AZ-3102

Phase 2Small molecule

AAzafaros A.G.

AZ-3102 is one of 8 small molecule programs targeting GM2 gangliosidosis. It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

GM2 GangliosidosisActive, not recruiting

Gaucher DiseaseNot yet recruiting

Niemann-Pick Disease Type CNot yet recruiting

GM2 GangliosidosisPhase 2

Active, not recruiting

Gaucher DiseasePhase 2

Not yet recruiting

Niemann-Pick Disease Type CPreclinical

Not yet recruiting

Competition

Competitive Landscape

5 competitors in Gaucher Disease

#DrugCompanyPhaseTrials

Busulfex

Akero Therapeutics

Approved

—2

Cytoxan

BBaxter Healthcare Corporation

Approved

—3

ELELYSO

PPfizer's Upjohn has merged with Mylan to form Viatris Inc.

Approved

3 4

AZ-3102

AAzafaros A.G.

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q2 2027

Phase 2Active, not recruiting

GM2 Gangliosidosis

NCT07399704

Q1 2028

Phase 3Active, not recruiting

Gangliosidoses, GM2

NCT07082543

Q2 2028

Phase 3Recruiting

Niemann-Pick Type C Disease

NCT07054515

Q3 2029

Phase 2Not yet recruiting

Gaucher Disease

NCT07715084

Clinical

Trials by Indication[1]

GM2 gangliosidosis2 trials

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease

Active, not recruiting·AAzafaros A.G.·Phase 2·NCT05758922·13 enrolled

Open-label Study to Evaluate the Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease, With or Without Previous Administration of Miglustat

Active, not recruiting·AAzafaros A.G.·Phase 2·NCT07399704·21 enrolled

Niemann-Pick Type C Disease1 trial

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 Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

Recruiting·AAzafaros A.G.·Phase 3·NCT07054515·147 enrolled

Niemann-Pick disease type C1 trial

An Open-label, Roll-over Trial to Evaluate the Long-term Safety and Tolerability of Nizubaglustat (AZ-3102) in Participants With Rare Lysosomal Disorders Who Have Participated in Previous Nizubaglustat Trials and Wish to Continue Nizubaglustat Treatment

Not yet recruiting·AAzafaros B.V.·Preclinical·NCT07885917·218 enrolled

Gangliosidoses, GM21 trial

Active, not recruiting·AAzafaros A.G.·Phase 3·NCT07082543·75 enrolled

Gaucher Disease1 trial

96-week, Open-label, Phase 2 Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)

Not yet recruiting·AAzafaros B.V.·Phase 2·NCT07715084·20 enrolled

Sources

References

[1]
ClinicalTrials.gov — 6 trial records for AZ-3102. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — AZ-3102 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for AZ-3102. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Azafaros A.G. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: AAzafaros A.G.

AZ-3102

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q2 2027

Q1 2028

Q2 2028

Q3 2029

Trials by Indication[1]

GM2 gangliosidosis2 trials

Niemann-Pick Type C Disease1 trial

Niemann-Pick disease type C1 trial

Gangliosidoses, GM21 trial

Gaucher Disease1 trial

References

Drug Info

Quick Facts

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