Entrada Therapeutics: positive Cohort 1 data in Phase 1/2 DMD study

What happened

• On Oct 14, 2026 Entrada Therapeutics issued a press release and announced positive topline data from Cohort 1 of the double-blind, placebo-controlled multiple ascending dose (MAD) portion of the Phase 1/2 ELEVATE-45-201 study of ENTR-601-45 in ambulatory participants aged 4 to 20 with DMD amenable to exon 45 skipping.
• Cohort 1 enrolled 8 participants randomized 3:1 to receive ENTR-601-45 at 5 mg/kg or placebo, with intravenous doses on days 1, 43 and 85 and muscle biopsies at screening and six weeks after the last dose; all participants continued into the open-label extension.
• Cohort 2 enrollment is complete and dosing at 10 mg/kg is ongoing; the company said it is on track to report Cohort 2 data in the first half of 2027, with Cohort 3 (up to 15 mg/kg) to follow.

Key details

• Dystrophin: mean increase of 0.10% from baseline in treated participants (n = 6, nominal p = 0.03) versus a 0.02% mean decrease in placebo; LLOQ for dystrophin 0.25%, LOD 0.1%; two treated participants had baseline dystrophin LOD.
• Exon skipping: mean increase of 0.15% from baseline in treated participants (n = 5) versus a 0.04% mean decrease in placebo.
• Safety and tolerability: headache was the most common TEAE (5 events in 2 participants); one participant had 2 serious TEAEs (vomiting and pyrexia) that resolved; no study discontinuations; no hypomagnesemia or renal safety concerns reported and renal markers and magnesium were within normal ranges.
• Pharmacokinetics: plasma ENTR-601-45 exposures were consistent with juvenile non-human primate predictions, which the company said supports expectations of higher dystrophin expression at higher doses.

Why it may matter

• Item 7.01 (Regulation FD disclosure) notes the company furnished a press release (Exhibit 99.1) on Oct 14, 2026. Item 8.01 (Other events) provides an update on ENTR-601-45, reporting topline safety, tolerability, pharmacokinetics, dystrophin production, exon skipping and functional assessments for Cohort 1 of ELEVATE-45-201.
• A filing does not show why the insider traded or why the company acted.