Immunic announced the first patient enrolled in a proof-of-concept clinical trial of IMU-838 for the treatment of primary sclerosing cholangitis, according to a press release.
The National Institutes of Health awarded a grant to Keith Lindor, MD, principal investigator for the trail from the College of Health Solutions at Arizona State University. Additionally, Elizabeth Carey, MD, from the Mayo Clinic in Arizona, sponsored the trial with an investigational new drug approval from the FDA to conduct the study.
“Recent studies indicate that the proinflammatory cytokine interleukin 17, or IL-17, may play a central role in the pathogenesis of PSC, as well as ulcerative colitis,” Lindor said in the release. “Significant increases in IL-17-expressing lymphocytes are found in the livers of PSC patients. These findings speak to the strong possibility of an overlap in therapeutic approaches to the two diseases.”
“Our goal with this study is to examine the safety, tolerability, and efficacy of daily dosing of IMU-838, an orally available, small molecule inhibitor of DHODH, a target known for its effect on Th17 cells, in order to establish proof-of-concept that IMU-838 shows activity for the treatment of PSC,” Lindor continued. “Establishing such a baseline should enable the design of more comprehensive clinical studies.”
The investigators will enroll 30 adult patients with PSC in the single-arm, open-label study. Patients will receive 30 mg of IMU-838 once daily for 6 months. The primary endpoint will be change in serum alkaline phosphatase at study end compared with baseline.