ELEVATE-44-201
- Indication
- Duchenne Muscular Dystrophy
- Stage
- phase1
- Event
- Phase 1/2
- Details
October 14, 2026: Entrada Therapeutics (NASDAQ: TRDA) said an independent Data Monitoring Committee reviewed available safety and pharmacokinetic data from eight participants in Cohort 2 of the Phase 1/2 ELEVATE-44-201 study of ENTR-601-44 for Duchenne muscular dystrophy amenable to exon 44 skipping, and recommended starting Cohort 3 at 18 mg/kg, up from 12 mg/kg in Cohort 2 and 6 mg/kg in Cohort 1.
ELEVATE-44-201 is a global randomized, double-blind, placebo-controlled multiple ascending dose study in ambulatory patients aged 4-20. Part A is assessing safety, PK, pharmacodynamics, and functional measures after IV dosing at sites in the U.K. and EU.
Upcoming catalysts: the company expects Cohort 1 open-label period data by year-end 2026 after one year of treatment, and says Cohort 2 enrollment is complete with data expected in Q1 2027.
Background noted in the release: ENTR-601-44 is an exon 44-skipping oligonucleotide conjugated with Entrada’s Endosomal Escape Vehicle platform, and the FDA granted it Rare Pediatric Disease Designation in December 2025.
- Source
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