AZ-3102

Phase 3Small molecule

AAzafaros A.G.

AZ-3102 is one of 11 small molecule programs targeting GM2 gangliosidosis. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

GM2 GangliosidosisActive, not recruiting

GM1 GangliosidosisActive, not recruiting

GM2 GangliosidosisPhase 2

Active, not recruiting

GM1 GangliosidosisPhase 3

Active, not recruiting

Competition

Competitive Landscape

9 competitors in GM1 Gangliosidosis

#DrugCompanyPhaseTrials

Cytoxan

BBaxter Healthcare Corporation

Busulfex

Medrol

1 4

Rituxan

Roche

Phase 2

1 5

Rapamune

PPrism Pharmaceuticals

Phase 2

1 6

Rayos

RRoxane Laboratories

Phase 2

1 7

AZ-3102

AAzafaros A.G.

Phase 3

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

Timeline

Expected Milestones

Q4 2027

Phase 3Recruiting

GM1 Gangliosidosis

NCT07054515

Phase 3Recruiting

GM1 Gangliosidosis

NCT07082543

Q3 2030

Phase 2Recruiting

GM2 Gangliosidosis

NCT07399704

Clinical

Trials by Indication[1]

GM1 gangliosidosis2 trials

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

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

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

Recruiting·AAzafaros A.G.·Phase 3·NCT07082543·75 enrolled

GM2 gangliosidosis2 trials

A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Recruiting·AAzafaros A.G.·Phase 2·NCT07399704·21 enrolled

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

Sources

References

[1]
ClinicalTrials.gov — 4 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 3[1]
Company: AAzafaros A.G.

AZ-3102

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2027

Q3 2030

Trials by Indication[1]

GM1 gangliosidosis2 trials

GM2 gangliosidosis2 trials

References

Drug Info

Quick Facts

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