Servier, an independent international pharmaceutical group run by a foundation, has announced a final agreement to buy KER-0193 from Kaerus Bioscience. KER-0193 is a promising treatment for Fragile X syndrome, the main genetic cause of autism spectrum disorder. This move highlights Servier’s focus on expanding its neurology portfolio by targeting rare diseases with significant unmet medical needs. KER-0193 was developed by Kaerus Bioscience, a UK-based biotech co-founded by Medicxi in 2016. In March 2025, Kaerus completed a Phase 1 trial in healthy volunteers showing the treatment to be safe, well-tolerated, and having favorable drug behavior. The U.S. Food and Drug Administration granted KER-0193 Orphan Drug and Rare Pediatric Drug designations for Fragile X syndrome, emphasizing its potential for this rare disorder.
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Under the terms of the agreement, Kaerus Bioscience will receive an upfront payment and may earn additional funds based on development milestones and commercial success, with the total deal value possibly reaching $450 million. Claude Bertrand, Executive Vice-President of Research and Development at Servier, described KER-0193 as the company’s first neurology asset acquisition. He called this a crucial step in Servier’s 2030 strategy to build a strong neurology business focused on rare diseases. Bertrand expressed hope that KER-0193 could provide new hope for patients with Fragile X syndrome, a condition currently lacking any approved treatments.
Dr. Robert Ring, Chief Executive Officer of Kaerus Bioscience, expressed strong confidence in the therapeutic potential of KER-0193 for Fragile X syndrome. He noted that Servier’s expertise in neurology and its global reach make it an ideal partner to advance KER-0193’s development and bring it to patients worldwide. Michèle Ollier, Venture Partner and co-founder of Medicxi, expressed pride in the scientific achievements of Kaerus Bioscience under Dr. Ring’s leadership. She emphasized that this agreement with Servier will accelerate the development of KER-0193 and bring the potential treatment closer to patients in need.
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KER-0193 is an orally available small molecule drug that targets BK channels to correct their abnormal function associated with Fragile X syndrome. Preclinical studies have demonstrated that KER-0193 improves behavioral, sensory, and cognitive deficits linked to the syndrome. Servier plans to launch a Phase 2 clinical trial in 2026, focusing on Fragile X patients in the United States and Europe. This trial is a significant step toward developing an effective treatment for the condition.
Fragile X syndrome is a rare genetic neurodevelopmental disorder that causes a wide range of cognitive and behavioral challenges. It is the most common inherited cause of intellectual disability and the leading single-gene cause of autism spectrum disorder. Worldwide, Fragile X affects approximately one in 7,000 males and one in 11,000 females. Currently, there are no approved therapies for Fragile X syndrome, which accounts for about one percent of autism spectrum disorder and intellectual disability cases globally.
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