U.S. FDA has granted Orphan Drug Designation to SPG601 for the Treatment of Fragile X

SPG601 is a novel small molecule that works by correcting specific synaptic dysfunctions in the brain to address core symptoms of Fragile X syndrome

LOS ANGELES, Calif., July 23, 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 enrollment is now open for their Phase 2 clinical trial evaluating SPG601 for the treatment of adult men with Fragile X syndrome (FXS), the leading inherited form of intellectual disability and a known cause of autism. The U.S. Food and Drug Administration (FDA) recently cleared the Investigational New Drug (IND) applicationand granted Orphan Drug Designation for SPG601 for the treatment of people with FXS.

“Fragile X patients and their families compose an incredible, yet still underserved community. That is why, during this year’s National Fragile X Awareness Month, we are especially excited to announce that we have begun recruiting for our Phase 2 trial evaluating SPG601 for the treatment of individuals with this difficult condition. This aligns with our commitment to advancing research and treatment options to help people with this condition live better-quality lives, as there are currently no FDA-approved drugs available for those with FXS,” said Craig Erickson, M.D., Spinogenix Chief Medical Advisor. “Notably, SPG601 is a novel once-a-day pill that works by binding to BK channels and increasing their activation to correct specific synaptic dysfunctions, a therapeutic approach that we believe can bring much needed hope to individuals and families struggling with FXS.”

Stella Sarraf, Ph.D., Spinogenix Chief Executive Officer and Founder, added, “People with FXS and their families face immense challenges every day. This milestone marks a significant step forward in our mission to bring new hope to the people and families affected by this condition.

At Spinogenix, we are passionately working to develop first-in-class therapeutics that restore
synapses to improve the lives of people worldwide and we are accomplishing this through our
trials evaluating SPG601 in FXS and SPG302 in ALS and Alzheimer’s disease. We look forward to exploring the potential of our small molecule platform to address a range of diseases involving synapse loss and dysfunction.”

This study is a Phase 2 randomized, double blind, placebo-controlled, cross over, single dose of SPG601 and matching placebo in patients with Fragile X syndrome. Enrollment is currently open
at the Cincinnati Children’s Hospital Medical Center in Cincinnati, Ohio, United States. Additional information on the Phase 2 trial is available on ClinicalTrials.gov (NCT06413537).

FXS results from the silencing of the Fmr1 gene which leads to changes in mRNA and protein expression that results in abnormalities in synaptic function. Some of these abnormalities have been shown to be a result of a reduction in the amount and activity of large-conductance, calcium-activated potassium (“BK”) channels. Many core symptoms of FXS have been linked to deficient activity of BK channels. In addition to FXS, BK channel expression and activity have been linked to a range of other conditions including neurodegenerative, vascular, and sensory disorders. SPG601 is a novel small molecule BK channel activator that works by binding to BK channels and increasing their activation to correct specific synaptic dysfunctions that underlie these conditions.

About Fragile X syndrome
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. Providing care for individuals with FXS often becomes a full-time commitment for at least one parent and imposes significant financial strain, with direct family healthcare costs totaling $4.1 billion annually in the United States alone. Despite the considerable impact of FXS, there are currently no FDA-approved drugs available for those with the condition.

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. More information on Spinogenix can be found at www.spinogenix.com or follow us on LinkedIn.

For more information contact: info@spinogenix.com