FDA Clears Spinogenix's Phase 2a Trial for Innovative Fragile X Syndrome Treatment

Spinogenix, announced that the US Food and Drug Administration (FDA) has approved their Investigational New Drug (IND) application for SPG601, a novel treatment aimed at individuals with Fragile X Syndrome (FXS). This approval marks a crucial step for the company as it begins its first US trial to evaluate the efficacy of SPG601 in treating core symptoms of this debilitating disorder.

SPG601 is a once-daily oral medication that targets these BK channels, enhancing their function to potentially restore normal synaptic activity. As the first of its kind, SPG601 operates as a small molecule BK channel activator, offering a groundbreaking approach to a largely unmet medical need.

Fragile X Syndrome, the most common inherited intellectual disability and a key genetic factor in autism, affects individuals with symptoms like anxiety, hyperactivity, and social aversion. These issues arise from the silencing of the Fmr1 gene, leading to impaired BK channel function, essential for neuron communication.

Dr. Stella Sarraf, CEO of Spinogenix, highlighted the critical gaps in current treatment options for neurodevelopmental conditions and expressed optimism about SPG601's potential. “Like many other conditions, loss of synaptic function remains a key driver of disease, and for that reason we are excited to launch our first U.S. trial to address this unmet need in FXS. 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,” she said.

The upcoming Phase 2a trial will crucially assess the neurophysiological and clinical impacts of SPG601 in adult men diagnosed with FXS. This trial aims to determine how effective the drug is in altering disease markers and alleviating symptoms compared to a placebo.

Dr. Craig Erickson, Spinogenix Chief Medical Advisor added, “SPG601’s novel mechanism works directly at BK channels and BK channel function has been shown to be abnormal in many animal studies in the Fragile X field. We look forward to the first study of a BK channel modulator in humans with Fragile X and taking the first step to evaluate this important drug mechanism in Fragile X Syndrome.”

Spinogenix was founded with the mission to develop transformative therapeutics for diseases involving synaptic loss and dysfunction. Their drug development strategy focuses on regenerating synapses to reverse declines in cognitive and motor function across various neurological and psychiatric diseases, including ALS, Alzheimer's disease, Parkinson's disease, and schizophrenia.