- Published: 16 June 2016
- Written by Editor
Capricor Therapeutics Reports Positive 12-Month Data from the DYNAMIC Clinical Trial
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.
|
Parameter |
Median Baseline (Range) |
Median Month 12 (Range) |
Median % Change (Range) |
p-value* |
|
Ejection Fraction (%) |
25 (17, 30) |
27.5 (17, 50) |
17.5 (-32.0, 66.7) |
0.02 |
|
LVESV (mL)** |
148.5 (92, 384) |
125 (53, 262) |
-14.3 (-57.6, 55.0) |
0.27 |
|
LVEDV (mL)** |
196.8 (128, 509) |
178 (70, 317) |
-4.2 (-56.4, 36.6) |
0.28 |
|
LSESV = left ventricular end-systolic volume; LVEDV = left ventricular end-diastolic volume |
|
* Based on signed rank tests of null hypotheses of percent change from baseline = 0. All tests were two-sided and were post hoc. N = 12 at baseline and at Month 12. |
|
** Decreased values indicate improvement. |
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).
About the DYNAMIC clinical trial
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. (CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class therapeutics. Capricor's lead candidate, CAP-1002 (allogeneic cardiosphere-derived cells), is in clinical development for the treatment of Duchenne muscular dystrophy-associated cardiomyopathy as well as adult cardiology indications. Capricor is also advancing its research of its exosomes technology for various therapeutic areas. Its Cenderitide product candidate, a dual natriuretic peptide receptor agonist, has shown promise for the outpatient management of heart failure. For additional information, visitwww.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 conduct, size, timing and results of discovery efforts and 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 offering, 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, and in its Registration Statement on Form S-3, as filed with the Securities and Exchange Commission on September 28, 2015 and in our Quarterly Report on Form 10-Q for the period ending March 31, 2016 as filed with the Securities and Exchange Commission on May 13, 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 has not yet been investigated in any clinical trial.
For more information, please contact:
Capricor Therapeutics, Inc.
AJ Bergmann, Vice President of Finance
+1-310-358-3200
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Investor Relations:
Argot Partners
Kimberly Minarovich
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