Spinogenix Announces Phase 2b Trial for Autism Using Novel Brain-Wave Screening to Target Treatment

The Trial, a First-of-Its-Kind Prospective Use of a Brain-Activity Inclusion Criterion in Neurodevelopmental Drug Development, Will Enroll Patients Exhibiting Elevated Gamma Band EEG Power

New Research Suggests Roughly 1 in 5 People with Autism, and Nearly 1 in 3 Adults, Share This Specific Biomarker linked to Potential Improvements in Executive Function

LOS ANGELES, Oct. 14, 2026 /PRNewswire/ -- Spinogenix, Inc., a clinical-stage biopharmaceutical company developing first-in-class synaptic regenerative and corrective therapeutics, today announced the addition of a Phase 2b study in idiopathic autism spectrum disorder (ASD) as part of the Phase 2b/3 CLARITY basket trial (NCT07439510) evaluating codabakalner (SPG601) in Fragile X syndrome (FXS). The expansion marks Spinogenix's first-ever study in autism and introduces a novel, biomarker-enriched enrollment design to treat cognitive, emotional, and sensory symptoms.

For the past 25 years, autism drug trials have struggled because they enrolled participants based on observable clinical criteria alone, diluting potential benefits across a highly heterogeneous population. The CLARITY trial's "biomarker enrichment" enrollment strategy prioritizes participants most likely to share the specific biology the drug is designed to target. Rather than enrolling on behavioral criteria alone, individuals with idiopathic ASD whose screening electroencephalogram (EEG) shows elevated resting gamma band power will be selected.

The basis for this approach derives from parallels between idiopathic ASD and FXS, a common syndromic cause of autism where elevated resting gamma power is one of the most robust brain findings. Evidence suggests that normalizing this elevated gamma may have therapeutic potential. In a Phase 2a study published in Scientific Reports, codabakalner reduced gamma power in adult men with FXS, alongside significant improvements in executive function and selective attention. Spinogenix will now test that hypothesis directly in the ASD Phase 2b portion of the CLARITY basket trial in participants who share the FXS gamma signature.

A study co-authored by researchers at Cincinnati Children's Hospital and Spinogenix, Defining a subgroup within autism leads to potential enrichment criteria in investigational therapeutic development: Comparing resting state gamma band power to a fragile X syndrome reference sample, compared resting-state EEGs from 159 people with autism with those of 41 males aged 13 and older with full-mutation FXS.

"For over two decades, autism drug trials have enrolled a highly diverse population as if it were one condition, testing promising medicines in many people who may not have the biology those medicines target," said Dr. Stella Sarraf, CEO and Founder of Spinogenix. "Fragile X gave us a measurable brain signature, and codabakalner a potential first-in-class medicine that engages it. CLARITY takes what we have learned in Fragile X and uses EEG to find the people with autism who share that biology and may experience therapeutic benefit in symptoms, including impaired executive function and heightened anxiety. We believe this is a fundamentally new way to develop autism therapeutics."

The study found that:

• 22% of participants with autism (35 of 159) met the Fragile X-like threshold, rising to 31.9% of adults (23 of 72).
• No clinical measure set this subgroup apart. None of the 27 clinical score comparisons, spanning IQ, social responsiveness, behavior and adaptive function, differed after statistical correction, meaning the subgroup would not be readily identified by clinical presentation alone.

"In our analysis, about one in five people with autism, and nearly one in three adults, carried the same elevated gamma signature we see in Fragile X, yet they showed no readily observable clinical differences from others with autism," said Craig Erickson, M.D., Chief Medical Advisor to Spinogenix and Principal Investigator of CLARITY. "In Fragile X, reducing elevated gamma with codabakalner was correlated with an improvement in executive function. CLARITY lets us ask whether people with autism who share that brain biology can benefit in the same way. This is exactly the kind of precision medicine approach we need to use in autism treatment development given the wide heterogeneity that characterizes the autism spectrum." 

Dr. Erickson's leadership in EEG-based precision medicine was recently recognized with a $17.25 million grant from the Aligning Research to Impact Autism (ARIA) initiative to Cincinnati Children's, one of 10 sites in the IMPACT precision clinical trials network. IMPACT combines EEG biomarkers, genomic analysis and deep phenotyping to lay the foundation for precision autism trials; the CLARITY autism Phase 2b study brings that approach into an interventional study.

Patients, families, and clinicians interested in learning more about the CLARITY trial and enrollment criteria can visit ClinicalTrials.gov (NCT07439510).

About Codabakalner

Codabakalner (SPG601), is a novel, oral investigational small molecule. It is the first positive modulator of BK channels to be clinically evaluated in Fragile X syndrome patients in a Phase 2a study and demonstrated the potential to improve cognitive, emotional, and sensory symptoms by boosting BK channel activity. CLARITY is an adaptive Phase 2b/3 trial of codabakalner, with grant support from the FRAXA Research Foundation, for FXS and Phase 2b for idiopathic autism spectrum disorder (NCT07439510). Codabakalner has received FDA Orphan Drug and Fast Track designations and EMA orphan designation for FXS.

About Fragile X Syndrome and Autism

FXS is the leading inherited cause of intellectual disability and a leading known genetic cause of autism. It results from silencing of the FMR1 gene and affects approximately 1 in 4,000–5,000 males and 1 in 6,000–8,000 females. ASD is a neurodevelopmental condition affecting an estimated 1 in 31 children aged 8 years and over. It is characterized by persistent difficulties in social communication and interaction, alongside restricted or repetitive behaviors and interests, with widely varying abilities and support needs. There are currently no FDA-approved treatments for FXS, and only two medications (both atypical antipsychotics) are approved for ASD to help with irritability, aggression and related behaviors.

About Spinogenix

Spinogenix, Inc. is a clinical-stage biopharmaceutical company developing first-in-class synaptic regenerative and corrective therapeutics for neurodegenerative, neuropsychiatric and neurodevelopmental diseases. Its pipeline includes codabakalner for Fragile X syndrome and autism, and tazbentetol for ALS, Alzheimer's disease and schizophrenia. Learn more at spinogenix.com.

Forward-Looking Statements

This press release contains forward-looking statements, including statements about the design, enrollment, timing and potential outcomes of the CLARITY trial, the potential of EEG-based enrichment, and the therapeutic potential of codabakalner. These statements are subject to risks and uncertainties that could cause actual results to differ materially. Codabakalner is investigational and has not been approved by any regulatory authority. Spinogenix undertakes no obligation to update these statements except as required by law.

Media Contact: 
Daniel Davis
FINN Partners
[email protected] 

Investor Relations Contact: 
Sasha Damouni Ellis
Spinogenix, Inc.
[email protected]

SOURCE Spinogenix