Roche Announces Positive Results from Year Two of EMBARK Trial

A Game-Changer in the Treatment of Duchenne Muscular Dystrophy

Basel, 27 January 2025 – Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today positive topline results from year two of the EMBARK trial, a global, randomised, double-blind phase III study of Elevidys™ (delandistrogene moxeparvovec), the first approved gene therapy for the treatment of individuals with Duchenne muscular dystrophy.

Two years after treatment with Elevidys, statistically significant and clinically meaningful improvements were observed across three key motor function measures of NSAA, TTR and 10MWR, when compared to a pre-specified propensity-weighted untreated external control group.* Functional differences between individuals treated with Elevidys and those in the external control group increased between one and two years after dosing. Together, these results demonstrate consistent, sustained benefit in favour of Elevidys.

A New Hope for Patients and Families

The news of the positive results from the EMBARK trial has brought new hope to patients and families affected by Duchenne muscular dystrophy. This breakthrough gene therapy offers a ray of light in the fight against this debilitating disease, providing a glimmer of hope for a brighter future.

Elevidys has the potential to revolutionize the treatment landscape for Duchenne muscular dystrophy, offering a promising alternative to traditional therapies. With its demonstrated efficacy and safety profile, Elevidys represents a significant step forward in the quest to improve the quality of life for patients with this rare genetic disorder.

Impact on Individuals

For individuals living with Duchenne muscular dystrophy, the approval of Elevidys marks a turning point in their treatment journey. This groundbreaking gene therapy has the potential to improve their motor function and overall quality of life, offering new possibilities for a better future.

Global Implications

The positive results from the EMBARK trial are not just significant for individuals with Duchenne muscular dystrophy, but also have far-reaching implications for the field of gene therapy and rare disease research. This breakthrough paves the way for future advancements in the treatment of genetic disorders, offering hope to patients worldwide.

Conclusion

In conclusion, the positive results from the EMBARK trial represent a major milestone in the treatment of Duchenne muscular dystrophy. The approval of Elevidys offers new hope for patients and families affected by this devastating disease, with the potential to transform lives and inspire future innovations in the field of gene therapy. As we celebrate this momentous achievement, let us look towards a brighter future filled with promise and possibility.