This is a prospective long-term follow-up (LTFU) study to evaluate the long-term safety and survival benefit for patients who received at least one of CARsgen's GM T cell product in a clinical trial or as a commercially available product. In this study, patients will be followed for up to 15 years after last GM T-cell infusion for the evaluation of delayed adverse events (AEs), vector persistence, potential risk for integration, and survival time due to GM T-cell exposure.

开始日期2025-03-31

申办/合作机构

科济生物医药（上海）有限公司

NCT06659770

/ Not yet recruitingN/A

A Real-World Observational Study: Zevorcabtagene Autoleucel Injection in Patients with Relapsed/Refractory Multiple Myeloma

This study is a single-arm, open-label, multicenter, post-marketing, Phase IV, prospective, observational clinical trial to evaluate the efficacy and safety of the post-marketing product zevor-cel as treatment for subjects with R/R MM in the real world.

开始日期2024-10-01

申办/合作机构

科济生物医药（上海）有限公司

NCT03915184

/ Active, not recruiting临床1/2期

Open Label, Multi-center, Phase 1b/2 Clinical Trial to Evaluate the Safety and Efficacy of Autologous CAR BCMA T Cells (CT053) in Patients With Relapsed and/or Refractory Multiple Myeloma (LUMMICAR STUDY 2)

A phase 1b/2, open label, multi-center, Clinical Study of Chimeric Antigen Receptor T Cells targeting BCMA in patients with relapsed and or refractory multiple myeloma.

开始日期2019-09-25

申办/合作机构

科济生物医药（上海）有限公司

100 项与泽沃基奥仑赛相关的临床结果

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100 项与泽沃基奥仑赛相关的转化医学

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100 项与泽沃基奥仑赛相关的专利（医药）

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项与泽沃基奥仑赛相关的文献（医药）

2024-07-01·Molecular Diagnosis & Therapy

Zevorcabtagene Autoleucel: First Approval

Review

作者: Dhillon, Sohita

Zevorcabtagene autoleucel () is a fully humanised B cell maturation antigen (BCMA)-targeting specific chimeric antigen receptor (CAR) T-cell therapy being developed by CARsgen for the treatment of multiple myeloma. Zevorcabtagene autoleucel is an autologous CAR T cell comprising a fully human BCMA-specific scFv (25C2), a CD8α hinge region and transmembrane domain, a 4-1BB costimulatory domain and a CD3-ζ T cell activation domain. Zevorcabtagene autoleucel recognizes and induces selective toxicity against BCMA-expressing tumour cells leading to their elimination. In February 2024, zevorcabtagene autoleucel received its first approval in China for the treatment of adults with relapsed or refractory multiple myeloma who have progressed after ≥ 3 prior lines of therapy (including ≥ 1 proteasome inhibitor and an immunomodulatory agent). Clinical studies of zevorcabtagene autoleucel are underway in Canada and the US. This article summarizes the milestones in the development of zevorcabtagene autoleucel leading to this first approval for relapsed or refractory multiple myeloma.

2022-07-01·Blood reviews2区 · 医学

Current state and next-generation CAR-T cells in multiple myeloma

2区 · 医学

Review

作者: Facon, Thierry ; Mitra, Suman ; Manier, Salomon ; Nudel, Morgane ; Prodhomme, Chloé ; Escure, Guillaume ; Ingegnere, Tiziano

Chimeric antigen receptor T cells (CAR-T cells) have emerged as a potentially transformative new approach to treating hematological malignancies. Ide-cel, an autologous B cell maturation antigen (BCMA) targeting CAR-T cells, has recently been approved to treat multiple myeloma (MM). Here, we review the main clinical trials of CAR-T cells in MM with the most advanced autologous BCMA-directed ide-cel and cilta-cel, the human CARs orva-cel and CT053, the alternative manufacturing process with P-BCMA-101 and bb21217, the dual CAR GC012F and the allogenic BCMA-directed CAR-T cells ALLO-715. In light of those clinical data, we provide an overview of CAR-T cells' main potential resistance mechanisms, including antigen loss, antigen spreading, anti-CAR antibodies, CAR-T cell exhaustion, and the emergence of a non-permissive microenvironment. Finally, we describe the principal area of research to build the next generation of CAR-T cells, with armored-, gated- or commuting-CARs, CARs associated with knock out of specific genes, and CAR-T cells made from γδT cells or NK cells.

2018-08-01·Journal of clinical oncology : official journal of the American Society of Clinical Oncology1区 · 医学

T Cells Genetically Modified to Express an Anti–B-Cell Maturation Antigen Chimeric Antigen Receptor Cause Remissions of Poor-Prognosis Relapsed Multiple Myeloma

1区 · 医学

Article

作者: Maric, Irina ; Mikkilineni, Lekha ; Brudno, Jennifer N ; Lam, Norris ; Stroncek, David F ; Hansen, Brenna G ; Yuan, Constance ; Pavletic, Steven ; Wang, Michael ; Patel, Rashmika ; Lawrence, Judith ; Gress, Ronald E ; Stetler-Stevenson, Maryalice ; Hartman, Steven D ; Rose, Jeremy J ; Salem, Dalia ; Kanakry, Jennifer A ; Kochenderfer, James N ; Ali, Syed Abbas ; Hakim, Frances ; Feldman, Steven A

Purpose:

Therapies with novel mechanisms of action are needed for multiple myeloma (MM). T cells can be genetically modified to express chimeric antigen receptors (CARs), which are artificial proteins that target T cells to antigens. B-cell maturation antigen (BCMA) is expressed by normal and malignant plasma cells but not normal essential cells. We conducted the first-in-humans clinical trial, to our knowledge, of T cells expressing a CAR targeting BCMA (CAR-BCMA).

Patients and Methods:

Sixteen patients received 9 × 106 CAR-BCMA T cells/kg at the highest dose level of the trial; we are reporting results of these 16 patients. The patients had a median of 9.5 prior lines of MM therapy. Sixty-three percent of patients had MM refractory to the last treatment regimen before protocol enrollment. T cells were transduced with a γ-retroviral vector encoding CAR-BCMA. Patients received CAR-BCMA T cells after a conditioning chemotherapy regimen of cyclophosphamide and fludarabine.

Results:

The overall response rate was 81%, with 63% very good partial response or complete response. Median event-free survival was 31 weeks. Responses included eradication of extensive bone marrow myeloma and resolution of soft-tissue plasmacytomas. All 11 patients who obtained an anti-MM response of partial response or better and had MM evaluable for minimal residual disease obtained bone marrow minimal residual disease–negative status. High peak blood CAR+ cell levels were associated with anti-MM responses. Cytokine-release syndrome toxicities were severe in some cases but were reversible. Blood CAR-BCMA T cells were predominantly highly differentiated CD8+ T cells 6 to 9 days after infusion. BCMA antigen loss from MM was observed.

Conclusion:

CAR-BCMA T cells had substantial activity against heavily treated relapsed/refractory MM. Our results should encourage additional development of CAR T-cell therapies for MM.

234

项与泽沃基奥仑赛相关的新闻（医药）

2025-04-01

·研发客

被调出港股通不跌反涨，背后大有玄机

// • 港股通对港股上市创新药公司股价的影响，已没有预想的那么显著；• 恒生创新药指数已触及自2023年12月以来近16个月的新高；• 根据研发客统计，在港上市创新药企业或可获得内地ETF总计达500亿的投资，足以缓解港股创新药流动性的大难题；• 除了存量ETF，投资于港股创新药的增量资金也在路上；• 前公募冠军基金经理创立的私募基金，正借助公募指数产品布局港股创新药赛道。沪港通下港股通的标的名单在3月初又有新调整。此次，多家创新药公司在调整范围之列。其中，同源康医药被纳入港股通，而宜明昂科、来凯医药、科济药业、科笛、艾美疫苗和康宁杰瑞制药6家公司由于月均市值低于40亿元被调出。众所周知，港股通是一个让内地投资者可以通过内地券商买卖港交所上市股票的机制。由于数量巨大的内地投资者（证券账户市值50万以上）可以投资港股，也使得港股通对在港上市的创新药企的流动性来讲显得尤其重要。然而，研发客从此次调整公司的股价变化发现，是否被纳入港股通，已经不能显著影响港股上市创新药公司的股价，同时，港股创新药赛道流动性难题正在缓解。六家被调出公司的表现先来看调出的几家公司2024年的成绩单。宜明昂科2024全年收入7415万元人民币，去年同期仅38.6万元。收入增长主要源于2024年8月与Instil Bio子公司Axion Bio达成的一项授权合作协议所获得首付款及近期付款。双方合作的产品是宜明昂科的PD-L1 x VEGF双抗IMM2510，以及下一代CTLA-4抗体IMM27M。尽管如此，宜明昂科在2024年的亏损仍然达到3.159亿元。截至3月24日，公司市值为19.96亿元。来凯医药在上周公布的2024年度财报显示，亏损2.543亿元，较上一年同期有所收窄。同时，来凯在去年缩减了研发开支，全年研发费用2.151亿元，同比减少6%。公司方面表示，2024年公司更专注于先进入临床的项目。来凯在本周一的公告称，LAE102针对肥胖症治疗的IND临床方案修订版已递交FDA。2024年11月，来凯与礼来加速LAE102针对肥胖症治疗的全球临床开发达成合作协议。截至3月24日，公司市值达到55亿元。拓展阅读减肥增肌研究与激活素药物：下一个重磅呼之欲出？| 新靶点的故事科济药业2024年亏损7.981亿元，亏损幅度较上一年继续扩大。全年研发支出4.66亿元，同比缩减29.5%。目前，科济已有一款CAR-T产品赛恺泽在中国上市，去年3月获批用于治疗多发性骨髓瘤。此外，公司正在推进多个通用型CAR-T产品的研发。截至3月24日，公司市值达到73亿元。康宁杰瑞在2024全年营收6.41亿元，同比增长192.58%；净利润1.663亿元，同比增长178.99%。这也是公司第一次扭亏为盈，截至3月24日，市值达到73.11亿元。涨和跌出人意料上述6家公司，在被技术性调出港股通投资标的后，按惯例，其股价均呈现不同程度短期下跌。以首周表现为例，其中宜明昂科在3月10日至3月14日创新药板块跌幅排名第一，跌幅达 23.51%。科济药业跌幅则达到20.80%，在创新药板块跌幅排名中位列第三。相对于被调出个股短期震荡下跌的股价表现，被调入的同源康医药股价则出现短期大涨，3月10日调入当天股价一度大涨超50%，最后收涨 16.18%。当然，同源康医药股价的突出表现，不完全因为被调入港股通投资标的，而更多缘于公司3月9日公布了其自主研发的TY–9591，对比奥希替尼作为一线治疗 EGFR 突变肺癌脑转移的关键Ⅱ期临床试验中，研究者数据显示达到研究预期，具有统计学显著意义和重大临床获益。但出乎市场意料的是，随后几个交易日，同源康医药股价出现回调。截止到3月24日，公司市值110.5亿元，与两则利好公告发布前市值基本保持一致。相反，康宁杰瑞制药在被调出港股通投资标的经历短暂下跌后，于之后几个交易日股价大幅上涨近30%。其他几只被调出港股通标的的个股，股价也逐步企稳，部分个股甚至收复了此前短暂下跌的跌幅。可见，是否纳入港股通对港股上市创新药公司股价的影响，已没有预想的那么显著。18A公司寻求破局之路有券商统计，早年港股通对这些创新药企成交量的贡献一度接近50%。尽管港股通的重要性可想而知，但由于绝大部分港股上市创新药公司在市值上均低于港股通规定的月均40亿规模，导致亟需市场流动性的中小型创新药企被排除在港股通标的范围之外。于是，在港上市的创新药企业开始寻求突破口。2024年12月1日，中国医药创新促进会向有关部门郑重发出一项倡议：提议将所有依据“18A章节”上市的公司直接纳入港股通，同时，那些已被移出港股通的公司也应重新被列为港股通交易标的。在这之前，通过“18A章节”规则上市的创新药企为争取更有利的市场环境，已三次向相关机构发出联名信。联名数量也由最初的19家发展到41 家。去年末的倡议收信方范围也大幅扩大，涵盖了中国证券监督管理委员会、香港证券及期货事务检查委员会、上海证券交易所、深圳证券交易所、香港交易及结算所有限公司、恒生指数有限公司。问题也就此产生，创新药企对于集体进入港股通投资标的范围的诉求为何在今年一季度偃旗息鼓，最新被剔除出去的创新药股票为何跌幅有限，甚至不跌反涨？恒生创新药指数走强这可能得益于恒生创新药指数今年以来良好的收益表现，以及相关内地发行的ETF规模水涨船高，使得非港股通的创新药企流动性问题已大为缓解。今年以来，恒生创新药指数涨幅在香港资本市场中仅次于恒生科技互联网指数，截至3月20日，该指数已触及自2023年12月以来近16个月的新高，不过近期该指数已出现明显回调。除了一系列政策的利好之外，业绩是推动本轮恒生创新药指数持续上涨的催化剂。2024年年报的陆续披露，多家港股Biotech公司进入盈利期。一家港股上市的新药研发公司CFO向研发客表示，资本正在引导行业向更健康的方向发展。过去几年的资本寒冬，推动了创新药企业在差异化布局上有更慎重的布局。他也看到，最近一段时间资本对行业又重拾了一些信心，有兴趣来投资Biotech公司。持续净流入是另一个重要因素。随着港股市场表现持续领涨全球股市，港股市场资金面趋松，以港股通为代表的南向资金加大对创新药等细分领域的配置。同时，各类在内地发行的恒生创新药主题ETF也出现持续净申购迹象。内地ETF承接在港创新药企500亿体量事实上，通过港股相关主题ETF借道投资港股，也成为内地机构投资者和没有港股通投资资格的中小投资者的首选。根据研发客统计，以恒生医疗ETF和港股创新药ETF为例，两只基金场内流通规模分别高达140亿元和123亿元，加上其他中小规模的港股创新药ETF、投资香港全市场的ETF，在港上市的创新药企业或可获得内地ETF总计达500亿的投资，足以缓解港股创新药流动性的大难题。除了存量ETF，投资于港股创新药的增量资金也在路上。易方达恒生港股通创新药基金于3月26日上市交易，此前该基金募集期仅7个交易日。值得一提的是，根据该基金已披露的上市交易公告书显示，前公募基金冠军张小仁创立的私募基金，成为该港股创新药指数基金的前十大份额持有人之一。创立私募的前公募冠军基金经理借助公募指数产品，布局港股创新药赛道的现象，凸显出连跌三年的创新药行业，已几乎成为港股市场最锋利的矛，而中小市值创新药公司则在其中弹性最高。随着港股通、ETF等内地跨市场交易工具的丰富，此前内地创新药企管理者和投资者最为担心的香港市场流动性难题正被逐步化解，解决这一难题的并不是之前预期的国际投资者，却是我们自己人——内地投资者。而一旦香港上市创新药企的市值和流动性被内地投资者逐渐盘活后，国际投资者的趋势性买入或将进一步提升创新药企的市值和流动性，从而形成良性循环。编辑 | 姚嘉yao.jia@PharmaDJ.com 总第2381期访问研发客网站，深度报道和每日新闻抢鲜看www.PharmaDJ.com

财报免疫疗法细胞疗法疫苗

2025-03-25

·生物制品圈

Hillstar Bio 获 6700 万美元融资直击强直性脊柱炎治疗痛点

导语：在自身免疫疾病治疗领域，一家由跨国药企高管与顶级风投联手打造的新锐公司正以“细胞清除革命”掀起技术革新。2025年3月，波士顿生物技术公司 Hillstar Bio 宣布完成 6700 万美元 A 轮融资，聚焦开发靶向致病性免疫细胞的创新疗法，旨在打破传统免疫抑制药物的局限，为强直性脊柱炎等疾病患者提供更安全、更持久的治疗选择。一、技术破局：从 “全面抑制” 到 “精准清除” Hillstar Bio 的核心技术源于对自身免疫病发病机制的深度解析。传统疗法（如 TNF-α 抑制剂）通过抑制整体免疫系统缓解症状，但长期使用可能引发感染风险。Hillstar 的突破性在于其 “精准免疫” 平台，可选择性清除特定致病性免疫细胞，保留健康细胞功能。核心靶点 TRBV9+ T 细胞：该细胞亚群在强直性脊柱炎（AxSpA）、银屑病关节炎等疾病中异常活跃。临床前研究显示，清除 TRBV9+ T 细胞可使患者病情长期缓解，甚至实现多年无复发。首款候选药物 HBS-101：计划 2026 年启动临床试验，针对 AxSpA 患者，目标实现 “长效缓解”，减少对免疫抑制剂的依赖。二、团队与资本：顶级阵容加持的 “独角兽” 雏形 Hillstar Bio 的高管团队堪称行业 “全明星组合”： CEO Robert Mabry：曾任武田全球生物药负责人，主导多款单抗药物上市，后在 Orna Therapeutics 推动环状 RNA 技术开发。首席开发官 Mitchell Keegan：波士顿制药临床开发前负责人，成功将多款创新药推进至 III 期临床。首席运营官 Lauren Mifflin：Frazier Life Sciences 前合伙人，擅长生物科技公司孵化与战略布局。 Robert Mabry, Ph.D., Chief Executive Officer, Hillstar Bio 本轮融资由 Frazier Life Sciences 领投，Droia Ventures、Novo Holdings（诺和诺德投资部门）等参投。值得注意的是，Novo Holdings 近期已连续注资两家自身免疫领域初创公司，显示其对该赛道的战略看好。三、市场潜力：2000 亿美元赛道的 “蓝海机遇” 全球自身免疫病患者超 3.5 亿，市场规模突破 2000 亿美元且年增速 8%。Hillstar 的技术若成功，将填补“长效、低毒”疗法的空白：强直性脊柱炎（AxSpA）：全球患者超 3000 万，现有疗法仅 30% 患者达标，HBS-101 的精准清除机制有望重塑治疗标准。管线扩展计划：融资将支持其他精准免疫管线开发，覆盖类风湿性关节炎、多发性硬化症等适应症。四、行业趋势：精准免疫疗法成资本新宠 Hillstar 的崛起反映出自身免疫领域的三大趋势：靶点精细化：从泛免疫抑制转向特定细胞亚群调控，如 JAK 抑制剂（辉瑞 Xeljanz）到更精准的 S1P 调节剂（百健 Zeposia）。技术融合：结合单细胞测序、AI 靶点发现与基因编辑技术，加速药物开发。资本理性化：投资者更青睐差异化技术平台，2024 年全球自身免疫领域融资中，40% 流向精准免疫疗法。五、挑战与未来尽管前景光明，Hillstar 仍需跨越多重障碍：临床验证风险：TRBV9+ T 细胞的致病性在不同患者中存在异质性，需大规模试验验证疗效。生产工艺优化：细胞清除疗法的规模化生产可能面临成本挑战，需开发高效、可重复的制造流程。竞争加剧：诺华、艾伯维等巨头正布局同类技术，如诺华的 CAR-T 细胞疗法 CT053 已进入 III 期临床。六、专家观点：改变游戏规则的潜力 “Hillstar 的技术代表了自身免疫病治疗的未来方向。”哈佛医学院免疫学家 Sarah Lee 评论道，“选择性清除致病性细胞而非抑制整个免疫系统，将显著降低副作用，改善患者生活质量。” 行业分析师指出，若 HBS-101 在临床试验中表现优异，Hillstar 的估值可能在 3 年内突破 50 亿美元，成为精准免疫领域的 “独角兽” 企业。七、结语当传统疗法遭遇瓶颈，精准免疫疗法正成为自身免疫病领域的 “破局者”。Hillstar Bio 的 6700 万美元融资不仅是资本对技术的认可，更是对患者需求的回应。在强直性脊柱炎等疾病中，这场 “细胞清除革命”或将重新定义治疗标准，为全球 3.5 亿自身免疫病患者带来更安全、更持久的治疗选择。而 Hillstar 的故事，也将成为生物技术领域 “创新驱动价值” 的又一经典案例。参考来源：https://www.biospace.com/drug-development/hillstar-bio-nets-67m-series-a-to-advance-autoimmune-and-arthritis-pipeline 识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观不本站。

临床3期免疫疗法

2025-03-24

·PRNewswire

CARVYKTI Continued Performance Reflects Growing Confidence in CAR-T Therapies for Multiple Myeloma | DelveInsight

As a BCMA-targeting CAR T-cell therapy, CARVYKTI addresses a high unmet need in relapsed or refractory multiple myeloma patients. With promising clinical efficacy, durable responses, and growing adoption in advanced treatment lines, CARVYKTI is poised for substantial revenue growth. LAS VEGAS, March 24, 2025 /PRNewswire/ -- DelveInsight's " CARVYKTI Market Size, Forecast, and Market Insight Report" highlights the details around CARVYKTI, a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient's T cells with a transgene encoding a CAR that identifies and eliminates cells that express BCMA. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of CARVYKTI. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Janssen's CARVYKTI (ciltacabtagene autoleucel) Overview CARVYKTI is an autologous T-cell immunotherapy that targets B-cell maturation antigen (BCMA). It works by genetically modifying a patient's T cells with a transgene that encodes a chimeric antigen receptor (CAR). This CAR helps the T cells recognize and destroy cells expressing BCMA, which is primarily found in malignant multiple myeloma B-lineage cells, late-stage B cells, and plasma cells. The CARVYKTI CAR protein includes two single-domain antibodies designed to bind strongly to human BCMA, enhancing T-cell activation, proliferation, and the destruction of target cells upon binding. Currently, CAR-T cell therapies are only available to UK patients through clinical trials. Janssen recently decided not to proceed with seeking approval for cilta-cel (CARVYKTI) for myeloma patients in the UK through the National Institute for Health and Care Excellence (NICE). This decision means cilta-cel will not be available on the NHS for now, although it remains accessible through clinical trials. Janssen's decision does not impact ongoing trials. The drug is currently being evaluated in Phase III trials: CARTITUDE-6 for frontline multiple myeloma transplant eligible vs ASCT and CARTITUDE-5 for frontline multiple myeloma TNI. In 2024, CARVYKTI generated sales of USD 963 million across the world. CARVYKTI Dosage and Administration CARVYKTI is administered as a single infusion containing a suspension of CAR-positive, viable T cells in one infusion bag. The recommended dosage ranges from 0.5 to 1.0 × 10⁶ CAR-positive viable T cells per kilogram of body weight, with a maximum limit of 1 × 10⁸ CAR-positive viable T cells per infusion. Learn more about CARVYKTI projected market size for multiple myeloma @ CARVYKTI Market Potential CAR-T cell immunotherapy is emerging as a revolutionary treatment for multiple myeloma, offering new hope to patients who have exhausted conventional treatment options. Currently, only two CAR-T cell therapies are approved for multiple myeloma: ABECMA (idecabtagene vicleucel) from Bristol-Myers Squibb and CARVYKTI (ciltacabtagene autoleucel) from Johnson & Johnson Innovative Medicine. Their approval highlights the potential of CAR-T therapy to transform multiple myeloma treatment. A key advantage of CAR-T therapies is their "one-and-done" nature, requiring a single administration, unlike bispecific antibodies such as TECVAYLI and TALVEY, which need ongoing dosing. Present CAR-T therapies use a patient's own T cells (autologous), but research is underway to develop allogeneic CAR-T cells derived from healthy donors. Allogeneic therapies could offer off-the-shelf solutions, enhancing accessibility and lowering costs. The approval of CAR-T therapies has created new opportunities for companies focused on advanced-stage multiple myeloma (fourth line and beyond). Several companies are progressing with their CAR-T candidates at different stages of development. The CAR-T cell therapy market for multiple myeloma is expected to grow significantly between 2024 and 2034, driven by its high efficacy and potential for expanded use. Collaboration between pharmaceutical companies and research institutions, along with ongoing innovation, is expected to accelerate market growth. However, challenges such as manufacturing complexity and pricing will need to be addressed to unlock the full potential of CAR-T therapies. Discover more about the CAR T-cell therapy for multiple myeloma market in detail @ CAR T-cell Therapy for Multiple Myeloma Market Report Emerging Competitors of CARVYKTI Some of the CAR-Ts in the multiple myeloma pipeline that will give fierce competition to CARVYKTI include Anito-cel (Arcellx), PHE885 (Novartis), BMS-986393 (Bristol-Myers Squibb), Zevorcabtagene Autoleucel (CARsgen Therapeutics), and GLPG5301 (Galapagos), among others In December 2024, Arcellx announced encouraging new data from its Phase II pivotal iMMagine-1 trial for anito-cel in multiple myeloma. The results were presented during an oral session at the 66th American Society of Hematology (ASH) Annual Meeting on Monday, December 9, 2024, at 5:30 p.m. PT. Also at ASH 2024, BMS shared the first overall survival (OS) and progression-free survival (PFS) results for arlo-cel in an oral presentation. The Phase I trial involved patients with relapsed or refractory multiple myeloma who had previously undergone at least three treatments, including a proteasome inhibitor, an immunomodulatory drug, and anti-CD38 therapy. Among 79 patients evaluable for efficacy, with a median follow-up of 16.1 months (range: 2.8–25.2), arlo-cel showed an overall response rate (ORR) of 87%, indicating durable responses. Minimal residual disease (MRD) was assessed as an exploratory measure, with 57% (48 of 84) of MRD-evaluable patients included. Of these, 46% (22 of 48) were MRD-negative and achieved a complete response (CR) or stringent CR (sCR). Across all treated patients, 27% (23 of 84) were MRD-negative and achieved a CR. Median PFS was 18.3 months (95% CI: 11.8–21.9), while the median OS had not yet been reached. To know more about the number of competing drugs in development, visit @ CARVYKTI Market Positioning Compared to Other Drugs Key Milestones of CARVYKTI In April 2024, Johnson & Johnson announced that the US FDA approved CARVYKTI (ciltacabtagene autoleucel; cilta-cel) for the treatment of adult patients with RRMM who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide. In March 2024, Johnson & Johnson announced that the US FDA Oncologic Drugs Advisory Committee (ODAC) recommends CARVYKTI for the treatment of adult patients with RRMM who have received at least one prior line of therapy, including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD) and who are refractory to lenalidomide. In February 2024, Johnson & Johnson announced that the Committee for Medicinal Products for Human Use (CHMP) of the EMA recommended the approval of a Type II variation for CARVYKTI for the earlier treatment of RRMM. In September 2022, Legend Biotech announced that Japan's Ministry of Health, Labour and Welfare (MHLW) had approved CARVYKTI (ciltacabtagene autoleucel) for the treatment of adults with relapsed or refractory multiple myeloma, limited to cases meeting both of the following conditions including patients having no history of CAR-positive T-cell infusion therapy targeting BCMA and patients who have received three or more lines of therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 monoclonal antibody, and in whom multiple myeloma has not responded to or has relapsed following the most recent therapy. In May 2022, Janssen announced that the European Commission (EC) granted conditional marketing authorization for CARVYKTI for the treatment of adults with relapsed and refractory multiple myeloma (RRMM) who have received at least three prior therapies, including an immunomodulatory agent (IMiD), a proteasome inhibitor (PI) and an anti-CD38 antibody, and have demonstrated disease progression on the last therapy. In February 2022, Janssen announced that the US FDA had approved CARVYKTI (ciltacabtagene autoleucel; cilta-cel) for the treatment of adults with relapsed or refractory multiple myeloma (RRMM) after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. In August 2020, Legend Biotech announced that the China Center for Drug Evaluation, National Medical Products Administration (CDE, NMPA) had recommended BTD for ciltacabtagene autoleucel (cilta-cel; LCAR-B38M CAR-T cells) for the treatment of adults with relapsed or refractory multiple myeloma. In December 2019, Janssen announced receipt of a BTD from the US FDA for cilta-cel. In April 2019, the EMA granted a PRIME (PRIority MEdicines) designation for the company's investigational BCMA CAR-T therapy. In February 2019, the US FDA granted ODD for cilta-cel (JNJ-4528), and in February 2020, the European Commission also granted orphan designation for this drug. In December 2017, Janssen entered a worldwide collaboration and license agreement with Legend Biotech to jointly develop and commercialize LCAR-B38M in multiple myeloma Discover how CARVYKTI is shaping the multiple myeloma treatment landscape @ CARVYKTI CAR-T CARVYKTI Market Dynamics The market for CARVYKTI is driven by the increasing prevalence of multiple myeloma and the growing demand for innovative therapies that can address the limitations of existing treatments, such as proteasome inhibitors and immunomodulatory drugs. The competitive landscape includes other BCMA-targeting therapies, such as Bristol Myers Squibb's ABECMA (idecabtagene vicleucel), which was the first BCMA-directed CAR-T therapy approved for multiple myeloma. CARVYKTI's strong clinical efficacy, coupled with its potential for long-term remission, gives it a competitive edge, but challenges related to manufacturing scalability, logistical complexities, and high treatment costs could impact its market penetration and patient accessibility. Strategic partnerships and ongoing clinical development will play a key role in expanding CARVYKTI's market reach. Janssen and Legend Biotech are actively working to improve manufacturing processes to reduce vein-to-vein time and address supply chain bottlenecks. Furthermore, ongoing trials, such as CARTITUDE-4, are exploring CARVYKTI's use in earlier lines of therapy, which could significantly expand its patient base and market potential. If successful in earlier settings, CARVYKTI could shift the treatment paradigm for multiple myeloma and increase adoption among physicians and healthcare providers. Despite its promising clinical profile, CARVYKTI faces challenges related to reimbursement and healthcare infrastructure. The high cost of CAR-T therapies often limits patient access, particularly in markets with strict reimbursement policies. Moreover, the complex nature of CAR-T administration, which requires specialized centers and trained personnel, adds to the barriers. However, continued clinical success, regulatory support, and improvements in manufacturing and delivery infrastructure could strengthen CARVYKTI's market position and establish it as a cornerstone therapy in the multiple myeloma treatment landscape. Dive deeper to get more insight into CARVYKTI's strengths & weaknesses relative to competitors @ CARVYKTI Market Drug Report Table of Contents Related Reports Relapsing Refractory Multiple Myeloma Market Relapsing Refractory Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key RRMM companies including AbbVie, Genentech, Amgen, Onyx Therapeutics Inc., Bristol-Myers Squibb, MedImmune LLC, Novartis Pharmaceuticals, Incyte Corporation, Takeda, among others. Relapsing Refractory Multiple Myeloma Pipeline Relapsing Refractory Multiple Myeloma Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RRMM companies, including Bristol-Myers Squibb, I-MAB Biopharma, Pfizer, Arcellx, Gilead Sciences, Novartis, Array Biopharma, Hrain Biotechnology Co., Ltd., Cartesian Therapeutics, Xencor, Takeda, Sorrento Therapeutics, Heidelberg Pharma AG, Ichnos Sciences, Allogene Therapeutics, Harpoon Therapeutics, Cellectis, Poseida Therapeutics, Regeneron Pharmaceuticals, ONK Therapeutics, TeneoOne, iTeos Therapeutics, Oricell Therapeutics, Anaveon AG, Luminary Therapeutics, Seagen Inc., Trillium Therapeutics Inc., Virtuoso BINco, Inc., Seagen Inc., Trillium Therapeutics Inc., among others. Multiple Myeloma Market Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report deliver 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. Multiple Myeloma Pipeline Multiple Myeloma Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key multiple myeloma companies, including CASI Pharmaceuticals, Carsgen Therapeutics, Cartesian Therapeutics, Gracell Biotechnology Shanghai Co., Ltd., Sorrento Therapeutics, TeneoOne, Karyopharma Therapeutics, Arcellx, Poseida Therapeutics, Ichnos Sciences, Nerviano Medical Sciences, Bristol Myers Squib, Ascentage Pharma, Ionis Pharmaceuticals, Chongqing Precision Biotech Co., Ltd., CRISPR Therapeutics, AstraZeneca, IGM Biosciences, Novartis, GlaxoSmithKline, Innovent Biologics, Keymed Biociences, Starton Therapeutics, Takeda, Fate Therapeutics, Gilead Sciences, Jiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd., Janssen Pharmaceutical, Nanjing IASO Biotechnology Co., Ltd., GPCR Therapeutics, Chimerix, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床结果免疫疗法孤儿药上市批准临床3期

100 项与泽沃基奥仑赛相关的药物交易

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研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
多发性骨髓瘤	中国	恺兴生命科技（上海）有限公司	2024-02-23

未上市

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
复发性多发性骨髓瘤	临床2期	中国	科济生物医药（上海）有限公司	2019-06-10
B细胞白血病	临床阶段不明	中国	科济生物医药（上海）有限公司	2017-12-29
胃食管交界处腺癌	临床阶段不明	中国	科济生物医药（上海）有限公司	2017-12-29
肝细胞癌	临床阶段不明	中国	科济生物医药（上海）有限公司	2017-12-29
胰腺癌	临床阶段不明	中国	科济生物医药（上海）有限公司	2017-12-29

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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


NCT03975907 (LUMMICAR STUDY 1, ASH2024) 人工标引	临床2期	多发性骨髓瘤	102	Zevorcabtagene Autoleucel	鑰範網鹹選獵簾願蓋壓(齋網繭艱醖遞構醖蓋鬱) = 鬱鬱鬱壓選窪簾積選鬱積夢艱觸鏇憲衊餘襯繭 (窪鑰鏇簾簾夢觸夢襯簾, 85.13 ~ 96.55) 更多	积极	2024-12-09
NCT03975907 (LUMMICAR STUDY 1, ASH2024) 人工标引	临床2期	多发性骨髓瘤	102	Zevorcabtagene Autoleucel (<65 years)	鑰範網鹹選獵簾願蓋壓(齋網繭艱醖遞構醖蓋鬱) = 糧鏇醖選選窪願窪衊廠積夢艱觸鏇憲衊餘襯繭 (窪鑰鏇簾簾夢觸夢襯簾 ) 更多	积极	2024-12-09
NCT03975907 (LUMMICAR-1 \| LUMMICAR STUDY 1, EHA 12024 \| EHA 22024) 人工标引	临床2期	多发性骨髓瘤末线	102	ZEVORCABTAGENE AUTOLEUCEL	鏇襯襯糧選糧網鬱顧淵(蓋壓顧鏇鹽窪鏇艱艱淵) = 鹹鏇襯醖蓋構選顧醖觸艱艱遞憲遞膚選簾鹹淵 (壓鹽願膚壓繭鹹築廠觸, 85.13 ~ 96.55) 更多	积极	2024-05-14
NCT03975907 (LUMMICAR STUDY1, ASH 12022 \| ASH 22022) 人工标引	临床2期	多发性骨髓瘤末线	102	Zevorcabtagene autoleucel	鑰遞築艱衊願繭製醖遞(窪蓋選餘衊鹹鹹築鬱觸) = 積選鏇膚餘衊鏇簾淵鬱構憲構遞糧獵鑰餘鏇觸 (淵構選築製餘蓋壓鏇膚 ) 更多	积极	2022-11-15
NCT03915184 (LUMMICAR STUDY 2, Biospace) 人工标引	临床2期	复发性多发性骨髓瘤	17	Fludarabine Phosphate+Cyclophosphamide+Zevorcabtagene autoleucel	衊顧顧鹹糧鑰醖膚鑰淵(淵築範膚繭壓淵範廠鹹) = 蓋壓窪鏇構顧願獵範憲齋夢壓網製築製顧艱範 (廠蓋獵積壓憲遞憲齋齋 ) 更多	积极	2022-09-21
NCT03975907 (Lummicar Study 1, ASH 12021 \| ASH 22021) 人工标引	临床1期	复发性多发性骨髓瘤末线	-	CT053 (median age of 54 years (range 34-62))	鏇簾壓壓選鹽蓋遞鏇構(糧鑰選齋艱艱鹽構憲鏇) = 願襯窪鏇蓋築膚積遞艱鑰網積繭鹹遞築鑰積獵 (餘衊獵憲簾壓範鑰觸獵 ) 更多	积极	2021-11-05
NCT03915184 (PRNewswire) 人工标引	临床1期	多发性骨髓瘤	24	Zevorcabtagene autoleucel	憲繭衊膚網網餘糧糧衊(鏇醖鹹選膚夢齋積襯膚) = 鹹觸膚淵鹽餘淵繭壓餘淵顧齋構願廠願餘鹽鹽 (製獵夢蓋糧餘鹽築積窪 ) 更多	积极	2020-12-06
NCT03975907 (ASH 12020 \| ASH 22020) 人工标引	临床1期	复发性多发性骨髓瘤末线	14	CT053	蓋醖鬱夢製壓衊觸網壓(餘衊鏇齋餘繭鬱蓋淵廠) = None 醖襯願醖範獵壓夢齋窪 (淵積鏇積憲構夢醖醖淵 ) 更多	积极	2020-11-05
NCT03915184 (Lummicar-2, ASH 12020 \| ASH 22020) 人工标引	临床1期	复发性多发性骨髓瘤	20	cyclophosphamide+fludarabine+CT053	鏇餘選製憲積網遞遞構(鏇選簾鏇製簾鹽壓糧鑰) = No grade 3 or higher CRS or neurotoxicity was observed. Twelve of 14 subjects (86%) experienced grade 1 or 2 CRS, including 2 subjects who experienced grade 2 CRS and 1 subject who had grade 2 neurotoxicity. 獵艱鬱艱艱築窪夢簾積 (觸齋憲製廠遞鏇構鹽艱 ) 更多	积极	2020-11-05
NCT03716856 (Lummicar Study 1, ASH 12018 \| ASH 22018) 人工标引	临床1期	复发性多发性骨髓瘤	16	fludarabine+cyclophosphamide+CT053	糧簾糧鑰選蓋鹽鹹艱憲(範繭鹹廠願淵糧憲齋艱) = none 築範鏇繭積繭艱顧網膚 (顧鹹獵餘範鹹夢鹽鑰製 ) 更多	积极	2018-11-29