Immunic's lead asset, vidofludimus calcium, is currently being evaluated in Phase 3 trials for relapsing multiple sclerosis (MS) and a Phase 2 trial for progressive MS, representing a potential advancement in MS therapy. The drug's mechanism of action targets both inflammation and neuroprotection, addressing critical unmet needs in MS treatment.
Addressing Unmet Needs in MS Treatment
Multiple sclerosis, affecting an estimated 2.9 million people worldwide, presents diverse symptoms and disease courses. Approximately 85% of patients initially experience relapsing MS, characterized by periods of remission and relapse. However, many transition to secondary progressive MS, marked by a steady worsening of health, even without relapses. A significant challenge is progression independent of relapse activity (PIRA), a gradual decline in physical and cognitive abilities observed across all MS forms, even with existing treatments. Currently, no therapies specifically target PIRA, highlighting a critical gap in MS care.
Chronic fatigue is another significant burden, affecting at least two-thirds of MS patients. This debilitating fatigue, distinct from ordinary tiredness, severely impacts daily life and social activities. Researchers suspect a link to constant viral reactivation, potentially involving the Epstein-Barr Virus (EBV).
Vidofludimus Calcium: A Dual-Action Approach
Vidofludimus calcium is a selective immune-modulator that activates the transcription factor nuclear receptor related 1 (Nurr1) and inhibits dihydroorotate dehydrogenase (DHODH). Nurr1 activation is associated with direct neuroprotective effects, while DHODH inhibition reduces the activity of overactive immune cells and virus-infected cells.
Dr. Daniel Vitt, CEO of Immunic, explains, "In microglia, resident immune cells in the brain, activation of Nurr1 leads to a reduction of pro-inflammatory cytokines and blocks the production of direct neurotoxic substances. Increased Nurr1 activity in neurons can directly promote neuronal survival and differentiation as well as improve neurotransmission."
Clinical Trial Milestones
Immunic has published Phase 2 EMPhASIS trial results with vidofludimus calcium in relapsing-remitting MS in "Neurology® Neuroimmunology & Neuroinflammation" and "Annals of Clinical and Translational Neurology." The company anticipates top-line data from its Phase 2 CALLIPER trial, focusing on progressive MS, in April 2025. "If the data from our Phase 2 CALLIPER trial, expected in April 2025, are convincing, Immunic could discuss accelerated approval with regulatory authorities," says Dr. Vitt.
The ongoing Phase 3 ENSURE trials in relapsing MS are expected to yield top-line data in 2026. These trials assess the impact on disability worsening, in addition to the primary endpoint of time to first relapse.
