Capricor Reports Positive 6-month Results From Duchenne Muscular Dystrophy Trial

(RTTNews) - Capricor Therapeutics, Inc. (CAPR), a clinical-stage biotechnology company for cardiac and other medical conditions, announced Tuesday positive six-month results from the randomized phase I/II HOPE clinical trial in Duchenne muscular dystrophy or DMD.

The randomized Halt cardiomyOPathy progrEssion in Duchenne or HOPE clinical trial was designed to evaluate the safety and exploratory efficacy of CAP-1002.

CAP-1002 is an investigational candidate for the treatment of patients with DMD, a rare, life-threatening genetic disorder for which treatment options are limited.

The HOPE Trial was evaluating CAP-1002 in patients 12 years and older with DMD who had cardiomyopathy, or heart disease, secondary to DMD as evidenced by scar in four or more left ventricular segments as detected by late gadolinium-enhancement MRI.

In a 25-patient, randomized, single-dose, three-center clinical trial, patients treated with CAP-1002 demonstrated statistically-significant improvement compared to usual care controls in certain measures of cardiac and upper limb function, the company noted.

According to the firm, the drug candidate was generally safe and well-tolerated over the initial six-month follow-up period.

Capricor said it has submitted a meeting request to the U.S. Food and Drug Administration, or FDA, to discuss potential product registration strategies for CAP-1002 in the DMD indication.

Capricor intends to submit HOPE-Duchenne data to FDA and to request Breakthrough Therapy or Regenerative Medicine Advanced Technology, or RMAT, designations for CAP-1002.

John Jefferies, Principal Investigator of the HOPE Trial, said, "In HOPE, we saw potential effects in both the heart and skeletal muscle that appear quite compelling in an exploratory trial. These results clearly support the conduct of a confirmatory clinical trial in DMD to further evaluate the potential of CAP-1002. We look forward to an effective medication becoming available for people with this progressive and fatal disease, one that is poorly met by current options."

Linda Marbán, Capricor's president and chief executive officer, stated that the initial positive clinical results build upon a large body of preclinical data which illustrate CAP-1002's potential to broadly improve the condition of those afflicted by DMD.

Capricor shares closed Monday's trading at $3.09, up 2 percent. In the extended trading, shares gained 19.7 percent to $3.70.