Capricor Therapeutics has announced additional analyses and new functional outcomes data from the Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne. Deramiocel is the company’s investigational cell therapy for the treatment of Duchenne cardiomyopathy.
Cardiac MRI analyses demonstrated Deramiocel’s impact on the heart, with data showing reduction in fibrotic segments in patients treated with Deramiocel versus placebo, measured by late gadolinium enhancement (LGE). According to Capricor, in patients with baseline cardiomyopathy, Deramiocel demonstrated even greater treatment effect on cardiac function.
Additionally, a composite measure including Performance of Upper Limb (PUL v2.0), left ventricular ejection fraction (LVEF), and Patient Global Impression of Severity (PGI-S), demonstrated a statistically significant overall treatment benefit favoring Deramiocel.
This update comes after Capricor shared on March 10, 2026, that the U.S. Food and Drug Administration (FDA) has lifted the July 2025 Complete Response Letter and resumed review of the company’s Biologics License Application (BLA) for Deramiocel. The FDA has assigned a new Prescription Drug User Fee Act (PDUFA) target action date of August 22, 2026, the date by which the agency plans to complete its review and decide whether to approve the therapy.
PPMD is encouraged by this update from Capricor, and we are hopeful that the submission of this latest positive HOPE-3 data to the FDA will support a thorough review of Deramiocel’s BLA. We will continue to share additional information with the community as soon as updates become available.
Read Capricor’s press release here.
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