Chemistry:Vatiquinone
Vatiquinone (INN, USAN, JAN), also known as α-tocotrienol quinone or vincerenone and by its developmental code names EPI-743 and PTC-743, is a synthetic analogue of coenzyme Q10, antioxidant, and oxidoreductase inhibitor which is under development for the treatment of Friedreich's ataxia, mitochondrial disorders, epilepsy, Leigh disease, methylmalonic acidemia, noise-induced hearing loss, Parkinson's disease, Rett syndrome, and Tourette's syndrome.[1][2][3][4][5] It is taken orally.[6]
The drug inhibits 15-lipoxygenase (15-LOX; ALOX15) and/or other oxidoreductases, enzymes involved in inflammation, ferroptosis, and oxidative stress.[3][4][5] It has also been described as a selective 15-LOX inhibitor however.[6] The antioxidant activities of vatiquinone are said to be 1,000- to 10,000-fold greater than those of resveratrol, idebenone, or coenzyme Q10.[3][5] Mitochondrial dysfunction results in excessive production of reactive oxygen and reactive nitrogen species, which causes redox imbalance and glutathione deficiency.[5] Vatiquinone may increase intracellular glutathione levels and thereby theoretically improve redox balance to treat mitochondrial disorders.[5]
Vatiquinone is or has been under development by Edison Pharmaceuticals and PTC Therapeutics, among other organizations.[1][2] As of May 2026, it is in preregistration for Freidreich's ataxia, phase 3 clinical trials for mitochondrial disorders, phase 2/3 trials for epilepsy, and phase 2 trials for Leigh disease, methylmalonic acidemia, noise-induced hearing loss, Parkinson's disease, and Rett syndrome, whereas no recent development has been reported for Tourette's syndrome.[1]
See also
- List of investigational Parkinson's disease drugs
- List of investigational Tourette's syndrome drugs
- Utreloxastat (EPI-857; PTC-857)
- CU-13001
- Mitoquinone
References
- ↑ 1.0 1.1 1.2 "Vatiquinone -PTC Therapeutics". 18 May 2026. https://adisinsight.springer.com/drugs/800033222.
- ↑ 2.0 2.1 "Delving into the Latest Updates on Vatiquinone with Synapse". 13 May 2026. https://synapse.patsnap.com/drug/7e5d44ca4976435bb93403859189a7a8.
- ↑ 3.0 3.1 3.2 "Emerging antioxidant therapies in Friedreich's ataxia". Frontiers in Pharmacology 15. 2024. doi:10.3389/fphar.2024.1359618. PMID 38379897.
- ↑ 4.0 4.1 "Therapies for Mitochondrial Disease: Past, Present, and Future". Journal of Inherited Metabolic Disease 48 (4). July 2025. doi:10.1002/jimd.70065. PMID 40714961. "Vatiquinone (also known as PTC-743 and EPI-743) is another synthetic analogue of CoQ10, which can bind and modulate oxidoreductases. It has been reported to improve glutathione balance and redox status [96] and was suggested to suppress ferroptosis, an iron-dependent form of cell death [97].".
- ↑ 5.0 5.1 5.2 5.3 5.4 "Clinical Trials in Mitochondrial Disease: An Update on EPI-743 and RP103". Journal of Inborn Errors of Metabolism and Screening 5: 232640981773301. 2017. doi:10.1177/2326409817733013. ISSN 2326-4098.
- ↑ 6.0 6.1 "Characterizing Population Pharmacokinetics of Vatiquinone in Healthy Volunteers and Patients with Friedreich's Ataxia". Pharmaceuticals 18 (9). September 2025. doi:10.3390/ph18091339. PMID 41011210.
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