Entrada Therapeutics has secured authorisation from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee to commence Phase I/II ELEVATE-45-201 trial of ENTR-601-45 for treating Duchenne muscular dystrophy (DMD).
The randomised, placebo-controlled, two-part, double-blind trial will assess the therapy in individuals with a DMD gene mutation amenable to exon 45 skipping.
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It aims to evaluate the tolerability, effectiveness and safety of the therapy and will include ambulatory DMD subjects.
The initial phase, Part A, is said to be a multiple-ascending dose (MAD) study to assess the pharmacodynamics, pharmacokinetics and safety, inclusive of the exon skipping and dystrophin production in an estimated 24 subjects.
Administration of the doses will take place every six weeks, with expected ranges from 5mg/kg to 15mg/kg across three cohorts.
Following this phase, Part B will aim to determine the optimal dose identified in Part A for efficacy and safety, considering functional results and subject-reported quality of life measures.
Subjects may have the opportunity to join an open-label extension study, where the long-term efficacy, tolerability, and safety of the therapy will be assessed.
The commencement of the trial is scheduled for the third quarter of this year.
Entrada Therapeutics CEO Dipal Doshi said: “We are pleased to receive UK clearance for the second clinical programme in our growing Duchenne franchise.
“Building on the momentum of our ELEVATE-44 programme, this authorisation brings us closer to having three Duchenne programmes in clinical development by the end of this year.”
The therapy is an Endosomal Escape Vehicle (EEV)-conjugated phosphorodiamidate morpholino oligomer (PMO) and claims to be the second candidate in the company’s DMD franchise from its EEV-therapeutic pipeline.
Last month, the company announced that the US Food and Drug Administration (FDA) had lifted a clinical hold on DMD therapy, ENTR-601-44, and authorised the commencement of ELEVATE-44-102, a Phase Ib MAD clinical study for the therapy.
