Entrada Therapeutics, today announced it has received authorization from the Health Authorities and Ethics Committees of multiple countries under the European Union Clinical Trial Regulation (EU-CTR) to initiate ELEVATE-45-201, a Phase 1/2 multiple ascending dose (MAD) clinical study of ENTR-601-45 in patients living with Duchenne muscular dystrophy (DMD) who are amenable to exon 45 skipping.
Entrada Therapeutics Receives EU Authorization
“ELEVATE-45-201 is the most advanced clinical study of a conjugated exon skipping therapy for individuals amenable to exon 45 skipping and offers the potential to address a large Duchenne subpopulation with significant unmet medical needs,” said Dipal Doshi, Chief Executive Officer at Entrada Therapeutics. “The EU authorization builds on the growing clinical momentum behind our novel Duchenne programs. With the recent authorizations of ELEVATE-44 and ELEVATE-45, and a planned filing for ELEVATE-50 later this year, we are on track to significantly expand the scope of our DMD franchise by year-end.”
Read More: Next Generation Exon Skipping Therapies Developed for the Treatment of Duchenne Muscular Dystrophy
Exon 45 Skipping Clinical Trial Starts in EU
ELEVATE-45-201 is a global, two-part, randomized, double-blind placebo-controlled Phase 1/2 study evaluating the safety, tolerability and effectiveness of ENTR-601-45 in ambulatory patients with Duchenne who are exon 45 skipping amenable. Part A is a multiple ascending dose study designed to evaluate the safety, pharmacokinetics and pharmacodynamics, including exon skipping and dystrophin production in approximately 24 patients. Dosing will be administered every six weeks, with the planned doses across three cohorts anticipated to range from 5 mg/kg up to 15 mg/kg. Part B of the study will further evaluate the optimal dose established in Part A for safety and efficacy, including functional outcomes and patient reported quality of life measures. Study participants may be eligible to enter an open label extension study (OLE), in which the safety, efficacy and tolerability of ENTR-601-45 will be evaluated over a longer period of time. The Company is on track to initiate ELEVATE-45-201 in Q3 2025.
Learn More: Cures of Duchenne (List of All Researches)
