Drugs / Nizubaglustat
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 21 trial | ||||||
| Phase 2 | NCT07715084 | Sep 2026 → Jan 2028 expected | Gaucher disease type III | Azafaros B.V. | Not yet recruiting | No outcome recorded |
| Phase 33 trials | ||||||
| Phase 3 | NCT07054515 | Jun 2025 → May 2028 expected | GM1 gangliosidosis, GM2 gangliosidosis, Niemann-Pick disease type C | Azafaros B.V. | Recruiting | No outcome recorded |
| Phase 3 | NCT07082543 | Jun 2025 → Feb 2028 expected | GM1 gangliosidosis, GM2 gangliosidosis | Azafaros B.V. | Recruiting | No outcome recorded |
| Phase 3 | NCT07082725 | Jun 2025 → May 2028 expected | Niemann-Pick disease type C | Azafaros B.V. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 10
| Date | Issuer | Release |
|---|---|---|
| 2026-06-05 | Azafaros B.V. | Results Azafaros Announces Publication of Phase 2 RAINBOW Study Data for Nizubaglustat in Molecular Genetics and Metabolism Journal azafaros.com ↗
The study also showed encouraging signs of clinical efficacy, reducing disease progression and seizure burden in patients treated with nizubaglustat . |
| 2026-02-25 | Azafaros B.V. | Results Azafaros Announces Publication of Preclinical Efficacy Data with Nizubaglustat in GM2 Gangliosidosis azafaros.com ↗
The research assessed how the drug’s exposure relates to its effects in the brain, demonstrating a significant increase in both survival (22%; 26 days) and assessments of movement and behavior at 16 weeks versus untreated Sandhoff diseased mice. |
| 2025-01-08 | Azafaros B.V. | Regulatory Azafaros granted important regulatory designations and clearance by European authorities for global Phase 3 studies, to be initiated in 2025 azafaros.com ↗
Azafaros B.V. today announced that its lead asset, nizubaglustat, has been granted orphan drug designation from regulatory authorities in both the United States and the European Union for the treatment of GM1 gangliosidosis. |
| 2024-09-10 | Azafaros B.V. | Results 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 ↗
The results of part one of the study, designed to determine the safety, pharmacodynamics, and pharmacokinetics of the Company’s lead asset nizubaglustat, demonstrated the compound had a positive safety profile and was well-tolerated in the 13 participants in the study. |
| 2024-07-16 | Azafaros B.V. | Results Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C azafaros.com ↗
Azafaros B.V. today 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). |
| 2023-02-20 | Azafaros B.V. | Regulatory Azafaros Receives Additional Regulatory Designations for AZ-3102 from FDA, EMA and MHRA azafaros.com ↗
In the last two weeks, Azafaros has been granted the following designations: Two Rare Pediatric Disease Designations (RPDD) by the United States Food and Drug Administration (FDA) for the treatment of GM1 and GM2 gangliosidoses Orphan Medicinal Product Designation (OMPD) by the European Medicines Agency (EMA) for the treatment of GM2 gangliosidosis An Innovation Passport by the UK Medicines and Healthcare Products Regulatory Agency (MHRA) for the treatment of GM1 and GM2 gangliosidoses |
| 2023-01-05 | Azafaros B.V. | Regulatory BREAKING NEWS: Azafaros Receives FDA’s IND Clearance and Fast Track Designation azafaros.com ↗
In addition to the IND clearance, the FDA has granted Fast Track Designation for the investigation of AZ-3102 for GM1 and GM2 gangliosidoses and NP-C. |
| 2022-04-24 | Azafaros B.V. | Regulatory Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease azafaros.com ↗
Leiden, The Netherlands, April 24, 2022 – Azafaros B.V. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102, a novel small molecule with a unique dual mode of action, in Niemann-Pick disease type C (NP-C). |
| 2022-02-14 | Azafaros B.V. | Results 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 ↗
Azafaros B.V. announced positive clinical data from its first-in-human Phase 1 study with AZ-3102, the company’s lead program in development as a potential treatment for paediatric neurogenetic lysosomal storage disorders (LSDs). |
| 2022-02-01 | Azafaros B.V. | Regulatory Azafaros Receives FDA Orphan Drug Designation for AZ-3102 in GM2 Gangliosidosis azafaros.com ↗
Azafaros B.V. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102, a novel oral small molecule, in GM2 gangliosidosis including both Sandhoff and Tay-Sachs diseases. |
All press releases naming this drug 17 releases
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Nizubaglustat | “A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis” NCT07082543 ↗3“Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)” NCT07715084 ↗ “oral nizubaglustat (AZ-3102)” NCT07054515 ↗ |
| Known as | AZ-3102 | “An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and...” NCT07082725 ↗3“A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis” NCT07082543 ↗ “Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)” NCT07715084 ↗ “oral nizubaglustat (AZ-3102)” NCT07054515 ↗ |
| Route | Oral | “Oral dispersible tablets” NCT07054515 ↗ |