An Open-label, Multi-center, Single-arm Phase 1/2 Study to Assess Tolerability, Safety and Efficacy of CRC01 in Adult Patients With Relapsed or Refractory Large B-cell Lymphoma

This is a multi-center, phase I/II study to determine the efficacy and safety of CRC01 in adult patients with relapsed or refractory large B-cell lymphoma.

开始日期2021-03-02

申办/合作机构

Curocell, Inc.

100 项与安基奥仑赛相关的临床结果

登录后查看更多信息

100 项与安基奥仑赛相关的转化医学

登录后查看更多信息

100 项与安基奥仑赛相关的专利（医药）

登录后查看更多信息

项与安基奥仑赛相关的文献（医药）

2021-06-01·The Lancet Oncology1区 · 医学

Trastuzumab deruxtecan (DS-8201) in patients with HER2-expressing metastatic colorectal cancer (DESTINY-CRC01): a multicentre, open-label, phase 2 trial

1区 · 医学

Article

作者: Fotios Loupakis ; Maria Di Bartolomeo ; Emarjola Bako ; Fortunato Ciardiello ; Taito Esaki ; Kanwal Raghav ; Ki Chung ; Marwan Fakih ; Kapil Saxena ; Kensei Yamaguchi ; Tomohiro Nishina ; Javad Shahidi ; Elena Elez ; Andrea Sartore-Bianchi ; Zev Wainberg ; Javier Rodriguez ; Salvatore Siena ; Toshiki Masuishi ; Axel Grothey ; Yoshito Komatsu ; Destiny-Crc investigators ; Eriko Yamamoto ; Yasuyuki Okuda ; Hisato Kawakami ; Takayuki Yoshino

BACKGROUNDHER2 amplification has been identified in 2-3% of patients with colorectal cancer, although there are currently no approved HER2-targeted therapies for colorectal cancer. We aimed to study the antitumour activity and safety of trastuzumab deruxtecan (an antibody-drug conjugate of humanised anti-HER2 antibody with topoisomerase I inhibitor payloads) in patients with HER2-expressing metastatic colorectal cancer.METHODSDESTINY-CRC01 is an open-label, phase 2 study that recruited patients from 25 clinics and hospitals in Italy, Japan, Spain, the UK, and the USA. Eligible patients had centrally confirmed HER2-expressing metastatic colorectal cancer that had progressed on two or more previous regimens (HER2-targeted therapies other than trastuzumab deruxtecan permitted), were aged 18 years or older (≥20 years in Japan), had an Eastern Cooperative Oncology Group score of 0 or 1, and had RAS and BRAF^V600E wild-type tumours. Patients were enrolled into one of three cohorts by HER2 expression level: cohort A (HER2-positive, immunohistochemistry [IHC] 3+ or IHC2+ and in-situ hybridisation [ISH]-positive), cohort B (IHC2+ and ISH-negative), or cohort C (IHC1+). Patients received 6·4 mg/kg trastuzumab deruxtecan intravenously every 3 weeks until disease progression, unacceptable adverse events, withdrawal of consent, or death. The primary endpoint was confirmed objective response rate in cohort A by independent central review which was assessed in the full analysis set and safety was assessed in the safety analysis set. Both the full analysis set and the safety analysis set included all patients who received one or more doses of trastuzumab deruxtecan. This ongoing trial is registered with ClinicalTrials.gov, number NCT03384940.FINDINGSBetween Feb 23, 2018, and July 3, 2019, 78 patients were enrolled in the study (53 in cohort A, seven in cohort B, and 18 in cohort C), all of whom received at least one dose of study drug. For the 53 (68%) patients with HER2-positive tumours (cohort A), a confirmed objective response was reported in 24 (45·3%, 95% CI 31·6-59·6) patients after a median follow-up of 27·1 weeks (IQR 19·3-40·1). Grade 3 or worse treatment-emergent adverse events that occurred in at least 10% of all participants were decreased neutrophil count (17 [22%] of 78) and anaemia (11 [14%]). Five patients (6%) had adjudicated interstitial lung disease or pneumonitis (two grade 2; one grade 3; two grade 5, the only treatment-related deaths).INTERPRETATIONTrastuzumab deruxtecan showed promising and durable activity in HER2-positive metastatic colorectal cancer refractory to standard treatment, with a safety profile consistent with that reported in previous trastuzumab deruxtecan trials. Interstitial lung disease and pneumonitis are important risks requiring careful monitoring and prompt intervention.FUNDINGDaiichi Sankyo.

项与安基奥仑赛相关的新闻（医药）

2023-12-18

·药明康德

GLP-1基因疗法减重27%，缓解率达100%的CAR-T疗法

近期，全球细胞和基因疗法（CGT）领域迎来系列进展。其中：CAR-T疗法不但在治疗多发性骨髓瘤的临床试验中达到100%的总缓解率，在治疗自身免疫疾病的临床试验中也让所有患者症状达到持续缓解。基于GLP-1受体激动剂的基因疗法在动物实验中将体重降低27%，带来无需长期接受治疗就可维持减重的希望。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。 ◇德国埃尔兰根大学医院（University Hospital Erlangen）研究人员在2023美国血液学会（ASH）年会上，公布了靶向CD19的CAR-T疗法治疗自身免疫性疾病患者的研究结果。该试验入组系统性红斑狼疮（SLE）、特发性炎性肌炎（IIM）和系统性硬化症（SSc）难治性患者，这些患者有活动性器官受累的迹象、经历多种免疫调节疗法失败且其疾病严重、有立即危及生命的可能。共有15例患者（8例SLE、4例SSc和3例IIM）接受了CAR-T细胞单次输注治疗。分析显示，所有8名SLE患者在3个月后均达到完全缓解，并自此维持SLE疾病活动指数（SLEDAI）为0。3/3的IIM患者在3个月后体内肌酸肌酶（CK）水平呈现显著改善和正常化，且这一情况仍在持续中。在4名SSc患者中，根据欧洲抗风湿病联盟（EULAR）标准，3名随访时间超过3个月患者的疾病活动性降低了4.3。所有15例患者均完全停止使用免疫抑制药物。◇Arcellx公布了旗下细胞疗法CART-ddBCMA（现名为anitocabtagene autoleucel，anito-cel）治疗多发性骨髓瘤的1期扩展研究的最新临床数据。试验结果显示，患者的客观缓解率（ORR）达到100%，38例可评估患者中有29例获得了完全缓解（CR）或严格的完全缓解（sCR）（>CR率，76%）。38例患者中有35例获得了非常好的部分缓解或更高水平的缓解（>VGPR率，92%）。 ◇Affimed在ASH年会上宣布了其先天细胞衔接蛋白（ICE）acimtamig，与同种异体自然杀伤（NK）细胞联用的最新临床试验数据。数据显示，在接受2期临床试验推荐剂量（RP2D）的复发/难治性霍奇金淋巴瘤（HL）患者中，客观缓解率达97%，完全缓解率高达78%。 Acimtamig是一款潜在“first-in-class"的先天细胞衔接蛋白，可独特地激活先天免疫系统破坏CD30阳性血液肿瘤。Acimtamig通过结合并激活自然杀伤细胞和巨噬细胞，利用先天免疫系统的力量，诱导CD30阳性肿瘤细胞的特异性和选择性杀伤。 ◇Poseida Therapeutics宣布了与罗氏（Roche）共同开发的同种异体CAR-T疗法P-BCMA-ALLO1的1期临床试验的早期疗效和安全性结果，该疗法用于治疗复发/难治性多发性骨髓瘤患者。分析显示，在接受充分淋巴细胞耗竭的强化预治疗患者中，接受CAR-T疗法P-BCMA-ALLO1治疗患者的ORR为82%（n=9/11），并具有深度临床缓解。而在既往未接受过BCMA靶向双特异性T细胞衔接抗体治疗患者的ORR则为100%。 ◇Editas Medicine公司在ASH年会上公布了17例患者接受CRISPR基因编辑疗法EDIT-301治疗的最新安全性和有效性数据，EDIT-301又称为renizgamglogene autogedtemcel（reni-cel），在RUBY试验中用于治疗严重镰刀型细胞贫血病（SCD）（n=11），在EdiTHAL试验中用于治疗输血依赖性β地中海贫血（TDT）（n=6）。试验结果显示，在RUBY试验中，所有患者均未发生血管闭塞事件（VOE）。在EdiTHAL试验中，所有患者的总血红蛋白均早期稳健升高，高于输血非依赖性阈值9 g/dL（n=6）。 ◇Obsidian Therapeutics公司公布了其工程化肿瘤浸润淋巴细胞（TIL）疗法OBX-115治疗晚期或转移性黑色素瘤的积极1期临床试验结果。中位随访时间为18周时，在对PD-1抑制剂耐药的6名患者中，ORR达到50%，完全缓解率为33%，疾病控制率为100%。 OBX-115是一款表达与细胞膜结合的IL-15的工程化TIL细胞疗法。它无需使用高剂量IL-2激活和扩增TIL细胞，减轻了疗法的潜在毒性。安全性方面，目前尚未观察到剂量限制毒性，治疗后出现的不良事件与淋巴细胞清除的特征一致。 ◇Carisma Therapeutics宣布，确定该公司与Moderna公司研发合作中的首款体内CAR巨噬细胞（CAR-M）候选疗法。这款疗法靶向具有显著未竟医疗需求的实体瘤上一个尚未公开的靶点。Carisma和Moderna的研发合作利用Moderna的脂质纳米颗粒（LNP）技术在体内制造CAR-M细胞。近日发表的临床前概念验证实验数据显示，Moderna公司使用LNP装载的优化mRNA能够在动物体内将髓系细胞重新定向，展现出抗肿瘤活性。在2022药明康德全球论坛上，Carisma公司首席医学官Debora Barton博士表示，在实体瘤的微环境中，T细胞耗竭，同时巨噬细胞大量存在，因此，对于实体瘤的治疗，探索CAR-M是有意义的。 ◇Fractyl Health日前在2023年世界胰岛素抵抗、糖尿病和心血管疾病大会上展示了其Rejuva基因治疗平台的临床前研究成果。在啮齿类动物肥胖模型中，该公司开发的基因疗法GLP-1 PGTx单剂输注后28天，将小鼠体重降低27%，而持续接受司美格鲁肽（10 nmol/kg/day）治疗的对照组在同一阶段体重降低21%。这一差异具有统计学显著性。这些发现表明，针对性的GLP-1 PGTx递送可以在肥胖动物中通过仅一次给药实现持久的减重。目前获批的GLP-1受体激动剂需要患者长期接受注射或者口服疗法，一旦停药，体重可能会逐渐反弹。GLP-1 PGTx是一款基于腺相关病毒（AAV）载体的基因疗法，目标是在胰岛素启动子的驱动下，在胰岛β细胞中增加GLP-1受体激动剂的表达，从而达到持久调节机体代谢功能的效果。该公司预计在2024年进行GLP-1 PGTx的毒理学和支持IND申请的临床前研究。 ◇CSL公司在ASH年会上公布了血友病B基因疗法Hemgenix的3年长期随访结果。在这项开放标签3期临床试验中，52名中重度血友病B患者完成了三年随访。Hemgenix在一年后产生的平均凝血因子IX水平为41.5 IU/dL，两年后因子IX平均水平为36.7 IU/dL，三年后平均水平为38.6 IU/dL。此外，94%接受治疗的患者无需持续接受预防性治疗。三年数据同时显示，在接受治疗后7-36个月期间，平均年化出血率（ABR）降低64%。 ◇Tome Biosciences公司宣布完成2.13亿美元的A轮和B轮融资。该公司致力于开发名为程序化基因组整合（PGI）的新技术，用于精确插入任何大小的DNA序列到目标基因组位置。 PGI技术结合了CRISPR/Cas9系统的位点特异性与不需切断DNA双链，就能够插入或写入DNA序列（包括完整基因）的酶。公司最先进的PGI技术称为整合酶介导的PGI（I-PGI），使用独特的整合酶。到目前为止，I-PGI已成功实现了在各种分裂和非分裂细胞类型中超过30 kb的遗传物质的精确位点特异性插入。它还提供了多重编辑的潜力，可进行复杂的细胞工程化改造，为细胞疗法开发的未来铺平了道路。 ◇Shinobi Therapeutics宣布完成5100万美元的A轮融资。该公司独特的方法基于共同科学创始人Tobias Deuse博士和Shin Kaneko博士的研究，首先编辑诱导多能干细胞（iPSCs）使其具有高度免疫逃逸能力，然后利用公司的专有技术将其分化为CD8αβ iPS-T细胞。这一技术平台有望让同种异体细胞疗法逃避免疫系统的排斥，降低细胞疗法的生产成本并提高疗法的可及性。除了上述动态，本周细胞和基因疗法领域还有一些其它进展，限于篇幅，本文不再一一介绍。希望该领域迎来更多的新进展和突破，惠及更多患者。参考资料： [1] Immusoft Administers the First Engineered B Cell in a Human Clinical Trial. Retrieved December 15, 2023, from https://www.businesswire.com/news/home/20231215132846/en [2] Precision BioSciences Announces Approval of First Clinical Trial Application of ARCUS Gene Insertion Program by Partner iECURE. Retrieved December 15, 2023, from https://www.businesswire.com/news/home/20231212100358/en [3] Ocugen, Inc. Announces First Patient Dosed in Phase 1/2 Clinical Trial Evaluating the Safety and Efficacy of OCU410—Modifier Gene Therapy—for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration. Retrieved December 15, 2023, from https://www.globenewswire.com/news-release/2023/12/13/2795480/0/en/Ocugen-Inc-Announces-First-Patient-Dosed-in-Phase-1-2-Clinical-Trial-Evaluating-the-Safety-and-Efficacy-of-OCU410-Modifier-Gene-Therapy-for-Geographic-Atrophy-Secondary-to-Dry-Age-.html [4] Tome Biosciences Launches with Over $200 Million in Funding to Advance Programmable Genomic Integration Platform. Retrieved December 15, 2023, from https://www.globenewswire.com/news-release/2023/12/12/2794660/0/en/Tome-Biosciences-Launches-with-Over-200-Million-in-Funding-to-Advance-Programmable-Genomic-Integration-Platform.html [5] CSL Behring's HEMGENIX® (etranacogene dezaparvovec-drlb) Demonstrates at Three Years Post-Treatment Long-Term Durability, Safety and Greater Bleed Protection Versus Prophylactic Treatment in People Living with Hemophilia B. Retrieved December 15, 2023, from https://www.prnewswire.com/news-releases/csl-behrings-hemgenix-etranacogene-dezaparvovec-drlb-demonstrates-at-three-years-post-treatment-long-term-durability-safety-and-greater-bleed-protection-versus-prophylactic-treatment-in-people-living-with-hemophilia-b-302011606.html [6] Fractyl Health Demonstrated That a Single Dose of a GLP-1-Based Pancreatic Gene Therapy Candidate (GLP-1 PGTx) Durably Maintained Weight Loss After Semaglutide Withdrawal in a Murine Model of Obesity at WCIRDC 2023 Annual Meeting. Retrieved December 15, 2023, from https://www.globenewswire.com/news-release/2023/12/11/2793883/0/en/Fractyl-Health-Demonstrated-That-a-Single-Dose-of-a-GLP-1-Based-Pancreatic-Gene-Therapy-Candidate-GLP-1-PGTx-Durably-Maintained-Weight-Loss-After-Semaglutide-Withdrawal-in-a-Murine.html [7] Poseida Therapeutics Presents Positive Early Results from its Phase 1 Trial of Allogeneic CAR-T P-BCMA-ALLO1 in Relapsed-Refractory Multiple Myeloma at the 65th American Society of Hematology (ASH) Annual Meeting. Retrieved December 15, 2023, from https://www.prnewswire.com/news-releases/poseida-therapeutics-presents-positive-early-results-from-its-phase-1-trial-of-allogeneic-car-t-p-bcma-allo1-in-relapsed-refractory-multiple-myeloma-at-the-65th-american-society-of-hematology-ash-annual-meeting-302010725.html [8] Long-term Follow-up Data From bluebird’s Gene Therapy Program in Sickle Cell Disease Support Durable, Potentially Curative Benefits Through Stable Production of Anti-Sickling Adult Hemoglobin and Resolution of Vaso-Occlusive Events. Retrieved December 15, 2023, from https://www.businesswire.com/news/home/20231209650405/en [9] First In Vivo CAR-M Lead Candidate Nominated Under Carisma-Moderna Collaboration. Retrieved December 15, 2023, from https://www.prnewswire.com/news-releases/first-in-vivo-car-m-lead-candidate-nominated-under-carisma-moderna-collaboration-302014783.html [10] Shinobi Therapeutics Launches with Completion of $51M Series A to Advance Hypoimmune iPS-T Cell Therapy Platform. Retrieved December 15, 2023, from https://www.prnewswire.com/news-releases/shinobi-therapeutics-launches-with-completion-of-51m-series-a-to-advance-hypoimmune-ips-t-cell-therapy-platform-302012188.html [11] Obsidian Therapeutics Announces Positive Interim Top-Line Clinical Data for OBX-115 Engineered TIL Cell Therapy in Advanced or Metastatic Melanoma Post-Anti-PD1 Therapy. Retrieved December 15, 2023, from https://www.businesswire.com/news/home/20231212231588/en [12] Gracell Biotechnologies Presents Updated Clinical Data from FasTCAR-T GC012F Demonstrating Deep and Durable Responses in Newly Diagnosed Multiple Myeloma at ASH 2023. Retrieved December 15, 2023, from https://www.globenewswire.com/news-release/2023/12/12/2794331/0/en/Gracell-Biotechnologies-Presents-Updated-Clinical-Data-from-FasTCAR-T-GC012F-Demonstrating-Deep-and-Durable-Responses-in-Newly-Diagnosed-Multiple-Myeloma-at-ASH-2023.html [13] Nexcella Announces 100% Overall Response Rate (n=10); 23.7 months Best Response Duration (ongoing) for CAR-T NXC-201 in Relapsed/Refractory AL Amyloidosis Patients at ASH 2023. Retrieved December 15, 2023, from https://www.globenewswire.com/news-release/2023/12/11/2793986/0/en/Nexcella-Announces-100-Overall-Response-Rate-n-10-23-7-months-Best-Response-Duration-ongoing-for-CAR-T-NXC-201-in-Relapsed-Refractory-AL-Amyloidosis-Patients-at-ASH-2023.html [14] Affimed Announces Updated Phase 1/2 Data from Acimtamig in Combination with Allogeneic NK in Hodgkin Lymphoma Patients Who Failed Prior Chemotherapy and Are Double-Refractory to Brentuximab Vedotin (BV) and Checkpoint Inhibitors (CPIs). Retrieved December 15, 2023, from https://www.globenewswire.com/news-release/2023/12/11/2793672/0/en/Affimed-Announces-Updated-Phase-1-2-Data-from-Acimtamig-in-Combination-with-Allogeneic-NK-in-Hodgkin-Lymphoma-Patients-Who-Failed-Prior-Chemotherapy-and-Are-Double-Refractory-to-Br.html 内容来源于网络，如有侵权，请联系删除。

细胞疗法免疫疗法基因疗法

2023-12-17

·药明康德

完全缓解率达100%的多款细胞疗法、可在1周内完成从制备到给药的CAR-T疗法... | 一周盘点

▎药明康德内容团队编辑本期看点1. 用于治疗复发性或难治性多发性骨髓瘤（MM）患者的CAR-T细胞疗法CART-ddBCMA的1期临床研究结果积极，所有受试者的总缓解率（ORR）达100%，估计的无进展生存期（PFS）为28个月。2. 在自体CAR-T细胞疗法MB-106针对复发或难治性惰性B细胞非霍奇金淋巴瘤（NHL）患者的1/2期临床试验中，滤泡性淋巴瘤（FL）和华氏巨球蛋白血症（WM）患者的ORR为100%，100%的FL患者（n=5）实现了完全缓解（CR）。3. 在自体CAR-T细胞疗法NXC-201用于治疗复发/难治性轻链（AL）淀粉样变性的1b/2a期临床试验中，患者的ORR为100%，CR率为70%。4. 在同种异体γ-δ T细胞疗法INB-100用于治疗接受异体造血干细胞移植的高危白血病患者的1期试验中，100%接受治疗的患者实现了持久的CR，包括高危和复发性急性髓系白血病（AML）患者以及既往多线治疗（包括CAR-T）失败的患者。5. 可在护理点制造的CAR-T细胞疗法GLPG5201和GLPG5101的1期临床试验结果积极，且从制备到回输到患者体内的中位时间仅需7天。药明康德内容团队整理CART-ddBCMA（anito-cel）：公布1期扩展试验数据 Arcellx公司公布了旗下细胞治疗产品CART-ddBCMA（现名为anitocabtagene autoleucel，anito-cel）的1期扩展研究的最新临床数据。Anito-cel是一款BCMA特异性CAR修饰T细胞疗法，旨在治疗复发性或难治性MM患者。此前，该疗法已被美国FDA授予快速通道资格、孤儿药资格和再生医学先进疗法认定。截至2023年10月15日的数据，中位随访时间为26.5个月，共有38名患者可进行疗效和安全性分析评估。根据国际骨髓瘤工作组（IMWG）标准，所有受试者的ORR达100%，并且带有预后不良因素的患者出现了深入而持久的缓解。中位PFS和OS尚未达到，Kaplan-Meier模型估计的PFS为28个月。▲根据Kaplan-Meier法估算的PFS率（图片来源：参考资料[6]）MB-106：公布1/2期临床试验数据Mustang Bio公司公布了其靶向CD20的自体CAR-T细胞疗法MB-106在针对复发或难治性惰性B细胞NHL患者的1/2期临床试验的数据。结果显示，MB-106在此类患者中的安全性良好，尽管未使用预防性药物，也没有发生1级以上的细胞因子释放综合征（CRS）和任何级别的免疫效应细胞相关神经毒性综合征（ICANS），且门诊给药是可行的。所有9名患者在接受MB-106治疗后均有缓解。FL和WM患者的ORR为100%。100%的FL患者（n=5）实现了CR。在WM患者中观察到1例非常好的部分缓解（PR）和2例PR。1名毛细胞白血病变异型患者此前一直严重依赖输血，治疗后病情持续稳定，不再需要输血。NXC-201（HBI0101）：公布1b/2a期临床试验的新数据Nexcella公司公布了其靶向BCMA的自体CAR-T细胞疗法NXC-201的1b/2a期临床试验的新数据。该研究旨在评估NXC-201用于治疗复发/难治性AL淀粉样变性的安全性和有效性。此前，NXC-201已被FDA授予孤儿药资格，用于治疗AL淀粉样变性和MM。截至2023年12月10日的数据，既往接受过大量治疗（中位治疗线数为6线）的患者的ORR为100%（10/10），CR率为70%（7/10）。患者的最佳缓解持续时间为23.7个月，并且缓解仍在持续。Nexcella公司计划在40名患者接受NXC-201治疗后，向FDA递交NXC-201的生物制品许可申请（BLA），用于治疗AL淀粉样变性。INB-100：公布1期临床试验的新数据 IN8bio公司公布了其同种异体γ-δ T细胞疗法INB-100在接受异体造血干细胞移植的高危白血病患者中的1期试验的新数据。结果显示，INB-100在这类患者中具有提供持久无复发治疗的潜力。截至2023年11月3日的数据，100%接受治疗的患者（n=10）实现了持久的CR，包括高危和复发性AML患者以及既往多线治疗（包括CAR-T）失败的患者。截至上次评估，所有受试者均存活，且没有复发，6名患者已经一年多没有复发。此外，在单次施用INB-100后365天，同种异体γ-δ T细胞依然保持着长期的体内扩增和持久性。该研究目前正在扩大患者招募，以进一步评估2期推荐剂量的疗效。GLPG5201、GLPG5101：公布1期临床试验数据Galapagos公司公布了其可在护理点制造的CAR-T细胞疗法GLPG5201和GLPG5101的1期临床试验结果。GLPG5201旨在治疗复发/难治性慢性淋巴细胞白血病（CLL）患者或小淋巴细胞淋巴瘤（SLL）患者，这些患者伴或不伴Richter转化（RT）。GLPG5101旨在治疗复发/难治性NHL患者。截至2023年9月6日的数据，接受GLPG5201治疗的14名疗效可评估的CLL患者的ORR为93%（13/14），CR率为57%（8/14）。9名RT患者的ORR为89%（8/9），CR率为67%（6/9）。接受较高剂量水平（DL2）的患者的ORR为100%（8/8），CR率为63%（6/8），6名RT患者的ORR为100%。在最初获得缓解后进展的3名患者中有2名已证实为CD19阴性疾病。截至2023年9月1日的数据，在研究的第一部分，接受GLPG5101治疗的14例可评估的NHL患者的ORR为86%（12/14），CR率为79%（11/14）。接受较高剂量水平（DL2）治疗的患者的ORR为86%（6/7），CR率也为86%。中位随访时间为8.6个月时，12名有缓解的患者中有8例（67%）保持持续缓解，持续时间长达15个月。在最初获得缓解后进展的4名患者中，有2例为CD19阳性复发，1例已证实为CD19阴性疾病。在研究的第二部分，7例可评估患者的ORR为86%（6/7），CR率为57%（4/7）。中位随访时间为3.2个月时，所有6名有缓解的患者均保持缓解。安全性方面，GLPG5201和GLPG5101均显示出令人鼓舞的安全性，大多数治疗伴发不良事件为1级或2级。此外，数据表明，这两种疗法从制备到回输到患者体内的中位时间仅需7天。P-BCMA-ALLO1：公布1期临床试验数据Poseida Therapeutics公司公布了其与罗氏（Roche）共同开发的在研BCMA靶向同种异体CAR-T疗法P-BCMA-ALLO1的1期临床试验的早期疗效和安全性积极结果，该疗法用于治疗复发/难治性MM患者。在该试验中，可评估患者（n=33）的随访时间至少4周，且皆接受了大量预治疗，中位既往治疗线数为7线。分析显示，在接受充分淋巴细胞耗竭的强化预治疗患者中，接受CAR-T疗法P-BCMA-ALLO1治疗患者的ORR为82%（9/11），并具有深度临床缓解。在既往未接受过BCMA靶向双特异性T细胞衔接抗体治疗的患者中，其ORR为100%。该疗法展现良好的安全性和可靠性特征，所有意向治疗（ITT）患者均未观察到移植物抗宿主病（GvHD）或剂量限制性毒性，且观察到的CRS和神经毒性发生率较低（均≤2级）。Revumenib：公布1/2期临床试验数据Syndax Pharmaceuticals公司公布了其选择性小分子menin抑制剂revumenib联合氟达拉滨及阿糖胞苷用于携带NPM1突变或KMT2A重排的复发/难治性AML患者的积极结果。截至2023年11月1日的数据，所有9名患者均获得了形态学缓解，ORR为100%，其中78%的患者获得了复合完全缓解（CRc），其中44%的患者获得CR或是完全缓解伴部分血液学恢复（CRh）。试验中67%（6/9）的患者达到最小残留病（MRD）阴性状态。5名患者在获得缓解后接受了造血干细胞移植。两名患者开始接受revumenib的移植后维持治疗，并已持续缓解超过11个月。该组合疗法在复发和难治性患者人群中的耐受性良好，没有观察到新的安全信号。Acimtamig（AFM13）：公布1/2期临床试验数据Affimed公司公布了其潜在“first-in-class"的先天细胞衔接蛋白（ICE）acimtamig与同种异体自然杀伤（NK）细胞联用治疗复发/难治性霍奇金淋巴瘤（HL）的1/2期临床试验的最新数据。此次公布的数据显示，在所有剂量水平中，治疗方案的ORR为93%，CR率为67%；在接受推荐的2期剂量（RP2D）治疗的32例HL患者中，治疗方案的ORR为97%，CR率为78%。在所有剂量水平，中位无事件生存期（EFS）为8.8个月，中位总生存期尚未达到。对于接受RP2D治疗的HL患者，中位EFS为9.8个月，84%的患者在12个月时存活。接受RP2D治疗的HL患者的中位缓解持续时间为8.8个月。此外，治疗方案显示了良好的安全性和耐受性特征，无任何级别的CRS、ICANS或GvHD。WU-CART-007：公布1/2期临床试验数据Wugen公司公布了其同种异体现货型CAR-T细胞疗法WU-CART-007用于治疗复发/难治性T细胞急性淋巴细胞白血病（T-ALL）或淋巴细胞淋巴瘤（LBL）的全球1/2期临床试验数据。WU-CART-007靶向CD7，并能够防止CAR-T细胞自相残杀，降低发生GvHD的风险。此次公布的结果显示，WU-CART-007表现出可控的安全性，未观察到GvHD。在剂量水平≥2的18例疗效可评估的患者中，CRc为67%，接受RP2D治疗的患者的CRc为73%。没有患者产生针对供体的新型抗HLA抗体，在接受检测的18名患者中，未检测到针对CAR的抗药抗体。大家都在看作为药明康德旗下专注于细胞和基因疗法的CTDMO，药明生基致力于加速和变革基因和细胞治疗及其他高端治疗的开发、测试、生产和商业化。药明生基能够助力全球客户将更多创新疗法早日推向市场，造福病患。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看）[1] Molecular Partners Presents Positive Initial Data from First Four Dosing Cohorts of Ongoing Phase 1/2a Trial of MP0533 for Patients with Relapsed/Refractory AML and AML/MDS at ASH Annual Meeting. Retrieved December 11, 2023, from https://www.globenewswire.com/news-release/2023/12/10/2793463/0/en/Molecular-Partners-Presents-Positive-Initial-Data-from-First-Four-Dosing-Cohorts-of-Ongoing-Phase-1-2a-Trial-of-MP0533-for-Patients-with-Relapsed-Refractory-AML-and-AML-MDS-at-ASH-.html[2] Century Therapeutics Presents Initial Data from CNTY-101 Phase 1 ELiPSE-1 Trial Supporting the Potential for a Multi-Dosing Strategy for CAR iNK Enabled by Allo-Evasion™ Edits. Retrieved December 11, 2023, from https://www.globenewswire.com/news-release/2023/12/09/2793435/0/en/Century-Therapeutics-Presents-Initial-Data-from-CNTY-101-Phase-1-ELiPSE-1-Trial-Supporting-the-Potential-for-a-Multi-Dosing-Strategy-for-CAR-iNK-Enabled-by-Allo-Evasion-Edits.html[3] Corvus Pharmaceuticals Presents New Interim Soquelitinib Data from its Phase 1/1b T Cell Lymphoma Trial. Retrieved December 11, 2023, from https://www.globenewswire.com/news-release/2023/12/09/2793436/0/en/Corvus-Pharmaceuticals-Presents-New-Interim-Soquelitinib-Data-from-its-Phase-1-1b-T-Cell-Lymphoma-Trial.html[4] Kymera Therapeutics Presents Interim Results from STAT3 Degrader Phase 1 Clinical Trial at American Society of Hematology Annual Meeting. Retrieved December 11, 2023, from https://www.globenewswire.com/news-release/2023/12/11/2793490/0/en/Kymera-Therapeutics-Presents-Interim-Results-from-STAT3-Degrader-Phase-1-Clinical-Trial-at-American-Society-of-Hematology-Annual-Meeting.html[5] Innate Pharma Shares Efficacy and Safety Phase 1 /2 Results of NK Cell Engager SAR443579 / IPH6101 Developed by Sanofi at ASH 2023. Retrieved December 11, 2023, from https://www.businesswire.com/news/home/20231210199351/en[6] Arcellx Announces Continued Robust Long-Term Responses from Its CART-ddBCMA (anito-cel) Phase 1 Expansion Trial in Patients with Relapsed or Refractory Multiple Myeloma at ASH. Retrieved December 11, 2023, from https://www.prnewswire.com/news-releases/arcellx-announces-continued-robust-long-term-responses-from-its-cart-ddbcma-anito-cel-phase-1-expansion-trial-in-patients-with-relapsed-or-refractory-multiple-myeloma-at-ash-302010481.html[7] TILT Biotherapeutics Announces Positive Clinical Data in Checkpoint Resistant Metastatic Melanoma Phase I Trial at ESMO Immuno-Oncology 2023. Retrieved December 11, 2023, from https://www.globenewswire.com/news-release/2023/12/08/2793021/0/en/TILT-Biotherapeutics-Announces-Positive-Clinical-Data-in-Checkpoint-Resistant-Metastatic-Melanoma-Phase-I-Trial-at-ESMO-Immuno-Oncology-2023.html[8] TScan Therapeutics Presents Initial Phase 1 Clinical Results on TSC-100 and TSC-101 at the 65th American Society of Hematology Annual Meeting and Exposition. Retrieved December 11, 2023, from https://ir.tscan.com/news-releases/news-release-details/tscan-therapeutics-presents-initial-phase-1-clinical-results-tsc[9] Poseida Therapeutics Presents Positive Early Results from its Phase 1 Trial of Allogeneic CAR-T P-BCMA-ALLO1 in Relapsed-Refractory Multiple Myeloma at the 65th American Society of Hematology (ASH) Annual Meeting. Retrieved December 11, 2023, from https://www.prnewswire.com/news-releases/poseida-therapeutics-presents-positive-early-results-from-its-phase-1-trial-of-allogeneic-car-t-p-bcma-allo1-in-relapsed-refractory-multiple-myeloma-at-the-65th-american-society-of-hematology-ash-annual-meeting-302010725.html[10] Affimed Announces Updated Phase 1/2 Data from Acimtamig in Combination with Allogeneic NK in Hodgkin Lymphoma Patients Who Failed Prior Chemotherapy and Are Double-Refractory to Brentuximab Vedotin (BV) and Checkpoint Inhibitors (CPIs). Retrieved December 11, 2023, from https://www.globenewswire.com/news-release/2023/12/11/2793672/0/en/Affimed-Announces-Updated-Phase-1-2-Data-from-Acimtamig-in-Combination-with-Allogeneic-NK-in-Hodgkin-Lymphoma-Patients-Who-Failed-Prior-Chemotherapy-and-Are-Double-Refractory-to-Br.html[11] Electra Therapeutics presents first clinical data from ongoing Phase 1b study of ELA026 for treatment of secondary hemophagocytic lymphohistiocytosis (sHLH). Retrieved December 11, 2023, from https://www.businesswire.com/news/home/20231211552878/en[12] Syndax Announces Positive Data for Revumenib in Patients with Acute Leukemias from the BEAT AML, SAVE AML and AUGMENT-102 Phase 1 Combination Trials. Retrieved December 11, 2023, from https://www.prnewswire.com/news-releases/syndax-announces-positive-data-for-revumenib-in-patients-with-acute-leukemias-from-the-beat-aml-save-aml-and-augment-102-phase-1-combination-trials-302011153.html[13] Mustang Bio Presents Updated Phase 1/2 Multicenter Clinical Data for MB-106 at the 2023 American Society of Hematology (ASH) Annual Meeting. Retrieved December 11, 2023, from https://www.globenewswire.com/news-release/2023/12/11/2793874/0/en/Mustang-Bio-Presents-Updated-Phase-1-2-Multicenter-Clinical-Data-for-MB-106-at-the-2023-American-Society-of-Hematology-ASH-Annual-Meeting.html[14] Initial Phase 1 Dose Escalation Data for ORIC-533 in Relapsed/Refractory Multiple Myeloma Demonstrates Clinical Activity and Strong Safety Profile Supporting Potential for Combination Development. Retrieved December 12, 2023, from https://www.globenewswire.com/news-release/2023/12/11/2794106/0/en/Initial-Phase-1-Dose-Escalation-Data-for-ORIC-533-in-Relapsed-Refractory-Multiple-Myeloma-Demonstrates-Clinical-Activity-and-Strong-Safety-Profile-Supporting-Potential-for-Combinat.html[15] Disc Presents Initial Positive Data from Ongoing Phase 1b/2 Trial of DISC-0974 in Patients with Myelofibrosis (MF) and Anemia at the 65th American Society of Hematology (ASH) Annual Meeting. Retrieved December 12, 2023, from https://www.globenewswire.com/news-release/2023/12/11/2794107/0/en/Disc-Presents-Initial-Positive-Data-from-Ongoing-Phase-1b-2-Trial-of-DISC-0974-in-Patients-with-Myelofibrosis-MF-and-Anemia-at-the-65th-American-Society-of-Hematology-ASH-Annual-Me.html[16] Wugen Presents Latest Data from First-In-Human Phase 1/2 Trial of WU-CART-007 in Patients with Difficult-to-Treat Blood Cancers at American Society of Hematology Annual Meeting. Retrieved December 12, 2023, from https://www.globenewswire.com/news-release/2023/12/11/2794234/0/en/Wugen-Presents-Latest-Data-from-First-In-Human-Phase-1-2-Trial-of-WU-CART-007-in-Patients-with-Difficult-to-Treat-Blood-Cancers-at-American-Society-of-Hematology-Annual-Meeting.html[17] MEI Pharma Reports Clinical Data on Oral CDK9 Inhibitor Voruciclib at ASH2023. Retrieved December 12, 2023, from https://www.businesswire.com/news/home/20231211089962/en[18] Biomea Fusion Presents Achievement of Minimal Residual Disease Negativity (MRD-neg) in First Complete Responder from Ongoing Phase I Study (COVALENT-101) of BMF-219 in Patients with Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) at the 2023 ASH. Retrieved December 12, 2023, from https://investors.biomeafusion.com/news-releases/news-release-details/biomea-fusion-presents-achievement-minimal-residual-disease[19] Sumitomo Pharma Presents Encouraging New Data on DSP-5336 Clinical Activity at the American Society of Hematology Annual Meeting. Retrieved December 12, 2023, from https://www.prnewswire.com/news-releases/sumitomo-pharma-presents-encouraging-new-data-on-dsp-5336-clinical-activity-at-the-american-society-of-hematology-annual-meeting-302011396.html[20] IN8bio Announces Positive Clinical Update Demonstrating Continued Durable Complete Remission in 100% of Evaluable Patients in Phase 1 Trial of INB-100 in Leukemia. Retrieved December 12, 2023, from https://investors.in8bio.com/news-releases/news-release-details/in8bio-announces-positive-clinical-update-demonstrating[21] Nurix Therapeutics Presents Positive Clinical Data from Its Novel Bruton’s Tyrosine Kinase (BTK) Degrader Programs, NX-5948 and NX-2127, at the 65th American Society of Hematology (ASH) Annual Meeting. Retrieved December 12, 2023, from https://ir.nurixtx.com/news-releases/news-release-details/nurix-therapeutics-presents-positive-clinical-data-its-novel[22] NovalGen presents NVG-111 clinical data in hematological malignancies in an oral session and preclinical data for next-generation AR T cell engager NVG-222 at the 65th American Society of Hematology Annual Meeting. Retrieved December 12, 2023, from https://www.globenewswire.com/news-release/2023/12/11/2793495/0/en/NovalGen-presents-NVG-111-clinical-data-in-hematological-malignancies-in-an-oral-session-and-preclinical-data-for-next-generation-AR-T-cell-engager-NVG-222-at-the-65th-American-Soc.html[23] Immix Biopharma Announces 100% Overall Response Rate (n=10); 23.7 months Best Response Duration (ongoing) for CAR-T NXC-201 in Relapsed/Refractory AL Amyloidosis Patients at ASH 2023. Retrieved December 12, 2023, from https://www.globenewswire.com/news-release/2023/12/11/2793969/0/en/Immix-Biopharma-Announces-100-Overall-Response-Rate-n-10-23-7-months-Best-Response-Duration-ongoing-for-CAR-T-NXC-201-in-Relapsed-Refractory-AL-Amyloidosis-Patients-at-ASH-2023.html[24] NKARTA PRESENTS NKX101 CLINICAL DATA AT THE 2023 AMERICAN SOCIETY OF HEMATOLOGY ANNUAL MEETING & EXPOSITION. Retrieved December 12, 2023, from https://ir.nkartatx.com/news-releases/news-release-details/nkarta-presents-nkx101-clinical-data-2023-american-society[25] Adicet Bio Highlights ADI-001 Expansion, Persistence and Pharmacodynamic Profile from Ongoing Phase 1 Study at the 65th American Society of Hematology (ASH) Annual Meeting. Retrieved December 12, 2023, from https://www.businesswire.com/news/home/20231210950496/en[26] ImmunoGen Presents Findings from Newly Diagnosed Acute Myeloid Leukemia Cohorts in Phase 1b/2 Study of Pivekimab Sunirine in Combination with Azacitidine and Venetoclax at ASH. Retrieved December 12, 2023, from https://www.businesswire.com/news/home/20231210537316/en[27] Aptose Tuspetinib Clinical Data Featured in Oral Presentation at the 2023 ASH Annual Meeting. Retrieved December 12, 2023, from https://www.aptose.com/investors/news-events/press-releases/detail/283/aptose-tuspetinib-clinical-data-featured-in-oral[28] Aptose Tuspetinib Clinical Data Featured in Oral Presentation at the 2023 ASH Annual Meeting. Retrieved December 12, 2023, from https://www.aptose.com/investors/news-events/press-releases/detail/283/aptose-tuspetinib-clinical-data-featured-in-oral[29] Galapagos presents new encouraging data at ASH 2023 from ongoing CD19 CAR-T studies with GLPG5201 and GLPG5101. Retrieved December 12, 2023, from https://pipelinereview.com/index.php/2023121084951/DNA-RNA-and-Cells/Galapagos-presents-new-encouraging-data-at-ASH-2023-from-ongoing-CD19-CAR-T-studies-with-GLPG5201-and-GLPG5101.html[30] SIRPant Immunotherapeutics Announces FDA Clearance of IND Application for SIRPant-MTM for the Treatment of Solid Tumors. Retrieved December 12, 2023, from https://www.globenewswire.com/news-release/2023/12/12/2794702/0/en/SIRPant-Immunotherapeutics-Announces-FDA-Clearance-of-IND-Application-for-SIRPant-MTM-for-the-Treatment-of-Solid-Tumors.html[31] GigaGen Receives FDA Clearance of IND to Begin Phase 1 Trial of Oncology Drug Candidate, GIGA-564, in Solid Tumors. Retrieved December 12, 2023, from https://www.globenewswire.com/news-release/2023/12/12/2794694/0/en/GigaGen-Receives-FDA-Clearance-of-IND-to-Begin-Phase-1-Trial-of-Oncology-Drug-Candidate-GIGA-564-in-Solid-Tumors.html[32] Immuneering Announces FDA Clearance of IND Application for Phase 1/2a Trial of IMM-6-415 to Treat Advanced Solid Tumors with RAF or RAS Mutations. Retrieved December 12, 2023, from https://www.globenewswire.com/news-release/2023/12/12/2794706/0/en/Immuneering-Announces-FDA-Clearance-of-IND-Application-for-Phase-1-2a-Trial-of-IMM-6-415-to-Treat-Advanced-Solid-Tumors-with-RAF-or-RAS-Mutations.html[33] C4 Therapeutics Announces Positive Data from CFT7455 Phase 1 Trial in Relapsed/Refractory Multiple Myeloma. Retrieved December 12, 2023, from https://www.globenewswire.com/news-release/2023/12/12/2795107/0/en/C4-Therapeutics-Announces-Positive-Data-from-CFT7455-Phase-1-Trial-in-Relapsed-Refractory-Multiple-Myeloma.html[34] Obsidian Therapeutics Announces Positive Interim Top-Line Clinical Data for OBX-115 Engineered TIL Cell Therapy in Advanced or Metastatic Melanoma Post-Anti-PD1 Therapy. Retrieved December 12, 2023, from https://www.businesswire.com/news/home/20231212231588/en[35] Black Diamond Therapeutics Announces Topline Results from Phase 1 Dose Escalation Trial of BDTX-1535 in Patients with Recurrent GBM. Retrieved December 14, 2023, from https://www.globenewswire.com/news-release/2023/12/13/2795402/0/en/Black-Diamond-Therapeutics-Announces-Topline-Results-from-Phase-1-Dose-Escalation-Trial-of-BDTX-1535-in-Patients-with-Recurrent-GBM.html[36] Black Diamond Therapeutics Announces Topline Results from Phase 1 Dose Escalation Trial of BDTX-1535 in Patients with Recurrent GBM. Retrieved December 14, 2023, from https://www.globenewswire.com/news-release/2023/12/13/2795402/0/en/Black-Diamond-Therapeutics-Announces-Topline-Results-from-Phase-1-Dose-Escalation-Trial-of-BDTX-1535-in-Patients-with-Recurrent-GBM.html[37] Shattuck Labs Announces Positive Initial Topline Data from Ongoing Phase 1 A/B Dose Expansion Clinical Trial of SL-172154 with Azacitidine in Frontline Higher-Risk Myelodysplastic Syndromes (HR-MDS) and TP53 mutant (TP53m) Acute Myeloid Leukemia (AML) Patients. Retrieved December 14, 2023, from https://www.globenewswire.com/news-release/2023/12/13/2795366/0/en/Shattuck-Labs-Announces-Positive-Initial-Topline-Data-from-Ongoing-Phase-1-A-B-Dose-Expansion-Clinical-Trial-of-SL-172154-with-Azacitidine-in-Frontline-Higher-Risk-Myelodysplastic-.html[38] BioAtla Hosting Virtual R&D Day to Highlight BA3071 CAB-CTLA-4 Phase 1 Data in Multiple Solid Tumor Types. Retrieved December 14, 2023, from https://www.globenewswire.com/news-release/2023/12/13/2795508/0/en/BioAtla-Hosting-Virtual-R-D-Day-to-Highlight-BA3071-CAB-CTLA-4-Phase-1-Data-in-Multiple-Solid-Tumor-Types.html[39] Avidity Biosciences Reports Positive Data Demonstrating AOC 1044 Delivers Unprecedented Concentrations of PMO in Muscle Following a Single Dose in Healthy Volunteers from Phase 1/2 EXPLORE44™ Trial for Duchenne Muscular Dystrophy. Retrieved December 14, 2023, from https://www.prnewswire.com/news-releases/avidity-biosciences-reports-positive-data-demonstrating-aoc-1044-delivers-unprecedented-concentrations-of-pmo-in-muscle-following-a-single-dose-in-healthy-volunteers-from-phase-12-explore44-trial-for-duchenne-muscular-dystrophy-302013456.html[40] IECURE SECURES CLEARANCE FROM AUSTRALIAN THERAPEUTIC GOODS ADMINISTRATION FOR ITS CLINICAL TRIAL APPLICATION FOR THE OTC-HOPE PHASE 1/2 STUDY OF ECUR-506. Retrieved December 14, 2023, from https://iecure.com/news/iecure-secures-clearance-from-australian-therapeutic-goods-administration-for-its-clinical-trial-approval-for-the-otc-hope-phase-1-2-study-of-ecur-506/[41] SonALAsense Presents Preliminary Data From Clinical Study in Patients With Deadly Pediatric Brain Tumor. Retrieved December 14, 2023, from https://www.biospace.com/article/releases/sonalasense-presents-preliminary-data-from-clinical-study-in-patients-with-deadly-pediatric-brain-tumor/[42] SonALAsense Presents Preliminary Data From Clinical Study in Patients With Deadly Pediatric Brain Tumor. Retrieved December 14, 2023, from https://www.businesswire.com/news/home/20231213836300/en[43] Agomab Starts Phase 1 Clinical Study for AGMB-447 in Healthy Subjects and Patients with Idiopathic Pulmonary Fibrosis. Retrieved December 14, 2023, from https://www.businesswire.com/news/home/20231214772174/en/免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

细胞疗法临床1期免疫疗法临床结果快速通道

2023-12-14

·药明康德

速递 | 总缓解率达100%！创新BCMA靶向性CAR-T疗法展现长期疗效

▎药明康德内容团队编辑近日，Arcellx公布了旗下细胞治疗产品CART-ddBCMA（现名为anitocabtagene autoleucel，anito-cel）1期扩展研究的最新临床数据。根据国际骨髓瘤工作组（IMWG）标准，所有受试者的总缓解率（ORR）达到100%，并且带有预后不良因素的患者出现了深入而持久的缓解。最新披露的数据持续显示出强劲的长期缓解，具体表现为中位缓解持续时间、无进展生存期（PFS）和总生存期在数据截止时尚未达到。这些数据截止到2023年10月15日，anito-cel输注后的中位随访时间为26.5个月。最新的研究结果在2023年12月11日举行的第65届美国血液学会（ASH）年会暨博览会上以口头报告的形式公布。截至2023年10月15日，根据治疗后中位时长为26.5个月的随访结果，共有38名患者可进行疗效和安全性分析评估。这些可评估患者包括第一剂量水平（1亿CAR+ T细胞，n=6）和第二剂量水平（3亿CAR+ T细胞，n=6）的剂量递增队列，以及剂量为1亿CAR+ T细胞的剂量扩展队列（n=26）。Anito-cel的1期临床试验中期结果（截止日期为2023年10月15日）显示，带有预后不良因素的患者出现了深入而持久的缓解。具体的试验结果如下：根据IMWG标准，所有患者的ORR达到100%。38例可评估患者中有29例获得了完全缓解（CR）或严格的完全缓解（sCR）（>CR率，76%）。38例患者中有35例获得了非常好的部分缓解或更高水平的缓解（>VGPR率，92%）。在可进行最小残留病（MRD）检测的患者（28人）中，25人（89%）为MRD阴性，灵敏度至少为10-5。在2023年10月15日数据截止时，中位缓解持续时间、PFS和总生存期尚未达到。虽然中位PFS尚未达到，但在数据截止时，Kaplan-Meier模型估计的PFS为28个月。根据Kaplan-Meier法估算，6、12、18和24个月的PFS率分别为92%、76%、64%和56%。在具有高危特征（基线时患有EMD、BMPC≥60%或B2M≥5.5 mg/L）的患者和具有高危细胞遗传学特征的患者中也观察到了持久的缓解。▲根据Kaplan-Meier法估算的PFS率（图片来源：参考资料[1]）Anito-cel是Arcellx公司开发的BCMA特异性CAR修饰T细胞疗法，这款疗法利用Arcellx公司的新型BCMA靶向型结合域治疗复发性或难治性多发性骨髓瘤患者。Anito-cel目前正在进行2期临床研究，它已被美国FDA授予快速通道资格、孤儿药资格和再生医学先进疗法认定。在2023年10月15日数据截止时，剂量为1.15亿（+/-10）个CAR+ T细胞的给药方案仍然耐受良好。使用anito-cel出现的不良反应，包括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），均在可控范围内。试验过程中无3级（或以上）CRS病例，仅有一例（3%）3级ICANS病例，此前未报告其他不良反应病例。试验中未观察到组织靶向毒性，未发现迟发性神经毒性事件或帕金森病症状。大家都在看作为药明康德旗下专注于细胞和基因疗法的CTDMO，药明生基致力于加速和变革基因和细胞治疗及其他高端治疗的开发、测试、生产和商业化。药明生基能够助力全球客户将更多创新疗法早日推向市场，造福病患。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料：[1] Arcellx Announces Continued Robust Long-Term Responses from Its CART-ddBCMA (anito-cel) Phase 1 Expansion Trial in Patients with Relapsed or Refractory Multiple Myeloma at ASH，Retrieved December 12, 2023 from https://ir.arcellx.com/news/news-details/2023/Arcellx-Announces-Continued-Robust-Long-Term-Responses-from-Its-CART-ddBCMA-anito-cel-Phase-1-Expansion-Trial-in-Patients-with-Relapsed-or-Refractory-Multiple-Myeloma-at-ASH/default.aspx[2] Arcellx, Kite aim to keep chipping away at Carvykti advantage with new multiple myeloma data: #ASH23，Retrieved December 12, 2023 from https://endpts.com/arcellx-kite-aim-to-keep-chipping-away-at-carvykti-advantage-with-new-multiple-myeloma-data-at-ash/免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

细胞疗法临床结果免疫疗法孤儿药快速通道

100 项与安基奥仑赛相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
滤泡性淋巴瘤	临床2期	韩国	Curocell, Inc.	2021-03-02
高级别B细胞淋巴瘤	临床2期	韩国	Curocell, Inc.	2021-03-02
大B细胞淋巴瘤	临床2期	韩国	Curocell, Inc.	2021-03-02
纵隔大b细胞淋巴瘤	临床2期	韩国	Curocell, Inc.	2021-03-02

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ESMO2023	临床2期	Carney复合症	-	Anbal-cel	(鹽淵膚構憲餘廠夢憲範) = 餘網襯觸齋遞鏇範製遞築鏇鑰顧構觸衊衊廠顧 (繭窪構遞鹽醖範範鏇網 )	-	2023-10-23
NCT04836507 (ASCO2022) 人工标引	临床1/2期	B细胞淋巴瘤	9	CRC-01	(鹽廠壓襯簾選築蓋糧艱) = 製製糧簾壓範鹹膚淵網廠範網遞醖網蓋繭艱製 (衊衊窪範簾襯鬱廠鹽糧 ) 更多	积极	2022-06-02
NCT04836507 (ESMO_ASIA2022) 人工标引	临床1/2期	弥漫性大B细胞淋巴瘤	11	CRC-01	(遞壓鑰醖鏇鬱淵廠壓鬱) = Anemia 22%, neutropenia 82%, thrombocytopenia 55% 鹹蓋鏇蓋蓋鏇醖觸艱膚 (憲積觸壓積觸觸憲醖網 )	积极	2022-12-04
ICML2023	N/A	-	-	Anbal-cel	(糧壓餘鑰衊鏇襯齋遞範) = Responder group (CR) reported higher incidence and severity of CRS compared to non-responder group (Non-CR), but it was insignificant. 膚選製獵齋願夢壓衊築 (餘遞願獵廠窪鏇窪憲鏇 ) 更多	-	2023-06-09