CAPRICOR THERAPEUTICS, INC. Q3 FY2024 earnings call

• Regulatory Update: Filing a BLA for full approval of deramiocel for DMD cardiomyopathy, using HOPE-2 data vs natural history data from DMD Cardiac Consortium. Expect FDA decision by end of Q1 2025, potential PDUFA Q2 2025. Combining HOPE-3 cohorts A and B for skeletal muscle label later.
• Manufacturing: In-house manufacturing, San Diego facility operational, planning new facility within a year of launch, using transplant-qualified hearts, modular clean rooms, preparing for pre-licensing inspection.
• Corporate/Commercial: Completed oversubscribed public offering, partnership with Nippon Shinyaku for U.S. and European commercialization, exosomes program progress with StealthX platform, StealthX vaccine collaboration with NIAID for COVID-19 prevention.

Revenues for the third quarter of 2024 were approximately $2.3 million compared with approximately $6.2 million for the third quarter of 2023. The revenue source is the ratable recognition of the $40 million from the U.S. exclusive commercialization and distribution agreement with Nippon Shinyaku. As of September 30, 2024, cash, cash equivalents and marketable securities totaled approximately $85 million. Subsequent to September 30, 2024, a public offering of common stock raised approximately $80.8 million in net proceeds, and on a pro forma basis, cash, cash equivalents and marketable securities totaled approximately $166 million.

• Anticipate FDA decision on BLA by end of Q1 2025, potential PDUFA Q2 2025.
• Plan to expand manufacturing to meet post-launch demand.
• 2025 goal to explore indication expansion for Becker muscular dystrophy.

• Uncertainties in regulatory approval timelines.
• Dependence on successful manufacturing scale-up and pre-licensing inspection.
• Market adoption and reimbursement risks for deramiocel.

Q: What are you and Nippon Shinyaku doing to prepare the market and scale manufacturing for deramiocel launch?

A: Nippon Shinyaku has a pre-established team for DMD, plug-and-play model with Capricor's expertise. Manufacturing has been in-house, San Diego facility ready, planning new facility with modular clean rooms.

Q: Regarding second half '25 guidance for approval and RMAT designation, what's the expectation?

A: RMAT designation includes priority review, building timelines around it.

Q: What constitutes clinically meaningful benefit in cardiac function for deramiocel, especially in severe patients?

A: Improvement in all patients, with those below 45% LVEF showing less improvement but still benefit.

Q: Thoughts on AdCom for deramiocel BLA?

A: Relationship with FDA is collaborative, data is strong, but preparing for AdCom if needed.

Q: Potential impact of HOPE-3 readout on deramiocel sales?

A: Expanding the label could add to sales, as any additional eligible patients bring in dollars.

Q: Development plans for 2025 regarding Becker muscular dystrophy?

A: Planning to explore indication, working with KOLs and agency to design trial.