Novel BK Channel Activator Fast Tracked for Fragile X Syndrome

The Food and Drug Administration (FDA) has granted Fast Track designation to SPG601 for the treatment of people with Fragile X syndrome (FXS), a rare inherited cause of intellectual disability and autism.

SPG601 is an investigational small molecule large-conductance, calcium-activated potassium (BK) channel activator. It is designed to correct specific synaptic dysfunctions in FXS patients by binding to BK channels and increasing their activation.

A phase 2 trial (ClinicalTrials.gov Identifier: NCT06413537) evaluating the safety and efficacy of oral SPG601 in adult men with FXS was recently completed. According to developer Spinogenix, topline results from the double-blind, placebo-controlled crossover study are expected by the end of the first quarter of 2025.

“Receiving Fast Track designation for SPG601 demonstrates its potential to impact patients’ lives and brings us one step closer to providing a new therapeutic to combat FXS,” said Dr Stella Sarraf, Spinogenix Chief Executive Officer and Founder. “We remain focused on developing SPG601 as a first-in-class treatment for FXS capable of restoring synapse function, and the completion of this trial and this designation will allow us to accelerate its development to offer a much-needed therapy that can improve patients’ quality of life.”

There are currently no FDA-approved treatments for FXS, a disease that affects approximately 1 in 4-5000 men and 1 in 6-8000 women globally. Symptoms of FXS, which include severe anxiety, attention deficit, aggression, and intellectual disability, have been linked to BK channel deficiency.

This article originally appeared on MPR