Capricor Therapeutics (CAPR) Leerink’s Global Healthcare Conference 2025 summary

Key product and clinical development updates

• Deramiocel, an allogeneic cell therapy for Duchenne muscular dystrophy cardiomyopathy, is the primary focus, with a commercial-scale facility and partnerships in the US and Japan.

• Exosome-based pipeline programs are expected to enter the clinic in 2026.

• BLA for deramiocel was completed in late 2024, accepted for priority review in March 2025, with a PDUFA date of August 31, 2025.

• Multiple regulatory designations (orphan, RMAT, ATMP, rare pediatric disease) support the pathway to approval.

• Manufacturing uses explanted, transplant-qualified human hearts, with a proprietary process and quarterly dosing.

Clinical efficacy and safety data

• Over 200 patients have been treated; mechanism validated by FDA-approved potency assays.

• HOPE-2 trial showed 71% slowing of skeletal muscle disease and 107% slowing of cardiac disease progression, with significant p-values.

• Open-label extension (OLE) patients show sustained stabilization and improvement in cardiac and skeletal muscle function over up to four years.

• Side effects are mild, including flu-like symptoms, headache, and nausea in about 30% of patients.

• No immune rejection issues due to the transient nature of the infused cells and exosome-mediated mechanism.

Regulatory and commercial strategy

• FDA has been closely engaged, reviewing patient-level data and supporting the BLA despite small sample size due to robust, objective MRI endpoints.

• Post-marketing requirements will be discussed prior to PDUFA.

• Commercial launch is anticipated before the end of 2025, with 100 OLE patients likely to transition first.

• Revenue share with partner Nippon Shinyaku is 30%-50% of net sales; partner handles all SG&A, while manufacturing remains in-house.

• Expansion plans include Becker muscular dystrophy and other orphan cardiomyopathies, with manufacturing capacity scaling up.