IRVING, Texas, June 05, 2019 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, Inc. (RETA), a clinical-stage biopharmaceutical company, today announced the United States Food and Drug Administration (FDA) has granted orphan drug designation to bardoxolone methyl (bardoxolone) for the treatment of autosomal dominant polycystic kidney disease (ADPKD).
ADPKD is the most common inherited form of kidney disease affecting approximately 140,000 patients in the United States. It is characterized by the development of pathologic fluid-filled cysts throughout the kidneys, which leads to organ enlargement and chronic kidney disease (CKD). Despite standard of care treatment, approximately 50% of these patients will progress to end-stage kidney disease and require dialysis or a kidney transplant by 60 years of age.
“Obtaining orphan drug designation for bardoxolone for the treatment of ADPKD is an important milestone for Reata. This is the third orphan drug designation obtained for bardoxolone in the United States for the treatment of diseases characterized by mitochondrial dysfunction and inflammation, and it is the second designation for the treatment of patients with rare forms of CKD,” said Warren Huff, Reata’s Chief Executive Officer and President. “We believe that, if approved, bardoxolone may prove to be a meaningful new treatment option for patients with ADPKD.”
Last year, Reata released positive data from the ADPKD cohort of the PHOENIX Phase 2 study of bardoxolone in four rare forms of CKD. After 12 weeks of bardoxolone treatment, we observed a mean increase from baseline in estimated glomerular filtration rate (eGFR) of 9.3 mL/min/1.73 m2 (n=31; p<0.0001). Notably, 96% of patients who reached Week 12 demonstrated an improvement in eGFR. The observed improvement represents a recovery of approximately two years of average eGFR loss. Based on these encouraging results, Reata launched FALCON, an international, double-blind, placebo-controlled, parallel group, Phase 3 study of bardoxolone in approximately 300 patients with ADPKD. The primary efficacy endpoint, which may support accelerated approval under Subpart H, is the change from baseline in eGFR compared to placebo after 48 weeks of treatment followed by a 4-week drug withdrawal period.
Orphan drug status is granted to treatments for diseases that affect fewer than 200,000 people in the United States and provides specific incentives for the development of therapies intended for the treatment, diagnosis, or prevention of rare diseases. Such designation will provide Reata with certain development incentives, including tax credits for clinical testing, exemption from a prescription drug user fee, and seven years of market exclusivity.
Bardoxolone is an experimental, oral, 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. The FDA has granted orphan drug designation to bardoxolone for the treatment of Alport syndrome, ADPKD, and pulmonary arterial hypertension. The European Commission has granted orphan drug designation to bardoxolone for the treatment of Alport syndrome. In addition to FALCON, bardoxolone is currently being studied in CARDINAL, a Phase 3 study for the treatment of Alport syndrome, CATALYST, a Phase 3 study for the treatment of connective tissue disease-associated pulmonary arterial hypertension, and AYAME, a Phase 3 study for the treatment of diabetic kidney disease in Japan. AYAME is being conducted by our licensee Kyowa Hakko Kirin Co., Ltd.
About Reata Pharmaceuticals, Inc.
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, bardoxolone and omaveloxolone, target the important transcription factor Nrf2 that promotes restoration of mitochondrial function, reduction of oxidative stress, and inhibition of pro-inflammatory signaling. Bardoxolone and omaveloxolone are investigational drugs, and their safety and efficacy have not been established by any agency.
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, 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,” 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) 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 (iv) other factors set forth in Reata’s filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K, 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.
Reata Pharmaceuticals, Inc.
Vice President, Strategy
Matt Middleman, M.D.
LifeSci Public Relations