Immunic Announces Vidofludimus Calcium Reduced Risk of Disability Worsening by 30% in Primary Progressive Multiple Sclerosis Patients from Phase 2 CALLIPER Trial
Immunic announced positive results from its phase 2 CALLIPER trial of vidofludimus calcium in progressive multiple sclerosis patients. The drug showed a 20% reduction in disability worsening risk across all patients, with an impressive 30% reduction specifically in primary progressive multiple sclerosis (PPMS) patients.
Key findings include a 29% risk reduction in patients without gadolinium-enhancing lesions and a 20% decrease in thalamic brain volume loss compared to placebo. The trial involved 467 patients across North America and Europe, focusing on PPMS and non-active secondary progressive MS patients.
The drug demonstrated a favorable safety profile with no new safety concerns identified. Treatment-related adverse events were similar between drug (69.4%) and placebo (68.5%) groups. The company plans to present detailed results at upcoming scientific meetings, while phase 3 trials in relapsing multiple sclerosis continue with expected completion in 2026.
Immunic ha annunciato risultati positivi dal suo studio di fase 2 CALLIPER sul vidofludimus calcio in pazienti con sclerosi multipla progressiva. Il farmaco ha mostrato una riduzione del 20% del rischio di peggioramento della disabilità in tutti i pazienti, con una riduzione impressionante del 30% specificamente nei pazienti con sclerosi multipla primaria progressiva (PPMS).
I risultati chiave includono una riduzione del rischio del 29% nei pazienti senza lesioni con gadolinio e una diminuzione del 20% della perdita di volume del talamo rispetto al placebo. Lo studio ha coinvolto 467 pazienti in Nord America e Europa, concentrandosi su pazienti con PPMS e sclerosi multipla secondaria progressiva non attiva.
Il farmaco ha dimostrato un profilo di sicurezza favorevole senza nuovi problemi di sicurezza identificati. Gli eventi avversi correlati al trattamento sono stati simili tra il gruppo trattato (69,4%) e il gruppo placebo (68,5%). L'azienda prevede di presentare i risultati dettagliati nei prossimi congressi scientifici, mentre proseguono gli studi di fase 3 sulla sclerosi multipla recidivante, con completamento previsto nel 2026.
Immunic anunció resultados positivos de su ensayo de fase 2 CALLIPER con vidofludimus calcio en pacientes con esclerosis múltiple progresiva. El medicamento mostró una reducción del 20% en el riesgo de empeoramiento de la discapacidad en todos los pacientes, con una impresionante reducción del 30% específicamente en pacientes con esclerosis múltiple primaria progresiva (EMPP).
Los hallazgos clave incluyen una reducción del riesgo del 29% en pacientes sin lesiones con realce de gadolinio y una disminución del 20% en la pérdida de volumen del tálamo en comparación con el placebo. El ensayo involucró a 467 pacientes en Norteamérica y Europa, enfocándose en pacientes con EMPP y esclerosis múltiple secundaria progresiva no activa.
El medicamento demostró un perfil de seguridad favorable sin nuevas preocupaciones de seguridad identificadas. Los eventos adversos relacionados con el tratamiento fueron similares entre el grupo de medicamento (69,4%) y el grupo placebo (68,5%). La compañía planea presentar resultados detallados en próximas reuniones científicas, mientras continúan los ensayos de fase 3 en esclerosis múltiple remitente-recidivante, con finalización prevista para 2026.
Immunic은 진행성 다발성 경화증 환자를 대상으로 한 비도플루디무스 칼슘의 2상 CALLIPER 임상시험에서 긍정적인 결과를 발표했습니다. 이 약물은 모든 환자에서 장애 악화 위험을 20% 감소시켰으며, 특히 원발성 진행성 다발성 경화증(PPMS) 환자에서는 30%의 인상적인 감소를 보였습니다.
주요 결과로는 가돌리늄 증강 병변이 없는 환자에서 위험이 29% 감소했고, 위약 대비 시상(thalamic) 뇌 부피 손실이 20% 감소한 점이 포함됩니다. 이 임상시험은 북미와 유럽에서 467명의 환자를 대상으로 진행되었으며, PPMS 및 비활성 이차 진행성 다발성 경화증 환자에 초점을 맞추었습니다.
약물은 새로운 안전성 문제 없이 우수한 안전성 프로파일을 나타냈습니다. 치료 관련 이상반응 발생률은 약물군(69.4%)과 위약군(68.5%)에서 유사했습니다. 회사는 향후 과학 회의에서 상세 결과를 발표할 계획이며, 재발성 다발성 경화증을 대상으로 한 3상 시험은 2026년 완료될 예정입니다.
Immunic a annoncé des résultats positifs de son essai de phase 2 CALLIPER sur le vidofludimus calcium chez des patients atteints de sclérose en plaques progressive. Le médicament a montré une réduction de 20 % du risque d'aggravation du handicap chez tous les patients, avec une réduction impressionnante de 30 % spécifiquement chez les patients atteints de sclérose en plaques primaire progressive (SPPP).
Les résultats clés incluent une réduction de 29 % du risque chez les patients sans lésions rehaussées au gadolinium et une diminution de 20 % de la perte de volume du thalamus par rapport au placebo. L'essai a impliqué 467 patients en Amérique du Nord et en Europe, se concentrant sur les patients atteints de SPPP et de sclérose en plaques secondaire progressive non active.
Le médicament a démontré un profil de sécurité favorable sans nouvelles préoccupations identifiées. Les événements indésirables liés au traitement étaient similaires entre le groupe médicament (69,4 %) et le groupe placebo (68,5 %). La société prévoit de présenter des résultats détaillés lors de prochaines réunions scientifiques, tandis que les essais de phase 3 sur la sclérose en plaques récurrente se poursuivent, avec une fin prévue en 2026.
Immunic gab positive Ergebnisse seiner Phase-2-Studie CALLIPER mit Vidofludimuscalcium bei Patienten mit progredienter Multipler Sklerose bekannt. Das Medikament zeigte eine 20%ige Reduktion des Risikos für eine Verschlechterung der Behinderung bei allen Patienten, mit einer beeindruckenden 30%igen Reduktion speziell bei Patienten mit primär progredienter Multipler Sklerose (PPMS).
Wesentliche Ergebnisse umfassen eine 29%ige Risikoreduktion bei Patienten ohne gadoliniumaufhellende Läsionen und eine 20%ige Verringerung des thalamischen Hirnvolumenverlusts im Vergleich zu Placebo. Die Studie umfasste 467 Patienten in Nordamerika und Europa, mit Fokus auf PPMS und nicht-aktive sekundär progrediente MS-Patienten.
Das Medikament zeigte ein günstiges Sicherheitsprofil ohne neue Sicherheitsbedenken. Behandlungsbedingte Nebenwirkungen traten ähnlich häufig bei der Wirkstoffgruppe (69,4%) und der Placebogruppe (68,5%) auf. Das Unternehmen plant, detaillierte Ergebnisse auf bevorstehenden wissenschaftlichen Tagungen vorzustellen, während Phase-3-Studien bei schubförmiger Multipler Sklerose fortgesetzt werden, deren Abschluss für 2026 erwartet wird.
- 30% reduction in disability worsening risk for PPMS patients - a significant clinical benefit in high unmet need population
- 20% reduction in disability worsening risk across all progressive MS patients
- 29% reduction in disability worsening for patients without inflammatory lesions - showing effectiveness in harder-to-treat cases
- 20% reduction in thalamic brain volume loss compared to placebo
- Strong safety profile with no new safety signals and similar adverse event rates to placebo
- Potential first-in-class neuroprotective treatment for progressive MS with unique Nurr1 activation mechanism
- Modest 5% improvement in primary MRI endpoint (brain volume change)
- Lower effectiveness in secondary progressive MS patients with only 15% reduction in disability worsening
- Phase 2 results still require confirmation in larger Phase 3 trials before potential approval
Insights
Immunic's phase 2 trial shows promising 30% disability reduction in primary progressive MS, demonstrating potential neuroprotective effects through novel Nurr1 activation mechanism.
The phase 2 CALLIPER trial results for vidofludimus calcium demonstrate significant clinical progress in progressive multiple sclerosis (PMS), particularly in the highest-need population. The drug reduced 24-week confirmed disability worsening events by 20% in the overall study population, with an even stronger 30% reduction in primary progressive MS patients. What's particularly remarkable is the 29% reduction in patients without gadolinium-enhancing lesions at baseline, a population historically unresponsive to current anti-inflammatory therapies.
These results validate vidofludimus calcium's novel mechanism as a Nurr1 activator, suggesting neuroprotective benefits beyond anti-inflammation. The drug also reduced thalamic brain volume loss by 20%, a sensitive marker strongly associated with clinical disability progression in MS. While the primary MRI endpoint (brain volume change) showed modest improvement (
The favorable safety profile with no new signals further strengthens the clinical potential. With disability worsening being the recognized regulatory approval endpoint for registrational studies in progressive MS, these results position vidofludimus calcium as a promising candidate for PMS where treatment options remain extremely limited. The company will now discuss these results with regulators while continuing their ongoing phase 3 trials in relapsing MS.
Vidofludimus calcium shows first true neuroprotective potential in MS through Nurr1 activation, addressing crucial unmet need in progressive forms.
The CALLIPER trial results represent a potential breakthrough for progressive MS patients. The 30% reduction in disability worsening in PPMS is clinically meaningful in a disease form with extremely limited treatment options. Most current MS therapies target inflammation, but progressive MS is driven primarily by neurodegeneration independent of inflammatory activity.
What makes vidofludimus calcium unique is its mechanism as a Nurr1 activator, which plays a key role in neuroprotection. The drug demonstrated efficacy in patients without inflammatory lesions (29% reduction in disability worsening), suggesting it addresses the neurodegenerative component that current therapies fail to target. This is further supported by the 20% reduction in thalamic atrophy - a critical brain region whose volume loss correlates strongly with disability progression.
The low prevalence of inflammatory activity in the study population (
For progressive MS patients who currently have minimal options, these results suggest vidofludimus calcium could potentially become the first therapy directly targeting the neurodegenerative processes driving disability. The favorable safety profile further supports its potential as a long-term treatment option in this difficult-to-treat population.
– Reduced Relative Risk of 24-Week Confirmed Disability Worsening Events by
– Showed Consistent Reduction of Disability Worsening in Subpopulations Without Inflammatory Lesions at Baseline in Overall Study Population; Reduced Relative Risk of 24-Week Confirmed Disability Worsening Events in Patients Without Gadolinium-Enhancing Lesions at Baseline by
– Reduced Annualized Rate of Thalamic Brain Volume Loss by
– Confirmed Favorable Safety and Tolerability Observed in Previous Clinical Trials; No New Safety Signals Identified –
– Webcast to be Held Today, April 30, at 8:00 am ET –
Clinical Endpoints
In the overall PMS patient population (n=467), vidofludimus calcium reduced the relative risk of 24-week confirmed disability worsening (24wCDW) events based on changes in the expanded disability status scale (EDSS) by
Immunic believes that these reductions in 24wCDW events are remarkable in light of the overwhelming non-activity of the CALLIPER population. In support, the evidence of focal inflammatory disease (gadolinium-enhancing lesions at baseline in
A consistent reduction of disability worsening was observed in the different subpopulations with or without inflammatory gadolinium-enhanced lesion activity at baseline and during the study. Vidofludimus calcium reduced the relative risk of 24wCDW events in patients without gadolinium-enhancing lesions at baseline by
Magnetic Resonance Imaging (MRI) Endpoints
While vidofludimus calcium had a modest benefit on the exploratory primary MRI endpoint (annualized rate of percent brain volume change:
The total volume of new or enlarging T2 lesions showed a substantial difference between vidofludimus calcium and placebo over time, with vidofludimus calcium decreasing and placebo increasing (mean percent change,
"The tremendous reduction of confirmed disability worsening in PMS patients is a wonderful confirmation of the objectives of this exploratory phase 2 trial. CALLIPER was designed to evaluate the clinical efficacy, safety and tolerability of vidofludimus calcium in a broad set of PMS patients to determine the suitability of advancing to a confirmatory phase 3 program," said Daniel Vitt, Ph.D., Chief Executive Officer of Immunic. "We are particularly thrilled to see such a clinically meaningful effect in the PPMS population, with a
"The most significant unmet need in MS continues to be the lack of safe and effective therapies that can slow or halt disease progression, especially in PPMS and non-active SPMS. Vidofludimus calcium is the only medicine in development for MS that has been shown to be a potent activator of Nurr1, which plays a key role in neuroprotection. With this unique mode of action, vidofludimus calcium could become the first real neuroprotective treatment option for patients with progressive forms of MS," added Andreas Muehler, M.D., M.B.A., Chief Medical Officer of Immunic. "In particular, the
Safety and Tolerability
The top-line CALLIPER data set confirmed the favorable safety and tolerability profile of vidofludimus calcium already observed in previous clinical trials. No new safety signals were identified. The occurrence of treatment-emergent adverse events and serious adverse events showed a similar frequency between both treatment arms. The rate of treatment-emergent adverse events was
About CALLIPER
CALLIPER is an international, multicenter, randomized, double-blind, placebo-controlled, exploratory phase 2 trial which enrolled 467 patients at more than 70 sites throughout
Analysis of the full CALLIPER data set is ongoing and will be presented at upcoming scientific meetings. The company's phase 3 clinical trial program of vidofludimus calcium in relapsing multiple sclerosis is ongoing and expected to be completed in 2026.
Webcast Information
Immunic will host a webcast today at 8:00 am ET to discuss these results. To participate in the webcast, please register in advance at: https://imux.zoom.us/webinar/register/WN_tzX8otsFRAawwEmjwgCjeA or on the "Events and Presentations" section of Immunic's website at: ir.imux.com/events-and-presentations. Registrants will receive a confirmation email containing a link for online participation or a telephone number for dial in access.
An archived replay of the webcast will be available approximately one hour after completion on Immunic's website at: ir.imux.com/events-and-presentations.
About Progressive Multiple Sclerosis
While multiple sclerosis (MS) in general is an autoimmune disease characterized by immune-mediated demyelination that affects the brain, spinal cord and optic nerve, the progressive phase of the disease seems to be dominated by significant neurodegenerative mechanisms. Progressive multiple sclerosis (PMS) is a clinical form of MS characterized by gradual accrual of disability independent of relapses over time. PMS includes both primary progressive MS (PPMS) and secondary progressive MS (SPMS). PPMS is characterized by steadily worsening neurologic function from the onset of symptoms without initial relapse or remissions. SPMS is identified following an initial relapsing-remitting course, after which the disease becomes more steadily progressive, with (active SPMS) or without (non-active SPMS) MRI lesions and/or relapses present.
About Vidofludimus Calcium (IMU-838)
Vidofludimus calcium is an orally administered investigational small molecule drug being developed for chronic inflammatory and autoimmune diseases, currently in late-stage clinical trials for multiple sclerosis (MS). Uniquely, vidofludimus calcium's first-in-class, dual mode of action combines neuroprotective, anti-inflammatory and anti-viral effects to target the complex pathophysiology of MS. As a selective immune modulator, it activates the neuroprotective transcription factor, nuclear receptor-related 1 (Nurr1), which provides direct and indirect neuroprotective effects. Additionally, vidofludimus calcium achieves anti-inflammatory and anti-viral effects through highly selective inhibition of the enzyme dihydroorotate dehydrogenase (DHODH). Vidofludimus calcium is currently being evaluated in phase 3 and phase 2 clinical trials for the treatment of relapsing and progressive MS, respectively. In a phase 2 clinical trial, it has shown therapeutic activity in patients suffering from relapsing-remitting MS, significantly reducing MRI lesions and demonstrating encouraging results in reducing confirmed disability worsening. Additionally, vidofludimus calcium has demonstrated clinical benefits in progressive MS patients by showing substantial reductions in the relative risks of 24-week confirmed disability progression events and in thalamic brain volume in a phase 2 clinical trial. To date, vidofludimus calcium has been exposed to approximately 2,700 individuals and has shown an attractive pharmacokinetic, safety and tolerability profile. Vidofludimus calcium is not yet licensed or approved in any country.
About Immunic, Inc.
Immunic, Inc. (Nasdaq: IMUX) is a biotechnology company developing a clinical pipeline of orally administered, small molecule therapies for chronic inflammatory and autoimmune diseases. The company's lead development program, vidofludimus calcium (IMU-838), is currently in phase 3 clinical trials for the treatment of relapsing multiple sclerosis, which are expected to be completed in 2026. It has already shown therapeutic activity in phase 2 clinical trials in patients suffering from relapsing-remitting multiple sclerosis and progressive multiple sclerosis. Vidofludimus calcium combines neuroprotective effects, through its mechanism as a first-in-class nuclear receptor related 1 (Nurr1) activator, with additional anti-inflammatory and anti-viral effects, by selectively inhibiting the enzyme dihydroorotate dehydrogenase (DHODH). IMU-856, which targets the protein Sirtuin 6 (SIRT6), is intended to restore intestinal barrier function and regenerate bowel epithelium, which could potentially be applicable in numerous gastrointestinal diseases, such as celiac disease as well as inflammatory bowel disease, Graft-versus-Host-Disease and weight management. IMU-381, which currently is in preclinical testing, is a next generation molecule being developed to specifically address the needs of gastrointestinal diseases. For further information, please visit: www.imux.com.
Cautionary Statement Regarding Forward-Looking Statements
This press release contains "forward-looking statements" that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, future financial position, future revenue, projected expenses, sufficiency of cash and cash runway, expected timing, development and results of clinical trials, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to Immunic's development programs and the targeted diseases; the potential for vidofludimus calcium to safely and effectively target diseases; preclinical and clinical data for vidofludimus calcium; the feasibility of advancing vidofludimus calcium to a confirmatory phase 3 clinical trial in progressive multiple sclerosis; the timing of current and future clinical trials and anticipated clinical milestones; the nature, strategy and focus of the company and further updates with respect thereto; and the development and commercial potential of any product candidates of the company. Immunic may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management's current expectations and involve substantial risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, increasing inflation, tariffs and macroeconomics trends, impacts of the
Contact Information
Immunic, Inc.
Jessica Breu
Vice President Investor Relations and Communications
+49 89 2080 477 09
jessica.breu@imux.com
US IR Contact
Rx Communications Group
Paula Schwartz
+1 917 633 7790
immunic@rxir.com
US Media Contact
KCSA Strategic Communications
Caitlin Kasunich
+1 212 896 1241
ckasunich@kcsa.com
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SOURCE Immunic, Inc.
FAQ
What were the results of IMUX's Phase 2 CALLIPER trial for multiple sclerosis in 2025?
How effective is IMUX's vidofludimus calcium in treating PPMS patients without inflammatory lesions?
What safety concerns were reported in IMUX's CALLIPER trial for MS treatment?
When will IMUX complete its Phase 3 trials for multiple sclerosis treatment?
How does IMUX's vidofludimus calcium work differently from other MS treatments?
