GM2 Gangliosidosis
2
Pipeline Programs
2
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
2
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
AzafarosAZ-3102
IntraBioIB1001
Clinical Trials (2)
Total enrollment: 43 patients across 2 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
Start: Apr 2023Est. completion: Mar 202613 patients
Phase 2Active Not Recruiting
N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
Start: Jun 2019Est. completion: Jan 202330 patients
Phase 2Completed
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
2 companies competing in this space
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.