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Capricor Therapeutics Announces Late-Breaking HOPE-3 Data at the 2026 MDA Conference Demonstrating Significant Functional Benefits of Deramiocel for Duchenne Muscular Dystrophy
Reduced progression of myocardial fibrosis measured by LGE on cardiac MRI (p=0.022)Significant improvement in LVEF versus placebo in patients with baseline
About this update from Capricor Therapeutics, Inc.
[{"type":"text","content":"Reduced progression of myocardial fibrosis measured by LGE on cardiac MRI (p=0.022)Significant improvement in LVEF versus placebo in patients with baseline cardiomyopathy (p=0.017)Global Statistical Test (GST) composite endpoint showed significant overall treatment benefitDuchenne Video Assessment (DVA) showed approximately 83% slowing of disease progression (p=0.018), consistent with mid-level (elbow) PUL v2.0 resultsDeramiocel BLA currently under FDA review with a PDUFA target action date of August 22, 2026 SAN DIEGO, March 12, 2026 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced additional analyses and new functional outcomes data from the Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne muscular dystrophy (DMD), which were presented yesterday at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Orlando, Florida. MDA Presentation Highlights: Cardiac MRI analyses demonstrated Deramiocel’s impact on cardiac structure in patients with DMD. Evaluation of late gadolinium enhancement (LGE), a marker of myocardial fibrosis, showed a significant reduction in fibrotic segments in patients treated with Deramiocel versus placebo, corresponding to a three-segment treatment difference at 12 months (p=0.022). The presence of LGE reflects replacement of viable myocardium with fibrotic tissue and is associated with progressive cardiac dysfunction and heart failure risk in DMD cardiomyopathy.In patients with baseline cardiomyopathy, Deramiocel demonstrated an even greater treatment effect on cardiac function. In this subgroup, treatment resulted in a 3.3 percentage-point improvement in LVEF versus placebo, corresponding to greater than 100% attenuation of expected cardiac decline (p=0.017).A Global Statistical Test (GST), a patient-level composite 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 (p=0.017). This composite integrates multiple clinically meaningful domains of disease, reflecting how patients feel and function.Additional functional outcomes evaluating hand-to-mouth acti...