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 company founded in 2005 and public since 2014.

• Progressed from early clinical validation to late-stage trials and platform expansion, including exosome-based therapeutics.

• Lead program targets Duchenne muscular dystrophy (DMD), a rare genetic disease affecting 15,000 in the U.S.

• Extensive intellectual property portfolio with ~150 patents and applications.

• In-house GMP manufacturing facility in San Diego, with FDA pre-license inspection completed in 2025.

Strategic priorities and pipeline

• Focus on advancing Deramiocel for DMD approval and expanding pipeline into additional indications and global markets.

• Long-term vision to lead in cell and exosome-based medicines for rare diseases.

• Pipeline includes Deramiocel for DMD and Becker muscular dystrophy, and StealthX exosome platform for infectious diseases and other indications.

• Phase 1 study of StealthX exosome-based vaccine in collaboration with NIH.

Deramiocel DMD program and clinical results

• Deramiocel is an allogeneic CDC therapy, administered intravenously, with over 800 doses given in trials.

• HOPE-3 Phase 3 trial (n=106) met primary (PUL v2.0, p=0.03) and key cardiac secondary endpoints (LVEF, p=0.04), with all Type I error-controlled secondary endpoints achieved.

• Deramiocel BLA under FDA review, PDUFA action date set for August 22, 2026.

• Demonstrated 54% slowing of decline in skeletal muscle function and 91% slowing in cardiac function decline versus placebo.

• Favorable safety profile with no severe or fatal treatment-related adverse events in pivotal trial.