Entrada Therapeutics DMC Greenlights 10 mg/kg Dose for ELEVATE-45-201 Cohort 2
TRDA•An independent Data Monitoring Committee reviewed safety and PK data from eight participants in Cohort 1 of Entrada Therapeutics’ Phase 1/2 ELEVATE-45-201 study, recommending escalation to a 10 mg/kg dose for Cohort 2. The company is on track to report Cohort 1 data in mid-2026 with higher-dose results to follow.
1. DMC Recommendation and Dose Escalation
The independent Data Monitoring Committee evaluated all available safety and pharmacokinetic data from the eight participants in Cohort 1 (5 mg/kg) of the Phase 1/2 ELEVATE-45-201 multiple ascending dose study. After confirming tolerability and favorable PK profiles, the committee approved initiating Cohort 2 at an increased dose level of 10 mg/kg.
2. ELEVATE-45-201 Study Design and Timeline
ELEVATE-45-201 is a global, randomized, double-blind, placebo-controlled MAD study enrolling 24 ambulatory participants aged four to 20 with Duchenne muscular dystrophy amenable to exon 45 skipping. Dosing occurs every six weeks across three cohorts (5, 10 and up to 15 mg/kg), with Cohort 1 data due mid-2026 and subsequent cohort readouts to follow.
3. Potential Clinical Impact and Next Steps
ENTR-601-45’s once-every-six-weeks regimen aims to improve dystrophin production while reducing treatment burden. With Cohort 2 initiation approved, the company will collect additional safety, PK and functional data to inform the open-label Phase 2 portion and support further development of the program.
