Roche has revealed encouraging topline results from the second year of the EMBARK trial, a global, randomized, double-blind Phase III study evaluating Elevidys (delandistrogene moxeparvovec), the first approved gene therapy for Duchenne muscular dystrophy (DMD).
The trial demonstrates significant, sustained improvements in key motor functions for children treated with Elevidys, supporting its potential as a disease-modifying treatment for DMD.
Significant Improvements in Motor Function
After two years of treatment, Elevidys showed statistically significant and clinically meaningful improvements in three crucial motor function measures: the North Star Ambulatory Assessment (NSAA), time to rise (TTR), and the 10-meter walk/run (10MWR).
These improvements were compared to a pre-specified propensity-weighted untreated external control group. Importantly, the differences between individuals treated with Elevidys and those in the control group continued to increase over the second year, highlighting the sustained benefit of Elevidys.
Promising Results from Year Two
The detailed results from the second year of the EMBARK study will be presented at an upcoming medical meeting and discussed with health authorities. The one-year data from part one of the EMBARK study were previously published in Nature Medicine in October 2024.
Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer, emphasized the significance of these results. “These sustained benefits in standing, walking, and running demonstrate Elevidys’ disease-modifying potential for children with Duchenne,” Garraway stated. “We are eager to share these findings with health authorities as quickly as possible.”
Sustained Benefits in Functional Motor Improvements
In part one of the EMBARK study, 63 children received Elevidys and showed meaningful improvements in their NSAA scores, which remained significant even two years post-treatment.
Professor Francesco Muntoni, Director of the Dubowitz Neuromuscular Centre at Great Ormond Street Hospital for Children, UK, highlighted the importance of these findings.
He noted, “As Duchenne progresses, children lose the ability to walk and face severe health challenges. These encouraging results from year two of the EMBARK trial suggest that innovative treatments like Elevidys could extend mobility and independence, easing the physical and emotional burden on families.”
Similar Benefits for Crossover Group
Participants in part two of the EMBARK study, who were previously treated with a placebo in part one, received Elevidys after 52 weeks. Despite being one year older than the original Elevidys group, the crossover group (59 participants) experienced similar motor function improvements after one year of treatment. These findings indicate that Elevidys can deliver consistent benefits even when administered later in the disease progression.
Continued Micro-Dystrophin Expression and Minimal Muscle Progression
A subset of muscle biopsies from part one participants at 64 weeks after treatment revealed sustained expression of micro-dystrophin, a protein crucial for muscle function. Additionally, MRI scans showed minimal progression of muscle pathology, consistent with the observed functional improvements.
Safety Profile Remains Consistent
No new safety signals emerged from the study, reinforcing Elevidys’ manageable safety profile. The data continues to support the one-time treatment’s safety and efficacy in individuals with Duchenne.
Regulatory Approvals and Global Availability
Elevidys is approved in several countries for individuals aged four and older, regardless of ambulatory status, including the US, United Arab Emirates, Qatar, Kuwait, Bahrain, and Oman. In Brazil and Israel, Elevidys is approved for ambulatory individuals aged four to seven. Applications for approval are pending in the European Union, Japan, Switzerland, Singapore, Hong Kong, and Saudi Arabia.
As reported by pharmabiz.com, in 2019, Roche entered into a global collaboration with Sarepta Therapeutics, Inc. to commercialize Elevidys outside the US.