Safety and efficacy of delpacibart zotadirsen in Duchenne muscular dystrophy mutations amenable to exon 44 skipping in the USA (EXPLORE44): a randomised, placebo-controlled, double-blind, phase 1/2 trial.

Delpacibart zotadirsen (del-zota) in USA participants with DMD44 produced mean exon 44 skipping increases of 36·6% at 5 mg/kg and 43·4% at 10 mg/kg.

• Why it matters: No approved exon-skipping therapies exist for Duchenne muscular dystrophy variants amenable to exon 44 skipping, leaving a gap for restoring dystrophin expression.
• What they did: In EXPLORE44, a randomized, double-blind, placebo-controlled phase 1/2 trial, del-zota was given intravenously to men and boys with DMD44 (3:1) in two dose cohorts with three infusions.
• The result: Del-zota showed an acceptable safety profile and increased dystrophin levels by 25·0% (5 mg/kg) and 25·6% (10 mg/kg), while placebo showed −0·7%, supporting phase 3 evaluation.

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