Capricor Therapeutics Presents Positive 12-Month Results from DYNAMIC at TCT 2016

LOS ANGELES, Oct. 31, 2016 /PRNewswire/ -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other serious medical conditions, today announced detailed results from its DYNAMIC clinical trial of CAP-1002 in patients with advanced heart failure. The results were featured at a poster session held on Saturday, October 29^th at the 28^th Transcatheter Cardiovascular Therapeutics (TCT), the annual scientific symposium of the Cardiovascular Research Foundation, in Washington, D.C. The poster may be accessed at www.capricor.com. Capricor had announced top-line 12-month data from DYNAMIC in June. 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 secondary to dilated cardiomyopathy, and who demonstrated a left ventricular ejection fraction (LVEF) ≤35% despite maximal medical and device-based therapy, were treated with a single administration of CAP-1002. The study medication was given at one of four escalating dose levels (37.5 million, 50.0 million, 62.5 million, or 75 million cells) and was delivered directly to the three major coronary arteries via intracoronary infusion. Patients were then followed for 12 months for periodic safety and efficacy assessments.

Efficacy was evaluated at six and 12 months following CAP-1002 infusion. In pooled-dose analyses (N=10-13), measures of functional status and capacity, cardiac function and dimension, and quality-of-life broadly showed trends of improvement from baseline at both time points. At six months, statistically-significant improvements in NYHA Class (p=0.01) and in left ventricular ejection fraction (LVEF) (p=0.02), well as in the Minnesota Living with Heart Failure Questionnaire (MLHFQ) score (p=0.01), were demonstrated. The level of significance for LVEF improvement was maintained at twelve months (p=0.02).

Eleven of twelve (92%) patients assessed improved by at least one NYHA Class at six months (p=0.01). All five patients who received the highest dose of CAP-1002 improved. Among this group, at six months three improved by one class to Class II and two improved by two classes to Class I; at 12 months, two were at Class II and three were at Class I, demonstrating further improvement and indicating durability of the benefit of CAP-1002 on heart failure status for as long as 12 months following administration.

High Dose Group (N=5):

CAP-1002 was well-tolerated in DYNAMIC. There were no events recorded for the primary safety endpoint, which was a composite of five cardiac event types.

"Patients with NYHA Class III heart failure experience marked limitations in their physical activity, and suffer fatigue and shortness of breath even at less than ordinary levels of exertion. Although current options enable us to provide some support for their condition, the ability to therapeutically alter their progressive decline over the long-term is still lacking. Therefore, it is very encouraging to see evidence that CAP-1002 may be capable of modifying the underlying disease process in heart failure, the leading cause of hospitalization among U.S. citizens over 65 years of age," said Raj R. Makkar, M.D., Director, Interventional Cardiology and Cardiac Catheterization Laboratory, Cedars-Sinai Medical Center.

Dr. Linda Marbán, president and chief executive officer of Capricor, added, "We are very encouraged by both the safety as well as the signs of efficacy shown by CAP-1002 in DYNAMIC. This trial indicates, for the first time, that the cells can work in patients with longstanding heart failure, potentially providing entry into the large and poorly-met heart failure market. It also delineates a dose-response relationship that will inform the design of future trials in this and other indications. In fact, as in DYNAMIC, CAP-1002 at a dose level of 75 million cells was given by triple-coronary infusion in our ongoing HOPE trial in Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. We expect to report top-line six-month data from HOPE in the first quarter of 2017."

About DYNAMIC

The Phase I DYNAMIC (Dilated cardiomYopathy iNtervention with Allogeneic MyocardIally-regenerative Cells) clinical trial evaluated CAP-1002 (allogeneic cardiosphere-derived cells) in patients with advanced heart failure. The trial was open to patients with New York Heart Association (NYHA) Class III or ambulatory Class IV heart failure characterized by ischemic or non-ischemic dilated cardiomyopathy in which left ventricular ejection fraction was 35% or less. Suitable patients underwent sequential intracoronary infusion of CAP-1002 in up to three coronary territories. This triple vessel infusion was designed to broadly deliver cells to the myocardium, since patients with advanced heart failure have diffuse fibrosis throughout the heart. 

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapies for the treatment of cardiac and other serious medical conditions. Capricor's lead candidate, CAP-1002, is a cardiac cell therapy that is currently being evaluated for the treatment of heart disease associated with Duchenne muscular dystrophy and myocardial infarction (heart attack). Capricor is advancing its proprietary exosome product candidate, CAP-2003, for the treatment of ophthalmic disorders and is exploring other therapeutic areas. Capricor's portfolio also features Cenderitide, a dual natriuretic peptide receptor agonist, which may have application for the outpatient treatment of advanced heart failure and other potential indications. For additional information, visit www.capricor.com.

Cautionary Note Regarding Forward-Looking Statements

Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams, expectations with respect to the expected use of proceeds from the recently completed offering and the anticipated effects of the offerings, and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words "believes," "plans," "could," "anticipates," "expects," "estimates," "should," "target," "will," "would" and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor's business are set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2015, as filed with the Securities and Exchange Commission on March 30, 2016, in its Registration Statement on Form S-3, as filed with the Securities and Exchange Commission on September 28, 2015, and in its Quarterly Report on Form 10-Q for the quarter ended June 30, 2016, as filed with the Securities and Exchange Commission on August 15, 2016. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.

CAP-1002 and Cenderitide are Investigational New Drugs and are not approved for any indications. Capricor's exosomes technology, including CAP-2003, has not yet been investigated in any clinical trial.

For more information, please contact:

Corporate
Capricor Therapeutics, Inc.
AJ Bergmann, Vice President of Finance
+1-310-358-3200
abergmann@capricor.com 

Investor Relations
Argot Partners
Kimberly Minarovich
+1-212-600-1902
kimberly@argotpartners.com

Media
Argot Partners
Eliza Schleifstein
+1-917-763-8106
eliza@argotpartners.com

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SOURCE Capricor Therapeutics, Inc.