Capricor Reports Encouraging Results in its DYNAMIC Trial

Capricor Therapeutics, Inc., located in Beverly Hills, CA, has announced their six-month safety and adverse event data from a Phase I clinical trial of their CAP-1002 product for patients with advanced heart failure. This clinical trial is part of the DYNAMIC or which is short for Dilated cardiomYopathy iNtervention with Allogeneic MyocardIally-regenerative Cells trial whose goal is to evaluate CAP-1002 in patients with advanced heart failure.

CAP-1002 is Capricor’s lead investigational allogeneic, cardiosphere-derived cell (CDC) therapy. Allogeneic means that the cells come from someone other than the patient. The advantage of allogeneic cells is that they come from healthy donors whose cells have not been ravaged by old age or other conditions. These cells do not need to be matched to the patient’s immune system in this case because they help the heart through indirect means (see Tseliou E, et al., J Am Coll Cardiol. 2013 Mar 12;61(10):1108-19).  Cardiospheres are cells taken from the hearts of healthy patients that grow in culture as small balls of cells. Because these cells are derived from the heart and grow as spheres, they are called cardiospheres (see Cheng K, et al., JACC Heart Fail. 2014 Feb;2(1):49-61.).

Cardiospheres have been shown in small clinical trials (the CADUCEUS trial) to replace the heart scar with heart muscle (see Malliaras K, et al., Am Coll Cardiol. 2014 Jan 21;63(2):110-22).  Animal studies in rats showed similar results (see above).

CAP-1002 is an off-the-shelf “ready to use” cardiac cell therapy that consists of cells that come from donor heart tissue and is infused directly into a patient’s coronary artery during a catheterization procedure. This Phase I study is meant to determine if CAP-1002 is safe and effective in treating heart function and structure. In particular, Capricor scientists are interested in determining if CAP-1002 cells can decrease heart scar tissue and promote the growth of heart muscle. In doing so, this regenerative treatment might delay or even prevent the onset of heart failure. The US Food and Drug Administration has granted CAP-1002 an orphan drug designation for the treatment of cardiomyopathy associated with Duchenne Muscular Dystrophy.

Capricor’s Cardiosphere-Derived Cells are a unique therapeutic product that were created in the laboratory of company Co-Founder and Scientific Advisory Board Chairman, Dr. Eduardo Marbán, who is the Director of the Heart Institute at Cedars-Sinai Medical Center.

All patients in this trial have advanced heart failure and have progressed to a more advanced stage of the disease. Patients received CAP-1002 in up to three coronary arteries, which delivers the cells to the more of the diseased parts of the heart. Since these patients have significant fibrosis in all areas of the heart, this delivery system is optimal for these patients. Cell delivery will also utilize methods that do not stop blood flow, which will decrease patient discomfort during cell delivery.

The data from this trial, so far, comes from 14 patients who were diagnosed with either dilated cardiomyopathy or non-ischemic dilated cardiomyopathy. These patients have ejections fractions of 35% or less and are classified as New York Heart Association class III or Ambulatory Class IV heart failure.

The data collected to date show that CAP-1002 cells are safe and well tolerated and produced no adverse cardiac events at one month or six months after they were infused into the patient’s hearts. Although DYNAMIC was designed as a Phase I clinical trial that does not assess the efficacy of CAP-1002 cells, patients have also been tested for their subject wellbeing, exercise capacity (six-minute walk test), ejection fraction, and ventricular volumes.

According to the principal investigator Dr. Raj Makkar of Cedar-Sinai Medical Center, the data so far are rather encouraging, even beyond the positive safety data, since they are seeing “concordance between the clinical improvement and the physiological measurements of trends for improved ejection fraction and reverse re-modeling.” Dr. Makkar, however, emphasized that this clinical trial only tested a small cohort of patients, and these data must be confirmed in larger clinical trials.