Capricor Therapeutics today announced positive topline results from its pivotal Phase 3 HOPE-3 clinical trial evaluating Deramiocel, the company’s investigational cell therapy for the treatment of Duchenne muscular dystrophy.
According to Capricor, the study met both its primary skeletal muscle endpoint, Performance of Upper Limb (PUL v2.0) and the key secondary cardiac endpoint, left ventricular ejection fraction (LVEF%), each achieving statistical significance. Data indicates that treatment with Deramiocel resulted in a nearly 54% slowing of skeletal muscle disease progression compared to placebo. Results also demonstrated preservation of cardiac function, with a significant preservation of LVEF% over 12-months on Deramiocel compared to placebo.
Capricor noted that it plans to submit a response to the U.S. Food and Drug Administration’s (FDA) July 2025 Complete Response Letter incorporating HOPE-3 data, consistent with FDA guidance that HOPE-3 results should be sufficient to support regulatory approval.
These HOPE-3 topline results are encouraging for the Duchenne community, particularly non-ambulatory individuals for whom there are limited therapeutic options. Evidence of slowed decline in upper limb function and preservation of cardiac function is promising—having novel cardiac therapies that can slow disease progression and provide more time for patients is incredibly meaningful, given the central role cardiomyopathy plays in long-term outcomes for people with Duchenne.
We look forward to seeing the full results as they become available and remain encouraged by continued progress toward new therapeutic options for families living with Duchenne.
Read Capricor’s press release here.
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