Mirdametinib(Mirdametinib) - 药物靶点：MEK1 x MEK2_在研适应症：神经纤维瘤病1型，NF1突变型丛状神经纤维瘤，去分化脂肪肉瘤_专利_临床

概要

基本信息

药物类型

小分子化药

别名

Mirdametinib (USAN)、PD 901、PD-0325901

+ [3]

靶点

MEK1 x MEK2

作用方式

抑制剂

作用机制

MEK1抑制剂(双特异性丝裂原活化蛋白激酶激酶1抑制剂)、MEK2抑制剂(双特异性丝裂原活化蛋白激酶激酶2抑制剂)

治疗领域

肿瘤神经系统疾病遗传病与畸形+ [6]

在研适应症

神经纤维瘤病1型NF1突变型丛状神经纤维瘤去分化脂肪肉瘤+ [11]

非在研适应症

晚期乳腺癌晚期癌症晚期非小细胞肺癌+ [4]

原研机构

Springworks Therapeutics, Inc.

在研机构

BeOne Medicines Ltd.

Springworks Therapeutics, Inc.SPRINGWORKS THERAPEUTICS IRELAND LTD

非在研机构

Pfizer Inc.

权益机构

SpringWorks Therapeutics LLCMerck KGaA

Springworks Therapeutics, Inc.

+ [2]

最高研发阶段批准上市

首次获批日期

美国 (2025-02-11),

神经纤维瘤病1型

最高研发阶段(中国)-

特殊审评优先审评 (美国)、快速通道 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、罕见儿科疾病 (美国)

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结构/序列

分子式C16H14F3IN2O4

InChIKeySUDAHWBOROXANE-SECBINFHSA-N

CAS号391210-10-9

关联

24

项与 Mirdametinib 相关的临床试验

NCT06876142

/ Recruiting临床1/2期IIT

A Phase 1b/2 Study of Combination Therapy (Mirdametinib and Sirolimus) for RAS Mutated Relapsed Refractory Multiple Myeloma (RRMM)

Background:
Multiple myeloma (MM) is a type of blood cancer that affects a person s immunity. MM returns after treatment (relapse) in almost all people; MM may also not respond to initial treatment (refractory). Many people with relapsed refractory MM (RRMM) also have changes in their KRAS and NRAS genes. Researchers want to try a new drug treatment that targets cancer with these changed genes.
Objective:
To test 2 drugs (mirdametinib and sirolimus) in people with RRMM.
Eligibility:
People aged 18 and older with RRMM who have changes in their KRAS or NRAS genes.
Design:
Participants will be screened. They will have blood tests and imaging scans. They will have an eye exam and a test of their heart function. They will need to provide proof of their disease status and of their KRAS or NRAS status. If neither is available, the tests will be repeated.
Participants will have a bone marrow biopsy: A needle will be inserted into a hipbone to draw out some soft tissue.
This study will be done in two parts. In the first part of this study, we will find a safe dose of mirdametinib combined with sirolimus. In the second part, we will learn more about how mirdametinib combined with sirolimus may work against RRMM.
Mirdametinib (capsules) and sirolimus (tablets) are taken by mouth. Participants will take both drugs at home on a 4-week cycle. They will take mirdametinib twice a day for the first 3 weeks of each cycle. They will take sirolimus once a day, every day, during each cycle.
Participants will have study visits once a week during the first cycle, and then on the first day of subsequent cycles. Blood, heart, imaging scans, and other tests will be repeated.
Treatment with the study drugs will go on for 1 year. Then participants will have follow-up visits every 3 months for 4 more years.

开始日期2025-07-09

申办/合作机构

National Cancer Institute

NCT06997276

/ Recruiting临床1期

A Phase 1 Open-Label Study to Assess the Pharmacokinetics of Mirdametinib and Its Metabolite PD-0315209 in Participants With Impaired Hepatic Function and Participants With Normal Hepatic Function

The purposes of this study are to determine:
* The pharmacokinetics (the amount of study drug in your blood and how long it takes the body to get rid of it) of the study drug and its metabolites (substances produced as the body breaks down the study drug) in participants with moderate or severe liver function impairment compared to participants with normal liver function (also known as a healthy volunteer). Pharmacokinetics (or PK) is the study of how your body absorbs, breaks down, and removes a study drug.
* How well the study drug is tolerated and any side effects that may occur in participants with moderate or severe liver function impairment compared to participants with normal liver function.
This study is for research purposes only and is not intended to treat any medical condition.

开始日期2025-05-07

申办/合作机构

Springworks Therapeutics, Inc.

NCT06666348

/ Not yet recruiting临床1/2期

A Phase 1/2 Study of Mirdametinib and Vinblastine for Newly Diagnosed or Previously Untreated Patients With Pediatric Low-grade Glioma and Activation of the MAPK Pathway

This is a 3-part open-label study (feasibility phase, treatment phase and follow-up phase) of orally administered mirdametinib in combination with intravenous vinblastine chemotherapy in patients with PLGG with activation of MAPK pathway.
Feasibility Phase:
The maximum tolerated/recommended phase 2 dose (MTD/RP2D) of the mirdametinib plus vinblastine combination will be assessed using a modified Rolling-6 design.
Treatment Phase:
Patients will receive mirdametinib twice daily (continuously) at a fixed dose (2mg/m2 po BID up to 4 mg BID) for a total of 13 cycles (28 days cycle). Weekly intravenous vinblastine at MTD will be given for a total of 17 cycles. If adverse events occur, two dose reductions are allowed.
Follow-up Phase:
Following the end of treatment, patients will be scheduled for a follow-up visit every 6 months for 36 months to evaluate PFS, TTP and OS.

开始日期2025-04-01

申办/合作机构

C17 Council

100 项与 Mirdametinib 相关的临床结果

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100 项与 Mirdametinib 相关的转化医学

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100 项与 Mirdametinib 相关的专利（医药）

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988

项与 Mirdametinib 相关的文献（医药）

2025-08-01·GENE

Exploring the functional and prognostic roles of EPHX4 in pancreatic cancer: Insights from bioinformatics and experimental validation

Article

作者: Gu, Dandan ; Xiong, Wei ; Huang, Shaoyang

BACKGROUND:

Epoxide hydrolase-4 (EPHX4) belongs to the epoxide hydrolase enzyme family, but its biological function remains unclear, especially its potential involvement in the development of pancreatic tumours. This research sought to examine the function of EPHX4 in pancreatic adenocarcinoma (PAAD).

METHODS:

The expression of EPHX4 across cancers was examined through The Cancer Genome Atlas (TCGA). Bioinformatics was employed, leveraging multiple databases to investigate EPHX4 gene expression in pancreatic cancer and its association with survival prognosis, functional enrichment, immune infiltration, tumour mutation load, and drug sensitivity, among other variables. To evaluate EPHX4 expression in PAAD cells, Western blotting and reverse transcription quantitative PCR were used. A series of in vitro functional experiments was performed to assess the proliferation, migration, and invasion of PAAD cells.

RESULTS:

EPHX4 expression was markedly elevated in a number of malignancies, including PAAD, and was associated with patient sex, clinical stage, and metastasis to the lymph nodes. High EPHX4 expression was significantly associated with a worse outcome in PAAD patients. According to functional and enrichment studies, EPHX4 is involved in many signalling pathways linked to cancer. The study of immune infiltration revealed that EPHX4 was connected to the existence of a variety of immune cells inside the tumour. Our study revealed that EPHX4 knockdown significantly decreased PAAD cell migration, proliferation, and invasion.

CONCLUSION:

EPHX4 is highly expressed in PAAD and independently predicts poor survival. Functional experiments demonstrate its tumor-promoting effects. Its involvement in multiple cancer-related signaling pathways and immune regulation mechanisms highlights the dual prognostic and therapeutic potential of EPHX4 in PAAD.

2025-07-01·DEVELOPMENTAL BIOLOGY

Characterisation of the avascular mesenchyme during digit outgrowth

Article

作者: Batho-Samblas, Cameron ; Smith, Jonathan ; Keavey, Lois ; Clancy, Noah ; McTeir, Lynn ; Davey, Megan G

The avascular mesenchyme at the tip of the developing digit contributes to digit outgrowth and patterning, however, it has been poorly characterised. Using newly developed fate mapping approaches, tissue manipulation and single-cell mRNA sequencing data, we explore the transcriptional nature and developmental potential of this tissue. We find that the avascular mesenchyme is essential to normal segmental patterning of the digit and has a distinct transcriptional identity. In addition, we uncover an unexpected relationship between the unspecified tissue of the avascular mesenchyme and the committed phalanx forming region, which controls patterning, but not outgrowth of the digit. This multifaceted approach provides insights into the mechanics and genetic pathways that regulate digit outgrowth and patterning.

2025-06-01·Animal Bioscience

Key events in the process of sex determination and differentiation in early chicken embryos

Article

作者: Gong, Wei ; Li, Zeyu ; Sun, Hongyan ; Qian, Hongwu ; Liu, Xin ; Zuo, Qisheng ; Li, Bichun ; Han, Wei ; Song, Jiuzhou ; Liu, Guanzheng ; Chen, Guo Hong ; Niu, Yingjie ; Zhu, Xilin ; Lv, Xiaoqian ; Wu, Jun ; Jin, Kai ; Sun, Changhua

Objective: Current understanding of sex determination and differentiation mechanisms during early chicken embryonic development remains incomplete. To address this, we applied RNA sequencing to identify male-female expression differences at critical developmental stages (E0 blastocysts, E3.5-E6.5 genital ridges, E18.5 gonads), focusing on glycolysis, histone acetylation, and DNA methylation. This approach aims to unravel key regulatory mechanisms and advance developmental biology insights.Methods: We analyzed molecular mechanisms of chicken sex determination at key stages (E0 blastocysts, E3.5-E6.5 genital ridges, E18.5 gonads) using RNA sequencing. Glycolysis, histone acetylation, and DNA methylation levels were assessed in embryonic stem cells and chicken embryonic fibroblasts. E18.5 gonads were treated with glycolytic activators (SB431542 and PD0325901 [2i]), a DNA demethylation activator (Vitamin C [Vc]), or an inhibitors of histone acetylation modification (valproic acid [VPA]). Sex-related gene expression, hormone levels, and gonad morphology were evaluated to determine treatment effects.Results: Key findings revealed that sex differences emerged as early as the blastocyst stage, intensified with embryonic development and were marked by a surge in sexually dimorphic gene expression. Gene Ontology and Kyoto encyclopedia of genes and genomes analyses highlighted the pivotal roles of energy metabolism and epigenetic modification process during this critical period. 2i, VC, or VPA interventions targeting E18.5 embryo gonads, induced a remarkable transformation of ovarian tissue into a testis-like structure, characterized by cortical thinning, medulla densification, downregulation of female-specific genes (FOXL2, WNT4), upregulation of male-specific genes (SOX9, AMH), and increased testosterone secretion. This phenotypic and molecular shift underscores the ability of metabolic and epigenetic modulators to reprogram ovarian development towards a male-like pattern, preserving male sexual characteristics.Conclusion: Our study establishes energy metabolism and epigenetic regulation as central drivers of avian sex determination. These findings advance understanding of vertebrate developmental biology and provide a framework for dissecting regulatory networks in avian sexual development.

132

项与 Mirdametinib 相关的新闻（医药）

2025-07-02

·GBIHealth

医保局启动2025年医保谈判和商业健康创新药目录调整工作

药械追踪No.1 / 武田低IgA含量即用型免疫球蛋白GAMMAGARD LIQUID ERC在美获批2025年6月30日，武田宣布，GAMMAGARD LIQUID ERC（人免疫球蛋白输注液）获得美国食品药品监督管理局（FDA）批准，作为替代疗法用于治疗两岁及以上人群的原发性免疫缺陷。GAMMAGARD LIQUID ERC是一种即用型免疫球蛋白输注液，临床使用时无需复溶，可通过静脉或皮下给药，有望减轻用药负担。其10%溶液中IgA含量≤2µg/mL，是目前唯一一款低IgA含量的即用型免疫球蛋白溶液。GAMMAGARD LIQUID ERC预计将于2026年在美国商业上市，随后将于2027年在欧盟上市；其在欧盟已获得批准，商品名为DEQSIGA。->点击文末阅读原文，解锁完整双语新闻企业动态No.1 / 德国默克完成对SpringWorks的收购，对价34亿美元2025年7月2日，德国默克宣布完成对SpringWorks Therapeutics, Inc.（简称“SpringWorks”）的收购，对价34亿美元（约30亿欧元）。这是2025年迄今全球生物制药领域规模最大的并购交易之一。双方此前于2025年4月28日宣布达成最终协议。此次业务合并将助推默克的营收增长，并预计到2027年将提升默克的每股摊薄前收益（EPS pre）。SpringWorks的产品组合包含两款极具创新性的罕见肿瘤治疗药物。一款是Ogsiveo（nirogacestat），这是一种口服γ分泌酶抑制剂，也是FDA批准的首个成人硬纤维瘤治疗药物；另一款是Gomekli（mirdametinib），这是一种口服小分子MEK抑制剂，已获FDA批准用于治疗无法完全切除的患有1型神经纤维瘤病（NF1）且伴有症状性丛状神经纤维瘤（PN）的成人和儿童患者。Gomekli和Ogsiveo分别于2025年5月和6月获得欧洲药品管理局（EMA）人用药品委员会（CHMP）积极意见推荐批准。SpringWorks的产品组合将补充默克的罕见肿瘤业务管线，目前默克还拥有与和誉医药合作开发的腱鞘巨细胞瘤（TGCT）疗法pimicotinib，该产品已在中国提交上市申请。->点击文末阅读原文，解锁完整双语新闻No.2 / 镁信健康拟赴港交所上市近日，镁信健康递交招股书，拟赴香港联交所上市。镁信健康是目前中国最大的医药多元支付平台。公司致力于解决患者、保司与药企面临的筹资与支付挑战，助力推动中国医疗支付体系变革。公司拥有自研AI中枢mind42.ai、医疗供应链多元支付平台MediTrust Rx以及商保医师网络MediTrust Healthcare三大核心产品，依托AI技术为保司、药企及患者提供了智能化的解决方案。截至2024年12月31日，镁信健康已经累计服务了约160万名患者、超过90家保司以及超140家药企。公司2022~2024年业绩持续增长，年度收入分别为10.69亿元、12.55亿元和20.35亿元。->点击文末阅读原文，解锁完整双语新闻行业政策No.1 / 医保局启动2025年医保谈判和商业健康创新药目录调整工作2025年7月1日，国家医保局发布《2025年国家基本医疗保险、生育保险和工伤保险药品目录及商业健康保险创新药品目录调整工作方案（征求意见稿）》以及《2025年国家基本医疗保险、生育保险和工伤保险药品目录及商业健康保险创新药品目录调整申报指南（征求意见稿）》，并修订完善了《谈判药品续约规则》，于7月7日前向社会公开征求意见。2025年制定第一版商业健康保险创新药品目录，主要纳入超出保基本定位、暂时无法纳入基本目录，但创新程度高、临床价值大、患者获益显著的创新药，推荐商业健康保险、医疗互助等多层次医疗保障体系参考使用。满足以下条件之一的协议期内谈判药品，可以纳入常规目录管理：①非独家药品（以国家药监部门批准的同通用名药品数量为准），截至目录调整当年 6 月30 日（含），下同；②截至目录调整当年12月31 日，连续纳入目录“协议期内谈判药品部分”时间达到8年的药品。以2025年目录调整为例（截至2025年12月31日），2017年至2024年谈判新增的药品，纳入目录的时间分别为8年、7年、6年、5年、4年、3年、2年、1年。目录外新药申报进入医保谈判和目录内续约的规则与往年基本一致，其中条件①2020年1月1日（含，下同）至2025年6月30日（含，下同）期间，经国家药监部门批准上市的新通用名药品和⑤2025年6月30日前，经国家药监部门批准上市的罕见病治疗药品，可单独申报商保创新药目录或同时申报商保创新药目录、基本目录。参与商保创新药目录调整的专家主要通过相关部门、行业学协会、商业保险公司和省级医保部门、人力资源社会保障部门推荐。续约规则也与往年一致，从2025年起，调整支付范围的药品且连续在谈判药品目录中不够4年的独家药，药品销售额上限开始调整为往年的1.5倍，即纳入支付范围的药品费用预算年均增加值3亿元（含）以内的（往年为2亿元），按简易续约阶梯降价调整；年均增加值在3~15亿元（含）之间，支付标准的下调比例增加2%。年均增加值在15~30亿元（含）之间，支付标准的下调比例增加4%。年均增加值在30~60亿元（含）之间，支付标准的下调比例增加6%。年均增加值在60亿元以上的，支付标准的下调比例增加10%。截至目录调整当年12月31日，连续纳入目录“协议期内谈判药品部分”达到或超过4年的品种，其支付标准的下调比例在前述计算值基础上减半。->点击阅读原文，解锁完整双语新闻全球医疗情报领导者解锁隐藏在数据中的商业潜力关于 G B I”自从2002年成立以来，GBI始终以技术为驱动，为药企、器械及行业相关服务商提供贯穿生命周期的全球药品市场竞争数据、全球行业资讯、HCPs洞察、全国医疗器械数据等商业信息与洞察，助力企业在进行战略布局和决策时，脱颖而出。历经20余年的深耕细作GBI已成为95%以上跨国药企、国内头部药企、咨询与投资机构等医疗圈灯塔用户值得信赖的长期合作伙伴。联系我们投稿 | 发稿 | 媒体合作▶ olivia.xu@generalbiologic.com数据库 | 咨询服务 | 资讯追踪▶ 点击左下“阅读原文”完成表单填写点击阅读原文，解锁完整双语新闻

并购申请上市上市批准免疫疗法

2025-07-02

·Pharmaceutical Technology

Merck concludes SpringWorks acquisition for $3.4bn

Merck executive board chair and CEO Belén Garijo. Credit: Merck KGaA. Merck KGaA has concluded the previously announced acquisition of SpringWorks Therapeutics for an enterprise value of €3bn ($3.4bn), after receiving regulatory approvals and fulfilling customary closing conditions. The merger, announced in April 2025 , will positively impact Merck’s financial position by contributing to revenues immediately, and is anticipated to be accretive to its earnings per share by 2027. Merck will add two SpringWorks’ products targeting rare tumours with limited treatment options. Ogsiveo (nirogacestat) is approved by the Food and Drug Administration (FDA) for adults with progressing desmoid tumours requiring systemic treatment. The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) also recommended its approval in June 2025. Gomekli (mirdametinib) is approved by the FDA for neurofibromatosis type 1 (NF1)-related symptomatic plexiform neurofibromas (PN) in adults and children aged two years and above deemed unsuitable for complete resection. Mirdametinib also received a positive opinion from CHMP in May 2025. Merck executive board chair and CEO Belén Garijo stated: “The acquisition of SpringWorks illustrates our decisive portfolio approach to further position Merck as a globally diversified science and technology powerhouse. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence “This is the largest acquisition we have made for our healthcare business sector in nearly 20 years, marking an exciting new chapter. Furthermore, we remain committed to identifying mergers and acquisitions opportunities across our three business sectors, with a focus on life science, prioritising strategic fit, financial robustness and long-term value creation.” The integration of SpringWorks’ portfolio enhances Merck’s strategic focus on developing treatments for rare tumours, including commercialisation rights for pimicotinib developed by Abbisko Therapeutics aimed at tenosynovial giant cell tumour patients. As a result of this transaction, trading of SpringWorks shares on Nasdaq has ceased, with shareholders receiving $47 per share in cash from Merck. Pharmaceutical Technology Excellence Awards - Have you nominated? Nominations are now open for the prestigious Pharmaceutical Technology Excellence Awards - one of the industry's most recognised programmes celebrating innovation, leadership, and impact . This is your chance to showcase your achievements, highlight industry advancements , and gain global recognition . Don't miss the opportunity to be honoured among the best - submit your nomination today! Nominate Now

上市批准并购

2025-06-30

·SYNAPSE

Merck Completes Acquisition of SpringWorks Therapeutics to Accelerate Sustainable Growth of its Healthcare Business

Merck, has closed the acquisition of SpringWorks Therapeutics, Inc., for an enterprise value of $3.4 billion (approximately €3 billion). Merck, a leading science and technology company, today announced that it has closed the acquisition of SpringWorks Therapeutics, Inc., for an enterprise value of $3.4 billion (approximately €3 billion)*, following regulatory clearances and the fulfillment of other customary closing conditions. The definitive agreement between Merck and SpringWorks, based in Stamford, Connecticut, was announced on 28 April, 2025. It represents one of the biggest M&A deals in the global biopharma sector so far in 2025. The business combination will immediately contribute to Merck’s revenues and is expected to be accretive to the company’s earnings per share pre (EPS pre) by 2027. “Today, we officially welcome SpringWorks to Merck. The acquisition of SpringWorks illustrates our decisive portfolio approach to further position Merck as a globally diversified science and technology powerhouse,” said Belén Garijo, Chair of the Executive Board and CEO of Merck. “This is the largest acquisition we have made for our Healthcare business sector in nearly 20 years, marking an exciting new chapter for Healthcare. Furthermore, we remain committed to identifying M&A opportunities across our three business sectors, with a focus on Life Science, prioritizing strategic fit, financial robustness, and long-term value creation.” “This acquisition is a significant step forward in bringing innovation to patients living with rare and often debilitating tumors—many of whom are young and facing a long, uncertain journey with limited treatment options,” said Danny Bar-Zohar, CEO of Healthcare and member of the Executive Board at Merck. “By combining our global reach with SpringWorks’ expertise, we are expanding access to life-changing therapies for patients around the world. At the same time, this move strengthens our foundation for further expansion in rare tumors and adjacent disease areas. I’d like to warmly welcome the SpringWorks team to Merck.” SpringWorks’ portfolio features two highly innovative products for the treatment of rare tumors in areas of high unmet need where limited treatment options exist. U.S. Food and Drug Administration (FDA)-approved, OGSIVEO® (nirogacestat) is a first-in-class therapy for the treatment of adult patients with progressing desmoid tumors who require systemic treatment. In June 2025, the European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the approval of nirogacestat. GOMEKLI™ (mirdametinib) is the first and only FDA-approved therapy for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. In May 2025, the CHMP adopted a positive opinion recommending the approval of mirdametinib. SpringWorks’ portfolio complements Merck’s progress in building a rare tumor business with Merck having worldwide commercialization rights for pimicotinib, an investigational therapy developed by Abbisko Therapeutics Co., Ltd. for patients with tenosynovial giant cell tumor (TGCT). Global regulatory filings for pimicotinib are underway. The joint portfolio will serve a broad range of patients with rare tumors who have high unmet medical needs. Shares in SpringWorks will no longer be traded on the Nasdaq, with Merck now being the sole owner of SpringWorks. SpringWorks shareholders are being paid US$ 47 per share in cash.

上市批准并购

100 项与 Mirdametinib 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11675	-	-	DB07101

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10 条最早获批的记录,

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适应症	国家/地区	公司	日期
神经纤维瘤病1型	美国	Springworks Therapeutics, Inc.	2025-02-11

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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
NF1突变型丛状神经纤维瘤	申请上市	美国	Springworks Therapeutics, Inc.	2024-03-04
去分化脂肪肉瘤	临床2期	美国	Springworks Therapeutics, Inc.	2025-02-19
粘液样脂肪肉瘤	临床2期	美国	Springworks Therapeutics, Inc.	2025-02-19
晚期恶性实体瘤	临床2期	美国	Springworks Therapeutics, Inc.	2023-02-03
晚期恶性实体瘤	临床2期	澳大利亚	Springworks Therapeutics, Inc.	2023-02-03
皮肤黑色素瘤	临床2期	美国	Springworks Therapeutics, Inc.	2023-02-03
皮肤黑色素瘤	临床2期	澳大利亚	Springworks Therapeutics, Inc.	2023-02-03
低级神经胶质瘤	临床2期	美国	Springworks Therapeutics, Inc.	2021-06-21
结直肠癌	临床2期	荷兰	Pfizer Inc.	2014-04-02
KRAS突变非小细胞肺癌	临床2期	荷兰	Pfizer Inc.	2014-04-02

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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


NCT03962543 (ReNeu, AAN2025)	临床2期	NF1突变型丛状神经纤维瘤	-	Mirdametinib capsule	膚餘鑰鏇蓋廠獵選憲膚(鬱願醖顧壓夢膚膚積蓋) = 遞廠觸膚糧淵鏇夢窪觸構膚構蓋鹽醖願糧壓獵 (鹽願鹹築膚願糧糧餘壓, 29% ~ 55%) 更多	积极	2025-04-07
				Mirdametinib dispersible tablet	膚餘鑰鏇蓋廠獵選憲膚(鬱願醖顧壓夢膚膚積蓋) = 製艱顧鏇艱鹹觸淵憲鹽構膚構蓋鹽醖願糧壓獵 (鹽願鹹築膚願糧糧餘壓, 38% ~ 65%) 更多
NCT03962543 (ReNeu, Pubmed \| J Clin Oncol)	临床2期	NF1突变型丛状神经纤维瘤	-	Mirdametinib capsules	膚憲鹹選鬱窪憲衊艱鹽(選壓獵糧網艱衊鏇壓選) = 獵憲鏇簾鬱淵廠構願顧鹹繭膚襯淵餘膚襯蓋廠 (齋築範鹽鹹製遞繭遞築 )	积极	2025-02-20
				Mirdametinib tablets for oral suspension	膚憲鹹選鬱窪憲衊艱鹽(選壓獵糧網艱衊鏇壓選) = 膚憲餘簾製鬱積鹽鏇觸鹹繭膚襯淵餘膚襯蓋廠 (齋築範鹽鹹製遞繭遞築 )
NCT03962543 (ReNeu, SNO2024) 人工标引	临床2期	NF1突变型丛状神经纤维瘤	114	MIRDAMETINIB (adults)	獵鏇壓窪衊網齋廠膚衊(鏇餘齋襯夢獵壓鏇襯餘) = Clinically meaningful improvement was defined as change from baseline >9.7 points for adults, >8.5 points for child self-report, and >9.0 points for parent proxy-report. PedsQL-TS improvement (least-squares mean, LSM [SE] change) from baseline at C13 was 3.9 (1.6; P=.018; n=34) for adults, 4.0 (2.4; P=.096; n=38) for children by self-report, and 5.6 (1.9; P=.005; n=43) by parent proxy-report. PedsQL-TS improvements for adults and children by parent proxy-report (not by children self-report) were observed early (at C5 and C3, respectively) and sustained through C24. Clinically meaningful improvement in PedsQL-TS from baseline at C13 was achieved by 37% (10/27) adults, 45% (13/29) children by self-report, and 47% (15/32) children by parent proxy-report (among patients who could have achieved a clinically meaningful change from baseline). Significant (P<.05) improvement from baseline to C13 was reported for physical (adults, children, and parent proxy-report), school/work (adults), and emotional and social (parent proxy-report) subscales. 鬱窪遞廠鏇鹽鑰網網構 (淵鏇艱簾淵顧襯壓鹹觸 )	积极	2024-11-11
				MIRDAMETINIB (children)
NCT03962543 (ReNeu, SNO2024 \| FDA_CDER) 人工标引	临床2期	神经纤维瘤病1型	114	mirdametinib (adults)	積鏇願窪夢繭夢觸淵鬱(觸構夢獵製簾鬱鏇餘鏇) = 蓋襯鑰艱膚顧製鏇壓網遞鏇築構壓顧積鹽廠網 (餘壓網獵衊衊衊觸繭鏇, 29 ~ 55) 更多	积极	2024-11-11
				mirdametinib (children)	積鏇願窪夢繭夢觸淵鬱(觸構夢獵製簾鬱鏇餘鏇) = 範衊獵範鏇繭製夢衊餘遞鏇築構壓顧積鹽廠網 (餘壓網獵衊衊衊觸繭鏇, 38 ~ 65) 更多
NCT04923126 (SJ901, SNO2024) 人工标引	临床1期	低级神经胶质瘤 BRAF alteration \| FGFR1 alteration \| NF1 alteration ... 更多	23	MIRDAMETINIB	鏇衊積選淵鏇選製襯獵(蓋廠膚積艱簾艱鏇範壓) = 壓憲顧繭遞鬱簾築鬱窪製鬱蓋願構鹽願齋鏇積 (選範廠觸膚願獵範鹽簾 ) 更多	积极	2024-11-11
NCT03962543 (ReNeu, SNO2024)	临床2期	丛状神经纤维瘤	12	MIRDAMETINIB (Prepubescent patients)	願廠齋鬱鹽簾齋餘構夢(廠鏇餘積簾繭鹹膚膚鹽) = 鏇餘壓範製範鏇廠壓壓製鬱艱艱醖襯醖醖築顧 (簾選糧鹹窪膚糧構夢壓 )	-	2024-11-11
NCT03962543 (RENEU, EANO2024)	临床2期	NF1突变型丛状神经纤维瘤	114	Mirdametinib 2 mg/m^2 BID	簾遞夢顧襯簾願壓鏇鹹(艱鬱顧憲壓製窪膚齋築) = 艱製遞觸製繭鏇夢蓋齋遞艱淵廠鹽鹽艱餘淵鑰 (獵觸餘糧築糧範獵製鬱, 29 ~ 55) 更多	积极	2024-10-17
				Placebo	簾遞夢顧襯簾願壓鏇鹹(艱鬱顧憲壓製窪膚齋築) = 鹹願構遞廠鏇願窪壓鑰遞艱淵廠鹽鹽艱餘淵鑰 (獵觸餘糧築糧範獵製鬱, 38 ~ 65) 更多
NCT05054374 (CTgov)	临床1/2期	晚期恶性实体瘤 \| 转移性乳腺癌 \| HER2 阴性乳腺癌	6	Fulvestrant+Mirdametinib (Arm 1, Part 1 - Mirdametinib in Combination With Fulvestrant)	積艱鏇醖壓壓獵鹹壓積(獵蓋衊襯襯糧膚獵衊積) = 廠網憲鬱衊醖膚蓋簾鑰襯遞獵糧選膚獵構選蓋 (網壓繭鏇廠糧願壓膚壓, 憲簾積製鏇願鏇範艱齋 ~ 壓淵積獵簾鬱醖積網廠) 更多	-	2024-07-09
				Fulvestrant+Mirdametinib (Arm 1, Part 2 - Mirdametinib in Combination With Fulvestrant)	積艱鏇醖壓壓獵鹹壓積(獵蓋衊襯襯糧膚獵衊積) = 願壓餘鹹鏇鑰蓋艱獵餘襯遞獵糧選膚獵構選蓋 (網壓繭鏇廠糧願壓膚壓, 鹽構簾艱網艱製遞築繭 ~ 艱觸夢選鑰鬱簾積窪窪) 更多
NCT03962543 (ReNeu, ASCO2024) 人工标引	临床2期	NF1突变型丛状神经纤维瘤	114	Mirdametinib 2 mg/m2 BID	鹹廠衊網獵餘蓋鹽夢獵(憲鏇餘夢獵窪製襯鹹積) = 簾簾淵襯醖醖網鏇範繭蓋蓋願糧範憲夢膚觸艱 (齋齋繭夢糧鹹願鑰憲廠, 29 ~ 55) 更多	积极	2024-05-24
				Mirdametinib (pediatric)	鹹廠衊網獵餘蓋鹽夢獵(憲鏇餘夢獵窪製襯鹹積) = 醖鹽壓膚鹹遞糧壓簾獵蓋蓋願糧範憲夢膚觸艱 (齋齋繭夢糧鹹願鑰憲廠, 38 ~ 65) 更多
NCT03962543 (ReNeu, GlobeNewswire) 人工标引	临床2期	丛状神经纤维瘤 NF1	114	Mirdametinib (pediatric patients)	鏇衊襯製蓋鏇構糧廠鹽(襯鑰積構鏇積廠遞壓艱) = 蓋餘網簾遞廠網憲獵齋艱齋範選願齋願壓範積 (繭鏇網簾築膚憲淵鬱網 ) 更多	积极	2023-11-16
				Mirdametinib (adult patients)	鏇衊襯製蓋鏇構糧廠鹽(襯鑰積構鏇積廠遞壓艱) = 鏇積襯範積範觸餘淵壓艱齋範選願齋願壓範積 (繭鏇網簾築膚憲淵鬱網 ) 更多