FDA Clears Phase 1/2 Clinical Trial for Spinogenix’s ALS Treatment SPG302


The cleared phase 1/2 clinical trial will assess SPG302 as a once-a-day pill among patients with amyotrophic lateral sclerosis.

Stella Sarraf, PhD  (Credit: LinkedIn)

Stella Sarraf, PhD

(Credit: LinkedIn)

According to a recent announcement, the FDA has cleared an investigational new drug (IND) application paving the way for Spinogenix’s phase 1/2 clinical trial assessing the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics SPG302 as a potential treatment for patients with amyotrophic lateral sclerosis (ALS).1

SPG302 is a once-a-day pill in development as a 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.

“Having completed the phase 1 safety study in healthy participants in Australia, we are thrilled to have gained FDA acceptance of our U.S. IND for SPG302 in ALS,” Stella Sarraf, PhD, founder and chief executive officer at Spinogenix, said in a statement.1 “SPG302’s unique approach to regenerate synapses offers a fundamentally different treatment modality, focusing on synapse loss which is central to ALS. Current treatments have not sufficiently met the needs of ALS patients, as slowing disease progression alone is not enough. We are committed to advancing SPG302 with the hope of providing a new, transformative therapeutic that can significantly improve the lives of those battling this devastating disease.”

READ MORE: EU Approves Biogen’s Tofersen as First Treatment for SOD1 ALS

Top Clinical Takeaways

  • SPG302 aims to treat ALS by regenerating synapses, potentially reversing declines in cognitive, respiratory, and motor functions.
  • The FDA has granted orphan drug designation to SPG302, providing benefits like exemption from user fees and tax credits for clinical research.
  • The phase 1/2 clinical trial will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of SPG302, with initial dosing in healthy volunteers showing promising results.

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. Currently, SPG302 is being tested in an ongoing phase 1/2 study (NCT05882695) in Australia, which has completed dosing of healthy volunteer cohorts. Reports from the trial have indicated that the treatment demonstrated tolerability and had alignment of plasma levels as observed with its efficacy in animal models. The company noted that the dosing of patients with ALS for the trial in Australia initiated earlier this year in April.

In the Australian trial, investigators randomly assigned healthy volunteers aged between 18 and 55 years to either SPG302 or a placebo. The first part of the trial features only single ascending doses of SPG302 while the second tests multiple ascending doses of SPG302 for 5 days.

In phase 2 of the study, patients with ALS aged between 18 and 80 will be randomly assigned to by investigators to receive once daily SPG302 or a placebo for 28 days. In this part of the trial, researchers will aim 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 will have the opportunity to enter the open-label extension study where they would receive the therapy for up to 1 year.

“ALS is a complex and varied disease, affecting cognitive and motor functions as well as speech and respiration. Spinogenix’s new approach works at the synaptic level to regenerate synapses. This first study in people with ALS is an important step towards determining whether SPG302 helps recover lost functions in motor and cognitive symptom domains,” Merit Cudkowicz, MD, MSc, chair of neurology at Massachusetts General Hospital, said in a statement.1

1. Spinogenix Announces FDA Clearance of IND Application for SPG302, a Novel Therapy for the Treatment of ALS. News Release. Published May 29, 2024. Accessed June 14, 2024. https://www.spinogenix.com/spinogenix-announces-fda-clearance-of-ind-application-for-spg302-a-novel-therapy-for-the-treatment-of-als/
2. Spinogenix Granted FDA Orphan Drug Designation for SPG302 for the Treatment of Amyotrophic Lateral Sclerosis. News Release. Published June 7, 2024. Accessed June 14, 2024. https://www.biospace.com/article/releases/spinogenix-granted-fda-orphan-drug-designation-for-spg302-for-the-treatment-of-amyotrophic-lateral-sclerosis/
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