MS drug shows promise in progressive cases, despite missed trial goal

US biotech Immunic (Nasdaq: IMUX) has reported top-line results from a mid-stage study of its lead candidate vidofludimus calcium in patients with progressive multiple sclerosis, delivering mixed news that sent its share price into a sharp decline.

The Phase II CALLIPER trial failed to meet its primary exploratory MRI endpoint, showing only a 5% improvement over placebo in whole brain volume change after 24 months of treatment. Despite this modest finding, the company emphasized signs of efficacy on disability-related measures and said the results justify advancing to Phase III.

The drug, an oral activator of nuclear receptor Nurr1, reduced the risk of 24-week confirmed disability worsening by 20% in the full study group of 467 patients. That effect increased to 30% in the subgroup with primary progressive MS, a form of the disease with few treatment options.