Dive Brief:
- Capricor Therapeutics announced on Feb. 16 it would terminate a license agreement with the Mayo Clinic for rights to natriuretic peptide receptor agonists, including the Los Angeles-based company's Phase 2 heart failure candidate cenderitide.
- "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 Capricor President and CEO Linda Marbán.
- Capricor anticipates it will read out top-line results next quarter for a Phase 1/2 trial evaluating CAP-1002 in people with Duchenne muscular dystrophy (DMD)-associated heart disease, and also plans to move a exosome-based therapeutic into the clinic.
Dive Insight:
Capricor, as it now stands, was formed in November 2013 through the merger of privately held Capricor and Nile Therapeutics.
Since entering into an amended license agreement around the time of the 2013 merger, Capricor had completed two small Phase 2 studies of cenderitide in people with chronic, stable heart failure. The trials were aimed at treating heart failure patients on an outpatient basis.
Progress from CAP-1002, however, led Capricor to expand its clinical development program for that candidate to include skeletal muscle aspects of DMD, as well as DMD-related cardiac complications.
In addition to the expected top-line results from the Phase 1/2 trial in DMD-associated heart disease, Capricor is planning a trial to evaluate CAP-1002's ability to improve peripheral and respiratory muscle in DMD patients.
The company is also accelerating its exosomes program, indicating in its Feb. 16 statement it would file an investigational new drug application for CAP-2003 sometime in the second half of the year.
Capricor's strategic shift may have been signaled by the lack of mention of cenderitide or the natriuretic peptide receptor program in a Jan. 25 letter from Marbán to shareholders.
CAP-1002 and CAP-2003 were similarly the focus of a company presentation at the recent BIO CEO conference.
Capricor believes it can "generate thousands of doses of CAP-1002 from each donor heart" through a commercial process being developed with Janssen, CFO Leland Gershall said in the presentation. Janssen is expected to decide on its license option for CAP-1002’s use in adult heart disease by mid-2017.