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更新于：2026-06-04

Alpelisib

阿培利司

更新于：2026-06-04

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 Pharma Stein AG

Novartis Pharmaceuticals Corp.Novartis Pharma AG

+ [6]

非在研机构

The University of California, San Francisco

权益机构

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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989

项与阿培利司相关的文献（医药）

2026-08-01·EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Benzothiophene-based, orally active PIK3CA H1047R mutant-selective inhibitors for the treatment of HR+/HER2- breast cancer

Article

作者: Xu, Hang ; Jin, Lei ; Yang, Fanglong ; Wang, Siqin ; Jia, Xiangyu ; Fan, Xing ; Xia, Yuanfeng ; Zhao, Sheng ; Lu, Biao

PI3Kα plays a key role in a variety of cellular processes, and its gene mutations are closely related to the occurrence and development of many types of cancer. Although two orthosteric PI3Kα inhibitors including Alpelisib and Inavolisib have been launched onto market, they often cause toxic and side effects due to insufficient selectivity for PIK3CA mutant protein. The development of allosteric PI3Kα inhibitors provides new ideas for overcoming these problems. Our research focuses on optimizing a novel series of allosteric PI3Kα inhibitors derived from STX-478. By integrating scaffold hopping and comprehensive structural modification strategies, we obtained the lead compound allosteric PI3Kα inhibitor 11f with a benzothiophene scaffold. The inhibitor demonstrates high selectivity for PIK3CA mutant protein, low hERG inhibition, minimal CYP inhibition, excellent in vivo efficacy, good safety and no impact on insulin balance. Collectively, these findings confirm that compound 11f is a highly promising drug candidate for the targeted therapy of PIK3CA H1047R mutant HR+/HER2-breast cancer.

2026-07-01·INTERNATIONAL IMMUNOPHARMACOLOGY

Targeting NBL1/PI3K/AKT axis to enhance the chemosensitivity and radiosensitivity of esophageal squamous cell carcinoma

Article

作者: Wang, Bing ; Feng, Bin ; Zeng, Renya ; Lei, Lingli ; Yang, Zhe

BACKGROUND:

The development of radioresistance and chemoresistance remains a huge obstacle for the prognosis of esophageal squamous cell carcinoma (ESCC) patients. When investigating novel therapeutic targets involved in ESCC radioresistance and chemoresistance, we identified that ESCC patients expressing lower levels of neuroblastoma suppression of tumorigenicity 1 (NBL1) tended to have short overall survival. This research aimed to elucidate the functional roles of NBL1 in ESCC progression and further identify its downstream signaling pathway.

METHODS:

The expression level of NBL1 in ESCC tissues and non-tumor tissues was evaluated using enzyme-linked immunosorbent assay (ELISA) and immunohistochemistry (IHC). The correlation between NBL1 expression and patient survival was analyzed using the Kaplan-Meier survival curve method. Knockdown and overexpression of NBL1 in ESCC cells were achieved via short hairpin RNA (shRNA) and lentiviral transduction, respectively. The survival capacity of NBL1-silenced cells under chemotherapy and radiotherapy was assessed through colony formation assay and Cell Counting Kit-8 (CCK-8) assay. Western blotting was employed to identify downstream signaling pathways of NBL1 involved in chemoresistance and radioresistance in ESCC, revealing that the PI3K-AKT (phosphoinositide 3-kinase-AKT signaling) pathway was activated in NBL1-silenced cells. Colony formation and CCK-8 assays were further applied to evaluate cell survival after combined treatment with chemotherapy and PI3K-AKT inhibitors (LY294002 or BYL719) or radiotherapy combined with LY294002/BYL719. Levels of (Bone Morphogenetic Protein) BMP2, 4, 6, and 7 were interfered with using siRNA in NBL1-knockdown cells, followed by qPCR validation and Western blot analysis to determine the expression of PI3K, AKT, and p-AKT proteins. A mouse xenograft model was established to investigate the antitumor efficacy of LY294002 and BYL719 in enhancing chemosensitivity and radiosensitivity in esophageal squamous cell carcinoma.

RESULTS:

Kaplan-Meier survival analysis revealed that ESCC patients with lower NBL1 expression had a shorter overall survival (P = 0.0141), suggesting NBL1 as a potential prognostic biomarker for ESCC. To investigate the functional role of NBL1 in ESCC progression, we performed knockdown of NBL1 expression in ESCC cells for functional assays. The results demonstrated that downregulation of NBL1 significantly promoted cell proliferation and migration, and enhanced resistance to multiple chemotherapeutic agents (chemoresistance) as well as radiotherapy (radioresistance). These phenotypes indicate that NBL1 may function as a tumor suppressor in ESCC. Mechanistically, both in vitro and in vivo studies confirmed that the PI3K-AKT signaling pathway was markedly activated in NBL1-knockdown ESCC cells. Further rescue experiments showed that treatment with PI3K-AKT pathway inhibitors (LY294002 or BYL719) significantly sensitized ESCC cells, particularly NBL1-silenced cells, to chemotherapy and radiotherapy. The qPCR results verified effective reduction in BMP2, 4, 6, and 7 levels following siRNA interference. Western blot analysis indicated that knockdown of BMP7 led to increased expression of PI3K, AKT, and p-AKT proteins. Taken together, these findings suggest that NBL1 may negatively regulate the PI3K-AKT signaling pathway through BMP2,4,7 thereby suppressing malignant progression and therapy resistance in ESCC.

CONCLUSION:

NBL1 functions as a tumor suppressor in ESCC. Its downregulation enhances radioresistance and chemoresistance in ESCC by activating the PI3K-AKT signaling pathway through BMP2,4,7. Inhibition of the PI3K-AKT pathway with LY294002 or BYL719 increases the cytotoxic effects of chemotherapy and radiation, offering a novel therapeutic strategy for ESCC patients, particularly those with low NBL1 expression.

2026-06-01·NEOPLASIA

PIK3CA mutant cervical cancer is selectively suppressed by PI3Kα inhibition (Alpelisib/BYL-719 and Inavolisib/GDC-0077) and cooperates with HPV directed T cell therapy

Article

作者: Syracuse, Nick ; Langan, David ; Robinson, Emma ; Oberley, Matthew R ; Xie, Yi ; Lou, Hong ; Kim, Sojung ; Dean, Michael ; Rossi, Nicole M ; Oelke, Mathias ; Murphy, Elisabeth A ; Rao, Nina ; Rodriguez, Isabel ; Joe, Tawnjerae ; Tulsyan, Sonam

Cervical cancer is largely driven by human papillomavirus (HPV) infection, yet clinically actionable molecular subtypes and effective targeted therapies remain limited. Here, we define biologically and clinically relevant subtypes and evaluate targeted therapeutic strategies. Analysis of public datasets, coupled with functional studies in cervical cancer cell lines and immune assays, identified three subtypes: (I) PIK3CA wild-type without YAP1 amplification, (II) PIK3CA-mutant without YAP1 amplification, and (III) PIK3CA wild-type with YAP1 amplification. Notably, YAP1 amplification is associated with poorer patient survival. The PI3Kα-specific inhibitors, Alpelisib (BYL-719) and Inavolisib (GDC-0077), selectively inhibited proliferation in multiple PIK3CA-mutant cervical cancer cell lines but had minimal effect in PIK3CA wild-type cells. Alpelisib further reduced expression of HPV16 E7, PD-L1 (CD274), YAP1, and EGFR specifically in PI3Kα-mutant models. In an HPV16-positive, HLA-A2-positive, PIK3CA-mutant cell line (CaSki), antigen-specific donor T cells (NexImmune) induced cytotoxicity in a dose-dependent manner. Importantly, combining BYL-719 with T-cell therapy enhanced tumor cell killing, with maximal effects observed following drug pretreatment prior to T-cell exposure. These findings identify actionable molecular subtypes of cervical cancer and support targeting PI3Kα in PIK3CA-mutant tumors. Moreover, combining PI3K inhibition with antigen-specific immunotherapy represents a promising strategy to improve outcomes in advanced HPV16-associated cervical cancer.

574

项与阿培利司相关的新闻（医药）

2026-06-04

·研发客

PFS翻倍，股价却跌超25%！是ASCO赢家还是输家？

ASCO期间，戏剧性的一幕再度上演。 Celcuity公司的乳腺癌候选药gedatolisib在III期研究中将无进展生存期（PFS）延长一倍，死亡或疾病进展风险降低一半。尽管如此，这家生物科技公司的股价仍下跌超过25%。 gedatolisib是一款多靶点 PAM 抑制剂，它能有效作用于所有四种 I 类 PI3K 亚型、mTORC1 和 mTORC2，从而全面阻断 PAM 通路。这也使其与目前获批的针对 PAM 通路的单靶点抑制剂有着显著不同。在VIKTORIA-1 III 期研究中，Celcuity公司招募了700多名携带PIK3CA突变的HR阳性、HER2阴性晚期或转移性乳腺癌患者。这类患者目前的标准治疗方案为氟维司群联合诺华的PI3Kα抑制剂alpelisib（Piqray）。来源 | Celcuity官网最新公布的结果显示，gedatolisib与氟维司群的二联方案，与标准治疗相比，中位PFS显著延长，分别为11.3个月和5.6个月，将疾病进展或死亡风险降低了49%（HR=0.51）；gedatolisib二联方案的ORR为35.7%，中位DoR为24.2个月。在gedatolisib与氟维司群及辉瑞CDK4/6抑制剂palbociclib（Ibrance）联合使用的三联方案中，与标准治疗相比，患者中位PFS显著改善，无疾病进展或死亡风险降低50%（HR=0.50）。两组经BICR评估的中位PFS分别为11.1个月和5.6个月，ORR分别为48.9%和26.0%，中位DoR分别为15.7个月和7.5个月。 Jefferies在一份投资者报告中表示，这种程度的PFS改善“在我们看来将改变临床实践”。分析师们继续指出，“与Piqray对照组相比，geda三联疗法组的PFS提高了一倍”，“与标准疗法（SOC）相比，这是一个显著的改善，可能会在短期内推动该疗法的快速普及。” 据外媒BioSpace报道，Celcuity公司回应了此次抛售，将其股价疲软归因于“市场对绝对PFS数值的预期过高”。 Jefferies还指出股价暴跌的另一个原因是，与双药疗法相比，gedatolisib三药疗法“似乎没有PFS优势”——两组的PFS结果非常相似——“使得实际商业用途变得不太明确”。然而，分析师们认为，三药联合疗法能带来更高的客观缓解率和更早的总生存期曲线分离，“这可能支持在二线治疗中对身体状况较好的患者或更广泛的患者群体使用三药联合疗法”。在Celcuity公司公布研究结果时，总生存期数据尚不成熟。 Jefferies仍然非常看好gedatolisib，认为该药物“可以在乳腺癌领域占据主导地位”，在二线治疗中“占有重要地位”，甚至有可能在一线治疗中也占有重要地位。目前，gedatolisib已在接受FDA审评，用于治疗HR阳性、HER2阴性且不携带PIK3CA突变的晚期乳腺癌患者，预计将于7月17日做出决定。Celcuity公司还表示，计划将VIKTORIA-1数据作为该申请的补充材料提交。拓展阅读让信达恒瑞受挫的PI3K，何去何从？ 11款新药前瞻：谁会成为重磅炸弹？ PI3Kα 迷雾渐散？礼来25亿美元入局总第2942期访问研发客网站www.PharmaDJ.com 深度报道和每日新闻抢鲜看

2026-06-03

·药番茄Pharmato

Celcuity: Gedatolisib联合疗法使PIK3CA突变乳腺癌PFS翻倍

在2026年ASCO大会上，美国生物技术公司Celcuity公布了III期关键注册研究VIKTORIA-1的最新结果。数据显示，其核心产品Gedatolisib联合方案在HR阳性、HER2阴性、PIK3CA突变晚期乳腺癌患者中取得突破性疗效，相较目前标准治疗方案Alpelisib联合氟维司群，将疾病进展或死亡风险降低约50%，无进展生存期（PFS）实现翻倍改善。这一结果不仅创造了PI3K/AKT/mTOR（PAM）通路靶向治疗领域的重要里程碑，也使Gedatolisib有望成为该患者群体新的治疗标准。晚期HR+/HER2-乳腺癌仍面临巨大未满足需求 HR阳性、HER2阴性乳腺癌是乳腺癌中最常见的亚型，约占全部乳腺癌病例的70%。近年来，CDK4/6抑制剂联合内分泌治疗已经成为该类患者的一线标准治疗方案。然而，大多数患者最终仍会出现耐药和疾病进展。其中，PIK3CA突变是最常见的获得性驱动事件之一，在HR+/HER2-晚期乳腺癌中的发生率约为40%。目前获批的PI3Kα抑制剂Alpelisib虽然能够改善患者预后，但疗效提升有限，同时高血糖、皮疹等毒性反应显著，影响长期治疗依从性。因此，如何更有效地抑制PI3K/AKT/mTOR信号通路，成为乳腺癌研究的重要方向。VIKTORIA-1：首个PI3K通路抑制剂“头对头”III期研究 Gedatolisib正是在这一背景下开发的新一代PAM通路抑制剂。与现有药物不同，Gedatolisib并非针对单一靶点，而是能够同时抑制全部四种I类PI3K亚型以及mTORC1和mTORC2，实现对PI3K/AKT/mTOR信号网络的全面封锁。从机制上看，这种多靶点抑制策略有望避免单靶点治疗过程中常见的代偿性激活和旁路耐药问题。 VIKTORIA-1是全球首个直接比较两种PAM通路抑制剂疗效的III期临床研究。研究共纳入701例HR阳性、HER2阴性晚期乳腺癌患者，并按照PIK3CA状态进行独立分析。其中PIK3CA突变队列共纳入350例患者，这些患者均已接受过CDK4/6抑制剂联合芳香化酶抑制剂治疗并出现疾病进展。患者按照3:3:1比例随机分配至三组，分别接受Gedatolisib联合氟维司群和Palbociclib三联治疗、Alpelisib联合氟维司群标准治疗以及Gedatolisib联合氟维司群双联治疗。核心数据：无进展生存期实现“翻倍” 研究最核心的数据来自Gedatolisib三联方案与Alpelisib对照组的比较。经独立影像学评估后发现，Gedatolisib三联方案将疾病进展或死亡风险降低了50%，风险比（HR）达到0.50（95%CI：0.37-0.68，P<0.0001）。更值得关注的是，中位无进展生存期从Alpelisib组的5.6个月延长至11.1个月，实现了接近翻倍的改善。对于经历CDK4/6抑制剂治疗失败后的患者而言，后线治疗PFS能够突破10个月一直被认为是极具挑战性的目标。因此，11.1个月的中位PFS不仅具有统计学意义，更具有重要的临床价值。Celcuity表示，这是目前所有包含内分泌治疗的HR+/HER2-晚期乳腺癌二线III期研究中报告的最长PFS数据。除了延缓疾病进展外，Gedatolisib方案还显著提高了肿瘤缓解率。三联方案的客观缓解率达到48.9%，而Alpelisib组仅为26.0%。换言之，几乎每两名患者中就有一名实现了肿瘤显著缩小。与此同时，缓解持续时间也从对照组的7.5个月延长至15.7个月，再次实现翻倍改善。双联方案同样展现出令人印象深刻的疗效。Gedatolisib联合氟维司群组的中位PFS达到11.3个月，相较对照组的5.6个月同样实现超过两倍提升，风险比为0.51。其客观缓解率达到35.7%，而中位缓解持续时间更是达到24.2个月，显示出极强的长期疾病控制能力。安全性评价：更强效，亦更“温柔” 如果仅从疗效数据来看，Gedatolisib已经建立起当前PAM通路治疗领域的新标杆。但更令人意外的是，其安全性表现同样优于现有标准治疗。长期以来，高血糖一直是Alpelisib最受关注的不良反应之一，也是导致患者减量甚至停药的重要原因。在本研究中，3级及以上高血糖发生率在Alpelisib组达到14.5%，而Gedatolisib三联组仅为2.6%，双联组甚至未观察到相关事件。严重皮疹发生率方面，Gedatolisib三联组和双联组分别为6.5%和5.8%，显著低于Alpelisib组的15.1%。从治疗持续性来看，因治疗相关不良事件导致停药的比例在Gedatolisib三联组仅为2.6%，双联组为3.8%，而Alpelisib组达到7.1%。这意味着更多患者能够长期接受治疗并持续获得临床获益。值得注意的是，目前VIKTORIA-1的总生存期数据尚未成熟，但研究者已经观察到积极趋势。如果未来OS分析能够进一步验证生存获益，那么Gedatolisib有望成为近年来HR+/HER2-晚期乳腺癌领域最具影响力的新药之一。从产业角度看，VIKTORIA-1的成功具有超越单个产品的意义。过去十余年，PI3K/AKT/mTOR通路始终被认为是肿瘤治疗领域最具潜力但又最具挑战性的靶点之一。大量药物开发项目因疗效不足或毒性问题折戟，而Gedatolisib首次在III期研究中证明，多靶点PAM通路抑制不仅能够带来更强疗效，而且能够实现更好的安全性管理。未来展望目前Celcuity已经计划基于VIKTORIA-1结果向FDA提交补充新药申请。与此同时，公司针对PIK3CA野生型患者的上市申请已获得FDA优先审评资格，PDUFA日期为2026年7月17日。此外，两项一线治疗III期研究VIKTORIA-2也正在推进中，分别针对内分泌耐药和内分泌敏感患者群体。对于HR+/HER2-晚期乳腺癌治疗而言，CDK4/6抑制剂时代之后的竞争焦点，正在逐步转向如何延长患者后线治疗获益。VIKTORIA-1的数据表明，通过对PAM通路进行更全面、更彻底的抑制，或许能够为这一难题提供新的解决方案。如果后续监管审评顺利，Gedatolisib很可能成为未来几年乳腺癌领域最值得关注的新标准治疗之一。 --- 以上正文 --- 往期好文《新药价值评估模型 3.0》提示词 MASH 新药竞争格局-2026年3月口服GLP-1竞争格局 JPM值得关注的CNS领域进展：Kv7 钾离子通道诺和诺德：中国最高法院对司美格鲁肽化合物专利作出了积极的裁决报告：肿瘤浸润淋巴细胞（TIL）疗法综述 - 2025年12月 Treg：调节性T细胞（Treg）疗法演进过程、竞争格局（含附件）体内CAR-T：竞争格局全析，交易、管线、主流递送平台 TSLP竞争格局：SHR-1905启动特应性皮炎2期临床报告：FGF21的研究进展-2025年10月报告：T细胞衔接器（TCE）在自身免疫性疾病的研究进展 2026年的创新药，需要关注的重点药番茄追踪新药情报，分享AI在制药领域落地应用，欢迎感兴趣的朋友加群交流。若如上二维码添加失败，也可以添加助理微信进群。声明：本文内容供业内同行交流学习，不代表药番茄表平台立场，不构成任何投资意见和建议。药番茄不对任何主体因使用本文内容而导致的任何损失承担责任。内容如有疏漏，欢迎沟通指出！

临床结果ASCO会议临床3期临床2期

2026-06-03

·生物制品圈

ASCO 2026重磅：PFS 翻倍！Celcuity 击败诺华新药，改写乳腺癌治疗格局

2026年美国临床肿瘤学会（ASCO）年会重磅更新，美国生物科技企业Celcuity公布其核心靶点药物gedatolisib的III期临床关键数据。作为全新PAM通路抑制剂，gedatolisib在头对头研究中，疗效全面超越诺华经典PI3K抑制剂Piqray（阿培利司），为难治性晚期HR+/HER2-乳腺癌带来突破性治疗方案，有望颠覆该领域现有治疗格局。核心数据出炉：疗效翻倍，刷新同类最佳纪录本次ASCO大会上，Celcuity首次完整披露核心III期 VIKTORIA-1研究数据。该研究聚焦临床高发的HR+/HER2-晚期及转移性乳腺癌人群，重点针对存在PIK3CA基因突变的患者群体，精准解答了这类难治患者的临床治疗难题。数据显示：对照组（Piqray + 氟维司群）中位无进展生存期（PFS）为5.6 个月 gedatolisib + 氟维司群双药组中位 PFS 达到11.3 个月，疗效近乎翻倍 gedatolisib + 氟维司群 + 哌柏西利（Ibrance）三联组中位 PFS 为11.1 个月疗效终点数据亮眼，gedatolisib联合氟维司群双药方案客观缓解率（ORR）达35.7%，在此基础上联合哌柏西利的三联方案ORR更是高达48.9%。Celcuity CEO Brian Sullivan公开表示，该组缓解数据是当前该适应症领域的最优临床成果。尽管整体数据略低于行业机构此前的市场预期，但PFS近乎翻倍的核心临床获益，依然获得了在场肿瘤领域专家的高度认可。Sullivan指出，在实体瘤靶向治疗中，实现患者核心预后指标的翻倍提升实属罕见，充分印证了这款药物的极高临床价值。安全性优势显著：解决同类药物最大痛点安全性短板是传统PI3K抑制剂临床应用的主要桎梏，其中高血糖不良反应为该类药物最核心、最常见的痛点。而gedatolisib凭借差异化的作用机制，实现了同类药物中最低的高血糖发生率，有效破解了长期困扰临床的用药安全难题。 Celcuity首席医学官Igor Gorbatchevsky在报告中表示，两种gedatolisib治疗方案均展现出优异的耐受性，因不良反应停药的患者比例极低。药物主要不良反应为中性粒细胞减少、口腔炎，均为临床可控性不良反应，整体安全性显著优于阿培利司联合治疗方案。在用药便捷性方面，gedatolisib采用每月3次静脉输注的给药模式，彻底摆脱传统靶向药物28天不间断口服的繁琐方案，有效减少患者药物暴露时长，降低长期用药负担，实现疗效、安全性与依从性的多重优化。机制创新：一次性阻断整条 PAM 通路 gedatolisib的突破性优势，核心源于其独一无二的全程通路抑制机制。区别于传统药物仅靶向PAM通路单一节点的治疗模式，该药可同时精准抑制通路三大核心靶点PI3K、AKT、mTOR，全面阻断肿瘤增殖的异常信号通路。业内曾形象比喻，RAS靶点是难以靶向的“油球”，而PAM通路则是结构复杂、多节点联动的“多足蜘蛛”与“魔方”，长期被视作难以攻克的成药难点，而gedatolisib成功实现了整条复杂通路的精准抑制。 PAM通路是调控肿瘤细胞增殖、分化、存活的核心信号通路，其异常激活广泛参与多种肿瘤的发生进展。gedatolisib的研发突破，顺利攻克这一经典“不可成药”靶点难题，填补了该领域的精准治疗空白。值得一提的是，这款潜力重磅药物，是Celcuity于2021年以1000万美元现金+股票的超低成本从辉瑞授权引进，成为全球生物医药行业低成本斩获重磅管线的经典案例。审批在即，剑指 100 亿美元市值目前，gedatolisib针对无PIK3CA突变的HR+/HER2-晚期乳腺癌适应症的上市申请已提交FDA，预计2026年7月17日迎来最终审批结果。依托本次ASCO公布的PIK3CA突变人群优异数据，Celcuity计划在2026年第三季度提交补充新药申请，进一步拓宽药物适用人群。与此同时，该药两项一线治疗III期临床试验正在稳步推进。HR+/HER2-乳腺癌一线治疗市场空间广阔，若一线研究顺利取得阳性结果，Celcuity有望跻身百亿市值生物科技企业行列。 Sullivan表示，依托gedatolisib的全场景治疗潜力，冲击百亿市值是企业清晰的战略发展目标。伴随FDA审批进入倒计时，gedatolisib有望彻底重塑HR+/HER2-晚期乳腺癌的临床治疗体系。凭借疗效翻倍、安全性优异、用药便捷的多重核心优势，该药极大概率成为该亚型乳腺癌全新标准治疗方案，为广大晚期乳腺癌患者带来更优质的生存获益。参考来源：https://www.fiercebiotech.com/biotech/asco-10-billion-ambitions-celcuity-details-breast-cancer-win-over-piqray 识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观不本站。

临床结果ASCO会议临床3期突破性疗法

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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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	網糧積艱襯願觸襯淵艱(製觸觸鬱艱範積衊構襯) = 衊鬱鬱窪鏇糧積鹽鏇積網窪顧觸壓鹽鏇壓範築 (選壓鬱製憲餘鏇窪網襯 ) 更多	积极	2026-04-21
				Camizestrant 75mg	網糧積艱襯願觸襯淵艱(憲夢構觸蓋窪繭鹽壓衊) = 鏇鏇淵蓋鏇醖構餘鏇範獵艱獵艱製範構蓋製膚 (淵觸製繭鹹糧艱製憲鬱 ) 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))	窪簾築簾壓觸願網製顧(製鹽壓顧蓋廠壓淵簾獵) = 鏇鬱製膚鏇壓築壓膚壓糧淵窪鑰糧鏇艱膚膚鹹 (醖願壓蓋鏇築範醖蓋範, 餘餘醖選鑰範齋繭醖築 ~ 壓鑰膚鏇觸觸餘範構糧) 更多	-	2026-01-22
				Fulvestrant (Cohort 2: Fulvestrant (Control Arm))	窪簾築簾壓觸願網製顧(製鹽壓顧蓋廠壓淵簾獵) = 齋遞醖積蓋憲選願繭遞糧淵窪鑰糧鏇艱膚膚鹹 (醖願壓蓋鏇築範醖蓋範, 窪構襯鹽網廠鏇齋願淵 ~ 衊蓋觸醖窪鏇獵艱鹹憲) 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	獵製鬱顧醖膚艱鏇網製(膚鹹鬱繭鬱糧積齋顧簾) = 壓夢憲齋壓願製艱範襯製鹽製簾鹹衊鹽壓鑰醖 (積鬱廠淵憲選鏇齋壓簾, 9 ~ 28)	积极	2025-12-10
				alpelisib alone	獵製鬱顧醖膚艱鏇網製(膚鹹鬱繭鬱糧積齋顧簾) = 選膚範築膚襯鹽顧鹹構製鹽製簾鹹衊鹽壓鑰醖 (積鬱廠淵憲選鏇齋壓簾, 5 ~ 8)
SABCS2025	N/A	HR阳性乳腺癌 PIK3CA \| ESR1	107	alpelisib+fulvestrant	壓齋鬱築鹹製蓋襯壓構(獵顧憲鑰選齋糧遞鑰鹹) = One patient with alpelisib experienced treatment interruption and dose reduction due to skin toxicity, while Elacestrant was well-tolerated without major adverse effects. 簾壓製窪築憲選構襯夢 (憲鬱廠顧餘遞選築襯餘 )	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	鹽顧願構夢襯蓋廠憲簾(簾觸構積網夢夢夢醖鹽) = G3 17%, G4 0% 鹽蓋窪艱蓋壓糧窪艱艱 (構壓餘艱鹽淵願遞衊範 ) 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	鑰壓襯遞鏇範繭網觸簾(鑰觸鹹獵獵鏇獵廠淵繭) = 37 (29%) of 127 patients 膚蓋憲構廠選鬱鏇艱簾 (網窪選憲膚遞鹽醖夢膚 ) 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	積夢範鏇夢繭艱壓鏇築(夢襯餘餘遞憲膚餘繭顧) = 獵築選鏇醖網積襯鏇製願繭鏇鑰簾選鏇壓鹽憲 (齋鑰醖繭鑰夢餘鑰醖製, 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	鏇夢遞獵夢淵夢簾糧積 = 選鑰網構襯範衊蓋構餘選積願蓋構壓築餘構齋 (齋鹽淵簾憲廠齋範壓糧, 鏇範蓋壓簾築襯繭衊選 ~ 獵築夢網選積襯醖繭憲) 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)	淵簾艱鬱鑰艱願鹽鏇範(獵襯淵網淵壓襯選觸餘) = 糧齋築製繭觸衊壓醖築壓選憲鏇夢製構夢繭簾 (鑰餘醖網鏇願淵鏇製顧, 鏇願獵蓋繭獵簾網顧膚 ~ 繭鏇顧構範蓋壓餘鏇遞) 更多	-	2024-07-31
				placebo+nab-paclitaxel (Part A: Placebo + Nab-paclitaxel)	淵簾艱鬱鑰艱願鹽鏇範(獵襯淵網淵壓襯選觸餘) = 鏇構觸廠蓋鑰窪壓夢鹹壓選憲鏇夢製構夢繭簾 (鑰餘醖網鏇願淵鏇製顧, 淵積蓋糧構糧網憲糧衊 ~ 廠膚獵餘衊鑰壓淵齋齋) 更多
ASCO2024	N/A	HR阳性乳腺癌 PIK3CA-mutated \| CDK4/6i	115	Alpelisib + Endocrine Therapy	觸膚選鹽齋製選鹽艱醖(顧製衊淵鏇淵憲構簾壓) = 顧鑰鹹壓簾積觸淵築築範壓簾範獵衊糧齋構選 (網築齋醖淵艱鬱醖遞淵, 5.5 ~ 13.0) 更多	不佳	2024-05-24
				Everolimus + Endocrine Therapy	觸膚選鹽齋製選鹽艱醖(顧製衊淵鏇淵憲構簾壓) = 築構憲觸鏇膚觸積壓鹹範壓簾範獵衊糧齋構選 (網築齋醖淵艱鬱醖遞淵, 7.0 ~ 14.0) 更多