Entrada Therapeutics has secured authorisation from the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) to begin the randomised, two-part Phase I/II trial, ELEVATE-44-201, to evaluate ENTR-601-44, aimed at treating Duchenne muscular dystrophy (DMD).
This marks a significant step forward in the potential treatment of DMD in individuals with a confirmed mutation in the DMD gene amenable to exon 44 skipping.
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The global, double-blind, placebo-controlled study is designed to assess the tolerability, effectiveness and safety of the therapy in these subjects.
Part A of ELEVATE-44-201 is a multiple ascending dose study that will assess the therapy’s pharmacodynamics, safety, and pharmacokinetics in nearly 24 subjects.
In this part, the subjects’ exon skipping and production of dystrophin will also be assessed. The dosing, scheduled every six weeks, is expected to range from 6mg/kg to 18mg/kg across three cohorts.
Following the completion of Part A, Part B will aim to further determine the ideal dose for efficacy and safety, incorporating subject-reported results along with quality-of-life measures.
Subjects may also have the opportunity to join an open-label extension study to monitor the long-term effects of the therapy.
The company anticipates initiating the ELEVATE-44-201 study in the second quarter of this year.
Entrada Therapeutics CEO Dipal Doshi said: “As the first authorisation for our global MAD clinical study of ENTR-601-44 in patients, we are pleased to be initiating the study at what we believe to be an effective therapeutic dose.
“This is even more important since families living with Duchenne do not have time on their side as the progressive decline in function profoundly impacts the quality of life for patients and their care partners. It is this urgency that drives our work each day.”
An Endosomal Escape Vehicle (EEV)-conjugated phosphorodiamidate morpholino oligomer, ENTR-601-44, is tailored to restore the mRNA reading frame and facilitate the translation of a slightly shortened but functional dystrophin protein.
In November 2023, the company completed dosing for the first and second cohorts in the ENTR-601-44-101 Phase I study of ENTR-601-44 for DMD treatment.
