Regulatory Pathway for Pyridorin™ Therapy

NephroGenex continues to discuss the regulatory pathway for approval of Pyridorin™ as a treatment for diabetic nephropathy with the FDA. NephroGenex is currently exploring novel strategies for accelerated approvable, such as the use of surrogate markers (subpart H approval) that would subsequently be confirmed with hard clinical endpoints. Such strategies are incorporated into the design of the new PYR 210 protocol.

PYR 210

NephroGenex has launched a Phase 2b clinical trial investigating the safety and efficacy of Pyridorin™ therapy in slowing the progression of diabetic overt nephropathy in patients with type 2 diabetes. The treatment period for this trial is 12 months, and the primary endpoint is year-1 serum creatinine progression. Details of this trial can be viewed at www.clinicaltrials.gov

The objective of this trial is to confirm the safety and efficacy of Pyridorin™ therapy in slowing the progression of overt nephropathy as observed in two previous Phase 2 trials. The trial is being conducted by the Collaborative Research Group, a site-management organization of leading nephrologists who specializes in kidney diseases, in collaboration with NephroGenex.

Phase 3 Program

At the conclusion of the current Phase 2b trial, 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.