Alexander Disease
1
Pipeline Programs
1
Companies
2
Clinical Trials
1 recruiting
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
0
0
1
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
RNA Therapeutic
1100%
+ 1 programs with unclassified modality
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Ionis PharmaceuticalsCARLSBAD, CA
2 programs1
zilganersenPhase 3RNA Therapeutic1 trial
Evaluation of Outcome Metrics in Alexander DiseaseN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
Ionis Pharmaceuticalszilganersen
Ionis PharmaceuticalsEvaluation of Outcome Metrics in Alexander Disease
Clinical Trials (2)
Total enrollment: 254 patients across 2 trials
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
Start: Jun 2021Est. completion: Sep 202954 patients
Phase 3Active Not Recruiting
Evaluation of Outcome Metrics in Alexander Disease
Start: Jan 2016Est. completion: Dec 2030200 patients
N/ARecruiting
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
1 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 254 patients
1 companies competing in this space