ENTR-601-45 - (ELEVATE-45-201)

October 14, 2026: Entrada Therapeutics (NASDAQ: TRDA) reported positive topline Phase 1/2 Cohort 1 data from ELEVATE-45-201 for ENTR-601-45 in ambulatory Duchenne muscular dystrophy patients with DMD mutations amenable to exon 45 skipping. In the double-blind, placebo-controlled multiple ascending dose portion, participants received three 5 mg/kg doses or placebo. The primary objectives of safety and tolerability were met: headache was the most common TEAE, one participant had two serious TEAEs (vomiting and pyrexia) that resolved, there were no discontinuations, and no hypomagnesemia or renal safety concerns were reported.

Secondary measures showed a 0.10% mean increase in dystrophin from baseline in treated participants (n=6; nominal p=0.03) versus a 0.02% mean decrease with placebo, and a 0.15% mean increase in exon skipping (n=5) versus a 0.04% mean decrease with placebo. No functional benefit was expected or observed after three doses at this low dose level.

All Cohort 1 participants moved into the open-label Phase 2 portion. Cohort 2 enrollment is complete and 10 mg/kg dosing is ongoing, with data expected in 1H 2027; Cohort 3 is planned at up to 15 mg/kg. The company also said an independent data monitoring committee recommended proceeding to 18 mg/kg in the separate ELEVATE-44-201 study.