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