Capricor Therapeutics, Inc. Reports Third Quarter 2015 Business & Financial Highlights

LOS ANGELES, Nov. 12, 2015 /PRNewswire/ -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), a biotechnology company focused on the discovery, development and commercialization of first-in-class therapeutics, today provided a business and financial update for the third quarter ended September 30, 2015.

Third Quarter and Recent Operational Highlights

• Capricor presented positive six-month DYNAMIC (Dilated cardiomYopathy iNtervention with Allogeneic MyocardIally-regenerative Cells) clinical trial results of its cardiosphere derived cell (CDC) therapy, CAP-1002, for the treatment of advanced heart failure.
• The Company appointed Deborah Ascheim, M.D., as its Chief Medical Officer.  Dr. Ascheim is a heart failure cardiologist with significant experience directing national and international clinical trials.

"We have made significant advances for the CAP-1002 clinical development program this quarter," said Linda Marban, Ph.D., Chief Executive Officer of Capricor.  "Data from the DYNAMIC clinical trial presented at the American Heart Association Scientific Sessions earlier this week confirmed the bioactivity of our CDC therapy as seen in earlier clinical trials.  In addition, the multi-vessel intracoronary infusion technique used in the DYNAMIC trial was safe and well tolerated and will be used in our HOPE-Duchenne clinical trial, which is now open for enrollment.  We are encouraged by the concordance of the clinical data with the physiologic outcomes in the previous CDC clinical trials, CADUCEUS, ALLSTAR and now DYNAMIC, and are hopeful that the reduction of scarring in damaged hearts will also translate into positive outcomes for DMD-related cardiomyopathy patients in the HOPE-Duchenne clinical trial."

Clinical Development Program Update

CAP-1002 Program

DYNAMIC Clinical Trial Results
The Phase I DYNAMIC trial is evaluating CDCs (CAP-1002) in patients with advanced heart failure. The trial enrolled 14 patients with either ischemic or non-ischemic dilated cardiomyopathy with left ventricular ejection fraction (LVEF) of 35% or below and New York Heart Association (NYHA) Class III or Ambulatory Class IV heart failure. Suitable patients underwent sequential intracoronary infusion of CAP-1002 in up to three coronary territories.  The triple vessel infusion was designed to deliver cells to wide areas of myocardium since patients with advanced heart failure have significant fibrosis in all areas of the heart.  The Phase I trial is being funded in part through a grant of approximately $3.0 million from the National Institutes of Health (NIH).

Results of the DYNAMIC trial were presented on Monday, November 9, 2015 at the American Heart Association Scientific Sessions. 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.

HOPE-Duchenne Clinical Trial Initiation
The HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) clinical trial is a randomized open-label usual care controlled multi-center study evaluating the safety and preliminary efficacy of CAP-1002 in up to 24 male patients with DMD who have significant cardiac involvement.  Patients will receive CAP-1002 in all three coronary arteries using the "non-stop flow technique" safely used in the DYNAMIC trial. The first six subjects randomized will be 18 years or older. Pending a safety review, the trial will then be open to boys 12 years of age or older with cardiomyopathy secondary to DMD, with left ventricular scar involving at least four cardiac segments and ejection fraction equal to 35% or greater.  Cardiomyopathy is currently the leading cause of death in patients with DMD, having recently supplanted respiratory causes as a result of treatment improvements for that aspect of the disease. 

The HOPE-Duchenne clinical trial is open for enrollment. The Company expects to report topline data from HOPE-Duchenne by the first quarter of 2017.

ALLSTAR Clinical Trial
Capricor is now enrolling ALLSTAR Phase II, a clinical trial evaluating the effectiveness of CAP-1002 in reducing infarct (scar) size in patients who have suffered a myocardial infarction (heart attack) more than 30 days and less than 12 months prior to treatment with CAP-1002. The trial is a randomized, double-blind, placebo-controlled trial powered to detect a reduction in infarct size as measured by MRI. It is Capricor's intent to perform an interim analysis, which, if successful, could result in a reduction in the number of patients necessary for achieving statistical significance for the primary endpoint, a reduction in infarct size at 12 months post infusion.  This trial is intended as a proof-of-concept trial to validate the results of CADUCEUS using an allogeneic product while also looking for potential efficacy in patients.  Results from the CADUCEUS trial showed that patients treated with autologous CDCs, CAP-1001, had a significant reduction in infarct size and an increase in healthy heart muscle mass.

ALLSTAR is open for enrollment at approximately 24 centers in the United States. If the interim analysis plan is implemented, we expect the trial to be fully enrolled in the third quarter of 2016 and the Company expects to report six-month data in the first quarter of 2017. 

Janssen Biotech, Inc., a wholly-owned subsidiary of Johnson & Johnson, has entered into an exclusive option to license CAP-1002, which can be exercised anytime until 60 days after the six-month ALLSTAR Phase II data is available.

Phase II of the ALLSTAR study is being funded in part through the support of the California Institute for Regenerative Medicine.

Cenderitide (CD-NP) Program
Cenderitide belongs to a class of drugs called natriuretic peptides. Preclinical and clinical data have shown that the natriuretic peptide class can act on multiple disease processes that play a role in negative outcomes associated with heart failure. A Phase II clinical study of Cenderitide was initiated in 14 patients with heart failure in January 2015 and completed enrollment in March 2015. The drug was tolerated and there were no significant adverse events. Capricor has decided to conduct an additional small study to further assess the safety and efficacy of higher dose levels of Cenderitide. This study will assess the safety and tolerability, pharmacokinetics profile and pharmacodynamic response to increasing dose levels of Cenderitide. Following these studies, Capricor will determine whether to conduct additional clinical studies to further assess the safety and efficacy of this product candidate.

Exosome Program
Exosomes are nano-sized, membrane-enclosed vesicles or "bubbles" that are filled with RNAs and proteins, which, when released, send messages to neighboring cells to regulate cellular functions. Exosomes act as a transport vehicle out of the cell for microRNA, other fragments of genetic material and proteins that act as messengers between cells, ultimately providing regulatory function for many cell processes, including inflammation, angiogenesis, programmed cell death (apoptosis) and scarring.

Capricor's preclinical exosome program is progressing and the Company expects to identify the first clinical target and clinical development plan in the first half of 2016 and plans to initiate a Phase I clinical trial before the end of 2016.

Results of Operations for the Quarter Ended September 30, 2015
For the quarter ended September 30, 2015, the Company had cash, cash equivalents and marketable securities of approximately $17.2 million. The increase in cash equivalents and marketable securities from the approximately $8.0 million reported as of December 31, 2014 is a result of approximately $17.0 million raised in two private placements of Capricor common stock, less cash used to fund operations.

For the quarter ended September 30, 2015, the Company reported a net loss of approximately $2.9 million, or $0.18 per basic and diluted share, compared to a net loss of approximately $1.5 million, or $0.13 per basic and diluted share, for the same period in the prior year. Research and development expenses increased to approximately $3.2 million in the quarter ended September 30, 2015, compared to approximately $2.0 million for the same period in the prior year. The increase was primarily due to increased expenses related to ongoing or planned clinical trials. General and administrative expenses increased to approximately $1.0 million in the quarter ended September 30, 2015, compared to approximately $0.8 million for the same period in the prior year. The increase was primarily due to increases in compensation costs and non-cash stock-based compensation.

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