A Servier muscular dystrophy drug fell short of the goals of a pivotal study, a surprising outcome that comes months after the company closed the deal for the molecule it acquired to become the centerpiece of its emerging neuromuscular disease strategy.
The drug, sevasemten, was being evaluated as a treatment for Becker muscular dystrophy. Servier’s announcement after Tuesday’s market close provided no details about the results, other than to state that no statistically significant benefit was observed according to the rating scale used to assess muscle movement in activities such as standing up and walking. The privately held company added that the drug did not meet any of the trial’s secondary goals.
Becker muscular dystrophy is caused by an inherited deficiency of dystrophin, a key muscle protein. This rare disease has no FDA-approved treatments. Sevasemten is a small molecule designed to block a protein that leads to fast contractions of muscle fibers that damage muscle. By blocking this protein, sevasemten is intended to protect and preserve muscles.
In results from a placebo-controlled Phase 2 trial called CANYON, the study drug led to a reduction from baseline in circulating levels of a biomarker associated with skeletal muscle damage. This reduction measured at 12 months met the main goal of this part of the study. The trial expanded to add a pivotal cohort named GRAND CANYON that evaluated the study drug for 18 months. The is the part of the study that failed.
“The results of the pivotal GRAND CANYON study are not aligned with certain observations made in previous clinical studies, including the Phase 2 CANYON study,” the company said in its announcement. “Servier is currently conducting an in-depth analysis of all the data from the GRAND CANYON study, as well as relevant prior clinical data, to better understand and more accurately characterize the results of the study.”
Those analyses could be key to determining whether there is readthrough to Duchenne muscular dystrophy, which also stems from lack of dystrophin and has limited treatment options. A separate mid-stage test of sevasemten is ongoing in Duchenne.
Sevasemten came from Edgewise Therapeutics, which conducted the drug’s Phase 2 trial. In June, Servier announced a deal to buy Edgewise’s muscular dystrophy assets for $1.55 billion up front. Edgewise is eligible for up to $1.1 billion in milestone payments, including as much as $200 million tied to regulatory approval in Becker.
The Servier pipeline has early-stage neuromuscular assets from business deals as well as internal R&D. The Edgewise deal was Servier’s first late-stage bet in rare neuromuscular disease, Frederic Scaerou, Servier’s global head of business development, said in an interview during the BIO International Convention in June. He described sevasemten as a “late-stage anchor” around which Servier could build out its neuromuscular indications.
“If Edgewise works, we will look at then additional Phase 3, Phase 2, Phase 1, preclinical assets in the same disease area so that we start building our expertise, experience, network in this area,” Scaerou said.
The Edgewise transaction was Servier’s second big M&A move this year. In March, Servier agreed to pay $2.5 billion to buy Day One Biopharmaceuticals, which brought a commercialized brain cancer drug and a pipeline of antibody drug conjugates. Servier has been active in business development as part of a strategy to increase annual revenue to €10 billion (about $11.6 billion) by 2030. For the fiscal year that ended Sept. 30, 2025, Servier reported €6 billion (about $8 billion) in revenue.
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