Entrada Therapeutics Announces Positive Topline Results from Cohort 1 of Participants with Duchenne Muscular Dystrophy Treated with ENTR-601-44 in Phase 1/2 ELEVATE-44-201 Study
Entrada Therapeutics reported positive topline data from Cohort 1 of its Phase 1/2 ELEVATE-44-201 study of ENTR-601-44 in pediatric Duchenne patients, showing a favorable safety profile and statistically significant improvement in Time to Rise velocity. Treated participants showed a 2.36% increase in dystrophin over a 4.00% baseline, though plasma exposure was lower than expected compared to healthy adults.
Key figures
- Drug Name
- ENTR-601-44
- Phase Of Trial
- Phase 1/2
- Dystrophin baseline percent
- 4
- Dystrophin increase percent
- 2.36
- Exon skipping baseline percent
- 2.66
- Exon skipping increase percent
- 2.31
- Mean change ttr velocity vs placebo
- 0.115
- Mean change ttr velocity treatment group
- 0.08
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