IMU-856

IMU-856 is an orally available and systemically acting small molecule modulator that targets a protein which serves as a transcriptional regulator of intestinal barrier function and regeneration of bowel epithelium. Immunic has not yet disclosed the molecular target for IMU-856. Based on preclinical data, the compound may represent a unique treatment approach, as the mechanism of action targets the restoration of the intestinal barrier function and bowel wall architecture in patients suffering from gastrointestinal diseases such as celiac disease, inflammatory bowel disease, irritable bowel syndrome with diarrhea and other intestinal barrier function associated diseases. Immunic believes that, because IMU-856 has been shown in preclinical investigations to avoid suppression of immune cells, it may therefore have the potential to maintain immune surveillance for patients during therapy, an important advantage versus immunosuppressive medications.

IMU-856 was discovered by Daiichi Sankyo. In November 2018, Immunic and Daiichi Sankyo entered into a global option and license agreement, granting Immunic the exclusive right to license IMU-856. Under this agreement, Immunic has the rights to commercialization of IMU-856 in all countries. Immunic exercised the option in January 2020.

A double-blind, randomized, placebo-controlled phase 1 clinical trial comprised of three parts is currently ongoing. In September 2022, Immunic announced positive results from Part A (single ascending doses, or SAD) and Part B (multiple ascending doses, or MAD) of the trial in healthy human subjects. The data revealed a favorable safety, tolerability and pharmacokinetic profile for IMU‑856 in single and 14-day multiple dosing. No maximum tolerated dose was reached and the investigated doses are expected to exceed the required therapeutic dosing of IMU-856.

The ongoing Part C of the phase 1 program is a double-blind, randomized, placebo-controlled trial designed to assess the safety and tolerability of IMU-856 during 28 days of treatment with 80 and 160 mg of IMU-856 or placebo, once daily, in patients with celiac disease during periods of gluten-free diet and gluten challenge. Secondary objectives include pharmacokinetic as well as acute and chronic disease markers, including those evaluating acute response on the biomarker interleukin-2, or IL-2, and gastrointestinal architecture and inflammation. Initial results are expected to be available in mid-2023.