Reata Announces Initiation of Phase 2/3 Study of Bardoxolone Methyl in the Treatment of Chronic Kidney Disease Due to Alport Syndrome
IRVING, Texas, Feb. 23, 2017 (GLOBE NEWSWIRE) — Reata Pharmaceuticals, Inc. (NASDAQ:RETA) (“Reata” or the “Company”) today announced the initiation of patient screening in a Phase 2/3 trial to evaluate bardoxolone methyl (“bard”) in patients with chronic kidney disease (“CKD”) caused by Alport syndrome. The purpose of this study is to determine the safety and efficacy of bard in Alport syndrome patients, and to determine if Alport syndrome patients experience improvements in kidney function similar to those observed in multiple, previous trials of bard in patients with other forms of CKD. Reata expects data from the Phase 2 portion of the trial to be available, and to decide on entering the Phase 3 portion, by year-end 2017.
The Alport Syndrome Foundation’s Executive Director Gina Parziale said, “As there are currently no FDA approved treatments for those with Alport Syndrome, the Alport Syndrome Foundation encourages the development of therapies that will delay or prevent the need for dialysis and transplantation. We are grateful to Reata for engaging us in this process and for recognizing the crucial role of the patient perspective.”
“Based on our extensive clinical experience with bardoxolone methyl in patients with diabetic CKD, as well as in our ongoing Phase 2 and Phase 3 trials for bardoxolone in other orphan diseases, we hope to demonstrate that bardoxolone methyl can serve as a meaningful new treatment option for patients with Alport syndrome,” said Warren Huff, Reata’s Chief Executive Officer and President.
Overview of Clinical Trial Program in Alport Syndrome
The Phase 2 portion of the study is open-label and will enroll up to 30 patients from 12 to 60 years old with estimated glomerular filtration rates (eGFR) between 30 to 90 mL/min/1.73 m2. Fifteen patients with microalbuminuria will receive up to 20 mg of bard once daily, and 15 patients with macroalbuminuria will receive up to 30 mg of bard once daily. The primary endpoint for the Phase 2 portion of the study is change in eGFR at week 12 compared to baseline.
The Phase 3 portion is designed to support regulatory approval of bard. This portion will be double-blind and placebo-controlled and will randomize approximately 180 patients on a 1:1 basis to once-daily, oral bard or placebo. Similar to the Phase 2 portion of the trial, the study will assess dose escalation of bard from 5 mg to a maximum daily dose of 20 mg or 30 mg based on baseline proteinuria at randomization. The primary efficacy endpoint is the on-treatment change from baseline in eGFR in bardoxolone methyl-treated patients relative to placebo after 48 weeks. The key secondary endpoints will be the change from baseline in eGFR following a 4-week withdrawal of drug after one and two years of treatment. Based on FDA guidance, if the trial is positive, the year one off-treatment data could support accelerated approval under subpart H of the Food, Drug, and Cosmetic Act, and the year two off-treatment data could support full approval.
About Alport Syndrome
Alport syndrome is a rare, genetic disease caused by mutations in the genes encoding type IV collagen, a major structural component of the glomerular basement membrane (“GBM”) in the kidney. The abnormal expression of type IV collagen causes loss of GBM integrity, abnormal leakage of proteins such as albumin through the GBM, and excessive reabsorption of protein in the proximal tubules of the kidney. Like other forms of CKD, excessive reabsorption of protein in the tubules induces oxidative stress, chronic inflammation, and renal interstitial inflammation and fibrosis.
Alport syndrome affects approximately 12,000 people in the United States and 40,000 globally. Almost all patients with Alport syndrome develop end-stage renal disease (“ESRD”), and approximately 50% of male patients require dialysis or kidney transplant by the age of 25. There are currently no approved therapies to treat Alport syndrome.
About Bardoxolone Methyl
Bardoxolone methyl is an experimental, 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. Bardoxolone methyl is currently being studied in CATALYST, a Phase 3 study for the treatment of connective tissue disease associated pulmonary arterial hypertension (CTD-PAH).
About Reata Pharmaceuticals, Inc.
Reata Pharmaceuticals, Inc., 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 methyl and omaveloxolone) target an important transcription factor, called Nrf2, to restore mitochondrial function, reduce oxidative stress, and resolve inflammation.
This press release includes certain disclosures which 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 ability to obtain and retain regulatory approval of our product candidates, estimates of our expenses and our needs for additional financing, and our ability to obtain additional financing for our product development activities and existing and future clinical trials and pre-clinical programs. 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 are set forth in Reata’s filings with the U.S. Securities and Exchange Commission, including its Registration Statement on Form S-1, as amended from time to time, 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.