GNE Myopathy
3
Pipeline Programs
2
Companies
3
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
3
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Ultragenyx PharmaceuticalNOVATO, CA
2 programs2
SA-ER 500 mgPhase 21 trial
Sialic Acid Extended ReleasePhase 21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Leadiant BiosciencesManNAc
Ultragenyx PharmaceuticalSA-ER 500 mg
Ultragenyx PharmaceuticalSialic Acid Extended Release
Clinical Trials (3)
Total enrollment: 159 patients across 3 trials
Multi-Center Study of ManNAc for GNE Myopathy
Start: Apr 2022Est. completion: Oct 202554 patients
Phase 2Active Not Recruiting
An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy
Start: Jun 2013Est. completion: Feb 201759 patients
Phase 2Completed
A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
Start: May 2012Est. completion: Nov 201346 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