The results from part 1 of a clinical trial investigating a potential therapy for Fredreich’s ataxia have been published. Omaveloxolone, a drug that we reported in July after it gained a new Orphan drug designation for Friedreich’s ataxia with Reata Pharmaceuticals (see here), has been in the process of being tested in 2-part international, multi-centre, randomized, double-blind, placebo-controlled phase 2 trial, referred to as MOXIe.

The results of part 1 of the study were published in the Annals of Clinical and Translational Neurology journal (see here). The study found Omaveloxolone was well tolerated, and adverse events were generally mild. Treatment of Friedreich ataxia patients with omaveloxolone at the optimal dose level of 160 mg/day appeared to improve neurological function.

The investigation is continuing in part two of the trial at various sites around the world including the London ataxia centre. Results from part two of the trial are expected in second half of 2019.

Posted on 23/11/2018