Overview
- Topline Phase 3 results showed deramiocel met two main goals by preserving upper limb function and heart pumping ability in Duchenne muscular dystrophy.
- The randomized HOPE-3 study enrolled 106 largely non-ambulatory adolescents, dosing 150 million cells intravenously every three months for 12 months.
- Capricor reported that nearly 54% of patients had slower skeletal muscle decline and about 91% showed a treatment effect on cardiomyopathy.
- An investigator said HOPE-3 is the first Phase 3 trial in a largely non-ambulatory Duchenne population to meet its primary endpoint, and a cardiology expert highlighted significant preservation of left ventricular ejection fraction.
- Shares jumped sharply on the news, trading as high as $40 before settling around $25 by late morning, as the company prepares an FDA response incorporating the new data.