Spinogenix to Present Complete Phase 2a Clinical Trial Results for SPG601, a First-in-Class Fragile X Syndrome Treatment, at AACAP Conference

SPG601, Granted Fast Track Designation by the FDA, Reduced Signature Changes in High-Frequency Gamma Band Activity in Fragile X Syndrome Patients, a Common Inherited Form of Intellectual Disability and Autism, and Improved Measures of Cognitive Impairment 

Dr. Craig Erickson, Spinogenix Chief Medical Advisor, to Receive Prestigious George Tarjan Award for Contributions in Developmental Disabilities

LOS ANGELES, Oct. 22, 2025 /PRNewswire/ -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, announced it will present the full data set of the Phase 2a clinical trial at the American Academy of Child & Adolescent Psychiatry Conference (AACAP) for SPG601, a treatment for adults with Fragile X syndrome (FXS), a condition for which there is currently no approved medicine.

SPG601 is a first-in-class, oral, small molecule formulated as a tablet, and is 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.

The Phase 2a randomized, double-blind, placebo-controlled, crossover study utilized a single dose of SPG601 and a matching placebo in 10 adult male participants exhibiting core FXS attributes. Treatment with SPG601 resulted in improvements in a signature neurophysiological biomarker of FXS and in a core behavioral deficit. At a neurophysiological level, SPG601 reduced high-frequency gamma band activity, an abnormality seen in electroencephalogram (EEG) recordings of FXS patients that occur at the expense of normal brain activity levels used for learning and memory. Behaviorally, FXS participants treated with SPG601 exhibited improved performance on a task that measures selective attention, a domain of cognitive function impaired in those with FXS. 

"We are excited to continue demonstrating SPG601's potential as a first-in-class treatment for FXS," said Stella Sarraf, Ph.D., Chief Executive Officer and Founder at Spinogenix. "These trial results, together with the recent positive Type C meeting with the FDA, provide a clear path forward for the development of SPG601."

Dr. Craig Erickson, Director of the Cincinnati Fragile X Research and Treatment Center, and Spinogenix Chief Medical Advisor, will present the results at the AACAP conference. Dr. Erickson will also be honored with the prestigious AACAP George Tarjan, MD, Award for Contributions in Developmental Disabilities. The award recognizes child and adolescent psychiatrists who have made significant contributions over the course of a career or single seminal work to the understanding or care of those with developmental disabilities. Dr. Erickson's work studying FXS has been instrumental to the development of SPG601.

"I am honored by the recognition from the AACAP and its members. With no current approved treatment and an urgent global need for effective options, SPG601 has the potential to represent an important step forward in FXS treatment development," said Dr. Erickson. "The successful implementation of objective neurophysiological outcomes in the clinical testing of SPG601 represents a significant milestone in the development of novel therapeutics for FXS and gratifying application of work I have pursued throughout my career."

About SPG601

SPG601 is an oral 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 and has the potential to improve cognitive, emotional, and sensory symptoms by boosting BK channel activity. SPG601 has received Fast Track designation from the FDA and Orphan Drug Designation from the EMA and 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 4-5,000 men and 1 in 6-8,000 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: SPG302, which triggers neurons to produce new glutamatergic synapses and restore cognitive, motor, and other functions in Amyotrophic lateral sclerosis, Alzheimer's disease, schizophrenia and other diseases; and SPG601, which works at the synaptic level to correct specific dysfunctions in FXS that underlie many core symptoms. The company has received FDA and EMA Orphan Drug designations for both ALS and FXS as well as FDA Fast Track designation for FXS. More information on Spinogenix can be found at www.spinogenix.com or follow us on LinkedIn. 

Media Contact 
Daniel Davis
FINN Partners
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Investor Relations 
Dan Albosta
Spinogenix, Inc.
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SOURCE Spinogenix