Servier acquires treatment for autism genetic cause Fragile X syndrome
Posted: 8 September 2025 | Dominic Tyer (European Pharmaceutical Review) | No comments yet
Deal with Kaerus Bioscience for KER-0193 could be worth $450m to the UK-based biotech.
Servier has strengthened its neurology pipeline with the acquisition of a potential treatment for Fragile X syndrome, the most common genetic cause of autism spectrum disorder.
The French pharma company struck a deal with Kaerus Bioscience that could be worth $450m to the UK biotech for its oral small molecule KER-0193 should all the deal’s development and commercial milestones be reached.
Claude Bertrand, Executive Vice-President of R&D at Servier, said: “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. It reflects our determination to build a differentiated pipeline of innovative therapies for patients with underserved needs.
“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.”
KER-0193 successfully cleared a phase I clinical trial in March 2025 and has both Orphan Drug and Rare Pediatric Drug designations from the US Food and Drug Administration (FDA).
Dr Robert Ring, Chief Executive Officer of Kaerus Bioscience, said: “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.”
There are currently no approved treatments for Fragile X, a rare genetic syndrome of neurodevelopment that’s characterised by a wide range of cognitive and behavioural challenges. It is the most common genetic cause of intellectual disability and the leading single-gene form of autism spectrum disorders, affecting approximately 1 in 7,000 males and 1 in 11,000 females globally.
Co-founded by European life sciences investment firm Medicxi in 2016, Kaerus Bioscience was created with the ambition of developing the first therapy for Fragile X syndrome. The deal with Servier for KER-0193 and Kaerus Bioscience’s wider BK modulator research programme that it’s part of should see KER-0193 move into phase II trials for Fragile X syndrome in the US and Europe next year.
