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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.
On Stocktwits, retail sentiment around the stock improved to ‘extremely bullish’ from ‘bullish’ territory the previous day amid ‘extremely high’ message volume levels.
On the primary measure, upper limb performance (PUL v2.0), those receiving Deramiocel showed a roughly 54% slower rate of deterioration than those receiving placebo. On the key secondary cardiac measure, left ventricular ejection fraction (LVEF), the treatment group saw a 91% preservation in heart function relative to placebo.
“A nearly 54 percent slowing of skeletal muscle disease progression is extraordinary in Duchenne and directly linked to maintaining independence and quality of life in the most severely affected patients with greatest unmet need.”
-Craig McDonald, Distinguished Professor at UC Davis Health
Capricor plans to submit the HOPE-3 data in response to the regulatory agency’s prior concerns to secure approval.
CAPR stock has gained over 103% in 2025 and over 69% in the last 12 months.
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