Regulatory Pathway for Pyridorin™ Therapy

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.

The new surrogate endpoint was determined from a comprehensive analysis of prior large clinical trials in diabetic nephropathy patients that demonstrated a highly significant relationship between increases in SCr and the future progression of patients to end stage renal disease (ESRD).

In the Pyridorin™ Phase 3 Subpart H program, the FDA has agreed that approval will be based on a new surrogate endpoint that includes SCr changes after one year that predict progression to ESRD. These patients will subsequently be followed to confirm the clinical benefit of Pyridorin™ in delaying progression to ESRD. The patient population for the study will be type 2 diabetic patients with mild to moderate overt nephropathy who are on an established standard of care consisting of adequate blood pressure control and a stable regimen of ACEi/ARB therapy prior to initial screening.

Phase 3 Program

NephroGenex will secure a corporate partner for Phase 3 development and commercialization.

2nd Generation Candidates

Structure-function relationships defining pyridoxamine’s inhibitory activity toward pathogenic oxidative chemistries have been established by NephroGenex scientists. These relationships have been used to design, synthesize, and evaluate numerous proprietary 2nd-generation candidates. NephroGenex has also acquired commercial rights to these compounds. One promising compound, which exhibits improved potency over Pyridorin™, has completed initial preclinical efficacy and toxicity studies with encouraging results.