New Peer-Reviewed Research Demonstrates Neurophysiological Efficacy of Spinogenix's SPG601 in Patients with Fragile X Syndrome

Study Results, Published in Nature Scientific Reports, Found SPG601 Reduced Signature Changes in High-Frequency Gamma Band Activity and Improved Cognition in FXS Patients

LOS ANGELES, April 9, 2026 /PRNewswire/ -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced the publication of a peer-reviewed article in Nature Scientific Reports, supporting the efficacy of SPG601 in participants with Fragile X Syndrome (FXS). The report shares the results of the Phase 2 clinical trial conducted at Cincinnati Children's Hospital demonstrating that synapse correction may offer a new approach to addressing the cognitive and neurophysiological deficits associated with FXS.

SPG601 is a first-in-class, small-molecule oral tablet designed to modulate the activity of large-conductance, calcium-activated potassium ("BK") channel to correct specific synaptic dysfunctions that underlie many core symptoms of FXS.

"Seeing these clinical trial results published in Nature Scientific Reports further validates the promise and potential of SPG601 as a first-in-class treatment for FXS," said Dr. Stella Sarraf, Chief Executive Officer and Founder at Spinogenix. "This latest milestone, together with continued support from the industry-leading FRAXA Research Foundation on this mechanism of action, provides a strong foundation as we advance this drug into a Phase 2b/3 trial. With no therapies currently FDA approved for people living with this condition, we are committed to leading the path by furthering the development of SPG601 to help people and their families who have been searching for new treatments."

In the published Phase 2a study, 10 adult male participants with genetically confirmed FXS were administered a single 800mg dose of SPG601 and placebo separated by a one-week washout period. SPG601 was well-tolerated with a favorable safety profile. For the first time in FXS treatment, SPG601 demonstrated improvement across the EEG power spectrum including reduction in excessive high frequency gamma band activity and increase in alpha band power that is reduced at baseline in fragile X. These corrected EEG deficits show a normalization of brain function in the direction of normal activity levels required for learning and memory.   

SPG601 treatment was also associated with significant improvement in the NIH TB Flanker task, which measures inhibitory control and attention. FXS is associated with lower overall Flanker scores than other intellectual disabilities, highlighting attention as a significant deficit in the disease population. In addition, cognitive performance as measured by the Fluid Cognition Composite and Total Cognition Composite indices showed an SPG601-associated trend towards cognitive improvement.  

"These results are the strongest sign of positive target engagement we have ever noted in the fragile X field using our single-dose trial design which has been used to test over a dozen small molecules over the last decade plus. In fact, the combination of positive EEG change and cognitive change has not been seen to this degree in the field ever before in single or chronic dosing trials," Dr. Craig Erickson, principal investigator of the planned Phase 2b trial, Director of the Cincinnati Fragile X Research and Treatment Center and Spinogenix Chief Medical Advisor commented. "The rapid improvements in key neurophysiological biomarkers, as measured by EEG, together with improvements in cognitive measures, support the viability of targeting BK channels in FXS. I am eager to continue studying SPG601 in the next trial to assess the potential added benefits of chronic dosing and further validate these findings."

SPG601 is on an expedited path to regulatory approval, having received Fast Track Designation and Orphan Drug Designation by the FDA, as well as Orphan Disease Designation by the EMA.

About SPG601
SPG601 is an oral investigational medication being developed to treat FXS, and potentially other conditions on the autism spectrum, by mitigating key underlying abnormalities in synaptic function and neural excitability. FXS involves a reduction in the activity of large conductance, calcium-activated potassium (BK) channels, which contributes to synaptic dysfunction, cortical hyperexcitability, and multiple symptoms of FXS.  SPG601, a novel small molecule, is the first positive modulator of BK channels to be clinically evaluated in FXS patients in a Phase 2a study and demonstrated the potential to improve cognitive, emotional, and sensory symptoms by boosting BK channel activity.  SPG601 has received both Orphan Drug designation and Fast Track designation from the FDA for FXS, as well as orphan disease designation from the EMA. Plans for a registrational-directed Phase 2b/3 trial have been agreed to with the FDA. 

About Fragile X Syndrome
Fragile X Syndrome (FXS) is the leading inherited form of intellectual disability and a known cause of autism that results from the silencing of the Fmr1 gene. FXS is an orphan disease affecting approximately 1 in 4-5000 men and 1 in 6-8000 women globally. In addition to intellectual disability, FXS patients endure a wide range of disabling symptoms, including severe anxiety, social aversion, hyperactivity and attention deficit, sensory hypersensitivity, aggression, developmental seizures, and others. Providing care for individuals with FXS often becomes a full-time commitment for at least one parent and imposes significant financial strain, with direct family healthcare costs totaling $4.1 billion annually in the United States alone. Despite the considerable impact of FXS, there are currently no FDA-approved drugs available for those with the condition.

About Spinogenix  
Current treatments for neurodegenerative, neuropsychiatric and neurodevelopmental conditions primarily focus on slowing disease progression or minimizing symptoms, leaving many without hope for improvement. Spinogenix is aiming to transform the treatment of these conditions through its pioneering first-in-class and paradigm-shifting synaptic regenerative and synaptic corrective therapeutics designed to restore depleted synapses and reverse synaptic degeneration and dysfunction – offering patients and their families a new reality of hope.

Spinogenix is developing two novel therapeutics: Tazbentetol (formerly SPG302), which triggers neurons to produce new glutamatergic synapses and restore cognitive, motor, and other functions in ALS, Alzheimer's disease, schizophrenia and other diseases; and SPG601, which works at the synaptic level to correct specific dysfunctions in Fragile X Syndrome (FXS) that underlie many core symptoms. The company has received FDA Orphan Drug and EMA designations for ALS as well as FDA Orphan Drug and Fast Track designations for FXS. More information on Spinogenix can be found at www.spinogenix.com or follow us on LinkedIn.

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Spinogenix, Inc.
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SOURCE Spinogenix