Positive Clinical Trial Results for a New Fragile X Treatment

Research By: Craig Erickson, MD

Post Date: February 11, 2025 | Publish Date:

A therapy intended to improve core symptoms and challenging behaviors related to fragile X syndrome — a known cause of autism and the leading inherited form of intellectual disability — demonstrated positive results in a recent clinical trial involving adult men, according to an announcement Feb. 11, 2025, from the treatment maker Spinogenix, Inc.

The announcement provides topline results from a Phase 2 study of its SPG601 therapy for fragile X syndrome (FXS) (NCT06413537). The small molecule treatment targets large-conductance, calcium-activated potassium (“BK”) channels, increasing their activation to correct specific synaptic dysfunctions that occur in FXS.

Craig Erickson, MD, Division of Psychiatry at Cincinnati Children’s, served as principal investigator for the clinical trial. He also serves as Spinogenix chief medical advisor.

“This is the strongest test result to date demonstrating a therapy normalizing gamma band activity, which is directly associated with learning, memory, and typical brain activity,” Erickson says.

The FDA granted Orphan Drug designation to SPG601 for the treatment of FXS in May 2024 and granted Fast Track designation in December 2024. Spinogenix and Cincinnati Children’s are completing analysis of the full study results in preparation for publication.

Read the entire Spinogenix announcement