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Potential treatment for Fragile X syndrome expected to enter Phase 2 trial in America and Europe.
Servier, an international pharmaceutical group, has entered into a definitive agreement with Kaerus Bioscience to acquire KER-0193, a potential treatment for Fragile X syndrome (FXS), the most common genetic cause of autism spectrum disorder (ASD), in a deal valued at up to $450 million.
KER-0193 was discovered and developed by Kaerus Bioscience, a UK headquartered biotechnology company co-founded by Medicxi in 2016. A Phase 1 clinical study of KER-0193 in healthy volunteers has demonstrated the treatment to be safe and well tolerated. Additionally, KER-0193 has been granted both Orphan Drug Designation and Rare Pediatric Drug Designations for the treatment of FXS by the U.S. FDA.
Under the agreement, Kaerus Bioscience will receive an upfront payment for the sale of KER-0193 and will also be eligible for development and commercial earn-out payments.
KER-0193 is an orally bioavailable small molecule modulator of BK channels that specifically addresses abnormal function of BK channels linked to FXS. In preclinical studies, KER-0193 has already demonstrated broad therapeutic-like effects on improving syndrome-relevant behavioral, sensory and cognitive deficits. As part of the development strategy, Servier will prepare the launch of a Phase 2 clinical trial in 2026 in FXS patients in America and Europe.
FXS is a rare syndrome of neurodevelopment characterized by a wide range of cognitive and behavioral challenges. It is the most common genetic cause of intellectual disability and the leading single-gene form of autism spectrum disorder (ASD), affecting approximately 1 in 7,000 males and 1 in 11,000 females globally. There are currently no approved treatments for FXS, which accounts for approximately 1% of ASD and intellectual disability cases globally.
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