Entrada Therapeutics has secured the Health Authorities and Ethics Committees’ authorisation within the European Union (EU) Clinical Trial Regulation to begin the randomised Phase I/II trial, ELEVATE-45-201, evaluating ENTR-601-45 for those with Duchenne muscular dystrophy (DMD).
This multiple ascending dose (MAD) trial targets patients amenable to exon 45 skipping.
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It is structured as a two-part, double-blind, placebo-controlled global trial, aiming to assess the tolerability, effectiveness, and safety of the therapy in ambulatory DMD subjects.
Part A of the trial will focus on pharmacodynamics, safety, and pharmacokinetics, including exon skipping and the production of dystrophin, in approximately 24 subjects.
The doses will be administered every six weeks, at levels ranging from 5mg/kg to 15mg/kg across three cohorts.
The subsequent phase, Part B, will assess the ideal dose determined in the earlier part for efficacy and safety by considering functional outcomes and quality of life measures reported by subjects.
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By GlobalDataIn addition, subjects may have the opportunity to join an open-label extension trial to monitor the long-term effects of ENTR-601-45.
The company plans to launch ELEVATE-45-201 in the third quarter of this year.
Entrada Therapeutics CEO Dipal Doshi said: “The EU authorisation builds on the growing clinical momentum behind our novel Duchenne programmes. With the recent authorisations 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.”
An Endosomal Escape Vehicle (EEV)-conjugated phosphorodiamidate morpholino oligomer, ENTR-601-45 is the company’s second DMD product candidate.
The company noted that each EEV-PMO therapeutic is specifically tailored for targeted subpopulations.
ENTR-601-45 aims to correct the root cause of DMD by addressing mutations or absences in the DMD gene.
The investigational therapy seeks to restore the mRNA reading frame, enabling the translation of a slightly shortened, yet functional, dystrophin protein.
This February, the US Food and Drug Administration (FDA) lifted a two-year clinical hold on Entrada Therapeutics’ DMD therapy, ENTR-601-44.
