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Deramiocel Significantly Slows Upper Limb Functional Decline in Duchenne Muscular Dystrophy: Skeletal Muscle Outcomes from the Phase 3 HOPE-3 Trial (LS2.003)
Abstract   Peer reviewed

Deramiocel Significantly Slows Upper Limb Functional Decline in Duchenne Muscular Dystrophy: Skeletal Muscle Outcomes from the Phase 3 HOPE-3 Trial (LS2.003)

Susan Iannaccone, Craig McDonald, Aravindhan Veerapandiyan, Arun Varadhachary, Leigh Ramos-Platt, Kathryn Gambetta, Russell Butterfield, Mindy Leffler, kati maharry, Nathaniel J. Hogan, …
Neurology, Vol.106(11_Supplement_1)
06/09/2026
DOI: 10.1212/WNL.0000000000217921

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Abstract

Objective Confirmation of musculoskeletal efficacy of deramiocel in DMD using clinic and home-based assessments Background Deramiocel is a novel cellular therapy previously shown to benefit skeletal muscle function in Duchenne muscular dystrophy (DMD) in a phase 2 randomized placebo-controlled trial. The mechanism is pleiotropic, likely involving extracellular vesicles and soluble factors that confer immune-modulatory, anti-inflammatory, and anti-fibrotic properties. This phase-3 study assessed efficacy and safety of deramiocel in late-ambulatory and non-ambulatory DMD subjects. Design/Methods HOPE-3 was a randomized, double-blind, placebo-controlled Phase 3 study enrolling 106 patients with DMD. The primary endpoint was mean percent change from baseline in the Performance of Upper Limb (PUL2.0) Total Score at Month 12. Secondary endpoints included the PUL 2.0 mid-level domain and the Duchenne Video Assessment (DVA) score of Eating 10 Bites, a measure of sustained feeding performance in the home environment, blinded to timepoint. Results At Month 12, mean percent change from baseline in PUL2.0 Total Score was −3.86 in the deramiocel group versus −8.41 in the placebo group, a difference of 4.55 (p = 0.029) which corresponds to 54% slowing of disease progression over 12 months. Mid-level PUL2.0, function closely related to independence, differed by 8.12 (p = 0.008), a 60% slowing of progression. Deramiocel also significantly reduced deterioration in independent feeding as measured by the Eat 10 Bites score at 12 months (p = 0.0176) for which data were available on 90 patients. Deramiocel was safe and well tolerated. Conclusions Treatment with deramiocel resulted in a clinically meaningful and statistically significant slowing of upper limb disease progression over 12 months. The magnitude of effect, concordance across standardized functional scales and home-based real-world video assessments support deramiocel as a therapy with the potential to modify the course of skeletal muscle decline and preserve independent function in patients with DMD.

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