ITCC-104: HEM-iSMART International Proof of Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory HEMatological Malignancies in Children, Sub-Protocol E: Capivasertib + Venetoclax + Dexamethasone in Pediatric Patients With Relapsed or Refractory Hematological Malignancies

HEM-iSMART is a master protocol which investigates multiple investigational medicinal products in children, adolescents and young adults (AYA) with relapsed/refractory (R/R) ALL and LBL. Sub-protocol E is a phase I/II trial evaluating the safety and efficacy of capivasertib + venetocolax in combination with dexamethasone in children and AYA with R/R ped ALL/LBL whose tumor present with alterations of the PAM pathway, or lacking any mutations.

开始日期2026-10-01

申办/合作机构

Princess Maxima Centre For Pediatric Oncology

[+2]

CTIS2025-523460-21-00

/ Not yet recruiting临床2期IIT

A multicenter, multicohort, phase 2 platform trial to personalize second-line treatment intensity and targeting in HR-positive, HER2-negative metastatic breast cancer through an integrated liquid biopsy algorithm.

开始日期2026-06-01

申办/合作机构-

NCT07486648

/ Not yet recruiting临床1/2期IIT

The Safety and Efficacy of Osimertinib Plus Capivasertib in EGFRm Advanced Non-small Cell Lung Cancer (NSCLC) Participants With PIK3CA/AKT1/PTEN Alterations Who Had Progressed on First-line Osimertinib Monotherapy or Plus Chemotherapy: a First-in-human, Phase Ib/Ⅱa Study (PRECISION)

The goal of this clinical trial is to learn if Osimertinib plus Capivasertib works to treat EGFRm advanced non-small cell lung cancer (NSCLC) in participants with PIK3CA/AKT1/PTEN alterations after progression on first-line Osimertinib (monotherapy or plus chemotherapy).
The main questions it aims to answer are:
Part A:
* Number of Dose-limiting toxicities (DLTs)
* Adverse events (AEs)/serious adverse events (SAEs) (graded by CTCAE Version 5.0)
* Recommended combined dose (RCD)
Part B:Confirmed ORR assessed by the Investigator per RECIST 1.1 criteria.
Participants will:
Part A:Take Capivasertib twice daily from day 1 to 4 of a 7-day cycle, Osimertinib will be given orally QD(once daily) at 80 mg throughout the study treatment period.
Part B: Take Osimertinib (80mg QD, continuously) and Capivasertib(RCD,orally BID from day1-day 4 in 7-day cycle , 4 days on /3 days off) till disease progression (PD) or unacceptable toxicity.

开始日期2026-05-15

申办/合作机构

山西省肿瘤医院（山西省癌症中心）（山西医科大学附属肿瘤医院（山西医科大学肿瘤医学院）、山西省第三人民医院、山西省肿瘤研究所）

[+1]

100 项与卡匹色替相关的临床结果

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100 项与卡匹色替相关的转化医学

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100 项与卡匹色替相关的专利（医药）

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394

项与卡匹色替相关的文献（医药）

2026-12-31·Hematology

Neutrophil extracellular trap-related genes in PTCL: identification, prognosis and drug interaction prediction via bioinformatics-machine learning

Article

作者: Chen, Jing ; Cheng, Fanjun ; Fang, Jun

OBJECTIVE:

This study aimed to identify neutrophil extracellular trap-related genes (NET-RGs), explore their prognostic significance, and predict drug interactions in peripheral T-cell lymphoma (PTCL).

METHODS:

Differentially expressed NET-RGs (DE-NRGs) between PTCL and normal tissues were screened. Functional enrichment analysis was conducted. Bioinformatics and machine learning were used to identify hub genes and assess their diagnostic value. Univariate and multivariate analyses were used to evaluate prognostic roles. Correlation and immune infiltration analyses were performed to explore relationships with the tumor microenvironment (TME). Clinical data were collected from PTCL patients who received potential agents (lenalidomide) as first-line treatment.

RESULTS:

A total of 31 DE-NRGs were identified (18 upregulated and 13 downregulated), enriched in inflammatory response, extracellular matrix organization, and infection involvement. Four hub genes (AKT2, MAPK14, IRF1, and TNF) were identified as effective PTCL diagnostic markers. Higher AKT2/MAPK14 expression correlated with poorer overall survival (OS), while elevated TNF expression associated with better OS; AKT2 and TNF independently predicted OS. These genes were implicated in modulating TME remodeling. Potential therapeutic agents (e.g. capivasertib, lenalidomide) were predicted, and lenalidomide may represent a feasible initial treatment option for PTCL, with an objective response rate (ORR) of 40.0% and a maximum survival duration exceeding 50 months.

CONCLUSION:

NET-RGs play crucial roles in diagnosis, prognosis, and TME regulation, and lenalidomide, a putative TNF-targeting agent, may represent a feasible initial treatment option in PTCL.

2026-12-31·JOURNAL OF MEDICAL ECONOMICS

Cost-effectiveness analysis of capivasertib plus fulvestrant in the PIK3CA/AKT1/PTEN -altered subgroup with HR+/HER2− advanced breast cancer: a United States payer perspective

Article

作者: Tan, Chia Jie ; Liang, Xi ; Malone, Daniel C.

AIMS:

Capivasertib plus fulvestrant has emerged as a promising targeted therapy for HR+/HER2- advanced breast cancer (ABC). This study evaluated the cost-effectiveness of capivasertib plus fulvestrant vs. fulvestrant monotherapy in PI3K/AKT/PTEN pathway-altered patients with HR+/HER2- ABC from a U.S. payer's perspective.

MATERIALS AND METHODS:

A partitioned survival model with three health states (progression-free, progressive disease, and death) was developed over a 5-year time horizon. Progression-free survival (PFS) and overall survival (OS) data were derived from the CAPItello-291 trial (NCT04305496). Utility, disutility, and direct medical costs were obtained from published literature and the Centers for Medicare and Medicaid Services. Capivasertib cost was based on wholesale acquisition cost. Costs were presented in 2025 U.S. dollars. Both costs and outcomes were discounted by 3%. Incremental cost-effectiveness ratios (ICERs) based on quality-adjusted life-year (QALY) gained and equal value of life years gained (evLYG) were compared using a willingness-to-pay (WTP) threshold of $150,000. Sensitivity analyses, scenario analyses (10-year horizon and phase II trial data) and threshold analysis were conducted.

RESULTS:

Capivasertib plus fulvestrant yielded more QALYs (1.89 vs. 1.29) and life years (3.31 vs. 2.49) but higher costs ($539,625 vs. $158,679) than fulvestrant alone, resulting in ICERs of $636,455/QALY gained and $459,358/evLYG over a 5-year time horizon. The cost of capivasertib ($28,332/4 weeks) had the greatest impact and would need to be reduced to $5,598/4 weeks to achieve cost-effectiveness.

LIMITATIONS:

Extrapolation of survival data reflects limited long-term trial data and increases uncertainty surrounding model parameters. Efficacy based on clinical trials may differ from real-world effectiveness.

CONCLUSIONS:

Despite clinical benefits, capivasertib plus fulvestrant was not cost effective at current price from a U.S. payer's perspective. The cost of capivasertib per 4 weeks would be required to decrease by ∼80% from $28,332 to $5,598 (about $87 per 200 mg) to meet the WTP threshold of $150,000.

2026-12-01·JOURNAL OF COMPUTER-AIDED MOLECULAR DESIGN

AI-driven peptide discovery for endometrial cancer: deep generative modeling and molecular simulation in the big data era

Article

作者: Meng, Yuxuan ; Fatima, Israr ; Ur Rehman, Hafeez ; Aldaw, Mohamed ; Rehman, Abdur ; Wang, Zhibo ; Li, Yan ; Liao, Mingzhi ; Warraich, Dawood Ahmed

The integration of artificial intelligence (AI) with molecular modeling offers new opportunities to accelerate therapeutic discovery. In this study, we developed an AI-driven generative pipeline combining deep reinforcement learning (DRL), generative adversarial networks (GANs), and variational autoencoders (VAEs) to design novel peptide-like molecules targeting major proteins implicated in endometrial cancer (EC), including AKT1, ESR1, Connexin-43, and CTNNB1. From over 14,200 generated structures, approximately 2313 peptides met drug-likeness and structural criteria and were screened using deep learning-enhanced docking. Top-ranked peptides, such as Gitoxoside (- 11.53 kcal/mol) and 9-Fluoro-11 (- 11.38 kcal/mol), demonstrated stronger binding to AKT1 than the reference inhibitor Capivasertib (- 8.50 kcal/mol). Similar high-affinity interactions were observed for CTNNB1-SCHEMBL (- 12.33 kcal/mol) and ESR1-1Estra-1,3 (- 11.05 kcal/mol). Molecular dynamics (MD) simulations confirmed the stability of these complexes with RMSD values below 2.5 Å and minimal residue fluctuations. WaterSwap free energy calculations yielded highly favorable binding energies (- 34 to - 37 kcal/mol), further validating stable ligand-protein interactions. ADMET predictions indicated acceptable pharmacokinetic properties and low predicted toxicity for most candidates. Collectively, this integrative AI framework efficiently explores peptide chemical space, enabling the rapid identification of peptide-based and peptidomimetic inhibitors with strong binding affinity and stability. The findings highlight the potential of AI-assisted peptide design as a scalable and cost-effective strategy for developing next-generation therapeutics against endometrial cancer.

769

项与卡匹色替相关的新闻（医药）

2026-05-29

·医药速览

股价上涨10倍！明星AI制药跌下神坛后，终于杀回来了

过去几年，AI制药行业经历了一轮剧烈的估值起伏。 Relay Therapeutics曾是最受关注的明星公司之一。这家由知名对冲基金D.E.Shaw创始人DavidE.Shaw创办的AI药物发现公司，凭借其“蛋白质动态构象预测”技术路线，获得大量市场关注。但随后，公司核心管线数据不及预期、合作终止、持续裁员，市场对于AI制药平台价值的质疑也随之而来，股价一度跌至低位。这一年来，公司股价强势修复，从最低1.77美元，一路走高到近期最高17.32美元，股价上涨约10倍，不过近期有所回落。其中最重要的是核心药物zovegalisib（RLY-2608）临床试验中数据转正，中期实验数据得到市场逐步的认可。近期RLY-2608关于血管畸形II期数据发布后，公司又趁热打铁融了2.75亿美元，账上现金流充足。 Relay的故事，也折射出AI制药行业正在发生的变化，对于AI biotech而言，真正决定公司价值的，依然是药物本身。转机仍然蕴藏着危机，等待着Relay的，还有更大的关要闯。曾经的至暗时刻药物失败，又被罗氏退货 Relay Therapeutics成立于2016年，总部位于美国马赛诸塞州，是一家以分子动力学模拟见长的AI药物发现公司。该公司由著名对冲基金大佬David E. Shaw 博士创办，他也是D.E. Shaw Research的创始人兼首席科学家。 Relay上市时因 “蛋白质动态预测+难成药靶点”故事，股价一路走高，最高冲到了64美元。然而此后，公司接连遭遇了一系列打击。首先是2023年，Relay在AACR2023会议上披露了其核心管线选择性PI3Kα抑制剂RLY-2608的临床数据有效性不佳。在27名患有PIK3CA突变型乳腺癌的受试者中，只有一名患者出现部分缓解（PR），而且这一点尚未得到证实，因为该患者曾经接受过12线治疗。随后公司调整管线，又暂停了一款CDK2抑制剂的开发。紧接着，2024年7月，罗氏旗下基因泰克终止其与SHP2变构抑制剂RLY-1971的合作，将权益退还给Relay。此前，基因泰克与Relay Therapeutics达成协议，获得Relay旗下的SHP2抑制剂RLY-1971的开发和商业化权益。Relay获得7500万美元的预付款，近期付款2500万美元和里程碑付款6.95亿美元，总价值为7.95亿美元。这意味着Relay失去重要外部管线以及后续的里程碑收入，AI药物对外授权变现的故事也讲不下去了。一连串的打击，让Relay Therapeutics受到了外界的质疑，尤其是对其AI平台和长期生存能力的担忧。公司更是在2024年到2025年间进行多次裁员，2024年在7月，裁员约5%；紧接着，10月又有30人被裁，约占员工的10%，2025年裁员70人。公司还选择剥离其他资产。 2024年底，Relay宣布将一款FGFR2抑制剂lirafugratinib（RLY-4008）的全球权益授权给Elevar Therapeutics。在这笔交易中，Relay获得 7500万美元的预付款，以及未来4.25亿美元的潜在商业里程碑+10%的分层特许权使用费。靠一款药物，翻身了但与此同时，Relay临床管线就仅剩zovegalisib(RLY-2608)，这是一款PI3Kα突变选择性抑制剂。当时，RLY-2608 几乎成了Relay的“全部身家”，如果不能成功，将严重动摇了公司的股价基石。 PI3Kα 是PI3K家族中作为癌症治疗的主要靶点，它的高表达与结肠癌、乳腺癌、前列腺癌、胃癌、直肠癌、卵巢癌等肿瘤的发生发展密切相关。尤其是在HR+/HER2-乳腺癌中，约40%的患者都存在PIK3CA突变，是重要的治疗靶点。由于副作用和耐受性等情况，PI3K抑制剂疗效以及安全性一直是难题。在治疗乳腺癌方面，诺华已上市的PI3Kα抑制alpelisib就存在高血糖、肺部问题、过敏反应、皮肤问题、腹泻等副作用。而RLY-2608作为首个口服泛突变选择性PI13Ka抑制剂，能够降低毒性，以及早期疗效数据，具备同类最佳（BIC）的潜力。公司正在RLY-2608全力推进血管畸形与乳腺癌双适应症开发。 RLY-2608成为全球首个在血管畸形（VA）中显示出显著疗效与安全性的PI3Kα选择性抑制剂。近期该药物二期临床数据亮眼：20 名可评估患者中60%病灶体积缩小，低剂量组缓解率也达 62%，既往用药患者缓解率同样为62%。95%患者病灶均有缩小、无人病情进展；同时患者症状、疼痛及生活质量均得到明显改善。不过血管畸形是个罕见病，美国每年新发患者大概17万人，核心开发场景还是乳腺癌。在乳腺癌领域，zovegalisib联合氟维司群治疗PIK3CA突变、CDK4/6抑制剂经治的HR+/HER2-晚期乳腺癌的3期试验正在开展，且已获FDA突破性疗法认定。这项研究直接头对头对比获批不久的“Capivasertib+氟维司群”，非常值得期待。挑战继续目前股价回升，属于明显的阶段性估值修复。随着核心药物临床数据表现亮眼，叠加账面资金充裕、管线规划明确，公司重新回归到正常biotech的估值区间。但如今，公司的命运与RLY-2608紧紧地绑定在一起。因为公司股价只依赖于单一核心资产，但这也意味着一旦这项产品失败，容易遭受重挫。如今的股价已经反映了市场大部分乐观情绪，后续想要继续上涨，只能依靠实打实的临床数据，无法再单纯依靠AI概念驱动。作为AI+新药研发长期的行业研究和观察者，在我看来，Relay的故事和当下火热的AI制药公司版本有些不一样了。尤其是，Relay 的核心叙事是MBDD（基于运动的药物设计），即突破传统静态靶点思维，用分子动力学模拟蛋白质“动态构象”，精准发现变构位点，攻克“不可成药靶点”。但如今，新一代公司如Chai Discovery正在利用生成式AI，直接零样本直接生成抗体等大分子。同时在商业模式上作为技术核心提供服务，为礼来等大药企构建定制模型，不依赖自研管线。两种路线没有绝对的优劣，却折射出AI制药行业当下的发展分野。早期行业普遍依靠前沿技术概念推高估值，自研药物的AI biotech最终要接受医药行业临床为王的铁律。主打技术服务与平台的AI企业，则凭借灵活的商业模式走出另一条道路，但也意味着技术迭代和开拓客户的难题。不过，从Relay的例子可以看出，公司可以趁着市场接受度良好的时候，多进行融资。从历史看，Relay几乎是每出一次关键阳性数据，就紧跟着一次大额融资，助力其跨越低谷。近期，Relay公布关于血管畸形2期优秀数据后，公司立即完成约2.75亿美元的增发。截至2026年Q1，公司账面现金约7亿美元，能够支撑到2029年。推文用于传递知识，如因版权等有疑问，请于本文刊发30日内联系医药速览。原创内容未经授权，禁止转载至其他平台。 ©2021 医药速览保留所有权利往期链接 “小小疫苗”养成记 | 医药公司管线盘点人人学懂免疫学| 人人学懂免疫学（语音版）综述文章解读 | 文献略读 | 医学科普|医药前沿笔记 PROTAC技术| 抗体药物| 抗体药物偶联-ADC 核酸疫苗 | CAR技术| 化学生物学温馨提示医药速览公众号目前已经有近12个交流群（好学，有趣且奔波于医药圈人才聚集于此）。进群请扫描上方二维码，备注“姓名/昵称-企业/高校-具体研究领域/专业”，此群仅为科研交流群，非诚勿扰。简单操作即可星标⭐️医药速览，第一时间收到我们的推送 ①点击标题下方“医药速览” ②至右上角“...” ③点击“设为星标

2026-05-29

·峰秀生物

neladalkib NDA受理，GIST与PBC优先审评推进

峰秀生物 | 2026-05-29 生物医药行业日报 📅 2026-05-29 | ⏱️ 过去 48 小时 🎯 专注生物创新药 · 重大事件 · 每日精选今日引语：凡事预则立，不预则废。——《礼记·中庸》今日重点 1. Nuvalent的neladalkib用于ALK阳性非小细胞肺癌NDA获FDA优先审评，PDUFA日期为2026-11-27，后线耐药和颅内活性是注册看点。 2. Durvalumab联合BCG获FDA批准用于BCG初治高危非肌层浸润性膀胱癌，POTOMAC III期研究显示DFS改善，HR为0.68。 3. Cogent的bezuclastinib联合sunitinib用于GIST的NDA获FDA优先审评，PEAK III期mPFS为16.5个月对9.2个月，ORR为46%对26%。 4. Menarini披露elacestrant联合capivasertib在ER阳性/HER2阴性转移性乳腺癌ELEVATE研究数据，重点指向ESR1与PIK3CA共突变患者。 5. Zydus的saroglitazar用于原发性胆汁性胆管炎获FDA优先审评，EPICS-III研究52周复合生化应答率为56.7%对9.8%。 🛡️ 自免·炎症 1）Saroglitazar用于原发性胆汁性胆管炎获FDA优先审评事件与核心事实：Zydus的saroglitazar用于PBC（原发性胆汁性胆管炎，primary biliary cholangitis）的NDA获得FDA优先审评，目标用于UDCA应答不足或不耐受成人患者。EPICS-III数据显示，52周复合生化应答率为56.7%对9.8%，并观察到ALP等胆汁淤积相关指标改善。行业判断：PBC项目提醒研发团队，罕见和慢性免疫相关疾病的价值往往先由可靠替代终点建立，再等待更长期临床结局验证。对国内自免和肝病项目而言，生化终点、瘙痒等症状终点、安全性和真实世界长期管理必须同步规划，而不是等注册前再补证据。来源：Zydus / HCPLive，事件日期：2026-05-28 / 来源发布日期：2026-05-28 🎯肿瘤免疫 2）Durvalumab联合BCG获批用于高危非肌层浸润性膀胱癌事件与核心事实：FDA批准durvalumab联合BCG（卡介苗，Bacillus Calmette-Guerin）用于BCG初治、高危NMIBC（非肌层浸润性膀胱癌，non-muscle-invasive bladder cancer）成人患者。批准依据POTOMAC III期研究，联合方案相较BCG单独治疗显著改善DFS（无病生存期，disease-free survival），HR为0.68，且中位DFS两组均未达到。行业判断：膀胱癌局部治疗正在从单纯灌注标准疗法走向免疫组合强化，但这并不意味着所有高危患者都应无差别加用免疫治疗。真正的开发重点在于找到复发风险、CIS比例、毒性承受能力和治疗依从性之间的平衡，避免把统计获益机械外推到低风险人群。来源：FDA / CancerNetwork，事件日期：2026-05-28 / 来源发布日期：2026-05-28 3）Elacestrant联合capivasertib在ER阳性乳腺癌披露ELEVATE数据事件与核心事实：Menarini披露ELEVATE 1b/2期研究中elacestrant联合capivasertib用于ER（雌激素受体，estrogen receptor）阳性、HER2（人表皮生长因子受体2，human epidermal growth factor receptor 2）阴性mBC（转移性乳腺癌，metastatic breast cancer）的安全性和初步疗效。RP2D队列显示DCR、CBR24和ORR信号，并特别指向ESR1与PIK3CA共突变患者的联合治疗潜力。行业判断：乳腺癌内分泌耐药正在从“换一个内分泌药”转向以基因突变和通路补偿为基础的组合设计。该类项目后续能否成立，关键取决于突变分层是否足够清楚、联合毒性是否可长期管理，以及早期缓解信号能否转化为稳定的PFS获益。来源：Menarini Group / GlobeNewswire，事件日期：2026-05-28 / 来源发布日期：2026-05-28 📊 转化评价 / 监管与临床策略 4）Neladalkib用于ALK阳性非小细胞肺癌获FDA优先审评事件与核心事实：Nuvalent的neladalkib针对ALK（间变性淋巴瘤激酶，anaplastic lymphoma kinase）阳性NSCLC（非小细胞肺癌，non-small cell lung cancer）获FDA（美国食品药品监督管理局）NDA（新药上市申请，new drug application）优先审评，PDUFA目标日期为2026-11-27。申报主要基于ALKOVE-1研究中TKI经治人群的缓解和颅内活性数据，项目进入商业化准备和一线扩展并行阶段。行业判断：这类事件的价值不只在“又一个ALK抑制剂进入审评”，而在于它把耐药后线、颅内病灶和TKI序贯策略同时放进注册证据链。对国内靶向药项目而言，下一阶段竞争不再只是靶点选择，而是能否把患者分层、脑转移活性、剂量耐受和审评节奏提前设计成可验证路径。来源：CancerNetwork / Nuvalent，事件日期：2026-05-27 / 来源发布日期：2026-05-28 5）Bezuclastinib联合sunitinib用于GIST获FDA优先审评事件与核心事实：Cogent宣布FDA受理bezuclastinib联合sunitinib用于既往接受imatinib治疗后GIST（胃肠道间质瘤，gastrointestinal stromal tumor）患者的NDA，并授予优先审评，PDUFA日期为2026-11-30。PEAK III期数据显示，组合治疗mPFS（中位无进展生存期，median progression-free survival）为16.5个月，对照为9.2个月；ORR（客观缓解率，objective response rate）为46%对26%。行业判断：GIST二线治疗长期缺乏明确突破，bezuclastinib组合的看点在于用互补KIT突变覆盖来解释临床获益，而不是单纯叠加两个TKI。对精准治疗项目来说，突变谱、耐药机制和组合毒性必须在III期前形成闭环，否则进入注册阶段后很难支撑清晰的标签和用药顺序。来源：Cogent Biosciences / GlobeNewswire，事件日期：2026-05-28 / 来源发布日期：2026-05-28 峰秀观察今天的主线不是某个单一靶点突然升温，而是多个项目同时进入“证据能不能支撑下一步”的检验场。ALK阳性肺癌和GIST项目把耐药机制、患者分层和注册节奏推到台前；NMIBC和PBC则把治疗获益放回更具体的疾病场景、终点选择和长期安全管理中判断。从转化科学角度看，项目推进越来越依赖一条可追溯的证据链：机制假设要能解释患者筛选，早期疗效要能连接关键终点，安全窗要能支撑真实使用场景，监管沟通要能反映标签边界。没有这条链条，单点数据再亮，也很难转化为稳定的注册和商业化价值。对国内药企和项目负责人来说，今天这些事件的启发是：不要把“进入临床”或“获得数据”当成终点，而要把每个节点都设计成下一次决策的输入。能不能继续投入、是否需要调整适应症、组合是否值得放大、患者分层是否足够清楚，应该在项目早期就被当作管理问题来回答。情报说明时间窗：过去 48 小时（2026-05-27 至 2026-05-29）覆盖主题：肿瘤精准治疗、肿瘤免疫、乳腺癌联合治疗、自免炎症、监管与临床策略来源说明：本稿基于 5 条精选事件成稿，正文事件段落不使用外链。完整来源网址如下： 1. CancerNetwork / Nuvalent：https://www.cancernetwork.com/view/neladalkib-earns-fda-priority-review-in-alk-positive-nsclc 2. FDA / CancerNetwork：https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-durvalumab-combination-bacillus-calmette-guerin-high-risk-non-muscle-invasive-bladder 3. Cogent Biosciences / GlobeNewswire：https://www.globenewswire.com/news-release/2026/05/28/3302753/0/en/cogent-biosciences-announces-fda-acceptance-of-new-drug-application-nda-with-priority-review-for-bezuclastinib-in-combination-with-sunitinib-for-patients-with-gist.html 4. Menarini Group / GlobeNewswire：https://www.globenewswire.com/news-release/2026/05/28/3302984/21662/en/Menarini-Group-Presents-New-Data-from-the-Phase-1b-2-ELEVATE-Study-of-Elacestrant-ORSERDU-in-Combination-with-Capivasertib-in-Patients-with-ER-HER2-Metastatic-Breast-Cancer-mBC-at-.html 5. Zydus / HCPLive：https://www.hcplive.com/view/fda-grants-priority-review-to-saroglitazar-nda-for-primary-biliary-cholangitis 免责声明：本情报仅供行业参考，不构成投资建议或临床用药建议。报告生成时间：2026-05-29 订阅提醒：关注「峰秀生物」获取最新动态

2026-05-29

·BioSpace

FDA delays verdict on Beren’s rare disease drug by three months

iStock, Nauval Wildani Beren Therapeutics is seeking approval in Neimann-Pick disease type C, an application the FDA was set to decide on by August 17. The new target action date is November 17. The FDA has pushed back its decision date for Beren Therapeutics’ intrathecal injection for infantile-onset Niemann-Pick disease type C. The regulator accepted Beren’s data package for priority review in February, giving an initial target action date of August 17. Then, in March, Beren filed additional data and documentation in response to a previous FDA request. The agency considered this submission to be a major adjustment to the original drug application, necessitating an extension, according to the company’s release on Thursday . The new decision data is November 17. Niemann-Pick disease type C (NPC) is a rare and severe neurodegenerative disorder that arises from pathogenic mutations to the NPC1 or NPC2 genes, impairing the movement of cholesterol molecules inside cells. Patients with NPC suffer from progressive neurological decline and early death. Beren’s answer to NPC is adrabetadex, a proprietary mixture of various isomers of the oligomer 2-hydroxypropyl-β-cyclodextrin. The drug candidate, according to the company, can restore cholesterol trafficking in the cells. Beren is specifically proposing adrabetadex for infantile-onset NPC, which refers to cases that manifest in children younger than 6 years of age. This NPC subtype is typically linked to more rapid disease progression and worse prognosis, according to the biotech. The drug elicited a 71% reduction in the risk of death versus external controls, Beren said in its February news release , noting that this effect was highly statistically significant. The FDA in December last year granted adrabetadex its Breakthrough Therapy designation . If approved, adrabetadex will share the NPC market with Zevra Therapeutics’ Miplyffa, which became the first-ever drug cleared for the indication in September 2024, and IntraBio’s Aqneursa, which followed just days later . Azafaros is also advancing an NPC therapy dubbed nizubaglustat. The molecule entered late-stage development last July. Beren’s delay comes just days after the FDA similarly extended its review of AstraZeneca’s breast cancer pill camizestrant, citing the need for more time to review additional submissions. The pharma is developing camizestrant, in combination with the AKT blocker Truqap, as a first-line therapy for patients with HER2-negative, HR-positive, ESR1-mutated breast cancer. It is unclear whether the exits of FDA Commissioner Marty Makary and several other senior leaders in recent weeks contributed to the delays. Regulatory Makary’s reforms will live on at FDA even as leadership turns over Under the temporary reign of top food executive Kyle Diamantas, the FDA will sustain programs initiated by former Commissioner Marty Makary, including the Commissioner’s National Priority Voucher initiative. May 27, 2026 · 2 min read · Tristan Manalac Read more

优先审批突破性疗法加速审批

100 项与卡匹色替相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11371	-	-	DB12218

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
ER阳性/HER2阴性乳腺癌	欧盟	AstraZeneca AB	2024-06-17
ER阳性/HER2阴性乳腺癌	冰岛	AstraZeneca AB	2024-06-17
ER阳性/HER2阴性乳腺癌	列支敦士登	AstraZeneca AB	2024-06-17
ER阳性/HER2阴性乳腺癌	挪威	AstraZeneca AB	2024-06-17
乳腺癌	加拿大	AstraZeneca Canada, Inc.	2024-01-26
HR阳性/HER2阴性乳腺癌	美国	AstraZeneca Pharmaceuticals LP	2023-11-16

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
去势敏感前列腺癌	申请上市	美国	AstraZeneca PLC	2025-08-01
HER2 阴性乳腺癌	临床3期	比利时	AstraZeneca PLC	2025-11-27
HER2 阴性乳腺癌	临床3期	德国	AstraZeneca PLC	2025-11-27
HER2 阴性乳腺癌	临床3期	葡萄牙	AstraZeneca PLC	2025-11-27
HR阳性乳腺癌	临床3期	比利时	AstraZeneca PLC	2025-11-27
HR阳性乳腺癌	临床3期	德国	AstraZeneca PLC	2025-11-27
HR阳性乳腺癌	临床3期	葡萄牙	AstraZeneca PLC	2025-11-27
转移性乳腺癌	临床3期	比利时	AstraZeneca PLC	2025-11-27
转移性乳腺癌	临床3期	德国	AstraZeneca PLC	2025-11-27
转移性乳腺癌	临床3期	葡萄牙	AstraZeneca PLC	2025-11-27

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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


AACR2026	临床2/3期	HR阳性/HER2阴性乳腺癌 HR Positive \| HER2 Negative	16	Imlunestrant alone	憲淵蓋淵襯顧網衊鑰網(選築簾壓範艱願築窪簾) = 淵餘夢糧遞壓網憲選醖襯鏇鹽襯繭鑰築壓繭齋 (選壓選壓餘觸鏇壓構夢 ) 更多	积极	2026-04-21
				Camizestrant 75mg	憲淵蓋淵襯顧網衊鑰網(糧憲艱積蓋選鏇築積衊) = 鬱築醖窪選鹽築遞廠襯鑰顧襯壓繭製繭願糧齋 (積齋願構餘壓齋糧積選 ) 更多
NCT04862663 (CAPItello-292, SABCS2025)	-	HR阳性/HER2阴性乳腺癌 HR+ \| HER2-	48	Capivasertib + CDK4/6i + fulvestrant	製夢夢憲繭鏇積選夢壓(廠壓壓鑰鬱鬱網顧衊鑰) = 鏇憲壓壓淵醖遞鬱齋願鑰獵齋糧壓襯鏇壓鬱築 (憲鑰廠構網鏇艱壓遞選 ) 更多	积极	2025-12-11
SABCS2025	N/A	HR阳性乳腺癌 PIK3CA \| ESR1	107	alpelisib+fulvestrant	齋廠顧網築製憲鬱鬱夢(廠獵衊構夢膚窪顧蓋構) = One patient with alpelisib experienced treatment interruption and dose reduction due to skin toxicity, while Elacestrant was well-tolerated without major adverse effects. 鏇鬱齋選廠積網顧夢顧 (築網鬱鑰獵遞蓋鏇壓積 )	积极	2025-12-10
				capivasertib+fulvestrant
SABCS2025	N/A	HR阳性/HER2阴性乳腺癌 HR+ \| HER2- \| PI3K/AKT/PTEN pathway alterations	72	≥2 pathway inhibitors sequentially	鹹鏇醖築艱鹹鬱蓋築衊(觸顧餘鹽憲積網窪廠網) = 窪選鹽繭簾觸選遞築積顧獵積鑰衊鏇鏇積構壓 (繭構襯窪積夢糧衊憲鹽, 9 ~ 28)	积极	2025-12-10
				alpelisib alone	鹹鏇醖築艱鹹鬱蓋築衊(觸顧餘鹽憲積網窪廠網) = 窪膚製襯壓夢膚構夢窪顧獵積鑰衊鏇鏇積構壓 (繭構襯窪積夢糧衊憲鹽, 5 ~ 8)
NCT03997123 (CAPItello-290, ESMO_ASIA2025)	临床3期	转移性三阴性乳腺癌一线 PIK3CA \| AKT1 \| PTEN	128	Capivasertib + paclitaxel	鏇鬱獵壓膚艱糧簾壓繭(膚選餘範製齋製築壓遞) = 艱遞窪廠窪壓膚窪鑰網蓋選襯壓選廠繭餘製襯 (網積醖遞膚鑰廠夢廠蓋 ) 更多	积极	2025-12-06
				Placebo + paclitaxel	鏇鬱獵壓膚艱糧簾壓繭(膚選餘範製齋製築壓遞) = 艱構鑰積齋衊觸鬱鏇積蓋選襯壓選廠繭餘製襯 (網積醖遞膚鑰廠夢廠蓋 ) 更多
NCT03801369 (AMTEC, CTgov)	临床2期	转移性乳腺癌 \| 转移性三阴性乳腺癌	24	Quality-of-Life Assessment+Olaparib+Durvalumab (Arm I (Olaparib, Durvalumab))	願艱願網齋鹹醖選廠積 = 襯選糧蓋淵鏇齋網窪網積夢鹹淵範壓遞夢選糧 (衊艱壓淵廠衊糧廠範鑰, 鹽醖衊積範憲積製憲築 ~ 顧廠簾膚鹽製醖繭獵淵) 更多	-	2025-11-21
				Quality-of-Life Assessment+Olaparib+selumetinib (Arm II (Olaparib, Selumetinib))	窪淵齋窪製網憲窪艱壓(選憲獵醖鹽觸製膚蓋鏇) = 網願鏇製網衊糧鹽鑰鹹蓋範簾鏇獵鏇窪選膚觸 (構夢醖鬱夢簾鹽選構壓, 顧繭夢糧蓋願遞艱鏇膚 ~ 築鬱壓獵觸壓鹹壓憲鹽) 更多
NCT03660826 (NRG GY012, ESMO2025)	临床2期	转移性子宫内膜恶性肿瘤	168	Olaparib 300 mg PO BID + Durvalumab 1500mg q28 days	憲範築鬱觸艱簾積膚餘(顧廠願齋餘膚淵餘艱窪) = 獵繭製繭窪憲壓構遞鑰構遞鏇選齋餘蓋觸淵積 (簾觸觸憲繭壓構艱觸築 ) 更多	不佳	2025-10-17
				Cediranib 30 mg PO Daily	憲範築鬱觸艱簾積膚餘(顧廠願齋餘膚淵餘艱窪) = 鑰願構夢淵窪窪淵壓艱構遞鏇選齋餘蓋觸淵積 (簾觸觸憲繭壓構艱觸築 ) 更多
ESMO2025	N/A	晚期乳腺癌 HR+ \| HER2-	16	Camizestrant	-	积极	2025-10-17
				Ribociclib plus ET
NCT04742036 (Pubmed) 人工标引	临床1期	晚期恶性实体瘤	16	Capivasertib (Part A)	遞醖網鬱簾鹽範積鏇製(鹹襯構蓋壓網繭製醖艱) = Hyperglycemia, diarrhea, and rash were the most common. Most adverse events were Grade 1-2. 遞鹹夢範糧觸醖憲糧獵 (壓淵夢壓鏇餘範願壓糧 )	积极	2025-10-14
				Capivasertib + Paclitaxel (Part B)
CAPItello-291 (ASCO2025)	N/A	HR阳性/HER2阴性乳腺癌 PIK3CA \| AKT \| PTEN ... 更多	34	Capivasertib	齋蓋憲鏇獵構遞夢廠鹹(廠鹹淵鏇餘鹹鹽憲範壓) = 淵鹽艱繭艱願鹹鑰遞觸艱膚鏇蓋鏇壓憲築觸鑰 (鏇襯獵網鑰簾遞網鑰廠, 2.4 ~ 7.6) 更多	积极	2025-05-30
				Alpelisib	-