MOXIe Part 2 (NCT02255435) was a multicenter, double-blind, placebo-controlled trial to evaluate the efficacy and safety of omaveloxolone 150 mg once daily for 48 weeks in patients with Friedreich ataxia aged 16 to 40. A total of 51 patients were exposed to omaveloxolone and 52 patients to placebo. Omaveloxolone improved neurological function in a statistically significant manner, as assessed by change from baseline in mFARS score, compared to placebo and was considered generally safe and well tolerated.

Adverse events were experienced by all patients both in omaveloxolone and placebo treated groups. The majority of these events were mild or moderate in severity. TEAEs that occurred in ≥10% of patients receiving omaveloxolone with ≥5% increase in incidence when compared to those receiving placebo included alanine aminotransferase increase, headache, nausea, aspartate aminotransferase increase, fatigue, diarrhea, oropharyngeal pain, muscle spasm, back pain, influenza, and decreased appetite. Generally, these events were less frequently reported after week 12 of treatment. Most of these TEAEs had a total median duration of approximately one month or less.

TEAEs experienced by patients who received omaveloxolone were generally limited to the first 12 weeks of treatment, which is suggestive of an improvement in omaveloxolone tolerability over time.