Capricor Therapeutics (CAPR) Completes Patient Enrollment in Phase II ALLSTAR Trial

October 5, 2016 7:02 AM EDT

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Capricor Therapeutics, Inc. (NASDAQ: CAPR) announced that its Phase II ALLSTAR clinical trial has completed patient enrollment. ALLSTAR (ALLogeneic Heart STem Cells to Achieve Myocardial Regeneration) is evaluating CAP-1002 (allogeneic cardiosphere-derived cells, or CDCs) in adults with cardiac dysfunction following a large heart attack (myocardial infarction, or MI).

"As we and others have shown, CAP-1002 possesses the ability to promote therapeutic regeneration in the injured heart, a powerful concept for the treatment of heart disease. In the CADUCEUS clinical trial, CDCs decreased scar size and increased viable tissue in the hearts of patients who had suffered a large heart attack. In ALLSTAR, not only are we studying a population similar to the one that delivered such astounding results in CADUCEUS (30 – 90 days post-MI), but we have also included patients that were 91 – 365 days post-MI to see if we could extend the indication window. We have also moved to an allogeneic platform from autologous cells. With the last patient in ALLSTAR having been dosed on September 30th, we expect to report top-line 12-month primary efficacy outcome results in the fourth quarter of 2017," said Linda Marbán, Ph.D., president and chief executive officer of Capricor. "In the nearer-term, based on our current projections, we expect to learn of Janssen's decision on its exclusive license option by next summer. We are very much looking forward to seeing the results of the ALLSTAR trial because they may show, for the first time in a Phase II clinical trial, that cells can reduce scar and potentially improve outcomes."

"We recently reported 12-month results from our DYNAMIC clinical trial in patients with advanced heart failure, a condition that affects over five million people in the U.S. alone. Patients who received CAP-1002 had consistent and durable improvements in overall functional status, cardiac function and dimensions, as well as quality-of-life. We believe these patients represent a large share of the adult cardiology market, and our results appear to validate the bioactivity of the cells in improving heart function," added Dr. Marbán.

The randomized, double-blind, placebo-controlled Phase II ALLSTAR trial enrolled adults with a history of MI within the prior 12 months that resulted in scar tissue replacing at least 15 percent of total left ventricular mass and that was accompanied by cardiac dysfunction as evidenced by left ventricular ejection fraction (LVEF) no greater than 0.45. One hundred forty-two subjects were randomized to the active or control treatment groups in a 2:1 ratio, respectively, of whom 134 received a single infusion of either CAP-1002 or placebo into the infarct-associated coronary artery. Patients were stratified according to time between infarct event and study medication infusion (Recent MI = 30 – 90 days; Chronic MI = 91 – 365 days). Following infusion, patients are to be followed for periodic evaluations over the course of one year. Patients in the trial were enrolled at 30 centers in the U.S. and Canada.

For the pre-specified primary efficacy analysis, ALLSTAR is powered to detect a reduction in infarct size in the CAP-1002 group, relative to the placebo group, at 12 months post-infusion. Infarct size will be assessed by magnetic resonance imaging (MRI). ALLSTAR will also explore additional efficacy parameters, such as ejection fraction and cardiac volumes, as well as evaluate the safety and tolerability of CAP-1002.

ALLSTAR generally follows the design of the randomized CADUCEUS trial, which demonstrated a statistically significant reduction in infarct size as well as a statistically significant increase in viable heart muscle tissue, as measured at six and 12 months, in patients treated with a single intracoronary infusion of CAP-1001 (autologous CDCs) as compared to standard-of-care controls (Makkar et al, Lancet 2012;379:895–904). In contrast to CAP-1001, which was used in CADUCEUS and which had been prepared on an individual basis from each patient's own heart tissue, CAP-1002, which is being used in ALLSTAR, was prepared in advance from donor heart tissue and was stored under controlled conditions.

Under the terms of Capricor's Collaboration Agreement and Exclusive License Option with Janssen Biotech, at any time until 60 days following delivery to Janssen of six-month interim data from the Phase II ALLSTAR trial, Janssen has the right to enter into an exclusive, worldwide license agreement for the development and commercialization of CAP-1002. If Janssen exercises its option rights, Capricor will be eligible to receive an upfront license fee and milestone-based payments totaling up to $325 million, as well as royalties on any commercial product sales.

The Phase II portion of ALLSTAR clinical trial is funded in part through the support of the California Institute For Regenerative Medicine. The Phase I portion of the trial was funded in part by the National Institutes of Health.

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