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Spinogenix Receives FDA Approval for Phase 2a Clinical Trial of SPG601 in Fragile X Syndrome

Tuesday, April 16, 2024

Spinogenix, Inc., a company specializing in developing innovative therapies aimed at restoring synaptic function to enhance the lives of patients worldwide, has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for SPG601. This signifies a significant advancement in the treatment of Fragile X Syndrome (FXS), a neurodevelopmental disorder characterized by synaptic dysfunction. SPG601 targets the core symptoms of FXS by restoring synaptic function. The Phase 2a trial will assess the neurophysiological and clinical effects of a single dose of SPG601 compared to a placebo in adult men with FXS.

Stella Sarraf, Ph.D., Founder and Chief Executive Officer of Spinogenix, expressed excitement about the FDA approval, stating, “The clearance of our U.S. IND for SPG601 for FXS signifies a major milestone for our company as we aim to expand our portfolio of innovative therapeutics targeting synaptic function restoration. Existing treatments fall short in addressing the needs of patients with neurodevelopmental conditions. By focusing on restoring synaptic function, we hope to fill this critical gap in FXS treatment options. The initiation of clinical trials with SPG601 marks a significant advancement in our mission to introduce innovative therapies that bring hope to patients.”

FXS is the leading inherited cause of intellectual disability and is associated with autism spectrum disorder, resulting from the silencing of the Fmr1 gene. It is considered an orphan disease, affecting a relatively small number of individuals globally. FXS patients experience a range of debilitating symptoms, including anxiety, social difficulties, hyperactivity, attention deficits, sensory sensitivities, aggression, and seizures. SPG601, a novel small molecule BK channel activator, addresses these symptoms by targeting BK channels to restore synaptic function.

Dr. Craig Erickson, M.D., Chief Medical Advisor at Spinogenix, emphasized the significance of SPG601’s mechanism of action in addressing FXS. He stated, “Despite the significant impact of FXS, there are currently no FDA-approved drugs available for this condition. SPG601 targets BK channels, which have been implicated in FXS pathology. This trial marks the first investigation of a BK channel modulator in humans with Fragile X, representing a crucial step in evaluating this promising drug mechanism for FXS.”

In summary, Spinogenix’s FDA clearance for the IND application of SPG601 for FXS represents a significant advancement in the field of neurodevelopmental disorder therapeutics, offering hope for improved treatments for patients affected by FXS.



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