Omavelexolone’s Potential in Friedreich Ataxia: David R. Lynch, MD, PhD


The professor of neurology at the University of Pennsylvania Perelman School of Medicine discussed his presentation at MDS 2021, which highlighted the therapeutic benefit of omaveloxolone in Friedreich ataxia. [WATCH TIME: 2 minutes]

WATCH TIME: 2 minutes

"If you look at the slopes as they changed in the extension period, you find that they were equivalent and were roughly about one-fourth as fast as natural history data. In addition, responder analysis showed that people responded during this period like natural history data. This again shows that omav [omaveloxolone] is beneficial to people with Friedrich Ataxia in this delayed-start approach.”

The MOXIe trial (NCT02255435), featuring a 2-part study design, is the largest global interventional trial ever conducted in Friedreich Ataxia. At the International Parkinson and Movement Disorders Society (MDS) Virtual Congress 2021, September 17-22, data from the open-label, delayed-start period of the MOXIe trial was presented. The study evaluated the efficacy and safety of omaveloxolone (Reata Pharmaceuticals) in a cohort of 73 patients with Friedrich Ataxia (FA), using differences in modified Friedrich’s Ataxia Rating Scale (mFARS) scores as the primary end point.

Previously reported results showed a statistically significant 2.40-point improvement in mFARS score for those treated with the investigational agent compared with placebo (P = .014), otherwise a mean improvement of 1.55 points from baseline.1 Results reported at MDS 2021 by David R. Lynch, MD, PhD, continued to show similar therapeutic benefit, with a difference in mFARS of –2.18 points (±0.96) between the omaveloxolone and placebo groups at the end of the placebo-controlled MOXIe Part 2.2

Lynch, professor of neurology, University of Pennsylvania Perelman School of Medicine, sat down with NeurologyLive to provide background on the results, including the notable topline findings and the substantial improvement in those who previously switched from placebo.

For more coverage of MDS 2021, click here.

1. Raeta Announces Positive Topline Results from the MOXIe Registrational Trial of Omaveloxolone ir Patients with Friedreich’s Ataxia. news release. Raeta Pharmaceuticals. October 14, 2019. Accessed September 22, 2021.
2. Lynch DR, Chin MP, Boesch S, et al. Efficacy of omaveloxolone in patients with Friedrich’s ataxia: delayed-start study. Presented at MDS Virtual Congress; September 17-22, 2021. Poster LBA 2.
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