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Is Reata’s Friedreich’s Ataxia Study a Failure? – Rare Disease Report

Data from a Phase 2 Friedreich’s ataxia (FA) study by Reata Pharmaceuticals is making its rounds on social media today. The data, according to Reata, is exceptional. However, according to many on Twitter, the trial is a failure.

At the core of the dispute between the company and its critics is that Reata reported statistically significant improvements from baseline in FA patients receiving the drug for 12 weeks in the phase 2 clinical trial testing omaveloxolone. Unfortunately, the study is a placebo-controlled trial and when you compare the outcome measure [modified Friedreich’s Ataxia Rating Scale (mFARS)] between the treatment group and the placebo group, the differences are not statistically different.

Friedreich’s ataxia, an inherited rare disease, causes damage to the nervous system and issues with bodily movement and functions. It typically begins in childhood and leads to impaired muscle coordination that worsens over time.

In a news release, the company stated that across all doses, ‘omaveloxolone significantly reduced mFARS by 2.5 points from baseline (P < .001) and by 1.1 points versus placebo (non-significant). At the higher 160 mg dose, omaveloxolone improved mFARS by 3.8 points versus baseline (P = .0001) and by 2.3 points versus placebo (P = .06)."

Reata further noted that “omaveloxolone produced greater improvements in mFARS in patients that did not have a preexisting musculoskeletal foot deformity that causes high arched feet, called pes cavus.”

There was also plenty abuzz on Twitter as a reaction to that second analysis:

In spite of what the critics think, the company and the advocacy group Friedreich’s Ataxia Research Alliance (FARA) are cautiously optimistic.

“We are greatly appreciative of Reata, the clinical investigators, and the study volunteers for conducting and participating in a well-designed and robust dose-escalation study. We find these results to be very exciting, and they are the ideal outcome for an early Phase 2 study,” said Jennifer Farmer, executive director of FARA. “They exceed expectations in terms of safety and by demonstrating dose-dependent and clinically meaningful activity that correlated with biological activity.”


Source: Is Reata’s Friedreich’s Ataxia Study a Failure? – Rare Disease Report

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