VivoSeq
Other

Mitochondrial Disease

14

Pipeline Programs↗

2

Active Trials↗

12

Companies Involved↗

Mitochondrial Disease is an emerging landscape with 14 programs across 12 companies. The most active players are Minovia Therapeutics Ltd., Omeicos Therapeutics GmbH, Horizon Therapeutics Ireland DAC. 3 therapies are approved with 4 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

41/100

Pipeline Velocity

0/100

Mech. Concentration

20/100

Completion Rate

57/100

Attrition Rate

38/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

M

Minovia Therapeutics Ltd.

1 program

Phase 1O

Omeicos Therapeutics GmbH

1 program

Phase 2H

Horizon Therapeutics Ireland DAC

1 program

Phase 2Eli Lilly logo

Eli Lilly

1 program

Phase 2S

Stealth BioTherapeutics Inc.

1 program

Phase 3P

PTC Therapeutics

1 program

Phase 3

Pipeline

Programs by Phase

Analysis

Modality Breakdown

93%

Small molecule

14 of 15 classified programs — the dominant modality here.

7%ALL OTHERSPeptide17%

Small molecule leads; the ring shows all 15 classified programs, each share of the total. Modality classified for 94% of 18 pipeline programs.

Analysis

Trial Status

16

clinical trials

Completed8 · 50%
Terminated5 · 31%
Recruiting2 · 13%
Withdrawn1 · 6%

Clinical Trials

Trial Activity

16 trials

Applying pGz in Mitochondrial Disease
Recruiting·—·Phase 1·NCT05569122·90 enrolled
An Interventional, Randomised, Double-blind, Parallel-group, Placebo-controlled, Flexible-dose, Adaptive Study of the Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease
Recruiting·PPharming Technologies B.V.·Phase 2·NCT05650229·180 enrolled
OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)
Completed·OOmeicos Therapeutics GmbH·Phase 2·NCT05972954·28 enrolled
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
Completed·PPTC Therapeutics·Phase 3·NCT05218655·101 enrolled
DNA QUANTIFICATION TECHNIQUE AS A INTERPRETATION TOOL IN MITOCHONDRIAL DISEASES
Completed·—·N/A·NCT03216252·10 enrolled
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
Completed·SStealth BioTherapeutics Inc.·Phase 3·NCT05162768·102 enrolled
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
Completed·Amgen logoAmgen·Phase 2·NCT02023866·36 enrolled
Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study
Completed·SStealth BioTherapeutics Inc.·Phase 2·NCT02805790·30 enrolled
Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
Completed·UUniversity of Florida·Phase 3·NCT00432744·24 enrolled
A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease
Completed·AAbliva AB·Phase 1·NCT03888716·72 enrolled
Anesthesia in Patients With Mitochondrial Disease
Terminated·TThe University of Texas Health Science Center, Houston·N/A·NCT02053766·19 enrolled
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
Terminated·PPTC Therapeutics·Phase 2/3·NCT04378075·68 enrolled
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
Terminated·Amgen logoAmgen·Phase 2·NCT02473445·22 enrolled
RG2133 (2',3',5'-Tri-O-Acetyluridine) in Mitochondrial Disease
Terminated·RRepligen Corporation·Phase 1·NCT00060515·12 enrolled
Anesthetic Effects in Mitochondrial Disease
Terminated·—·N/A·NCT01001585·55 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)