You are here: Home / News / Gene therapy / Roche Shares Top-line Results from Year Two of the EMBARK Phase 3 Clinical Trial.
Roche Shares Top-line Results from Year Two of the EMBARK Phase 3 Clinical Trial.

March 12, 2025 by John Marrin

Roche have provided an update on the latest top-line results from the second year of their EMBARK Phase 3 clinical trial (NCT05096221) investigating delandistrogene moxeparvovec for boys with Duchenne muscular dystrophy (DMD) aged 4-7.

EMBARK Clinical Trial Results:

The phase 3 EMBARK trial evaluated Elevidys in 125 ambulant boys aged 4-7 with DMD. Participants were randomly assigned to receive either Elevidys (63 boys) or a placebo (59 boys).

Initial 52-Week Findings:

  • As communicated in October 2023, the trial did not meet its primary endpoint, which was to demonstrate a statistically significant improvement in motor function as measured by the NorthStar Ambulatory Assessment (NSAA) score. While Elevidys showed improvement, the results could not be shown to be statistically significant.
  • However, Elevidys did demonstrate statistically significant improvements in secondary endpoints, specifically in time to rise from the floor and 10-meter walk test.

Two-Year Follow-up Data:

  • After two years, participants receiving Elevidys showed improvements in NSAA scores compared to an external control group. This external control group consisted of data from individuals with DMD, gathered from other trials and natural history studies, who had shared characteristics but did not receive Elevidys.
  • It’s important to acknowledge that comparing trial data to external data has limitations. A placebo-controlled comparison would provide more robust evidence, but ethical considerations regarding long-term placebo use are a factor.
  • Further results from the EMBARK trial will be released as they become available.

Next Steps:

  • Boys participating in the EMBARK trial will have the option to continue follow-up for at least five years for safety and clinical outcomes.
  • Full safety and efficacy data from year two will be presented at an upcoming medical conference.

Implications for the UK:

All medicines in the UK require a license from the Medicines and Healthcare products Regulatory Agency (MHRA). However, to gain access to the treatment via the NHS, further assessments by bodies such as the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) are needed for NHS availability.

Licensing decisions are ordinarily reliant on the primary endpoint of a clinical trial being met. Whilst EMBARK did not meet its primary endpoint, Roche remain committed to discussing the year one and year two results with the relevant authorities in the UK and continue to evaluate the available options to determine the best path forward for a potential licence and reimbursement for delandistrogene moxeparvovec in the UK. 

We will continue to provide updates as more information becomes available.

Share this: