Early Phase 2 Data Support Testing GKT831 in IPF, Continuing Work with Other Fibrotic Diseases

Alice Melão, MSc avatar

by Alice Melão, MSc |

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Genkyotex’s anti-fibrotic candidate GKT831 appears to be safe and hold therapeutic activity in an ongoing Phase 2 trial in patients with primary biliary cholangitis, an autoimmune-triggered fibrotic disease of the liver.

These positive interim results support the development of GKT831 as a possible treatment of other fibrotic diseases, including idiopathic pulmonary fibrosis (IPF) and nonalcoholic steatohepatitis (NASH).

In July 2018, the National Institutes of Health (NIH) awarded $8.9 million to Victor Thannickal, MD, a professor at the University of Alabama at Birmingham, to lead a multi-year research program to assess the role of NOX enzymes in IPF. NOX enzymes are involved in the generation of reactive oxygen species (ROS), and high levels of ROS have been associated with tissue damage.

The program’s core component will be a Phase 2 trial evaluating the safety and efficacy of GKT831 in IPF patients, which is expected to be launched in the first half of 2019. GKT831 is an inhibitor of NOX1 and NOX4 enzymes.

“The clinical data [obtained so far] … establish GKT831 as a novel anti-fibrotic candidate and confirm the therapeutic value of NOX inhibition for patients,” Elias Papatheodorou, CEO of Genkyotex, said in a press release. “NOX inhibitors are an emerging therapeutic class able to address many human diseases with unmet medical need.”

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Genkyotex is currently evaluating the clinical potential of GKT831 in an ongoing Phase 2 trial in primary biliary cholangitis (PBC, NCT03226067), and in a Phase 2 study in diabetes and kidney disease (DKD, NCT02010242).

Recent preliminary data collected during the study in 111 patients with PBC support treatment with GKT831 being generally safe. One serious adverse event was reported, and found to be unrelated to the treatment.

Patients are being randomized to receive a stable dose of ursodiol (sold under the brand names Actigall, among others) plus GKT831 capsules, 400 mg once or twice a day, or matching placebo for 24 weeks.

Interim, six-week results had patients treated with GKT831 showing significant improvements in several markers of liver and bile duct injury, as well as of liver inflammation.

“[These results provide] clinical confirmation of GKT831’s mechanism of action,” said Philippe Wiesel, chief medical officer of Genkyotex. “We are looking forward to the launch of the NIH funded IPF Phase 2 trial … the continuation of the JDRF funded DKD Phase 2 trial, and of course, seeing the final data of the PBC Phase 2 trial at week 24 in Spring 2019.”