other material
confidence high
sentiment positive
materiality 0.65
Entrada's ENTR-601-45 shows safe 5 mg/kg profile, 0.10% dystrophin rise in DMD Cohort 1
Entrada Therapeutics, Inc.
- Cohort 1 of Phase 1/2 ELEVATE-45-201 enrolled 8 ambulatory DMD patients (exon 45 skipping), randomized 3:1 to IV ENTR-601-45 5 mg/kg or placebo on days 1, 43 and 85.
- Dystrophin rose a mean 0.10% from baseline in treated patients (n=6, nominal p=0.03) vs a 0.02% mean decrease on placebo; exon skipping rose 0.15% (n=5) vs -0.04% on placebo.
- Favorable safety: headache most common TEAE (5 events in 2 participants); 1 participant had 2 serious TEAEs (vomiting, pyrexia) that resolved; no discontinuations; no hypomagnesemia or renal concerns.
- No functional benefit seen after three doses, as expected given patient age and baseline function; Q6W dosing equals about nine infusions a year vs 50+ for weekly regimens.
- Cohort 2 (10 mg/kg) enrollment complete and dosing ongoing, with data due H1 2027; Cohort 3 up to 15 mg/kg to follow, targeting higher exposure and dystrophin.