Clinical-stage biopharmaceutical company Spinogenix Inc announced on Thursday that it will present the full data set of the Phase 2a clinical trial for SPG601, a treatment for adults with Fragile X syndrome (FXS), at the American Academy of Child & Adolescent Psychiatry Conference (AACAP).

There is currently no FDA-approved medicine for FXS, the leading inherited form of intellectual disability and a known cause of autism that results from the silencing of the Fmr1 gene..

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 randomised, double-blind, placebo-controlled, crossover study utilised 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 behavioural 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. Behaviourally, 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., Spinogenix CEO and founder. "These trial results, together with the recent positive Type C meeting with the FDA, provide a clear path forward for the development of SPG601."