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Abstract Details

EMBARK, a Phase 3 Trial Evaluating Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Duchenne Muscular Dystrophy (DMD): Study Design and Baseline Characteristics
Neuromuscular and Clinical Neurophysiology (EMG)
P5 - Poster Session 5 (11:45 AM-12:45 PM)
10-012
Delandistrogene moxeparvovec is an investigational gene transfer therapy developed to address the root cause of DMD through targeted skeletal and cardiac muscle expression of SRP-9001 dystrophin protein, which contains key functional domains of dystrophin.
To describe the EMBARK (Study 301; NCT05096221) study design, a Phase 3, global, randomized, double-blind, two-part, placebo-controlled study assessing the safety and efficacy of intended commercial process delandistrogene moxeparvovec (SRP-9001) material in ambulatory individuals with a confirmed Duchenne muscular dystrophy (DMD) mutation within exons 18-79 (excluding individuals with a mutation fully contained within exon 45), aged ≥4 to <8 years (N=125). 

In Part 1, participants will be stratified by age at randomization (≥4 to <6 years or ≥6 to <8 years) and North Star Ambulatory Assessment total score (NSAA ≤22 points or >22 points) at screening and randomized (1:1) to receive a single intravenous dose of intended commercial process delandistrogene moxeparvovec material (1.33x1014 vg/kg by linear standard qPCR) or placebo. Participants will be evaluated at Week 52. In Part 2 (52-week follow-up period), participants randomized to placebo in Part 1 will receive delandistrogene moxeparvovec and participants randomized to delandistrogene moxeparvovec in Part 1 will receive placebo.

The primary endpoint is change from baseline to Week 52 in NSAA total score (Part 1). Secondary endpoints include safety; SRP-9001 dystrophin protein production at Week 12 by western blot and change from baseline to Week 52 in: key timed function tests, stride velocity 95th centile measured by a wearable device, and Patient-Reported Outcomes Measurement Information Score®.

For the first time, we present baseline characteristics of participants enrolled in EMBARK, a Phase 3 study of delandistrogene moxeparvovec.
EMBARK will provide placebo-controlled information about efficacy and safety of delandistrogene moxeparvovec in a large population of ambulatory patients with DMD aged ≥4 to <8 years. 
Authors/Disclosures
Francesco Muntoni, MD (UCL Institute of Child Health)
PRESENTER
Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Pfizer. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Avexis. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. The institution of Dr. Muntoni has received research support from European Commission. The institution of Dr. Muntoni has received research support from Medical Research Council. The institution of Dr. Muntoni has received research support from Biogen. The institution of Dr. Muntoni has received research support from Muscular Dystrophy UK. The institution of Dr. Muntoni has received research support from MDA USA. The institution of Dr. Muntoni has received research support from Sarepta. The institution of Dr. Muntoni has received research support from Association Francoise Myopathies. Dr. Muntoni has received personal compensation in the range of $0-$499 for serving as a Clinical expert with UK NICE Committee.
Eugenio Mercuri Eugenio Mercuri has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta, Santhera, PTC, Pfizer, Roche, Biogen, Avexis Novartis, Scholar Rock, Cytokinetics. Eugenio Mercuri has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Sarepta, Santhera, PTC, Pfizer, Roche, Biogen, Avexis Novartis. The institution of Eugenio Mercuri has received research support from biogen.
Ulrike Schara Schmidt, MD (University of Essen / Germany) Prof. Schara Schmidt has received personal compensation for serving as an employee of PTC. Prof. Schara Schmidt has received personal compensation for serving as an employee of Pfizer. Prof. Schara Schmidt has received personal compensation for serving as an employee of Sarepta. Prof. Schara Schmidt has received personal compensation for serving as an employee of Roche. Prof. Schara Schmidt has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC. Prof. Schara Schmidt has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Prof. Schara Schmidt has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Prof. Schara Schmidt has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Prof. Schara Schmidt has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Sarepta.
Hirofumi Komaki Hirofumi Komaki has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Nippon Shinyaku. Hirofumi Komaki has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. The institution of Hirofumi Komaki has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC therapeutics Inc. Hirofumi Komaki has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Chugai .
James Antony Richardson, MBBS (Sarepta Therapeutics) Dr. Richardson has received personal compensation for serving as an employee of Sarepta Therapeutics. Dr. Richardson has stock in Sarepta Therapeutics.
Tejdip Singh (Sarepta) Tejdip Singh has received personal compensation for serving as an employee of Sarepta Therapeutics . Tejdip Singh has received stock or an ownership interest from Sarepta Therapeutics.
Maitea Guridi, PhD (Hoffman-La Roche) Dr. Guridi has received personal compensation for serving as an employee of Hoffmann-La Roche.
Stefanie Mason, MD Dr. Mason has received personal compensation for serving as an employee of Sarepta. Dr. Mason has stock in Sarepta. An immediate family member of Dr. Mason has stock in Sarepta. The institution of an immediate family member of Dr. Mason has received research support from Biogen. The institution of Dr. Mason has received research support from NIH. The institution of Dr. Mason has received research support from Chest Foundation.
Alexander Peter Murphy, PhD,MBBS (Roche Pharmaceuticals) Dr. Murphy has nothing to disclose.
Lixi Yu Mrs. Yu has nothing to disclose.
Carol Reid Carol Reid has received personal compensation for serving as an employee of Roche Products Ltd. Carol Reid has stock in Roche Products Ltd.
Eddie Darton, MD (Sarepta Therapeutics) Dr. Darton has received personal compensation for serving as an employee of Sarepta Therapeutics. Dr. Darton has stock in Sarepta Therapeutics.
Christoph Wandel, MD, PhD Christoph Wandel, MD, PhD has received personal compensation for serving as an employee of F Hoffmann-LaRoche AG. Christoph Wandel, MD, PhD has stock in F Hoffmann-La Roceh AG, Basel, CH; Bayer AG, Leverkusen, FRG.
Jerry R. Mendell, MD, FAAN (The Research Institute at Nationwide Children's Hospital) Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Vertex. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Mendell has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta Therapeutics . The institution of Dr. Mendell has received research support from Sarepta.