AwesomeCapital: Capricor projects FDA priority review decision for deramiocel by August 2025

Wednesday, March 19, 2025

CEO Linda Marban highlighted the acceptance by the FDA of Capricor's Biologics License Application (BLA) for deramiocel, targeting treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy. The application has been granted priority review with a PDUFA date set for August 31, 2025.
The BLA submission is supported by data from the Phase 2 HOPE-2 and HOPE-2 open-label extension trials, combined with natural history data from the DMD Cardiac Consortium.
Capricor plans to begin commercial production of deramiocel at its San Diego facility, which can handle up to 500 patients annually, and is expanding its manufacturing by leasing an additional 25,000 square feet to increase capacity to 2,000-3,000 patients annually by mid-2026.
CFO A.J. Bergmann reported a strong financial position with approximately $151.5 million in cash, cash equivalents, and marketable securities as of December 31, 2024, and an additional $10 million milestone payment received post-quarter end from Nippon Shinyaku.

Capricor anticipates FDA feedback on the need for an advisory committee (AdCom) for deramiocel's review but is preparing for such a possibility.
If approved, the company expects reimbursement for deramiocel to align with other DMD therapies. Initial market entry aims to transition approximately 100 patients currently in open-label extension trials to commercial therapy, with a total addressable market of around 7,500 eligible patients in the U.S.
The company continues collaboration with its commercial partner, NS Pharma, for U.S. market access and reimbursement strategies.

Revenues for Q4 2024 were $11.1 million, primarily from recognition of distribution agreement milestones.
Research and development expenses increased to $13.6 million compared to $9.4 million in Q4 2023, reflecting heightened development activity.
Net loss for Q4 2024 was $7.1 million, compared to $0.8 million in Q4 2023, driven by increased operational costs.

Edward Tenthoff, Piper Sandler: Questioned the collaboration dynamics with Nippon Shinyaku in preparation for deramiocel’s launch. CEO Marban explained that Capricor is providing product expertise while Nippon Shinyaku focuses on sales and distribution using its existing DMD-focused infrastructure.
Leland Gershell, Oppenheimer: Asked about payer feedback regarding premium-priced therapies. Marban reiterated favorable responses due to deramiocel addressing an unmet need in DMD cardiomyopathy.
Kristen Kluska, Cantor Fitzgerald: Inquired about non-ambulatory patient data and potential label extensions. Marban noted that year-over-year functional improvements in upper limb performance support deramiocel's broader therapeutic potential.

Analysts displayed positive sentiment, emphasizing Capricor’s progress in regulatory and commercial readiness.
Management maintained a confident tone, particularly on regulatory pathways and manufacturing expansion. Notable expressions included "high confidence" in facility readiness and "strong statistical significance" of trial data.

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