NephRx initiates NX001 Phase I clinical trial to prevent DGF in kidney transplant patients

NephRx CorporationNov 11 2010

NephRx Corporation today announced that it has initiated a Phase I clinical trial of its lead compound NX001 and has successfully dosed the first subject in the study.  NX001, a kidney growth factor peptide that has demonstrated good safety and promising efficacy in two animal models of acute renal failure, is in development for the prevention of delayed graft function (DGF) in kidney transplantation patients.

Separately, NephRx announced that it has been awarded a total of $488,000 in two federal Qualifying Therapeutic Discovery Project (QTDP) grants.  One of the grants is to support the clinical development program for NX001, and the other is for NX002, NephRx's derivative of a naturally occurring growth factor in preclinical development as a treatment for oral mucositis, a common, painful and dose-limiting side effect of chemotherapy and radiation therapy in patients with head and neck cancers.

"NX001 has demonstrated promising activity in preclinical models of acute renal failure, and this clinical program is designed to test its effectiveness in helping to prevent or treat delayed graft function in renal transplant patients, a condition that currently lacks effective treatment options," said James Koziarz, Ph.D., CEO of NephRx Corp.  "We also welcome the recently announced federal grants to support the development of both NX001 and NX002."

DGF affects an estimated 20-25% of the approximately 14,000 renal transplants performed annually in the U.S.  Patients are put on dialysis to "rest" the transplanted kidney in the hope that this will correct metabolic imbalances and allow kidney function to return spontaneously.  Studies have shown that DGF can negatively affect future graft function and can increase the incidence of acute rejection episodes.  Kidney grafts with impaired function require the most intense follow-up and therapeutic management and are accordingly the most costly.  

The NX001 Phase I trial is a randomized, double-blind, placebo-controlled, single ascending dose level study of the safety, tolerability and pharmacokinetics of NX001 in healthy volunteers.  

"The launch of our first human trial represents an important milestone for NephRx," said F. Gary Toback, M.D., Ph.D., Founding Scientist of NephRx and Professor of Medicine and Cell Physiology at the University of Chicago Medical School.  "Because the kidney is one of the few human organs with the potential for self-repair, our kidney growth factor peptide has the potential to stimulate improved renal function in a number of disorders.  We chose DGF as our initial indication because it represents a well-defined condition where NX001 has the potential to improve near-term kidney function.  If these Phase I results are positive, Phase II trials in DGF patients could get underway by mid-2011."

NX001 has been designated an orphan drug by the U.S. FDA for this indication.  

NX002 has been shown to stimulate the growth of epithelial cells and to promote mucosal wound healing after injury.  In a recent study in a well-validated mucositis model, animals treated with NX002 had reduced levels of oral ulceration and a more rapid recovery, with no evidence of toxicity.  NephRx intends to initiate preclinical toxicology studies of NX002 in the coming months, with clinical trials targeted for late 2011.

The Qualifying Therapeutic Discovery Project, enacted as part of the federal Patient Protection and Affordable Care Act of 2010, is designed to provide grants or tax credits to qualified biotechnology company projects that demonstrate the potential to result in new therapies to treat areas of unmet medical need; to prevent, detect, or treat chronic or acute diseases or conditions; to reduce long-term health care costs in the United States; or to significantly advance the goal of curing cancer within 30 years.