Dec 3 (Reuters) – Capricor Therapeutics said on Wednesday its cell therapy for a heart condition related to a rare muscular disorder met the main goal of a late-stage study, renewing hopes for its U.S. approval and sending shares up 18% in premarket trading.
In July, the U.S. Food and Drug Administration had declined to approve the therapy, deramiocel, as it sought additional data after saying the treatment did not meet efficacy requirements.
Capricor said it plans to use the new results in its response to the agency’s so-called “complete response letter.” It had previously agreed with the FDA that this trial could support approval of its treatment for cardiomyopathy in patients with Duchenne muscular dystrophy.
In the 106-patient trial, those given deramiocel saw a 54% slower decline in upper limb function versus placebo, while there was a 91% slower worsening of heart function, both statistically significant, the company said. The therapy was generally well tolerated.
More than 75% of participants had already been diagnosed with cardiomyopathy.
Duchenne, a rare genetic disorder that causes muscle degeneration, affects fewer than 50,000 people in the U.S., according to the National Institutes of Health. Heart complications are the leading cause of death in patients, typically in the twenties or thirties.
(Reporting by Mariam Sunny and Kamal Choudhury in Bengaluru; Editing by Shinjini Ganguli and Leroy Leo)
