US-based biopharmaceutical company Spinogenix has published top line outcomes from a Phase II trial of SPG601 for treating Fragile X syndrome (FXS), a genetic disorder linked to autism and intellectual disability, in adult male patients.
The randomised, placebo-controlled study compared the single dose of the therapy with a matching placebo in ten adult men with core attributes of FXS.
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The trial met its primary goal of showing the therapy’s potential in decreasing high-frequency gamma band activity, an electroencephalogram (EEG) abnormality characteristic of FXS that impedes the brain activity needed for learning and memory.
It was conducted at Cincinnati Children’s Hospital Medical Center in Cincinnati, Ohio, where extensive research has been done on gamma band EEG as a marker of brain activity.
Spinogenix CEO and founder Dr Stella Sarraf said: “This is an exciting milestone for SPG601 at a time when there are no approved treatments for FXS.
“We are focused on developing SPG601 as a first-in-class treatment for FXS capable of restoring synapse function.
“The completion of this trial and our FDA fast track designation allow us to accelerate its development to offer a much-needed therapy that can improve patients’ quality of life in an underserved community.”
SPG601 works at the synaptic level and is claimed to target large-conductance, calcium-activated potassium channels.
By increasing the activation of these channels, the therapy is designed to improve the core symptoms and challenging behaviours associated with this condition.
It received orphan drug designation for the treatment of FXS in May 2024 and fast track designation in December from the US Food and Drug Administration (FDA).
Based in California, Spinogenix develops first-in-class therapeutics to treat neurological conditions by regenerating synapses or correcting defects in synaptic function.
In July 2023, the company received approval from Australia’s Human Research Ethics Committee to enrol subjects in a Phase I trial of SPG302 for treating amyotrophic lateral sclerosis.
