Immunic Therapeutics reported positive top-line data from the phase 2 EMPhASIS trial evaluating IMU-838 versus placebo in patients with relapsing relapsing-remitting multiple sclerosis (RRMS). The study met primary and key secondary endpoints with high statistical significance, indicating activity for IMU-838 in relapsing-remitting multiple sclerosis. Analysis of the full EMPhASIS data is ongoing and will be presented at an upcoming scientific meeting.
EMPhASIS trial was an international, multicenter, double-blind, placebo-controlled, randomized, parallel-group study. Of the 210 patients randomized in 36 centers across four European countries, 209 patients received at least one dose of IMU-838 or placebo (placebo n=69, 30mg IMU-838 n=71, 45mg IMU-838 n=69), and 197 patients completed the blinded 24-week treatment period. All enrolled patients were required to have shown disease activity based on clinical evidence of relapse and additional MRI criteria. The study met its primary endpoint, demonstrating a statistically significant reduction in the cumulative number of combined unique active (CUA) magnetic resonance imaging (MRI) lesions up to week 24 in patients receiving 45mg of IMU-838 once daily, by 62%, as compared to placebo. The study also met its key secondary endpoint, showing a statistically significant reduction in the cumulative number of CUA MRI lesions for the 30mg once daily dose, by 70%, as compared to placebo. All other secondary endpoints, including those based on other MRI parameters and on clinical endpoints such as relapse events, also provided a noticeable signal and numerical benefit for the IMU-838 treatment groups, as compared to placebo. Treatment was determined to be safe and well tolerated.
Immunic is preparing a clinical phase 3 program for IMU-838 in RRMS and, after a full review of the data, anticipates providing a further update on development strategy.
IMU-838 is an orally available, next-generation selective immune modulator that inhibits the intracellular metabolism of activated immune cells by blocking the enzyme dihydroorotate dehydrogenase (DHODH). IMU-838 acts on activated T and B cells while leaving other immune cells largely unaffected and allows the immune system to stay functioning, e.g. in fighting infections. DHODH inhibitors, such as IMU-838, are known to possess a host-based antiviral effect, which is independent with respect to specific virus proteins and their structure. Therefore, DHODH inhibition may be broadly applicable against multiple viruses. IMU-838 was successfully tested in two phase 1 clinical trials in 2017 and is currently being tested in phase 2 trials in patients with COVID-19, relapsing-remitting multiple sclerosis and ulcerative colitis.
About Relapsing-Remitting Multiple Sclerosis
Multiple sclerosis (MS) is an autoimmune disease that affects the brain, spinal cord and optic nerve. In MS, myelin, the coating that protects the nerves, is attacked and damaged by the immune system. Relapsing-remitting MS (RRMS) is the most common form of the disease. Approximately 85% of patients with MS are expected to develop RRMS, with some of these patients later developing more progressive forms of the disease. RRMS is characterized by clearly defined attacks of new or increasing neurologic symptoms. These relapses are followed by periods of remission, or partial or complete recovery. MS affects more than 700,000 people in the United States, and more than 2.2 million people worldwide.