Phase III Double Blinded Trial of Immune-Based Therapy With a Live Biotherapeutic MO-03 or Placebo for Frontline Therapy of Advanced Clear Cell Renal Cell Carcinoma [BioFront Trial]

This phase III trial compares the effect of adding live biotherapy, MO-03, to standard of care (SOC) immunotherapy, including ipilimumab, nivolumab, axitinib, pembrolizumab, cabozantinib, and lenvatinib, to SOC immunotherapy alone in treating patients with clear cell renal cell cancer that may have spread from where it first started (primary site) to nearby tissue, lymph nodes, or distant parts of the body (advanced) or that has spread from where it first started to other places in the body (metastatic). Studies have shown that gut health (the gut microbiome) may impact the effectiveness of immunotherapy. The microbiome includes all of the bacteria and organisms naturally found in the digestive tract. MO-03, a type of biotherapy, contains material from living organisms that may help keep the digestive tract healthy and may help to increase the effect of immunotherapy. Immunotherapy with monoclonal antibodies, such as ipilimumab, nivolumab, pembrolizumab, may help the body's immune system attack the tumor, and may interfere with the ability of tumor cells to grow and spread. Axitinib, cabozantinib, and lenvatinib are a type of angiogenesis inhibitor and tyrosine kinase inhibitor (TKI) that block certain proteins which may help keep tumor cells from growing and may also help prevent the growth of new blood vessels that tumors need to grow. Adding MO-03 to SOC immunotherapy may be more effective than SOC immunotherapy alone in treating patients with advanced or metastatic clear cell renal cell cancer.

开始日期2026-06-10

申办/合作机构

SWOG

[+1]

NCT07187869

/ Not yet recruiting临床1期IIT

Modulation of the Bone Immune Microenvironment Following Cabozantinib Treatment of Bone Metastatic Clear Cell Renal Cell Carcinoma: Proof of Concept Study

The goal of our study is to explore how treatments affect the bone immune microenvironment and determine the best treatment options for patients with metastatic kidney cancer to the bone. This could prevent skeletal complications and improve patient outcomes.

开始日期2026-03-31

申办/合作机构

The University of Texas MD Anderson Cancer Center

[+1]

NCT07293351

/ Not yet recruiting临床1/2期

ROSETTA RCC-208: A Phase 1/2 Open-label, Multi-center, Randomized Study of Pumitamig Alone or in Combination With Ipilimumab or Cabozantinib in Participants With Advanced Renal Cell Carcinoma (RCC)

The purpose of this study is to evaluate the safety, tolerability, and efficacy of Pumitamig alone or in combination with Ipilimumab or Cabozantinib in participants with advanced Renal Cell Carcinoma (RCC)

开始日期2026-03-26

申办/合作机构

Bristol Myers Squibb Co.

[+1]

100 项与苹果酸卡博替尼相关的临床结果

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100 项与苹果酸卡博替尼相关的转化医学

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100 项与苹果酸卡博替尼相关的专利（医药）

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2,090

项与苹果酸卡博替尼相关的文献（医药）

2026-04-01·LUNG CANCER

RET Fusion–Positive Lung Adenocarcinoma: Partner-Specific Clinicopathological Characteristics, Co-Mutation Profiles, and Implications for Targeted and Immunotherapy

Article

作者: Wang, Bingzhi ; Gao, Lina ; Ying, Jianming ; Liu, Tianyi ; Li, Weihua ; Guo, Lei ; Zhao, Zuxuan ; Sun, Zihan ; Xu, Jiaxi

BACKGROUND:

RET fusions represent actionable oncogenic drivers in lung adenocarcinoma (LUAD). However, the clinicopathological features, co-mutation landscape, and therapeutic outcomes across different fusion partners remain incompletely characterized.

METHODS:

We retrospectively analyzed 268 patients with RET fusion-positive LUAD diagnosed between July 2017 and December 2024. Clinicopathological features, metastatic patterns, and concomitant genomic alterations were compared between KIF5B and non-KIF5B subgroups. Treatment outcomes of selective RET inhibitors, multikinase inhibitors (MKIs), and immunotherapy combined with chemotherapy were evaluated, with subgroup analyses according to fusion partners.

RESULTS:

The median age at diagnosis was 58 years; most patients were female (57.0%) and never/light smokers (60.8%). Bone (12.3%), pleural (11.9%), and brain metastases (6.7%) were the most common metastatic sites. KIF5B-RET fusions were more frequently detected in earlier disease stages compared with non-KIF5B (p = 0.0531). Among 274 RET fusion events, KIF5B-RET was predominant (65%), followed by CCDC6-RET (16%) and NCOA4-RET (1%). Concomitant mutations were identified in 28.7% of patients, most commonly TP53 (39.0%) and CDKN2A (13.0%), with CDKN2A alterations predominantly consisting of functionally inactivating SNVs concurrent with shallow deletions more enriched in non-KIF5B (p = 0.0393). Nineteen patients received targeted therapy, including pralsetinib, selpercatinib, and cabozantinib, achieving an overall ORR of 57.9% and mPFS of 12.0 months. Notably, non-KIF5B patients demonstrated longer median PFS than KIF5B patients under pralsetinib (17.0 vs. 5.5 months, p = 0.0473). Fifteen patients received first-line immunochemotherapy, achieving a median PFS of 17.0 months, ORR of 40.0%, and DCR of 80.0%, comparable to targeted therapy (p = 0.3871).

CONCLUSIONS:

RET fusion-positive LUAD comprises biologically heterogeneous subsets defined by fusion partners. KIF5B-RET tend to occur at earlier stages, whereas non-KIF5B are more frequently associated with CDKN2A co-mutations and may derive greater benefit from selective RET inhibition. Immunochemotherapy demonstrates comparable efficacy regardless of fusion partner, highlighting the need for partner-specific therapeutic strategies in RET fusion-positive LUAD.

2026-03-01·DRUG RESISTANCE UPDATES

UBL3 governs VEGFR inhibitor resistance by activating NOTCH signaling in renal cell carcinoma

Article

作者: Zhang, Xiaoping ; Fang, Kanglin ; Shi, Jian ; Yang, Hongmei ; Miao, Daojia ; Gong, Jinshuo ; Tan, Diaoyi ; Xiong, Zhiyong ; Wu, Songming ; Zhao, Chuanyi ; Yang, Junkai ; Ruan, Hailong ; Xiao, Wen ; Ye, Yuzhong ; Lv, Qingyang ; Lu, Feiyi

BACKGROUND:

Targeted therapy is the first-line treatment for patients with metastatic renal cell carcinoma (RCC), with vascular endothelial growth factor receptor inhibitors (VEGFRis) constituting the bulk of regimens used. Although the repertoire of VEGFRis for RCC now spans from sunitinib to cabozantinib, resistance to treatments has emerged as a common and prominent challenge. Thus, identifying novel therapeutic targets has become essential for enhancing the antitumor efficacy of current treatments and inhibiting RCC progression.

METHOD:

To investigate the potential mechanisms underlying VEGFRi resistance in RCC, we performed a genome-wide CRISPR/Cas9 library screen under sunitinib and cabozantinib treatment and identified UBL3 as a key driver of VEGFRi resistance in RCC cells. The critical role of UBL3 in promoting VEGFRi resistance was validated using CCK8 assays, flow cytometry, TUNEL assays, and bioinformatics analyses. To elucidate the molecular mechanisms underlying UBL3, we utilized western blotting, RNA sequencing, chromatin immunoprecipitation, small extracellular vesicles (sEVs) isolation, and Astral-DIA proteomics. The contribution of UBL3 to VEGFRi resistance was further confirmed through comprehensive in vitro and in vivo experiments.

RESULTS:

UBL3 was confirmed to suppress apoptosis and promote VEGFRi resistance through NOTCH signaling activation. Further investigations highlighted the importance of NOTCH signaling in VEGFRi resistance in RCC via the NOTCH-PTEN-AKT and NOTCH-FOS pathways and revealed the mechanisms by which UBL3 activated NOTCH signaling. On the one hand, UBL3 formed complex with NOTCH2 and ADAM17 simultaneously, accelerating ADAM17-mediated cleavage of NOTCH2. On the other hand, UBL3-modified NOTCH2 was sorted into sEVs, which were taken up by recipient cells, activating NOTCH signaling and thereby transmitting VEGFRi resistance. Finally, lipid nanoparticle-mediated delivery of the CRISPR/Cas9 knockout system targeting UBL3 effectively restored the sensitivity of RCC tumors to VEGFRis.

CONCLUSION:

This study emphasized the importance of UBL3 in VEGFRi resistance in RCC and proposed that UBL3 activated NOTCH signaling through two distinct pathways, thereby suppressing cancer apoptosis and promoting resistance to VEGFRis. These findings provided a solid scientific foundation and paved the way for the development of novel therapeutic strategies for patients with advanced RCC.

2026-03-01·LIFE SCIENCES

Cabozantinib induces NLRP3-CASP1-GSDMD-dependent pyroptosis in hepatocellular carcinoma

Article

作者: Ou, Da-Liang ; Chang, Hsin-Yi ; Feng, Zhi-Rui ; Hsu, Chiun ; Chung, Chih-Hung ; Lin, Li ; Yang, Muh-Hwa ; Cheng, Ann-Lii ; Cheng, Han-Ying ; Jian, Cheng-Zhe ; Hsu, Chia-Lang

AIMS:

Cabozantinib is a multi-target tyrosine kinase inhibitor approved for second-line treatment of advanced hepatocellular carcinoma (HCC). While its antiangiogenic and antiproliferative effects are well known, its immunomodulatory mechanisms remain incompletely understood. Here, we aimed to investigate the novel immunomodulatory mechanism of cabozantinib, focusing on its role in inducing pyroptotic cell death in HCC cells and myeloid-derived suppressor cells (MDSCs).

MATERIALS AND METHODS:

Cabozantinib-induced pyroptosis was evaluated in HCC cell lines and MDSCs. The involvement of the canonical NLRP3-CASP1-GSDMD pathway was validated using MDSCs induced from Nlrp3 knockout mice and Gsdmd knockout HCC cells. In vivo efficacy and immunomodulation were assessed using Gsdmd knockout Hepa1-6 tumors in immunocompetent mice, analyzed via flow cytometry, single-cell RNA sequencing, and phosphoproteomics.

KEY FINDINGS:

We show that cabozantinib induces pyroptotic cell death in both HCC cells and MDSCs via the canonical NLRP3-CASP1-GSDMD pathway. Disruption of this pathway using MDSCs induced from Nlrp3 knockout mice or Gsdmd knockout HCC cells abrogated cabozantinib-induced pyroptosis. Critically, implantation of Gsdmd knockout tumors in vivo abolished the antitumor and immunomodulatory effects of cabozantinib, confirming the necessity of GSDMD-mediated pyroptosis. Furthermore, cabozantinib reduced MDSC accumulation and enhanced cytotoxic CD8⁺ T cell responses, findings further supported by phosphoproteomic analysis showing pyroptosis-associated signaling alterations.

SIGNIFICANCE:

These findings reveal a mechanistic link between cabozantinib-induced pyroptosis and immune activation. Our results provide a strong rationale for the clinical combination of cabozantinib with immunotherapy in the treatment of HCC.

882

项与苹果酸卡博替尼相关的新闻（医药）

2026-02-27

·Biotech前瞻

共识解读丨2026年《原发性肝细胞癌后线规范化用药专家共识》

点击蓝字关注我们本期看点我国是肝癌高发国家，一线治疗后患者的后线诊疗需求巨大，但长期存在耐药界定不统一、进展模式对应换药时机模糊，以及对于一线接受免疫联合方案治疗的患者，现有后线治疗方案缺乏高级别循证医学支持等问题，影响患者治疗及生存获益。近日，由中国老年学及老年医学学会转化医学分会、北京医学会肝病学分会、中国临床肿瘤学会肝癌专家委员会共同制定的《原发性肝细胞癌后线规范化用药专家共识》（以下简称《共识》）于《中华肝脏病杂志》正式发布。声明：本文内容仅供学术交流与个人学习参考。完整版权归原作者及中华肝脏病杂志所有。引用链接如下：中国老年学及老年医学学会转化医学分会, 北京医学会肝病学分会，中国临床肿瘤学会肝癌专家委员会. 原发性肝细胞癌后线规范化用药专家共识[J]. 中华肝脏病杂志, 2026, 34(1):32-48. DOI: 10.3760/cma.j.cn501113-20250919-00397 . 通信作者：陆荫英，解放军总医院第五医学中心肝病医学部本期内容 01 一线治疗进展的界定及换药时机 02 二线治疗 03 三线等后续治疗 04 总结【01 一线治疗进展的界定及换药时机】推荐意见 1：肝癌系统治疗进展可由原发性耐药和继发性耐药所致。原发性耐药指系统治疗过程中未见肿瘤退缩至肿瘤进展，继发性耐药指系统治疗过程中肿瘤退缩或长期稳定（靶向治疗 3 个月、免疫治疗 6 个月）后出现肿瘤进展。原发性耐药定义：靶向治疗 3 个月内出现疾病进展；免疫单药或免疫联合治疗最佳反应为疾病进展或疾病稳定时间<6 个月（专家共识度 93.16%）。推荐意见 2：靶向联合免疫治疗后，优先采用 RECIST1.1标准进行疗效评价。如果存在假性进展的可能性，需结合iRECIST标准进行综合评估，以确保对治疗反应的准确判断（专家共识度95.83%）。推荐意见3：通过影像学检查、血清标志物和临床表现综合评估，准确区分进展模式，为治疗决策提供依据。寡进展定义为整体疾病稳定或缓解，所有器官出现新发或进展的经影像学证实的病灶不多于5个且受累器官不超过3个（专家共识度98.61%）。推荐意见4：确定换药时机需综合多方面因素，且患者需规范完成影像学检查（每6～8周进行1次）。对于原发性耐药患者，需在达到肿瘤评估进展后及时更换系统治疗方案；对于继发性耐药的患者，建议根据进展模式对肝癌患者实行精细化管理。对于寡进展患者，建议采用局部治疗联合现有系统治疗，若3个月内再次出现进展，则应更换系统治疗方案；对于非寡进展患者，建议切换至后线治疗方案或入组临床试验（专家共识度98.61%）。推荐意见5：若患者出现严重的药物不良反应，即便无明确病情进展，也应根据通用不良事件术语标准（CTCAE）分级调整或停用药物，并根据后续恢复及发作情况评估继续或调整系统治疗方案（专家共识度97.22%）。推荐意见6：对于腹膜转移、癌性腹水/胸水、肠系膜/下腔静脉癌栓等出现复发/进展时，按照非寡进展原则予以更换系统治疗方案（专家共识度95.83%）。推荐意见7：肿瘤进展后需基于具体耐药种类及机制实施精准干预，推荐选用靶点更丰富、疗效更强的药物，同时也可考虑换用或联合应用不同作用机制的药物。靶向治疗耐药时，继续使用原药物可能导致永久性耐药。建议换用另一种多靶点、适度抗血管生成、深度阻断通路间交互作用的靶向药物或化学治疗药物。免疫治疗耐药时，仅更换ICIs效果有限。建议在维持或更换ICIs的同时，换用能够逆转肿瘤免疫抑制性微环境（例如解除M2型免疫抑制等），且与免疫治疗协同增效的靶向及化学治疗药物（专家共识度98.61%）。 02 二线治疗目前已经上市的二线治疗药物包括瑞戈非尼、阿帕替尼、卡瑞利珠单抗、帕博利珠单抗等（表1）。推荐意见8：对于索拉非尼治疗失败的患者，可按照国家卫生健康委员会及中国临床肿瘤学会2024版推荐选择用药，包括瑞戈非尼、卡博替尼、仑伐替尼、雷莫西尤（AFP≥400 ng/mL）、阿帕替尼、纳武利尤单抗联合伊匹木单抗、帕博利珠单抗、卡瑞利珠单抗、替雷利珠单抗治疗，也可以尝试将TKIs联合ICIs作为二线治疗方案（专家共识度97.22%）。推荐意见9：对于仑伐替尼治疗失败的患者，优先选择联合ICIs的治疗包括瑞戈非尼/仑伐替尼（等TKIs）联合ICIs、阿替利珠联合贝伐珠单抗方案，亦可尝试瑞戈非尼、替雷利珠等单药方案，索拉非尼单药不作为治疗推荐，并重视多学科综合治疗的应用（专家共识度98.61%）。推荐意见10：对于多纳非尼治疗失败的患者，可参考索拉非尼的后续治疗策略。鉴于当前相关临床数据有限，亟须进一步积累更多的临床证据以优化此类患者的治疗方案（专家共识度97.22%）。推荐意见11：对于 “T+A” 方案治疗失败的患者，推荐优先选择瑞戈非尼/仑伐替尼等TKIs±PD-1抑制剂作为后续治疗方案。可考虑其他二线药物，如雷莫西尤单抗、卡博替尼、仑伐替尼单药以及纳武利尤单抗联合伊匹木单抗，但需进一步积累临床证据以优化治疗决策（专家共识度95.83%）。推荐意见12：对于信迪利单抗联合贝伐珠单抗生物类似物、阿帕替尼联合卡瑞利珠单抗、替西木单抗联合度伐利尤单抗以及纳武利尤单抗联合伊匹木单抗治疗失败的患者，目前尚缺乏明确的二线治疗方案。基于疗效概率排名，可选择瑞戈非尼、卡博替尼、仑伐替尼、帕博利珠单抗以及阿帕替尼单药或联合治疗。同时建议密切关注相关临床研究进展，并与患者充分沟通治疗选择和预期效果，以便制定最佳的个体化治疗策略（专家共识度97.22%）。推荐意见13：对于TKIs联合ICIs治疗失败的患者，目前尚缺乏明确的二线治疗方案，可考虑瑞戈非尼+ICIs、卡博替尼、雷莫西尤单抗、瑞戈非尼单药及纳武利尤单抗联合伊匹木单抗治疗，对于PD-1/PD-L1原发性耐药的患者也可考虑PD-1/PD-L1与CTLA-4联合治疗，并重视多学科综合治疗的应用，同时也需要进一步积累相关临床证据以优化此类患者的治疗方案（专家共识度98.61%）。推荐意见14：对于ICIs单药治疗失败的患者，如无禁忌推荐选择TKIs联合ICIs或者TKIs单药作为后续治疗方案，TKIs可考虑瑞戈非尼、卡博替尼及仑伐替尼。纳武利尤单抗联合伊匹木单抗治疗可作为PD-1/PD-L1继发性耐药后治疗选择，但目前缺乏循证学依据（专家共识度100%）。推荐意见15：对于一线方案不限的患者，推荐选择瑞戈非尼联合PD-1抑制剂或阿帕替尼联合卡瑞利珠单抗作为后续治疗方案，同时需要进一步积累其他治疗方案的数据以优化临床实践（专家共识度95.83%）。推荐意见16：对于肝癌合并门静脉高压患者，在选择系统治疗方案时，应综合考虑胃镜及影像检查的结果，在使用卡维地洛/普萘洛尔降低门静脉压力的同时，优先选择瑞戈非尼、索拉非尼等可以协同降低门静脉高压的治疗方案或者ICIs这些对于门静脉压力影响较小的方案，避免选择明显加重静脉曲张或门静脉高压的治疗方案。ICIs对于门静脉压力的影响还有待进一步证实及探索（专家共识度97.22%）。推荐意见17：对于肝癌合并消化道出血患者，在选择系统治疗方案时，应优先选择出血风险较低的系统治疗方案，在出血停止后优先建议ICIs单药及联合治疗，谨慎使用如瑞戈非尼、阿帕替尼、卡博替尼、贝伐珠单抗等靶向治疗方案。条件允许时可通过多学科综合治疗协作（multidisciplinary team， MDT）评估经颈静脉肝内门体静脉分流术、内镜下治疗等降低出血风险（专家共识度97.22%）。推荐意见18：对于Child-Pugh B级患者，需要重视个体化评估，推荐选择瑞戈非尼、卡博替尼、纳武利尤单抗或阿可拉定作为Child-Pugh B7级患者二线治疗，推荐纳武利尤单抗、卡博替尼或阿可拉定作为B8患者二线治疗， B9患者目前缺乏循证学依据但可尝试阿可拉定或纳武利尤单抗等ICIs治疗，同时重视保肝和支持治疗，根据患者耐受性个体化调整剂量；对于Child-Pugh C级患者，仅有阿可拉定可作为推荐，同时建议优先参与临床研究，并加强保肝和支持治疗。鼓励所有失代偿患者积极参与相关临床研究，以获取潜在新疗法带来的获益，并为后续治疗策略的优化提供宝贵的临床数据（专家共识度95.83%）。推荐意见19：对于肝癌合并肝性脑病反复发作的患者，慎重使用仑伐替尼、阿帕替尼等靶向药物治疗，在脱氨、蛋白饮食、调节肠道菌群的基础上，可联合ICIs治疗，通便、低应用时需基于个体化评估并严密监测患者的反应及任何可能的不良事件（专家共识度98.61%）。推荐意见20：对于肝移植后肿瘤复发而一线治疗进展的患者，优先推荐瑞戈非尼、卡博替尼、仑伐替尼等TKIs，贝伐珠单抗、雷莫西尤单抗、化学治疗单药或联合使用作为二线治疗选择。在其他系统治疗药物无法有效控制肿瘤的情况下，建议完善正常肝组织PD-L1水平表达的检测，对于阴性表达患者经MDT会诊及充分沟通后，可考虑使用ICIs单药或联合作为挽救性治疗，不推荐使用免疫联合治疗方案。同时需密切监测可能的排斥反应：与CTLA-4单抗相比，PD-1单抗治疗的肝移植受者更容易出现排斥反应，移植术后时间短（＜2年）、免疫抑制药物减量、对供体特异性抗体的敏感性增加都是ICIs治疗后继发排斥反应的高危因素（专家共识度94.44%）。推荐意见21：对于肝癌合并蛋白尿患者，推荐ICIs单药及联合治疗、瑞戈非尼单药作为二线治疗选择，慎重选择含仑伐替尼、贝伐珠单抗以及雷莫西尤单抗方案，同时需警惕后续治疗仍可能导致肾功能持续损伤至不可逆改变（专家共识度97.22%）。【03 三线等后续治疗】推荐意见22：肝癌的三线及以上治疗要尽可能地保证患者生活质量，最大限度的延长生存获益，可考虑使用既往未曾使用过的一、二线方案，也可考虑在MDT指导下，合理选择联合策略（专家共识度100%）。推荐意见23：在肝癌治疗的多线模式中，应始终将肝功能的保护和管理作为核心考量因素，选择对肝功能影响较小的治疗方案，并优化现有治疗手段，以确保后续治疗的可行性并改善整体预后（专家共识度98.61%）。 04 总结中晚期肝细胞癌系统治疗已进入免疫联合的新时代，而现有指南推荐的二线治疗方案远远不能满足一线免疫联合治疗后进展患者的实际需求，亟待开展相关临床研究获得更高级别循证依据。连续肝脏病理检查及基因测序，有助于寻找可靠的疗效预测生物标志物，识别特定方案的优势人群，实现个性化精准诊疗。随着多线治疗的逐步完善，合理规划三线及更后期的治疗方案变得至关重要。鉴于HCC耐药的核心在于肿瘤微环境的动态适应性和肿瘤细胞的高度异质性，靶向广泛表达的免疫调控靶点或重塑抗肿瘤免疫微环境等药物或治疗方法的研发已成为后线治疗的重要突破口；未来的临床研究可在深入解析耐药机制的基础上，开发更多新型抗肿瘤药物联合的后线治疗方案，通过多种治疗策略相结合进一步提高患者的生存期和生活质量。本共识结合国内外最新临床研究结果，重点讨论中晚期肝细胞癌患者各种一线系统治疗方案耐药后的临床管理策略，提出优选方案推荐，供临床医师参考。随着未来新的循证医学证据出现，也将不断修订和完善这一共识，以期更好地服务于患者。针对于高度异质性的肝癌，Biotech前瞻团队也整理了精美幻灯。肝癌如何诊断？如何分期？各分期肝癌如何治疗？随着运营内容的日渐丰富，Biotech前瞻公众号已然制备了肿瘤基础类、研究进展类、研究设计小课堂及行业研究类幻灯。此外，ASCO和ESMO为代表的国际大会会后简版幻灯材料也会在会后一周左右时间发给会员。都为可编辑无水印资料。不定期还有会员福利惊喜赠送或享限时优惠价格哦！联系方式如下，可添加企业微信后台沟通：选择成为我们的会员，不仅是一次知识的积累，更是一场专业成长的加速之旅。在这里，您将与众多行业精英汇聚一堂，共享前沿资讯，交流实践经验，共同推动医药行业的发展与进步。 2025年，让我们携手共进，以知识为翼，以专业为刃，开启医药行业的全新篇章。立即注册成为会员，开启您的专属福利之旅，我们期待与您共同见证成长与突破！ ★

免疫疗法临床结果

2026-02-25

·医药投资并购俱乐部

Ipsen SA 益普生

Ipsen SA 益普生公司在全球拥有超过 5,300 名员工。全球领导团队中，女性占比达到 55%。在 35 个国家设有直接机构。公司崇尚去风险化与聚焦，旨在解决长期困扰公司的Somatuline（兰瑞肽）专利悬崖问题。通过内部与外部创新丰富管线，例如利用重组神经毒素领域的全球领导地位，开发长效神经毒素。在罕见病和肿瘤领域推出了多款重要疗法，巩固了其在细分领域的领导地位；在治疗复杂罕见癌症，包括上皮样肉瘤、神经内分泌肿瘤（NETs）和肾细胞癌（RCC）。专注于改善特定儿童脑肿瘤患者的治疗效果。内部开发下一代神经毒素等核心技术。通过战略伙伴关系探索前沿治疗方式。利用端到端的研发能力，高效推进管线中 “first-in-class” 和 “best-in-class” 的分子，优先选择能快速惠及患者的项目。战略与商业模式 Ipsen 已彻底完成从一家混合型药企向纯粹的生物制药公司（Global Specialty Care Biopharma）的转型。通过果断剥离非核心的消费者健康业务（Consumer HealthCare），公司将全部资源集中在高壁垒、高附加值的三大核心领域：肿瘤、罕见病和神经科学。这种“做减法”的战略极大地提高了资本效率，使其核心运营利润率维持在35%左右的行业高位。外部创新（External Innovation）已取代内部发现，成为增长的第一引擎。不同于Big Pharma依赖庞大的内部研发中心，Ipsen 将自己定位为“资产整合者”。其战略逻辑利用强劲的资产负债表，通过许可引进（In-licensing）或并购（M&A）获取处于临床中后期（Mid-to-late stage）的资产。这种模式显著降低了早期药物发现的高失败率风险，同时利用其覆盖全球（特别是中、美、欧）的商业化网络实现资产价值最大化。 Ipsen 强调“全球化规模，本地化聚焦”。特别是在美国市场，Ipsen 正在积极扩张其商业足迹，以承接新购入资产（如Iqirvo和Bylvay）的落地。Ipsen的战略愿景并非追求全覆盖，而是在细分市场（Niche Market）做到全球第一，成为特定疾病领域的“特种部队”。核心领域肿瘤学 (Oncology)（占比约70%） Cabometyx（卡博替尼片） (RCC（肾细胞癌）/HCC（肝细胞癌）) 和 Onivyde（伊立替康脂质体注射液），胰腺癌，是当前的支柱。正在从传统的实体瘤向血液瘤（血液系统肿瘤）拓展（通过Epizyme（艾匹zyme公司）收购获得Tazverik他泽司他，并布局下一代免疫疗法ImCheck（艾姆克公司）的ICT01，针对γ9δ2 T细胞（伽马9德尔塔2 T细胞））罕见病 (Rare Disease) 建立了全球领先的罕见肝病（Rare Liver，罕见肝病）特许经营权。 • Iqirvo（艾启沃） (Elafibranor，伊拉非布兰)：治疗原发性胆汁性胆管炎 (PBC，原发性胆汁性胆管炎)，Best-in-class（同类最佳）潜力。 • Bylvay（蓓尔唯） (Odevixibat，奥德昔巴特)：覆盖 PFIC（进行性家族性肝内胆汁淤积症）和 ALGS（阿拉杰里综合征）等儿科胆汁淤积症。 • 罕见骨病： Sohonos（索霍诺斯） (Palovarotene，帕洛伐罗汀) 是全球首个获批用于 FOP（进行性骨化性纤维发育不良）的药物。神经科学 (Neuroscience) 围绕 Dysport（吉适） (肉毒毒素) 构建。不仅在医美（医疗美容），更在治疗性适应症（痉挛）上拥有极强统治力。正在开发长效版本（LANT，长效肉毒毒素候选药物）以对抗竞品。其他/前沿探索：通过与 Marengo 和 Skyhawk 的合作，探索 RNA剪接（RNA剪切）和 T细胞激活平台（T细胞激活平台），意图攻克“不可成药”靶点。图2：三大治疗领域的临床管线概览（来源：Ipsen-Integrated-annual-report-2024-1.pdf，第24页）技术平台肉毒毒素工程 (Toxin Engineering) Ipsen 在英国 Wrexham 拥有一流的研发与制造中心，专注于肉毒杆菌毒素（BoNT）的重组与修饰。这是公司的核心护城河。毒素生产涉及极高的生物安全标准和复杂的发酵工艺，极难被仿制。长效神经毒素（Long-Acting Neurotoxins）。其在研平台致力于通过蛋白工程技术，开发半衰期更长的重组毒素（如IPN10200），旨在减少患者注射频率，直接对标艾伯维的Botox。 RNA 剪接调节平台 (RNA Splicing)：通过与 Skyhawk Therapeutics 的合作，Ipsen 进入了小分子靶向RNA的领域。该技术旨在纠正神经系统罕见病中的RNA错误剪接，这为Ipsen在肉毒毒素之外，打开了治疗神经退行性疾病（如亨廷顿舞蹈症等）的新窗口。 T细胞重编程平台 (T-Cell Engagers)：通过与 Marengo Therapeutics 的合作，引入 TriSTAR 平台。该技术不同于传统的CAR-T或双抗，它通过特异性激活Vβ T细胞亚群来攻击“冷肿瘤”。这标志着Ipsen的技术触角延伸至下一代免疫肿瘤学。病毒载体技术 (Viral Vectors)：针对罕见病基因治疗的递送系统探索。总结： Ipsen 的技术平台策略是实用主义的。它不追求拥有所有底层技术，而是通过掌握核心制造工艺（毒素、缓释制剂）确立防守优势，同时通过灵活的BD接入前沿技术（RNA、免疫平台）以博取未来的爆发。核心产品产品组合协同性强： Dysport、 Somatuline提供稳定现金流（合计20亿欧元/年），Iqirvo、Bylvay贡献长期增量，Cabometyx主导肿瘤领域增长。2. 中国市场成关键变量：多款产品纳入医保，渗透率快速提升，成为全球增长核心极。3. 壁垒支撑估值：独家适应症、技术工艺、临床数据构建核心竞争优势，保障长期商业化价值。 Bylvay® (odevixibat) 全球推出的首款用于进行性家族性肝内胆汁淤积症（PFIC）的治疗药物。已获批用于治疗 Alagille 综合征相关的胆汁淤积性瘙痒（美国），并在欧洲以 Kayfanda® 品牌名上市。 IQIRVO® (elafibranor) 作为首款原发性胆汁性胆管炎（PBC）一线疗法，在美欧加速获批。其全球上市确立了益普生在罕见胆汁淤积性肝病领域的领导地位。 Onivyde® (irinotecan) NALIRIFOX 在美国推出，作为转移性胰腺腺癌（mPDAC）成人患者的一线治疗。有望成为该领域新的标准治疗方案，为患者提供更优选择。 Somatuline®（Lanreotide）长效生长抑素类似物，GEP-NET治疗一线标杆，肽自组装凝胶剂型，无需冷链、可自我注射，GEP-NET领域中位PFS 32.8个月（显著优于竞品奥曲肽微球）。2024年全球销售额12亿欧元，中国市场销售额8亿元（+25% YoY），医保覆盖后渗透率加速提升。全球SSA市场份额40%，凭借剂型与疗效优势，持续追赶诺华（58%份额） Dysport®（AboBoNT-A）全球第二大A型肉毒毒素品牌，神经肌肉治疗与医美领域双栖现金牛，工艺壁垒极高。无血清发酵工艺构建技术壁垒，扩散半径适中（3cm），适配肢体痉挛与面部除皱，免疫原性低。2024年全球销售额8亿欧元，新兴市场（中国、东南亚）增速超15%；市场份额约25%，性价比优于保妥适。拓展儿童脑瘫、下尿路障碍新适应症，研发长效制剂提升依从性 Iqirvo®（Elafibranor）首个获批的PPARα/δ双重激动剂，Ipsen未来5年核心增长引擎，填补PBC二线治疗近十年空白。双重激活PPAR受体，调节胆汁酸代谢、抗炎抗纤维化，瘙痒症状改善优于传统FXR激动剂。ELATIVE研究中，63%患者ALP降低≥40%，51%患者瘙痒评分改善≥30%，不良反应停药率＜5%，耐受性优异。全球PBC患者20-30万人，2029年销售额预计超10亿美元；同步拓展NASH、PSC适应症，延长生命周期。 Bylvay®（Odevixibat）首创IBAT抑制剂，儿科罕见病领域独家性产品，拥有罕见病优先审评券（PRV）战略价值。选择性抑制回肠胆汁酸重吸收，降低肝脏毒性，不影响脂溶性维生素吸收，适配儿童患者。全球唯一PFIC治疗药物、首个ALGS瘙痒治疗药物，患者忠诚度100%，无替代疗法竞争。儿科罕见病定价弹性大，2029年全球销售额预计8亿美元；胆道闭锁适应症进入III期，进一步打开增长空间。 Cabometyx®（Cabozantinib）多靶点TKI，晚期肾癌一线联合免疫标准疗法，Ipsen肿瘤管线核心支柱。抑制MET、VEGFR等9个靶点，调节肿瘤微环境，与免疫治疗协同增效，降低进展/死亡风险。CheckMate 9ER研究中，联合纳武利尤单抗使晚期RCC中位PFS达16.6个月（vs 对照组8.3个月），ORR 55%。2024年全球销售额12亿欧元，2029年预计超15亿欧元；拓展肝癌、神经内分泌肿瘤适应症，联合用药延长生命周期。核心在研管线 IPN10200 (LANT，肉毒素)：处于 Phase III 准备阶段。目标是在医美（眉间纹）和治疗（痉挛、偏头痛）领域替代现有短效毒素。这是 Ipsen 最大的潜在重磅产品之一。 Bylvay (IBAT抑制剂，治疗胆道闭锁 - BA)：正在进行 Phase III (BOLD) 试验。胆道闭锁的患儿数量远多于 PFIC 和 ALGS，若获批，将显著提升 Bylvay 的销售峰值潜力. Fidrisertib (FOP)： Phase II (FALKON) 试验中。作为 ALK2 激酶抑制剂，旨在阻断 FOP 的病理核心——骨形态发生蛋白（BMP）信号通路的过度激活，有望比第一代药物 Sohonos 更有效. ICT01 (ImCheck)：即将进入注册性临床。针对一线不适合化疗的 AML 患者，有望填补巨大的临床空白。 Tovorafenib (pLGG)：正在进行 Phase III (FIREFLY-2) 试验，针对一线需要治疗的儿童低级别胶质瘤，旨在替代传统的化疗方案. IPN01194 / IPN01195：针对 MAPK 通路的下一代抑制剂，旨在克服现有 BRAF/MEK 抑制剂的耐药性问题，用于治疗实体瘤. 重点交易合作伙伴合作形式领域核心资产/技术交易结构与战略逻辑 ImCheck Therapeutics 2025年12月收购 (Acquisition) 肿瘤 (IO) ICT01 (γ9δ2 T细胞激活剂) 结构：€3.5亿首付 + 里程碑付款，总额可达€10亿。逻辑：获得 First-in-class 的免疫治疗资产，补充血液肿瘤管线。这是对“非传统T细胞疗法”的一次豪赌，旨在突破 PD-1/L1 的治疗瓶颈. Day One Biopharmaceuticals 2024年7月许可引进 (In-licensing) 肿瘤 (儿科) Tovorafenib (pLGG) 结构：$1.11亿首付（含股权投资） + 里程碑。逻辑：获取除美国以外的全球权益。利用 Ipsen 强大的欧洲及全球商业网络变现，Day One 则保留美国市场。这是典型的“地域互补”型合作. Marengo Therapeutics 2024年6月扩大战略合作 (Strategic Partnership) 肿瘤 (实体瘤) TriSTAR 平台 (TCEs) 结构：高达$12亿的里程碑付款。逻辑：针对“冷肿瘤”的攻坚战。通过特异性激活 Vβ T 细胞，解决传统免疫疗法无效的难题。这属于“技术平台型”合作，旨在储备未来资产. Skyhawk Therapeutics 2024年4月研发合作 (R&D Collaboration) 神经科学 RNA 剪接调节小分子结构：高达$18亿的里程碑付款。逻辑：布局未来技术。利用小分子靶向 RNA，治疗难以成药的神经系统罕见病，补充毒素以外的神经管线，实现技术路线的多元化. 重点案例 1：收购 Albireo (2023年 M&A) • 交易金额：约 9.52亿美元（含预付款和CVR）。 • 核心资产： Bylvay (Odevixibat) 及整个胆汁酸调节剂管线。 • 交易解读：这是一笔教科书式的**“补强收购 (Bolt-on)”**。￮战略契合：完美契合 Ipsen 的罕见病战略。￮协同效应： Ipsen 利用其现有的全球消化/肝病销售网络，迅速放大了 Bylvay 的商业价值。￮价值判断：直接确立了 Ipsen 在儿科罕见肝病领域的全球统治地位。重点案例 2：与 Day One Biopharmaceuticals 的独家许可 (2024年 Licensing) • 涉及资产： Tovorafenib (泛RAF抑制剂，治疗儿科脑胶质瘤 pLGG)。 • 交易结构： 1.11亿美元首付款 (含股权投资) + 约 3.5亿美元里程碑 + 销售分成。 • 交易解读：典型的**“地域互补 (Geographic Split)”** 模式。￮逻辑： Day One 保留美国权益，Ipsen 获得除美国以外的全球权益。￮双赢： Day One 这种小型 Biotech 难以在欧洲建立商业团队，而 Ipsen 在欧洲拥有强大的肿瘤销售网络。这是一笔低风险、高杠杆的交易，直接变现了 Ipsen 的渠道价值。价值判断罕见病（Bylvay/IQIRVO）与神经科学（Dysport）提供中短期增长；肿瘤领域通过 Onivyde 一线拓展、MAPK 管线与外部引入资产形成催化剂。 END 扫码关注医药投资并购俱乐部获得更多讯息

并购申请上市

2026-02-25

·investors.arcusbio.com

Arcus Biosciences Reports Fourth-Quarter and Full-Year 2025 Financial Results and Provides a Pipeline Update

Updated data for casdatifan in late-line kidney cancer demonstrated median progression-free survival (PFS) of 15.1 months and a confirmed overall response rate (cORR) of 45% for the 100mg once-daily (QD) cohort At least two additional data presentations for casdatifan are expected in 2026: updated data for casdatifan plus cabozantinib and initial data in early-line kidney cancer In addition to the ongoing Phase 3 PEAK-1 study, Arcus plans to initiate a Phase 3 study in the first-line (1L) metastatic setting evaluating casdatifan in a tyrosine kinase inhibitor (TKI)-free combination by the end of 2026 With $1.0 billion in cash, cash equivalents and marketable securities at year-end, Arcus is well positioned to advance casdatifan with cash runway until at least the second half of 2028 HAYWARD, Calif.--(BUSINESS WIRE)-- Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for patients with cancer and inflammatory and autoimmune diseases, today reported financial results for the year and quarter ended December 31, 2025, and provided a pipeline update on its clinical-stage investigational molecules and discovery programs. “This week’s updated data at ASCO GU continue to validate casdatifan’s profile as the best-in-class HIF-2a inhibitor and new potential standard-of-care therapy for ccRCC,” said Terry Rosen, Ph.D., chief executive officer of Arcus. “We are focused on rapidly enrolling PEAK-1, our Phase 3 study evaluating casdatifan plus cabozantinib in IO-experienced patients, as well as determining the optimal TKI-free casdatifan-based regimen for a registrational trial in the 1L setting by the end of the year. Together, the IO-experienced and first line settings represent an over $5 billion peak sales opportunity for casdatifan. We remain extremely well-positioned to execute on our casdatifan program and our emerging inflammation portfolio, with cash runway until at least the second half of 2028.” Casdatifan (HIF-2a inhibitor) Recent Data Update: Earlier this week, Arcus shared updated ORR and PFS data from the cohorts of the Phase 1/1b ARC-20 study evaluating casdatifan monotherapy in late-line ccRCC. These data will be presented by Dr. Toni Choueiri of the Dana-Farber Cancer Institute at ASCO GU on February 28, 2026. In the 100mg QD cohort, with 17.9 months of median follow up, cORR increased to 45.2% with an mPFS of 15.1 months as of the data cut-off (DCO) date of January 30, 2026. In the pooled analysis (n=121), with 20.8 months of median follow up, cORR increased to 34.7% and mPFS was stable at 12.2 months as of the DCO. New biomarker data demonstrated a strong correlation between the magnitude and durability of erythropoietin suppression by casdatifan and clinical benefit, including rate of primary progression, cORR and PFS. The safety profile of casdatifan remained consistent with prior analyses. In the 100mg QD cohort, with 17.9 months of median follow up, cORR increased to 45.2% with an mPFS of 15.1 months as of the data cut-off (DCO) date of January 30, 2026. In the pooled analysis (n=121), with 20.8 months of median follow up, cORR increased to 34.7% and mPFS was stable at 12.2 months as of the DCO. New biomarker data demonstrated a strong correlation between the magnitude and durability of erythropoietin suppression by casdatifan and clinical benefit, including rate of primary progression, cORR and PFS. The safety profile of casdatifan remained consistent with prior analyses. Casdatifan Development Program: IO-experienced ccRCC: PEAK-1, a Phase 3 study evaluating casdatifan + cabozantinib versus cabozantinib in immunotherapy (IO)-experienced metastatic ccRCC, started enrollment in 3Q25 and is now enrolling globally. Similar Phase 3 trials in this setting have completed enrollment in 18 months or less, and Arcus’s goal is to achieve a similar timeline. 1L ccRCC: Arcus is focused on TKI-free regimens, which are enabled by the consistently low rate of primary progression observed with casdatifan across all cohorts and settings evaluated to date. Emerging data from the following cohorts are designed to determine the optimal 1L registrational strategy with the goal of initiating a Phase 3 study by the end of 2026: Casdatifan plus zimberelimab (Arcus’s anti-PD-1 antibody) and ipilimumab: Arcus is now enrolling a cohort evaluating this combination as part of ARC-20. Casdatifan plus zimberelimab: The cohort evaluating this combination in ARC-20 has completed enrollment. Arcus plans to present data from this cohort in the second half of 2026. Arcus expects this to be the backbone of its front-line strategy. Casdatifan plus volrustomig (AstraZeneca’s anti-PD-1/CTLA-4 bispecific antibody): This combination is being evaluated in the eVOLVE-RCC02 study (which is being operationalized by AstraZeneca). Additional combinations in ccRCC and tumor types: This year, Arcus plans to evaluate the potential of casdatifan in at least one additional combination in 1L ccRCC and in another tumor type. Casdatifan plus zimberelimab (Arcus’s anti-PD-1 antibody) and ipilimumab: Arcus is now enrolling a cohort evaluating this combination as part of ARC-20. Casdatifan plus zimberelimab: The cohort evaluating this combination in ARC-20 has completed enrollment. Arcus plans to present data from this cohort in the second half of 2026. Arcus expects this to be the backbone of its front-line strategy. Casdatifan plus volrustomig (AstraZeneca’s anti-PD-1/CTLA-4 bispecific antibody): This combination is being evaluated in the eVOLVE-RCC02 study (which is being operationalized by AstraZeneca). Planned Data Readouts: Arcus expects to have at least two data readouts for casdatifan in 2026: More mature ORR data and initial PFS data for approximately 45 patients treated in the ARC-20 cohort evaluating casdatifan plus cabozantinib in the IO-experienced setting will be presented at an investor event or at a medical conference. All patients will have had at least 12 months of follow-up. Initial data from the ARC-20 cohorts evaluating casdatifan in early-line settings, including the cohort evaluating casdatifan plus zimberelimab in 1L ccRCC. Quemliclustat (small-molecule CD73 inhibitor) Enrollment was completed for PRISM-1, a Phase 3 trial of quemliclustat combined with gemcitabine/nab-paclitaxel versus gemcitabine/nab-paclitaxel in 1L metastatic pancreatic ductal adenocarcinoma, in September 2025. Results from this study are expected in the first half of 2027. Emerging I&I Portfolio Arcus expects to advance its first inflammation program, a potential best-in-class oral MRGPRX2 antagonist for atopic dermatitis and chronic spontaneous urticaria, into clinical development in 2026. The molecule was designed to have exquisite potency, enabling the ability to elicit maximal pharmacology at substantially lower exposure relative to the most advanced MRGPRX2 antagonist in clinical development, thereby avoiding potential exposure-limiting toxicities. Clinical development will begin with a first-in-human healthy volunteer study followed quickly by a proof-of-concept study, with potential for data within a year from entry into the clinic. Arcus also expects to advance an oral small-molecule TNF inhibitor, a potential treatment for rheumatoid arthritis, psoriasis and inflammatory bowel disease, into the clinic in late 2026 or early 2027. The molecules are being designed to selectively block TNFR1 signaling in order to achieve better safety and efficacy than approved anti-TNF antibodies. The molecule was designed to have exquisite potency, enabling the ability to elicit maximal pharmacology at substantially lower exposure relative to the most advanced MRGPRX2 antagonist in clinical development, thereby avoiding potential exposure-limiting toxicities. Clinical development will begin with a first-in-human healthy volunteer study followed quickly by a proof-of-concept study, with potential for data within a year from entry into the clinic. The molecules are being designed to selectively block TNFR1 signaling in order to achieve better safety and efficacy than approved anti-TNF antibodies. Domvanalimab (Fc-silent anti-TIGIT antibody) plus Zimberelimab (anti-PD-1 antibody) Status Update: Arcus and Gilead are in the process of rapidly winding down activities related to the Phase 3 STAR-221 and Phase 2 EDGE-Gastric studies. A futility analysis of STAR-121, a Phase 3 study evaluating domvanalimab plus zimberelimab and chemotherapy in 1L non-small cell lung cancer, will be conducted in the next couple of months to determine next steps for the program. Financial Results for Fourth Quarter and Full Year 2025: Cash, Cash Equivalents and Marketable Securities were $1.0 billion as of December 31, 2025, compared to $992 million as of December 31, 2024. The increase is primarily due to $429 million in net proceeds from issuances of our common stock, the additional $50 million drawdown under our term loan facility, and $37 million from Taiho for development milestones and option payments, partially offset by cash used in research and development activities. Arcus expects its cash and investments will be sufficient to provide funding until at least the second half of 2028. Revenues were $33 million for the fourth quarter 2025, compared to $36 million for the same period in 2024. Fourth quarter 2025 revenues included (i) $23 million related to programs under the Gilead collaboration, (ii) $7 million related to Taiho's exercise of its option to casdatifan, and (iii) $3 million related to Gilead’s ongoing rights to access Arcus’s research and development pipeline. GAAP revenue for the full year 2026 is expected to be between $45 to $55 million. Research and Development (R&D) Expenses were $121 million for the fourth quarter 2025, compared to $111 million for the same period in 2024. The net increase of $10 million was primarily attributable to higher late-stage development costs, driven by increased enrollment and clinical activities in our Phase 3 studies for casdatifan and quemliclustat, partially offset by lower early-stage development costs, as earlier study activities for domvanalimab and etrumadenant approached completion. For the fourth quarter 2025 and 2024, Arcus recognized gross reimbursements of $28 million and $41 million, respectively, for shared collaboration expenses, primarily from Gilead. Gross reimbursements were $127 million for the full year 2025, compared to $165 million for 2024. Our partnership reimbursements decreased compared to the prior year, primarily due to Gilead-led activities representing a larger share of total joint development costs and an increase in programs fully funded by us. R&D expenses are expected to decrease meaningfully for the full year 2026 relative to 2025; the magnitude of the decrease will be determined in part by the results of the futility analysis of STAR-121. Non-cash stock-based compensation expense was $8 million for each of the fourth quarter 2025 and 2024. General and Administrative (G&A) Expenses were $26 million for the fourth quarter 2025, compared to $28 million for the same period in 2024. Non-cash stock-based compensation expense was $7 million for the fourth quarter 2025, compared to $9 million for the same period in 2024. Net Loss was $106 million for the fourth quarter 2025, compared to $94 million for the same period in 2024. Conference Call Information: Arcus will host a conference call and webcast today, February 25, 2026, at 1:30 PM PT/4:30 PM ET to discuss its fourth-quarter and full-year 2025 financial results and pipeline updates. To access the call, please dial +1 (646) 844-6383 (local) or +1 (833) 470-1428 (toll-free), using Access Code: 190500. Participants may also register for the call online using the following link: https://events.q4inc.com/attendee/640976415. To access the live webcast and accompanying slide presentation, please visit the “Investors & Media” section of the Arcus Biosciences website at www.arcusbio.com. A replay of the webcast will be available following the live event. About Arcus Biosciences Arcus Biosciences is a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules for the treatment of cancer and inflammatory and autoimmune diseases. In partnership with industry collaborators, patients and physicians around the world, Arcus is expediting the development of its late-stage portfolio of first- and/or best-in-class medicines against well-characterized biological targets and pathways and studying novel, biology-driven combinations that have the potential to help people with cancer live longer. Founded in 2015, the company has advanced multiple investigational medicines into registrational clinical trials including casdatifan, a HIF-2a inhibitor for clear cell renal cell carcinoma, and quemliclustat, a small-molecule CD73 inhibitor for pancreatic cancer. For more information about Arcus Biosciences’ clinical and preclinical programs, please visit www.arcusbio.com. Forward-Looking Statements This press release contains forward-looking statements. All statements regarding events or results to occur in the future contained herein are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, the timing of milestones, including future data presentations, initiation of new cohorts and clinical studies and completion of enrollment for ongoing studies; potential for casdatifan to become a standard-of-care therapy; and advantages of Arcus’s inflammation and immunology programs. All forward-looking statements involve known and unknown risks and uncertainties and other important factors that may cause Arcus’s actual results, performance or achievements to differ materially from those expressed or implied by the forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, risks associated with: interim data not being guarantees of future data or replicated in other studies evaluating casdatifan, including the Phase 3 PEAK-1 study; the unexpected emergence of adverse events or other undesirable side effects with casdatifan; risks associated with manufacturing or supplying product for clinical trials evaluating casdatifan; adverse data from toxicology studies that affect Arcus’s ability to advance development candidates from its immunology and inflammation programs, uncertainties in timelines associated with the conduct of clinical studies and with respect to the regulatory approval process; changes in the competitive landscape for Arcus’s programs; and the inherent uncertainty associated with pharmaceutical product development and clinical trials. Risks and uncertainties facing Arcus are described more fully in the “Risk Factors” section of Arcus’s most recent periodic report filed with the U.S. Securities and Exchange Commission (SEC) and in other filings that Arcus makes with the SEC from time to time, which are available at www.sec.gov. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. Arcus disclaims any obligation or undertaking to update, supplement or revise any forward-looking statements contained in this press release, except to the extent required by law. The Arcus name and logo are trademarks of Arcus Biosciences, Inc. All other trademarks belong to their respective owners. ARCUS BIOSCIENCES, INC. Consolidated Statements of Operations (unaudited) (In millions, except per share amounts) Three Months Ended December 31, Years Ended December 31, 2025 2024 2025 2024 Revenues: License and development service $ 29 $ 28 $ 221 $ 222 Other collaboration 4 8 26 36 Total revenues 33 36 247 258 Operating expenses: Research and development 121 111 523 448 General and administrative 26 28 110 120 Impairment of long-lived assets — — — 20 Total operating expenses 147 139 633 588 Loss from operations (114 ) (103 ) (386 ) (330 ) Non-operating income (expense): Interest and other income, net 10 12 41 52 Interest expense (2 ) (2 ) (8 ) (4 ) Total non-operating income, net 8 10 33 48 Loss before income taxes (106 ) (93 ) (353 ) (282 ) Income tax expense — (1 ) — (1 ) Net loss $ (106 ) $ (94 ) $ (353 ) $ (283 ) Net loss per share: Basic and diluted $ (0.89 ) $ (1.03 ) $ (3.29 ) $ (3.14 ) Shares used to compute net loss per share: Basic and diluted 118.5 91.7 107.4 90.1 Selected Consolidated Balance Sheet Data (unaudited) (In millions) December 31, 2025 December 31, 2024 Cash, cash equivalents and marketable securities $ 1,010 $ 992 Total assets 1,139 1,150 Total liabilities 508 665 Total stockholders’ equity 631 485 Derived from the audited financial statements included in the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 25, 2026. Investor Inquiries: Pia Eaves VP of Investor Relations & Strategy (617) 459-2006 peaves@arcusbio.com Media Inquiries: Holli Kolkey VP of Corporate Affairs (650) 922-1269 hkolkey@arcusbio.com Maryam Bassiri AD, Corporate Communications (510) 406-8520 mbassiri@arcusbio.com

临床3期临床结果财报临床2期

100 项与苹果酸卡博替尼相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10095	苹果酸卡博替尼	L01XE26	DB08875

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
神经内分泌肿瘤	英国	Ipsen SA	2025-09-26
高分化胰腺内分泌肿瘤	欧盟	Ipsen Pharma SAS	2025-07-24
高分化胰腺内分泌肿瘤	冰岛	Ipsen Pharma SAS	2025-07-24
高分化胰腺内分泌肿瘤	列支敦士登	Ipsen Pharma SAS	2025-07-24
高分化胰腺内分泌肿瘤	挪威	Ipsen Pharma SAS	2025-07-24
胰腺外神经内分泌肿瘤	澳大利亚	Ipsen Pty Ltd.	2018-01-19
胰腺神经内分泌肿瘤	澳大利亚	Ipsen Pty Ltd.	2018-01-19
分化型甲状腺癌	韩国	Ipsen Ltd.	2017-09-26
肾细胞癌	韩国	Ipsen Ltd.	2017-09-26
肝细胞癌	欧盟	Ipsen Pharma SAS	2016-09-09
肝细胞癌	冰岛	Ipsen Pharma SAS	2016-09-09
肝细胞癌	列支敦士登	Ipsen Pharma SAS	2016-09-09
肝细胞癌	挪威	Ipsen Pharma SAS	2016-09-09
晚期肾细胞癌	美国	Exelixis, Inc.	2016-04-25
甲状腺髓样癌	美国	Exelixis, Inc.	2012-11-29

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
实体瘤	申请上市	中国	江苏豪森药业集团有限公司	2020-09-28
难治性分化型甲状腺癌	临床3期	美国	ECOG-ACRIN Medical Research Foundation, Inc.	2024-08-22
难治性甲状腺癌	临床3期	美国	ECOG-ACRIN Medical Research Foundation, Inc.	2024-08-22
转移性骨肉瘤	临床3期	美国	National Cancer Institute	2023-03-03
转移性骨肉瘤	临床3期	澳大利亚	National Cancer Institute	2023-03-03
转移性骨肉瘤	临床3期	加拿大	National Cancer Institute	2023-03-03
转移性骨肉瘤	临床3期	新西兰	National Cancer Institute	2023-03-03
晚期尿路上皮癌	临床3期	美国	National Cancer Institute	2022-06-03
晚期尿路上皮癌	临床3期	加拿大	National Cancer Institute	2022-06-03
膀胱癌	临床3期	美国	National Cancer Institute	2022-06-03

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临床结果

适应症

分期

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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


NCT01068782 (CTgov)	临床2期	胶质母细胞瘤	19	cabozantinib (Arm 1)	餘鬱獵廠醖範願淵範繭 = 淵製糧醖糧繭廠鏇齋鹽鏇選窪網壓鑰選膚簾窪 (廠襯淵襯遞糧廠壓顧鬱, 積簾窪襯願鑰顧壓餘艱 ~ 積窪艱獵衊醖簾鹽願廠) 更多	-	2026-02-25
				cabozantinib (Arm 2)	餘鬱獵廠醖範願淵範繭 = 築夢蓋糧鑰鑰觸製鏇鏇鏇選窪網壓鑰選膚簾窪 (廠襯淵襯遞糧廠壓顧鬱, 鹹鏇顧範築顧網醖膚窪 ~ 醖鏇觸範廠鏇蓋憲獵淵) 更多
NCT04200443 (Pubmed \| Lancet Oncol)	临床2期	平滑肌肉瘤 \| 软组织肉瘤	72	Cabozantinib 40 mgTemozolomide 40 mg + Cabozantinib 40 mgTemozolomide	顧觸築築窪鑰鏇膚積鹹(築繭簾築築鹽獵醖顧簾) = 鑰鏇製網鹹廠鹽憲艱鬱獵窪憲窪窪壓遞構憲餘 (艱壓蓋夢齋鏇鑰鹹憲艱 ) 更多	积极	2026-02-01
				Temozolomide (Cohort 1)	壓鹽夢範壓築廠鹽範願(廠廠遞範餘鬱廠餘鹽選) = 製鏇構齋選選衊範積繭獵網鏇蓋鹹蓋壓襯夢選 (憲製築簾繭餘艱膚願憲 ) 更多
NCT00596648 (CTgov)	临床1/2期	非小细胞肺癌	92	erlotinib+cabozantinib (Phase 1 - Cohort 1)	積淵範壓願願齋獵醖蓋(齋艱鏇艱齋糧夢蓋艱繭) = 鬱鬱壓糧構鏇襯選夢範壓壓醖衊夢憲網鬱淵築 (齋襯壓簾獵醖窪糧製窪, 顧窪淵窪構鹹衊網艱衊 ~ 壓鏇壓構窪鬱選淵壓鑰) 更多	-	2026-01-13
				erlotinib+cabozantinib (Phase 1 - Cohort 2A)	積淵範壓願願齋獵醖蓋(齋艱鏇艱齋糧夢蓋艱繭) = 積簾範窪築壓構糧鑰艱壓壓醖衊夢憲網鬱淵築 (齋襯壓簾獵醖窪糧製窪, 觸艱顧襯積蓋膚艱簾鑰 ~ 憲糧鑰積鹽獵窪觸觸膚) 更多
NCT03375320 (CABINET, Pubmed \| Endocr Relat Cancer)	临床3期	神经内分泌肿瘤	96	Cabozantinib-Malate	範築襯蓋製糧餘糧鏇襯(範醖鬱膚鹹鹹構糧餘鹽) = 淵鏇蓋齋觸鬱選願獵願構鏇築齋鹹膚願襯醖網 (窪淵網繭積製壓鏇鑰膚 )	积极	2026-01-12
				Placebo	範築襯蓋製糧餘糧鏇襯(範醖鬱膚鹹鹹構糧餘鹽) = 淵構襯窪網願觸鑰製積構鏇築齋鹹膚願襯醖網 (窪淵網繭積製壓鏇鑰膚 )
NCT01995058 (CTgov)	临床2期	转移性去势抵抗性前列腺癌	54	prednisone+abiraterone+Cabozantinib (Arm 1)	觸築衊鹽廠獵壓餘鬱顧(繭醖鏇願窪艱鬱餘顧艱) = 構淵鏇構艱餘構醖膚遞簾夢憲願鹽願鹽繭顧窪 (遞鏇積艱壓選淵繭膚網, 鹽鬱醖遞憲艱製壓襯築 ~ 糧遞簾鏇膚選窪壓膚遞) 更多	-	2026-01-08
				prednisone+abiraterone+Cabozantinib (Arm 2)	觸築衊鹽廠獵壓餘鬱顧(繭醖鏇願窪艱鬱餘顧艱) = 鏇艱糧蓋襯窪鏇鬱願艱簾夢憲願鹽願鹽繭顧窪 (遞鏇積艱壓選淵繭膚網, 餘糧願窪願築膚鹽構網 ~ 齋艱蓋餘艱築淵鹹範襯) 更多
NCT03141177 (CheckMate 9ER, Pubmed \| J Immunother Cancer)	临床3期	晚期肾细胞癌一线	651	Nivolumab+Cabozantinib	顧憲蓋範衊艱糧淵網醖(築鏇繭築夢鑰襯簾網簾) = 窪鑰廠簾蓋廠網網製衊選齋醖願選築鑰觸築網 (鹹鹽蓋繭憲鬱製鹽選築 ) 更多	积极	2026-01-01
				Sunitinib	顧憲蓋範衊艱糧淵網醖(築鏇繭築夢鑰襯簾網簾) = 選衊積膚遞齋積夢襯艱選齋醖願選築鑰觸築網 (鹹鹽蓋繭憲鬱製鹽選築 ) 更多
NCT04446117 (CONTACT-02 \| CONTACT-2, CTgov)	临床3期	转移性去势抵抗性前列腺癌 \| 前列腺腺癌	575	Cabozantinib+Atezolizumab (Experimental Arm: Cabozantinib and Atezolizumab)	繭淵範壓積簾壓鬱膚醖(憲繭窪簾願選蓋襯衊積) = 壓遞選壓醖繭蓋獵膚製獵積憲襯壓醖製網觸遞 (遞壓遞餘膚顧憲積衊選, 襯鏇積鏇繭蓋糧憲獵築 ~ 餘鏇鹽製遞艱構獵齋鹹) 更多	-	2025-12-18
				enzalutamide+prednisone+abiraterone (Control Arm: Second NHT)	繭淵範壓積簾壓鬱膚醖(憲繭窪簾願選蓋襯衊積) = 醖膚糧醖廠構鏇襯鹽夢獵積憲襯壓醖製網觸遞 (遞壓遞餘膚顧憲積衊選, 獵夢窪廠簾繭簾鬱窪蓋 ~ 艱淵鏇獵襯觸願淵遞獵) 更多
NCT04079712 (CTgov)	临床2期	大细胞神经内分泌癌 \| 小细胞癌 \| 胰岛细胞癌 ... 更多	17	Ipilimumab+Nivolumab+Cabozantinib S-malate	襯觸艱選餘窪網壓夢積(顧壓積餘窪醖襯願壓膚) = 醖壓齋築窪蓋鑰簾網範窪鹹構憲憲顧範願壓蓋 (選鹹鏇鹹製選餘繭構簾, 範願襯顧衊憲糧選鏇廠 ~ 窪膚積艱糧觸範夢遞製) 更多	-	2025-12-12
NCT03755791 (COSMIC-312, CTgov)	临床3期	晚期肝细胞癌	837	Cabozantinib+Atezolizumab (Experimental Arm: Cabozantinib + Atezolizumab)	鹽選窪醖鏇壓鹹膚範鬱(膚壓構餘蓋網範餘鬱夢) = 顧窪築餘艱獵觸壓齋蓋鹹齋衊鏇願餘壓鬱繭壓 (壓醖鑰齋壓艱觸醖簾蓋, 膚憲簾積窪醖獵鹹獵窪 ~ 壓窪鑰範鑰構襯淵壓積) 更多	-	2025-12-11
				Sorafenib (Control Arm: Sorafenib)	鹽選窪醖鏇壓鹹膚範鬱(膚壓構餘蓋網範餘鬱夢) = 廠艱鑰膚鬱願鏇窪繭網鹹齋衊鏇願餘壓鬱繭壓 (壓醖鑰齋壓艱觸醖簾蓋, 衊構憲範積遞顧淵鏇築 ~ 鑰廠糧襯顧遞鏇齋艱網) 更多
NCT05052723 (CTgov)	临床2期	转移性胰腺癌	21	Cabozantinib+Pembrolizumab	夢積簾顧範艱夢蓋願襯 = 餘選築壓鏇顧積繭夢範顧鏇糧餘糧鏇築鹹範齋 (壓廠願醖獵構鹽蓋窪糧, 鏇廠積憲餘積醖簾願憲 ~ 蓋窪築鹽選構繭顧膚憲) 更多	-	2025-11-19