Kaerus Bioscience announced today that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation and Rare Pediatric Drug Designation to its lead candidate KER-0193 for the treatment of Fragile X syndrome (FXS). These significant regulatory milestones follow the recent successful completion of Phase 1 clinical trials and position the company to advance its novel therapeutic approach for this rare genetic condition.
Fragile X syndrome affects approximately 1 in 7,000 males and 1 in 11,000 females globally, making it the most common inherited cause of autism and intellectual disability. Despite its prevalence, there are currently no FDA-approved treatments specifically for FXS, highlighting the substantial unmet medical need.
Novel Mechanism Targeting Underlying Genetic Dysfunction
KER-0193 represents a pioneering approach to treating Fragile X syndrome by targeting BK channels (calcium-activated potassium channels), which function as master regulators of excitability throughout the nervous system. The genetic cause of FXS is directly linked to reduced BK channel function, resulting in the hyperexcitability of brain function characteristic of the condition.
Dr. Robert Ring, CEO of Kaerus Bioscience, explained the significance of the FDA designations: "The FDA's granting of Orphan Drug Designation and Rare Pediatric Drug Designation for KER-0193 is an important step towards our objective of delivering an effective treatment for people with Fragile X syndrome. Not only do these two FDA designations affirm the therapeutic potential of KER-0193 in Fragile X syndrome, 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."
Promising Phase 1 Results with Proof of Mechanism
The Phase 1 trial of KER-0193 in healthy volunteers confirmed that the drug is safe, well-tolerated, and exhibits excellent pharmacokinetics. Notably, the trial included a pre-planned sub-study using electroencephalography (EEG) to investigate the effects of KER-0193 on brain activity.
This EEG study provided compelling evidence that KER-0193 successfully crosses the blood-brain barrier and produces pharmacodynamic effects in brain regions often implicated in FXS patients. These findings replicated observations from preclinical animal studies, demonstrating proof of mechanism for the novel therapeutic.
"These important FDA designations are more excellent news for Kaerus, coming after the impressive Phase 1 results showing KER-0193 to be safe and well-tolerated. These developments put the Company in a superb position as we look to move into Phase 2 trials," said Paul Sekhri, Chairman of Kaerus Bioscience.
Addressing Multiple Aspects of Fragile X Syndrome
KER-0193 is an orally-bioavailable small molecule specifically designed to address the hyperexcitability of brain function commonly associated with Fragile X syndrome. Based on its broad efficacy profile in preclinical genetic models, Kaerus believes KER-0193 has the potential to address the diverse behavioral, sensory, and cognitive challenges that adversely impact individuals with FXS.
The company's preclinical studies demonstrated that KER-0193 improves syndrome-relevant behavioral, sensory, and cognitive deficits observed in genetic animal models of Fragile X. These comprehensive improvements across multiple symptom domains suggest potential for meaningful clinical benefits.
Expanding Therapeutic Applications
Beyond Fragile X syndrome, reduced BK channel activity has been implicated in several other neurological conditions, including epilepsy and rare genetic epileptic encephalopathies. This creates potential for broader applications of Kaerus's BK modulator platform.
"We are actively exploring opportunities to expand the therapeutic potential of our BK modulator platform to these other indications, where patient populations struggle with significant unmet treatment needs," noted Dr. Ring.
Path Forward to Phase 2 Trials
With these regulatory designations secured, Kaerus is finalizing preparations for a Phase 2 proof-of-concept study in FXS patients. The Orphan Drug and Rare Pediatric Drug Designations provide significant incentives, including tax credits for clinical trial costs, exemption from user fees, and potential eligibility for seven years of market exclusivity upon approval.
Kaerus Bioscience, backed by healthcare-focused investment firm Medicxi, is a clinical-stage biotechnology company focused on developing treatments for rare genetic syndromes of neurodevelopment. The company's pipeline includes targeted, small molecule therapeutics addressing underlying ion channel dysfunction in conditions like Fragile X syndrome.
As KER-0193 advances toward Phase 2 trials, it represents one of the most promising therapeutic candidates in development for Fragile X syndrome, potentially offering hope to patients and families affected by this challenging condition.
