Year Two of This Alport Syndrome Trial Concludes with Encouraging Findings

According to a story from GlobeNewswire, the biopharmaceutical company Reata Pharmaceuticals, Inc., recently announced that year two of its phase 3 clinical trial has been completed. This trial is testing the company’s experimental therapy bardoxolone methyl as a treatment for chronic kidney disease caused by Alport syndrome. The trial was able to successfully achieve its primary and secondary endpoints.

About Alport Syndrome

Alport syndrome is a genetic disorder which is characterized by end stage kidney disease, hearing loss, and glomerulonephritis, a type of kidney disease in which the glomeruli or small blood vessels in the kidneys become inflamed. Alport syndrome is a heritable disease and is caused by mutations of the COL4A3, COL4A4, and COL4A5 genes. The symptoms of the disorder include blood and protein in the urine, leiomyomatosis (coughing, vomiting, bronchitis, stomach pain, dysphagia), various eye conditions, and, rarely, aortic dissection. Since kidney failure inevitably develops eventually, treatment is similar to that of chronic kidney disease, and patients will eventually require dialysis or a kidney transplant. ACE inhibitors appear to slow the decline of kidney function. There is a serious need for more effective treatments for this disorder. To learn more about Alport syndrome, click here.

About The Clinical Trial

This clinical trial included a total of 157 patients living with chronic kidney disease linked to Alport syndrome. This study was international in scope and included a total of 50 trial sites spread throughout the US, Australia, Japan, and Europe. The primary endpoint was change to estimated glomerular filtration rate (eGFR) from baseline after 100 weeks of treatment (the end-of-treatment period). The secondary endpoint was change to eGFR after 104 weeks. For both of these endpoints, treatment with bardoxolone was able to cause statistically significant improvements in eGFR from baseline.

Data from a long term extension study suggests that the drug can produce long term improvements to eGFR, a widely accepted measurement of kidney function.

The safety profile of bardoxolone was similar to earlier studies and the drug was generally well-tolerated. Ten percent of patients being treated with the therapy experienced a serious adverse event. 

About Bardoxolone Methyl

Bardoxolone methyl is classified as an activator of the transcription factor Nrf2 which is known to reduce inflammation by inhibiting inflammatory signaling, restoring the function of mitochondria, and reducing oxidative stress. It has earned Orphan Drug designation in the US and the EU for treating Alport syndrome. The drug is currently being trialed for a variety of other diseases as well, including diabetic kidney disease, autosomal dominant polycystic kidney disease (ADPKD), COVID-19, IgA nephropathy, and focal segmental glomerulosclerosis (FSGS).

Following the results of this trial, Reata aims to file a New Drug Application with the FDA in early 2021.

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