Entrada Therapeutics reports positive Phase 1/2 topline data and advances DMD program to highest dose
Entrada Therapeutics announced that its Phase 1/2 ELEVATE‑45‑201 study in Duchenne muscular dystrophy met safety and tolerability goals and showed a modest increase in dystrophin and exon skipping at a 5 mg/kg dose. All eight participants moved to the open‑label Phase 2 portion, and the company is enrolling Cohort 2 at a 10 mg/kg dose with results expected in the first half of 2027. In a separate DMD program, an independent data monitoring committee cleared Cohort 3 of the ENTR‑601‑44 study to receive the highest planned dose of 18 mg/kg, with data from earlier cohorts due by the end of 2026 and early 2027.
Why it matters
The company said the safety clearance and early signals of increased dystrophin support continued development and could drive future regulatory milestones, which may be reflected in the stock’s recent price gain.
Key facts
- 1The ELEVATE‑45‑201 study enrolled eight participants aged four to 20 years. investing.com
- 2Participants received three doses of 5 mg/kg ENTR‑601‑45. investing.com
- 3Mean dystrophin production rose 0.10% from baseline in the treatment group. investing.com
- 4The placebo group showed a mean dystrophin change of –0.02%. investing.com
- 5The nominal p‑value for the dystrophin difference was 0.03. investing.com
- 6Mean exon‑skipping increased 0.15% in treated participants versus a 0.04% decrease in placebo. investing.com
Summary written by AlphAI AI Desk from 5 of 5 sources. Not investment advice. Figures are as stated by the linked sources.