Spinogenix's SPG302, the First Synaptic Regenerative Therapy to Treat ALS, Granted Orphan Drug Designation by the European Medicines Agency

LOS ANGELES, June 3, 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 SPG302 for the treatment of people living with Amyotrophic Lateral Sclerosis (ALS). SPG302 is being developed as the first synaptic regenerative approach for the treatment of ALS, with the potential to restore function in cognition, movement, and respiration.

"Every 90 minutes someone is diagnosed with ALS, highlighting the urgent need for new innovative treatment options," Dr. Stella Sarraf, Spinogenix Chief Executive Officer and Founder. "At Spinogenix, our team is working tirelessly to help provide access to SPG302 to as many people battling ALS as we can. Achieving this new significant milestone with EMA ODD designation for SPG302 in ALS, takes us one step closer in furthering that commitment, allowing us the possibility to now expand development of   SPG302 to the thousands of individuals living with ALS across Europe."

In its mission to help patients around the world, Spinogenix recently received FDA authorization for an Expanded Access Program, providing access to 200 ALS individuals ineligible for clinical study in the United States. The company recently announced the completion of a Phase 2 trial in Australia and has offered patients the opportunity for continued treatment in an open label extension. Additional information on the trial (NCT05882695) and open label extension (NCT06903286) may be found on ClinicalTrials.gov.

"SPG302 has the potential to reverse an unaddressed, yet critical, aspect of ALS pathogenesis: an early and progressive loss of glutamatergic synapses," said Dr. Peter Vanderklish, Spinogenix Chief Scientific Officer. "The evidence for a central role of synapse loss in ALS continues to grow, underscoring the need to evaluate a synaptic regenerative approach in the disease. Such an approach has the added translational advantage of eliciting changes that are measurable by objective, quantitative neurophysiological tools, such as EEG, which can track signature changes in brain activity that correlate with ALS symptoms."  

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 an ODD benefit from a 10-year period of market exclusivity following authorization, protocol assistance, and regulatory fee reductions.

About SPG302
SPG302 is a once-a-day pill being developed as a regenerative treatment for neurodegenerative and neuropsychiatric diseases with the unique ability to restore synapses, the key connections between neurons that allow people to think, plan, remember, and control movement. The synaptic regenerative activity of SPG302 represents a first-in-class approach to treating these diseases and has the potential to reverse declines in cognitive, respiratory, and motor function. SPG302 has been granted U.S. FDA and EMA Orphan Drug Designation for the treatment of ALS and has received preclinical support from the U.S. National Institutes of Health and the Department of Defense.

Spinogenix has completed a Phase 1/2 study in Australia (NCT05882695) assessing the safety, pharmacokinetics and pharmacodynamics of once-daily dosing of SPG302 in healthy volunteers and minimally 24 weeks of treatment in ALS patients with open label extension up to one year. Additional information on the global clinical trials evaluating SPG302 for the treatment of schizophrenia and Alzheimer's disease can be found on ClinicalTrials.gov (NCT06442462 and NCT06427668).

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 ALS as well as FDA Orphan Drug and Fast Track designations 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