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Capricor Therapeutics’ stock skyrocketed 371% following positive results from its Phase III HOPE-3 trial of deramiocel in Duchenne muscular dystrophy (DMD). The trial enrolled 106 ambulatory and non-ambulatory patients, who received 150 million cells per infusion or placebo every three months for 12 months.
Results showed a 54% slowing of skeletal muscle disease progression (PUL v2.0) and a 91% reduction in progression of left ventricular ejection fraction decline, with a favorable safety profile consistent with prior studies. The therapy’s impact on both skeletal muscle and cardiomyopathy may improve long-term independence and survival, particularly benefiting non-ambulatory patients.
HOPE-3 data contrasts with the FDA’s earlier complete response letter for HOPE-2, which Capricor contested due to statistical analysis issues. The new results reinforce deramiocel’s durable benefits seen over more than 48 months in HOPE-2 and its open-label extension.
Capricor plans to present detailed HOPE-3 data at future scientific meetings and in a peer-reviewed journal.
07-12-2025