Entrada Therapeutics (NASDAQ:TRDA) has announced positive preliminary data from its Phase 1 clinical trial evaluating the safety and tolerability of a single dose of ENTR-601-44-101 in patients with Duchenne Muscular Dystrophy (DMD).
A total of 32 healthy male volunteers participated in the trial, which also evaluated pharmacokinetics and target engagement, as measured by exon skipping in the skeletal muscle. The company reported no serious adverse events and no clinically significant changes or trends in vital signs, ECGs, physical exams, or laboratory assessments observed in the trial.
In a statement, Francesco Muntoni, M.D., Professor of Paediatric Neurology, said, “I am encouraged to see the preliminary results of Entrada’s Phase 1 clinical trial of ENTR-601-44-101. These data in healthy volunteers represent a potentially transformative treatment option for boys and young men living with Duchenne who are exon 44 skipping amenable, a population for which there are currently exon skipping options.”
Entrada plans to submit regulatory applications in the fourth quarter of 2024 to initiate separate global Phase 2 clinical trials for ENTR-601-44-101 in patients with DMD who are exon 44 skipping amenable.
