Entrada Therapeutics Announces Topline Data from ELEVATE-44-201 Clinical Trial

Entrada Therapeutics announced topline data from Cohort 1 of the double-blind, placebo-controlled, multiple ascending dose portion of the Phase 1/2 ELEVATE-44-201 clinical study. ELEVATE-44-201 is a clinical study of ENTR-601-44 in ambulatory participants ages four to 20 with a confirmed mutation in the DMD gene amenable to exon 44 skipping. Study participants in Cohort 1 were randomized 3:1 to receive three doses of 6 mg/kg of ENTR-601-44, the lead investigational product in Entrada's Duchenne muscular dystrophy franchise, or placebo. Muscle biopsies were performed at the time of screening and six weeks after the last dose. The average age of treated participants in the Cohort 1 study was 9.3 years old with a mean age of disease onset of 2.2 years. Per protocol, all participants were ambulatory and all were on a stable dose of steroids. Baseline dystrophin in both the placebo and treatment population was also lower than that reported in competitive exon 44 skipping clinical studies. This is also notable, as treatment response generally correlates with higher baseline dystrophin levels. The results demonstrated a favorable safety and tolerability profile with no reported serious adverse events (SAEs) and no adverse events leading to discontinuation from the study. Markers of kidney function were normal. All study participants in Cohort 1 have now progressed to the open-label, Phase 2 portion of the study, where they will receive six additional doses of 6 mg/kg of ENTR-601-44. Additional study participants are now being dosed in Cohort 2, in which they will receive three doses of 12 mg/kg of ENTR-601-44 or placebo. The Company expects to report results from the Cohort 1 open-label study and Cohort 2 MAD study by year-end 2026, with data from Cohort 3 to follow. Favorable safety and tolerability with ENTR-601-44 at the 6 mg/kg dose. All treatment emergent adverse events were mild to moderate. No reported SAEs and no AEs leading to discontinuation from the study. The most common AE was headache. Markers of kidney function including eGFR, Cystatin C and magnesium were within normal ranges and comparable to placebo. There were no discontinuations and all eight Cohort 1 participants have transitioned to the open-label portion of the study.