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Primary Mitochondrial Disease

3
Pipeline Programs
4
Companies
6
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
3
0
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Peptide
4100%
+ 2 programs with unclassified modality

Competitive Landscape

4 companies ranked by most advanced pipeline stage

BioTherapeutics Inc
BioTherapeutics IncVA - Blacksburg
2 programs
2
ElamipretidePhase 2Peptide1 trial
elamipretidePhase 2Peptide1 trial
Active Trials
NCT02805790Completed30Est. Mar 2017
NCT02976038Terminated28Est. Apr 2020
Abliva
AblivaSweden - Lund
1 program
1
KL1333Phase 23 trials
Active Trials
NCT05650229Recruiting180Est. Nov 2027
NCT04643249Completed14Est. Dec 2020
NCT03888716Completed72Est. Mar 2021
Stealth BioTherapeutics
2 programs
ElamipretidePHASE_2Peptide
elamipretidePHASE_2Peptide
OMEICOS Therapeutics
OMEICOS TherapeuticsGermany - Berlin
1 program
OMT-28PHASE_21 trial
Active Trials
NCT05972954Completed28Est. Jul 2025

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
OMEICOS TherapeuticsOMT-28
AblivaKL1333
BioTherapeutics Incelamipretide
BioTherapeutics IncElamipretide
AblivaKL1333
AblivaKL1333

Clinical Trials (6)

Total enrollment: 352 patients across 6 trials

OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)

Start: May 2023Est. completion: Jul 202528 patients
Phase 2Completed

Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

Start: Dec 2022Est. completion: Nov 2027180 patients
Phase 2Recruiting

Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)

Start: Dec 2016Est. completion: Apr 202028 patients
Phase 2Terminated

Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study

Start: Aug 2016Est. completion: Mar 201730 patients
Phase 2Completed

Drug-drug Interaction Study of KL1333 in Healthy Subjects

Start: Nov 2020Est. completion: Dec 202014 patients
Phase 1Completed

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

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 actively recruiting trials targeting 352 patients
Peptide is the dominant modality (100% of programs)
4 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.