Vatiquinone

Phase 3Small molecule

PPTC Therapeutics

Vatiquinone is one of 34 small molecule programs targeting Friedreich ataxia. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Friedreich AtaxiaActive, not recruiting

Mitochondrial DiseaseActive, not recruiting

Friedreich's AtaxiaNot yet recruiting

Friedreich AtaxiaPhase 2/Phase 3

Active, not recruiting

Mitochondrial DiseasePhase 2/Phase 3

Active, not recruiting

Friedreich's AtaxiaPhase 3

Not yet recruiting

Competition

Competitive Landscape

24 competitors in Friedreich Ataxia

#DrugCompanyPhaseTrials

Skyclarys

Biogen

Approved

1 2

Actos

Takeda

Approved

—3

Ferriprox

Chiesi Farmaceutici S.p.A.

Approved

—4

Chantix

PPrism Pharmaceuticals

Approved

—5

Actimmune

HHorizon Therapeutics Ireland DAC

Phase 3

1 6

Epogen

Johnson & Johnson Pharmaceutical Research & Development, L.L.C.

Phase 2

1 7

Intelence

WWim Janssens

Phase 2

1 8

Aquasol E

NNational Eye Institute (NEI)

Vatiquinone

3 10

Medrol

Pfizer

Phase 1

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2027

Phase 3Active, not recruiting

Friedreich Ataxia

NCT05515536

Q1 2029

Phase 3Not yet recruiting

Friedreich's Ataxia

NCT07681713

Clinical

Trials by Indication[1]

Friedreich ataxia3 trials

A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

Completed·PPTC Therapeutics·Phase 2·NCT05485987·5 enrolled

A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

Completed·PPTC Therapeutics·Phase 2/3·NCT04577352·146 enrolled

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

Active, not recruiting·PPTC Therapeutics·Phase 3·NCT05515536·130 enrolled

Mitochondrial Disease2 trials

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 Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

Completed·PPTC Therapeutics·Phase 3·NCT05218655·101 enrolled

ESS1 trial

Vatiquinone Expanded Access Protocol

Available·MMedical University of South Carolina··NCT07159139

Friedreich's Ataxia1 trial

Evaluation of Long-Term Efficacy of Vatiquinone for the Treatment of Friedreich's Ataxia

Not yet recruiting·PPTC Therapeutics·Phase 3·NCT07681713·120 enrolled

Sources

References

[1]
ClinicalTrials.gov — 7 trial records for Vatiquinone. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Vatiquinone compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Vatiquinone. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — PTC Therapeutics company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: PPTC Therapeutics

Vatiquinone

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2027

Q1 2029

Trials by Indication[1]

Friedreich ataxia3 trials

Mitochondrial Disease2 trials

ESS1 trial

Friedreich's Ataxia1 trial

References

Drug Info

Quick Facts

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