Mito research around the world

Vatiquinone (PTC-743)

Vatiquinone is an investigational treatment that is structurally related to vitamin E and can be broadly classified as an antioxidant. It is being developed by PTC Therapeutics, who acquired BioElectron in October 2019.

How does it work?

When the mitochondria don’t function properly, this results in the production of abnormally high amounts of reactive oxygen and nitrogen species, which causes a redox imbalance and glutathione deficiency.  It is thought to improve the redox imbalance by increasing levels of glutathione within cells.

What is it supposed to do?

Vatiquinone is supposed to increase the amount of energy the mitochondria can produce which could reduce some of the symptoms of mitochondrial disease.

Is it a cure?

Unfortunately not, Vatiquinone does not correct the inherited disorder of energy metabolism or mitochondrial disease and is not expected to be a cure.

What types of mitochondrial disease could it treat?

Vatiquinone has been investigated in a number of clinical trials and an emergency access program. The majority of studies have been exploratory, open label trials involving relatively few patients. Studies have been conducted in a number of mitochondrial diseases, including polymerase γ (POLG) deficiency, Leigh syndrome, Mitochondrial Encephalopathy (MELAS), mtDNA deletion syndrome, Leber’s hereditary optic neuropathy (LHON) and RARS2 deficiency.

Are there any current trials?

Vatiquinone is currently being tested in a phase 3 trial (MIT-E) to evaluate the safety and efficacy in patients with mitochondrial disease and refractory epilepsy. The study will be conducted in an estimated 60 patients up to 18 years of age with mitochondrial disease and drug resistant epilepsy, including Leigh syndrome, MELAS, Pontocerebellar Hypoplasia Type 6 (PCH6) and Alpers syndrome.

The study is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in stage to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase. After completion of the randomized, placebo-controlled phase, participants may enter a 48-week, long-term, extension phase during which they will receive open-label treatment with vatiquinone. Further details are available here.

Back to Mito Research Around the World