FDA declines to lift hold on Entrada's Phase I DMD study
22 Nov 2023
Phase 1OligonucleotideINDPhase 2
Entrada Therapeutics announced Wednesday that the investigational new drug (IND) application for a Phase I trial of its proposed Duchenne muscular dystrophy (DMD) therapy ENTR-601-44 remains on clinical hold by the FDA. Testing on the drug - which uses Entrada's Endosomal Escape Vehicle (EEV) technology to increase cellular uptake of oligonucleotides for patients amenable to exon 44 skipping - was barred from getting under way in the US when the FDA hold was issued last December.
However, a Phase I trial dubbed ENTR-601-44-101 did kick off this past September in the UK, where it remains ongoing. "Despite providing additional information…the agency declined to lift the clinical hold," the company said, noting that the information it submitted was also what was used to support the start of the UK trial.
"We are disappointed that the US clinical hold has not been lifted, especially given the strength of the data package submitted to the FDA," commented CEO Dipal Doshi, adding "we will re-engage the FDA to discuss next steps in due course." Entrada also noted Wednesday that it has completed dosing of the first and second UK cohorts in the ENTR-601-44-101 trial and that it is anticipating data in the second half of 2024.
The study is evaluating the safety and tolerability of a single dose of ENTR-601-44 in healthy male volunteers, with a target enrollment of approximately 40 participants. The trial will also evaluate pharmacokinetics and target engagement, as measured by exon skipping in the skeletal muscle.
Meanwhile, Entrada has a partnership with Vertex Pharmaceuticals to develop intracellular EEV therapeutics for myotonic dystrophy type 1, including its preclinical asset ENTR-701. That deal, announced last December, came with a $224-million upfront payment and up to $485 million in potential milestones. Entrada has two other IND-stage oligonucleotide programmes for DMD, including ENTR-601-45 and ENTR-601-50.
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