预约演示
更新于：2026-04-02
Tebotelimab
特泊利单抗
更新于：2026-04-02
概要
基本信息
药物类型 双特异性抗体 |
别名 Anti-LAG-3 bispecific DART protein、PD-1 x LAG-3 bispecific DART molecule、MGD-013 + [2] |
作用方式 抑制剂 |
作用机制 LAG3抑制剂(淋巴细胞活化基因3蛋白抑制剂)、PD-1抑制剂(细胞程序性死亡-1抑制剂) |
非在研机构 |
最高研发阶段临床2/3期 |
首次获批日期- |
最高研发阶段(中国)临床2/3期 |
特殊审评- |
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结构/序列
Sequence Code 13030948
来源: *****
Sequence Code 13030956
来源: *****
关联
10
项与 特泊利单抗 相关的临床试验NCT04634825
A Phase 2 Open-Label Trial to Evaluate Enoblituzumab in Combination With Retifanlimab or Tebotelimab in the First-Line Treatment of Patients With Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck
This is a Phase 2 study of enoblituzumab combined with either retifanlimab or tebotelimab administered as first-line treatment to patients with recurrent or metastatic squamous cell carcinoma of the head and neck.
开始日期2021-03-17 |
申办/合作机构 |
NCT04653038
An Open-label, Multi-cohort, Multi-center Phase I Study Evaluating the Efficacy and Safety of MGD013 in Patients With Unresectable, Recurrent or Metastatic Malignant Melanoma
This is an open-label, multi-cohort, multi-center Phase I clinical trial to evaluate the efficacy and safety of MGD013 in ① Cohort 1: patients with unresectable, recurrent or metastatic melanoma who have failed prior immune checkpoint inhibitor therapy; ② Cohort 2: patients with untreated, unresectable recurrent or metastatic, mucosal or acral lentiginous melanoma.
开始日期2020-10-29 |
申办/合作机构 |
NCT04212221
A Multicenter, Open-label, Phase I/II Dose Escalation and Expansion Clinical Study to Assess the Safety and Efficacy of MGD013 Monotherapy and in Combination With Brivanib Alaninate (ZL-2301) in Patients With Advanced Liver Cancer
This study consists of two parts: Phase I is a dose escalation study to determine the Recommended Phase II Dose (RP2D) of MGD013 monotherapy and that of MGD013 when in combination with Brivanib Alaninate (ZL-2301) in subjects with advanced liver cancer (including hepatocellular carcinoma and intrahepatic cholangiocarcinoma). Phase II is a dose expansion study and consists of two parts: Part 1 is to assess the safety and efficacy of MGD013 monotherapy and MGD013 in combination with ZL-2301 in subjects with advanced hepatocellular carcinoma (HCC); in Part 2, a therapeutic method (MGD013 monotherapy or MGD013 in combination with ZL-2301, determined by the sponsor according to the obtained data) will be selected for dose expansion study in HCC subjects who have previously failed immune checkpoint inhibitor treatment, to further evaluate the safety and efficacy of the study treatments in the specific group of subjects.
开始日期2020-04-20 |
申办/合作机构 |
100 项与 特泊利单抗 相关的临床结果
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100 项与 特泊利单抗 相关的转化医学
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100 项与 特泊利单抗 相关的专利（医药）
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6
项与 特泊利单抗 相关的文献（医药）2025-07-01·Journal for ImmunoTherapy of Cancer
Therapeutic potential of targeting LAG-3 in cancer
Review
作者: Anderson, Ana Carrizosa ; Davar, Diwakar ; Diaz-Padilla, Ivan
Immune checkpoint inhibitors targeting negative regulatory checkpoints including programmed death-1 (PD-1) and cytotoxic T-lymphocyte-associated protein 4 have produced significant improvements in progression-free survival (PFS) and overall survival in multiple solid tumors. Lymphocyte activation gene 3 (LAG-3) is an inhibitory receptor that is highly expressed by exhausted T cells. Dual blockade of LAG-3 and PD-1 with monoclonal antibodies relatlimab and nivolumab has improved PFS in advanced melanoma, leading to Food and Drug Administration approval for this indication. Concurrently, enthusiasm for targeting LAG-3 has been tempered by negative results in multiple indications, although novel approaches including LAG-3-directed bispecifics tebotelimab continue to demonstrate promise. In this review, we discuss the current understanding of LAG-3 in regulating antitumor immunity and the ongoing state of clinical development of LAG-3-directed agents in cancer.
2025-06-01·CANCER
Tebotelimab plus niraparib in previously treated locally advanced or metastatic solid tumors: A phase 1b dose escalation and expansion study
Article
作者: Cheng, Jing ; Hang, Wenzhao ; Zheng, Yi ; Gao, Yunong ; Wang, Li ; Wu, Xinhong ; Pan, Hongming ; Zheng, Yu ; Hou, Jianmei ; Xu, Rui‐Hua ; Bao, Lequn ; Yan, Min ; Wang, Jufeng ; Lam, Ka On ; Li, Huiping ; Shi, Yanxia ; Zhen, Xiaoa ; Luo, Huiyan ; Qiu, Miao‐Zhen
Abstract:
Background:
This open‐label, single‐arm, phase 1b dose escalation and expansion study (ClinicalTrials.gov identifier NCT04178460) explored the safety, tolerability, and antitumor activity of tebotelimab, a programmed cell death protein 1 × lymphocyte‐activation gene 3 bispecific monoclonal antibody, in combination with niraparib, a poly(adenosine diphosphate ribose) polymerase inhibitor, in patients with gastric cancer, triple‐negative breast cancer (TNBC), biliary tract carcinoma (BTC), and endometrial carcinoma.
Methods:
In the escalation phase, patients with locally advanced or metastatic gastric cancer who failed ≥2 prior systemic treatments received tebotelimab (120, 300, or 600 mg) intravenously once every 2 weeks (Q2W). In the expansion phase, patients with previously treated gastric cancer, TNBC, BTC, or endometrial carcinoma received tebotelimab at the recommended phase 2 dose (RP2D). All patients received niraparib orally once daily with an individualized starting dose.
Results:
From June 11, 2020, to January 29, 2022, 60 patients were enrolled. All received ≥1 dose of the study drug. The RP2D for tebotelimab was determined to be 600 mg Q2W, without dose‐limiting toxicities observed. Grade ≥3 and serious treatment‐emergent adverse events (TEAEs) occurred in 39 patients (65.0%) and 25 patients (41.7%), respectively, with no treatment‐related death. Immune‐related TEAEs occurred in 28 patients (46.7%). In those who had target lesions at baseline and received the RP2D, the investigator‐assessed, confirmed overall response rate per Response Evaluation Criteria in Solid Tumors, version 1.1, was 5.3% (one of 19), 20.0% (two of 10), 8.3% (one of 12), and 0% (zero of three) for gastric cancer, TNBC, BTC, and endometrial carcinoma, respectively.
Conclusions:
Tebotelimab plus niraparib preliminarily demonstrated a tolerated and manageable safety profile and limited antitumor activity in patients with previously treated solid tumors.
2024-04-01·Cancer communications (London, England)
Revealing the secret behind Epstein‐Barr virus‐specific tumor immune contexture
Article
作者: Chu-Xia Deng
Immune checkpoint inhibitor (ICI) therapy has significantly revolutionized cancer treatment across various malignancies, offering distinct and enduring clinical advantages [1]. Gastric cancer (GC) represents a widespread and life-threatening malignancy with substantial global health implications [2]. However, ICI therapy has not produced satisfactory therapeutic responses in GC patients. Presently, monotherapy targeting the programmed cell death protein 1/programmed death-ligand 1 (PD-1/PD-L1) pathway demonstrates clinical efficacy in approximately 10% of GC patients [3]. Exploration of novel immunotherapeutic targets is urgently needed. Notably, Epstein-Barr virus (EBV)-positive GC constitutes a distinct GC subset, exhibiting a remarkably higher objective response rate to anti-PD-1 inhibitors—an intriguing phenomenon [4]. Several studies have found that EBV-positive GC has more innate and adaptive immune cell infiltration than EBV-negative GC [5, 6]. In their recent publication titled "Dynamic single-cell mapping reveals Epstein-Barr virus-imprinted T-cell exhaustion and on-treatment response", Qiu et al. [7] unveiled, for the first time, the cellular basis underlying these remarkably heightened responses in EBV-positive GC patients undergoing immunochemotherapy. Leveraging single-cell profiling technologies, this study deepened our comprehension of the intricate and heterogeneous tumor microenvironment in GC. In this study, the authors conducted a comprehensive characterization of the cellular dynamics of tumor-infiltrating immune cells in GC patients, distinguishing between those with EBV-positive and EBV-negative statuses. EBV-positive GC exhibited a heightened immune phenotype characterized by elevated infiltration of T cells and B cells, highlighting the intricate interplay among EBV infection, multi-cellular ecosystems, and tumor development. EBV-negative GC exhibited an immune-suppressive tumor microenvironment characterized by an abundance of plasma cells, myeloid cells, and mast cells. After treatment, the evaluation of the dynamic changes in EBV-positive GC showed an increased presence of cytotoxic CD8+ T cells and effector memory/memory CD8+ T cells. Notably, there was clear evidence of clonal revival and reinvigoration of CD8+ T cells in EBV-positive GC patients, indicating an active T cell-mediated immune reaction enhancing treatment responses. Furthermore, the authors observed a distinct EBV-imprinted CD8+ T cell population, ISG-15+CD8+ T cells, which exhibited significantly higher expression of interferon-stimulated genes, such as ISG-15, IFIT1-3, RASD2, and MX1. Significantly, ISG-15+CD8+ T cells demonstrated the ability to recognize EBV antigens and coordinated exhausted T cell responses. STARTRAC-tran analysis revealed a robust association between ISG-15+CD8+ T cells and actively proliferating T cells, effector CD8+ T cells (CXCL13+CD8+ T cells) and cytotoxic CD8+ T cells within EBV-positive GC. It is worth mentioning that EBV epitopes were detected within ISG-15+CD8+ T cells, suggesting that this particular cell population consists of T cells specifically targeting EBV. In the context of ICI-responsive GCs, their investigation revealed a significant proportion of ISG-15+CD8+ T clonotypes prior to treatment within the effector T cell populations of EBV-positive tumors after the treatment. In contrast, there was no evidence of re-emerged clonotypes of pre-existing ISG-15+CD8+ T cells in EBV-negative tumors following therapy. More research is required to validate these observations, as these data suggest that ISG-15+CD8+ T cells function as intermediate precursor exhausted T cells, and fluctuations in the proportions of exhausted T cells and effector T cell populations are associated with the responses to PD-1-based therapies. By deciphering the association between the dynamics of ISG-15+CD8+ T cells and clinical response following treatment, the authors discovered that ISG-15+CD8+ T cells could be a significant predictor of a positive treatment response. Their data also revealed that an elevated baseline presence of intratumoral ISG-15+CD8+ T cells may be indicative of a potential benefit from immunochemotherapy. Moreover, ISG15+CD8+ T cell population played a crucial role in the transition to T cell exhaustion, accompanied by escalating levels of LAG-3. In comparison to EBV-negative GC, LAG-3 expression may be upregulated due to an exhaustion expression program triggered by EBV-associated activation. This suggests that LAG-3 could potentially act as a crucial checkpoint for revitalizing the terminal exhausted/dysfunctional T cell subset in EBV-positive GC. Moreover, the author described the results of two clinical trials assessing the efficacy of MGD013 and KL-A289 in treating metastatic GC patients. One patient who participated in a phase Ib clinical trial of MGD013, a bi-specific antibody targeting PD-1 and LAG-3, achieved partial response within a month of treatment. Another patient with spleen and multiple lymph node metastases was enrolled in a phase I clinical trial of KL-A289, a LAG-3 inhibitor. After two cycles of KL-A289 treatment, the patient achieved stable disease followed by a significant decrease in the EBV-DNA copy number. These data support further exploration of a LAG-3 antibody in patients with EBV-positive GC and other EBV-associated tumors. Importantly, their longitudinal single-cell view on EBV-positive GC during treatment provided physicians with a direct view on response to treatment, although more detailed investigations on the early effects of anti-LAG-3 and anti-PD-1/PD-L1 are still required. However, the use of biopsies does not allow for direct comparison of the effects of different perturbations, which complicates efforts to answer mechanistic questions. Therefore, alternative approaches that enable direct comparison of the effects of different perturbations should be considered to address this limitation. Of note, ex vivo tumor culture systems that maintain the tumor microenvironment and architecture as found in the patient, but that enable perturbation by immunotherapies, offer a unique opportunity to study the dynamics of treatment response and intrinsic properties of a tumor [8]. In particular, air-liquid interface organoids consisting of both tumor and stroma components have been shown to enhance the function of intratumoral T cells on PD-1 blockade [9]. Similarly, various ex vivo models such as patient-derived tumor fragment platform [10], microfluidic cancer-on-chip platform [11], and three-dimensional tumor slice culture platform [12] have been documented with the potential to assess the effect of various antitumor treatment strategies. Using ex vivo tumor culture systems, it is feasible and valuable to assess the effect of anti-LAG3 treatment on the tumor microenvironment and thereby directly test the functional importance of specific cell types in the treatment response. Overall, this study represents a significant advancement in our understanding of the EBV-specific tumor immune contexture. The authors propose that the ISG15+CD8+ T cell signature may predict a good response to immunotherapy. More importantly, their findings reveal the necessity of dual blockade of PD-1 and LAG-3 in EBV-associated GC (Figure 1). The comprehensive insights gained from this research pave the way for future research aimed at developing innovative and personalized therapeutic approaches in the fight against cancer. Chu-Xia Deng wrote and proofread the manuscript. The author would like to thank Faculty of Health Sciences, University of Macau for the support. This work was supported by the Macao Science and Technology Development Fund (0009/2022/AKP). The authors declare that they have no competing interests. Not applicable. Not applicable. Not applicable.
50
项与 特泊利单抗 相关的新闻（医药）2026-03-27
康心药物临床试验
康心药物临床试验
【胆管癌】
患者招募
patient enrolment for drug clinical trials
康心药物临床试验患者招募中心：依托于国家疾病防治中心，为各类癌症肿瘤疾病患者、血液疾病患者、慢性疾病患者、传染病疾病患者、罕见病等患者，提供专业国内外药物临床试验免费治疗项目的咨询、药物查询，项目报名、匹配、筛选、对接、入组、帮扶等服务。为患者创造出一条崭新的出路和新的选择。
01
癌症肿瘤项目
晚期胆管癌-前沿医学新药项目
——TQB2450注射液联合安罗替尼胶囊Ib期临床研究
一、基本信息
药物名称：TQB2450注射液、盐酸安罗替尼胶囊（福可维）
药物类型：TQB2450注射液为生物制品，盐酸安罗替尼胶囊为化学药物
适应症：晚期胆管癌（经组织学或病理学确诊的不能手术切除或转移性胆管癌，包括肝内胆管癌、肝外胆管癌和胆囊癌）
试验专业题目：TQB2450联合安罗替尼胶囊在晚期胆管癌患者中药代动力学、安全性及有效性的Ib期临床研究
试验分类：药代动力学/药效动力学试验
试验分期：Ib期
其他说明：无
设计类型：单臂试验
随机化：非随机化
盲法：开放
试验范围：国内试验
受试者信息：年龄18岁(最小年龄)至无岁(最大年龄)，性别：男+女，健康受试者：无
二、治疗原理
1. 药物剂型优势：本试验采用TQB2450注射液（静脉滴注）联合盐酸安罗替尼胶囊（口服）的给药方式，两种剂型互补，静脉给药可确保TQB2450快速吸收、精准递送，口服胶囊剂型便捷易服，便于患者长期坚持治疗，同时两种药物协同作用，提升治疗的便捷性与安全性。
2. 抗肿瘤作用机制：TQB2450注射液作为生物制品，可通过调节机体免疫功能，激活免疫细胞对胆管癌肿瘤细胞的杀伤能力；盐酸安罗替尼胶囊可抑制VEGFR靶点，阻断肿瘤血管生成，减少肿瘤营养供应，二者联合可从免疫调节、抗血管生成双重途径抑制肿瘤细胞增殖、诱导肿瘤细胞凋亡，精准作用于晚期胆管癌病灶，控制疾病进展。
3. 临床治疗价值：针对既往标准一线或一线以上化疗失败、不适合标准一线治疗的晚期胆管癌患者，该联合用药方案可提供新的治疗选择。本次Ib期临床研究核心目的是评价TQB2450注射液联合安罗替尼胶囊治疗晚期胆管癌患者的安全性、初步疗效及免疫原性，同时探索相关生物标志物，为后续临床试验设计和给药方案确定提供科学依据，改善晚期胆管癌患者的治疗预后。
三、入选标准
1. 年满18周岁；ECOG体力状况0~1分；预计生存期≥3个月；
2. 经组织学或病理学确诊的不能手术切除或转移性胆管癌患者，包括肝内胆管癌（IHCC）、肝外胆管癌（EHCC）和胆囊癌（GBC）；
3. 患者至少具有一个可评估病灶（RECIST 1.1：包括可测量病灶与不可测量病灶）；
4. 患者既往标准一线或一线以上化疗失败或不适合标准一线治疗，一线化疗失败定义为：治疗过程中或末次治疗后6个月内出现疾病进展，或治疗过程中因为毒副作用不可耐受；允许前期进行新辅助或辅助化疗，若新辅助/辅助治疗期间或治疗结束后6个月内出现疾病进展/复发，视为一线全身化疗失败；既往一线标准化疗不包含抗血管生成的小分子抑制剂或者单抗，队列1患者还不包含肿瘤免疫相关药物；拒绝标准一线治疗者也可纳入；
5. 实验室检查需满足：血常规检查（血红蛋白Hb≥90g/L，14天内未输血；绝对中性粒细胞计数NEUT≥1.5×10⁹/L；血小板PLT≥100×10⁹/L）；生化检查（谷丙转氨酶ALT及谷草转氨酶AST≤2.5×ULN，肿瘤肝脏转移者≤5×ULN；血清总胆红素TBIL≤2×ULN，Gilbert综合症患者≤3×ULN；血清肌酐Cr≤1.5×ULN，或肌酐清除率＞50μmol/L）；凝血功能（活化部分凝血活酶时间APTT、国际标准化比值INR、凝血酶原时间PT≤1.5×ULN）；多普勒超声评估（左室射血分数LVEF≥50%）；
6. 女性应同意在研究期间和研究结束后6个月内采用避孕措施（如宫内节育器、避孕药或避孕套），入组前7天内血清或尿妊娠试验阴性，且为非哺乳期患者；男性应同意在研究期间和研究期结束后6个月内采用避孕措施；
7. 患者自愿加入本研究，签署知情同意书，依从性好。
四、排除标准
1. 既往接受了包括盐酸安罗替尼胶囊在内的VEGFR靶点抑制剂药物治疗的患者（队列1和队列2），或使用了其他PD-1/PD-L1/CTLA-4抗体治疗或其他针对PD-1/PD-L1/CTLA-4的免疫治疗（队列1）；
2. 其他单克隆抗体给药后出现重度超敏反应者（队列1）；
3. 5年内受试者既往或同时患有其它恶性肿瘤（已治愈的皮肤基底细胞癌和宫颈原位癌除外）；
4. 存在任何活动性自身免疫病或有自身免疫病病史（如自身免疫性肝炎、间质性肺炎、肠炎、血管炎、肾炎；需要支气管扩张剂进行医学干预的哮喘不能纳入）；但无需全身治疗的白癜风、银屑病、脱发，控制良好的I型糖尿病，经替代治疗甲状腺功能正常的甲减患者允许入组；
5. 需使用免疫抑制剂、或全身、或可吸收的局部激素治疗以达到免疫抑制目的（剂量>10mg/天泼尼松或其他等疗效激素），并首次给药2周内仍在继续使用的；
6. 具有影响口服药物的多种因素（比如无法吞咽、胃肠道切除术后、慢性腹泻和肠梗阻等）者；
7. 不能控制的需要反复引流的胸腔积液、心包积液或腹水；
8. 不管严重程度如何，存在任何出血体质迹象或病史的患者；在首次给药前4周内，出现任何出血或流血事件≥CTCAE 3级的患者；或存在未愈合创口、骨折、胃及十二指肠活动性溃疡、溃疡性结肠炎等消化道疾病或未切除的肿瘤存在活动出血，或研究者判定的可能引起消化道出血、穿孔的其他状况；
9. 首次给药前4周内出现不可控制的脑转移症状、脊髓压迫、癌性脑膜炎，或筛选时CT或MRI检查发现脑或者软脑膜的疾病者；
10. 研究治疗开始前4周内接受过已获批的或在研的抗肿瘤治疗，包括但不限于化疗、手术、放疗、生物靶向治疗、介入治疗、免疫治疗和抗肿瘤中药治疗（以中药说明书适应症为准，经过2周洗脱期可入组）；口服靶向药不足5个药物半衰期者、口服氟尿嘧啶类药物不足14天、丝裂霉素C以及亚硝基脲不足6周者，先前治疗引起的不良事件（脱发除外）未恢复至≤CTCAE 1度的患者也需排除；
11. 存在任何重度和/或未能控制的疾病的患者，包括：a)血压控制不理想（收缩压≥150 mmHg或舒张压≥90mmHg）；b)首次给药6个月内出现不稳定心绞痛、心梗、≥2级充血性心功能衰竭，或需治疗的心律失常（包括QTc≥480ms）；c)活动性或未能控制的严重感染（≥CTC AE 2级感染）；d)已知有临床意义的肝病病史，包括病毒性肝炎，HBV携带者需排除活动性HBV感染（HBV DNA阳性>1×10³拷贝/mL或>500 IU/mL），HCV感染且HCV RNA阳性（>1×10³拷贝/mL或>100 IU/mL），或其它失代偿性肝病、慢性肝炎需接受抗病毒治疗；e)HIV检测阳性；f)糖尿病控制不佳（空腹血糖≥CTCAE 2级）；g)尿常规提示尿蛋白≥++，且证实24小时尿蛋白定量＞1.0 g者；
12. 首次给药4周内接种过预防疫苗或减毒疫苗；首次给药4周内接受过粒细胞集落刺激因子（G-CSF）或粒细胞-巨噬细胞集落刺激因子（GM-CSF）治疗者（队列1）；
13. 经研究者判断，受试者有其他可能导致本研究被迫中途终止的因素，如其他严重疾病（含精神疾病）需要合并治疗、有严重的实验室检查异常、伴有家庭或社会等因素影响受试者安全或资料及样品收集。
五、试验分组
试验药1：中文通用名:TQB2450注射液；英文通用名:TQB2450 injection；商品名称:NA；剂型:注射剂；规格:10mL：100mg、20mL：600mg；用法用量:静脉滴注，一次1200mg，21天一次；用药时程:最长使用时间不超过24个月，按方案规定使用。
试验药2：中文通用名:盐酸安罗替尼胶囊；英文通用名:Anlotinib Hydrochlride Capsules；商品名称:福可维；剂型:胶囊剂；规格:8mg、10mg、12mg；用法用量:连续口服2周停1周；用药时程:21天为一治疗周期，按方案规定使用。
对照药:无
六、患者获益
1. 免费体检；
2. 有一定的交通补助；
3. 免费使用试验用药品（TQB2450注射液、盐酸安罗替尼胶囊）及相关治疗、检测；
4. 就诊知名三甲医院，全程专家团队长期跟踪服务，持续监测晚期胆管癌治疗效果与安全性，享受规范的临床诊疗服务。
七、需要资料
肿瘤项目需要的资料（通用）
1. 详细记录了患者治疗经过的出院记录（距离现在时间最近的一次）；
2. 最近两次复查的CT或者核磁报告；
3. 确诊时的病理报告以及基因检测（没做基因检测的话就不发）；
4. 血常规，血生化，出凝血，肿瘤标志物，传染病检查（乙肝艾滋梅毒）。
八、研究中心所在地区
河北、山西、黑龙江、吉林、辽宁、江苏、浙江、安徽、福建、江西、山东、河南、湖北、湖南、广东、海南、四川、贵州、云南、陕西、甘肃、青海、北京、上海、天津、重庆等上百个城市均有项目中心。
全国上百家三甲医院负责主治，上千家上市药企供药，根据晚期胆管癌患者实际病情和项目匹配筛选安排。招募符合条件的患者，您可直接联系我们进行匹配筛选。
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02
癌症肿瘤项目
携带FGFR2重排的晚期胆管癌-前沿医学新药项目
——Pemigatinib片III期临床研究
一、基本信息
药物名称：Pemigatinib片
药物类型：化学药物
适应症：携带FGFR2重排的、手术不可切除或转移性胆管癌（针对既往未接受过治疗的晚期胆管癌患者）
试验专业题目：一项评价Pemigatinib对比吉西他滨联合顺铂一线治疗携带FGFR2重排的、手术不可切除或转移性胆管癌患者疗效和安全性的随机、开放、阳性药物对照、多中心III期临床研究
试验分类：安全性和有效性
试验分期：III期
其他说明：无
设计类型：平行分组
随机化：随机化
盲法：开放
试验范围：国际多中心试验
受试者信息：年龄18岁(最小年龄)至无上限(最大年龄)，性别：男+女，健康受试者：无
二、治疗原理
1. 药物剂型优势：本试验所用Pemigatinib片为口服片剂，剂型便捷、服用稳定，相较于静脉注射类药物，可显著提升患者治疗的依从性，减少给药相关的侵入性损伤，同时药物吸收平稳，可维持有效血药浓度，为晚期胆管癌患者提供更具可及性的治疗选择。
2. 抗肿瘤作用机制：Pemigatinib是针对FGFR（成纤维细胞生长因子受体）的小分子抑制剂，可特异性阻断携带FGFR2重排的胆管癌肿瘤细胞的信号传导通路，抑制肿瘤细胞增殖、迁移与血管生成，诱导肿瘤细胞凋亡，精准杀伤FGFR2重排阳性的胆管癌病灶，有效控制疾病进展。
3. 临床治疗价值：针对携带FGFR2重排的手术不可切除或转移性胆管癌患者，Pemigatinib片可提供一线靶向治疗新选择。本次III期临床研究核心目的是对比Pemigatinib与吉西他滨联合顺铂的疗效和安全性，为该类患者的精准治疗提供高级别循证依据，改善患者长期生存预后。
三、入选标准
1. 能够理解并且愿意签署研究的书面知情同意书（ICF）；
2. 签署ICF时≥18岁的男性或女性受试者；日本的合法未成年受试者需要获得父母的书面同意；
3. 经组织学或细胞学证实为胆管癌，既往未接受过治疗，且视为手术不可切除和/或转移（按照AJCC癌症分期手册[AJCC 2010]为IV期）；
4. 经CT或MRI评估且根据RECIST v1.1标准确定为放射性可测量或可评价疾病；
5. ECOG体能状态为0~1分；
6. 自愿签署知情同意书，依从性良好，可随访，自愿遵守研究规定。
四、排除标准
1. 既往针对不可手术切除和/或转移性疾病接受过全身抗癌治疗（不包括在入组前至少6个月完成辅助/新辅助治疗的患者、接受过经动脉化疗栓塞或选择性体内放射治疗但在入组前观察到放射学进展的局部晚期患者，或接受了1个周期的吉西他滨 + 顺铂治疗的受试者[接受研究药物的开始时间{第1周期第1天}必须距离末次吉西他滨 + 顺铂给药至少14天且≤4周{28天}]，该情况不适用于中国的研究中心）；
2. 对于存在肝硬化的受试者，为Child-Pugh B和C级（注：对于胆管癌导致的（例如：存在腹膜转移）腹水，不应归纳在评分中）；
3. 筛选时既往治疗相关毒性＞1级；
4. 除接受本研究规定的抗癌治疗外，还同时进行其他抗癌治疗者（包括化疗、放疗、手术、免疫疗法、生物疗法、激素治疗、试验性治疗或肿瘤栓塞术等）；
5. 有可能接受根治性手术治疗者；
6. 研究者认为受试者存在其他原因而不适合参加本临床研究。
五、试验分组
试验药：中文通用名:Pemigatinib片；英文通用名:Pemigatinib Tablets；商品名称:PEMAZYRE；剂型:片剂；规格:4.5mg、9mg、13.5mg（剂量调整时使用）；用法用量:13.5mg QD，21天为一个疗程；用药时程:直至疾病进展，按方案规定使用。
对照药：中文通用名:注射用吉西他滨+注射用顺铂；英文通用名:Gemcitabine Hydrochloride for Injection + Cisplatin For Injection；商品名称:健择+注射用顺铂；剂型:注射剂+注射用粉针剂；规格:1g/支、200mg/支（吉西他滨）；20mg/支（顺铂）；用法用量:吉西他滨1000mg/m²，每周期D1及D8给药；顺铂25mg/m²，每周期D1及D8给药；用药时程:8个周期，按方案规定使用。
六、患者获益
1. 免费体检；
2. 有一定的交通补助；
3. 免费使用试验用药品（Pemigatinib片或吉西他滨联合顺铂）及相关治疗、检测；
4. 就诊知名三甲医院（含国际多中心研究机构），全程专家团队长期跟踪服务，持续监测携带FGFR2重排的晚期胆管癌治疗效果与安全性，接受国际前沿的靶向治疗方案。
七、需要资料
肿瘤项目需要的资料（通用）
1. 详细记录了患者治疗经过的出院记录（距离现在时间最近的一次）；
2. 最近两次复查的CT或者核磁报告；
3. 确诊时的病理报告以及FGFR2基因检测报告（必须提供，为入组关键依据）；
4. 血常规，血生化，出凝血，肿瘤标志物，传染病检查（乙肝艾滋梅毒）。
八、研究中心所在地区
本研究为国际多中心试验，全球多国家、多地区开展，国内涵盖北京、上海、广州、南京、杭州、成都、武汉等数十个核心城市的知名三甲医院。
全国上百家三甲医院负责主治，上千家上市药企供药，根据携带FGFR2重排的晚期胆管癌患者实际病情和项目匹配筛选安排。招募符合条件的患者，您可直接联系我们进行匹配筛选。
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03
癌症肿瘤项目
晚期胆管癌-前沿医学新药项目
——M7824（Bintrafusp alfa）II/III期临床研究
一、基本信息
药物名称：M7824（Bintrafusp alfa）
药物类型：生物制品
适应症：晚期胆管癌（针对局部晚期或转移性胆管癌患者，包括肝内胆管癌、肝外胆管癌、胆囊癌和壶腹部癌）
试验专业题目：吉西他滨加顺铂联合或不联合Bintrafusp alfa（M7824）方案作为胆管癌一线治疗的II/III期多中心、随机、安慰剂对照研究
试验分类：安全性和有效性
试验分期：其它（其他说明:II期+III期）
其他说明：无
设计类型：平行分组
随机化：随机化
盲法：双盲
试验范围：国际多中心试验
受试者信息：年龄18岁(最小年龄)至无上限(最大年龄)，性别：男+女，健康受试者：无
二、治疗原理
1. 药物剂型优势：本试验所用M7824为无菌浓缩注射液，经稀释后静脉注射给药，剂型稳定、生物利用度高，相较于传统化疗药物，其靶向性更强，全身暴露更可控，同时可减少对正常组织的非特异性损伤，提升治疗的安全性与患者耐受性。
2. 抗肿瘤作用机制：M7824是一种双功能融合蛋白，可同时靶向PD‑L1和TGF‑β两大免疫调节通路，通过阻断PD‑1/PD‑L1通路解除T细胞免疫抑制，同时抑制TGF‑β介导的免疫抑制与肿瘤间质纤维化，双重机制激活机体自身免疫系统，精准杀伤胆管癌肿瘤细胞，有效控制肿瘤进展。
3. 临床治疗价值：针对未曾接受化疗、免疫治疗的晚期或转移性胆管癌患者，M7824联合吉西他滨及顺铂的方案可提供一线治疗新选择。本次II/III期临床研究核心目的是评估该联用方案对比安慰剂联合吉西他滨加顺铂方案对患者总生存期和无进展生存期的影响，为晚期胆管癌的免疫联合治疗提供高级别循证依据，改善患者长期生存预后。
三、入选标准
1. 受试者在签署知情同意书时至少年满18岁（日本和中国台湾地区，≥20岁或法律认为已成年的年龄）。在日本，如果受试者年龄<20岁但≥18岁，除受试者知情同意书外，还需父母或监护人的知情同意书方可参加研究；
2. 组织学或细胞学确认的局部晚期或转移性胆管癌（BTC），包括肝内CCA、肝外CCA、胆囊癌和壶腹部癌（需收集壶腹癌组织学起源）；排除吉西他滨或顺铂治疗不被视为标准治疗的组织学亚型（如肉瘤样肿瘤或HCC混合型）；
3. 局部晚期或转移性BTC以前未曾接受化疗、免疫治疗和放射介入治疗（经动脉内化疗栓塞、经动脉内栓塞、经动脉内输注）；新辅助或辅助治疗结束至少6个月后疾病复发的受试者可参加研究；
4. 研究性干预首次给药前，提供肿瘤组织（原发性或转移性，新鲜或保存的活检标本）；安全性导入部分除外，不接受刷片细胞学和细胞块；肿瘤组织需适合用于生物标志物评估；
5. 至少具有一个根据RECIST 1.1判断为可测量的病灶；安全性导入阶段的受试者不要求基线时有可测量病变；
6. 进入研究时和给药前第1周第1天，ECOG体能状态为0或1分；
7. 研究者判断预期寿命≥12周；
8. 进入研究时和给药前第1周第1天具有充分的造血功能，定义为：白细胞计数≥2.0 x 10^9 /L，中性粒细胞绝对计数≥1.5 x 10^9 /L，淋巴细胞计数≥0.5 x 10^9 /L，血小板计数≥100 x 10^9 /L，血红蛋白（Hgb）≥9 g/dL；既往曾输血的受试者若进入研究时Hgb稳定≥9 g/dL，可参加研究；
9. 具有足够的肝功能，即总胆红素水平≤1.5×正常值上限（ULN），谷草转氨酶水平≤3.0×ULN，谷丙转氨酶水平≤3.0×ULN；肝脏受累的受试者可接受谷草转氨酶≤5.0 x ULN和谷丙转氨酶≤5.0 x ULN；
10. 具有足够的肾功能，定义为根据Cockcroft‑Gault公式或24小时尿液收集样本的CrCl检测估算得出肌酐清除率（CrCl）≥50 mL/ min；CrCl （mL/min） = （140-年龄）x 体重（kg）/（72 x 血清肌酐 [Crjaffe]）；如果是女性则x 0.85；如果通过酶法检测肌酐，需加0.2，采用以下公式：Crjaffe = 0.2 + Crenzyme；
11. 白蛋白≥2.8 g/dL；
12. 足够的凝血功能，即凝血酶原时间或国际标准化比率≤1.5 x ULN，除非受试者正在接受抗凝治疗；
13. 乙肝病毒（HBV）脱氧核糖核酸（DNA）阳性受试者，进入研究时必须接受治疗并服用稳定剂量的抗病毒药物（恩替卡韦、替诺福韦或拉米夫定；不允许阿德福韦或干扰素），并接受有计划的监测和管理，包括基线HBV DNA含量；接受活性丙肝病毒（HCV）治疗的受试者，进入研究时需使用稳定的药物剂量，并接受有计划的监测和管理；
14. 男性或女性受试者需满足避孕要求：男性受试者干预期间以及研究干预末次给药后至少4个月内，同意避免捐献精子，且避免任何导致射精的活动，或使用男用避孕套；当与目前未妊娠的育龄妇女发生性关系时，应建议她采用一种年失败率<1%的高效避孕方法；女性受试者非妊娠期或哺乳期，且至少符合以下条件之一：不是育龄期女性；或育龄期女性在第一次研究干预前、干预期间、研究干预末次给药后至少65天内采用年失败率<1%的高效避孕方法（使用激素避孕法者，需完成至少一次为期4周的口服避孕药周期且已/开始月经期，或已使用长效避孕药/延长周期口服避孕药至少28天，并记录妊娠试验阴性）；育龄妇女从开始给药直至研究干预停止后2个月内，避免捐献卵子；研究治疗第一次给药前24小时内的血清或高敏感性尿液妊娠试验结果阴性，尿检不明确时需进行血清妊娠试验；
15. 能够提供签署的知情同意书，遵循知情同意书（ICF）和本方案中所列出的要求和限制条件。
四、排除标准
1. 既往和/或间发性癌症（已治愈且超过3年无复发的癌症或已治愈性治疗的早期癌症除外），包括但不限于原位宫颈癌、浅表性非侵袭性膀胱癌、基底细胞癌、原位鳞状细胞癌，或局限于粘膜层且已内镜下切除的胃肠道肿瘤；
2. 与恶性肿瘤无关的快速临床恶化，研究者认为在研究入组和第1周第1天给药前可能导致受试者无法耐受治疗或研究操作；
3. 症状性中枢神经系统（CNS）转移的受试者；有已治疗的CNS转移（手术治疗或放疗）病史的受试者，除非据判断在治疗后已完全恢复；
4. 接受任何器官移植（包括同种异体干细胞移植），无需免疫抑制的移植（如角膜移植、头发移植）除外；
5. 重大急性或慢性感染，包括：已知人类免疫缺陷病毒（HIV）或获得性免疫缺陷综合征检测阳性史（筛选时无需检测，怀疑感染拒绝检测需与医学监查员讨论）；活动性结核（有临床症状、体格检查或放射影像学发现）；未控制的胆道感染（胆道梗阻需通过支架置入术或经皮穿肝胆管引流术（PTBD）解除，入组时无持续感染证据）；筛选期间需要全身治疗的活动性细菌、真菌或病毒感染（乙肝和丙肝除外）；
6. 接受免疫刺激性药物可能恶化的活动性自体免疫疾病；不需要免疫抑制治疗的1型糖尿病、白癜风、脱发、银屑病、甲状腺功能减退或亢进的受试者可入选；需要皮质类固醇激素替代治疗且剂量≤10 mg泼尼松或等效剂量/天的受试者可入选；局部、鼻内、眼内或吸入途径应用甾体类激素可接受；
7. 间质性肺病的病史或目前合并间质性肺病；
8. 已知对bintrafusp alfa或其制品有过敏反应史，或已知对单克隆抗体有严重过敏反应（≥3级美国国立癌症研究所（NCI）不良事件通用术语标准（CTCAE）5.0版），任何过敏性休克史，或近期（5个月内）未控制的哮喘病史；
9. 临床明显心血管/脑血管疾病，包括入组前<6个月内的脑血管意外/卒中、心肌梗死，不稳定性心绞痛，充血性心衰（纽约心脏协会分级≥II级），或严重心律失常；
10. 其他重度、急性或慢性医学状况，包括免疫性结肠炎、炎症性肠病、免疫性肺炎或精神疾病（近期1年内或活跃的自杀意念或行为）；有出血倾向病史或近期大出血事件，研究者判断研究干预有高风险的受试者；
11. 随机分组前30天内，因慢性阻塞性肺病加重或其他呼吸系统疾病而需要住院治疗或无法进行研究治疗；
12. 肝移植候选者以及在医学可接受的时期内能够接受移植的受试者；
13. 合用不允许使用的药物；随机前>6个月已完成既往辅助治疗的受试者符合标准；
14. 既往使用过作用于T细胞共调节蛋白（免疫检查点）的任何抗体/药物，包括但不限于抗PD-1、抗PD-L1、抗细胞毒性T淋巴细胞相关抗原4（CTLA-4）抗体、抗-4-1BB抗体（含局部用药）；
15. 既往使用过作用于TGFβ/TGFβ受体的任何抗体/药物；
16. 过去28天内接受过除局灶性姑息性骨放疗外的放疗；
17. 研究干预开始前7天内，接受免疫抑制剂全身治疗；或研究干预开始前28天内，使用任何研究性药物；
18. 研究干预给药前4周内使用活疫苗；
19. 同时参加任何干预性临床研究；
20. 研究者认为受试者无法耐受CT或核磁共振成像（MRI），和/或对造影剂过敏；
21. 研究干预开始前28天内接受大手术（不包括既往的诊断性活检以及为了解除胆管梗阻而进行的支架放置/PTBD）；
22. 妊娠或哺乳期女性；
23. 已知酒精或药物滥用；
24. 受试者无民事行为能力或为限制行为能力；
25. 研究者认为受试者存在其他原因而不适合参加本临床研究。
五、试验分组
试验药：中文通用名:M7824；英文通用名:Bintrafusp alfa (M7824)；商品名称:NA；剂型:无菌浓缩注射液；规格:10 mg/mL；用法用量:无菌用水稀释后静脉注射，每三周一次，每次2400mg，用药时长1-2小时；用药时程:共15个周期（43周），直到治疗结束，按方案规定使用。
对照药：中文通用名:M7824安慰剂注射液；英文通用名:M7824 Placebo；商品名称:NA；剂型:无菌浓缩注射液；规格:不适用；用法用量:无菌用水稀释后静脉注射（成分：海藻糖二水合物, L-组氨酸, 氯化钠, L-甲硫氨酸, 聚山梨酯20（吐温20）, 盐酸, 注射用水），每三周一次，用药时长1-2小时；用药时程:共15个周期（43周），直到治疗结束，按方案规定使用。
六、患者获益
1. 免费体检；
2. 有一定的交通补助；
3. 免费使用试验用药品（M7824或M7824安慰剂联合吉西他滨及顺铂）及相关治疗、检测；
4. 就诊知名三甲医院（含国际多中心研究机构），全程专家团队长期跟踪服务，持续监测晚期胆管癌治疗效果与安全性，接受国际前沿的免疫联合治疗方案。
七、需要资料
肿瘤项目需要的资料（通用）
1. 详细记录了患者治疗经过的出院记录（距离现在时间最近的一次）；
2. 最近两次复查的CT或者核磁报告；
3. 确诊时的病理报告以及相关生物标志物评估所需的肿瘤组织标本（新鲜或保存的活检标本，不接受刷片细胞学和细胞块）；
4. 血常规，血生化，出凝血，肿瘤标志物，传染病检查（乙肝艾滋梅毒），肝肾功能相关检测报告。
八、研究中心所在地区
本研究为国际多中心试验，全球多国家、多地区开展，国内涵盖北京、上海、广州、南京、杭州、成都、武汉、济南、西安、长沙等数十个核心城市的知名三甲医院。
全国上百家三甲医院负责主治，上千家上市药企供药，根据晚期胆管癌患者实际病情和项目匹配筛选安排。招募符合条件的患者，您可直接联系我们进行匹配筛选。
官方渠道：扫码加微信咨询详情或报名
04
癌症肿瘤项目
晚期胆管癌（肝内胆管癌）-前沿医学新药项目
——MGD013注射液I/II期临床研究
一、基本信息
药物名称：MGD013注射液
药物类型：生物制品
适应症：晚期胆管癌（含肝内胆管癌，同时涵盖肝细胞癌；针对不适合手术或局部治疗，或经手术和/或局部治疗后进展的晚期肝内胆管癌患者）
试验专业题目：多中心、开放、I/II期剂量递增和剂量扩展研究，评估MGD013单药及MGD013联合丙氨酸布立尼布在晚期肝癌患者中的安全性和有效性
试验分类：安全性和有效性
试验分期：其它（其他说明:I/II期）
其他说明：无
设计类型：平行分组
随机化：非随机化
盲法：开放
试验范围：国内试验
受试者信息：年龄18岁(最小年龄)至75岁(最大年龄)，性别：男+女，健康受试者：无
二、治疗原理
1. 药物剂型优势：本试验所用MGD013注射液为静脉输注剂型，搭配口服丙氨酸布立尼布片，两种剂型互补，给药方式灵活，静脉输注可确保药物快速起效，口服制剂便于患者长期遵医嘱用药，提升治疗依从性，同时降低给药相关不良反应。
2. 抗肿瘤作用机制：MGD013注射液联合丙氨酸布立尼布片采用联合治疗方案，通过协同作用靶向晚期肝内胆管癌肿瘤细胞，精准作用于肿瘤相关靶点，抑制肿瘤细胞增殖、阻断肿瘤血管生成，同时激活机体免疫应答，双重作用杀伤肿瘤细胞，有效控制肝内胆管癌病灶进展，延缓疾病恶化。
3. 临床治疗价值：针对不适合手术或局部治疗、经手术和/或局部治疗后进展的晚期肝内胆管癌患者，MGD013单药及联合丙氨酸布立尼布方案可提供新的治疗选择。本次I/II期临床研究核心目的是明确II期推荐剂量，评估该药物单药及联合用药的安全性和有效性，为晚期肝内胆管癌患者提供更具针对性的治疗方案，改善患者生存质量与预后。
三、入选标准
1. 自愿签署知情同意书，依从性良好，可随访，自愿遵守研究规定；
2. 18-75岁，性别不限；
3. 经病理组织/细胞学或临床诊断确诊的晚期肝内胆管癌（I期可包括混合型肝细胞癌-胆管细胞癌），不适合手术或局部治疗，或经过手术和/或局部治疗后进展；
4. 根据RECIST v1.1标准证实具有至少一个可测量病灶；
5. I期患者应至少接受过一线系统治疗（包括免疫检查点抑制剂、分子靶向药物或系统化疗，单药或联合治疗），治疗失败（经影像学确认进展）或研究者评估不耐受；
6. 前一次抗肿瘤治疗结束距开始本研究用药≥2周，且既往治疗相关不良事件恢复至CTCAE≤1级；既往接受免疫检查点抑制剂治疗的患者，若发生过免疫相关的内分泌腺体病，需应用激素替代治疗控制良好；
7. I期：Child-Pugh A级；
8. ECOG体力状态评分为0或1；
9. 预期生存时间≥12周；
10. 患有慢性HBV感染的受试者HBV-DNA必须＜500 IU/ml，且在研究治疗开始之前至少接受14天抗HBV治疗（如恩替卡韦、替诺福韦等）且愿意在研究期间全程接受抗病毒治疗；丙型肝炎病毒（HCV）核糖核酸（RNA）阳性患者必须接受标准抗病毒治疗且肝功能在CTCAE 1级升高以内；
11. 重要器官功能符合下列要求：（1）血液系统功能（筛选检查前14天内未输血、未使用细胞生长因子纠正）：中性粒细胞计数≥1.5×10⁹/L，血小板计数≥75×10⁹/L，血红蛋白≥90 g/L；（2）肝肾功能（筛选检查前14天内未输白蛋白）：血清总胆红素≤2.5倍正常值上限（ULN），血清白蛋白≥29 g/L，ALT和AST ≤5×ULN；血清肌酐≤1.5×ULN或内生肌酐清除率（Cockcroft-Gault公式）≥60ml/min；（3）凝血功能：国际标准化比率（INR）≤2.3 或凝血酶原时间（PT）超过正常对照的范围≤6秒；（4）二维心脏超声检查左心室射血分数（LVEF）≥50%；
12. 有生育可能的女性患者（除外已进行手术绝育和绝经超过1年的女性），需在研究治疗期间和研究治疗期结束后120天内采用一种经医学认可的避孕措施；非手术绝育的育龄期女性患者在研究入组前7天内血清或尿HCG检查必须为阴性，且为非哺乳期；伴侣为育龄妇女的男性患者，应在试验期间和末次给药后120天内采用有效方法避孕；
13. 愿意提供肿瘤学组织（如果有）进行生物标志物检测。
四、排除标准
1. I期需除外已知肝纤维板层癌；
2. 筛选期头部MRI检查证实有脑转移或软脑膜转移的患者；
3. 在首次给药前5年内诊断为其他恶性肿瘤，经过根治的皮肤基底细胞癌、皮肤鳞状细胞癌和/或经过根治切除的原位癌除外；
4. 在首次给药前4周内接受过肝脏或者其它部位的局部治疗（包括经导管肝动脉化疗栓塞[TACE]、经导管肝动脉栓塞[TAE]等）；在首次给药前4周内进行过肝脏或其他部位的大手术；在首次给药前1周内接受小手术操作；2周以内针对骨转移病灶接受姑息性放疗，并且放疗相关毒性反应≥CECAE 2级；
5. 中度或重度的腹水（B超或CT证实），或需要治疗性的腹腔穿刺或引流；
6. 有肝性脑病病史；
7. 研究入选前3个月内出现未治愈伤口或溃疡、或骨折病史；
8. 准备进行或既往有异体器官移植或异体骨髓移植史；
9. 有出血倾向和血栓史：（1）筛选前3个月内出现显著临床意义的出血症状或具有明确的出血倾向；（2）筛选前6个月内发生消化道出血病史或有明确的消化道出血倾向；（3）筛选前6个月内发生的动/静脉血栓事件；（4）患者需要用华法林或肝素等药物进行抗凝治疗；（5）患者需要长期接受抗血小板治疗（如阿司匹林≥100mg/天、氯吡格雷等）；
10. 严重心血管病史：（1）NYHA（纽约心脏协会）3级和4级充血性心力衰竭；（2）筛选前12个月内患不稳定心绞痛或新诊断的心绞痛或心肌梗死；（3）需要治疗干预的心律失常（服用β-受体阻断剂的患者可以入组）；（4）CTCAE≥2级瓣膜性心脏病；（5）药物控制不良的高血压（收缩压＞150 mmHg或舒张压＞90 mmHg）；
11. 有症状的肺纤维化史、间质性肺炎、尘肺、放射性肺炎、药物相关肺炎、肺功能严重受损或可能会干扰可疑的药物相关肺毒性的检测和处理的受试者；
12. 筛查前7天内患有需要系统治疗的活动性细菌或真菌感染；活动性结核感染；
13. 活动性共感染：乙型肝炎和丙型肝炎，通过可检测的HBV表面抗原或HBV DNA、HCV RNA证实；
14. 受试者具有任何活动性、已知或可疑的自身免疫性疾病；
15. 给予研究药物前14天之内，要求使用皮质类固醇（>10 mg/天泼尼松等效剂量）或其它免疫抑制药物进行系统治疗的受试者（无活动性自身免疫疾病时，允许吸入或局部使用类固醇替代剂量>10 mg/天泼尼松等效剂量）；
16. 其他实验室检查异常：（1）首次给药前发生低钠血症、低钾血症、低磷血症，使用电解质补充疗法后无法恢复至正常水平；（2）明确诊断的甲状腺功能异常，使用甲状腺素替代治疗不能将甲状腺功能维持在正常范围内；（3）人类免疫缺陷病毒（HIV）检测阳性；
17. 连续两份ECG中QTc间期>480ms；
18. 妊娠或哺乳期妇女；育龄期女性或男性在研究期间无避孕意愿或无避孕措施；
19. 既往接受过两线或以上的肿瘤免疫检查点抑制剂治疗（主要包括抗PD-1、抗PD-L1、抗CTLA-4抗体，或包含上述靶点的双特异性抗体）；或患者接受过抗LAG-3抗体治疗；其他肿瘤免疫治疗，需与申办方确认是否可以入组；
20. 高敏体质，可能对试验药物产生严重过敏反应的患者；
21. 在首次服药前4周内接受过减毒活疫苗或者任何在中国未上市的试验性药物；
22. 既往应用过免疫检查点抑制剂的患者，如出现过下列免疫检查点相关的不良事件，无论恢复与否，都不适合入组：a) ≥3级眼部不良事件；b) 4级肝功能异常；c) ≥3级神经系统毒性；d) ≥3级结肠炎；e) ≥3级肾脏毒性；f) ≥3级肺部炎症；
23. 经研究者判断认为不适合参与本试验。
五、试验分组
试验药1：中文通用名:MGD013注射液；剂型:注射液；规格:300mg/15ml/支；用法用量:静脉输注60-75分钟，每两周一次，起始剂量120mg，分别递增至240mg、400mg、600mg，最大不超过600mg；用药时程:连续给药直至疾病进展或发生不可接受的毒性。
试验药2：中文通用名:丙氨酸布立尼布片；剂型:片剂；规格:200mg/片；用法用量:口服，每日一次，可随餐或不随餐，起始剂量200mg，分别递增至400mg、600mg、800mg，最大不超过800mg；用药时程:连续给药直至疾病进展或发生不可接受的毒性。
对照药:无
六、患者获益
1. 免费体检；
2. 有一定的交通补助；
3. 免费使用试验用药品（MGD013注射液、丙氨酸布立尼布片）及相关治疗、检测；
4. 就诊知名三甲医院，全程专家团队长期跟踪服务，持续监测晚期肝内胆管癌治疗效果与安全性，接受国内前沿的联合治疗方案。
七、需要资料
肿瘤项目需要的资料（通用）
1. 详细记录了患者治疗经过的出院记录（距离现在时间最近的一次）；
2. 最近两次复查的CT或者核磁报告；
3. 确诊时的病理报告以及基因检测（没做基因检测的话就不发）；
4. 血常规，血生化，出凝血，肿瘤标志物，传染病检查（乙肝艾滋梅毒）。
八、研究中心所在地区
本研究为国内多中心试验，涵盖北京、上海、江苏、浙江、安徽、福建、山东、河南、湖北、湖南、广东、四川、重庆等上百个城市均有项目中心。
全国上百家三甲医院负责主治，上千家上市药企供药，根据晚期肝内胆管癌（胆管癌）患者实际病情和项目匹配筛选安排。招募符合条件的患者，您可直接联系我们进行匹配筛选。
官方渠道：扫码加微信咨询详情或报名
05
癌症肿瘤项目
晚期胆管癌（FGFR2基因融合）-前沿医学新药项目
——E7090片II期临床研究
一、基本信息
药物名称：E7090片
药物类型：化学药物
适应症：晚期胆管癌（针对具有FGFR2基因融合、经以吉西他滨为基础的联合化疗失败的不可切除晚期或转移性胆管癌患者）
试验专业题目：一项用E7090治疗患不可切除的晚期或转移性胆管癌（FGFR2基因融合）受试者的多中心、非盲、2期试验
试验分类：安全性和有效性
试验分期：II期
其他说明：无
设计类型：单臂试验
随机化：非随机化
盲法：开放
试验范围：国际多中心试验
受试者信息：年龄20岁(最小年龄)至无上限(最大年龄)，性别：男+女，健康受试者：无
二、治疗原理
1. 药物剂型优势：本试验所用E7090片为口服片剂，剂型便捷，便于患者居家遵医嘱用药，无需静脉输注，可显著提升治疗依从性，同时减少静脉给药相关的不良反应，降低患者治疗期间的就医负担。
2. 抗肿瘤作用机制：E7090片是针对FGFR2基因融合的靶向药物，可特异性结合FGFR2靶点，阻断FGFR2信号通路的异常激活，从而抑制晚期胆管癌肿瘤细胞的增殖、迁移和侵袭，诱导肿瘤细胞凋亡，精准靶向杀伤携带FGFR2基因融合的胆管癌病灶，有效控制疾病进展。
3. 临床治疗价值：针对经以吉西他滨为基础的联合化疗失败、具有FGFR2基因融合的不可切除晚期或转移性胆管癌患者，E7090片可提供全新的靶向治疗选择。本次II期临床研究核心目的是基于独立影像检查，评估该药物的客观缓解率，进一步验证其安全性和有效性，为该类胆管癌患者提供更具针对性的治疗方案，改善患者生存质量。
三、入选标准
1. 自愿签署知情同意书，依从性良好，可随访，自愿遵守研究规定；
2. 20岁及以上，性别不限；
3. 经组织学或细胞学诊断为肝内或肝门周围胆管癌，同意提供存档肿瘤样本或残余活检样本，或同意肿瘤活检；
4. 在中心实验室已通过FISH确认肿瘤FGFR2基因融合；在其他检查/研究中通过相同FISH检验确认的FGFR2基因融合，需经申办方逐例批准；
5. 患有无法手术切除或晚期/转移性胆管癌，既往至少接受过一种化疗，包括以吉西他滨为基础的联合化疗（如吉西他滨和顺铂）；
6. 具有可测量的疾病，符合以下标准：a. 使用CT/MRI进行连续测量，根据RECIST 1.1，至少有1处病灶，非淋巴结病灶最长直径≥1.0 cm，淋巴结病灶短轴直径≥1.5 cm；b. 接受过体外放射治疗或局部区域治疗的病灶，根据RECIST 1.1必须显示出疾病进展证据，方可视为靶病灶；
7. 校正后血清钙≤正常值上限（ULN）；
8. 磷酸盐≤正常值上限（ULN）；
9. 根据东部肿瘤协作组（ECOG）标准，体力状态（PS）评分为0-1；
10. 开始服用研究药物后，预计可存活3个月或更长时间；
11. 从既往治疗结束到开始E7090给药需满足洗脱期要求：a. 抗体和其他研究药物：≥4周；b. 既往化疗（小分子靶向治疗除外）、手术治疗、放射治疗：≥3周；c. 内分泌治疗、免疫治疗、小分子靶向治疗：≥2周。
四、排除标准
1. 患有脑部或硬膜下转移的受试者，除非已完成局部治疗且在开始本研究治疗前至少4周已停止服用用于此适应症的皮质类固醇，且脑转移的任何体征或症状在开始研究治疗前稳定至少4周；
2. 伴随需要系统治疗的活动性感染（接受抗病毒治疗的乙型或丙型肝炎病毒感染患者除外）；
3. 筛选期人体免疫缺陷病毒（HIV抗体）检查呈阳性；
4. Child-Pugh分级评分B或C；
5. 存在从骨盆延伸至肝表面的中度或重度腹水；
6. 患有如下眼科疾病：a. 当前具有2级或更高级别角膜疾病的证据；b. 当前具有活动性黄斑疾病的证据（如与年龄有关的黄斑变性、中央性浆液性脉络膜视网膜疾病）；
7. 除脱发、不育和入选标准中的不良事件外，根据不良事件通用术语标准（CTCAE v4.03），既往治疗毒性未恢复至1级或更低；
8. 既往针对FGFR2进行过治疗；
9. 需要使用强效抑制或诱导代谢酶细胞色素P450（CYP）3A4的药物或食物；
10. 研究者认为受试者存在其他原因而不适合参加本临床研究。
五、试验分组
试验药：中文通用名:E7090片；英文通用名:E7090 Tablets；商品名称:NA；剂型:片剂；规格:35mg；用法用量:口服，一天一次，每次140mg；用药时程:连续用药，每28天为一疗程，直到满足个别受试者的任何停药标准。
对照药:无
六、患者获益
1. 免费体检；
2. 有一定的交通补助；
3. 免费使用试验用药品（E7090片）及相关治疗、检测；
4. 就诊知名三甲医院（含国际多中心研究机构），全程专家团队长期跟踪服务，持续监测晚期胆管癌治疗效果与安全性，接受国际前沿的靶向治疗方案。
七、需要资料
肿瘤项目需要的资料（通用）
1. 详细记录了患者治疗经过的出院记录（距离现在时间最近的一次）；
2. 最近两次复查的CT或者核磁报告；
3. 确诊时的病理报告以及基因检测（需包含FGFR2基因融合检测结果，没做基因检测的话就不发）；
4. 血常规，血生化，出凝血，肿瘤标志物，传染病检查（乙肝艾滋梅毒）。
八、研究中心所在地区
本研究为国际多中心试验，全球多国家、多地区开展，国内涵盖北京、上海、广州、南京、杭州、成都、武汉、济南、西安、长沙等数十个核心城市的知名三甲医院。
全国上百家三甲医院负责主治，上千家上市药企供药，根据晚期胆管癌（FGFR2基因融合）患者实际病情和项目匹配筛选安排。招募符合条件的患者，您可直接联系我们进行匹配筛选。
官方渠道：扫码加微信咨询详情或报名
06
癌症肿瘤项目
晚期胆管癌-前沿医学新药项目
——EOC317片I期临床研究
一、基本信息
药物名称：EOC317片
药物类型：化学药物
适应症：晚期胆管癌（针对经标准治疗后进展、不能耐受标准治疗或尚无标准治疗，且有目标FGFR改变的晚期胆管癌患者）
试验专业题目：在中国晚期实体瘤患者中开展的EOC317片口服给药的I期临床研究
试验分类：其他（其他说明:安全性及药代动力学/药效动力学试验）
试验分期：I期
其他说明：无
设计类型：单臂试验
随机化：非随机化
盲法：开放
试验范围：国内试验
受试者信息：年龄18岁(最小年龄)至无上限(最大年龄)，性别：男+女，健康受试者：无
二、治疗原理
1. 药物剂型优势：本试验所用EOC317片为口服片剂，剂型便捷，便于患者居家遵医嘱用药，无需静脉输注，可显著提升治疗依从性，同时减少静脉给药相关的不良反应，降低患者治疗期间的就医负担，适配晚期胆管癌患者长期治疗需求。
2. 抗肿瘤作用机制：EOC317片针对有目标FGFR改变的晚期胆管癌，可特异性作用于FGFR信号通路，抑制信号通路异常激活，从而阻断胆管癌肿瘤细胞的增殖、迁移和侵袭，诱导肿瘤细胞凋亡，精准靶向杀伤肿瘤病灶，延缓疾病进展，同时探索FGFR信号通路改变与药物疗效的相关性。
3. 临床治疗价值：针对经标准治疗后进展、不能耐受标准治疗或尚无标准治疗的晚期胆管癌患者，EOC317片可提供新的治疗选择。本次I期临床研究核心目的是评估该药物的安全性和耐受性，确定最大耐受剂量和/或扩展研究推荐剂量，研究其药代动力学特点，初步评估治疗晚期胆管癌的疗效，为后续临床试验给药方案提供依据，为晚期胆管癌患者带来新的治疗希望。
三、入选标准
1. 自愿签署知情同意书，依从性良好，可随访，自愿遵守研究规定；
2. 18周岁及以上，性别不限；
3. 经组织病理学或细胞病理学证实的晚期胆管癌患者（剂量递增部分可包含胆管癌，剂量扩展部分需为有目标FGFR改变的晚期胆管癌患者）；
4. 既往经标准治疗后进展、或不能耐受标准治疗、或尚无标准治疗的患者；
5. 剂量递增部分：可测量或不可测量的肿瘤病灶均可接受；剂量扩展部分：至少有一个可测量的肿瘤病灶（根据RECIST v1.1版，可测量病灶定义为经CT或MRI检查显示最长径≥10 mm且扫描厚度不超过5 mm的肿瘤病灶；对于淋巴结病灶，短径须≥15 mm）；
6. ECOG评分为0-1分；
7. 预期生存期大于3个月；
8. 无严重血液学、肝、肾功能异常，符合以下实验室检测结果：血液学：中性粒细胞≥1.5×l0⁹/L，血小板≥75×10⁹/L，血红蛋白≥90g/L；肝功能：谷丙转氨酶（ALT）和谷草转氨酶（AST）≤正常值上限×3.0，碱性磷酸酶（ALP）≤正常值上限×2.5，总胆红素（TBIL）≤正常值上限×1.5（≤3mg/dL，如有Gilbert综合征）；如有肝部肿瘤病灶时，ALT和AST≤正常值上限×5.0；如有骨转移或肝部肿瘤病灶时，ALP≤正常值上限×5.0；肾功能：根据Cockcroft-Gault公式计算的肌酐清除率≥50mL/min；
9. 既往系统抗癌治疗的所有不良事件恢复到基线或≤1级（除外：脱发和白癜风；既往抗癌治疗诱发的神经病变稳定或≤2级）；
10. 男性或女性患者在治疗期间以及末次给药后6个月内进行有效的避孕；
11. 剂量递增部分：是否收集肿瘤组织样本依据患者意愿而定；剂量扩展部分：筛选期FGFR靶点异常未知的患者能够提供肿瘤组织样本，其他时期是否收集肿瘤组织样本，研究者根据患者具体情况而定；
12. Child-Pugh肝功能评级为A级或较好的B级（≤7分）；
13. 使用0或1种降压药物即可有效控制血压，定义为：筛选时BP ≤150/90 mm Hg，且在第1个周期/第1天前的1周内没有更换降压药物。
四、排除标准
1. 既往接受过针对FGFR通路的药物；
2. 患有晚期胆管癌以外的其他恶性肿瘤疾病（例外情况包括：治愈且在研究入选前3年内没有复发的恶性肿瘤；完全切除的基底细胞和鳞状细胞皮肤癌；完全切除的任何类型的原位癌）；
3. 原有病灶侵及中枢神经系统（CNS）并有症状，不稳定或需要高剂量类固醇（≥10 mg地塞米松或等效剂量）以达到控制；
4. 在研究治疗首次给药前患者即便进行过医学干预，仍存在有临床意义的实验室钙/磷异常，或者合并甲状旁腺障碍、肿瘤溶解综合征等；
5. 已知可能影响视觉灵敏度的眼部疾病，如视网膜/角膜/晶体病变、严重青光眼等；
6. 需要全身性治疗的活动性感染（例如，病毒、细菌或真菌）；
7. 在开始试验药物前，使用以下伴随治疗：在首次给药前7天内，使用可以延长QT间期和/或伴有尖端扭转型室性心动过速(TdP)风险的药物；在首次给药前90天内使用胺碘酮；
8. 心功能受损或是有临床意义的心血管疾病，包括任何以下内容：脑血管意外/卒中（入组前6个月内）；心肌梗死（入组前6个月内）；不稳定型心绞痛、充血性心力衰竭（纽约心脏学会分级≥II）或需要药物治疗的严重心律失常（包括QT间期[QTc]延长超过470 ms、安装起搏器等）；超声心电图扫描显示左心室射血分数（LVEF）小于50%；
9. 近4周内有活动性出血病史或有胃肠道穿孔危险或近期手术尚未愈合的患者；
10. 研究治疗首次给药前的以下规定时间内患者接受了抗癌治疗：接受其它试验药物治疗≤4周或5个已知药物半衰期（以时间长者为准）；在开始试验药物前≤4周内，接受了大手术的患者；
11. 长期类固醇治疗，每天使用≥10mg泼尼松或等效剂量；
12. 既往慢性腹泻病史或在接受试验药物前仍存在腹泻症状者；
13. 乙肝表面抗原阳性，且HBV DNA拷贝数>检测正常值；丙型肝炎病毒抗体阳性，且HCV RNA阳性者；HBV检测显示HBsAg阳性或HbcAb阳性，且HBV-DNA≥10000 拷贝数/ml或≥2000 IU/ml（经标准抗病毒治疗2周后HBV-DNA不可测的患者可以入组）；丙型肝炎病毒（HCV）RNA> 1000 拷贝/ml（经标准抗病毒治疗2周后HCV-RNA不可测的患者可以入组）；乙肝表面抗原（HbsAg）与抗HCV抗体同时阳性；
14. 有感染人类免疫缺陷病毒史，或患有其它获得性、先天免疫缺陷疾病，或有器官移植史者；
15. 已知有酒精和/或药物依赖者；
16. 既往有明确的神经或精神障碍史，如癫痫，依从性差者；
17. 血妊娠试验阳性的女性患者；
18. 剂量扩展阶段，肝性脑病患者；研究者判断无法缓解的中度或重度的腹水（需在随机前1周内经B超检查或CT扫描确认），或需要治疗性的腹腔穿刺或引流；
19. 研究者认为受试者存在其他原因而不适合参加本临床研究。
五、试验分组
试验药1：中文通用名:EOC317片；剂型:片剂；规格:20mg；用法用量:口服；剂量递增部分拟纳入5mg、10mg、20mg、30mg、45mg、60mg 6个剂量组，DLT观察期第1天给药1次（单次给药），第4天开始进行多次给药，每天1次，连续21天，停药7天；后延长治疗期每28天为一个治疗周期；研究持续给药直至出现DLT、病情进展或脱落；剂量扩展部分根据递增部分试验结果固定剂量服药，28天为一个治疗周期。
试验药2：中文通用名:EOC317片；剂型:片剂；规格:2.5mg；用法用量:口服；剂量递增部分拟纳入5mg、10mg、20mg、30mg、45mg、60mg 6个剂量组，DLT观察期第1天给药1次（单次给药），第4天开始进行多次给药，每天1次，连续21天，停药7天；后延长治疗期每28天为一个治疗周期；研究持续给药直至出现DLT、病情进展或脱落；剂量扩展部分根据递增部分试验结果固定剂量服药，28天为一个治疗周期。
对照药:无
六、患者获益
1. 免费体检；
2. 有一定的交通补助；
3. 免费使用试验用药品（EOC317片）及相关治疗、检测；
4. 就诊知名三甲医院，全程专家团队长期跟踪服务，持续监测晚期胆管癌治疗效果与安全性，接受国内前沿的靶向治疗相关临床试验方案。
七、需要资料
肿瘤项目需要的资料（通用）
1. 详细记录了患者治疗经过的出院记录（距离现在时间最近的一次）；
2. 最近两次复查的CT或者核磁报告；
3. 确诊时的病理报告以及基因检测（需包含FGFR靶点检测结果，没做基因检测的话就不发）；
4. 血常规，血生化，出凝血，肿瘤标志物，传染病检查（乙肝艾滋梅毒）。
八、研究中心所在地区
本研究为国内多中心试验，涵盖北京、上海、江苏、浙江、安徽、福建、山东、河南、湖北、湖南、广东、四川、重庆等上百个城市均有项目中心。
全国上百家三甲医院负责主治，上千家上市药企供药，根据晚期胆管癌患者实际病情和项目匹配筛选安排。招募符合条件的患者，您可直接联系我们进行匹配筛选。
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我司平台丨康心药物临床试验患者招募平台
招募公司丨江西康心医疗科技有限公司
背书单位丨国家疾病防治中心
咨询报名丨雍主任:0799-6888661、189 7999 1644、186 7998 8528
2026-03-26
2026AI 时代已经来临，它对医药行业的冲击也变得日渐明显。除了众所周知的不同LLM 对话，智能体工作流之类，加载skill的LLM 确实能极大的提高医药行业的工作效率，尽可能避免既往普遍的AI 幻觉问题。 笔者最近尝试了一个半专业级别的skill， 作为一个在医药行业摸爬滚打20载的老牛马，被 AI+skill 的能力震惊了！！！
与君共赏如下：
输入：
1.LAG3 药物研发最新情况
2.请再次检查汇总文件的精确性，挑选可靠引文内容，如无法判断引文是否可靠，请不要引用。不清楚或不确定的内容务必不能编造。以上检查和校正后重新生成综合分析报告，谢谢
AI 输出如下（用时1分钟！！！）：
LAG3药物研发综合分析报告（校正版）
报告日期：2026年3月26日
执行摘要
LAG3（淋巴细胞激活基因-3）作为继PD-1和CTLA-4之后的第三个获批的免疫检查点抑制剂，正处于"从希望到质疑"的关键转折点。本报告基于权威学术期刊和可靠媒体公开信息，系统梳理LAG3靶点的机制、已上市药物、在研管线、临床进展及未来前景。
一、LAG3靶点基础概况
1.1 靶点发现历程
1990年：法国免疫学家Frédéric Triebel首次克隆LAG3基因【来源：中国通讯社报道，2026-03-17】
2022年3月：BMS的Relatlimab联合Nivolumab获FDA批准，成为首个上市的LAG3靶向药【来源：PubMed文献，PMID: 38269271】
1.2 分子机制
根据权威文献，LAG3具有以下特征：
表达特征：主要表达在活化的T细胞、NK细胞、B细胞和神经元细胞，特定生理状态下也在DC细胞表达【来源：第三个免疫检查点抑制剂的春天，2025-12-15】
主要配体：MHC II类分子是核心配体，此外还有FGL1、LSECtin、Galectin-3等【来源：第三个免疫检查点抑制剂的春天，2025-12-15】
2025年Cell期刊重要突破：中国科学院生物物理研究所娄继忠研究组发现，LAG3并非简单依赖配体结合，而是依赖MHC-II介导的TCR空间邻近效应，提出"顺式共定位依赖性免疫检查点"新概念【来源：中国科学院生物物理研究所官网，2025-07-02】二、已上市药物分析
2.1 Relatlimab + Nivolumab（Opdualag）
基本信息
表格
项目
内容
来源商品名
Opdualag
PubMed文献（PMID: 38269271）开发商
Bristol Myers Squibb (BMS)
PubMed文献（PMID: 38269271）获批时间
2022年3月18日（FDA）
PubMed文献（PMID: 38269271）获批适应症
不可切除或转移性黑色素瘤（12岁及以上）
PubMed文献（PMID: 38269271）
关键临床数据（RELATIVITY-047研究）
根据《Im Fokus Onkologie》2025年9月23日发布的长期随访数据：中位随访时间
：33.8个月无进展生存期（PFS）
：联合治疗组10.2个月 vs 单药组4.6个月（HR=0.79）总生存期（OS）
：联合治疗组51.0个月 vs 单药组34.1个月（HR=0.80）客观缓解率（ORR）
：联合治疗组43.7% vs 单药组33.7%安全性
：未发现新的安全信号，治疗相关不良事件发生率与既往报道一致【来源：Im Fokus Onkologie，2025-09-23】商业化表现
2025年销售额
：11.85亿美元，保持28%增速【来源：中国通讯社报道，2026-03-17】2023年销售额
：6.27亿美元；2024年销售额9.28亿美元，同比增长48%【来源：第三个免疫检查点抑制剂的春天，2025-12-15】给药方案与安全性
根据药物说明书信息：
推荐剂量：每4周静脉输注一次，Relatlimab 160mg + 纳武利尤单抗 480mg【来源：瑞拉利单抗Opdualag详细中文药品说明书，2026-03-23】
常见不良反应：肌肉骨骼疼痛、疲劳、皮疹、腹泻【来源：瑞拉利单抗Opdualag详细中文药品说明书，2026-03-23】
严重不良反应：免疫介导反应（肺炎、结肠炎、肝炎、内分泌疾病、肾炎、心肌炎、皮肤反应）、输液相关反应、干细胞移植并发症【来源：瑞拉利单抗Opdualag详细中文药品说明书，2026-03-23】三、2026年3月关键临床事件：Immutep Efti折戟
3.1 事件概况
根据多家权威媒体报道：
时间
：2026年3月13日公司
：Immutep（澳大利亚Biotech）药物
：Eftilagimod Alpha（Efti，LAG3融合蛋白）试验
：TACTI-004（一线非小细胞肺癌III期）结果
：独立数据监察委员会（IDMC）判定，即使继续推进，也不太可能在PFS和OS两个主要终点上达到预设目标，建议停止新入组【来源：钛媒体APP，2026-03-20；中国通讯社，2026-03-17】
3.2 入组情况
原计划入组
：756例截至2026年2月已入组
：378例【来源：钛媒体APP，2026-03-20】
四、LAG3靶点面临的挑战
4.1 单药疗效有限
根据文献报道：
在MC38和Sa1N肿瘤小鼠模型中，LAG3单药治疗效果甚微，仅能轻微抑制肿瘤生长，肿瘤清除率极低【来源：钛媒体APP，2026-03-20】
另一项临床试验分析了PD-1和/或LAG3先导疗法在转移性黑色素瘤的表现，结果显示先导入LAG3，后引入PD-1抑制作用有限，反过来却生效【来源：钛媒体APP，2026-03-20】
结论：LAG3的定位更像是PD-1抑制剂的"搭档"，而不是单独驱动疗效的开关【来源：钛媒体APP，2026-03-20】
4.2 "边际效用递减"规律
根据专业媒体分析：
PD-1/PD-L1通路的独特性被严重低估了——它是肿瘤免疫逃逸的主干道，而LAG3、TIGIT、TIM-3等靶点更多扮演辅助性调节角色【来源：又一轮"希望—失望"：LAG-3与TIGIT在III期试验中再次折戟，2026-03-20】
在PD-1已经构筑的免疫底色上，这些"补丁"很难再挤出统计学意义上的增量获益【来源：又一轮"希望—失望"：LAG-3与TIGIT在III期试验中再次折戟，2026-03-20】
4.3 机制复杂性
根据专业报道：
LAG3的表达还受到多种酶的调控，其中金属蛋白酶ADAM10和ADAM17能够切割细胞表面的LAG3，使其以可溶形式脱落。这种动态变化使得LAG3在不同肿瘤微环境中的表现不同【来源：钛媒体APP，2026-03-20】
4.4 适应症拓展困难
根据市场数据：
Opdualag自2022年获批以来，无新增适应症获批，甚至其针对黑色素瘤的辅助治疗的III期临床也没有达到主要终点【来源：钛媒体APP，2026-03-20】
BMS在结直肠癌、肝癌及胃癌等大癌种上的尝试也相继折损【来源：中国通讯社，2026-03-17】五、在研管线进展
5.1 Regeneron - Fianlimab
根据行业报道：
开发商
：Regeneron + Sanofi关键三期临床
：一线黑色素瘤，与Libtayo（PD-1抑制剂）联用，并包含与BMS的Opdualag的"头对头"比较预计数据读出
：2026年上半年行业意义
：所有压力都集中到了再生元身上，该数据预计将决定LAG3靶点在未来数年内的商业命运【来源：lag-3先锋三期临床告吹：热门肿瘤靶点再遭重创，immutep股价暴跌80%，2026-03-14】
5.2 维立志博 - LBL-007
根据腾讯新闻2025年5月28日报道和《Clinical Cancer Research》期刊2026年1月31日发表的研究：
基本信息
药物类型
：LAG3全人源IgG4单克隆抗体机制
：靶向LAG3的独特表位，以高亲和力与LAG3结合，并阻断LAG3与所有四种已识别免疫抑制配体结合临床进度
：II期（LBL-007-CN-004研究，NCT05516914）
鼻咽癌II期关键数据
该研究为单臂、多中心、开放标签的1b/2期临床研究：
入组情况
：2023年3月20日至2024年9月20日期间共筛选53例患者，最终纳入42例符合标准的患者中位年龄
：50岁（范围：29-71岁）中位随访时间
：19.0个月客观缓解率（ORR）
：83.3%（95% CI：68.6%-93.0%），其中包括1例CR（2.4%）和34例PR（82.9%）疾病控制率（DCR）
：97.6%（95% CI：87.4%-99.9%）中位无进展生存期（PFS）
：15.8个月（95% CI：9.9个月-未达到）12个月PFS率
：55.1%（95% CI：41.7%-72.9%）中位起效时间（TTR）
：1.4个月缓解持续时间（DOR）
：14.6个月（95% CI：10.3个月-未达到）12个月总生存（OS）率
：85.6%（95% CI：75.6%-97.0%）中位OS
：尚未达到
安全性数据
停药比例：4.8%，低于替雷利珠单抗+化疗5.3%的历史数据【来源：腾讯新闻，2025-05-28】
不良事件谱：与既往研究一致，未发现新风险【来源：丁香园肿瘤时间，2026-01-31】
其他适应症探索
在食管鳞癌、头颈部鳞癌及鳞状非小细胞肺癌患者中也观察到较好的疗效，特别在鳞状非小细胞肺癌患者中，LBL-007联用疗法ORR达到25.0%【来源：腾讯新闻，2025-05-28】
行业地位
截至2024年11月，LBL-007是全球临床进度前三的靶向LAG3的临床阶段单克隆抗体之一【来源：腾讯新闻，2025-05-28】
是同类抗体中首款被证实对鼻咽癌有效的抗体【来源：腾讯新闻，2025-05-28】
5.3 中国企业双抗管线（已公开信息）
根据可靠媒体报道：
表格
企业
药物
类型
临床进度
目标适应症
康方生物
AK129
PD-1/LAG3双抗
II期
胃癌、淋巴瘤
信达生物
IBI323
LAG-3/PD-L1双抗
II期
多种实体瘤
三生制药
SSGJ-709
PD-1/LAG3双抗
I期
泛实体瘤
【来源：中国通讯社，2026-03-17；LAG3赛道重挫：Immutep三期折戟，双抗等创新形式能否破局？，2026-03-14】六、全球LAG3在研管线概况
根据学术文献统计：
全球仅1款含LAG3靶点单抗药物：BMS的Opdualag【来源：第三个免疫检查点抑制剂的春天，2025-12-15】
世界卫生组织临床试验网站：查询到1-3期试验至少91个，其中2024-2025年开展中34个，待开展45个【来源：第三个免疫检查点抑制剂的春天，2025-12-15】
LAG3靶点在研药物：涉及38个，关联30家企业【来源：第三个免疫检查点抑制剂的春天，2025-12-15】
Immutep：专注于LAG3靶点药物研发的企业，开发4款LAG3靶点候选药物，其中2款产品分别授权默沙东（部分适应症）和诺华【来源：第三个免疫检查点抑制剂的春天，2025-12-15】
3期在研药物：5个，其中默沙东的MK-4280A和MacroGenics的MGD013（联合PD-1）已终止研究，开展中3个涉及诺华与Immutep合作的IMP701、默沙东与Immutep合作的IMP321和Regeneron的REGN3767【来源：第三个免疫检查点抑制剂的春天，2025-12-15】
2期在研药物：14个，其中2款联合PD-1，2款联合PD-L1【来源：第三个免疫检查点抑制剂的春天，2025-12-15】
1期在研药物：15个，新联合靶点涉及CD73、CTLA-3和TIGIT【来源：第三个免疫检查点抑制剂的春天，2025-12-15】
七、关键结论
基于可靠信息来源，得出以下结论：
7.1 已验证的疗效
Opdualag在黑色素瘤中的疗效已确证：III期RELATIVITY-047研究的3年随访数据显示，联合治疗相比PD-1单药在PFS（10.2个月 vs 4.6个月）和OS（51.0个月 vs 34.1个月）方面均有显著改善【来源：Im Fokus Onkologie，2025-09-23】
Opdualag商业化成功：2025年销售额达11.85亿美元，成为"重磅炸弹"药物，并保持28%的健康增速【来源：中国通讯社，2026-03-17】
7.2 主要挑战
Immutep Efti在肺癌III期失败：标志着"踩油门派"路线在非小细胞肺癌一线战场未能跑赢现有标准疗法【来源：中国通讯社，2026-03-17】
BMS适应症拓展困难：Opdualag在黑色素瘤辅助治疗、结直肠癌、肝癌、胃癌等大癌种上的尝试均未成功【来源：钛媒体APP，2026-03-20；中国通讯社，2026-03-17】
LAG3单药疗效有限：临床前和临床数据均显示，LAG3必须与PD-1联合使用才能发挥作用【来源：钛媒体APP，2026-03-20】
7.3 中国企业的优势
维立志博LBL-007在鼻咽癌中展现优异疗效：II期研究显示ORR达83.3%，中位PFS 15.8个月，显著优于历史对照【来源：腾讯新闻，2025-05-28；丁香园肿瘤时间，2026-01-31】
双抗管线全球领先：康方生物、信达生物、三生制药等企业的LAG3双抗项目处于全球第一梯队【来源：中国通讯社，2026-03-17】
7.4 未来关键时间节点
Regeneron Fianlimab 2026年上半年数据读出
：这一数据将决定LAG3靶点在未来数年内的商业命运【来源：lag-3先锋三期临床告吹：热门肿瘤靶点再遭重创，immutep股价暴跌80%，2026-03-14】
7.5 新兴方向
小分子抑制剂：2025年研究发现首类LAG3小分子结合剂，为口服药物开发奠定基础【来源：From Virtual Screens to Cellular Target Engagement: New Small Molecule Ligands for the Immune Checkpoint LAG-3，PMC11571005，2026-03-08】
自身免疫疾病应用：2025年Cell期刊揭示LAG3顺式激活机制，为LAG3激动剂在自身免疫治疗中提供理论支持【来源：中国科学院生物物理研究所官网，2025-07-02】
八、重要风险提示
Immutep III期失败证明LAG3在肺癌等大型癌种中的疗效存在高度不确定性【来源：钛媒体APP，2026-03-20；中国通讯社，2026-03-17】
Opdualag适应症拓展严重受限：除黑色素瘤外，在肺癌、结直肠癌、肝癌、胃癌等大癌种上的尝试均未成功【来源：钛媒体APP，2026-03-20；中国通讯社，2026-03-17】
LAG3单药疗效有限，必须依赖与PD-1联合使用【来源：钛媒体APP，2026-03-20】
Regeneron 2026年上半年的Fianlimab数据是LAG3靶点的关键考验【来源：lag-3先锋三期临床告吹：热门肿瘤靶点再遭重创，immutep股价暴跌80%，2026-03-14】
"边际效用递减"规律表明，在PD-1已经构筑的免疫底色上，LAG3等辅助靶点很难再挤出统计学意义上的增量获益【来源：又一轮"希望—失望"：LAG-3与TIGIT在III期试验中再次折戟，2026-03-20】
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免责声明：本报告由AI生成，未人工核验所有内容。AI仅基于已公开的信息来源整理，不构成使用建议。药物研发存在高度不确定性，临床结果可能与预期存在显著差异。研究决策需结合更多专业咨询和调查。首见上述报告， 笔者一开始还不以为然，毕竟AI 特别擅长结构化的信息输出，结构好会自圆其说没用，毕竟既往它在医学专业领域的使用存在致命缺陷：即常见半壶幻觉。可是，当AI +专业skill 使用后， 笔者查验了一些研究资料进行信息比对， 发现比对信息居然基本还是靠谱的，当然受限于时间和精力，笔者未作全文核对。但就已验证部分笔者确实是已经被震惊到了！！！往常这样的一篇分析报告往往需要一定经验的专业人士大几天或上周才能出来，但AI+skill 在这里用时仅仅1 分钟！！！ 考虑到医学专业的严谨性，后续如真的使用还需要不少时间进行人工核验，但AI +skill 确实打了一个非常好用的底板，而这无疑可以巨幅提高工作效率 。未来已来，还在医药行业打拼的牛马们，大家准备好了吗.......
2026-02-18
New-generation ICIs set to break biological ceiling
With PD-1/CTLA-4 franchises such as Merck’s Keytruda (>$30bn in sales in 2025) reaching maturity, incremental value is shifting to next-generation checkpoints that can address adaptive resistance, extend duration of response, expand responder populations and support lifecycle management. Bristol Myers Squibb’s (BMS’s) Opdualag (LAG-3/PD-1 combination) provides the first commercial proof that dual-checkpoint strategies can extend durability and defend PD-1 backbones. We expect assets showing additive efficacy with PD-1/CTLA-4s and favourable safety are most likely to secure partnering and premium valuations.
Cancer vaccines as immune-priming platforms
Next-generation cancer vaccines are transitioning from monotherapy setbacks to high-value immune-priming partners capable of generating high-avidity, tumour-specific responses and converting immunologically cold tumours into ‘hot’ tumours. Moderna/Merck’s mRNA vaccine V940/mRNA-4157 provides the clearest validation in melanoma studies. We see cancer vaccines’ strongest commercial positioning in combination treatments, earlier-stage and minimal residual disease settings, where durable immune memory supports long treatment windows. Platform scalability, precision biomarker strategies and consistent combination benefit across tumour types will be critical to partnering activity and peak-sales realisation.
CAR-T therapies at an inflection point
CAR-T therapies (such as Gilead’s Yescarta and Novartis’s Kymriah) deliver potentially curative outcomes in select haematological cancers, but uptake remains constrained by limited solid-tumour efficacy, manufacturing complexity, high cost and toxicities. Next-wave strategies, including allogeneic platforms, bi-specific/multi-targeting constructs programmes and CRS-mitigation approaches, promise to reduce infrastructure intensity and enable earlier-line use, and will determine CAR-T’s evolution from niche therapy to a broader commercial modality.
Immuno-oncology: The next phase of value creation
Our immune system is naturally built to detect and eliminate abnormal cells while sparing healthy tissue. However, cancer has evolved several ways to evade immune recognition, allowing unchecked tumour growth. While traditional treatments like chemotherapy and radiation can be effective, they are associated with significant side effects. In contrast, IO is a class of cancer therapies that harness, activate or engineer the body’s natural defence mechanisms to recognise and destroy tumour cells, effectively removing the ‘brakes’ that cancer places on the immune system. Since approval, IO has reshaped cancer care, led by ICIs that have become the backbone treatment across multiple solid tumour types. Yet the first ICI wave has also exposed clear constraints: response rates remain limited in many indications, resistance emerges frequently and some tumour types remain stubbornly ‘cold’. In parallel, haematological malignancies remain an untapped frontier where ICIs have delivered more modest outcomes.
The growing challenge is no longer whether immunotherapy works, but how to make it work for the majority of patients, across a broader range of tumours, with acceptable safety, cost and scalability. The next phase of value creation is therefore less about a single breakthrough drug and more about expanding the responder pool and durability of benefit through new checkpoint targets beyond the current PD-(L)1 and CTLA-4 combination strategies that address tumour-resistance mechanisms and new immunotherapy modalities such as therapeutic cancer vaccines and next-generation cell/CAR-T therapies.
Commercially, this evolution should continue to drive partnering: big pharma’s PD-(L)1/CTLA-4 franchises, broad oncology footprints and overhang from the upcoming patent cliff (>$200bn in revenues at risk by 2030) create a natural pull for differentiated add-ons (novel checkpoints, vaccines, intratumoural immunotherapies) and for technologies that reduce toxicity and the healthcare burden in cell therapies.
This report explores the scientific rationale for next-generation IO approaches, showcases leading biotech innovators advancing the field, examines the commercial dynamics driving deal-making and presents the investment case for biotechs at the forefront of this evolution.
Market context: Oncology dominates therapeutic development
Oncology remains the largest and most innovation-intensive therapeutic area in pharma, reflecting a combination of large addressable patient populations, persistent unmet need and the high economic value of clinically meaningful survival improvements. According to Evaluate Pharma, oncology accounted for 39% of global pharmaceutical R&D spend in 2023, with projected sales of $375bn by 2030, more than twice that of the next largest therapeutic area, immunology, at $165bn (Exhibit 1).
Exhibit 1: Therapeutic area market size projections (2030)
Source: Evaluate Pharma, Edison Investment Research
Within oncology, IO has become a central pillar of R&D and commercial strategy. PD-(L)1 inhibitors have created a large, durable market, but the competitive dynamics have shifted from ‘who has a PD-1’ to ‘who has the best combination ecosystem and differentiated biology’.
This matters for how value is likely to accrue. In the next phase, winners are likely to be those that can: (a) take immunotherapy into harder-to-treat tumours and patient subgroups; (b) move effective approaches earlier in the disease course (adjuvant/maintenance/minimal residual disease); and (c) reduce toxicity and delivery friction so that therapies can be used more widely and earlier.
ICIs: Revolution and limitations
What are ICIs?
ICIs are immunotherapies or drugs that modulate immune ‘brakes’ that ordinarily prevent excessive immune activation, such as by T cells. Tumours exploit these brakes to avoid immune destruction. By blocking these inhibitory brakes, ICIs restore anti-tumour immune activity, thereby enabling the immune system to recognise and attack cancer cells (Exhibit 2). The two foundational inhibitory pathways to date have been PD-1/PD-L1 (dominant in the tumour microenvironment (TME); affects peripheral effector function) and CTLA-4 (more upstream; affects T-cell priming/activation in lymphoid tissues). By releasing these brakes, ICIs can convert an existing anti-tumour immune response into deep, durable remissions in a subset of patients, an outcome rarely achieved with historical cytotoxic treatments or reliably matched by targeted therapy.
Exhibit 2: ICIs’ mechanism of action
Source: Adapted from Poto R, Troiani T, Criscuolo G, Marone G, Ciardiello F, Tocchetti CG and Varricchi G (2022) Holistic Approach to Immune Checkpoint Inhibitor-Related Adverse Events. Front. Immunol. 13:804597. (CC by 4.0)https://www.frontiersin.org/journals/immunology/articles/10.3389/fimmu.2022.804597/full
Clinically, the impact has been most visible in immunogenic tumours (with multiple neoantigens; proteins exclusively expressed by cancer cells) and where a pre-existing anti-tumour immune response can be amplified (eg ‘hot’ tumours such as melanoma, non-small cell lung cancer (NSCLC), renal cell carcinoma and classical Hodgkin lymphoma). However, mechanistically this is not a simple ‘on/off’ switch: the effectiveness of ICIs depends on antigen presentation, T-cell trafficking and infiltration, TME suppressive factors and the dynamic expression of multiple immune checkpoints.
Current ICI landscape limited to PD-(L)1s
The current ICI market (Exhibit 3) has been dominated by PD-1 and PD-L1 inhibitors, which have achieved broad approvals, frequent label expansions and deep integration into treatment pathways, with Merck’s Keytruda (generic name: pembrolizumab) leading the pack with $31.7bn in annual sales in 2025 and approval across 20+ indications, with an additional 20+ indications in clinical development. CTLA-4 inhibition (BMS’s Yervoy) remains important in select high-intensity combinations. More recently, LAG-3 joined the approved checkpoint set through BMS’s Opdivo (nivolumab) + relatlimab fixed-dose combination, Opdualag, in melanoma (approved in 2022), the first successful next-generation checkpoint beyond CTLA-4. Opdualag’s approval validates the potential clinical relevance of additional checkpoints, in our view.
Exhibit 3: Approved ICIs
Source: Evaluate Pharma, Edison Investment Research
According to Global Markets Insights, the global ICI market was valued at $66.2bn in 2025, with double-digit growth expected over the next decade, driven by earlier lines/adjuvant settings, new combinations and next-generation mechanisms that can extend benefit beyond today’s responder ceiling (discussed in further detail below).
Limitations of current ICIs
Hitting the biological ceiling: Despite transformative outcomes in some tumour types, only a minority of patients benefit from PD-(L)1 and CTLA-4 blockade, in what is termed a ‘biological ceiling’ issue. Large reviews commonly cite 20–40% of the patients benefiting across broad populations depending on tumour type, biomarker enrichment and treatment regimen. While response rates can vary, the key issue is that a large proportion of patients either do not respond at all or respond transiently before disease progression. This constrains the ceiling of benefit for monotherapy and forces the field toward combination strategies, better patient selection and immune priming approaches.
Resistance mechanisms – innate and acquired resistance: The variability in ICI efficacy is fundamentally driven by tumour immune resistance mechanisms, which can be broadly classified as innate (primary) or acquired (secondary). In primary resistance, tumours lack the biological prerequisites for response, including effective antigen presentation, baseline immune activation and pre-existing T-cell infiltration. In acquired resistance, initial responders subsequently relapse as tumours undergo immune editing, downregulate antigen presentation, activate alternative inhibitory checkpoints or remodel the TME to exclude effector T cells. Together, these adaptive escape processes cap response rates and durability.
Haematological malignancy limitations: While ICIs have transformed outcomes in multiple solid tumours, their performance has often been more variable in haematological cancers. Many haematological malignancies have lower levels of tumour-specific mutations (so the immune system has fewer abnormal targets to recognise) and exist in the bone marrow and lymph node environment, which is naturally immunosuppressive and can physically limit effective T-cell activity. As a result, releasing a single immune brake is often not enough to generate a meaningful anti-tumour response. The key exception is classical Hodgkin lymphoma, where the PD-1 pathway is genetically overactive, making it particularly sensitive to checkpoint blockade.
This has created space for other immunotherapy modalities (including cellular therapies and vaccines) to become central to innovation and value creation in haematology.
Toxicity and immune-related adverse events: ICIs work by activating the immune system, but this broad activation can also cause the immune system to attack normal healthy tissues, leading to immune-related adverse events (irAEs). These side effects can affect multiple organs (most commonly the skin, gut, liver, lungs and hormone-producing glands) and range from mild inflammation to, in rare cases, severe or life-threatening complications. In clinical practice, most irAEs are manageable with early detection and immunosuppressive treatment, but they remain a key factor in treatment selection and in the design of next-generation IO combinations.
TME complexity: ICIs are also less effective in many cancers because of the complex TME (the community of non-cancer cells, blood vessels and signalling molecules that surround the tumour). In some tumours, this environment acts like a protective shield, keeping cancer-killing immune cells out, switching them off, or flooding the area with signals that suppress immune activity. This means that even if an ICI releases the ‘brakes’ on the immune system, the T cells either cannot reach the tumour or cannot function properly once they get there. As a result, the treatment has little effect. Overcoming this barrier is a major focus for next-generation IO, with new strategies aimed at making these tumours more immune-friendly (such as intratumoral therapies, vaccines and myeloid-targeting approaches) so checkpoint inhibitors can work more effectively.
Cold tumours (eg pancreatic, prostate, ovarian, colorectal cancer): ICIs tend to work best when there is already an active immune response against the tumour. In so-called ‘cold’ tumours, this immune activity is largely absent (there are very few cancer-fighting T cells in or around the tumour), and the cancer often does not produce enough abnormal signals to attract them. As a result, giving an ICI is like releasing the brakes on a car that has no engine running – the drug removes the immune block, but there are not enough immune cells present to attack the cancer. In addition, these tumours often create a strongly suppressive environment that keeps immune cells out. To address this, a key focus of current research is on combination approaches that first ‘heat up’ the tumour by bringing immune cells in, and then use ICIs to activate them.
Upcoming newer generation ICI strategies
To transcend current limitations, the field is aggressively exploring novel immune checkpoints and innovative modalities, with the goal of improving response rates, overcoming resistance and broadening benefit to more tumour types and patients.
Expansion into earlier treatment lines: One of the key commercial upsides for ICIs will come from increasing use in the adjuvant, peri-operative and minimal residual disease (MRD) settings, where patient numbers are higher, treatment duration is longer and curative intent supports premium pricing. This is already evident with pembrolizumab in adjuvant melanoma and peri-operative NSCLC (KEYNOTE-671), nivolumab in adjuvant melanoma and resected oesophageal/gastroesophageal junction (GEJ) cancer (CheckMate-577) and atezolizumab in adjuvant NSCLC (IMpower010). With metastatic markets largely penetrated, future growth and blockbuster potential for next-generation IO will depend on demonstrating meaningful benefit in earlier-stage disease.
New-generation checkpoint targets – LAG-3, TIGIT, TIM-3, VISTA: Most solid tumours can express multiple inhibitory checkpoints at the same time, often five to 10 or more across tumour cells, building the biological basis for a ‘multiple brakes’ concept and for the move toward dual- and multi-checkpoint strategies. Beyond PD-(L)1 and CTLA-4, multiple inhibitory pathways have emerged as potential levers to overcome resistance and improve response. However, translating target biology into consistent clinical benefit has been uneven.
Combination treatments – two better than one?: With growing proof of the ability of tumours to activate multiple immune ‘brakes’ simultaneously, combination checkpoint strategies are increasingly being evaluated. These aim to widen the responder pool by targeting complementary resistance pathways, deepen responses by enhancing T-cell activation and reduce the likelihood of tumour escape via single-pathway adaptation.
One thing is clear from the above – to win adoption, combinations need either a clear efficacy step-up (eg overall survival benefit) or a tolerability/convenience advantage that enables broader eligible populations.
Bispecific and multi-specific agents: Bispecific and multi-specific agents attempt to integrate the aforementioned combination logic into a single molecule, potentially improving pharmacology and co-targeting engagement, convenience of administration and intellectual property differentiation. These agents can be designed to simultaneously block multiple inhibitory pathways, bring immune effector cells into proximity with tumours, or combine inhibitory checkpoint blockade with immune stimulation. Early clinical and regulatory validation is emerging, with PD-1 × CTLA-4 bispecifics such as Akeso’s cadonilimab approved in China in first-line treatment in gastric/GEJ cancer in combination with chemotherapy. PD-1 × LAG-3 bispecifics represent a mechanistically validated optionality following Opdualag, with assets such as EpimAb/BeiGene’s EMB-02, aiming to co-target the commonly co-expressed pathways and show activity in PD-1–refractory disease. However, challenges remain, as evidenced by the clinical discontinuations of MacroGenics’s tebotelimab and Roche’s tobemstomig, underscoring execution risk. PD-1 × TIGIT formats, led by AstraZeneca’s rilvegostomig, are higher risk but offer potential upside if bispecific architecture can outperform prior TIGIT monoclonal antibodies. The challenge will be to demonstrate meaningful differentiation, efficacy or tolerability versus simply co-administering two agents, while maintaining manufacturability and safety.
More recently business development activity in ICI bispecifics has increasingly converged on the PD-(L)1 × VEGF axis, which has emerged as one of the most competitive post-PD-1 backbone strategies. VEGF (vascular endothelial growth factor) is a key signalling protein that tumours use to stimulate the formation of new blood vessels (angiogenesis). VEGF-targeted therapies disrupt this process, effectively starving the tumour and normalising its abnormal vasculature. By simultaneously blocking immune checkpoints and normalising tumour vasculature, these agents may help address two of the key limitations of first-generation PD-1 therapy: poor T-cell infiltration and an immunosuppressive microenvironment. A series of large transactions, including multi-billion-dollar collaborations, the largest being the $11.1bn deal between BMS and BioNTech (for pumitamig) in June 2025, highlights big pharma’s urgency to secure next-generation platforms that combine immune activation with TME normalisation. Exhibit 4 lists the major ICI bispecific licensing deals since 2022, the largest volume and value of which have been in PD-1 x VEGF bispecifics.
Exhibit 4: Major ICI bispecific licensing deals
Source: Edison Investment Research, Evaluate Pharma
Value in the ICI space is shifting from new PD-1 monotherapies to combination-enabling assets that expand responder populations, overcome resistance and move treatment into earlier lines. The biological ceiling creates strong partnering demand for next-generation checkpoints, TME modulators and immune-priming approaches. Bispecific deal activity highlights big pharma’s urgency to secure differentiated, franchise-extending platforms. Therapies that demonstrate additive efficacy with manageable toxicity and clear biomarker strategy are most likely to achieve premium valuations and early monetisation.
Cancer vaccines: From disappointment to resurgence
Unlike traditional preventative vaccines, therapeutic cancer vaccines are designed to treat patients who already have cancer by training the immune system to recognise and attack tumour-specific targets, mainly by activating cancer-killing T cells. This is more challenging because cancer comes from the body’s own cells and often hides from the immune system, but the same fact also makes these vaccines a strong partner for checkpoint inhibitors. The vaccine helps create more tumour-fighting immune cells, and the checkpoint drug keeps them active. Earlier-generation cancer vaccines delivered only modest clinical benefit and struggled commercially due to weak immunogenicity, suboptimal antigen selection and deployment in late-stage disease, where immune dysfunction is profound.
However, recent randomised clinical data provide the first clear evidence that newer-generation vaccines can significantly reduce recurrence risk when used in the early or minimal residual disease setting and/or in combination with PD-1 inhibitors. This represents a fundamental shift in how the modality is positioned: not as a monotherapy for metastatic disease, but as an immune-priming platform capable of inducing durable, tumour-specific T-cell responses.
Historical clinical development and limited early impact
First-generation cancer vaccines were based on several technological approaches, including peptide vaccines, whole-cell tumour vaccines and autologous dendritic cell therapies. While these strategies demonstrated proof of principle that immune responses could be generated against cancer, they failed to produce consistent and clinically meaningful tumour regression.
The approval of Dendreon Corp’s sipuleucel-T (Provenge) in 2010 for metastatic castration-resistant prostate cancer represented a landmark for the field, as it showed an overall survival benefit in a randomised trial. However, the absence of objective tumour responses, the complexity of autologous manufacturing, high cost of goods and logistical challenges limited physician adoption and ultimately constrained commercial success.
More broadly, these early programmes suffered from a lack of biomarker-driven patient selection (such as MSI-H/dMMR), poor antigen immunogenicity due to immune tolerance and clinical trial designs that evaluated vaccines in heavily pre-treated metastatic patients with advanced immune dysfunction. Collectively, these factors led to the perception that cancer vaccines were biologically interesting but clinically impractical.
Core scientific and clinical limitations of earlier approaches
Antigen selection (shared TAAs) and immune tolerance: Most first-generation therapeutic cancer vaccines were built around shared tumour-associated antigens (TAAs), proteins overexpressed in cancer cells but also present at low levels in normal tissues. Because the immune system is trained to tolerate self-antigens, these targets had limited capacity to generate strong cytotoxic T-cell responses. This biological constraint was reflected in several high-profile late-stage failures including GSK’s MAGE-A3 programme (in melanoma and NSCLC), Merck KGaA’s tecemotide (L-BLP25), which targeted the MUC1 glycoprotein in NSCLC, and Bavarian Nordic’s PROSTVAC, a PSA-targeted viral vaccine for prostate cancer.
TME and immune escape: A second major limitation was the inability of early vaccine strategies to overcome the TME. First-generation cancer vaccines were commonly administered as monotherapies in patients with advanced metastatic disease, where tumour burden is high and immunosuppressive signalling is well established. Even when tumour-specific T cells were successfully generated in the periphery, their infiltration and cytotoxic function within the tumour were actively inhibited by a complex network of suppressive signals. This included upregulation of immune checkpoints such as PD-L1, recruitment of regulatory T cells and myeloid-derived suppressor cells (MDSCs) and secretion of inhibitory cytokines such as TGF-β and IL-10.
Disease setting and tumour burden: Clinical positioning was another critical determinant of failure. Early vaccine trials were conducted predominantly in late-stage, heavily pre-treated patients with high tumour burden and often impaired immune function. In this context, the time required to generate an adaptive immune response, typically several weeks to months, is biologically and clinically mismatched with the rapid disease progression typical of advanced cancer. In contrast, it is now widely recognised that cancer vaccines are most effective in the minimal residual disease or adjuvant setting, where tumour burden and subclonal diversity are lower and immune competence is relatively preserved.
Platform and delivery limitations: Many peptide-based vaccines lacked sufficiently potent or appropriately tailored adjuvants to stimulate dendritic cell maturation and effective cross-presentation through MHC Class I pathways, which is essential for generating CD8+ cytotoxic T-cell responses. Without robust innate immune activation, adaptive responses remained quantitatively and qualitatively inadequate. Modern platforms, including mRNA, viral vectors and next-generation dendritic cell approaches (Exhibit 5) are specifically designed to address this limitation by integrating antigen delivery with strong innate immune stimulation.
Exhibit 5: Different types of therapeutic cancer vaccines
Source: mRNA vaccines for cancer immunotherapy, Yashavantha Vishweshwaraiah, Nikolay Dokholyan. 2022. Frontiers of Immunology
Field at an inflection point
The resurgence of cancer vaccines is not driven by a single breakthrough but by the convergence of multiple enabling technologies.
Neoantigen targeting – moving beyond shared self-antigens: Next-generation cancer vaccines prioritise tumour-specific neoantigens rather than shared TAAs, avoiding central immune tolerance and enabling high-avidity cytotoxic T-cell responses. This shift materially improves the probability of meaningful tumour control, as neoantigens are recognised as non-self and can drive polyclonal immune activation through multi-epitope designs. Clinical validation is emerging across multiple platforms. Moderna/Merck’s V940 has demonstrated a significant reduction in melanoma recurrence in the adjuvant setting in a randomised Phase IIb trial, while BioNTech/Genentech’s autogene cevumeran has generated strong neoantigen-specific T-cell responses in pancreatic cancer. Viral and epitope-based approaches from Nouscom (NOUS-PEV), OSE Immunotherapeutics (Tedopi) and Transgene (TG4050) further underscore the central importance of antigen selection, alongside optimised delivery technologies, in driving immunogenicity and clinical activity.
Genetic delivery platforms – mRNA and DNA as scalable backbones: New-generation cancer vaccines are increasingly using genetic material, such as mRNA or DNA, to instruct the body’s own cells to produce tumour antigens, rather than introducing the antigens directly as proteins or peptides. This approach helps the immune system recognise the targets more effectively and generates both killer (CD8+) and helper (CD4+) T cell responses. It also naturally activates the early immune signals needed for a strong and durable reaction, reducing the need for additional adjuvants. Moderna’s personalised mRNA vaccines and BioNTech’s personalised and shared antigen programmes (including BNT111) highlight the speed and flexibility of this technology, while Scancell’s DNA ImmunoBody platform (iSCIB1+) shows how a potent off-the-shelf option can be combined with checkpoint inhibitors.
Designed for checkpoint inhibitor synergy: Newer cancer vaccines are also being positioned as immune primers for checkpoint blockade rather than as standalone therapies. The vaccine trains the immune system to recognise the tumour and brings T cells into the tumour, while drugs such as PD-1, PD-L1 or CTLA-4 inhibitors stop those T cells from being switched off. Together, this helps overcome two key problems: many tumours do not already have tumour-fighting T cells, and the tumour environment suppresses immune activity. The positive data for V940 with pembrolizumab show this approach can work in practice. Similar combination strategies, including Scancell’s iSCIB1+ with dual checkpoint blockade, also make it easier for vaccines to fit into current treatment pathways.
Earlier disease settings – MRD, adjuvant and maintenance therapy: Cancer vaccine development is moving away from use in very late-stage disease and focusing instead on earlier settings, such as after surgery or when only small amounts of cancer remain (minimal residual disease). At this stage, the immune system is stronger and the tumour burden is low, giving vaccine-activated T cells a better chance to clear remaining cancer cells and provide long-term protection. This also fits better with how vaccines work, as they need time to build an immune response and are less effective in fast-growing metastatic disease. Mendus’s vididencel in acute myeloid leukaemia (AML) after remission and V940 in post-surgery melanoma are good examples. Earlier use also means larger patient populations and longer treatment duration, which improves commercial potential.
Exhibit 6 presents a list of the most clinically advanced cancer vaccine programmes in development.
Exhibit 6: Selection of ongoing clinical-stage cancer vaccine programmes
Source: Edison Investment Research, Evaluate Pharma
Deal activity in cancer vaccines is re-accelerating after a prolonged period of limited partnering, reflecting renewed confidence in the modality as clinical data and platform maturity improve. The c $1.25bn BioNTech acquisition of German mRNA vaccine developer CureVac in December 2025 is a clear inflection point. This follows earlier large platform alliances such as BioNTech-Genentech and Moderna-Merck and signals that large pharma now views cancer vaccines as a core long-term pillar of IO. For the broader sector, the transaction raises the strategic value of companies with validated antigen-selection technologies, combination data with checkpoint inhibitors and scalable manufacturing infrastructure, and points to a more competitive partnering and M&A environment.
Cancer vaccines are transitioning from a historically high-risk modality to a combination-driven, platform opportunity, supported by randomised clinical validation in the adjuvant and MRD settings. The shift to neoantigen targeting, genetic delivery and PD-1 synergy materially improves probability of success and commercial scalability. Earlier-line positioning expands patient populations and treatment duration, while recent large-scale transactions signal renewed big pharma appetite. Companies with biomarker-led development, checkpoint-combination data and scalable manufacturing are therefore best placed to secure premium partnering and valuation re-rating.
CAR-Ts: Expanding beyond first-generation constraints
CAR-Ts – mechanism and therapeutic concept
Chimeric antigen receptor-T cell therapies (CAR-Ts) are a form of adoptive cell therapy in which a patient’s immune cells are genetically engineered to recognise and kill cancer cells. The process begins with the collection of T cells from the patient, which are then modified ex vivo to express a synthetic receptor, the chimeric antigen receptor, that binds to a specific tumour surface antigen independently. These engineered cells are expanded and reinfused into the patient, where they proliferate, directly eliminate antigen-expressing tumour cells and can persist as a living drug capable of long-term immune surveillance (Exhibit 7).
Exhibit 7: How CAR-Ts work
Source: Zhou Y, Wei S, Xu M, Wu X, Dou W, Li H, Zhang Z and Zhang S (2024) CAR-T cell therapy for hepatocellular carcinoma: current trends and challenges. Front. Immunol. 15:1489649. (CC BY 4.0) https://www.frontiersin.org/journals/immunology/articles/10.3389/fimmu.2024.1489649/full
Limitations and next-generation solutions
Curative potential but concentrated to haematology: To date, CAR-Ts have been applied in the treatment of haematological cancers by targeting the CD19 and BCMA antigens. Approved CAR-T therapies from companies such as Gilead/Kite (Yescarta, Tecartus), BMS (Breyanzi, Abecma) and Johnson & Johnson/Legend (Carvykti) have transformed outcomes in relapsed or refractory B-cell malignancies and multiple myeloma, delivering deep and durable responses in heavily pre-treated patients (Exhibit 8). This success stems from several structural advantages over conventional therapies: high target specificity, in vivo expansion after infusion and the potential for durable remission following a single treatment. However, the treatment is also associated with unique immune-mediated toxicities. Complex autologous manufacturing and the need for specialised centres for treatment delivery currently limit scalability. More importantly, clinical activity in solid tumours has been modest (due to tumour heterogeneity), highlighting that first-generation autologous CAR-T designs are highly effective in liquid cancers but not yet broadly applicable across oncology.
Exhibit 8: FDA-approved CAR-T therapies
Source: Edison Investment Research
Antigen escape – The shift to multi-target constructs: Antigen escape in CAR-T cell therapy is a mechanism of treatment resistance where tumour cells evade destruction by losing or reducing the specific surface protein (antigen) that the CAR-T cells are engineered to recognise. Relapse driven by antigen loss is a major limitation of single-target CAR-Ts. Dual and multi-antigen targeting is now a central development focus, designed to address this limitation by simultaneously recognising more than one tumour antigen, reducing the probability of immune evasion and improving depth and duration of response. Academic and China-based programmes evaluating dual CD19/CD22 CAR-Ts, as well as BCMA/CD19 constructs being explored by multiple developers, exemplify this strategy. In parallel, next-generation single-target approaches such as Autolus’s obe-cel (Aucatzyl), approved by the FDA in November 2024 for adults with relapsed/refractory B-cell precursor ALL, use a fast off-rate CD19 binder to enhance T-cell persistence and reduce exhaustion, representing an alternative strategy to improve durability of response.
This strategy is particularly relevant in B-cell malignancies and multiple myeloma, where antigen downregulation is well documented. These designs are expected to become increasingly important as CAR-Ts move into earlier lines of therapy where durability requirements are higher.
Toxicity and safety – Enabling broader adoption: Cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) remain key barriers to treatment in community settings
Engineering approaches to reduce tonic signalling and control CAR activation are being pursued by Autolus and Allogene, while pharmacological strategies targeting inflammatory pathways are emerging as a complementary solution.
In parallel, adjunctive therapies specifically targeting CRS are being developed. A notable example is Poolbeg Pharma’s POLB-001, an oral p38 MAP kinase inhibitor, being developed to prevent CRS associated with CAR-T and bispecific antibodies. The asset has completed Phase I human challenge work demonstrating broad cytokine suppression and is now entering a Phase IIa (TOPICAL) proof-of-concept study in r/r multiple myeloma patients receiving the bispecific T-cell engaging antibody teclistamab, with interim data expected in summer 2026. The programme has received FDA Orphan Drug Designation for CRS prevention, positioning it as a potential first-in-class prophylactic that could enable outpatient delivery and broader adoption of cell and T-cell-redirecting therapies.
Manufacturing bottlenecks – the move to allogeneic, off-the-shelf platforms: Off-the-shelf (allogeneic) CAR-T therapies use pre-made immune cells from healthy donors instead of creating a custom product for each patient. This could allow treatment to be given much faster, at lower cost and with more consistent quality. Gene editing removes the donor cell’s natural receptor so it does not attack the patient and is less likely to be rejected, an approach being developed by companies such as Allogene and Cellectis. Newer ‘immune-cloaked’ versions use CRISPR to help the cells stay in the body longer, which is important for long-lasting benefit. Another step forward is induced pluripotent stem cell (iPSC)-derived CAR-Ts, where stem cells are used to produce a large, standardised supply. If these therapies work as well as current CAR-T therapies, they could be delivered more widely and earlier in treatment.
Alternative effector cells: CAR-NK and CAR-iNKT: Other immune cells such as natural killer (NK) cells and invariant natural killer T (iNKT) cells offer several structural merits over conventional CAR-T cells. They are inherently allogeneic, have a lower risk of graft-versus-host disease and appear to produce less severe CRS. Nkarta and Fate Therapeutics are advancing CAR-NK programmes in both haematological and solid tumours, while Arovella Therapeutics is developing CAR-iNKT therapies, with encouraging early data. These platforms may be particularly important for solid tumours and outpatient delivery models. A key current limitation however is the relatively short in-vivo persistence and limited clinical durability particularly for CAR-NK approaches, which may necessitate repeat dosing to match the long-term efficacy achieved with autologous CAR-T therapies.
CAR-T for solid tumours – the largest expansion opportunity: Extending CAR-T therapies into solid tumours represents the biggest long-term growth opportunity for the modality, but also the most complex biologically due to the immunosuppressive TME and heterogeneous antigen expression. Armoured CAR-T cells, designed to secrete stimulatory cytokines such as IL-12 or IL-15, aim to remain active within this hostile environment and recruit other immune cells; this approach is being explored in multiple early-stage clinical and academic–industry programmes. To improve tumour specificity and safety, logic-gated CAR designs that require the presence of more than one tumour antigen for activation are also under development. Modular adaptor CAR platforms, such as Prescient Therapeutics’ OmniCAR, provide an alternative strategy by enabling controllable and retargetable tumour recognition. Most of these approaches remain in early phases of clinical development.
Expanding beyond oncology: CAR-T therapies are emerging as a potentially transformative modality in autoimmune disease by delivering deep and durable depletion of pathogenic B cells, creating the possibility of a one-time immune reset rather than chronic immunosuppression. Early academic CD19 CAR-T studies in systemic lupus erythematosus have demonstrated drug-free remissions, catalysing rapid industry investment. A key requirement for broader adoption in immunology is an improved safety and tolerability profile suitable for outpatient use. Immix Biopharma’s NXC-201, a BCMA-targeted CAR-T in Phase Ib/II for AL amyloidosis with registrational intent, uses a sterically optimised design that has shown low neurotoxicity and short-duration CRS. This profile underpins planned expansion into B-cell-driven non-oncology indications, where risk tolerance is lower but the commercial opportunity is significantly larger.
In-vivo CAR-T: Next-generation cell therapy without ex-vivo manufacturing: An area of development that has been gaining increasing interest is in-vivo CAR-T therapy, an emerging approach in which a patient’s T cells are genetically programmed directly inside the body using targeted delivery technologies such as lipid nanoparticles or viral vectors, removing the need for complex ex-vivo cell processing. This has the potential to significantly reduce manufacturing time, lower cost of goods and enable treatment in outpatient or community settings, addressing key access and scalability barriers associated with current autologous CAR-T therapies. The modality may also allow repeat dosing and more controlled cell expansion, although this remains to be demonstrated clinically. This field has seen increasing M&A and licensing activity in the past year and is becoming one of the most commercially attractive areas of development within the cell therapy space. Exhibit 9 highlights some of the major deals in this space, the most recent being the acquisition of Orna Therapeutics by Eli Lilly for up to $2.4bn in February 2026.
Exhibit 9: Recent in-vivo CAR-T deals
Source: Edison Investment Research, Evaluate Pharma
Competitive pressure from bispecific antibodies: Bispecific T-cell engagers from companies such as Amgen (Blincyto), Regeneron and Roche are achieving high response rates in haematological malignancies with an off-the-shelf format, creating both a competitive threat and a complementary treatment option. Their ease of administration and lower upfront cost are accelerating adoption, particularly in earlier lines of therapy. As a result, CAR-T developers are increasingly focused on demonstrating superior durability and curative potential to maintain differentiation.
The long-term value of CAR-T therapy will depend on its evolution from an autologous, late-line therapy into a scalable, earlier-line treatment modality. Off-the-shelf manufacturing, improved safety profiles and expansion into solid tumours are the key inflection points. Companies that can demonstrate durable responses with faster delivery and outpatient administration are likely to capture a disproportionate share of future growth.
Other promising treatment modalities
Emerging intratumoural modalities
Intratumoural delivery is gaining traction as a strategy to activate the anti-tumour immune response directly at the tumour site while minimising systemic toxicity. By converting immune-cold lesions into inflamed, T cell-infiltrated tumours, these approaches can generate systemic immunity and improve responses to checkpoint inhibitors. Rather than competing with established backbones, most intratumoural therapies are being developed as immune-priming combination partners, with their greatest potential in accessible solid tumours and earlier treatment settings where immune function is less compromised.
Immuno-modulatory antibodies – reprogramming the TME
Immuno-modulatory antibodies are an emerging class of IO agents that work by changing the tumour’s immune environment rather than attacking cancer cells directly. They act on both the innate and adaptive immune system to improve antigen presentation, boost antibody-driven tumour killing and help overcome resistance to PD-1 inhibitors and other targeted antibodies, making them important combination partners. BioInvent’s BI-1206, a first-in-class FcγRIIB-blocking antibody in Phase I/II, is a key example, restoring sensitivity to rituximab in lymphoma with early signs of re-sensitising tumours to pembrolizumab in solid tumours. Overall, these therapies act as immune amplifiers that increase response rates to established checkpoint backbones and are an important source of the next wave of IO value.
Pharma patent cliff – implications
The healthcare sector is approaching an unprecedented loss-of-exclusivity cycle, with >$200bn of branded drug revenue at risk by 2030 (Exhibit 10). The concentration of exposure in a small number of megabrands means the impact will be both financially material and strategically disruptive for large pharma, which will be looking to acquire new assets to replenish pipelines and future revenues.
Exhibit 10: Worldwide sales at risk from patent expiration (2025–30)
Source: Edison Investment Research, Evaluate Pharma
The notable event in this cycle is the expected biosimilar exposure of Merck’s Keytruda from 2028, currently the world’s largest oncology drug with c $31.7bn in sales in 2025. BMS’s Opdivo, with over $10bn in revenue in 2025, faces a similar timeline. Together, these two PD-1 inhibitors account for over $40bn of at-risk revenue, making IO one of the largest contributors to the late-decade patent cliff. This is structurally different from previous cycles because PD-1 therapy is not a single indication franchise, but a treatment backbone across multiple tumour types and lines of therapy.
What it means for the biotech sector
Lifecycle management is necessary but not sufficient: Large pharma is already implementing lifecycle strategies to defend these franchises, most visibly through subcutaneous formulations of PD-1 antibodies such as Keytruda and Opdivo. However, lifecycle management alone is unlikely to offset the scale of the revenue decline. The more durable strategy is to build proprietary combination regimens that extend the clinical relevance of the checkpoint backbone, which in turn increases reliance on external innovation.
Combination-driven innovation – a structural tailwind for biotechs: The Merck-Moderna collaboration around the personalised cancer vaccine V940 illustrates how the patent cliff is reshaping deal-making. Positive Phase II melanoma data in combination with Keytruda led to a global profit-share agreement, reflecting the value of assets that increase responder rates and enable earlier-line use. BioNTech’s neoantigen vaccine partnership with Genentech follows the same logic. These therapies do not compete with PD-1; they expand its addressable population, which makes them strategically attractive and allows smaller companies to monetise early clinical proof of concept.
Cell therapy/CAR-T platform acquisitions and modality leadership: A similar dynamic is evident in cell therapy, where the focus has shifted to scalable next-generation platforms. AbbVie’s acquisition of Capstan for up to $2.1bn, AstraZeneca’s purchase of EsoBiotec for up to $1bn and Gilead/Kite’s acquisition of Interius for $350m all target in-vivo cell-engineering technologies that remove the manufacturing bottlenecks of autologous CAR-T therapies. Eli Lilly’s recent acquisition of Orna for up to $2.4bn reinforces the strategic importance of programmable immune-engineering platforms. These transactions show that modality leadership alone can crystallise significant value for small biotechs, often before pivotal data.
Targeting resistance – the rise of immune-modulatory combinations: Overcoming resistance to PD-1 and other targeted therapy is another major investment theme. BioInvent’s BI-1206, which restores sensitivity to rituximab and pembrolizumab by targeting FcγRIIB. CD47/SIRPα programmes, including assets originating from Trillium (Pfizer) and the AbbVie/I-Mab collaboration, aim to enhance macrophage-mediated tumour cell clearance, although recent clinical setbacks across the class have increased the focus on differentiated safety and combination positioning. By increasing response rates rather than competing with established backbones, these therapies extend the commercial lifespan of PD-1 and are therefore highly attractive combination partners.
From single-asset licensing to platform consolidation: The urgency to replace multi-billion-dollar revenues is also changing the structure of deal-making. Large pharma is increasingly acquiring entire technology platforms, as seen in BioNTech’s $1.25bn acquisition of CureVac and Eli Lilly’s acquisition of Orna. Owning the underlying technology allows rapid generation of multiple clinical assets and shortens time to impact. For small biotechs, this creates an opportunity to monetise platform value earlier and reduces dependence on late-stage, capital-intensive development.
Financing and valuation implications for the biotech sector: The patent cliff creates a multi-year period of structural demand for external innovation. Assets that demonstrate clear synergy with PD-1, durable responses, or the ability to move into earlier lines of therapy are likely to attract partnering interest earlier, at Phase I or II, bringing forward value inflection points. At the same time, the bar for differentiation is rising, with increasing emphasis on biomarker selection, durability and health-economic benefit.
Tougher evidence bar and payer friction post-loss of exclusivity: As pricing dynamics intensify around megabrands, payers will push harder on incremental benefit. Biotechs should expect increasing emphasis on: clear biomarkers, durable endpoints, health-economic outcomes (reduced hospital time, fewer cycles) and differentiated safety/route of administration. In the US, the Inflation Reduction Act and Medicare price negotiation for high-spend biologics further increase long-term pricing pressure, reinforcing the need for clinically differentiated, earlier-line and combination-driven strategies to sustain premium reimbursement.
What it means for investors
Lower commercial risk: From an investor standpoint, IO remains one of the most compelling areas in biotechnology because it combines large commercial markets, multiple value-creation inflection points and strong strategic demand from large pharma. Unlike many therapeutic areas where new entrants must displace entrenched standards of care, most next-generation IO assets are largely developed as combination partners to existing treatments. This significantly lowers commercial adoption risk and increases the probability of partnering at an early clinical stage. For investors, this creates a model in which Phase I/II proof-of-concept data can drive disproportionate valuation re-rating, well ahead of registrational timelines.
Pipeline-in-a-product optionality: Many IO modalities are inherently modular and can be expanded across multiple tumour types and treatment settings. Personalised cancer vaccines, cell therapies and immune-modulatory antibody platforms can generate several clinical programmes from a single technology base. This supports higher risk-adjusted valuations, multiple deal opportunities and lifecycle expansion, which is particularly valuable for small and mid-cap companies.
Accelerated development pathways: IO assets often target high-unmet-need or biomarker-selected populations, making them well suited for expedited regulatory pathways and smaller, faster clinical trials. The use of durable endpoints such as long-term survival or MRD clearance can shorten development timelines and reduce funding requirements, improving the return profile for investors.
Premium pricing: Therapies that deliver deep and durable responses, particularly those with curative potential, can command premium pricing despite high upfront costs. This is already evident in the CAR-T space (eg Kymriah and Yescarta come with list price of $47k and $373k, respectively) and is likely to extend to immune-reset approaches in both oncology and autoimmune disease. Strong pricing power translates into attractive peak-sales potential and supports higher valuation multiples.
Structural deal flow driven by the patent cliff: The loss of exclusivity for major ICIs is creating a multi-year need for revenue replacement among large pharmaceutical companies. This is accelerating licensing and M&A activity for assets that can expand the PD-1 ecosystem or overcome resistance. For investors, this provides a clear and recurring exit pathway through strategic transactions.
Multiple value inflection points across the development cycle: IO programmes typically generate several catalysts, including early immune-response readouts, combination data, expansion into new indications and regulatory designations. This steady flow of milestones supports sustained investor engagement and allows value to be realised progressively rather than only at approval. From a capital markets perspective, IO offers earlier value inflection than most therapeutic areas. Immune-response readouts, durability signals and initial combination data can drive meaningful re-rating well ahead of approval, while sustained M&A demand provides a clear monetisation pathway.
Risk-reward considerations
Investment risks
Despite its transformational clinical impact and strong deal activity, immuno-oncology investments continue to carry meaningful risk.
Clinical development risks:
Commercial risks:
Company-specific risks:
Mitigating factors
Several factors help mitigate traditional IO investment risks.
Strong external demand for innovation
Increasingly validated biology
Large and durable market opportunity
Platform and lifecycle optionality
Investment conclusions
The IO landscape represents one of the most compelling long-term investment themes in healthcare today, driven by scientific validation, platform technologies and the need to replace multibillion-dollar checkpoint revenues. The shift from monotherapy checkpoints to combination-based, immune-ecosystem control marks the next phase of value creation.
Several factors make the current investment environment particularly attractive. The upcoming PD-1 patent cliff is accelerating external innovation and increasing partnering appetite for assets that expand responder populations or overcome resistance. At the same time, advances in biomarker-driven development, cell-engineering, mRNA and in-situ tumour-priming approaches are improving clinical success probabilities and shortening development timelines.
The commercial opportunity remains substantial. Oncology continues to be the largest therapeutic area globally, with immuno-oncology capturing a disproportionate share of drug spending. Movement into earlier lines of therapy, adjuvant and MRD settings is expanding the addressable population, while durable responses and treatment-free remission support premium pricing and strong pharmacoeconomic value.
For investors, immuno-oncology offers multiple avenues for exposure. Large-cap pharmaceutical companies provide franchise stability and lifecycle management of checkpoint backbones, mid-cap innovators offer focused exposure to next-generation modalities and small-cap platform companies present the most asymmetric risk-reward, particularly where early data can unlock partnering or acquisition.
The companies highlighted in this report reflect the breadth of innovation across the field. From personalised cancer vaccines and next-generation cell therapies to immune-modulatory antibodies and intratumoural immune-priming approaches, these platforms are designed to extend the PD-1 ecosystem rather than compete with it, which lowers commercial adoption risk.
Risk mitigation comes from the structural demand for external innovation and the modular nature of many IO technologies. Multiple value inflection points, accelerated regulatory pathways and the ability to monetise assets at Phase I/II through partnerships help offset traditional biotech funding and development risks.
As this next phase of immuno-oncology unfolds, investors who position themselves early in differentiated platforms and combination-relevant assets are likely to benefit from sustained deal flow and valuation re-rating. The sector combines a large market opportunity, strong strategic buyer demand and scalable technologies, creating a highly attractive long-term investment landscape.
Appendix: Company showcase
The following companies represent a selection of the most innovative approaches across the immuno-oncology landscape, each targeting a key biological or commercial bottleneck such as responder expansion, resistance reversal, immune priming or scalable cell therapy delivery (Edison clients are indicated with an asterisk).
BioInvent International
BioInvent International AB (Nasdaq Stockholm: BINV) is a clinical-stage biotech focused on developing first-in-class immune-modulatory antibodies to address key limitations in cancer immunotherapy, particularly treatment resistance and low response rates. Its antibodies target immune checkpoints and tumour microenvironment biology to unlock new mechanisms of action.
The company’s discovery engine is built on its proprietary F.I.R.S.T platform, which enables the simultaneous identification of novel targets and corresponding antibodies directly from patient-derived tumour material. This is complemented by the n-CoDe antibody library, supporting rapid generation of differentiated candidates.
Following a strategic portfolio prioritisation in August 2025, BioInvent is focusing resources on its two lead assets, BI-1206 and BI-1808. BI-1206 (anti-FcγRIIB) is designed to restore sensitivity to anti-CD20 therapies in non-Hodgkin’s lymphoma and enhance responses to PD-1 inhibitors in solid tumours. In a Phase I/IIa trial with rituximab and acalabrutinib, early data from the triple-combination arm showed 100% disease control and a 63% objective response rate. Subsequent data for the Phase IIa safety run-in (n=15) showed an overall response rate of 80%, with 47% achieving complete responses. The disease control rate was 100%.
A second Phase IIa study combining BI-1206 with pembrolizumab in first-line NSCLC and uveal melanoma marks its entry into treatment-naïve settings. The asset has also been reformulated for subcutaneous delivery to improve convenience, safety and durability.
BI-1808 targets TNFR2 to counter tumour immune evasion by depleting regulatory T cells and expanding CD8+ T cells. In Phase IIa cutaneous T-cell lymphoma, it has demonstrated strong immune engagement with 100% disease control and a 45% response rate, alongside ongoing evaluation in combination with pembrolizumab in solid tumours.
Both programmes have received supportive FDA designations, including Orphan Drug and Fast Track status, highlighting their potential in high-unmet-need indications. BioInvent’s integrated GMP manufacturing provides scalable, cost-efficient biologics production and ensures reliable global clinical supply, strengthening its operational and partnering position.
Immix Biopharma*
Immix Biopharma (NASDAQ: IMMX) is a clinical-stage company focused on advancing next-generation CAR-T therapies for patients with relapsed/refractory AL amyloidosis, a rare plasma cell disorder characterised by rapid organ deterioration and poor outcomes with conventional treatments. Its lead and wholly owned programme, NXC-201, is a BCMA-directed autologous CAR-T therapy designed with the objective of inducing deep haematologic responses and enabling meaningful and potentially durable organ recovery.
Clinical data generated to date highlight compelling efficacy in a heavily pretreated patient population, including high overall and complete response rates and early signals of organ improvement. Recent interim data (20 patients) presented from the ongoing Phase Ib/II NEXICART-2 trial (n=40) showed a 75% complete response (CR) rate, with 15/20 patients achieving deep haematological responses. Of the five patients without a CR, four were MRD negative. Importantly, NXC-201 has demonstrated a favourable safety and tolerability profile, with low rates of high-grade CRS and neurotoxicity, a critical consideration in AL amyloidosis patients who are often frail and ineligible for intensive therapies. This safety profile, combined with rapid manufacturing turnaround and reliable product delivery, supports the potential use of NXC-201 in a broader treatment setting and in patients with significant organ involvement.
The ongoing US-based NEXICART-2 study is intended to support a registrational pathway for AL amyloidosis with a biologics licence application planned before end-FY26. The company was recently granted the Breakthrough Therapy Designation for NXC-201 by the FDA based on the strength of the interim data. NXC-201 also holds the Regenerative Medicine Advanced Therapy designation by the US FDA and Orphan Drug Designation by FDA and the EMA.
With CAR-T increasingly recognised as a transformative modality in non-oncology indications, NXC-201 is positioned to address a critical gap for AL amyloidosis patients who have limited therapeutic options and poor prognosis following relapse.
Immutep
Immutep (ASX: IMM) is a late-stage biotechnology company and a pioneer in therapies targeting the MHC Class II and LAG-3 pathways for cancer and autoimmune diseases. Its scientific leadership is rooted in the discovery of LAG-3 by CSO Dr Frédéric Triebel, positioning the company at the forefront of this immune checkpoint field. Immutep’s approach focuses on modulating key regulators of the immune response to enhance anti-tumour activity and improve outcomes for patients who derive limited benefit from existing immunotherapies.
The oncology pipeline is anchored by two clinical-stage assets that employ complementary strategies to stimulate anti-cancer immunity. Efti (eftilagimod alpha) is a first-in-class MHC Class II agonist that directly activates antigen-presenting cells (APCs), including dendritic cells and monocytes. By engaging this central immune control point, efti drives a broad and durable immune response, priming CD8+ cytotoxic T cells and expanding multiple immune effector populations. This mechanism has demonstrated strong synergy with anti-PD-(L)1 therapy, chemotherapy and radiotherapy, alongside a favourable safety profile across combinations.
Efti is currently being evaluated in TACTI-004 (KEYNOTE-F91), a pivotal Phase III trial in partnership with MSD (n=750), in combination with pembrolizumab and chemotherapy as first-line treatment for advanced/metastatic NSCLC (50% enrolment completed). The study builds on positive data from TACTI-002 and INSIGHT-003 (>165 patients), which showed high response rates and encouraging survival outcomes. Notably, efficacy has been observed across the full PD-L1 expression spectrum, including the large low/negative PD-L1 population with significant unmet need. Efti is also being explored in multiple additional solid tumours, including head and neck, urothelial, breast cancer and soft tissue sarcoma.
Immutep’s second clinical asset, LAG525 (ieramilimab), the first anti-LAG-3 antibody, is out-licensed to Novartis, validating the company’s leadership in LAG-3 biology. Earlier-stage programmes include a small-molecule LAG-3 inhibitor and IMP761, the first LAG-3 agonist antibody for autoimmune disease, which has generated promising initial Phase I data (single ascending dose part completed).
Lytix Biopharma
Lytix Biopharma (Euronext Growth Oslo: LYTIX) is developing a novel class of oncolytic immunotherapies designed to harness the full potential of the immune system by directly reprogramming the tumour microenvironment. Its lead asset, ruxotemitide (LTX-315), is a first-in-class intratumoral therapy that rapidly disrupts tumour cell membranes, releasing a broad repertoire of tumour antigens and danger signals that drive immune activation. By initiating immune recognition rather than relying on pre-existing anti-tumour immunity, the approach is positioned to convert immunologically ‘cold’ tumours into ‘hot’ tumours and expand the benefit of checkpoint inhibitors beyond current responder populations.
This mechanism enables broad T-cell priming and durable systemic immune responses, providing a strong biological rationale for combination with PD-1/PD-L1 inhibitors in indications where response rates to checkpoint blockade alone remain limited. Clinically, the company is prioritising injectable and resectable solid tumours, where intratumoral delivery is both feasible and therapeutically meaningful.
In partnership with Verrica Pharmaceuticals, ruxotemitide has completed a Phase II study in non-metastatic skin cancer, demonstrating a 97% objective response rate, 51% pathological complete responses and an 86% mean tumour reduction across treated lesions, alongside a favourable safety profile with no serious adverse events. Based on these data and FDA alignment on trial design, Verrica is preparing a pivotal Phase III programme in basal cell carcinoma. Lytix retains rights in metastatic and neoadjuvant settings, including melanoma, triple-negative breast cancer and Merkel cell carcinoma, where combination strategies with checkpoint inhibitors represent a key value driver.
The pipeline also includes LTX-401, an oncolytic candidate targeting liver metastases, extending the platform to deep-seated tumours. With a validated mechanism, encouraging clinical activity and a flexible partnering strategy, Lytix is positioning intratumoral immunotherapy as a complementary modality within the immuno-oncology landscape, aimed at delivering a durable systemic benefit for patients underserved by current treatments.
Mendus*
Mendus (OMX: IMMU) is a clinical-stage immuno-oncology company developing off-the-shelf cellular cancer vaccines designed to induce durable, tumour-specific immune responses and reduce relapse in haematological malignancies and solid tumours. Its lead programme, vididencel, is an allogeneic dendritic cell-based therapy aimed at establishing long-term immune surveillance in patients with AML and CML.
By priming and expanding tumour-specific T cells, vididencel is intended to eliminate residual malignant cells and deliver sustained disease control without the toxicity associated with intensive consolidation treatments. The off-the-shelf format enables rapid treatment, scalable manufacturing and favourable logistics compared with autologous cell therapies.
Clinical data to date from the ADVANCE II trial has demonstrated encouraging immune responses, a favourable safety profile and signals of relapse reduction in AML patients following first line intensive induction chemotherapy. The programme is now being repositioned to address a broader AML population, including ‘chemo-unfit’ patients receiving venetoclax-based regimens, with new studies (CADENCE and DIVA) expected to inform the design of a potential global registrational trial.
Mendus is also expanding into CML, where vididencel is being developed as a potential treatment-free remission, enabling therapy for patients on long-term TKI therapy, representing a large and well-defined commercial opportunity. Vididencel is also being explored in ovarian cancer and has potential applicability in additional tumour settings where immune priming in the minimal residual disease setting could improve long-term outcomes.
Manufacturing is supported through a dedicated GMP partnership with NorthX Biologics, providing scalable, commercial-ready production capabilities and strengthening the pathway to late-stage development and potential out-licensing. The company is funded into 2027 and is seeking partnerships to support late-stage development and maximise the platform’s potential.
See below for our most recently conducted Edison TV executive interview with Mendus.
Exhibit 11: Executive interview with Erik Manting, CEO of Mendus, and Tariq Mughal, CMO
Source: Edison Investment Research
OSE Immunotherapeutics*
OSE Immunotherapeutics (PAR: OSE) is a clinical-stage biotechnology company developing first-in-class immunotherapies across oncology and immuno-inflammation, with a strategy centred on biomarker-driven precision medicine and differentiated T-cell modulation. Its lead oncology programme, Tedopi, is an off-the-shelf, multi-epitope therapeutic cancer vaccine designed to induce tumour-specific cytotoxic T-cell responses in HLA-A2–positive patients and address resistance to ICIs.
Tedopi is being advanced in NSCLC after ICI failure, a setting with high unmet need and limited treatment options. The vaccine targets five tumour-associated antigens and is engineered to generate durable CD8+ T-cell immunity with a favourable tolerability profile compared with chemotherapy. In the Phase III ATALANTE-1 study, Tedopi demonstrated a clinically meaningful survival benefit versus standard chemotherapy in the ICI-resistant population, establishing proof of concept and supporting its ongoing registrational strategy. The confirmatory ARTEMIA Phase III trial, comparing Tedopi with docetaxel, represents the final step toward potential approval, with a futility analysis expected in 2026.
Beyond oncology, OSE’s immunology pipeline provides additional value creation and partnering optionality. The most advanced asset, lusvertikimab (anti-IL-7R), targets a central pathway in T-cell expansion and is being developed in chronic inflammatory diseases (ulcerative colitis, chronic pouchitis and hidradenitis suppurativa) offering a complementary and potentially less capital-intensive route to late-stage development through partnerships.
See below for our most recently conducted Edison TV executive interview with OSE Immunotherapeutics.
Exhibit 12: Executive interview with Silvia Comis MD, chief clinical and medical research officer at OSE Immunotherapeutics
Source: Edison Investment Research
Percheron Therapeutics*
Percheron Therapeutics (ASX: PER) is a clinical-stage immuno-oncology company developing next-generation checkpoint inhibitors to address resistance to PD-1/PD-L1 therapies. Its lead asset, HMBD-002, targets VISTA (V-domain Ig suppressor of T-cell activation), an emerging immune checkpoint strongly associated with poor response to existing immunotherapies and with tumours that remain largely unresponsive to current treatment, including pancreatic, ovarian and prostate cancers. By expanding the number of patients who can benefit from checkpoint blockade, VISTA inhibition represents a key opportunity in the evolution of the IO landscape.
HMBD-002 is differentiated by its IgG4 backbone, the same isotype used in pembrolizumab and nivolumab, which is designed to minimise unwanted immune activation and improve tolerability, an important advantage in a class where earlier candidates have been limited by toxicity. Preclinical studies have demonstrated broad anti-tumour activity both as monotherapy and in combination with PD-1 inhibitors, including in triple-negative breast cancer models (UT Southwestern) and in radiotherapy combinations in head and neck cancer (Stanford), supporting a strong biological rationale for use in CPI-resistant disease.
The completed US Phase I study (n=48) showed an excellent safety profile across doses from 20–1,400mg, with no maximum tolerated dose reached due to the low incidence of dose-limiting toxicities. Early signs of clinical activity included tumour shrinkage and durable stable disease in heavily pre-treated patients, supporting advancement into later-stage trials. A modular Phase II study is expected to commence within 2026.
See below for our most recently conducted Edison TV executive interview with Percheron Therapeutics.
Exhibit 13: Executive interview with Percheron Therapeutics CEO, Dr James Garner
Source: Edison Investment Research
Poolbeg Pharma
Poolbeg Pharma (LSE: POLB) is developing POLB 001, a first-in-class, orally administered p38 MAPK inhibitor designed to prevent cancer immunotherapy-induced CRS, one of the most serious and resource-intensive toxicities associated with T-cell-engaging bispecific antibodies and CAR-T therapies. CRS affects more than 70% of patients receiving certain T-cell-redirecting treatments and currently necessitates administration in specialised centres, leading to prolonged hospitalisation, high healthcare utilisation and constrained patient access. With no approved preventative therapies, POLB 001 is positioned to address a critical bottleneck in the delivery and scalability of next-generation immuno-oncology treatments.
By selectively modulating excessive inflammatory signalling without causing broad immunosuppression, POLB 001 aims to improve the safety profile of T-cell-engaging therapies while preserving anti-tumour immune function, an important consideration in heavily pre-treated cancer populations. Enabling safer outpatient or community-hospital administration could significantly expand treatment capacity, improve patient quality of life and unlock additional commercial value across the rapidly growing cell therapy and bispecific antibody markets.
The initial commercial opportunity, focused on multiple myeloma and diffuse large B-cell lymphoma, is estimated to exceed US$10bn, with further upside as T-cell-engaging therapies move into earlier lines of treatment and into solid tumours. The programme has demonstrated a favourable safety and pharmacodynamic profile in preclinical studies and in a human lipopolysaccharide (LPS) challenge model, providing important translational validation ahead of patient studies.
The Phase IIa open-label, single-arm trial is in advanced preparation and will evaluate POLB 001 in approximately 30 relapsed/refractory multiple myeloma patients receiving an approved bispecific antibody (teclistamab), with interim data expected in summer 2026. The study is being led by The Christie NHS Foundation Trust and the Accelerating Clinical Trials network. FDA Orphan Drug Designation provides additional regulatory validation and commercial optionality. On positive data, Poolbeg expects to pursue a partnering strategy, positioning POLB 001 as a potential enabling therapy for the broader immuno-oncology ecosystem.
Scancell
Scancell (LSE: SCLP) is a clinical-stage immuno-oncology company developing targeted active immunotherapies designed to generate durable, tumour-specific T-cell responses and address the large proportion of patients who fail to benefit from checkpoint inhibitors (ICIs). With approximately half of patients not responding to ICIs and five-year survival in stage IV melanoma below 23%, Scancell’s off-the-shelf platforms aim to enhance efficacy without adding toxicity while improving accessibility versus personalised approaches.
The lead asset, iSCIB1+ (DNA Immunobody), is a needle-free DNA vaccine that induces high-avidity T cells against gp100 and TRP-2 melanoma antigens, selected from patients with spontaneous tumour regression. In the Phase II SCOPE study (n=140), iSCIB1+ in combination with ICIs has demonstrated clinically meaningful improvements over historical CPI outcomes. Progression-free survival in the target HLA population reached 78% at 11 months versus ~46% for nivolumab/ipilimumab, with 69% PFS at 22 months across cohorts. Updated data remained encouraging with PFS of 74% at 16 months in the target population. These data support the potential to establish a new standard of care in advanced melanoma. With FDA IND clearance in place, development is being accelerated towards a randomised registrational study in 2026, likely incorporating a precision biomarker. A commercial-scale GMP manufacturing process with strong stability has already been established, supporting future scalability.
The second clinical programme, Modi-1 (Moditope), is a peptide-based active immunotherapy targeting post-translationally modified tumour antigens and is currently in Phase II evaluation in hard-to-treat solid tumours, including head and neck cancer, with further data expected in early 2026.
Beyond vaccines, Scancell’s GlyMab platform, developed within its Glymab Therapeutics subsidiary, generates high-affinity antibodies against tumour-specific glycans, with two assets already licensed to Genmab, providing external validation and potential non-dilutive value.
See below for our recent Edison TV executive interview with Scancell.
Exhibit 14: Executive Interview with Scancell Holdings’ chief medical officer, Dr Nermeen Varawalla
Source: Edison Investment Research
Transgene
Transgene (FRA: TNG) is a clinical-stage biotechnology company developing virus-based immunotherapies, with a strategic focus on individualised neoantigen therapeutic vaccines (INTVs) to prevent cancer relapse in early-stage disease. Its lead programme, TG4050, is based on the proprietary myvac platform and is currently being evaluated in the randomised Phase II part of a Phase I/II trial in operable head and neck cancer (HNSCC).
The myvac platform integrates viral vector engineering, artificial intelligence and translational data to deliver precision immunotherapy at clinically relevant timelines. Using a Modified Vaccinia Ankara (MVA) vector, TG4050 encodes patient-specific neoantigens identified through AI-driven algorithms developed with NEC. This approach is designed to generate potent and durable CD8+ T-cell responses against tumour mutations unique to each patient, with the aim of establishing long-term immune surveillance following surgery and standard-of-care treatment. A digitalised, in-house GMP manufacturing process supports scalable production within a timeframe compatible with routine clinical practice.
Clinical data presented at ASCO 2025 demonstrated the feasibility, safety and strong immunogenicity of TG4050, with 100% disease-free survival at a median 30-month follow-up (n=32). Neoantigen-specific CD8+ T-cell responses persisted for more than two years and showed effector and memory phenotypes consistent with durable protection against relapse. Additional translational analyses presented at SITC 2025 confirmed the induction of polyclonal cytotoxic T cells, expansion of both de novo and pre-existing tumour-reactive clones and the development of tissue-resident immune populations, highlighting the potential to overcome resistance in immunologically ‘cold’ tumours.
With proof of concept established, Transgene is advancing TG4050 towards a registrational pathway and exploring expansion into additional early-stage solid tumours. More broadly, myvac represents a scalable platform for a pipeline of individualised vaccines, positioning the company at the forefront of next-generation precision immuno-oncology.
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 转移性头颈部鳞状细胞癌 | 临床3期 | - | 2019-10-01 | |
| 胃食管交界处癌 | 临床3期 | 美国 | 2019-09-30 | |
| 胃食管交界处癌 | 临床3期 | 中国 | 2019-09-30 | |
| 胃食管交界处癌 | 临床3期 | 德国 | 2019-09-30 | |
| 胃食管交界处癌 | 临床3期 | 意大利 | 2019-09-30 | |
| 胃食管交界处癌 | 临床3期 | 波兰 | 2019-09-30 | |
| 胃食管交界处癌 | 临床3期 | 新加坡 | 2019-09-30 | |
| 胃食管交界处癌 | 临床3期 | 韩国 | 2019-09-30 | |
| 胃食管交界处癌 | 临床3期 | 中国台湾 | 2019-09-30 | |
| 胃食管交界处癌 | 临床3期 | 英国 | 2019-09-30 |
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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 | 60 | 鏇顧鏇廠製製醖製鹽壓(襯夢構築窪鑰膚壓範襯) = 醖憲遞蓋廠夢艱簾淵顧 夢鹽構鹹廠範願壓糧壓 (製夢齋艱衊觸夢繭觸鹹 ) | 不佳 | 2025-06-01 | |||
鏇顧鏇廠製製醖製鹽壓(襯夢構築窪鑰膚壓範襯) = 鑰觸衊鹹蓋壓觸選膚願 夢鹽構鹹廠範願壓糧壓 (製夢齋艱衊觸夢繭觸鹹 ) | |||||||
临床2/3期 | 82 | (Chemotherapy-free Arm) | 醖鑰齋遞築鏇顧廠願壓 = 獵膚構壓願鹽獵積艱衊 襯獵窪鹽鑰網積簾構選 (簾選廠選糧簾醖蓋構鏇, 築顧衊淵膚糧鑰網廠廠 ~ 製鑰壓壓鬱鹹衊膚鏇積) 更多 | - | 2025-04-22 | ||
Chemotherapy+Trastuzumab (Trastuzumab and Chemotherapy Arm) | 蓋網範窪鹽廠憲壓蓋築 = 蓋積網膚鬱簾顧網襯廠 餘獵艱構蓋築鏇鹽顧齋 (鏇廠願膚鏇窪觸艱繭獵, 積積簾鬱鹽蓋積願淵願 ~ 遞憲觸鏇願觸憲繭簾鹹) 更多 | ||||||
临床1/2期 | 89 | (Phase 1: MGD013 120mg) | 衊鬱積顧蓋餘願鏇顧淵 = 淵鹹憲憲窪廠顧襯網鹹 憲繭遞鹽夢淵壓遞網衊 (鹹蓋鹽襯衊蓋鑰鹹鏇蓋, 餘窪簾廠顧憲簾積衊鏇 ~ 顧艱糧構夢餘鹽築構繭) 更多 | - | 2024-07-23 | ||
(Phase 1: MGD013 240mg) | 衊鬱積顧蓋餘願鏇顧淵 = 選膚夢糧醖鹽蓋壓齋壓 憲繭遞鹽夢淵壓遞網衊 (鹹蓋鹽襯衊蓋鑰鹹鏇蓋, 窪製範膚衊製鹽鏇積觸 ~ 廠蓋觸繭範簾襯餘獵壓) 更多 | ||||||
临床2期 | 62 | (Retifanlimab Cohort) | 築廠願憲蓋願構憲廠積 = 構鹹餘選廠廠製繭鹽醖 獵鏇壓簾獵觸鏇簾築襯 (簾糧範窪鹽鏇觸獵鹽選, 醖鹹醖獵鬱糧製選網醖 ~ 鬱糧鹽糧網繭鏇製廠鑰) 更多 | - | 2023-12-20 | ||
(Tebotelimab Cohort) | 選廠構餘鬱鏇廠衊膚夢 = 壓淵壓築蓋夢觸膚艱鹽 觸壓壓簾膚窪鑰觸淵鏇 (遞顧鏇衊獵繭窪範衊鬱, 鑰憲蓋範鏇簾餘膚鬱鹹 ~ 蓋衊構顧築範窪鹽繭淵) 更多 | ||||||
临床1期 | 25 | (efficacy analysis set) | 製夢繭顧齋憲願艱餘築(廠繭廠顧蓋遞蓋築遞糧) = 鏇襯鏇遞憲窪壓獵衊憲 膚齋廠製範獵襯觸壓網 (簾醖簾淵網積築窪積衊, 9 ~ 45) 更多 | 积极 | 2023-04-14 | ||
(IRC-response evaluable set) | 製夢繭顧齋憲願艱餘築(廠繭廠顧蓋遞蓋築遞糧) = 築築憲艱艱糧鬱蓋選願 膚齋廠製範獵襯觸壓網 (簾醖簾淵網積築窪積衊, 12 ~ 54) 更多 | ||||||
临床1/2期 | 69 | (dose escalation phase) | 繭淵鑰遞構製鑰壓鏇選(鹹鹽壓膚鏇夢築範網膚) = 襯鹹鏇鹽鹹膚醖鬱選簾 襯選積餘淵齋壓築遞積 (衊鏇獵鏇築艱蓋簾鏇蓋 ) 更多 | 积极 | 2023-01-24 | ||
(expansion phase) | 膚艱鹹夢壓鹽鬱簾觸淵(製遞網夢觸糧醖顧壓壓) = 願築鏇鬱壓憲夢糧糧鬱 廠鹽構衊獵艱繭鬱鹽獵 (構壓廠構夢壓鬱遞獵憲 ) 更多 | ||||||
临床1期 | 27 | 鑰範顧鹹艱廠鏇遞網餘(積醖選鑰願網獵憲廠糧) = 鬱製鏇願齋艱夢壓醖鹽 壓夢艱艱齋鹽蓋膚簾鹹 (顧艱廠構糧齋簾憲繭範 ) 更多 | 积极 | 2023-01-24 | |||
临床2/3期 | - | 構窪窪構淵鹽觸繭鹽窪(鹽窪醖繭鹽範壓膚簾構) = 壓鑰選鑰蓋醖範淵遞膚 壓鏇選觸醖齋憲蓋鑰遞 (膚艱鏇艱遞遞壓範築襯 ) | 积极 | 2021-07-03 | |||
临床1期 | 20 | 壓餘衊艱構鬱膚遞廠積(鬱觸製膚醖鏇壓選積艱) = 積鏇製鑰獵鏇繭範願壓 鹽網構餘衊鏇糧齋製積 (鹽構糧獵壓鬱獵築選選 ) 更多 | 积极 | 2020-12-07 | |||
临床1期 | 31 | 積憲鑰鬱鹽鏇鹽醖鏇選(憲齋構鑰網壓醖網膚繭) = 網繭鏇壓窪積蓋獵顧遞 膚網觸壓窪鬱衊鹽網築 (簾廠膚糧觸壓鹽範鹹顧 ) | 积极 | 2020-11-09 | |||
積憲鑰鬱鹽鏇鹽醖鏇選(憲齋構鑰網壓醖網膚繭) = 簾窪鹹衊製糧齋構願範 膚網觸壓窪鬱衊鹽網築 (簾廠膚糧觸壓鹽範鹹顧 ) |
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