Pharma Focus Asia

Capricor Therapeutics Provides Update on ALLSTAR Trial

Saturday, May 13, 2017

Capricor Therapeutics, Inc. announced that a pre-specified administrative interim analysis performed on six-month follow-up data from the ALLSTAR Trial, an ongoing randomized, double-blind, placebo-controlled, 142-patient Phase II clinical trial of CAP-1002 (allogeneic cardiosphere-derived cells) in adults who have experienced a large heart attack with residual cardiac dysfunction, has demonstrated a low probability (futility) of achieving a statistically-significant difference in the 12-month primary efficacy endpoint of percent change from baseline infarct size as a percent of left ventricular mass, measured by cardiac magnetic resonance imaging (MRI). Capricor will continue to perform analyses of the cumulative ALLSTAR data to better understand the basis for this outcome. At six months, a near-statistically-significant (p=0.05) reduction of mean end-diastolic volume, as well as a trend of reduction of mean end-systolic volume, were seen in the CAP-1002 treatment group. There was no notable difference between treatment groups with respect to the change in ejection fraction. There were no safety signals in the CAP-1002 treatment cohort.

Raj Makkar, M.D., Associate Director, Interventional Technologies in the Heart Institute at Cedars-Sinai Medical Center and Co-Principal Investigator of the ALLSTAR Trial, said, "We are disappointed that the ALLSTAR six-month data did not demonstrate evidence of scar size improvement with CAP-1002, given the robust findings demonstrated on this measure in the randomized Phase I CADUCEUS clinical trial of cardiosphere-derived cells in a similar patient population. We believe it is important to note that the observed improvements in scar size in the placebo group are markedly inconsistent with the well-established natural history of this disease process. It is certainly possible that, for a variety of reasons, the greater number of sites involved in the conduct of ALLSTAR contributed to an increase in variability seen in the scar measurements as determined by MRI."

Tim Henry, M.D., Director, Division of Cardiology in the Heart Institute at Cedars-Sinai Medical Center and Co-Principal Investigator of the ALLSTAR Trial, added, "We are encouraged to see reductions in left ventricular volume measures in the CAP-1002 treated patients, an important indicator of reverse remodeling of the heart. These findings support the biological activity of CAP-1002."

Following Capricor's recent report of positive six-month data on clinical measures of skeletal muscle performance and cardiac biomarkers in the ongoing randomized 25-patient Phase I/II HOPE Trial of CAP-1002 in boys and young men with Duchenne muscular dystrophy (DMD), the Company plans to initiate enrollment into a randomized, double-blind, placebo-controlled, repeat-dose clinical trial of intravenous CAP-1002 in DMD in the second half of 2017, subject to regulatory approval. This anticipated trial will primarily evaluate skeletal (non-cardiac) muscle function.

"The lack of a clear difference in the change in scar size from baseline to six months between the active and control groups in the interim observations from ALLSTAR was unexpected. These results diverge from the consistent and extensive record of activity observed with our cell technology in the setting of cardiac fibrosis as demonstrated by both preclinical and clinical studies, and we hope to gain an understanding of the factors that led to these observations through the conduct of further analyses," said Linda Marbán, president and CEO of Capricor.

"Although we are disappointed, the favorable safety profile demonstrated by CAP-1002 in ALLSTAR supports the prospect of its chronic, repeat administration in patients with Duchenne muscular dystrophy. Also, the potent anti-inflammatory properties of CAP-1002 may be well-suited to mitigate DMD progression, for which chronic inflammation is believed to play a causative role," added Dr. Marbán.

Capricor plans to reduce the scope of its operations, including the size of its workforce, in order to focus its financial resources primarily on its DMD program.

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