Servier supercharges neuro pipeline with $450M deal for Fragile X syndrome candidate

Servier is souping up its neurology pipeline with a clinical-stage candidate sourced from U.K. biotech Kaerus Bioscience for a rare genetic disease. The French pharma is offering as much as $450 million for KER-0193, a potential therapy for Fragile X syndrome.

Kaerus will be paid an undisclosed amount upfront for the asset and can also earn additional payments through development and commercial milestones, Servier announced in a Sept. 8 release.

Servier plans to launch a phase 2 clinical trial of KER-0193 in Europe and the U.S. next year, according to the release.

“KER-0193 is Servier’s first asset acquisition in neurology and so marks a significant milestone in our 2030 strategy, reinforcing our long-term commitment to establishing a leading neurology franchise focused on rare diseases,” Claude Bertrand, Ph.D., the pharma’s chief scientific officer and executive vice president of R&D, said in the release. “We are particularly excited to advance KER-0193 as we believe it holds meaningful promise for patients living with Fragile X syndrome, a condition for which no approved treatment options exist today.”

The new candidate joins two preclinical assets in Servier’s pipeline, one for a neurodevelopmental disorder and another for a rare movement disorder.

Fragile X syndrome is caused by mutations in the FMR1 gene, which makes a protein important for brain development. Patients with Fragile X tend to be male and usually develop intellectual disabilities, with seizures also a possible symptom. About one-third of patients with the disease develop autism, making it the most common genetic cause of autism.

Medicxi-backed Kaerus created KER-0193 and has already shepherded it through a phase 1 trial, finding the small molecule to be safe in 56 healthy volunteers. The molecule is designed to restore the function of potassium channels in neurons called BK channels, which usually interact with FMR1 and are disrupted in Fragile X syndrome.

In May, Kaerus announced that KER-0193 had received both an orphan drug designation and a rare pediatric drug tag from the FDA.

The acquisition marks a key step in Servier’s long-term strategy. The company has detailed its ambition to become an innovative, patient-focused and highly profitable pharma by 2030, organized around three main pillars: oncology, cardiometabolism and venous diseases, and neurology and immuno-inflammation.

To that end, Servier has been hungry for deals. Earlier this week, the drugmaker shelled out a hefty $210 million upfront payment for ex-U.S. rights to a potential first-in-class cancer drug from Ideaya Biosciences. That candidate, a protein kinase C inhibitor called darovasertib, is already being put through its paces in several mid- and late-stage trials.

The Ideaya deal was predated by another cancer move in March, when Servier paid $70 million to license a stalled solid tumor candidate from Black Diamond Therapeutics.