This is a long-term follow-up study for participants treated with Galapagos (GLPG) CAR T-cell therapies to evaluate the long-term safety and efficacy of GLPG CAR T-cell products for 15 years post infusion.
Per Health Authorities guidelines for gene therapy medicinal products that utilize integrating vectors (e.g. lentiviral vectors), long term safety and efficacy follow up of treated patients is required. The purpose of this study is to monitor all participants exposed to GLPG CAR T-cell therapies for 15 years following their last CAR T-cell infusion to assess the risk of delayed adverse events (AEs) and the long-term benefit/risk profile and to monitor for replication-competent lentivirus (RCL) and CAR-T cell persistence.

开始日期2024-09-09

申办/合作机构

Galapagos NV

NCT06561425 / Recruiting临床1/2期

A Phase I/II, Multicenter Study Evaluating the Feasibility, Safety, and Efficacy of Point-of-care Manufactured GLPG5101 (19CP02) in Subjects With Relapsed/Refractory B-cell Non-Hodgkin Lymphoma

This study is evaluating whether an experimental treatment called GLPG5101 helps to treat non-Hodgkin lymphoma (NHL) and if it is safe to use.
This study will be carried out in 2 phases:
* The first phase is to see which dose of GLPG5101 works best with the least number of side effects.
* In the second phase, all participants will get the best dose of the first phase.

开始日期2022-03-09

申办/合作机构

Galapagos NV

CTIS2023-504422-19-00 / Not yet recruiting

- GLPG5101-CL-104

开始日期-

申办/合作机构

Galapagos NV

100 项与 GLPG-5101 相关的临床结果

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100 项与 GLPG-5101 相关的转化医学

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100 项与 GLPG-5101 相关的专利（医药）

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项与 GLPG-5101 相关的文献（医药）

2021-08-01·Journal of Managed Care & Specialty Pharmacy

Sustained long-term benefits of patient support program participation in immune-mediated diseases: improved medication-taking behavior and lower risk of a hospital visit.

Article

作者: Rubin, David T ; Brixner, Diana ; Mease, Philip ; Fendrick, A Mark ; Mittal, Manish ; Davis, Matthew ; Liu, Harry

BACKGROUND: Patient support programs (PSPs) improve medication-taking behavior in the first 12 months of treatment for patients with immune-mediated diseases, but it is unknown if these benefits are sustained. As immune-mediated diseases continue to increase in prevalence and economic burden, understanding the potential value of PSPs in helping patients adhere to their long-term treatment plan and avoid costly hospital visits is crucial. Launched nationally in 2015, HUMIRA Complete (a PSP for adalimumab patients) provides an opportunity to study long-term effects of PSP participation, including the impact on medication-taking behavior and hospital visits. OBJECTIVE: To evaluate the sustained relationship between PSP participation, long-term medication-taking behavior, and hospital visits. METHODS: A longitudinal, retrospective matched-cohort study was conducted of patients initiating adalimumab between January 2015 and February 2016 with or without enrolling in the PSP, using patient-level data from the HUMIRA Complete PSP linked with Symphony Health claims. The sample included adult, commercially insured patients diagnosed with an indicated disease who were biologic-naive and had data available for ≥ 6 months before and ≥ 12 months after initiating adalimumab. Adherence (proportion of days covered) and hospital visits were assessed at 12, 24, and 36 months for patients with sufficient follow-up data. Multivariable generalized models estimated differences between cohorts, controlling for baseline characteristics and hospital visits. Duration of persistence and time to a hospital visit were compared using Kaplan-Meier analyses. Hazard ratios were estimated using multivariable Cox proportional hazards models. RESULTS: The matched cohort included 2,268 patients (1,134 per cohort), and patient attrition was similar across cohorts. The PSP cohort consistently demonstrated higher adalimumab adherence than the non-PSP cohort at 12 (64.8% vs. 50.1%, P < 0.0001; 29% greater), 24 (49.4% vs. 38.4%; P < 0.0001; 29% greater), and 36 (39.4% vs. 35.1%; P = 0.02; 12% greater) months. PSP participation was associated with a 30% lower hazard of discontinuation (P < 0.0001), and median duration of persistence was 4.8 months longer for the PSP cohort (13.2 vs. 8.4 months; P < 0.0001). The PSP cohort had lower rates of hospital visits at 12 (30% vs. 37%; P < 0.001; 19% lower), 24 (44% vs. 53%; P = 0.01; 17% lower), and 36 (55% vs. 65%; P < 0.01; 16% lower) months, and PSP participation was associated with a 25% lower hazard of a hospital visit (P < 0.0001). Median time to a hospital visit was 10.8 months longer for the PSP cohort (32.7 vs. 21.9 months; P < 0.0001). Findings were consistent across therapeutic areas: hazard of a hospital visit was 28%, 27%, and 37% lower for rheumatology, gastroenterology, and dermatology patients participating in the PSP (all P < 0.05). CONCLUSIONS: Patients with immune-mediated diseases receiving adalimumab and utilizing this PSP had improved long-term medication-taking behavior and lower risk of hospital visits, demonstrating the potential of PSPs to improve patient outcomes and lower the burden to the health care system. DISCLOSURES: Design, study conduct, and financial support for the study were provided by AbbVie Inc., which participated in the interpretation of data, review, and approval of the manuscript. Fendrick has received personal fees from Merck, AstraZeneca, Trizetto, Amgen, Lilly, AbbVie, Johnson & Johnson, and Sanofi; grants from the National Pharmaceutical Council, PhRMA, the Gary and Mary West Health Foundation, the states of New York and Michigan, the Laura and John Arnold Foundation, the Robert Wood Johnson Foundation, and the Agency for Healthcare Research and Quality; and equity in Zansors, Sempre Health, Wellth, and V-BID Health. Brixner has received consulting fees from AbbVie, Novartis, Xcenda, Elevar Therapeutics, Sanofi, UCB Pharma, and the Millcreek Outcomes Group. Rubin has received consulting fees from AbbVie, Abgenomics, Allergan Inc., Amgen, Celgene Corporation, Forward Pharma, Genentech/Roche, Janssen Pharmaceuticals, Merck & Co., Miraca Life Sciences, Mitsubishi Tanabe Pharma Development America, Napo Pharmaceuticals, Pfizer, Salix Pharmaceuticals Inc., Samsung Bioepis, Sandoz Pharmaceuticals, Shire, Takeda, and Target Pharmaceuticals; and research support from AbbVie, Genentech/Roche, Janssen Pharmaceuticals, Prometheus Laboratories, Shire, Takeda, and UCB Pharma. Mease has received grant/research support from AbbVie, Amgen, BMS, Celgene, Janssen, Lilly, Merck, Novartis, Pfizer, SUN Pharma, and UCB; consulting fees from AbbVie, Amgen, BMS, Boehringer Ingelheim, Celgene, Galapagos, Genentech, Gilead, GlaxoSmithKline, Janssen, Lilly, Novartis, Pfizer, SUN Pharma, and UCB; and has served on the speakers bureau for AbbVie, Amgen, Celgene, Janssen, Lilly, Novartis, Pfizer, and UCB. Liu has no financial conflict of interest. Davis is an employee of Medicus Economics, which received payment from AbbVie to participate in this research. Mittal is an employee and stockholder of AbbVie. This study used a cohort of patients previously described in Brixner D, Rubin DT, Mease P, et al. Patient support program increased medication adherence with lower total health care costs despite increased drug spending. J Manag Care Spec Pharm. 2019 Jul;25(7):770-79 (doi: 10.18553/jmcp.2019.18443). As such, the sample selection and select baseline characteristics and 12-month outcomes have been published previously; however, the hospital visit outcomes and the longer-term medication-taking behavior outcomes have not been previously published or presented.

项与 GLPG-5101 相关的新闻（医药）

2024-11-28

·PRNewswire

Follicular Lymphoma Clinical Trial Pipeline Appears Robust With 50+ Key Pharma Companies Actively Working in the Therapeutics Segment | DelveInsight

Advances in immunotherapy have ushered in a new era of treatment for follicular lymphoma (FL), particularly for patients who have experienced relapse after conventional chemotherapy regimens. This paradigm shift is characterized by the emergence of innovative therapies like CAR T-cell therapy and bispecific antibodies, which have demonstrated remarkable efficacy in targeting and eradicating lymphoma cells. CAR T-cell therapy represents a groundbreaking approach where a patient's own T-cells are genetically engineered to recognize and attack cancer cells. This personalized treatment has shown significant promise, especially for individuals who have undergone extensive previous therapies. One notable example is Axicabtagene ciloleucel (Yescarta), a CAR T-cell therapy approved for use in patients with relapsed or refractory follicular lymphoma. Clinical trials have highlighted its ability to induce durable remissions, with some patients experiencing long-term remission even after multiple prior therapies. LAS VEGAS, Nov. 28, 2024 /PRNewswire/ -- DelveInsight's ' Follicular Lymphoma Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline follicular lymphoma therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the follicular lymphoma pipeline domain. Key Takeaways from the Follicular Lymphoma Pipeline Report DelveInsight's follicular lymphoma pipeline report depicts a robust space with 50+ active players working to develop 55+ pipeline therapies for follicular lymphoma treatment. Key follicular lymphoma companies such as Chia Tai Tianqing Pharmaceutical Group, Incyte Corporation, Mabworks, Johnson & Johnson Innovative Medicine, Nektar Therapeutics, Xynomic Pharmaceuticals, Merck Sharp & Dohme, Jiangsu HengRui Medicine Co., Ltd., ADC Therapeutics, Bristol-Myers Squibb, Swedish Orphan Biovitrum, Zhejiang DTRM Biopharma, Immpact Bio, Galapagos, Immunitas Therapeutics, Enterome, CRISPR Therapeutics, CHO Pharma, Shanghai Blueray Biopharma, BeiGene, AstraZeneca, AVM Biotechnology, Mustang Bio, Merck, Pell Bio-Med Technology, Schrodinger Inc., Sana Biotechnology, Poseida Therapeutics, OncoNano Medicine, Inc., Nurix Therapeutics, Inc., NovalGen, Pfizer, Xencor, Wellington Zhaotai Therapies, MEI Pharma, Sutro Biopharma, Synthekine, Verismo Therapeutics, Vincerx Pharma, Novartis, Century Therapeutics, Newave Pharmaceuticals, Bio-Path Holdings, Affimed GmbH, Iksuda Therapeutics, Allogene Therapeutics, Adicet Bio, Carna Biosciences, Inc., BeiGene, Boryung Pharmaceutical, Accutar Biotechnology, Atara Biotherapeutics, AbbVie, InnoCare Pharma, NeoImmuneTech, and others are evaluating new follicular lymphoma drugs to improve the treatment landscape. Promising follicular lymphoma pipeline therapies such as Parsaclisib, TQ-B3525, Tafasitamab, MIL62, Ibrutinib, AZD0486, NKTR-255, Abexinostat, Acalabrutinib, Zilovertamab vedotin, SHR2554, Nivolumab, Loncastuximab tesirine, Golcadomide, Emapalumab, DTRM-555, Capivasertib, IMPT 314, GLPG5101, IMT-009, EO2463, CTX112, CHO-H01, BR1733, BGB-16673, BGB-10188, AZD5492, AVM0703, MB106, MK-1026, PL001, SGR-1505, SC291, SC262, P-CD19CD20-ALLO1, ONM 501, NX-5948, NX 2127, NVG-111, Mevrometostat, Plamotamab, WZTL-002, Voruciclib, STRO001, SYNCAR-001 + STK-009, SynKIR-310, VIP-152, VAY736, CNTY-101, LP-168, BP1002, IOA-244, IKS 03, ALLO-501, ADI-001, AS-1763, BGB-21447, BR101801, AC676, ATA3219, ABBV-319, ABBV-101, ICP-B02, ICP-B05, C-CAR039, Efineptakin Alfa, and others are under different phases of follicular lymphoma clinical trials. In August 2024, the company expects to file a supplemental Biologics License Application for tafasitamab for the treatment of patients with FL who have failed at least one prior systemic anti-CD20 immunotherapy or chemo-immunotherapy by the end of 2024 based on the positive Phase III results. In June 2024, a consortium of Eugene Private Equity and Korea Development Bank Private Equity announced that they would acquire 80% of South Korean vaccine maker Boryung Biopharma Co. for 320 billion won (USD 231 million). In February 2024, Incyte announced that it had entered into an asset purchase agreement with MorphoSys AG, which gives Incyte exclusive global rights for tafasitamab. Incyte will now recognize revenue and cost for all US commercialization and clinical development and MorphoSys will no longer be eligible to receive future milestone, profit split and royalty payments. In September 2023, OncoNano Medicine, Inc. announced a clinical trial supply agreement with Regeneron for the use of Libtayo (cemiplimab), a PD-1 inhibitor, in the combination stage of the first human trial of ONM-501, a dual-activating STING (STimulator of INterferon Genes) agonist and lead therapeutic development candidate. The ONM-501 first-in-human trial is a multicenter Phase Ia/b dose escalation and dose expansion study of intratumoral ONM-501 as monotherapy and in combination with Libtayo in patients with advanced solid tumors and lymphomas. OncoNano is the sponsor of the clinical trial, and Regeneron will supply cemiplimab. In May 2023, Janssen Pharmaceutical Companies of Johnson & Johnson announced that it had entered into a worldwide collaboration and license agreement with Cellular Biomedicine Group Inc. (CBMG) to develop, manufacture, and commercialize next-generation chimeric antigen receptor (CAR) T-cell therapies for the treatment of B-cell malignancies. Under the terms of the agreement, CBMG will grant Janssen a worldwide license to develop and commercialize the CAR-T assets, except in Greater China. Janssen and CBMG will negotiate an option for Janssen to commercialize the products in the Chinese territory. Janssen will make an upfront payment of USD 245 million that will be accounted for in the second quarter as a research and development expense. Additional future payments will be based upon achieving certain development, regulatory, and sales milestones, as well as tiered royalty payments on worldwide net trade sales, excluding Greater China. Request a sample and discover the recent advances in follicular lymphoma treatment drugs @ Follicular Lymphoma Pipeline Report The follicular lymphoma pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage follicular lymphoma drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the follicular lymphoma clinical trial landscape. Follicular Lymphoma Overview Follicular lymphoma is a common type of non-Hodgkin lymphoma (NHL) that originates from B-cells, a kind of white blood cell responsible for producing antibodies. This cancer is characterized by the growth of abnormal lymphoid follicles, which can disrupt normal lymphatic function. While the exact causes of follicular lymphoma are not fully understood, certain factors may increase the risk, including age (most commonly affecting adults over 60), a family history of lymphoma, and potential environmental factors such as exposure to pesticides or chemicals. Symptoms of follicular lymphoma can vary widely but may include painless swelling of lymph nodes in the neck, armpit, or groin, unexplained weight loss, night sweats, fatigue, and fever. Some patients may remain asymptomatic for extended periods, as this type of lymphoma can progress slowly. Diagnosis typically involves a combination of physical examinations, blood tests, imaging studies (like CT scans), and a biopsy of affected lymph nodes. The biopsy helps determine the subtype and stage of the lymphoma, which is crucial for planning treatment. Treatment for follicular lymphoma depends on the stage of the disease and the patient's overall health. Options may include watchful waiting for asymptomatic cases, chemotherapy, immunotherapy (such as monoclonal antibodies), and targeted therapies. In some cases, stem cell transplants may be considered for patients with more aggressive forms or those who do not respond to initial treatments. Given the indolent nature of follicular lymphoma, ongoing management, and regular monitoring are vital for achieving the best possible outcomes. Find out more about follicular lymphoma treatment drugs @ Drugs for Follicular Lymphoma Treatment A snapshot of the Follicular Lymphoma Pipeline Drugs mentioned in the report: Learn more about the emerging follicular lymphoma pipeline therapies @ Follicular Lymphoma Clinical Trials Follicular Lymphoma Therapeutics Assessment The follicular lymphoma pipeline report proffers an integral view of the follicular lymphoma emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Follicular Lymphoma Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Intra-articular, Intraocular, Intrathecal, Intravenous, Oral, Parenteral, Subcutaneous, Topical, Transdermal Therapeutics Assessment By Molecule Type: Oligonucleotide, Peptide, Small molecule Therapeutics Assessment By Mechanism of Action: Phosphatidylinositol 3 kinase delta inhibitors, Phosphatidylinositol 3 kinase gamma inhibitors, Emt protein-tyrosine kinase inhibitors, Interleukin 15 receptor agonists, Histone deacetylase inhibitors, Rad51 recombinase inhibitors, Agammaglobulinaemia tyrosine kinase inhibitors, Apoptosis stimulants, Cell cycle inhibitors, Tubulin inhibitors, Tubulin polymerisation inhibitors, Enhancer of zeste homolog 2 protein inhibitors, Antibody-dependent cell cytotoxicity, Programmed cell death 1 receptor antagonists, T lymphocyte stimulants, Alkylating agents, DNA cross-linking agents Key Follicular Lymphoma Companies: Chia Tai Tianqing Pharmaceutical Group, Incyte Corporation, Mabworks, Johnson & Johnson Innovative Medicine, Nektar Therapeutics, Xynomic Pharmaceuticals, Merck Sharp & Dohme, Jiangsu HengRui Medicine Co., Ltd., ADC Therapeutics, Bristol-Myers Squibb, Swedish Orphan Biovitrum, Zhejiang DTRM Biopharma, Immpact Bio, Galapagos, Immunitas Therapeutics, Enterome, CRISPR Therapeutics, CHO Pharma, Shanghai Blueray Biopharma, BeiGene, AstraZeneca, AVM Biotechnology, Mustang Bio, Merck, Pell Bio-Med Technology, Schrodinger Inc., Sana Biotechnology, Poseida Therapeutics, OncoNano Medicine, Inc., Nurix Therapeutics, Inc., NovalGen, Pfizer, Xencor, Wellington Zhaotai Therapies, MEI Pharma, Sutro Biopharma, Synthekine, Verismo Therapeutics, Vincerx Pharma, Novartis, Century Therapeutics, Newave Pharmaceuticals, Bio-Path Holdings, Affimed GmbH, Iksuda Therapeutics, Allogene Therapeutics, Adicet Bio, Carna Biosciences, Inc., BeiGene, Boryung Pharmaceutical, Accutar Biotechnology, Atara Biotherapeutics, AbbVie, InnoCare Pharma, NeoImmuneTech, and others. Key Follicular Lymphoma Pipeline Therapies: Parsaclisib, TQ-B3525, Tafasitamab, MIL62, Ibrutinib, AZD0486, NKTR-255, Abexinostat, Acalabrutinib, Zilovertamab vedotin, SHR2554, Nivolumab, Loncastuximab tesirine, Golcadomide, Emapalumab, DTRM-555, Capivasertib, IMPT 314, GLPG5101, IMT-009, EO2463, CTX112, CHO-H01, BR1733, BGB-16673, BGB-10188, AZD5492, AVM0703, MB106, MK-1026, PL001, SGR-1505, SC291, SC262, P-CD19CD20-ALLO1, ONM 501, NX-5948, NX 2127, NVG-111, Mevrometostat, Plamotamab, WZTL-002, Voruciclib, STRO001, SYNCAR-001 + STK-009, SynKIR-310, VIP-152, VAY736, CNTY-101, LP-168, BP1002, IOA-244, IKS 03, ALLO-501, ADI-001, AS-1763, BGB-21447, BR101801, AC676, ATA3219, ABBV-319, ABBV-101, ICP-B02, ICP-B05, C-CAR039, Efineptakin Alfa, and others. Dive deep into rich insights for new drugs for follicular lymphoma treatment, visit @ Follicular Lymphoma Drugs Table of Contents For further information on the follicular lymphoma pipeline therapeutics, reach out @ Follicular Lymphoma Treatment Drugs Related Reports Follicular Lymphoma Market Follicular Lymphoma Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key follicular lymphoma companies including Epizyme, Eisai, Bayer Healthcare Pharmaceuticals, Verastem Oncology, Gilead Sciences, TG Therapeutics, Bristol-Myers-Squibb, Roche, Incyte Corporation, Bristol Myers Squibb, ADC Therapeutics, MorphoSys, Nordic Nanovector, AbbVie, Regeneron Pharmaceuticals, Janssen Research & Development, Novartis, MEI Pharma, and BeiGene, among others. Non-Hodgkin Lymphoma Market Non-Hodgkin Lymphoma Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key Non-Hodgkin lymphoma companies, including Roche, Pfizer, Amgen, Novartis, Teva Pharmaceuticals, Genentech, ACD Therapeutics, among others. Non-Hodgkin Lymphoma Pipeline Non-Hodgkin Lymphoma Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Non-Hodgkin lymphoma 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. 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, market share of the individual therapies, and key DLBCL companies, including AbbVie, Genmab, Merck, Roche, Xencor, Janssen, Denovo Biopharma, Calithera Biosciences IMV, Biogen, Autolus Therapeutics, Allogene Therapeutics, Novartis, Miltenyi Biomedicine, Regeneron Pharmaceuticals, Debiopharm, Seagen, Takeda, AstraZeneca, Gilead Sciences, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

免疫疗法引进/卖出临床3期疫苗细胞疗法

2024-11-22

·药智网

CAR-T疗法在美国，不打价格战

前有诺华、吉利德两大巨头的CAR-T疗法获批多年，作为后来者的biotech，应该如何定价？对于国内大部分企业来说，答案可能不假思索：价格战。这也是过去大部分药物国产替代的逻辑。不过，英国生物科技企业Autolus给出了另外一个答案。近日，Autolus靶向CD19抗原的自体CAR-T疗法Aucatzyl获FDA批准上市，用于治疗复发性/难治性成人B细胞急性淋巴细胞白血病的成人患者。 Autolus对于这款CAR-T疗法的定价为：52.5万美元。这一价格，低于诺华Kymriah同适应症的58万美元，但要显著高于吉利德Tecartus的46万美元。在Autolus看来，高价是因为Aucatzyl具备显著的安全性优势，理应获得价值溢价。虽然Aucatzyl能否顺利商业化还不得而知，但这份勇气可嘉。国产CAR-T疗法跌破百万，美国却不打价格战。这背后，固然与支付环境紧密有关，但其中biotech关于创新的理念，或许才是更值得我们关注的。 <01> CD19靶点的后来者 CD19是CAR-T疗法中最成功的靶点，目前已批准上市的CAR-T疗法中，大部分针对CD19。仅海外就多达4款，因此，在CD19 CAR-T领域，Aucatzyl并没有先发优势。即便是在急性淋巴细胞白血病（ALL）领域，Aucatzyl也只能排到第三。在其之前，吉利德的Tecartus、诺华的Kymriah已经分别获批上市。其中，Kymriah早在2017年8月就已经在美国获批，而Tecartus的美国获批时间为2021年10月。可以说，在获批时间层面，诺华、吉利德处于断层式领先。当然，诺华的Kymriah获批的是儿童适应症，Aucatzyl直接的竞争对手是吉利德的Tecartus。相比于Tecartus，Aucatzyl不仅获批时间落后，在疗效层面也没有优势。 Aucatzyl的关键临床FELIX主要疗效指标，是输注后3个月内达到的完全缓解（CR）率和持续时间。结果显示，共有42%的患者达成CR，中位缓解持续时间（DOR）为14.1个月。在非头对头的情况下，吉利德Tecartus的ZUMA-3临床中，3个月内达到的完全缓解的患者比例为52%。那么，面对这样的市场格局，Aucatzyl仍选择高定价的逻辑是什么呢？答案在于安全性。 <02> 安全性是看点安全性，是CAR-T疗法难以回避的一个核心问题，尤其是细胞因子释放综合征（CRS）和免疫效应细胞相关神经毒性综合征（ICANS）相关的毒副作用。吉利德德Tecartus，在急性淋巴细胞白血病的成人患者身上，CRS发生率为92%（≥3级，26%），ICANS发生率为87%（≥3级，35%）。也正因此，CAR-T疗法都需要风险评估缓解策略计划（REMS）。 REMS是美国FDA为管理某些创新药已知或潜在严重风险，要求制药公司制定并实施的一系列措施和干预手段。多年来，REMS为治疗中心带来了“持续的义务”和额外的工作，算是一个明确的负担。因为，管理CRS和ICANS相关的反应，都需要付出医疗保健资源利用和成本。也正因此，Autolus希望解决这一痛点。Aucatzyl的独特之处在于，设计的嵌合抗原受体在与CD19结合后具有较快的解离速度，从而能够尽量减少T细胞的过度激活，在降低毒副作用。临床证据也揭示了这一点。安全性方面，Aucatzyl表现出低水平的CRS，3级事件发生率为3%，无4级或5级事件，并且，只有7%的患者出现≥3级ICANS。基于此，Aucatzyl是获美国FDA批准的首个无需风险评估缓解策略计划的CAR-T疗法。在公司CEO看来，这一优势真正提供了一个让医生控制治疗的计划，对于扩大CAR-T在ALL适应症中的应用非常重要。也正因此，Autolus艺高人胆大地定出了高价。 <03> 乐观来自差异化关于Aucatzyl的商业化推进情况，Autolus极为乐观。在三季报的电话会议上，公司表示已经为商业化做好了充分准备，预计在未来几周内，Autolus授权的30个治疗中心能够覆盖大约60%的目标人群；而在接下来的12个月里，这个数字将增加到60个中心，进而覆盖90%的患者。虽然结果最终如何还不得而知，但这种基于差异化优势带来的乐观情绪，无疑是值得肯定的。这样的故事，未来必然不会只是在Autolus身上得到体现。可以看到，因为“差异化”，今年的ASH年会上，CD19 CAR-T将重回焦点，核心是在快速制备层面：Fast car-T。Galapagos的GLPG5101，以及诺华的Rapcabtagene autoleucel，都将带来口头报告。过去，CAR-T疗法的一个痛点是供应周期太长，需要数周时间才能预订到生产细胞，这意味着患者需要等待数周或数月才能真正接受治疗。一些患者等待了很长时间，以至于他们不得不通过化疗来弥补。不过，办法总比困难多。也正因此，目前Fast car-T成为热点，未来的制备周期可能缩短至2天甚至更短。而这些差异化选手，也必然会让市场对CAR-T疗法更乐观。 CAR-T疗法的疗效有目共睹，但目前仍有许多不足。一方面，适应症探索层面仍有极大的空间；另一方面，定制化生产、昂贵的上游原材料和生产设备、复杂的供应链体系等综合因素是CAR-T疗法价格居高不下、供应周期偏长的核心原因，自然也是改进的方向。期待更多国内企业，在差异化层面迈出更扎实的步伐。支付能力的限制，是客观的；但是，通过解决更多痛点的方式突围，也是值得一试的。来源 | 氨基观察（药智网获取授权转载）撰稿 | 郑晓责任编辑 | 八角声明：本文系药智网转载内容，图片、文字版权归原作者所有，转载目的在于传递更多信息，并不代表本平台观点。如涉及作品内容、版权和其它问题，请在本平台留言，我们将在第一时间删除。商务合作 | 王存星 19922864877（同微信）阅读原文，是受欢迎的文章哦

细胞疗法免疫疗法上市批准临床结果

2024-11-14

·氨基观察

不打价格战，CAR-T疗法在美国

氨基观察-创新药组原创出品作者 | 郑晓前有诺华、吉利德两大巨头的CAR-T疗法获批多年，作为后来者的biotech，应该如何定价？对于国内大部分企业来说，答案可能不假思索：价格战。这也是过去大部分药物国产替代的逻辑。不过，英国生物科技企业Autolus给出了另外一个答案。上周五， Autolus靶向CD19抗原的自体CAR-T疗法Aucatzyl获FDA批准上市，用于治疗复发性/难治性成人B细胞急性淋巴细胞白血病的成人患者。 Autolus对于这款CAR-T疗法的定价为：52.5万美元。这一价格，低于诺华Kymriah同适应症的58万美元，但要显著高于吉利德Tecartus的46万美元。在Autolus看来，高价是因为Aucatzyl具备显著的安全性优势，理应获得价值溢价。虽然Aucatzyl能否顺利商业化还不得而知，但这份勇气可嘉。国产CAR-T疗法跌破百万，美国却不打价格战。这背后，固然与支付环境紧密有关，但其中biotech关于创新的理念，或许才是更值得我们关注的。 / 01 / CD19靶点的后来者 CD19是CAR-T疗法中最成功的靶点，目前已批准上市的CAR-T疗法中，大部分针对CD19。仅海外就多达4款，因此，在CD19 CAR-T领域，Aucatzyl并没有先发优势。即便是在急性淋巴细胞白血病（ALL）领域，Aucatzyl也只能排到第三。在其之前，吉利德的Tecartus、诺华的Kymriah已经分别获批上市。其中，Kymriah早在2017年8月就已经在美国获批，而Tecartus的美国获批时间为2021年10月。可以说，在获批时间层面，诺华、吉利德处于断层式领先。当然，诺华的Kymriah获批的是儿童适应症，Aucatzyl直接的竞争对手是吉利德的Tecartus。相比于Tecartus，Aucatzyl不仅获批时间落后，在疗效层面也没有优势。 Aucatzyl的关键临床FELIX主要疗效指标，是输注后3个月内达到的完全缓解（CR）率和持续时间。结果显示，共有42%的患者达成CR，中位缓解持续时间（DOR）为14.1个月。在非头对头的情况下，吉利德Tecartus的ZUMA-3临床中，3个月内达到的完全缓解的患者比例为52%。那么，面对这样的市场格局，Aucatzyl仍选择高定价的逻辑是什么呢？答案在于安全性。 / 01 / 安全性是看点安全性，是CAR-T疗法难以回避的一个核心问题，尤其是细胞因子释放综合征（CRS）和免疫效应细胞相关神经毒性综合征（ICANS）相关的毒副作用。吉利德德Tecartus，在急性淋巴细胞白血病的成人患者身上，CRS发生率为 92%（≥3 级，26%），ICANS发生率为87%（≥3 级，35%）。也正因此，CAR-T疗法都需要风险评估缓解策略计划（REMS）。 REMS是美国FDA为管理某些创新药已知或潜在严重风险，要求制药公司制定并实施的一系列措施和干预手段。多年来，REMS为治疗中心带来了“持续的义务”和额外的工作，算是一个明确的负担。因为，管理CRS和ICANS相关的反应，都需要付出医疗保健资源利用和成本。也正因此，Autolus希望解决这一痛点。Aucatzyl的独特之处在于，设计的嵌合抗原受体在与CD19结合后具有较快的解离速度，从而能够尽量减少T细胞的过度激活，在降低毒副作用。临床证据也揭示了这一点。安全性方面，Aucatzyl表现出低水平的CRS，3级事件发生率为3%，无4级或5级事件，并且，只有7%的患者出现≥3级ICANS。基于此，Aucatzyl是获美国FDA批准的首个无需风险评估缓解策略计划的CAR-T疗法。在公司CEO看来，这一优势真正提供了一个让医生控制治疗的计划，对于扩大CAR-T在 ALL 适应症中的应用非常重要。也正因此，Autolus艺高人胆大地定出了高价。 / 03 / 乐观来自差异化关于Aucatzyl的商业化推进情况，Autolus极为乐观。在三季报的电话会议上，公司表示已经为商业化做好了充分准备，预计在未来几周内，Autolus授权的30个治疗中心能够覆盖大约60%的目标人群；而在接下来的12个月里，这个数字将增加到60个中心，进而覆盖90%的患者。虽然结果最终如何还不得而知，但这种基于差异化优势带来的乐观情绪，无疑是值得肯定的。这样的故事，未来必然不会只是在Autolus身上得到体现。可以看到，因为“差异化”，今年的ASH年会上，CD19 CAR-T将重回焦点，核心是在快速制备层面：Fast car-T。Galapagos的GLPG5101，以及诺华的Rapcabtagene autoleucel，都将带来口头报告。过去，CAR-T疗法的一个痛点是供应周期太长，需要数周时间才能预订到生产细胞，这意味着患者需要等待数周或数月才能真正接受治疗。一些患者等待了很长时间，以至于他们不得不通过化疗来弥补。不过，办法总比困难多。也正因此，目前Fast car-T成为热点，未来的制备周期可能缩短至2天甚至更短。而这些差异化选手，也必然会让市场对CAR-T疗法更乐观。 CAR-T疗法的疗效有目共睹，但目前仍有许多不足。一方面，适应症探索层面仍有极大的空间；另一方面，定制化生产、昂贵的上游原材料和生产设备、复杂的供应链体系等综合因素是CAR-T疗法价格居高不下、供应周期偏长的核心原因，自然也是改进的方向。期待更多国内企业，在差异化层面迈出更扎实的步伐。支付能力的限制，是客观的；但是，通过解决更多痛点的方式突围，也是值得一试的。 PS：欢迎扫描下方二维码，添加氨基君微信号交流。

细胞疗法上市批准免疫疗法临床结果ASH会议

100 项与 GLPG-5101 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
血液肿瘤	临床3期	比利时	Galapagos NV	2024-09-09
血液肿瘤	临床3期	荷兰	Galapagos NV	2024-09-09
血液肿瘤	临床3期	西班牙	Galapagos NV	2024-09-09
难治性B细胞淋巴瘤	临床2期	美国	Galapagos NV	2022-03-09
难治性B细胞淋巴瘤	临床2期	比利时	Galapagos NV	2022-03-09
难治性B细胞淋巴瘤	临床2期	荷兰	Galapagos NV	2022-03-09
伯基特淋巴瘤	临床2期	美国	Galapagos NV	2022-03-09
伯基特淋巴瘤	临床2期	比利时	Galapagos NV	2022-03-09
伯基特淋巴瘤	临床2期	荷兰	Galapagos NV	2022-03-09
弥漫性大B细胞淋巴瘤	临床2期	美国	Galapagos NV	2022-03-09

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ASH2024	N/A	非霍奇金淋巴瘤	-	GLPG5101	餘淵顧鹹鏇衊膚築蓋膚(憲鬱鬱廠鏇齋構餘遞鑰) = Most Grade ≥3 treatment-emergent adverse events (reported up to 14 weeks post-infusion) were hematological. CRS was reported in 9/20 (45%) Ph1 pts (Grade ≤2 n=8; Grade 3 n=1) and 10/25 (40%) Ph2 pts (all Grade ≤2) 簾構壓築獵製鏇醖鏇築 (繭願鹽鏇壓艱鏇築衊築 ) 更多	-	2024-12-07