The investigational allogeneic cell therapy deramiocel helps mitigate the progression of upper limb disease in young patients with Duchenne muscular dystrophy (DMD), as shown in the pivotal phase 3 HOPE-3 study.
“HOPE-3 met all its primary and secondary efficacy endpoints,” reported one of the study authors Dr Aravindhan Veerapandiyan from Arkansas Children's Hospital in Little Rock, Arkansas, US, in a late-breaking science session at the annual AAN meeting.
Compared with those who received placebo, deramiocel-treated patients saw a significantly smaller percent change in the primary endpoint of the Performance of Upper Limb (version 2 [PUL 2.0]) total score at 12 months (mean, –3.86 vs −8.41), with a difference of 4.55 (p=0.029). [AAN 2026, abstract 6188]
The change seen with deramiocel vs placebo corresponded to a 1.2-point improvement in PUL 2.0 total score and a 54-percent reduction in upper limb disease progression over 1 year, which Veerapandiyan described as a clinically meaningful benefit previously unseen in other DMD clinical trials.
Results for additional functional outcomes similarly favoured deramiocel over placebo.
Mid-level PUL 2.0 score at 12 months, which assessed elbow function, differed by 8.12 percent between the two treatment groups (p=0.008), which translated to a 65-percent slowing of progression.
Furthermore, the Duchenne Video Assessment “Eat 10 Bites” score (1.68 vs 9.52; p=0.018), which measured sustained feeding performance in a home setting, represented an 83-percent reduced deterioration in independent feeding.
“In addition to upper limb function, deramiocel provided treatment benefits on cardiac function and fibrosis,” Veerapandiyan said.
Compared with placebo, the allogeneic cell therapy preserved left ventricular ejection fraction (LVEF; treatment difference, 2.4 percent; p=0.041) and slowed late gadolinium enhancement (treatment difference, –3.0 segments; p=0.022).
These findings suggest that deramiocel may mitigate the decline in activities of daily living, including the ability to transfer, turn, and eat independently, according to Veerapandiyan. Morever, “the preservation of cardiac function is likely to translate into survival benefit.”
HOPE-3
Conducted at 20 sites across US, HOPE-3 included 106 DMD patients at least 10 years of age who were late or nonambulatory, had a PUL 2.0 entry item score of ≥2, and an LVEF of ≥35 percent. All patients were on background stable corticosteroid therapy.
The patients were randomly assigned to receive quarterly infusions of deramiocel 150 million cells (n=54) or placebo (n=52).
Deramiocel has acceptable tolerability and safety, Veerapandiyan noted.
The frequency of treatment-emergent adverse events (TEAEs) was 94.3 percent with deramiocel and 82.7 percent with placebo, of which 83 percent and 36.5 percent, respectively, were related to the investigational product or administration procedure. Overall, most TEAEs were mild or moderate in severity.
Serious TEAEs occurred in 1.9 percent of patients treated with deramiocel and 9.6 percent of those who received placebo. None of the TEAEs led to death.
Deramiocel is under active FDA review for the treatment of DMD, supported by the findings from the pivotal HOPE-3 study. FDA is expected to issue its decision by 22 August 2026.