Shares of Capricor Therapeutics more than doubled after the biotechnology company said the Food and Drug Administration has agreed to review new data supporting its investigational Duchenne muscular dystrophy therapy, deramiocel, giving the company a potential path forward following a rocky advisory committee review last month.
Capricor said the FDA indicated it will extend the current Prescription Drug User Fee Act target action date of August 22, 2026, once the company formally submits a planned amendment to its Biologics License Application. The amendment seeks a narrower indication focused specifically on upper-limb skeletal muscle function, rather than the broader cardiomyopathy indication the company had originally pursued.
The company said it is finalizing the timing of the submission and plans to include 24-month open-label extension data from its pivotal Phase 3 HOPE-3 clinical trial, along with additional analyses of its existing data package, to bolster the amended application.
The update follows a difficult stretch for Capricor. In late July, the FDA's Cellular, Tissue and Gene Therapies Advisory Committee voted 9 to 3 against the effectiveness of deramiocel for treating cardiomyopathy in Duchenne muscular dystrophy patients, a non-binding recommendation that nonetheless raised significant doubts about the therapy's approval prospects. Capricor has argued the committee's voting question addressed a narrower indication than the one it had originally proposed and did not fully capture the therapy's overall benefit-risk profile.
Notably, in a separate discussion during that same advisory committee meeting, feedback on deramiocel's effect on upper-limb function was described as directionally supportive of the HOPE-3 trial data, including on the therapy's primary endpoint, a measure known as PUL 2.0. That more favorable feedback appears to be the basis for Capricor's pivot toward pursuing a narrower, skeletal-muscle-focused indication rather than abandoning the program after the cardiomyopathy vote.
Deramiocel is an allogeneic cardiac-derived cell therapy designed to preserve both cardiac and skeletal muscle function in patients with Duchenne muscular dystrophy, a rare and severe genetic disorder that predominantly affects boys and has limited treatment options. The therapy previously received a Complete Response Letter from the FDA in July 2025 before the agency resumed review following submission of additional HOPE-3 data earlier this year. The therapy has also been published in The Lancet, a peer-reviewed medical journal, which the company has cited as external validation of its trial design and findings.
If ultimately approved, deramiocel would become one of the first therapies to address skeletal muscle manifestations of Duchenne muscular dystrophy through a cell-therapy approach, and the company has said it expects to be eligible for a Priority Review Voucher upon approval given the therapy's orphan drug and rare pediatric disease designations.
Friday's rally reflects investors' view that a narrower approval pathway may meaningfully improve deramiocel's odds of reaching the market, even as the broader cardiomyopathy indication faces a steeper regulatory road following the advisory committee's negative vote.