MDA 2025: Deramiocel linked to sustained arm function preservation
Researchers analyzed data from HOPE-2, extension studies and gap period
Capricor Therapeutics’ investigational therapy deramiocel, formerly CAP-1002, which is being considered for approval in the U.S., continued to show an ability to preserve upper limb function in boys and men with Duchenne muscular dystrophy (DMD) over the long term.
That’s according to analyses of about five years worth of data spanning the Phase 2 HOPE-2 trial (NCT03406780), its open-label extension study (NCT04428476), and a gap period without treatment.
The findings were presented in a Capricor-sponsored poster titled, “Treatment with Deramiocel in DMD: Evidence of Long-Term Stabilization in Performance of Upper Limb Function Compared to Matched External Comparators,” at the Muscular Dystrophy Association’s (MDA) Clinical & Scientific Conference, being held this week in Dallas and virtually.
“For patients and families battling DMD, time is muscle,” Linda Marbán, PhD, CEO of Capricor, said in a company press release. “This data … reinforces that deramiocel is not just slowing the disease — it is rewriting the trajectory of what’s possible for those living with Duchenne.”
The U.S. Food and Drug Administration (FDA) is reviewing an application for deramiocel’s approval for DMD patients with heart disease, having placed it under priority review this month. A decision is expected by the end of August.
If deramiocel is approved, NS Pharma, a subsidiary of Nippon Shinyaku, will handle its marketing.
Effects of deramiocel in Duchenne MD
People with DMD lack dystrophin, a protein that helps protect muscle cells against contraction-related damage. This leads to progressive muscle damage and weakness, affecting the skeletal muscles needed for movement, along with the heart and muscles for breathing.
Deramiocel contains cardiosphere-derived cells, a type of immature heart cell harvested from healthy donors. The cells secrete tiny vesicles called exosomes, which contain signaling molecules that help inhibit inflammation and scar tissue buildup, modulate the immune system, and promote blood vessel growth, all activities that should promote muscle regeneration.
HOPE-2 enrolled 20 boys and men with DMD, ages 10 and older, who were in relatively advanced disease stages and most were unable to walk. They were randomly assigned to receive deramiocel or a placebo, given via infusions into the bloodstream once every three months for a year.
The results showed deramiocel was associated with improved arm and heart function compared with the placebo after a year.
After about a one-year gap without treatment, those who’d completed HOPE-2 could enter the OLE, where they are all receiving long-term deramiocel treatment. Seven participants initially assigned to the placebo group and six given deramiocel in the main study entered the OLE.
Arm function was evaluated using the Performance of the Upper Limb (PUL 2.0) measure. The participants who’d been on a placebo in HOPE-2 exhibited a mean 3.6-point decline, that is, worsening, in PUL 2.0 scores during the main trial and saw a further 3.7-point decline during the gap period. Those who’d received deramiocel in the main HOPE-2 study exhibited a mean 1.5-point PUL 2.0 decline in the initial trial and a 2.8-point decline during the gap.
The relatively slower rate of decline during the gap period for those first given deramiocel relative to those first on a placebo, “highlights the potential disease-modifying effect of deramiocel, as patients who were previously on treatment and later discontinued continue to show an attenuated rate of decline,” the researchers wrote.
Sustained benefits to upper limb function
Consistent with previous interim analyses, data over three years in the OLE demonstrated the sustained benefits of deramiocel on upper limb function.
Across all 13 patients treated in the OLE, the mean decline in PUL 2.0 scores was 3.46 points per year. That equated to a 52% slowing of disease progression relative to a similarly aged external comparator group receiving standard-of-care steroid therapy, who saw a mean decline of 7.19 points a year.
The effects of deramiocel on disease progression appeared to deepen over time, with PUL 2.0 scores declining, on average, by 1.8 points in the first year of the OLE, 1.2 points in the second year, and 1.1 points in the third year.
Deramiocel was well tolerated with no new safety signals identified. Capricor previously indicated that heart function was also preserved during the OLE.
“We have continued to see durable, long-term benefits, enabling patients to maintain skeletal muscle and cardiac function when decline was once inevitable,” Marbán said.
Ongoing analyses of the OLE will help confirm the benefits of deramiocel beyond three years, the researchers said. Meanwhile, the ongoing Phase 3 HOPE-3 trial (NCT05126758) is further evaluating the investigational therapy in DMD patients, ages 10 and older, with impaired skeletal muscle function. Top-line data are due by year’s end.
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