NephroGenex initiates phase 3 trial for Pyridorin for diabetic nephropathy – July 21, 2014

In late June, NephroGenex, a North Carolina-based company focused on new therapies for kidney disease, announced the initiation of patient enrollment in the first of two phase 3 studies examining the safety and efficacy of the oral drug Pyridorin in slowing the progression of diabetic nephropathy. Pyridorin acts by inhibiting the formation of advanced glycation end products (AGEs) and inhibiting lipidoxidation while simultaneously trapping hydroxide radicals and toxic carbonyl compounds that are thought to be causative factors in the development of kidney disease. While normally we do not necessarily write about first patients enrolled in trials, we draw this to your attention because, notably, this study – part of the larger PIONEER program – has received fast track designation from the FDA and is designed to evaluate the efficacy of twice-daily administration of Pyridorin in mitigating the progression of renal disease in type 2 diabetes patients. The study (ClinicalTrials.gov Identifier: NCT02156843) is slated to enroll 600 patients, randomized to Pyridorin 300 mg twice daily or placebo, across 100 centers worldwide (the majority located within the US). Primary endpoints of the study are latency to a 50% increase in serum creatinine levels or progression to end stage renal disease. The primary completion date is slated for December 2017. Given the roughly six million patients who suffer from diabetic nephropathy in the US alone, we are excited about the late-stage development of this drug and its potential to address a significant unmet need. A phase 2 trial of this drug that was published in J Am Soc Nephrol (Lewis et al., 2012), found that Pyridorin 150 mg twice daily or 300 mg twice daily treatment produced a statistically significant treatment effect in people with less severe renal impairment (lowest tertile of baseline serum creatinine concentration) and not those with severe renal impairments. However, it does not appear that the company is limiting phase 3 enrollment to people with mild renal impairment.

• NephroGenex is collaborating with the collaborative study group (CSG) and Medpace to conduct the phase 3 program for Pyridorin.
• Also notably, NephroGenex recently went public in February 2014, raising $32.7 million; the current market capitalization of the company is $48.2 million. This company has flown under our radar screen for some time, and we look forward to learning much more now that they will have an impossible time staying stealth, given that they will have to disclose so much more as a public company.

-- by Varun Iyengar, Jessica Dong, Manu Venkat, and Kelly Close