Les Laboratoires Servier said that it agreed to acquire a potential treatment for Fragile X syndrome (FXS), the most common genetic cause of autism spectrum disorder (ASD), from its original developer, Kaerus Bioscience, for up to $450 million.
The deal for KER-0193 comes six months after its initial discoverer and developer, Kaerus Bioscience, generated positive results in a Phase I trial in 56 healthy volunteers, which showed the oral small molecule modulator of BK channels to be safe and well tolerated with excellent pharmacokinetics across a range of doses.
Kaerus also said KER-0193 demonstrated proof of mechanism in a biomarker substudy that profiled brain activity using electroencephalography (EEG). Specifically, KER-0193 generated pharmaco-EEG data showing a topography of effects on brain excitability that specifically mapped to cortical regions commonly reported as abnormal by EEG in patients with FXS.
According to Kaerus, that finding replicated observations from EEG profiling of KER-0193 in preclinical animal studies, in which the drug candidate showed broad therapeutic-like effects on improving syndrome-relevant behavioral, sensory, and cognitive deficits in genetic animal models of Fragile X.
At the time it announced the Phase I results in March, Kaerus said it was finalizing preparations for a Phase II proof of concept study of KER-0193 in FXS patients. Servier said it will prepare the launch of a Phase II clinical trial in 2026 in FXS patients in the United States and Europe.
In May, the FDA granted KER-0193 both its Orphan Drug and Rare Pediatric Drug designations for the treatment of FXS, which is not only the most common cause of inherited autism but of intellectual disability as well.
There are currently no approved treatments for FXS, an inherited condition that affects about 1 in 7,000 males and 1 in 11,000 females. FXS accounts for an estimated 1% to 6% of ASD, according to a 2017 study.
Servier said its acquisition of KER-0193—until now Kaerus’ lead pipeline candidate—showed its commitment to building a neurology franchise by expanding its pipeline with assets targeting rare indications for patients with high unmet need.
That pipeline is now dominated by oncology programs, of which nine are designed to treat solid tumors and the other six, blood cancers. Servier’s pipeline also includes two preclinical neurology programs: A neurodevelopmental disorder candidate designated as Project S230815 and an unspecified rare movement disorder candidate designated as Project S233107.
2030 strategy
“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, executive vice president of R&D at Servier, said in a statement.
Servier’s 2030 strategy commits the French-based pharma to addressing global therapeutic needs by developing treatments in therapeutic areas that include neurology as well as oncology, cardiometabolism and venous diseases, and immuno-inflammation.
“Furthermore, we aim to develop a third therapeutic area, in addition to oncology and cardiovascular, metabolism and venous diseases, neurology, an area in which needs are on the rise,” Servier stated on its website.
Through its strategy, Servier aims to generate global revenue of €10 billion ($11.7 billion) by 2030, of which €4 billion ($4.7 billion) would come from branded drugs in neurology and oncology combined. Another €4 billion is expected to come from cardiometabolism and venous disease treatments, with the remaining €2 billion ($2.35 billion) to come from generic medicines.
Servier has agreed to pay Kaerus an undisclosed upfront payment, plus additional potential payments tied to achieving development and commercial milestones.
“We strongly believe in the therapeutic potential of KER-0193 in Fragile X syndrome. Servier’s firm commitment to neurology and global capabilities make it the ideal partner to further develop KER-0193 for patients worldwide,” stated Kaerus CEO Robert Ring, PhD.
Kaerus is a U.K.-based biotech focused on developing treatments for FGX and other rare genetic neuro disorders. The company was co-founded in 2016 by Medicxi, a European investment firm with a reported $1.4 billion in assets under management.
“Kaerus Bioscience was created by Medicxi to advance a clear product vision to develop a potential first-in-disease therapy for Fragile X syndrome,” added Michèle Ollier, venture partner and co-founder of Medicxi. “We are extremely proud of the cutting-edge scientific progress achieved by the Kaerus team under Dr. Ring’s leadership and are excited that this deal with Servier will accelerate the development of KER-0193, bringing us closer to ensuring this innovative new therapeutic can reach those patients who are most in need.”
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