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Associated Press
4 days ago
- Business
- Associated Press
Ifinatamab Deruxtecan Granted Breakthrough Therapy Designation by U.S. FDA for Patients with Pretreated Extensive-Stage Small Cell Lung Cancer
BASKING RIDGE, N.J. & RAHWAY, N.J.--(BUSINESS WIRE)--Aug 18, 2025-- Ifinatamab deruxtecan (I-DXd) has been granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with extensive-stage small cell lung cancer with disease progression on or after platinum-based chemotherapy. Ifinatamab deruxtecan is a specifically engineered, potential first-in-class B7-H3 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being jointly developed by Daiichi Sankyo and Merck (NYSE: MRK), known as MSD outside of the United States and Canada. The FDA BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. The medicine is required to have shown encouraging preliminary clinical results that demonstrate substantial improvement on a clinically significant endpoint over currently available medicines. The FDA granted the BTD based on data from the IDeate-Lung01 phase 2 trial, with support from the IDeate-PanTumor01 phase 1/2 trial. Results from the primary analysis of IDeate-Lung01 will be presented in a late-breaking oral presentation at the IASLC 2025 World Conference on Lung Cancer hosted by the International Association for the Study of Lung Cancer (#WCLC25). This is the first BTD for ifinatamab deruxtecan and represents the first BTD since the start of the Daiichi Sankyo and Merck collaboration. 'This Breakthrough Therapy Designation granted by the FDA to ifinatamab deruxtecan highlights the urgent need for new treatment options for patients with pretreated extensive-stage small cell lung cancer,' said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. 'We are committed to advancing this medicine with the goal of bringing the first B7-H3 directed antibody drug conjugate to patients in order to transform the outcomes of those facing this aggressive disease.' 'Patients living with extensive-stage small cell lung cancer often have limited therapeutic options following disease progression after standard of care treatments,' said Eliav Barr, MD, Senior Vice President, Head of Global Clinical Development and Chief Medical Officer, Merck Research Laboratories. 'This Breakthrough Therapy Designation reinforces our confidence in the promise of ifinatamab deruxtecan to play an important role in the treatment of extensive-stage small cell lung cancer, and we are looking forward to sharing data at the upcoming IASLC 2025 World Conference on Lung Cancer that show the potential of this novel option.' In the first part of the trial (dose optimization), patients were randomized 1:1 to receive ifinatamab deruxtecan 8 or 12 mg/kg intravenously Q3W. In the second part of the trial (dose expansion), patients received ifinatamab deruxtecan 12 mg/kg intravenously Q3W. The primary endpoint is objective response rate (ORR) as assessed by blinded independent central review (BICR) per RECIST v1.1. Secondary endpoints included duration of response, progression-free survival, disease control rate, time to response, overall survival, pharmacokinetics and safety. Intracranial ORR was assessed by BICR as an exploratory analysis. IDeate-Lung01 enrolled 187 patients in Asia, Europe and North America. For more information about the trial, visit The phase 1 part of the trial (dose escalation) is assessing the safety and tolerability of increasing doses of ifinatamab deruxtecan to determine the maximum tolerated dose and recommended dose for expansion (RDE). The phase 2 part of the trial (dose expansion) is evaluating the safety and efficacy of ifinatamab deruxtecan at the RDE of 12 mg/kg in patients with squamous non-small cell lung cancer, metastatic castration-resistance prostate cancer or esophageal squamous cell carcinoma. The dose escalation part of the trial is evaluating dose-limiting toxicity and safety. The dose expansion part of the trial is evaluating overall response rate, duration of response, disease control rate, progression-free survival, overall survival and safety. Pharmacokinetic endpoints, exploratory biomarker and immunogenicity endpoints will also be assessed. IDeate-PanTumor01 enrolled approximately 250 patients in Asia and North America. For more information about the trial, visit Ifinatamab deruxtecan has been granted orphan drug designation by the U.S. FDA, European Commission, Japan Ministry of Health, Labour and Welfare and Taiwan Food and Drug Administration for the treatment of SCLC. The ADC platform furthest in clinical development is Daiichi Sankyo's DXd ADC Technology where each ADC consists of a monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. The DXd ADC portfolio currently consists of ENHERTU ®, a HER2 directed ADC, and DATROWAY ®, a TROP2 directed ADC, which are being jointly developed and commercialized globally with AstraZeneca. Patritumab deruxtecan (HER3-DXd), a HER3 directed ADC, ifinatamab deruxtecan (I-DXd), a B7-H3 directed ADC, and raludotatug deruxtecan (R-DXd), a CDH6 directed ADC, are being jointly developed and commercialized globally with Merck & Co., Inc, Rahway, N.J., USA. DS-3939, a TA-MUC1 directed ADC, is being developed by Daiichi Sankyo. The second Daiichi Sankyo ADC platform consists of a monoclonal antibody attached to a modified pyrrolobenzodiazepine (PBD) payload. DS-9606, a CLDN6 directed PBD ADC, is the first of several planned ADCs in clinical development utilizing this platform. Ifinatamab deruxtecan, patritumab deruxtecan, raludotatug deruxtecan, DS-3939 and DS-9606 are investigational medicines that have not been approved for any indication in any country. Safety and efficacy have not been established. About Merck At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit and connect with us on X (formerly Twitter), Facebook, Instagram, YouTube and LinkedIn. Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company's ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company's patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions. The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company's Annual Report on Form 10-K for the year ended December 31, 2024, and the company's other filings with the Securities and Exchange Commission (SEC) available at the SEC's Internet site ( ). ____________________________ References: View source version on CONTACT: Media Contacts:Daiichi Sankyo Global/US: Jennifer Brennan Daiichi Sankyo [email protected] +1 (908) 900-3183 (mobile)Japan: Daiichi Sankyo Co., Ltd. [email protected] Relations Contact: [email protected] Media: Julie Cunningham (617) 519-6264 [email protected] McArdle (908) 447-9453 [email protected]: Peter Dannenbaum (732) 594-1579 [email protected] Graziano (732) 594-1583 [email protected] KEYWORD: UNITED STATES NORTH AMERICA NEW JERSEY INDUSTRY KEYWORD: SCIENCE BIOTECHNOLOGY RESEARCH PHARMACEUTICAL ONCOLOGY HEALTH FDA CLINICAL TRIALS SOURCE: Daiichi Sankyo Copyright Business Wire 2025. PUB: 08/18/2025 07:00 AM/DISC: 08/18/2025 07:00 AM
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
03-06-2025
- Business
- Yahoo
ASCO 2025 Oral Presentation: Innovent Biologics Announces Updated Data of IBI343 (Novel Anti-CLDN18.2 ADC) From the Phase 1 Clinical Study in Patients with Advanced Pancreatic Cancer
SAN FRANCISCO and SUZHOU, China, June 2, 2025 /PRNewswire/ -- Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncology, cardiovascular and metabolic, autoimmune, ophthalmology and other major diseases, updated the Phase 1 study results of IBI343, a novel anti-CLDN18.2 ADC, for the treatment of advanced pancreatic cancer at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting. With extended follow-up and more mature data on progression-free survival (PFS) and overall survival (OS), IBI343 has demonstrated promising therapeutic efficacy in patients with CLDN18.2-positive advanced pancreatic cancer. These encouraging results suggest the potential of IBI343 in this challenging-to-treat malignancy. Supported by these robust clinical findings, IBI343 has been granted Breakthrough Therapy Designation (BTD) by China's National Medical Products Administration (NMPA). Concurrently, the Phase 1 clinical trial of IBI343 is also being conducted in the United States, where the drug candidate has received Fast Track Designation (FTD) from the U.S. Food and Drug Administration (FDA). Pancreatic cancer is one of the most aggressive malignancies worldwide. Most patients are diagnosed in the middle and late stages and often develop resistance to standard chemotherapy, resulting in a 5-year survival rate of less than 10%1. According to the GLOBOCAN 2022 statistics2, there are approximately 510,000 new cases and 467,000 deaths globally from pancreatic cancer each year, with China accounting for 120,000 new cases and 110,000 deaths annually. This Phase 1/1b study is a multi-regional, dose escalation and expansion clinical trial (NCT05458219). Preliminary data were presented at ASCO 2025 and ESMO Asia 2024 and the updated results from the study's dose-expansion cohort were presented at the 2025 ASCO as follows: As of March 14, 2025, a total of 83 patients with pancreatic cancer had received at least one dose of IBI343 with a median follow-up time of 11.1 months. As the data cutoff date, in patients with CLDN18.2 1+2+3+≥60% expression treated at the 6mg/kg dose (N=44), the confirmed overall objective response rate (cORR) was 22.7% and the disease control rate (DCR) was 81.8%. The median progression-free survival (mPFS) was 5.4 months, and the median overall survival (mOS) was 9.1 months. Among them, 17 patients had received only one line of prior treatment, achieving a mPFS of 5.4 months and a mOS of 12.1 months; and 18 patients had received two lines of prior treatment, the mPFS was 5.3 months and the mOS was 9.1 months. The updated safety results demonstrated the favorable safety profile of IBI343 with a consistently low rate of gastrointestinal toxicity and no new safety signals. 98.8% of the patients experienced treatment-emergent adverse events (TEAEs), with the most common TEAEs being anemia, neutrophil count decreased, and white blood cell count decreased. Notably, no ≥ grade 3 nausea and vomiting occurred. Professor Xianjun Yu from Fudan University Cancer Hospital, said, "Pancreatic cancer is one of the most malignant tumors of the digestive tract. Most patients are already in the advanced stage when diagnosed, and the 5-year survival rate is only about 10%1. Currently, chemotherapy is still the main first- and second-line treatment for advanced pancreatic cancer. The clinical options for second-line treatment are particularly limited, with a chemotherapy response rate of only 6-16%, median progression free survival of 2 to 5 months, and a median survival of approximately 6 to 9 months3, representing an urgent clinical need. With longer follow-up, the mature PFS and OS data from the latest IBI343 update demonstrate promising therapeutic potential, suggesting a breakthrough in this difficult-to-treat malignancy." Dr. Hui Zhou, Senior Vice President of Innovent, said, "We are pleased to present an oral update on IBI343's clinical data at this year's ASCO conference. With the unique Fc-silent antibody design, stable linker and potent TOPO1i payload, IBI343 is the first ADC candidate to show encouraging efficacy and a favorable safety profile in the treatment of advanced pancreatic cancer. We hope to continue advancing the clinical trials of IBI343 for pancreatic cancer patients. Innovent will leverage its unique strengths in R&D innovation and clinical translation to develop a new generation of globally competitive oncology - focused innovative pipeline to benefit patients worldwide.." About IBI343（Anti-CLDN18.2 ADC） IBI343 is a recombinant human anti-CLDN18.2 monoclonal antibody-drug conjugate (ADC) developed by Innovent Biologics. IBI343 binds to the CLDN18.2-expressing tumor cells, the CLDN18.2 dependent ADC internalization will occur and the drug is released resulting in DNA damage and eventually apoptosis of the tumor cells. The freed drug can also diffuse across the plasma membrane to reach and kill the neighboring cells, resulting in "bystander killing effect". As an innovative TOPO1i ADC, IBI343 has demonstrated tolerable safety and encouraging efficacy signals in Phase 1 clinical studies. The therapeutic potential of IBI343 is currently being explored in tumor types such as gastric and pancreatic cancer. The Phase 3 trial of IBI343 for advanced gastric / gastroesophageal junction adenocarcinoma is now recruiting patients. The relevant indication has been included in China's NMPA breakthrough therapy list. IBI343's Phase 1 trial for advanced pancreatic ductal adenocarcinoma is enrolling patients in an multi-regional study. This indication has received Fast Track designation from the FDA and been included in the NMPA's BTD list. About Innovent Biologics Innovent is a leading biopharmaceutical company founded in 2011 with the mission to empower patients worldwide with affordable, high-quality biopharmaceuticals. The company discovers, develops, manufactures and commercializes innovative medicines that target some of the most intractable diseases. Its pioneering therapies treat cancer, cardiovascular and metabolic, autoimmune and eye diseases. Innovent has launched 15 products in the market. It has 3 new drug applications under regulatory review, 4 assets in Phase III or pivotal clinical trials and 15 more molecules in early clinical stage. Innovent partners with over 30 global healthcare companies, including Eli Lilly, Sanofi, Incyte, LG Chem and MD Anderson Cancer Center. Guided by the motto, "Start with Integrity, Succeed through Action," Innovent maintains the highest standard of industry practices and works collaboratively to advance the biopharmaceutical industry so that first-rate pharmaceutical drugs can become widely accessible. For more information, visit or follow Innovent on Facebook and LinkedIn. Statement: 1. Innovent Biologics does not recommend the use of unapproved drugs/indications. 2. Ramucirumab injection (Ciranza®), selpercatinib capsules (Ritu®) and pirtobrutinib tablets (Capra®) were developed by Eli Lilly and Company Forward-Looking Statements This news release may contain certain forward-looking statements that are, by their nature, subject to significant risks and uncertainties. The words "anticipate", "believe", "estimate", "expect", "intend" and similar expressions, as they relate to Innovent Biologics, Inc. ("Innovent" or "Company"), are intended to identify certain of such forward-looking statements. The Company does not intend to update these forward-looking statements regularly. These forward-looking statements are based on the existing beliefs, assumptions, expectations, estimates, projections and understandings of the management of the Company with respect to future events at the time these statements are made. These statements are not a guarantee of future developments and are subject to risks, uncertainties and other factors, some of which are beyond the Company's control and are difficult to predict. Consequently, actual results may differ materially from information contained in the forward-looking statements as a result of future changes or developments in our business, the Company's competitive environment and political, economic, legal and social conditions. The Company, the Directors and the employees of the Company assume (a) no obligation to correct or update the forward-looking statements contained in this site; and (b) no liability in the event that any of the forward-looking statements does not materialize or turn out to be incorrect. References 1 Siegel RL, Miller KD, Fuchs HE, et al. Cancer statistics, 2022. CA Cancer J Clin. 2022;72:7-33. doi: 10.3322/caac.21708. 2 Bray F, Laversanne M, Sung H, et al. Global cancer statistics 2022: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2024 May-Jun;74(3):229-263. 3 Jemal A, Bray F, Center MM, et al. Global cancer stastics. CA Cancer J Clin, 2011, 61(2): 69-90. View original content: SOURCE Innovent Biologics
Yahoo
26-05-2025
- Health
- Yahoo
Abbisko Therapeutics Receives CDE Approval of Breakthrough Therapy Designation for Irpagratinib (ABSK011) in the Treatment of HCC
SHANGHAI, May 26, 2025 /PRNewswire/ -- Abbisko Therapeutics (HKEX Code: 02256) announced that its self-developed, highly selective small molecule FGFR4 inhibitor, irpagratinib (ABSK011), has received approval of Breakthrough Therapy Designation from the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for the treatment of Hepatocellular Carcinoma (HCC). Irpagratinib is the first therapeutic agent to leverage molecularly defined biomarkers for precision-targeted treatment in patients with HCC. During clinical trials, innovative drugs or modified new drugs intended to prevent or treat life-threatening diseases or conditions that severely impact quality of life—where no effective prevention or treatment exists, or where substantial evidence shows significant clinical advantages over existing therapies—may be eligible to apply for the CDE's Breakthrough Therapy Designation program[1]. The approval of irpagratinib for Breakthrough Therapy Designation is based on its promising Phase I clinical trial data. Patients with advanced or unresectable HCC currently lack effective treatment options following treatment with ICI- and mTKI-based therapies. Those with FGF19 overexpression often face significantly worse prognosis, and thus new treatment options are urgently needed. The Breakthrough Therapy Designation granted to irpagratinib will expedite its subsequent application and approval process with the CDE, bringing renewed hope and transformative possibilities to patients. Recently, Abbisko launched a pivotal registrational clinical study of irpagratinib for the treatment of HCC patients with FGF19 overexpression at Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology, and Nanjing Tianyinshan Hospital. About Irpagratinib (ABSK-011) Irpagratinib is a highly-selective FGFR4 small molecule inhibitor designed to target overexpression of the FGF19 signaling pathway. Several epidemiological studies indicate that approximately 30% of HCC patients worldwide exhibit FGF19 overexpression. Development of targeted therapies against FGFR4 represent an innovative and novel approach to the treatment of HCC. To date, no FGFR4 inhibitor has been granted regulatory approval globally. According to Frost & Sullivan, irpagratinib is expected to become the first breakthrough treatment for the treatment of HCC patients with FGF19 overexpression. In addition to monotherapy, Abbisko Therapeutics is exploring irpagratinib in combination with atezolizumab, an anti-PD-L1 antibody manufactured by F. Hoffmann-La Roche and Roche (China), in a Phase II study. At the previous 2024 ESMO GI Congress, Abbisko presented clinical data demonstrating 220mg irpagratinib BID in combination with atezolizumab achieved a 50% objective response rate (ORR) in FGF19+ HCC patients who had previously received immune checkpoint inhibition therapy. About Abbisko Therapeutics Founded in April 2016, Abbisko Therapeutics Co., Ltd. (HKEX: is an oncology-focused biopharmaceutical company based in Shanghai that is dedicated to the discovery and development of innovative medicines to treat unmet medical needs in China and globally. The Company was established by a group of seasoned drug hunters with rich research & development and managerial expertise from top multinational pharmaceutical companies. Since its founding, Abbisko Therapeutics has built an extensive pipeline of innovative programs focused on precision oncology and immuno-oncology. Please visit for more information. References: 1. NMPA突破性治疗药物审评工作程序. View original content: SOURCE Abbisko Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
20-05-2025
- Business
- Yahoo
Viridian Therapeutics Announces Positive Long-Term Durability Data from the Veligrotug Phase 3 THRIVE Clinical Trial in Patients with Active Thyroid Eye Disease (TED)
- 70% of patients treated with veligrotug in THRIVE who were proptosis responders at week 15 maintained their response at week 52 - - Veligrotug recently received Breakthrough Therapy Designation (BTD), supporting eligibility for Priority Review; the BTD request was based on veligrotug's (i) consistent and robust improvement and resolution of diplopia in chronic TED, and (ii) rapid onset of proptosis response - - Biologics License Application (BLA) submission for veligrotug is on track for second half 2025 - - Actively preparing organization for planned U.S. commercial launch in 2026 - WALTHAM, Mass., May 20, 2025--(BUSINESS WIRE)--Viridian Therapeutics, Inc. (Nasdaq: VRDN), a biopharmaceutical company focused on discovering, developing, and commercializing potential best-in-class medicines for serious and rare diseases, today announced positive long-term durability data from the THRIVE phase 3 clinical trial of veligrotug ("veli"), an intravenously delivered anti-insulin-like growth factor-1 receptor (IGF-1R) antibody, in patients with active thyroid eye disease (TED). TED is an autoimmune condition characterized by inflammation, growth, and damage to tissues around and behind the eye. The THRIVE phase 3 clinical trial in active TED evaluated 5 infusions of veli or placebo every three weeks with primary topline analysis at week 15 and then followed patients through week 52. Positive Veligrotug Durability at 52 Weeks 70% of veligrotug patients (21/30) in THRIVE, who were proptosis responders at week 15 and continued follow-up to the end of the study at week 52, maintained their proptosis response. Maintenance of response is defined as responders at week 15 who still had at least a 2-millimeter (mm) reduction in proptosis compared to baseline at week 52, without worsening in the fellow eye (≥2 mm increase), as measured by exophthalmometry. There were no changes to the safety profile in the follow-up period. The vast majority of adverse events reported at the week 15 primary analysis had resolved by week 52. "We view the strength of today's durability and safety resolution data as reinforcing veli's strong and consistently robust clinical profile," said Steve Mahoney, Viridian's President and CEO. "We believe that the totality of veligrotug's clinical data continues to demonstrate its potential to be the treatment-of-choice for patients living with TED. We believe these data, together with a streamlined dosing regimen of five infusions, position veli to become a market leading TED therapeutic, if approved. We continue to make great progress towards submitting the BLA in the second half of this year and preparing for a potential launch in 2026." Robust Veligrotug Topline Clinical Profile for Active and Chronic TED As announced in late 2024, veligrotug met all of its primary and secondary endpoints and was generally well-tolerated in its pivotal phase 3 clinical trials, THRIVE and THRIVE-2, for active and chronic TED, respectively. Veligrotug demonstrated a rapid onset of treatment effect and statistically significant and clinically meaningful reduction and resolution of diplopia in both clinical trials. THRIVE-2 was the first data set from a global phase 3 clinical trial in chronic TED patients to demonstrate statistically significant diplopia response and resolution. Together, THRIVE and THRIVE-2 comprise the largest pivotal program to date in TED. About Veligrotug Veligrotug is an intravenously (IV) delivered, anti-insulin-like growth factor-1 receptor (IGF-1R) antibody in phase 3 development for thyroid eye disease, with the potential to be the IV treatment-of-choice for active and chronic TED patients. IGF-1R is a clinically and commercially validated target for thyroid eye disease (TED) with U.S. revenues of approximately $2 billion in 2024. Veligrotug has the potential to improve patient experience with a differentiated dosing regimen that features a shorter infusion time and fewer infusions compared to the currently approved and marketed IGF-1R inhibitor. In its pivotal phase 3 clinical trials, THRIVE and THRIVE-2, veligrotug met all of its primary and secondary endpoints. Veligrotug demonstrated a rapid onset of treatment effect and statistically significant and clinically meaningful reduction and resolution of diplopia in both clinical trials. THRIVE-2 was the first demonstration in a global phase 3 clinical trial of a statistically significant diplopia response and resolution in chronic TED patients. Veligrotug was generally well tolerated. Viridian believes that the robust veligrotug clinical profile has the potential to establish a strong position in the TED commercial market, if approved, and may help facilitate the introduction of VRDN-003, its potential best-in-class subcutaneous IGF-1R antibody for TED. About Viridian Therapeutics Viridian is a biopharmaceutical company focused on discovering, developing and commercializing potential best-in-class medicines for patients with serious and rare diseases. Viridian's expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates for previously validated drug targets in commercially established disease areas. Viridian is advancing multiple candidates in the clinic for the treatment of patients with thyroid eye disease (TED). The company is conducting a pivotal program for veligrotug (VRDN-001), including two global phase 3 clinical trials (THRIVE and THRIVE-2), to evaluate its efficacy and safety in patients with active and chronic TED. Both THRIVE and THRIVE-2 reported positive topline data, meeting all the primary and secondary endpoints of each study. Viridian is also advancing VRDN-003 as a potential best-in-class subcutaneous therapy for the treatment of TED, including two ongoing global phase 3 pivotal clinical trials, REVEAL-1 and REVEAL-2, to evaluate the efficacy and safety of VRDN-003 in patients with active and chronic TED. In addition to its TED portfolio, Viridian is advancing a novel portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008, which has the potential to be developed in multiple autoimmune diseases. Viridian is based in Waltham, Massachusetts. For more information, please visit Follow Viridian on LinkedIn and X. Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as, but not limited to, "anticipate," "believe," "become," "continue," "could," "design," "estimate," "expect," "intend," "may," "might," "on track," "plan," "potential," "predict," "project," "should," "target," "will," or "would" or other similar terms or expressions that concern our expectations, plans and intentions. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations, and assumptions. Forward-looking statements include, without limitation, statements regarding: clinical development and anticipated commercialization of Viridian's product candidates, including veligrotug (formerly VRDN-001) and VRDN-003; the potential utility, efficacy, potency, safety, clinical benefits, clinical response, convenience, and number of indications of veligrotug and VRDN-003, including Viridian's view of the strength of the THRIVE durability and safety resolution data and veligrotug's robust clinical profile; veligrotug's potential to be the IV treatment-of-choice for active and chronic TED; the impact of Breakthrough Therapy Designation, including eligibility for Priority Review, or any other FDA designations; regulatory interactions and anticipated timing of regulatory submissions, including the anticipated BLA submission for veligrotug in the second half of 2025; potential market sizes and market opportunities for Viridian's product candidates, including Viridian's belief that veligrotug is positioned to become a market leading TED therapeutic, if approved; Viridian's product candidates potentially being best-in-class; whether veligrotug will serve an unmet need; veligrotug's potential to improve patient experience over existing therapies; and Viridian's expectations regarding the potential commercialization of veligrotug and VRDN-003, if approved, including the potential U.S. launch of veligrotug in 2026. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to: potential utility, efficacy, potency, safety, clinical benefits, clinical response, and convenience of Viridian's product candidates; that results or data from completed or ongoing clinical trials may not be representative of the results of ongoing or future clinical trials; that preliminary data may not be representative of final data; expectations and changes regarding the timing for regulatory filings; regulatory interactions; uncertainty and potential delays related to clinical drug development; the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates; competition from other therapies or products; estimates of market size; our future operating results and financial performance; Viridian's intellectual property position; that our product candidates may not be commercially successful, if approved; and other risks described from time to time in the "Risk Factors" section of our filings with the Securities and Exchange Commission (SEC), including those described in our most recent Annual Report on Form 10-K or Quarterly Report on Form 10-Q, as applicable, and supplemented from time to time by our Current Reports on Form 8-K. Any forward-looking statement speaks only as of the date on which it was made. Neither the company, nor its affiliates, advisors, or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing the company's views as of any date subsequent to the date hereof. View source version on Contacts IR@ Media@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
