[Ad hoc announcement pursuant to Art. 53 LR] Roche announces EMBARK trial in Duchenne muscular dystrophy (DMD) did not reach primary endpoint, but shows positive efficacy outcomes on all timed functional key endpoints

This study is ongoing.Study 301 (EMBARK), a Phase 3 global, randomised, double-blinded and placebo-controlled study of Elevidys in ambulatory Duchenne patients aged 4-<8 years old.The ENVOL trial (Study 302) a Phase 2 study in children with Duchenne. The study aims to enrol 21 participants who are under 4 years of age, including newborns. Not yet started.The ENVISION trial (Study 303), a Phase 3 study in older ambulatory/non-ambulatory patients which is now recruiting.The EXPEDITION long-term (5 year) follow up study (Study 305) of participants who have received Elevidys in a previous clinical study, which is not yet recruiting.About EMBARKEMBARK is a multinational, Phase 3, randomised, double-blind, two-part crossover, placebo-controlled study assessing the safety and efficacy of Elevidys in ambulatory boys with a confirmed mutation in theDMD gene, aged between 4 and 7 years. Eligible participants received a single dose of Elevidys during either Part 1 or Part 2 of the study. The study is ongoing.Participants (n=125) received 1.33x1014 vg/kg of delandistrogene moxeparvovec or placebo. In Part 1, participants were randomised according to age (4-5 or 6-7) or NSAA total score at screening ( ≤22 or>22) to receive either Elevidys or placebo, with a follow-up period for 52 weeks. In Part 2, participants crossed over - meaning, those who were previously treated with placebo in Part 1 received Elevidys and participants who were previously treated with placebo received Elevid...
Source: Roche Media News - Category: Pharmaceuticals Source Type: news