Vidofludimus Calcium Significantly Reduces Lesions of Multiple Sclerosis With Minimal Side Effects

As published in Annals of Clinical and Translational Neurology, vidofludimus calcium (VFC) (IMU-838; Immunic Therapeutics, New York, NY) significantly reduced disease activity in relapsing-remitting multiple sclerosis (MS). These data come from the phase 2 EMPHASIS trial (NCT03846219), in which adults with evidence of active RRMS (as defined by the 2017 McDonald criteria) were treated with VFC vs placebo. 

Participants treated with 45 mg/day VFC for 24 weeks had a 62% reduction in combined active lesions (CUA) on MRI compared with those who received placebo (P=.0002). In those treated with 30 mg/day VFC vs placebo, there was a 70% (P<.0001) reduction in CUA. 

Notably, no serious adverse events, including hepatotoxicity, occurred and the overall adverse event rate was similar between participants treated with VFC or placebo. Discontinuation rates were low at 2.8% (2/71) and 5.8% (4/69) with 30 or 45 mg/day VFC, respectively, and 7.2% (5/69) with placebo. 

“The results from this phase 2 trial of vidofludimus calcium in patients with RRMS are encouraging, as the trial met its primary and key secondary endpoints for suppressing the number of CUA MRI lesions,” stated coordinating investigator, Robert J. Fox, MD, staff neurologist, Mellen Center for Multiple Sclerosis, vice-chair for research, Neurologic Institute, Cleveland Clinic, Cleveland, Ohio, “Importantly, vidofludimus calcium was found to be safe and well-tolerated as compared to placebo, with no increase in the rate of infections, effects on liver or blood cell laboratory parameters and with a very low treatment discontinuation rate.” Dr. Fox receives consulting fees for serving as an advisor to Immunic.

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