Deramiocel (CAP-1002), a new experimental therapy for Duchenne muscular dystrophy (DMD), has shown effectiveness and safety in significantly slowing skeletal and heart muscle disease progression, according to a recently published press release.
Capricorn Therapeutics reported on the findings of a four-year ongoing study on deramiocel in patients with DMD. Deramiocel consists of a rare population of cardiac cells that have been shown to be effective in preserving skeletal and heart muscle function. It has received Orphan Drug Designation from both the U.S. Food and Drug Administration and the European Medicines Agency.
At the four-year mark of this study, researchers found that patients experienced positive changes. For example, patients had a median change of –0.5 points compared to the time when they first started treatment. In addition, patients who had a baseline left ventricular ejection fraction greater than 45% (a measure of heart health) had even greater clinical improvement.
Other findings from this study included a reduced progression in skeletal muscle disease. This was confirmed by measuring upper limb function; patients experienced a smaller average decline in their fourth year compared to their first.
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Combined, the study’s findings indicated that deramiocel was effective in alleviating disease progression over time, and was associated with cardiac stabilization. Furthermore, deriamocel had a favorable safety profile.
These findings are significant because cardiomyopathy (i.e., poor heart muscle health) is a leading cause of death in DMD.
“These four-year data reinforce the strength and durability of Deramiocel’s clinical benefit and favorable safety profile across both cardiac and skeletal muscle function,” Linda Marbán, CEO of Capricor Therapeutics, said. “With our [Biologics License Application] under priority review and several key regulatory steps now completed, we are executing with focus and urgency as we move toward potential approval.”
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