Mitochondrial Disease Therapeutic Pipeline
Several treatments for mitochondrial diseases are already making their way along this path. Now, 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
- KSS-CPEO
- Leigh Syndrome
- LHON
- MELAS
- MERRF
- Primary Mitochondrial Myopathies (PMM)
- MIDD
- Pearson Syndrome
- Pyruvate Dehydrogenase Complex Deficiency (PDCD)
- Seizures
- TK2 Deficiency

Pre-Clinical Development

Phase I

Phase II

Phase III

Regulatory Approval
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
Study Name:
TAZPOWER
Status:
Study Complete

KSS-CPEO
KL1333
Abliva AB
Study Name:
FALCON
Status:
Actively Recruiting

Leigh Syndrome
MNV-201
Minovia
Status:
Progressing to clinical development

NV354
Abliva AB
Status:
Progressing to clinical development

LHON
LUMEVOQ (GS010)
GenSight Biologics
Study Name:
REFLECT
Status:
Study active but not recruiting

Elamipretide (SS-31/MTP-131)
Stealth BioTherapeutics
Status:
Study complete

MELAS
IW-6463
Cyclerion Therapeutics
Status:
Study complete, additional studies in planning

Sonlicromanol (KH176)
Khondrion
Study Name:
KHENEREXT
Status:
Actively Recruiting

KL1333
Abliva AB
Study Name:
FALCON
Status:
Actively Recruiting

MERRF
KL1333
Abliva AB
Study Name:
FALCON
Status:
Actively Recruiting

MIDD
Sonlicromanol (KH176)
Khondrion
Study Name:
KHENEREXT
Status:
Actively Recruiting

KL1333
Abliva AB
Study Name:
FALCON
Status:
Actively Recruiting

Pearson Syndrome
MNV-201
Minovia
Status:
Progressing to clinical development

PDCD
SL-1009
(Sodium dichloroacetate)
Saol
Status:
Study fully recruited

Primary Mitochondrial Myopathies (PMM)
Elamipretide (SS-31/MTP-131)
Stealth BioTherapeutics
Study Name:
NuPower
Status:
Actively Recruiting

Bocidelpar (ASP0367)
Astellas Pharmaceuticals
Study Name:
Mountainside
Status:
Actively Recruiting

Mavodelpar (REN001)
Reneo Pharmaceuticals
Study Name:
Stride
Status:
Study fully recruited

Seizures
Vatiquinone (PTC-743)
PTC Therapeutics
Study Name:
MIT-E Study
Status:
Study fully recruited

Please Note: This trial is focused on Drug Resistant Epilepsy, may include: Leigh Syndrome, Mitochondrial Encephalopathy (MELAS), Pontocerebellar Hypoplasia Type 6 (PCH6), or Alpers Syndrome patients, as well as any other mitochondrial disease subtype with seizures as part of the disease.
TK2 Deficiency
MT1621
UCB
Status:
Open label study active, not recruiting
