Servier has confirmed that its recently acquired muscular dystrophy drug has failed its first Phase III test.
In the GRAND CANYON study (NCT05291091), which investigated sevasemten in patients with Becker muscular dystrophy (BMD), the study did not meet the primary endpoint, with no statistically significant benefit on the North Star Ambulatory Assessment (NSAA). Secondary endpoints were also not met.
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Servier only got its hands on the muscular dystrophy drug five months ago as part of its $1.55bn buyout of Edgewise Therapeutics’ muscular dystrophy business. The company paid $1.55bn upfront, with a potential further $1.1bn in regulatory and commercial milestone payments.
While the efficacy results were not favourable in the 18-month global placebo-controlled trial, the study did demonstrate a continued favourable safety and tolerability profile.
According to Servier, the results of the pivotal GRAND CANYON study do not align with certain findings observed in prior clinical studies, including the Phase II CANYON study.
As a result, Servier is conducting a comprehensive analysis of the GRAND CANYON study dataset, together with relevant prior clinical data, to better understand and further characterise the study findings. Full data are set to be presented in the future.
Commenting on the results, a spokesperson for the Muscular Dystrophy Association (MDA) said: “While we are disappointed that this trial did not demonstrate efficacy in adults with BMD, clinical research advances our understanding of the disease and informs the path forward. The GRAND CANYON study has contributed to advancements in outcome measures and clinical trial design that can help inform future research. We learn from every trial conducted, and the knowledge gained is an important part of advancing the science and developing future treatment opportunities for the BMD community.”
Sevasemten is an investigational orally administered first-in-class fast skeletal myosin inhibitor designed to preserve and protect unstable muscle against contraction-induced damage in individuals living with rare muscular dystrophy.
If successful, it could become the very first approved therapy for patients with BMD, answering a significant unmet need. Currently, treatment focuses on managing symptoms, preserving muscle function, and protecting heart and lung health.
Sevasemten is also being investigated in a Phase II trial in Duchenne muscular dystrophy (DMD). The LYNX study (NCT05540860) has enrolled 76 patients with DMD aged four to nine years and is due to complete in January 2027.
Both DMD and BMD are X-linked genetic disorders caused by mutations in the same gene that affects the production of dystrophin – a protein crucial for protecting muscle fibres.
GlobalData analysis predicts that the DMD market across the seven major markets (7MM: US, France, Germany, Italy, Spain, UK, Japan) will grow from $2.3bn in 2023 to $5.2bn in 2033. There is no comparable analysis for the BMD market.
GlobalData is the parent company of Clinical Trials Arena.
