On October 14, 2026, Entrada Therapeutics, Inc. filed a Form 8-K announcing positive topline results from the first cohort of its Phase 1/2 ELEVATE-45-201 clinical study. The study is evaluating ENTR-601-45, an investigational therapy for Duchenne Muscular Dystrophy (DMD), in patients with mutations amenable to exon 45 skipping.
The ELEVATE-45-201 study is a global, randomized, double-blind, placebo-controlled trial. The Multiple Ascending Dose (MAD) Part A portion of the study enrolled eight participants with DMD, aged four to 20, who were randomized 3:1 to receive ENTR-601-45 at a dose of 5 mg/kg or placebo. Participants received three intravenous doses on days one, 43, and 85.
The primary objectives of the study were met, with results indicating favorable safety and tolerability at the 5 mg/kg dose. The most common treatment-emergent adverse event was headache, occurring in five events among two participants. One participant experienced two serious adverse events—vomiting and pyrexia—which subsequently resolved. Importantly, there were no study discontinuations, and all participants have progressed to the Phase 2 open-label portion of the study.
Regarding efficacy, the study demonstrated a mean increase of 0.10% in dystrophin production from baseline in participants receiving ENTR-601-45, compared to a 0.02% decrease in the placebo group. A mean increase of 0.15% in exon skipping was observed in the treatment group versus a 0.04% decrease in the placebo group. Based on the age of the participants and baseline function, no observable functional benefit was expected or observed after the initial three doses.
Entrada reported that plasma ENTR-601-45 exposures were consistent with predictions based on juvenile non-human primate data. The company noted that modeled plasma exposures provide confidence in a significant increase in dystrophin expression at higher doses. Cohort 2 enrollment is complete, and dosing at 10 mg/kg is ongoing. The company expects to report data from Cohort 2 in the first half of 2027, with data from Cohort 3 (up to 15 mg/kg) to follow.
