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Azafaros is a clinical stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms, a compound library from Leiden University, and led by a team of highly experienced industry experts. Azafaros aims to build a pipeline of disease-modifying therapeutics to offer patients and their families new treatment options. The company’s lead clinical-staged program is AZ-3102 a small molecule, orally available, brain penetrant azasugar, with the potential to treat GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) and Niemann-Pick disease type C (NP-C). By applying its know-how, network, and courage, the Azafaros team challenges traditional development pathways to rapidly bring new drugs to the rare disease patients who need them.

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Patient and Trial Information

Gisela Linthorst, Head of Patient Engagement

Latest News

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.

Read complete announcement.



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.



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


NPC Community Meeting with Azafaros hosted by NNPDF & NTSAD
February 8, 2023  |  8:00 pm EST


Updates 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.



Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease type C

Read complete announcement.



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.



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