Entrada Therapeutics announced that an independent Data Monitoring Committee (DMC), per study protocol, has reviewed all available safety and PK data from the eight patients who completed dosing in Cohort 1 of the double-blinded, placebo-controlled, multiple ascending dose (MAD) portion of ELEVATE-44-201.
Dose increase recommendation is an indicator of positive results
“We are pleased that after reviewing the Cohort 1 data from our ELEVATE-44-201 study, the independent Data Monitoring Committee supports the initiation of Cohort 2 dosing at an increased dose of 12 mg/kg,” said Natarajan Sethuraman, PhD, President of Research and Development at Entrada Therapeutics. “Because Entrada’s neuromuscular programs all leverage the same Endosomal Escape Vehicle, we are confident that this study progression substantially de-risks our clinical programs and marks a significant clinical inflection point for our company. Establishing that ENTR-601-44 is safe at 6 mg/kg is a clear milestone for the program. We expect our Cohort 1 data at 6 mg/kg to show double-digit dystrophin production when we disclose the data in the second quarter. As we dose escalate to 12 mg/kg, we believe we will achieve best-in-class dystrophin restoration with the readout of our Cohort 2 data later this year.”
ELEVATE-44-201 is a global, two-part, randomized, double-blind, placebo-controlled Phase 1/2 study evaluating the safety, tolerability and effectiveness of ENTR-601-44 in ambulatory patients with DMD who are exon 44 skipping amenable. The Company completed dosing of the MAD portion of the study for Cohort 1, and participants have transitioned to the 6-dose open label, Phase 2 portion of the study. The Company is on track to report data from Cohort 1 (6 mg/kg) in the second quarter of 2026, data from Cohort 2 (12 mg/kg) by year-end, and data from Cohort 3 (up to 18 mg/kg) to follow. The Company also intends to open an expansion cohort later in the year to increase the number of participants treated in the ELEVATE-44-201 study, as this study has been designed to support an accelerated approval in the U.S. In December 2025, the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to ENTR-601-44.
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