Entrada Therapeutics has announced positive topline data from Cohort 1 of the double-blind, placebo-controlled, multiple ascending dose portion of the Phase 1/2 ELEVATE-44-201 study evaluating ENTR-601-44 in ambulatory participants ages 4–20 living with Duchenne muscular dystrophy amenable to exon 44 skipping. ENTR-601-44 is an investigational therapy combining their cell penetrating peptide with an exon skipping oligonucleotide designed to restore dystrophin production.
According to Entrada, results from Cohort 1 demonstrated a favorable safety and tolerability profile at the 6 mg/kg dose level. The study also showed a statistically significant improvement in mean time-to-rise (TTR) velocity in treated participants compared with placebo, with positive changes observed across most participants regardless of age or disease severity. Treated participants demonstrated a 2.36% increase in dystrophin levels over a 4% baseline expression in patients.
According to the company, the delivery mechanism of ENTR-601-44 may offer the potential to access satellite cells, supporting both the repair of existing muscle fibers and the formation of new healthy muscle fibers.
Entrada reports that all participants in Cohort 1 have now transitioned into the open-label Phase 2 portion of the study, where they will receive six additional doses of ENTR-601-44 at 6 mg/kg. Enrollment and dosing are also ongoing in Cohort 2, where participants will receive three doses of either 12 mg/kg ENTR-601-44 or placebo.
Entrada expects to report data from the Cohort 1 open-label portion and the Cohort 2 MAD portion by the end of 2026, with results from Cohort 3, evaluating doses up to 18 mg/kg, expected to follow.
PPMD is encouraged by this update and we look forward to future updates from Entrada as study of ENTR-601-44 continues.
Read Entrada’s press release here. Access Entrada’s community newsletter here.
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