European Medicines Agency Grants Orphan Drug Designation to Spinogenix's SPG601 for Treatment of Fragile X Syndrome (FXS)

FXS is a Common Inherited Form of Autism

EMA Designation Offers People with FXS Throughout European Union Access to Novel Therapeutic

LOS ANGELES, July 7, 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 European Medicines Agency (EMA) has granted orphan drug designation (ODD) to SPG601 for the treatment of people with Fragile X syndrome (FXS), a condition for which there is currently no approved medicine.

FXS, a known cause of autism, is the leading inherited form of intellectual disability caused by the silencing of the Fmr1 gene. FXS 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, addressing core symptoms to improve challenging behaviors and the overall quality of life of 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.

"We are grateful to the EMA for recognizing the importance of developing treatments for FXS and supporting the development and path to market for our novel drug, SPG601," said Dr. Stella Sarraf, Spinogenix Chief Executive Officer and Founder. "We want to ensure that every person living with this disabling condition can access treatment. Having already obtained ODD and Fast Track designations from the U.S. FDA, this new grant from the EMA takes us one step forward to achieving our mission to help patients across the globe."

Topline results from a Phase 2 randomized, double-blind, placebo-controlled, crossover study of SPG601 therapy in adult men with FXS were announced earlier this year. The study met its primary goal, significantly reducing high-frequency gamma band activity in the FXS subjects, a key indicator that occurs in FXS patients at the expense of normal brain activity levels that are used for learning and memory.

"Currently, FXS has no approved treatment, making indications like the EMA's ODD crucial for advancing therapies and accelerating development," said Dr. Craig Erickson, Spinogenix Chief Medical Advisor and principal investigator of the recently completed Phase 2 study conducted at Cincinnati Children's Hospital. "SPG601 has the potential to improve the underlying synaptic deficits central to this condition, offering immense hope to the whole patient community."

Dr. Erickson will present results from the Phase 2 trial at the Cincinnati Fragile X Family Conference, taking place on Saturday, July 12 at Cincinnati Children's Hospital. Families and researchers can attend to learn about the latest research and treatments and receive advice on planning and managing behaviors. Registration for the event is free to the public and further details can be found here: https://web.cvent.com/event/3c926471-8622-401c-86c8-06bb384d3bb8/summary .

The EMA grants ODD to drugs and biologics intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating disease that affects fewer than five in 10,000 people in the European Union, and with either no currently approved method of diagnosis, prevention or treatment or with significant benefit to those affected by the disease. Companies that secure ODD benefit from a 10-year period of market exclusivity following authorization, protocol assistance, and regulatory fee reductions.

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 

Current treatments for neurodegenerative, neuropsychiatric and neurodevelopmental conditions primarily focus on slowing disease progression or minimizing symptoms, leaving many without hope for improvement. Spinogenix is aiming to transform the treatment of these conditions through its pioneering first-in-class and paradigm-shifting synaptic regenerative and synaptic corrective therapeutics designed to restore depleted synapses and reverse synaptic degeneration and dysfunction – offering patients and their families a new reality of hope.

Spinogenix is developing two novel therapeutics: SPG302, which triggers neurons to produce new glutamatergic synapses and restore cognitive, motor, and other functions in ALS, Alzheimer's disease, schizophrenia and other diseases; and SPG601, which works at the synaptic level to correct specific dysfunctions in Fragile X Syndrome (FXS) that underlie many core symptoms. The company has received FDA Orphan Drug and EMA designations for both ALS and FXS as well as FDA Fast Track designation for FXS. More information on Spinogenix can be found at www.spinogenix.com or follow us on LinkedIn. 

Media Contact

Arielle Bernstein Pinsof
FINN Partners
arielle.pinsof@finnpartners.com 

Investor Relations
Dan Albosta
Spinogenix, Inc.
dan@spinogenix.com

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SOURCE Spinogenix