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
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval
KSS-CPEO
KL1333
Abliva AB
Pre-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
Phase III
Regulatory Approval
NV354
Abliva AB
Pre-Clinical
Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval
LHON
LUMEVOQ (GS010)
GenSight Biologics
Pre-Clinical
Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval
Elamipretide (SS-31/MTP-131)
Stealth BioTherapeutics
Pre-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
Phase III
Regulatory Approval
IW-6463
Cyclerion Therapeutics
Pre-Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval
Sonlicromanol (KH176)
Khondrion
Pre-Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval
KL1333
Abliva AB
Pre-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
Phase III
Regulatory Approval
KL1333
Abliva AB
Pre-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
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
Phase III
Regulatory Approval
REN001
Reneo Pharmaceuticals
Pre-Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval
MIDD
Sonlicromanol (KH176)
Khondrian
Pre-Clinical
Phase I
Phase I/II
Phase II
Phase II/III
Phase III
Regulatory Approval
KL1333
Abliva AB
Pre-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
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
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
Phase III
Regulatory Approval
TK2 Deficiency
MT1621
Modis Therapeutics