Capricor Therapeutics today announced that researchers found that repeat dosing of the company’s proprietary cellular therapy yields an increase in exercise performance in a disease model of Duchenne muscular dystrophy, the mdx mouse. The company has previously presented data showing that single doses lead to significant improvement in treadmill run times.

In an abstract presentation at the 11th Annual Neuromuscular Translational Research Conference in Cambridge, England, researchers reported that while the therapy, CAP-1002, is recognised by the immune system, its low immunogenic profile and immunomodulatory capabilities allowed it to be administered multiple times without significant safety issues. They also reported that repeat dosing was shown to sustain the effect of the therapeutic.

“These important findings were the foundation for the design of the HOPE-2 clinical trial we will be initiating shortly,” said Linda Marbán, Ph.D., Capricor president and chief executive officer. “The research found improvements in absolute force in soleus (leg) and in the diaphragm of a mouse model of Duchenne muscular dystrophy, the mdx mouse. Additionally, the researchers observed decreased fibrosis in skeletal and cardiac muscles after three administrations of CAP-1002.”

A previous clinical trial, the HOPE-Duchenne trial, found a single intracoronary dose of CAP-1002 was generally safe, well tolerated and demonstrated significant and sustained signals of improvement in cardiac and skeletal muscle function in boys and young men in advanced stages of Duchenne muscular dystrophy.

The HOPE-2 clinical trial will test the safety and efficacy of repeat doses of CAP-1002 in boys and young men with Duchenne muscular dystrophy, a devastating and fatal genetic disorder with limited treatment options and no cure. Up to 84 boys and young men with Duchenne muscular dystrophy will be enrolled in the Phase 2, randomized, double-blind, placebo-controlled trial. HOPE-2 will test repeat doses of CAP-1002 in participants with advanced stages of Duchenne muscular dystrophy.

“CAP-1002 is one of the very few clinical initiatives to focus on helping boys and young men that are in later stages of the disease process and are unable or close to being unable to ambulate any longer,” said Dr. Marbán. “We are eager to get the HOPE-2 trial underway as we believe it may potentially be a registration trial and because we have seen the potential for improvements in muscle function in both pre-clinical studies and in our earlier HOPE-Duchenne clinical trial.”

Capricor has been granted the RMAT and orphan disease designations by the U.S. Food and Drug Administration (FDA). These designations will enable the company to work closely with the FDA in finalising the regulatory approval pathway for CAP-1002 and to receive expedited FDA reviews.

“CAP-1002 also has the potential to work synergistically with gene and other therapies for Duchenne muscular dystrophy,” she said. “While these other therapies have the potential to restore dystrophin expression and sustain muscle function, there will still be significant inflammation and fibrosis which can offset the restorative effects. CAP-1002 may be able to work with these therapies because its primary mechanism of action is immunomodulatory, meaning it can help balance inflammation in this chronic inflammatory disease.”

For more information about the HOPE-2 trial, please visit www.HOPE2Trial.com.

The poster presented in Cambridge is available on the Events & Presentations section of Capricor's website.

About CAP-1002

CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a unique population of cells that contains cardiac progenitor cells. CAP-1002 has been shown to exert potent immunomodulatory activity and stimulate cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials.

Capricor's Press Release