Genkyotex, the leading developer of NOX enzyme inhibitors, has received the US Food and Drug Administration (FDA) approval for its Investigational New Drug (IND) application to begin a phase II clinical study of GKT137831 in patients with diabetic nephropathy. GKT137831 is a first in class inhibitor targeting NOX1 and NOX4 enzymes.

Enrollment of patients into the multinational phase II study is expected to begin during Q4, 2013.

“This IND approval is a major milestone for Genkyotex. As the first company developing an inhibitor of the NOX enzyme pathway, GKT137831, we are delighted to receive validation by the FDA of our extensive preclinical and Phase I data package as well as approval for our phase II clinical trial design,” said Ursula Ney, Genkyotex's chief executive officer. “Our focus is now on completing preparations for enrollment of the first patients with diabetic nephropathy into the global phase II clinical proof of concept study, which will include investigational centres in North America, Europe and Australia.”

NOX enzymes exist in seven isoforms and produce reactive oxygen species (ROS). ROS can cause tissue damage and modify biological pathways that are important in a number of pathologies, including metabolic, cardiovascular, pulmonary and neurological diseases. In the kidney, NOX4 is the most abundantly expressed isoform and even further upregulated in diabetic nephropathy. The causal role of NOX enzymes in diabetic complications is well recognised. NOX4 plays a key role in glomerular damage and kidney fibrosis, which lead to albuminuria and end-stage renal disease, respectively. NOX1 is also involved in angiogenesis, atherosclerosis and other diabetic co-morbidities, making the inhibition of both the NOX1 and NOX4 enzymes by GKT137831, an attractive therapeutic option for this hard to treat and growing global disease. This competitive therapeutic profile of GKT137831 has been validated in several animal models of diabetes and diabetic nephropathy.

Genkyotex is developing first in class, small molecule therapeutics that specifically and selectively inhibit the NOX family of enzymes.