Genkyotex, a biopharmaceutical company and the leader in NOX therapies, announced today that world-renowned diabetes experts Professor Mark Cooper, Head of the Department of Diabetes at Monash University, and Professor Jonathan Shaw, Deputy Director (Clinical and Population Health) at the Baker Heart and Diabetes Institute in Melbourne, Australia, will lead the conduct of a phase 2 clinical trial to evaluate the efficacy and safety of the Company’s lead product candidate, GKT831, in patients with type 1 diabetes and kidney disease (diabetic kidney disease).

This investigator-initiated study will be based at the Baker Institute and will include multiple study sites across Australia. This research is financially supported by JDRF Australia, the recipient of the Australian Research Council Special Research Initiative in Type 1 Juvenile Diabetes funding, with additional financial support by the Baker Institute. Genkyotex shall provide GKT831 Good Manufacturing Practice (GMP) material for the trial. The trial is expected to begin patient enrollment during the second half of 2017.

Diabetic kidney disease is a fibrotic disorder where progressive glomerulosclerosis and interstitial fibrosis leads to end stage renal disease. GKT831 is a NOX 1 and 4 enzyme inhibitor that has shown potent anti-fibrotic activity in a broad range of preclinical models including several DKD models [1-4]. In a previous, short-term phase 2 trial in patients with type 2 diabetes and kidney disease, GKT831 demonstrated an excellent safety profile and achieved statistically significant reductions in several secondary efficacy endpoints. However, improvements in albuminuria, the study’s primary efficacy endpoint, was not achieved after 12 weeks of treatment.

The Baker Institute study will be a placebo-controlled, double blind, randomized, parallel group phase 2 trial to evaluate the effect of oral GKT831 on the urine albumin-to-creatinine ratio (UACR) in patients with type 1 diabetes and persistent albuminuria despite treatment with optimal standard of care. The primary endpoint of the study will be UACR difference between means at the end of treatment period of 48 weeks, adjusted for baseline. A key secondary endpoint of the study will be the effect of GKT831 on renal function, as defined by changes in estimated glomerular filtration rate. Patients will receive 200mg of oral GKT831 or placebo twice a day for 48 weeks. A total of 142 patients are planned to be enrolled into the study at up to 15 investigational centers in Australia.

Professor Cooper has stated that “We are very excited to be commencing this study which arises in part from original research performed in our laboratories and which was initially supported by JDRF. This work is a classic example of bench to bedside clinical translation. We appreciate JDRF greatly assisting us in providing us with an opportunity to bring this new treatment forward for what is a major burden of T1D kidney disease.”

“We are delighted to be working with Professor Cooper and his team to pursue the clinical evaluation of GKT831 in this severe diabetic complication,” said Dr. Philippe Wiesel, chief medical officer of Genkyotex. “The design of this phase 2 trial was informed by previous phase 2 results in patients with type 2 diabetes and kidney disease performed by Genkyotex, in particular the extended 48-week treatment duration, a more homogenous and earlier stage patient population, and a higher dose throughout the dosing period. We also wish to thank JDRF for supporting this study, as well as previous preclinical studies, which has enabled a number of investigators to evaluate GKT831’s impact on ophthalmic, vascular, and renal complications caused by type 1 diabetes.”