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

CoQ10 deficiency

BPM31510IV
BPG Bio, Inc.

Status:
Received Rare Pediatric Disease
designation from FDA

Mitochondrial Disease Therapeutic Pipeline

Conditions in the Pipeline

Pre-Clinical Development

Phase I

Phase II

Phase III

Regulatory Approval

Barth Syndrome

CoQ10 deficiency

KSS-CPEO

Leigh Syndrome

LHON

MELAS

MERRF

MIDD

Pearson Syndrome

PDCD

Primary Mitochondrial Myopathies (PMM)

TK2 Deficiency

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