A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Remibrutinib in Patients With Generalized Myasthenia Gravis, Followed by an Open-label Extension Phase

A study to evaluate the efficacy, safety and tolerability of Remibrutinib versus placebo in adult patients with Generalized Myasthenia Gravis who are on stable, standard-of-care (SOC) treatment.

Start Date23 Dec 2024

Sponsor / Collaborator

Novartis Pharmaceuticals Corp.

CTIS2023-505739-12-01 / Recruiting

A 52-week multi-center, randomized, double-blind, placebo controlled, basket study with an open-label extension to investigate the efficacy, safety, and tolerability of remibrutinib (LOU064) in Chronic Inducible Urticaria (CINDU) in adults inadequately controlled by H1-antihistamines - CLOU064M12301

Start Date30 Sep 2024

Sponsor / Collaborator

Novartis Pharma AG

CTRI/2024/08/072230 / Not yet recruitingPhase 3

Start Date20 Aug 2024

Sponsor / Collaborator

Novartis Healthcare Pvt Ltd.

100 Clinical Results associated with Remibrutinib

100 Translational Medicine associated with Remibrutinib

100 Patents (Medical) associated with Remibrutinib

Literatures (Medical) associated with Remibrutinib

01 Jul 2024·Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology

Bruton's tyrosine kinase inhibition for the treatment of allergic disorders

Review

Author: Suresh, Ragha V ; Dispenza, Melanie C ; Lin, Erica V

IgE signaling through its high-affinity receptor FcεRI is central to the pathogenesis of numerous allergic disorders. Oral inhibitors of Bruton's tyrosine kinase (BTKis), which are currently Food and Drug Administration-approved for treating B cell malignancies, broadly inhibit the FcεRI pathway in human mast cells and basophils, and therefore may be effective allergen-independent therapies for a variety of allergic diseases. The application of these drugs to the allergy space was previously limited by the low kinase selectivity and subsequent toxicities of early-generation compounds. Fortunately, next-generation, highly selective BTKis in clinical development appear to have more favorable risk-benefit profiles, and their likelihood of being Food and Drug Administration-approved for an allergy indication is increasing. Recent clinical trials have indicated the remarkable and rapid efficacy of the second-generation BTKi acalabrutinib in preventing clinical reactivity to peanut ingestion in adults with peanut allergy. In addition, next-generation BTKis including remibrutinib effectively reduce disease activity in patients with antihistamine-refractory chronic spontaneous urticaria. Finally, several BTKis are currently under investigation in early clinical trials for atopic dermatitis and asthma. In this review, we summarize recent data supporting the use of these drugs as novel therapies in food allergy, anaphylaxis, urticaria, and other allergic disorders. We also discuss safety data derived from trials using both short-term and chronic dosing of BTKis.

01 May 2024·The Journal of allergy and clinical immunology

BTK signaling—a crucial link in the pathophysiology of chronic spontaneous urticaria

Review

Author: Maurer, Marcus ; Bernstein, Jonathan A ; Saini, Sarbjit S

Chronic spontaneous urticaria (CSU) is an inflammatory skin disorder that manifests with itchy wheals, angioedema, or both for more than 6 weeks. Mast cells and basophils are the key pathogenic drivers of CSU; their activation results in histamine and cytokine release with subsequent dermal inflammation. Two overlapping mechanisms of mast cell and basophil activation have been proposed in CSU: type I autoimmunity, also called autoallergy, which is mediated via IgE against various autoallergens, and type IIb autoimmunity, which is mediated predominantly via IgG directed against the IgE receptor FcεRI or FcεRI-bound IgE. Both mechanisms involve cross-linking of FcεRI and activation of downstream signaling pathways, and they may co-occur in the same patient. In addition, B-cell receptor signaling has been postulated to play a key role in CSU by generating autoreactive B cells and autoantibody production. A cornerstone of FcεRI and B-cell receptor signaling is Bruton tyrosine kinase (BTK), making BTK inhibition a clear therapeutic target in CSU. The potential application of early-generation BTK inhibitors, including ibrutinib, in allergic and autoimmune diseases is limited owing to their unfavorable benefit-risk profile. However, novel BTK inhibitors with improved selectivity and safety profiles have been developed and are under clinical investigation in autoimmune diseases, including CSU. In phase 2 trials, the BTK inhibitors remibrutinib and fenebrutinib have demonstrated rapid and sustained improvements in CSU disease activity. With phase 3 studies of remibrutinib ongoing, it is hoped that BTK inhibitors will present an effective, well-tolerated option for patients with antihistamine-refractory CSU, a phenotype that presents a considerable clinical challenge.

01 Mar 2024·Annals of the rheumatic diseases

Efficacy and safety of remibrutinib, a selective potent oral BTK inhibitor, in Sjögren’s syndrome: results from a randomised, double-blind, placebo-controlled phase 2 trial

Article

Author: Cenni, Bruno ; Kaul, Martin ; Hanser, Malika ; Papas, Athena S ; Grioni, Andrea ; Gergely, Peter ; Tseng, Jui-Cheng ; Hillenbrand, Rainer ; Szántó, Antónia ; Pylvaenaeinen, Ilona ; Abdallah, Nasri ; Ferrero, Enrico ; Avrameas, Alexandre ; Siegel, Richard M ; Dörner, Thomas ; Santos Da Costa, Aida

Objectives:

To evaluate the safety and efficacy of remibrutinib in patients with moderate-to-severe Sjögren’s syndrome (SjS) in a phase 2 randomised, double-blind trial (NCT04035668; LOUiSSE (LOU064 in Sjögren’s Syndrome) study).

Methods:

Eligible patients fulfilling 2016 American College of Rheumatology/European League Against Rheumatism (EULAR) criteria for SjS, positive for anti-Ro/Sjögren’s syndrome-related antigen A antibodies, with moderate-to-severe disease activity (EULAR Sjögren’s Syndrome Disease Activity Index (ESSDAI) (based on weighted score) ≥ 5, EULAR Sjögren’s Syndrome Patient Reported Index (ESSPRI) ≥ 5) received remibrutinib (100 mg) either one or two times a day, or placebo for the 24-week study treatment period. The primary endpoint was change from baseline in ESSDAI at week 24. Key secondary endpoints included change from baseline in ESSDAI over time, change from baseline in ESSPRI over time and safety of remibrutinib in SjS. Key exploratory endpoints included changes to the salivary flow rate, soluble biomarkers, blood transcriptomic and serum proteomic profiles.

Results:

Remibrutinib significantly improved ESSDAI score in patients with SjS over 24 weeks compared with placebo (ΔESSDAI −2.86, p=0.003). No treatment effect was observed in ESSPRI score (ΔESSPRI 0.17, p=0.663). There was a trend towards improvement of unstimulated salivary flow with remibrutinib compared with placebo over 24 weeks. Remibrutinib had a favourable safety profile in patients with SjS over 24 weeks. Remibrutinib induced significant changes in gene expression in blood, and serum protein abundance compared with placebo.

Conclusions:

These data show preliminary efficacy and favourable safety of remibrutinib in a phase 2 trial for SjS.

News (Medical) associated with Remibrutinib

13 Nov 2024

·www.prnewswire.com

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

Phase 3Clinical ResultFast TrackCell TherapyImmunotherapy

25 Sep 2024

·endpts.com

Celldex reveals Phase 2 responses for chronic hives drug and higher rates of neutropenia

Celldex Therapeutics has unveiled 52-week data from a mid-stage study of its anti-KIT drug in chronic hives, raising anticipation for ongoing registrational studies. The drug, dubbed barzolvolimab, is in a pair of Phase 3 trials in chronic spontaneous urticaria (CSU) following positive topline Phase 2 data announced in February. Some Wall Street analysts have said Celldex could challenge Novartis’ remibrutinib, which has already completed its registrational studies. Celldex’s latest results show 71% of 52 CSU patients dosed with 150 mg barzolvolimab every four weeks had a complete response, defined as no itch or hives, at 52 weeks. In the cohort of 51 patients given 300 mg every eight weeks, just over half of them had a complete response. Celldex’s Wednesday release did not mention p-values. “We believe this data set is a landmark event for the barzolvolimab program and for the treatment of CSU,” Celldex CMO Diane Young said in a statement. The candidate is being investigated in patients struggling with symptoms despite antihistamine treatment. The responses were seen regardless of patients’ prior exposure to Novartis and Genentech’s monoclonal antibody Xolair. For context, almost 56% of patients dosed twice daily with Novartis’ BTK inhibitor achieved a complete response at 52 weeks in a Phase 2b extension trial of the drug in CSU. After reporting positive long-term Phase 3 data in May, the drugmaker has said it will file remibrutinib with regulatory agencies in the second half of the year. But at 52 weeks, Celldex’s candidate has a higher incidence of neutropenia (17%) versus its earlier 16-week data (9%). Neutropenia was a big concern for investors going into the Phase 2 trial, TD Cowen analysts said in February. Celldex’s shares $CLDX were down by as much as 27% Wednesday, right after the company’s data announcement. Celldex said that neutrophil counts didn’t drop further with continued dosing in the latest Phase 2 data, and there was no link between neutropenia and infections. The Wednesday release did not mention concerns with anaphylaxis. The company said around 8% of patients in the 150 mg arm and 13% in the 300 mg arm discontinued treatment due to an adverse event. The company reported topline Phase 2 results in February that showed three doses, including the lowest dose of 75 mg, met the primary endpoint of mean change in urticaria activity score 7 (UAS7) at 12 weeks. At the time, this result was described by Cowen analysts as “superb.” The two identical Phase 3 studies of barzolvolimab in CSU, which were initiated in July, are aiming to enroll 915 patients each and will test 150 mg and 300 mg doses. Both trials are using the same primary endpoint as the Phase 2. Barzolvolimab is also in development for prurigo nodularis, atopic dermatitis and chronic inducible urticaria. Earlier this month, GlobalData analysts said barzolvolimab could achieve $874 million in US sales by 2039.

Phase 2Clinical ResultPhase 3

11 Sep 2024

·www.fiercepharma.com

After FDA rejection, Sanofi and Regeneron show data that could pave way for Dupixent to treat hives condition

New data from a phase 3 trial could pave the way for Sanofi and Regeneron's Dupixent to score an FDA approval to treat chronic spontaneous urticaria. The U.S. regulator rejected the companies' previous bid for a nod in October of last year. Eleven months after sustaining a rejection from the FDA for Dupixent to treat chronic spontaneous urticaria (CSU), Sanofi and Regeneron have presented data from a phase 3 trial that could help push the megablockbuster across the finish line in the indication.The LIBERTY-CUPID C study met its primary and secondary endpoints in testing patients with Dupixent who were already receiving background antihistamine therapy. It is the third phase 3 trial for Dupixent in CSU, a severe inflammatory condition that causes hives and deep swelling on or under the skin.In the trial of 151 children and adults, those who took Dupixent plus standard-of-care (SOC) antihistamines experienced an average 8.6-point reduction in itch severity (on a scale of 21) compared to a 6.1-point reduction for patients who received SOC plus placebo.The Dupixent group also experienced a 15.9-point reduction in itch and hive severity (on a 42-point scale) versus an 11.2-point reduction for the control group. The measurements were taken after 24 weeks on therapy.Additionally, 30% of Dupixent-treated patients reported a complete response compared to 18% of those on placebo.“The positive pivotal data from this study reinforce the potential of Dupixent to offer a new treatment option for the many people suffering from chronic spontaneous urticaria who do not respond to standard-of-care antihistamines,” Sanofi’s chief medical officer Dietmar Berger, M.D., Ph.D., said in a release.The companies will share the data with the FDA in the hopes of bringing Dupixent to CSU patients “as soon as possible," Berger added. The partners plan to file their regulatory resubmission before the end of the year. Earlier this year, Dupixent was approved for CSU in Japan.The trial confirms results from the successful LIBERTY-CUPID A study, which tested Dupixent plus SOC antihistamines against antihistamines alone in patients with moderate-to-severe CSU. In the LIBERTY-CUPID B trial, however, Dupixent came up short, failing to significantly reduce symptoms of people who did not have success with the lone treatment approved in for CSU—Novartis and Roche’s Xolair. While the trial was stopped for futility, the companies noted that Dupixent provided numerical improvements in the each of the main evaluation criteria, including itching.The result helped convince the FDA to reject Sanofi and Regeneron’s application for approval and ask the companies in a complete response letter for more efficacy data.More than 300,000 people in the U.S. suffer from CSU that is inadequately controlled by antihistamines, the companies said. Dupixent has been on the market for seven years and has been approved in atopic dermatitis, asthma, rhinosinusitis, eosinophilic esophagitis and prurigo nodularis. Another indication may soon be coming as the FDA is reviewing Dupixent as a treatment for chronic obstructive pulmonary disease. If approved in CSU, Dupixent would become the first new treatment in the indication in more than a decade, according to Sanofi and Regeneron. In 2023, Dupixent’s sales increased by 33% to $11.6 billion. More than 1 million patients have been treated with the monoclonal antibody, the companies said.Xolair, which was approved for CSU in 2014 and covers most of the same indications as Dupixent, generated sales of $4 billion last year.With Xolair soon to face biosimilar competition, Novartis is working to bring a new CSU treatment to the market as it recently presented data from two successful phase 3 trials for oral BTK inhibitor remibrutinib. The company expects to submit for approval of remibrutinib next year.

Clinical ResultPhase 3Drug Approval

100 Deals associated with Remibrutinib

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Myasthenia Gravis	Phase 3	-	Novartis Pharmaceuticals Corp.	23 Dec 2024
Multiple sclerosis relapse	Phase 3	US	China Novartis Institutes for BioMedical Research Co., Ltd.	13 Dec 2021
Multiple sclerosis relapse	Phase 3	US	Novartis Pharmaceuticals Corp.	13 Dec 2021
Multiple sclerosis relapse	Phase 3	CN	Novartis Pharmaceuticals Corp.	13 Dec 2021
Multiple sclerosis relapse	Phase 3	JP	Novartis Pharmaceuticals Corp.	13 Dec 2021
Multiple sclerosis relapse	Phase 3	JP	China Novartis Institutes for BioMedical Research Co., Ltd.	13 Dec 2021
Multiple sclerosis relapse	Phase 3	AR	Novartis Pharmaceuticals Corp.	13 Dec 2021
Multiple sclerosis relapse	Phase 3	AR	China Novartis Institutes for BioMedical Research Co., Ltd.	13 Dec 2021
Multiple sclerosis relapse	Phase 3	BR	China Novartis Institutes for BioMedical Research Co., Ltd.	13 Dec 2021
Multiple sclerosis relapse	Phase 3	BR	Novartis Pharmaceuticals Corp.	13 Dec 2021

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05030311 (REMIX-1, CTgov)	Phase 3	Chronic Urticaria	470	LOU064 25 mg (b.i.d) (LOU064 25mg b.i.d.)	wfesxrxzdn(byjhvoftrn) = agbdydqxgm tpgsfixiep (datkqchiif, xxwmvlccbg - kyqqaecnhg) View more	-	02 Dec 2024
				Placebo+LOU064 25 mg (b.i.d) as a tablet. (Placebo)	wfesxrxzdn(byjhvoftrn) = xmtdbsmoxm tpgsfixiep (datkqchiif, qoubehlhas - darmymirgg) View more
NCT05048342 (BISCUIT, CTgov)	Phase 3	Chronic Urticaria	71	LOU064	spdjdjymvb(ydwxkfqjoe) = yadqafrlzo aqcdqxkxji (gfnjxotvow, yamnpsjmml - ocaiwfolqu) View more	-	22 Nov 2024
NCT05032157 (REMIX-2, CTgov)	Phase 3	Chronic Urticaria	455	LOU064 (open-label)+Placebo	pxowytjxma(pvtiavqnfw) = awcerjdeks pnxyjgttzz (reensywfra, tqvpechbop - ocbuhtqwfk) View more	-	01 Nov 2024
REMIX-1/-2 (ACAAI2024)	Phase 3	Chronic Urticaria Bruton's tyrosine kinase (BTK)	-	Remibrutinib 25 mg twice daily	zyymvsrwee(lqxehiucmw) = xupjbyvubp ckzqjgrncu (pwtngtacam ) View more	Positive	24 Oct 2024
NCT03926611 (Phase 2b core and extension study, EAN2024)	Phase 2	Serum Sickness	309	Remibrutinib 100 mg b.i.d.	aiakqgplxh(pzwrmydkfk) = yahhsqoxps dmbhdiazyt (gxrfmyhlph, 2.47) View more	Positive	28 Jun 2024
NCT05032157 (REMIX-2, GlobeNewswire) Manual	Phase 3	Chronic Urticaria	455	Remibrutinib	ttpxxkrxoa(xdgrdapdoe) = Almost half of patients were completely free of itch and hives (UAS7=0) as assessed at Week 52 aqgoxkyagv (ivakqbkxpa ) View more	Positive	30 May 2024
				Placebo
NCT05030311 (REMIX-1, GlobeNewswire) Manual	Phase 3	Chronic Urticaria	470	Remibrutinib	lilutbwenp(fckkqigxqs) = Almost half of patients were completely free of itch and hives (UAS7=0) as assessed at Week 52 zpzextnkpx (kinenriqpr ) View more	Positive	30 May 2024
				Placebo
NCT03926611 (REMODEL, AAN2024)	Phase 2	Chronic Urticaria	309	Remibrutinib 100mg b.i.d.	oathhnxqiq(ntdoqskjwt) = hflnmfapjl vomoplrlib (ksumhecyfw, 2.49) View more	Positive	09 Apr 2024
NEWS Manual	Phase 2	Hidradenitis Suppurativa	77	Remibrutinib 100 mg	fxshpfobya(nbokxakbam) = faxibliiso moaybhrgbz (oawtkqxflc ) View more	Positive	09 Mar 2024
				Remibrutinib 25 mg	fxshpfobya(nbokxakbam) = vfllpwnfan moaybhrgbz (oawtkqxflc ) View more
NEWS Manual	Phase 2	Hidradenitis Suppurativa	66	Remibrutinib 25 mg	zbmmejafdw(uevlnsbvml) = otbodztyia mpcjdnzxgg (vktqnpsuxj ) View more	Positive	09 Mar 2024
				Remibrutinib 100 mg	zbmmejafdw(uevlnsbvml) = veizjipkzi mpcjdnzxgg (vktqnpsuxj ) View more

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