Servier's $1.55 billion Becker drug fails late stage trial

Servier paid $1.55 billion in cash for sevasemten in July, according to Edgewise Therapeutics’ July 10 filing. On Nov. 3, Servier said the drug failed the late stage trial meant to prove it slows Becker muscular dystrophy.

The study, called GRAND CANYON, showed no statistically significant benefit on its primary measure. It also missed every secondary goal.

What the trial tested

GRAND CANYON ran 18 months and compared sevasemten with a placebo in adults with Becker. The main measure was the North Star Ambulatory Assessment, a standard score of how well patients walk, rise and climb.

The study record on ClinicalTrials.gov planned about 175 adults, randomized two to one toward the drug.

Servier said the drug kept a favorable safety and tolerability profile. It also said the results do not line up with findings from earlier studies, including the smaller Phase 2 CANYON study. The company is reviewing the full dataset and will present complete results later.

The disease

Becker muscular dystrophy is a rare, inherited disease that wears down muscle over time. The faulty gene sits on the X chromosome. Lost muscle function does not come back. There is no approved treatment, Servier noted when it announced the deal.

Sevasemten is a pill. It blocks fast skeletal myosin, a protein that drives quick muscle contractions, to protect fragile muscle from damage during use.

The money

Servier announced the purchase of Edgewise’s muscular dystrophy business on June 1 for up to $2.65 billion. That was $1.55 billion upfront plus up to $1.1 billion in milestones. The deal closed July 10.

The June 1 filing set the milestone terms:

• $200 million for U.S. approval in Becker covering adults and adolescents, or $100 million for adults only.
• $600 million for U.S. approval in Duchenne muscular dystrophy.
• $300 million once yearly U.S. sales top $550 million.

In June, Servier listed a Phase 2 study of sevasemten in Duchenne, the more severe form of the disease. Its Nov. 3 statement did not say how the Becker result affects that work.

What it means for you

For families living with Becker, the treatment gap stays open. Patients in the trial should ask their study site what happens next with their supply and follow up visits.

Care teams should expect questions from patients who followed the earlier Phase 2 data. The honest answer today is that the larger trial did not confirm it.

Health plan and pharmacy leaders tracking rare disease pipelines should move sevasemten out of near term budget models for Becker until Servier releases full data.