Healthcare Business

Why This Neuromuscular Disorder Study Could Be the Next Big Thing

Reata Pharmaceuticals Inc. (NASDAQ: RETA) shares jumped on Tuesday after the firm announced positive results from its midstage neuromuscular disorder drug trial.

Specifically, the results are from Reata’s MOXIe Phase 2 trial of omaveloxolone in patients with Friedreich’s ataxia (FA). The study met its primary endpoint of change in the modified Friedreich’s Ataxia Rating Scale (mFARS) relative to placebo after 48 weeks of treatment. Patients treated with omaveloxolone demonstrated a statistically significant improvement in mFARS after 48 weeks of treatment.

Based on these positive results, and subject to discussions with regulatory authorities, the company plans to proceed with the submission of regulatory filings for marketing approval in the United States and internationally.

Ronald Bartek, president of the Friedreich’s Ataxia Research Alliance (FARA), commented:

The results of MOXIe represent a truly historic moment for the patients, families, and caregivers that comprise the Friedreich’s ataxia community. 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.  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.  We are also grateful to the clinical teams who conducted the trial and to our Reata colleagues.  We look forward to continuing the Reata-FARA partnership as we work in pursuit of approval of the first FA therapy.

Shares of Reata traded up about 57% at $157.57 on Tuesday, in a 52-week range of $47.50 to $162.50. The consensus price target is $141.25.