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. 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

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

Study Name:
TAZPOWER

Status:
Study Complete

KSS-CPEO

KL1333
Abliva AB

Status:
New study in development

Leigh Syndrome

MNV-BM-BLD
Minovia

Status:
Study Complete

NV354
Abliva AB

Status:
Progressing to clinical development

LHON

LUMEVOQ (GS010)
GenSight Biologics

Status:
Study complete

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

Status:
Study complete

MELAS

IW-6463
Cyclerion Therapeutics

Status:
Study complete

Sonlicromanol (KH176)
Khondrion

Study Name:
KHENEREXT

Learn More

KL1333
Abliva AB

Status:
New study in development

MERRF

KL1333
Abliva AB

Status:
New study in development

MIDD

Sonlicromanol (KH176)
Khondrion

Study Name:
KHENEREXT

Learn More

KL1333
Abliva AB

Status:
New study in development

Pearson Syndrome

MNV-BM-BLD
Minovia Therapeutics

Status:
Enrollment by invitation only

PDCD

Dichloroacetate
Saol

Status:
Study complete

Primary Mitochondrial Myopathies (PMM)

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

Study Name:
NuPower

Learn More

Bocidelpar (ASP0367)
Astellas Pharmaceuticals

Study Name:
Mountainside

Learn More

REN001
Reneo Pharmaceuticals

Study Name:
Stride

Learn More

Seizures

Inherited mitochondrial disease with associated epilepsy phenotype

Vatiquinone (PTC-743)
PTC Therapeutics

Study Name:
MIT-E Study

Learn More

Status: Focused on Drug Resistant Epilepsy, may include Leigh Syndrome, Mitochondrial Encephalopathy (MELAS), Pontocerebellar Hypoplasia Type 6 (PCH6), or Alpers Syndrome patients.

TK2 Deficiency

MT1621
UCB

Status:
Sponsor considering next steps

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