FRAXA Research Foundation recently spoke with Robert Ring, PhD, Chief Executive Officer of Kaerus Bioscience, about their investigational new drug for Fragile X syndrome. Kaerus Bioscience is working on BK channel openers, which may help reduce core symptoms of Fragile X. Phase 1 clinical trials are underway in Belgium to test the safety of KER-0193 in healthy volunteers. If all goes well, Phase 2 clinical trials for individuals with Fragile X could begin in 2025, moving one step closer to an innovative new treatment.
A Master Regulator of Excitability
BK channels, also known as large-conductance calcium-activated potassium channels, play a critical role in regulating brain activity. “In Fragile X, we know that hyper-excitability contributes to many of the features that patients struggle with,” Ring said. This over-excitability leads to hyperactivity, anxiety, and sensory overload. BK channels help control how neurons send electrical signals. When they don't function properly, the nervous system becomes overstimulated, as seen in Fragile X syndrome.
Kaerus’ lead compound, KER-0193, is a BK channel opener. “Our lead program is focused on this unique potassium channel in the brain, a master regulator of excitability across the nervous system,” said Ring. By opening these channels, KER-0193 works to calm the brain’s overactivity. Preclinical testing has shown encouraging reductions in hyperactivity and anxiety behaviors in animal models of Fragile X, offering hope that these results could translate to human patients.
From Lab to Clinical Trials
This new drug from Kaerus emerged from years of research supported by FRAXA. FRAXA’s involvement with BK channel research dates back more than a decade, with early studies by Dr. Peter Vanderklish at Scripps Research Institute and Dr. Vitalie Klyachko at Washington University. Then Dr. Silvain Briault and Dr. Jacques Pichon at the University of Orleans went on to launch Kaerus based on discoveries they made, with FRAXA funding. “Our co-founders received a FRAXA grant over ten years ago working on this BK channel story,” Ring explained.
Recent testing at FRAXA’s Drug Validation Initiative (FRAXA-DVI) at the University of Chile demonstrated further success, paving the way for the drug’s transition into clinical trials.
FRAXA remains committed to collaborating with Kaerus as they continue to develop KER-0193. Collaborations such as this are essential to our mission to bring effective treatments to families affected by Fragile X syndrome.
