Entrada Therapeutics Advances Duchenne Study To Highest Planned Dose Level
(RTTNews) - A favorable safety review has cleared the way for Entrada Therapeutics to begin testing its Duchenne muscular dystrophy candidate at the highest planned dose level in an ongoing early-stage clinical study.
Entrada Therapeutics, Inc. (TRDA) announced that an independent Data Monitoring Committee (DMC) has recommended initiation of Cohort 3 at a dose of 18 mg/kg in the Phase 1/2ELEVATE-44-201 study of ENTR-601-44, a therapy for Duchenne muscular dystrophy patients whose genetic mutation is amenable to exon 44 skipping.
The recommendation followed a review of available safety and pharmacokinetic data from eight participants enrolled in Cohort 2 of the study's multiple ascending dose portion. Cohort 3 will evaluate the highest planned dose level, increasing from 12 mg/kg in Cohort 2 and 6 mg/kg in Cohort 1.
Duchenne muscular dystrophy (DMD) is a rare genetic disease that causes progressive muscle weakness and loss of muscle function. ENTR-601-44 is designed to promote exon 44 skipping, a genetic approach intended to help the body produce a shorter but functional form of dystrophin, a protein that is lacking in patients with Duchenne.
ELEVATE-44-201 is global, two-part, randomized, double-blind, placebo-controlled Phase 1/2 study evaluating the safety, tolerability and effectiveness of ENTR-601-44 in ambulatory participants aged 4 to 20 years. The study is being conducted at sites in the United Kingdom and European Union.
The company said enrollment in Cohort 2 has been completed and expects to report data from Cohort 1 participants in the open label portion of the study by the end of 2026. Data from Cohort 2 are anticipated in the first quarter of 2027.
According to the company, advancing to the 18 mg/kg dose level will allow researchers to further evaluate the therapy's safety profile and determine whether higher exposure may lead to increased dystrophin production and potential functional benefit.
TRDA has traded between $4.83 and $16.45 over the past year. The stock closed Tuesday's trading at $4.86, down 11.64%. In pre-market trading the stock is at $4.88, up 0.41%.
