In October 2019, Reata Pharmaceuticals announced positive top-line results from their MOXIe trial testing Omaveloxolone (or Omav) in Friedreich’s ataxia (FA).
In the MOXIe trial, Reata used the modified Friedreich’s Ataxia Rating Scale (mFARS) to measure the symptoms of those taking Omav vs those taking placebo (a placebo is a substance that has no therapeutic effect, used as a control in testing new drugs). The mFARS results showed that patients taking Omav for 48 weeks had a statistically significant improvement in their symptoms associated with FA than those on the placebo trial.
Back in August the Food and Drug Administration (FDA), which is the drug regulatory body in the USA, suggested that while it does not have concerns that the mFARS data is reliable, it was not convinced that the data will be enough to support approval of Omav based on this trial alone.
On the 24th of November 2020 Reata Pharmaceuticals announced that the FDA completed its internal review of the Omav study and concluded that the results do not strengthen the results of Part 2 of the MOXIe study. Here’s the outcome:
- The FDA proposed additional exploratory analyses using patients randomised to placebo during the MOXIe Part 2 study, but stated that the potential for these analyses to strengthen the study results was questionable due to the small number of patients available for analysis.
- The FDA remain interested in reviewing the results of the additional exploratory analyses as those may inform the future development program.
Reata plans to submit to the FDA the analyses that they proposed and to request a meeting with the FDA to discuss the development program. In addition, based on the FDA’s conclusion, Reata is considering the next steps for the development programme, including whether to conduct a second pivotal study in patients with FA.
You can read the press release here: