Capricor Therapeutics (CAPR) Piper Sandler 36th Annual Healthcare Conference summary

Key product and clinical development updates

• Deramiocel, composed of cardiosphere-derived cells, targets cardiomyopathy in Duchenne muscular dystrophy and works via anti-inflammatory, immunomodulatory, and anti-fibrotic mechanisms through exosome release.

• Phase 2 HOPE-2 trial met its primary endpoint in upper limb function and showed improvement in 21 of 22 cardiac MRI measures; open-label extension data demonstrated durable stabilization and improvement in both skeletal and cardiac muscle function, with benefits maintained for up to three years.

• FDA engagement led to a shift in regulatory focus, with a BLA for cardiomyopathy planned by year-end, priority review expected, and potential approval and market launch targeted for late 2025.

• Phase 3 HOPE-3 trial, with 104 patients, will support post-approval label expansion and European approval, with readout expected at the end of 2025.

Manufacturing, partnerships, and commercialization

• Manufacturing is modular, scalable, and based on robust potency assays, with initial capacity for 500 patients per year and plans to triple capacity in the first year post-launch.

• Recent fundraising and upfront payments from Nippon Shinyaku have enabled expansion of manufacturing and commercial infrastructure, providing a cash runway through 2027.

• Nippon Shinyaku holds marketing rights in the US, Japan, and Europe, with $80 million already paid and additional milestones and royalties (30%-50%) expected; their US team is prepared for launch.

Pipeline and future directions

• Engineered exosome platform StealthX is advancing, with a COVID vaccine program entering phase 1 in early 2025 and plans to deliver nucleic acid therapeutics to muscle tissue using targeted exosomes.

• Expansion into Becker muscular dystrophy and other rare cardiomyopathies is planned, leveraging both clinical and manufacturing capabilities.