Capricor Therapeutics stock surged more than 300 percent Wednesday after the company reported that its Duchenne muscular dystrophy therapy, Deramiocel, met key goals in a Phase three trial involving non-ambulatory patients.
Deramiocel Shows Strong Functional Gains in HOPE-3
Capricor said Deramiocel slowed disease progression, showing a fifty-four percent reduction in skeletal muscle decline based on the PUL v2.0 assessment. Shares closed at $28.05 after briefly nearing $40 amid heavy morning trading.
The HOPE-3 study enrolled 106 participants at 20 U.S. sites. Patients received intravenous Deramiocel or placebo for 12 months while remaining on stable corticosteroid therapy. The company said safety outcomes were consistent with previous trials.
“These results demonstrate clinically meaningful preservation of function in a population with urgent needs,” Capricor Chief Executive Linda Marbán said. “We believe HOPE-3 offers clear evidence to support our regulatory path.”
More than 75 percent of participants had cardiomyopathy, and nearly 90 percent were taking cardiac medications. Capricor Therapeutics said those real-world factors underscore the relevance of the findings, particularly the therapy’s ability to maintain heart function.
Company Looks to Address FDA Feedback
The study showed stable left ventricular ejection fraction, an outcome cardiologists view as critical because cardiac failure remains the leading cause of death in Duchenne patients.
Dr. Aaron Feldman, a pediatric cardiologist unaffiliated with the company, said the results merit close regulatory attention. “Any therapy that stabilizes cardiac function in Duchenne marks a major advance,” he said. “Cardiac decline accelerates in later stages, so maintaining function can extend quality of life.”
The company said HOPE-3 findings align with earlier data from the HOPE-2 study and its open-label extension, reflecting consistent therapeutic benefit over nearly a decade of development. Capricor Therapeutics plans to present results to the Food and Drug Administration as it works to address concerns raised in a recent Complete Response Letter.
“We intend to use the strength of these data to move quickly in discussions with regulators,” Marbán said. “The consistency across studies supports Deramiocel’s long-term potential.”
Market Responds to Clearer Approval Path
Analysts said Wednesday’s rally reflected both the clinical significance of the results and market expectations of an expedited FDA review. “The magnitude of the stock move indicates investor confidence in a clearer approval path,” said biotech analyst Morgan Tate. “Regulators will still scrutinize the data, but this positions Capricor Therapeutics far stronger than before.”
Capricor said Deramiocel aims to preserve independence for severely affected patients with limited mobility. The therapy improves skeletal and cardiac function, two areas where current treatments offer limited benefit.
The company did not provide a timeline for submitting additional data to the FDA but said preparation is underway. Analysts expect further updates in early 2026.
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