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

Study Name:
FALCON

Status:
Actively Recruiting

Learn More

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

Learn More

KL1333
Abliva AB

Study Name:
FALCON

Status:
Actively Recruiting

Learn More

MERRF

KL1333
Abliva AB

Study Name:
FALCON

Status:
Actively Recruiting

Learn More

MIDD

Sonlicromanol (KH176)
Khondrion

Study Name:
KHENEREXT

Status:
Actively Recruiting

Learn More

KL1333
Abliva AB

Study Name:
FALCON

Status:
Actively Recruiting

Learn More

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

Learn More

Bocidelpar (ASP0367)
Astellas Pharmaceuticals

Study Name:
Mountainside

Status:
Actively Recruiting

Learn More

Mavodelpar (REN001)
Reneo Pharmaceuticals

Study Name:
Stride

Status:
Study fully recruited

Seizures

Inherited mitochondrial disease with associated epilepsy phenotype

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

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