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Capricor Therapeutics (CAPR) announced on Wednesday that its experimental cell therapy Deramiocel achieved both its primary and key secondary goals in the pivotal Phase 3 trial for Duchenne muscular dystrophy (DMD).
The HOPE-3 study, involving 106 participants across 20 U.S. centres, delivered strong signals of benefit for muscle and heart function.
HOPE-3 was a randomized, double-blind, placebo-controlled trial that administered Deramiocel infusions every three months over a 12-month period. The average age of enrolled boys and young men was around 15, and all continued their standard corticosteroid therapy.
At baseline, about 90% were on cardiac medications, and over 75% already had clinical cardiomyopathy. Following the study results, Capricor’s stock surged by over 440% on Wednesday morning.
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