Spinogenix has broadened its ongoing Phase IIb/III CLARITY basket trial to include a Phase IIb study of codabakalner (SPG601) in idiopathic autism spectrum disorder (ASD).
The trial marks the biopharmaceutical firm’s first study in autism, assessing the oral compound’s effects on sensory, emotional and cognitive symptoms.
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It is backed by grant funding from the FRAXA Research Foundation, previously focused on Fragile X syndrome (FXS).
Rather than relying purely on behavioural observations, the new autism cohort will select participants through a biomarker-enriched recruitment method.
Patients with idiopathic ASD will be screened via electroencephalogram (EEG) and enrolled if they exhibit heightened resting gamma band power.
This inclusion strategy relies on research conducted jointly by Spinogenix and Cincinnati Children’s Hospital.
The study examined resting-state EEGs from 159 individuals with autism alongside 41 males aged 13 and older diagnosed with full-mutation FXS.
Investigators identified the FXS-like brain signature in 22% of the overall autism cohort (35 out of 159) and in 31.9% of adult participants (23 out of 72).
Across 27 clinical evaluations assessing adaptive function, behaviour, social responsiveness, and IQ, no clinical measures distinguished this subgroup after statistical correction, indicating that standard clinical evaluations alone cannot detect the population.
Spinogenix CEO and founder Dr Stella Sarraf said: “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. Fragile X gave us a measurable brain signature, and codabakalner is 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.”
Codabakalner functions as a positive modulator of BK channels. In a prior Phase IIa study published in Scientific Reports, the therapy lowered resting gamma power and led to notable gains in selective attention and executive function among adult males with FXS.
FXS represents a primary inherited source of intellectual disability and a prominent genetic contributor to autism. Codabakalner has secured orphan drug and fast track designations from the FDA, alongside orphan status from the EMA, specifically for FXS.
The Phase IIb study also integrates elements of the IMPACT framework, which combines deep phenotyping, genomic analysis, and EEG biomarkers.
Spinogenix develops therapies targeting neurodevelopmental, neurodegenerative, and neuropsychiatric conditions.
Alongside codabakalner for autism and FXS, the company’s clinical pipeline features tazbentetol, which is under assessment for Alzheimer’s disease, ALS, and schizophrenia.
