A Multi-Center, Randomized, Double-Blinded Phase 3 Study to Evaluate the Efficacy, Safety, and Tolerability of IMU-838 versus Placebo in Adults with Relapsing Multiple Sclerosis (ENSURE-1) - ENSURE 1

开始日期2023-06-01

申办/合作机构

Immunic AG

CTRI/2022/04/041596 / Recruiting临床3期

A Multi-Center, Randomized, Double-Blinded Phase 3 Study to Evaluate the Efficacy, Safety, and Tolerability of IMU-838 versus Placebo in Adults with Relapsing Multiple Sclerosis (ENSURE-2) - nil

开始日期2022-05-25

申办/合作机构

Immunic AG

CTRI/2022/02/040605 / Recruiting临床3期

A Multi-Center, Randomized, Double-Blinded Phase 3 Study to Evaluate the Efficacy, Safety, and Tolerability of IMU-838 versus Placebo in Adults with Relapsing Multiple Sclerosis (ENSURE 1)

开始日期2022-04-18

申办/合作机构

Immunic AG

100 项与 Vidofludimus calcium 相关的临床结果

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100 项与 Vidofludimus calcium 相关的转化医学

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100 项与 Vidofludimus calcium 相关的专利（医药）

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项与 Vidofludimus calcium 相关的文献（医药）

2024-10-01·ChemMedChem

Synthesis and Characterization of DHODH Inhibitors Based on the Vidofludimus Scaffold with Pronounced Anti‐SARS‐CoV‐2 Activity

Article

作者: Kohlhof, Hella ; Issmail, Leila ; Herrmann, Alexandra ; Uhlig, Nadja ; Häge, Sigrun ; Grunwald, Thomas ; Klopfleisch, Robert ; Eberlein, Valentina ; Vitt, Daniel ; Marschall, Manfred ; Wangen, Christina ; Hahn, Friedrich ; Gege, Christian

AbstractNew strategies for the rapid development of broad‐spectrum antiviral therapies are urgently required for emerging and re‐emerging viruses like the severe acute respiratory syndrome coronavirus 2 (SARS‐CoV‐2). Host‐directed antivirals that target universal cellular metabolic pathways necessary for viral replication present a promising approach with broad‐spectrum activity and low potential for development of viral resistance. Dihydroorotate dehydrogenase (DHODH) was identified as one of those universal host factors essential for the replication of many clinically relevant human pathogenic viruses. DHODH is the rate‐limiting enzyme catalyzing the fourth step in the de novo pyrimidine synthesis. Therefore, it is also developed as a therapeutic target for many diseases relying on cellular pyrimidine resources, such as cancer, autoimmune diseases and viral or bacterial infection. Thus, several DHODH inhibitors, including vidofludimus calcium (VidoCa, IMU‐838), are currently in development or have been investigated in clinical trials for the treatment of virus infections such as SARS‐CoV‐2‐mediated coronavirus disease 19 (COVID‐19). Here, we report the medicinal chemistry optimization of VidoCa that resulted in metabolically more stable derivatives with improved DHODH target inhibition in various mammalian species, which translated into improved efficacy against SARS‐CoV‐2.

2024-05-01·Journal of Neuroimmunology

Recent advances in the treatment of primary and secondary progressive Multiple Sclerosis

Review

作者: Jaiswal, Shruti ; Amatya, Suban ; Sriwastava, Shitiz ; Bhatia, Dipika ; Elkhooly, Mahmoud ; Lisak, Robert P ; Shrestha, Kriti ; Kagzi, Yusuf ; Gupta, Rajesh

BACKGROUNDThe article highlights upcoming potential treatments, which target different phases of inflammation and offer remyelinating strategies as well as direct and indirect neuroprotective and oligodendrocyte protective effects, providing a hopeful outlook for patients with primary and secondary progressive multiple sclerosis (PPMS and SPMS).OBJECTIVESThe review aims to identify potential treatments and ongoing clinical trials for PPMS and SPMS, and compare their mechanisms of action, efficacy, and side effects with current treatments.METHODSWe reviewed ongoing clinical trials for PPMS and SPMS on the NIH website, as well as articles from PubMed, Embase, and clinicaltrails.gov since 2010.RESULTSBTKIs like, tolebrutinib, and fenebrutinib are being explored as potential PMS treatments. Vidofludimus calcium, an orally available treatment, has shown a reduction of active and new MRI lesions. Other treatments like simvastatin, N-acetylcysteine (NAC), and alpha-lipoic acid are being explored for their antioxidant properties. AHSCT and mesenchymal stem cell therapy are experimental options for younger patients with high inflammatory activity.CONCLUSIONSSPMS and PPMS are being studied for new treatments and future trials should consider combination therapies targeting inflammation, demyelination, and neuronal death, as the pathogenesis of PMS involves complex factors.

2024-05-01·Neurology Neuroimmunology & Neuroinflammation

Safety and Dose-Response of Vidofludimus Calcium in Relapsing Multiple Sclerosis

Article

作者: De Stefano, Nicola ; Gryb, Viktoriia ; Kohlhof, Hella ; Sciacca, Valentina ; Fox, Robert J ; Vitt, Daniel ; Wolf, Christian ; Sellner, Johann ; Slizgi, Jason ; Ondrus, Matej ; Bozhinov, Plamen S ; Wiendl, Heinz ; Rejdak, Konrad ; Muehler, Andreas R

BACKGROUND AND OBJECTIVESVidofludimus 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.METHODSIn 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.RESULTSAcross 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.DISCUSSIONCompared 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 EVIDENCEThis 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 INFORMATIONClinicalTrials.gov (NCT03846219) and EudraCT (2018-001896-19).

项与 Vidofludimus calcium 相关的新闻（医药）

2024-11-13

·PRNewswire

75+ Key Companies Charting New Frontiers in Multiple Sclerosis Therapeutic Space | DelveInsight

Multiple sclerosis (MS) is a chronic neurological disorder affecting the central nervous system (CNS), particularly the brain, spinal cord, and optic nerves. Continuous research has led to new drug developments, particularly disease-modifying therapies (DMTs) that slow MS progression. Monoclonal antibodies, oral therapies, and infusion treatments are among the most promising options and drives the multiple sclerosis market. LAS VEGAS, Nov. 13, 2024 /PRNewswire/ -- DelveInsight's ' Multiple Sclerosis Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline multiple sclerosis therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the multiple sclerosis pipeline domain. Key Takeaways from the Multiple Sclerosis Pipeline Report DelveInsight's multiple sclerosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for multiple sclerosis treatment. Key multiple sclerosis companies such as Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Promising multiple sclerosis pipeline therapies such as GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. In October 2024, Immunic, Inc. reported favorable results from the non-binding interim futility analysis of its Phase 3 ENSURE trial, which is evaluating its lead candidate, vidofludimus calcium (IMU-838), a nuclear receptor-related 1 (Nurr1) activator, for the treatment of relapsing multiple sclerosis (RMS). In October 2024, Immunic, Inc. announced a positive outcome of the non-binding, interim futility analysis of its Phase III ENSURE program, investigating lead asset, nuclear receptor related 1 (Nurr1) activator, vidofludimus calcium (IMU-838), for the treatment of relapsing multiple sclerosis (RMS). Based on the outcome of the interim futility analysis, an unblinded Independent Data Monitoring Committee (IDMC) has recommended that the trials are not futile and should continue as planned. In September 2024, Roche, secured FDA approval for an injectable form of its blockbuster multiple sclerosis treatment, Ocrevus, reducing patient treatment time and potentially helping the company stay ahead of increasing competition. In September 2024, Sanofi announced the outcomes of Phase III clinical trials of its investigational oral brain-penetrant BTK inhibitor, tolebrutinib, aimed at treating multiple sclerosis (MS). These studies, namely HERCULES, GEMINI 1, and GEMINI 2, have provided mixed results, with the HERCULES trial meeting its primary endpoint while the GEMINI trials did not. The HERCULES study focused on the efficacy and safety of tolebrutinib in individuals with non-relapsing secondary progressive MS (nrSPMS) compared to a placebo. In August 2024, Abata Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ABA-101 for the treatment of patients with progressive multiple sclerosis (MS). The FDA recently cleared ABA-101's Investigational New Drug (IND) application, and initiation of a first-in-human (FIH) Phase I study is imminent. In August 2024, TG Therapeutics announced that the FDA has cleared an investigational new drug (IND) application submitted by TG Therapeutics for azercabtagene zapreleucel (azer-cel), an investigational allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, for a phase 1 clinical trial in patients with progressive multiple sclerosis (MS). In January 2024, Kyverna Therapeutics, Inc. announced it received fast-track designation by the U.S. Food and Drug Administration (FDA) for its autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate, KYV-101, to be used for the treatment of multiple sclerosis (MS). Request a sample and discover the recent advances in multiple sclerosis treatment drugs @ Multiple Sclerosis Pipeline Report The multiple sclerosis pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage multiple sclerosis drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the multiple sclerosis clinical trial landscape. Multiple Sclerosis Overview Multiple sclerosis is a chronic autoimmune disorder that affects the central nervous system, leading to the deterioration or permanent damage of myelin, the protective sheath surrounding nerve fibers. While the exact cause of multiple sclerosis remains unclear, it is believed to involve a combination of genetic predisposition, environmental factors, and possibly viral infections. Common symptoms of multiple sclerosis include fatigue, difficulty walking, numbness or tingling in the limbs, muscle weakness, and vision problems, which can vary significantly among individuals and may evolve over time. Diagnosis typically involves a combination of neurological examinations, magnetic resonance imaging (MRI) to detect lesions in the brain and spinal cord, and sometimes lumbar puncture to analyze cerebrospinal fluid for inflammatory markers. While there is no cure for multiple sclerosis, treatment options aim to manage symptoms, reduce the frequency and severity of relapses, and slow disease progression. This may include disease-modifying therapies (DMTs), corticosteroids for acute exacerbations, and symptomatic treatments such as physical therapy, pain management, and lifestyle modifications. Early diagnosis and a tailored treatment plan are crucial for improving the quality of life for those living with multiple sclerosis. Find out more about multiple sclerosis treatment drugs @ Drugs for Multiple Sclerosis Treatment A snapshot of the Multiple Sclerosis Pipeline Drugs mentioned in the report: Learn more about the emerging multiple sclerosis pipeline therapies @ Multiple Sclerosis Clinical Trials Multiple Sclerosis Therapeutics Assessment The multiple sclerosis pipeline report proffers an integral view of the multiple sclerosis emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Multiple Sclerosis Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Immunomodulators, MHC class II gene modulators, Agammaglobulinaemia tyrosine kinase inhibitors, Dihydroorotate dehydrogenase inhibitors, Nuclear receptor subfamily 4 group A member 2 agonists, CD20 antigen inhibitors, Energy metabolism stimulants; Immunomodulators, Lysine-specific demethylase 1 inhibitors, Monoamine oxidase B inhibitors, Protein-arginine deiminase inhibitors, Immunostimulants Key Multiple Sclerosis Companies: Mapi Pharma, Novartis, Immunic, Biocad, Apimeds, Genentech (Roche), AB Science, Celltrion, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, BrainStorm Cell Therapeutics, NeuroSense Therapeutics, Denali Therapeutics Inc., Sanofi, MediciNova, Biogen, Tiziana Life Sciences, Antisense Therapeutics, RemeGen, Atara Biotherapeutics, Contineum Therapeutics, Eli Lilly and Company, Axsome Therapeutics, Accure therapeutics, Stem Cell Medicine Ltd., Ever Supreme Bio-Technology Co., Ltd., ImCyse, Lucid Psycheceuticals, Immune Response BioPharma, Guangzhou Lupeng Pharmaceutical, Kyowa Kirin, Nura Bio, Bristol-Myers Squibb, ImStem Biotechnology, Hoffmann-La Roche, LAPIX Therapeutics, Motric Bio, Immunophage Biotech, NervGen, FibroBiologics, Kyverna Therapeutics, GlaxoSmithKline, PolTREG, Cyxone, Biohaven Pharmaceuticals, HuniLife Biotechnology, GlobeStar Therapeutics, Anokion, Medsenic, ZyVersa Therapeutics, Alumis Inc., Axoltis Pharma, Barricade Therapeutics, BioNxt Solutions, Eikonoklastes Therapeutics, 4D Pharma, Gossamer Bio, Pasithea Therapeutics, Synaptogenix, Monte Rosa Therapeutics, Immix Biopharma, f5 Therapeutics, Autobahn Therapeutics, Biosenic, Polpharma Biologics, Find Therapeutics, TeraImmun, AptaTargets, Trethera, Aditxt, SetPoint Medical, Sarepta Therapeutics, Nucleome Therapeutics, Vaccinex Inc., and others are evaluating new multiple sclerosis drugs to improve the treatment landscape. Key Multiple Sclerosis Pipeline Therapies:GA Depot, Remibrutinib, Tolebrutinib, IMU 838, BCD-132, Apitox, Fenebrutinib, SAR 441344, CT-P53, CNM-Au8, Orelabrutinib, Vafidemstat, Temelimab, Elezanumab, NurOwn (MSC-NTF cells), PrimeC, SAR4433820, Ibudilast, BIIB091, Foralumab, ATL1102, RC18, ATA188, PIPE-307, Pirtobrutinib, LY3541860, Solriamfetol, Act-01, SCM-010, UMSC01, IMCY-0141, Lucid-MS, NeuroVax, LP-168, NB-4746, CC-97540, IMS-001, RO7121932, BMS-986196, LPX-TI641, MTR-601, IPG-11406, IPG-1094, NVG-291, CYMS-101, KYV-101, PIPE-791, GSK 3888130B, PTG-007, T 20K, BHV-8000, HuL001, Amethyst™, ANK 700, Arscimed, A-005, NX210c, Research programme: CAR-TREG, TASIN-1, Cladribine ODF, ET-101, MRx0002, GB7208, PAS-002, Bryostatin-1, MRT-6160, NXC-201, Research Program: Cytokine reduction, ABX-002, Arsenic trioxide, LL-341070, PB018, FTX 101, TI-235, TRE 515, NT-0007, NT-0002, NVG300, and others are under different phases of multiple sclerosis clinical trials. Dive deep into rich insights for new drugs for multiple sclerosis treatment, visit @ Multiple Sclerosis Drugs Table of Contents For further information on the multiple sclerosis pipeline therapeutics, reach out @ Multiple Sclerosis Treatment Drugs Related Reports Multiple Sclerosis Market Multiple Sclerosis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key multiple sclerosis companies including Immune Response BioPharma, Inc., Clene Nanomedicine, Genzyme, ImStem Biotechnology, Rho, Inc., Bristol-Myers Squibb, TG Therapeutics, Inc., Hoffmann-La Roche, Atara Biotherapeutics, Immunic AG, Celgene, Anokion SA, Sanofi, Actelion, Biogen, Supernus Pharmaceuticals, Inc., GeNeuro SA, HuniLife Biotechnology, Inc., Emerald Health Pharmaceuticals, RemeGen Co., Ltd., Antisense Therapeutics, Biocad, AB Science, Genentech, Novartis, Pipeline Therapeutics, ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, Mapi Pharma, f5 Therapeutics, Autobahn Therapeutics, AstraZeneca, ZyVersa Therapeutics, RedHill Biopharma, Gossamer Bio, Sarepta Therapeutics, BrainStorm Cell Limited, CytoDyn, Pear Therapeutics, Solstice Neurosciences, ProJenX, among others. Multiple Sclerosis Epidemiology Forecast Multiple Sclerosis Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted multiple sclerosis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Relapsing Multiple Sclerosis Pipeline Relapsing Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key relapsing multiple sclerosis companies, including ASLAN Pharmaceuticals, GlaxoSmithKline, Lucid Psycheceuticals, Voronoi, Hanmi Pharmaceutical, among others. Relapsing-Remitting Multiple Sclerosis Pipeline Relapsing-Remitting Multiple Sclerosis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RRMS companies, including TG Therapeutics, Novartis, Sanofi, Cinnagen, Polpharma Biologics, Immunic, Mapi Pharma, Biocad, Apimeds, Genentech (Roche), Merck, Immune Response BioPharma, Clene Nanomedicine, InnoCare Pharma, Oryzon Genomics, GeNeuro SA, AbbVie, Emerald Health Pharmaceuticals, GlaxoSmithKline, RedHill Biopharma, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床3期临床结果快速通道细胞疗法免疫疗法

2024-11-13

·PRNewswire

Immunic Announces Publication of Data From Phase 1/1b Clinical Trial of IMU-856 in the Peer Reviewed Journal, The Lancet Gastroenterology & Hepatology

– Includes Phase 1 Data in Healthy Human Subjects and Phase 1b Data in Celiac Disease Patients – NEW YORK, Nov. 13, 2024 /PRNewswire/ -- 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 that the data from its phase 1/1b clinical trial of IMU-856, an orally available and systemically acting small molecule modulator that targets SIRT6 (Sirtuin 6), has been published in the peer reviewed journal, The Lancet Gastroenterology & Hepatology. Lead authored by Dr. A. James Daveson, Gastroenterologist, Wesley Research Institute and Coral Sea Clinical Research Institute, Queensland, Australia, the paper is entitled, "Safety, clinical activity, pharmacodynamics, and pharmacokinetics of IMU-856, a SIRT6 modulator, in coeliac disease: a first-in-human, randomised, double-blind, placebo-controlled, phase 1 trial." It can be accessed through the following link: (24)00248-6/fulltext. Dr. Daveson stated, "Celiac disease affects approximately 1.4% of the world's population. The only current treatment option is a strict, lifelong gluten-free diet, which poses significant challenges due to dietary and social restrictions and the risk of cross-contamination, which leads to persistent intestinal inflammation with villous atrophy in many patients. IMU-856's potential ability to improve the integrity and function of the intestinal barrier represents a promising, novel approach to treat this condition. Importantly, this phase 1b clinical trial is the first study to show that IMU-856 can mitigate the gluten-related effects in celiac disease patients. Based on this result, in conjunction with the drug's favorable safety and tolerability profile, we have concluded that it warrants further clinical investigation." "The publication of our phase 1/1b clinical data in healthy human subjects and patients with celiac disease, in such a prestigious peer reviewed journal, confirms that IMU-856's novel mechanism modulating SIRT6, a protein which serves as a transcriptional regulator of intestinal barrier function and physiological regeneration of bowel epithelium, can represent an entirely new approach to treating gastrointestinal diseases," added Daniel Vitt, Ph.D., Chief Executive Officer of Immunic. "In our phase 1b clinical trial, IMU-856 showed the first clinical signals of its potential ability to restore a healthy gut by renewal of the gut wall, demonstrating meaningful improvements over placebo in four key dimensions of celiac disease pathophysiology: histology, disease symptoms, biomarkers and nutrient absorption. Together with a favorable safety and tolerability profile, it may set the stage for a potential first-in-class, oral celiac disease therapy. Additionally, we believe that this data provides initial clinical proof-of-concept for a potentially new, oral therapeutic approach to a range of gastrointestinal diseases with high unmet needs, beyond celiac disease." The first two portions of the phase 1 clinical trial, Parts A and B, were single ascending dose and multiple ascending dose, double-blind, placebo-controlled studies in a total of 71 healthy human subjects. Single and multiple ascending doses of IMU-856 were found to be safe and well-tolerated, with no investigational medicinal product (IMP)-related serious or severe treatment-emergent adverse events. No maximum tolerated dose was reached in either part. Part C was structured as a double-blind, randomized, placebo-controlled trial, designed to assess the safety and tolerability of IMU-856 in patients with celiac disease during periods of gluten-free diet and a 15-day gluten challenge with 6 g of gluten given daily. Further objectives included pharmacokinetics and initial clinical activity assessments, including malabsorption parameters, biomarkers for enterocyte functional mass, such as citrulline, disease-related symptoms as well as histological changes. The trial was conducted at sites in Australia and New Zealand. A total of 43 patients were enrolled in two consecutive cohorts with 80 mg or 160 mg of IMU-856 or placebo given once-daily over 28 days. The data demonstrated positive effects for IMU-856 over placebo in four key dimensions of celiac disease pathophysiology: protection of the gut architecture, improvement of patients' symptoms, biomarker response, and enhancement of nutrient absorption. IMU-856 was also observed to be safe and well-tolerated in this trial. There were no IMP-related serious or severe treatment-emergent adverse events, nor was there any dose-dependency in adverse events. Moreover, the rates of treatment-emergent adverse events in non-disease-related parameters were comparable between the active treatment groups and placebo. About IMU-856 IMU-856 is an orally available and systemically acting small molecule modulator that targets SIRT6 (Sirtuin 6), a protein which serves as a transcriptional regulator of intestinal barrier function and regeneration of bowel epithelium. Based on preclinical data, the compound may represent a unique treatment approach, as the mechanism of action targets the restoration of the intestinal barrier function and bowel wall architecture in patients suffering from gastrointestinal diseases such as celiac disease, inflammatory bowel disease and other intestinal barrier function associated diseases. Based on preclinical investigations demonstrating no suppression of immune cells, IMU-856 may have the potential to maintain immune surveillance for patients during therapy, which would be an important advantage versus immunosuppressive medications. IMU-856 demonstrated positive results in a phase 1b clinical trial in celiac disease patients in four key dimensions of the disease's pathophysiology: histology, disease symptoms, biomarkers and nutrient absorption. Currently, the company is preparing for phase 2 clinical testing. IMU-856 is an investigational drug product that has not been approved in any jurisdiction. 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 and phase 2 clinical trials for the treatment of relapsing and progressive multiple sclerosis, respectively, and has shown therapeutic activity in phase 2 clinical trials in patients suffering from relapsing-remitting multiple sclerosis, progressive multiple sclerosis and moderate-to-severe ulcerative colitis. 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, for which it is currently in preparations for a phase 2 clinical trial. 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: . 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 IMU-856 to safely and effectively target diseases; preclinical and clinical data for IMU-856; 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, the COVID-19 pandemic, increasing inflation, impacts of the Ukraine – Russia conflict and the conflict in the Middle East on planned and ongoing clinical trials, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient financial and other resources to meet business objectives and operational requirements, including the ability to satisfy the minimum average price and trading volume conditions required to receive funding in tranche 2 and 3 of the January 2024 private placement, the fact that the results of earlier preclinical studies and clinical trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by Immunic's intellectual property, risks related to the drug development and the regulatory approval process and the impact of competitive products and technological changes. A further list and descriptions of these risks, uncertainties and other factors can be found in the section captioned "Risk Factors," in the company's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, filed with the SEC on February 22, 2024, and in the company's subsequent filings with the Securities and Exchange Commission. Copies of these filings are available online at or ir.imux.com/sec-filings. Any forward-looking statement made in this release speaks only as of the date of this release. Immunic disclaims any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. Immunic expressly disclaims all liability in respect to actions taken or not taken based on any or all the contents of this press release. Contact Information Immunic, Inc. Jessica Breu Vice President Investor Relations and Communications +49 89 2080 477 09 [email protected] US IR Contact Rx Communications Group Paula Schwartz +1 917 633 7790 [email protected] US Media Contact KCSA Strategic Communications Caitlin Kasunich +1 212 896 1241 [email protected] SOURCE Immunic, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床1期临床结果临床2期

2024-11-09

·药事纵横

6家专注于多发性硬化症治疗的biotech

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。多发性硬化症是中枢神经系统（CNS）的一种慢性疾病。作为一种不可预测的疾病，对人的影响不同，一些多发性硬化症患者可能只有轻微的症状，而另一些患者可能会在大脑与身体其他部位之间的交流中断时失去清晰地看、写、说或走路的能力。以下是六家专注于多发性硬化症的生物技术公司，几家公司和研究人员试图通过不同的方法来治疗多发性硬化症，比如BTK抑制剂、CAR-T疗法、双特异性抗体，甚至干细胞疗法。 Biogen 主要多发性硬化候选药物：BIIB091 Biogen以其在神经科学领域的开创性工作而闻名。40多年来，它一直在发现和开发中枢神经系统疾病的治疗方法，包括多发性硬化、肌萎缩侧索硬化（ALS）、阿尔茨海默病、抑郁症、狼疮和脊髓性肌萎缩（SMA）的治疗方法。至于多发性硬化症，该公司已经有几种已获批准的治疗方法，包括Avonex、Plegridy和Vumerity，这些药物都用于治疗多发性硬化症的复发。此外，在其现有的研发过程中，百健正致力于开发下一代伯顿酪氨酸激酶（BTK）抑制剂，称为BIIB091，用于复发性多发性硬化成人患者。目前正处于开发的第二阶段。 BTK是一种非受体酪氨酸激酶，可调节被认为与多发性硬化发病机制有关的B细胞和髓样细胞的发育和信号传导。BIIB091除对B细胞产生作用外，还对髓系细胞中的BTK产生可逆性抑制作用，因此预计BIIB091与仅B细胞疗法相比具有更多的临床益处，并且可以解决共价BTK抑制剂中观察到的耐药性。 Immunic Therapeutics 主要多发性硬化候选药物：Vidofludimus calcium immunictherapeutics正致力于慢性炎症性和自身免疫性疾病的小分子疗法的开发。其主要候选药物是vidofludimus calcium（IMU-838），目前正在开发用于治疗多发性硬化。 IMU-838通过有效激活称为核受体相关1 （Nurr1）的神经保护转录因子发挥作用，Nurr1与直接的神经保护特性相关，并可能增强患者的潜在获益。IMU-838还抑制二氢乳清酸脱氢酶（DHODH），后者是过度活跃的免疫细胞和病毒感染细胞代谢的关键酶。这一双重作用不仅针对炎症和抗病毒过程，而且还解决了多发性硬化潜在的神经退行性方面，使其从现有疗法中脱颖而出，现有疗法虽然能够控制多发性硬化的复发，但不能阻止患者的残疾随着时间的推移而恶化。数项正在进行的多发性硬化试验正在对IMU-838进行测试，包括复发性多发性硬化的两项3期试验和进展性多发性硬化的支持性2期试验。2023年10月，Immunic报告了2期试验的阳性中期数据，因为有生物标志物证据表明，IMU-838的活性超出了之前观察到的抗炎作用。最终，这进一步加强了它的神经保护潜力。此外，在2024年1月，Immunic宣布了高达2.4亿美元的私募。 Kyverna Therapeutics 主要多发性硬化候选药物：KVY-101 Kyverna Therapeutics公司正致力于开发针对b细胞驱动的自身免疫性疾病患者的细胞疗法。这家多发性硬化症公司拥有靶向CD19的自体和异体CAR化合物，该分子在B淋巴细胞的发育、激活和分化中起着重要作用，旨在减少细胞因子释放，消耗B细胞，提高临床耐受性。其主要候选药物KYV-101目前正在一项2期试验中评估用于治疗多发性硬化以及重症肌无力。今年1月，它被美国食品药品监督管理局（FDA）授予快速通道认定，用于治疗难治性进行性多发性硬化患者。此外，Kyverna在2024年2月通过首次公开发行（IPO）获得了.66亿美元的总收益。 Progentos Therapeutics 几个月前，Progentos Therapeutics才成立，获得了6500万美元的A轮融资，用于推进多发性硬化和其他退行性疾病的治疗。在多发性硬化症等疾病中，残疾是由支持轴突功能和生存的脱髓鞘（即髓鞘受损）引起。在这里，免疫系统攻击髓鞘，导致炎症和神经细胞功能受损。正因为如此，Progentos正在开发一种新型小分子，旨在诱导多发性硬化影响的轴突的髓鞘再生。该公司从麻省理工学院（MIT）的子公司Frequency Therapeutics收购了多发性硬化症项目，后者于2023年与RNA编辑公司Korro Bio进行了反向合并。根据Progentos的说法，当涉及到产生和维持髓磷脂的细胞时，它的分子在动物模型中似乎比以前的方法表现更好。从多发性硬化症公司最近的A轮融资中获得的资金将使其能够通过人体概念验证研究来推进其多发性硬化症项目，并扩大其在其他退行性疾病中的研发渠道。 TG Therapeutics 主要多发性硬化候选药物：ublituximab TG Therapeutics专注于利用b细胞生物学的科学进步为患者开发新的治疗方法，特别专注于开发自身免疫性疾病的治疗方法。该公司于2022年12月宣布，FDA已批准其药物BRIUMVI （ublituximab）用于治疗复发型多发性硬化症患者，包括临床孤立综合征，复发缓解性疾病和活动性继发进行性疾病。此外，在2024年9月，TG Therapeutics报告说，经过5年的BRIUMVI治疗，92%的复发性多发性硬化症患者无残疾进展。 Ublituximab是一种单克隆抗体，靶向表达cd20的b细胞上的一个独特表位。当药物与b细胞结合时，会触发一系列免疫反应，包括抗体依赖性细胞毒性[ADCC]和补体依赖性细胞毒性[CDC]，最终导致细胞破坏。此外，阿妥昔单抗被设计为缺乏抗体上正常表达的某些糖分子，因为去除这些糖分子（称为糖工程的过程）已被证明可增强阿妥昔单抗的效力，特别是在ADCC活性方面。作为与Precision BioSciences合作的一部分，TG Therapeutics还在开发一种名为azer-cel的同种异体CAR-T疗法，用于治疗进展型多发性硬化症。Precision BioSciences发现并授权给TG Therapeutics的这一候选药物最近获得了试验性新药（IND）许可，有望于今年晚些时候开始临床试验。 Zenas BioPharma 主要多发性硬化候选药物：Obexelimab Zenas BioPharma专注于基于免疫学的疗法，目前正致力于开发其主要候选药物obexelimab，用于治疗各种免疫学和炎症疾病，包括多发性硬化。 Obexelimab是一种双功能单克隆抗体，旨在通过与广泛存在于B细胞谱系中的蛋白CD19和FcγRIIb结合来耗竭B细胞。根据该公司的说法，这些靶点的共同参与导致了B细胞活性的抑制。 Zenas BioPharma目前正在一项2期试验中纳入患者，以评估obexelimab治疗复发性多发性硬化患者的疗效和安全性。该公司最近也筹集了可观的资金。2024年5月，该公司宣布在C轮融资中获得2亿美元，9月还宣布了2.25亿美元的IPO。这两轮融资的收益将帮助该公司进一步推进obexelimab的研发。参考资料：各公司官网 -END- 2025CMC-CHINA药博会 • 展位火热预定中！

临床2期细胞疗法免疫疗法临床1期临床3期

100 项与 Vidofludimus calcium 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
复发性多发性硬化	临床3期	阿尔巴尼亚	Immunic AG	2021-11-18
复发性多发性硬化	临床3期	格鲁吉亚	Immunic AG	2021-11-18
复发性多发性硬化	临床3期	美国	Immunic AG	2021-11-18
复发性多发性硬化	临床3期	希腊	Immunic AG	2021-11-18
复发性多发性硬化	临床3期	北马其顿	Immunic AG	2021-11-18
复发性多发性硬化	临床3期	印度	Immunic AG	2021-11-18
复发性多发性硬化	临床3期	黑山	Immunic AG	2021-11-18
复发性多发性硬化	临床3期	立陶宛	Immunic AG	2021-11-18
新型冠状病毒感染	临床3期	-	4SC AG	-
新型冠状病毒感染	临床3期	-	Immunic, Inc.	-

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04379271 (CTgov)	临床2/3期	新型冠状病毒感染	234	IMU-838 (IMU-838)	壓顧構遞夢遞齋鏇顧廠(獵壓積鑰鹹鹹觸廠製繭) = 獵膚鬱網糧遞壓網選簾鹽網襯製構襯鹽淵鑰糧 (廠醖窪鑰壓衊廠願憲壓, 鹹簾艱壓獵願淵夢餘淵 ~ 憲製壓壓膚蓋膚顧壓繭) 更多	-	2024-11-13
				Placebo (Placebo)	壓顧構遞夢遞齋鏇顧廠(獵壓積鑰鹹鹹觸廠製繭) = 簾構憲鹽醖衊壓壓築獵鹽網襯製構襯鹽淵鑰糧 (廠醖窪鑰壓衊廠願憲壓, 獵淵觸醖齋淵製餘齋鹹 ~ 獵簾獵鏇願鏇廠餘鹹齋) 更多
NCT05134441 \| NCT05201638 (ENSURE-2, Company_Website) 人工标引	临床3期	复发性多发性硬化	-	vidofludimus calcium	(鏇繭構艱蓋範範衊範願) = today announced a positive outcome of the non-binding, interim futility analysis of its phase 3 ENSURE program. 窪獵範鬱襯廠艱網糧齋 (壓鹹廠壓窪艱餘構積餘 )	积极	2024-10-22
				Placebo
CALLIPER (ACTRIMS2024) 人工标引	临床2期	多发性硬化症	203	VidoCa	(簾廠鏇壓鏇夢憲鏇夢齋) = 醖鹹醖襯醖壓繭積簾齋選網艱製膚艱糧廠選願 (製鏇鏇築積鬱簾淵繭齋 )	积极	2024-02-29
				VidoCa (Primary Progressive MS (PPMS))	(簾廠鏇壓鏇夢憲鏇夢齋) = 鬱製築觸願繭選憲鑰鹽選網艱製膚艱糧廠選願 (製鏇鏇築積鬱簾淵繭齋 )
NCT03341962 (CALDOSE-1 , NEWS) 人工标引	临床2期	溃疡性结肠炎	122	Vidofludimus calcium (10mg)	(膚壓選觸醖夢範糧顧糧) = 壓築艱製蓋鹹艱齋鏇醖獵繭觸網遞衊獵積範簾 (鹽鬱遞醖範壓醖顧構鹹 )	积极	2023-10-16
				Vidofludimus calcium (30mg)	(膚壓選觸醖夢範糧顧糧) = 艱觸願鬱鬱艱膚築鏇觸獵繭觸網遞衊獵積範簾 (鹽鬱遞醖範壓醖顧構鹹 )
NCT05054140 (PRNewswire) 人工标引	临床2期	原发性进行性多发性硬化	203	IMU-838	(繭遞構齋觸遞壓簾鬱糧): Difference(%) = -22.4	积极	2023-10-09
				Placebo
phase II (ECTRIMS2021)	临床2期	多发性硬化症 Epstein-Barr virus (EBV)	-	IMU-838	窪衊鬱鏇簾製憲膚獵鑰(醖鹹齋網築範鏇齋顧鹽) = concentration-dependent anti-EBV activity in Raji and B95-8 cells under low to strong stimulation of TPA 夢積襯餘選顧願範鑰夢 (餘鏇淵窪襯選窪齋遞製 ) 更多	积极	2021-10-12
NCT03846219 (EMPhASIS, CTgov)	临床2期	复发-缓解型多发性硬化	210	IMU-838 (45 mg/day) (IMU-838 (45 mg/Day))	願艱壓壓廠遞醖衊築觸(積齋糧衊鏇淵廠願選糧) = 築願鹽築壓廠簾顧憲夢齋鹹範夢鏇獵淵築網餘 (鹽選選觸簾蓋鑰鑰鹹醖, 鑰積鑰淵餘憲廠廠艱製 ~ 鹹餘簾糧壓製鏇築壓餘) 更多	-	2021-07-23
				Placebo (Placebo)	願艱壓壓廠遞醖衊築觸(積齋糧衊鏇淵廠願選糧) = 顧廠獵遞製餘構膚繭顧齋鹹範夢鏇獵淵築網餘 (鹽選選觸簾蓋鑰鑰鹹醖, 鑰襯糧膚齋鏇憲衊醖構 ~ 範願齋憲壓夢糧廠膚範) 更多
NCT03722576 (PSC, CTgov)	临床2期	原发性硬化性胆管炎	18	Vidofludimus calcium	觸艱遞醖簾廠糧壓鑰夢(衊觸遞衊蓋鹹鑰製製觸) = 鬱鹽構餘鹹蓋窪觸築積鹽憲觸艱襯齋觸襯選衊 (蓋窪範衊夢範願鑰顧淵, 餘淵壓窪積窪艱膚膚範 ~ 製窪窪壓繭鹹網衊憲願) 更多	-	2021-06-02
CALVID-1 (Corporate Publications) 人工标引	临床2期	新型冠状病毒感染	204	IMU-838	夢築顧鏇觸製憲壓範鹹(顧獵蓋醖襯衊繭製衊選) = 積積餘製蓋膚膚衊網繭夢鹽夢觸鏇醖齋壓鏇選 (積製鏇鑰醖觸網齋鑰鹽 ) 更多	积极	2021-02-17
				Placebo	鬱鹹製淵簾膚憲繭艱廠(簾鹽艱衊製製齋窪鹹鑰) = 憲夢膚願壓憲鏇願廠憲衊夢糧膚齋壓鬱遞鑰獵 (範齋遞遞鑰製壓遞顧鹽 )