CAPRICOR THERAPEUTICS, INC. Q1 FY2025 earnings call

• Linda Marban provided updates on the BLA for deramiocel in treating Duchenne muscular dystrophy cardiomyopathy, highlighting data from the HOPE-2 and HOPE-2 open-label extension studies compared to a natural history dataset. - Capricor is transitioning to a commercial stage, actively working with commercial partner NS Pharma on launch readiness for the U.S. - Appointed Dr. Michael Binks as Chief Medical Officer. - Discussed the exosome program and Project NextGen vaccine for COVID-19 prevention. - Noted a cash balance of approximately $145 million with a runway into 2027; potential $80 million milestone and Priority Review Voucher if FDA approves deramiocel.

In the first quarter of 2025, Capricor Therapeutics had zero revenue, compared to approximately $4.9 million in the first quarter of 2024. The revenue in Q1 2024 was from the ratable recognition of the $40 million under the U.S. distribution agreement with Nippon Shinyaku, which was fully recognized by December 31, 2024. As of March 31, 2025, cash, cash equivalents, and marketable securities totaled approximately $144.8 million.

• Aim to have over 100 patients transition from clinical to commercial product following potential BLA approval. - Plan to use HOPE-3 study data for potential label expansion and evaluate international expansion of HOPE-3. - Anticipate an adcom meeting related to deramiocel's BLA, though specific date is unknown; await PDUFA date. - Potential non-dilutive cash infusions over $200 million if FDA approves deramiocel, which could be used to enhance the therapeutic pipeline.

• Regulatory uncertainties, including potential Complete Response Letters (CRLs) from the FDA. - Dependence on successful BLA approval and favorable regulatory actions. - Uncertainties in the commercial launch and market reception of deramiocel.

Q: Have you had your site inspection in San Diego and preparations for the adcom?

A: The pre-licensing inspection is coming up this quarter within the next few weeks; actively preparing for the adcom, viewing it as a positive sign for the agency's engagement with the BLA.

Q: Update on Nippon Shinyaku, Europe, and NS Pharma's role?

A: Evaluating direct entry into Europe, still in negotiations with Nippon Shinyaku; NS Pharma has 125 full-time employees focusing on deramiocel's U.S. launch.

Q: Key drivers of physicians' confidence in the data?

A: Statistical significance from cardiac MRI, objective measure of disease progression, and long-term stabilization seen in data.

Q: Plan if FDA issues a CRL in August?

A: Submit HOPE-3 study data for the skeletal muscle function indication.

Q: FDA feedback on LVF as a surrogate endpoint?

A: FDA is not using left ventricular fraction (LVF) as a surrogate endpoint, instead relying on real-world evidence and natural history data.

Q: HOPE-3 data disclosure and label expansion?

A: Plan to take HOPE-3 data to Europe and may unblind it in the third or fourth quarter; could expand the label based on HOPE-3 findings.

Q: Plan for the PRV voucher?

A: Intend to sell the Priority Review Voucher to strengthen the balance sheet.

Q: Profile of patients eligible for deramiocel?

A: Patients with presence of late gadolinium enhancement (LGE) or cardiac dysfunction with an ejection fraction of 55% or less.

Q: Progress with Japanese regulators?

A: Actively working with Japanese regulators, with upcoming meetings with CMIC and plans for PMGA meetings.

Q: Adcom committee composition?

A: Panelists from the Cell and Gene Therapy Advisory Committee; practicing with prepared panelists to be ready for the adcom.