Capricor Therapeutics (CAPR) Corporate Presentation summary

Company evolution and scientific foundation

• Originated from discovery of cardiosphere-derived cells (CDCs) at Johns Hopkins in 2004, with key clinical and preclinical milestones over 20 years.

• Developed Deramiocel, a cellular therapy for Duchenne muscular dystrophy (DMD), and StealthX exosome platform for drug delivery.

• Built an extensive IP portfolio with ~150 patents and established in-house GMP manufacturing.

• Secured commercial partnerships with Nippon Shinyaku for U.S. and Japan distribution.

• Uplisted to NASDAQ in 2014 and maintains a strong leadership team with deep industry experience.

Deramiocel DMD program and clinical data

• Deramiocel is an allogeneic CDC therapy with orphan drug and advanced therapy designations in the U.S. and Europe.

• Phase 2 HOPE-2 trial showed statistically significant slowing of DMD progression in both skeletal and cardiac function, with favorable safety profile.

• 3-year open-label extension data demonstrated 99% slowing of cardiac and 52% slowing of skeletal disease progression compared to external controls.

• Phase 3 HOPE-3 trial fully enrolled, topline data expected Q4 2025; FDA alignment allows data submission to current BLA.

• Manufacturing process enables multi-dose, cryopreserved product; FDA completed pre-license inspection in June 2025.

Financials and commercial outlook

• Cash balance of $123 million as of June 30, 2025, with runway into Q4 2026.

• Eligible for $80 million milestone at U.S. approval and up to $605 million in sales-based milestones from NS Pharma.

• Potential for $150 million from sale of a priority review voucher if received.

• Annual revenue estimates could exceed $1.5 billion with small market penetration; pricing targeted similar to exon skipping therapies.

• Commercial partnerships provide 30-50% of product revenue in the U.S. and double-digit share in Japan.