In ALS, the accumulation of abnormal proteins is thought to contribute to disease progression. Previous research has shown that the pathways responsible for regulating healthy protein levels and clearing abnormal proteins can be dysregulated in the disease. This dysregulation contributes to abnormal protein aggregation inside cells, disrupting normal cellular function. VRG50635 is a small-molecule inhibitor that reduces the activity of a molecule called PIKfyve. Reduction in PIKfyve activity is believed to promote pathways for clearing these toxic and aggregated proteins inside cells. In the lab, VRG50635 has been shown to extend survival in cell and animal models of ALS. This Phase 1 study will investigate VRG50635 in a clinical setting by recruiting 50 participants. The study is open-label with no placebo group, meaning all participants will receive the active drug for up to 80 weeks. Researchers will monitor participants to ensure the drug is safe, determine the appropriate dosage, learn how the body metabolizes the drug, and look for exploratory indications of its biological effects on ALS to determine the next steps.
View the ALS Clinical Trials Unboxed webinar on VRG50635 here.