Shares of a small biotech company skyrocketed Tuesday following the release of positive data from its registration-directed Phase II study of a fatal genetic disorder that causes muscle weakness.

Irving, Texas-based Reata Pharmaceuticals rose 60 percent on the Nasdaq Tuesday morning and remained up 57.5 percent through the afternoon after the company announced positive results from the registrational second part of its Phase II MOXIe trial of the drug omaveloxolone in patients with Friedreich’s ataxia.

The company said that patients treated with the drug at 150mg per day showed a statistically significant, placebo-corrected 2.40-point improvement on the modified Friedreich’s Ataxia Rating Scale, or mFARS after 48 weeks of treatment. The company plans to submit regulatory filings to seek approval in the U.S. and in other countries.

FA, also abbreviated FRDA, affects about 1-in-40,000 people and is the most common inherited ataxia – or loss of control of the limbs – in Europe, the Middle East, South Asia and North Africa, according to the National Organization for Rare Disorders. Ataxia results in an unsteady gait and poor control of fine movements of the limbs, along with slurred speech and difficulty swallowing. Patients with FA ultimately progress from needing walking aids to requiring a wheelchair. Most patients are diagnosed before the age of 25, though late-onset FA can affect people between the ages of 26 and 39, and very late-onset FA can appear after age 40.

“Based on the results reported today for omaveloxolone, we are hopeful that our community will finally have its first approved therapy that can slow this relentlessly progressive disease,” Friedreich’s Ataxia Research Alliance President Ronald Bartek said in a statement. “We are extremely proud of, and grateful for, the FA community, including all those who have participated in this clinical trial and in the natural history study important in designing the trial.”

The news comes five days after Reata said it had reacquired from Chicago-based drugmaker AbbVie worldwide rights to omaveloxolone and other drugs belonging to its class, known as Nrf2 activators, along with ex-U.S. rights to the drug bardoxolone. Reata is paying AbbVie $330 million to the rights for bardoxolone, which includes a $75 million upfront payment this year and installments paid next year and in 2021, plus low single-digit tiered royalties from worldwide sales of omaveloxolone and other Nrf2 activators. The ClinicalTrials.gov page for MOXIe lists AbbVie and the Friedreich’s Ataxia Research Alliance as collaborators.

Another company, Barcelona, Spain-based Minoryx Therapeutics, is running a 36-patient, placebo-controlled Phase II study of its own drug for FA, MIN-102, which it launched in March of this year and which is scheduled to reach initial completion next March.

Photo: FotografiaBasica, Getty Images



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