Rare Daily Staff
Reata Pharmaceuticals and its partner Kyowa Hakko Kirin reported positive data on bardoxolone, an experimental treatment for a rare chronic kidney disease known as Alport syndrome, during a presentation at the 2017 American Society of Nephrology Kidney Week meeting.
Reata said in a mid-stage study bardoxolone met its primary efficacy endpoint of significantly increasing estimated glomerular filtration rate, a standard measure of kidney function, after 12 weeks of treatment. A late-stage study is currently underway, and data are expected in the second half of 2019.
Bardoxolone demonstrated a favorable safety profile with no effect on blood pressure, urinary volume or sodium retention, and no evidence of overt fluid overload or cardiac toxicity.
Based upon these results, Reata has activated sites in a mid-stage trial of bardoxolone in patients with other rare forms of chronic kidney disease, including autosomal dominant polycystic kidney disease, IgA nephropathy, CKD associated with type 1 diabetes, and focal segmental glomerulosclerosis. This is an open-label trial of bardoxolone orally-administered once-daily for 12 weeks as in the Alport syndrome study.
The primary efficacy endpoint of the study is change from baseline in the estimated glomerular filtration rate at week 12. Approximately 20 to 30 patients will be enrolled per cohort. The company anticipates that data from the individual cohorts of the study will be released throughout the second half of 2018 and 2019.
“Extensive clinical and preclinical development and insights from prior clinical studies have allowed us to rapidly generate phase 2 data in Alport syndrome and initiate a registrational trial in that indication,” said Colin Meyer, chief medical officer of Reata. “We believe that bardoxolone may impact a diverse set of kidney indications where inflammation, remodeling, and fibrosis are also central to the loss of kidney function, and we are optimistic that bardoxolone can be a meaningful treatment option for patients with these severe and underserved forms of CKD.”
November 6, 2017