Servier has struck a deal to buy Edgewise Therapeutics’ muscular dystrophy business for $1.55 billion upfront, gaining control of a program that is on course to deliver pivotal data this year.
Colorado-based Edgewise chose Becker muscular dystrophy (BMD) as the lead indication for sevasemten, an oral drug candidate designed to modulate contraction of fast muscle fibers. Pivotal data in BMD, a rare genetic condition that causes progressive muscle weakness, are expected in the fourth quarter. Edgewise is also running a phase 2 trial of sevasemten in Duchenne muscular dystrophy (DMD).
Edgewise’s pipeline features cardiovascular assets, including the midphase hypertrophic cardiomyopathy candidate EDG-7500. The Servier deal narrows Edgewise’s focus onto the cardiovascular pipeline while giving the biotech the cash to fuel development. Edgewise estimates the deal will fully fund EDG-7500 development through potential approval.
In addition to the upfront fee, Servier has committed up to $1.1 billion in regulatory and commercial milestones to acquire Edgewise’s muscular dystrophy business. Sevasemten is Edgewise’s only molecule in clinical development as a treatment for muscular dystrophy.
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In a note to investors last week, Guggenheim Securities analysts modeled a 55% probability of success for sevasemten in BMD. The forecast reflected mechanistic and functional proof of concept that Edgewise obtained through two clinical trials. In March, the biotech reported persistent stabilization of function for up to 3.5 years and no new safety concerns.
After reviewing the data, Guggenheim analysts said they remained confident in a statistically significant outcome for Grand Canyon, the pivotal BMD cohort scheduled to deliver data this year. The evidence on sevasemten in DMD is less developed, leading the analysts to assign a 25% probability of success to the indication.
Sevasemten has a different mechanism than DMD drugs such as Sarepta Therapeutics’ Exondys 51, which aim to boost levels of functional dystrophin. Rather than targeting the protein at the root of muscular dystrophies, sevasemten seeks to limit contraction of fast skeletal muscle fibers and protect them from injury and fibrosis. The mechanism could slow muscle breakdown and disease progression.
Betting on the hypothesis ahead of pivotal data bolsters Servier’s neurology pipeline while exposing the French drugmaker to the risk of a late-phase failure. Neurology is one of Servier’s three pillars. However, Servier’s neurology pipeline is underdeveloped compared to the other pillars, with three clinical projects versus (PDF) 22 in oncology as of January. Expanding the neurology pipeline is a priority for Servier.
