This phase II trial assesses the feasibility (including both safety and tolerability) of conducting Next Generation Sequencing and administering targeted therapy (infigratinib) in the preoperative setting for patients with intrahepatic cholangiocarcinoma that can be removed by surgery (resectable). Chemotherapy drugs, such as nab-paclitaxel, cisplatin, and gemcitabine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Targeted therapy with infigratinib will bind to FGFR which can help stop tumor cell growth and cause tumor cell death. Giving chemotherapy and/or targeted therapy before surgery may make the tumor smaller for resection and may help prevent the cancer from coming back. If a molecular profiling test shows a genetic change called an FGFR2 fusion, patients receive both chemotherapy and targeted therapy while patients without a FGFR2 fusion just receive chemotherapy. Giving targeted therapy based on molecular profile testing results prior to attempted resection of an intrahepatic cholangiocarcinoma that has a risk for either not being able to be removed or for coming back after it has been removed may help improve treatment outcomes.

开始日期2024-03-01

申办/合作机构

Emory University

[+1]

NCT06164951 / Enrolling by invitation临床3期

A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children 3 to <18 Years of Age With Achondroplasia: PROPEL 3

This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of participation in the QED-sponsored study PROPEL (QBGJ398-001).

开始日期2023-11-10

申办/合作机构

QED Therapeutics, Inc.初创企业

CTR20233585 / Recruiting临床2期

一项评价口服Infigratinib单药治疗伴有成纤维细胞生长因子受体2(FGFR2)基因扩增的局部晚期或转移性胃癌或胃食管结合部腺癌患者的多中心、开放、单臂II期临床试验

主要目的：评价Infigratinib单药治疗伴有FGFR2基因扩增的GC/GEJ患者的有效性次要目的：评价Infigratinib单药治疗伴有FGFR2基因扩增的GC/GEJ患者的有效性；评价Infigratinib单药治疗伴有FGFR2基因扩增的GC/GEJ患者的安全性和耐受性描述在伴有FGFR2基因扩增的GC/GEJ患者中Infigratinib和活性代谢产物的药代动力学（PK）特征探索性目的： 1.探索采用不同检测方法检测患者肿瘤组织中FGFR2基因扩增状态的结果。 2.在接受至少一次Infigratinib治疗的患者中，探索肿瘤生物标志物的变化及其与有效性和/或耐药性的潜在关系。

开始日期2023-11-07

申办/合作机构

Alcami Carolinas Corp.

[+2]

100 项与英菲格拉替尼相关的临床结果

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100 项与英菲格拉替尼相关的转化医学

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100 项与英菲格拉替尼相关的专利（医药）

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199

项与英菲格拉替尼相关的文献（医药）

2024-05-01·Anti-cancer agents in medicinal chemistry

An Expedition on Synthetic Methodology of FDA-approved Anticancer Drugs (2018-2021)

Review

作者: Gupta, Ghanshyam Das ; Jain, Ankit ; Vishakha, S ; Navneesh, N ; Verma, Sant Kumar ; Patel, Preeti ; Kurmi, Balak Das

Abstract::

New drugs being established in the market every year produce specified structures for selective biological targeting. With medicinal insights into molecular recognition, these begot molecules open new rooms for designing potential new drug molecules. In this review, we report the compilation and analysis of a total of 56 drugs including 33 organic small molecules (Mobocertinib, Infigratinib, Sotorasib, Trilaciclib, Umbralisib, Tepotinib, Relugolix, Pralsetinib, Decitabine, Ripretinib, Selpercatinib, Capmatinib, Pemigatinib, Tucatinib, Selumetinib, Tazemetostat, Avapritinib, Zanubrutinib, Entrectinib, Pexidartinib, Darolutamide, Selinexor, Alpelisib, Erdafitinib, Gilteritinib, Larotrectinib, Glasdegib, Lorlatinib, Talazoparib, Dacomitinib, Duvelisib, Ivosidenib, Apalutamide), 6 metal complexes (Edotreotide Gallium Ga-68, fluoroestradiol F-18, Cu 64 dotatate, Gallium 68 PSMA-11, Piflufolastat F-18, 177Lu (lutetium)), 16 macromolecules as monoclonal antibody conjugates (Brentuximabvedotin, Amivantamab-vmjw, Loncastuximabtesirine, Dostarlimab, Margetuximab, Naxitamab, Belantamabmafodotin, Tafasitamab, Inebilizumab, SacituzumabGovitecan, Isatuximab, Trastuzumab, Enfortumabvedotin, Polatuzumab, Cemiplimab, Mogamulizumab) and 1 peptide enzyme (Erwiniachrysanthemi-derived asparaginase) approved by the U.S. FDA between 2018 to 2021. These drugs act as anticancer agents against various cancer types, especially non-small cell lung, lymphoma, breast, prostate, multiple myeloma, neuroendocrine tumor, cervical, bladder, cholangiocarcinoma, myeloid leukemia, gastrointestinal, neuroblastoma, thyroid, epithelioid and cutaneous squamous cell carcinoma. The review comprises the key structural features, approval times, target selectivity, mechanisms of action, therapeutic indication, formulations, and possible synthetic approaches of these approved drugs. These crucial details will benefit the scientific community for futuristic new developments in this arena.

2024-01-05·Clinical cancer research : an official journal of the American Association for Cancer Research

Landscape of Clinical Resistance Mechanisms to FGFR Inhibitors in FGFR2-Altered Cholangiocarcinoma

Article

作者: Ellis, Haley ; Fece de la Cruz, Ferran ; Bardeesy, Nabeel ; Norden, Bryanna L. ; Che, Jianwe ; Michel, Alexa G. ; Silveira, Vanessa S. ; Corcoran, Ryan B. ; Siravegna, Giulia ; Balasooriya, Eranga Roshan ; Zhen, Yuanli ; Wu, Qibiao

Abstract:

Purpose::

FGFR inhibitors are effective in FGFR2-altered cholangiocarcinoma, leading to approval of reversible FGFR inhibitors, pemigatinib and infigratinib, and an irreversible inhibitor, futibatinib. However, acquired resistance develops, limiting clinical benefit. Some mechanisms of resistance have been reported, including secondary FGFR2 kinase domain mutations. Here, we sought to establish the landscape of acquired resistance to FGFR inhibition and to validate findings in model systems.

Experimental Design::

We examined the spectrum of acquired resistance mechanisms detected in circulating tumor DNA or tumor tissue upon disease progression following FGFR inhibitor therapy in 82 FGFR2-altered cholangiocarcinoma patients from 12 published reports. Functional studies of candidate resistance alterations were performed.

Results::

Overall, 49 of 82 patients (60%) had one or more detectable secondary FGFR2 kinase domain mutations upon acquired resistance. N550 molecular brake and V565 gatekeeper mutations were most common, representing 63% and 47% of all FGFR2 kinase domain mutations, respectively. Functional studies showed different inhibitors displayed unique activity profiles against FGFR2 mutations. Interestingly, disruption of the cysteine residue covalently bound by futibatinib (FGFR2 C492) was rare, observed in 1 of 42 patients treated with this drug. FGFR2 C492 mutations were insensitive to inhibition by futibatinib but showed reduced signaling activity, potentially explaining their low frequency.

Conclusions::

These data support secondary FGFR2 kinase domain mutations as the primary mode of acquired resistance to FGFR inhibitors, most commonly N550 and V565 mutations. Thus, development of combination strategies and next-generation FGFR inhibitors targeting the full spectrum of FGFR2 resistance mutations will be critical.

2024-01-01·Journal of orthopaedic translation

Targeting FGFR3 signaling and drug repurposing for the treatment of SLC26A2-related chondrodysplasia in mouse model

Article

作者: Li, Pan ; Yang, Liu ; Luo, Zhuojing ; Jie, Qiang ; He, Xin ; Zhao, Chengxiang ; Yun, Haitao ; Wang, Dong ; Lu, Weiguang ; Hu, Jingyan

Background:

Mutations in Slc26a2 cause a spectrum of autosomal-recessive chondrodysplasia with a significant and negligible influence on the quality of life. It has been reported that Slc26a2 deficiency triggers the ATF6 branch of the UPR, which may, in turn, activate the negative regulator of the FGFR3 signaling pathway. However, the correlation between the deletion of Slc26a2 and the augmentation of downstream phosphorylation of FGFR3 has not been investigated in vivo.

Methods:

First, we constructed Slc26a2 and Fgfr3 double knockout mouse lines and observed gross views of the born mice and histological staining of the tibial growth plates. The second approach was to construct tamoxifen-inducible Cre-ER^T2 mouse models to replicate SLC26A2-related non-lethal dysplastic conditions. Pharmacological intervention was performed by administering the FGFR3 inhibitor NVP-BGJ398. The effect of NVP-BGJ398 on chondrocytes was assessed by Alcian blue staining, proliferation, apoptosis, and chondrocyte-specific markers and then verified by western blotting for variations in the downstream markers of FGFR3. The growth process was detected using X-rays, micro-CT examination, histomorphometry staining of growth plates, and immunofluorescence.

Results:

Genetic ablation of Fgfr3 in embryonic Slc26a2-deficient chondrocytes slightly attenuated chondrodysplasia. Subsequently, in the constructed mild dysplasia model, we found that postnatal intervention with Fgfr3 gene in Slc26a2-deficient chondrocytes partially alleviated chondrodysplasia. In chondrocyte assays, NVP-BGJ398 suppressed the defective phenotype of Slc26a2-deficient chondrocytes and restored the phosphorylation downstream of FGFR3 in a concentration-dependent manner. In addition, in vivo experiments showed significant alleviation of impaired chondrocyte differentiation, and micro-CT analysis showed a clear improvement in trabecular bone microarchitectural parameters.

Conclusion:

Our results suggested that inhibition of FGFR3 signaling pathway overactivation and NVP-BGJ398 has promising therapeutic implications for the development of SLC26A2-related skeletal diseases in humans.

The translational potential of this article:

Our data provide genetic and pharmacological evidence that targeting FGFR3 signaling via NVP-BGJ398 could be a route for the treatment of SLC26A2-associated skeletal disorders, which promisingly advances translational applications and therapeutic development.

127

项与英菲格拉替尼相关的新闻（医药）

2024-06-12

·E药经理人

百济“神秘”大股东，狂“买”中国创新药！这家对冲基金有何来头？

Seagen曾经最大股东，百济神州第二大股东，康方合作伙伴Summit第四大股东，如今所投资的海思科海外合作方再登IPO舞台，仅有25名员工却管理250亿美元，这家总资产20年翻百倍的海外对冲基金有何来头？撰文| Erin 编辑| 润屿顿河近日，海思科TYK2抑制剂ESK-001出海的合作伙伴美国生物技术公司Alumis提交了在纳斯达克的IPO申请，募集资金用以支持ESK-001的III期临床。在此之前，Alumis已凭借ESK-001完成了多轮过亿美元融资，其中B轮融资约3亿美元，投资方包括Matrix Capital、Baker Brothers等知名投资机构。值得一提的是，投资方之一的对冲基金Baker Brothers，其投资标的与中国公司有着千丝万缕的关系。上个月，Baker Brothers刚刚投资了康方生物的合作伙伴Summit，成为其第四大股东。此外，Baker Brothers还是百济神州的十大股东之一。而这家隐匿于生物技术公司背后的神秘“大佬”，眼光颇为独到，核药明星Bicycle、推出全球首款NASH新药的Madrigal都在持仓名单中。不仅如此，Baker Brothers还曾是ADC先驱Seagen的最大股东，昔日Seagen被辉瑞收购，为Baker Brothers带来了80亿美元的收益。盘点这家对冲基金在生物技术公司上的投资，不可谓一段传奇。2003年时总资产为2.5亿美元，但到了2023年，其资产已增百倍至257亿美元。这家对冲基金的独到眼光，究竟是怎样炼成的？又为何狂“买”中国药企创新资产？重仓中国创新药的海外基金，还有谁？长期持仓，高收益，总资产20年增百倍 Baker Brothers自创立以来，就专注于生物技术公司的投资。多年来，这家对冲基金几乎是生物技术超级爆款的“掘金队”，且因对生物技术赛道“长期持仓，获得高收益”的独到眼光而闻名，曾重仓的Seagen、Mirati、Alexion等生物技术公司，纷纷被MNC并购，更是重复验证了这一能力。 Baker Brothers在近年最出名的一次事件，莫过于在2023年辉瑞收购Seagen后，一下子获得了80亿美元的高额收益。据悉，Baker Brothers自2003年就投资了当时还名叫Seattle Genetics的Seagen，此后持续加仓，到了2005年，Baker Brothers的创始人之一Felix Baker就成为了Seattle Genetics的首席独立董事。截至辉瑞完成收购前，Baker Brothers是Seagen的最大股东，持股比例超过25%。这场长达20年的“陪跑”，也为Baker Brothers带来了80亿美元的收益。投资Seagen的案例并非Baker Brothers首次打响名号。 2015年，艾伯维以每股261.25美元、总额210亿美元收购了Pharmacyclics，获得了伊布替尼。彼时，Pharmacyclics是Baker Brothers最大的持仓，持有约900万股Pharmacyclics股票，总价值超过12亿美元。此后，Baker Brothers所投资的Alexion、ChemoCentryx、Mirati等相继被AZ、安进、BMS等MNC收入囊中。Baker Brothers的总资产也水涨船高，截至2023年年底，其总资产高达257亿美元，相较于2003年2.5亿美元，翻了100倍。值得一提的是，Baker Brothers虽然目前管理着超过250亿美元的资产，但其员工总数仅有25人，且一直保持神秘，迄今甚至连官网都没有。但Baker Brothers能获得如此高收益，离不开“人”的因素。据悉，Baker Brothers的创始人是Julian Baker、Felix Baker两兄弟，2000年成立之时的主要资金来自美国财团Tisch家族，之后还引入了耶鲁捐赠基金会。创始人两兄弟分别拥有斯坦福大学免疫学博士学位和瑞士信贷第一波士顿银行的私募部门工作背景，“生物技术+投资”两大专业背景的组合为他们在投资中建立了独特眼光，而两兄弟在投资后通常会加入被投公司的董事会，其专业背景也能保障为被投公司做好长期的投后管理。当然，其投资标的屡屡被MNC青睐，或许也与两兄弟的投后管理不无关系。根据Baker Brothers所提交给SEC的文件，这家对冲基金目前持有86只股票，前五大持股占其总资产的68%，这显示出“高度集中投资”的特点。而Baker Brothers持有的投资组合十分有意思：几乎都是生物技术各个领域的佼佼者。最大持仓的Incyte，已经是全球知名制药公司，还是JAK抑制剂的先驱，已获批8款创新药；第三持仓的Acadia Pharmaceuticals，也是全球中枢神经系统疾病领域的头部公司，多个产品都是全球首个且唯一的疗法；第四持仓的Madrigal Pharmaceuticals，研发出了全球首款NASH疗法；此外，Baker Brothers所持仓的Bicycle、Kymera、Immatics等公司也分别是核药、蛋白降解、TCR-T细胞疗法领域的明星公司，并被诺华、赛诺菲、BMS等MNC青睐。推荐阅读 * AZ、诺华密集抄底中国Biotech！罗氏开年4笔，BMS一月许出265亿美元，MNC并购背后的权谋 * 溢价154%！AZ打响第一枪！本土CAR-T四小龙之一“卖身”MNC，下一个是谁？重仓中国创新药的海外基金除了具有较高的被并购潜力外，Baker Brothers的投资标的，更与中国创新药关系非常紧密。 Baker Brothers的第二大持仓，正是中国的创新药“一哥”百济神州。截至今年4月19日，Baker Brothers持有百济神州10.2%的普通股，是百济的十大股东之一。 ▲来源：百济神州2024年年度股东大会会议资料而Baker Brothers最大持仓的Incyte，分别与信达、康哲、诺诚健华都有合作。此外，在亘喜生物被AZ收购前，Baker Brothers也是其股东之一。再加上其投资的海思科合作伙伴Alumis、康方合作伙伴Summit，Baker Brothers几乎可以称得上中国创新药出海背后神秘的“最大买家”之一。与Baker Brothers和中国创新药的“隐形”关系不同，海外基金RTW Investments、Perceptive Advisors则直接参与了中国Biotech公司的孵化。例如在2019年，由Perceptive Advisors孵化的联拓生物成立，并于2021年在纳斯达克上市，主要专注于将欧美的创新药引入中国，成立短短几年，联拓生物就建立了Mavacamten、TP-03、NBTXR3、infigratinib（英菲格拉替尼）多个管线，横跨心血管、眼科、肿瘤、炎症四大领域。虽然最终以联拓生物关停为结局，但也代表着一次国外基金到中国孵化Biotech的实践。而与Perceptive Advisors不同，RTW Investments与中国创新药的关系更加紧密，不仅孵化了箕星药业，还参与了恒瑞GLP-1的授权出海。 RTW所孵化的箕星药业也专注于将海外创新药带入中国，通过这条路径，箕星药业目前建立了心血管领域和眼科疾病领域的多个产品管线，并已经有9个临床后期阶段在研产品。值得注意的是，箕星药业还在今年1月获得了拜耳与RTW的D轮融资，拜耳集团处方药事业部全球执行副总裁兼中国区总裁、拜耳集团大中华区总裁周晓兰更是加入到了箕星董事会。另外，今年5月，恒瑞的三款GLP-1产品授权给刚刚成立不久的美国Hercules公司。Hercules公司恰恰是由贝恩资本、Atlas Ventures、RTW、Lyra资本联合出资创立。除了Perceptive Advisors和RTW外，其实国内还有很多由海外基金参与孵化的Biotech，如信瑞诺、经久生物、维亚臻生物等，并且已经诞生了一笔海外基金成功退出的孵化：今年1月，诺华宣布收购信瑞诺医药，并将其将整体整合入诺华中国。据悉，信瑞诺成立于2021年，专注于肾脏疾病及相关治疗领域，由海外生物技术公司Chinook Therapeutics与鼎丰生科资本、Frazier Healthcare Partners等投资机构共同设立。其中，Chinook通过技术入股，以2个药(atrasentan、BION-1301)的大中华区权益换取合资公司信瑞诺50%A轮优先股，投资机构投资4000美元获得另外50%股份。而在2023年，诺华收购了Chinook。因此，诺华再收购信瑞诺的交易，也被行业解读为诺华“不舍” atrasentan和zigakibart两款重磅产品剩下50%的中国权益。回复“V”，了解电子期刊详情精彩推荐 CM10 | 集采 | 国谈 | 医保动态 | 药审 | 人才 | 薪资 | 榜单 | CAR-T | PD-1 | mRNA | 单抗 | 商业化 | 国际化｜猎药人系列专题 | 出海启思会 | 声音·责任 | 创百汇 | E药经理人理事会 | 微解药直播 | 大国新药 | 营销硬观点 | 投资人去哪儿 | 分析师看赛道 | 药事每周谈 | 医药界·E药经理人 | 中国医药手册创新100强榜单 | 恒瑞 | 中国生物制药 | 百济 | 石药 | 信达 | 君实 | 复宏汉霖｜翰森 | 康方生物 | 上海医药 | 和黄医药 | 东阳光药 | 荣昌 | 亚盛医药 | 齐鲁制药 | 康宁杰瑞 | 贝达药业 | 微芯生物 | 复星医药｜再鼎医药｜亚虹医药跨国药企50强榜单 | 辉瑞 | 艾伯维 | 诺华 | 强生 | 罗氏 | BMS | 默克 | 赛诺菲 | AZ | GSK | 武田 | 吉利德科学 | 礼来 | 安进 | 诺和诺德 | 拜耳 | 莫德纳 | BI | 晖致 | 再生元

并购临床3期抗体药物偶联物IPO

2024-06-04

·药明康德

每年多长高2.5厘米！潜在“best-in-class”疗法3期试验已启动

▎药明康德内容团队编辑 BridgeBio Pharma今天宣布，其在研口服疗法infigratinib在治疗软骨发育不全（achondroplasia）儿童患者的2期临床试验PROPEL 2中表现出潜在“best-in-class”的持续疗效和安全性。队列5中的患儿在18个月时，年平均身高生长速度（AHV）与基线相比提高2.50厘米/年。Infigratinib的的全球3期注册试验PROPEL 3已经启动，预计今年年底会完成患者招募。迄今为止，PROPEL 2试验中第5组剂量递增队列的主要结果包括：在12个月时，患儿的AHV相对于基线的平均增长为+2.51厘米/年，在18个月时为+2.50厘米/年，增长持续且具有统计学意义（p=0.0015）。 ▲Infigratinib持续改善患儿身高增长（图片来源：参考资料[2]）患儿的身体比例（上半身与下半身平均比例）显著改善，从基线时的2.02改善到第18个月时的1.88（与基线相比的平均变化，p=0.001）。 ▲Infigratinib持续改善患儿身体比例（图片来源：参考资料[2]）该疗法展现持续良好的安全性与耐受性，未发生与infigratinib相关的治疗相关不良事件。 ▲Infigratinib的作用机制（图片来源：参考资料[2]）软骨发育不全是侏儒症的常见类型，主要表现为四肢、脊柱和头骨底部的骨骼生长缓慢，患儿每年只能生长约4厘米（正常为6-7厘米）。全世界约有25万人被这种疾病困扰，但只能通过手术解决一些症状。该病发病的背后病理机制是由于编码成纤维细胞生长因子受体3（FGFR3）的基因出现突变，FGFR3蛋白过度活跃而造成的。FGFR3是骨骼发育的负向调节分子，可影响软骨细胞的增殖活化和分化。而infigratinib通过阻断FGFR3的活性，就好像松开抑制儿童生长的“刹车”，直接靶向软骨发育不全的潜在病理生理学，从而促进软骨内骨形成，让患儿的生长速率恢复正常。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料： [1] BridgeBio Announces Durable Month 12 and 18 Phase 2 Cohort 5 Results of Oral Infigratinib in Achondroplasia, and First Participant Consented in ACCEL for Hypochondroplasia. Retrieved June 4, 2024 from https://www.globenewswire.com/news-release/2024/06/04/2892934/0/en/BridgeBio-Announces-Durable-Month-12-and-18-Phase-2-Cohort-5-Results-of-Oral-Infigratinib-in-Achondroplasia-and-First-Participant-Consented-in-ACCEL-for-Hypochondroplasia.html [2] Investor webcast: PROPEL 2 data update and ACCEL program initiation. Retrieved June 4, 2024 from https://investor.bridgebio.com/static-files/54364352-e46a-49a3-a749-8ddf8f3be134 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

临床结果临床2期

2024-06-04

·FierceBiotech

BridgeBio dwarfism drug increases height in children, beating BioMarin's Voxzogo

BridgeBio is sufficiently encouraged by 12- and 18-month data on daily oral FGFR3 inhibitor infigratinib to expand development. BridgeBio Pharma has bounced back from the withdrawal of infigratinib in oncology, linking the molecule to a BioMarin-beating height increase in children with a growth disorder in a midphase clinical trial. The FDA withdrew accelerated approval of infigratinib in May, completing a process that began one year earlier when BridgeBio’s then-partner Helsinn asked the agency to pull the authorization. Helsinn made the request after struggling to enroll subjects for a required confirmatory clinical trial. As the cancer program went off the rails, BridgeBio continued to advance low-dose infigratinib in a form of dwarfism. BridgeBio reported top-line data from the midphase PROPEL 2 trial in March 2023 and followed up with six-month data around one year ago. Tuesday, the biotech unwrapped 12- and 18-month data that add to evidence that the daily oral FGFR3 inhibitor infigratinib may have an edge over BioMarin’s approved dwarfism therapy Voxzogo. On infigratinib, children with achondroplasia, the most common type of dwarfism, had mean increases in annualized height velocity (AHV) of 2.51 cm/year after 12 months and 2.50 cm/year after 18 months. The FDA approved (PDF) Voxzogo on the basis of a trial that reported a 1.40 cm/year increase in AHV at 12 months. BridgeBio also shared data on a secondary endpoint. The ratio of the upper to lower body improved in children on infigratinib, falling from 2.02 at baseline to 1.88 after 18 months. The 0.14 improvement is greater than the change BioMarin reported across two years of follow up. Ravi Savarirayan, M.D., Ph.D., of Murdoch Children’s Research Institute, said in a statement that changes in body ratio are encouraging and suggest infigratinib “has the potential to enhance functionality for people living with achondroplasia in addition to increasing growth.” Infigratinib appears capable of beating Voxzogo, with the usual caveats about cross-trial comparisons, but its effect on AHV fell between the six- and 12-month updates. After six months, the mean increase in AHV was 3.38 cm/year from baseline. Even so, with no patients having treatment-related adverse events, BridgeBio is sufficiently encouraged by the data to expand development. A phase 3 study in achondroplasia is already underway and on track to complete enrollment by the end of the year. BridgeBio is now working to enter phase 3 in hypochondroplasia, a form of short-limbed dwarfism. After talking to regulators, BridgeBio has begun a small, open-label phase 2 trial in hypochondroplasia. The study will test infigratinib at one dose, the level that has shown most promise in achondroplasia, to support progression into a placebo-controlled registrational phase 3 study in hypochondroplasia. Last month, BioMarin said it aimed to start a phase 3 trial in hypochondroplasia by midyear. BioMarin reported a 74% increase in Voxzogo revenue in the first quarter, causing sales of the product to reach $153 million, but multiple companies are aiming to challenge the biotech for the achondroplasia market. Ascendis Pharma and Sanofi are among the drug developers with achondroplasia assets in the clinic.

临床结果临床3期上市批准加速审批

100 项与英菲格拉替尼相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11589	-	L01XE	DB11886

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
FGFR2融合或重排的胆管癌	美国	Helsinn Healthcare SA	2021-05-28

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
胆管上皮癌	申请上市	瑞士	Helsinn Birex Pharmaceuticals Ltd.	2022-01-30
软骨发育不全	临床3期	美国	BridgeBio Pharma, Inc.	2023-03-07
肾盂和输尿管尿路上皮癌	临床3期	美国	QED Therapeutics, Inc.初创企业	2020-03-11
肾盂和输尿管尿路上皮癌	临床3期	比利时	QED Therapeutics, Inc.初创企业	2020-03-11
肾盂和输尿管尿路上皮癌	临床3期	保加利亚	QED Therapeutics, Inc.初创企业	2020-03-11
肾盂和输尿管尿路上皮癌	临床3期	加拿大	QED Therapeutics, Inc.初创企业	2020-03-11
肾盂和输尿管尿路上皮癌	临床3期	法国	QED Therapeutics, Inc.初创企业	2020-03-11
肾盂和输尿管尿路上皮癌	临床3期	德国	QED Therapeutics, Inc.初创企业	2020-03-11
肾盂和输尿管尿路上皮癌	临床3期	希腊	QED Therapeutics, Inc.初创企业	2020-03-11
肾盂和输尿管尿路上皮癌	临床3期	意大利	QED Therapeutics, Inc.初创企业	2020-03-11

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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


NCT04265651 (PROPEL2, Biospace) 人工标引	临床2期	软骨发育不全 \| 软骨发育不良	-	Infigratinib (Cohort 5)	膚憲鹹簾顧餘淵獵蓋範(鹹鏇衊廠繭糧範選鑰顧) = 壓觸醖製壓蓋選餘醖窪遞壓餘衊壓艱膚觸鹽鏇 (網選醖簾簾蓋壓鏇積壓 ) 更多	积极	2024-06-11
NCT04197986 (PROOF302, CTgov)	临床3期	肾盂和输尿管尿路上皮癌 \| 膀胱尿路上皮癌	39	Infigratinib (Infigratinib 125 mg)	艱獵鑰獵選獵範壓襯觸(窪糧製遞壓餘網艱蓋遞) = 積顧窪願獵夢築衊範鹹範觸齋鏇選衊醖選繭製 (構蓋積廠構淵鏇選遞鏇, 鏇憲網鏇襯襯齋蓋積醖 ~ 簾築築鑰憲醖憲蓋壓淵) 更多	-	2024-03-13
NCT04197986 (PROOF302, CTgov)	临床3期	肾盂和输尿管尿路上皮癌 \| 膀胱尿路上皮癌	39	Placebo (Placebo)	艱獵鑰獵選獵範壓襯觸(窪糧製遞壓餘網艱蓋遞) = 顧鑰網鏇憲積廠衊觸選範觸齋鏇選衊醖選繭製 (構蓋積廠構淵鏇選遞鏇, 艱選窪遞範簾觸糧鑰範 ~ 襯觸繭窪糧積鹹鑰顧壓) 更多	-	2024-03-13
NCT04265651 (PROPEL 2, Biospace) 人工标引	临床2期	软骨发育不全	-	infigratinib	鏇憲遞壓壓鏇齋獵窪築(鑰醖淵壓糧艱膚膚夢觸) = 鹽製廠範遞顧鬱壓觸襯壓艱鑰顧簾構繭選憲顧 (製膚觸鑰齋餘餘獵遞繭 )	积极	2024-02-07
NCT04197986 (PROOF302, ASCO_GU2024) 人工标引	临床3期	尿路上皮癌辅助 FGFR3 Fusion \| FGFR3 Mutation	39	Infigratinib	膚積鹽餘醖壓壓艱鑰齋(簾蓋艱糧襯鹹鹽顧鹽繭) = No significant differences 選鹹繭觸憲憲衊艱遞膚 (衊獵鏇蓋鹽齋餘憲繭鏇 ) 更多	不佳	2024-01-25
NCT04197986 (PROOF302, ASCO_GU2024) 人工标引	临床3期	尿路上皮癌辅助 FGFR3 Fusion \| FGFR3 Mutation	39	Placebo		不佳	2024-01-25
NCT03773302 (PROOF 301, ASCO_GI2024) 人工标引	临床3期	FGFR2融合或重排的胆管癌一线 FGFR2 Fusion \| FGFR2 Rearrangement	48	Infigratinib 125 mg	鹹艱顧鹹簾網網襯願繭(繭願齋鹹繭淵築夢築膚) = 蓋簾廠糧範構構繭獵膚鬱廠鹽鹽窪鏇範憲選憲 (選鹹壓壓繭艱網積構衊, 5.4 ~ NE) 更多	积极	2024-01-18
NCT03773302 (PROOF 301, ASCO_GI2024) 人工标引	临床3期	FGFR2融合或重排的胆管癌一线 FGFR2 Fusion \| FGFR2 Rearrangement	48	Gemcitabine + Cisplatin	鹹艱顧鹹簾網網襯願繭(繭願齋鹹繭淵築夢築膚) = 夢壓廠願餘獵網憲鑰製鬱廠鹽鹽窪鏇範憲選憲 (選鹹壓壓繭艱網積構衊, 3.4 ~ NE) 更多	积极	2024-01-18
NCT05019794 (LB1001-201, ESMO2023) 人工标引	临床2期	FGFR2阳性胃癌 \| 胃食管交界处腺癌三线 FGFR2 amplification	19	Infigratinib 125mg	鬱襯網窪衊鹹鹽衊遞鏇(糧築糧壓觸糧窪範齋製) = 繭繭獵壓夢衊餘願鏇衊簾顧獵壓衊獵鏇憲積積 (夢鏇築醖蓋憲觸壓選網, 56.3, 94.3) 更多	积极	2023-10-23
NCT04265651 (PROPEL 2, ESPE2023)	临床2期	软骨发育不全 fibroblast growth factor receptor 3 (FGFR3) gene \| collagen X marker	-	Infigratinib	窪壓鏇獵憲繭觸鏇鏇糧(觸醖夢構鬱夢網衊築積) = 顧鏇顧壓鏇糧醖壓網製願膚憲淵醖齋窪鏇蓋鹽 (築蓋選網獵淵鬱鏇糧艱 )	-	2023-09-21
NCT04504331 (CTgov)	临床1期	PR 阳性乳腺癌 \| HER2 阴性乳腺癌 \| ER阳性乳腺癌 ... 更多	4	Omnipaque 350+Tamoxifen+Infigratinib+Iopamidol (Cohort 1: Infigratinib (100mg) + Tamoxifen)	憲襯餘鏇醖網鹹醖鏇網(壓衊觸鏇廠鏇遞襯夢壓) = 顧齋築憲顧獵選構憲艱齋鬱壓艱製鏇夢遞窪觸 (蓋廠選選廠顧網鏇製獵, 衊膚鏇鏇鬱積衊製願遞 ~ 製積膚鏇鏇繭網繭簾網) 更多	-	2023-06-01
NCT04504331 (CTgov)	临床1期	PR 阳性乳腺癌 \| HER2 阴性乳腺癌 \| ER阳性乳腺癌 ... 更多	4	Omnipaque 350+Tamoxifen+Infigratinib+Iopamidol (Cohort 2: Infigratinib (125mg) + Tamoxifen)	憲襯餘鏇醖網鹹醖鏇網(壓衊觸鏇廠鏇遞襯夢壓) = 選憲夢範簾簾艱膚夢鏇齋鬱壓艱製鏇夢遞窪觸 (蓋廠選選廠顧網鏇製獵, 蓋繭鏇鏇廠鏇繭窪鑰積 ~ 廠窪構顧鑰餘構製壓壓) 更多	-	2023-06-01
NCT04424966 (ASCO2023) 人工标引	早期临床1期	复发性恶性胶质瘤	7	Infigratinib 125 mg	鬱遞餘構簾簾艱醖範蓋(觸鏇積鹽鏇簾齋範鹽憲) = 糧繭製衊願窪獵網衊遞衊夢願襯網簾製廠觸遞 (選積範獵衊廠蓋願積範 ) 更多	不佳	2023-05-31
NCT04265651 (Biospace) 人工标引	临床2期	软骨发育不全	11	Infigratinib	壓構網鑰壓繭鏇壓襯窪(繭膚網艱鏇鹹餘鏇夢憲) = 鹹製夢衊鑰醖窪衊餘繭鬱簾襯廠壓廠繭範範憲 (餘廠衊構網夢構憲餘觸 ) 更多	积极	2022-07-26