Reata Pharmaceuticals Inc. said its experimental drug omaveloxolone significantly improved certain symptoms of an inherited neuromuscular disorder compared to placebo in a mid-stage study.
Reata's stock price was up 37.4% to $138.25 in after-hours trading on Oct. 14.
The Irving, Texas-based company was studying the drug to treat Friedreich's ataxia in part 2 of the phase 2 trial, named MOXIe. The disorder is characterized by progressive loss of coordination, muscle weakness and fatigue that commonly progresses to motor incapacitation and wheelchair reliance.
Friedreich's ataxia affects about 5,000 children and adults in the U.S. and 22,000 globally, according to Reata.
Reata enrolled 103 patients with Friedreich's ataxia in the trial and randomly assigned them to receive either a 150-milligram dose of omaveloxolone or a placebo.
Omaveloxolone met the primary goal of the study after 48 weeks by showing significant improvement versus placebo in patients' ability to perform activities such as speaking, swallowing, upper limb coordination, lower limb coordination and standing as well as walking.
Reata said that 8% of omaveloxolone patients and 4% of placebo patients stopped treatment due to an adverse event. The overall rate of serious adverse events was low, with three patients in each group reporting such events during treatment.
The pharmaceutical company intends to submit applications for marketing approval in the U.S. and internationally based on the results of the study.
Reata has received orphan drug designation from the U.S. Food and Drug Administration and the European Commission for omaveloxolone to treat Friedreich's ataxia. The FDA has not approved any treatment for this disorder.