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

Name:
FORZINITY

Status:
Approved by FDA

Pipeline_Approved

CoQ10 deficiency

BPM31510IV
BPG Bio, Inc.

Status:
Study in Development

Pipeline_PhaseII

KSS-CPEO

KL1333
Abliva/Pharming

Study Name:
FALCON

Status:
Actively Recruiting

Pipeline_PhaseII-III

Leigh Syndrome

MNV-201
Minovia

Status:
Progressing to clinical development

Pipeline_Leigh_Minovia-3

NV354
Abliva/Pharming

Status:
Progressing to clinical development

Pipeline_Leigh_Abliva_line

LHON

Idebenone
Chiesi Global Rare Diseases

Status:
New Drug Application Pending with FDA, Target Action Date Feb. 2026

Pipeline_Approved_LightGreen

LUMEVOQ (GS010)
GenSight Biologics

Study Name:
REFLECT

Status:
Study active but not recruiting

Pipeline_LHON_GenSight-3

NR082
Neurophth Biotechnology

Status:
Actively Recruiting
(Site in China)

Pipeline_PMM_Astellas-4

MELAS

OMT-28
Omeicos Therapeutics

Study Name:
PMD-OPTION

Status:
Fully Recruited

Pipeline_PhaseII_FullyRecruited

Zagociguat
Tisento Therapeutics

Study Name:
PRIZM

Status:
Actively Recruiting

Pipeline_MELAS_Khondrion

Sonlicromanol (KH176)
Khondrion

Study Name:
KHENERFIN

Status:
Active, Not Yet Recruiting

Pipeline_PhaseIII

KL1333
Abliva/Pharming

Study Name:
FALCON

Status:
Actively Recruiting

Pipeline_PhaseII-III

MERRF

OMT-28
Omeicos Therapeutics

Study Name:
PMD-OPTION

Status:
Fully Recruited

Pipeline_PhaseII_FullyRecruited

KL1333
Abliva/Pharming

Study Name:
FALCON

Status:
Actively Recruiting

Pipeline_PhaseII-III

MIDD

OMT-28
Omeicos Therapeutics

Study Name:
PMD-OPTION

Status:
Fully Recruited

Pipeline_PhaseII_FullyRecruited

Sonlicromanol (KH176)
Khondrion

Study Name:
KHENERFIN

Status:
Active, Not Yet Recruiting

Pipeline_PhaseIII

KL1333
Abliva/Pharming

Study Name:
FALCON

Status:
Actively Recruiting

Pipeline_PhaseII-III

Pearson Syndrome

MNV-201
Minovia

Status:
Actively Recruiting
(Site in Israel)

Pipeline_PhaseII

PDCD

SL-1009
(Sodium dichloroacetate)
Saol

Status:
Under Regulatory Review

Pipeline_Approved_LightGreen

Primary Mitochondrial Myopathies (PMM)

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

Study Name:
NuPower

Status:
Fully Recruited

Pipeline_PhaseIV_FullyRecruited

OMT-28
Omeicos Therapeutics

Study Name:
PMD-OPTION

Status:
Fully Recruited

Pipeline_PhaseII_FullyRecruited

TK2 Deficiency

MT1621
UCB

Name:
Kygevvi

Status:
Approved by FDA 11/3/25

Pipeline_Approved

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