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Primary Mitochondrial Myopathy

5
Pipeline Programs
4
Companies
5
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
2
1
1
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Peptide
2100%
+ 4 programs with unclassified modality

Competitive Landscape

4 companies ranked by most advanced pipeline stage

BioTherapeutics Inc
BioTherapeutics IncVA - Blacksburg
1 program
1
elamipretidePhase 3Peptide1 trial
Active Trials
NCT03323749Terminated218Est. Feb 2020
OnKure Therapeutics
3 programs
1
1
1
REN001Phase 2/31 trial
MavodelparPhase 21 trial
REN001Phase 11 trial
Active Trials
NCT03862846Terminated23Est. Apr 2020
NCT04535609Completed213Est. Oct 2023
NCT05267574Terminated155Est. Jan 2024
Astellas
AstellasChina - Shenyang
1 program
1
BocidelparPhase 21 trial
Active Trials
NCT04641962Terminated34Est. May 2024
Stealth BioTherapeutics
1 program
elamipretidePHASE_3Peptide

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
BioTherapeutics Incelamipretide
OnKure TherapeuticsREN001
AstellasBocidelpar
OnKure TherapeuticsMavodelpar
OnKure TherapeuticsREN001

Clinical Trials (5)

Total enrollment: 643 patients across 5 trials

A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension

Start: Oct 2017Est. completion: Feb 2020218 patients
Phase 3Terminated

An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)

Start: Feb 2022Est. completion: Jan 2024155 patients
Phase 2/3Terminated

A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy

Start: May 2021Est. completion: May 202434 patients
Phase 2Terminated

An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients

Start: May 2021Est. completion: Oct 2023213 patients
Phase 2Completed

A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy

Start: May 2019Est. completion: Apr 202023 patients
Phase 1Terminated

Phase Legend

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

Key Insights

1 late-stage (Phase 3) programs, potential near-term approvals
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.