Capricor Therapeutics has regained rights to develop and commercialize its lead candidate CAP-1002 (allogeneic cardiosphere-derived cells), after Janssen Biotech opted not to license the cardiac cell therapy, ending a three-and-a-half-year collaboration between the companies.

Capricor received $12.5 million upfront from Janssen, and could have received up to another $325 million, under an exclusive license option agreement signed by the companies in January 2014 to develop Caprcor’s cell therapy program for cardiovascular applications, including CAP-1002.

CAP-1002 has been in clinical development for the treatment of heart disease associated with Duchenne muscular dystrophy (DMD), as well as two other adult cardiology indications—postmyocardial infarction (for which it has reached Phase II) and advanced heart failure (Phase I).

Capricor said in March that Janssen’s decision on its license option would follow delivery of a six-month administrative analysis of results from the Phase II ALLSTAR trial, designed to assess CAP-1002 in patients with myocardial infarction.

In May and again yesterday, Capricor acknowledged that the analysis “showed a low probability that CAP-1002 would achieve the primary endpoint of scar size reduction, as measured by late gadolinium-enhanced magnetic resonance imaging, at 12 months.”

However, Capricor said a potential beneficial effect from CAP-1002 may yet be shown on remodeling, a key factor lined to chronic heart failure. The company cited nonsignificant group differences in certain prespecified secondary endpoints, including changes in left ventricular end-systolic and end-diastolic volumes (LVESV and LVEDV).

Duchenne Trial Top-Line Results

In April, Capricor reported more positive six-month top-line results from the Phase I/II Halt cardiomyOPathy progrEssion in Duchenne (HOPE-Duchenne) clinical trial, designed to assess the safety and preliminary efficacy of CAP-1002 in 25 subjects. Capricor cited statistically significant improvements in systolic thickening of the inferior wall of the heart and in the function of the middle and distal upper limb in CAP-1002 patients compared with control patients receiving usual care.

The HOPE-Duchenne trial also found differences in several other cardiac and skeletal muscle measures, including cardiac scar, that Capricor said were consistent with a treatment effect.

“Over the last few years, and during the term of the Janssen option period, we believe that significant value for our CAP-1002 asset has been created through the demonstration of clinical proof-of-concept to treat DMD, and also from the progress that has been made toward the development of a commercial-scale manufacturing process for the cells,” Capricor president and CEO Linda Marbán, Ph.D., said in the statement.

As a result, she said, Capricor will continue to develop CAP-1002, either alone or with a new collaboration partner. After announcing HOPE results, Capricor discussed potential product registration strategies for this indication at a recent meeting with FDA officials.

Subject to regulatory approval, Dr. Marbán said, Capricor expects to begin a randomized, double-blind, placebo-controlled clinical trial of repeat administrations of intravenous CAP-1002 in boys and young men with DMD in the second half of this year, subject to regulatory approval.

“We look forward to providing an update on our clinical development plans in DMD very shortly,” Dr. Marbán said. “Although Janssen's decision removes a potential corporate partner for Capricor, this decision also resolves uncertainty concerning the scope of the license for CAP-1002 and provides Capricor the freedom to enter into new licensing and/or business development opportunities around this promising therapeutic candidate.”

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