阿培利司(Alpelisib) - 药物靶点：PI3Kα_在研适应症：PIK3CA相关的过度生长现象，乳腺癌，晚期乳腺癌_专利_临床

概要

基本信息

药物类型

小分子化药

别名

Alpelisib (JAN/USAN/INN)、阿吡利塞、BYL-719

+ [4]

靶点

PI3Kα

作用方式

抑制剂

作用机制

PI3Kα抑制剂(磷脂酰肌醇3-激酶α抑制剂)

治疗领域

肿瘤皮肤和肌肉骨骼疾病其他疾病+ [6]

在研适应症

PIK3CA相关的过度生长现象乳腺癌晚期乳腺癌+ [23]

非在研适应症

肺腺癌晚期胃癌晚期非小细胞肺癌+ [27]

原研机构

Novartis Pharma AG

在研机构

Novartis Europharm Ltd.Novartis Pharma AG诺华（中国）生物医学研究有限公司

+ [6]

非在研机构

AstraZeneca PLC

The University of California, San Francisco

Brown University

权益机构

Kura Oncology, Inc.

Novartis AG

Olema Pharmaceuticals, Inc.

最高研发阶段批准上市

首次获批日期

美国 (2019-05-24),

PIK3CA突变/HR阳性/HER2阴性乳腺癌

最高研发阶段(中国)临床3期

特殊审评孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (澳大利亚)

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结构/序列

分子式C19H22F3N5O2S

InChIKeySTUWGJZDJHPWGZ-LBPRGKRZSA-N

CAS号1217486-61-7

关联

112

项与阿培利司相关的临床试验

NCT07573696

/ Not yet recruiting临床2期IIT

A Multicenter, Phase 2 Non-Randomized Study of Unresectable Stage III ALK+ NSCLC Treated With Neoadjuvant Alectinib Plus Chemotherapy Followed by Multidisciplinary Approach for Optimal Local Treatment

This is a multicenter, phase 2 non-randomized study to investigate the clinical feasibility and therapeutic efficacy of employing a MDT-based strategy in unresectable stage III ALK positive NSCLC following neoadjuvant alectinib in combination with platinum-based chemotherapy. Participants in this study must not have received any previous systemic anticancer therapy before enrollment.
The study will consist of a 42-day screening period, a neoadjuvant treatment period, a local radical treatment period, a post-local treatment period, a safety follow-up visit occurring 28 days after the final dose of alectinib, and a survival follow-up period.
In the neoadjuvant treatment period, participants will be provided with alectinib (600mg PO BID for 3 cycles) plus platinum-based chemotherapy for a maximum of 3 cycles (each cycle is 21 days).
Following the completion of neoadjuvant therapy, all participants who are reassessed by MDT to be resectable after neoadjuvant treatment and have adequate lung functions would be provided with definite surgery. Otherwise, patients would be provided with radical radiotherapy through MDT discussion.
For the surgery cohort, participants meet both the R0 resection, the pathological assessment criteria of pCR and have two consecutive landmark ctDNA tests that are negative will receive surveillance after surgery. Participants who do not meet all the above conditions will receive alectinib after surgery, adjuvant treatment should be initiated ideally 4-12 weeks after surgery, or according to local standard of care, treatment will continue until completion of treatment period (24 months), disease recurrence, unacceptable toxicity, death, or withdrawal from the study, whichever occurs first.
For the radical radiotherapy cohort, participants will receive alectinib after radiotherapy, adjuvant treatment should be initiated ideally 4-12 weeks after surgery, or according to local standard of care, the treatment will continue until completion of treatment period (24 months), disease progression, unacceptable toxicity, death, or withdrawal from the study, whichever occurs first.

开始日期2026-06-15

申办/合作机构

广东省人民医院

NCT06997588

/ Recruiting临床2期

Study Assessing the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)

This study is designed to demonstrate the efficacy and assess safety and tolerability of oral daily alpelisib in participants with PIK3CA-related overgrowth spectrum (PROS).

开始日期2025-10-09

申办/合作机构

Novartis Pharmaceuticals Canada, Inc.

NCT07543822

/ Active, not recruiting临床2期

VATCH (Vascular Anomaly Analysis for Therapy Choice): Phase II Study of Alpelisib Treatment in Subjects With TIE2/PIK3CA Pathway Driven Vascular Anomalies

The study will enroll participants 2 months of age up to 30 years of age. The purpose of this study is to assess the effectiveness and safety of Alpelisib (the "Study Drug") in patients with PIK3CA/TIE-2/TEK pathway driven vascular anomalies (VA). Alpelisib has been approved by the U.S. Food and Drug Administration (FDA) for treating adults and children with certain types of breast cancer. Its use in this study is considered experimental because FDA has not approved the study drug for treating people with VAs.
Study participation will last for up to 3 years and will involve regular study visits to Children's Hospital of Philadelphia (CHOP)'s Philadelphia Campus. Participants will need to take the study drug Alpelisib for at least 2 years, or up to 3 years in total if there is a positive response. Participating in this research means you will attend up to 16 clinic visits for the purposes of the study. Most visits will take approximately 30 minutes, but some visits will take approximately 2 hours, because you will be asked to complete questionnaires about your experience. Participating in this research also means taking the study drug, having pictures taken, and completing study drug diaries. There is also an optional portion to this study that involves collecting blood for biomarker testing.

开始日期2025-08-27

申办/合作机构

The Children's Hospital of Philadelphia

[+21]

100 项与阿培利司相关的临床结果

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100 项与阿培利司相关的转化医学

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100 项与阿培利司相关的专利（医药）

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916

项与阿培利司相关的文献（医药）

2026-06-01·BREAST

Integrating PIK3CA Testing into Clinical Practice for Advanced HR+/HER2- Breast Cancer: An Expert Consensus

Review

作者: Curigliano, Giuseppe ; Guarneri, Valentina ; Castellano, Isabella ; de Biase, Dario ; Gennari, Alessandra ; Gerratana, Lorenzo ; Zamagni, Claudio ; De Angelis, Carmine ; Del Mastro, Lucia ; Fusco, Nicola ; Arpino, Grazia ; Puglisi, Fabio ; Malapelle, Umberto ; Bianchini, Giampaolo ; Zambelli, Alberto ; Fabi, Alessandra

BACKGROUND:

Alterations in the PI3K/AKT/mTOR signaling pathway represent a mechanism of resistance to endocrine therapy in advanced HR+/HER2-breast cancer. The identification of activating mutations in PIK3CA has gained therapeutic relevance, guiding the use of PIK3CA-targeted inhibitors such as alpelisib and inavolisib.

METHODS:

A multidisciplinary panel of Italian experts conducted a structured consensus process to develop shared recommendations for molecular testing of PIK3CA in advanced HR+/HER2-breast cancer, addressing preanalytical, analytical, and clinical-therapeutic areas of concern.

RESULTS:

A total of 23 out of 29 statements reached complete agreement (100%). The resulting recommendations encompass sample selection, analytical methodologies, sensitivity thresholds, result reporting, and clinical interpretation and integration. Additional guidance is provided for the management of non-canonical gene variants and for other genes involved in the PI3K/AKT/mTOR pathway.

CONCLUSIONS:

This consensus document provides operational and interpretative guidelines aimed at standardizing PIK3CAtesting and assessing related genes in clinical practice. These recommendations promote a harmonized approach to patient care, in line with the latest scientific evidence.

2026-05-01·BREAST CANCER RESEARCH AND TREATMENT

Alpelisib and Fulvestrant in PIK3CA-mutated hormone receptor-positive HER2-negative advanced breast cancer included in the German PRAEGNANT trial

Article

作者: Hörner, Manuel ; Köpke, Melitta B ; Uhrig, Sabrina ; Tegeler, Christian M ; Deutsch, Thomas M ; Eichstädt, Kathleen ; Lüftner, Diana ; Häberle, Lothar ; Tesch, Hans ; Banys-Paluchowski, Maggie ; Müller, Annika ; Roth, Jonas ; Schäffler, Henning ; Rody, Achim ; Hack, Carolin C ; Lux, Michael P ; Michel, Laura L ; Seitz, Stephan ; Brucker, Sara Y ; Sych, Lea ; Tauber, Nikolas ; Princk, Henriette ; Goossens, Chloë ; Hadji, Peyman ; Wichmann, Catharina ; Reinhardt, Kristin ; Müller, Volkmar ; Ditsch, Nina ; Tretschock, Lara M ; Ziegler, Philipp ; Wallwiener, Diethelm ; Ettl, Johannes ; Dannehl, Dominik ; Krückel, Annika ; Link, Theresa ; Fasching, Peter A ; Englisch, Alexander ; Cieslik, Jan-Philipp ; Dimpfl, Moritz ; Hein, Alexander ; Hartkopf, Andreas ; Fehm, Tanja ; Amann, Niklas ; Marmé, Frederik ; Taran, Florin-Andrei ; Belleville, Erik ; John, Nelson ; Janni, Wolfgang W ; Schneeweiss, Andreas ; Ehlert, Max ; Kolberg, Hans-Christian ; Wallwiener, Markus

Abstract:

Purpose:

Mutations in PIK3CA are one of several actionable mutations for patients with hormone receptor positive, human epidermal growth factor receptor 2 negative breast cancer. Alpelisib in combination with fulvestrant was the first approved PI3K inhibitor and was introduced in clinical practice in 2019. A lack of evidence for the use of alpelisib in the context of current treatment options like cyclin-dependent 4/6 inhibitor (CDK4/6i), highlights the importance of this analysis. We provide a real-world analysis of the use of alpelisib with the prospective German PRAEGNANT registry (NCT02338167).

Methods:

57 patients with advanced breast cancer receiving alpelisib and fulvestrant were identified. 55 Patients had received prior CDK4/6i therapy. Progression-free survival (PFS) and overall survival (OS) were calculated for all patients, and stratified according CDK4/6i pre-treatment, using the Kaplan–Meier method. Subgroups (age, line of therapy, concomitant disease among others), somatic PIK3CA mutations, reasons for discontinuation and adverse events (AEs) were analyzed.

Results:

The median PFS was 5.0 (95% confidence interval [CI], 3.1–9.4) months, and the median OS was 20.1 (95% CI, 14.6–30.8) months. Line of therapy and concomitant diseases appeared to affect PFS, while the line of therapy and preexisting diabetes influenced OS. However, subgroups were too small for statistical testing. Discontinuation was mainly due to tumor progression (56.1%). Hyperglycemia, rash and diarrhea were the most documented AEs.

Conclusion:

This prospective real-world analysis shows slightly shorter median PFS and OS times compared with the pivotal trials. Patients in our analyses received alpelisib in later therapy lines, which may explain the poorer outcome.

2026-05-01·JOURNAL OF MOLECULAR GRAPHICS & MODELLING

Discovery of a novel PI3Kα inhibitor for breast cancer therapy via virtual screening method, molecular dynamics simulation and biological evaluation

Article

作者: Nutho, Bodee ; Rajchakom, Chananya ; Kanjanasirirat, Phongthon ; Boonma, Thitiya ; Nunthaboot, Nadtanet

Phosphatidylinositol-4,5-bisphosphate 3-kinase alpha (PI3Kα) is a central signaling enzyme driving cell proliferation and growth in cancers including breast cancer. Selective inhibition of PI3Kα isoform has become a promising therapeutic approach. In this work, 2000 in-house natural compounds were virtually screened against the ATP-binding site of PI3Kα. Of these, 618 compounds were predicted to have acceptable drug-likeness, pharmacokinetic, and toxicity properties based on in silico ADMET screening. Docking analysis highlighted four candidates forming stable hydrogen bonds with key residues V851, S854, and Q859 in the PI3Kα binding pocket. Molecular dynamics simulations were then used to assess their structural features and dynamic stability. Hit 2 was found to form strong hydrogen bonds with E849 and V851 of the PI3Kα protein. MM/GBSA-based binding free energy analysis supported that Hit 2 possessed the most favorable binding affinity to PI3Kα among the identified candidates. In vitro cytotoxicity assays were then performed in MCF-7 and MDA-MB-231 breast cancer cell lines, with alpelisib as a reference compound. Hit 2 reduced cell viability in both cell lines, but its effect was particularly pronounced in MDA-MB-231 cells, a model of triple-negative breast cancer (TNBC). These results suggest that Hit 2 represents a promising natural scaffold for further design and development in breast cancer therapy, with particular relevance for aggressive TNBC.

519

项与阿培利司相关的新闻（医药）

2026-05-10

·今日头条

暴涨近40%！乳腺癌新药3期临床大获成功，惠及更多晚期患者

想象一下，对于晚期乳腺癌患者来说，每多一个月的无进展生存期，就意味着多一份与家人相处的时光，多一次看到希望的机会。近日，Celcuity公司宣布的一项重磅临床试验结果，让无数PIK3CA野生型晚期乳腺癌患者的这份期待变成了现实——他们自主研发的泛PI3K/mTOR抑制剂gedatolisib在Viktoria-1 3期临床试验中，不仅达到了具有临床意义的无进展生存期（PFS）改善终点，更打破了PI3K/mTOR通路药物在野生型患者中的十年研发瓶颈。在激素受体阳性、HER2阴性的晚期乳腺癌治疗中，PIK3CA基因突变是常见的驱动因素，约35%-45%的患者携带这种突变。长期以来，PI3K抑制剂主要针对这类突变型患者，但占比更高的野生型患者却一直缺乏有效的靶向治疗选择。Viktoria-1试验的突破性就在于，它首次聚焦于这一被忽视的群体，采用了极具说服力的头对头对照设计，直接挑战当前的临床标准方案。试验中，一组患者接受了gedatolisib联合阿斯利康氟维司群（Faslodex）与辉瑞哌柏西利（Ibrance）的三联疗法，另一组则使用诺华的Piqray（阿培利司）联合氟维司群这一标准方案。结果令人振奋：三联疗法组患者的中位PFS达到9.3个月，而对照组仅为2.0个月，疾病进展或死亡风险显著降低76%（HR=0.24，P<0.001）。更值得一提的是，即使是gedatolisib联合氟维司群的双联方案，也展现出优异疗效，中位PFS达7.4个月，较对照组降低67%的疾病进展风险，这进一步验证了gedatolisib在该患者人群中的核心治疗价值。消息一出，资本市场迅速给出了积极回应。Celcuity公司股价盘前大幅拉升近40%，每股报价一度达到145美元，市值飙升至70亿美元，这一涨幅充分体现了市场对这款药物突破性价值的高度认可。 gedatolisib的成功绝非偶然，它的背后是一段从被搁置到重获新生的传奇故事。这款药物最初由辉瑞公司开发，名为PF-05212384，后因耐受性问题被暂时搁置。2021年，Celcuity以1000万美元预付款的价格将其授权引入，并通过创新性的联合用药方案优化，最终让这款"沉睡"的药物焕发新生。作为一款多靶点PI3K/AKT/mTOR（PAM）通路抑制剂，gedatolisib的独特之处在于它能够同时强效靶向所有四种I类PI3K亚型（α、β、γ、δ）以及mTORC1和mTORC2两个复合物，实现对PAM通路的全面阻断。这与传统的PI3K抑制剂有本质区别——以往药物多只靶向单一PI3K亚型（如PI3Kα），容易导致通路适应性交叉激活，最终引发耐药性。而gedatolisib的"全面封锁"策略，正是为了从根源上解决这一临床难题，帮助患者克服内分泌治疗耐药性。在乳腺癌治疗中，内分泌耐药是医生和患者面临的最大挑战之一。PI3K/AKT/mTOR通路的过度激活是导致内分泌治疗失效的核心机制，它能通过磷酸化雌激素受体，使癌细胞在缺乏雌激素的环境下依然存活并增殖。gedatolisib的多靶点设计，恰好精准打击了这一耐药根源，为那些对传统治疗无响应的患者提供了新的治疗选择。根据Celcuity的规划，公司将基于本次阳性结果，向美国FDA提交补充新药申请。值得一提的是，FDA已经授予gedatolisib优先审评资格，目前正在审评其用于PIK3CA野生型乳腺癌的上市申请，预计审批决定将于7月17日作出。若进展顺利，该药有望在获批后快速将适用人群拓展至PIK3CA突变型患者，让更多乳腺癌患者受益。同时，公司还计划于今年第四季度向欧洲提交上市申请，并同步推进全球商业化合作，加速这款创新药物的全球可及性。从辉瑞的实验室到Celcuity的临床试验，从被搁置的候选药物到可能改写乳腺癌治疗指南的突破性疗法，gedatolisib的逆袭之路不仅彰显了药物研发创新的价值，更给无数晚期乳腺癌患者带来了新的希望。Viktoria-1试验的成功，标志着PI3K/mTOR通路药物研发进入了一个新的时代——不再局限于突变型患者，而是能够惠及更广泛的野生型患者群体。我们期待着7月的审批结果，更期待着这款药物能够早日走进临床，为全球乳腺癌患者带来更长、更有质量的生存时间。免责声明本文旨在科普医药健康领域的最新研究进展，不构成任何医疗建议或诊疗方案。Gedatolisib目前仍处于临床试验阶段，其安全性和有效性尚未完全确认，最终获批适应症和使用方法需以美国FDA及其他国家药品监管机构的正式批准文件为准。晚期乳腺癌患者的治疗方案应在专业医生指导下，结合患者具体病情、身体状况和基因检测结果综合制定，切勿自行用药或更改治疗方案。本文内容仅供读者参考，不承担因使用本文信息而产生的任何医疗或法律责任。参考文献 1. 生物通. Gedatolisib联合疗法显著改善HR+/HER2-、PIK3CA野生型晚期乳腺癌患者的无进展生存期:III期VIKTORIA-1研究结果[EB/OL]. 2026-03-11. 2. 医脉通. 打破靶点限制！VIKTORIA-1研究揭示泛Ⅰ类PI3K/mTOR双重抑制剂Gedatolisib在PIK3CA野生型晚期乳腺癌治疗中的突破性价值[EB/OL]. 2026-03-26. 3. 世易医健. FDA授予Gedatolisib优先审评资格，用于HR阳性、HER2阴性晚期乳腺癌[EB/OL]. 2026-03-24. 4. 药智新闻. 乳腺癌新药，3期成功[EB/OL]. 2026-05-06. 5. 中国抗癌协会乳腺癌专业委员会. PI3K/AKT/mTOR信号通路抑制剂治疗乳腺癌临床应用专家共识（2025版）[J]. 中国癌症杂志, 2025, 35(10): 945-962.

2026-05-09

·制药网

乳腺癌治疗迎来爆发期，5月初中外创新药密集突破

【制药网行业动态】2026年5月初，国内外乳腺癌治疗领域已迎来了多项里程碑式的进展。不仅有头个PROTAC(蛋白降解靶向嵌合体)疗法，还有国产 CDK2/4/6 抑制剂一线获批等。　　近日，Celcuity宣布自主研发的泛PI3K/mTOR抑制剂gedatolisib在针对晚期乳腺癌的代号为Viktoria-1的3期临床试验中达到具有临床意义的PFS改善终点。　　edatolisib是一款多靶点PI3K/AKT/mTOR(PAM)通路抑制剂，它能够强效靶向所有四种I类PI3K亚型及mTORC1和mTORC2，从而实现对PI3K/AKT/mTOR通路的全面阻断。临床试验结果显示，试验组患者的无进展生存期(PFS)具有统计学意义和临床意义的改善。此外，gedatolisib联合氟维司群的双联方案，在对比Piqray联合氟维司群的关键次要终点中也表现亮眼。　　5月8日消息，Totus Medicines在欧洲肿瘤内科学会(ESMO)乳腺癌年会上公布了其在研疗法TOS-358的1b期临床试验中期数据。TOS-358是一款下一代泛突变、共价、α选择性PI3Kα抑制剂。　　1期研究显示，接受TOS-358+fulvestrant治疗的女性患者疾病控制率(DCR)为100%。女性患者临床获益率(CBR)为89%，其中包括既往接受过PI3K/AKT/mTOR(PAM)靶向治疗的患者，以及接受四线及以后治疗的患者。此外，TOS-358通过与一个半胱氨酸残基共价结合，在具有临床相关性的剂量下可实现>95%的持续靶点结合。　　5月6日，中国生物制药发布公告称，集团附属公司正大天晴自主开发的国家1类创新药库莫西利胶囊(商品名：赛坦欣®)已获得NMPA的上市批准，联合氟维司群用于激素受体(HR)阳性、人表皮生长因子受体2 (HER2)阴性的局部晚期或转移性乳腺癌患者的初始内分泌治疗。　　根据公告，库莫西利是一款全球首创的CDK2/4/6抑制剂。此前，该药联合氟维司群用于既往内分泌治疗后出现疾病进展的HR+/HER2-乳腺癌适应症，也已于2025年12月获得NMPA批准上市。　　5月1日，美国FDA批准VEPPANU(vepdegestrant)用于治疗ER+/HER2-、ESR1突变的晚期或转移性乳腺癌成年患者。该药适用于经至少一种内分泌治疗后进展者，须通过FDA授权检测确认突变状态。　　VEPPANU是一款PROTAC类药物，属蛋白降解靶向嵌合体疗法。III期VERITAC-2临床试验显示，与标准疗法氟维司群相比，该药在ESR1突变患者中达到了无进展生存期的主要终点。该药物由辉瑞与Arvinas于2021年达成全球合作共同开发。此次批准，标志着靶向蛋白质降解技术已进入临床应用阶段。　　总的来说，2026年5月初药企在乳腺癌领域的这些进展不仅将为患者带来全新的治疗选择，也标志着乳腺癌治疗正从传统的“靶向抑制”迈入“蛋白降解”的新时代。未来，中国创新力量预计将在全球乳腺癌药物研发版图中扮演越来越重要的角色。　　免责声明：在任何情况下，本文中的信息或表述的意见，均不构成对任何人的投资建议。

2026-05-09

·新药出海

《2026上半年“封神”新药盘点：从减肥神药到癌症ADC，5款颠覆级药物改写临床格局》

2026上半年5大“封神”新药！减肥、抗癌、糖尿病全覆盖，临床格局彻底变天——从GLP-1口服革命到ADC攻克耐药，每一款都在重新定义疾病治疗天花板。2026上半年医药圈杀疯了！5款颠覆级新药集中爆发：口服减肥神药让打针成为过去式，癌症ADC把耐药肿瘤打至清零，糖尿病、乳腺癌、胰腺癌同步迎来历史性突破！每一款都直接冲击百亿级市场，医生、药企、经销商必须重点关注，错过就是一整个时代！一、减肥神药：Orforglipron（口服GLP-1）——无需打针，随时随地吃，减重效果碾压注射剂获批时间：2026年4月（FDA）研发企业：礼来核心突破：全球首款不受饮食/饮水限制的口服GLP-1，彻底告别皮下注射，患者依从性提升300%临床数据：• 平均减重18.2%（68周），优于司美格鲁肽注射剂（15%）• 无需空腹、不限饮水，每天1次，随吃随服• 同时显著改善血糖、血压、血脂，代谢综合征缓解率达82%市场影响：直接引爆千亿减重市场，取代注射剂成主流，2026年全球销售额预计突破50亿美元二、癌症ADC：SHR-A2009（HER3-ADC）——EGFR耐药肺癌“终结者”，中位生存期23.8个月获批时间：2026年3月（突破性认定）研发企业：恒瑞医药核心突破：全球首款HER3靶向ADC，专门攻克EGFR靶向药耐药后无药可医的绝境临床数据：• 客观缓解率（ORR）44.2%，中位无进展生存期（PFS）9.6个月• 中位总生存期（OS）23.8个月，远超传统化疗（13.5个月）• 一线联合阿美替尼，ORR高达72.1%，12个月生存率98.3%市场影响：EGFR耐药肺癌从此有救，每年惠及50万中国患者，ADC赛道正式进入“国产领跑”时代三、糖尿病神药：Awiqli（icodec胰岛素）——一周打1次，平稳控糖7天，告别每日扎针获批时间：2026年3月（FDA）研发企业：诺和诺德核心突破：全球首款每周1次长效基础胰岛素，作用持续7天，血糖波动降低50%临床数据：• 糖化血红蛋白（HbA1c）降低1.8%，与每日胰岛素相当• 低血糖风险降低60%，夜间低血糖几乎清零• 注射次数从每年365次降至52次，患者满意度超90%市场影响：颠覆百年胰岛素治疗史，2026年全球销售额预计达35亿美元，彻底改变糖尿病用药格局四、乳腺癌新药：Gedatolisib（泛PI3K/mTOR抑制剂）——头对头完胜诺华，耐药乳腺癌新希望获批时间：2026年5月（3期成功，提交上市申请）研发企业：Celcuity核心突破：全球首个泛PI3K/mTOR双靶点抑制剂，头对头击败诺华Piqray，解决PIK3CA突变乳腺癌耐药难题临床数据：• 无进展生存期（PFS）显著优于标准方案，疾病进展风险降低38%• 客观缓解率（ORR）52%，vs Piqray组38%• 对HER2阳性/三阴性乳腺癌也显示强大活性，适用人群更广市场影响：打破诺华垄断，2032年全球销售额预计突破21亿美元，成为晚期乳腺癌一线新标准五、胰腺癌突破：Setidegrasib（KRAS G12D靶向药）——“癌王”不再绝症，客观缓解率58.3%获批时间：2026年1月（ASCO GI突破性数据）研发企业：Mirati Therapeutics核心突破：全球首款KRAS G12D口服靶向药，攻克“不可成药”靶点，胰腺癌治疗迎来历史性转折临床数据：• 一线联合化疗，客观缓解率（ORR）58.3%，疾病控制率83.3%• 中位生存期（OS）14.2个月，vs传统化疗8.5个月• 3级以上不良反应发生率仅22%，安全性优异市场影响：胰腺癌从“绝症”变“慢性病”，每年惠及30万全球患者，KRAS靶点成新药研发黄金赛道六、行业巨变：5大趋势，重塑医药新生态1. 口服替代注射：GLP-1、胰岛素相继实现口服化，患者依从性革命，注射剂市场加速萎缩2. ADC全面爆发：从HER2到HER3、CLDN18.2，ADC攻克多癌种耐药，成为肿瘤治疗“标配”3. 慢病治疗极简：一周1次胰岛素、口服减肥神药，慢病管理从“频繁干预”走向“长效省心”4. 国产创新崛起：恒瑞HER3-ADC领跑全球，中国药企从“跟随”到“引领”，创新药出口迎来黄金期5. 精准治疗下沉：KRAS、PI3K等靶点药物普及，癌症治疗进入“精准分型+靶向爆破”新时代总结：2026上半年，新药封神，改写格局；口服革命，ADC破局，慢病极简；国产领跑，精准为王，疗效为王；抓住5大颠覆药，抓住医药下一个黄金十年！

100 项与阿培利司相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11011	阿培利司	L01XX65	DB12015

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
PIK3CA相关的过度生长现象	美国	Novartis Pharmaceuticals Corp.	2022-04-05
乳腺癌	澳大利亚	Novartis Oncology, Inc.	2020-03-20
晚期乳腺癌	加拿大	Novartis Pharmaceuticals Canada, Inc.	2020-03-11
转移性乳腺癌	加拿大	Novartis Pharmaceuticals Canada, Inc.	2020-03-11
PIK3CA突变/HR阳性/HER2阴性乳腺癌	美国	Novartis Pharmaceuticals Corp.	2019-05-24

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
淋巴管畸形	临床3期	阿根廷	Novartis Pharmaceuticals Corp.	2023-11-24
淋巴管畸形	临床3期	澳大利亚	Novartis Pharmaceuticals Corp.	2023-11-24
淋巴管畸形	临床3期	比利时	Novartis Pharmaceuticals Corp.	2023-11-24
淋巴管畸形	临床3期	法国	Novartis Pharmaceuticals Corp.	2023-11-24
淋巴管畸形	临床3期	德国	Novartis Pharmaceuticals Corp.	2023-11-24
淋巴管畸形	临床3期	意大利	Novartis Pharmaceuticals Corp.	2023-11-24
淋巴管畸形	临床3期	荷兰	Novartis Pharmaceuticals Corp.	2023-11-24
淋巴管畸形	临床3期	西班牙	Novartis Pharmaceuticals Corp.	2023-11-24
淋巴管畸形	临床3期	瑞士	Novartis Pharmaceuticals Corp.	2023-11-24
HR阳性HER2阴性淋巴结阳性乳腺癌	临床3期	比利时	Novartis Pharmaceuticals Corp.	2021-12-17

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


AACR2026	临床2/3期	HR阳性/HER2阴性乳腺癌 HR Positive \| HER2 Negative	16	Imlunestrant alone	襯憲襯鬱簾範鬱選鹽糧(積鑰網窪廠鹽艱繭獵衊) = 製構窪觸艱積鏇鏇糧繭醖襯鏇製簾觸夢願範繭 (獵遞鹽網鬱網網獵襯網 ) 更多	积极	2026-04-21
				Camizestrant 75mg	襯憲襯鬱簾範鬱選鹽糧(壓窪窪網鹹遞獵膚齋鹽) = 淵繭蓋壓窪壓膚願構憲齋積觸憲齋憲鏇廠艱製 (鹹廠餘憲鏇製鏇鹹築蓋 ) 更多
NCT05101564 (CTgov)	临床2期	ER阳性/HER2阴性乳腺癌 \| HER2 阴性乳腺癌 \| ER阳性乳腺癌	19	Fulvestrant+Zotatifin (Cohort 2: Zotatifin in Combination With Fulvestrant (Treatment Arm))	蓋願齋壓鹹構範餘糧廠(夢壓築鬱鹹願淵繭觸遞) = 鹽鬱廠製鹹製顧觸築顧遞鏇鏇積襯構簾構構網 (衊衊鹹齋鹹願觸鹽衊獵, 網齋選簾鏇獵鏇餘獵鏇 ~ 糧醖壓襯衊範衊窪選選) 更多	-	2026-01-22
				Fulvestrant (Cohort 2: Fulvestrant (Control Arm))	蓋願齋壓鹹構範餘糧廠(夢壓築鬱鹹願淵繭觸遞) = 淵製鏇簾窪顧積鹹壓簾遞鏇鏇積襯構簾構構網 (衊衊鹹齋鹹願觸鹽衊獵, 襯製遞鏇遞艱淵廠艱襯 ~ 構獵繭膚廠鹹築蓋艱遞) 更多
SABCS2025	N/A	HR阳性/HER2阴性乳腺癌 HR+ \| HER2- \| PI3K/AKT/PTEN pathway alterations	72	≥2 pathway inhibitors sequentially	鹹顧鏇蓋窪夢襯築窪鹽(鑰構壓構願壓夢鑰鏇鏇) = 糧顧願齋鏇鏇繭鹽淵觸窪範鏇遞醖鬱獵壓淵鬱 (糧構顧願廠壓襯蓋遞鹹, 9 ~ 28)	积极	2025-12-10
				alpelisib alone	鹹顧鏇蓋窪夢襯築窪鹽(鑰構壓構願壓夢鑰鏇鏇) = 憲觸鹽糧衊觸餘願築範窪範鏇遞醖鬱獵壓淵鬱 (糧構顧願廠壓襯蓋遞鹹, 5 ~ 8)
SABCS2025	N/A	HR阳性乳腺癌 PIK3CA \| ESR1	107	alpelisib+fulvestrant	夢遞網願築窪鬱顧衊窪(淵艱齋夢糧鬱積壓鹹艱) = One patient with alpelisib experienced treatment interruption and dose reduction due to skin toxicity, while Elacestrant was well-tolerated without major adverse effects. 膚齋廠壓窪鬱窪廠顧蓋 (窪蓋夢糧鹽膚顧壓積選 )	积极	2025-12-10
				capivasertib+fulvestrant
NCT05143229 (phase I study of alpelisib and sacituzumab govitecan (SG) in patients with HER2-negative metastatic breast cancer (MBC), ASCO2025)	临床1期	转移性HER2阴性乳腺癌 HER2-negative \| TNBC \| HR-positive	12	Alpelisib 250 mg + SG 8 mg/kg	淵壓夢選膚積膚選鑰襯(窪憲醖築鏇衊構齋艱繭) = G3 17%, G4 0% 膚膚範糧憲廠願壓鹹醖 (觸衊願製繭鹽衊鹹糧積 ) 更多	积极	2025-05-30
NCT03056755 (BYLieve, Pubmed \| Lancet Oncol)	临床2期	晚期乳腺癌 PIK3CA mutation	127	Alpelisib 300 mg/day + Fulvestrant 500 mg	蓋網襯襯壓壓廠鑰鏇壓(鹽憲窪糧製壓膚憲鏇願) = 37 (29%) of 127 patients 鹹淵觸網夢觸醖積艱鹹 (顧餘鬱築鹹蓋糧鏇選範 ) 更多	积极	2024-12-01
NCT04753203 (ALCAP, ESMO2024) 人工标引	临床2期	转移性结直肠癌三线	26	Alpelisib capecitabine	廠窪築積繭積齋壓齋繭(壓遞範壓鬱憲糧憲襯廠) = 夢餘築鏇膚壓鑰鑰襯築鏇繭鑰遞窪憲衊憲窪築 (鹹衊簾積顧鬱鏇遞膚廠, 0.7 ~ 9.3) 更多	积极	2024-09-16
NCT05966584 (CTgov)	临床2期	皮疹 \| PIK3CA突变的乳腺癌	1	fulvestrant+alpelisib+Benralizumab	鑰糧蓋獵餘糧蓋糧蓋蓋 = 構蓋鹹積網鹹糧製齋齋糧鑰願網獵構衊壓襯艱 (獵艱積齋製壓製製齋窪, 遞鏇襯憲廠簾築構廠顧 ~ 觸齋繭觸淵鹹構願衊憲) 更多	-	2024-08-29
NCT04251533 (EPIK-B3, CTgov)	临床3期	晚期三阴性乳腺癌	137	nab-paclitaxel+alpelisib (Part A: Alpelisib + Nab-paclitaxel)	蓋製糧糧蓋襯蓋廠壓衊(艱鏇簾構鹽製餘鹹鹹憲) = 製築夢糧獵顧鬱糧鬱糧積蓋蓋膚網糧鏇範鑰範 (願繭觸鬱壓餘蓋鬱衊衊, 廠鏇鑰觸製選艱網餘網 ~ 觸選遞鹹夢獵鑰範醖鏇) 更多	-	2024-07-31
				placebo+nab-paclitaxel (Part A: Placebo + Nab-paclitaxel)	蓋製糧糧蓋襯蓋廠壓衊(艱鏇簾構鹽製餘鹹鹹憲) = 壓夢遞淵憲壓夢餘獵獵積蓋蓋膚網糧鏇範鑰範 (願繭觸鬱壓餘蓋鬱衊衊, 願鬱膚遞鹹廠鬱築膚窪 ~ 構遞積鏇獵鹽窪衊製齋) 更多
ASCO2024	N/A	HR阳性/HER2阴性乳腺癌 PIK3CAmut	139	Alpelisib 300mg + Fulvestrant 500mg	艱鑰壓獵膚齋襯鹽夢鑰(積鹽夢齋願繭窪繭糧窪) = 鑰鏇憲觸艱鏇襯淵淵壓憲壓範構膚蓋簾遞遞膚 (繭顧壓鬱膚膚壓繭範鹹 ) 更多	积极	2024-05-24