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Webinar: FA Community Call to Action
on January 5, 2021
Have Your Voices Heard: Be a Part of the FA Community's Request for Access to Reata and FDA
Tune in on January 5, 2021 at 7pm (EST) to a webinar outlining FARA’s proposed response from the FA community to the positive results of Reata Pharmaceuticals’ MOXIe Part 2 clinical study. (See below for more detailed information about MOXIe.)

After consultation with regulatory advisors, FARA plans to submit a letter to both Reata and the Food and Drug Administration (FDA) that requests they work together to provide access to omaveloxolone for individuals with Friedreich's Ataxia as soon as possible. The letter will highlight the voice of the patient with a request for Reata to submit a marketing application to the FDA and for the FDA to review and consider approval of this application. The strength of this letter will depend in large part on input and testimony from the FA community. The voices and lived experiences of this community matter and will help add critical perspective and context to the MOXIe trial data points that are being discussed. Even if you did not participate in this clinical trial, it is important to hear your voices too. It is crucial that our response is respectful, informative, and representative of the FA patient experience.

We are at an important crossroads in Reata's development of omaveloxolone as a potential treatment for FA. Our clinical investigators and FARA have confidence in the data from the completed studies and would like FDA to use tools given to it under the law to exercise regulatory flexibility to avoid a delay of at least several years for access which would occur if Reata conducts another clinical trial prior to the drug being approved for use by FA patients in the U.S. Please register for this important session and learn how you can participate. Please also share this message within the FA community. The greater our unity, the stronger our message.
Background Information:
Reata Pharmaceuticals, Inc. is developing omaveloxolone which has the potential to prevent long-term consequences and improve FA symptoms by addressing the underlying pathologic processes associated with inflammation, mitochondrial dysfunction, and oxidative stress. Omaveloxolone is a Nrf2 activator that targets the mitochondrial dysfunction associated with FA.

Reata has shared the results of the MOXIe trials with the FDA and sought their feedback, specifically, to understand if these results could support FDA approval of omaveloxolone in FA. While FDA views the MOXIe study results as positive, FDA's advice and feedback has been that they don't see these results as sufficiently persuasive, and as a result recommends that Reata conduct another clinical trial. It is important to note that Reata has not submitted a marketing application for omaveloxolone and, therefore, FDA has not declined approval of the drug; FDA has only provided advice.

Here is a timeline of key events that have led up to this point:

2015
Reata enrolled the first patient in Part 1 of the MOXIe study. This was a Phase 2 dose-ranging study examining the safety, tolerability, and efficacy of omaveloxolone versus placebo for the treatment of patients with Friedreich’s Ataxia.

2017
Reata Pharmaceuticals, Inc. announced positive data from Part 1 of the Company’s Phase 2 trial (MOXIe) of omaveloxolone for the treatment of Friedreich’s Ataxia, and they also announced plans to initiate Part 2 of MOXIe.

2018
Part 2 of MOXIe is a randomized, placebo-controlled, double-blind study to evaluate the safety and efficacy of 150mg omaveloxolone in individuals with FA over 48 weeks. This is the part of the trial that was designed to establish effectiveness of the investigational drug. The trial completed enrollment of >100 individuals with FA ages 16-40 years who are able to perform 10-15 minutes of exercise on a recumbent exercise bike during 2018 at the following sites: Children's Hospital of Philadelphia, University of Florida, Ohio State University, University of Iowa, University of South Florida, Emory University, University of California Los Angeles, Murdoch Children's Research Institute (Melbourne, Australia), and Medical University Innsbruck (Innsbruck, Austria), University College London (United Kingdom) and Neurological Institute Carlo Besta (Milan, Italy).

2019
Topline results of this trial were reported by Reata in a press release, October 2019. Part 2 of the MOXIe Phase 2 met its primary endpoint of change in the modified Friedreich’s Ataxia Rating Scale (mFARS) relative to placebo after 48 weeks of treatment. Individuals with FA treated with omaveloxolone (150 mg/day) demonstrated a statistically significant, placebo-corrected 2.40 point improvement in mFARS after 48 weeks of treatment (p=0.014). The mFARS is a physician-assessed neurological rating scale used to measure FA disease progression.

2020
The FDA requested additional evidence to support the results of Part 2 of the MOXIe Phase 2 trial. In response to the FDA's request, Reata proposed a "baseline-controlled analysis" to measure the effect of omaveloxolone on mFARS in patients who were previously randomized to placebo in the MOXIe Part 2 study or were enrolled in MOXIe Part 1 and are being treated with omaveloxolone in the MOXIe open-label extension study. In early November 2020, Reata shared positive results of this analysis, these results further supported the positive mFARS results observed in the Part 2 study and provide additional evidence of the effectiveness of omaveloxolone in FA.

In November 2020, Reata announced that the FDA completed its internal review of the Baseline-Controlled analysis results of omaveloxolone for the treatment of patients with Friedreich’s ataxia (FA) and concluded that the results do not strengthen the results of Part 2 of the MOXIe study. The FDA stated that they remain interested in reviewing the results of additional exploratory analyses as those may inform the future development program.