Edison Pharmaceuticals and Bambino Gesu Children’s Hospital have initiated a Phase II trial of EPI-743 in patients with cobalamin C defect.

The orally bioavailable small molecule, EPI-743, is being developed to treat Friedreich’s ataxia and other inherited mitochondrial diseases.

Go deeper with GlobalData

Premium Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

The study is intended to identify if EPI-743, a member of the para-benzoquinone class of drugs, holds a therapeutic benefit for intermediary metabolism disorders that also result in redox disturbances.

"The study is intended to identify if EPI-743, a member of the para-benzoquinone class of drugs, holds a therapeutic benefit for intermediary metabolism disorders that also result in redox disturbances."

EPI-743, which serves as a cofactor for the novel drug target NADPH quinone oxidase 1, enhances endogenous glutathione biosynthesis through a redox-based mechanism, required for oxidative stress control.

Bambino Gesu Children’s Hospital, Divsion of Metabolism and trial principal investigator Professor Carlo Dionisi-Vici said Cobalamin C deficiency is an inherited disorder inducing perturbations in the metabolism of glutathione.

See Also:

"Given the central role of glutathione in cellular redox balance and antioxidant defense systems, we are eager to explore whether a therapeutic that increases glutathione such as EPI-743 will provide clinical benefit," Dionisi-Vici said.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

The trial is a 12-month placebo-controlled study, preceded by a six-month run-in phase for all patients to establish a defined clinical and metabolic baseline, according to the company.

Improvement in visual function is primary endpoint. Secondary outcome measurements will evaluate neurologic and neuromuscular function, glutathione biomarkers and quality of life, while establishing safety parametres.