SPG302 shows potential to slow disease progression and improve neuronal function in ALS patients
Spinogenix, Inc., a clinical-stage biopharmaceutical company developing synaptic regenerative therapeutics, has reported positive topline results from its completed Phase 2a trial of SPG302 in patients with Amyotrophic Lateral Sclerosis (ALS).
SPG302 is an oral, once-daily therapy designed to restore synaptic connections between neurons, potentially improving cognitive and motor function in patients with neurodegenerative diseases.
Overview of the Study
The randomized, double-blind, placebo-controlled Phase 2a study (NCT05882695) enrolled 23 participants with ALS. The trial included a 28-day placebo-controlled phase, followed by 140 days of open-label treatment with 300 mg SPG302.
The study aimed to evaluate safety, tolerability, and pharmacodynamic biomarkers to support future, larger clinical trials.
Key Findings
According to results presented at the Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) Annual Meeting, SPG302 demonstrated encouraging safety and early efficacy signals:
- Well-tolerated: No treatment-related serious adverse events were reported over six months of dosing.
- Biomarker support: Objective biomarker data confirmed dose selection and effects in brain regions impacted by ALS.
- EEG improvement: Electroencephalogram results indicated improved brain activity patterns associated with ALS.
- Slower disease decline: 82% of patients treated with SPG302 exhibited stable or improved ALS Functional Rating Scale (ALSFRS-R) scores.
- Comparative results: When compared with historical controls from the PRO-ACT database, patients treated with SPG302 showed a 76% slower rate of decline over six months.
The complete dataset, including exploratory endpoints, will be presented by Prof. Dominick B. Rowe of Macquarie University at the International Symposium on ALS/MND on December 5 in San Diego, California.
Executive and Expert Commentary
Dr. Stella Sarraf, CEO and Founder of Spinogenix, said the results provide early proof of concept for SPG302 as a regenerative therapy for ALS.
“This trial demonstrates the potential of SPG302 to address synaptic loss in ALS,” Sarraf said. “As we prepare for a larger registrational trial, we remain committed to making this therapy accessible through our FDA-cleared Expanded Access Program.”
Dr. Merit Cudkowicz, Executive Director of the Mass General Brigham Neuroscience Institute and a member of the company’s advisory board, emphasized the therapy’s novel mechanism.
“SPG302 represents a new approach targeting neuronal repair in ALS,” Cudkowicz noted. “It’s encouraging to see Spinogenix also extend access to patients not eligible for clinical trials.”
Expanded Access and Regulatory Designations
Spinogenix received FDA authorization earlier this year for an Expanded Access Program, enabling up to 200 ALS patients who are ineligible for clinical trials to receive SPG302 treatment in the United States.
The therapy has also earned Orphan Drug Designation from both the U.S. FDA and the European Medicines Agency (EMA).
About SPG302
SPG302 is a first-in-class synaptic regenerative therapeutic under development for ALS, Alzheimer’s disease, schizophrenia, and other neurodegenerative or neuropsychiatric disorders.
It aims to restore lost synapses and improve neuronal communication. The drug has shown strong preclinical support from the U.S. National Institutes of Health and the Department of Defense.
Spinogenix has completed a Phase 1/2 trial in Australia assessing SPG302’s safety, pharmacokinetics, and pharmacodynamics, with compassionate use extended to participants for up to one year. Additional trials are underway for schizophrenia (NCT06442462) and Alzheimer’s disease (NCT06427668).
About Spinogenix
Spinogenix, Inc. is a U.S.-based biopharmaceutical company developing therapeutics to restore and correct synaptic function in neurodegenerative, neuropsychiatric, and neurodevelopmental disorders. The company’s pipeline includes:
- SPG302 – a synapse-regenerating therapy for ALS and other neurological conditions
- SPG601 – a synaptic corrective therapy for Fragile X Syndrome (FXS)
Both programs have received Orphan Drug and Fast Track designations from regulatory authorities.
