Azafaros press release:  Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C

We are pleased to share the latest press release from Azafaros related to their study with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C.  

The full press release can be read here and the highlights are below:

 Azafaros B.V. announced positive topline data from its RAINBOW study, a Phase 2 clinical trial investigating nizubaglustat in patients with a genetic diagnosis of either GM2 gangliosidosis or Niemann-Pick disease type C (NPC). 

 The trial was conducted across three sites in Brazil, involving 13 patients older than 12 years of age, with the aim of determining the safety, pharmacodynamics, and pharmacokinetics of two different doses of nizubaglustat. The results will guide the identification of the target dose for Azafaros’ planned Phase 3 pivotal studies. 

With the 12-week, double-blind and placebo-controlled part of the study complete, patients have now entered the extension phase, where all participants receive the study drug. 

The data from the RAINBOW study will be presented at the SSIEM Annual Symposium 2024, which will take place on 3 – 6 September in Porto, Portugal.