Spinogenix Presents Topline Phase 2a Clinical Trial Results for SPG302, a First-in-Class ALS Treatment

The First Synaptic Regenerative Drug, SPG302, Led to Slowed Disease Progression in ALS Patients

LOS ANGELES, Nov. 4, 2025 /PRNewswire/ -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics designed to restore synapses to improve health in the lives of patients, presented initial positive topline results from its completed Phase 2a study for SPG302, in patients with Amyotrophic Lateral Sclerosis (ALS). SPG302 is a once-a-day pill being developed as a regenerative treatment with the potential to restore synapses, the key connections between neurons that allow people to think, plan, remember and control movement.

The randomized, double-blind, placebo-controlled Phase 2a study of SPG302 (NCT05882695) assessed safety, tolerability, and pharmacodynamic biomarkers in 23 ALS participants. The trial started with a double-blind placebo-controlled period of 28 days, after which all participants received active treatment for an additional 140 days.  The goal of this first in ALS study was to bridge the gap in the translation from robust preclinical activity to humans in a small study to help with the planning of larger human trials.

Topline results from the trial presented as a poster at the recent Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) Annual Meeting, indicated:

• SPG302 was well tolerated with no treatment-related serious adverse events over 6 months of oral daily dosing of 300mg
• Quantitative and objective biomarker evidence supported dose selection and a drug effect on brain areas impacted by ALS
• Electroencephalogram (EEG) recordings of brain activity revealed improvements in ALS-associated patterns, supporting apparent benefits observed in the rate of functional decline
• 82% of patients treated with SPG302 exhibited a stable or improved rate of decline at end of treatment, as assessed with the ALS Functional Rating Scale - Revised (ALSFRS-R)
• When compared to historical controls from the PRO-ACT database, these patients exhibited on average a 76% slower rate of decline through 6 months

The full data set for the study inclusive of all exploratory endpoints will be shared at an oral presentation by Principal Investigator Dominick B. Rowe, Professor of Neurology in the Faculty of Medicine and Health Sciences at Macquarie University in Sydney, Australia at the International Symposium on ALS/MND on December 5 in San Diego, CA.

"We are very encouraged with the positive Phase 2a trial results for SPG302, in our initial proof of concept ALS study, demonstrating the potential of this new regenerative therapy," said Dr. Stella Sarraf, CEO and Founder at Spinogenix. "As we plan our larger registrational directed ALS trial, we also remain committed to making this investigation therapy available to patients who do not qualify for clinical trials through our FDA-cleared Expanded Access Program."

Spinogenix received FDA authorization earlier this year for an Expanded Access Program, providing access to 200 ALS individuals ineligible for clinical trials in the United States. SPG302 has also been granted FDA and EMA Orphan Drug Designation for the treatment of ALS.

Dr. Merit Cudkowicz, inaugural Executive Director of the Mass General Brigham Neuroscience Institute, Director of the Sean M. Healey & AMG Center for ALS at Mass General Hospital and member of Spinogenix's scientific advisory (institutional consulting agreement) board commented, "there is a need to develop new therapies that target neuronal repair pathways for people with ALS. SPG302 represents an important new approach to target synapse loss in ALS. I look forward to the next phase of drug development to assess the potential of SPG302 and I am grateful to the leadership at Spinogenix for their additional commitment of a parallel expanded access program for people not eligible for their planned clinical efficacy trials."

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 is positioned as a first-in-class approach to treating ALS and has the potential to stabilize or reverse declines in cognitive 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. Compassionate use has been offered to all trials patients for 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
Daniel Davis
FINN Partners for Spinogenix
daniel.davis@finnpartners.com 

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

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SOURCE Spinogenix