Immunic Reports Positive Interim Analysis in Phase 3 ENSURE Study of Vidofludimus Calcium for Relapsing MS

Immunic, Inc., a biotechnology firm focusing on the development of oral small molecule therapies for chronic inflammatory and autoimmune diseases, has reported a positive interim outcome for its phase 3 ENSURE program. This program investigates the efficacy of vidofludimus calcium (IMU-838), a nuclear receptor related 1 (Nurr1) activator, for the treatment of relapsing multiple sclerosis (RMS).

The interim futility analysis, conducted by an Independent Data Monitoring Committee (IDMC), has concluded that the trials are not futile and should proceed as planned. This analysis was designed to evaluate whether the trials, ENSURE-1 and ENSURE-2, would likely meet their primary endpoint of time to first relapse. The IDMC, after reviewing unblinded data, affirmed that the current sample size is sufficient, and no changes, including sample size increases, are necessary.

Dr. Andreas Muehler, Chief Medical Officer of Immunic, highlighted the importance of this interim analysis. He noted that the IDMC's recommendations align with the initial design and assumptions of the trials, suggesting that the data collected thus far are consistent with expectations. This positive outcome supports the continuation of the trials, with completion anticipated in 2026.

Dr. Daniel Vitt, CEO of Immunic, expressed enthusiasm about reaching this significant milestone in the ENSURE program. He emphasized the potential of vidofludimus calcium to make a substantial impact on the oral treatment market for multiple sclerosis. The next milestone for the drug will be the top-line results of the phase 2 CALLIPER trial for progressive multiple sclerosis (PMS), expected in April of the following year. If the data demonstrate a neuroprotective effect, vidofludimus calcium could become a pioneering oral treatment for PMS, addressing a critical unmet need in this severe form of multiple sclerosis.

The interim analysis was based on a pre-specified assessment point, after roughly half of the planned first relapse events had occurred in both ENSURE-1 and ENSURE-2 trials. The IDMC was tasked with determining whether the trials were futile and if a sample size adjustment was needed. Both recommendations were based on the trials’ conditional power at the interim analysis stage. Immunic has remained blinded to the data reviewed by the IDMC.

The ENSURE program consists of two identical multicenter, randomized, double-blind phase 3 trials. These trials are evaluating the efficacy, safety, and tolerability of vidofludimus calcium compared to a placebo in RMS patients. Each trial aims to enroll around 1,050 adults across more than 100 sites in over 15 countries, including the United States, India, and regions in Latin America, Central, and Eastern Europe. Participants are randomly assigned to receive either 30 mg of vidofludimus calcium daily or a placebo, with the primary endpoint being the time to first relapse within 72 weeks. Secondary endpoints include time to confirmed disability worsening, volume of new T2-lesions, changes in cognition, and brain volume changes.

Immunic will host a webcast to discuss these findings. Vidofludimus calcium, the company’s leading investigational drug, is being developed as a treatment for multiple sclerosis and other chronic inflammatory and autoimmune diseases. It functions by activating the neuroprotective transcription factor Nurr1 and inhibiting the enzyme dihydroorotate dehydrogenase (DHODH), which plays a role in the immune response and viral infections. So far, the drug has demonstrated a favorable pharmacokinetic, safety, and tolerability profile in over 1,800 individuals. However, it is not yet approved for use in any country.

Immunic, Inc. continues to advance its clinical pipeline, seeking to address significant medical needs in the field of autoimmune and inflammatory diseases through innovative oral therapies.