Drug From Servier's $1.55B Deal Misses Goal in 175-Patient Study

A muscular dystrophy drug that drugmaker Servier bought from Edgewise Therapeutics has failed its pivotal study. Servier said on Nov. 3 that sevasemten did not meet the main goal of the GRAND CANYON study in Becker muscular dystrophy.

The result comes less than four months after Servier completed the deal. It paid $1.55 billion upfront for Edgewise's muscular dystrophy business.

What happened

GRAND CANYON was a global, placebo-controlled study that ran for 18 months. According to European Biotechnology, it enrolled 175 adults with Becker muscular dystrophy.

The main measure was the North Star Ambulatory Assessment, a scale of motor function. Servier said the drug showed no statistically significant benefit on that scale. It also missed its secondary goals.

Servier said the drug's safety and tolerability were favourable over the course of the study. The company has not shared detailed efficacy numbers. It said it is analyzing the GRAND CANYON data alongside data from earlier studies, and will present full results later.

• Drug: sevasemten, an oral small molecule designed to protect muscle from damage caused by contraction.
• Deal: $1.55 billion upfront, plus up to $1.1 billion in milestones, according to MedCity News and European Biotechnology.
• Closing: the deal completed July 13, European Biotechnology reported.

Why it matters

Becker muscular dystrophy is caused by an inherited lack of the protein dystrophin. MedCity News reported that it has no FDA-approved treatments.

Part of the deal's value depended on Becker. MedCity News reported that up to $200 million of the milestones was tied to regulatory approval in Becker.

Earlier data had looked more hopeful. In the Phase 2 CANYON study, the drug cut a muscle damage marker over 12 months, which was that study's main goal. But its effect on the North Star scale was not statistically significant, with a p value of 0.16, according to European Biotechnology.

Servier itself said the new results do not line up with some findings from earlier studies, including CANYON, MedCity News reported.

In an interview at the BIO convention in June, Frederic Scaerou, Servier's global head of business development, called sevasemten a "late-stage anchor," MedCity News reported. Servier, which is privately held, also agreed in March to pay $2.5 billion for Day One Biopharmaceuticals.

What it means for people in the industry

A mid-stage study of sevasemten in Duchenne muscular dystrophy is still running. Servier has given no word yet on what the Becker miss means for that work, so its next update on Duchenne is the one to watch.

Also watch for the full GRAND CANYON data, which Servier said it will present at a later date.

See open pharma jobs in the US and Canada

Sources

• Servier Announces Topline Results from Pivotal Sevasemten Study in Becker, Parent Project Muscular Dystrophy
• Servier Stumbles in Muscle Disease as Drug From $1.5B Deal Fails Pivotal Test, MedCity News
• Servier's $1.55B Edgewise acquisition backfires after Phase 2 setback, European Biotechnology