Mitochondrial Disease Therapeutic Pipeline

All new treatments or therapeutics proceed through a defined development path. This path begins with preclinical development and progresses through clinical trials all the way to FDA-approval before ending in patients’ hands.

Several treatments for mitochondrial diseases are already making their way along this path. Soon mito patients and their families will be able to see the treatment approaches being developed, where they are in the development pipeline, and what mitochondrial diseases they target.

Conditions in the Pipeline

  • Barth Syndrome
  • Inherited Mitochondrial Disorders
  • KSS-CPEO
  • Leigh Syndrome
  • LHON
  • MELAS
  • MERRF
  • Mitochondrial Myopathies
  • MIDD
  • Pearson Syndrome
  • PDCD
  • Seizures
  • TK2 Deficiency

Pre-Clinical Development

Non-human lab testing

Phase I

Human safety trial

Phase II

Preliminary human efficacy trial

Phase III

Definitive human safety & efficacy trial

Regulatory Approval

New drug approved & available to patients

Phase I/II tests the safety, side effects, and best dose of a new treatment.
Phase II/III tests how well a new treatment works and compares the new treatment with a standard treatment.

Barth Syndrome

Elamipretide (SS-31/MTP-131)
Stealth BioTherapeutics

Pre-Clinical
Clinical
Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval

KSS-CPEO

KL1333
Abliva AB

Pre-Clinical
Clinical
Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval

Leigh Syndrome

MNV-BM-BLD
PTC Therapeutics

Pre-Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Actively Recruiting
Phase III
Regulatory Approval

NV354
Abliva AB

Pre-Clinical
Clinical
Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval

LHON

LUMEVOQ (GS010)
GenSight Biologics

Pre-Clinical
Clinical
Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Not Recruiting
Regulatory Approval

Elamipretide (SS-31/MTP-131)
Stealth BioTherapeutics

Pre-Clinical
Clinical
Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval

MELAS

Vatiquinone (PTC-743)
PTC Therapeutics

Study Name:
MIT-E Study

Pre-Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Actively Recruiting
Phase III
Regulatory Approval

IW-6463
Cyclerion Therapeutics

Pre-Clinical
Phase I
Phase I/II
Phase II
Actively Recruiting
Phase II/III
Phase III
Regulatory Approval

Sonlicromanol (KH176)
Khondrion

Pre-Clinical
Phase I
Phase I/II
Phase II
Actively Recruiting
Phase II/III
Phase III
Regulatory Approval

KL1333
Abliva AB

Pre-Clinical
Clinical
Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval

MERRF

Vatiquinone (PTC-743)
PTC Therapeutics

Study Name:
MIT-E Study

Pre-Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Actively Recruiting
Phase III
Regulatory Approval

KL1333
Abliva AB

Pre-Clinical
Clinical
Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval

Mitochondrial Myopathies/PMM

Elamipretide (SS-31/MTP-131)
Stealth BioTherapeutics

Pre-Clinical
Clinical
Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval

Bocidelpar (ASP0367)
Astellas Pharmaceuticals

Pre-Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Actively Recruiting
Phase III
Regulatory Approval

REN001
Reneo Pharmaceuticals

Pre-Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Actively Recruiting
Phase III
Regulatory Approval

MIDD

Sonlicromanol (KH176)
Khondrian

Pre-Clinical
Phase I
Phase I/II
Phase II
Actively Recruiting
Phase II/III
Phase III
Regulatory Approval

KL1333
Abliva AB

Pre-Clinical
Clinical
Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval

Pearson Syndrome

MNV-BM-BLD
Minovia Therapeutics

Pre-Clinical
Phase I
Phase I/II
Invitation Only
Phase II
Phase II/III
Phase III
Regulatory Approval

PDCD

Dichloroacetate

Pre-Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Actively Recruiting
Regulatory Approval

Seizures

Vatiquinone (PTC-743)
PTC Therapeutics

Study Name:
MIT-E Study

Pre-Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Actively Recruiting
Phase III
Regulatory Approval

TK2 Deficiency

MT1621
Modis Therapeutics

Pre-Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Not Recruiting
Regulatory Approval

Looking for additional resources?

View our video library to learn more about clinical trials.