Sarepta Therapeutics announced positive topline results from Part 2 of the EMBARK (Study SRP-9001-301) Phase 3 clinical trial evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for Duchenne muscular dystrophy (DMD). This global, randomized, double-blind, placebo-controlled study provides further evidence supporting the long-term benefits of ELEVIDYS for patients with DMD.
Key Findings from EMBARK Part 2:
- Significant Improvement in Motor Function in Crossover Group: Patients who initially received placebo in Part 1 of the EMBARK trial and subsequently crossed over to receive ELEVIDYS in Part 2 demonstrated a statistically significant 2.34-point improvement from baseline on the North Star Ambulatory Assessment (NSAA) at 52 weeks after starting ELEVIDYS treatment (p<0.0001). This improvement is particularly noteworthy considering that these crossover patients were, on average, one year older (7.18 years) at the start of their ELEVIDYS treatment compared to the patients treated in Part 1 (5.98 years). The NSAA is a validated rating scale used to assess motor function in individuals with DMD. This improvement suggests that even with a slight delay in treatment initiation, patients can still experience substantial benefits from ELEVIDYS. Furthermore, these crossover patients also showed significant improvements in other key functional measures, including Time to Rise (TTR, a measure of how quickly a patient can stand up from a seated position) and the 10-meter walk/run test (10MWR), compared to a pre-specified, propensity-weighted external control group.
- Sustained Benefits Over Two Years in Part 1 Treated Group: Patients who received ELEVIDYS in Part 1 of the EMBARK trial and were followed for two years continued to demonstrate clinically meaningful and statistically significant benefits across all three primary functional outcome measures (NSAA, TTR, and 10MWR) compared to the external control group. Critically, the magnitude of the benefit, as measured by the difference between the ELEVIDYS-treated group and the control group, increased from year one to year two. This widening divergence from the natural history of DMD suggests that ELEVIDYS may not only slow down disease progression but potentially alter the long-term trajectory of the disease. This is a crucial finding, as it hints at the potential for sustained benefit from a one-time gene therapy. Biopsies taken at 64 weeks (two years) after dosing in the Part 1 group confirmed consistent and sustained expression of the ELEVIDYS micro-dystrophin protein compared to biopsies taken at 12 weeks, providing biological support for the observed functional improvements.
- Minimal Progression of Muscle Pathology: Skeletal muscle MRI conducted on patients treated in Part 1 revealed minimal progression in underlying muscle pathology over the two-year period. This finding is consistent with the observed functional benefits and further strengthens the evidence that ELEVIDYS is having a positive impact on muscle health at a cellular level.
- Consistent Safety Profile: No new safety signals were identified in Part 2 of the EMBARK trial. This reinforces the previously observed manageable safety profile of ELEVIDYS, which is an important consideration for any gene therapy.
Significance and Next Steps:
These positive topline results from Part 2 of EMBARK provide further compelling evidence supporting the potential of ELEVIDYS to provide long-term, clinically meaningful benefits and improve the quality of life for individuals living with DMD. The fact that benefits were observed in patients who crossed over to treatment later, and that benefits continued to accrue in patients treated earlier, underscores the potential of this gene therapy. Sarepta plans to share detailed results from Part 2 of the EMBARK study at future medical meetings and in peer-reviewed publications. This additional data will provide a more comprehensive understanding of the impact of ELEVIDYS on DMD progression. Sarepta is collaborating with Roche to bring ELEVIDYS to patients worldwide. ELEVIDYS is currently approved in the U.S. and several other countries for individuals with DMD aged four and older, regardless of their ability to walk. The approval for non-ambulatory patients is under accelerated approval, contingent on confirmatory trials demonstrating clinical benefit.
About ELEVIDYS:
ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, intravenously administered AAV-based gene therapy designed to address the underlying genetic cause of DMD—mutations in the DMD gene that lead to a deficiency in the dystrophin protein. ELEVIDYS delivers a transgene that codes for a shortened, but functional, version of the dystrophin protein (micro-dystrophin) to skeletal muscle cells.

Check out our mRNA service to expedite your vaccine research
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
Related News
Navega Therapeutics Receives $4 Million CIRM Grant to Advance Epigenetic Gene Therapy for Chronic Pain
SAN DIEGO, CA – February 4, 2025 – Navega Therapeutics, a pioneering biotechnology company developing cutting-edge epigenetic gene therapies, today announced a significant milestone with the receipt of a $4 million Translational Science grant from the California...
Akribion Therapeutics Secures €8 Million in Seed Financing to Advance Novel RNA-Guided Cell Depletion Technology
ZWINGENBERG, Germany, February 4, 2025 – Akribion Therapeutics, a biotechnology company pioneering a unique, RNA-guided, nuclease-based technology for programmable cell depletion, today announced the closing of an €8 million Seed financing round. The round was led by...
UF-Kure19 CAR-T Cell Therapy Demonstrates High CR Rates, Low Toxicity in R/R NHL
Treatment with UF-Kure19, a rapidly manufactured CAR T-cell therapy, led to complete responses (CR) and low toxicity in patients with relapsed/refractory non-Hodgkin lymphoma, according to data from a single-arm, mult-center phase 1 study (NCT05400109) presented at...
Opinion: Companies Vie to Develop a Hunter Syndrome Therapy That Reaches the Brain
Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio—have products in the pipeline that could improve treatment options for this rare disease. Hunter syndrome is a rare, X‐linked disease caused by a deficiency of the lysosomal enzyme...
Related Services

AAV Packaging Services
READ MORE

Off-the-Shelf AAV Products
READ MORE