Entrada Therapeutics Reports Positive Preliminary Data in Healthy Volunteers from Phase 1 ENTR-601-44-101 Trial for Duchenne Muscular Dystrophy

– ENTR-601-44 was well-tolerated in healthy volunteers with no serious adverse events, no drug-related adverse events and no clinically significant changes or trends noted in vital signs, ECGs, physical exams or laboratory assessments –

– ENTR-601-44 demonstrated significant plasma concentration, muscle concentration and exon skipping, at levels that suggest the potential for a clinically meaningful starting dose in planned upcoming patient trials –

– Phase 2 planning underway for separate ENTR-601-44 and ENTR-601-45 clinical trials with regulatory filings anticipated in Q4 2024 –

Entrada Therapeutics, which received early funding from CureDuchenne, reported positive data from a Phase 1 ENTR-601-44-101 trial in healthy volunteers. ENTR-601-44, which is designed to skip exon 44, was well-tolerated, and the six individuals in the highest dose cohort (6 mg/kg) demonstrated an average of 0.44% exon skipping after a single dose. This was statistically significant compared to placebo control. Entrada plans to submit regulatory applications in Q4 2024 to initiate separate global Phase 2 clinical trials of ENTR-601-44 for Duchenne amenable to skipping exon 44, and for ENTR-601-45 for Duchenne amenable to skipping exon 45. They also plan to submit regulatory applications in 2025 for a global Phase 2 for Duchenne amenable to skipping exon 50.

Link to press release HERE

Related Posts

Share This Page

Make an Impact

You can advance the care, treatment and cure for Duchenne muscular dystrophy. Contributions in any amount can truly make a difference and can be credited to a fundraiser or event from the list below.