Entrada reports positive Cohort 1 safety data for DMD therapy ENTR-601-45

Entrada Therapeutics announced on October 14, 2026 topline results from Cohort 1 of the Phase 1/2 ELEVATE-45-201 study, in which participants received three doses of 5 mg/kg of ENTR-601-45 or placebo, meeting the primary objectives of favorable safety and tolerability.¹

Cohort 1 enrolled eight participants with Duchenne, randomized 3:1 to receive ENTR-601-45 at 5 mg/kg or placebo, administered intravenously.¹ On secondary measures, a mean increase of 0.10% (n = 6, nominal p = 0.03) from baseline in dystrophin production was observed in study participants who received ENTR-601-45 versus a 0.02% mean decrease from baseline in study participants who received placebo, and a mean increase of 0.15% (n = 5, no p-value calculated) from baseline in exon skipping was observed versus a 0.04% mean decrease in the placebo group.¹ The company noted that based on the age of Cohort 1 participants and baseline levels of function, no observable functional benefit was expected or observed after three doses.¹

On safety, headache (5 events in 2 participants) was the most common treatment-emergent adverse event, one participant experienced 2 serious TEAEs (vomiting and pyrexia) which subsequently resolved, and there were no study discontinuations due to any cause.¹ No hypomagnesemia or renal safety concerns were noted, with renal markers and magnesium levels within normal ranges.¹

All Cohort 1 participants have progressed to the open-label Phase 2 portion, where they will receive six additional doses of 5 mg/kg.¹ Cohort 2 enrollment is complete with dosing at 10 mg/kg ongoing, and the company is on track to report Cohort 2 data in the first half of 2027, with Cohort 3 data (up to 15 mg/kg) to follow.¹