Neurology Publishes Study Reporting Significant Improvements in Duchenne Muscular Dystrophy

Neurology, an international peer-reviewed journal, on Wednesday published the results of the HOPE-Duchenne clinical trial that evaluated the safety and efficacy of a single intracoronary dose of Capricor Therapeutics’ (NASDAQ:CAPR) novel cell therapy candidate, CAP-1002, in boys and young men in advanced stages of Duchenne muscular dystrophy.

As quoted in the press release:

The Phase I/II, randomized, controlled, open-label trial found that CAP-1002 demonstrated improvement in cardiac muscle function and reduction in cardiac scarring that were statistically-significant and sustained improvement of skeletal muscle functions in patients with Duchenne muscular dystrophy, a fatal genetic disease with limited treatment options. The HOPE-Duchenne trial also found no serious safety issues, according to the study published in the January 23, 2019, online issue of Neurology, the medical journal of the American Academy of Neurology.

“CAP-1002 is the only therapy so far that has shown reduction in myocardium scar and improvement of localized cardiac function in late stage Duchenne associated cardiomyopathy. Interestingly, it also showed improvement in upper limb strength,” said Michael Taylor, M.D., Ph.D., the study’s corresponding author and Director of Cardiac MR research at Children’s Healthcare of Atlanta. “These findings suggest that CAP-1002 could be an option for older patients who have scarring in the heart as a result of the underlying disease. As many of these patients are in later stages of the disease, the improvement in upper limb strength is important in order to maintain independence.”

Click here to read the full press release.