The Norwegian Immunotherapy in Multiple Myeloma Study - A Population-based Longitudinal Observational Multicenter Study on Effectiveness and Complications of Immunotherapy in Multiple Myeloma in the Norwegian Myeloma Cohort

The goal of this observational study is to study the effectiveness and complications of novel immunotherapies used in the treatment of multiple myeloma in routine care in Norway. The aim is to close knowledge gaps, generate evidence for future clinical trials and contribute to future consensus on how to monitor for adverse events, and what mitigation strategies should be implemented, so that we can increase patient survival and quality-of-life.

开始日期2025-01-15

申办/合作机构

St. Olavs Hospital HF

[+9]

NCT06698887

/ RecruitingN/A

Long-Term Safety Follow-Up Surveillance for Phase 3 Trial (KarMMA-9/CA089-1043) to Compare the Efficacy and Safety of Idecabtagene Vicleucel With Lenalidomide Maintenance Therapy Versus Lenalidomide Maintenance Therapy Alone in Adult Participants With Newly Diagnosed Multiple Myeloma Who Have Suboptimal Response After Autologous Stem Cell Transplantation

The purpose of this study is to monitor the long-term safety of participants who received idecabtagene vicleucel treatment as part of the KarMMa-9 (CA089-1043) Phase 3 clinical trial.

开始日期2024-09-11

申办/合作机构

Bristol Myers Squibb Co.

100 项与艾基维仑赛相关的临床结果

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100 项与艾基维仑赛相关的转化医学

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100 项与艾基维仑赛相关的专利（医药）

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192

项与艾基维仑赛相关的文献（医药）

2025-12-09·Blood Advances

Safety and efficacy of BCMA-directed chimeric antigen receptor T-cell therapy for the treatment of plasma cell leukemia

Article

作者: Shune, Leyla ; Ferreri, Christopher James ; Clark, William ; Khouri, Jack ; Hovanky, Vanna N. ; Forsberg, Peter ; De Menezes Silva Corraes, Andre ; Purvey, Sneha ; Dhakal, Binod ; Htut, Myo ; De Avilla, Gabriel ; Nazarenko, Elena ; Hashmi, Hamza ; Sborov, Douglas W. ; Galarza Fortuna, Gliceida M. ; Shambavi, Richard ; Hansen, Doris K. ; Dima, Danai ; Godara, Amandeep ; Anderson, Larry ; Atrash, Shebli ; Voorhees, Peter M. ; Herr, Megan M. ; Rossi, Adriana ; Pereira, Denise L. ; Beitinjaneh, Amer ; Lin, Yi ; Davis, James A. ; Afrough, Aimaz ; Sidana, Surbhi ; Mann, Hashim ; Kocoglu, Mehmet H. ; Gaballa, Mahmoud ; Peres, Lauren ; McGuirk, Joseph ; Patel, Krina

Abstract:

Despite significant therapeutic advances in multiple myeloma (MM), outcomes in patients with plasma cell leukemia (PCL) remain dismal. We conducted a multicenter retrospective analysis of patients with PCL who were treated with the B-cell maturation antigen–directed chimeric antigen receptor T-cell (CAR-T) products idecabtagene vicleucel (ide-cel) and ciltacabtagene autoleucel (cilta-cel). We identified 34 patients; 19 patients received ide-cel and 15 received cilta-cel. With a median follow-up of 11.9 months, the overall median progression-free survival (mPFS) was 9.0 months (95% confidence interval [CI], 4-15) and the median overall survival (mOS) was 13.0 months (95% CI, 8 to not estimable [NE]). The 1-year cumulative incidence of progression or death was 72%, and the 1-year cumulative incidence of death was 47%. Patients who received cilta-cel had a longer mPFS (19.0 months vs 6.0 months) and mOS (>23 months [NE] vs 9.0 months) when compared with those treated with ide-cel. Similarly, the 1-year cumulative incidence of disease progression or death was 37.5% (95% CI, 17.4-68.5) with cilta-cel, whereas all patients treated with ide-cel progressed or died within 12 months of infusion. The rates of hematologic and nonhematologic toxicities were similar between patients treated with cilta-cel and those treated with ide-cel and were consistent with those reported in patients with MM. In this first multicenter study that evaluated patients with PCL who were treated with standard-of-care CAR-T products, we show that CAR-T is safe, feasible, and associated with improved outcomes when compared with historic standards.

2025-11-01·Drug information journal

Post-marketing Safety Assessment of CAR T-cell Therapies: Analysis of Individual Case Safety Reports in the VigiBase

Article

作者: AlFadel, Nouf S ; AlRasheed, Meshael M ; AlOrf, Nora N ; AlHawas, Solaiman M ; AlHarbi, Fawaz F

PURPOSE:

Chimeric Antigen Receptor (CAR)-T cell therapies have emerged as potential therapy for hematological malignancies, however, their safety profiles should be monitored after approval. Therefore, we aimed in this study to explore and analyze individual case safety report (ICSRs) associated with CAR-T cell therapies and reported to the World Health Organization (WHO) global database (VigiBase).

METHODS:

A retrospective pharmacovigilance study was conducted to describe and characterize Adverse drug reactions (ADRs) reported to Vigibase from inception to March 31st, 2024 and associated with use of the following CAR-T cell therapies: Tisagenlecleucel, Axicabtagene ciloleucel, Brexucabtagene autoleucel, Lisocabtagene maraleucel, Idecabtagene vicleucel, and Ciltacabtagene autoleucel.

RESULTS:

A total of 11,693 ICSRs were identified with the use of CAR-T cell therapies in VigiBase (Axicabtagene ciloleucel (N = 5668, 48.5%), Tisagenlecleucel (N = 3364, 28.8%), Brexucabtagene autoleucal (N = 1027, 8.8%), Lisocabtagene maraleucel (N = 304, 2.6%), Idecabtagene vicleucel (N = 579, 4.9%), and Ciltacabtagene autoleucel (N = 751, 6.4%)). ICSRs completeness score was averaged between 0.2 and 0.57 among all included products and the majority of reported ADRs were serious (67-91%). Among serious ADRs, death was reported with an average percentage of (8.8-21.5%). The majority of ADR reports with fatal outcome occurred in accordance with their approved indications. About 18% of fatal events reported with Tisagenlecleucel as the suspected drug were in the pediatric population.

CONCLUSION:

Our study provides an overall exploration of the post-marketing safety profiles of currently approved CAR-T cell therapies. The significant proportion of fatalities occurred in accordance with approved indications, emphasizes the need for ongoing investigation into ADRs with fatal outcomes, particularly in the pediatric population.

2025-11-01·JOURNAL OF CLINICAL PHARMACOLOGY

Characterizing Cellular Expansion of Idecabtagene Vicleucel and Association with Clinical Efficacy and Safety in Patients with Triple‐Class‐Exposed Relapsed/Refractory Multiple Myeloma

Article

作者: Caia, Andrea ; Piasecki, Julia ; Zhang, Wanying ; Kondic, Anna ; Zhou, Jian ; Wu, Fan ; Zheng, Xirong ; Lamba, Manisha ; Cook, Mark ; Masilamani, Madhan ; Zhong, Xiaobo ; Burnett, Joseph

Abstract:

Idecabtagene vicleucel (ide‐cel, ABECMA) is an autologous, B‐cell maturation antigen‐directed, chimeric antigen receptor (CAR) T‐cell therapy, which has demonstrated significantly improved progression‐free survival (PFS) and overall response rate (ORR) in patients with triple‐class‐exposed relapsed/refractory multiple myeloma (TCE RRMM). Here, we characterize cellular expansion of ide‐cel in vivo and further evaluate associations between cellular expansion and clinical efficacy and safety endpoints. The exposure parameters of ide‐cel were evaluated through non‐compartmental analysis methods using the time course data of CAR transgene copy numbers collected from the ide‐cel arm of Study KarMMa‐3 (NCT03651128). Multivariable regression analyses were conducted between the exposure parameters and clinical responses to characterize relationships between cellular expansion in vivo and clinical outcomes and to evaluate potential effects of covariates on the exposure–response (E‐R) relationships. There appears to be lack of a strong association between actual ide‐cel dose and cellular expansion at the dose range evaluated in Study KarMMa‐3. The multivariable E‐R regression models suggest positive relationships between cellular expansion and clinical efficacy and safety endpoints, with higher exposure associated with longer PFS, higher ORR, and higher rates of cytokine release syndrome requiring tocilizumab or corticosteroids. The current analyses do not identify any clinically relevant covariate effects on the E‐R relationships. The positive exposure–response relationships were found to be overall similar between KarMMa‐3 and a previous study KarMMa. The modeling analyses, paired with clinical data, support extending the dose range from previously approved 300‐460 × 10 6 CAR+ T cells to 300‐510 × 10 6 CAR+ T cells for TCE RRMM patients.

767

项与艾基维仑赛相关的新闻（医药）

2025-12-04

·药研网

BCMA加速跨界自免蓝海

前言曾经在血液肿瘤领域“叱咤风云”的明星靶点 BCMA，正凭借其独特的生物学特性，从肿瘤领域加速迈向自身免疫，开启一场新的“免疫重塑”浪潮。今年6月，和铂医药与大塚制药达成全球战略合作，共同推进用于自身免疫性疾病的BCMAxCD3双特异性T细胞衔接器HBM7020开发。过去一年间，从岸迈生物、康诺亚到智翔金泰，短短10个月内已有四款BCMA/CD3双抗布局自免疾病，显示这一靶点正在成为新的热点。 01 BCMA：机制与靶向策略解析 B细胞成熟抗原（BCMA，TNFRSF17或CD269），是肿瘤坏死因子（TNF）受体（TNFR）超家族成员, 是维持浆细胞命运的重要跨膜糖蛋白。[1] BCMA 的主要配体包括TNFR超家族另外两个成员—增殖诱导配体（APRIL）和B细胞激活因子（BAFF），其中APRIL对BCMA的亲和力更高。 BCMA 在造血干细胞与其他非免疫组织中几乎不表达，而在成熟B细胞尤其是长寿命骨髓浆细胞（LLPC）中高度表达，对其生存和抗凋亡状态具有关键调控作用。在多发性骨髓瘤（MM）中，BCMA 在所有患者的恶性浆细胞上均呈高表达，而正常组织表达极低，使其兼具“高选择性”与“安全窗口”这一罕见特质。[2] 图1. BCMA在浆细胞（PC）中的生物学意义当BCMA与APRIL/BAFF结合后，可激活多种信号通路，主要包括 NF-κB、MAPK 和 PI3K/Akt，这些通路共同促进抗凋亡蛋白的上调、细胞增殖的增强，并诱导一系列黏附、血管生成和免疫抑制分子的表达，从而形成一个有利于浆细胞（尤其是骨髓瘤细胞）持续存活的微环境。 BCMA 还会与 CD138、HSPG 以及转录因子 IRF-4 等分子协同作用，进一步强化其在浆细胞生物学中的核心地位。与此同时，部分膜 BCMA 会被切割为可溶性的 sBCMA，它能够竞争性结合 APRIL/BAFF，在一定程度上调节并削弱 BCMA 的信号强度。图2. BCMA信号通路吉满生物自主研发了一系列BCMA靶点相关产品，包括稳定过表达细胞系、报告基因检测细胞系、抗体及蛋白相关产品，充分满足药物研发需求，推动临床申报加速落地。我们拥有HEK293、Jurkat E6.1、CHO-K1细胞商业化使用授权，同时拥有HEK293细胞中国内地定向用途分授权独家代理，可提供合规性授权解决方案。为客户从早期研发到产品上市保驾护航。咨询吉满客服联系同微信：18916119826 02 自免领域正在火速拥抱BCMA 针对BCMA的新型药物的研发正在快速进行，治疗方式多种多样，包括：CAR-T、双/三抗（含BiTE）、ADC（总结于表1），以及癌症疫苗。 CAR-T细胞疗法是目前针对BCMA的最活跃的研发领域之一，展现出显著疗效。迄今为止，已有四款CAR-T细胞（抗BCMA）免疫治疗产品获批用于治疗R/R MM患者：idecabtagene vicleucel(Abecma)、西达基奥仑赛(Carvykti)，国内信达生物/驯鹿医疗的伊基奥仑赛和科济生物/华东医药的泽沃基奥仑赛。 2025年10月16日，创新细胞疗法的生物制药公司驯鹿生物，宣布国际顶级学术期刊《Cell》在线发表了其自主研发的全人源靶向BCMA CAR-T细胞疗法伊基奥仑赛注射液治疗进展型多发性硬化症的研究结果。该研究不仅在全球首次证实CAR-T细胞疗法在治疗进展型多发性硬化中的安全性和有效性，同时也成为《Cell》首次发表靶向BCMA CAR-T细胞疗法成功治疗自身免疫性疾病的研究成果，标志着该领域的重要科学突破。[3] 图3.伊基奥仑赛注射液在治疗进展型多发性硬化症（PMS）方面表现出显著疗效和良好的安全性基于BCMA 在 MM 的成功，证明其作为免疫疗法靶点的强大杀伤力与安全窗口。目前多项临床试验正评估这些产品在自身免疫疾病中的安全性和有效性。以系统性红斑狼疮（SLE）为例，一项题为 B-Cell Maturation Antigen (BCMA) as a Biomarker and Potential Treatment Target in Systemic Lupus Erythematosus 的研究显示，SLE患者 B 细胞亚群的 BCMA 表达普遍升高，并与浆母细胞比例及血清自身抗体水平呈正相关，提示 BCMA 不仅可作为疾病活动的生物标志物，也具有潜在的治疗价值。此外，在Sanges等人的研究中，SSc患者的肺动脉高压患者sBCMA水平较研究人群其他患者更高（p = 0.03）,进一步支持了靶向 BCMA 的治疗可行性。[4,5] 表1.全球已上市/在研 BCMA靶点药物（据药研网不完全统计）虽然针对BCMA的CAR-T细胞疗法一直是重点，但BCMA双抗（含BiTE）、三抗等新型疗法已成为有前景的替代治疗方案。双特异性抗体通过同时结合BCMA和T细胞表面抗原，使这两种细胞相互靠近，增强T细胞对肿瘤细胞的杀伤能力。在BCMA双特异性抗体领域，已有三款药物Teclistamab、Elranatamab 和 Linvoseltamab 获批上市，用于RRMM 的治疗。其中Teclistamab，它是第一个获得FDA批准的靶向BCMA的双特异性抗体，也是全球首个BCMA/CD3双特异性抗体。此外，CM336、GR1803 等在研 BCMA 双特异性 T 细胞衔接器项目，伴随合作授权的落地，将加速向自身免疫性疾病等新适应症拓展。针对三抗药物，目前临床开发也在积极推进：JNJ-5322 (Johnson & Johnson) 已进入 III 期临床，用于复发/难治性多发性骨髓瘤 (RRMM)；ISB-2001 (Ichnos Glenmark Innovation) 和 QLS4131 (齐鲁制药) 处于 I 期临床，针对RRMM，后者已拓展SLE 适应症。 ADC则是将抗体与细胞毒性药物结合，通过靶向BCMA直接将药物传递至恶性浆细胞。这类药物如Belantamab mafodotin（商品名 Blenrep），已获美国FDA批准，显著改善了多发性骨髓瘤的治疗效果，是目前唯一获批的 BCMA‑ADC 药物。总结凭借高度特异的表达和安全窗口，BCMA正从血液肿瘤迈向自身免疫领域，CAR-T、双抗、ADC多种策略齐发，正加速开启一场免疫治疗的新纪元。吉满生物吉满生物自主研发研发了一系列BCMA靶点相关产品，包括稳定过表达细胞系、报告基因检测细胞系、抗体及蛋白相关产品，充分满足药物研发需求，推动临床申报加速落地。 1 靶点相关细胞系：适用于药物功能筛选、活性评价、药效学研究及信号通路验证 2 高活性重组蛋白：满足结合分析、筛选及免疫研究需求 3 高特异性抗体：提供经严格验证的抗体产品，支持流式细胞术、免疫组化、Western Blot、ELISA 等多种检测应用，确保实验结果准确可靠 BCMA 产品列表及数据展示 1 细胞系数据展示 GM-C24896 H_BCMA HEK-293 Cell Line，经过流式验证，表达稳定可靠，为BCMA 靶点研究、抗体筛选及机制解析提供高质量实验工具。使用Anti-BCMA hIgG1 Antibody流式验证结果 GM-C25349 H_BCMA Reporter Cell Line，传代稳定，25代信号响应表现仍然优秀,功能验证结果表现可靠，可以有效帮助用户进行药物筛选和活性评价。使用Human BAFF Protein;His Tag稳定性验证结果分别使用Anti-BCMA hIgG1 Antibody(SEA-BCMA)、 Anti-BCMA hIgG1 Antibody(Belantamab)抗体验证结果 2 蛋白数据展示吉满生物BCMA蛋白相关产品，经多次ELISA验证，表现出优异的灵敏度和特异性，活性高，稳定性强。 GM-88229RP Human BCMA Protein; His Tag ELISA验证 Human BCMA Protein; hFc Tag ELISA验证联系我们吉满生物(Genomeditech)成立于2011年，是专业从事生物科技服务和前沿技术研发的高新技术企业。吉满生物专注为生物药开发赋能，自主创立了国内知名的细胞系品牌-DDXCELL，目前已布局近400个热门靶向药靶点，超1000株现货单克隆细胞系，涵盖GPCR、细胞因子、免疫检查点、TAA等多领域，做到进口细胞的国产替代。此外，吉满生物还可在药物研发进程中提供一系列抗体、蛋白产品，旨在为客户提供高效的研发工具和解决方案! 扫码咨询扫码找现货参考资料 1.Cho SF, Anderson KC, Tai YT. Targeting B Cell Maturation Antigen (BCMA) in Multiple Myeloma: Potential Uses of BCMA-Based Immunotherapy. Front Immunol. 2018 Aug 10;9:1821. doi: 10.3389/fimmu.2018.01821. PMID: 30147690; PMCID: PMC6095983. 2.Shah, N., Chari, A., Scott, E. et al. B-cell maturation antigen (BCMA) in multiple myeloma: rationale for targeting and current therapeutic approaches. Leukemia 34, 985–1005 (2020). https://doi.org/10.1038/s41375-020-0734-z 3.www.cell.com/cell/fulltext/S0092-8674(25)01088-8 4.Martin, J.; Cheng, Q.; Laurent, S.A.; Thaler, F.S.; Beenken, A.E.; Meinl, E.; Krönke, G.; Hiepe, F.; Alexander, T. B-Cell Maturation Antigen (BCMA) as a Biomarker and Potential Treatment Target in Systemic Lupus Erythematosus. Int. J. Mol. Sci. 2024, 25, 10845. https://doi.org/10.3390/ijms251910845 5.Sanges, S.; Guerrier, T.; Duhamel, A.; Guilbert, L.; Hauspie, C.; Largy, A.; Balden, M.; Podevin, C.; Lefèvre, G.; Jendoubi, M.; et al. Soluble Markers of B Cell Activation Suggest a Role of B Cells in the Pathogenesis of Systemic Sclerosis-Associated Pulmonary Arterial Hypertension. Front. Immunol. 2022, 13, 954007. 6.https://finance.sina.com.cn/stock/hkstock/ggscyd/2025-06-23/doc-infazmin0775730.shtml 7.https://www.globenewswire.com/news-release/2024/09/04/2940171/0/en/EpimAb-Biotherapeutics-and-Vignette-Bio-Announce-Strategic-Collaboration-to-Develop-EMB-06-a-BCMA-CD3-Bispecific-Antibody.html 8.https://www.prnasia.com/story/475616-1.shtml 9.https://www.pharmadj.com/ssr/article/1930600199804284929

2025-12-02

·ETPharma

Bristol Myers must face $6.7 billion lawsuit over delayed cancer drug, US judge rules

New York: A US judge on Monday rejected Bristol Myers Squibb's bid to dismiss a $6.7 billion lawsuit claiming it cheated shareholders of the former Celgene by delaying federal approval for three drugs, including the cancer treatment Breyanzi . While dismissing some claims, U.S. District Judge Jesse Furman in Manhattan said the plaintiff, UMB Bank , may pursue some claims on behalf of the shareholders, including for Bristol Myers' alleged breach of contract and failure to act in good faith. Bristol Myers and lawyers for the Princeton, New Jersey-based company did not immediately respond to requests for comment. A lawyer for UMB declined to comment. The lawsuit arose from Bristol Myers' $80.3 billion purchase of Celgene in 2019. Bristol Myers agreed to pay shareholders who held "contingent value rights" (CVR) an extra $9 per share in cash if it won timely U.S. Food and Drug Administration approvals for Liso-cel, which is sold as Breyanzi , and the drugs Ozanimod and Ide-cel. UMB, in its role as a trustee, said Bristol Myers slow-rolled the process by not using "diligent efforts" to win approvals, and wrongly delisted the CVRs from the New York Stock Exchange before holders could enforce their rights. Bristol Myers won FDA approval for Breyanzi to treat non-Hodgkin lymphoma on February 5, 2021, five weeks after the relevant deadline. In a 24-page decision, Furman said UMB had legal standing to sue, after addressing concerns he raised in a September 2024 dismissal of its earlier lawsuit. The judge also rejected Bristol Myers' claim that its alleged lack of diligence did not trigger an "event of default," and said a jury should decide whether Bristol Myers breached an implied agreement to act in good faith by delisting the CVRs. Furman gave Bristol Myers three weeks to formally respond to UMB's claims. (Reporting by Jonathan Stempel in New York; Editing by Rod Nickel)

并购上市批准专利侵权

2025-12-02

·药时空

细胞疗法“大退潮”！6家企业逆势坚守，CAR-T三巨头领跑创新

2025年末，细胞治疗领域正经历一场“大退潮”。继武田（Takeda）宣布全面退出，诺和诺德（Novo Nordisk）终止所有项目，Vertex、Galapagos等企业的管线研发受挫，行业信心一度受挫。然而，据BioSpace的观察，在这场寒流中，仍有六家公司选择逆流而上——他们不仅没有收缩，反而加大投入，将细胞治疗从血液肿瘤拓展至实体瘤、自身免疫病等更广阔领域。其中，BMS、吉利德和强生是CAR-T领域的三大领军者，其凭借已上市产品的优势和持续的研发投入，牢牢占据行业头部位置。Immatics、Kyverna和Cabaletta 三家生物科技公司则凭借差异化布局，开辟了细分蓝海——Immatics则押注广谱靶点PRAME，攻坚实体瘤；Kyverna与Cabaletta引领CAR-T进军自身免疫病，开拓新疆域。下面，本文将带您了解这些“坚守者”的战略布局与技术突破。 1、BMS：CAR-T双获批，布局全面作为全球唯一一家拥有两款获批CAR-T细胞疗法且分别靶向不同靶点的公司——Abecma（靶向BCMA），用于复发或难治性多发性骨髓瘤；Breyanzi（靶向CD19），用于多种血液癌症。BMS在细胞治疗领域已建立显著优势。但BMS并未止步。2025年10月，公司以15亿美元收购Orbital Therapeutics，进入体内（in vivo）CAR-T领域。收购使BMS获得一款在研体内CAR T细胞治疗产品OTX-201以及Orbital的RNA平台技术。OTX-201旨在重置免疫系统以治疗自身免疫疾病。更令人瞩目的是，BMS正将CAR-T应用于自身免疫疾病。10月25日，BMS首次公布了其在2025美国风湿病学会（ACR）年会上展示的1期BREAKFREE-1研究更新数据，披露了在慢性难治性自身免疫疾病（包括系统性红斑狼疮、系统性硬化症、特发性炎性肌病和类风湿关节炎）中的结果。此次公布的1期初步安全性与疗效结果，与“免疫重置”潜力一致：整个治疗过程仅需一次性输注CD19 NEX-T，即可在所有三个队列中观察到强劲的CAR-T细胞扩增、B细胞完全清除以及初始B细胞表型的重新出现。截至分析时，94%的可评估患者已停用慢性免疫抑制治疗。 2、吉利德/Kite：押注体内CAR-T，联手中国创新力量吉利德通过子公司Kite Pharma，同样手握两款获批CAR-T——Yescarta与Tecartus，均靶向CD19，并适用于多种血癌。目前，其战略重心正转向下一代CAR-T技术： 2025年8月，以3.5亿美元收购Interius BioTherapeutics，获得体内CAR-T平台； 2025年10月，再与中国的生物公司普瑞金（Pregene）达成了一项价值高达16.4亿美元的合作协议，共同开发体内生成CAR-T的新疗法。同时，吉利德与Arcellx合作的anito-cel（靶向BCMA），目前正针对复发或难治性多发性骨髓瘤展开临床试验，双方正在开展II期iMMagine-1试验和III期iMMagine-3试验。今年5月公布的中期数据显示，该药物的总缓解率达到97%，其中完全缓解/严格完全缓解率为68%，数据极为亮眼。 3、强生：Carvykti强势崛起，双靶点CAR-T蓄势待发强生与传奇生物合作的Carvykti（靶向BCMA）已成为多发性骨髓瘤市场的重磅产品，2024年全球销售额达9.63亿美元，在该市场直追BMS，去年Breyanzi销售额为7.47亿美元，Abecma为4.06亿美元。 Carvykti的市场表现得益于其卓越的临床数据：在CARTITUDE-4 III期试验中，89%的Carvykti治疗参与者在中位随访三年时无可检测癌细胞，相较于现有标准疗法具有很高的统计显著性。此外，强生自研的JNJ-4496（双靶点CAR-T）在大B细胞淋巴瘤Ib期试验中实现100%客观缓解率，如果获批，其有望成为Yescarta的有力竞争者。 4、Immatics：聚焦PRAME靶点，攻坚实体瘤 Immatics则选择了一条差异化路径——瞄准PRAME蛋白，其在50多种癌症（如子宫内膜癌、子宫癌、卵巢癌和肺癌，以及黑色素瘤）中表达，而在健康组织几乎不表达，是理想的实体瘤靶点。公司针对PRAME的主导产品是anzu-cel，这是一种通过识别PRAME后触发强烈抗癌免疫反应的T细胞疗法。该资产进展如下：正在SUPRAME Ⅲ期试验中评估，作为皮肤黑色素瘤的二线治疗，Immatics预计将在2027年下半年推出该疗法；用于葡萄膜黑色素瘤启动了Ⅱ期试验，计划在美国和德国招募约30名患者，其Ib期读数显示，确诊的客观缓解率为67%，疾病控制率为88%；与Moderna合作，推进anzu-cel与mRNA癌症疫苗mRNA-4203的联合治疗方案，用于治疗皮肤黑色素瘤和滑膜肉瘤，探索协同增效。 5、Kyverna：用CAR-T治疗重症肌无力，疗效惊艳 Kyverna Therapeutics专注于自身免疫疾病，其候选药KYV-101是一款全人源、CD19靶向自体CAR-T疗法，具有CD28共刺激域，旨在实现高效活性与良好耐受性，目前正在用于治疗B细胞驱动的自身免疫性疾病。 10月29日，Kyverna 公布了KYV-101治疗全身型重症肌无力（gMG）的注册性2/3期临床试验KYSA-6的II期部分积极中期数据，100%（6/6）患者达到应答标准，被分析师称为“设定新疗效标准”，Kyverna预计在今年年底前启动3期试验部分的患者入组。此外，KYV-101还在僵人综合征（SPS）、狼疮肾炎、多发性硬化症、类风湿关节炎和系统性硬化症等多个罕见或重大自身免疫病中推进临床。其中，其针对僵人综合征处于注册Ⅱ期临床试验中，预计明年初将有主要数据。FDA的申请计划于2026年上半年提交。 6、Cabaletta：广谱布局自身免疫，机制独特 Cabaletta Bio的CAR-T产品rese-cel（CABA-201）同样靶向CD19，但采用4-1BB共刺激域，可实现完全但暂时的B细胞清除，触发“免疫系统重置”。在2025年ACR会议上，rese-cel的Ⅰ/Ⅱ期读数在三大适应症中展现潜力。在RESET-Myositis试验中，Cabaletta为6名患者进行了给药，其中4人患有皮肌炎，2人患有抗合成酶综合征。结果显示，在未使用免疫调节剂的情况下，接受rese-cel治疗16周后，患者的疾病应答实现了“中度或显著改善”。基于这些研究结果，公司正在RESET-Myositis试验中推进一个注册队列研究，针对上述两种适应症展开探索。公司计划在年底前为该队列招募14名患者。此外，rese-cel治疗让3名接受治疗的系统性红斑狼疮（SLE）患者实现病情缓解，另有1名狼疮性肾炎患者达到了肾脏完全应答。与此同时，在系统性硬化症（SSc）适应症中，该疗法实现了免疫系统重置，即便未使用基础免疫调节剂，也为患者带来了临床获益。公司计划在今年与FDA 就这些适应症的关键研究设计达成一致。 rese-cel其他适应症还包括全身性重症肌无力（gMG）、复发型和进展型多发性硬化症以及寻常型天疱疮。结语：寒冬中的火种，照亮细胞治疗未来尽管细胞疗法领域近期出现了一些巨头退出的现象，但这更像是一场行业从“淘金热”转向“理性深耕”的洗牌。这些坚守的企业，或将成为未来细胞疗法行业复苏的核心力量，推动这一疗法从“小众高端治疗”向更普惠、更多适应症的方向发展。也正是他们的坚守与创新，共同描绘了细胞疗法充满希望的未来图景。参考资料：6 Companies Hanging On in Cell Therapy.BioSpaceBy Tristan Manalac.December 1, 2025. 识别微信二维码，可添加药时空小编请注明：姓名+研究方向！

细胞疗法免疫疗法并购临床1期临床结果

100 项与艾基维仑赛相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11556	-	L01XX	-

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	美国	Celgene Corp.	2021-03-03

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3期	美国	中美上海施贵宝制药有限公司	2022-01-30
复发性多发性骨髓瘤	临床3期	美国	2seventy Bio, Inc.	2022-01-30
BCMA阳性多发性骨髓瘤	临床1期	美国	Celgene Corp.	2015-12-22
BCMA阳性多发性骨髓瘤	临床1期	美国	bluebird bio, Inc.	2015-12-22

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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


NCT03361748 (KarMMa, Pubmed \| Blood)	临床2期	复发性多发性骨髓瘤 high-risk cytogenetics	821	Standard-of-care idecabtagene vicleucel	遞鬱網窪衊壓鹹構夢壓(廠艱壓鬱顧遞艱觸鏇獵) = Clinically significant infections were seen in 45% of patients 壓膚齋淵鏇範艱鑰襯獵 (廠製糧衊願淵積製醖廠 ) 更多	积极	2025-07-10
ASCO2025	N/A	自身免疫性疾病 \| 多发性骨髓瘤 AIHA \| autoimmune thyroiditis \| ITP ... 更多	179	idecabtagene vicleucel (Patients with autoimmune disease (AD))	簾醖築網廠築壓鹽鬱選(襯膚齋範選鑰簾願衊觸) = 襯壓憲糧鏇積鹹廠膚艱繭壓選廠製鏇艱鑰襯廠 (糧憲鏇築構製餘願淵鬱 ) 更多	积极	2025-05-30
				idecabtagene vicleucel (Patients without autoimmune disease (AD))	簾醖築網廠築壓鹽鬱選(襯膚齋範選鑰簾願衊觸) = 憲壓蓋衊鹽鏇鹹夢網淵繭壓選廠製鏇艱鑰襯廠 (糧憲鏇築構製餘願淵鬱 ) 更多
ASCO2025	N/A	复发性多发性骨髓瘤	-	Idecabtagene vicleucel (IC)	願選憲襯顧膚簾壓鹹醖(鹹範齋範製鏇製繭餘艱) = 願醖膚遞壓鹹廠淵網構艱齋糧繭願網構醖壓糧 (範鹽網鹽構繭壓觸壓鑰 ) 更多	积极	2025-05-30
				Ciltacabtagene autoleucel (CC)	願選憲襯顧膚簾壓鹹醖(鹹範齋範製鏇製繭餘艱) = 鹹遞艱願糧窪糧築繭範艱齋糧繭願網構醖壓糧 (範鹽網鹽構繭壓觸壓鑰 ) 更多
ASH2024	N/A	复发性多发性骨髓瘤	-	Cilta-cel	願構醖網醖積壓鹹夢製(醖淵餘觸鹽網艱鏇衊壓) = 製壓憲鬱艱鑰鏇觸餘鑰鏇廠鬱蓋糧積壓願衊繭 (繭襯鏇憲憲繭膚鑰簾壓 ) 更多	-	2024-12-09
				Ide-cel	願構醖網醖積壓鹹夢製(醖淵餘觸鹽網艱鏇衊壓) = 襯願襯鹹醖夢醖網繭鏇鏇廠鬱蓋糧積壓願衊繭 (繭襯鏇憲憲繭膚鑰簾壓 ) 更多
3791CAR-T (ASH2024) 人工标引	N/A	多发性骨髓瘤	-	Ide-cel	築鑰製窪淵齋醖遞淵醖(齋簾選廠築餘齋襯餘衊) = 蓋憲壓廠醖鹽範鏇襯積觸簾鏇遞鬱餘齋襯鏇鬱 (簾鹹憲積窪獵顧襯夢窪 ) 更多	相似	2024-12-08
				Cilta-cel	築鑰製窪淵齋醖遞淵醖(齋簾選廠築餘齋襯餘衊) = 壓醖膚糧鹽鹽膚齋簾齋觸簾鏇遞鬱餘齋襯鏇鬱 (簾鹹憲積窪獵顧襯夢窪 ) 更多
ASH2024	N/A	多发性骨髓瘤	-	idecabtagene vicleucel (ide-cel)	構獵鹽壓齋壓獵遞壓餘(艱簾網範齋願艱鑰鑰簾) = 夢遞顧觸顧夢鑰衊艱齋壓餘憲顧襯齋鹹構餘獵 (繭膚蓋壓願鏇廠艱廠鑰 ) 更多	-	2024-12-08
ASH2024	N/A	复发性多发性骨髓瘤	-	Ciltacabtagene Autoleucel	鏇艱壓窪衊遞窪願窪簾(憲鑰壓簾繭鑰鑰獵願壓) = 獵襯壓顧膚顧簾積築夢鑰網蓋廠構壓製獵網鑰 (廠鑰膚廠顧襯蓋餘憲製 ) 更多	-	2024-12-07
				Idecabtagene Vicleucel	鏇艱壓窪衊遞窪願窪簾(憲鑰壓簾繭鑰鑰獵願壓) = 遞簾積夢願遞鏇鏇膚膚鑰網蓋廠構壓製獵網鑰 (廠鑰膚廠顧襯蓋餘憲製 ) 更多
not available (ASCO2024)	N/A	多发性骨髓瘤	351	idecabtagene vicleucel (Extramedullary disease (EMD))	憲觸範積窪築壓憲窪餘(膚鏇鑰蓋糧觸網遞鹹窪) = 築遞簾齋顧鬱襯憲鹹繭齋鹽憲獵夢衊觸構膚夢 (糧顧繭艱願築廠鬱夢構 ) 更多	不佳	2024-05-24
				idecabtagene vicleucel (Non-EMD)	憲觸範積窪築壓憲窪餘(膚鏇鑰蓋糧觸網遞鹹窪) = 築範觸衊憲遞憲壓鏇餘齋鹽憲獵夢衊觸構膚夢 (糧顧繭艱願築廠鬱夢構 ) 更多
NCT03601078 (KarMMa-2, EHA 12024 \| EHA 22024) 人工标引	临床2期	复发性慢性淋巴细胞白血病 \| 难治性慢性淋巴细胞白血病二线	35	Idecabtagene vicleucel	鑰範窪鹽膚蓋廠繭糧襯(蓋壓齋醖觸膚築窪夢顧) = 糧憲襯獵簾範築糧構繭淵衊壓蓋獵簾餘鏇膚壓 (鹹壓憲鹹簾繭遞餘膚簾, 52 ~ 86) 更多	积极	2024-05-14
CIBMTR Registry (EHA2024)	N/A	多发性骨髓瘤	817	IDECABTAGENE VICLEUCEL (IDE-CEL) (Very Good Partial Response (VGPR) or better)	襯網艱鏇構鹹簾鏇築鏇(淵窪窪壓網鹽觸淵選構) = observed in SD and PD groups 憲壓顧鹽願願蓋膚淵鬱 (憲憲艱糧窪餘壓艱憲範 ) 更多	积极	2024-05-14
				IDECABTAGENE VICLEUCEL (IDE-CEL) (Stable Disease (SD) or Progressive Disease (PD))