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Vidofludimus calcium consistently reduced neurofilament light chain (NfL) levels in the Phase 2 CALLIPER trial, suggesting it may slow disease progression across all progressive MS subtypes, the company reported.

The drug also showed potential to reduce fatigue in MS patients by preventing Epstein-Barr virus (EBV) reactivation, which could contribute to reduced fatigue in MS patients.

https://www.proactiveinvestors.com/comp ... 05646.html

Vidofludimus calcium is also helpful in RRMS.

A double-blind, randomized, placebo-controlled phase 2 trial evaluating the selective dihydroorotate dehydrogenase inhibitor vidofludimus calcium in relapsing-remitting multiple sclerosis
Ann Clin Transl Neurol. 2022 Jul;9(7):977-987.

Objective: Inhibition of dihydroorotate dehydrogenase suppresses magnetic resonance imaging brain lesions and disease activity in multiple sclerosis but has limiting tolerability. We assessed the safety and efficacy of vidofludimus calcium, a novel, selective dihydroorotate dehydrogenase inhibitor, in patients with relapsing-remitting multiple sclerosis.

Methods: This double-blind, 24 weeks, placebo-controlled, phase 2 trial (EMPhASIS) enrolled patients 18-55 years with relapsing-remitting multiple sclerosis. Eligible patients were randomly assigned (1:1:1) to once-daily vidofludimus calcium (30 mg or 45 mg) or placebo. The primary endpoint was the cumulative number of combined unique active lesions to week 24 between vidofludimus calcium 45 mg and placebo (clinicalTrials.gov number NCT03846219; EudraCT 2018-001896-19).

Results: After 24 weeks, the mean cumulative number of combined unique active lesions was 6.4 (95% CI: 2.8-13.9) with placebo compared to 2.4 (95% CI: 1.1-4.9) with vidofludimus calcium 45 mg (rate ratio 0.38, 95% CI: 0.22-0.64; p = 0.0002); the rate ratio between vidofludimus calcium 30 mg and placebo was 0.30 (95% CI: 0.17-0.53; p < 0.0001). Treatment-emergent adverse events occurred in 30 (44%) of patients assigned placebo and 60 (43%) of patients assigned vidofludimus calcium. Serious adverse events occurred in one (1%) assigned placebo and two (1%) assigned vidofludimus calcium. No increased incidence of infectious, hepatic, or renal treatment-emergent adverse events or serious adverse events was observed.

Interpretation: Treatment with vidofludimus calcium led to a reduction in new magnetic resonance imaging lesions in patients with relapsing-remitting multiple sclerosis and was well tolerated with a favorable safety profile. Assessment in longer, larger trials is justified.

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Treats RRMS, will be the second PPMS drug, and first to be approved for non-relapsing SPMS.
It reduces EBV reactivation, there is less viral infection of any kind and covid, compared to placebo group.
The opposite of what we get from current DMTs!
The Nurr1 effect stops the brain inflammation from microglia and astrocytes, protecting neurons.
Great at lowering Nfl levels, reducing lesions, significantly slows progression.
Plus it heals the gut lining.

https://filecache.investorroom.com/mr5i ... r_2024.pdf

Phase 3 ENSURE Program of Vidofludimus Calcium Continues Following Positive Futility Analysis

https://www.neurologylive.com/view/phas ... y-analysis

From Facebook:

"Just got a phone call. Are you sitting down?

I’ve been on the CALLIPER trial for a MS med called IMU838 since summer of 2023. just got a call that the med reached all its endpoints with positive results. Early!

It was reported at ECTRIMS in Italy in April so I get to end the study now instead of December 2025! moving to open label which means I get the meds and follow up for eight years . FREE!

I am thrilled beyond belief! exciting way to start 59. "

https://imux.com/pipeline/imu-838/

Safety and Dose-Response of Vidofludimus Calcium in Relapsing Multiple Sclerosis: Extended Results of a Placebo-Controlled Phase 2 Trial
Neurol Neuroimmunol Neuroinflamm. 2024 May;11(3):e200208.

Background and objectives: Vidofludimus calcium suppressed MRI disease activity compared with placebo in patients with relapsing-remitting multiple sclerosis (RRMS) in the first cohort of the phase 2 EMPhASIS study. Because 30 mg and 45 mg showed comparable activity on multiple end points, the study enrolled an additional low-dose cohort to further investigate a dose-response relationship.

Methods: In a randomized, placebo-controlled, phase 2 trial, patients with RRMS, aged 18-55 years, and with ≥2 relapses in the last 2 years or ≥1 relapse in the last year, and ≥1 gadolinium-enhancing brain lesion in the last 6 months. Patients were randomly assigned (1:1:1) vidofludimus calcium (30 or 45 mg) or placebo in cohort 1 and vidofludimus calcium (10 mg) or placebo (4:1) in cohort 2 for 24 weeks. The primary end point was the cumulative number of combined unique active (CUA) lesions at week 24. Secondary end points were clinical outcomes and safety.

Results: Across cohorts 1 and 2, 268 patients were randomized to placebo (n = 81), 10 mg (n = 47) vidofludimus calcium, 30 mg (n = 71) vidofludimus calcium, or 45 mg (n = 69) vidofludimus calcium. The mean cumulative CUA lesions over 24 weeks was 5.8 (95% CI 4.1-8.2) for placebo, 5.9 (95% CI 3.9-9.0) for 10 mg treatment group, 1.4 (95% CI 0.9-2.1) for 30 mg treatment group, and 1.7 (95% CI 1.1-2.5) for 45 mg treatment group. Serum neurofilament light chain decreased in a dose-dependent manner. The number of patients with confirmed disability worsening after 24 weeks was 3 (3.7%) patients receiving placebo and 3 (1.6%) patients receiving any dose of vidofludimus calcium. Treatment-emergent adverse events occurred in 35 (43%) placebo patients compared with 11 (23%) and 71 (37%) patients in the 10 mg or any dose of vidofludimus calcium groups, respectively. The incidence of liver enzyme elevations and infections were similar between placebo and any dose of vidofludimus calcium. No new safety signals were observed.

Discussion: Compared with placebo, vidofludimus calcium suppressed the development of new brain lesions with daily doses of 30 mg and 45 mg, but not 10 mg, establishing the lowest efficacious dose is 30 mg.

Classification of evidence: This study provides Class II evidence that among adults with active RRMS and ≥1 Gd+ brain lesion in the past 6 months, the cumulative number of active lesions decreased with vidofludimus calcium.

Trial registration information: ClinicalTrials.gov (NCT03846219) and EudraCT (2018-001896-19).

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Immunic to Participate in Investor and Scientific Conferences in February

https://www.prnewswire.com/news-release ... 67302.html

Immunic Presents Key Vidofludimus Calcium Data at the ACTRIMS Forum 2025, Highlighting Its Potential in Multiple Sclerosis

https://imux.com/immunic-presents-key-v ... sclerosis/

NEW YORK, February 26, 2025 – Immunic, Inc. (Nasdaq: IMUX), a biotechnology company developing a clinical pipeline of orally administered, small molecule therapies for chronic inflammatory and autoimmune diseases, today announced the presentation of data on its lead asset, nuclear receptor-related 1 (Nurr1) activator, vidofludimus calcium (IMU-838), in two poster presentations at the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum 2025, taking place from February 27 to March 1, in West Palm Beach, Florida.

“Having two poster presentations on vidofludimus calcium at the prestigious ACTRIMS Forum highlights the unique importance of our drug candidate and its promise as a potential new treatment option for multiple sclerosis (MS),” stated Daniel Vitt, Ph.D., Chief Executive Officer of Immunic. “For the first time, we analyzed the baseline characteristics of patients in subpopulations from our phase 2 CALLIPER trial in progressive multiple sclerosis (PMS), and compared to those from four major PMS trials. The goal of this analysis was to identify how the differences may impact comparability of trial outcomes, including our top-line data readout of the phase 2 CALLIPER trial, expected in April. The CALLIPER data is expected to provide valuable insights into the effects of vidofludimus calcium in a non-active PMS population. We believe any impact of vidofludimus calcium on 24-week confirmed disability worsening in this group would likely primarily reflect its influence on compartmentalized pathology within the central nervous system, expressed clinically as progression independent of relapse activity (PIRA).”

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Here are Immunic's posters at ACTRIMS 2025.

▪︎ Poster Title: Baseline Characteristics Across Major Clinical Trials in Progressive Multiple Sclerosis: Insights from ORATORIO, EXPAND, MS-STAT2, HERCULES, and CALLIPER

Presenting Author: Robert J. Fox, Staff Neurologist, Mellon Center for Multiple Sclerosis, Vice-Chair for Research, Neurological Institute, Cleveland Clinic, Cleveland, Ohio

Poster, Abstract


▪︎ Poster Title: Vidofludimus Calcium Shows a Potential Neuroprotective Function in Multiple Sclerosis through its Activity on Nurr1 in Preclinical Models

Presenting Author: Evelyn Peelen, Ph.D., Head of Research, Immunic

Poster, Abstract

Immunic Presented Key Vidofludimus Calcium Data at the 41st Congress of ECTRIMS, Highlighting Its Potential in Multiple Sclerosis

September 25, 2025

https://imux.com/immunic-presented-key- ... sclerosis/

• Vidofludimus Calcium Demonstrated Statistically Significant 24-Week Confirmed Disability Improvement in Phase 2 CALLIPER Trial in Progressive Multiple Sclerosis, With Over Two-Fold Probability Over Placebo in the Overall Study Population and Consistent Effects Across Subtypes

• CALLIPER Data Showed Positive and Consistent Signals for Slowing Disability Progression Across Disability Endpoints, Patient Populations and Subgroups Without Evidence of Focal Inflammation, Reinforcing the Drug’s Neuroprotective Potential and Promise to Slow Disease Progression

• CALLIPER Data Supports Nurr1 Activation as New Mechanism to Prevent Neurodegeneration in Multiple Sclerosis

• Long-Term Data From Phase 2 EMPhASIS Trial in Relapsing-Remitting Multiple Sclerosis Showed High Rates of Patients Remaining Free of 12- and 24-Week Confirmed Disability Worsening as well as Low Discontinuation Rates and Favorable Long-Term Safety and Tolerability

NEW YORK, September 25, 2025 – Immunic, Inc. (Nasdaq: IMUX), a biotechnology company developing a clinical pipeline of orally administered, small molecule therapies for chronic inflammatory and autoimmune diseases, today announced the presentation of key data in an oral and four poster presentations, including one late-breaking poster, at the 41st Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), being held September 24-26, 2025 in Barcelona, Spain. The data highlight Immunic’s orally available lead asset, nuclear receptor-related 1 (Nurr1) activator, vidofludimus calcium’s (IMU-838) therapeutic potential in multiple sclerosis (MS). The presentation and all posters are accessible on the “Events and Presentations” section of Immunic’s website at: https://ir.imux.com/events-and-presentations.

“Having such a broad presence at the prestigious ECTRIMS Congress is a major achievement for Immunic and underscores both the strength of our clinical and preclinical data and the potential of vidofludimus calcium to transform the oral MS therapy landscape,” stated Daniel Vitt, Ph.D., Chief Executive Officer of Immunic. “We were especially honored to showcase, in an oral presentation, the positive efficacy and safety data from our phase 2 CALLIPER trial in progressive multiple sclerosis (PMS), offering much needed hope for this high unmet medical need patient population.”

Dr. Vitt continued, “The CALLIPER results, also selected for the Best of ECTRIMS 2025 slide deck, highlight vidofludimus calcium’s neuroprotective potential and its promise to slow disease progression in patients with or without focal inflammation. Importantly, the consistent 24-week confirmed disability worsening (24wCDW) results across disability endpoints, patient populations and subgroups, including in patients without evidence of baseline inflammatory gadolinium-enhancing (Gd+) lesions during MRI, were seen both in the overall population and in the primary progressive multiple sclerosis (PPMS) and non-active secondary progressive multiple sclerosis (naSPMS) subgroups. Additionally, newly available data regarding 24-week confirmed disability improvement (24wCDI) showed an over two-fold probability for vidofludimus calcium over placebo, statistically significant in the overall PMS population, with consistent trends across the subtypes. The CALLIPER findings support clinically measurable neuroprotective effects of vidofludimus calcium, consistent with its Nurr1 activation mechanism. We believe that, since 24wCDW is an accepted regulatory endpoint to demonstrate clinical benefit in PMS, this evidence of clinical activity merits further investigation and de-risks a potential phase 3 program.”

“Moreover, additional long-term data from our phase 2 EMPhASIS trial in relapsing-remitting multiple sclerosis (RRMS) has further reinforced the robust efficacy signals and favorable safety and tolerability observed, to date,” concluded Dr. Vitt. “Importantly, our fully enrolled twin phase 3 ENSURE trials in relapsing multiple sclerosis (RMS), for which top-line data is expected by the end of 2026, are designed to evaluate multiple endpoints regarding patient disability. With its unique neuroprotective, anti-inflammatory, and anti-viral profile, along with clean safety and tolerability observed in clinical trials to date, we believe vidofludimus calcium has the potential to become a differentiated oral therapy in the multi-billion-dollar MS market.”

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