VRG50635

VRG50635 is a potent, orally bioavailable small molecule inhibitor of Phosphoinositide kinase FYVE-type zinc finger containing enzyme (PIKfyve), a protein involved in the endolysosomal pathway responsible for cellular cleaning and recycling. The drug was discovered and developed by Verge Genomics using an AI-enabled platform that analyzed human disease tissue data to identify novel therapeutic targets for amyotrophic lateral sclerosis (ALS). By inhibiting PIKfyve, VRG50635 aims to restore endolysosomal function in ALS patient neurons, promoting the clearance of toxic protein aggregates that contribute to motor neuron degeneration. Preclinical studies have shown efficacy in ALS-relevant models, and clinical trials have demonstrated safety and tolerability in healthy volunteers. VRG50635 is currently being evaluated in Phase 1b proof-of-concept studies for both sporadic and familial forms of ALS.

Other names

PIKfyve inhibitor