Capricor Therapeutics Reports Positive 12-Month Data from the DYNAMIC Clinical Trial

LOS ANGELES, Capricor Therapeutics, Inc. a biotechnology company focused on the discovery, development and commercialization of first-in-class therapeutics, today announced positive preliminary 12-month data from its DYNAMIC (Dilated cardiomYopathy iNtervention with Allogeneic MyocardIally-regenerative Cells) clinical trial, which evaluated CAP-1002 in patients with advanced heart failure. CAP-1002 is Capricor's investigational allogeneic cardiosphere-derived cell (CDC) therapy. 

In DYNAMIC, 14 patients with New York Heart Association (NYHA) Class III heart failure were treated with multi-vessel intracoronary infusion of CAP-1002 in four escalating dose cohorts. All patients received a one-time, triple-vessel infusion of CAP-1002 at doses ranging from 37.5 million to 75 million cells. Patients were followed for 12 months, and all echocardiographic studies were read by a core lab.

As observed at six months, directional improvements from baseline in key efficacy measures, including assessments of functional status, cardiac function and dimensions, and quality-of-life, were previously reported. Among these, statistically-significant (p<0.05) improvements were achieved in NYHA Class, left ventricular ejection fraction and end-systolic volume, and the Minnesota Living with Heart Failure (MLHFQ) score. These data were highlighted in a poster presentation at the 2015 American Heart Association Scientific Sessions.

For the 12 patients available for follow-up at one year, improvements from baseline in key cardiac function and dimensional indices were directionally maintained. Importantly, the change in median left ventricular ejection fraction from baseline to 12 months maintained its level of statistical-significance at six months (p=0.02 at both timepoints) and, on an absolute basis, continued to improve from six to 12 months.

Of the five NYHA Class III subjects who received the highest dose of CAP-1002, two subjects improved by two Classes (to Class I) and three improved by one Class (to Class II) at six months. At 12 months, three of these five subjects were assessed as Class I and two as Class II, demonstrating further improvement and indicating durability of the benefit of CAP-1002 on heart failure status for as long as 12 months following administration. This 75 million cell dose is currently being evaluated in Capricor's HOPE-Duchenne clinical trial in boys with Duchenne muscular dystrophy (DMD)-associated cardiomyopathy.

CAP-1002 infusion was well-tolerated in DYNAMIC. Two of the 14 patients, who were in the lower two of the four dose cohorts, died from progressive heart failure approximately one and three months prior to study conclusion.

"I am very impressed with all aspects of these preliminary data, which show important and consistent improvements in cardiac structure and function. Class III patients are known to follow an inevitable course of clinical deterioration with progression of their heart failure. To see sustained improvements out to 12 months as shown in the DYNAMIC study is significant, and may herald a new treatment paradigm for the more than one million patients with advanced heart failure," said Raj R. Makkar, M.D., Director, Interventional Cardiology and Cardiac Catheterization Laboratory, Cedars-Sinai Medical Center, and principal investigator of the trial.

Dr. Linda Marbán, president and chief executive officer of Capricor, commented, "We are very encouraged by the initial results demonstrated by CAP-1002 in this Class III population of advanced heart failure patients. Ejection fraction was improved and ventricular volumes were reduced, implying that CAP-1002 effected reverse remodeling. Importantly, the benefits of CAP-1002 were durable to 12 months. These data support the potential of Capricor's CDCs to alter the prognosis of patients with chronic, advanced heart failure for whom current therapeutic options are limited."

Dr. Marbán added, "Furthermore, the safety record of the triple-coronary infusion procedure used in DYNAMIC is supportive of the design of our randomized, 24-patient HOPE-Duchenne clinical trial, for which we expect top line data in the first quarter of 2017."

In addition to the HOPE-Duchenne trial, Capricor is also evaluating CAP-1002 in the ALLSTAR clinical trial in adults with cardiac dysfunction following a myocardial infarction (heart attack).

Source: Capricor Therapeutics, Inc.