Vatiquinone
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| Other names | α-Tocotrienol quinone; Vincerenone; EPI-743; EPI743; PTC-743; PTC743 |
| Routes of administration | Oral[1] |
| Drug class | ALOX15 inhibitor; Antioxidant |
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| Formula | C29H44O3 |
| Molar mass | 440.668 g·mol−1 |
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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.[2][3][4][5][6] It is taken orally.[1]
The drug inhibits 15-lipoxygenase (15-LOX; ALOX15) and/or other oxidoreductases, enzymes involved in inflammation, ferroptosis, and oxidative stress.[4][5][6] It has also been described as a selective 15-LOX inhibitor however.[1] The antioxidant activities of vatiquinone are said to be 1,000- to 10,000-fold greater than those of resveratrol, idebenone, or coenzyme Q10.[4][6] Mitochondrial dysfunction results in excessive production of reactive oxygen and reactive nitrogen species, which causes redox imbalance and glutathione deficiency.[6] Vatiquinone may increase intracellular glutathione levels and thereby theoretically improve redox balance to treat mitochondrial disorders.[6]
Vatiquinone is or has been under development by Edison Pharmaceuticals and PTC Therapeutics, among other organizations.[2][3] 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.[2]
See also
References
- 1 2 3 Hu Y, Gao L, Lee L, Cherry JJ, Kong R (September 2025). "Characterizing Population Pharmacokinetics of Vatiquinone in Healthy Volunteers and Patients with Friedreich's Ataxia". Pharmaceuticals. 18 (9). doi:10.3390/ph18091339. PMC 12472293. PMID 41011210.
- 1 2 3 "Vatiquinone -PTC Therapeutics". AdisInsight. 18 May 2026. Retrieved 1 June 2026.
- 1 2 "Delving into the Latest Updates on Vatiquinone with Synapse". Synapse. 13 May 2026. Retrieved 1 June 2026.
- 1 2 3 Edzeamey FJ, Ramchunder Z, Pourzand C, Anjomani Virmouni S (2024). "Emerging antioxidant therapies in Friedreich's ataxia". Frontiers in Pharmacology. 15 1359618. doi:10.3389/fphar.2024.1359618. PMC 10876797. PMID 38379897.
- 1 2 Ball M, van Bergen NJ, Compton AG, Thorburn DR, Rahman S, Christodoulou J (July 2025). "Therapies for Mitochondrial Disease: Past, Present, and Future". Journal of Inherited Metabolic Disease. 48 (4) e70065. doi:10.1002/jimd.70065. PMC 12301291. 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].
- 1 2 3 4 5 Enns GM, Cohen BH (2017). "Clinical Trials in Mitochondrial Disease: An Update on EPI-743 and RP103". Journal of Inborn Errors of Metabolism and Screening. 5: 232640981773301. doi:10.1177/2326409817733013. ISSN 2326-4098.