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Azafaros Media Release | March 12, 2024

Azafaros announces completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients.

Read the full statement from Azafaros here:

Here is a summary of the article:

  • Study Completion: Azafaros has completed a 12-week Phase 2 clinical study, RAINBOW, evaluating nizubaglustat in patients with GM2 gangliosidosis or Niemann-Pick disease type C (NPC).

  • Study Details: The study was randomized, double-blind, placebo-controlled, conducted in Brazil, and involved 13 patients. It aimed to determine the safety, pharmacodynamics, and pharmacokinetics of nizubaglustat.

  • Next Steps: Patients are now entering the extension phase where all will receive the study drug. Top line results are expected in Q2 2024, with presentations to the scientific community planned for later this year.

  • Drug Information: Nizubaglustat is a small molecule with a dual mode of action, developed for rare lysosomal storage disorders with neurological involvement. It has received several designations and support from regulatory agencies.


To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere."

Ways to support NP-C in Australia:

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