Servier wagers $2.65bn on Edgewise muscular dystrophy drug

Servier has signed the largest licensing deal in its history, paying $1.55 billion upfront for Edgewise Therapeutics' muscular dystrophy business, headed by late-stage therapy sevasemten.

The French pharma group is on the hook for another $1.1 billion in regulatory and commercial milestones, including up to $200 million on US approval of the drug in lead indication, Becker muscular dystrophy (BMD), and $600 million if it gets an FDA green light to extend its use into Duchenne muscular dystrophy (DMD).

Another $300 million would be payable if sevasemten makes US sales above $550 million in one year, according to an Edgewise financial filing.

Servier said in a statement that sevasemten could be a first-in-class oral myosin inhibitor for the two forms of the muscle-wasting disease and position the company as "a global player with strong capabilities and a pipeline in neuromuscular disorders."

It is in the pivotal GRAND CANYON trial in BMD, a rare, X-linked disorder that causes slowly progressive muscle degeneration and weakness, primarily affecting the legs, pelvis, and heart, with results expected in the fourth quarter of this year.

Edgewise is also testing the drug in two phase studies in DMD, which tends to be more severe. The dose-ranging LYNX trial is comparing sevasemten to placebo, while FOX is enrolling patients who have previously been treated with Sarepta and Roche's DMD gene therapy Elevidys (delandistrogene moxeparvovec).

Servier's president, Olivier Laureau, said the acquisition is a key component of the company's ambitious 2030 growth plan, which has been built around growing its presence in neurology and cancer and has also just seen it agree to buy Day One Biopharmaceuticals and its fast-growing brain cancer therapy Ojemda (tovorafenib) for up to $2.5 billion.

"Developing treatments for young and adult patients, with highly debilitating rare conditions and limited to no options today, is at the heart of our mission," added Laureau.

For Edgewise, handing over the muscular dystrophy business will allow it to focus its attention on its cardiac sarcomere modulator pipeline, which includes EDG-7500 in phase 2 for hypertrophic cardiomyopathy and EDG-15400 for heart failure, due to start phase 2 later this year.

The company was already pretty well-funded as it had had around $500 million in cash at the end of the first quarter, and if the Servier deal completes as planned in the third quarter, it will have plenty of resources to advance its cardiovascular assets through development.

"Servier's focus on precision therapeutics and its dedication to rare neurological and neuromuscular diseases make them the ideal steward for the programme," said Edgewise chief executive Kevin Koch.

"This transaction delivers immediate, significant value…while placing sevasemten with an acquirer that has the global scale, patient commitment, and commercial reach to maximise its potential," he added.