D.4 Safety and efficacy of delandistrogene moxeparvovec versus placebo in Duchenne muscular dystrophy (EMBARK): Pivotal Phase 3 primary results

• DOI: 10.1017/cjn.2024.95
• Publication Date: 2024-05-24T01:46:43Z
• Authors: JR Mendell; F Muntoni; CM McDonald; EM Mercuri; E Ciafaloni; H Komaki; C Leon-Astudillo; A Nascimento; C Proud; U Schara-Schmidt; A Veerapandiyan; CM Zaidman; M Guridi; AP Murphy; C Reid; C Wandel; E Darton; S Mason; RA Potter; T Singh; W Zhang; P Fontoura; JS Elkins; LR Rodino-Klapac
• Keywords: Clinical endpoint

Background: Duchenne muscular dystrophy (DMD) is caused by DMD gene mutations. Delandistrogene moxeparvovec an investigational transfer therapy, developed to address the underlying cause of DMD. We report findings from Part 1 (52 weeks) two-part EMBARK trial (NCT05096221). Methods: Key inclusion criteria: Ambulatory patients aged ≥4-<8 years with a confirmed mutation within exons 18–79 (inclusive); North Star Assessment (NSAA) score >16 and <29 at screening. Eligible were randomized 1:1 intravenous delandistrogene (1.33×1014 vg/kg) or placebo. The primary endpoint was change baseline in NSAA total Week 52. Results: At 52 (n=125), did not reach statistical significance, although there nominal difference (2.6, n=63) versus placebo groups (1.9, n=61). secondary endpoints (time rise, micro-dystrophin expression, 10-meter walk/run) demonstrated treatment benefit both age (4-5 6-7 years; p<0.05).There no new safety signals, reinforcing favorable manageable profile observed date. Conclusions: Based on totality functional assessments including timed function tests, indicates beneficial modification disease trajectory.