Kaerus Bioscience Successfully Completes Phase 1 Trial and Demonstrates Proof of Mechanism with its Novel BK Channel Modulator KER-0193 being developed for Fragile X Syndrome

LONDON, March 11, 2025 (GLOBE NEWSWIRE) — Kaerus Bioscience (www.kaerusbio.com), a clinical-stage biopharmaceutical company created by Medicxi for the development of therapeutics for rare genetic syndromes of neurodevelopment, today announces the successful completion of its Phase 1 clinical trial of KER-0193, a novel BK channel modulator the company is developing for Fragile X syndrome (FXS) and other neurodevelopmental conditions.

In the trial, KER-0193 was found to be safe and well-tolerated in both single ascending dose and multiple ascending dose cohorts involving 56 healthy volunteers. The drug also exhibited an excellent, dose-proportional pharmacokinetic profile across a wide range of doses.

A planned biomarker substudy profiling brain activity using electroencephalography (EEG) was also completed as part of the Phase 1 clinical trial. The results of this pharmaco-EEG study revealed significant pharmacodynamic effects of KER-0193 on multiple, translationally-relevant parameters of brain activity, providing clear clinical evidence of central target engagement for KER-0193.

Pharmaco-EEG also revealed a unique topography of KER-0193 effects on brain excitability that specifically maps to cortical regions commonly reported as abnormal by EEG in patients with FXS. This pattern of regional effects on brain excitability replicates observations from EEG profiling of KER-0193 in preclinical animal studies, demonstrating proof of mechanism.

“These results are a significant milestone for Kaerus,” said Dr. Robert Ring, CEO, Kaerus Bioscience. “The data clearly demonstrate our first-in-class BK channel modulator is active in the CNS, and is both safe and well-tolerated. Taken together with the positive ‘proof of mechanism’ with EEG, we feel extremely excited and confident heading into Phase 2 in the target population.”

The company is currently finalizing preparations for a Phase 2 proof of concept study in FXS patients.

About Kaerus Bioscience
Kaerus Bioscience Ltd is a clinical-stage biotechnology company committed to turning scientific advances into treatment realities for patients with rare genetic syndromes of neurodevelopment. Kaerus has developed a pipeline of targeted, small-molecule therapeutics that address an underlying ion channel dysfunction in Fragile X syndrome, the most common inherited cause of intellectual disability and autism globally.

About KER-0193
KER-0193 is a novel, proprietary, and orally-bioavailable small molecule modulator of BK channels discovered by Kaerus. KER-0193 specifically addresses abnormal function of BK channels linked to the genetic cause of Fragile X syndrome. KER-0193 has already demonstrated broad effects on improving syndrome-relevant behavioral, sensory and cognitive deficits observed in genetic animal models of Fragile X. Fragile X syndrome is the most common inherited cause of autism and intellectual disability globally. There are currently no approved treatments for Fragile X.

About Medicxi
Medicxi is a healthcare-focused investment firm with the mission to create and invest in companies across the full drug development continuum. Leveraging deep expertise in drug development and company creation spanning over two decades, Medicxi invests in early and late-stage therapeutics with a product vision that can fulfil a clear unmet medical need. For more information, please visit: https://www.medicxi.com

Contact
For more information, please visit www.kaerusbio.com or contact info@kaerusbio.com