Mitochondrial Diseases
2
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
1
0
0
1
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
PTC TherapeuticsWARREN, NJ
2 programs1
VatiquinonePhase 2/31 trial
EPI-743N/A1 trial
Active Trials
NCT01370447No Longer Available
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
PTC TherapeuticsVatiquinone
AblivaKL1333
Clinical Trials (3)
Total enrollment: 140 patients across 3 trials
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
Start: Sep 2020Est. completion: Dec 202368 patients
Phase 2/3Terminated
A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease
Start: Mar 2019Est. completion: Mar 202172 patients
Phase 1Completed
EPI-743 for Mitochondrial Respiratory Chain Diseases
N/ANo Longer Available
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