise awareness.
• OCU410ST for Stargardt disease received Rare Pediatric Disease Designation, IND Amendment cleared in June, and first patient dosed in July in the Phase II/III GARDian3 registration trial; Phase I data for OCU410ST and OCU410 showed favorable safety and efficacy.
• OCU410 for geographic atrophy secondary to dry age-related macular degeneration (dAMD) had Phase I 12-month data showing 23% slower lesion growth and stabilization/gain in visual function.
• Signed binding term sheet for exclusive Korean rights to OCU400, expecting to close definitive agreement by September; proposed reverse merger with OrthoCellix to create a NASDAQ-listed regenerative cell therapy company focused on orthopedic diseases.
• New Board appointments and updates to Retina Scientific Advisory Board; strengthened internal expertise with new Chief Development Officer, Executive Vice President of Commercial and Business Development, and VP of Manufacturing.

No traditional product segment financial performance with revenue contributions detailed; focus is on gene therapy candidates advancing in clinical trials.

• On track to meet goal of 3 BLA and MAA filings in the next 3 years.
• OCU400 Phase III liMeliGhT trial is on target for BLA and MAA filings in 2026.
• OCU410ST Phase II/III trial is potentially accelerating clinical development, with BLA expected in 2027.
• Expect to close the definitive agreement for the OCU400 Korean licensing by September.
• Proposed reverse merger with OrthoCellix planned with $25 million in private financing, and OrthoCellix estimated to have a value of $135 million.

• Forward-looking statements subject to risks and uncertainties described in Ocugen's periodic filings with the SEC.
• Uncertainties in clinical development timelines and regulatory approvals for gene therapy candidates.
• Dependence on strategic partnerships and adequate funding to advance clinical programs and secure regulatory approvals.

Q: Are there any other deals being explored, such as regional license agreements, asset sales, or spin-outs?

A: We are continuously looking for potential partnership opportunities, including regional partnerships for all our gene therapy programs.

Q: Will the rare nature of Stargardt disease make enrollment challenging?

A: No, there are approximately 44,000 Stargardt patients in the US with no approved product, inclusion/exclusion criteria are amenable, and we have 15 centers activated with plans to activate more.

Q: What was the DSMB's review of OCU400?

A: It was purely a safety analysis, no futility analysis, and no serious adverse events related to the investigational product were reported.

Q: When is the interim update for OCU410 expected?

A: The interim update for OCU410 will be in the fourth quarter, analyzing structural and functional outcomes.

Q: What is the approval path for OCU400 in Korea?

A: Korea can potentially use our US FDA approval to get approval for OCU400 without further clinical trials.

Q: Progress of OCU200 Phase I trial?

A: Dosing in Cohort 3, data expected later this year on safety and efficacy.