In a recent press release, Capricor Therapeutics shared that deramiocel demonstrated positive topline results in their HOPE-3 clinical trial, improving cardiac and skeletal muscle function in patients with Duchenne muscular dystrophy (DMD).
Earlier this year, Capricor submitted a Biologics License Application to the U.S. Food & Drug Administration (FDA), which, if approved, would allow the company to market deramiocel. However, the FDA denied the initial application, citing the need for more evidence of its efficacy.
Capricor plans to include the new HOPE-3 data in a response to the rejection. “We believe these pivotal study results, in addition to the evidence from the HOPE-2 and HOPE-2 OLE studies, position us to address the clinical issues in the Complete Response Letter received earlier this year, consistent with prior FDA guidance that HOPE-3 results should be sufficient to support regulatory approval,” said Dr. Linda Marbán, chief executive officer at Capricor Therapeutics.
The study randomized 106 patients with DMD to receive either deramiocel or a placebo via an intravenous infusion every three months for one year. All participants, who had an average age of 15, also received corticosteroids for the duration of the study.
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Compared to those receiving the placebo, those taking deramiocel experienced a 54% reduction in progression of upper limb weakness. Patients in the treatment group also demonstrated a 91% slowing in cardiac function decline, measured via left ventricular ejection fraction.
“The HOPE-3 study is the first-ever Phase 3 trial in a largely non-ambulatory population with DMD to successfully meet its primary endpoint,” explained Dr. Craig McDonald, the primary investigator of the HOPE-3 trial.
Given that cardiomyopathy is one of the major complications among individuals with DMD, these findings suggest that deramiocel may have the potential to extend not only life expectancy but also overall quality of life.
Deramiocel consists of a specialized type of heart cell known as cardiosphere-derived cells, which are provided by healthy donors. When administered to patients, these cells release molecules that reduce fibrosis and inflammation. To date, over 250 publications have studied cardiosphere-derived cells, according to Capricor.
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