Kaerus wins two FDA designations for KER-0193 in Fragile X syndrome

UK-based Kaerus Bioscience, a biopharma created by European investment firm Medicxi for the development of therapeutics for rare genetic syndromes of neurodevelopment, has announced that its lead candidate KER-0193 has been granted both Orphan Drug designation and Rare Pediatric Drug designations for the treatment of Fragile X syndrome (FXS) by the US Food and Drugs Administration (FDA).

This follows the recent successful completion of a Phase I trial of KER-0193 in healthy volunteers, which confirmed the drug to be safe, well tolerated and exhibit excellent pharmacokinetics.

FXS is an inherited condition, affecting about one in 7,000 boys and one in 11,000 girls, for which there are currently no approved treatments. It is the most common cause of inherited autism and intellectual disability, accounting for around 1% of such cases globally.