Reata Pharmaceuticals Completes Rolling Submission of New Drug Application for Omaveloxolone for the Treatment of Patients with Friedreich’s Ataxia

Rhea-AI Summary

Reata Pharmaceuticals has completed a rolling submission of a New Drug Application (NDA) for omaveloxolone, aimed at treating Friedreich’s ataxia, affecting approximately 5,000 patients in the U.S. Currently, there are no approved therapies for this degenerative disorder. The FDA has granted Fast Track and Orphan Drug Designations for omaveloxolone. This submission is based on data from the MOXIe studies, signaling a potential breakthrough for patients with this condition, which typically leads to severe motor incapacitation and reduced life expectancy.

Positive

• Completion of NDA submission for omaveloxolone for Friedreich’s ataxia.
• FDA granted Fast Track and Orphan Drug Designations for omaveloxolone.
• Potential to be the first approved therapy for Friedreich’s ataxia.

Negative

• None.

If Approved, Omaveloxolone Would Become the First Therapy Indicated for the Treatment of Patients with Friedreich’s Ataxia

Friedreich’s Ataxia Affects Approximately 5,000 Patients in the United States with an Estimated 4,000 Diagnosed Patients

PLANO, Texas--(BUSINESS WIRE)-- Reata Pharmaceuticals, Inc. (Nasdaq: RETA), (“Reata,” the “Company,” “our,” “us,” or “we”), a clinical-stage biopharmaceutical company, today announced the completion of the rolling submission of a New Drug Application (“NDA”) to the U.S. Food and Drug Administration (“FDA”) for omaveloxolone for the treatment of patients with Friedreich’s ataxia. This NDA is supported by the efficacy and safety data from the MOXIe Part 1, Part 2, and MOXIe Extension studies. The FDA has granted Fast Track Designation and Orphan Drug Designation to omaveloxolone for the treatment of Friedreich’s ataxia.

“This NDA submission marks an important step toward making a treatment available for patients with Friedreich’s ataxia, a serious degenerative neuromuscular disorder with no approved therapies,” said Warren Huff, Reata’s Chief Executive Officer. “I want to thank all those who made this possible, especially our patients with Friedreich’s ataxia, their families, and investigators, as well as our employees, for their tenacity and unwavering belief in the omaveloxolone clinical program. We look forward to the next steps on the path to making omaveloxolone available as a first-in-class therapy for Friedreich’s ataxia, pending approval.”

About Friedreich's Ataxia

Friedreich’s ataxia is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder typically caused by a trinucleotide repeat expansion in the first intron of the frataxin gene, which encodes the mitochondrial protein frataxin. Pathogenic repeat expansions can lead to impaired transcription and reduced frataxin expression, which can result in mitochondrial iron overload and poor cellular iron regulation, increased sensitivity to oxidative stress, and impaired mitochondrial ATP production. Patients with Friedreich’s ataxia typically experience symptoms in childhood, including progressive loss of coordination, muscle weakness, and fatigue that commonly results in motor incapacitation with patients requiring a wheelchair in their teens or early 20s. Patients with Friedreich’s ataxia may also experience visual impairment, hearing loss, diabetes, and cardiomyopathy. On average, patients with Friedreich’s ataxia die in the mid-thirties. Based on literature and proprietary research, we believe Friedreich’s ataxia affects approximately 5,000 children and adults in the United States and 22,000 individuals globally. There are an estimated 4,000 patients diagnosed with FA in the United States. Currently, there are no approved therapies for the treatment of Friedreich’s ataxia.

About Omaveloxolone

Omaveloxolone is an investigational, oral, once-daily, activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. The FDA has granted Orphan Drug and Fast Track Designations to omaveloxolone for the treatment of Friedreich’s ataxia. The European Commission has granted Orphan Drug Designation in Europe to omaveloxolone for the treatment of Friedreich’s ataxia.

About Reata

Reata is a clinical-stage biopharmaceutical company that develops novel therapeutics for patients with serious or life-threatening diseases by targeting molecular pathways involved in the regulation of cellular metabolism and inflammation. Reata’s two most advanced clinical candidates, omaveloxolone and bardoxolone methyl (“bardoxolone”), target the important transcription factor Nrf2 that promotes the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. Omaveloxolone and bardoxolone are investigational drugs, and their safety and efficacy have not been established by any agency.

Forward-Looking Statements

This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding the success, cost and timing of our product development activities and clinical trials, our plans to research, develop, and commercialize our product candidates, our plans to submit regulatory filings, and our ability to obtain and retain regulatory approval of our product candidates. You can identify forward-looking statements because they contain words such as “believes,” “will,” “may,” “aims,” “plans,” “model,” and “expects.” Forward-looking statements are based on Reata’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks, and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include, but are not limited to, (i) the timing, costs, conduct, and outcome of our clinical trials and future preclinical studies and clinical trials, including the timing of the initiation and availability of data from such trials; (ii) the timing and likelihood of regulatory filings and approvals for our product candidates; (iii) whether regulatory authorities determine that additional trials or data are necessary in order to obtain approval; (iv) the potential market size and the size of the patient populations for our product candidates, if approved for commercial use, and the market opportunities for our product candidates; and (v) other factors set forth in Reata’s filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K for the fiscal year ended December 31, 2021, under the caption “Risk Factors.” The forward-looking statements speak only as of the date made and, other than as required by law, we undertake no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.

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Reata Pharmaceuticals, Inc.

(972) 865-2219

https://www.reatapharma.com/

Investor Relations & Media Relations:

Andres Lorente ir@reatapharma.com

Wendy Segal media@reatapharma.com

https://www.reatapharma.com/contact-us/

Source: Reata Pharmaceuticals, Inc.

FAQ

What is the significance of Reata Pharmaceuticals’ NDA submission for omaveloxolone?

The NDA submission is crucial as it could make omaveloxolone the first therapy approved for Friedreich’s ataxia, addressing a significant unmet medical need.

How many patients are affected by Friedreich’s ataxia in the United States?

Friedreich’s ataxia affects approximately 5,000 patients in the United States, with an estimated 4,000 diagnosed.

What designations has the FDA granted for omaveloxolone?

The FDA has granted Fast Track and Orphan Drug Designations for omaveloxolone.

What studies support Reata Pharmaceuticals’ NDA for omaveloxolone?

The NDA is supported by the efficacy and safety data from the MOXIe Part 1, Part 2, and MOXIe Extension studies.

What condition does omaveloxolone aim to treat?

Omaveloxolone aims to treat Friedreich’s ataxia, a rare and debilitating neuromuscular disorder.