Servier’s decision to bet $1.55 billion on Edgewise Therapeutics’ muscular dystrophy program ahead of pivotal data has backfired. Five months after striking the deal, the French drugmaker has reported that a Becker muscular dystrophy (BMD) trial missed its primary and secondary endpoints.
Edgewise designed the pivotal cohort of its phase 2 trial to compare sevasemten to placebo in 175 adults with BMD, a genetic condition that causes progressive muscle weakness. After 18 months of daily oral dosing, patients receiving the study drug performed statistically no better than their peers on placebo on the North Star Ambulatory Assessment scale, causing the trial to miss its primary endpoint. The study also missed secondary endpoints.
The pivotal results are not aligned with certain observations made in previous clinical studies, including the prior part of the phase 2 trial, Servier said. The company is analyzing the pivotal data, plus relevant prior clinical results, to better understand and more accurately characterize the findings. Servier has yet to publish any data, but plans to share the full results later.
Sevasemten maintained a favorable safety and tolerability profile, but otherwise, Servier’s topline readout is devoid of encouraging findings. The drugmaker said it remains fully committed to bringing innovations for rare neurological diseases to patients. However, the failure of the BMD trial eliminates a key part of its current neurological disease pipeline.
Neurology is one of Servier’s three pillars. Yet the company has fewer clinical-phase assets in neurology than in its other core areas of oncology and cardiometabolic diseases. Servier has prioritized expanding its neurology pipeline, leading to the bet on sevasemten.
The bet always looked risky. In a note to investors back in May, Guggenheim Securities analysts modeled a 55% chance of success for sevasemten in BMD, reflecting mechanistic and functional proof-of-concept data generated in earlier trials. The analysts assigned a 25% probability of success to Duchenne muscular dystrophy (DMD), an indication where a pair of trials are scheduled to finish next year.
Sevasemten’s failure in BMD dents Edgewise’s chances of receiving more money from Servier. The deal features up to $1.1 billion in milestones, including $200 million tied to the U.S. approval of the candidate in BMD. Servier will pay a further $600 million if the FDA approves sevasemten for DMD, with the remaining $300 million tied to annual U.S. net sales.
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