Announcement • May 10

Entrada Therapeutics Announces Positive Topline Results From Cohort 1 Of Participants With Duchenne Muscular Dystrophy Treated With ENTR-601-44 In Phase 1/2 Elevate-44-201 Study

Entrada Therapeutics, Inc. announced positive 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. The results demonstrated a favorable safety and tolerability profile with no reported serious adverse events 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 multiple ascending dose study by year-end 2026, with data from Cohort 3 (up to 18 mg/kg) to follow. Highlights from the topline results of Cohort 1 ELEVATE-44-201 include: 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 serious adverse events and no adverse events leading to discontinuation from the study. The most common adverse event 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. Consistent with the recently received data in juvenile nonhuman primates, the Company observed a lower-than-expected plasma Cmax and area under the curve in pediatric Duchenne muscular dystrophy participants when compared with that seen in healthy adult volunteers and the adult nonhuman primates. The levels observed in Cohort 1 were in line with the exposures observed in juvenile nonhuman primates, thus providing confidence on future modeling of exposure. Updated modeling, following review of the juvenile nonhuman primate data, suggest that the area under the curve will significantly increase in Cohort 2, resulting in higher muscle concentration, exon skipping and dystrophin production. Despite the drug plasma concentration and dystrophin levels, the Company obtained earlier-than-expected functional responses that were both statistically significant and clinically meaningful. Mean change in Time to Rise velocity is a robust, low variability measure which carries the largest absolute and proportional annual signal and is used as an early prognostic factor for disease progression and loss of ambulation. Cohort 1 results demonstrated a statistically significant improvement in mean Time to Rise velocity in treated versus placebo participants.

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Entrada Therapeutics Announces Positive Topline Results From Cohort 1 Of Participants With Duchenne Muscular Dystrophy Treated With ENTR-601-44 In Phase 1/2 Elevate-44-201 Study

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