Entrada Therapeutics announced on May 28, 2025, that it received authorization from Health Authorities and Ethics Committees of multiple countries under the European Union Clinical Trial Regulation (EU-CTR). This authorization is 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.
The company is on track to initiate ELEVATE-45-201 in Q3 2025. ELEVATE-45-201 is a global, two-part, randomized, double-blind placebo-controlled study designed to evaluate the safety, tolerability, and effectiveness of ENTR-601-45 in ambulatory DMD patients. Part A will assess safety, pharmacokinetics, and pharmacodynamics, including exon skipping and dystrophin production, in approximately 24 patients with planned doses ranging from 5 mg/kg up to 15 mg/kg.
Entrada's CEO, Dipal Doshi, stated that ELEVATE-45-201 is the most advanced clinical study of a conjugated exon skipping therapy for individuals amenable to exon 45 skipping, a subpopulation representing approximately nine percent of the estimated 41,000 people with Duchenne in the U.S. and Europe. With recent authorizations for ELEVATE-44 and ELEVATE-45, and a planned filing for ELEVATE-50 later this year, Entrada is on track to significantly expand its DMD franchise by year-end.
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