Capricor to amend deramiocel BLA following adcomm blow

After a July advisory committee meeting raised concerns about its application, Capricor Therapeutics plans to amend its BLA for deramiocel, its cell therapy for Duchenne muscular dystrophy (DMD).

In an earnings call last week, Capricor CEO Linda Marbán, Ph.D., said the amendment will narrow the application’s focus to upper-limb skeletal muscle function — the primary efficacy endpoint of the phase 3 HOPE-3 trial — rather than cardiomyopathy. The company plans to include 24-month open-label extension data from HOPE-3, along with additional analyses of the existing data package, to support the refined indication.

“HOPE-3 was actually designed with a skeletal functional primary endpoint and was powered to assess efficacy and upper limb function,” Marbán said. “Importantly, the advisory committee was not asked to vote on whether they believed the data on the HOPE-3 primary efficacy endpoint could support approval of the product, nor whether the overall benefit-risk profile of deramiocel was favorable.”

In a closely watched adcomm meeting last month, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted 9-3 that available evidence did not support the effectiveness of Capricor’s deramiocel for the treatment of cardiomyopathy in patients with Duchenne muscular dystrophy. However, in a separate discussion on upper limb function, the committee's feedback was directionally supportive of the clinical evidence from the phase 3 HOPE-3 trial, including results on its primary endpoint, PUL 2.0.

The FDA has indicated that it is willing to review the amendment and will extend the August 22 PDUFA action date accordingly upon receiving it.