Immunic Plans Phase 3 Progressive MS Trial After Mixed Phase 2 Results

Key Points

• Immunic plans a Phase 3 trial of vidofludimus calcium in about 1,400 people with progressive MS, with enrollment expected to begin in the first half of 2027. The study will compare the drug with placebo and assess disability outcomes.
• The plan follows mixed Phase 2 CALLIPER results: the trial missed its primary brain-volume endpoint but showed exploratory signals on disability measures. Those findings are not definitive, and researchers said a larger trial is needed to establish benefit.
• Immunic expects results from two Phase 3 trials in relapsing MS in December; favorable results could support a drug application next year and a potential 2028 launch, subject to regulatory approval. The company also cites a potential progressive-MS market of up to $18 billion, contingent on demonstrating benefit and securing approval.

Immunic NASDAQ: IMUX is preparing a Phase 3 trial of its experimental multiple sclerosis drug vidofludimus calcium in progressive disease, with the first patients expected to enroll in the first half of 2027.

The planned study follows mixed results from a Phase 2 trial that missed its primary goal but showed exploratory signals on disability measures. Separately, Immunic expects results from two Phase 3 trials in relapsing MS, ENSURE-1 and ENSURE-2, in December.

CEO Erik Lundgren said successful relapsing MS studies could support a new drug application next year and a potential market launch in 2028. Both the filing and launch depend on favorable results and regulatory approval.

Progressive MS Trial Design

The Phase 3 study, called ProTEct-MS, is expected to enroll approximately 1,400 patients with progressive MS. It will compare a 45-milligram dose of vidofludimus calcium with placebo and include patients with primary progressive MS and secondary progressive MS.

Chief Medical Officer Mike Panzara said the study will evaluate time to confirmed disability worsening over 24 weeks using a disability scale and a manual dexterity test known as the 9-Hole Peg Test. Secondary measures will include confirmed disability improvement, imaging results and patient-reported outcomes.

The trial will end based on the number of disability events observed rather than at a fixed time. Interim analyses will assess whether the study should stop for futility or efficacy and whether its sample size should be adjusted, Panzara said.

During the question-and-answer session, University of Toronto neurologist Dr. Jiwon Oh said a placebo-controlled design was appropriate for the non-active progressive patients being studied because there is no standard of care for that population.

Phase 2 Results Inform Plan

Immunic’s Phase 2 CALLIPER trial enrolled 467 patients with progressive MS and missed its primary endpoint, which measured change in brain volume. Panzara said the study nevertheless produced encouraging trends in disability outcomes and imaging measures.

For confirmed disability improvement, the pooled patient population showed a nominally significant result, with a p-value of 0.034, Panzara said. He cautioned that because the trial missed its primary endpoint, the subsequent findings could not be considered statistically significant within the study’s testing hierarchy.

Dr. Maria Pia Sormani, a biostatistics professor at the University of Genoa, discussed an analysis combining disability improvement and worsening. That approach produced a win ratio of 1.55 in favor of vidofludimus calcium. She said CALLIPER was not designed with enough patients to establish an effect on disability and that a larger Phase 3 trial would be needed.

Oh said whole-brain atrophy can be a problematic measure because it is not specific to a single disease process and may lag underlying changes. She also cautioned that it is difficult to separate the drug’s potential neuroprotective effects from its anti-inflammatory effects.

Commercial Ambitions

Chief Scientific Officer Hella Kohlhof said vidofludimus calcium is designed to activate Nurr1, a protein involved in neuronal maintenance and survival, while also inhibiting DHODH, a mechanism associated with anti-inflammatory effects. The company presented preclinical findings supporting potential neuroprotection; those effects remain to be established in the Phase 3 study.

President and Chief Operating Officer Jason Tardio estimated an annual progressive MS market opportunity of up to $18 billion across the United States, Europe and the United Kingdom, based on Immunic’s pricing assumptions. He said success in progressive MS could add billions of dollars in peak annual sales, but those projections depend on demonstrating clinical benefit and gaining approval.

Oh described treatments that definitively alter progression as a major unmet need. She said disability can worsen independently of relapses throughout the disease course, including in patients classified as having relapsing MS.

About Immunic (NASDAQ:IMUX)

Immunic, Inc NASDAQ: IMUX is a clinical-stage biopharmaceutical company developing orally administered therapies for chronic inflammatory and autoimmune diseases. The company focuses on treatments designed to address significant unmet needs while potentially offering convenient alternatives to injectable or infusion-based therapies.

Immunic's lead product candidate is vidofludimus calcium, formerly known as IMU-838. It is a selective oral inhibitor of dihydroorotate dehydrogenase (DHODH) being developed for multiple sclerosis, including relapsing and progressive forms of the disease.

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