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:
Fully Recruited
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
NR082
Neurophth Biotechnology
Status:
Actively Recruiting
(Beijing, China)
NFS-02
Neurophth Biotechnology
Status:
Actively Recruiting
( Sites in China and Aurora, CO)
NR082
Neurophth Biotechnology
Status:
Actively Recruiting
(Sites: Palo Alto, CA;
Aurora, CO; Philadelphia, PA)
MELAS
OMT-28
Omeicos Therapeutics
Status:
Actively Recruiting
(Site in Germany & Italy)
IW-6463
Tisento Therapeutics
Status:
Study complete, additional studies in planning
Sonlicromanol (KH176)
Khondrion
Study Name:
KHENEREXT
Status:
Fully Recruited
KL1333
Abliva AB
Study Name:
FALCON
Status:
Fully Recruited
MERRF
OMT-28
Omeicos Therapeutics
Status:
Actively Recruiting
(Site in Germany & Italy)
KL1333
Abliva AB
Study Name:
FALCON
Status:
Fully Recruited
MIDD
OMT-28
Omeicos Therapeutics
Status:
Actively Recruiting
(Site in Germany & Italy)
Sonlicromanol (KH176)
Khondrion
Study Name:
KHENEREXT
Status:
Fully Recruited
KL1333
Abliva AB
Study Name:
FALCON
Status:
Fully Recruited
Pearson Syndrome
MNV-201
Minovia
Status:
Actively Recruiting
(Sites in Israel)
PDCD
SL-1009
(Sodium dichloroacetate)
Saol
Status:
Fully recruited
OMT-28
Omeicos Therapeutics
Status:
Actively Recruiting
(Site in Germany & Italy)
Primary Mitochondrial Myopathies (PMM)
Elamipretide (SS-31/MTP-131)
Stealth BioTherapeutics
Study Name:
NuPower
Status:
Fully Recruited
Bocidelpar (ASP0367)
Astellas Pharmaceuticals
Study Name:
Mountainside
Status:
Actively Recruiting
OMT-28
Omeicos Therapeutics
Status:
Actively Recruiting
(Site in Germany & Italy)
TK2 Deficiency
MT1621
UCB
Status:
Open label study active, not recruiting