PLANO, Texas – Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” or “we”), a clinical-stage biopharmaceutical company, today announced that it has submitted a New Drug Application (“NDA”) for bardoxolone methyl (“bardoxolone”) for the treatment of chronic kidney disease (“CKD”) caused by Alport syndrome to the U.S. Food and Drug Administration (“FDA”).
This NDA submission is based on the efficacy and safety data from the CARDINAL Phase 3 clinical trial. The submission includes a request for Priority Review, which, if granted, would shorten the FDA’s review of the NDA to eight months from the time of submission, versus a standard review timeline of 12 months. If approved, bardoxolone would become the first therapy specifically indicated for the treatment of CKD caused by Alport syndrome.
“This NDA submission marks an important step toward making a treatment available for patients with Alport syndrome, a serious, progressive disease with an urgent need for new therapeutic options,” said Warren Huff, Reata’s President and Chief Executive Officer. “I want to thank all those who made this moment possible, especially Alport syndrome patients and their families. We look forward to next steps on the path to making bardoxolone available as a first-in-class therapy for Alport syndrome, pending NDA acceptance, review, and drug approval.”
About Alport Syndrome
Alport syndrome is a rare, genetic form of CKD caused by mutations in the genes encoding type IV collagen, which is a major structural component of the glomerular basement membrane in the kidney. The kidneys of patients with Alport syndrome progressively lose the capacity to filter waste products out of the blood, which can lead to end-stage kidney disease and the need for chronic dialysis treatment or a kidney transplant. Alport syndrome affects both children and adults. In patients with the most severe forms of the disease, approximately 50% progress to dialysis by age 25, 90% by age 40, and nearly 100% by age 60. According to the Alport Syndrome Foundation, Alport syndrome affects approximately 30,000 to 60,000 people in the United States. There are currently no approved therapies to treat CKD caused by Alport syndrome.
About the CARDINAL Clinical Study
CARDINAL was an international, multi-center, Phase 2/3 study that enrolled patients from 12 to 60 years old with a confirmed genetic or histological diagnosis of Alport syndrome, baseline estimated glomerular filtration rate (“eGFR”) values between 30 to 90 mL/min/1.73 m2 , and on stable renin-angiotensin-aldosterone system blockade unless contraindicated. The Phase 3 portion of CARDINAL was a double-blind, placebo-controlled, randomized trial that enrolled 157 patients with CKD caused by Alport syndrome at approximately 50 study sites in the United States, Europe, Japan, and Australia. Patients were randomized 1:1 to once-daily, oral bardoxolone or placebo. The primary endpoint for Year 2 of the study was the change from baseline in eGFR after 100 weeks of treatment. The key secondary endpoint for Year 2 of the study was the change from baseline in eGFR at Week 104 (four weeks after last dose in second year of treatment).
Results from CARDINAL demonstrated that patients treated with bardoxolone experienced a statistically significant improvement in kidney function as measured by eGFR at Week 100 and Week 104, compared to patients treated with placebo. Bardoxolone was generally reported to be well tolerated in this study, and the safety profile was similar to that observed in prior trials. The reported adverse events (“AE”) were generally mild to moderate in intensity, and the most common AEs observed more frequently in patients treated with bardoxolone compared to patients treated with placebo were muscle spasms and increases in aminotransferases.
Bardoxolone is an investigational, once-daily, orally administered 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 designation to bardoxolone for the treatment of Alport syndrome and autosomal dominant polycystic kidney disease (“ADPKD”). The European Commission has granted Orphan Drug designation in Europe to bardoxolone for the treatment of Alport syndrome.
In addition to the CARDINAL Phase 3 study, bardoxolone is currently being studied in FALCON, a Phase 3 study for the treatment of ADPKD, MERLIN, a Phase 2 study for the treatment of patients with CKD at risk of rapid progression, and AYAME, a Phase 3 study for the treatment of diabetic kidney disease that is being conducted by our licensee, Kyowa Kirin Co., Ltd., in Japan. Bardoxolone treatment has produced positive results in Phase 2 studies in patients with CKD caused by ADPKD, IgA nephropathy, focal segmental glomerulosclerosis, and type 1 diabetes.
About Reata Pharmaceuticals, Inc.
Reata is a clinical-stage biopharmaceutical company that develops novel therapeutics for patients with serious or lifethreatening 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 the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. Bardoxolone and omaveloxolone are investigational drugs, and their safety and efficacy have not been established by any agency.
Reata Pharmaceuticals, Inc.
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