Fragile X Research in the News:  Kaerus Bioscience’s Successful Phase 1 Trial

In exciting news for the Fragile X community, Kaerus Bioscience has announced the successful completion of its Phase 1 clinical trial for KER-0193, a novel BK channel modulator being developed to treat Fragile X syndrome (FXS).  This achievement marks a significant milestone in the search for effective treatments for those living with FXS.

What’s the Big News?

The Phase 1 trial demonstrated “proof of mechanism” for KER-0193, meaning that the drug works as expected at a biological level.  More importantly, the trial found that KER-0193 was safe and well-tolerated by participants, which is a crucial step in the development of any new treatment.

This progress is an exciting development in the ongoing efforts to find better treatments for FXS.  As researchers continue to push the boundaries of what is possible, this trial success brings us one step closer to more effective therapies for this neurodevelopmental disorder.

The National Fragile X Foundation talked to Dr. Robert Ring, CEO of Kaerus Bioscience, and he shared:
“Kaerus’s BK channel modulator KER-0193 has been developed specifically to address underlying hyperexcitability of the brain unique to people with Fragile X syndrome.” 
 
“We are tremendously excited by the results of our successfully completed Phase 1 study. It provides the company, our research partners, and importantly participants in future trials, confidence in the safety and tolerability of the drug. The Phase 1 also clearly demonstrated that the drug has effects on relevant measurements of brain activity using EEG. This is very exciting.” 
 
“We are looking forward to working closely with the NFXF, and both the foundation’s Clinical Trial and Family Advisory Committees, as we prepare to move the program into the next phase of testing in patients.”

Why Does This Matter?

For families living with Fragile X, new treatment options are a hopeful sign that progress is being made.  With no cure currently available, every step forward in research offers the possibility of better management and quality of life for individuals with FXS.

Looking Ahead

We look forward to our continued partnership with Kaerus Bioscience as they move forward with this groundbreaking research.  For more information on KER-0193 and Kaerus Bioscience’s work, you can read the full announcement on their website.

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This is an exciting time for Fragile X research, and we’ll continue to bring you the latest updates as progress unfolds.  Stay tuned!

learn more

Visit the RECONNECT MyFXReserach post for more infromation:

Clinical Trial RECONNECT: ZYN002 Gel | MyFXResearch Portal (fragilex.org)

Study: Mechanisms and biomarkers of disease progression in Fragile X-associated tremor/ataxia syndrome (FXTAS)

The University of Kansas BRAIN Lab is conducting a research study to learn about behavioral and brain differences associated with the Fragile X premutation. Males and females ages 50-80 living with the Fragile X premutation, with or without FXTAS, may be eligible to participate. The study includes remote & in-person visits at the University of Kansas.