Azafaros reports positive data from Phase II trial of nizubaglustat

Azafaros has announced positive results from the Phase II RAINBOW trial of nizubaglustat for treating GM2 gangliosidosis and Niemann-Pick disease type C (NPC). Conducted at three sites in Brazil, the trial enrolled 13 participants aged 12 and older. It assessed the safety, pharmacokinetics, and pharmacodynamics of two doses of nizubaglustat. Following a 12-week, placebo-controlled, double-blind phase, all participants entered an extension phase to receive the drug. The latest data will help determine the target dose for future Phase III trials.

Nizubaglustat, an orally available, brain-penetrant azasugar, targets rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and NPC. It has received various designations from the US FDA, such as rare pediatric disease, orphan drug, IND clearance, and fast-track designations for GM1/GM2 gangliosidoses and NPC. Additionally, the European Medicines Agency (EMA) has granted it orphan medicinal product designation for GM2 gangliosidosis.