Reata gets another shot in Friedreich’s ataxia

EvaluatePharma. August 06, 2019.

Pharmaceuticals will soon find out if its decision to persevere with omaveloxolone despite an earlier failure has paid off.

The signs are not auspicious: the first part of the same study did not show a benefit with omaveloxolone on the primary endpoint, peak work during maximal exercise testing, or its secondary endpoint, change versus placebo on the modified Friedreich’s ataxia rating scale (mFARS), when it reported two years ago (Friedreich's failure highlights thinning pipeline, 2 June 2017).

Despite this, Reata claimed that the data were positive, and Vinny Jindal, the group’s vice-president of strategy, told Vantage: “I wouldn’t classify part one of Moxie as a failure.” He added that the study had found a “meaningful” effect on patients, as measured by mFARS, a 100-point scale on which a higher score represents more severe disease.

Mr Jindal pointed to a 2.3-point placebo-corrected reduction on mFARS after 12 weeks' treatment with omaveloxolone 160mg, the best-performing dose in part one. This missed statistical significance with a p value of 0.06.

Nevertheless, Mr Jindal maintained: “Patients typically progress 1-2 points per year, so that improvement [could save patients] more than a year of progression.”