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 contributing 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.
