Entrada reports ENTR-601-45 DMD trial data

• Entrada Therapeutics (NASDAQ:TRDA) reported that ENTR-601-45 achieved safety and tolerability objectives in the first cohort of its Phase 1/2 Duchenne muscular dystrophy (DMD) study.
• The treatment produced a 0.10% mean increase in dystrophin, a muscle-supporting protein, compared with a decrease in placebo-treated participants.
• Entrada said Cohort 2 dosing at 10 mg/kg is ongoing, with data expected in the first half of 2027.

Entrada Therapeutics (NASDAQ:TRDA) announced initial results from the first cohort of its Phase 1/2 ELEVATE-45-201 study evaluating ENTR-601-45 in patients with Duchenne muscular dystrophy.

Eight participants received three doses of ENTR-601-45 at 5 mg/kg or placebo. The company reported that the study met its initial safety and tolerability objectives.

Participants treated with ENTR-601-45 showed a mean 0.10% increase in dystrophin levels, compared with a 0.02% mean decrease among placebo participants.

Exon skipping increased by a mean 0.15% in treated participants compared with a 0.04% mean decline in the placebo group.

One participant experienced two serious adverse events involving vomiting and fever, which resolved. No participants discontinued treatment, and all participants entered the open-label extension phase.

Entrada said no functional improvement was observed after three doses, which was consistent with the company’s expectations for the early study timeframe.

Cohort 2 enrollment has been completed, with participants receiving 10 mg/kg dosing.

Entrada expects to report Cohort 2 data in the first half of 2027, followed by results from Cohort 3 evaluating doses up to 15 mg/kg.

The company expects higher doses of ENTR-601-45 could increase dystrophin production and potentially deliver functional improvements.

Entrada Therapeutics develops RNA-based and genetic medicines designed to treat serious diseases, including neuromuscular disorders.