Kaerus Bioscience Achieves Key FDA Designations for Fragile X Syndrome Therapy
Kaerus Bioscience has announced a significant milestone in its mission to tackle fragile X syndrome (FXS), with the U.S. Food and Drug Administration (FDA) granting both orphan drug designation and rare paediatric disease designation for its lead candidate, KER-0193.
The designations mark a pivotal step forward for the biotech firm, following the successful completion of a phase 1 clinical trial that confirmed the drug’s safety and encouraging pharmacokinetics.
Fragile X syndrome is the most common inherited cause of autism and intellectual disability, affecting approximately 1 in 7,000 males and 1 in 11,000 females. Despite its prevalence, there are currently no approved treatments for the condition – an unmet medical need Kaerus aims to address.
KER-0193 is a novel, orally bioavailable small molecule designed to modulate BK (Big Potassium) channels, which are critical in regulating neuronal excitability throughout the nervous system.
In individuals with FXS, reduced BK channel activity is believed to contribute to the neurological dysfunctions commonly observed, including sensory hypersensitivity, cognitive impairments, and behavioural issues.
The phase 1 trial included a pre-planned electroencephalography (EEG) sub-study that assessed the compound’s ability to penetrate the brain and elicit a pharmacodynamic response.
Results demonstrated that KER-0193 successfully entered the brain and produced measurable effects in regions associated with FXS pathology. These findings mirrored earlier preclinical animal studies and offer a strong foundation for the drug’s continued development.
The CEO of Kaerus Bioscience commented that they are pleased that the FDA has recognised the promise of KER-0193 by granting both orphan drug and rare paediatric disease designations. This is an important validation of their scientific approach and brings them closer to providing an effective therapy for individuals and families living with fragile X syndrome.
The company is now preparing for a phase 2 proof-of-concept study to further evaluate the therapeutic potential of KER-0193 in FXS patients. Beyond this lead indication, Kaerus is exploring broader applications of its BK channel modulator platform.
Reduced BK channel activity is also implicated in a number of other neurological disorders, including epilepsy and rare genetic epileptic encephalopathies – offering exciting avenues for future clinical research and therapeutic expansion.
Conclusion
With its recent FDA designations and promising early clinical results, Kaerus Bioscience is forging a path toward a much-needed therapy for fragile X syndrome.
The development of KER-0193 not only brings new hope to patients and families affected by this challenging condition but also signals broader potential for BK channel modulation in treating complex neurological disorders.
As the company advances into phase 2 trials, it continues to cement its role at the forefront of neuroscience innovation.
News Credits: Kaerus Bioscience secures orphan drug designation for fragile x syndrome treatment
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