FDA Authorizes Expanded Access Program for ALS Treatment SPG302

Stella Sarraf, PhD

(Credit: Spinogenix)

According to a new announcement, the FDA has authorized Spinogenix’s expanded access program (EAP) for its investigational amyotrophic lateral sclerosis (ALS) agent SPG302 in the United States for patients living with the disease that meet the program’s eligibility criteria. The company also noted that it recently completed a phase 1/2 trial (NCT05882695) in Australia, and patients were offered continuation of the treatment in the open label extension trial (NCT05882695).¹

“Every 90 minutes someone is diagnosed with ALS, the leading adult-onset motor neuron disease. Tragically, most patients survive only 2 to 5 years post diagnosis and current therapies offer only modest extensions of survival and are often poorly tolerated. Spinogenix is working toward improving the standard of care by addressing, for the first time, synapse loss as a fundamental contributor to disease onset and progression,” Stella Sarraf, PhD, founder and chief executive officer at Spinogenix, told NeurologyLive^®.

The phase 1/2 clinical trial, cleared by the FDA in June 2024, assesses the safety, tolerability, pharmacokinetics, and pharmacodynamics of SPG302 in healthy volunteers and patients living with ALS.² Regarding healthy volunteers, previously announced results showed that treatment with the agent was tolerable and led to changes in plasma levels that aligned with its efficacy in animal models. In the study, investigators randomly assigned healthy volunteers aged between 18 and 55 years to either SPG302 or a placebo. The first part of the trial featured only single ascending doses of SPG302 and the second tested multiple ascending doses of SPG302 for 5 days.

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In phase 2 of the study, patients with ALS aged between 18 and 80 were randomly assigned to by investigators to receive once daily SPG302 or a placebo for 28 days. In this part of the trial, researchers aimed to assess the treatment’s safety and pharmacological properties as well as its effects on disease progression, lung function, and disease biomarkers. Following the completion of the trial, participants had the opportunity to enter the open-label extension study where they would receive the therapy for up to 1 year.

In July 2021, the FDA granted orphan drug designation to SPG302 for the treatment of ALS and has also received preclinical support from the U.S. National Institutes of Health and the Department of Defense.³ SPG302 is a a small molecule compound in development as a once-a-day pill regenerative treatment for ALS and other neurodegenerative diseases. The company noted that the therapy has a unique ability to restore synapses, which are the key connections between neurons that allow patients to think, plan, remember, and control motor functions. The synaptic regenerative activity of SPG302 may represent a first-in-class approach to treating ALS and shows promise to reverse decline in cognitive, respiratory, and motor function, according to Spinogenix.

“Our pioneering synaptic regenerative approach is central to all our clinical programs. With the FDA's support, we are advancing solutions for patients with neurodegenerative, neuropsychiatric, and neurodevelopmental conditions. Significant milestones are on the horizon for 2025, reflecting our commitment to offering patients and their families new treatment options and a new reality of hope,” Sarraf said.

REFERENCES
1. Spinogenix Announces FDA-Authorized Expanded Access Program for SPG302, the First Synaptic Regenerative Therapy to Treat ALS. News Release. Spinogenix. Published May 5, 2025. Accessed May 5, 2025.
2. Spinogenix Announces FDA Clearance of IND Application for SPG302, a Novel Therapy for the Treatment of ALS. News Release. Spinogenix. Published May 29, 2024. Accessed May 1, 2025. https://www.spinogenix.com/spinogenix-announces-fda-clearance-of-ind-application-for-spg302-a-novel-therapy-for-the-treatment-of-als/
3. Spinogenix Granted FDA Orphan Drug Designation for SPG302 for the Treatment of Amyotrophic Lateral Sclerosis. News Release. Spinogenix. Published June 7, 2021. Accessed May 1, 2025. https://www.spinogenix.com/spinogenix-granted-fda-orphan-drug-designation-for-spg302-for-the-treatment-of-amyotrophic-lateral-sclerosis/