FDA clears investigational new drug application for Fragile X treatment

The FDA has cleared an investigational new drug application for a small molecule therapeutic to treat those with Fragile X syndrome, according to the manufacturer.

In a press release, San Diego-based Spinogenix Inc. said SPG601 works by binding to BK (calcium activated potassium) synaptic channels and increasing their activation to restore synaptic function.

Fragile X syndrome (FXS) is a genetic condition that causes mild to severe intellectual disability. Symptoms of the condition, including severe anxiety, social aversion, sensory hypersensitivity, aggression and developmental seizures, have been linked to deficiencies in the activity of large conductance, calcium-activated potassium channels.

A known cause of autism, FXS is the leading inherited form of intellectual disability, according to the release. The disease affects about one in 4,000 to 5,000 men and one in 6,000 to 8,000 women worldwide.

A pending phase 2a clinical trial is expected to evaluate neurophysiological and clinical effects of single-dose SPG601 compared with placebo in adult males with FXS.

“The FDA approval of our U.S. [investigational new drug application] for SPG601 for FXS represents a significant milestone for the company as we look to expand our pipeline of game-changing therapeutics that aim to restore synaptic function,” Spinogenix founder and CEO Stella Sarraf, PhD, said in the release. “The expansion of clinical programs with SPG601 represents an important step in our progress to bringing innovative treatments that offer new hope, and we look forward to dosing the first patient in the trial this year.”