drugset

Drugs / AZ-3102

Follow last change Jun 2026 re-read 3 minutes ago

AZ-3102

also known as Nizubaglustat

Developed for: GM2 gangliosidosis · Niemann-Pick disease type C
Investigated by: Azafaros B.V.

Trials 2

today NCT07399704 · Ph 2 NCT05758922 · Ph 2

2024202520262027202820292030

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 2	NCT07399704	Feb 2026 → Apr 2030 expected	GM2 gangliosidosis, Niemann-Pick disease type C	Azafaros B.V.	Recruiting	No outcome recorded
Phase 2	NCT05758922	Apr 2023 → Apr 2024	GM2 gangliosidosis, Niemann-Pick disease type C	Azafaros B.V.	Completed	Met primary

Press releases naming this drug 15 releases

Date	Issuer	Release
2026-06-05	Azafaros B.V.	Azafaros Announces Publication of Phase 2 RAINBOW Study Data for Nizubaglustat in Molecular Genetics and Metabolism Journal azafaros.com ↗
2026-02-25	Azafaros B.V.	Azafaros Announces Publication of Preclinical Efficacy Data with Nizubaglustat in GM2 Gangliosidosis azafaros.com ↗
2026-01-22	Azafaros B.V.	Azafaros Announces Key Scientific Oral and Poster Presentations on Nizubaglustat Accepted for WORLDSymposium™ 2026 azafaros.com ↗
2025-07-28	Azafaros B.V.	Azafaros Announces Initiation of two Global Phase 3 studies with Nizubaglustat in Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses, respectively azafaros.com ↗
2024-11-19	Azafaros B.V.	Azafaros Announces Key Scientific Oral and Poster Presentations on Nizubaglustat Accepted for WORLDSymposium™ 2025 azafaros.com ↗
2024-09-10	Azafaros B.V.	Positive Niemann-Pick disease type C (NPC) and GM2 gangliosidosis data from nizubaglustat Phase 2 RAINBOW study conducted by Azafaros presented at major metabolic disease conference azafaros.com ↗
2024-07-16	Azafaros B.V.	Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C azafaros.com ↗
2024-03-12	Azafaros B.V.	Azafaros announces completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients azafaros.com ↗
2023-12-11	Azafaros B.V.	Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled azafaros.com ↗
2023-07-08	Azafaros B.V.	Azafaros Announces Enrollment of First Patient in Phase 2 RAINBOW Study Evaluating AZ-3102 in GM2 and NP-C Patients azafaros.com ↗
2023-02-20	Azafaros B.V.	Azafaros Receives Additional Regulatory Designations for AZ-3102 from FDA, EMA and MHRA azafaros.com ↗
2022-04-24	Azafaros B.V.	Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease azafaros.com ↗
2022-02-14	Azafaros B.V.	Azafaros Presents Positive Clinical and Preclinical Data Supporting Development of Lead Compound AZ-3102 in Lysosomal Storage Disorders at the 18th Annual WORLDSymposium™ Conference azafaros.com ↗
2022-02-01	Azafaros B.V.	Azafaros Receives FDA Orphan Drug Designation for AZ-3102 in GM2 Gangliosidosis azafaros.com ↗
2021-04-06	Azafaros B.V.	Azafaros enters clinical stage with AZ-3102, an oral small molecule being developed for rare neurogenetic disorders azafaros.com ↗

Evidence & citations 3 cited values

Every value below carries the sentence it was read from. 2 sources stand behind the page.

Field	Value	Cited text
Known as	AZ-3102	ClinicalTrials.gov intervention name — accepted as the source's own label NCT07399704 ↗ 1 NCT05758922 ↗
Known as	Nizubaglustat	“A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease” NCT07399704 ↗
Route	Oral	“Route of administration: oral” NCT05758922 ↗