Capricor Therapeutics is ready to follow through with their plans to develop CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD) following a successful meeting with the U.S. Food and Drug Administration (FDA).
The meeting was held to discuss the drug’s current safety and efficacy data, as well as the use of a unique outcomes measure for an upcoming pivotal clinical trial(s).
DMD is the result of a patient not having a properly functional dystrophin protein, which assists in keeping muscles intact. Boys with this progressive muscle condition have been known to lose walking abilities as early as age 10, and as the disease progresses, cardiovascular problems also become a concern. It’s typical of DMD patients to die in their 20s due to heart disease.
While other companies are seemingly focused on developing drugs to attenuate the skeletal muscle degradation associated with the condition, Capricor has remained focused on the heart problems that develop in DMD.
CAP-1002, a cardiac cell therapy to treat heart disease associated with DMD, contains allogeneic cardiosphere-derived progenitor cells that exert potent immuno-modulatory activity.
Linda Marbán, Ph.D., president and chief executive officer of Capricor, said in a news release
, "The cells in CAP-1002 release exosomes that are immunomodulatory and exert anti-inflammatory, anti-fibrotic, and anti-apoptotic effects. By ameliorating the myocyte damage induced by dystrophin mutations, our product has been demonstrated to preserve and improve the structure and function of dystrophic skeletal muscle. Its differentiated mechanism of action supports its potential to be a standalone therapy as well as an adjunct to dystrophin-modulating agents."
Based on minutes released from the meeting, the FDA is willing to:
accept the use the Performance of the Upper Limb (PUL) as the primary efficacy endpoint for clinical studies intended for CAP-1002’s Biologics License Application (BLA); and,
accept the existing nonclinical safety and efficacy database to support submission of an Investigational New Drug application (IND) to clinically evaluate repeat intravenous administration of CAP-1002.
In the ongoing Phase 1/2 HOPE-Duchenne study, CAP-1002 has shown that PUL was an effective measurement tool and the drug was well tolerated.
Moving forward, the company plans to begin a randomized, double-blind, placebo-controlled Phase 2 clinical trial of intravenous, repeat-dose CAP-1002 in the second half of 2017.
For more updates on CAP-1002 and other potential DMD therapies, follow Rare Disease Report