- Significant functional benefits for 8- and 9-year-olds with Duchenne in Part 2 of the EMBARK study, contributing to the evidence of stabilization or slowing of disease progression in later childhood when muscle weakness typically progresses
- Statistically significant differences were observed on all key endpoints including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort
- Five abstracts, including two oral presentations at American Society of Gene & Cell Therapy Conference, span Sarepta’s portfolio of approved and pipeline therapies across Duchenne and limb-girdle muscular dystrophy
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today presented new data from Part 2 of the EMBARK study that continue to support the clinical benefits of ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy. These data are among other ELEVIDYS data from Sarepta’s portfolio presented during the 28th annual meeting of the American Society of Gene & Cell Therapy (ASGCT) Conference.
In the recent analysis of Part 2 of the EMBARK study, participants with Duchenne muscular dystrophy who had received a placebo in Part 1 and were aged 8 to 9 years (n=14) at crossover were included. At one year post ELEVIDYS treatment, there were between-group differences (least square means) on all key endpoints that were statistically significant, including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort.
"The latest data from the EMBARK study highlighting motor function improvements in 8- and 9-year-old boys is encouraging and adds to the growing body of evidence supporting ELEVIDYS," said Aravindhan Veerapandiyan, M.D., Associate Professor of Pediatrics at the University of Arkansas for Medical Sciences and Arkansas Children’s Hospital. "What stands out is that these patients were treated at an age when motor decline is typically expected in those with Duchenne. Yet, those who received ELEVIDYS demonstrated statistically significant and clinically meaningful functional improvements compared to external controls."
The results presented at ASGCT are from the ongoing analysis of results from Part 2 of EMBARK, which compared two-year outcomes from 63 participants against data from an external control group of untreated individuals with Duchenne. Results at two years post-treatment showed that individuals treated with ELEVIDYS had better outcomes in multiple motor function measures, compared to a well-matched external control group. Additionally, no new safety signals were observed in the EMBARK study over the two-year duration and, in a subset of patients (n=16), micro-dystrophin expression and sarcolemmal localization was sustained from Week 12 to Week 64.
“This has been a significant year for our neuromuscular portfolio, with multiple, ongoing analyses and longer-term data on efficacy and safety presented for ELEVIDYS,” said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research and development, Sarepta Therapeutics. “Building on the topline EMBARK Part 2 data from earlier this year, we’re committed to sharing ongoing analyses as fast as possible. The one-year results of patients treated with ELEVIDYS at 8 to 9 years old provide evidence that those treated with gene therapy outperform those who don’t receive it at a critical point when more dramatic functional decline is expected.”
A full listing of Sarepta’s presentations at ASGCT are below.
Abstracts can be found at https://annualmeeting.asgct.org/. Data from presentations are embargoed until 6:00 AM CT on the presentation day for oral abstracts and until 6:00 AM CT on May 13, 2025 for poster abstracts.
Oral Presentations (*Previously presented at MDA 2025 and supplemented with additional data)
Title |
Date, Time |
Long-term Functional Outcomes and Safety Following Delandistrogene Moxeparvovec Treatment in DMD: EMBARK 2-Year Results* |
May 16 4:30 – 4:45 p.m. CST Room 393-396 |
Cardiovascular Investigation of SRP-9005 (AAVrh74.MHCK7.hSGCG) in Non-Human Primates: A Gene Therapy for Limb-Girdle Muscular Dystrophy 2C/R5 |
May 14 5 – 5:15 p.m. CST New Orleans Theater B |
Poster Presentations (*Denotes encore presentation)
Poster # |
Title |
#1350 |
3-Year Functional Outcomes of Patients with Duchenne Muscular Dystrophy: Pooled Delandistrogene Moxeparvovec Clinical Trial Data vs. External Controls* |
#1353 |
Assessment of Cardiac Outcomes in Delandistrogene Moxeparvovec Clinical Trials for Duchenne Muscular Dystrophy* |
#1422 |
In Situ Biodistribution and Localization of Bidridistrogene Xeboparvovec (SRP-9003) in LGMD2E/R4 Mice After 1 Year of Follow-up |
About EMBARK, Study SRP-9001-301
Study SRP-9001-301, also known as EMBARK, is a multinational, phase 3, randomized, two-part crossover, placebo-controlled study of ELEVIDYS in individuals with Duchenne muscular dystrophy between the ages of 4 to 7 years. The primary endpoint is change from baseline in NSAA Total Score at Week 52 following treatment. Eligible participants received a single dose of ELEVIDYS during either Part 1 or Part 2 of the study.
In Part 1, participants (n=125) were randomized according to age (≥4 to 16 to