Vidofludimus Calcium a Selective T- and B-Cell Reduced Multiple Sclerosis Disease Activity 

In the phase 2 EMPhASIS trial (NCT03846219) vidofludimus calcium (VFC)(IMU-838; Immunic, New York, NY) reduced new combined unique active (CUA) MRI lesions in individuals with multiple sclerosis (MS). CUA is a measure that counts unique lesions that are new or enlarging on T2 MRI or gadolinium-enhancing (Gd+) on T1 MRI without counting any lesion more than once.

Individuals treated with 30 or 45 mg/day VFC for 12 weeks, respectively, had 70% and 62% reductions in CUA compared with placebo (P<.001 for both). Similar reductions were seen when counting Gd+ lesions only and the annualized relapse rate. 

Rates of overall treatment-emergent adverse events were similar among all 3 groups. Of note, there were no serious adverse events with 30 mg/day of VFC, although 3 individuals (4.3%) treated with 45 mg/day had liver enzyme elevations (n=2) or rash (n=1). There were 5 adverse events in the placebo group (liver enzyme elevation [n=2], cervical carcinoma [n=1], and hematuria [n=1].

Based on all available data, we believe that the dose of 30mg once-daily IMU-838 should be considered the most appropriate dose for relapsing-remitting MS patients,” said Andreas Muehler, MD, chief medical officer, Immunic. “While we continue our discussions with major regulatory authorities, we will move ahead with formal phase 3 feasibility activities.”

In this double-blind randomized placebo-controlled study, adults with relapsing-remitting MS as defined by the McDonald 2017 criteria with evidence of active disease were randomly assigned to receive oral VFC (30 or 45 mg/day) or placebo. Low discontinuation rates of 5.8% (4/69), 2.8% (2/71) with VFC 40 and 30 mg/day, respectively, and 7.2% (5/69) with placebo were seen.

VFC is a dihydro-orotate dehydrogenase (DHODH) inhibitor that selectively inhibits activated T and B cells selectively, leaving other immune cells largely unaffected and allowing the immune system to continue functioning. 

“Data thus far continues to convince us that IMU-838 may become an important, new oral therapeutic option with an outstanding combination of safety, tolerability, and robust efficacy for the treatment of patients suffering from RRMS, and we are eager to move ahead with its final clinical development steps,” said Daniel Vitt, PhD, chief executive officer and president, Immunic. 

These data were presented at the virtual 2021 American Academy of Neurology (AAN) Virtual Annual Meeting April 17-22, 2021.