Entrada Therapeutics has concluded the first and second cohorts dosing for the ENTR-601-44-101 Phase I clinical study of its investigational therapy ENTR-601-44 to treat Duchenne muscular dystrophy (DMD).
DMD is a serious form of muscular dystrophy that mainly impacts boys. Weakness in the muscles typically starts around the age of four and progresses rapidly.
ENTR-601-44 is an Endosomal Escape Vehicle (EEV)-conjugated phosphorodiamidate morpholino oligomer (PMO).
ENTR-601-44-101 is a single ascending dose and double-blind study, which is anticipated to enrol around 40 subjects.
Assessing the tolerability and safety of a single ENTR-601-44 dose in healthy male volunteers is the primary objective of the Phase I study.
Furthermore, the trial will assess pharmacokinetics and target engagement, as measured by exon skipping in the skeletal muscle.
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The company intends to reveal data from this trial in the second half of next year.
Entrada Therapeutics president and CEO Dipal Doshi said: “Our strategy has always been to run a single Phase I clinical trial for ENTR-601-44 and, notably, that trial is progressing in the UK.
“We are pleased to have completed dosing of the first and second cohorts of participants. In parallel with the Phase I clinical trial, we continue to plan for the global development of ENTR-601-44 which will include clinical trials in patients with Duchenne who are exon 44 skipping amenable.”
Entrada received prior notification from the US Food and Drug Administration (FDA) about the Phase I clinical trial of ENTR-601-44 being placed on hold.
Even after providing more details to the FDA, the company was informed that the agency decided not to remove the clinical hold.
The information submitted backed the start of the Phase I study in the UK in September.
During that month, Entrada dosed the first subject in the trial to assess ENTR-601-44.