Treatments in Phase 3 Trials for FA
Omaveloxolone, or RT 408, is a small molecule Nrf2 activator that could potentially be beneficial for patients with Friedreich’s ataxia. It is being evaluated in a Phase 2/3 trial called MOXIe, for which positive top-line results of the Phase 2 portion were published in 2019.
RT001 is an experimental fatty acid aimed at stabilizing the mitochondrial and cellular membranes against attacks and restores cellular health. It is being tested in Friedreich’s ataxia patients in a Phase 3 trial, which began in October 2019.