Capricor Therapeutics, Inc. (CAPR), on Monday, announced positive data from its ongoing HOPE-2 open-label extension trial, demonstrating the long-term efficacy of Deramiocel for the treatment of Duchenne Muscular Dystrophy.
DMD is a devastating genetic disorder that leads to progressive muscle weakness and eventually death, typically due to cardiomyopathy. There are currently limited treatment options available.
The long-term trial data showed that patients treated with deramiocel over three years experienced a 52 percent slower decline in muscle function, as measured by the Performance of the Upper Limb or PUL 2.0 score, compared to a cohort-matched external comparator group.
The average decline in the deramiocel group was 3.46 points, compared to a 7.19-point decline in the comparator group.
Notable findings include:
-- Patients treated with deramiocel showed a reduction in disease progression, with the annual decline in PUL 2.0 scores decreasing over the three years.
--- The data suggests that deramiocel may slow disease progression even in the absence of ongoing treatment.
--- Deramiocel was well tolerated with no new safety concerns identified.
Deramiocel, an allogeneic cardiac-derived cell therapy, has demonstrated potent immunomodulatory and antifibrotic actions in both preclinical and clinical studies, preserving cardiac and skeletal muscle function in DMD.
Capricor has sought FDA approval for deramiocel to treat the cardiomyopathy associated with DMD, with a decision expected on August 31, 2025.
Currently, CAPR is trading at $13.52 up by 2.18 percent on the Nasdaq.
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