CAPRICOR THERAPEUTICS, INC. Q4 FY2024 earnings call

• Regulatory: BLA for DMD cardiomyopathy accepted by FDA for priority review with PDUFA date of August 31, 2025; prelicensing inspection of manufacturing facility scheduled for Q2 2025. - Commercial: Working with NS Pharma on US launch readiness, focusing on market access, reimbursement; ~50%-60% of US DMD population (~7,500 boys/young men) eligible for treatment. - Manufacturing: Expanding San Diego facility to support 2,000-3,000 patients/year by mid-2026. - Exosomes pipeline: StealthX exosome platform in development, proof-of-concept data presented, Phase 1 clinical trial for vaccine candidate planned by NIAID.

In the fourth quarter of 2024, revenues were approximately $11.1 million compared to approximately $12.1 million in the fourth quarter of 2023. The revenue source was the ratable recognition of the $40 million from the US distribution agreement with Nippon Shinyaku and the $10 million milestone payment triggered in December 2024. Research and development expense excluding stock-based compensation for Q4 2024 was approximately $13.6 million compared to approximately $9.4 million in Q4 2023. G&A expense excluding stock-based compensation was approximately $3 million in Q4 2024 compared to approximately $2.1 million in Q4 2023. Net loss for Q4 2024 was approximately $7.1 million compared to a net loss of approximately $800,000 in Q4 2023. For the full year 2024, net loss was approximately $40.5 million compared to a net loss of approximately $22.3 million in 2023.

• FDA PDUFA date for BLA is August 31, 2025. - Potential $80 million milestone payment from Nippon Shinyaku upon FDA approval. - Anticipated revenue from ~50%-60% of US DMD population eligible for deramiocel treatment.

• Regulatory: Uncertainty around AdCom necessity, potential delays due to government turmoil. - Commercial: Reimbursement challenges, competition, and manufacturing scaling issues.

Q: Ted Tenthoff asks about commercial preparation for deramiocel and division of labor with NS Pharma.

A: Linda Marban states NS has ~125 employees in US dedicated to deramiocel commercialization, working on market access, meeting payers, and building out launch activities with Capricor shepherding deramiocel to market and NS selling/distributing.

Q: Leland Gershell asks about capital investment plans.

A: Linda Marban mentions evaluating label extension for Duchenne and Becker muscular myopathy, and focusing on exosomes pipeline with updates to come.

Q: Joseph Pantginis asks about new facility costs and EMA discussions.

A: A.J. Bergmann says expansion plans are underway with construction operations, and Linda Marban discusses working with EMA in Q2 2025 and ongoing negotiations with NS for definitive agreement in Europe.

Q: Kristen Kluska asks about FDA view on single-arm studies in HOPE-2 OLE.

A: Linda Marban explains use of Vanderbilt Natural History Cardiac Consortium data as a natural history comparator, noting cardiac MRI is non-volitional and strong statistical significance supports the data.

Q: Aydin Huseynov asks about Becker MD and other heart diseases.

A: Linda Marban says working on Becker program, evaluating orphan cardiomyopathies for deramiocel expansion with more updates in next quarters.

Q: Madison El-Saadi asks about ejection fraction and eligibility.

A: Linda Marban discusses early treatment for better outcomes, messaging early intervention to care providers, and that eligibility is a shared decision among care providers.