Capricor Therapeutics, Inc. (OTCBB: NLTXD) today announced that it has received notification from the National Heart Lung and Blood Institute (NHLBI) Gene and Cell Therapy (GST) Data Safety Monitoring Board (DSMB) that the 14-patient Phase I portion of the Company’s ALLSTAR Phase I/II clinical trial has met its safety endpoints and that the Company is now cleared to begin the Phase II portion of the ALLSTAR clinical trial.
Capricor’s ALLSTAR Phase I/II clinical trial is evaluating its CAP-1002 allogeneic cardiac-derived cell (CDC) product candidate in reducing infarct (scar) size in patients who have suffered a heart attack. The estimated 300-patient, double-blind, randomized, placebo-controlled Phase II portion of the ALLSTAR clinical trial is powered to detect a reduction in infarct size as measured by MRI in patients following a large myocardial infarction more than 30 days and less than 12 months prior to treatment with CAP-1002. The trial is sub-randomized into an ‘early’ group (30-90 days post-heart attack) and a ‘late’ group (91-365 days post-heart attack). Each group is powered sufficiently to provide both a safety and efficacy readout. Phase I of the ALLSTAR trial was funded in part by a grant received from the NIH. Phase II is being funded with the support of the California Institute for Regenerative Medicine.
“The fact that donor heart CDC cells were introduced into patients following a myocardial infarction and produced no adverse clinical effects in the 14-patient Phase I cohort marks a major milestone for the field of cardiac cell therapy,” said Timothy D. Henry, MD, Co-Principal Investigator of ALLSTAR and Chief of Cardiology at Cedars Sinai Medical Center in Los Angeles. “We are excited about entering the randomized portion of the trial and probing both the safety and effectiveness of these cardiac-derived allogeneic stem cells in a larger group of patients.”
Capricor CEO Dr. Linda Marbán stated, “Meeting the safety endpoints in the Phase I portion of the ALLSTAR trial is a giant leap forward for the field and for Capricor Therapeutics. By moving into the Phase II portion of this trial, we can now attempt to replicate the results of the previously conducted Phase Ib CADUCEUS autologous trial in a larger population using the significantly less expensive allogeneic cells.”
In the prospective, randomized Phase I Intracoronary Cardiosphere-Derived Cells for Heart Regeneration after Myocardial Infarction (CADUCEUS) clinical trial, 24 patients received CDC treatment. The trial provided early evidence for cardiac regeneration as autologous CDC patients saw scars left by myocardial infarction shrink over the course of 12 months and scientists observed a significant correlation between scar shrinkage and increases in viable tissue suggesting regeneration of new viable heart muscle. CDC patients also demonstrated improved regional function and trends pointing toward the reversal of adverse cardiac remodeling.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (CAPR), a publicly traded biotechnology company, is focused on the development of novel therapeutics to prevent and treat heart disease. The Company has two leading product candidates: CAP-1002 and Cenderitide. The Company was formed through the November 2013 merger between Capricor, Inc., a privately held company whose mission is to improve the treatment of heart disease by commercializing cardiac stem cell therapies for patients, and Nile Therapeutics, Inc., a clinical-stage biopharmaceutical company developing innovative products for the treatment of cardiovascular diseases. Capricor Therapeutics’ stock will begin trading under the symbol “CAPR” starting December 20, 2013. For additional information visit www.capricor.com.
CAP-1002, Capricor’s lead product candidate, is a proprietary allogeneic adult stem cell therapy for the treatment of heart disease. The product is derived from donor heart tissue. The cells are expanded in the laboratory using a specialized process and then introduced directly into a patient’s heart via infusion into a coronary artery using standard cardiac catheterization techniques.
CAP-1002 is currently not an approved product and is strictly for investigational purposes.
Cenderitide, a novel chimeric natriuretic peptide, is a first-in-class dual guanylyl cyclase receptor activator. Chronic dual receptor activation using a continuous subcutaneous infusion of Cenderitide is being studied to test whether its combination of tissue protective, renal protective and cardiac unloading effects will help stabilize the patient’s heart and kidney function, preventing the worsening of heart failure symptoms and re-admissions to the hospital. Cenderitide is under clinical development to treat heart failure patients during the post-acute heart failure (P-AHF) period, which is the 90-day period immediately after discharge from a hospitalization for acute decompensated heart failure. The P-AHF period is often associated with a worsening of heart failure symptoms or renal function and rates of re-admission and mortality can be as high as 40%.
Cenderitide is currently not an approved product and is strictly for investigational purposes.
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; future royalty streams, 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," "plans," "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 our business are set forth in our Form 10-K for the year ended December 31, 2012, as filed with the Securities and Exchange Commission on June 21, 2013, in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2013, as filed with the Securities and Exchange Commission on November 14, 2013, and in our Definitive Proxy Statement on Schedule 14A, as filed with the Securities and Exchange Commission on October 10, 2013. All forward-looking statements in this press release are based on information available to us as of the date hereof, and we assume no obligation to update these forward-looking statements.
Capricor Therapeutics, Inc.
AJ Bergmann, +1-310-358-3200