First-In-Human Study

Our Phase I First-In-Human Study with AZ-3102 (now nizubaglustat)

Our Phase I Study in healthy volunteers assessed the safety and tolerability of nizubaglustat, with specific measurements on drug levels and biomarkers in plasma and cerebrospinal fluid (CSF). The study design served the future development of nizubaglustat in neurological lysosomal storage disorders, including GM1 and GM2 Gangliosidoses, and Niemann-Pick type C disease. Nizubaglustat successfully completed this study and the results have been published in the peer-reviewed journal Molecular Genetics and Metabolism.