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
-- Achieved the primary objective with favorable safety and tolerability, no discontinuations and no serious adverse events -- -- Markers of kidney function via eGFR, Cystatin C and magnesium were all within normal ranges and comparable to placebo -- -- Observed lower plasma exposure in Cohort 1 participants who are all between six and 17 years of age when compared with healthy adult volunteers; A similar trend was seen between recently received juvenile and adult NHP PK data -- -- Consequently, Cohort 1 demonstrated an increase of 2.36% in dystrophin over a baseline of 4.00% and an increase of 2.31% in exon skipping over a baseline of 2.66% in treated participants -- -- Statistically significant and potentially differentiated improvement in treated participants versus placebo in Time to Rise velocity, a clinically validated functional measurement -- -- Company's updated PK modeling predicts Cohort 2, building upon Cohort 1 data and combined with the recently received juvenile NHP data, will result in a significant increase of plasma AUC and substantially higher dystrophin levels with continued benefit in muscle function -- -- Company has initiated dosing of ELEVATE-44-201 Cohort 2 at the increased dose of 12 mg/kg and is on track to report data by year-end 2026 -- -- Entrada to host investor webcast and conference call today, Thursday, May 7, at 8:30 a.m. ET -- BOSTON, May 07, 2026 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA) today announced positive topline data from Cohort 1 of the double-blind, placebo-controlled, multiple ascending dose (MAD) portion of the Phase 1/2 ELEVATE-44-201 clinical study.