Reata Pharmaceuticals has announced positive results from part two of the registrational portion of its Phase II clinical trial, MOXIe, assessing omaveloxolone in patients with Friedreich’s ataxia (FA), an inherited, debilitating, and degenerative neuromuscular disorder.
Omaveloxolone is an experimental, oral, once-daily activator of Nrf2, a transcription factor that induces molecular pathways, promoting the restoration of mitochondrial function, reduction of oxidative stress, and inhibition of pro-inflammatory signalling.
Part II of MOXIe trial enrolled 103 patients with FA, who were randomised 1:1 to receive 150mg of omaveloxolone or placebo.
The international, multi-centre, double-blind, placebo-controlled, randomised registrational trial has been carried out at 11 study sites in the US, Europe, and Australia. It is claimed to be the largest global, interventional study undertaken in FA.
The trial met its primary endpoint of the change in the modified FA Rating Scale (mFARS) at week 48 versus placebo.
Reata said the observed placebo-corrected enhancements in mFARS were time-dependent, increasing over the course of treatment. The largest improvement was observed after 48 weeks of treatment.
Reata president and CEO Warren Huff said: “Patients living with Friedreich’s ataxia experience a devastating and progressive loss of neurological function. 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.”
Reata said it will present the full MOXIe study results at a future medical meeting.
Omaveloxolone has the potential to avoid long-term consequences and improve FA symptoms by addressing the underlying pathologic processes associated with inflammation, mitochondrial dysfunction, and oxidative stress.
Omaveloxolone has secured orphan drug designation from the US Food and Drug Administration and the European Commission to treat FA, which affects about 5,000 children and adults in the US and 22,000 globally.