IRVING, Texas, May 30, 2019 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, Inc. (RETA), a clinical-stage biopharmaceutical company, today announced the enrollment of the first patient in the Phase 3 FALCON trial of bardoxolone methyl (bardoxolone) in patients with autosomal dominant polycystic kidney disease (ADPKD).
FALCON is an international, multi-center, randomized, double-blind, placebo-controlled trial studying the safety and efficacy of bardoxolone in approximately 300 patients with ADPKD randomized evenly to active drug or placebo. The trial will enroll a broad range of patients from 18 to 70 years old with an estimated glomerular filtration rate (eGFR) between 30 to 90 mL/min/1.73 m². The primary efficacy endpoint for FALCON is the change from baseline in eGFR compared to placebo after 48 weeks of treatment followed by a 4-week drug withdrawal period, which is also known as the retained eGFR benefit. Based upon guidance from the United States Food and Drug Administration (FDA), an analysis of retained eGFR demonstrating an improvement versus placebo after one year of bardoxolone treatment may support accelerated approval, and an improvement versus placebo after two years of treatment may support full approval.
“ADPKD is the most common inherited form of chronic kidney disease, and despite standard of care treatment, approximately 50% of these patients will progress to end-stage kidney disease by 60 years of age,” said Warren Huff, Reata’s Chief Executive Officer and President. “We observed significant improvements in kidney function in the ADPKD cohort of the Phase 2 PHOENIX study, and historically we have observed strong correlations between changes in eGFR after 12 weeks of bardoxolone treatment and one-year retained eGFR benefit in other forms of chronic kidney disease. We are hopeful that bardoxolone may serve as a meaningful new treatment option for patients with ADPKD.”
About Autosomal Dominant Polycystic Kidney Disease
ADPKD is a genetic form of chronic kidney disease (CKD) caused by mutations in PKD1 and PKD2 genes leading to the formation of fluid-filled cysts in the kidneys and other organs. The cysts continue to grow and can cause the kidneys to expand up to five to seven times their normal volume leading to pain and progressive loss of kidney function. As in other forms of CKD, decreased mitochondrial function and chronic inflammation are key drivers of disease progression.
ADPKD affects both men and women of all racial and ethnic groups and is the leading inheritable cause of kidney failure with an estimated diagnosed population of 140,000 patients in the United States. As an autosomal dominant disease, an affected parent has a 50% chance of passing ADPKD on to their children. Despite current standard of care treatment, an estimated 50% of ADPKD patients progress to end-stage kidney disease and require dialysis or a kidney transplant by 60 years of age.
Bardoxolone is an experimental, oral, once-daily activator of Nrf2, a transcription factor that induces molecular pathways that restore mitochondrial function, reduce oxidative stress, and inhibit pro-inflammatory signaling. The FDA has granted orphan drug designation to bardoxolone for the treatment of Alport syndrome 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 restores mitochondrial function, reduces oxidative stress, and inhibits 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 United States 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