Overview

NephroGenex's focus is the development of novel therapies for kidney disease. The Company is headquartered in Research Triangle Park, North Carolina.

NephroGenex has acquired commercial rights to Pyridorin™ (pyridoxamine dihydrochloride). Pyridorin™ targets diabetes-induced carbonyl and oxidative chemistries that are a principal causative factor in the development of diabetic nephropathy and other diabetic complications.

Pyridorin™ has demonstrated a significant treatment effect in slowing the progression of diabetic nephropathy in three Phase 2 clinical trials, and has been awarded Fast Track status by the FDA. NephroGenex recently completed a Phase 2b clinical trial (PYR-210) that evaluated the safety and efficacy of Pyridorin™ in slowing the progression of overt nephropathy in patients with type 2 diabetes. This trial incorporated a year-one SCr change endpoint as a surrogate marker to predict time to ESRD. In patients on previously established standard of care, a 300 mg BID dose of Pyridorin showed a 57% treatment effect on patients with mild to moderate disease. This effect was dose dependent and highly statistically significant (P=0.009).

NephroGenex has reached an agreement with the FDA on the design of a new Phase 3 Subpart H program that evaluates Pyridorin™ in diabetic nephropathy patients. This Subpart H program uses a novel surrogate endpoint based on an increase in serum creatinine (SCr), and provides for an accelerated regulatory pathway to approval.

Diabetic kidney disease afflicts about 33% of all diabetics and is the major cause of end-stage renal disease (ESRD). There are an estimated 2.8 million diabetic overt nephropathy patients in the US, and approximately 3.5 million diabetic patients exhibiting signs of developing kidney disease. Pyridorin™ is one of only two drug candidates in advanced clinical trials for diabetic kidney disease.

NephroGenex is also studying the application of Pyridorin™ to specific types of acute renal failure where carbonyl and oxidative chemistries have been identified as causative factors in the onset and progression of this condition.

Corporate Objectives

NephroGenex’s principal corporate objective is the acquisition of clinical candidates with application to kidney disease. NephroGenex’s business model leverages the enormous medical need and market opportunity that exist for treatments that can slow the progression of renal disease and thus delay or avoid the onset of end-stage renal disease (ESRD).

NephroGenex acquired commercial rights to Pyridorin™ and is currently investigating the safety and efficacy of Pyridorin™ therapy for diseases in which pathogenic carbonyl and oxidative chemistries are an established causative factor in kidney disease. These include diabetic nephropathy and acute renal injury.

The Company aims to undertake its subsequent evaluation in Phase 2 safety and efficacy trials against relevant patient populations identified using the latest advances in clinical research and kidney-disease diagnostics.

The Company will seek corporate partners for Phase 3 development and commercialization.