Synapse-Generating Small Molecule for ALS Receives Nod from FDA for Clinical Trial

The Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for SPG302 (Spinogenix, San Diego, CA) for evaluation as a potential treatment for people with amyotrophic lateral sclerosis (ALS) as a once-daily pill. The safety, tolerability, and pharmacokinetics of SPG302 will be investigated in a planned phase 1/2 clinical trial. SPG302 is a synthetic small molecule drug capable of penetrating the blood-brain barrier with a unique mechanism of action that induces synaptogenesis.

SPG302 was previously granted Orphan Drug Designation (ODD) for the treatment of ALS in 2021. According to a statement from Spirogenix, the National Institutes of Health (NIH) and Department of Defense (DoD) have issued preclinical support for the investigational treatment, with the latter providing nearly $1 million in grant funding to advance clinical testing. An ongoing phase1/2 randomized, double-blind, placebo-controlled, single and multiple ascending dose study (NCT05882695) is underway in Australia to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of SPG302 in healthy volunteers and participants with ALS. Outcome measures for this study include electrophysiological, biomarker, respiratory and behavioral assessments. The phase 1 portion of this study, evaluating safety, has concluded.

“ALS is a complex and varied disease, affecting cognitive and motor functions as well as speech and respiration,” said Dr. Merit Cudkowicz, Chair of the Massachusetts General Hospital Department of Neurology and Spinogenix Advisory Board member. “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.”