Omaveloxolone for Freidreich’s Ataxia Provides Clinical Improvment in Phase 2 Trial 

Participants treated with omaveloxolone (Reata Pharmaceuticals, Irving, TX) daily had a statistically significant placebo-corrected 2.40-point improvement on the modified Friedrich’s ataxia (FA) rating scale (mFARS) after 48 weeks of treatment (P =.014).  Those treated experienced a mean improvement in mFARS of 1.55 points from baseline, whereas participants treated with placebo had a mean worsening of 0.85 points from baseline.    

The mFARS includes 4 sections that measure a person’s performance of activities such as speaking and swallowing, upper limb coordination, lower limb coordination, standing, and walking.  

 “Patients living with Friedreich’s ataxia experience a devastating and progressive loss of neurological function,” said Warren Huff, president and chief executive officer, “The MOXIe trial with omaveloxolone is the first study to demonstrate a significant improvement in neurological function in patients with FA. We believe that the MOXIe findings announced today bring us closer to our goal of providing an urgently needed therapy to patients with FA.” 

“The results of MOXIe represent a truly historic moment for the patients, families, and caregivers who comprise the Friedreich’s ataxia community,” said Ronald Bartek, president of the Friedreich’s Ataxia Research Alliance (FARA). “Based on the results reported today for omaveloxolone, we are hopeful that our community will finally have its first approved therapy that can slow this relentlessly progressive disease.” 
With these results, omaveloxolone met its primary endpoint of the MOXIe clinical trial (NCT02255435). The MOXIe trial is a global placebo-controlled study that enrolled 103 participants with FA. Participants were randomly assigned on a 1:1 basis to receive 150 mg omaveloxolone or placebo.  

Omaveloxolone is an experimental oral activator of Nrf2, a transcription factor that induces molecular pathways that promote restoration of mitochondrial function, reduction of oxidative stress, and inhibition of pro-inflammatory signaling.