FDA Grants Fast Track Designation to Spinogenix's SPG601 for Treatment of Fragile X Syndrome, a Common Inherited Form of Autism

Milestone Highlights Urgent Need for a Novel Therapeutic to Treat People with FXS

FDA Designation Enables Expedited Clinical Development and Regulatory Review Timelines for SPG601

LOS ANGELES, Jan. 13, 2025 /PRNewswire/ -- 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 Fast Track designation to SPG601 for the treatment of people with Fragile X syndrome (FXS).

FXS, a known cause of autism, is the leading inherited form of intellectual disability that can produce a wide range of disabling symptoms, with many individuals requiring lifelong around-the-clock supportive care. SPG601 works at the synaptic level, targeting a well-established molecular dysfunction in FXS, to address core symptoms with the hope to improve the overall quality of life for those affected. It is a small molecule large-conductance, calcium-activated potassium ("BK") channel activator that works by binding to BK channels and increasing their activation to correct specific synaptic dysfunctions that underlie many core symptoms of FXS.

"I am thrilled that the FDA has granted Fast Track designation to SPG601 for FXS, highlighting its potential as a novel therapeutic option for a disease that, to this day, has no approved treatment," said Dr. Craig Erickson, Spinogenix Chief Medical Advisor and principal investigator of the recently completed Phase 2 study conducted at Cincinnati Children's. Dr. Erickson is also the research director and an associate professor in the Division of Child and Adolescent Psychiatry at Cincinnati Children's where he and his lab team study translational treatment development for neurodevelopmental disorders, with a focus on FXS and autism spectrum disorder. "The possibility that SPG601 could address underlying synaptic deficits central to this condition in people with FXS brings immense hope, and with this designation, we are even closer to making it available to patients sooner."

In 2024, the FDA granted Orphan Drug designation to SPG601 for the treatment of FXS. With this Fast Track designation, Spinogenix can expedite the development and review of SPG601 in FXS.

Spinogenix recently completed its Phase 2 study of SPG601 in adult men with FXS (NCT06413537), with topline results expected by end of Q1 2025. This study was designed to enable the detection of meaningful brain target engagement in first-in-human FXS trials, utilizing validated neurophysiologic markers of brain activity. These markers have been extensively validated over the past decade by Dr. Erickson and colleagues, supported by the National Institutes of Health Centers for Collaborative Research in Fragile X program. The Phase 2 trial was a randomized, double blind, placebo-controlled, cross over study utilizing a single dose of SPG601 and a matching placebo, in patients with FXS.

"Receiving Fast Track designation for SPG601 demonstrates its potential to impact patients' lives and brings us one step closer to providing a new therapeutic to combat FXS," said Dr. Stella Sarraf, Spinogenix Chief Executive Officer and Founder. "We remain focused on developing SPG601 as a first-in-class treatment for FXS capable of restoring synapse function, and the completion of this trial and this designation will allow us to accelerate its development to offer a much-needed therapy that can improve patients' quality of life. We also believe our core technology can play a similar future restorative role across additional disease states."

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 pioneering first-in-class therapeutics that restore brain connections (synapses) to improve the lives of patients worldwide. The company has designed small molecules to help restore synapses in neurodegenerative, neuropsychiatric and neurodevelopmental conditions including amyotrophic lateral sclerosis (ALS), Alzheimer's disease, schizophrenia, Fragile X syndrome and others. To date, Spinogenix has received a U.S. FDA Orphan Drug designation for the treatment of both ALS and Fragile X syndrome. More information on Spinogenix can be found at www.spinogenix.com or follow us on LinkedIn. 

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SOURCE Spinogenix