Capricor Regains Rights to CAP-1002 as Janssen Ends Collaboration – Genetic Engineering & Biotechnology News

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 Marbn, 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. Marbn 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. Marbn 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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Capricor Regains Rights to CAP-1002 as Janssen Ends Collaboration - Genetic Engineering & Biotechnology News