Capricor to seek deramiocel’s FDA approval for DMD heart disease
Looking to expand label for skeletal muscle myopathy after potential approval
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Capricor Therapeutics will soon begin filing an application seeking U.S. approval of its cell therapy deramiocel for cardiomyopathy, a disease of the heart’s muscle, in people with Duchenne muscular dystrophy (DMD).
Following recent meetings with the U.S. Food and Drug Administration (FDA), Capricor will file a rolling application, meaning the company can submit parts of it as they become ready, instead of waiting to submit the entire thing all at once. Capricor will start this process in October, with a full submission expected to be complete by the end of the year.
Capricor also has eyes on a potential label expansion for the use of deramiocel for treating DMD muscle disease in the rest of the body — or skeletal muscle myopathy — down the line, but that wouldn’t be until after a submission and approval for cardiomyopathy, for which there are currently no approved treatments.
‘Novel, first-in-class treatment to patients in need’
“Based on the strength of our cardiac data … we are seeking approval for the cardiomyopathy associated with DMD and will look to expand the label for skeletal muscle myopathy post-approval,” Linda Marbán, PhD, Capricor’s CEO, said in a company press release. “We believe this is the best path forward to potential approval, allowing us to bring this novel, first-in-class treatment to patients in need in the most expeditious manner.”
Findings from the ongoing Phase 3 HOPE-3 trial (NCT05126758) are intended to back an eventual application for a label expansion in skeletal muscle myopathy.
To best support that effort, Capricor is waiting to analyze trial results until it can combine findings from two separate groups of study participants, delaying previously indicated plans to announce top-line results from one of the groups by year’s end.
Previously known as CAP-1002, deramiocel contains what’s called cardiosphere-derived cells from a healthy donor. These immature heart cells release beneficial signaling molecules which have shown to modulate the immune system, prevent scar tissue buildup, and have regenerative properties.
In DMD patients, where cardiomyopathy is a leading cause of death, the therapy is expected to be able to improve both cardiac and skeletal muscle function through these signaling activities, overall slowing disease progression. Deramiocel is infused directly into the bloodstream once every three months.
The regulatory filing in the U.S. will be based on cardiac data from the Phase 2 HOPE-2 trial (NCT03406780) and its ongoing open-label extension (NCT04428476), which involved boys and young men, 10 and older, with advanced DMD.
Trial data show deramiocel significantly improved heart function
Findings from the main HOPE-2 trial showed deramiocel significantly improved heart function and lowered levels of a marker of heart damage relative to a placebo after a year of treatment.
After an up to 1.5-year pause in treatment, participants could join the extension, where all are receiving deramiocel.
Data presented earlier this year showed after three years of treatment in the extension study, deramiocel had stabilized cardiac function, compared to a steady decline observed in an external group of untreated patients seen at two U.S. hospitals (a natural history comparator).
Findings from both parts of the study also showed deramiocel could improve upper limb function relative to the placebo or the natural history comparator group.
The main goal of HOPE-3, which enrolled a similar patient population, is to evaluate the effects of deramiocel on arm and hand function after a year, with secondary goals related to heart health and other functional evaluations.
Two groups of patients (Groups A and B) will be treated with deramiocel and a matching placebo manufactured at two different facilities, but are otherwise expected to be the same. Although initial plans were to analyze data from Group A first, analyses will now be delayed until the two groups can be analyzed together.
Deramiocel previously received orphan drug, regenerative medicine advanced therapy, and rare pediatric drug designations in the U.S., which are regulatory statuses intended to help speed its clinical development.
“We want to extend our appreciation to the patients, their families and advocates who continue to work with us and to the FDA for their commitment to accelerating treatments for DMD,” Marbán said.
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