NephroGenex has started patient enrolment for the first of two pivotal Phase III studies in the PIONEER programme for oral Pyridorin, which is indicated for slow progression of diabetic kidney disease.
The trial aims to assess the safety and efficacy of Pyridorin in slowing disease progression in patients with diabetic nephropathy, a chronic and degenerative disease of the kidney caused by diabetes.
Pyridorin inhibits pathogenic oxidative chemistries, which are collectively elevated in diabetic patients and induces pathological changes implicated in the development of diabetic nephropathy, according to the company.
NephroGenex CEO Pierre Legault said with initiation of this study, the company is now in late-stage development with a first-in-class compound that targets and scavenges pathogenic oxidative chemistries, which are fundamental causative factors in the development and progression of diabetic nephropathy.
"Of the 19 million people in the US diagnosed with diabetes, about one third, or roughly six million people, exhibit signs and symptoms of kidney disease," Legault said.
"Pyridorin is the leading drug candidate for diabetic nephropathy that targets an underlying cause of this disease."
The two identical double-blind placebo controlled Phase III trials will evaluate the safety and efficacy of Pyridorin at 300mg twice a day compared with placebo in reducing the renal disease progression rate in type 2 diabetic patients.
Each study will recruit approximately 600 patients who are randomized in a 1:1 ratio to receive either Pyridorin or placebo.
The trial will be conducted at approximately 100 centres across the globe, with the majority being situated in the US.
Primary efficacy endpoints of the study are time to a 50% increase in serum creatinine (SCr) levels, or end-stage renal disease (ESRD).
The Phase III study is 90% powered to identify a 28% treatment effect, while the previous Phase IIb study showed a greater than 50% treatment efficacy in the target patient population.
NephroGenex reached an agreement with the FDA on a special protocol assessment (SPA) for the PIONEER clinical programme. It received fast-track designation from the FDA for Pyridorin in diabetic nephropathy.