Vatiquinone is a Small Molecule owned by PTC Therapeutics, and is involved in 23 clinical trials, of which 15 were completed, 7 are ongoing, and 1 is planned.

EPI-743 serves as a cofactor for the enzyme NADPH quinone oxidase 1 (NQO1) and arachidonate 15-lipoxygenase inhibitor. Through a redox-based mechanism, it augments endogenous glutathione biosynthesis essential for the control of oxidative stress. Glutathione plays a central role of in cellular redox balance and antioxidant defense system. The drug candidate exhibit therapeutic intervention in the treatment of the disease by checking the accumulation of methylmalonic acid and homo-cysteine as well as to the reduced synthesis of methionine. It aids in controlling the breathing and coordination of movement, thereby reducing the loss of strength and increasing coordination of cellular energy metabolism.15-Lipoxygenase is a key regulator of inflammation and oxidative stress pathways in CNS diseases. Oxidative stress activates 15-lipoxygenase resulting in increased oxidative stress and inflammation. The drug candidate inhibits 15-lipoxygenase to decrease oxidative stress and inflammation and improve cell viability.

The revenue for Vatiquinone is expected to reach a total of $1.3bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Vatiquinone NPV Report.

Vatiquinone was originated by BioElectron Technology and is currently owned by PTC Therapeutics.

Vatiquinone Overview

Vatiquinone (EPI-743) is under development for the treatment of Alpers-Huttenlocher syndrome, Friedreich ataxia, inherited mitochondrial disorders including Leigh syndrome, Alpers syndrome, mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), myoclonic epilepsy with ragged-red fibers (MERRF), pontocerebellar hypoplasia type 6 (PCH6), or other mitochondrial disease, mitochondrial diseases and refractory epilepsy. It is administered orally. It is a small molecule that readily crosses into the central nervous system. It works by targeting the enzyme NADPH quinone oxidoreductase 1 (NQO1) and arachidonate 15-lipoxygenase. It was also under development for the treatment of Rett syndrome, Melas syndrome, Leber’s hereditary optic neuropathy and mitochondrial diseases such as Pearson syndrome, a form of mitochondrial myopathy and Tourette syndrome. It was under development for the treatment of cobalamin C deficiency syndrome (methylmalonic acidemia), RARS2 syndrome, Parkinson's disease, metabolic disease (disorders of energy utilization or oxidation-reduction),Leigh's disease in children and noise-induced hearing loss.

PTC Therapeutics Overview

PTC Therapeutics discovers, develops, and markets medicines for the treatment of rare disorders. Its marketed products include Evrysdi (risdiplam) for spinal muscular atrophy, Translarna (ataluren) and Emflaza (deflazacort) for the treatment of Duchenne muscular dystrophy. The company’s pipeline candidates target the treatment of Huntington’s disease; children with inherited mitochondrial disease; leiomyosarcoma; acute myelogenous leukemia; and aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics works in collaboration with various biotechnology and pharmaceutical companies for advancing its pipeline candidates. The company operates through its subsidiaries in the US and Europe. PTC Therapeutics is headquartered in South Plainfield, New Jersey, the US.

The company reported revenues of (US Dollars) US$538.6 million for the fiscal year ended December 2021 (FY2021), an increase of 41.4% over FY2020. The operating loss of the company was US$374.4 million in FY2021, compared to an operating loss of US$431.8 million in FY2020. The net loss of the company was US$523.9 million in FY2021, compared to a net loss of US$438.2 million in FY2020. The company reported revenues of US$217.1 million for the third quarter ended September 2022, an increase of 31.2% over the previous quarter.

Quick View – Vatiquinone

Report Segments
  • Innovator
Drug Name
  • Vatiquinone
Administration Pathway
  • Oral
Therapeutic Areas
  • Central Nervous System
  • Ear Nose Throat Disorders
  • Genetic Disorders
  • Hematological Disorders
  • Metabolic Disorders
  • Musculoskeletal Disorders
  • Ophthalmology
Key Companies
Highest Development Stage
  • Phase III

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.