Capricor Therapeutics Presents Positive Data from the DYNAMIC Clinical Trial at 2015 American Heart Association Scientific Sessions

Capricor Therapeutics, Inc. CAPR, a biotechnology company focused on the discovery, development and commercialization of first-in-class therapeutics, today announced positive six-month safety and adverse event data from its ongoing DYNAMIC (Dilated cardiomYopathy iNtervention with Allogeneic MyocardIally-regenerative Cells) clinical trial evaluating CAP-1002 in patients with advanced heart failure. The data were highlighted in a poster presentation at the 2015 American Heart Association (AHA) Scientific Sessions taking place November 7-11, 2015 in Orlando, FL. CAP-1002 is Capricor's lead investigational allogeneic, cardiosphere-derived cell (CDC) therapy. Multi-vessel intracoronary infusion of CAP-1002 in subjects with dilated cardiomyopathy was shown to be safe in this study with no major adverse cardiac events reported at one month or at six months post-infusion. Though this trial was intended as an early safety study, the six-month data demonstrated encouraging and congruent preliminary efficacy signals in multiple parameters, including subjective well being, exercise capacity, ejection fraction and ventricular volumes. Table 1 Parameter Median Baseline (Range) Median Month 6 (Range) Median % Change (Range) p-value* Ejection Fraction (%) 24 (17, 30) 28 (18, 41) +14.3 (-20, 65) 0.02 LVESV (mL)** 169 (92, 384) 131 (79, 218) -20.4 (-47, 35) 0.03 LVEDV (mL)** 208 (128, 509) 175 (109, 293) -15.2 (-44, 37) 0.08 6 minute walk test (m) 375 (180, 525) 400 (225, 525) 0.0 (-13, 113) 0.68 VO2 Max (m/kg/min) 13.9 (4.4, 22.4) 13.7 (10.6, 28.5) 3.0 (-25, 141) 0.46 Minnesota Living with Heart Failure (MLHFQ)** 48 (7, 105) 18 (1, 69) -38.1 (-98, 79) 0.01 Table 2 NYHA (Class) n (%) Baseline n (%) Month 6 p-value* Class I** 0 (0) 2 (17) 0.006 Class II** 0 (0) 9 (75) Class III** 14 (100) 1 (8) * Based on t-tests or signed rank tests of null hypotheses of absolute change from baseline = 0. For NYHA, null hypothesis was probability of improvement = 0.5. All tests were two-sided and were post hoc. Analyses were based only on subjects for whom complete data were available in time for the AHA presentation data cutoff. N = 13 or 14 at baseline, 11 or 12 at Month 6 (depending on parameter). ** Decreased values indicate improvement. "I am absolutely delighted with the preliminary safety data and efficacy signals of the DYNAMIC trial in highly symptomatic patients with severe cardiac dysfunction. These data justify further investigation of the CDC cells in larger clinical trials of patients with advanced heart failure of multiple etiologies. Heart failure is a malignant disease and one of the greatest unmet needs in all of medicine" said Raj Makkar, M.D., Director, Interventional Cardiology and Cardiac Catheterization Laboratory, Cedars-Sinai Medical Center, and principal investigator of the trial. Dr. Makkar added, "what is specifically encouraging in this data set beyond the safety is the concordance between the clinical improvement and the physiologic measurements of trends for improved ejection fraction and reverse remodeling. These data are based on a small cohort and need to be confirmed in larger trials, but we do know that advanced heart failure patients do not, in general, spontaneously improve." Dr. Linda Marbán, Chief Executive Officer of Capricor, commented "these data from the DYNAMIC trial continue to support our prior published findings that CAP-1002 has great potential to improve the outcomes for patients with advanced heart failure. Currently, there are more than 6 million Americans suffering from this disorder. Capricor has programs in place for advanced heart failure in adults, as well as in the cardiomyopathy of Duchenne Muscular Dystrophy (DMD) in young men. The FDA has granted CAP-1002 orphan status for DMD cardiomyopathy and the company is planning to initiate the upcoming HOPE trial for this indication. We look forward to significant progress in our quest to provide a new class of therapeutics for a large spectrum of patients suffering with cardiac disease and to address the very large market opportunities." Presentation details: Title: Dilated cardiomYopathy iNtervention with Allogeneic MyocardIally-regenerative Cells (DYNAMIC): Six Month Safety and Exploratory Efficacy Results Date & Time: Monday, November 9, 2015, from 5:30-6:45PM ET Location: Poster Hall, A2, BS Poster #: M 1041 Session ID: APS.06.02. Stem/Progenitor Cells II