Randomized, Open-label, Multicenter Phase 3 Study to Assess the Efficacy and Safety of GIVinostat Versus Hydroxyurea IN JAK2V617F-positive High-risk Polycythemia Vera Patients: the GIV-IN PV TRIAL

The goal of this clinical trial is to compare in the efficacy and safety of givinostat to hydroxyurea in Jak2V617F-positive high risk polycythemia vera patients.

开始日期2024-03-26

申办/合作机构

Italfarmaco SpA

NCT05933057 / Recruiting临床3期

Randomised, Double-blind, Placebo-controlled, Multicentre Study to Evaluate the Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy

This is a randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety, and tolerability of givinostat in non-ambulant male paediatric (aged 9 to <18 years) patients with DMD. 138 patients will be randomised 2:1 to givinostat or placebo and will be treated for 18 months.
Planned screening duration: approximately 4 weeks (±14 days)
Planned treatment duration: 18 months (approximately 72 weeks)
Planned follow-up duration: 4 weeks (±7 days) (for patients not participating in the long-term safety study)
Total duration of study participation: up to 83 weeks (ie, 20-21 months)

开始日期2024-02-19

申办/合作机构

Italfarmaco SpA

[+1]

NCT05492318 / Completed临床1期

An Open-label, Single-center, Three-part Study in Healthy Subjects to Investigate the Effect of Givinostat on the Pharmacokinetics of Midazolam and Dabigatran, the Effect of Clarithromycin on the Pharmacokinetics of Givinostat and the Pharmacokinetics of Single and Multiple Doses of Givinostat

The study will evaluate the givinostat (ITF2357) potential drug-drug interaction (DDI) at level of CYP3A-mediated metabolism and P-glycoprotein (P-gp) transport.

开始日期2022-03-21

申办/合作机构

Italfarmaco SpA

100 项与吉维诺司他相关的临床结果

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100 项与吉维诺司他相关的转化医学

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100 项与吉维诺司他相关的专利（医药）

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131

项与吉维诺司他相关的文献（医药）

2023-08-01·Advanced science (Weinheim, Baden-Wurttemberg, Germany)

A T Cell-Engaging Tumor Organoid Platform for Pancreatic Cancer Immunotherapy.

Article

作者: Yu, Tao ; Sharma, Samantha ; Zhou, Zhuolong ; Liu, Sheng ; Li, Yujing ; Opyrchal, Mateusz ; Wan, Jun ; Moulana, Ishara ; Van der Jeught, Kevin ; Wan, Guohui ; Territo, Paul R ; Lu, Xiongbin ; Zhang, Xinna

Pancreatic ductal adenocarcinoma (PDA) is a clinically challenging disease with limited treatment options. Despite a small percentage of cases with defective mismatch DNA repair (dMMR), PDA is included in the most immune-resistant cancer types that are poorly responsive to immune checkpoint blockade (ICB) therapy. To facilitate drug discovery combating this immunosuppressive tumor type, a high-throughput drug screen platform is established with the newly developed T cell-incorporated pancreatic tumor organoid model. Tumor-specific T cells are included in the pancreatic tumor organoids by two-step cell packaging, fully recapitulating immune infiltration in the immunosuppressive tumor microenvironment (TME). The organoids are generated with key components in the original tumor, including epithelial, vascular endothelial, fibroblast and macrophage cells, and then packaged with T cells into their outside layer mimicking a physical barrier and enabling T cell infiltration and cytotoxicity studies. In the PDA organoid-based screen, epigenetic inhibitors ITF2357 and I-BET151 are identified, which in combination with anti-PD-1 based therapy show considerably greater anti-tumor effect. The combinatorial treatment turns the TME from immunosuppressive to immunoactive, up-regulates the MHC-I antigen processing and presentation, and enhances the effector T cell activity. The standardized PDA organoid model has shown great promise to accelerate drug discovery for the immunosuppressive cancer.

2023-07-12·Cells

Chemokine/ITGA4 Interaction Directs iPSC-Derived Myogenic Progenitor Migration to Injury Sites in Aging Muscle for Regeneration.

Article

作者: Kim, Joung Woul ; Ashraf, Muhammad ; Tipparaju, Srinivas M ; Xuan, Wanling

The failure of muscle to repair after injury during aging may be a major contributor to muscle mass loss. We recently generated muscle progenitor cells (MPCs) from human-induced pluripotent stem-cell (iPSC) cell lines using small molecules, CHIR99021 and Givinostat (Givi-MPCs) sequentially. Here, we test whether the chemokines overexpressed in injured endothelial cells direct MPC migration to the site by binding to their receptor, ITGA4. ITGA4 was heavily expressed in Givi-MPCs. To study the effects on the mobilization of Givi-MPCs, ITGA4 was knocked down by an ITGA4 shRNA lentiviral vector. With and without ITGA4 knocked down, cell migration in vitro and cell mobilization in vivo using aged NOD scid gamma (NSG) mice and mdx/scid mice were analyzed. The migration of shITGA4-Givi-MPCs was significantly impaired, as shown in a wound-healing assay. The knockdown of ITGA4 impaired the migration of Givi-MPCs towards human aortic endothelial cells (HAECs), in which CX3CL1 and VCAM-1 were up-regulated by the treatment of TNF-α compared with scramble ones using a transwell system. MPCs expressing ITGA4 sensed chemokines secreted by endothelial cells at the injury site as a chemoattracting signal to migrate to the injured muscle. The mobilization of Givi-MPCs was mediated by the ligand-receptor interaction, which facilitated their engraftment for repairing the sarcopenic muscle with injury.

2023-06-13·Communications biology

Complete remission of diabetes with a transient HDAC inhibitor and insulin in streptozotocin mice.

Article

作者: Okano, Junko ; Kojima, Hideto ; Nakagawa, Takahiko ; Nakae, Yuki ; Fujino, Kazunori ; Miyazawa, Itsuko ; Ohashi, Natsuko ; Katagi, Miwako

Despite the growing epidemic worldwide, diabetes is an incurable disease. We have been focusing on why diabetes manifests refractoriness to any therapy. We recently found that abnormal bone marrow-derived cells (BMDCs), namely, Vcam-1⁺ST-HSCs, was a key mechanism for diabetic complications. We then hypothesize that those aberrant BMDCs sustainedly impair pancreatic β cells. Here we show that eliminating abnormal BMDCs using bone marrow transplantation results in controlling serum glucose in diabetic mice, in which normoglycemia is sustained even after cessation of insulin therapy. Alternatively, abnormal BMDCs exhibiting epigenetic alterations are treated with an HDAC inhibitor, givinostat, in diabetic mice. As a result, those mice are normoglycemic along with restored insulin secretion even following the cessation of both insulin and givinostat. Diabetic cell fusion between abnormal BMDCs and resident cells is significantly blocked by the combination therapy in the pancreatic islets and thymus while surgical ablation of the thymus completely eliminates therapeutic protection in diabetic mice. In conclusion, diabetes is an epigenetic stem cell disorder with thymic disturbances. The combination may be applied to patients aiming at complete remission from diabetes in clinical medicine.

项与吉维诺司他相关的新闻（医药）

2024-07-08

·药时代

2024上半年，中美批准新药对比

2024年上半年，美国食品药品监督管理局（FDA）批准了21款1类新药，中国国家药品监督管理局（NMPA）批准了26款1类新药。本文将分别盘点中美获批的这些新药，并比较两国获批新药在药物类型、适应症等方面的异同。 21款新药FDA批准上市截至2024年6月底，美国食品药品监督管理局(FDA)药品评价研究中心(CDER)共批准了21种新药，包括17个化药和6个生物药，批准数量比2023年同期略低，但仍处于历史平均水平以上。图1. 美国FDA历年批准的新药数量数据来源：FDA官网、博药整理表1. 2024H1美国FDA CDER批准的新药商品名称活性成分适应症研发企业批准日期小分子 ZELSUVMI BERDAZIMER SODIUM 传染性软疣 LNHC 1/5/24 EXBLIFEP CEFEPIME;ENMETAZOBACTAM 复杂性尿路感染;肾盂肾炎 ALLECRA THERAPEUTICS SAS 2/22/24 REZDIFFRA RESMETIROM MASH MADRIGAL 3/14/24 TRYVIO APROCITENTAN 高血压 IDORSIA PHARMA LTD 3/19/24 DUVYZAT GIVINOSTAT 杜氏肌营养不良 ITALFARMACO SA 3/21/24 VAFSEO VADADUSTAT 慢性肾病引起的贫血 AKEBIA THERAPEUTICS INC 2024-03-27 VOYDEYA DANICOPAN 阵发性睡眠性血红蛋白尿症（PNH）阿斯利康 3/29/24 ZEVTERA CEFTOBIPROLE MEDOCARIL SODIUM 多种感染 BASILEA PHARM ALLSCH 4/3/24 OJEMDA TOVORAFENIB 儿童低级别胶质瘤 DAY ONE BIOPHARMACEUTICALS 4/23/24 XOLREMDI MAVORIXAFOR WHIM综合征 X4 PHARMACEUTICALS 4/26/24 IQIRVO ELAFIBRANOR 原发性胆汁性胆管炎（PBC） IPSEN BIOPHARM LTD 6/10/24 SOFDRA SOFPIRONIUM 原发性腋窝多汗症 BOTANIX SB INC. 6/18/24 OHTUVAYRE ENSIFENTRINE 慢性阻塞性肺病（COPD） Verona Pharma 6/26/24 显像剂 LUMISIGHT PEGULICIANINE 乳腺癌手术中辅助检测 LUMICELL 4/17/24 寡核苷酸 RYTELO IMETELSTAT SODIUM 骨髓增生异常综合征（MDS） GERON 6/6/24 单抗 TEVIMBRA TISLELIZUMAB替雷利珠单抗食管鳞状细胞癌百济神州 3/13/24 PIASKY CROVALIMAB-AKKZ 阵发性睡眠性血红蛋白尿症（PNH）罗氏 6/20/24 其他生物药 WINREVAIR SOTATERCEPT-CSRK 肺动脉高压默沙东 3/26/24 ANKTIVA Nogapendekin alfa inbakicept-pmln 非肌层浸润性膀胱癌 ALTOR BIOSCIENCE 4/22/24 IMDELLTRA TARLATAMAB-DLLE 非小细胞肺癌安进 5/16/24 LETYBO LETIBOTULINUMTOXINA-WLBG 成人中度至重度眉间纹 HUGEL INC 2/29/24 注：清单包括CDER批准的多种新药及部分生物制品，不包括疫苗、过敏性产品、血液和血液制品、血浆衍生物、细胞和基因治疗产品，以及生物制品评估和研究中心在2024年批准的其他产品。数据来源：FDA官网、博药整理 26款新药NMPA批准上市截至2024年6月底，中国国家药品监督管理局(NMPA)药品评价研究中心(CDE)共批准了26种新药，包括15个化药、8个生物药和3个中药，批准数量比2023年同期（24个）有一定增长。表2. 2024H1 NMPA获批上市的1类新药药品名称企业名称适应症批准日期小分子脯氨酸加格列净片惠升生物 2型糖尿病 1/16/24 富马酸泰吉利定注射液恒瑞医药腹部手术后中重度疼痛 1/30/24 妥拉美替尼胶囊科州药物黑色素瘤 3/12/24 盐酸伊普可泮胶囊诺华成人阵发性睡眠性血红蛋白尿症（PNH） 4/24/24 恩替司他片亿腾景昂 HR阳性、HER-2阴性，经内分泌治疗复发或进展的局部晚期或转移性乳腺癌 4/24/24 富马酸安奈克替尼胶囊正大天晴 ROS1阳性的局部晚期或转移性非小细胞肺癌 4/24/24 瑞普替尼胶囊 BMS ROS1阳性的局部晚期或转移性非小细胞肺癌 5/8/24 苯磺酸克利加巴林胶囊海思科糖尿病性周围神经病理性疼痛 5/15/24 甲磺酸瑞齐替尼胶囊倍而达药业既往经EGFR-TKI治疗时或治疗后出现疾病进展，并且经检测确认存在EGFR T790M 突变阳性的局部晚期或转移性非小细胞肺癌 5/15/24 枸橼酸依奉阿克胶囊正大天晴 ALK阳性的局部晚期或转移性非小细胞肺癌 6/11/24 甲磺酸瑞厄替尼片圣和药业 EGFR T790M突变阳性的局部晚期或转移性非小细胞肺癌 6/11/24 戈利昔替尼胶囊迪哲医药复发或难治的外周T细胞淋巴瘤 6/18/24 考格列汀片海思科 2型糖尿病 6/18/24 注射用舒巴坦钠/注射用度洛巴坦钠组合包装 Entasis Therapeutics/再鼎医药 18 岁及以上患者由鲍曼-醋酸钙不动杆菌复合体敏感分离株所致医院获得性细菌性肺炎（HABP）、呼吸机相关性细菌性肺炎（VABP） 5/15/24 多肽枸橼酸倍维巴肽注射液百奥泰急性冠脉综合征 6/25/24 依柯胰岛素注射液诺和诺德 2型糖尿病 6/18/24 单抗仑卡奈单抗注射液卫材/渤健阿尔茨海默病 1/5/24 可伐利单抗注射液罗氏阵发性睡眠性血红蛋白尿症 2/6/24 贝莫苏拜单抗注射液正大天晴广泛期小细胞肺癌 4/30/24 泽美洛韦玛佐瑞韦单抗注射液兴盟生物狂犬病病毒暴露者的被动免疫治疗 6/4/24 恩朗苏拜单抗注射液石药集团含铂方案化疗失败的PD-L1表达阳性的复发或转移性宫颈癌 6/25/24 双抗依沃西单抗注射液康方生物联合培美曲塞和卡铂，用于经EGFR-TKI治疗后进展的EGFR基因突变阳性的局部晚期或转移性非鳞状非小细胞肺癌 5/21/24 CAR-T 泽沃基奥仑赛注射液科济药业复发或难治性多发性骨髓瘤 2/23/24 中药儿茶上清丸齐进药业口腔溃疡 1/8/24 九味止咳口服液卓和药业急性气管-支气管炎中医辨证属风热证的咳嗽 2/20/24 秦威颗粒成都华西天然药物急性痛风性关节炎 3/12/24 数据来源：药智数据、药智头条整理中美异同适应症、药物类型对比分析从批准数量来看，今年上半年NMPA批准的新药有26个，比美国CDER批准的新药（21个）多近20%。如果剔除中国特色的中药和不属于CDER批准范畴的细胞疗法，则NMPA批准的新药数为22个，比美国CDER批准的新药多1个。从药物类型来看，无论是中国还是美国，小分子都是获批新药的主要类型，占比均超过一半。其次为发展较为成熟的抗体药物。另外，新型药物如双特异性T细胞接合器、双抗、CAR-T疗法中美各有涉猎，但数量不多。图2. 2024上半年美国CDER批准新药药物类型分布数据来源：FDA官网、博药整理图3. 2024上半年NMPA批准新药药物类型分布数据来源：药智数据、博药整理从某种程度上而言，在研药物领域的创新技术与获批上市的药品技术存在一定时间差，目前较热门ADC、CAR-T等领域在新药获批层面上还未体现，而从目前新药研发领域由小分子药向生物药转变的趋势上来看，后续生物药新药获批数量或许会越来越高。从适应症来看，中美获批的新药中都是抗肿瘤新药最多，但是中国占比更高，接近50%，而美国为29%。从具体癌种来看，中国获批的12个肿瘤新药中有7个用于肺癌（6个非小细胞肺癌、1个小细胞肺癌）治疗，血液瘤2个，宫颈癌、乳腺癌、黑色素瘤各1个。而在美国获批的肿瘤新药中癌种较分散，包括食管癌、乳腺癌、胶质瘤等。这一系列中美差异点，或许正是国内新药同质化的原因之一，拥抱热门不可厚非，但强调差异化发展，如艾伯维一般进行建立核心疾病领域优势，对于国内创新药企业或许更值得学习。其次，抗感染、血液疾病、代谢性疾病、心血管疾病用药也是新药研发的热门领域。另外，罕见病作为美国新药开发和获批的重点领域，2024上半年获批的新药中有10个新药曾获得孤儿药认证，包括4个罕见肿瘤用药，2个血液罕见病用药，以及4个其他罕见病用药。而在中国，2024上半年获批上市的1类新药中，仅有2个进口1类罕见病新药获批用于治疗成人阵发性睡眠性血红蛋白尿症（PNH）患者，尚未出现国产自主研发的罕见病新药。当然，还有部分罕见病新药以5.1类新药申报并获批进口，不在本文统计范围内。这一点上，或许某种程度上也意味着中国新药“源头创新”意愿的不足，对于罕见病等临床上需求迫切的领域，反而因为市场空间、技术难度等问题而推进缓慢，这一点上或许有待加强。图4. 2024上半年美国CDER批准新药疾病领域分布数据来源：FDA官网、博药整理图5. 2024上半年NMPA批准新药疾病领域分布数据来源：药智数据、博药整理从企业来看，罗氏（Roche）开发的新一代C5循环抗体Piasky（crovalimab，可伐利单抗）同时在中美获批上市，用以治疗阵发性睡眠性血红蛋白尿症（PNH）患者。美国FDA批准的新药少部分来自跨国大药企，如默沙东、阿斯利康、安进等，多数来自美国的生物技术公司，例如MADRIGAL、AKEBIA、ALTOR BIOSCIENCE等。 NMPA批准的新药同样少部分来自跨国大药企，如BMS、诺华等，多数来自中国的药企和生物技术公司，例如恒瑞医药、石药集团、正大天晴、倍而达药业、科济药业、康方生物等。从审评方式来看，2024上半年美国有10个新药通过快速审批方式获得批准，占所有获批新药的48%；中国有9个新药通过优先审批获得批准，占所有获批新药的35%，可见快速审批、优先审批已是新药获得批准的重要途径。小结近年来，在特别审批、突破性治疗药物、附条件批准、优先审评审批等药品加快上市注册程序下，NMPA批准新药数量快速增长。2022年NMPA批准了23个1类新药，2023年这一数字增长到40个，而今年上半年就已批准了26个1类新药，超过美国FDA批准的1类新药数量，今年全年批准新药数量有望再创新高。但是，在适应症方面，国内新药主要集中在抗肿瘤领域，尤其是肺癌领域。而FDA批准的新药适应症罕见病和罕见肿瘤占据很大比例。这与国内外市场环境差异、创新药发展历程长短有关。目前而言，国外市场更鼓励去探索一些更新的靶点和适应症，对于比较新的靶点，药企也偏向于出海去寻找机会。未来，相信随着政策的支持、支付环境的改善，国内也有望迎来更多创新疗法。资料来源：美国FDA官网 NMPA官网药智数据各企业公开资料封面图来源：pixabay 版权声明/免责声明本文为授权转载文章。本文仅作信息交流之目的，不提供任何商用、医用、投资用建议。文中图片、视频、字体、音乐等素材或为药时代购买的授权正版作品，或来自微信公共图片库，或取自公司官网/网络，部分素材根据CC0协议使用，版权归拥有者，药时代尽力注明来源。如有任何问题，请与我们联系。衷心感谢! 药时代官方网站:www.drugtimes.cn 联系方式: 电话:13651980212 微信:27674131 邮箱:contact@drugtimes.cn 罗氏TIGIT再败肺癌后，决定提前终止这一研究一家CNS领域 biotech 的红与黑 mRNA CAR-T二期临床试验成功，公司股价却暴跌35%！点击这里，发现价值信息

上市批准寡核苷酸疫苗核酸药物临床研究

2024-06-28

·药智网

基因疗法的胜利

Sarepta股价上涨了32%。背后的助推原因是，Sarepta自主研发的用于治疗DMD（杜氏肌营养不良症）的基因疗法产品——Elevidys，被FDA完全批准用于4岁及以上经确定携带DMD基因突变的患者。 2023年10月时，这款药的顶线结果公布时，由于未达主要终点，Sarepta股价还曾一度下跌37.5%。此外，针对Elevidys，FDA还同时对疾病发展至严重、不能行走的DMD患者的适应症给予了加速批准。作为加速批准的正常流程，Sarepta承诺了将提交一项随机对照试验的结果，以作为完全批准的确证性研究。 DMD领域，Elevidys一骑绝尘 DMD是由DMD基因突变引起，导致肌萎缩蛋白缺失，而发展形成的一种进行性的遗传性肌肉疾病，主要影响男性。基因疗法直接作用于遗传物质，理论上来讲，直指DMD这类罕见遗传病的根本致病原因，是当前医学水平下最直接的治疗方式。目前，Sarepta的Elevidys仍是全球范围内唯一一款用于治疗DMD的基因疗法。随着此次FDA对这款药的两项新批准，Elevidys几乎将覆盖4岁及以上的所有携带DMD突变的DMD患者（第8号外显子和/或第9号外显子缺失的患者禁用）。公开信息得知，DMD患者一般在2岁左右时出现症状，3~5岁左右确诊，10~14岁患者将逐渐失去行走能力，并出现心脏和呼吸肌功能减弱症状，最终危机患者生命。也就是说，从年龄段来看，Elevidys已经覆盖了大部分DMD患者。另外，从基因突变特征类型来看Elevidys的覆盖面也已经远远甩开其他DMD产品。早前获批的，包括Sarepta的另外三款基于RNA技术平台开发的DMD产品、以及Amondys 45、Vyondys 53等产品均仅适用于个别外显子跳跃的DMD患者，仅分别占全部DMD人群的8%~13%左右。不过在今年3月，FDA批准了首款适用于所有遗传变异的DMD患者的非甾体类药物——Duvyzat。Duvyzat是一款组蛋白脱乙酰酶（HDAC）抑制剂，通过靶向致病过程来减少炎症和肌肉流失。值得注意的是，这款药是口服给药。但目前，Duvyzat仅被批准用于6岁及以上的DMD患者。一波三折的完全批准回顾Elevidys此次被FDA完全批准的整个过程，可谓一波三折。早在2023年10月，Sarepta曾公布了SRP-9001-301研究，结果并不乐观。数据显示，与安慰剂对照组相比，接受Elevidys治疗的受试者在52周时北极星移动评估（NSAA）分数有所增加，但其试验仍未达主要终点。当时的Sarepta认为这项试验仍然可以证明了Elevidys在所有年龄组中具有相似的临床获益，因为试验中所有次要终点（包括上升时间（p=0.0025）和10米步行测试（p=0.0048））均取得了积极且具有统计学意义的结果。在2023年10月发布的公告中，Sarepta强调公司将基于顶线结果立即采取行动，扩大标签，也提及了FDA的积极态度，但股票仍下跌了约37.5%。两个月后，即2023年12月，Sarepta又发公告，称已针对Elevidys提交疗效补充申请，以扩大适应症标签。也是在这份公告中，Sarepta提到已完成了SRP-9001-301研究的上市后要求（PMR），并向FDA提交了PMR，以此推进完全批准。 2024年2月，Sarepta再发公告，称FDA已经接受了对其疗效补充申请的审查，并将PDUFA日期定为2024年6月21日。最终结果正如文章开篇提到的，FDA通过了这项疗效补充申请，Elevidys的适应症范围得以扩展至更广泛人群。从Sarepta先后发布的三篇公告中可得知，此次Elevidys的这两项批准主要基于SRP-9001-301、SRP-9001-103、及SRP-9001-303三项研究。其中，SRP-9001-301纳入4-7岁的可正常行走的DMD患者，SRP-9001-103纳入了2岁及以上可正常行走的DMD患者。SRP-9001-303共有两个队列，分别纳入了8-18岁的可正常行走的DMD患者以及不能行走的DMD患者（无年龄限制）。据悉，SRP-9001-303从2023年6月才正式开始在美国的患者入组，尚未有更多相关临床数据公布。在此次FDA的官方公告，FDA也对未达主要终点这一点作出了回应：尽管研究未能达到主要终点，但对次要终点和探索性终点的审查“令人信服”，足以证明临床益处。基因疗法的胜利将Elevidys的境况放在非基因疗法类别的其他药品身上，那这款药能被FDA完全批准的可能性就得大打折扣。近年来，在FDA生物制品审评与研究中心（CBER）主任Peter Marks的鼎力相助下，基因疗法在FDA的审查过程中常亮绿灯。就在不久前，Peter Marks曾公开表明在之后的审查中，加速批准将成为基因疗法的常用批准方式，尤其是在患者人数少的罕见病领域。同时，为进一步助力基因疗法的研发热情，2024年1月8日，FDA宣布了一项名为“基因治疗全球试点合作”（CoGenT Global）的试点计划。计划提到，FDA将于其他全球监管合作伙伴（包括世界卫生组织和国际协调委员会（ICH）成员（包括欧盟（EU）、日本、加拿大和瑞士）合作，共同探索出一个通用的审批程序，以此为基因疗法领域提供更有利的监管环境。（不禁让人想到近日的“韩美日印欧生物制药联盟”）来源 | 药时代（药智网获取授权转载）撰稿 | 药时代多面体团队责任编辑 | 八角声明：本文系药智网转载内容，图片、文字版权归原作者所有，转载目的在于传递更多信息，并不代表本平台观点。如涉及作品内容、版权和其它问题，请在本平台留言，我们将在第一时间删除。商务合作 | 王存星 19922864877（同微信）阅读原文，是昨天最受欢迎的文章哦

基因疗法临床研究加速审批

2024-06-27

·BioSpace

KAZIA ANNOUNCES UPCOMING DIPG DATA PRESENTATIONS AT ISPNO AND PUBLICATION IN EUROPEAN JOURNAL OF CANCER

SYDNEY, June 27, 2024 /PRNewswire/ -- Kazia Therapeutics Limited (NASDAQ: KZIA), a biotechnology company specialising in oncology, is pleased to announce the presentation of new data from its lead program, paxalisib, at the upcoming 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) June 29 – July 2, 2024, in Philadelphia, PA. Kazia concurrently announces publication of an article in European Journal of Cancer highlighting the need for evaluating mutation-specific, CNS penetrant, inhibitors to treat pediatric patients with Diffuse Midline Glioma (DMG). There will be three paxalisib-related presentations in total at ISPNO, including data from the Phase 2 PNOC DMG-ACT (DMG-Adaptive Combination Trial, PNOC022) study evaluating the efficacy and safety of paxalisib in combination with ONC201. As a follow up to data presented last year at the Society for NeuroOncology, 28th Annual Meeting, lead researchers will discuss survival, pharmacokinetics, and tumor biomarkers from 132 diffuse midline glioma (DMG) patients enrolled in the Phase 2 study. Highlights of the abstract include median overall survival of 13.2 months in Cohort 1 (newly diagnosed, enrolled pre-radiation n=33), 15.8 months in Cohort 2 (newly diagnosed, enrolled post-radiation n=69) and 8.8 months in Cohort 3 (relapsed patients, enrolled after progression n=30). The second presentation is based on novel preclinical data utilizing the addition of a novel HDAC inhibitor to the backbone therapy of paxalisib in DMG models. The third presentation will highlight preclinical data results of the combination therapy of paxalisib and gemcitabine for patients with relapsed/recurrent atypical teratoid/rhabdoid tumors AT/RT by Johns Hopkins University researchers. Based on these findings, the Pacific Pediatric Neuro-Oncology Consortium is planning to include this combination therapy in its next AT/RT international clinical trial. Summary of Abstracts ( ) Clinical Trials; July 2, 2024; 8:15am TRLS-14: PNOC022 report: a combination therapy trial using an adaptive platform design for patients with diffuse midline glioma at initial diagnosis, post-radiation therapy, or progression Cassie Kline, Andrea Franson, Anuradha Banerjee, Alyssa T Reddy, et al Poster Session I; June 30, 2024; 5pm ATRT-15 Combining the PI3K inhibitor paxalisib with nucleoside analog gemcitabine to improve survival of atypical teratoid/rhabdoid tumors Tyler Findlay, Kristen Malebranche, Anupa Geethadevi, Charles Eberhart, Jeffrey Rubens, Eric Raabe DIPG-21 Preclinical assessment of a multimodal treatment approach with Givinostat, Paxalisib, and radiotherapy for Diffuse Midline Glioma (DMG) Aimée du Chatinier, Michaël H Meel, Piotr Waranecki, Dennis S Metselaar, Esther The European Journal of Cancer publication titled Paediatric Strategy Forum for Medicinal Product Development of PI3-K, mTOR, AKT and GSK3β Inhibitors in Children and Adolescents with Cancer is the output from a two-day forum in April 2023 at Dana Farber Cancer Institute. Consisting of patient advocates, regulators, researchers and pediatric clinicians, the publication concludes "Evaluation of mutation-specific, CNS-penetrant PI3-K inhibitors in children with DMG should be prioritised and innovative regulatory approaches are needed in view of the rarity of the population." The paper can be accessed at the following website: (24)00801-3/fulltext About Kazia Therapeutics Limited Kazia Therapeutics Limited (NASDAQ: KZIA) is an oncology-focused drug development company, based in Sydney, Australia. Our lead program is paxalisib, an investigational brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway, which is being developed to treat multiple forms of brain cancer. Licensed from Genentech in late 2016, paxalisib is or has been the subject of ten clinical trials in this disease. A completed Phase 2 study in glioblastoma reported early signals of clinical activity in 2021, and a pivotal study in glioblastoma, GBM AGILE, has completed enrollment, with final data expected imminently. Other clinical trials are ongoing in brain metastases, diffuse midline gliomas, and primary CNS lymphoma, with several of these having reported encouraging interim data. Paxalisib was granted Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA for diffuse intrinsic pontine glioma in August 2020, and for atypical teratoid / rhabdoid tumours in June 2022 and July 2022, respectively. For more information, please visit or follow us on Twitter @KaziaTx. Forward-Looking Statements This announcement may contain forward-looking statements, which can generally be identified as such by the use of words such as "may," "will," "estimate," "future," "forward," "anticipate," or other similar words. Any statement describing Kazia's future plans, strategies, intentions, expectations, objectives, goals or prospects, and other statements that are not historical facts, are also forward-looking statements, including, but not limited to, statements regarding: the timing for results and data related to Kazia's clinical and preclinical trials and investigator-initiated trials of Kazia's product candidates, and Kazia's strategy and plans with respect to its programs, including paxalisib and EVT801. Such statements are based on Kazia's current expectations and projections about future events and future trends affecting its business and are subject to certain risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements, including risks and uncertainties: associated with clinical and preclinical trials and product development, related to regulatory approvals, and related to the impact of global economic conditions. These and other risks and uncertainties are described more fully in Kazia's Annual Report, filed on form 20-F with the United States Securities and Exchange Commission (SEC), and in subsequent filings with the SEC. Kazia undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required under applicable law. You should not place undue reliance on these forward-looking statements, which apply only as of the date of this announcement. This announcement was authorized for release by Dr John Friend, CEO. Company Codes: NASDAQ-SMALL:KZIA

临床2期孤儿药放射疗法ASCO会议

100 项与吉维诺司他相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	吉维诺司他	-	DB12645

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
杜氏肌营养不良症	美国	Italfarmaco SpA	2024-03-21

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
真性红细胞增多症	临床3期	美国	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	奥地利	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	保加利亚	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	克罗地亚	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	法国	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	德国	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	以色列	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	意大利	Italfarmaco SpA	2024-03-26
真性红细胞增多症	临床3期	西班牙	Italfarmaco SpA	2024-03-26
贝克型肌营养不良	临床2期	意大利	Italfarmaco SpA	2017-12-12

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT02851797 (EPIDYS, Literature) 人工标引	临床3期	杜氏肌营养不良症	120	givinostat	糧醖鏇齋憲製簾築鏇淵(壓廠窪淵觸壓網糧繭蓋) = 網顧遞構網獵憲鑰醖夢膚壓憲簾艱鏇遞壓簾鹹 (選夢夢願顧壓願衊壓窪, 1.17 ~ 1.37)	积极	2024-04-01
				Placebo	糧醖鏇齋憲製簾築鏇淵(壓廠窪淵觸壓網糧繭蓋) = 艱廠鹹餘餘願願網艱顧膚壓憲簾艱鏇遞壓簾鹹 (選夢夢願顧壓願衊壓窪, 1.17 ~ 1.37)
NCT02851797 (FDA_CDER) 人工标引	临床3期	杜氏肌营养不良症	179	DUVYZAT	願鹽膚範窪繭鏇夢窪夢(鹽淵願範廠膚糧網築鑰) = 膚願積願觸襯襯鬱繭構衊鑰艱艱製鏇齋夢憲鏇 (艱築淵鏇夢簾製繭鑰壓 )	积极	2024-03-21
NCT05845567 (CTgov)	临床1期	药物相互作用	20	Givinostat (Givinostat Alone (Day 1))	構壓鬱獵觸鹽憲膚鏇壓(蓋糧鹹憲壓淵壓蓋製鹹) = 積壓積選鹽顧糧齋獵鹽衊糧壓廠憲範鏇築顧積 (築蓋構膚積窪醖顧窪積, 觸餘積鏇願壓衊膚齋鑰 ~ 鏇積遞繭鏇夢夢鏇壓觸) 更多	-	2024-02-09
				Givinostat+Clarithromycin (Givinostat Co-Administered With Clarithromycin)	構壓鬱獵觸鹽憲膚鏇壓(蓋糧鹹憲壓淵壓蓋製鹹) = 簾齋膚範網鬱顧鹽蓋憲衊糧壓廠憲範鏇築顧積 (築蓋構膚積窪醖顧窪積, 糧構窪壓遞顧糧鬱積鬱 ~ 積網淵簾襯顧選遞淵遞) 更多
NCT00792740 (CTgov)	临床1/2期	克罗恩病	51	Placebo (Placebo)	觸齋餘繭顧糧網製鏇網(選蓋醖製觸襯簾鏇鬱願) = 願醖壓憲獵獵觸遞窪觸築窪鹽淵淵廠遞艱築繭 (襯網壓遞齋齋鏇顧憲鬱, 艱襯網願齋蓋蓋構選憲 ~ 衊淵淵顧簾繭製簾襯鏇) 更多	-	2023-07-19
				ITF2357 (ITF2357)	觸齋餘繭顧糧網製鏇網(選蓋醖製觸襯簾鏇鬱願) = 鬱製鑰醖齋簾餘壓簾遞築窪鹽淵淵廠遞艱築繭 (襯網壓遞齋齋鏇顧憲鬱, 廠餘糧製積選鹽膚醖顧 ~ 淵鹽糧繭繭獵膚憲餘選) 更多
NCT02851797 (Epidys, MDA2023)	临床3期	杜氏肌营养不良症 fat fraction of the vastus lateralis (VL)	179	Givinostat	積淵淵蓋憲餘構夢膚構(範廠蓋窪齋夢願窪膚淵) = 獵糧觸壓網築構構廠鬱醖憲遞衊襯積壓網範艱 (齋窪簾顧憲淵顧淵衊齋 )	积极	2023-03-19
				Placebo	積淵淵蓋憲餘構夢膚構(範廠蓋窪齋夢願窪膚淵) = 鹽衊襯範簾遞遞積繭鹽醖憲遞衊襯積壓網範艱 (齋窪簾顧憲淵顧淵衊齋 )
NCT02851797 (Epidys Study, MDA2023)	临床3期	杜氏肌营养不良症	179	Givinostat	積窪夢鹹網遞壓顧願鑰(選淵膚鏇願糧膚選獵顧) = reversible and manageable 壓構艱壓憲範襯鹹糧糧 (顧淵選簾築築壓蓋鏇鹹 ) 更多	积极	2023-03-19
NCT02851797 (Study 1 \| Epidys Study \| EPIDYS, CTgov)	临床3期	杜氏肌营养不良症	179	givinostat (Givinostat)	顧淵網蓋鹹構顧淵齋廠(醖夢觸簾夢顧獵衊餘廠) = 顧廠鬱構齋繭築壓製鑰齋構鏇構鹽顧膚鏇鑰鹹 (鑰觸膚廠積鹽艱遞鹽鏇, 窪醖鏇選鏇膚選壓製艱 ~ 網鏇觸製憲膚壓鑰鹹糧) 更多	-	2023-02-02
				placebo (Placebo)	顧淵網蓋鹹構顧淵齋廠(醖夢觸簾夢顧獵衊餘廠) = 積蓋廠鑰簾憲齋獵範鹹齋構鏇構鹽顧膚鏇鑰鹹 (鑰觸膚廠積鹽艱遞鹽鏇, 範壓醖顧選範築膚築齋 ~ 觸齋憲構觸構簾膚顧窪) 更多
NCT02851797 (EPIDYS, PipelineReview) 人工标引	临床3期	杜氏肌营养不良症	179	Givinostat	範淵夢醖鏇選願顧鬱網(廠夢醖壓艱蓋鹹憲蓋遞): difference = 1.78, P-Value = 0.0345 更多	积极	2022-06-26
				Placebo
NCT03238235 (BusinessWire) 人工标引	临床2期	贝克型肌营养不良	47	Givinostat	襯壓艱憲夢選構壓衊夢(鑰積積製襯醖憲範鏇蓋) = No serious Adverse Events (AE) occurred during the course of the trial. 糧廠夢製廠憲膚艱鹽顧 (構遞顧憲蓋鏇願壓顧鬱 ) 更多	积极	2021-06-26
				Placebo
NCT00792467 (CTgov)	临床1/2期	霍奇金淋巴瘤	24	ITF2357	築鏇積鑰簾鑰顧鏇製鑰(獵鬱餘簾繭鹹簾艱鹽憲) = 積醖鏇製夢膚蓋壓膚襯蓋顧齋餘廠觸壓鹹積餘 (衊壓築網醖鏇衊築廠積, 衊遞鏇鑰鹽繭襯衊遞鹽 ~ 鑰顧選顧壓觸衊簾壓範) 更多	-	2021-04-19