Edison Pharmaceuticals begins Ataxia Clinical Trial

Edison Pharmaceuticals has started a phase 2B study, which will investigate new treatments for Friedreich's ataxia.

The trial is entitled Safety and Efficacy Study of EPI-743 on Visual Function in Patients with Friedreich's Ataxia. It is placebo-controlled and will last six months, before an extension phase begins where subjects will receive EPI-743.

Those contruibuting to the clinical laboratory work need to be between 18 and 45 years of age, possess confirmation of the disease and meet particular disease severity criteria.

Its primary endpoint is visual function, while secondary endpoints include neurological and neuromuscular function, alongside disease-relevant biomarkers.

The trial is being carried out with help from Friedreich's Ataxia Research Alliance (FARA) Clinical Research Network and FARA's Patient Registry.

FARA president Ronald Bartek said that the company is looking forward to the commencement of the research.

"FARA is excited about the commencement of the EPI-743 Friedreich's ataxia clinical trial, and is working closely with Edison Pharmaceuticals and Clinical Research Network investigators to expedite enrolment.

"This trial represents the importance of public-private partnership in drug development and the culmination of FARA-sponsored translational research, initiated with both FARA and National Institutes of Health (NIH) support."

FARA clinical research network investigator Theresa Zesiewicz, professor of Neurology, director of the University of South Florida Ataxia Research Centre and one of the trial's principal investigators, also commented on the work.

"Given the encouraging clinical findings reported to date for EPI-743 in mitochondrial disease, we are eager to determine whether these results are distensible to Friedreich's ataxia," Ms Zesiewicz explained.

News of Edison Pharmaceuticals' latest trial comes after it received EP1-743 orphan designation from European Medicines Agency (EMA) to help treat Leigh Syndrome.

Edison has studied EPI-743 in 80 subjects with mitochondrial disease, with this data being used along with a phase 2A study to form the basis of the application.

The company will also soon release the results of its phase 2A study entitled Prospective Open Label Study of EPI-743 in Children with Leigh Syndrome (Subacute Necrotizing Encephalomyelopathy).


I'm always encouraged when I see clinical trials or studies for ataxia! Fantastic! Thanks for the info Alan! ;o)