S-606001

S-606001 is a **small molecule inhibitor of glycogen synthase 1 (GYS1)** under development by Shionogi for the treatment of **Pompe disease**, specifically as an add-on therapy to enzyme replacement therapy (ERT) for late-onset Pompe disease (LOPD)[10][5]. Its mechanism of action involves inhibition of the GYS1 gene, which encodes glycogen synthase, thereby aiming to reduce glycogen accumulation in muscle tissues—a core pathology in Pompe disease[10]. Phase 1 and Phase 2 clinical trials have been conducted to evaluate safety, pharmacokinetics, pharmacodynamics, and exploratory efficacy in adults with LOPD on stable ERT regimens[1][9]. S-606001 has received orphan drug designation from the FDA[2]. Primary outcomes in trials include changes in forced vital capacity and 6-minute walk test performance as measures of respiratory and muscle function[1][5][8].

Brand names

S-606001S606001S 606001

Other names

S-606001S606001S 606001