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更新于：2026-07-14
Bleximenib
布莱昔替尼
更新于：2026-07-14
概要
基本信息
药物类型 小分子化药 |
别名 Bleximenib、JNJ 6617、JNJ 75276617 + [4] |
作用方式 抑制剂 |
作用机制 MLL1抑制剂(lysine methyltransferase 2A inhibitors)、menin抑制剂(Menin蛋白抑制剂) |
在研适应症 |
非在研机构- |
权益机构- |
最高研发阶段临床3期 |
首次获批日期- |
最高研发阶段(中国)临床3期 |
特殊审评孤儿药 (美国)、孤儿药 (日本) |
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结构/序列
分子式C32H50FN7O3 |
InChIKeyPDUGAXSIWNMIBQ-HHHXNRCGSA-N |
CAS号2654081-35-1 |
关联
7
项与 布莱昔替尼 相关的临床试验NCT07223814
Bleximenib or Placebo in Combination With Standard Induction and Consolidation Therapy Followed by Maintenance for the Treatment of Patients With Newly Diagnosed KMT2A-rearranged or NPM1-mutant Acute Myeloid Leukemia Eligible for Intensive Chemotherapy: a Double-blind Phase 3 Study
The current standard of care treatment for adult patients with acute myeloid leukemia (AML) consists of chemotherapy and, if indicated, donor stem cell transplantation.
Bleximenib blocks the interaction between a protein called menin and another protein called KMT2A in the leukemia cells. When this interaction is disrupted in AML with mutations in the NPM1 or KMT2A gene, bleximenib can cause leukemia cells to die.
The main objective is to assess if treatment with bleximenib, when added to chemotherapy treatment will improve treatment outcome in adult participants with newly diagnosed AML who present with mutations in the NPM1 or KMT2A genes.
This is a randomized, double-blind, placebo-controlled, phase 3 clinical trial. All of the participants will receive standard chemotherapy treatment, combined with either bleximenib or a placebo. A placebo is a substance that looks like the study medicine but has no active ingredients (e.g., a sugar pill). In a double blind trial neither the participant nor the doctor know if placebo or active study drug is given.
After the end of the protocol treatment there will be an observational follow-up of 4 years from the time of inclusion of the last patient. The results of the different treatment groups will be compared.
875 previously untreated patients with AML with a specific change in the DNA of the leukemia cells (a KMT2A rearrangement or a NPM1 mutation) will be included. Participants must be 18 years or older and considered eligible for intensive chemotherapy.
Bleximenib blocks the interaction between a protein called menin and another protein called KMT2A in the leukemia cells. When this interaction is disrupted in AML with mutations in the NPM1 or KMT2A gene, bleximenib can cause leukemia cells to die.
The main objective is to assess if treatment with bleximenib, when added to chemotherapy treatment will improve treatment outcome in adult participants with newly diagnosed AML who present with mutations in the NPM1 or KMT2A genes.
This is a randomized, double-blind, placebo-controlled, phase 3 clinical trial. All of the participants will receive standard chemotherapy treatment, combined with either bleximenib or a placebo. A placebo is a substance that looks like the study medicine but has no active ingredients (e.g., a sugar pill). In a double blind trial neither the participant nor the doctor know if placebo or active study drug is given.
After the end of the protocol treatment there will be an observational follow-up of 4 years from the time of inclusion of the last patient. The results of the different treatment groups will be compared.
875 previously untreated patients with AML with a specific change in the DNA of the leukemia cells (a KMT2A rearrangement or a NPM1 mutation) will be included. Participants must be 18 years or older and considered eligible for intensive chemotherapy.
开始日期2026-03-01 |
NCT05521087
A Phase I/Ib Study of JNJ-75276617 in Combination With Conventional Chemotherapy for Pediatric and Young Adult Participants With Relapsed/Refractory Acute Leukemias Harboring KMT2A, NPM1, or Nucleoporin Gene Alterations
The purpose of this study is to determine the recommended Phase 2 dose(s) (RP2Ds) of JNJ-75276617 in combination with a conventional chemotherapy backbone in pediatric and young adult participants with relapsed/refractory acute leukemia harboring histone-lysine N-methyltransferase 2A1 ([KMT2A1], nucleophosmin 1 gene (NPM1), or nucleoporin alterations in Part 1 (Dose Escalation) and to further evaluate safety at the RP2D(s) of JNJ-75276617 in combination with chemotherapy in pediatric and young adult participants with relapsed/refractory acute leukemia harboring KMT2A1, NPM1, or nucleoporin alterations and safety at the RP2D(s) of JNJ-75276617 as monotherapy in a select low burden of disease cohort in Part 2 (Dose Expansion).
开始日期2025-12-26 |
NCT07295951
An Open-Label Study to Investigate the Absorption, Metabolism, And Excretion (AME) Of 14C-Bleximenib (JNJ-75276617) in Participants With Acute Leukemia
The purpose of this study is to assess how the body absorbs, breaks down (metabolism), and removes (excretes) radiolabeled bleximenib (a drug molecule that has been chemically bonded with a radioactive isotope which emits radiation making it easier to track in the body) in participants with acute leukemia (highly aggressive blood cancer typically characterized by large numbers of immature white blood cells in the bone marrow).
开始日期2025-11-18 |
100 项与 布莱昔替尼 相关的临床结果
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100 项与 布莱昔替尼 相关的转化医学
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100 项与 布莱昔替尼 相关的专利（医药）
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14
项与 布莱昔替尼 相关的文献（医药）2026-12-01·CURRENT TREATMENT OPTIONS IN ONCOLOGY
Menin Inhibitors: A New Era of Targeted Therapies in Acute Myeloid Leukemia
Review
作者: Reese, Mollie ; Hatfield, Jess ; Zeidner, Joshua F ; Peters, Daniel T
OPINION STATEMENT: Menin is involved in protein-protein interactions that are critical in the leukemogenesis of AML, particularly in patients with KMT2A rearrangements (KMT2Ar) and mutations in NPM1 (NPM1m). Revumenib is a first-in-class menin inhibitor approved by the U.S. Food and Drug Administration (FDA) in November 2024 for relapsed/refractory (R/R) acute leukemia with KMT2Ar. Revumenib was subsequently FDA-approved in October 2025 for R/R NPM1m AML, followed by approval of another oral menin inhibitor, ziftomenib, for R/R NPM1m AML in November 2025. These agents, along with other investigational menin inhibitors including bleximenib and enzomenib are being investigated in ongoing clinical trials as both single-agents and in combination with other chemotherapy agents in AML. Menin inhibitors have demonstrated clinical activity as single agents in KMT2Ar and NPM1m AML. However, the pharmacokinetics, safety profile, and selective activity varies among the available and investigational menin inhibitors. Despite these encouraging results, resistance mechanisms have been identified via mutations of the MEN1 gene and structural mutations in the menin binding pocket. As current and future clinical trials are completed, menin inhibitors are likely to play a large role in frontline and R/R treatment in KMT2Ar and NPM1m AML with likely expansion into populations of other genetic abnormalities.
2026-03-10·Blood Advances
Outcomes of relapsed or refractory acute myeloid leukemia after menin inhibition failure
Article
作者: Ali, Moiez ; Haque, Tamanna Z. ; Goldberg, Aaron D. ; Geyer, Mark B. ; Chin, Kuo-Kai ; Abaza, Yasmin ; Ball, Brian J. ; Thakur, Rahul K. ; Tallman, Martin S. ; Altman, Jessica K. ; Park, Jae H. ; Stein, Eytan M. ; Thompson, Meghan C. ; Aldoss, Ibrahim T. ; Wang, Eunice S. ; Cai, Sheng F. ; Derkach, Andriy
Abstract:
Menin inhibitors (MENINi) show promise for relapsed or refractory (R/R) acute myeloid leukemia (AML) with KMT2A rearrangements (KMT2Ar) and nucleophosmin 1 mutations (NPM1c). Outcomes after MENINi failure are poorly understood. To characterize the mutational landscape and subsequent outcomes, we conducted a multicenter retrospective study of adults from 4 US centers with R/R AML after MENINi failure (relapse after response or primary refractory). The 84 patients (63% KMT2Ar, n = 53; 23% NPM1c, n = 19) who received MENINi were heavily pretreated: 86% (n = 72) had previous intensive chemotherapy (IC) and 77% venetoclax (VEN; n = 67). After MENINi failure, 40% of patients (n = 34) received supportive care. For the treated patients (n = 50), common regimens included hypomethylating agent (HMA)/VEN (26%, n = 13), clinical trial (26%, n = 13), and gilteritinib-based therapy (18%, n = 9). The complete response (CR)/CR with incomplete hematologic recovery (CRi) rate for nontrial therapies was 19% (n = 7); overall response rate was 32% (n = 12). All CR/CRi occurred with HMA/VEN (n = 2, 15%), IC + VEN (n = 4, 67%), or MENINi switching (bleximenib to revumenib, n = 1 [50%]). No patient with FLT3-ITD mutation responded to gilteritinib (0/6 gilteritinib-naïve). Median overall survival from start of next therapy was 4.4 months and superior for patients who achieved CR/CRi, (15.4 vs 3.4 months, P = .048). Outcomes after MENINi failure are poor, but responses occur with VEN-based regimens or MENINi switching. FLT3-ITD, WT1, and MEN1 mutations are associated with resistance.
2025-12-05·Hematology-American Society of Hematology Education Program
The promise of menin inhibitors: from approval to triplet regimens
Review
作者: Thakur, Rahul K. ; Wang, Eunice S.
Abstract:
Pharmacologic targeting of the menin-KMT2A protein–protein interaction has emerged as a therapeutic breakthrough for acute leukemias harboring KMT2A rearrangements or NPM1 mutations. The first-in-class menin inhibitor (revumenib) achieved accelerated regulatory approval in November 2024 for monotherapy of relapsed/refractory KMT2A rearranged leukemia. Next-generation agents (ziftomenib, bleximenib, enzomenib, BMF-219) have displayed similar composite complete remission rates (20-35%) and overall response rates (45-65%) in heavily pretreated KMT2Ar and NPM1m acute myeloid leukemia (AML) with measurable residual disease (MRD) negativity and prolonged overall survival (5–7 months). While all menin inhibitors result in on-target blast differentiation with clinical sequelae (“differentiation syndrome”) in 10% to 20% of patients, not all menin inhibitors are the same, with differing safety, toxicity (heartrate corrected QT interval (QTc) prolongation, cytopenias), and pharmacokinetic profiles. Acquired mutations in the menin gene described in 39% of post-revumenib relapses have not been identified following other inhibitors (ziftomenib, bleximenib), prompting new questions about resistance mechanisms. These promising results swiftly led to the launch of multiple trials of menin inhibitors combined with intensive (cytarabine and anthracycline) and nonintensive (venetoclax and hypomethylating) chemotherapy backbones. To date, these triplets have yielded high response rates (ie, ≥80% in de novo, 50-70% in relapsed) with most patients achieving MRD negativity and prolonged one-year survival. Ongoing/pending phase 3 trials will clarify whether menin blockade should be incorporated into frontline and maintenance regimens for all patients with KMT2A rearranged or NPM1 mutant disease. In the current era, menin inhibition remains a key pillar of the success of precision medicine for AML therapy.
33
项与 布莱昔替尼 相关的新闻（医药）2026-07-08
·北三血液
尊敬的患者朋友们：
北京大学第三医院血液科整理了一批值得关注的血液系统疾病相关临床试验信息，大家可根据下面的信息进行推荐及咨询。我们会根据具体病情推荐适合的临床试验，安排筛选入组。
以下临床试验信息会持续更新，目前更新至2026年7月7日。请各位患者朋友们关注微信公众号“北三血液”了解最新资讯，如果您对下列临床试验有兴趣，可以通过血液科门诊进行咨询，由研究医生判断您是否符合加入研究的情况。
目前在北京大学第三医院的本部院区、海淀北部院区、首都机场院区均开设有血液科门诊，详情请见北京大学第三医院微信公众号。弥漫大B细胞淋巴瘤（初治）
1、项目名称：一项评估 DZD8586 联合用药在弥漫大 B 细胞淋巴瘤患者中的疗效及安全性的 Ib/II期、 多中心研究（队列1）
申办方/发起者：迪哲（江苏）医药股份有限公司
入选标准（简版）：
（1）病理确诊的弥漫性大 B 细胞淋巴瘤。A 部分纳入既往接受≤1 线治疗，且适合接受 R-CHOP 化疗的患者。B 部分纳入既往未接受过抗淋巴瘤治疗的患者，分期为II至IV期；
（2）预期寿命≥12 个月；
（3）年龄≥ 18 周岁；
（4）ECOG 评分 0-2 分；
（5）经 CT 或 MRI评估 ，有至少一个影像学可测量病灶且 PET/CT 扫描显示阳性病变并与 CT 或 MRI 确定的肿瘤部位一致。
（6）足够的骨髓造血功能储备（入组前访视 7 天内未输血、 且未使用任何刺激因子或促红细胞生成素）及脏器功能 ，具体如下：
a)中性粒细胞计数绝对值≥ 1.0×109/L，或ANC≥ 0.5×109/L,（累及骨髓时）；血小板计数 ≥ 75 ×109/L或 ≥ 50×109/L（累及骨髓时）；血红蛋白≥ 80 g/L；
b)APTT、PT、INR ≤ 1.5 × ULN;
c)总胆红素≤ 1.5 × ULN；若患有 Gilbert 综合征（非结合高胆红素血症）,或有影像证据显示肝脏受累,则总胆红素应 ≤ 3 × ULN；ALT 及 AST ≤ 2.5 × ULN; 影像证据显⽰肝脏受累时，ALT 及 AST ≤ 3 × ULN;
d)肌酐≤ 1.5 × ULN,或测量所得的肌酐清除率 ≥30 毫升 / 分（mL /min）；
e)LVEF ≥ 50%。
2、项目名称：一项在既往未经治疗的弥漫性大B 细胞淋巴瘤（DLBCL)受试者中比较ZilovertamabVedotin（MK-2140）联合R-CHP 与 R-CHOP 的随机开放性、多中心、III 期研究。
申办方/发起者：默沙东有限责任公司
入选标准（简版）：
（1）确诊为 DLBCL，包括但不限于：DLBCL，NOS 生发中心B细胞型或活化B细胞型；DLBCL 腿型；EBV+ DLBCL，NOS；和富含T细/组织细胞的 DLBCL。注：排除 HGBL 患者；
（2）筛选时PET 阳性，定义为 Lugano5分量表上 4-5 分；
（3）既往未接受过 DLBCL 治疗；
（4）年龄≥18 周岁；
（5）已提供既往未接受过放疗的肿瘤病灶的存档肿瘤组织样本或新采集的空心针穿刺或切除活检样本；
（6）ECOG 为 0 至 2 分；
（7）IPI 评分为 2 至 5 分；
（8）LVEF≥45%；
（9）HBsAg 阳性的受试者需HBV 病毒载量低于检测下限；
（10）HCV 感染史需HCV 病毒载量低于检测下限；
（11）具有充分的器官功能，包括：
a)中性粒细胞绝对计数≥1.0×109/L；血小板≥50×109/L；血红蛋白≥80g/L；
b)肌酐清除率≥30 mL/min ；
c)总胆红素≤1.5×ULN ，或对于总胆红素水平＞1.5×ULN 的受试者 ，直接胆红素≤ULN；
d)AST和ALT ≤2.5×ULN（肝脏累及受试者≤5×ULN）；
e)INR或PT、APTT ≤1.5×ULN；弥漫大B细胞淋巴瘤（复发难治）
1、项目名称：一项评估洛布替尼单药对比研究者选择的方案治疗复发或难治性non-GCB型弥漫大B细胞淋巴瘤疗效和安全性的开放标签、随机对照、多中心II期临床研究
申办者：广州麓鹏制药有限公司
入选标准（简版）：
（1）年龄≥18 周岁；
（2）病理确诊为 DLBCL（非特指）且经 Hans 法明确为非生发中心B细胞样（non-GCB）的患者；
（3）经过至少两线治疗（至少有一线治疗需含抗 CD20 抗体的治疗方案）后难治或复发受试者。
（4）至少具有一个可测量病灶（淋巴结病灶长径>1.5cm ，结外病灶长径>1.0 cm）。
（5）不计划进行自体造血干细胞移植（ASCT）治疗。
（6）ECOG体能状态评分≤2。
（7）经研究者判定，受试者预期生存≥12 周。
（8）受试者需要有足够的凝血功能和肝肾功能，当地实验室检查需要满足如下标准：
a)PT和APTT ≤1.5 倍正常值上限（ULN）；
b)肝脏：AST和ALT≤3×ULN；血清总胆红素≤1.5×ULN，若有明确的 Gilbert 综合症（非结合型高胆红素血症），总胆红素≤3.0×ULN；
c)胰腺：血清淀粉酶和血清脂肪酶≤1.5×ULN（结合 B 超、核磁共振或 CT 等影像学检查及临床 ，研究者不考虑胰腺炎者除外）；
d)肾脏：肌酐清除率（Ccr） ≥50 mL/min。
（9）有足够的骨髓造血功能，血液学检查必须满足以下标准：
a)中性粒细胞绝对计数≥1.0×109/L
b)血小板≥75×109/L
c)血红蛋白≥80 g/L
（10）既往治疗的毒性反应及外科手术并发症缓解至≤1 级
2、项目名称：BEBT-908 联合利妥昔单抗（ R）对比利妥昔单抗 - 吉西他滨 - 奥沙利铂（ R-GemOx）或利妥昔单抗 - 异环磷酰胺 - 卡铂 - 依托泊苷（ R-ICE）治疗复发 / 难治性弥漫性大 B细胞淋巴瘤（ r/r DLBCL）有效性和安全性的多中心、随机、对照、开放性 III 期临床研究
申办者：广州必贝特医药股份有限公司
入选标准（简版）：
（1）年龄≥18岁且≤75岁；
（2）病理学确诊为弥漫大 B 细胞淋巴瘤；
（3）采用 Lugano2014 标准经 PET-CT 和 CT 或 MRI 评估 ，具有可测量的病灶；
（4）确诊后须至少经过 1 种系统治疗后复发或难治性弥漫大 B 细胞淋巴瘤，并且至少有 1 种系统治疗中包含 CD20 抗体；
（5）ECOG评分为 0-2 分；
（6）预期寿命 >12 周；
（7）外周血：
a)中性粒细胞绝对计数≥1.0×109/L；
b)血红蛋白≥80g/L；
c)血小板计数≥100×109/L；
（8）肝肾功能：
a)血清总胆红素≤1.5×ULN（对于 Gilbert 综合征试验参与者,总胆红素<1.5×ULN；
b)ALT、AST≤2.5×ULN（有肝受累时≤5×ULN）；
3、项目名称：评估HP-001 㬵囊在复发难治的多发性骨髓瘤和非霍奇金淋巴瘤患者中的安全性、 耐受性、药代动力学和有效性的I期临床研究
申办方/ 发起者: 上海超阳药业有限公司
入组标准(简版):
（1）根据2022版WHO淋巴瘤病理分类，病理组织形态学和免疫组化确诊的DLBCL；
（2）既往1线治疗后进展且不适合移植或接受过≥2线系统性抗肿瘤治疗且既往方案包括CD20单抗；
（3）末次治疗后疾病复发或者末次治疗后未达缓解；
（4）具有可测量病灶：要求淋巴结病灶任一长径＞1.5 cm或结外病灶任一长径＞1.0cm；
（5）预计生存期至少 3 个月 ；
（6）年龄 18-75 岁；
（7）ECOG 体能状态评分为 0-2；
（8）重要器官的功能符合下列要求：
a)骨髓功能需满足：中性粒细胞计数≥1.0×109/L（检查前2周内未接受过输血或造血刺激因子治疗）；血红蛋白≥70g/L（骨髓瘤患者不强制要求）；血小板≥50×109/L（检查前1周内未输注血小板或使用血小板生成素等药物）
b)肝功能：丙氨酸氨基转移酶（ALT）≤3×ULN；天门冬氨酸氨基转移酶（AST）≤3×ULN；血清总胆红素（TBIL）≤1.5×ULN（除外 Gilbert 综合征，总胆红素≤3.0×ULN)；检查前2周内未使用保肝降酶药物；
c)肾功能：肌酐清除率（CrCl） ≥60ml/min（Cockcroft/Gault 公式，MM受试者肾脏受累者放宽至≥45ml/min）
d)凝血功能：国际标准化比率（INR） ≤1.5×ULN；活化部分凝血活酶时间（APTT） ≤1.5×ULN；
4、项目名称：项目名称: 一项评估 SCTB35 联合吉西他滨和奥沙利铂（GemOx）对比利妥昔单抗联合吉西他滨和奥沙利铂（ R-GemOx）在复发 / 难治性弥漫大 B 细胞淋巴瘤患者中的有效性和安全性的多中心、 随机对照、 开放的 III 期临床研究
申办者：神州细胞工程有限公司
入选标准（简版）：
（1）年龄18-80岁；
（2）组织病理学确诊符合2022年WHO诊断标准的弥漫大B细胞淋巴瘤[包括弥漫大B细胞淋巴瘤，非特指（DLBCL，NOS）、EBV阳性（EBV+）DLBCL、惰性淋巴瘤亚型转化的DLBCL、高级别B细胞淋巴瘤（HGBCL），NOS、双打击/三打击HGBCL、滤泡性淋巴瘤（FL） 3b级及原发纵隔大B细胞淋巴瘤（PMBCL）]，且免疫组化或流式免疫分型示CD20表达阳性；
（3）既往至少接受过一线含CD20单抗（如利妥昔单抗）的系统性方案充分治疗后复发、进展和/或难治性疾病（CAR-T或造血干细胞移植加桥接治疗为一个治疗线；局部疗法如放疗不视为一个治疗线）；
（4）基于CT或MRI至少有一个可测量病灶（淋巴结病灶定义为长轴＞1.5 cm；结外病灶定义为长轴＞1.0 cm）；
（5）ECOG体力状态评分0、1或2；
（6）具备充足的肝功能/造血功能/肾/心功能：
a)中性粒细胞绝对计数≥1.0×109/L；对于骨髓侵犯的试验参与者，ANC必须≥0.75 x 109/L；
b)血红蛋白≥90g/L（未经生长因子支持或血制品输注）；
c)血小板计数≥75×109/L（未经生长因子支持或血制品输注）。对于骨髓侵犯的试验参与者，PLT必须≥50 x 109/L；
c)血清总胆红素≤1.5×ULN（除非诊断为 Gilbert 综合征）；
d)ALT、AST≤3×ULN；
e)血肌酐≤1.5×ULN或肌酐清除率≥45 mL/min；原发中枢神经系统淋巴瘤（复发难治）
1、项目名称：克耐替尼治疗复发/难治性B 细胞淋巴瘤的探索性临床研究（队列1为原发中枢神经系统淋巴瘤）
申办者：江苏迈度药物研发有限公司
入选标准（简版）：
（1）年龄≥18 周岁；
（2）疾病诊断符合下列所有条件：
a)经病理确诊的原发中枢神经系统淋巴瘤；
b)复发或难治性 PCNSL ，针对 CNS 病灶，必须至少接受过1线治疗；
c)颅脑 MRI 或颅脑 CT 显⽰有疾病进展的实质性病灶
（3）ECOG评分为 0-2 分
（4）预计生存时间≥4 个月
（5）受试者有适宜的器官功能，主要器官功能符合以下标准：
a)血常规：中性粒细胞绝对值≥1.0×10^9/L，血小板≥75×10^9/L，血红蛋白≥80g/L；
b)血生化：总胆红素≤2×ULN（除非确诊吉尔伯特综合征），天冬氨酸氨基转移酶和丙氨酸氨基转移酶≤2.5×ULN；血清肌酐≤2×ULN 或肌酐清除率≥50ml/min；
c)凝血功能：国际标准化比率和活化部分凝血活酶时间≤1.5×ULN套细胞淋巴瘤（初治）
1、项目名称：一项比较奥布替尼联合苯达莫司汀和利妥昔单抗(奥布替尼+BR)对比苯达莫司汀联合利妥昔单抗(BR)治疗初治套细胞淋巴瘤受试者的随机、双盲、多中心、安慰剂对照III期研究
申办者：北京诺诚健华医药科技有限公司
入选标准（简版）：
（1）年龄≥65 岁，或≥60 岁且<65 岁因以下原因不适合或拒绝干细胞移植的受试者：
a)左室射血分数≤40%；
b)肌酐清除率 >30 mL/min ，但 <70 mL/min；
c)有其他不适合高强度诱导化疗的合并症；
d)预计干细胞动员和收集失败的高危受试者（如骨髓侵袭、糖尿病、血小板减少症等）；
e)受试者不愿意接受自体干细胞移植，顾虑移植后并发症（长期输血、免疫缺陷、出血和严重感染并发症等）和复发。
（2）既往未接受过针对MCL的治疗（包括以治疗为目的的手术、放疗、化疗、靶向治疗、免疫治疗等）。
（3）修订版 Ann Arbor II-IV 期；II 期受试者基于研究者判断需要系统治疗的才可入组。
（4）组织病理确诊的 MCL：
（5）有可用的存档组织样本，或愿意进行肿瘤活检以诊断 MCL（仅适用随机阶段）。
（6）至少有一个可测量病灶（淋巴结病灶长径 >1.5 cm ，结外病灶长径 >1.0 cm）。
（7）ECOG 评分 0-2 分。
（8）受试者必须有足够的器官功能，如下所⽰：
a)血常规（不依赖生长因子支持或1周内输血）：ANC≥1×109/L，血小板≥75×109/L，血红蛋白≥80 g/L；如伴骨髓侵犯，ANC≥0.75×109/L，血小板≥50×109/L，血红蛋白 ≥50g/L。
b)血生化：总胆红素≤1.5×ULN ，AST 或 ALT≤2×ULN；肌酐清除率≥50 mL/min；（Gilbert 综合征患者 ，总胆红素≤3×ULN）。
c)凝血功能：活化部分凝血酶时间（APTT）/ 凝血酶原时间（PT）/ 国际标准化比值（INR） ≤1.5×ULN。套细胞淋巴瘤（复发难治）
1、项目名称：评估IM19 CART 细胞治疗复发或难治 CD19 阳性套细胞淋巴瘤患者的安全性和有效性的 II 期临床研究。
申办者：艺妙神州医疗科技有限公司
入选标准（简版）：
（1）复发或难治的套细胞淋巴瘤患者，经细胞学或组织学确诊为 CD19 阳性，对患者既往治疗情况的规定如下：
a)需至少二线治疗后复发、未缓解或进展；
b)至少两个周期的含 CD20 单抗的治疗（如受试者对 CD20 单抗过敏 ，则 CD20 治疗周期可为两次以下）；
c)至少两个周期的含蒽环类药物或苯达莫司汀的治疗；
d)应接受过 BTK 抑制剂治疗；
（2）患者必须有可评估的疾病证据（根据 Lugano2014 标准确定的 CT 可测量病灶和PET-CT 可评估阳性病灶）；
（3）年龄≥18 岁；
（4）预计生存期 3 个月以上；
（5）ECOG评分 0-1 分；
（6）既往治疗导致的毒性已稳定或恢复到≤1 级（研究者判断无临床意义的情况除外）；
（7）血细胞分析（3 天内未进行过输血治疗）：血红蛋白≥80g/L；中性粒细胞>1×109/L；血小板≥50×109/L；淋巴细胞绝对数＞ 0.6×109/L；
（8）肝、肾、心、肺功能满足以下要求：肌酐清除率≥30ml/min 或血清肌酐≤ 1.5 ×正常值上限（upper limit of normal ,ULN）；ALT≤2.5×ULN ，总胆红素≤1.5×ULN（对于患有吉尔伯特氏综合征或淋巴瘤侵犯肝脏的受试者 ，ALT ≤5×ULN ，总胆红素≤3×ULN）；左心室射血分数≥ 50%；非吸氧状态下血氧饱和度＞ 95%；
（9）血管通路足以进行细胞采集，对于已有中心静脉导管的患者管路可用。
2、项目名称：一项评估ICP-248 在复发或难治性套细胞淋巴瘤受试者中疗效和安全性的单臂、 多中心、 开放标签的 II 期研究
申办者：北京诺诚建华医药科技有限公司
入选标准（简版）：
（1）年龄≥18 周岁
（2）组织病理确诊 MCL，表达 Cyclin D1 和/或 t(11;14)染色体易位的受试者；
（3）既往接受过 MCL 的系统性治疗：既往至少1种抗 CD20 治疗，且既往至少1种含 BTK 抑制剂的治疗。
（4）对于最近的治疗，充分治疗后未达到部分缓解或更佳疗效，或在治疗期间/治疗之后有疾病进展的记录。不能耐受最近一次治疗（在剂量调整和/或最佳支持治疗后仍因毒性不能耐受需终止治疗的患者也可入组。
（5）根据 Lugano2014 标准，至少有一个可测量病灶（淋巴结病灶的最长轴> 1.5 cm，或结外病灶的最长直径 > 1.0 cm）。
（6）ECOG 评分为 0-2；
（7）受试者必须具有以下充分的器官功能：
a)绝对中性粒细胞计数≥1.0×109/L，至少7天内不依赖生长因子支持。骨髓受累患者 ANC 须≥0.75×109/L；
b)血小板计数≥75×109/L，至少7天内不依赖生长因子支持或输血。骨髓受累患者PLT计数须≥50×109/L；
c)肌酐清除率≥ 40 mL/min；
d)天门冬氨酸氨基转移酶（AST）和丙氨酸氨基转移酶（ALT）≤3×正常值上限 （ULN）；
e)总胆红素≤2×ULN（有记录的 Gilbert 综合症除外）。
（8）预期生存时间>3 个月。
3、项目名称：克耐替尼治疗复发/难治性B 细胞淋巴瘤的探索性临床研究（队列3为复发难治MCL）
申办者：江苏迈度药物研发有限公司
入选标准（简版）：
（1）年龄≥18 周岁
（2）受试者疾病诊断符合下列所有条件：
a)组织病理确诊的套细胞淋巴瘤：包括细胞遗传学检测 t(11;14)（q13;q32） 阳性和/或免疫组织化学 Cyclin D1 高表达的套细胞淋巴瘤；
b)既往接受过>1 种，但≤3种不同化疗和/或靶向药物治疗失败；
c)有确认对最近一次治疗无效或缓解后发生疾病进展的病历记录；
d)CT/MRI 显⽰可测量病灶；可测量病灶定义为≥1个淋巴结的最长径>1.5cm，且2个垂直直径清晰可测；
（3）ECOG评分为 0-2 分；
（4）预计生存时间≥4 个月；
（5）受试者有适宜的器官功能，在最近7天内无生长因子支持治疗或输血的情况下，主要器官功能符合以下标准：
a)血常规：中性粒细胞绝对值≥0.75×10^9/L，血小板≥50×10^9/L（如伴随骨髓侵犯除外）；
b)血生化：总胆红素≤2×ULN（除非确诊吉尔伯特综合征），天冬氨酸氨基转移酶和丙氨酸氨基转移酶≤2.5×ULN；血清肌酐≤2×ULN 或肌酐清除率≥50ml/min（按照 Cockcroft-Gault 公式计算）；
c)凝血功能：国际标准化比率和活化部分凝血活酶时间≤1.5×ULN
4、项目名称：一项评估SCTB35 在复发 / 难治性 CD20 阳性 B 细胞非霍奇金淋巴瘤患者中的安全性、耐受性、 药代动力学、初步有效性的 Ia/Ib 期临床剂量递增及剂量扩展研究
申办者：神州细胞工程有限公司
入选标准（简版）：
（1）年龄18-80岁；
（2）依据组织病理学确诊为 CD20+ 成熟 B 细胞 NHL 患者；
a)Ia 期剂量递增研究：i. 弥漫大 B 细胞淋巴瘤（原发或转化）；ii. 高级别 B 细胞淋巴瘤；iii. 原发纵隔大 B 细胞淋巴瘤；iv. 滤泡性淋巴瘤；v. 套细胞淋巴瘤；vi. 慢性淋巴细胞白血病 / 小淋巴细胞淋巴瘤；vii. 边缘区淋巴瘤（结内 ，结外或粘膜型）
b)Ib期剂量递增研究：i. FL：经组织学确诊为 1、2 或 3a 级 FL ，且无临床或病理转化；ii. LBCL：包括组织学确诊的 DLBCL ，NOS、 EBV+ DLBCL、惰性淋巴瘤亚型转化而来的DLBCL、 HGBCL ，NOS、双打击 / 三打击 HGBCL、 FL3b 级及 PMBCL；iii. CLL/SLL：经病理确诊为慢性淋巴细胞白血病 / 小淋巴细胞淋巴瘤（CLL/SLL）且具有治疗指征；iv. MCL：经病理确诊为套细胞淋巴瘤（ MCL）有 Cyclin D1 过表达或t（11;14） 的证据。
（3）既往接受过含CD20 单抗（如利妥昔单抗）方案充分治疗后复发、进展和/ 或难治性疾病；
（4）基于 CT 或MRI至少有一个可测量病灶（淋巴结病灶定义为长轴＞ 1.5 cm；结外病灶定义为长轴＞ 1.0 cm）。 既往接受过放疗的病灶必须证实放疗后存在病灶进展或残留病变 ，才可视为可测量病灶（ Ia 期允许至少存在一个可评估病灶）。
（5）ECOG 体力状态评分 0、 1 或 2；
（6）具备充足的肝功能 / 造血功能 / 肾 / 心功能：
a. 首次给予试验药物前 7 天内中性粒细胞绝对值（ANC） ≥1.0 x 109/L（未经生长因子支持）。对于骨髓侵犯的受试者 ，ANC 必须≥0.75 x 109/L。
b. 首次给予试验药物前 7 天内 PLT 计数≥75 x109/L（未经生长因子支持或输注）。对于骨髓侵犯的受试者 ，PLT 必须≥50 x 109/L。
c. 首次给予试验药物前 7 天内血红蛋白 （ HGB） ≥90 g/L（未经生长因子支持或输注）。
d. 血肌酐≤1.5 ×ULN 或肌酐清除率≥45 mL/min ，
e. 充足的肝功能储备：i. 天门冬氨酸氨基转移酶（AST）/ 血清 AST≤3 倍正常值上限(ULN）；ii. 丙氨酸氨基转移酶（ALT）/ 血清 ALT≤3xULN；iii. 总胆红素≤1.5xULN（除非诊断为Gilbert’ s 综合征）；
f. 左室射血分数（ LVEF） ≥50%。
（7）预期生存期 3 个月以上。慢性淋巴细胞白血病/小淋巴细胞淋巴瘤（初治）
1、项目名称：一项在未经治疗的慢性淋巴细胞白血病/ 小淋巴细胞淋巴瘤受试者中比较Nemtabrutinib 与研究者选择的伊布替尼或阿可替尼的 III 期随机研究（ BELLWAVE-011）
申办者：默沙东有限责任公司
入选标准（简版）：
（1）确诊为 CLL/SLL 且有明确记录的活动性病变，需要启动治疗。应符合以下至少一个标准：
a)进行性骨髓衰竭证据，新发贫血和/或血小板减少症，或者贫血和/或血小板减少症恶化。
b)巨脾（如左肋缘下≥6 cm）或进行性或症状性脾肿大。
c)巨块型淋巴结肿大（如最长直径≥10 cm）或进行性或症状性淋巴结肿大。
d)在 2 个月内进行性淋巴细胞增多≥50% ，或淋巴细胞倍增时间＜ 6 个月。
e)自身免疫性并发症，包括贫血或血小板减少症，研究者认为对皮质类固醇反应不佳。
f)症状性或功能性结外受累（如皮肤、肾脏、肺、脊髓）。
g)有以下任一项定义的疾病相关症状：过去 6 个月内非故意体重减轻≥10%；严重疲劳（无法工作或无法进行常规活动）；发热（＞38.0℃) ≥2 周，无感染迹象；盗汗≥1个月，无感染证据。
（2）具有以下至少 1 种疾病负荷标志物：
a)恶性淋巴结是由 CLL/SLL 引起 ，轴向平面二维清晰、 可重复测量 ，且通过 CT/MRI扫描最长直径＞ 1.5 cm。
b)淋巴细胞绝对计数＞4×109/L
c)血小板计数＜100×109/L
d)血红蛋白＜110g/L
（3）提供外周血、骨髓穿刺和/或淋巴结样本，用于确定del(17p)状态和TP53突变状态 ，两者均由中心实验室检测确定。 需要在随机分组前采样。
a)只有当存在 TP53 突变时 ，Del(17p)状态不确定的受试者才有入组资格。
b)只有在检出 del(17p)时 ，TP53 突变状态不确定的受试者才有入组资格。
（4）随机分组前 7 天内 ECOG PS 为0 至 2 分。
（5）能够吞咽并保留口服药物。
（6）HBsAg 阳性的受试者需HBV-DNA 病毒载量低于检测下限。
（7）有 HCV 感染史的受试者需HCV RNA 病毒载量检测不到。
（8）具有定义的充分的器官功能
a)血常规：ANC≥0.75×109/L（骨髓累及≥0.5×109/L）、血小板≥50×109/L（骨髓累及≥30×109/L）、血红蛋白≥80 g/L
b)肾功：CrCl≥30 mL/min
c)肝功：总胆红素≤1.5×ULN ，或对于总胆红素水平＞1.5×ULN 的受试者 ，直接胆红素≤ULN（有 CLL/SLL 肝脏累及和 /或 Gilbert 综合征的受试者中 ，总胆红素≤3×ULN），AST（SGOT）和 ALT（SGPT）≤2.5×ULN（有CLL/SLL肝脏累及的受试者，≤5×ULN）
d)凝血：INR 或 PT、aPTT≤1.5×ULN
（9）年龄≥18 周岁。慢性淋巴细胞白血病/小淋巴细胞淋巴瘤（复发难治）
1、项目名称：克耐替尼治疗复发/难治性B细胞淋巴瘤的探索性临床研究（队列2为复发难治CLL/SLL）
申办者：江苏迈度药物研发有限公司
入选标准（简版）：
（1）性别不限，年龄≥18 周岁
（2）受试者疾病诊断符合下列所有条件：
a)根据 IWCLL2008 标准，确诊的 CLL/SLL；
b)既往接受过至少一线的系统治疗的难治或复发性 CLL/SLL。一线治疗定义为至少完成≥2 个周期的依据目前指南的标准方案或临床试验的研究方案；
c)至少符合一条 CLL/SLL 需要治疗的指征；
d)有确认对最近一次治疗无效或缓解后发生疾病进展的病历记录；
e)CT/MRI 显⽰可测量病灶；可测量病灶定义为至少有一个淋巴结最大轴线超过 1.5cm，并且有2个可测量的垂直维度；
f)允许纳入累及中枢神经系统但病情稳定者
（3）ECOG状态评分为 0-2 分
（4）预计生存时间≥4 个月
（5）受试者有适宜的器官功能，在最近 7 天内无生长因子支持治疗或输血的情况下，主要器官功能符合以下标准：
a)血常规：中性粒细胞绝对值≥0.75×10^9/L，血小板≥50×10^9/L（如果 CLL 患者有骨髓受累 ，则可以血小板≥30×10^9/L，输血依赖性血小板减少排除）；
b)血生化：总胆红素（TBIL）≤2×ULN（除非确诊吉尔伯特综合征），天冬氨酸氨基转移酶（AST）和丙氨酸氨基转移酶（ALT）≤2.5×ULN；血清肌酐≤2×ULN 或肌酐清除率（Ccr）≥50ml/min
c)凝血功能：国际标准化比率（INR）和活化部分凝血活酶时间（APTT）≤1.5×ULN
2、项目名称：一项III期、开放标签、随机、多中心研究以评估DZD8586对比研究者选择治疗在复发难治性慢性淋巴细胞白血病/小淋巴细胞淋巴瘤患者中的抗肿瘤疗效
申办者：迪哲（江苏）医药股份有限公司
入选标准（简版）：
（1）年龄≥18 周岁；
（2）ECOG评分 0-2 分；
（3）确诊为CLL/SLL的受试者，且病情符合以下条件：在既往任何一线 BTK 抑制剂治疗期间或之后出现复发、难治或不耐受。
（4）预期寿命≥ 3 个月。
（5）受试者需满足基于iwCLL 2018 标准规定的 r/r CLL/SLL 需要临床治疗的条件，研究者判断需要临床治疗：
a)符合启动后续治疗的标准，包括前线治疗后疾病负荷持续存在、前线治疗的治疗指征尚未缓解。
b)进行性骨髓衰竭的证据：表现为血红蛋白和（或）血小板进行性减少。
c)巨脾（如左肋缘下≥6 cm）、进行性或有症状的脾肿大。
d)巨块型淋巴结肿大（如最长直径≥10 cm）或有症状的淋巴结肿大。
e)进行性淋巴细胞增多：2个月内淋巴细胞增多≥50％，或淋巴细胞倍增时间（LDT）< 6 个月，排除感染和药物等原因。
f)CLL/SLL 导致的有症状的脏器功能异常（如：皮肤、 肾、肺、脊柱等）。
g)自身免疫性溶血性贫血（AIHA）和（或）免疫性血小板减少症（ ITP）对皮质类固醇反应不佳。
h)至少存在下列一种疾病相关症状：既往 6 个月内无明显原因的体重下降≥10％ ；或严重疲乏（如 ECOG 体能状态评分≥ 2 分；或不能进行常规活动）；或无感染证据，体温≥ 38.0°C 持续≥2 周；或无感染证据，夜间盗汗≥1 个月。
（6）SLL 受试者至少存在一个可测量的病灶（淋巴结最长直径> 1.5 cm 或结外病灶最长直径> 1.0 cm）。
（7）足够的骨髓造血功能储备（入组前访视 7 天内未输血、 且未使用任何刺激因子或促红细胞生成素）及脏器功能 ，具体如下：
a)中性粒细胞计数绝对值（ANC）≥ 0.75×109/L。
b)血小板计数≥50×109/L。若计划接受 BR 方案作为组 2 的治疗，血小板计数需≥75×109/L。
c)活化的部分凝血活酶时间（APTT）、凝血酶原时间（PT）和国际标准化比值（INR） ≤1.5 × 正常值上限（ ULN）
d)总胆红素≤ 1.5 × ULN；若患有 Gilbert 综合征（非结合高胆红素血症），或有影像证据显⽰肝脏受累，则总胆红素应≤3 × ULN。
e)丙氨酸氨基转移酶（ALT）及天冬氨酸氨基转移酶（AST） ≤ 2.5 ×ULN。 影像证据显⽰肝脏受累时，ALT 及 AST ≤ 3 × ULN。
f)肌酐清除率≥ 30 mL/min（Cockcroft-Gault 方法）。
g)超声心动图（ECHO）评估（左心室射血分数） LVEF ≥ 50%。
3、项目名称：一项评估ICP-248 单药或联合奥布替尼治疗在成熟 B 细胞恶性肿瘤患者中的安全性、耐药性、 药代动力学、 药效动力学和初步疗效的 I 期研究（复发难治CLL/SLL队列）
申办方/发起者：北京诺诚健华医药科技有限公司
主要入组标准（简版）:
（1）年龄≥ 18 岁且≤80 岁。
（2）经组织病理学和/或流式细胞学确诊的 CLL/SLL；
（3）复发性疾病：经充分系统性治疗达到完全缓解（CR）或部分缓解（PR）后 ，出现疾病进展（PD）；或难治性疾病：对充分系统性治疗无反应（系统性治疗过程中疾病出现进展 ，或经过充分周期的系统性治疗后尚未取得 PR 或 CR 的最佳缓解）；充分系统性治疗定义为由研究者判定的足够疗程足够剂量且包括抗 CD20 单抗（CLL/SLL 患者除外） 的系统性治疗 ，除非治疗中疾病进展或不可耐受毒性。
（4）剂量扩展阶段：
· 队列 A 入组此前未采用 BTKi 治疗的 R/R CLL/SLL 受试者；
· 队列 B 入组此前采用过 BTKi 治疗的 R/R CLL/SLL 受试者；
· 队列 E 入组 R/R CLL/SLL 受试者 ，不论 BTKi 暴露史；
· 队列 I 入组经 BTKi 治疗的 R/R CLL/SLL 受试者；
· 队列 J 入组 R/R CLL/SLL 受试者 ，无论 BTKi 暴露史 ，同时筛选期 TLS 风险评估须为低危或中危；
（5）对于 B-NHL 受试者：根据 Lugano2014 评估标准 ，患者必须有可测量病灶，定义为至少1个结节病灶的最长径> 1.5 cm，或为至少 1 个结外病灶的最长径> 1 cm，且至少有 1 对可以准确测量的垂直径。
（6）ECOG≤ 2 分，并且预计生存时间≥6 个月的患者。
（7）血液学功能充分，定义为：
a)绝对中性粒细胞计数（ANC）≥ 1.0×109/L ，检测前7天内不得使用生长因子支持；
b)PLT 计数 ≥ 50×109/L ，检测前7天内不得使用生长因子支持或进行输血；
c)血红蛋白 > 80 g/L ，检测前7天内不得进行输血。
（8）凝血功能基本正常的患者：活化部分凝血活酶时间（APTT） ≤ 1.5×正常值上限（ULN）；国际标准化比值（INR） ≤ 1.5 × ULN。
（9）肝、肾、肺、心脏功能基本正常的患者：
a)肝功能：血清总胆红素≤ 1.5 × ULN （除非有证据表明患者患有 Gilbert 综合征 ，则 ≤ 3.0 × ULN）；谷草转氨酶（AST）和谷丙转氨酶（ALT） ≤ 3× ULN（如果有肿瘤继发性改变影响到肝脏 ，则 ≤ 5.0 × ULN）；
b)肾功能：肌酐清除率≥ 40 mL/min；
c)心脏功能：左室射血分数（ LVEF） ≥ 50% ，无显著临床意义的心包积液；
d)肺功能：无重度阻塞性肺病和急慢性呼吸衰竭。
（10）对于绝对淋巴细胞计数（ALC） ≥50 × 109/L 且合并任何淋巴结（ LN）长径≥5 cm 的CLL/SLL 患者或任何淋巴结长径≥10 cm 的 CLL/SLL 及 B-NHL 患者需与申办方 MM 共同权衡风险 - 获益后方可入组研究。
（11）经研究者判断，CLL/SLL 受试者需有治疗的指征。滤泡细胞淋巴瘤（复发难治）
1、项目名称：一项评估SCTB35 联合来那度胺对比利妥昔单抗联合来那度胺（ R2）在复发 / 难治性滤泡性淋巴瘤患者中的有效性和安全性的多中心、 随机、 开放的 III 期临床研究
申办者：神州细胞工程有限公司
入选标准（简版）：
（1）年龄18-80岁；
（2）依据组织病理学确诊符合2022年WHO诊断标准的1，2 或3a级FL，无临床或病理转化证据，且免疫组化或流式免疫分型示CD20表达阳性；
（3）既往至少接受过一线含CD20单抗（如利妥昔单抗）的系统性方案充分治疗后复发、进展和/或难治性疾病；
（4）研究者根据肿瘤大小和/ 或GELF标准评估需要进行全身治疗；
（5）基于CT 或MRI至少有一个可测量病灶（淋巴结病灶定义为长轴＞1.5 cm；结外病灶定义为长轴＞1.0 cm），且正电子发射断层造影术（PET）显⽰为FDG 亲和性阳性病灶。既往接受过放疗的病灶必须证实放疗后存在病灶进展或残留病变，才可视为可测量病灶；
（6）ECOG评分0~2；
（7）具备充足的肝功能/造血功能/ 肾/心功能：
（8）预期生存期6个月以上。边缘区淋巴瘤（复发难治）
1、项目名称：一项评估ICP-248 单药或联合奥布替尼治疗在成熟 B 细胞恶性肿瘤患者中的安全性、耐药性、 药代动力学、 药效动力学和初步疗效的 I 期研究
申办方/发起者：北京诺诚健华医药科技有限公司
主要入组标准（简版）:
（1）年龄≥ 18 岁且≤80 岁。
（2）经病理学确诊的 MZL。
（3）复发性疾病：经充分系统性治疗达到完全缓解（CR）或部分缓解（ PR）后 ，出现疾病进展（ PD）；或难治性疾病：对充分系统性治疗无反应（系统性治疗过程中疾病出现进展 ，或经过充分周期的系统性治疗后尚未取得 PR 或 CR 的最佳缓解）；充分系统性治疗定义为由研究者判定的足够疗程足够剂量且包括抗 CD20 单抗（CLL/SLL 患者除外） 的系统性治疗 ，除非治疗中疾病进展或不可耐受毒性。
（4）剂量扩展阶段：
· 队列 H 入组非 BTKi 难治的 R/R MZL 受试者；
（5）对于 B-NHL 受试者：根据 Lugano2014 评估标准 ，患者必须有可测量病灶，定义为至少1个结节病灶的最长径> 1.5 cm，或为至少 1 个结外病灶的最长径> 1 cm，且至少有 1 对可以准确测量的垂直径。
（6）ECOG≤ 2 分，并且预计生存时间≥6 个月的患者。
（7）血液学功能充分，定义为：
a)绝对中性粒细胞计数（ANC）≥1.0×109/L ，检测前7天内不得使用生长因子支持；
b)PLT 计数≥50×109/L ，检测前 7 天内不得使用生长因子支持或进行输血；
c)血红蛋白 > 80 g/L ，检测前 7 天内不得进行输血。
（8）凝血功能基本正常的患者：活化部分凝血活酶时间（APTT） ≤ 1.5×正常值上限（ULN）；国际标准化比值（INR） ≤ 1.5 × ULN。
（9）肝、肾、肺、心脏功能基本正常的患者：
a)肝功能：血清总胆红素≤ 1.5 × ULN （除非有证据表明患者患有 Gilbert 综合征 ，则 ≤ 3.0 × ULN）；谷草转氨酶（AST）和谷丙转氨酶（ALT） ≤ 3× ULN（如果有肿瘤继发性改变影响到肝脏 ，则 ≤ 5.0 × ULN）；
b)肾功能：肌酐清除率≥ 40 mL/min；
c)心脏功能：左室射血分数（ LVEF） ≥ 50% ，无显著临床意义的心包积液；
d)肺功能：无重度阻塞性肺病和急慢性呼吸衰竭外周T/NKT细胞淋巴瘤（复发难治）
1、项目名称：一项III 期 ，开放标签 ，随机 ，多中心研究以评估戈利昔替尼对比研究者选择治疗在复发难治性外周 T 细胞淋巴瘤成人患者中的抗肿瘤疗效
申办方/ 发起者: 迪哲（江苏）医药股份有限公司
入组标准(简版):
（1）年龄≥ 18 岁；
（2）ECOG评分 0 - 2 分；
（3）预期寿命应≥ 12 周。
（4）组织病理确诊为 PTCL，符合入选标准的组织学亚型限于以下几种：
a)PTCL ，非特指型（PTCL ，NOS）
b)血管免疫母细胞型 T 细胞淋巴瘤（AITL）
c)间变大细胞型淋巴瘤 ALK+（ALK+ALCL）
d)间变大细胞型淋巴瘤 ALK-（ALK-ALCL）
e)滤泡性辅助 T 细胞淋巴瘤 ，或 TFH 表型 PTCL（FTCL或PTCL-TFH）
f)肠病相关性 T 细胞淋巴瘤（EATL）
g)单形性嗜上皮性肠道 T 细胞淋巴瘤（MEITL）
h)NK/T 细胞淋巴瘤（NK/TCL）
i)肝脾 T 细胞淋巴瘤（HSTCL）
j)皮下脂膜炎样 T 细胞淋巴瘤（SPTCL）
（5）接受过至少一线系统性标准治疗后出现疾病进展、无效或不能耐受（CD30+ 的 ALCL ,必须经过维布妥昔单抗治疗；NK/TCL 受试者,既往必须经过含门冬酰胺酶 / 培门冬酶 / 左旋门冬酰胺酶治疗。受试者进入本研究时应不具备造血干细胞移植（自体或异体）指征。
（6）具有较为充足的骨髓造血功能储备和器官系统功能，如下：
a)中性粒细胞计数绝对值≥ 1.5 ×10^9 /L（若基线骨髓累及,应 ≥1.0 ×10^9 /L）
b)血小板计数≥ 100×10^9 /L（若基线骨髓累及,应 ≥ 50 ×10^9 /L）
c)血红蛋白≥ 8 g/dL
d)总胆红素应≤ 1.5 ×ULN（患有 Gilbert 综合征或基线肝脏累及的情况下 ，应 ≤ 3 ×ULN）
e)丙氨酸氨基转移酶（ALT）和天门冬氨酸氨基转移酶（AST） ≤ 2.5 ×ULN
f)γ- 谷氨酰转移酶 (γ- GGT） ≤ 2.5 ×ULN
g)肌酐≤ 1.5 ×ULN ，或肌酐清除率 ≥ 50 mL/min，或 24 小时测量的尿肌酐清除率 ≥ 50 mL/min。
（7）左心室射血分数（LVEF） ≥ 50%。
2、项目名称：评估HP-001 㬵囊在复发难治的多发性骨髓瘤和非霍奇金淋巴瘤患者中的安全性、 耐受性、 药代动力学和有效性的 I 期临床研究
申办方/ 发起者: 上海超阳药业有限公司
入组标准(简版):
（1）根据 2022 版 WHO 淋巴瘤病理分类，病理组织形态学和免疫组化确诊的非霍奇金淋巴瘤；
（2）NHL受试者：（目前仅入组PTCL患者）受试者须已接受可用的所有标准治疗，经研究者评估可能从试验药物治疗中获益 ;
（3）末次治疗后疾病复发或者末次治疗后未达缓解；
（4）具有可测量病灶：要求淋巴结病灶任一长径＞1.5cm 或结外病灶任一长径＞1.0cm；
（5）预计生存期至少 3 个月 ；
（6）男女不限，年龄 18-75 岁；
（7）ECOG 体能状态评分为 0-2；
（8）重要器官的功能符合下列要求：
a)骨髓功能需满足：中性粒细胞计数≥1.0×109/L（检查前2周内未接受过输血或造血刺激因子治疗）；血红蛋白≥70g/L（ NHL 受试者检查前1周内未输注红细胞）；血小板≥50×109/L（检查前1周内未输注血小板或使用血小板生成素等药物）
b)肝功能：丙氨酸氨基转移酶（ALT）≤3×ULN；天门冬氨酸氨基转移酶（AST） ≤3×ULN；血清总胆红素（TBIL） ≤1.5×ULN（除外 Gilbert 综合征，总胆红素≤3.0×ULN)；检查前2周内未使用保肝降酶药物；
c)肾功能：肌酐清除率（CrCl） ≥60ml/min（Cockcroft/Gault 公式 ， MM 受试者肾脏受累者放宽至≥45ml/min）
d)凝血功能：国际标准化比率（INR） ≤1.5×ULN；活化部分凝血活酶时间（APTT） ≤1.5×ULN；
3、项目名称：一项在晚期实体瘤或血液系统恶性肿瘤患者中评价口服APG-5918 的安全性、 药代动力学和疗效的 I 期研究（复发难治外周T细胞淋巴瘤）
申办方/ 发起者: 苏州亚盛药业有限公司
入组标准(简版):
（1）年龄≥18 岁；
（2）ECOG体能状态为0-1；
（3）预期寿命 > 3 个月；
（4）患有恶性肿瘤：根据 NHL 的非霍奇金淋巴瘤 Cheson 缓解标准，具有可测量的病灶。
（5）具有充分的血液学功能，定义为：
a)在研究药物首次给药前7天内 ANC≥1.0×10^9/L ，但不依赖于生长因子支持。
b)研究药物首次给药前7天内血红蛋白≥9 g/dL 且未进行输血或生长因子支持。
c)研究药物首次给药前 7 天内血小板计数≥75×10^9/L 且未进行输血支持。
（6）肝功能和肾功能良好，定义为：
a)AST 和 ALT≤3×ULN（正常值上限）（如果存在肝转移，则≤5×ULN）
b)使用 Cockcroft-Gault 公式计算的肌酐清除率≥50 mL/min
c)总胆红素≤1.5×ULN（除非认为继发于 Gilbert 综合征和主要为间接胆红素血症）
（7）PT 和aPTT≤2×ULN。
（8）肌钙蛋白≤2×ULN。
（9）对于所有性别的 QTcF 间期≤470ms（需测量3次，取平均值，测量间隔时间为 2-5 分钟）
4、项目名称：HH2853 对照西达本胺治疗复发 / 难治性外周 T 细胞淋巴瘤的随机、双盲、对照、 III期临床研究
申办方/发起者:上海海和药物研究开发股份有限公司
入组标准(简版):
（1）年龄≥20 岁且≤70 岁；
（2）必须提供足量且合格的组织样本（新鲜制备或留档的肿瘤组织样本），且经中心临床病理证实的外周 T 细胞淋巴瘤，具体亚型如下：1)外周 T 细胞淋巴瘤 - 非特指型；2) 间变性大细胞型淋巴瘤 ALK+；3) 间变性大细胞型淋巴瘤 ALK-；4)结内滤泡辅助 T 细胞淋巴瘤 ，血管免疫母型；5)结内滤泡辅助 T 细胞淋巴瘤 ，非特指型；6)结内滤泡辅助 T 细胞淋巴瘤 ，滤泡型；7)结外 NK/T 细胞淋巴瘤；8)肠病相关性 T 细胞淋巴瘤；9)单形性嗜上皮性肠道 T 细胞淋巴瘤；10)肝脾 T 细胞淋巴瘤；11)肠道 T 细胞淋巴瘤，非特指型
（3）ECOG体能状态为0-1；
（4）既往至少接受过一线充分治疗的复发或难治性受试者（对于CD30+ 的ALCL受试者，既往应接受过维布妥昔单抗治疗；对于ENKTCL受试者既往应该接受过含门冬酰胺酶/培门冬酶方案的治疗）。
（5）既往接受过放疗的受试者可以入组，单独放疗将不被视为系统性全身治疗方案。
（6）受试者进入本研究时应不具备造血干细胞移植（自体或异体）指征或拒绝接受移植。
（7）具有可测量病灶（接受过放疗的病灶在放疗后如果有明显的疾病进展证据，则可作为靶病灶）：可测量淋巴结需长径＞ 1.5cm；可测量结外病灶需长径＞1.0cm。
（8）具有足够的骨髓、肝肾器官功能：多发性骨髓瘤（复发难治）
1、项目名称：伊基奥仑赛注射液治疗来那度胺耐药的复发/ 难治性多发性骨髓瘤患者的随机对照Ⅲ临床研究临床试验方案
申办方/ 发起者: 南京驯鹿生物医药有限公司
入组标准(简版):
（1）年龄18 岁至 75 岁（包含临界值），性别不限；
（2）受试者既往诊断为多发性骨髓瘤，经过 1-2 线治疗（包含蛋白酶体抑制剂和免疫调节剂为基础的化疗方案，每一线治疗至少接受≥1 个完整周期；在最近的抗骨髓瘤治疗期间或之后 12 个月内出现有检查资料证明的疾病进展；（既往仅接受过一线治疗的受试者：如果接受过自体干细胞移植，需在自体造血干细胞移植后24 个月内疾病进展或经研究者判断受试者不适合接受再次自体造血干细胞治疗；如果没有接受过自体造血干细胞移植，需经研究者判断不适合行自体造血干细胞移植治疗。）
（3）受试者既往对来那度胺治疗耐药：既往任何一线含来那度胺方案治疗过程中未达到微小缓解（MR）及以上疗效，或在来那度胺用药期间或用药结束后 60 天内（包括来那度胺维持治疗）出现疾病进展（PD）；
（4）根据以下任一标准确定筛选时存在可测量病灶：
a)血清单克隆蛋白（M-蛋白） 水平：≥5g/L；
b)尿 M 蛋白水平≥200 mg/24 小时；
c)受累的血清游离轻链≥100 mg/L 且血清κ/λ游离轻链比异常；
（5）ECOG 评分为 0 或 1 分；
（6）受试者必须有适当的器官功能，入组前符合下列所有实验室检查结果：
a)血常规：中性粒细胞绝对计数（ANC）≥1×109/L（允许使用过生长因子支持 ，但在实验室检查前 7 天内必须未接受过支持治疗）；绝对淋巴细胞计数（ALC） ≥0.3×109/L；血小板≥50×109/L（实验室检查前 7 天内必须未接受过输血小板支持）；血红蛋白≥60g/L（实验室检查前 7 天内没有输注过红细胞；允许使用重组人红细胞生成素）；
b)肝功能：丙氨酸氨基转移酶（ALT）和天冬氨酸氨基转移酶（AST）≤2.5×正常值上限（ULN）；血清总胆红素≤1.5×ULN；
c)肾功能：根据 Cockcroft-Gault 公式计算的肌酐清除率（CrCl）≥ 40 ml/min；
d)凝血功能：纤维蛋白原≥1.0g/L；活化的部分凝血活酶时间(aPTT) ≤1.5×ULN ，凝血酶原时间（PT）≤1.5×ULN；
e)血氧饱和度 >91%；
f)左心室射血分数(LVEF）≥50%；
2、项目名称：一项在复发/ 难治性多发性骨髓瘤（ RRMM）受试者中评估注射用 QLS32015 单药对比研究者选择的方案的有效性和安全性的随机、对照、 多中心、 开放的 III 期研究
申办方/ 发起者: 齐鲁制药有限公司
入组标准(简版):
（1）年龄≥18 周岁 ，性别不限；
（2）根据 2016 年国际骨髓瘤工作组（IMWG）诊断标准确诊的多发性骨髓瘤患者
（3）既往接受过≥3 线治疗（包括一种蛋白酶体抑制剂、一种免疫调节剂和一种抗 CD38 单抗）失败或不耐受的复发性或难治性多发性骨髓瘤患者；
（4）多发性骨髓瘤患者筛选时存在可测量病灶，定义为符合以下任何一项：1) 血清 M 蛋白水平≥1.0 g/dL（10 g/L）；2) 尿 M 蛋白水平≥200 mg/24 h；3) 血清免疫球蛋白游离轻链≥10 mg/dL（100 mg/L）且血清免疫球蛋白 κ/λ游离轻链比异常。
（5）血液学满足以下条件：
a)血红蛋白：≥80 g/L（实验室检查前7天内不能输注过红细胞；允许使用重组人促红细胞生成素）；
b)血小板计数≥75×109/L，在基线浆细胞占骨髓有核比例≥50% 的受试者中，血小板计数≥50×109/L即可入组；（实验室检查前7天内不得接受过输血支持或血小板刺激因子）；
c)中性粒细胞绝对计数≥1.0×109/L（允许既往使用过生长因子，但在实验室检查前7天内不得接受过生长因子的支持治疗）。
（6）生化实验室检查值满足以下条件：
a)天门冬氨酸氨基转移酶和丙氨酸氨基转移酶（ALT/AST）：≤3.0×正常上限（ULN）；
b)肌酐清除率（CrCl）：≥40 mL/min；
c)总胆红素（TBIL）： ≤2.0×ULN（先天性胆红素血症患者除外，例如 Gilbert 综合征，在这种情况下，要求直接胆红素≤1.5×ULN）；
d)校正血清钙：≤3.5 mmol/L或游离离子钙≤6.5 mg/dL ( ≤1.6 mmol/L）；
e)凝血：国际标准化比值（INR）≤1.5×ULN；凝血酶原时间（PT）延长≤1.5×ULN；活化部分凝血活酶时间（APTT）≤1.5×ULN。
（7）ECOG评分 0-2 分；
3、项目名称：评价注射用BL-M24D1 在复发或难治性多发性骨髓瘤及其他血液系统恶性肿瘤患者中的安全性、耐受性、 药代动力学特征和初步疗效的 I 期临床研究
申办方/ 发起者: 成都百利多特生物药业有限责任公司
入组标准(简版):
（1）预期生存时间≥3 个月；
（2）经病理组织学和/或细胞学确诊的标准治疗失败或目前尚无标准治疗的多发性骨髓瘤（根据 IMWG 标准)；既往治疗线数≥2 线，并对至少1种免疫调节剂（IMiDs）和至少1种蛋白酶体抑制剂（PIs）耐药；
（3）必须具有以下至少一项可测量指标：1）血清 M 蛋白≥5 g/L ，2）尿 M 蛋白≥200 mg/24小时 ，3）在血清游离轻链比率异常的情况下，受累血清游离轻链≥100 mg/L；
（4）既往抗肿瘤治疗的毒性已恢复至 NCI-CTCAE v5.0 定义的≤1 级（研究者考虑无症状性实验室检查异常除外，如 ALP 升高、淋巴细胞绝对值降低、高尿酸血症、血清淀粉酶/脂肪酶升高、血糖升高等；研究者判断无安全风险的毒性除外，如脱发、经激素替代治疗稳定的甲状腺功能减退等）；
（5）无严重心脏功能异常，左心室射血分数≥50%；
4、项目名称：评估HP-001 㬵囊在复发难治的多发性骨髓瘤和非霍奇金淋巴瘤患者中的安全性、 耐受性、药代动力学和有效性的I期临床研究
申办方/ 发起者: 上海超阳药业有限公司
入组标准(简版):
（1）根据 IMWG 的标准 ，诊断为活动性多发性骨髓瘤；
（2）既往接受过至少一种蛋白酶体抑制剂、一种免疫调节剂和一种 CD38 单抗治疗（允许经研究者判断不合适进行 CD38 单抗治疗或因经济原因无法接受 CD38 单抗治疗的受试者入组）；
（3）末次治疗后疾病复发或者末次治疗后未达缓解；
（4）具有可测量病，MM 受试者满足3条中至少1条：①血清 M 蛋白≥5g/L；②尿 M 蛋 白≥200mg/24 h；③⾎清游离轻链（ FLC）分析：如果血清 FLC 比例 异常 ，则涉及 FLC 水平≥100mg / L；
（5）预计生存期至少 3 个月 ；
（6）年龄 18-75 岁；
（7）ECOG 体能状态评分为 0-2；
（8）重要器官的功能符合下列要求：
e)骨髓功能需满足：中性粒细胞计数≥1.0×109/L（检查前2周内未接受过输血或造血刺激因子治疗）；血红蛋白≥70g/L（骨髓瘤患者不强制要求）；血小板≥50×109/L（检查前1周内未输注血小板或使用血小板生成素等药物）
f)肝功能：丙氨酸氨基转移酶（ALT）≤3×ULN；天门冬氨酸氨基转移酶（AST）≤3×ULN；血清总胆红素（TBIL）≤1.5×ULN（除外 Gilbert 综合征，总胆红素≤3.0×ULN)；检查前2周内未使用保肝降酶药物；
g)肾功能：肌酐清除率（CrCl） ≥60ml/min（Cockcroft/Gault 公式，MM受试者肾脏受累者放宽至≥45ml/min）
h)凝血功能：国际标准化比率（INR） ≤1.5×ULN；活化部分凝血活酶时间（APTT） ≤1.5×ULN；
5、项目名称：评估HP-001 胶囊联合地塞米松在复发难治的多发性骨髓瘤患者中的安全性、耐受性、 药代动力学和有效性的 I/II 期临床研究
申办方/ 发起者: 上海超阳药业有限公司
入组标准(简版):
（1）根据 IMWG 的标准 ，诊断为活动性多发性骨髓瘤；
（2）接受过至少 3 线多发性骨髓瘤治疗 ，既往接受过至少一种蛋白酶体抑制剂、一种免疫调节剂和一种 CD38 单抗治疗；
（3）末次治疗后疾病进展或者未达缓解；
（4）具有可测量病灶；
（5）ECOG体能状态为0-2；
（6）预计生存期至少 3 个月；
（7）中性粒细胞计数≥1.0*109/L ，血红蛋白≥10g/L ，血小板≥75*109/L；ALT≤3*ULN， AST≤3*ULN ，TBIL≤1.5*ULN；CrCl≥45ml/min ；INR≤1.5ULN,APTT≤1.5ULN
6、项目名称：一项在晚期实体瘤或血液系统恶性肿瘤患者中评价口服APG-5918 的安全性、 药代动力学和疗效的 I 期研究（复发进展骨髓瘤）
申办方/ 发起者: 苏州亚盛药业有限公司
入组标准(简版):
（1）年龄≥18 岁；
（2）ECOG体能状态为0-1；
（3）预期寿命 > 3 个月；
（4）患有恶性肿瘤：2022 年第 5 版造血与淋巴组织肿瘤分类的浆细胞肿瘤，符合疾病进展或者临床复发（IWMG 2016）
（5）具有充分的血液学功能，定义为：
a)在研究药物首次给药前7天内 ANC≥1.0×10^9/L ，但不依赖于生长因子支持。
b)研究药物首次给药前7天内血红蛋白≥9 g/dL 且未进行输血或生长因子支持。
c)研究药物首次给药前 7 天内血小板计数≥75×10^9/L 且未进行输血支持。
（6）肝功能和肾功能良好，定义为：
a)AST 和 ALT≤3×ULN（正常值上限）（如果存在肝转移，则≤5×ULN）
b)使用 Cockcroft-Gault 公式计算的肌酐清除率≥50 mL/min
c)总胆红素≤1.5×ULN（除非认为继发于 Gilbert 综合征和主要为间接胆红素血症）
（7）PT 和aPTT≤2×ULN。
（8）肌钙蛋白≤2×ULN。
（9）对于所有性别的 QTcF 间期≤470ms（需测量3次，取平均值，测量间隔时间为 2-5 分钟
7、项目名称：评价注射用QLS4131 联合用药治疗恶性浆细胞肿瘤的多中心、 开放性II期研究
申办方/ 发起者: 齐鲁制药有限公司
入组标准(简版):
（1）年龄≥18 岁；
（2）根据 2016 年国际骨髓瘤工作组(IMWG)诊断标准确诊的多发性骨髓瘤患者 及相关指南明确诊断的浆细胞白血病和原发性轻链型淀粉样变性等患者；对于多发性骨髓瘤和浆细胞白血病患者筛选时存在可测量病灶；对于轻链型淀粉样变性患者：存在可测量病灶，定义为：受累的血清游离轻链≥50mg/L 且轻链比值异常或血清游离轻链差值(dFLC, 受累轻链和未受累轻链 差值)≥50 mg/L；
（3）ECOG评分 0~1 分；预计生存期≥3 个月 ；
（4）具有充分的血液学、肝肾、凝血功能。急性髓系白血病（初治）
1、项目名称：一项评价Bleximenib、维奈克拉和阿扎胞苷治疗携带 KMT2A 重排或 NPM1 突变且不适合强化疗的新诊断急性髓系白血病受试者的 III 期、随机、双盲、安慰剂对照研究
申办方/ 发起者: Janssen Research & Development, LLC
入组标准(简版):
（1）年龄≥18 岁。
（2）根据 2022 年国际共识分类(ICC）标准，为骨髓原始细胞≥10% 的既往未接受过治疗的 KMT2Ar或 NPM1m AML 患者。 KMT2A 部分串联重复或扩增的受试者无资格参加研究。有共突变（例如,IDH1 共突变） 的受试者必须接受获批的 SoC 治疗,除非他们不适合或拒绝这些治疗。 允许在研究治疗首次给药前进行紧急白细胞分离或使用羟基脲 /1剂阿糖胞苷（最高 2 g/m2）进行细胞减灭治疗。注：在筛选期骨髓评估后才能给予这些治疗。白细胞分离和阿糖胞苷治疗必须在研究治疗首次给药前≥24 小时停止。
（3）基于以下标准判定不适合强化疗：
a.年龄≥75 岁且医师判断其不适合强化疗,美国东部肿瘤协作组（ ECOG）体能状态评分为0-2
b.年龄≥18 岁至＜ 75 岁,且存在≥1 种以下合并症： ECOG 体能状态评分为2；重度心脏疾病（例如，需要治疗的充血性心力衰竭，或慢性稳定型心绞痛）；重度肺部疾病（例如，肺一氧化碳弥散量[DLCO]≤65%或一秒用力呼吸容量[FEV1]≤65%）；肾功能损害，定义为估算的肾小球滤过率（eGFR≥30 mL/min 至＜ 60 mL/min；
在研究者看来，导致受试者不适于进行强化疗的合并症，必须记录在案并在入组之前得到申办方批准。
（4）随机化前有足够的肾功能和肝功能：
a.天门冬氨酸氨基转移酶（AST）和丙氨酸氨基转移酶（ALT）＜3×正常上限（ULN）；对于白血病器官受累（通过活检或影像学证实）的受试者，允许 AST 和 ALT＜ 5×ULN
b.总胆红素≤1.5×ULN，除非胆红素升高是由先天性非溶血性高胆红素血症（例如Gilbert 综合征）所致或属于非肝源性升高
c. eGFR≥30 mL/min。
（5）白细胞（WBC）计数＜ 25×109/L。
2、项目名称：一项在新诊断的FLT3-ITD（+）急性髓系白血病完全缓解成年患者中评价奎扎替尼作为维持治疗的两个剂量水平的安全性和有效性的2期、多中心、随机、开放标签研究
申办方/ 发起者: 第一三共（中国）投资有限公司
入组标准(简版):
（1）年龄≥18 岁；
（2）根据世界卫生组织（WHO）2008/2016分类 ，形态学确诊的、新诊断的原发性 AML 或继发于骨髓增生异常综合征或骨髓增殖性肿瘤的 AML。
（3）受试者初始诊断时的骨髓或外周血中证实存在 FLT3-ITD 阳性（SR≥0.05 或 VAF≥5%）激活突变。
（4）受试者必须在诱导/巩固化疗后，根据当地实验室结果，在维持治疗 C1D1 前 28 天内进行的、最近一次的骨穿（BMA）确认为形态学 CR（CR1）。
（5）受试者必须符合以下既往治疗要求：
a.接受了至少1个周期但不超过2个周期的诱导治疗达到 CR1。诱导周期可采用相同的治疗方案或不同的治疗方案 ，可以仅包含常规药物（例如阿糖胞苷 + 柔红霉素或伊达比星：“7+3”或“5+2”或与 FLT3 抑制剂联合用药。
b.未接受超过4个周期的巩固治疗。治疗方案可仅包含常规药物。
c.FLT3 抑制剂可作为诱导或巩固治疗的一部分。在入组试验前,接受 FLT3 抑制剂治疗的受试者需要洗脱 14 天。
（6）能够在巩固治疗的最后一个周期 D1 的 60 天内开始维持治疗。
（7）ECOG为 0-2 分。
（8）基线当地实验室数据（试验干预首次给药前 7 天内）需符合以下要求： ALT 和 AST ≤2.5 × ULN；肌酐清除率 >50 mL/min；总胆红素（TBL） ≤1.5×ULN ，对于 Gilbert 综合征或溶血受试者 ，直接胆红素≤1.5×ULN且 TBL<3.0×ULN；中性粒细胞绝对计数≥1.0×109/L，4周未接受 G-CSF 支持治疗血小板 ≥100×109/L ，14 天内未接受血小板输注支持治疗
（9）随机化前 28 天内LVEF>45%。急性髓系白血病（复发难治）
1、项目名称：HEC73543 片治疗 FLT3-ITD 突变的复发/难治急性髓系白血病的随机、开放、挽救性化疗对照、多中心III期临床试验
申办方/ 发起者: 广东东阳光药业股份有限公司
入组标准(简版):
（1）年龄≥18 周岁 ，性别不限；
（2）按照世界卫生组织(WHO)标准确诊的原发性 AML 或骨髓增生异常综合征(MDS)继发性 AML；
（3）受试者接受一线 AML 治疗(进行或未进行 HSCT)后出现难治或复发；
（4）由中心实验室检测确认受试者骨髓或血液FLT3-ITD 突变阳性；
（5） ECOG 评分为 0-2 分；
（6）预期生存时间≥12 周；
（7）经研究者评估，受试者适合接受试验方案选定的挽救性化疗；
（8）受试者必须有适当的器官功能，入组前符合下列所有实验室检查结果：
a.凝血功能：国际标准化比值(INR)<1.5xULN；
b.电解质：血钾≥3.0 mmol/L；血镁≥0.5 mmol/L；
c.肝功能：ALT 和 AST≤2.5xULN；血清胆红素≤1.5xULN；
d.肾功能：血清肌酐≤1.5xULN；或根据 Cockcroft-Gault 公式计算的肌酐清除率(Ccr) >50mL/min。急性淋巴细胞白血病（复发难治）
1、项目名称：靶向CD19 嵌合抗原受体 T 淋巴细胞（ pCAR-19B） 自体回输制剂治疗 CD19 阳性复发 / 难治性 B 细胞急性淋巴细胞白血病（22-70 周岁） 的 Ⅱ期临床试验
申办方/ 发起者: 重庆精准生物技术有限公司
入组标准(简版):
（1）年龄22-70周岁（含界值），性别不限；
（2）确诊为B 细胞急性淋巴细胞白血病 ，且符合下述条件之一的：
a.难治性B-ALL：初诊经标准诱导化疗方案 2 个疗程骨髓未达到完全缓解的早期难治者；
b.复发性B-ALL：①完全缓解后早期复发（<12 月）；②完全缓解后晚期复发（≥12月)需经再次诱导治疗，治疗后未获完全缓解或早期治疗反应不佳；③经历 2 次及以上骨髓复发；④异基因造血干细胞移植后复发；
c.对于 Ph+ALL 患者 ，除满足复发/难治的标准外，还应至少接受两种 TKI治疗后未获完全缓解者或完全缓解后复发（无法耐受TKI治疗或有TKI治疗禁忌症或存在 T315i 突变对TKI类药物耐药者除外）；
（3）经流式细胞术确认骨髓中的恶性细胞表达 CD19 ，若既往接受过靶向 CD19 抗体治疗（如blinatumomab），则 CD19 阳性恶性细胞比例需≥90%；
（4）筛选时骨髓形态学提⽰原幼淋巴细胞≥5%；
（5）ECOG 0-1 分；
（6）预期生存时间 12 周以上；
（7）重要脏器功能基本正常：
a.心功能：心脏超声提⽰心脏射血分数≥50% ，心电图未见明显异常；
b.肾功能：血清肌酐≤2.0×ULN ，或肌酐清除率≥60ml/min（Cockcroft-Gault 公式）；
c.肝功能：ALT 和 AST ≤5.0×ULN；
d.总胆红素≤2.0×ULN (Gilbert 综合征者需总胆红素≤3.0×ULN)；
e.非吸氧状态下血氧饱和度≥92%。
（8）具备单采采血标准，并且没有其他细胞采集禁忌症；急性T 淋巴母细胞白血病 / 淋巴瘤（T-ALL/LBL）（复发难治）
1、项目名称：PA3-17 注射液治疗复发 / 难治性 T 淋巴母细胞白血病 / 淋巴瘤受试者的单臂、 开放标签的 II 期临床研究）
申办方/ 发起者: 博生吉安科细胞技术有限公司
入组标准(简版):
（1）年龄≥18 岁（包括界值），性别不限；
（2）ECOG 评分 0-1 分；
（3）确诊为 WHO 第五版《血液淋巴肿瘤分类淋巴肿瘤》中分类的急性T淋巴母细胞白血病/淋巴瘤（T-ALL/LBL），包括早期前T淋巴母细胞白血病/淋巴瘤（ETP-ALL/LBL）；确诊的方式包括但不限于组织学、细胞形态学、免疫学、细胞遗传学或分子遗传学等；
（4）经标准治疗失败或缺乏有效治疗手段的复发/难治性 T-ALL/LBL（包括 ETP-ALL/LBL）受试者 ，符合以下任一标准：
a.复发性 T-ALL/LBL：获得完全缓解的受试者出现新发的髓外病变或出现骨髓复发，且再经一个方案的治疗后，受试者仍未获得缓解；或二次及以上复发者；
b.难治性 T-ALL/LBL：初诊经至少两个疗程的规范诱导治疗后未获得缓解的早期难治受试者，且再经一个方案的治疗后，受试者仍未获得缓解；经三线及以上治疗后未获缓解者。
（5）筛选时：①受试者应用多色流式检测骨髓和 / 或外周血中有异常肿瘤细胞 ，无论影像学⽰有无髓外病灶，只要求骨髓异常肿瘤细胞表面 CD7 阳性 ，外周血异常肿瘤细胞表面 CD7 阳性 和 CD4 阴性和 CD8 阴性表达。② 受试者应用多色流式检测骨髓和 / 或外周血中无异常肿瘤细 胞 ，影像学⽰受试者仅有髓外病灶（例如淋巴结肿大）的情况下，要求病灶病理免疫组化明确为 CD7 阳性 ，且肿物 CD7 阳性率≥70%。
（6）受试者仅有髓外病灶时，具有可评估（PET-CT）和/或可测量（增强 CT）的病灶；
（7）肝肾功能、心肺功能满足以下要求：① 肌酐≤×1.5ULN；②左室射血分数≥45%，且超声心动图检查未见显著的（需临床干预的）心包积液，心电图检查未见有临床意义的异常波段；③ 非吸氧状态下血氧饱和度>91%；④总胆红素≤2×ULN；ALT 和 AST≤2.5×ULN；研究者判断由于疾病导致的（例如肝浸润或胆管阻塞）引起的 ALT 和 AST 异常 ，其指标可放宽至≤5×ULN；如诊断为 Gilbert综合征，总胆红素指标可放宽至≤3.0×ULN 且直接胆红素≤1.5×ULN；骨髓纤维化
1、项目名称：评价JAB-8263 在晚期恶性肿瘤患者的安全性、耐受性、 药代动力学特征和抗肿瘤活性的多中心 ，开放的 I/IIa 期临床研究
申办方/发起者：北京加科思新药研发有限公司
主要入组标准（简版）:
（1）原发性骨髓纤维化(PMF)、继发性骨髓纤维化(包括 post-PV MF 和 post-ET MF)患者
（2）既往对 JAK 抑制剂治疗出现耐药、难治、不耐受
（3）血小板≥100x109/L:血红蛋白≥70g/L; 中性粒细胞绝对计数≥1.5x109/L
（4）既往未进行过脾切除手术 , 患者脾脏最突出的部位必须离肋骨下缘≥5 cm 或通过核磁测量脾的体积≥450cm³原发免疫性血小板减少症
1、项目名称：一项在原发免疫性血小板减少症成年受试者中评价艾加莫德α注射液的疗效和安全性的Ⅲ期、多中心、随机、双盲、安慰剂对照、平行组序贯开放标签组研究
申办方/发起者：argenx BV
主要入组标准（简版）:
（1）年满 18 岁 ；
（2）有记录的原发性ITP诊断，根据IWG 定义：在没有其他可能与血小板减少症相关的原因或疾病的情况下，血小板计数 <100 × 10^9/L
（3）在签署 ICF 之日，记录的原发性 ITP 持续时间超过 12 个月；
（4）有既往 ITP 治疗记录，包括接受过至少1种以下治疗：皮质类固醇；IVIg；抗 D 免疫球蛋白（适用于未接受脾切除术的 Rho(D)阳性受试者）；TPO-RA；或利妥昔单抗；
（5）具有对既往 ITP 治疗应答不充分的记录，包括：皮质类固醇；IVIg；抗 D 免疫球蛋白 （适用于未接受脾切除术的 Rho(D)阳性受试者）；TPO-RA；利妥昔单抗；或脾切除术 ，符合以下任一标准：
a)治疗后无血小板计数≥50×10^9/L；
b)治疗后任何血小板计数≥50×10^9/L ，随后任何血小板计数 <50×10^9/L；
c)血小板计数相对于治疗前计数值增加不到2倍；
d)脾切除术后无血小板计数≥30×10^9/L；
e)脾切除术后任何血小板计数≥30×10^9/L ，随后任何血小板计数 <30×10^9/L；
f)如果皮质类固醇逐渐减量，血小板计数降低至阈值30×10^9/L 以下；
g)需要持续给予泼尼松（或皮质类固醇等效药物）5 mg/d ，以维持血小板计数≥30×10^9/L和/或避免出血；
h)需要重复给予皮质类固醇，持续至少2个月，以维持血小板计数≥30×10^9/L和/或避免出血；
（6）有既往应答记录，其定义为在ICF签署日期前3年内，接受以下至少1种ITP治疗后出现 1次血小板计数≥50×10^9/L：泼尼松；地塞米松；其他或非特定皮质类固醇；IVIg；或抗D免疫球蛋白（适用于未接受脾切除术的 Rho(D)阳性受试者）
（7）在研究第1 日随机化前记录了基线平均血小板计数 <30 × 10^9/L
（8）在研究第1 日随机化前有1次合格的血小板计数记录（即算术平均值计算公式中用到的血小板计数）。
（9）在研究第 -14 日至研究第1 日随机化前 ，至少有 2 次合格的血小板计数记录
（10）在研究第1 日随机化前 3 个月内 ，至少有 3 次合格的血小板计数记录
（11）在研究第1 日随机化前 30 日内 ，未记录血小板计数 >35×10^9/L
（12）血红蛋白水平 >90 g/L
（13）凝血酶原时间和活化部分凝血活酶时间在正常实验室检查值范围内
2026-06-27
2026年AACR/ASCO等国际肿瘤会议 热门药物靶点TOP 20综合报告
报告日期：2026年6月27日
数据来源：2026年AACR年会、ASCO年会及近期相关学术会议公开信息
日期：2026年6月27日
数据来源：2026年AACR年会（4月17-22日，圣地亚哥）、2026年ASCO年会（5月29日-6月2日，芝加哥）及近期相关学术会议公开信息
报告性质：基于公开会议摘要及行业分析整理的科研参考，不构成临床用药或投资建议
目录
1.[报告概述与研究方法](#一报告概述与研究方法)
2.[TOP 20靶点总览表](#二top-20靶点总览表)
3.[TOP 20靶点详细信息（含在研公司、管线及常用细胞系）](#三top-20靶点详细信息)
4.[常用细胞系汇总表](#四top-20靶点常用细胞系汇总表)
5.[关键趋势分析与展望](#五关键趋势分析与展望)
一、报告概述与研究方法1.1 背景
2026年AACR年会共收录超过9,500篇研究摘要，覆盖全球40余个国家和地区的研究机构与企业。2026年ASCO年会中国学者主导入选报告达95项，创历史新高。两大会议集中反映了当前肿瘤药物研发的最新趋势与核心靶点竞争格局。1.2 靶点排名依据
本报告TOP 20靶点排名综合参考以下维度：
评价维度
权重说明
会议摘要数量
AACR/ASCO中与该靶点相关的研究摘要数量
临床阶段进展
是否有Phase III数据公布或上市申请
在研管线丰富度
全球范围内针对该靶点的在研药物数量
BD交易热度
相关管线的License-out/BD交易金额及频次
创新模态覆盖
涵盖ADC、双抗、PROTAC、CAR-T等多模态
行业分析评级
赛业生物、缔码生物等第三方分析报告的靶点梯队划分
1.3 数据来源说明
·AACR 2026官方摘要及New Drugs on the Horizon session
·ASCO 2026口头报告、快速口头报告及壁报
·赛业生物AACR 2026研究报告（7562篇摘要分析）
·缔码生物AACR 2026 ADC靶点分析
·NovaPharmaNews、Labiotech.eu、VCBeat等行业媒体报道
·ClinicalTrials.gov及各企业官方公开信息
二、TOP 20靶点总览表
排名
靶点
靶点类型
热度评级
核心适应症
最热模态
代表性事件
1
KRAS/RAS (Pan-RAS)
小分子GTP酶
⭐⭐⭐⭐⭐
胰腺癌、NSCLC、结直肠癌
Pan-RAS分子胶/抑制剂
Daraxonrasib Phase 3死亡风险↓60%
2
HER2
受体酪氨酸激酶
⭐⭐⭐⭐⭐
乳腺癌、NSCLC、胃癌、结直肠癌
ADC/双payload ADC
AACR 64篇摘要，ADC最热靶点
3
TROP2
跨膜糖蛋白
⭐⭐⭐⭐⭐
三阴性乳腺癌、NSCLC、尿路上皮癌
ADC
Sac-TMT+K药一线NSCLC Phase 3达终点
4
B7-H3 (CD276)
免疫检查点分子
⭐⭐⭐⭐⭐
SCLC、前列腺癌、卵巢癌、胰腺癌
ADC/双抗/NK衔接器
被誉为"肿瘤最热门抗原"
5
CLDN18.2
紧密连接蛋白
⭐⭐⭐⭐
胃癌、胰腺癌
ADC/单抗/CAR-T
信达IBI343全球首个CLDN18.2 ADC申报上市
6
EGFR×HER3 (双抗)
双靶点ADC
⭐⭐⭐⭐
TNBC、食管癌、NSCLC
双抗ADC
百利天恒BL-B01D1多Phase 3口头报告
7
PD-1/VEGF (双抗)
双特异性抗体
⭐⭐⭐⭐
NSCLC、子宫内膜癌
双抗
Summit Ivonescimab BLA受理；三生12.5亿美元交易
8
DLL3
Notch配体
⭐⭐⭐⭐
小细胞肺癌、神经内分泌癌
CAR-T/ADC/TCE
传奇生物DLL3 CAR-T实体瘤数据
9
EGFR×MET (双抗)
双靶点抗体
⭐⭐⭐⭐
NSCLC、头颈癌、结直肠癌
双抗
J&J Rybrevant皮下制剂头颈癌ORR 42%
10
PRMT5
表观遗传酶
⭐⭐⭐
MTAP缺失实体瘤、血液瘤
小分子（MTA协同型）
131个在研药物，65项注册临床
11
Nectin-4
细胞黏附分子
⭐⭐⭐
尿路上皮癌、宫颈癌、TNBC
ADC
迈威9MW2821全球首个宫颈癌III期Nectin-4 ADC
12
GPC3
磷脂酰肌醇聚糖
⭐⭐⭐
肝细胞癌
ADC
乐普MRG006A全球首创GPC3 ADC
13
HER3
受体酪氨酸激酶
⭐⭐⭐
EGFR突变NSCLC、乳腺癌
ADC
恒瑞SHR-A2009全球首个III期成功HER3 ADC
14
SEZ6
跨膜蛋白
⭐⭐⭐
小细胞肺癌
ADC
AbbVie ABBV-706 Phase 3 ORR 82%
15
c-Met
受体酪氨酸激酶
⭐⭐⭐
NSCLC、卵巢癌、头颈癌
ADC/双抗
AbbVie ABBV-400卵巢癌ORR 80%
16
EP300/CBP
组蛋白乙酰转移酶
⭐⭐
多种实体瘤及血液瘤
PROTAC/降解剂
阿斯利康布局，AACR T4前沿潜力靶点
17
CDH17
钙黏蛋白17
⭐⭐
结直肠癌、胃癌
ADC
AACR T3核心BD方向，竞争极早期
18
METTL3
RNA甲基转移酶
⭐⭐
肉瘤、AML及多种实体瘤
小分子
Storm STC-15 Phase 1→2
19
Menin (KMT2A)
蛋白质相互作用分子
⭐⭐
AML（KMT2A重排/NPM1突变）
小分子
Syndax Revumenib 2年OS率90%
20
WRN (Werner螺旋酶)
DNA解旋酶
⭐⭐
MSI-H癌症（结直肠癌、子宫内膜癌等）
小分子
DepMap 2026 AACR团队科学奖；合成致死新靶点
三、TOP 20靶点详细信息TOP 1：KRAS / Pan-RAS靶点概述
KRAS是肿瘤学史上最重要的致癌基因之一，曾被称为"不可成药"靶点。2026年AACR/ASCO会议中，Pan-RAS分子胶成为本届最高频新药类别（AACR 37篇摘要）。Revolution Medicines的Daraxonrasib在ASCO 2026公布的Phase 3数据显示胰腺癌死亡风险降低60%，被称为"game changer"。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据
Revolution Medicines / Amgen
Daraxonrasib
RAS(ON)抑制剂（Pan-RAS分子胶）
胰腺导管腺癌
Phase 3
ORR 33.2% vs 化疗11.8%；死亡风险↓60%
Amgen
Lumakras (Sotorasib)
KRAS G12C抑制剂
胰腺腺癌
Phase 1/2
单药ORR 47%, DCR 92%；联合ORR 58%
劲方医药
GFH276
RAS分子胶
泛实体瘤
Phase 1
国产RAS分子胶FIC
MSK
mRNA疫苗
KRAS mRNA疫苗
胰腺癌
Phase 1
早期阶段
Mirati/BMS
Adagrasib (Krazati)
KRAS G12C抑制剂
NSCLC、结直肠癌
上市/Phase 3
已获批二线NSCLC
Eli Lilly
Olomorasib (LY3537982)
KRAS G12C抑制剂
NSCLC
Phase 1/2
联合K药数据
Novartis
JDQ443
KRAS G12C抑制剂
NSCLC
Phase 2
KontRASt-01研究
常用细胞系
细胞系
KRAS突变类型
肿瘤类型
来源
MIA PaCa-2
G12C
胰腺癌
人胰腺导管腺癌
Panc-1
G12D
胰腺癌
人胰腺导管上皮癌
AsPC-1
G12D
胰腺癌
人胰腺腺癌
NCI-H358
G12C
NSCLC
人肺腺癌
NCI-H23
G12C
NSCLC
人肺腺癌
NCI-H441
G12V
NSCLC
人肺乳头状腺癌
HCT-116
G13D
结直肠癌
人结肠癌
SW620
G12V
结直肠癌
人结肠癌（淋巴结转移）
SW1417
G12V
结直肠癌
人结肠癌
A549
G12S
NSCLC
人肺腺癌
CAPAN-1
G12V
胰腺癌
人胰腺导管腺癌
NCI-H2009
G12C
NSCLC
人肺腺癌
TOP 2：HER2 (ERBB2)靶点概述
HER2是AACR 2026中ADC领域摘要数量最多的靶点（64篇），热度明显高于其他方向。研发重点已从靶点验证进入设计升级阶段，聚焦HER2低表达拓展、双payload ADC、联合治疗及双特异性ADC。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
恒瑞医药
SHR-A1811
HER2 ADC
HER2突变NSCLC
Phase 3
ORR 81.3%，全球最佳数据，License-out标杆
第一三共/阿斯利康
T-DXd (Enhertu)
HER2 ADC
乳腺癌、胃癌、NSCLC等
上市/多适应症拓展
ADC霸主地位夯实，多适应症推进
Boehringer Ingelheim
Zongertinib
HER2 TKI
HER2+ NSCLC、mCRC
Phase 2
mCRC ORR 42%, DCR 95%；NSCLC一周内见效
康宁杰瑞
KN026 (安炳尼单抗)
HER2双抗
新辅助乳腺癌
Phase 2/3
LBA口头报告
荣昌生物
RC48 (维迪西妥单抗)
HER2 ADC
尿路上皮癌、胃癌
上市/Phase 3
中国首个原创ADC
Roche
Trastuzumab + Pertuzumab
单抗联用
HER2+乳腺癌
上市
标准治疗基础
百利天恒
BL-M07D1
HER2 ADC
乳腺癌
Phase 1/2
差异化设计
常用细胞系
细胞系
HER2状态
肿瘤类型
特征
SK-BR-3
HER2高表达（ER-/PR-）
乳腺癌
HER2研究金标准细胞系
BT-474
HER2高表达（ER+/PR+）
乳腺癌
适合研究HER2与激素受体交叉调控
HCC1954
HER2过表达
乳腺癌
HER2扩增，ER阴性
MDA-MB-453
HER2中表达
乳腺癌
HER2低表达模型
NCI-N87
HER2高表达
胃癌
HER2+胃癌经典模型
SK-OV-3
HER2过表达
卵巢癌
HER2+卵巢癌模型
NCI-H2170
HER2扩增
NSCLC
HER2+肺鳞癌
Calu-3
HER2过表达
NSCLC
HER2+肺腺癌
MCF-7
HER2低表达
乳腺癌
HER2阴性对照
MDA-MB-231
HER2阴性
乳腺癌
三阴性乳腺癌对照
TOP 3：TROP2靶点概述
TROP2是AACR 2026中第二大热门ADC靶点（33篇摘要）。在sacituzumab govitecan打开临床认知后，TROP2赛道呈现两类趋势：已上市分子拓展适应症和联合方案，新一代差异化TROP2 ADC解决疗效深度、旁观者效应和耐药问题。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
科伦博泰/默沙东
Sacituzumab Tirumotecan (sac-TMT)
TROP2 ADC
PD-L1+ NSCLC一线、TNBC
Phase 3（上市）
一线NSCLC联合K药：PFS风险↓65%（HR=0.35）
Gilead
Sacituzumab Govitecan (Trodelvy)
TROP2 ADC
TNBC、HR+/HER2-乳腺癌、尿路上皮癌
上市
TROP2 ADC开创者
康宁杰瑞
JSKN016
TROP2/HER3双抗ADC
HER2阴性乳腺癌
Phase 1
双靶点创新设计
第一三共
Datopotamab Deruxtecan (Dato-DXd)
TROP2 ADC
NSCLC、TNBC
Phase 3
TROP2 ADC竞争者
恒瑞医药
SHR-A1921
TROP2 ADC
多实体瘤
Phase 1/2
差异化设计
多禧生物
DX126-262
TROP2 ADC
实体瘤
Phase 1/2
新型连接子
百奥泰
BAT-8008
TROP2 ADC
实体瘤
Phase 1/2
国产TROP2 ADC
常用细胞系
细胞系
TROP2表达
肿瘤类型
特征
MDA-MB-468
高表达
三阴性乳腺癌
TROP2研究经典细胞系
HCC1806
高表达
三阴性乳腺癌
TROP2高表达
HCC38
高表达
乳腺癌
TROP2+乳腺癌
BT-549
中/高表达
乳腺癌
TNBC
T47D
中表达
乳腺癌
管腔型
BxPC-3
中表达
胰腺癌
TROP2+胰腺癌
NCI-H1993
中表达
NSCLC
肺腺癌
A431
高表达
表皮样癌
TROP2高表达
Calu-3
中表达
NSCLC
肺腺癌
SNU-16
中表达
胃癌
TROP2+胃癌
TOP 4：B7-H3 (CD276)靶点概述
B7-H3被2026年行业分析誉为"肿瘤学中最热门的抗原"。广泛高表达于前列腺癌、肺癌、乳腺癌、卵巢癌等高致死性实体瘤，正常组织几乎不表达。AACR 2026收录15篇相关摘要，竞争进入双靶设计、适应症细分和机制优化阶段。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
GSK
Risvutatug Rezetecan (GSK'227)
B7-H3 ADC
SCLC等多实体瘤
Phase 1/2
与Summit PD-1/VEGF双抗联用
IDEAYA Biosciences
IDE034
PTK7/B7-H3双特异ADC
肺癌、乳腺癌、卵巢癌
Phase 1
全球潜在首发双靶点ADC，需双抗原共表达
GT Biopharma
GTB-5550
B7-H3 TriKE（NK衔接器）
前列腺癌、卵巢癌、胰腺癌
Phase 1
全球首个皮下给药B7-H3 NK衔接器
第一三共/默沙东
Ifinatamab Deruxtecan (I-DXd)
B7-H3 ADC
SCLC
Phase 2
SCLC ORR数据优异
百利天恒
BL-M14D1
B7-H3 ADC
实体瘤
Phase 1
国产B7-H3 ADC
翰森制药
HS-20093
B7-H3 ADC
SCLC、骨肉瘤等
Phase 1/2
国产B7-H3 ADC领先
宜联生物
YL201
B7-H3 ADC
SCLC、鼻咽癌等
Phase 1/2
新型连接子技术
常用细胞系
细胞系
B7-H3表达
肿瘤类型
特征
A204
高表达
横纹肌肉瘤
B7-H3研究经典模型
NCI-H526
高表达
SCLC
B7-H3+小细胞肺癌
NCI-H446
高表达
SCLC
B7-H3+小细胞肺癌
NCI-H69
中/高表达
SCLC
SCLC经典模型
RD-ES
高表达
尤文肉瘤
B7-H3高表达
SK-N-DZ
高表达
神经母细胞瘤
B7-H3+神经母细胞瘤
LNCaP
中表达
前列腺癌
B7-H3+前列腺癌
DU145
中表达
前列腺癌
B7-H3+前列腺癌
OVCAR-3
中表达
卵巢癌
B7-H3+卵巢癌
MCF-7
低/中表达
乳腺癌
B7-H3低表达对照
TOP 5：CLDN18.2靶点概述
CLDN18.2（Claudin 18.2）是消化道肿瘤领域最重要的新兴靶点。2026年ASCO会议上，信达生物IBI343成为全球首个申报上市的CLDN18.2 ADC，在晚期胃癌III期临床中达到主要终点。AACR 2026收录7篇相关摘要，热度正从单抗/双抗向ADC设计扩展。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
信达生物/武田
IBI343 (TAK-921)
CLDN18.2 ADC
晚期胃/胃食管结合部腺癌
上市申请（NDA）
全球首个CLDN18.2 ADC，III期达主要终点
礼新医药/中国生物制药
LM-302 (维特柯妥拜单抗)
CLDN18.2 ADC
胃癌
上市申请
多项突破性疗法认定
德琪医药/默沙东
ATG-022
CLDN18.2 ADC
实体瘤
Phase 1/2
与K药全球临床合作
安斯泰来
Zolbetuximab (佐妥昔单抗)
CLDN18.2单抗
胃癌
上市（日本/中国）
全球首个CLDN18.2单抗获批
创胜集团
TST001 (Osemitamab)
CLDN18.2单抗
胃癌、胰腺癌
Phase 2/3
增强ADCC设计
科济药业
CT041 (Satricabtagene Autoleucel)
CLDN18.2 CAR-T
胃癌、胰腺癌
Phase 2
全球首个CLDN18.2 CAR-T
百利天恒
BL-M09
CLDN18.2 ADC
胃癌
Phase 1
新型ADC设计
常用细胞系
细胞系
CLDN18.2表达
肿瘤类型
特征
AGS
内源性表达
胃癌
CLDN18.2+胃癌经典模型
NUGC-4
内源性表达
胃癌
CLDN18.2+胃癌
KATO III
内源性表达
胃癌
弥漫型胃癌
SNU-216
低/中表达
胃癌
肠型胃癌
SNU-16
低表达
胃癌
CLDN18.2低表达对照
SNU-1
低/无表达
胃癌
CLDN18.2阴性对照
BxPC-3
需转染
胰腺癌
CLDN18.2基因工程模型
PANC-1
需转染
胰腺癌
CLDN18.2基因工程模型
HEK293-CLDN18.2
稳转
基因工程
过表达对照系统
CHO-CLDN18.2
稳转
基因工程
结合活性检测
TOP 6：EGFR×HER3（双抗ADC）靶点概述
EGFR×HER3双靶点ADC是2026年ASCO的里程碑式突破。百利天恒BL-B01D1（iza-bren）是全球首个进入III期临床的EGFR×HER3双抗ADC，在食管鳞癌III期研究中双终点（OS/PFS）均达统计学显著，是双抗ADC在消化道肿瘤领域的重大突破。海外权益授权给BMS，总交易额最高达84亿美元，为当年中国创新药出海授权最高纪录。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
百利天恒/BMS
BL-B01D1 (Iza-bren)
EGFR×HER3双抗ADC
TNBC、食管鳞癌、NSCLC
Phase 3
食管癌OS 9.8 vs 7.2个月（HR=0.64）；84亿美元BD交易
恒瑞医药
SHR-A2009
HER3 ADC
EGFR突变NSCLC
Phase 3
全球首个III期成功HER3 ADC，预计2027年获批
第一三共
Patritumab Deruxtecan (HER3-DXd)
HER3 ADC
EGFR突变NSCLC
Phase 3（已申报）
HER3-DXd已提交BLA
Immunomedics
Sacituzumab Govitecan (技术平台)
—
—
—
相关HER3平台技术
常用细胞系
细胞系
EGFR/HER3状态
肿瘤类型
特征
HCC827
EGFR突变/HER3+
NSCLC
EGFR外显子19缺失
HCC8205
EGFR突变/HER3+
NSCLC
EGFR-TKI耐药模型
NCI-H1975
EGFR T790M/HER3+
NSCLC
EGFR耐药突变
FaDu
EGFR+/HER3+
头颈鳞癌
食管/头颈模型
MDA-MB-468
EGFR高表达/HER3+
三阴性乳腺癌
TNBC模型
MCF-7
HER3+
乳腺癌
HER3+乳腺癌
SK-BR-3
HER3+
乳腺癌
HER2+/HER3+共表达
BxPC-3
EGFR+/HER3+
胰腺癌
胰腺癌模型
A431
EGFR高表达
表皮样癌
EGFR过表达
TOP 7：PD-1/VEGF（双特异性抗体）靶点概述
PD-1/VEGF双特异性抗体代表"后K药时代"免疫联合治疗的新范式，通过双靶点同时阻断免疫检查点和血管生成通路。2026年ASCO会议上，Summit/Ivonescimab的BLA获FDA受理，三生制药以12.5亿美元首付款将PD-1/VEGF双抗授权给辉瑞，印证该靶点的巨大商业价值。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
Summit Therapeutics/康方生物
Ivonescimab (AK112)
PD-1/VEGF双抗
NSCLC
BLA受理
PDUFA日期2026-11-14；市值约140亿美元
三生制药/辉瑞
SSGJ-707 (PF-08634404)
PD-1/VEGF双抗
NSCLC、子宫内膜癌
Phase 2
12.5亿美元首付款授权辉瑞
BioNTech/BMS
Pumitamig
PD-(L)1×VEGF双抗
NSCLC
Phase 2/3
各PD-L1表达水平均显示鼓舞活性
恒瑞医药
SHR-1701
PD-L1/TGF-β双抗
多实体瘤
Phase 3
相关双免平台
信达生物
IBI324
PD-1/VEGF双抗
实体瘤
Phase 1
早期开发
常用细胞系
细胞系
PD-L1/VEGF表达
肿瘤类型
应用场景
MC-38
PD-L1+/VEGF+
结直肠癌（小鼠）
体内药效模型（同基因型）
CT26
PD-L1+/VEGF+
结直肠癌（小鼠）
体内药效模型
B16-F10
PD-L1+/VEGF+
黑色素瘤（小鼠）
免疫治疗模型
4T1
PD-L1+/VEGF+
乳腺癌（小鼠）
转移模型
A549
PD-L1+/VEGF+
NSCLC
人源细胞体外验证
HUVEC
VEGFR+
血管内皮
VEGF通路功能验证
NCI-H1975
PD-L1+
NSCLC
PD-L1表达验证
MDA-MB-231
PD-L1+/VEGF+
三阴性乳腺癌
TNBC免疫模型
TOP 8：DLL3靶点概述
DLL3（Delta-like ligand 3）是小细胞肺癌（SCLC）领域验证最充分的靶点之一，85%-96%的SCLC肿瘤高表达DLL3，正常组织几乎不表达。2026年ASCO会议上，传奇生物率先读出DLL3 CAR-T在实体瘤中的早期临床数据，验证CAR-T技术向实体瘤拓展的可行性。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
传奇生物
LB2102
DLL3 CAR-T（装甲型）
SCLC/大细胞神经内分泌癌
Phase 1
实体瘤CAR-T突破
AbbVie
Rova-T (Rovalpituzumab Tesirine)
DLL3 ADC
SCLC
重新开发
早期失败后技术迭代
百利天恒
BL-M14D1
DLL3 ADC
SCLC
Phase 1
新型拓扑异构酶I抑制剂
Amgen
Tarlatamab (AMG 757)
DLL3×CD3 BiTE
SCLC
上市申请
FDA优先审评
Harpoon Therapeutics
HPN328
DLL3三特异性T细胞衔接器
SCLC
Phase 1/2
三特异性设计
翰森制药
HS-20093
B7-H3/DLL3双抗ADC
SCLC
Phase 1/2
—
常用细胞系
细胞系
DLL3表达
肿瘤类型
特征
NCI-H446
高表达
SCLC
SCLC经典模型，DLL3高表达
NCI-H526
高表达
SCLC
DLL3+小细胞肺癌
NCI-H69
高表达
SCLC
SCLC-A亚型（ASCL1高）
NCI-H82
中表达
SCLC
SCLC-N亚型（NEUROD1高）
NCI-H209
高表达
SCLC
SCLC-A亚型
DMS-114
低/无表达
NSCLC
DLL3阴性对照
DMS-153
中表达
SCLC
大细胞神经内分泌癌
TOP 9：EGFR×MET（双特异性抗体）靶点概述
EGFR×MET双特异性抗体通过同时靶向EGFR和c-Met，解决EGFR突变NSCLC的耐药问题，并拓展至头颈部鳞癌和结直肠癌。2026年ASCO会议上，强生Rybrevant Faspro（皮下制剂）在头颈癌中ORR达42%，超过1/3为完全缓解（CR）。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
Johnson & Johnson
Amivantamab (Rybrevant Faspro)
EGFR×MET双抗
NSCLC、头颈癌、结直肠癌
上市/Phase 3
头颈癌ORR 42%，中位OS 12.5个月
岸迈生物
EMB-01
EGFR×MET双抗
实体瘤
Phase 1/2
FIT-Ig平台
贝达药业
MCLA-129
EGFR×MET双抗
NSCLC
Phase 1/2
与JNJ竞争
AstraZeneca
AZD9592
EGFR×MET ADC
NSCLC
Phase 1
ADC化双抗
常用细胞系
细胞系
EGFR/MET状态
肿瘤类型
特征
HCC827
EGFR突变
NSCLC
EGFR外显子19缺失
HCC8205
EGFR突变/MET扩增
NSCLC
EGFR-TKI耐药模型
NCI-H1975
EGFR T790M
NSCLC
EGFR耐药突变
EBC-1
MET扩增
NSCLC
MET驱动
MKN-45
MET扩增
胃癌
MET扩增经典模型
SNU-5
MET扩增
胃癌
MET扩增
FaDu
EGFR+
头颈鳞癌
头颈癌模型
Calu-3
EGFR+/MET+
NSCLC
双靶共表达
TOP 10：PRMT5靶点概述
PRMT5（蛋白精氨酸甲基转移酶5）是MTAP缺失癌症中的关键合成致死靶点。AACR 2026将其列为T4前沿潜力靶点。截至2026年5月，全球已有131个在研药物和65项注册临床试验，尚无药物获批。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
BMS
Navlimetostat (BMS-986504)
MTA协同型PRMT5抑制剂
MTAP缺失实体瘤
Phase 1/2
第一代PRMT5抑制剂迭代
IDEAYA Biosciences
IDE892
PRMT5抑制剂
MTAP缺失实体瘤
Phase 1
Phase 1首例患者入组（2026年3月）
IDEAYA Biosciences
IDE397 (GSK3326595)
MAT2A抑制剂
MTAP缺失肿瘤
Phase 2
与IDE892双抑制显示持久肿瘤消退
Amgen
AMG 193
MTA协同型PRMT5抑制剂
MTAP缺失肿瘤
Phase 1/2
—
Pfizer
PF-06939999
PRMT5抑制剂
实体瘤
Phase 1
第一代分子
Tango Therapeutics
TNG908
PRMT5抑制剂
MTAP缺失肿瘤
Phase 1/2
MTA协同型
常用细胞系
细胞系
MTAP状态
肿瘤类型
特征
HCT-116
MTAP缺失
结直肠癌
MTAP缺失经典模型
LN-18
MTAP缺失
胶质瘤
MTAP缺失脑肿瘤
KP-4
MTAP缺失
胰腺癌
MTAP缺失胰腺癌
MDA-MB-468
MTAP缺失
三阴性乳腺癌
MTAP缺失乳腺癌
NCI-H460
MTAP正常
NSCLC
MTAP野生型对照
A549
MTAP正常
NSCLC
MTAP野生型对照
MIA PaCa-2
MTAP缺失
胰腺癌
MTAP缺失胰腺癌
U-87 MG
MTAP正常
胶质瘤
MTAP野生型对照
TOP 11：Nectin-4靶点概述
Nectin-4是细胞黏附分子IgSF家族成员，在尿路上皮癌、乳腺癌、肺癌等多种肿瘤中高表达。2026年ASCO会议上，迈威生物9MW2821成为全球首个进入宫颈癌III期的Nectin-4 ADC。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
Seagen/Astellas
Enfortumab Vedotin (Padcev)
Nectin-4 ADC
尿路上皮癌
上市
Nectin-4 ADC开创者
迈威生物
9MW2821
Nectin-4 ADC
尿路上皮癌、宫颈癌、TNBC
Phase 3
全球首个宫颈癌III期Nectin-4 ADC；FDA快速通道
恒瑞医药
SHR-A2102
Nectin-4 ADC
肌层浸润性膀胱癌围手术期
Phase 2/3
围手术期布局
石药集团
SYS6002
Nectin-4 ADC
实体瘤
Phase 1/2
—
常用细胞系
细胞系
Nectin-4表达
肿瘤类型
特征
T24
高表达
膀胱癌
尿路上皮癌经典模型
UM-UC-3
高表达
膀胱癌
尿路上皮癌
5637
中/高表达
膀胱癌
尿路上皮癌
HT-1197
中表达
膀胱癌
尿路上皮癌
MDA-MB-468
中表达
三阴性乳腺癌
TNBC
MCF-7
低表达
乳腺癌
Nectin-4低表达对照
A549
中表达
NSCLC
肺腺癌
HeLa
中表达
宫颈癌
宫颈癌模型
TOP 12：GPC3 (Glypican-3)靶点概述
GPC3是肝细胞癌（HCC）中高表达的磷脂酰肌醇聚糖，正常肝组织几乎不表达。乐普生物MRG006A是中国首个、全球首创（FIC）的GPC3 ADC，填补全球肝癌ADC治疗空白。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
乐普生物
MRG006A
GPC3 ADC
肝细胞癌
Phase 1/2
FIC GPC3 ADC，DAR=8，Hi-TOPi平台
Codia Biosciences
Codrituzumab
GPC3单抗
肝细胞癌
Phase 1/2
—
Bayer
BAY-2763
GPC3 ADC
肝细胞癌
Phase 1
—
AnaptysBio
ANA-002
GPC3双抗
实体瘤
临床前/Phase 1
—
常用细胞系
细胞系
GPC3表达
肿瘤类型
特征
HepG2
高表达
肝癌
GPC3+肝癌经典模型
Hep3B
高表达
肝癌
GPC3+肝癌
Huh-7
中/高表达
肝癌
GPC3+肝癌
PLC/PRF/5
中表达
肝癌
GPC3+肝癌
SK-HEP-1
低/无表达
肝癌
GPC3阴性对照
SNU-475
低表达
肝癌
GPC3低表达
SMMC-7721
中表达
肝癌
国产肝癌细胞系
BEL-7402
中表达
肝癌
国产肝癌细胞系
TOP 13：HER3 (ERBB3)靶点概述
HER3是EGFR家族成员，在多种肿瘤中过表达，与治疗耐药密切相关。恒瑞医药SHR-A2009成为全球首个取得III期临床成功的HER3 ADC，有望填补全球HER3靶向疗法上市空白。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
恒瑞医药
SHR-A2009
HER3 ADC
EGFR突变NSCLC
Phase 3
全球首个III期成功HER3 ADC，预计2027年获批
第一三共
Patritumab Deruxtecan (HER3-DXd)
HER3 ADC
EGFR突变NSCLC
上市申请
已提交BLA
Eli Lilly
Seribantumab
HER3单抗
多实体瘤
Phase 2
—
百利天恒
BL-B01D1（含HER3臂）
EGFR×HER3双抗ADC
多瘤种
Phase 3
双靶点设计
常用细胞系
细胞系
HER3表达
肿瘤类型
特征
MCF-7
高表达
乳腺癌
HER3+乳腺癌
SK-BR-3
高表达
乳腺癌
HER2+/HER3+共表达
HCC827
中表达
NSCLC
EGFR突变+HER3
NCI-H1975
中表达
NSCLC
EGFR T790M+HER3
T47D
高表达
乳腺癌
管腔型HER3+
BxPC-3
中表达
胰腺癌
HER3+胰腺癌
PC-9
中表达
NSCLC
EGFR外显子19缺失
TOP 14：SEZ6靶点概述
SEZ6（癫痫发作相关蛋白6 homolog）是SCLC和神经内分泌肿瘤中的新兴ADC靶点。AbbVie的ABBV-706在Phase 3中ORR达82%，显示出卓越疗效。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
AbbVie
ABBV-706
SEZ6 ADC
复发/难治性SCLC
Phase 3
ORR 82%（1.8 mg/kg）；与PD-1联用显示鼓舞活性
AbbVie
ABBV-706 + Budigalimab
SEZ6 ADC + PD-1
SCLC
Phase 1/2
联合策略
常用细胞系
细胞系
SEZ6表达
肿瘤类型
特征
NCI-H69
高表达
SCLC
SEZ6+小细胞肺癌
NCI-H526
高表达
SCLC
SEZ6+小细胞肺癌
NCI-H446
中/高表达
SCLC
SEZ6+小细胞肺癌
NCI-H82
中表达
SCLC
SEZ6中表达
NCI-H209
高表达
SCLC
SEZ6+小细胞肺癌
DMS-114
低/无表达
NSCLC
SEZ6阴性对照
TOP 15：c-Met (MET)靶点概述
c-Met（MET）是肝细胞生长因子受体，在多种实体瘤中过表达或扩增。AbbVie的ABBV-400（Temab-A）在c-Met筛选卵巢癌中ORR达80%。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
AbbVie
ABBV-400 (Temab-A)
c-Met ADC
铂耐药卵巢癌、头颈癌
Phase 1/2
卵巢癌ORR 80%；头颈癌ORR ~31%
AbbVie
Telisotuzumab Vedotin (Teliso-V)
c-Met ADC
NSCLC
Phase 3
MET过表达NSCLC
AstraZeneca
AZD9592
EGFR×MET ADC
NSCLC
Phase 1
双靶ADC化
和黄医药
Savolitinib
MET抑制剂
NSCLC、乳头状肾癌
上市/Phase 3
MET exon14跳跃突变
常用细胞系
细胞系
MET状态
肿瘤类型
特征
EBC-1
MET扩增
NSCLC
MET扩增经典模型
MKN-45
MET扩增
胃癌
MET扩增
SNU-5
MET扩增
胃癌
MET扩增
NCI-H1996
MET过表达
NSCLC
MET+肺腺癌
U-87 MG
MET+
胶质瘤
MET+脑肿瘤
SNU-449
MET+
肝癌
MET+肝癌
A2780
MET低表达
卵巢癌
MET低表达对照
SK-OV-3
MET中表达
卵巢癌
卵巢癌模型
TOP 16：EP300/CBP靶点概述
EP300和CBP是组蛋白乙酰转移酶（HAT），调节增强子介导的转录和蛋白质稳定性。AACR 2026将其列为T4前沿潜力靶点，阿斯利康等企业布局PROTAC降解剂。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
AstraZeneca
Vepdegestrant (ARV-471)
ER PROTAC降解剂
ER+乳腺癌
Phase 3
首个进入III期的ER PROTAC
Foghorn Therapeutics
FHD-609
BRG1/BRM ATP酶降解剂
SMARCB1缺失肿瘤
Phase 1
EP300/CBP相关染色质 remodel
Arvinas/Pfizer
ARV-766
AR PROTAC
前列腺癌
Phase 1/2
PROTAC平台
Kymera/Sanofi
KT-474
IRAK4降解剂
自身免疫/肿瘤
Phase 2
PROTAC平台技术
常用细胞系
细胞系
EP300/CBP状态
肿瘤类型
特征
MCF-7
EP300野生型
乳腺癌
ER+乳腺癌经典模型
T47D
EP300野生型
乳腺癌
ER+乳腺癌
K562
EP300突变
CML
EP300突变模型
HAP1
EP300/CBP编辑
近二倍体
基因编辑模型
HEK293T
过表达系统
基因工程
过表达/报告基因系统
MDA-MB-231
EP300低表达
三阴性乳腺癌
TNBC对照
LNCaP
AR+/EP300+
前列腺癌
AR驱动模型
VCaP
AR扩增
前列腺癌
AR过表达
TOP 17：CDH17（钙黏蛋白17）靶点概述
CDH17是消化道肿瘤特异性表达的钙黏蛋白家族成员，在结直肠癌和胃癌中高表达。AACR 2026将其列为T3核心BD方向，竞争极早期，先发优势下BD价值极高。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
天广实生物
MIL-141
CDH17单抗
结直肠癌
Phase 1
国产CDH17领先管线
药明生物/多家
多个CDH17 ADC
CDH17 ADC
结直肠癌、胃癌
临床前/Phase 1
竞争极早期
Abdera Therapeutics
ABD-401
CDH17 ADC
消化道肿瘤
临床前
差异化设计
常用细胞系
细胞系
CDH17表达
肿瘤类型
特征
HT-29
高表达
结直肠癌
CDH17+结直肠癌
SW480
高表达
结直肠癌
CDH17+结直肠癌
Caco-2
高表达
结直肠癌
CDH17+结直肠癌
LS174T
中/高表达
结直肠癌
CDH17+结直肠癌
HCT-116
低表达
结直肠癌
CDH17低表达
AGS
中表达
胃癌
CDH17+胃癌
NUGC-4
中表达
胃癌
CDH17+胃癌
HEK293-CDH17
稳转
基因工程
过表达对照
TOP 18：METTL3靶点概述
METTL3是RNA m6A甲基转移酶，在多种肿瘤中高表达，促进肿瘤增殖和耐药。Storm Therapeutics的STC-15是首个进入临床的METTL3抑制剂。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
Storm Therapeutics
STC-15
METTL3抑制剂
肉瘤及多种实体瘤
Phase 1→2
Phase 1耐受良好，单药显示疗效
Insilico Medicine
ISM0417
METTL3抑制剂
实体瘤
临床前
AI发现的METTL3抑制剂
常用细胞系
细胞系
METTL3表达
肿瘤类型
特征
MOLM-13
高表达
AML
METTL3+白血病
MV4-11
高表达
AML
METTL3+白血病
THP-1
高表达
AML
METTL3+单核细胞白血病
A549
中/高表达
NSCLC
METTL3+肺腺癌
HepG2
中表达
肝癌
METTL3+肝癌
HCT-116
中表达
结直肠癌
METTL3+结直肠癌
MIA PaCa-2
中表达
胰腺癌
METTL3+胰腺癌
U2OS
中表达
骨肉瘤
肉瘤模型
TOP 19：Menin (KMT2A)靶点概述
Menin是KMT2A（MLL）重排和NPM1突变急性髓系白血病（AML）中的关键蛋白相互作用分子。Menin抑制剂通过破坏Menin-KMT2A相互作用，恢复白血病细胞分化。Syndax的Revumenib在移植后维持治疗中2年OS率达90%。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
Syndax Pharmaceuticals
Revumenib
Menin抑制剂
R/R AML（KMT2A重排/NPM1突变）
上市/维持治疗拓展
2024年获批R/R急性白血病；2年OS率90%
Kura Oncology
Ziftomenib
Menin抑制剂
R/R AML
上市申请
NDA提交
Daiichi Sankyo
DS-1594
Menin抑制剂
AML
Phase 1/2
—
Janssen
JNJ-75276617
Menin抑制剂
AML
Phase 1/2
—
常用细胞系
细胞系
KMT2A状态
肿瘤类型
特征
MOLM-13
KMT2A-MLLT3重排
AML
Menin抑制剂敏感模型
MV4-11
KMT2A-AF4重排
AML
KMT2A重排经典模型
THP-1
KMT2A-MLLT3重排
AML
单核细胞白血病
NOMO-1
KMT2A重排
AML
KMT2A重排
OCI-AML3
NPM1突变
AML
NPM1突变模型
HL-60
无KMT2A重排
AML
阴性对照
Kasumi-1
AML1-ETO
AML
阴性对照
TOP 20：WRN (Werner螺旋酶)靶点概述
WRN（Werner综合征螺旋酶）是微卫星不稳定高（MSI-H）癌症中的合成致死靶点。Broad Institute的DepMap项目获2026年AACR团队科学奖，通过CRISPR-Cas9筛选发现WRN是MSI-H癌症的关键依赖。在研公司及管线
公司
药物名称
药物类型
适应症
临床阶段
关键数据/亮点
Novartis
HRO761
WRN抑制剂
MSI-H实体瘤
Phase 1
首个进入临床的WRN抑制剂
IDEAYA Biosciences
IDE849
WRN抑制剂
MSI-H实体瘤
临床前/Phase 1
与GSK合作开发
Roche
未公开
WRN抑制剂
MSI-H实体瘤
临床前
早期开发
Vividion Therapeutics
未公开
WRN抑制剂
MSI-H实体瘤
临床前
共价抑制剂平台
常用细胞系
细胞系
MSI状态
肿瘤类型
特征
HCT-116
MSI-H
结直肠癌
MSI-H经典模型，WRN依赖
RKO
MSI-H
结直肠癌
MSI-H结直肠癌
HCT-15
MSI-H
结直肠癌
MSI-H结直肠癌
LS174T
MSI-H
结直肠癌
MSI-H结直肠癌
LOVO
MSI-H
结直肠癌
MSI-H结直肠癌
SW48
MSI-H
结直肠癌
MSI-H结直肠癌
DLD-1
MSI-H
结直肠癌
MSI-H结直肠癌
HT-29
MSS
结直肠癌
MSS对照（WRN非依赖）
SW480
MSS
结直肠癌
MSS对照
HCT-8
MSS
结直肠癌
MSS对照
四、TOP 20靶点常用细胞系汇总表
排名
靶点
常用细胞系（核心模型）
常用细胞系（对照/验证模型）
1
KRAS/RAS
MIA PaCa-2, Panc-1, NCI-H358, NCI-H23, HCT-116, AsPC-1
A549 (G12S), NCI-H460 (WT), BEAS-2B (正常肺上皮)
2
HER2
SK-BR-3, BT-474, NCI-N87, HCC1954, SK-OV-3, Calu-3
MCF-7 (HER2低), MDA-MB-231 (HER2阴性)
3
TROP2
MDA-MB-468, HCC1806, HCC38, BT-549, A431
MCF-7 (TROP2低), T47D (中表达)
4
B7-H3
A204, NCI-H526, NCI-H446, RD-ES, SK-N-DZ
MCF-7 (低表达), HEK293 (对照)
5
CLDN18.2
AGS, NUGC-4, KATO III, SNU-216
SNU-1 (阴性), HEK293-CLDN18.2 (稳转)
6
EGFR×HER3
HCC827, NCI-H1975, FaDu, MDA-MB-468, BxPC-3
MCF-7 (HER3+), A431 (EGFR高)
7
PD-1/VEGF
MC-38, CT26, B16-F10, 4T1 (小鼠体内)
A549, NCI-H1975, HUVEC (体外)
8
DLL3
NCI-H446, NCI-H526, NCI-H69, NCI-H209
DMS-114 (阴性), DMS-153 (中表达)
9
EGFR×MET
HCC827, NCI-H1975, EBC-1, MKN-45, SNU-5
Calu-3 (双靶共表达), FaDu (EGFR+)
10
PRMT5
HCT-116, LN-18, KP-4, MDA-MB-468, MIA PaCa-2
A549 (MTAP WT), NCI-H460 (MTAP WT)
11
Nectin-4
T24, UM-UC-3, 5637, MDA-MB-468, HeLa
MCF-7 (低表达), A549 (中表达)
12
GPC3
HepG2, Hep3B, Huh-7, PLC/PRF/5
SK-HEP-1 (阴性), SNU-475 (低表达)
13
HER3
MCF-7, SK-BR-3, HCC827, NCI-H1975, T47D
MDA-MB-231 (HER3低), PC-9
14
SEZ6
NCI-H69, NCI-H526, NCI-H446, NCI-H209
DMS-114 (阴性)
15
c-Met
EBC-1, MKN-45, SNU-5, NCI-H1996, U-87 MG
A2780 (低表达), SK-OV-3 (中表达)
16
EP300/CBP
MCF-7, T47D, LNCaP, VCaP
K562 (EP300突变), MDA-MB-231 (低表达)
17
CDH17
HT-29, SW480, Caco-2, LS174T
HCT-116 (低表达), HEK293-CDH17 (稳转)
18
METTL3
MOLM-13, MV4-11, THP-1, A549, HepG2
U2OS (肉瘤), HCT-116
19
Menin (KMT2A)
MOLM-13, MV4-11, THP-1, NOMO-1, OCI-AML3
HL-60 (阴性), Kasumi-1 (阴性)
20
WRN
HCT-116, RKO, HCT-15, LS174T, LOVO, SW48
HT-29 (MSS), SW480 (MSS), HCT-8 (MSS)
五、关键趋势分析与展望5.1 模态创新趋势
模态类型
AACR 2026摘要数
核心特征
代表靶点
ADC
355篇
最热赛道；双payload、双抗ADC、新毒素
HER2, TROP2, B7-H3, CLDN18.2, Nectin-4
PROTAC/分子胶
178篇
正式进入III期时代
EP300/CBP, ER, AR
Pan-RAS分子胶
37篇
本届最高频新药类别
KRAS (Pan-RAS)
双特异性抗体
33项临床
多靶点组合探索
PD-1/VEGF, EGFR×MET, EGFR×HER3
CAR-T
13项临床
向实体瘤突破
DLL3, CLDN18.2, BCMA
5.2 靶点竞争梯队（基于赛业生物AACR 2026分析）
梯队
竞争状态
代表靶点
战略意义
T1（红海）
高度饱和
PD-1/PD-L1
已成红海，联合方案为主
T2（竞争激烈但仍有空间）
持续创新
KRAS, TROP2, HER2
后发创新仍有空间
T3（核心BD方向）
竞争尚未固化
B7-H3, CLDN18.2, CDH17
当前BD布局重点
T4（前沿潜力）
早期阶段
EP300/CBP, PRMT5, WRN
长期突破潜力
5.3 中国创新药全球化趋势
2026年AACR中国以866篇研究摘要位列全球第二（仅次于美国），ASCO中国学者报告95项创历史新高。标志性BD交易包括：
交易
金额
靶点/药物
意义
百利天恒 → BMS
84亿美元
EGFR×HER3 ADC (BL-B01D1)
当年中国出海最高纪录
科伦博泰 → 默沙东
14.1亿美元
TROP2 ADC (sac-TMT)
TROP2 ADC全球化
三生制药 → 辉瑞
12.5亿美元首付款
PD-1/VEGF双抗 (SSGJ-707)
双抗出海标杆
5.4 细胞系选择建议
针对不同靶点的药效学实验，建议遵循以下原则：
6.靶点表达验证先行：使用流式细胞术或Western blot确认细胞系靶点表达水平
7.多细胞系组合策略：同时使用高表达、中表达和阴性对照细胞系
8.体内模型配套：细胞系验证后配套CDX模型（异种移植瘤）进行体内药效验证
9.耐药模型构建：对于KRAS、EGFR等易耐药靶点，构建耐药细胞系进行耐药机制研究
10.基因工程细胞系：对于内源性表达低的靶点（如CLDN18.2），使用稳定转染细胞系
附录：数据来源与参考文献
11.AACR Annual Meeting 2026, April 17-22, 2026, San Diego, CA. (https://www.aacr.org/meeting/aacr-annual-meeting-2026/)
12.ASCO Annual Meeting 2026, May 29 - June 2, 2026, Chicago, IL. (https://www.asco.org/annual-meeting)
13.赛业生物. AACR 2026研究报告：7562篇摘要浓缩. 2026年4月29日. (https://www.cyagen.cn/articles/aacr2026-cancer-research-report)
14.缔码生物. AACR 2026热门ADC靶点分析. 2026年4月. (https://www.dimabio.cn/blog/popular-adc-targets-at-aacr-2026)
15.NovaPharmaNews. AACR 2026: ctDNA, KRAS Inhibitors, CAR-T Breakthroughs. 2026年4月23日.
16.Labiotech.eu. Highlights of ASCO 2026: how is cancer care evolving? 2026年6月3日.
17.VCBeat Health. ASCO 2026: Chinese ADCs make a powerful statement. 2026年6月2日.
18.ChinaMed Global. ASCO 2026中国创新药临床数据盘点. 2026年5月23日.
19.PR Newswire. B7-H3 Becomes The Hottest Antigen In Oncology. 2026年5月18日.
20.Broad Institute DepMap Project. (https://sites.broadinstitute.org/ccle)
21.ClinicalTrials.gov及各企业官方公开信息.
免责声明：本报告基于2026年AACR/ASCO等国际会议的公开摘要及行业分析整理，所提及的管线数据、临床数据均来源于会议摘要或企业公开信息，未经完整临床试验验证。报告内容不构成临床用药或投资建议。如需做出研发或投资决策，建议查阅原始会议摘要及最新临床试验数据。
整理日期：2026年6月27日
2026-05-12
SPRING HOUSE, Pa., (May 12, 2026) – Johnson & Johnson (NYSE: JNJ) announced today more than 30 hematology abstracts featuring data in multiple myeloma and leukemia, as well as in warm autoimmune hemolytic anemia (wAIHA), will be shared at the European Hematology Association (EHA) 2026 Congress from June 11-14 in Stockholm, Sweden. Among the results to be presented in a plenary session is the Phase 3 MonumenTAL-3 study investigating TALVEY® (talquetamab-tgvs), a GPRC5D bispecific antibody, in combination with DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) with or without pomalidomide in relapsed/refractory multiple myeloma. New pivotal data from the Phase 2/3 ENERGY study evaluating IMAAVY® (nipocalimab-aahu)a in wAIHA, a rare autoantibody-driven disease in which autoantibodies attach to and destroy red blood cells, will also be presented in an oral session.1 The U.S. Food and Drug Administration (FDA) recently granted Priority Review to IMAAVY ® in wAIHA.
Perspectives on key data readouts at EHA“The data we are sharing at EHA 2026 reflect the depth and breadth of our clinical innovations in blood cancer, led by the Phase 3 MonumenTAL-3 results, which are an exciting next step in exploring how we can continue to expand our portfolio of differentiated and combinable therapies in multiple myeloma,” said Yusri Elsayed, M.D., M.H.Sc., Ph.D., Global Therapeutic Area Head, Oncology, Johnson & Johnson. “Together with additional data in leukemia, these findings underscore our leadership in hematology and our continued commitment to advancing meaningful innovation for patients living with complex blood cancers.”
“wAIHA is marked by debilitating fatigue and serious complications of severe anemia. There is an unmet need for an approved treatment for wAIHA that may provide rapid control and sustained relief from symptoms while preserving critical immune functions,” said David Lee, M.D., Ph.D., Global Immunology Therapeutic Area Head, Johnson & Johnson. “The first presentation of Phase 2/3 results from the ENERGY study highlights the potential of an immunoselective treatment approach for wAIHA, which is a rare disease with no U.S. FDA-approved treatments.”
Updated data in multiple myelomaData at EHA will highlight advances in multiple myeloma treatment, including combination regimens and emerging therapies for patients with newly diagnosed and relapsed/refractory multiple myeloma.
New results from the Phase 3 MonumenTAL-3 study evaluating TALVEY® in combination with DARZALEX FASPRO® with or without pomalidomide versus DARZALEX FASPRO®, pomalidomide, and dexamethasone in relapsed/refractory multiple myeloma (Presented as “One of the six best abstracts” during the Plenary Abstracts Session #S100) Subgroup analysis based on cytogenetic and functional risk from the MajesTEC-3 study evaluating TECVAYLI® (teclistamab-cqyv) in combination with DARZALEX FASPRO® in relapsed/refractory multiple myeloma (Oral Abstract #S198) Cytogenetic subgroup analysis from the Phase 3 CARTITUDE-4 study evaluating CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) in patients with lenalidomide-refractory multiple myeloma responding to bridging therapy (Poster Abstract #PF767) [Encore of ASCO 2026] Subgroup analysis from the Phase 3 PERSEUS study evaluating the benefit of DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) in transplant-eligible patients with high- and standard-risk multiple myeloma (Poster Abstract #PS1869) [Encore of ASCO 2026] Final subgroup analysis from the Phase 3 CEPHEUS study evaluating DARZALEX FASPRO® in combination with bortezomib, lenalidomide, and dexamethasone in transplant-ineligible newly diagnosed multiple myeloma (Oral Abstract #S196) [Encore of ASCO 2026] Results from the Phase 3 MajesTEC-9 study of TECVAYLI® versus standard of care therapies as early as second line in patients with multiple myeloma (Oral Abstract #S195) [Encore of ASCO 2026]
New research in warm autoimmune hemolytic anemia (wAIHA)Pivotal study assessed hemoglobin response and fatigue improvement compared to placebo in patients with warm autoimmune hemolytic anemia.
Results from the pivotal ENERGY Phase 2/3 study evaluating the efficacy and safety of IMAAVY®in patients with wAIHA compared with placebo (Oral Abstract #S300) Assessment of the effects of IMAAVY®on fatigue in patients with wAIHA, measured using the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) scale (Oral Abstract #S325) Confirmatory evidence on the pharmacodynamic effect of IMAAVY®and the correlation with clinical improvement in patients with wAIHA (Poster Abstract #PF1297)
Key data presentations in leukemiaNew and updated data highlight clinical advances in leukemia, including acute myeloid leukemia (AML) and chronic lymphocytic leukemia (CLL), across targeted therapies and fixed-duration treatment approaches.
Safety analyses from the cAMelot-1 and ALE1002 clinical trials of bleximenib (JNJ-75276617), an investigational oral menin inhibitor, evaluating the presence of QTc interval prolongation when given as monotherapy or in combination with AML-directed therapies for KMT2A or NPM1 Altered AML (Poster Abstract #PF555) Primary analysis from the Phase 2 TAILOR study evaluating the safety and efficacy of dose-modified fixed-duration IMBRUVICA®(ibrutinib) plus venetoclax in previously untreated CLL (Poster Abstract #PF606) Real-world analysis from the prospective REALITY-WW study evaluating first-line fixed-duration IMBRUVICA® in combination with venetoclax in patients with CLL (Poster Abstract #PF613)
Information on Johnson & Johnson sponsored abstracts is available on JNJ.com.
Editor’s Notesa. IMAAVY® is not approved by the U.S. FDA for the treatment of wAIHAABOUT MULTIPLE MYELOMA Multiple myeloma is a complex blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.2 In multiple myeloma, these plasma cells proliferate and spread rapidly and replace normal cells in the bone marrow with tumors.3 Multiple myeloma is the third most common blood cancer worldwide.4 More than 180,000 new cases of multiple myeloma are diagnosed globally each year.5 People living with multiple myeloma have a 5-year survival rate of 59.8 percent.6 While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels and kidney problems or infections.7,8 In recent years, overall survival has improved from years to decades, with effective treatment options now available across every stage and line of therapy.9
ABOUT WARM AUTOIMMUNE HEMOLYTIC ANEMIAWarm autoimmune hemolytic anemia (wAIHA) is a rare, life-threatening condition where autoantibodies attach to and destroy red blood cells (RBCs), resulting in anemia. Approximately 1-3 new people per 100,000 are affected by wAIHA per year, and about 1 in 8,000 individuals are living with the condition.10,11 This condition affects both women and men, and can affect people at any age with incidence increasing over the age of 50.12,13 Additionally, people with wAIHA are at increased risk of other serious complications such as venous thrombotic events, acute renal failure, and infection.14 There are no U.S. FDA-approved drugs indicated for wAIHA, and treatment typically consists of unapproved corticosteroids, broad immunosuppressants, and B-cell directed therapies.11 With an unmet need for treatment in wAIHA, novel therapies like nipocalimab are being developed to potentially address this need.13
ABOUT ACUTE MYELOID LEUKEMIA Acute myeloid leukemia (AML) is an aggressive, fast-growing blood cancer that originates in the bone marrow and is marked by the uncontrolled proliferation of immature white blood cells known as myeloblasts.15,16 These malignant cells crowd out healthy blood-forming cells, leading to complications such as anemia, infections and bleeding.17 Acute myeloid leukemia progresses rapidly, often requiring immediate treatment after diagnosis.16 It is the most common type of acute leukemia in adults, with a median age of diagnosis around 70 years.15 Despite treatment advances, acute myeloid leukemia remains associated with poor patient outcomes, particularly in older adults or those with high-risk genetic profiles.18 The five-year survival rate remains the lowest among leukemias, with outcomes especially poor in patients with KMT2Ar or NPM1m where relapse/refractory disease survival can be as short as 2 to 3 months after a second relapse – highlighting a significant unmet medical need.18
ABOUT TALVEY® TALVEY® (talquetamab-tgvs) received approval from the U.S. FDA in August 2023 as a first-in-class GPRC5D-targeting bispecific antibody for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.19 Since U.S. FDA approval, 7,700 patients were treated with TALVEY®. The European Commission (EC) granted conditional marketing authorization (CMA) of TALVEY®(talquetamab-tgvs) in August 2023 as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma (RRMM) who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.20
TALVEY® is a bispecific T-cell engaging antibody that binds to the CD3 receptor expressed on the surface of T-cells and G protein-coupled receptor class C group 5 member D (GPRC5D), a novel multiple myeloma target which is highly expressed on the surface of multiple myeloma cells and non-malignant plasma cells, as well as some healthy tissues such as epithelial cells of the skin and tongue.
ABOUT DARZALEX FASPRO® AND DARZALEX® DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) received U.S. FDA approval in May 2020 and is approved for 11 indications in multiple myeloma, four of which are for frontline treatment in newly diagnosed patients who are transplant eligible or ineligible.21 It is the only subcutaneous CD38-directed antibody approved to treat patients with multiple myeloma. DARZALEX FASPRO® is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology.
DARZALEX® (daratumumab) received U.S. FDA approval in November 2015 and is approved in eight indications, three of which are in the frontline setting, including newly diagnosed patients who are transplant-eligible and ineligible.22 In 2025, DARZALEX FASPRO® was approved by the U.S. FDA and EMA as the first and only treatment for patients with high-risk smoldering multiple myeloma.
DARZALEX® is the first CD38-directed antibody approved to treat multiple myeloma.22 DARZALEX®-based regimens have been used in the treatment of more than 748,000 patients worldwide and more than 68,000 patients in the U.S. alone.
In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive license to develop, manufacture and commercialize daratumumab. For more information, visit www.DARZALEX.com. ABOUT IMAAVY®IMAAVY® (nipocalimab-aahu) is an immunoselective treatment designed to target, bind with high affinity, and block FcRn, reducing circulating immunoglobulin G (IgG) antibodies that drive disease while also preserving key immune functions. IMAAVY® is currently approved for the treatment of generalized Myasthenia Gravis (gMG) in adults and pediatric patients 12 years of age and older who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.23
Nipocalimab is being investigated across three key segments in the autoantibody space including Rheumatologic diseases, Rare Autoantibody diseases, and Maternal Fetal diseases mediated by maternal alloantibodies, in which blockade of IgG binding to FcRn in the placenta is believed to limit transplacental transfer of maternal alloantibodies to the fetus.24,25,26,27,28,29,30,31,32,33
The U.S. FDA and European Medicines Agency (EMA) have granted several key designations to nipocalimab including:
EU EMA Orphan medicinal product designation for HDFN in October 2019 and fetal and neonatal alloimmune thrombocytopenia (FNAIT in April 2025 U.S. FDA Fast Track designation in hemolytic disease of the fetus and newborn (HDFN) and warm autoimmune hemolytic anemia (wAIHA) in July 2019, gMG in December 2021, fetal and neonatal alloimmune thrombocytopenia (FNAIT) in March 2024, Sjögren’s disease (SjD) in March 2025, and systemic lupus erythematosus (SLE) in January 2026 U.S. FDA Orphan drug status for wAIHA in December 2019, HDFN in June 2020, gMG in February 2021, chronic inflammatory demyelinating polyneuropathy (CIDP) in October 2021 and FNAIT in December 2023 U.S. FDA Breakthrough Therapy designation for HDFN in February 2024 and for SjD in November 2024 U.S. FDA granted Priority Review in gMG in Q4 2024 and wAIHA in Q2 2026
The legal manufacturer for IMAAVY ® is Janssen Biotech, Inc.
ABOUT TECVAYLI®TECVAYLI® (teclistamab-cqyv) is a first-in-class, bispecific T-cell engager antibody therapy that uses innovative science to activate the immune system by binding to the CD3 receptor expressed on the surface of T-cells and to the B-cell maturation antigen (BCMA) expressed on the surface of multiple myeloma cells and some healthy B-lineage cells.34 TECVAYLI® received accelerated approval from the U.S. FDA in October 2022 as an off-the-shelf (or ready-to-use) antibody that is administered as a subcutaneous treatment for adult patients with relapsed or refractory multiple myeloma (RRMM) who received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.
In February 2024, the U.S. FDA approved the supplemental Biologics License Application (sBLA) for TECVAYLI® for a reduced dosing frequency of 1.5 mg/kg every two weeks in patients with RRMM who achieved and maintained a complete response (CR) or better for a minimum of six months.
In March 2026, the U.S. FDA approved TECVAYLI® in combination with DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent. The supplemental Biologics License Application was proactively selected for the Commissioner’s National Priority Voucher Pilot Program and also granted the application Breakthrough Therapy Designation and Real-Time Oncology Review. This approval expanded the use of TECVAYLI® into earlier lines of therapy and is the first bispecific antibody-based combination regimen in this setting, offering a potential new standard of care as early as second line.
To date, more than 26,000 patients have been treated worldwide with TECVAYLI®.
The European Commission (EC) granted TECVAYLI® conditional marketing authorization in August 2022 as monotherapy for the treatment of adult patients with RRMM who have received at least three prior therapies, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody, and have demonstrated disease progression since the last therapy. In August 2023, the EC approved a Type II variation application for TECVAYLI®, providing the option for a reduced dosing frequency of 1.5 mg/kg every two weeks (Q2W) in patients who have achieved a complete response or better for a minimum of six months.
For more information, visit www.TECVAYLI.com.
ABOUT CARVYKTI® CARVYKTI® (cilta-cel) received U.S. Food and Drug Administration approval in February 2022 for the treatment of adults with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.35
In April 2024, CARVYKTI® was approved in the U.S. for treatment of adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy including a proteasome inhibitor, an immunomodulatory agent, and who are refractory to lenalidomide, following a unanimous (11 to 0) U.S. FDA Oncologic Drugs Advisory Committee (ODAC) recommendation in support of this new indication. In April 2024, the European Medicines Agency (EMA) approved a Type II variation for CARVYKTI® for the treatment of adults with relapsed and refractory multiple myeloma who have received at least one prior therapy, including an immunomodulatory agent and a proteasome inhibitor, have demonstrated disease progression on the last therapy, and are refractory to lenalidomide. In September 2022, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved CARVYKTI® for the treatment of adults with relapsed or refractory multiple myeloma in patients that have no history of CAR-positive T cell infusion therapy targeting BCMA and 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.
CARVYKTI® is a BCMA-directed, autologous T-cell immunotherapy, which involves reprogramming a patient’s own T-cells with a transgene encoding chimeric antigen receptor (CAR) that directs the CAR-positive T cells to eliminate cells that express BCMA. BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B cells and plasma cells. The CARVYKTI® CAR protein features two BCMA-targeting single domains designed to confer high avidity against human BCMA. Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells. CARVYKTI® is available in 17 markets worldwide and has been used to treat more than 11,000 patients globally.
In December 2017, Janssen Biotech, Inc., a Johnson & Johnson company, entered into an exclusive worldwide license and collaboration agreement with Legend Biotech USA, Inc. to develop and commercialize CARVYKTI®. For more information, visit www.CARVYKTI.com.
ABOUT IMBRUVICA®IMBRUVICA® (ibrutinib) is a once-daily oral medication that is jointly developed and commercialized by Janssen Biotech, Inc., and Pharmacyclics LLC, an AbbVie company. IMBRUVICA® blocks the BTK protein, which is needed by normal and abnormal B cells, including specific cancer cells, to multiply and spread. By blocking BTK, IMBRUVICA® may help move abnormal B cells out of their nourishing environments and inhibit their proliferation.36,37,38
IMBRUVICA® has been used to treat more than 325,000 patients worldwide over the last decade. There are more 70 company-sponsored clinical trials, including 18 Phase 3 studies, spanning more than 13 years evaluating the efficacy and safety of IMBRUVICA®.
IMBRUVICA® was first approved by the U.S. FDA in November 2013, and today is indicated for adult patients in four disease areas. To date, IMBRUVICA® has been approved in more than 100 countries/territories for one or more of the following indications: the treatment of adult patients with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) with or without 17p deletion (del17p); adults with Waldenström’s macroglobulinemia (WM); adults with mantle cell lymphoma (MCL); adults with marginal zone lymphoma (MZL) who require systemic therapy and have received at least one prior anti-CD20-based therapy; and adult and pediatric patients aged one year and older with previously treated chronic graft versus host disease (cGVHD) after failure of one or more lines of systemic therapy.39
TALVEY® IMPORTANT SAFETY INFORMATION
INDICATION AND USAGE
TALVEY® (talquetamab-tgvs) is indicated for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.
This indication is approved under accelerated approval based on response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
IMPORTANT SAFETY INFORMATION
WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGIC TOXICITY, including IMMUNE EFFECTOR CELL-ASSOCIATED NEUROTOXICITY SYNDROME
Cytokine release syndrome (CRS), including life-threatening or fatal reactions, can occur in patients receiving TALVEY®. Initiate TALVEY® treatment with step-up dosing to reduce the risk of CRS. Withhold TALVEY® until CRS resolves or permanently discontinue based on severity.
Neurologic toxicity, including immune effector cell-associated neurotoxicity syndrome (ICANS), and serious and life-threatening or fatal reactions, can occur with TALVEY®. Monitor patients for signs and symptoms of neurologic toxicity including ICANS during treatment and treat promptly. Withhold or permanently discontinue TALVEY® based on severity.
Because of the risk of CRS and neurologic toxicity, including ICANS, TALVEY® is available only through a restricted program called the TECVAYLI® and TALVEY® Risk Evaluation and Mitigation Strategy (REMS).
CONTRAINDICATIONS: None.
WARNINGS AND PRECAUTIONS Cytokine Release Syndrome (CRS): TALVEY® can cause cytokine release syndrome, including life-threatening or fatal reactions. In the clinical trial, CRS occurred in 76% of patients who received TALVEY® at the recommended dosages, with Grade 1 CRS occurring in 57% of patients, Grade 2 in 17%, and Grade 3 in 1.5%. Recurrent CRS occurred in 30% of patients. Most events occurred following step-up dose 1 (29%) or step-up dose 2(44%) at the recommended dosages. CRS occurred in 33% of patients with step-up dose 3 in the biweekly dosing schedule (N=153). CRS occurred in 30% of patients with the first 0.4 mg/kg treatment dose and in 12% of patients treated with the first 0.8 mg/kg treatment dose. The CRS rate for both dosing schedules combined was less than 3% for each of the remaining doses in Cycle 1 and less than 3% cumulatively from Cycle 2 onward. The median time to onset of CRS was 27 (range: 0.1 to 167) hours from the last dose, and the median duration was 17 (range: 0 to 622) hours. Clinical signs and symptoms of CRS include but are not limited to pyrexia, hypotension, chills, hypoxia, headache, and tachycardia. Potentially life-threatening complications of CRS may include cardiac dysfunction, acute respiratory distress syndrome, neurologic toxicity, renal and/or hepatic failure, and disseminated intravascular coagulation (DIC). Initiate therapy with step-up dosing and administer pre-treatment medications (corticosteroids, antihistamine, and antipyretics) prior to each dose of TALVEY® in the step-up dosing schedule to reduce the risk of CRS. Monitor patients following administration accordingly. In patients who experience CRS, pre-treatment medications should be administered prior to the next TALVEY® dose.
Counsel patients to seek medical attention should signs or symptoms of CRS occur. At the first sign of CRS, immediately evaluate patient for hospitalization and institute treatment with supportive care based on severity, and consider further management per current practice guidelines. Withhold TALVEY® until CRS resolves or permanently discontinue based on severity.
Neurologic Toxicity including ICANS: TALVEY® can cause serious, or life-threatening neurologic toxicity or fatal neurologic toxicity including immune effector cell-associated neurotoxicity syndrome (ICANS), including fatal reactions. In the clinical trial, neurologic toxicity, including ICANS, occurred in 55% of patients who received the recommended dosages, with Grade 3 or 4 neurologic toxicity occurring in 6% of patients. The most frequent neurologic toxicities were headache (20%), encephalopathy (15%), sensory neuropathy (14%), and motor dysfunction (10%).
ICANS was reported in 9% of 265 patients where ICANS was collected and who received the recommended dosages. Recurrent ICANS occurred in 3% of patients. Most patients experienced ICANS following step-up dose 1 (3%), step-up dose 2 (3%), step-up dose 3 of the biweekly dosing schedule (1.8%), or the initial treatment dose of the weekly dosing schedule (2.6%) (N=156) or the biweekly dosing schedule (3.7%) (N=109). The median time to onset of ICANS was 2.5 (range: 1 to 16) days after the most recent dose with a median duration of 2 (range: 1 to 22) days. The onset of ICANS can be concurrent with CRS, following resolution of CRS, or in the absence of CRS. Clinical signs and symptoms of ICANS may include but are not limited to confusional state, depressed level of consciousness, disorientation, somnolence, lethargy, and bradyphrenia.
Monitor patients for signs and symptoms of neurologic toxicity during treatment and treat promptly. At the first sign of neurologic toxicity, including ICANS, immediately evaluate the patient and provide supportive care based on severity. Withhold or permanently discontinue TALVEY® based on severity and consider further management per current practice guidelines [see Dosage and Administration (2.5)].
Due to the potential for neurologic toxicity, patients receiving TALVEY® are at risk of depressed level of consciousness. Advise patients to refrain from driving or operating heavy or potentially dangerous machinery during the step-up dosing schedule and for 48 hours after completion of the step-up dosing schedule, and in the event of new onset of any neurological symptoms, until symptoms resolve.
TECVAYLI® and TALVEY® REMS: TALVEY® is available only through a restricted program under a REMS, called the TECVAYLI® and TALVEY® REMS because of the risks of CRS and neurologic toxicity, including ICANS. Further information about the TECVAYLI® and TALVEY® REMS program is available at www.TEC-TALREMS.com or by telephone at 1-855-810-8064.
Oral Toxicity and Weight Loss: TALVEY® can cause oral toxicities, including dysgeusia, dry mouth, dysphagia, and stomatitis. In the clinical trial, 80% of patients had oral toxicity, with Grade 3 occurring in 2.1% of patients who received the recommended dosages. The most frequent oral toxicities were dysgeusia (49%), dry mouth (34%), dysphagia (23%), and ageusia (18%). The median time to onset of oral toxicity was 15 (range: 1 to 634) days, and the median time to resolution to baseline was 43 (1 to 530) days. Oral toxicity did not resolve to baseline in 65% of patients.
TALVEY® can cause weight loss. In the clinical trial, 62% of patients experienced weight loss, regardless of having an oral toxicity, including 29% of patients with Grade 2 (10% or greater) weight loss and 2.7% of patients with Grade 3 (20% or greater) weight loss. The median time to onset of Grade 2 or higher weight loss was 67 (range: 6 to 407) days, and the median time to resolution was 50 (range: 1 to 403) days. Weight loss did not resolve in 57% of patients who reported weight loss.
Monitor patients for signs and symptoms of oral toxicity. Counsel patients to seek medical attention should signs or symptoms of oral toxicity occur and provide supportive care as per current clinical practice, including consultation with a nutritionist. Monitor weight regularly during therapy. Evaluate clinically significant weight loss further. Withhold TALVEY® or permanently discontinue based on severity.
Infections: TALVEY® can cause infections, including life-threatening or fatal infections. Serious infections occurred in 16% of patients, with fatal infections in 1.5% of patients. Grade 3 or 4 infections occurred in 17% of patients. The most common serious infections reported were bacterial infection (8%), which included sepsis and COVID-19 (2.7%). Monitor patients for signs and symptoms of infection prior to and during treatment with TALVEY® and treat appropriately. Administer prophylactic antimicrobials according to local guidelines. Withhold or consider permanent discontinuation of TALVEY® as recommended, based on severity.
Cytopenias: TALVEY® can cause cytopenias, including neutropenia and thrombocytopenia. In the clinical trial, Grade 3 or 4 decreased neutrophils occurred in 35% of patients, and Grade 3 or 4 decreased platelets occurred in 22% of patients who received TALVEY®. The median time to onset for Grade 3 or 4 neutropenia was 22 (range: 1 to 312) days, and the median time to resolution to Grade 2 or lower was 8 (range: 1 to 79) days. The median time to onset for Grade 3 or 4 thrombocytopenia was 12 (range: 2 to 183) days, and the median time to resolution to Grade 2 or lower was 10 (range: 1 to 64) days. Monitor complete blood counts during treatment and withhold TALVEY® as recommended, based on severity.
Skin Toxicity: TALVEY® can cause serious skin reactions, including rash, maculo-papular rash, erythema, and erythematous rash. In the clinical trial, skin reactions occurred in 62% of patients, with Grade 3 skin reactions in 0.3%. The median time to onset was 25 (range: 1 to 630) days. The median time to improvement to Grade 1 or less was 33 days.
Monitor for skin toxicity, including rash progression. Consider early intervention and treatment to manage skin toxicity. Withhold TALVEY® as recommended based on severity.
Hepatotoxicity: TALVEY® can cause hepatotoxicity. Elevated ALT occurred in 33% of patients, with Grade 3 or 4 ALT elevation occurring in 2.7%; elevated AST occurred in 31% of patients, with Grade 3 or 4 AST elevation occurring in 3.3%. Grade 3 or 4 elevations of total bilirubin occurred in 0.3% of patients. Liver enzyme elevation can occur with or without concurrent CRS.
Monitor liver enzymes and bilirubin at baseline and during treatment as clinically indicated. Withhold TALVEY® or consider permanent discontinuation of TALVEY®, based on severity [see Dosage and Administration (2.5)].
Embryo-Fetal Toxicity: Based on its mechanism of action, TALVEY® may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with TALVEY® and for 3 months after the last dose.
Adverse Reactions: The most common adverse reactions (≥20%) are pyrexia, CRS, dysgeusia, nail disorder, musculoskeletal pain, skin disorder, rash, fatigue, weight decreased, dry mouth, xerosis, dysphagia, upper respiratory tract infection, diarrhea, hypotension, and headache.
The most common Grade 3 or 4 laboratory abnormalities (≥30%) are lymphocyte count decreased, neutrophil count decreased, white blood cell decreased, and hemoglobin decreased.
Please read full Prescribing Information, including Boxed WARNING, for TALVEY®.
DARZALEX FASPRO® INDICATIONS AND IMPORTANT SAFETY INFORMATION INDICATIONS DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) is indicated for the treatment of adult patients with multiple myeloma: · In combination with bortezomib, lenalidomide, and dexamethasone for induction and consolidation in newly diagnosed patients who are eligible for autologous stem cell transplant · In combination with bortezomib, melphalan, and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant · In combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy · In combination with bortezomib, thalidomide, and dexamethasone in newly diagnosed patients who are eligible for autologous stem cell transplant · In combination with pomalidomide and dexamethasone in patients who have received at least one prior line of therapy including lenalidomide and a proteasome inhibitor (PI) · In combination with carfilzomib and dexamethasone in patients with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy · In combination with bortezomib and dexamethasone in patients who have received at least one prior therapy · As monotherapy in patients who have received at least three prior lines of therapy including a PI and an immunomodulatory agent or who are double refractory to a PI and an immunomodulatory agent
DARZALEX FASPRO® as monotherapy is indicated for the treatment of adult patients with high-risk smoldering multiple myeloma.
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS DARZALEX FASPRO® is contraindicated in patients with a history of severe hypersensitivity to daratumumab, hyaluronidase, or any of the components of the formulation.
WARNINGS AND PRECAUTIONS
Hypersensitivity and Other Administration Reactions Both systemic administration-related reactions, including severe or life-threatening reactions, and local injection-site reactions can occur with DARZALEX FASPRO®. Fatal reactions have been reported with daratumumab-containing products, including DARZALEX FASPRO®.
Systemic Reactions
In a pooled safety population of 1446 patients with multiple myeloma (N=1235) or light chain (AL) amyloidosis (N=193) who received DARZALEX FASPRO® as monotherapy or in combination, 7% of patients experienced a systemic administration-related reaction (Grade 2: 3%, Grade 3: 0.8%, Grade 4: 0.1%). In patients with high-risk smoldering multiple myeloma (N=193), systemic administration-related reactions occurred in 17% of patients in AQUILA (Grade 2: 7%, Grade 3: 1%).
In all patients (N=1639), systemic administration-related reactions occurred in 7% of patients with the first injection, 0.5% with the second injection, and cumulatively 1% with subsequent injections. The median time to onset was 3.2 hours (range: 4 minutes to 3.5 days). Of the 283 systemic administration-related reactions that occurred in 135 patients, 240 (85%) occurred on the day of DARZALEX FASPRO® administration. Delayed systemic administration-related reactions have occurred in 1% of the patients.
Severe reactions included hypoxia, dyspnea, hypertension, tachycardia, and ocular adverse reactions, including choroidal effusion, acute myopia, and acute angle closure glaucoma. Other signs and symptoms of systemic administration-related reactions may include respiratory symptoms, such as bronchospasm, nasal congestion, cough, throat irritation, allergic rhinitis, and wheezing, as well as anaphylactic reaction, pyrexia, chest pain, pruritus, chills, vomiting, nausea, hypotension, and blurred vision. Pre-medicate patients with histamine-1 receptor antagonist, acetaminophen, and corticosteroids. Monitor patients for systemic administration-related reactions, especially following the first and second injections. For anaphylactic reaction or life-threatening (Grade 4) administration-related reactions, immediately and permanently discontinue DARZALEX FASPRO®. Consider administering corticosteroids and other medications after the administration of DARZALEX FASPRO® depending on dosing regimen and medical history to minimize the risk of delayed (defined as occurring the day after administration) systemic administration-related reactions.
Ocular adverse reactions, including acute myopia and narrowing of the anterior chamber angle due to ciliochoroidal effusions with potential for increased intraocular pressure or glaucoma, have occurred with daratumumab-containing products. If ocular symptoms occur, interrupt DARZALEX FASPRO® and seek immediate ophthalmologic evaluation prior to restarting DARZALEX FASPRO®.
Local Reactions
In this pooled safety population of 1446 patients with multiple myeloma (N=1253) or light chain amyloidosis (N=193), injection-site reactions occurred in 8% of patients, including Grade 2 reactions in 1.1%. The most frequent (>1%) injection-site reaction were injection site erythema and injection site rash. In patients with high-risk smoldering multiple myeloma (N=193), injection-site reactions occurred in 28% of patients, including Grade 2 reactions in 3%. These local reactions occurred a median of 6 minutes (range: 0 minutes to 6.5 days) after starting administration of DARZALEX FASPRO®. Monitor for local reactions and consider symptomatic management.
Infections DARZALEX FASPRO® can cause serious, life-threatening, or fatal infections. In patients who received DARZALEX FASPRO® in a pooled safety population including patients with smoldering multiple myeloma and light chain (AL) amyloidosis (N=1639), serious infections, including opportunistic infections, occurred in 24% of patients, Grade 3 or 4 infections occurred in 22%, and fatal infections occurred in 2.5%. The most common type of serious infection reported was pneumonia (8.5%).
Monitor patients for signs and symptoms of infection prior to and during treatment with DARZALEX FASPRO® and treat appropriately. Administer prophylactic antimicrobials according to guidelines.
Neutropenia Daratumumab may increase neutropenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer’s prescribing information for background therapies. Monitor patients with neutropenia for signs of infection. Consider withholding DARZALEX FASPRO® until recovery of neutrophils. In lower body weight patients receiving DARZALEX FASPRO®, higher rates of Grade 3-4 neutropenia were observed.
Thrombocytopenia Daratumumab may increase thrombocytopenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer’s prescribing information for background therapies. Consider withholding DARZALEX FASPRO® until recovery of platelets.
Embryo-Fetal Toxicity Based on the mechanism of action, DARZALEX FASPRO® can cause fetal harm when administered to a pregnant woman. DARZALEX FASPRO® may cause depletion of fetal immune cells and decreased bone density. Advise pregnant women of the potential risk to a fetus. Advise females with reproductive potential to use effective contraception during treatment with DARZALEX FASPRO® and for 3 months after the last dose.
The combination of DARZALEX FASPRO® with lenalidomide, thalidomide, or pomalidomide is contraindicated in pregnant women because lenalidomide, thalidomide, and pomalidomide may cause birth defects and death of the unborn child. Refer to the lenalidomide, thalidomide, or pomalidomide prescribing information on use during pregnancy.
Interference With Serological Testing Daratumumab binds to CD38 on red blood cells (RBCs) and results in a positive indirect antiglobulin test (indirect Coombs test). Daratumumab-mediated positive indirect antiglobulin test may persist for up to 6 months after the last daratumumab administration. Daratumumab bound to RBCs masks detection of antibodies to minor antigens in the patient’s serum. The determination of a patient’s ABO and Rh blood type are not impacted.
Notify blood transfusion centers of this interference with serological testing and inform blood banks that a patient has received DARZALEX FASPRO®. Type and screen patients prior to starting DARZALEX FASPRO®.
Interference With Determination of Complete Response Daratumumab is a human immunoglobulin G (IgG) kappa monoclonal antibody that can be detected on both the serum protein electrophoresis (SPE) and immunofixation (IFE) assays used for the clinical monitoring of endogenous M-protein. This interference can impact the determination of complete response and of disease progression in some DARZALEX FASPRO®-treated patients with IgG kappa myeloma protein.
ADVERSE REACTIONS
In multiple myeloma, the most common adverse reaction (≥20%) with DARZALEX FASPRO® monotherapy is upper respiratory tract infection. The most common adverse reactions with combination therapy (≥20% for any combination) include fatigue, nausea, diarrhea, dyspnea, insomnia, headache, rash, pyrexia, cough, muscle spasms, back pain, vomiting, hypertension, musculoskeletal pain, upper respiratory tract infection, , peripheral neuropathy, peripheral sensory neuropathy, constipation, pneumonia, edema, peripheral edema, and anemia.
The most common adverse reactions (≥20%) in patients with high-risk smoldering multiple myeloma who received DARZALEX FASPRO® monotherapy are upper respiratory tract infection, musculoskeletal pain, fatigue, diarrhea, rash, sleep disorder, sensory neuropathy, and injection site reactions.
The most common hematology laboratory abnormalities (≥40%) with DARZALEX FASPRO® are decreased leukocytes, decreased lymphocytes, decreased neutrophils, decreased platelets, and decreased hemoglobin.
Please click here to read the full Prescribing Information for DARZALEX FASPRO®.
DARZALEX® INDICATIONS AND IMPORTANT SAFETY INFORMATION INDICATIONS DARZALEX® (daratumumab) is indicated for the treatment of adult patients with multiple myeloma: · In combination with bortezomib, melphalan, and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant · In combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy · In combination with bortezomib, thalidomide, and dexamethasone in newly diagnosed patients who are eligible for autologous stem cell transplant · In combination with pomalidomide and dexamethasone in patients who have received at least one prior line of therapy including lenalidomide and a proteasome inhibitor · In combination with carfilzomib and dexamethasone in patients with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy · In combination with bortezomib and dexamethasone in patients who have received at least one prior therapy · As monotherapy in patients who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent
CONTRAINDICATIONS DARZALEX® is contraindicated in patients with a history of severe hypersensitivity (eg, anaphylactic reactions) to daratumumab or any of the components of the formulation.
WARNINGS AND PRECAUTIONS
Infusion-Related Reactions DARZALEX® can cause severe and/or serious infusion-related reactions including anaphylactic reactions. These reactions can be life threatening, and fatal outcomes have been reported. In clinical trials (monotherapy and combination: N=2066), infusion-related reactions occurred in 37% of patients with the Week 1 (16 mg/kg) infusion, 2% with the Week 2 infusion, and cumulatively 6% with subsequent infusions. Less than 1% of patients had a Grade 3/4 infusion-related reaction at Week 2 or subsequent infusions. The median time to onset was 1.5 hours (range: 0 to 73 hours). Nearly all reactions occurred during infusion or within 4 hours of completing DARZALEX®. Severe reactions have occurred, including bronchospasm, hypoxia, dyspnea, hypertension, tachycardia, headache, laryngeal edema, pulmonary edema, and ocular adverse reactions, including choroidal effusion, acute myopia, and acute angle closure glaucoma. Signs and symptoms may include respiratory symptoms, such as nasal congestion, cough, throat irritation, as well as chills, vomiting, and nausea. Less common signs and symptoms were wheezing, allergic rhinitis, pyrexia, chest discomfort, pruritus, hypotension and blurred vision.
When DARZALEX® dosing was interrupted in the setting of ASCT (CASSIOPEIA) for a median of 3.75 months (range: 2.4 to 6.9 months), upon re-initiation of DARZALEX®, the incidence of infusion-related reactions was 11% for the first infusion following ASCT. Infusion-related reactions occurring at re-initiation of DARZALEX® following ASCT were consistent in terms of symptoms and severity (Grade 3 or 4: <1%) with those reported in previous studies at Week 2 or subsequent infusions. In EQUULEUS, patients receiving combination treatment (n=97) were administered the first 16 mg/kg dose at Week 1 split over two days, ie, 8 mg/kg on Day 1 and Day 2, respectively. The incidence of any grade infusion-related reactions was 42%, with 36% of patients experiencing infusion-related reactions on Day 1 of Week 1, 4% on Day 2 of Week 1, and 8% with subsequent infusions.
Pre-medicate patients with antihistamines, antipyretics, and corticosteroids. Frequently monitor patients during the entire infusion. Interrupt DARZALEX® infusion for reactions of any severity and institute medical management as needed. Permanently discontinue DARZALEX® therapy if an anaphylactic reaction or life-threatening (Grade 4) reaction occurs and institute appropriate emergency care. For patients with Grade 1, 2, or 3 reactions, reduce the infusion rate when re-starting the infusion.
To reduce the risk of delayed infusion-related reactions, administer oral corticosteroids to all patients following DARZALEX® infusions. Patients with a history of chronic obstructive pulmonary disease may require additional post-infusion medications to manage respiratory complications. Consider prescribing short- and long-acting bronchodilators and inhaled corticosteroids for patients with chronic obstructive pulmonary disease.
Ocular adverse reactions, including acute myopia and narrowing of the anterior chamber angle due to ciliochoroidal effusions with potential for increased intraocular pressure or glaucoma, have occurred with DARZALEX® infusion. If ocular symptoms occur, interrupt DARZALEX® infusion and seek immediate ophthalmologic evaluation prior to restarting DARZALEX®.
Interference With Serological Testing
Daratumumab binds to CD38 on red blood cells (RBCs) and results in a positive indirect antiglobulin test (indirect Coombs test). Daratumumab-mediated positive indirect antiglobulin test may persist for up to 6 months after the last daratumumab infusion. Daratumumab bound to RBCs masks detection of antibodies to minor antigens in the patient’s serum. The determination of a patient’s ABO and Rh blood type is not impacted. Notify blood transfusion centers of this interference with serological testing and inform blood banks that a patient has received DARZALEX®. Type and screen patients prior to starting DARZALEX®.
Neutropenia and Thrombocytopenia
DARZALEX® may increase neutropenia and thrombocytopenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer’s prescribing information for background therapies. Monitor patients with neutropenia for signs of infection. Consider withholding DARZALEX® until recovery of neutrophils or for recovery of platelets.
Interference With Determination of Complete Response
Daratumumab is a human immunoglobulin G (IgG) kappa monoclonal antibody that can be detected on both the serum protein electrophoresis (SPE) and immunofixation (IFE) assays used for the clinical monitoring of endogenous M-protein. This interference can impact the determination of complete response and of disease progression in some patients with IgG kappa myeloma protein.
Embryo-Fetal Toxicity
Based on the mechanism of action, DARZALEX® can cause fetal harm when administered to a pregnant woman. DARZALEX® may cause depletion of fetal immune cells and decreased bone density. Advise pregnant women of the potential risk to a fetus. Advise females with reproductive potential to use effective contraception during treatment with DARZALEX® and for 3 months after the last dose
The combination of DARZALEX® with lenalidomide, pomalidomide, or thalidomide is contraindicated in pregnant women because lenalidomide, pomalidomide, and thalidomide may cause birth defects and death of the unborn child. Refer to the lenalidomide, pomalidomide, or thalidomide prescribing information on use during pregnancy.
ADVERSE REACTIONS The most frequently reported adverse reactions (incidence ≥20%) were: upper respiratory infection, neutropenia, infusion related reactions, thrombocytopenia, diarrhea, constipation, anemia, peripheral sensory neuropathy, fatigue, peripheral edema, nausea, cough, pyrexia, dyspnea, and asthenia. The most common hematologic laboratory abnormalities (≥40%) with DARZALEX® are: neutropenia, lymphopenia, thrombocytopenia, leukopenia, and anemia.
Please click here to read the full Prescribing Information for DARZALEX®.
WHAT IS IMAAVY® (nipocalimab-aahu)?
IMAAVY® is a prescription medicine used to treat adults and children 12 years of age and older with a disease called generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.
It is not known if IMAAVY® is safe and effective in children under 12 years of age.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about IMAAVY®?
IMAAVY® is a prescription medicine that may cause serious side effects, including:· Infections are a common side effect of IMAAVY® that can be serious. Receiving IMAAVY® may increase your risk of infection. Tell your healthcare provider right away if you have any of the following infection symptoms:
· Allergic (hypersensitivity) reactions may happen during or up to a few weeks after your IMAAVY® infusion. Get emergency medical help right away if you get any of these symptoms during or after your IMAAVY® infusion:
· Infusion-related reactions are possible. Tell your healthcare provider right away if you get any of these symptoms during or a few days after your IMAAVY® infusion:
Do not receive IMAAVY® if you have a severe allergic reaction to nipocalimab-aahu or any of the ingredients in IMAAVY®. Reactions have included angioedema and anaphylaxis.
Before using IMAAVY®, tell your healthcare provider about all of your medical conditions, including if you:
· ever had an allergic reaction to IMAAVY®.· have or had any recent infections or symptoms of infection.· have recently received or are scheduled to receive an immunization (vaccine). People who take IMAAVY® should not receive live vaccines.· are pregnant, plan to become pregnant, or are breastfeeding. It is not known whether IMAAVY® will harm your baby.Pregnancy Safety Study. There is a pregnancy safety study for IMAAVY® if IMAAVY® is given during pregnancy or you become pregnant while receiving IMAAVY®. Your healthcare provider should report IMAAVY exposure by contacting Janssen at 1-800-526-7736 or www.IMAAVY®.com.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
What are the possible side effects of IMAAVY ®?IMAAVY® may cause serious side effects. See “What is the most important information I should know about IMAAVY®?”
The most common side effects of IMAAVY® include: respiratory tract infection, peripheral edema (swelling in your hands, ankles, or feet), and muscle spasms.
These are not all the possible side effects of IMAAVY®. Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please see the full Prescribing Information and Medication Guide for IMAAVY® and discuss any questions you have with your doctor.
Dosage Form and Strengths: IMAAVY® is supplied as a 300 mg/1.62 mL and a 1,200 mg/6.5 mL (185 mg/mL) single-dose vial per carton for intravenous injection.
TECVAYLI® IMPORTANT SAFETY INFORMATION
INDICATION AND USAGE
TECVAYLI® (teclistamab-cqyv) is a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager indicated for the treatment of adult patients with relapsed or refractory multiple myeloma:
· in combination with daratumumab and hyaluronidase-fihj in patients who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent.· as monotherapy, in patients who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody.
IMPORTANT SAFETY INFORMATION
WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGIC TOXICITY including IMMUNE EFFECTOR CELL-ASSOCIATED NEUROTOXICITY SYNDROME
Cytokine release syndrome (CRS), including life-threatening or fatal reactions, can occur in patients receiving TECVAYLI. Initiate treatment with TECVAYLI step-up dosing schedule to reduce risk of CRS. Withhold TECVAYLI until CRS resolves or permanently discontinue based on severity.
Neurologic toxicity, including Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) and serious, life-threatening, or fatal reactions, can occur in patients receiving TECVAYLI. Monitor patients for signs or symptoms of neurologic toxicity, including ICANS, during treatment. Withhold TECVAYLI until neurologic toxicity resolves or permanently discontinue based on severity.
TECVAYLI is available only through a restricted program called the TECVAYLI and TALVEY® Risk Evaluation and Mitigation Strategy (REMS).
WARNINGS AND PRECAUTIONS
Cytokine Release Syndrome - TECVAYLIcan cause cytokine release syndrome (CRS), including life-threatening or fatal reactions.
In the clinical trials (monotherapy and combination therapy; N=448), CRS occurred in 64% of patients who received TECVAYLI at the recommended dose, with Grade 1 CRS occurring in 46% of patients, Grade 2 in 18%, and Grade 3 in 0.2%. Recurrent CRS occurred in 27% of patients. Most patients experienced CRS during the initial step-up dosing schedule (step-up dose 1 [37%], step-up dose 2 [32%], or the initial treatment dose [20%]). CRS first occurred following subsequent doses of TECVAYLI in 2.5% of patients. The median time to onset of CRS was 2 (range: 1 to 9) days after the most recent dose and the median duration of CRS was 2 (range: 1 to 22) days.
Clinical signs and symptoms of CRS included, but were not limited to, fever, hypoxia, chills, hypotension, sinus tachycardia, headache, and elevated liver enzymes (aspartate aminotransferase and alanine aminotransferase elevation).
Initiate therapy according to TECVAYLI step-up dosing schedule to reduce risk of CRS. Administer pretreatment medications to reduce risk of CRS and monitor patients following administration of TECVAYLI accordingly.
At the first sign of CRS, immediately evaluate patient for hospitalization. Administer supportive care based on severity and consider further management per current practice guidelines. Withhold until CRS resolves or permanently discontinue TECVAYLI based on severity.
TECVAYLI is available only through a restricted program under a REMS.
Neurologic Toxicity including Immune Effector Cell-Associated Neurotoxicity Syndrome - TECVAYLI can cause serious, life-threatening, or fatal neurologic toxicity, including immune effector cell-associated neurotoxicity syndrome (ICANS).
In the clinical trials (monotherapy and combination therapy trials; N=448), neurologic toxicity occurred in 60% of patients who received TECVAYLI at the recommended dosage, with Grade 3 or 4 neurologic toxicity in 6%. Neurologic toxicities reported in ≥5% of patients included headache (27%), sensory neuropathy (16%), motor dysfunction (15%), insomnia (12%), encephalopathy (11%), and dizziness (8%). Fatal neurologic toxicity occurred in 0.4% of patients, including Guillain-Barré syndrome and status epilepticus (one patient each).
In MajesTEC-1, ICANS was reported in 6% of patients who received TECVAYLI as monotherapy at the recommended dosage. Recurrent ICANS occurred in 1.8% of patients. Most patients experienced ICANS following step-up dose 1 (1.2%), step-up dose 2 (0.6%), or the initial treatment dose (1.8%). Less than 3% of patients developed first occurrence of ICANS following subsequent TECVAYLI doses. The median time to onset of ICANS was 4 (range: 2 to 8) days after the most recent dose with a median duration of 3 (range: 1 to 20) days. The most frequent clinical manifestations of ICANS reported were confusional state and dysgraphia.
In MajesTEC-3, ICANS was reported in 1.1% of patients who received the recommended TECVAYLI dosage in combination with daratumumab and hyaluronidase-fihj, including Grade 4 ICANS in 1 patient. All events of ICANS occurred during the step-up dosing schedule. The median time to onset of ICANS was 2 (range: 1 to 3) days after the most recent dose and the median duration of ICANS was 2 (range: 1 to 2) days. The clinical manifestations of ICANS reported were amnesia, encephalopathy and delirium.
The onset of ICANS can be concurrent with CRS, following resolution of CRS, or in the absence of CRS.
Monitor patients for signs and symptoms of neurologic toxicity, including ICANS during TECVAYLI treatment. At the first sign of neurologic toxicity, including ICANS, immediately evaluate patient and provide supportive therapy based on severity. Withhold until neurologic toxicity resolves or permanently discontinue TECVAYLI based on severity per recommendations and consider further management per current practice guidelines.
Due to the potential for neurologic toxicity, patients receiving TECVAYLI are at risk of depressed level of consciousness. Advise patients to refrain from driving or operating heavy or potentially dangerous machinery during and for 48 hours after completion of TECVAYLI step-up dosing schedule and in the event of new onset of any neurologic toxicity symptoms until neurologic toxicity resolves.
TECVAYLI is available only through a restricted program under a REMS.
TECVAYLI and TALVEY REMS - TECVAYLI is available only through a restricted program under a REMS called the TECVAYLI and TALVEY REMS because of the risks of CRS and neurologic toxicity, including ICANS.
Hepatotoxicity - TECVAYLI can cause hepatotoxicity, including fatalities. There was one fatal case of hepatic failure in MajesTEC-1. In patients who received TECVAYLI at the recommended dose in the clinical trials (monotherapy and combination therapy trials; N=448) elevated aspartate aminotransferase (AST) occurred in 47% of patients, with Grade 3 or 4 elevations in 2.9%. Elevated alanine aminotransferase (ALT) occurred in 48% of patients, with Grade 3 or 4 elevations in 3.8%. Elevated total bilirubin occurred in 10% of patients with Grade 3 or 4 elevations in 0.7%. Liver enzyme elevation can occur with or without concurrent CRS.
Monitor liver enzymes and bilirubin at baseline and during treatment as clinically indicated. Withhold TECVAYLI or consider permanent discontinuation of TECVAYLI based on severity.
Infections - TECVAYLI can cause severe, life-threatening, or fatal infections.
In MajesTEC-1 (N=165), in patients who received the recommended TECVAYLI dosage, serious infections, including opportunistic infections, occurred in 30% of patients, Grade 3 or 4 infections in 35% of patients, and fatal infections in 4.2% of patients.
In MajesTEC-3 (N=283), in patients who received TECVAYLI in combination with daratumumab and hyaluronidase-fihj at the recommended dosage, serious infections, including opportunistic infections, occurred in 54% of patients, Grade 3 or Grade 4 infections in 54% of patients, and fatal infections in 4.6% of patients.
Monitor patients for signs and symptoms of infection prior to and during treatment with TECVAYLI and treat appropriately. Administer prophylactic antimicrobials according to current practice guidelines.
Withhold TECVAYLI or consider permanent discontinuation of TECVAYLI based on severity.
Monitor immunoglobulin levels prior to and during treatment with TECVAYLI and administer subcutaneous or intravenous immunoglobulin (IVIG) to maintain the serum levels >400 mg/dL.
Neutropenia - TECVAYLI can cause neutropenia and febrile neutropenia. In patients who received TECVAYLI at the recommended dose in the clinical trials (monotherapy and combination therapy trials; N=448), decreased neutrophils occurred in 88% of patients, with Grade 3 or 4 decreased neutrophils in 70%. Febrile neutropenia occurred in 6% of patients.
Monitor complete blood cell counts at baseline and periodically during treatment and provide supportive care per local institutional guidelines.
Monitor patients with neutropenia for signs of infection.
Withhold TECVAYLI based on severity.
Hypersensitivity and Other Administration Reactions - TECVAYLI can cause both systemic administration-related and local injection-site reactions.
Systemic Reactions - In patients who received the recommended TECVAYLI dosage in the clinical trials (monotherapy and combination therapy trials; N=448), 2.5% of patients experienced systemic-administration reactions, which included recurrent pyrexia and rash.
Local Reactions - In patients who received TECVAYLI at the recommended dosage in the clinical trials (monotherapy and combination therapy trials; N=448), injection-site reactions occurred in 37% of patients, with Grade 1 injection-site reactions in 29% and Grade 2 in 9%.
Withhold TECVAYLI or consider permanent discontinuation of TECVAYLI based on severity.
Embryo-Fetal Toxicity - Based on its mechanism of action, TECVAYLI may cause fetal harm when administered to a pregnant patient. Advise pregnant patients of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with TECVAYLI and for 5 months after the last dose.
ADVERSE REACTIONSThe most common adverse reactions (≥20%) in patients who received TECVAYLI monotherapy were pyrexia, CRS, musculoskeletal pain, injection site reaction, fatigue, upper respiratory tract infection, nausea, headache, pneumonia, and diarrhea. The most common adverse reactions (≥20%) in patients who received TECVAYLI in combination with daratumumab and hyaluronidase-fihj were hypogammaglobulinemia, upper respiratory tract infection, CRS, cough, diarrhea, musculoskeletal pain, COVID-19, pneumonia, injection site reaction, fatigue, pyrexia, headache, nausea, gastroenteritis, and weight decreased.
The most common Grade 3 to 4 laboratory abnormalities (≥20%) with TECVAYLI (as monotherapy or in combination with daratumumab and hyaluronidase-fihj) were decreased lymphocytes, decreased neutrophils, decreased white blood cells, decreased hemoglobin, and decreased platelets.
Please read full Prescribing Information, including Boxed WARNING, for TECVAYLI.
CARVYKTI® IMPORTANT SAFETY INFORMATION
WARNING: CYTOKINE RELEASE SYNDROME, NEUROLOGIC TOXICITIES, HLH/MAS, PROLONGED and RECURRENT CYTOPENIA, and SECONDARY HEMATOLOGICAL MALIGNANCIES
Cytokine Release Syndrome (CRS), including fatal or life-threatening reactions, occurred in patients following treatment with CARVYKTI®. Do not administer CARVYKTI® to patients with active infection or inflammatory disorders. Treat severe or life-threatening CRS with tocilizumab or tocilizumab and corticosteroids.
Immune Effector Cell-associated Neurotoxicity Syndrome (ICANS), which may be fatal or life-threatening, occurred following treatment with CARVYKTI®, including before CRS onset, concurrently with CRS, after CRS resolution, or in the absence of CRS. Monitor for neurologic events after treatment with CARVYKTI®. Provide supportive care and/or corticosteroids as needed.
Parkinsonism and Guillain-Barré syndrome (GBS) and their associated complications resulting in fatal or life-threatening reactions have occurred following treatment with CARVYKTI®.
Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome (HLH/MAS), including fatal and life-threatening reactions, occurred in patients following treatment with CARVYKTI®. HLH/MAS can occur with CRS or neurologic toxicities.
Prolonged and/or recurrent cytopenias with bleeding and infection and requirement for stem cell transplantation for hematopoietic recovery occurred following treatment with CARVYKTI®.
Immune Effector Cell-associated Enterocolitis (IEC-EC), including fatal or life-threatening reactions, occurred following treatment with CARVYKTI®.
Secondary hematological malignancies, including myelodysplastic syndrome and acute myeloid leukemia, have occurred in patients following treatment with CARVYKTI®. T-cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T-cell immunotherapies, including CARVYKTI®.
WARNINGS AND PRECAUTIONS
INCREASED EARLY MORTALITY - In CARTITUDE-4, a (1:1) randomized controlled trial, there was a numerically higher percentage of early deaths in patients randomized to the CARVYKTI® treatment arm compared to the control arm. Among patients with deaths occurring within the first 10 months from randomization, a greater proportion (29/208; 14%) occurred in the CARVYKTI® arm compared to (25/211; 12%) in the control arm. Of the 29 deaths that occurred in the CARVYKTI® arm within the first 10 months of randomization, 10 deaths occurred prior to CARVYKTI® infusion, and 19 deaths occurred after CARVYKTI® infusion. Of the 10 deaths that occurred prior to CARVYKTI® infusion, all occurred due to disease progression, and none occurred due to adverse events. Of the 19 deaths that occurred after CARVYKTI® infusion, 3 occurred due to disease progression, and 16 occurred due to adverse events. The most common adverse events were due to infection (n=12).
CYTOKINE RELEASE SYNDROME (CRS),including fatal or life-threatening reactions, occurred following treatment with CARVYKTI®. Among patients receiving CARVYKTI® for RRMM in the CARTITUDE-1 & -4 studies (N=285), CRS occurred in 84% (238/285), including ≥ Grade 3 CRS (ASTCT 2019) in 4% (11/285) of patients. Median time to onset of CRS, any grade, was 7 days (range: 1 to 23 days). CRS resolved in 82% with a median duration of 4 days (range: 1 to 97 days). The most common manifestations of CRS in all patients combined (≥10%) included fever (84%), hypotension (29%) and aspartate aminotransferase increased (11%). Serious events that may be associated with CRS include pyrexia, hemophagocytic lymphohistiocytosis, respiratory failure, disseminated intravascular coagulation, capillary leak syndrome, and supraventricular and ventricular tachycardia. CRS occurred in 78% of patients in CARTITUDE-4 (3% Grade 3 to 4) and in 95% of patients in CARTITUDE-1 (4% Grade 3 to 4).
Identify CRS based on clinical presentation. Evaluate for and treat other causes of fever, hypoxia, and hypotension. CRS has been reported to be associated with findings of HLH/MAS, and the physiology of the syndromes may overlap. HLH/MAS is a potentially life-threatening condition. In patients with progressive symptoms of CRS or refractory CRS despite treatment, evaluate for evidence of HLH/MAS.
Confirm that a minimum of 2 doses of tocilizumab are available prior to infusion of CARVYKTI®.
Of the 285 patients who received CARVYKTI® in clinical trials, 53% (150/285) patients received tocilizumab; 35% (100/285) received a single dose, while 18% (50/285) received more than 1 dose of tocilizumab. Overall, 14% (39/285) of patients received at least 1 dose of corticosteroids for treatment of CRS.
Monitor patients at least daily for 7 days following CARVYKTI® infusion for signs and symptoms of CRS. Monitor patients for signs or symptoms of CRS for at least 2 weeks after infusion. At the first sign of CRS, immediately institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids.
Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time.
NEUROLOGIC TOXICITIES, which may be severe, life-threatening, or fatal, occurred following treatment with CARVYKTI®. Neurologic toxicities included ICANS, neurologic toxicity with signs and symptoms of Parkinsonism, GBS, immune mediated myelitis, peripheral neuropathies, and cranial nerve palsies. Counsel patients on the signs and symptoms of these neurologic toxicities, and on the delayed nature of onset of some of these toxicities. Instruct patients to seek immediate medical attention for further assessment and management if signs or symptoms of any of these neurologic toxicities occur at any time.
Among patients receiving CARVYKTI® in the CARTITUDE-1 & 4 studies for RRMM, one or more neurologic toxicities occurred in 24% (69/285), including ≥ Grade 3 cases in 7% (19/285) of patients. Median time to onset was 10 days (range: 1 to 101) with 63/69 (91%) of cases developing by 30 days. Neurologic toxicities resolved in 72% (50/69) of patients with a median duration to resolution of 23 days (range: 1 to 544). Of patients developing neurotoxicity, 96% (66/69) also developed CRS. Subtypes of neurologic toxicities included ICANS in 13%, peripheral neuropathy in 7%, cranial nerve palsy in 7%, parkinsonism in 3%, and immune mediated myelitis in 0.4% of the patients.
Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS): Patients receiving CARVYKTI® may experience fatal or life-threatening ICANS following treatment with CARVYKTI®, including before CRS onset, concurrently with CRS, after CRS resolution, or in the absence of CRS.
Among patients receiving CARVYKTI® in the CARTITUDE-1 & -4 studies, ICANS occurred in 13% (36/285), including Grade ≥3 in 2% (6/285) of the patients. Median time to onset of ICANS was 8 days (range: 1 to 28 days). ICANS resolved in 30 of 36 (83%) of patients, with a median time to resolution of 3 days (range: 1 to 143 days). Median duration of ICANS was 6 days (range: 1 to 1229 days) in all patients, including those with ongoing neurologic events at the time of death or data cutoff. Of patients with ICANS, 97% (35/36) had CRS. The onset of ICANS occurred during CRS in 69% of patients, before and after the onset of CRS in 14% of patients, respectively.
Immune Effector Cell-associated Neurotoxicity Syndrome occurred in 7% of patients in CARTITUDE-4 (0.5% Grade 3) and in 23% of patients in CARTITUDE-1 (3% Grade 3). The most frequent (≥2%) manifestations of ICANS included encephalopathy (12%), aphasia (4%), headache (3%), motor dysfunction (3%), ataxia (2%), and sleep disorder (2%).
Monitor patients at least daily for 7 days following CARVYKTI® infusion for signs and symptoms of ICANS. Rule out other causes of ICANS symptoms. Monitor patients for signs or symptoms of ICANS for at least 2 weeks after infusion and treat promptly. Neurologic toxicity should be managed with supportive care and/or corticosteroids as needed. Advise patients to avoid driving for at least 2 weeks following infusion.
Parkinsonism: Neurologic toxicity with parkinsonism has been reported in clinical trials of CARVYKTI®. Among patients receiving CARVYKTI® in the CARTITUDE-1 & -4 studies, parkinsonism occurred in 3% (8/285), including Grade ≥3 in 2% (5/285) of the patients. Median time to onset of parkinsonism was 56 days (range: 14 to 914 days). Parkinsonism resolved in 1 of 8 (13%) of patients with a median time to resolution of 523 days. Median duration of parkinsonism was 243.5 days (range: 62 to 720 days) in all patients, including those with ongoing neurologic events at the time of death or data cutoff. The onset of parkinsonism occurred after CRS for all patients and after ICANS for 6 patients.
Parkinsonism occurred in 1% of patients in CARTITUDE-4 (no Grade 3 to 4) and in 6% of patients in CARTITUDE-1 (4% Grade 3 to 4).
Manifestations of parkinsonism included movement disorders, cognitive impairment, and personality changes. Monitor patients for signs and symptoms of parkinsonism that may be delayed in onset and managed with supportive care measures. There is limited efficacy information with medications used for the treatment of Parkinson’s disease for the improvement or resolution of parkinsonism symptoms following CARVYKTI® treatment.
Guillain-Barré Syndrome: A fatal outcome following GBS occurred following treatment with CARVYKTI® despite treatment with intravenous immunoglobulins. Symptoms reported include those consistent with Miller-Fisher variant of GBS, encephalopathy, motor weakness, speech disturbances, and polyradiculoneuritis.
Monitor for GBS. Evaluate patients presenting with peripheral neuropathy for GBS. Consider treatment of GBS with supportive care measures and in conjunction with immunoglobulins and plasma exchange, depending on severity of GBS.
Immune Mediated Myelitis: Grade 3 myelitis occurred 25 days following treatment with CARVYKTI® in CARTITUDE-4 in a patient who received CARVYKTI® as subsequent therapy. Symptoms reported included hypoesthesia of the lower extremities and the lower abdomen with impaired sphincter control. Symptoms improved with the use of corticosteroids and intravenous immune globulin. Myelitis was ongoing at the time of death from other cause.
Peripheral Neuropathy occurred following treatment with CARVYKTI®. Among patients receiving CARVYKTI® in the CARTITUDE-1 & -4 studies, peripheral neuropathy occurred in 7% (21/285), including Grade ≥3 in 1% (3/285) of the patients. Median time to onset of peripheral neuropathy was 57 days (range: 1 to 914 days). Peripheral neuropathy resolved in 11 of 21 (52%) of patients with a median time to resolution of 58 days (range: 1 to 215 days). Median duration of peripheral neuropathy was 149.5 days (range: 1 to 692 days) in all patients, including those with ongoing neurologic events at the time of death or data cutoff.
Peripheral neuropathies occurred in 7% of patients in CARTITUDE-4 (0.5% Grade 3 to 4) and in 7% of patients in CARTITUDE-1 (2% Grade 3 to 4). Monitor patients for signs and symptoms of peripheral neuropathies. Patients who experience peripheral neuropathy may also experience cranial nerve palsies or GBS.
Cranial Nerve Palsies occurred following treatment with CARVYKTI®. Among patients receiving CARVYKTI® in the CARTITUDE-1 & -4 studies, cranial nerve palsies occurred in 7% (19/285), including Grade ≥3 in 1% (1/285) of the patients. Median time to onset of cranial nerve palsies was 21 days (range: 17 to 101 days). Cranial nerve palsies resolved in 17 of 19 (89%) of patients with a median time to resolution of 66 days (range: 1 to 209 days). Median duration of cranial nerve palsies was 70 days (range: 1 to 262 days) in all patients, including those with ongoing neurologic events at the time of death or data cutoff. Cranial nerve palsies occurred in 9% of patients in CARTITUDE-4 (1% Grade 3 to 4) and in 3% of patients in CARTITUDE-1 (1% Grade 3 to 4).
The most frequent cranial nerve affected was the 7th cranial nerve. Additionally, cranial nerves III, V, and VI have been reported to be affected.
Monitor patients for signs and symptoms of cranial nerve palsies. Consider management with systemic corticosteroids, depending on the severity and progression of signs and symptoms.
HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH)/MACROPHAGE ACTIVATION SYNDROME (MAS): Among patients receiving CARVYKTI® in the CARTITUDE-1 & -4 studies, HLH/MAS occurred in 1% (3/285) of patients. All events of HLH/MAS had onset within 99 days of receiving CARVYKTI®, with a median onset of 10 days (range: 8 to 99 days), and all occurred in the setting of ongoing or worsening CRS. The manifestations of HLH/MAS included hyperferritinemia, hypotension, hypoxia with diffuse alveolar damage, coagulopathy and hemorrhage, cytopenia, and multi-organ dysfunction, including renal dysfunction and respiratory failure.
Patients who develop HLH/MAS have an increased risk of severe bleeding. Monitor hematologic parameters in patients with HLH/MAS and transfuse per institutional guidelines. Fatal cases of HLH/MAS occurred following treatment with CARVYKTI®.
HLH is a life-threatening condition with a high mortality rate if not recognized and treated early. Treatment of HLH/MAS should be administered per institutional standards.
PROLONGED AND RECURRENT CYTOPENIAS: Patients may exhibit prolonged and recurrent cytopenias following lymphodepleting chemotherapy and CARVYKTI® infusion.
Among patients receiving CARVYKTI® in the CARTITUDE-1 & -4 studies, Grade 3 or higher cytopenias not resolved by Day 30 following CARVYKTI® infusion occurred in 62% (176/285) of the patients and included thrombocytopenia 33% (94/285), neutropenia 27% (76/285), lymphopenia 24% (67/285), and anemia 2% (6/285). After Day 60 following CARVYKTI® infusion, 22%, 20%, 5%, and 6% of patients had a recurrence of Grade 3 or 4 lymphopenia, neutropenia, thrombocytopenia, and anemia, respectively, after initial recovery of their Grade 3 or 4 cytopenia. Seventy-seven percent (219/285) of patients had one, two, or three or more recurrences of Grade 3 or 4 cytopenias after initial recovery of Grade 3 or 4 cytopenia. Sixteen and 25 patients had Grade 3 or 4 neutropenia and thrombocytopenia, respectively, at the time of death.
Monitor blood counts prior to and after CARVYKTI® infusion. Manage cytopenias with growth factors and blood product transfusion support according to local institutional guidelines.
INFECTIONS: CARVYKTI® should not be administered to patients with active infection or inflammatory disorders. Severe, life-threatening, or fatal infections occurred in patients after CARVYKTI® infusion.
Among patients receiving CARVYKTI® in the CARTITUDE-1 & -4 studies, infections occurred in 57% (163/285), including Grade ≥3 in 24% (69/285) of patients. Grade 3 or 4 infections with an unspecified pathogen occurred in 12%, viral infections in 6%, bacterial infections in 5%, and fungal infections in 1% of patients. Overall, 5% (13/285) of patients had Grade 5 infections, 2.5% of which were due to COVID-19. Patients treated with CARVYKTI® had an increased rate of fatal COVID-19 infections compared to the standard therapy arm.
Monitor patients for signs and symptoms of infection before and after CARVYKTI® infusion and treat patients appropriately. Administer prophylactic, pre-emptive, and/or therapeutic antimicrobials according to the standard institutional guidelines. Febrile neutropenia was observed in 5% of patients after CARVYKTI® infusion and may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad-spectrum antibiotics, fluids, and other supportive care, as medically indicated. Counsel patients on the importance of prevention measures. Follow institutional guidelines for the vaccination and management of immunocompromised patients with COVID-19.
Viral Reactivation: Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, can occur in patients with hypogammaglobulinemia. Perform screening for Cytomegalovirus (CMV), HBV, hepatitis C virus (HCV), and human immunodeficiency virus (HIV) or any other infectious agents if clinically indicated in accordance with clinical guidelines before collection of cells for manufacturing. Consider antiviral therapy to prevent viral reactivation per local institutional guidelines/clinical practice.
Reactivation of John Cunningham (JC) virus, leading to progressive multifocal leukoencephalopathy (PML), including cases with fatal outcomes, have been reported following treatment. Perform appropriate diagnostic evaluations in patients with neurological adverse events.
HYPOGAMMAGLOBULINEMIA: can occur in patients receiving treatment with CARVYKTI®. Among patients receiving CARVYKTI® in the CARTITUDE-1 & -4 studies, hypogammaglobulinemia adverse event was reported in 36% (102/285) of patients; laboratory IgG levels fell below 500 mg/dL after infusion in 93% (265/285) of patients. Hypogammaglobulinemia either as an adverse reaction or laboratory IgG level below 500 mg/dL after infusion occurred in 94% (267/285) of patients treated. Fifty-six percent (161/285) of patients received intravenous immunoglobulin (IVIG) post CARVYKTI® for either an adverse reaction or prophylaxis.
Monitor immunoglobulin levels after treatment with CARVYKTI® and administer IVIG for IgG <400 mg/dL. Manage per local institutional guidelines, including infection precautions and antibiotic or antiviral prophylaxis.
Use of Live Vaccines: The safety of immunization with live viral vaccines during or following CARVYKTI® treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during CARVYKTI® treatment, and until immune recovery following treatment with CARVYKTI®.
HYPERSENSITIVITY REACTIONS occurred following treatment with CARVYKTI®. Among patients receiving CARVYKTI® in the CARTITUDE-1 & -4 studies, hypersensitivity reactions occurred in 5% (13/285), all of which were ≤2 Grade. Manifestations of hypersensitivity reactions included flushing, chest discomfort, tachycardia, wheezing, tremor, burning sensation, non-cardiac chest pain, and pyrexia. Serious hypersensitivity reactions, including anaphylaxis, may be due to the dimethyl sulfoxide (DMSO) in CARVYKTI®. Patients should be carefully monitored for 2 hours after infusion for signs and symptoms of severe reaction. Treat promptly and manage patients appropriately according to the severity of the hypersensitivity reaction.
IMMUNE EFFECTOR CELL-ASSOCIATED ENTERCOLITIS (IEC-EC) has occurred in patients treated with CARVYKTI®. Manifestations include severe or prolonged diarrhea, abdominal pain, and weight loss requiring parenteral nutrition. IEC-EC has been associated with fatal outcome from perforation or sepsis. Manage according to institutional guidelines, including referral to gastroenterology and infectious disease specialists. In cases of refractory IEC-EC, consider additional workup to exclude alternative etiologies, including T-cell lymphoma of the GI tract, which has been reported in the post marketing setting.
SECONDARY MALIGNANCIES: Patients treated with CARVYKTI® may develop secondary malignancies. Among patients receiving CARVYKTI® in the CARTITUDE-1 & -4 studies, myeloid neoplasms occurred in 5% (13/285) of patients (9 cases of myelodysplastic syndrome, 3 cases of acute myeloid leukemia, and 1 case of myelodysplastic syndrome followed by acute myeloid leukemia). The median time to onset of myeloid neoplasms was 447 days (range: 56 to 870 days) after treatment with CARVYKTI®. Ten of these 13 patients died following the development of myeloid neoplasms; 2 of the 13 cases of myeloid neoplasm occurred after initiation of subsequent antimyeloma therapy. Cases of myelodysplastic syndrome and acute myeloid leukemia have also been reported in the post marketing setting. T-cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T-cell immunotherapies, including CARVYKTI®. Mature T-cell malignancies, including CAR-positive tumors, may present as soon as weeks following infusions, and may include fatal outcomes.
Monitor lifelong for secondary malignancies. In the event that a secondary malignancy occurs, contact Janssen Biotech, Inc., at 1-800-526-7736 for reporting and to obtain instructions on collection of patient samples.
ADVERSE REACTIONS The most common nonlaboratory adverse reactions (incidence greater than 20%) are pyrexia, cytokine release syndrome, hypogammaglobulinemia, hypotension, musculoskeletal pain, fatigue, infections-pathogen unspecified, cough, chills, diarrhea, nausea, encephalopathy, decreased appetite, upper respiratory tract infection, headache, tachycardia, dizziness, dyspnea, edema, viral infections, coagulopathy, constipation, and vomiting. The most common Grade 3 or 4 laboratory adverse reactions (incidence greater than or equal to 50%) include lymphopenia, neutropenia, white blood cell decreased, thrombocytopenia, and anemia.
Please read full Prescribing Information, including Boxed Warning, for CARVYKTI®.
IMBRUVICA® IMPORTANT SAFETY INFORMATION
INDICATIONS
IMBRUVICA® is a kinase inhibitor indicated for the treatment of:
Adult patients with chronic lymphocytic leukemia (CLL)/Small lymphocytic lymphoma (SLL). Adult patients with chronic lymphocytic leukemia (CLL)/Small lymphocytic lymphoma (SLL) with 17p deletion. Adult patients with Waldenström’s macroglobulinemia (WM). Adult and pediatric patients age 1 year and older with chronic graft versus host disease (cGVHD) after failure of one or more lines of systemic therapy.
For more information, visit www.IMBRUVICA.com.
WARNINGS AND PRECAUTIONS
Hemorrhage: Fatal bleeding events have occurred in patients who received IMBRUVICA®. Major hemorrhage (≥ Grade 3, serious, or any central nervous system events; e.g., intracranial hemorrhage [including subdural hematoma], gastrointestinal bleeding, hematuria, and post procedural hemorrhage) occurred in 4.2% of patients, with fatalities occurring in 0.4% of 2,838 patients who received IMBRUVICA® in 27 clinical trials. Bleeding events of any grade including bruising and petechiae occurred in 39%, and excluding bruising and petechiae occurred in 23% of patients who received IMBRUVICA®, respectively.
The mechanism for the bleeding events is not well understood.
Use of either anticoagulant or antiplatelet agents concomitantly with IMBRUVICA® increases the risk of major hemorrhage. Across clinical trials, 3.1% of 2,838 patients who received IMBRUVICA® without antiplatelet or anticoagulant therapy experienced major hemorrhage. The addition of antiplatelet therapy with or without anticoagulant therapy increased this percentage to 4.4%, and the addition of anticoagulant therapy with or without antiplatelet therapy increased this percentage to 6.1%. Consider the risks and benefits of anticoagulant or antiplatelet therapy when co-administered with IMBRUVICA®. Monitor for signs and symptoms of bleeding.
Consider the benefit-risk of withholding IMBRUVICA® for at least 3 to 7 days pre- and post-surgery depending upon the type of surgery and the risk of bleeding.
Infections: Fatal and non-fatal infections (including bacterial, viral, or fungal) have occurred with IMBRUVICA® therapy. Grade 3 or greater infections occurred in 21% of 1,476 patients with B-cell malignancies who received IMBRUVICA® in clinical trials. Cases of progressive multifocal leukoencephalopathy (PML) and Pneumocystis jirovecii pneumonia (PJP) have occurred in patients treated with IMBRUVICA®. Consider prophylaxis according to standard of care in patients who are at increased risk for opportunistic infections. Monitor and evaluate patients for fever and infections and treat appropriately.
Cardiac Arrhythmias, Cardiac Failure, and Sudden Death: Fatal and serious cardiac arrhythmias and cardiac failure have occurred with IMBRUVICA®. Deaths due to cardiac causes or sudden deaths occurred in 1% of 4,896 patients who received IMBRUVICA® in clinical trials, including in patients who received IMBRUVICA® in unapproved monotherapy or combination regimens. These adverse reactions occurred in patients with and without preexisting hypertension or cardiac comorbidities. Patients with cardiac comorbidities may be at greater risk of these events. Grade 3 or greater ventricular tachyarrhythmias were reported in 0.2%, Grade 3 or greater atrial fibrillation and atrial flutter were reported in 3.7%, and Grade 3 or greater cardiac failure was reported in 1.3% of 4,896 patients who received IMBRUVICA® in clinical trials, including in patients who received IMBRUVICA® in unapproved monotherapy or combination regimens. These events have occurred particularly in patients with cardiac risk factors including hypertension and diabetes mellitus, a previous history of cardiac arrhythmias, and in patients with acute infections. Evaluate cardiac history and function at baseline, and monitor patients for cardiac arrhythmias and cardiac function. Obtain further evaluation (e.g., ECG, echocardiogram) as indicated for patients who develop symptoms of arrhythmia (e.g., palpitations, lightheadedness, syncope, chest pain), new onset dyspnea, or other cardiovascular concerns. Manage cardiac arrhythmias and cardiac failure appropriately, follow dose modification guidelines, and consider the risks and benefits of continued IMBRUVICA® treatment.
Hypertension: Hypertension occurred in 19% of 1,476 patients with B-cell malignancies who received IMBRUVICA® in clinical trials. Grade 3 or greater hypertension occurred in 8% of patients. Based on data from a subset of these patients, (N=1,124), the median time to onset was 5.9 months (range, 0 to 24 months). In a long-term safety analysis over 5 years of 1,284 patients with B-cell malignancies treated for a median of 36 months (range, 0 to 98 months), the cumulative rate of hypertension increased over time. The prevalence for Grade 3 or greater hypertension was 4% (year 0-1), 7% (year 1-2), 9% (year 2-3), 9% (year 3-4), and 9% (year 4-5); the overall incidence for the 5-year period was 11%. Monitor blood pressure in patients treated with IMBRUVICA®, initiate or adjust anti-hypertensive medication throughout treatment with IMBRUVICA® as appropriate, and follow dosage modification guidelines for Grade 3 or higher hypertension.
Cytopenias: In 645 patients with B-cell malignancies who received IMBRUVICA® as a single agent, grade 3 or 4 neutropenia occurred in 23% of patients, grade 3 or 4 thrombocytopenia in 8% and grade 3 or 4 anemia in 2.8%, based on laboratory measurements. Monitor complete blood counts monthly.
Second Primary Malignancies: Other malignancies (10%), including non-skin carcinomas (3.9%), occurred among the 1,476 patients with B-cell malignancies who received IMBRUVICA® in clinical trials. The most frequent second primary malignancy was non-melanoma skin cancer (6%).
Tumor Lysis Syndrome: Tumor lysis syndrome has been infrequently reported with IMBRUVICA®. Assess the baseline risk (e.g., high tumor burden) and take appropriate precautions. Monitor patients closely and treat as appropriate.
Embryo-Fetal Toxicity: Based on findings in animals, IMBRUVICA® can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with IMBRUVICA® and for 1 month after the last dose. Advise males with female partners of reproductive potential to use effective contraception during the same time period.
ADVERSE REACTIONS
B-cell malignancies: The most common adverse reactions (≥30%) in adult patients with B-cell malignancies were thrombocytopenia (55%)*, diarrhea (44%), fatigue (39%), musculoskeletal pain (39%), neutropenia (39%)*, rash (36%), anemia (35%)*, bruising (32%), and nausea (30%).
The most common Grade ≥ 3 adverse reactions (≥5%) in adult patients with B-cell malignancies were neutropenia (21%)*, thrombocytopenia (14%)*, pneumonia (8%), and hypertension (8%).
Approximately 9% (CLL/SLL), and 14% (WM) of adult patients had a dose reduction due to adverse reactions.Approximately 4-10% (CLL/SLL) and 5% (WM) of adult patients discontinued due to adverse reactions.cGVHD: The most common adverse reactions (≥20%) in adult or pediatric patients with cGVHD were fatigue (57%), anemia (49%)*, bruising (40%), diarrhea (36%), thrombocytopenia (33%)*, musculoskeletal pain (30%), pyrexia (30%), muscle spasms (29%), stomatitis (29%), hemorrhage (26%), nausea (26%), abdominal pain (23%), pneumonia (23%), and headache (21%).
The most common Grade 3 or higher adverse reactions (≥5%) reported in adult or pediatric patients with cGVHD were pneumonia (14%), anemia (13%)*, fatigue (12%), pyrexia (11%), diarrhea (10%), neutropenia (10%)*, sepsis (10%), osteonecrosis (9%), stomatitis (9%), hypokalemia (7%), headache (5%), and musculoskeletal pain (5%).
Discontinuation of IMBRUVICA® treatment due to an adverse reaction occurred in 24% of adult patients and 23% of pediatric patients. Adverse reactions leading to dose reduction occurred in 26% of adult patients and 19% of pediatric patients.
*Treatment-emergent decreases (all grades) were based on laboratory measurements.
DRUG INTERACTIONS
CYP3A Inhibitors: Co-administration of IMBRUVICA® with strong or moderate CYP3A inhibitors may increase ibrutinib plasma concentrations. Increased ibrutinib concentrations may increase the risk of drug-related toxicity. Dose modifications of IMBRUVICA® are recommended when used concomitantly with posaconazole, voriconazole, and moderate CYP3A inhibitors. Avoid concomitant use of other strong CYP3A inhibitors. Interrupt IMBRUVICA® if strong inhibitors are used short-term (e.g., for ≤ 7 days). Avoid grapefruit and Seville oranges during IMBRUVICA® treatment, as these contain strong or moderate inhibitors of CYP3A. See dose modification guidelines in USPI sections 2.3 and 7.1.
CYP3A Inducers: Avoid coadministration with strong CYP3A inducers.
SPECIFIC POPULATIONS
Pediatric Use: The safety and effectiveness of IMBRUVICA® have not been established for the treatment of cGVHD after failure of one or more lines of therapy in pediatric patients less than 1 year of age. The safety and effectiveness of IMBRUVICA® in pediatric patients have not been established in CLL/SLL, CLL/SLL with 17p deletion, WM, or in patients with mature B-cell non-Hodgkin lymphoma.
In the randomized population from a study that included 35 patients (26 pediatric patients age 5 to less than 17 years) with previously treated mature B-cell non-Hodgkin lymphoma, major hemorrhage and discontinuation of chemoimmunotherapy due to adverse reactions occurred more frequently in the ibrutinib plus chemoimmunotherapy arm compared to the chemoimmunotherapy alone arm.
Hepatic Impairment:Adult Patients with B-cell Malignancies: Hepatic Impairment (based on Child-Pugh criteria): Avoid use of IMBRUVICA® in patients with severe hepatic impairment. In patients with mild or moderate impairment, reduce recommended IMBRUVICA® dose and monitor more frequently for adverse reactions of IMBRUVICA®.
Patients with cGVHD: Avoid use of IMBRUVICA® in patients with total bilirubin level > 3x upper limit of normal (ULN) (unless of non-hepatic origin or due to Gilbert’s syndrome). Reduce recommended dose when administering IMBRUVICA® to patients with total bilirubin level > 1.5 to 3x ULN (unless of non-hepatic origin or due to Gilbert’s syndrome).
Please click here to see the full Prescribing Information.
ABOUT JOHNSON & JOHNSONAt Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow and profoundly impact health for humanity.
Learn more at https://www.jnj.com/ or at www.innovativemedicine.jnj.com.
Follow us at @JNJInnovMed.
Janssen Biotech, Inc., is a Johnson & Johnson company.
Cautions Concerning Forward-Looking StatementsThis press release contains “forward-looking statements” as defined in the Private Securities Litigation Reform Act of 1995 related to TALVEY ® (talquetamab), DARZALEX FASPRO® (daratumumab), IMAAVY ® (nipocalimab-aahu), TECVAYLI ® (teclistamab), CARVYKTI ® (ciltacabtagene autoleucel; cilta-cel), bleximenib (JNJ-75276617) and IMBRUVICA® (ibrutinib). The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson’s most recent Annual Report on Form 10-K, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in Johnson & Johnson’s subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov, www.jnj.com, www.investor.jnj.com or on request from Johnson & Johnson. Johnson & Johnson does not undertake to update any forward-looking statement as a result of new information or future events or developments.
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1 Rarediseases.org. Warm Autoimmune Hemolytic Anemia. Available at https://rarediseases.org/rare-diseases/warm-autoimmune-hemolytic-anemia/. Last accessed: May 2026.
2 Rajkumar SV. Multiple Myeloma: 2020 Update on Diagnosis, Risk-Stratification and Management. Am J Hematol. 2020;95(5):548-567.
3 National Cancer Institute. Plasma cell neoplasms. Avaliable at https://www.cancer.gov/types/myeloma/patient/myeloma-treatment-pdq. Last accessed May 2026.
4 City of Hope (2022) Multiple myeloma: Causes, symptoms & treatments. Avaliable at https://www.cancercenter.com/cancer-types/multiple-myeloma. Last accessed: May 2026.
5 International Agency for Research on Cancer (World Health Organization). (2024). Multiple myeloma fact sheet. Avaliable at https://gco.iarc.who.int/media/globocan/factsheets/cancers/35-multiple-myeloma-fact-sheet.pdf. Last accessed: May 2026.
6 SEER Explorer: An interactive website for SEER cancer statistics. Surveillance Research Program, National Cancer Institute. Avaliable at https://seer.cancer.gov/explorer/. Last accessed May 2026.
7 American Cancer Society. What is multiple myeloma? Avaliable at https://www.cancer.org/cancer/multiple-myeloma/about/what-is-multiple-myeloma.html. Last accessed: May 2026.
8 American Cancer Society. Multiple myeloma early detection, diagnosis, and staging. Avaliable at https://www.cancer.org/cancer/types/multiple-myeloma/detection-diagnosis-staging/detection.html. Last accessed May 2026.
9 Kazandjian D. Multiple myeloma epidemiology and survival: a unique malignancy. J Clin Oncol. 2018;36(15):1479–1487. Retrieved from https://pmc.ncbi.nlm.nih.gov/articles/PMC6119139/
10 Tranekær S, et al. Epidemiology of secondary warm autoimmune haemolytic anaemia-A systematic review and meta-analysis. J Clin Med. 2021 Mar 17;10(6):1244.
11 Sudulagunta SR, et al. Warm Autoimmune Hemolytic Anemia: Clinical Profile and Management. J Hematol. 2017 Mar; 6(1): 12–20. Published online 2017 Mar 21. doi: 10.14740/jh303w.
12 National Organization for Rare Disorders, Warm autoimmune Hemolytic Anemia. Available at: https://rarediseases.org/rare-diseases/warm-autoimmune-hemolytic-anemia/. Last accessed: May 2026.
13 Cherif H, Cai Q, Crivera C, Leon A, Rahman I, Leval A, Noel W, Kjellander C. Overall survival and treatment patterns among patients with warm autoimmune hemolytic anemia in Sweden: A nationwide population-based study. Eur J Haematol, 2025 Jan;114(1):129-138.
14 Fattizzo B, Barcellini W. New therapies for the treatment of warm autoimmune hemolytic anemia. Transfusion Medical Reviews. 2022;36(4).
15 The Leukemia & Lymphoma Society. Facts 2022–2023: Updated data on blood cancers. Avaliable athttps://www.lls.org/sites/default/files/2023-08/PS80_Facts_2022_2023.pdf. Last accessed: May 2026.
16 MD Anderson Cancer Center. Acute myeloid leukemia. Avaliable at https://www.mdanderson.org/cancer-types/acute-myeloid-leukemia.html. Last accessed: May 2026.
17 American Cancer Society. Signs and symptoms of acute myeloid leukemia (AML). Avaliable at https://www.cancer.org/cancer/types/acute-myeloid-leukemia/detection-diagnosis-staging/signs-symptoms.html. Last accessed: May 2026.
18 Shimony S, Stahl M, Stone RM. Acute myeloid leukemia: 2023 update on diagnosis, risk‑stratification, and management. Am J Hematol. 2023;98(3):502–526.https://doi.org/10.1002/ajh.26822. Accessed June 2026.
19 TALVEY® U.S. Prescribing Information. Horsham, PA: Janssen Biotech, Inc. August 2023.
20 European Medicines Agency. TALVEY Summary of Product Characteristics. August 2023.
21 DARZALEX FASPRO® U.S. Prescribing Information. 2025. Horsham, PA: Janssen Biotech, Inc.
22 DARZALEX® U.S. Prescribing Information. 2022. Horsham, PA: Janssen Biotech, Inc.
23 IMAAVY® U.S. Prescribing Information. 2025. Horsham, PA: Janssen Biotech, Inc.
24 ClinicalTrials.gov Identifier: NCT04951622. Available at: https://clinicaltrials.gov/ct2/show/NCT04951622. Last accessed: May 2026.
25 ClinicalTrials.gov Identifier: NCT03842189. Available at: https://clinicaltrials.gov/ct2/show/NCT03842189. Last accessed: May 2026.
26 ClinicalTrials.gov Identifier: NCT05327114. Available at: https://www.clinicaltrials.gov/study/NCT05327114. Last accessed: May 2026.
27 ClinicalTrials.gov Identifier: NCT04119050. Available at: https://clinicaltrials.gov/study/NCT04119050. Last accessed: May 2026.
28 ClinicalTrials.gov Identifier: NCT05379634. Available at: https://clinicaltrials.gov/study/NCT05379634. Last accessed: May 2026.
29 ClinicalTrials.gov Identifier: NCT05912517. Available at: https://www.clinicaltrials.gov/study/NCT05912517. Last accessed: May 2026.
30 ClinicalTrials.gov Identifier: NCT04968912. Available at: https://clinicaltrials.gov/study/NCT04968912. Last accessed: May 2026.
31 ClinicalTrials.gov Identifier: NCT04882878. Available at: https://clinicaltrials.gov/study/NCT04882878. Last accessed: May 2026.
32 ClinicalTrials.gov Identifier: NCT06449651. Available at: https://clinicaltrials.gov/study/NCT06449651. Last accessed: May 2026.
33 ClinicalTrials.gov Identifier: NCT06533098 Available at: https://clinicaltrials.gov/study/NCT06533098. Last accessed: May 2026.
34 U.S. FDA Approves TECVAYLI® (teclistamab-cqyv), the First Bispecific T-cell Engager Antibody for the Treatment of Patients with Relapsed or Refractory Multiple Myeloma. Avaliable at https://www.jnj.com/u-s-fda-approves-tecvayli-teclistamab-cqyv-the-first-bispecific-t-cell-engager-antibody-for-the-treatment-of-patients-with-relapsed-or-refractory-multiple-myeloma. Last accessed: May 2025.
35 CARVYKTI® U.S. Prescribing Information. 2025. Horsham, PA: Janssen Biotech, Inc.
36 Genetics Home Reference. Isolated growth hormone deficiency. Avaliable at http://ghr.nlm.nih.gov/condition/isolated-growth-hormone-deficiency. Last accessed: May 2026.
37 Turetsky A, et al. Single cell imaging of Bruton’s tyrosine kinase using an irreversible inhibitor. Scientific Reports. 2014;6:4782.
38 de Rooij MF, et al. The clinically active BTK inhibitor PCI-32765 targets B-cell receptor- and chemokine-controlled adhesion and migration in chronic lymphocytic leukemia. Blood. 2012;119(11):2590-2594.
39 IMBRUVICA® U.S. Prescribing Information, February 2024. Horsham, PA: Janssen Biotech, Inc.
Media contact:Bridget Kimmelbkimmel@its.jnj.com
Oncology Media Relationsoncology_media_relations@its.jnj.com
Investor contact:Jess Margevichinvestor-relations@its.jnj.com
临床3期临床结果临床2期上市批准孤儿药
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 伴有 11q23 异常的急性髓系白血病 | 临床3期 | 美国 | 2025-06-04 | |
| 伴有 11q23 异常的急性髓系白血病 | 临床3期 | 日本 | 2025-06-04 | |
| 伴有 11q23 异常的急性髓系白血病 | 临床3期 | 澳大利亚 | 2025-06-04 | |
| 伴有 11q23 异常的急性髓系白血病 | 临床3期 | 奥地利 | 2025-06-04 | |
| 伴有 11q23 异常的急性髓系白血病 | 临床3期 | 比利时 | 2025-06-04 | |
| 伴有 11q23 异常的急性髓系白血病 | 临床3期 | 巴西 | 2025-06-04 | |
| 伴有 11q23 异常的急性髓系白血病 | 临床3期 | 加拿大 | 2025-06-04 | |
| 伴有 11q23 异常的急性髓系白血病 | 临床3期 | 捷克 | 2025-06-04 | |
| 伴有 11q23 异常的急性髓系白血病 | 临床3期 | 丹麦 | 2025-06-04 | |
| 伴有 11q23 异常的急性髓系白血病 | 临床3期 | 法国 | 2025-06-04 |
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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 |
|---|
临床1期 | NPM1突变急性髓系白血病 NPM1 Alterations | KMT2A Alterations | FLT3 co-mutations | 44 | Bleximenib + intensive chemotherapy | 製觸願鏇憲繭鏇鑰鹹膚(淵壓獵鏇鏇鑰築構糧選) = 醖蓋選積鬱膚淵鏇簾膚 壓衊觸餘網獵廠鬱範願 (製製選顧糧繭餘構觸鑰 ) 更多 | 积极 | 2026-02-04 | |
临床1期 | NPM1突变急性髓系白血病 NPM1 mutations | KMT2A rearrangements | 44 | Bleximenib + '7+3' | 選憲憲製獵齋夢壓鑰獵(蓋蓋糧範糧製窪鑰築繭) = 艱憲壓觸齋夢膚網觸鬱 憲廠繭窪膚製獵襯廠築 (衊築醖鏇憲觸淵顧觸醖 ) | 积极 | 2025-12-06 | |
Bleximenib + '7+3' | 壓膚齋繭獵艱醖壓築窪(鹽蓋壓遞壓網醖膚鏇襯) = 構蓋網餘襯壓鹽夢簾醖 獵蓋網窪窪鹹觸壓範繭 (醖鏇膚艱網獵製築蓋觸 ) 更多 | ||||||
临床1期 | NPM1突变急性髓系白血病 KMT2A | NPM1 | 15 | 獵壓餘製選艱窪鹹廠淵(顧築膚築範醖壓選範鑰) = 構製簾廠窪鬱襯襯蓋製 觸醖鬱醖衊獵壓艱簾獵 (餘夢選築衊糧觸醖顧餘 ) 更多 | 积极 | 2025-12-06 | ||
(prior VEN exposure) | 觸繭淵網膚遞衊顧糧衊(選築壓壓繭壓網製網鬱) = 築憲獵襯獵築餘積糧構 築網醖憲艱遞壓醖蓋製 (鏇糧繭窪鬱餘願醖鑰壓 ) 更多 | ||||||
临床1期 | NPM1突变急性髓系白血病 NPM1m | KMT2Ar | 120 | 築觸積觸膚膚範鑰淵選(築選蓋願壓夢獵顧鹽鑰) = 5 events of differentiation syndrome (DS; 4%) were observed: 4 G2-3; 1 G5 (in a pt with adverse karyotype and progressive disease), which met dose-limiting toxicity (DLT) criteria 願獵淵齋繭醖築鏇鏇艱 (積廠選鬱糧餘憲簾壓遞 ) 更多 | 积极 | 2025-05-14 | ||
N/A | - | - | 淵憲築淵廠醖積構築廠(壓獵艱醖鬱衊選鑰獵艱) = TRAEs (all grades) attributed to bleximenib alone occurred in 18% (4/22) of pts, including thrombocytopenia and neutropenia (2/22; 9% each) and single cases (5% each) of anemia, diarrhea, cerebrovascular accident, dysesthesia and upper abdominal pain. 衊齋鏇壓選憲製壓願遞 (獵願選願簾築繭醖構築 ) 更多 | - | 2024-12-07 | ||
临床1期 | 急性白血病 KMT2A- | NPM1- | 121 | 齋膚醖觸糧壓餘窪壓積(鏇積願鑰衊願襯築膚艱) = Seventeen pts experienced DS (bothKMT2AandNPM1), with 8 (7%) DS events ≥G3, including 2 fatal events. One fatal DS AE occurred in a pt with recurrent DS after rapid dose escalation. 齋構襯膚積築獵鑰願顧 (觸觸醖願繭艱淵壓築願 ) 更多 | 积极 | 2024-12-07 | ||
临床1期 | 急性髓性白血病 KMT2A Mutation | NPM1 Mutation | 45 | 選網獵獵製鏇齋積網鏇(壓網蓋遞憲網餘醖窪淵) = 87% (39/45) of pts experienced ≥1 treatment-related AE (TRAE) attributed to any study treatment (allgrades); nausea (38%), vomiting (31%), and thrombocytopenia (31%) were the most common. 繭鹹淵糧淵夢鑰襯餘夢 (衊衊齋鹹淵鑰鬱簾糧積 ) 更多 | 积极 | 2024-05-14 |
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