Clinical-stage biopharmaceutical company Spinogenix Inc on Tuesday announced topline results from a Phase 2 study of its SPG601 therapy in adult men with Fragile X syndrome (FXS).

FXS, a genetic disorder caused by the silencing of the Fmr1 gene, is a known cause of autism and the leading inherited form of intellectual disability.

SPG601 is designed to work at the synaptic level, targeting a well-established molecular dysfunction in FXS, to improve core symptoms and challenging behaviours, which can include severe anxiety, social aversion, hyperactivity and attention deficit, sensory hypersensitivity, aggression and developmental seizures, with the hope to enhance the overall quality of life for those affected. It is a small molecule that targets large-conductance, calcium-activated potassium (BK) channels, increasing their activation to correct specific synaptic dysfunctions that are thought to underlie many core symptoms of FXS.

The Phase 2 trial was a randomised, double-blind, placebo-controlled crossover study utilising a single dose of SPG601 and a matching placebo in 10 adult male patients exhibiting core FXS attributes. A primary goal of the study was to determine if SPG601 reduced high frequency gamma band activity, an abnormality seen in electroencephalogram (EEG) recordings of FXS patients that occurs at the expense of normal brain activity levels that are more for learning and memory. The trial met this goal with SPG601 significantly reducing high frequency gamma band activity in the FXS subjects.

Topline results from the Phase 2 study were reported at the NIH Fragile X Centers of Excellence Update on 10 February 2025.