Caprock Mining Corp

CAP-1002, an investigational cell therapy, has shown to slow declines in upper limb and heart function in Duchenne muscular dystrophy (DMD) patients over two years in the HOPE-2 study. A Phase 3 trial is ongoing, with potential FDA approval pathways being explored. CAP-1002 aims to benefit late-stage DMD patients by slowing skeletal and cardiac muscle function decline, demonstrating disease-modifying potential and a favorable safety profile.

CAP-1002, cardiosphere-derived cells, shows long-term safety and efficacy in DMD patients, attenuating disease progression by 64% over 24 months. It also benefits cardiac function, with 67% of patients showing improvement in EF% after ~3 years, indicating potential in modifying DMD's underlying disease.

Capricor Therapeutics' CAP-1002 trial for Duchenne muscular dystrophy continues after positive DSMB review. Plans to discuss expedited FDA approval in 2024. CAP-1002, derived from heart cells, aims to improve arm and heart function in DMD patients. Recruitment ongoing; top-line data expected late 2024.

2023 saw significant progress in DMD treatment with Sarepta Therapeutics’ Elevidys approval, yet it benefits only a small patient group. Challenges remain in therapy's long-term efficacy, safety, and expanding access. Research continues for broader, more effective treatments, emphasizing the need for innovative strategies and precise outcome measures.

CAP-1002, an allogeneic cell therapy from donor heart tissue, aims to reduce inflammation, muscle degeneration, and enhance muscle regeneration, potentially prolonging muscle function. A Phase 3 study, Hope-3, is recruiting, sponsored by Capricor Therapeutics.

Capricor Therapeutics aligned with the FDA on the HOPE-3 trial design for CAP-1002, targeting Duchenne muscular dystrophy (DMD) treatment. The trial aims for 58 participants, with topline results expected in late 2024 and a BLA submission in 2025. Positive 24-month data from HOPE-2-OLE study showed CAP-1002's potential benefits in cardiac and skeletal function for DMD patients.