Entrada Therapeutics reports positive early Cohort 1 safety data for DMD drug ENTR-601-45
Entrada Therapeutics said Cohort 1 of its Phase 1/2 ELEVATE-45-201 study of ENTR-601-45 in Duchenne muscular dystrophy met its primary safety and tolerability objectives at a 5 mg/kg dose. Secondary results showed a 0.10% mean increase in dystrophin production from baseline in treated participants versus a 0.02% mean decrease in placebo, and the company said no observable functional benefit was expected or seen after three doses. The data are early and small, with eight participants in the cohort, and Cohort 2 data at 10 mg/kg are expected in the first half of 2027.
Key points
- Cohort 1 enrolled eight participants randomized 3:1 to ENTR-601-45 at 5 mg/kg or placebo, dosed on days one, 43 and 85.
- Mean dystrophin increase of 0.10% from baseline (n = 6, nominal p = 0.03) versus a 0.02% mean decrease for placebo.
- Headache (5 events in 2 participants) was the most common treatment-emergent adverse event; no study discontinuations occurred.
- Cohort 2 (10 mg/kg) data are expected in the first half of 2027, with Cohort 3 (up to 15 mg/kg) to follow.
The filing
Written by AI on Nov 9, 2026, 8:53 AM ET, from this filing only. Written later: this filing was briefed after the fact, when past filings were added to the site, so the brief may be read with hindsight; the filing itself is the record of what was known on October 14, 2026.
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