With Positive Phase II Data, Reata Eyes Potential NDA for Friedreich’s Ataxia Treatment

Clinical Trial Technology

Shares of Texas-based Reata Pharmaceuticals are up nearly 40% in premarket trading after the company announced its mid-stage trial of omaveloxolone in patients with Friedreich’s ataxia (FA) met its primary endpoint compared to placebo and sets up a potential regulatory filing.

Reata announced late Monday that the second part of its Phase II MOXIe trial met the goal of modified Friedreich’s Ataxia Rating Scale (mFARS) after 48 weeks of treatment. Patients treated with a 150 mg daily dose of omaveloxolone saw a “statistically significant, placebo-corrected 2.40 point improvement, a decrease, in mFARS at the 48-week mark, Reata said. Results from the Phase II trial show that patients treated with omaveloxolone experienced a mean improvement in mFARS of -1.55 points from baseline, while patients treated with placebo experienced a mean worsening in mFARS of +0.85 points from baseline. 

The mFARS is a physician-assessed neurological rating scale used to measure FA disease progression. The test includes four sections that measure the patient’s performance of activities, including speaking and swallowing, upper limb coordination, lower limb coordination, and standing and walking.  The United States Food and Drug Administration indicated that mFARS is an acceptable primary endpoint to evaluate the effect of omaveloxolone for the treatment of patients with FA, the company said.

The announcement of the Phase II data came less than a week after Reata reacquired all rights to omaveloxolone and other drugs in its platform from AbbVie. As part of the deal to reacquire the rights, Reata will pay AbbVie $330 million. The company will pay $75 million by the end of this year and the remainder in installments throughout 2020 and 2021.

Based on the results of the Phase II trial, Reata plans to begin talks FDA and other regulatory bodies regarding potential filing of New Drug Applications. Full results of the Phase II trial will be presented at a future medical conference, the company noted.

Reata’s omaveloxolone is a once-daily activator of Nrf2, a transcription factor that induces molecular pathways that promote restoration of mitochondrial function, reduction of oxidative stress, and inhibition of pro-inflammatory signaling, according to company data. Friedreich’s ataxia is a rare genetic neuromuscular disorder that causes progressive loss of coordination, muscle weakness, and fatigue. It can lead to reliance on a wheelchair for mobility and potential premature death. FA affects approximately 5,000 children and adults in the United States and 22,000 globally. There are no approved treatments for the disorder.

“Patients living with Friedreich’s ataxia experience a devastating and progressive loss of neurological function. The MOXIe trial with omaveloxolone is the first study to demonstrate a significant improvement in neurological function in patients with FA. We believe that the MOXIe findings announced today bring us closer to our goal of providing an urgently needed therapy to patients with FA,” Warren Huff, president and chief executive officer of Reata said in a statement.

Omaveloxolone was generally reported to be well tolerated in this study although four omaveloxolone patients and two placebo patients discontinued study drug due to an adverse event. The reactions were mild to moderate in intensity, Reata said.

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