PGL-001

PGL-001 (2-amino-4,7-dimethyl-benzothiazol-6-ol) is a small molecule benzothiazole derivative that was developed by PolyGene AG for the treatment of Huntington's disease (HD). Identified through a high-throughput screen for inhibitors of huntingtin (HTT) protein aggregation, PGL-001 is structurally related to riluzole. Its primary mechanism of action is the inhibition of the self-assembly of polyglutamine-expanded huntingtin exon 1 fragments into insoluble fibrils, which are thought to be neurotoxic. While PGL-001 showed promise in preclinical models, including cell culture and in vitro assays, its clinical development was halted. A Phase 2 clinical trial (NCT00216515) evaluating its safety and efficacy in HD patients was terminated in 2006 due to a lack of efficacy in preliminary analyses.

Other names

2-amino-4,7-dimethyl-benzothiazol-6-ol