NephroGenex is a pharmaceutical company focused on the development of therapeutics to treat kidney disease, an area of significant unmet medical need. Since our inception, we have collaborated with the world’s leading experts in kidney disease and leveraged our knowledge of pathogenic oxidative chemistries to build a strong portfolio of intellectual property and to advance the development of our drug candidates. We believe that our comprehensive effort to develop a new generation of therapeutics that target kidney disease provides us with a leadership position in addressing this disease.
Pathogenic oxidative chemistries, including advanced glycation end-products (AGE’s), reactive oxygen species, and toxic carbonyls, are generally agreed to be involved in the etiology of diabetic nephropathy, a common complication of diabetes. We are developing Pyridorin™ (“Pyridorin”), a small molecule drug that is a unique and broadly acting inhibitor of the pathogenic oxidative chemistries which are elevated in diabetic patients.
The oral form of Pyridorin (pyridoxamine dihydrochloride) is being developed as a drug to slow the progression of diabetic nephropathy. Diabetic nephropathy is a degenerative disease of the kidney caused by diabetes.
As of 2010, there were approximately 6 million patients with diabetic nephropathy in the United States. This population is expected to grow. Patients suffering from this disease progress to End Stage Renal Failure (and require dialysis) or death. There are currently no adequate treatments for diabetic nephropathy.
"Chronic kidney disease (CKD) represents an enormous burden on the health care system of the U.S. with treatment costs exceeding $24 billion"
(U.S. Renal Data System. USRDS 2005 Annual Data Report. National Institutes of Health, National Institute of Diabetes and Digestive Kidney Disease: Bethesda, MD, 2005)
Pyridorin has shown promise in slowing the progression of diabetic nephropathy in three Phase 2 studies and has earned Fast Track status from the FDA. Pyridorin is one of the few drug candidates in late stage development for diabetic kidney disease.
NephroGenex will launch the Pyridorin Phase 3 program in 2014. NephroGenex has reached an agreement with the FDA on a new endpoint for Phase 3. The use of this endpoint should reduce the follow-up time of the Phase 3 program by approximately 50% over the previously accepted endpoint. We believe that we will be the first company to use this novel endpoint in a Phase 3 trial for diabetic nephropathy.
We are also studying the application of an intravenous formulation of Pyridorin to specific types of acute kidney injury (AKI) where pathogenic oxidative chemistries have been identified as a possible contributing factor to the severity of this condition.