Latest news with #IMBRUVICA
Business Upturn
14-07-2025
- Business
- Business Upturn
Waldenstrom Macroglobulinemia Market Sees Surging Demand Across the 7MM Amid BTK Inhibitor Advancements
New York, USA, July 14, 2025 (GLOBE NEWSWIRE) — Waldenstrom Macroglobulinemia Market Sees Surging Demand Across the 7MM Amid BTK Inhibitor Advancements | DelveInsight The Waldenstrom macroglobulinemia market is witnessing steady growth driven by advances in targeted therapies, particularly BTK inhibitors like IMBRUVICA and BRUKINSA. Rising awareness, improved diagnostic techniques, and increasing clinical trial activity are further propelling market expansion. DelveInsight's Waldenstrom Macroglobulinemia Market Insights report includes a comprehensive understanding of current treatment practices, emerging Waldenstrom macroglobulinemia drugs, market share of individual therapies, and current and forecasted Waldenstrom macroglobulinemia market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Key Takeaways from the Waldenstrom Macroglobulinemia Market Report According to DelveInsight's analysis, the total Waldenstrom macroglobulinemia market size is expected to grow positively by 2034. The United States accounts for the largest market size of Waldenstrom macroglobulinemia, in comparison to EU4 (Germany, Italy, France, and Spain) and the UK, and Japan. Waldenström macroglobulinemia affects about 1 in 3.4 million American males and about half that number of American females. The incidence of waldenström macroglobulinemia is estimated to be about 5 per 1,000,000 people over the age of 50. American males and about half that number of American females. The incidence of waldenström macroglobulinemia is estimated to be about people over the age of 50. Prominent companies, including Cellectar Biosciences, Merck Sharp & Dohme, BeiGene, Nurix Therapeutics, Ascentage Pharma, TransThera Biosciences, and others, are actively working on innovative Waldenstrom macroglobulinemia drugs. and others, are actively working on innovative Waldenstrom macroglobulinemia drugs. Some of the key Waldenstrom macroglobulinemia therapies in the pipeline include Iopofosine, Nemtabrutinib, Sonrotoclax, NX-5948, Lisaftoclax, TT-01488 , and others. These novel Waldenstrom macroglobulinemia therapies are anticipated to enter the Waldenstrom macroglobulinemia market in the forecast period and are expected to change the market. , and others. These novel Waldenstrom macroglobulinemia therapies are anticipated to enter the Waldenstrom macroglobulinemia market in the forecast period and are expected to change the market. In June 2025, Cellectar Biosciences announced that the US FDA granted Breakthrough Therapy Designation (BTD) for iopofosine I 131 in Waldenstrom macroglobulinemia. announced that the US FDA granted Breakthrough Therapy Designation (BTD) for iopofosine I 131 in Waldenstrom macroglobulinemia. In June 2025, BeiGene showcased encouraging Phase I/II (CaDAnCe-101) results for BGB-16673 in waldenström macroglobulinemia at EHA 2025. showcased encouraging Phase I/II (CaDAnCe-101) results for BGB-16673 in waldenström macroglobulinemia at EHA 2025. In March 2025, the US FDA granted orphan drug designation to bexobrutideg (NX-5948) for the treatment of Waldenstrom macroglobulinemia. Discover which Waldenstrom macroglobulinemia medications are expected to grab the market share @ Waldenstrom Macroglobulinemia Market Report Waldenstrom Macroglobulinemia Overview Waldenstrom macroglobulinemia is a rare, slow-growing type of non-Hodgkin lymphoma characterized by the excessive production of abnormal white blood cells that secrete large amounts of a monoclonal IgM protein. These abnormal cells accumulate in the bone marrow and can interfere with the normal production of blood cells. The exact cause of Waldenstrom macroglobulinemia is not fully understood, but it is believed to arise due to genetic mutations, most commonly in the MYD88 gene, and possibly immune system dysregulation. Risk factors may include age, male gender, family history, and certain inherited conditions. Symptoms of Waldenstrom macroglobulinemia can vary significantly and often develop gradually. Common clinical signs include fatigue, weight loss, night sweats, and swollen lymph nodes. The excess IgM protein can cause hyperviscosity syndrome, leading to headaches, vision problems, dizziness, and bleeding issues. Some patients may also experience neuropathy, cold sensitivity, or enlargement of the spleen and liver. Diagnosis typically involves a combination of blood tests, bone marrow biopsy, and molecular testing to identify MYD88 or CXCR4 mutations. Imaging may also be used to assess organ involvement. Early and accurate diagnosis is key to managing this indolent but complex hematologic malignancy. Waldenstrom Macroglobulinemia Epidemiology Segmentation The Waldenstrom macroglobulinemia epidemiology section provides insights into the historical and current Waldenstrom macroglobulinemia patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The Waldenstrom macroglobulinemia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Incident Cases of Waldenstrom Macroglobulinemia Gender-specific Cases of Waldenstrom Macroglobulinemia Age-specific Cases of Waldenstrom Macroglobulinemia Gene Mutations in Waldenstrom Macroglobulinemia Download the report to understand which factors are driving Waldenstrom macroglobulinemia epidemiology trends @ Waldenstrom Macroglobulinemia Treatment Algorithm Waldenstrom Macroglobulinemia Treatment Market Significant progress has been made in the treatment of Waldenström macroglobulinemia, a rare form of cancer, with therapies increasingly tailored to its unique clinical challenges. The Waldenström macroglobulinemia treatment landscape is primarily led by BTK inhibitors such as IMBRUVICA and BRUKINSA, alongside rituximab-based regimens and BCL-2 inhibitors like VENETOCLAX, which serve as complementary options. These targeted therapies have revolutionized clinical outcomes by extending progression-free survival and enhancing response rates, while maintaining manageable side effects. BRUKINSA (zanubrutinib) from BeiGene represents a next-generation BTK inhibitor, engineered for improved selectivity and reduced off-target activity compared to earlier BTK inhibitors. It received U.S. FDA approval in 2021 for treating adults with Waldenström macroglobulinemia and has also been authorized by the EMA and other global regulatory bodies for use in this and other B-cell malignancies. IMBRUVICA (ibrutinib), developed by Janssen and AbbVie, was the first BTK inhibitor to gain FDA approval in 2015 for adult patients with Waldenström macroglobulinemia. As a pioneering agent in its class, it transformed the treatment approach by disrupting B-cell signaling pathways that drive malignant cell growth. That same year, the European Commission also approved its use. Rituximab remains the most commonly used monoclonal antibody for treating Waldenström macroglobulinemia. It works by binding to the CD20 protein found on the surface of lymphoma cells, triggering their destruction. Administered via intravenous infusion in clinical settings, rituximab may be used as a standalone therapy or in combination with chemotherapy, targeted treatments, or other agents. Learn more about the Waldenstrom macroglobulinemia treatment options @ Waldenstrom Macroglobulinemia Treatment Guidelines Waldenstrom Macroglobulinemia Emerging Drugs and Companies The potential drugs in the pipeline include Iopofosine (Cellectar Biosciences), Nemtabrutinib (Merck Sharp & Dohme), Sonrotoclax (BeiGene), NX-5948 (Nurix Therapeutics), Lisaftoclax (Ascentage Pharma), TT-01488 (TransThera Biosciences), and others. Iopofosine is a small-molecule Phospholipid Drug Conjugate (PDC) engineered to deliver iodine-131 directly to cancer cells in a targeted manner. It was recently assessed in the pivotal Phase II CLOVER-WaM study involving patients with relapsed/refractory Waldenstrom macroglobulinemia (R/R WM). The U.S. FDA has granted Fast Track Designation to iopofosine for use in patients with lymphoplasmacytic lymphoma (LPL) and Waldenstrom macroglobulinemia who have undergone at least two prior therapies. Furthermore, the drug has received Orphan Drug Designation (ODD) for LPL/WM. In the European Union, the European Commission has granted ODD for relapsed/refractory multiple myeloma and WM, along with PRIME designation specifically for WM, acknowledging its potential to address significant unmet clinical needs. In June 2025, the FDA also awarded Breakthrough Therapy Designation (BTD) to iopofosine I-131 for treating R/R WM. Nemtabrutinib is an experimental, reversible, non-covalent BTK inhibitor under investigation for its ability to block oncogenic B-cell receptor signaling. It is active against both wild-type BTK and BTK pathway mutations. Ongoing Phase II trials are evaluating its effectiveness in hematologic cancers, including chronic lymphocytic leukemia (CLL) and Waldenstrom macroglobulinemia. Unlike conventional BTK inhibitors, nemtabrutinib retains activity even against BTK proteins that have developed mutations. Sonrotoclax is a BH3 mimetic designed to inhibit BCL2, a protein that enables cancer cell survival. By mimicking natural signals that promote cell death, sonrotoclax induces apoptosis in B-cell malignancies. Preclinical and early clinical studies have demonstrated that it is a potent and selective BCL2 inhibitor, characterized by a short half-life and no accumulation in the body. It has shown encouraging clinical results across multiple B-cell cancers, with over 1,300 patients treated globally. The U.S. FDA has granted Fast Track Designation to sonrotoclax for treating Waldenstrom macroglobulinemia. The anticipated launch of these emerging Waldenstrom macroglobulinemia therapies are poised to transform the Waldenstrom macroglobulinemia market landscape in the coming years. As these cutting-edge Waldenstrom macroglobulinemia therapies continue to mature and gain regulatory approval, they are expected to reshape the Waldenstrom macroglobulinemia market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about new treatment for Waldenstrom macroglobulinemia, visit @ Waldenstrom Macroglobulinemia Management Waldenstrom Macroglobulinemia Market Dynamics The Waldenstrom macroglobulinemia market dynamics are anticipated to change in the coming years. The availability of targeted therapies such as BTK inhibitors (IMBRUVICA, BRUKINSA) and monoclonal antibodies (rituximab), along with the identification of MYD88 L265P and CXCR4 mutations, enables more personalized and effective treatment approaches, while emerging options like novel BTK inhibitors, CAR-T therapies, bispecific antibodies, and the development of new drug classes and combination regimens hold promise for significantly enhancing treatment outcomes. Furthermore, many potential therapies are being investigated for the treatment of Waldenstrom macroglobulinemia, and it is safe to predict that the treatment space will significantly impact the Waldenstrom macroglobulinemia market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate is expected to drive the growth of the Waldenstrom macroglobulinemia market in the 7MM. However, several factors may impede the growth of the Waldenstrom macroglobulinemia market. Targeted therapies like BTK inhibitors are often expensive, creating accessibility and reimbursement challenges, especially in lower-income countries, while resistance or intolerance to existing treatments like IMBRUVICA and BRUKINSA further necessitate alternative options, yet stringent approval processes and high clinical trial costs can significantly delay the launch of these new therapies. Moreover, Waldenstrom macroglobulinemia treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the Waldenstrom macroglobulinemia market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the Waldenstrom macroglobulinemia market growth. Waldenstrom Macroglobulinemia Report Metrics Details Study Period 2020–2034 Waldenstrom Macroglobulinemia Report Coverage 7MM [The United States, the EU-4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan] Key Waldenstrom Macroglobulinemia Companies Cellectar Biosciences, Merck Sharp & Dohme, BeiGene, Nurix Therapeutics, Ascentage Pharma, TransThera Biosciences, BioGene, Janssen, AbbVie, and others Key Waldenstrom Macroglobulinemia Therapies Iopofosine, Nemtabrutinib, Sonrotoclax, NX-5948, Lisaftoclax, TT-01488, BRUKINSA, IMBRUVICA, and others Scope of the Waldenstrom Macroglobulinemia Market Report Waldenstrom Macroglobulinemia Therapeutic Assessment: Waldenstrom Macroglobulinemia current marketed and emerging therapies Waldenstrom Macroglobulinemia current marketed and emerging therapies Waldenstrom Macroglobulinemia Market Dynamics: Conjoint Analysis of Emerging Waldenstrom Macroglobulinemia Drugs Conjoint Analysis of Emerging Waldenstrom Macroglobulinemia Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Waldenstrom Macroglobulinemia Market Access and Reimbursement Discover more about Waldenstrom macroglobulinemia drugs in development @ Waldenstrom Macroglobulinemia Clinical Trials Table of Contents 1. Waldenstrom Macroglobulinemia Market Key Insights 2. Waldenstrom Macroglobulinemia Market Report Introduction 3. Waldenstrom Macroglobulinemia Market Overview at a Glance 4. Waldenstrom Macroglobulinemia Market Executive Summary 5. Disease Background and Overview 6. Waldenstrom Macroglobulinemia Treatment and Management 7. Waldenstrom Macroglobulinemia Epidemiology and Patient Population 8. Patient Journey 9. Waldenstrom Macroglobulinemia Marketed Drugs 10. Waldenstrom Macroglobulinemia Emerging Drugs 11. Seven Major Waldenstrom Macroglobulinemia Market Analysis 12. Waldenstrom Macroglobulinemia Market Outlook 13. Potential of Current and Emerging Therapies 14. KOL Views 15. Unmet Needs 16. SWOT Analysis Related Reports Waldenstrom Macroglobulinemia Pipeline Waldenstrom Macroglobulinemia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Waldenstrom macroglobulinemia companies, including BeiGene, Cellectar Biosciences, Inc., Nurix Therapeutics, Inc., VelosBio Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA), Schrodinger, Inc., Loxo Oncology, Inc., Nkarta, Inc., Merck Sharp & Dohme LLC, ADC Therapeutics S.A., Bio-Path Holdings, Inc., Millennium Pharmaceuticals, Inc., Carna Biosciences, Inc., among others. Non-Hodgkin's Lymphoma Market Non-Hodgkin's Lymphoma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key NHL companies, including AbbVie, Genmab, Novartis, Angiocrine Bioscience, Autolus, Zentera Therapeutics, Jiangsu Hengrui Medicine, among others. Non-Hodgkin's Lymphoma Pipeline Non-Hodgkin's Lymphoma Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key NHL companies, including Novartis, AstraZeneca, Genentech, BioInvent, Genmab, SystImmune, Nordic Nanovector, Pacylex Pharmaceuticals, Artiva Biotherapeutics, Inc., Chipscreen Biosciences, Ltd., Timmune Biotech Inc., Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Gilead Sciences, Acerta Pharma BV, Adagene Inc, Conjupro Biotherapeutics, Inc., Rhizen Pharmaceuticals, Juventas Cell Therapy Ltd., Incyte Corporation, HUYA Bioscience International, SecuraBio, Genor Biopharma Co., Ltd., Kyowa Kirin Co., Ltd., Antengene Therapeutics Limited, Regeneron Pharmaceuticals, Jiangsu HengRui Medicine Co., Ltd., Xynomic Pharmaceuticals, Inc., BioTheryX, Inc., UWELL Biopharma, Kronos Bio, Bio-Thera Solutions, Spectrum Pharmaceuticals, Inc., Aptose Biosciences Inc., Miltenyi Biomedicine GmbH, Precision BioSciences, Inc., Teneobio, Inc., TCR2 Therapeutics, IGM Biosciences, Inc., among others. Multiple Myeloma Market Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key multiple myeloma companies, including Sanofi, Karyopharm Therapeutics, AbbVie, Takeda Pharmaceutical, Celgene, Bristol-Myers Squibb, RAPA Therapeutics, Pfizer, Array Biopharma, Cellectar Biosciences, BioLineRx, Celgene, Aduro Biotech, ExCellThera, Janssen Pharmaceutical, Precision BioSciences, Takeda, Glenmark (Ichnos Sciences SA), Poseida Therapeutics, Molecular Partners AG, Chipscreen Biosciences, AbbVie, Genentech (Roche), Janssen Biotech, Nanjing Legend Biotech, Merck Sharp & Dohme Corp., among others. Diffuse Large B-cell Lymphoma Market Diffuse Large B-cell Lymphoma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key DLBCL companies, including Roche (Genentech), Biogen, Nektar Therapeutics, Merck, Allogene Therapeutics, Miltenyi Biomedicine, AstraZeneca, BioVaxys, ImmunoVaccine Technologies, Cellectar Biosciences, Galapagos, Novartis, Lyell, ImmPACT Bio, Pfizer, Kartos Therapeutics, 2seventy bio, Regeneron Pharmaceuticals, BeiGene, Ranok Therapeutics, Constellation Pharmaceuticals, Genmab, IDP Discovery Pharma S.L., Immunitas Therapeutics, Monte Rosa Therapeutics, SymBio Pharmaceuticals, AVM Biotechnology, Autolus Therapeutics, Kymera Therapeutics, Otsuka Pharmaceutical, Caribou Biosciences, Adicet Bio, Gilead Sciences, Xynomic Pharmaceuticals, Amgen, among others. DelveInsight's Pharma Competitive Intelligence Service: Through its CI solutions, DelveInsight provides its clients with real-time and actionable intelligence on their competitors and markets of interest to keep them stay ahead of the competition by providing insights into the latest therapeutic area-specific/indication-specific market trends, in emerging drugs, and competitive strategies. These services are tailored to the specific needs of each client and are delivered through a combination of reports, dashboards, and interactive presentations, enabling clients to make informed decisions, mitigate risks, and identify opportunities for growth and expansion. Other Business Pharmaceutical Consulting Services Healthcare Conference Coverage Pipeline Assessment Healthcare Licensing Services Discover how a mid-pharma client gained a level of confidence in their soon-to-be partner for manufacturing their therapeutics by downloading our Due Diligence Case Study 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. Connect with us on LinkedIn|Facebook|Twitter Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same. Ahmedabad Plane Crash
Yahoo
12-06-2025
- Business
- Yahoo
4 Emerging Graft versus Host Disease Therapies That Could Change the Treatment Landscape
Key companies, such as CSL Behring, Equillium, Biocon, and medac, are advancing their assets through late-stage graft versus host disease clinical trials, driving innovation in the graft versus host disease market and creating significant growth opportunities. LAS VEGAS, June 12, 2025 /PRNewswire/ -- Graft versus host disease (GvHD) is an immune-driven disorder caused by a complex interplay between the donor's and recipient's adaptive immune systems. It typically manifests in two main forms: acute (aGvHD) and chronic (cGvHD). As per DelveInsight's analysis, approximately 60,000 allogeneic transplants and around 55,000 GvHD cases occurred in the 7MM in 2024. These numbers are projected to grow at a notable CAGR over the forecast period from 2025 to 2034. Corticosteroids like prednisone and methylprednisolone are the primary first-line treatments, often combined with other immunosuppressants. Mild GcHD is managed with topical steroids, while systemic cases require stronger immunosuppressive therapy. Several treatments for GvHD have received FDA approval in the US, including ORENCIA (abatacept), JAKAFI/JAKAVI (ruxolitinib), IMBRUVICA (ibrutinib), and REZUROCK (belumosudil), among others. In the upcoming GvHD treatment market landscape, there are a plethora of companies investigating agents in various stages of development. To know more about the graft versus host disease clinical trials market, visit @ Graft versus Host Disease Market DelveInsight estimates that the market size for GvHD is expected to grow from USD 1.4 billion in 2024 with a significant CAGR of 8.2% by 2034. This anticipated growth is driven by advancements in treatment options, greater healthcare access, and a rising prevalence of the condition, which together foster higher demand for innovative and effective therapies. The current pipeline for graft versus host disease includes a range of drugs with diverse mechanisms of action (MoA). CSL964 (Alpha 1 Antitrypsin), EQ001 (Itolizumab; Bmab600), MaaT013, and MC0518 are the most promising drugs that are in the late-stage of development for GvHD treatment. Ongoing research and current trials have the potential to change the GvHD market. Keen to know how the GvHD market will evolve by 2034? Find out @ Graft versus Host Disease (GvHD) Market Forecast Apart from this, several GvHD drugs currently in the early stages of development include RLS-0071 by ReAlta Life Sciences, Vimseltinib by Deciphera Pharmaceuticals, ASC-930 by ASC Therapeutics, RGI-2001 by REGiMMUNE, CYP-001 by Cynata Therapeutics, arsenic trioxide (As2O3) by BioSenic (Medsenic), TRX103 (Tregs) by Tr1X, TCD601 (Siplizumab) by ITB-MED, F-652 by Evive Biotech, RHPRG4 by Lubris BioPharma, XBI302 by Xbiome, RG6287 by Genentech, ALTB-168 by AltruBio, and SER-155 by Seres Therapeutics. Now, let's examine the late-stage pipeline therapies under investigation for GvHD treatment CSL Behring's ZEMAIRA CSL964 Alpha-1 Antitrypsin, an Alpha1-Proteinase Inhibitor (A1-PI) developed by CSL Behring, is being studied for the treatment of steroid-refractory acute graft-versus-host disease (aGvHD) and for the prevention of aGvHD in high-risk patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). It is currently in Phase III clinical trials for the treatment of steroid-refractory aGvHD and is also being evaluated in Phase II/III trials for its potential in preventing aGvHD. Equillium/Biocon's EQ001 EQ001 (Itolizumab; Bmab600) is a first-in-class immune-modulating antibody that targets CD6 to suppress the activation and migration of harmful T cells responsible for releasing pro-inflammatory cytokines in autoimmune and inflammatory conditions such as GvHD, moderate-to-severe uncontrolled asthma, and lupus nephritis. By acting on the CD6-ALCAM signaling pathway, Itolizumab selectively inhibits pathogenic effector T cells (Teffs) while preserving regulatory T cells (Tregs), which are essential for immune system balance. Currently, EQ001 is undergoing a Phase III clinical trial in combination with corticosteroids as a first-line therapy for March 2025, Equillium reported topline Phase III EQUATOR trial results for itolizumab in first-line aGVHD. While the study did not show a meaningful difference in CR or ORR at Day 29 versus placebo, itolizumab demonstrated statistically significant and clinically meaningful improvements in longer-term outcomes, including CR at Day 99, duration of response, and failure-free survival. In April 2025, the FDA declined to grant Breakthrough Therapy designation or support an Accelerated Approval pathway for itolizumab, citing limitations in the EQUATOR study data. Discover which therapies are expected to grab major GvHD market share @ Graft versus Host Disease Treatment Market MaaT Pharma's MaaT013 MaaT013 is a standardized, donor-derived microbiome ecosystem therapy characterized by high richness and diversity. It includes BUTYCORE, a consortium of bacterial species known for producing anti-inflammatory short-chain fatty acids. The therapy is designed to reestablish the balance between the patient's gut microbiome and immune system, aiming to enhance immune tolerance and responsiveness, thereby addressing steroid-resistant, gastrointestinal-dominant aGvHD. MaaT013 has been granted Orphan Drug Designation (ODD) by both the US FDA and the EMA. In December 2024, MaaT Pharma shared encouraging updated results from its Early Access Program at the ASH 2024 Annual Meeting. Subsequently, in January 2025, the company announced promising topline findings from the Phase III ARES trial, where MaaT013 achieved a 62% overall gastrointestinal response rate by Day 28, marking a significant advancement as a third-line treatment for GI-aGvHD. medac's MC0518 MC0518 is an investigational mesenchymal stromal cell (MSC) therapy developed by Medac GmbH, currently undergoing clinical trials for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGvHD) following allogeneic hematopoietic stem cell transplantation. This therapy leverages the immunomodulatory properties of MSCs to mitigate the severe inflammatory responses characteristic of SR-aGvHD, a condition where the donor's immune cells attack the recipient's tissues despite steroid treatment. The IDUNN trial, a pivotal Phase III study, is evaluating the efficacy and safety of MC0518 compared to the best available therapy (BAT) in pediatric and adolescent patients. The primary endpoint is the overall response rate (ORR) at Day 28, with secondary objectives including overall survival (OS) up to 24 months and freedom from treatment failure (FFTF) within six months. Preclinical assessments have demonstrated that MC0518 is well-tolerated, with no evidence of tumorigenicity or significant adverse effects in animal models. Discover more about drugs for GvHD in development @ Graft versus Host Disease Clinical Trials The anticipated launch of these emerging therapies for GvHD are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the GvHD market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight's latest published market report, titled as Graft versus Host Disease Market Insight, Epidemiology, and Market Forecast – 2034, will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the GvHD country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The GvHD market report offers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total allogenic transplant cases Total Graft Versus Host Disease Cases Type-specific Cases of Graft Versus Host Disease Acute Graft Versus Host Disease Cases by Grading Acute Graft Versus Host Disease Cases by Organ Involvement Chronic Graft Versus Host Disease Cases by Grading Chronic Graft Versus Host Disease Cases by Organ Involvement Total Treated Cases of Graft Versus Host Disease Mortality Adjusted Treated Cases of Graft Versus Host Disease The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM GvHD market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this GvHD market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the GvHD market. Also, stay abreast of the mitigating factors to improve your market position in the GvHD therapeutic space. Related Reports Graft versus Host Disease Epidemiology Forecast Graft versus Host Disease Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted GvHD epidemiology in the 7MM, i.e., the United States, EU4 (Germany, Spain, Italy, France) and the United Kingdom, and Japan. Graft versus Host Disease Pipeline Graft versus Host Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key GvHD companies, including Abbisko Therapeutics, Equillium, Theriva Biologics, Seres Therapeutics, CytoMed Therapeutics, Beijing Tide Pharmaceutical, CTI BioPharma, ViGenCell, Lipella Pharmaceuticals, Cellestia Biotech, Seres Therapeutics, Jiangsu HengRui Medicine Therapeutics, Genentech, AltruBio, Orca Bio, GSK, Amgen, among others. Acute Graft versus Host Disease Pipeline Acute Graft versus Host Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acute GvHD companies, including MaaT Pharma, Medac, CSL Behring, Humanigen, Ironwood Pharmaceuticals, ReAlta Life Sciences, Roche, Incyte Corporation, among others. Ocular Graft versus Host Disease Pipeline Ocular Graft versus Host Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key ocular GvHD companies, including Cambium Medical Technologies, Glia LLC., Ocular Discovery, Selagine, 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 UsShruti Thakur info@ +14699457679 Logo: View original content: SOURCE DelveInsight Business Research, LLP
Business Upturn
12-06-2025
- Business
- Business Upturn
Significant efficacy benefit of IMBRUVICA® (ibrutinib) plus venetoclax versus acalabrutinib plus venetoclax in frontline treatment of patients with chronic lymphocytic leukaemia suggested by indirect treatment comparison
Cross-study findings indicate significant clinical benefit of frontline fixed-duration ibrutinib plus venetoclax with improved likelihood of undetectable minimal residual disease and progression-free survival versus acalabrutinib plus venetoclax 1 Phase 2 CAPTIVATE long-term follow-up data further supports sustained efficacy and safety profile of fixed-duration ibrutinib plus venetoclax treatment in patients receiving frontline treatment for chronic lymphocytic leukaemia 2 Beerse, Belgium, June 12, 2025 (GLOBE NEWSWIRE) — Janssen-Cilag International NV, a Johnson & Johnson company, today announced new data from a matching-adjusted indirect comparison (MAIC) analysis assessing the efficacy of IMBRUVICA® (ibrutinib) in combination with venetoclax (I+V) vs acalabrutinib in combination with venetoclax (A+V) as fixed-duration (FD) treatments for adults with previously untreated chronic lymphocytic leukaemia (CLL).1 The data were featured in a poster presentation at the 30th European Hematology Association (EHA) Congress (Poster presentation #PF587) and reported that the I+V regimen yielded significantly better efficacy when compared to the A+V regimen.1 Patients treated with I+V were more likely to achieve disease clearance, as measured by undetectable minimal residual disease (uMRD) three months after the end of treatment (EOT+3), from both peripheral blood (PB) and bone marrow (BM).1 In addition to this, progression-free survival (PFS) significantly favoured I+V compared to A+V.1 'In the absence of head-to-head trials, clinicians need reliable tools to effectively compare treatment options and make the best possible choices for their patients,' said Talha Munir, M.D., Consultant in Clinical Haematology at St James's Hospital, Leeds, United Kingdom.* 'The matching-adjusted indirect comparison data presented at EHA suggests that ibrutinib plus venetoclax may offer meaningful clinical advantages over acalabrutinib plus venetoclax with patients more likely to achieve higher rates of undetectable minimal residual disease, three months after treatment. This may translate into more time in remission and longer progression-free survival – outcomes that matter deeply to both patients and the healthcare professionals who treat them.' Patients who met the AMPLIFY inclusion criteria from both the Phase 3 GLOW (NCT03462719) and Phase 2 CAPTIVATE (NCT02910583) studies were included in this analysis, and, after matching and balancing the treatment cohorts, comparative analyses between the trials suggested that I+V significantly reduced the risk of progression or death by 47 percent when compared to patients treated with A+V (hazard ratio [HR]: 0.53; 95 percent confidence interval [CI]: 0.33-0.85; p =0.0085).1,3,4,5 The results also suggested that patients treated with I+V were almost twice (95 percent CI: 1.47-2.41; p <0.0001) and 2.4 times (95 percent CI: 1.78-3.12; p <0.0001) more likely to achieve uMRD than A+V at EOT+3, in PB and BM, respectively.1 The GLOW and CAPTIVATE FD cohorts were based on individual patient-level data with a median follow-up of approximately 4.5 years, while the A+V cohort used aggregate level data from the AMPLIFY study with a median follow-up of approximately 3.4 years.1 Results from final analysis of CAPTIVATE Long-term follow-up results from the Phase 2 CAPTIVATE study data were presented as a poster at the American Society of Clinical Oncology (ASCO) 2025 Annual Meeting (Poster presentation #219) and will also be presented as an encore oral presentation at EHA 2025 (Oral presentation #S156).6 The data reinforced the durable clinical benefit of frontline I+V.2 Phase 2 CAPTIVATE results demonstrated patients in the I+V FD cohort displayed a clinically meaningful PFS and overall survival (OS) vs the MRD-guided cohort.2 After a median follow-up of 68.9 months, the 5.5-year PFS and OS rates for all treated patients were 66 percent (95 percent CI: 58-72) and 97 percent (95 percent CI: 93-99), respectively.2 Additionally, the 5.5 year PFS rate in patients who achieved uMRD in the PB at the EOT was 71 percent (95 percent CI: 60-80).2 Furthermore, 1-year PFS and OS rates from the start of retreatment (with single-agent ibrutinib or FD I+V) were both 100 percent, whilst 2-year PFS and OS rates from the start of retreatment were 91 percent and 96 percent, respectively.2 No new safety signals were observed during the CAPTIVATE study since the previous follow-up, with COVID-19, diarrhoea and hypertension being the most frequently reported adverse events (AEs).2 In the total pooled CAPTIVATE population, 32 percent (n=64/202) of patients had progressive disease following 5.5 years of follow-up.2 Of the 53 patients with available samples, none had acquired resistance-associated mutations in BTK or PLCG2 .2 'The final analysis of CAPTIVATE highlights how ibrutinib continues to raise the bar in the treatment of chronic lymphocytic leukaemia, with durable minimal residual disease response, extended treatment-free intervals, and a tolerable safety profile,' said Ester in't Groen, EMEA Therapeutic Area Head Haematology, Johnson & Johnson Innovative Medicine. 'With the longest follow-up of any oral fixed-dose regimen, ibrutinib is setting a new standard for what patients and clinicians can expect from targeted therapies. We remain committed to advancing science in complex blood cancers and improving outcomes across the cancer care landscape.' 'The updates presented at EHA add to the growing body of evidence in support of ibrutinib, the most extensively studied Bruton's tyrosine kinase inhibitor, as the standard of care in the frontline treatment of chronic lymphocytic leukaemia,' said Jessica Vermeulen, Vice President, Lymphoma & Leukemia Disease Area Stronghold Leader, Johnson & Johnson Innovative Medicine. 'Offering patients not only more time, but also the option for treatment-free remissions continues to be our goal and we are proud of the incredible contribution ibrutinib has made since its first European approval in 2014.' About the MAIC analysis The objective of this analysis was to compare the progression-free survival (PFS) and undetectable minimal residual disease (uMRD) data from the fixed-duration (FD) ibrutinib + venetoclax (I+V) cohorts from the Phase 3 GLOW (NCT03462719) and Phase 2 CAPTIVATE (NCT02910583) studies against the Phase 3 AMPLIFY (NCT03836261) data.1 In absence of prospective head-to-head trials investigating different Bruton's tyrosine kinase inhibitors (BTKis) plus B-cell lymphoma 2 (Bcl-2) strategies, this study utilised matching-adjusted indirect comparison (MAIC) to obtain useful insights on comparative efficacy.1 Individual patient data from the FD I+V cohorts of the GLOW and CAPTIVATE studies were pooled and compared to aggregate published Intent-to-Treat (ITT) data of the acalabrutinib plus venetoclax (A+V) arm of the AMPLIFY study.1,3,4,5 A MAIC was performed following method published by Signorovitch et al. and guidelines from the National Institute for Health and Care Excellence (NICE).1,7 Patients who did not meet the inclusion criteria of AMPLIFY were excluded from the I+V pooled cohort to establish the I+V patient population for analysis.1 The remaining I+V patients were then reweighted so that the average baseline characteristics of the pooled I+V cohort matched those of the A+V patients in AMPLIFY.1 The reweighted outcomes from I+V were then compared to the reported outcomes for A+V using indirect treatment comparison.1 Relative effects were quantified using relative risk (RR) and hazard ratios (HR) with 95 percent confidence intervals.1 There are potential sources of bias that cannot be accounted for in MAIC, that should be considered when interpreting such data. Specifically, in this comparison, the measurement of progression was stricter in GLOW and CAPTIVATE, requiring computer or magnetic imaging regardless of suspected progression and the median follow-up was longer in I+V population.1 Both may have biased the PFS results in favour of A+V.1 Additionally, AMPLIFY reported multicolour flow cytometry use but with no details on the number of colours and comparability is assumed with the 8-colour assay used in I+V studies.1 As in any non-randomised comparison there may be additional unreported clinically important prognostic patient baseline characteristics which cannot be accounted for.1 For example, Cumulative Illness Rating Scale score data was not collected in CAPTIVATE and therefore could not be used in matching.1 About CAPTIVATE The Phase 2 CAPTIVATE study ( NCT02910583 ) evaluated previously untreated adult patients with chronic lymphocytic leukaemia (CLL), who were 70 years or younger, including patients with high-risk disease, in two cohorts with combined median age of 60 years: a minimal residual disease (MRD)-guided cohort (n=43) and an FD cohort (n=159; median age).2,4,8 Patients in the FD cohort received three cycles of ibrutinib lead-in followed by 12 cycles of I+V (oral ibrutinib [420 mg/d]; oral venetoclax [five-week ramp-up to 400 mg/d]) and the primary endpoint was complete response rate.4 In the MRD cohort, after completion of three cycles ibrutinib lead-in followed by 12 cycles I+V, patients with confirmed uMRD were randomly assigned to double-blind treatment with placebo, or continuous ibrutinib.4 The primary endpoint was one-year disease-free survival.4 About the GLOW study The GLOW study ( NCT03462719 ) is a randomised, open-label, Phase 3 trial that evaluated the efficacy and safety of frontline, FD I+V versus chlorambucil plus obinutuzumab in adult patients with CLL who are (a) ≥65 years old, or (b) 18-64 years old with a Cumulative Illness Rating Scale score of greater than six or creatinine clearance less than 70 mL/min, who had active disease requiring treatment per the International Workshop on CLL criteria.3 About ibrutinib Ibrutinib is a once-daily oral medication that is jointly developed and commercialised by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company.9 Ibrutinib blocks the Bruton's tyrosine kinase (BTK) protein, which is needed by normal and abnormal B-cells, including specific cancer cells, to multiply and spread.10 By blocking BTK, ibrutinib may help move abnormal B-cells out of their nourishing environments and inhibits their proliferation.11 Ibrutinib is approved in more than 100 countries and has been used to treat more than 325,000 patients worldwide.12 There are more than 50 company-sponsored clinical trials, including 18 Phase 3 studies, over 11 years evaluating the efficacy and safety of ibrutinib.10,13 In October 2021, ibrutinib was added to the World Health Organization's Model Lists of Essential Medicines (EML), which refers to medicines that address global health priorities and which should be available and affordable for all.14 Ibrutinib was first approved by the European Commission (EC) in 2014, and approved indications to date include:10 As a single agent or in combination with rituximab or obinutuzumab or venetoclax for the treatment of adult patients with previously untreated CLL As a single agent or in combination with bendamustine and rituximab (BR) for the treatment of adult patients with CLL who have received at least one prior therapy As a single agent for the treatment of adult patients with relapsed or refractory (RR) mantle cell lymphoma (MCL) As a single agent for the treatment of adult patients with Waldenström's macroglobulinaemia (WM) who have received at least one prior therapy, or in first line treatment for patients unsuitable for chemo-immunotherapy. In combination with rituximab for the treatment of adult patients with WM For a full list of adverse events and information on dosage and administration, contraindications and other precautions when using ibrutinib please refer to the Summary of Product Characteristics . About Chronic Lymphocytic Leukaemia CLL is typically a slow-growing blood cancer of the white blood cells.15 The overall incidence of CLL in Europe is approximately 4.92 cases per 100,000 persons per year and it is about 1.5 times more common in men than in women (based on individuals diagnosed 2000-2002).16 CLL is predominantly a disease of the elderly, with a median age of 72 years at diagnosis.17 While patient outcomes have dramatically improved in the last few decades, the disease is still characterised by consecutive episodes of disease progression and the need for therapy.18 Patients are often prescribed multiple lines of therapy as they relapse or become resistant to treatments.19 About Johnson & Johnson At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity. Learn more at . Follow us at . Janssen-Cilag International NV, Janssen Pharmaceutica NV, Janssen-Cilag Limited, Janssen Biotech, Inc., and Janssen Research & Development, LLC are Johnson & Johnson companies. Cautions Concerning Forward-Looking Statements This press release contains 'forward-looking statements' as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of daratumumab. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialise, actual results could vary materially from the expectations and projections of Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; competition, including technological advances, new products and patents attained by competitors; challenges to patents; changes in behaviour and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's most recent Annual Report on Form 10-K, including in the sections captioned 'Cautionary Note Regarding Forward-Looking Statements' and 'Item 1A. Risk Factors,' and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at or on request from Johnson & Johnson. Johnson & Johnson does not undertake to update any forward-looking statement as a result of new information or future events or developments. *Talha Munir, M.D., Consultant in Clinical Haematology at St James's Hospital, Leeds, United Kingdom, has provided consulting, advisory, and speaking services to Janssen-Cilag International NV; he has not been paid for any media work. 1 Munir T, et al., Cross-Study Comparison of Ibrutinib in Combination with Venetoclax (I+V) vs Acalabrutinib in Combination with Venetoclax (A+V) in Subjects with Previously Untreated Chronic Lymphocytic Leukemia (CLL). Poster presentation at 2025 European Hematology Association (EHA) Congress; June 12–15, 2025. [Poster PF587]. 2 Ghia P, et al. Final Analysis of Fixed-Duration Ibrutinib + Venetoclax for Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL) in the Phase 2 CAPTIVATE Study. Oral presentation at 2025 European Hematology Association (EHA) Congress; June 12–15, 2025. [Oral S156]. 3 A Study of the Combination of Ibrutinib Plus Venetoclax Versus Chlorambucil Plus Obinutuzumab for the First-line Treatment of Participants With Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL) (GLOW). Available at: Last accessed: June 2025. 4 Ibrutinib Plus Venetoclax in Subjects With Treatment-naive Chronic Lymphocytic Leukemia /Small Lymphocytic Lymphoma (CLL/SLL) (CAPTIVATE). Available at: Last accessed: June 2025. 5 Study of Acalabrutinib (ACP-196) in Combination With Venetoclax (ABT-199), With and Without Obinutuzumab (GA101) Versus Chemoimmunotherapy for Previously Untreated CLL (AMPLIFY). Available at: Last accessed: June 2025. 6 Ghia P, et al. Final Analysis of Fixed-Duration Ibrutinib + Venetoclax for Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma in the Phase 2 CAPTIVATE Study. Poster presentation at 2025 American Society of Clinical Oncology (ASCO) Annual Meeting; May 30–June 3, 2025. [Poster #219]. 7 Signorovitch JE, et al. Matching-adjusted Indirect Comparisons: A New Tool for Timely Comparative Effectiveness Research. Value Health, 2012; 15: 940-947. 8 Jacobs R, et al., Outcomes in High-risk Subgroups After Fixed-Duration Ibrutinib + Venetoclax for Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma: Up To 5.5 years of Follow-up in the Phase 2 CAPTIVATE Study. Poster presentation at 2024 European Hematology Association (EHA) Hybrid Congress; June 13–16, 2024. [Poster P675]. 9 European Medicines Agency. IMBRUVICA Summary of Product Characteristics. April 2025. Available at: Last accessed: June 2025. 10 Turetsky A, et al. Single cell Imaging of Bruton's tyrosine kinase using an Irreversible Inhibitor. Sci Rep. 2014; 4: 4782. 11 de Rooij MF, et al. The Clinically Active BTK Inhibitor PCI-32765 targets B-cell Receptor- and Chemokine-controlled Adhesion and Migration in Chronic Lymphocytic Leukemia. Blood. 2012; 119(11): 2590-2594. 12 J&J Data on File (RF-465355). Patients Treated on Imbruvica Worldwide. May 2025. 13 Pollyea DA, et al. A Phase I Dose Escalation Study of the Btk Inhibitor PCI-32765 in Relapsed and Refractory B Cell Non-Hodgkin Lymphoma and Use of a Novel Fluorescent Probe Pharmacodynamic Assay. Blood. 2009; 114(22): 3713. 14 World Health Organization. WHO Prioritizes Access to Diabetes and Cancer Treatments in New Essential Medicines Lists. Available at: Last accessed: June 2025. 15 American Cancer Society. What is Chronic Lymphocytic Leukemia? Available at: Last accessed: June 2025. 16 Sant M, et al. Incidence of Hematologic Malignancies in Europe by Morphologic Subtype: Results of the HAEMACARE project. Blood. 2010; 116:3724–34. 17 Eichhorst B, et al. Chronic Lymphocytic Leukaemia: ESMO Clinical Practice Guidelines for Diagnosis, Treatment and Follow-up Ann Oncol. 2021; 32(1): 23-33. 18 Moreno C. Standard Treatment Approaches for Relapsed/refractory Chronic Lymphocytic Leukemia after Frontline Chemoimmunotherapy. Hematology Am Soc Hematol Educ Program. 2020; 2020: 33-40. 19 Bewarder M, et al. Current Treatment Options in CLL. Cancers (Basel). 2021;13(10): 2468. CP-523458 June 2025 Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same.
