Press Release

Spinogenix Announces U.S. FDA Approval of its Investigational New Drug Application for its Phase 2a Clinical Trial of SPG601 for Fragile X Syndrome

SPG601 is a once-a-day pill that works by restoring synaptic function to address core symptoms of Fragile X Syndrome

SAN DIEGO, Calif., April 15, 2024 — Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for SPG601 for the treatment of people with Fragile X Syndrome (FXS). SPG601 works by restoring synaptic function to address core symptoms of FXS. The Phase 2a trial will evaluate the neurophysiological and clinical effects of single-dose SPG601 and placebo in adult men with FXS.

“The FDA approval of our U.S. IND 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,” said Spinogenix Founder and Chief Executive Officer Stella Sarraf, Ph.D.
“Current treatments leave a critical gap in effective and patient-friendly solutions for neurodevelopmental conditions. 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.”

Fragile X Syndrome (FXS) is the leading inherited form of intellectual disability and a known cause of autism that results from the silencing of the Fmr1 gene. FXS is an orphan disease affecting approximately 1 in 4-5000 men and 1 in 6-8000 women globally. In addition to intellectual disability, FXS patients endure a wide range of disabling symptoms including severe anxiety, social aversion, hyperactivity and attention deficit, sensory hypersensitivity, aggression, developmental seizures, and others. Many core symptoms of FXS have been linked to deficient activity of large conductance, calcium-activated potassium (“BK”) channels. SPG601 is a novel small molecule BK channel activator that works by binding to BK channels and increasing their activation to restore synaptic function.

Dr. Craig Erickson, M.D., Spinogenix Chief Medical Advisor added, “Despite the considerable impact of FXS, there are currently no FDA-approved drugs available for those with the condition.
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.”

About Spinogenix 
Spinogenix was founded with the mission to develop transformative therapeutics for diseases involving synaptic loss and dysfunction. Our drugs are designed to regenerate synapses to reverse declines in cognitive and motor function and fundamentally change treatment paradigms by restoring neuronal connections regardless of the underlying cause of synapse loss. Synapse loss is associated with a variety of neurological and psychiatric diseases, such as ALS, Alzheimer’s disease, Parkinson’s disease, and schizophrenia. More information on Spinogenix can be found at

Spinogenix Contact

Media Contact
Kristin Politi, Ph.D.
LifeSci Communications
(646) 876-4783

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