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Reata surprises with positive data in challenging rare disease

In a surprise to many on Wall Street, Reata Pharmaceuticals said Monday afternoon that one of its drugs appears to work in challenging rare disease — results which spurred a more than 60% spike in the company’s stock value by Tuesday morning. Reata now intends to file the drug, known as omaveloxolone or omav, for approval in the U.S. and elsewhere as a treatment for Friedreich’s Ataxia, a genetic, neurodegenerative movement disorder that affects roughly one in 40,000 people.

Approvals would hinge on the pivotal, two-part MOXIe study. The second part, which Reata announced Monday, found that, after 48 weeks, Friedreich’s Ataxia patients who received omav as opposed to placebo had significantly greater improvements on a neurological rating scale that measures their disease severity.

Neuroscience, and especially neurodegenerative disorders, are a notoriously difficult area of drug development. With multiple Wall Street banks expecting a negative readout on omav, Reata’s positive announcement was met with a nearly $60 increase in its stock value, from $100 per share Monday to more than $160 by Tuesday morning.

But now that the program has shown promise, investors will surely be keeping a more watchful eye on it as Reata prepares approval applications and more complete data presentations.

So far, Reata has disclosed how the different treatment groups in MOXIe scored on the modified Friedreich’s Ataxia Rating Scale, or mFARS. Scores fell — which means the patients improved — by 1.55 points in the primary analysis group of omav-treated patients and increased by 0.85 points in the placebo arm, resulting in a placebo-adjusted change of 2.40.

The mFARS evaluates four categories of patient activity: a bulbar section, which includes speaking and swallowing, an upper limb coordination section, a lower limb coordination section, and a standing and walking section. On a Tuesday morning investor call, company leadership declined to comment on how patients performed on individual categories, but noted that omav-treated patients reported improvements in speech, swallowing and walking, among other daily activities.

On safety, four patients given omav and two given placebo discontinued the study due to an adverse event. Three patients from each group experienced a serious adverse event during the study, while another two were seen after the study in omav-treated patients.

One omav-treated patient had multiple serious adverse events, including a viral upper respiratory tract infection, laryngitis and non-cardiac chest pain. Liver enzymes called ALT and AST were also higher in a greater portion of the omav group compared to placebo, results that Reata leadership proposes are tied to the improved mitochondrial function associated with omav treatment.

Liver enzymes a pharmacological effect of Reata’s drug
Adverse event Placebo (n=52) Omaveloxolone (n=51)
Contusion 19 (37%) 17 (33%)
Headache 13 (25%) 19 (37%)
Upper respiratory tract infection 15 (29%) 14 (28%)
Excoriation 12 (23%) 13 (26%)
Nausea 7 (14%) 17 (33%)
ALT increased 1 (2%) 19 (37%)
Fatigue 7 (14%) 11 (22%)
Abdominal pain 3 (6%) 11 (22%)
AST increased 1 (2%) 11 (22%)


For the second part of the MOXIe study, 103 patients were randomized to receive either omav or placebo. Eighty-two didn’t have pes cavus, a musculoskeletal foot deformity that Reata says could have interfered with their leg component score on the mFARS exam.

After factoring in the 21 patients who did have pes cavus, omav appears to have improved patients’ mFARS scores by 1.93 points compared to placebo, which is still statistically significant.

Reata executives said that, if approved, the label for omav would likely include pes cavus patients. They also noted on the Tuesday call that the drug may be applicable to other neurodegenerative diseases such as ALS, Huntington’s and Alzheimer’s.