Azafaros
Azafaros is a clinical-stage biopharmaceutical company focused on developing innovative treatments for rare genetic diseases. With deep scientific expertise and a strong commitment to patients and families, Azafaros is taking a bold, patient-focused approach to accelerate the development of much-needed therapies for the rare disease community.
For more information, please visit azafaros.com.
Patient Information
Gisela Linthorst, Head of Patient Engagement
[email protected]
Trial Information
- RECRUITING: NAVIGATE Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)
Children’s Hospital and Research Center in Oakland, CA
clinicaltrials.gov/study/NCT07082725
Latest News
First U.S. Study Site Open for Azafaros’ Phase 3 clinical Trial NAVIGATE
The National Niemann-Pick Disease Foundation (NNPDF) is pleased to share the opening of the first U.S. study site for the Phase 3 NAVIGATE clinical trial, sponsored by Azafaros. NAVIGATE is a double-blind placebo-controlled study, investigating the safety and efficacy of oral nizubaglustat, an investigational product designed for the treatment of late-infantile/juvenile forms of Niemann- Pick type C disease and GM1 and GM2 (Tay Sachs and Sandhoff disease).
The opening of the first study site at Children’s Hospital and Research Center in Oakland, California under the leadership of Dr. Hastings marks another significant milestone in advancing treatment research for individuals and families affected by Niemann-Pick disease.
11/19/2025
Update from Azafaros
Azafaros is pleased to share the dosing of the first patient in its pivotal Phase 3 NAVIGATE program. The two global studies will evaluate the safety and efficacy of the company’s lead asset, nizubaglustat, in individuals living with Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses. This marks a significant milestone in Azafaros’ mission to deliver new treatment options for patients affected by these neurodegenerative disorders.
Read the complete announcement.
07/28/2025
Update from Azafaros
Azafaros announces its completion of an oversubscribed €132M Series B financing enabling accelerated development of its lead product nizubaglustat, scheduled to enter Phase 3 studies for Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses later this year.
05/14/2025
Update from Azafaros
Azafaros is pleased to share an update on PRONTO and Phase 2 RAINBOW study data with nizubaglustat, for patients with Niemann-Pick disease type C (NPC) GM1 and GM2 gangliosidoses.
01/08/2025
Update from Azafaros
Azafaros is pleased to share an update on PRONTO and Phase 2 RAINBOW study data with nizubaglustat, for patients with Niemann-Pick disease type C (NPC) GM1 and GM2 gangliosidoses.
12/19/2024
Update from Azafaros
Azafaros is pleased to announce positive topline data from its RAINBOW study, a Phase 2 clinical trial investigating nizubaglustat in patients with a genetic diagnosis of Niemann-Pick disease type C (NPC) or GM2 gangliosidosis.
07/16/2024
Update from Azafaros
Azafaros is pleased to announce the completion of its Phase 2 clinical trial RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients.
03/12/2024
Update from Azafaros
Azafaros is pleased to announce its Phase 2 clinical trial RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled. While unfortunately there are no actively recruiting studies with nizubaglustat in the US at the moment, Azafaros is working to initiate phase 3 studies globally, including the US.
Please see Azarafos’ complete message for the Niemann-Pick community below.
Read complete announcement.
Read the message.
12/20/2023
Update from Azafaros
Azafaros has announced enrollment of their first patient in Phase 2 RAINBOW Study evaluating AZ-3102 in GM2 and NP-C Patients. The RAINBOW study design enables rapid advancement into the company’s planned Phase 3 efficacy trial. Read the announcement.
06/09/2023
Update from Azafaros
Azafaros today announced that the first US site for the Phase II RAINBOW study, led by Dr. Marc Patterson has received Institutional Review Board (IRB) approval. The clinical trial (NCT05758922) is being conducted in the US and Brazil and will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics across two doses of its lead asset, AZ-3102, in patients with Niemann-Pick disease type C and GM2 gangliosidosis. Follow the links below for more information.
Press Release
Q & A on the RAINBOW Study
05/22/2023
NPC Community Meeting with Azafaros hosted by NNPDF & NTSADUpdates from Azafaros
Azafaros receives FDA IND clearance and Fast Track Designation for its lead asset AZ-3102 for treatment of Niemann-Pick disease type C (NPC). Read complete announcement.
Azafaros also announces organizational changes within the company. Read press release.
01/09/2023
Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease type C