Topline Results Announced at NIH Fragile X Centers of Excellence Conference

U.S. FDA Previously Granted Orphan Disease Designation and Fast Track Designation to SPG601

LOS ANGELES, Calif., February 11, 2025 — Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced topline results from a Phase 2 study of its SPG601 therapy in adult men with Fragile X syndrome (FXS) (NCT06413537).

FXS, a genetic disorder caused by the silencing of the Fmr1 gene, is a known cause of autism and the leading inherited form of intellectual disability. It 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 improve core symptoms and challenging behaviors, which can include severe anxiety, social aversion, hyperactivity and attention deficit, sensory hypersensitivity, aggression and developmental seizures, with the hope to enhance the overall quality of life for those affected. It is a small molecule that targets large-conductance, calcium-activated potassium (“BK”) channels, increasing their activation to correct specific synaptic dysfunctions that are thought to underlie many core symptoms of FXS.

The Phase 2 trial was a randomized, double-blind, placebo-controlled, crossover study utilizing a single dose of SPG601 and a matching placebo in 10 adult male patients exhibiting core FXS attributes. A primary goal of the study was to determine if SPG601 reduced high frequency gamma band activity, an abnormality seen in electroencephalogram (EEG) recordings of FXS patients that occurs at the expense of normal brain activity levels that are more for learning and memory. The Phase 2 trial met this goal with SPG601 significantly reducing high frequency gamma band activity in the FXS subjects.

“This is the strongest test result to date demonstrating a therapy normalizing gamma band activity, which is directly associated with learning, memory, and typical brain activity,” said Dr. Craig Erickson, Spinogenix Chief Medical Advisor and principal investigator of the study conducted at Cincinnati Children’s.

Gamma band EEG as a marker of brain activity has been extensively validated over the past decade by Dr. Erickson and colleagues, supported by the Fragile X program at the National Institutes of Health Centers for Collaborative Research. Dr. Erickson is also research director and a 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. He has received numerous NIH grants and has published extensively on the reliability of small clinical trial designs, applying this model to evaluate various drugs in development.

Topline results from the Phase 2 study were shared at the NIH Fragile X Centers of Excellence Update on Monday, February 10, 2025.

“This is an exciting milestone for SPG601 at a time when there are no approved treatments for FXS,” said Dr. Stella Sarraf, Spinogenix Chief Executive Officer and Founder. “We are focused on developing SPG601 as a first-in-class treatment for FXS capable of restoring synapse function. The completion of this trial and our FDA Fast Track designation allow us to accelerate its development to offer a much-needed therapy that can improve patients’ quality of life in an underserved community.”

The FDA granted Orphan Drug designation to SPG601 for the treatment of FXS in May 2024 and granted Fast Track designation in December 2024. Spinogenix and Cincinnati Children’s are completing analysis of the full study results in preparation for publication.

About Fragile X Syndrome
Fragile X Syndrome (FXS), a genetic disorder, is the leading inherited form of intellectual disability and a known cause of autism, affecting approximately 1 in 4-5,000 men and 1 in 6-8,000 women globally. Resulting from the silencing of the Fmr1 gene, FXS is an orphan disease that produces a range of debilitating symptoms beyond intellectual disability, including severe anxiety, social aversion, hyperactivity and attention deficit, sensory hypersensitivity, aggression and developmental seizures. These challenges often necessitate lifelong, full-time care, placing significant financial strain on families, with direct healthcare costs exceeding $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 regenerate brain connections (synapses) or correct defects in synaptic function 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 (FXS) and others. Spinogenix has received a U.S. FDA Orphan Drug designation for the treatment of both ALS and FXS, as well as U.S. FDA Fast Track designation for FXS. More information on Spinogenix can be found at www.spinogenix.com or follow us on LinkedIn.

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