Capricor Therapeutics is dropping further development of its Phase II-stage heart failure drug Cenderitide and is terminating its license agreement with the Mayo Clinic for the natriuretic peptide receptor agonist program. “Our decision to return these rights is a strategic move as we prioritize our efforts to advance our core cell and exosome-based therapeutic development programs,” said Linda Marbán, Ph.D., Capricor president and CEO.

Capricor said it expects to reach key clinical milestones with both the cell and exome therapy programs during 2017. Topline data from the Phase I/II HOPE clinical study with lead cell therapy candidate CAP-1002 (allogeneic cardiosphere-derived cells) is expected during Q2 2017. The trial is evaluating CAP-1002 in patients with Duchenne muscular dystrophy (DMD)-associated heart disease. The firm said it is separately planning a clinical trial, anticipated to start during 2017, to investigate whether CAP-1002 therapy can boost skeletal muscle function in men and boys with DMD.

“We are also committing increased attention to our exosomes program, and we expect to file an IND application for CAP-2003 (cardiosphere-derived cell exosomes) in the second half of this year,” added Dr. Marbán. Capricor is developing CAP-2003 for the potential treatment of ophthalmic disorders, including ophthalmic graft-versus-host disease (oGvHD).

CAP-1002 is an allogeneic cardiac cell therapy product manufactured from donor heart tissue. The treatment has been granted orphan drug designation by the FDA for the treatment of DMD. Capricor and partner Janssen Biotech are developing a commercial manufacturing process for CAP-1002 as part of their potentially $337.5 million collaboration, established in January 2014, to develop the cell therapy program for cardiovascular applications. In May of last year, Capricor received a $3.8 million grant from the California Institute for Regenerative Medicine to support the Phase I/II HOPE study.

CAP-2003 exosomes are obtained from CAP-1002 cardiosphere-derived cells (CDCs). The exosome technology is being developed under a 2014 license agreement with the Cedars-Sinai Medical Center. In October 2016, Capricor was granted up to $4.2 million from the NIH to evaluate CAP-2003 exosome therapy for treating hypoplastic left heart syndrome. The previous month the firm received a $2.4 million grant from the U.S. Department of Defense to establish a commercial manufacturing platform for CAP-2003.

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