Receiving Orphan Drug Designation for SPG601 emphasizes the pressing demand for novel therapies for individuals living with Fragile X Syndrome

LOS ANGELES, Calif., May 20, 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 granted Orphan Drug Designation (ODD) to SPG601 for the treatment of Fragile X Syndrome (FXS).

“Receiving ODD for SPG601 underscores the high unmet need for new treatment options for individuals with FXS,” said Craig Erickson, M.D., Spinogenix Chief Medical Advisor. “Despite the challenges individuals living with FXS endure, there are currently no FDA-approved therapies available for this condition. Spinogenix has chosen to focus on FXS due to the potential of SPG601 to correct specific synaptic dysfunctions by modulating calcium-activated potassium (“BK”) channels, which are a known driver of symptoms. This approach aims to address core symptoms of FXS with the goal of significantly improving the lives of those impacted by the condition.”

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 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.

Stella Sarraf, Ph.D., Spinogenix Chief Executive Officer, added, “There is growing sentiment among experts that synapse loss and dysfunction are central features of pathogenesis in a broad spectrum of diseases. We believe people living with Fragile X need a therapeutic option that works at the synaptic level to address their wide range of disabling symptoms. We are excited to be granted this designation for SPG601 as we continue to advance our small molecule therapies through clinical trials.”

Orphan Drug Designation is granted by the FDA to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of post-approval marketing exclusivity.

About Spinogenix 
Spinogenix is dedicated to developing transformative therapeutics for conditions involving the loss or dysfunction of synapses. Our lead clinical-stage synaptic regenerative candidate is a first-in-class therapeutic designed to reverse synapse loss and improve cognitive and motor functions in neurodegenerative and neuropsychiatric diseases such as ALS, Alzheimer’s disease, and schizophrenia. In parallel, we are also developing a synaptic function therapeutic designed to improve behavior in Fragile X Syndrome. For more information follow us on LinkedIn. 

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