UK-based Kaerus Bioscience has received US Food and Drug Administration’s (FDA) rare paediatric drug and orphan drug designations for its orally bioavailable small molecule, KER-0193 to treat Fragile X syndrome (FXS).
FXS is the most prevalent cause of inherited autism and intellectual disability.
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The announcement comes after a Phase I trial completion that confirmed the therapy’s safety, tolerability and favourable pharmacokinetics.
KER-0193, discovered by the company, is designed to address hyper-excitability in the brain, a common issue associated with FXS, by targeting BK channels, which are crucial in regulating nervous system excitability.
Its therapeutic potential is supported by its wide efficacy profile in preclinical genetic models of FXS.
Kaerus is optimistic about the therapy’s ability to address the diverse behavioural, cognitive and sensory challenges faced by those with FXS.
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By GlobalDataThe company’s belief is further supported by a pre-planned sub-study carried out during the Phase I trial, which used electroencephalography to investigate KER-0193’s effects on brain activity.
The study indicated that KER-0193 can penetrate the brain and impact brain activity by producing pharmacodynamic effects in areas often affected in FXS patients, providing proof of mechanism.
The implications of decreased BK channel activity extend beyond FXS, as it is also associated with epilepsy and various rare genetic epileptic encephalopathies.
Kaerus is preparing for a Phase II proof-of-concept trial for individuals with FXS.
Kaerus Bioscience CEO Dr Robert Ring stated: “The FDA’s granting of orphan drug designation and rare paediatric drug designation for KER-0193 is an important step towards our objective of delivering an effective treatment for people with FXS.
“Not only do these two FDA designations affirm the therapeutic potential of KER-0193 in FXS, they also provide Kaerus with access to significant regulatory and financial incentives as we look to progress the drug through clinical trials and ultimately take it to market.”
