Immunic, Inc. announced the start of the patient cohorts in its ongoing phase 1 clinical trial of IMU-856, the company's third clinical asset, in patients with celiac disease. IMU-856 is an orally available and systemically acting small molecule modulator that targets an undisclosed epigenetic regulator. Preclinical studies suggest that IMU-856 can restore barrier function in the gastrointestinal tract and also regenerate intestinal architecture while maintaining immunocompetency.

Based on preclinical and early clinical data available to date, the company believes that IMU-856 may represent a novel and potentially ground-breaking approach to the treatment of gastrointestinal diseases. Parts A and B of the ongoing phase 1 clinical trial are evaluating single and multiple ascending doses of IMU-856 in healthy human subjects. The now initiated Part C is structured as a 28-day, double-blind, placebo-controlled trial designed to assess the safety and tolerability of IMU-856 in patients with celiac disease during periods of gluten-free diet and gluten challenge.

Approximately 42 patients are planned to be enrolled in two consecutive cohorts with IMU-856 given once-daily over 28 days. Secondary objectives include pharmacokinetics and disease markers, including those evaluating gastrointestinal architecture and inflammation. Approximately 10 sites in Australia and New Zealand are expected to participate in Part C. The company also reiterates its prior guidance that phase 2 top-line data of vidofludimus calcium (IMU-838) in ulcerative colitis is expected to be available in June of 2022 and that initial clinical efficacy data of the Part C portion of the ongoing phase 1 clinical trial of IMU-935 in psoriasis is expected in the second half of 2022.

IMU-856, which Immunic believes to be novel, is an orally available small molecule modulator that targets a protein which serves as a transcriptional regulator of intestinal barrier function and regeneration of bowel epithelium. Based on preclinical data, the compound may represent a new 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 maintain immune surveillance for patients during therapy, an important advantage versus chronic treatment with potentially immunosuppressive medications.

IMU-856 is an investigational drug product that has not been approved in any jurisdiction. IMU-856 was discovered and initially developed by Daiichi Sankyo Co. Ltd. (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. The license also includes exclusivity on a patent application filed by Daiichi Sankyo, covering IMU-856's composition of matter. Immunic exercised the option in January 2020.