An Open-Label, Phase II Study Evaluating the Feasibility, Safety, and Efficacy of Oral Tetrahydrouridine-Decitabine in Patients With Advanced Myeloproliferative Neoplasms

This is a phase II, multicenter, open-label study evaluating the feasibility, safety, and efficacy of oral tetrahydrouridine-decitabine (THU-decitabine) in patients with advanced myeloproliferative neoplasms (MPN).

开始日期2027-01-01

申办/合作机构

Icahn School of Medicine at Mount Sinai

[+1]

NCT05816356

/ Completed临床1期

An Open Label, Randomized, Single Dose, Two Way Crossover, Bioavailability Study of a Combination Formulation of Decitabine/Tetrahydrouridine (2.5 mg/100 mg) Modified Release Capsules in Healthy, Fasting, Male Adults

This is an open-label, randomized, single-dose per period, two-period, crossover study to evaluate the relative bioavailability of decitabine and tetrahydrouridine ingested as a modified-release combination formulation compared to THU and decitabine ingested as immediate-release capsules

开始日期2023-03-24

申办/合作机构

EpiDestiny, Inc.

[+1]

NCT03233724

/ Terminated临床1/2期IIT

Phase I/II Evaluation of Oral Decitabine/Tetrahydrouridine as Epigenetic Priming for Pembrolizumab Immune Checkpoint Blockade in Inoperable Locally Advanced or Metastatic Non-Small Cell Lung Cancers, Esophageal Carcinomas, or Pleural Mesotheliomas

Background:
Non-small cell lung cancer (NSCLC) is the most common type of lung cancer. Lung cancer is the leading cause of cancer-related death in the United States. Most people with lung cancer are already in the advanced stages of the disease by the time they see a doctor. Researchers want to see if combining an approved drug with two new drugs can help.
Objective:
To study if tetrahydrouridine-decitabine (THU-DAC) with pembrolizumab is safe and effective in people with non-small cell lung cancer that cannot be removed by surgery.
Eligibility:
People 18 years and older who have NSCLC that cannot be removed by surgery
Design:
Participants will be screened with
* Medical history
* Physical exam
* Blood and urine tests
* Tests of heart and lung function
They may have a small tumor sample taken (biopsy). They may have tumor scans.
Before starting treatment, participants will repeat the screening tests. They will also give a stool sample.
The study will be done in 3-week cycles for up to 6 cycles.
* Participants will take the 2 study drugs by mouth 3-5 days a week.
* Participants will get pembrolizumab in a vein for 30 minutes 1 day each cycle.
Participants will keep a study medication diary.
During cycle 1, participants will have blood taken multiple times on days 1 and 2.
Every 3 cycles, participants will repeat screening tests.
Participants will have a mandatory tumor biopsy.
When they finish treatment, participants will have a physical exam and blood tests.

开始日期2018-04-11

申办/合作机构

National Cancer Institute

100 项与四氢尿苷相关的临床结果

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100 项与四氢尿苷相关的转化医学

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100 项与四氢尿苷相关的专利（医药）

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199

项与四氢尿苷相关的文献（医药）

2026-12-01·RENAL FAILURE

β-Thujaplicin Mobilizes Renal Tubular Iron to Alleviate Diabetic Kidney Disease.

Article

作者: Wu, Qian ; Yang, Fengning ; Huang, Shan ; Xu, Xiufang ; Wang, Kewu ; Chu, Jiayi ; Xiao, Wenbo

Diabetic kidney disease (DKD) is a major complication of diabetes mellitus. Iron metabolism is implicated in the pathogenesis of DKD; however, its mechanistic basis and therapeutic implications remain incompletely explored. Here, we employed unilateral nephrectomy combined with streptozotocin-induced DKD mice to identify distinct renal iron metabolic profiles in DKD versus controls. Notably, although total renal iron content remained unchanged, pronounced iron deposition was detected in proximal renal tubules of DKD mice compared with controls. This aberrant iron deposition correlated with elevated fibrotic markers, indicating a potential mechanistic link between iron dysregulation and renal fibrosis in DKD. Importantly, the iron ionophore β-Thujaplicin (hinokitiol) effectively mobilized tubular iron and significantly attenuated renal fibrosis. Transcriptome analysis revealed that TGF-β signaling, a key fibrotic pathway, was inhibited by β-Thujaplicin. To further elucidate the mechanism of β-Thujaplicin mitigating renal fibrosis, HK-2 cells were exposed to a combination of high glucose, palmitate, and TGF-β1. The results demonstrated that THU alleviated fibrosis in HK-2 cells by reducing oxidative stress, decreasing intracellular iron levels, and inhibiting the TGF-β signaling pathway. Collectively, our findings establish that iron metabolic reprogramming drives DKD progression, and pharmacological targeting of tubular iron deposition via iron ionophores represents a promising strategy against renal fibrosis in DKD and related nephropathies.

2026-10-01·Lancet Child & Adolescent Health

Paediatric Drug Optimization for Sickle Cell Disease: priorities for research and development in children

Review

作者: Morris, Claudia R ; Were, Wilson Milton ; Yaqub, Nuhu Omeiza ; Ross, Anna Laura ; Ubuguyu, Omary Said ; Segafredo, Giulia ; Kityo, Cissy ; Penazzato, Martina ; Hankins, Jane S ; Charnaud, Sarah ; Makani, Julie ; Odame, Isaac ; Masini, Tiziana

Sickle cell disease remains a major cause of childhood morbidity and mortality, particularly in sub-Saharan Africa. In September, 2025, WHO convened the Paediatric Drug Optimization for Sickle Cell Disease process to review approved therapies, pipeline candidates, and potentially curative approaches, and define priorities for children and adolescents. Hydroxyurea (hydroxycarbamide) was confirmed as the leading near-term priority, with age-appropriate soluble or dispersible formulations identified as essential to improve equitable paediatric access; preferred and minimum characteristics were defined through a formal target product profile. Among investigational agents, pyruvate kinase activators and decitabine plus tetrahydrouridine (NDec) emerged as promising candidates for paediatric investigation on the basis of emerging efficacy data and programmatic potential. Intersecting research priorities included identifying appropriate clinical trial endpoints, strengthening early and inclusive paediatric investigation, and proactively ensuring that the promise of potential cure through gene therapy does not delay investment in scalable disease-modifying treatments.

2026-08-09·XENOBIOTICA

Tetrahydrouridine pre-treatment increases tissue exposure of ¹⁴ C-decitabine in mice

Article

作者: Helleberg, Hans ; Beck Petersen, Signe ; Kjær Offenberg, Hanne ; Bjørnsdottir, Inga

Decitabine (DEC) is rapidly deaminated by cytidine deaminase (CDA); tetrahydrouridine (THU), a CDA inhibitor, reduces degradation and increases DEC exposure. This is clinically relevant in sickle cell disease (SCD), where DEC increases foetal haemoglobin in erythrocytes.The effect of THU pre-treatment on the tissue distribution of radiolabelled decitabine ([¹⁴C]-DEC) was studied in mouse by quantitative whole-body autoradiography and area under the concentration curve (AUC) as exposure metric.The decitabine-related exposure was highest in haematolymphoid tissues, particularly the bone marrow, followed by gastrointestinal tissues across all dosing groups, with up to 115‑ and 18‑fold higher levels than in blood, respectively.Furthermore, THU pre-treatment increased the exposure in haematolymphoid tissues, gastrointestinal tract and reproductive organs by up to 4.8-, 6.2- and 5.3-fold, respectively.In conclusion, THU pre-treatment enhances decitabine-related exposure in target tissues like bone marrow, supporting pre-dosing of THU prior to DEC treatment of SCD.

项与四氢尿苷相关的新闻（医药）

2024-02-02

·智慧芽新药科讯

读懂百年诺和诺德之创新、增长与影响非凡的第100年

近日，丹麦医药巨头诺和诺德公司迎来了其成立100周年的里程碑。诺和诺德官网更新了一份长度超过100页的2023年报，完整地回顾了这个百年药企走过的第100个年头。在庆祝这一非凡成就的同时，公司的管理层也深刻认识到过去的成功并不能保证未来的持续进步。作为全球领先的慢性病治疗企业，诺和诺德致力于通过创新药物改变严重慢性疾病患者的生活，并以此塑造其长远愿景，即不仅要在核心治疗领域保持领先地位，还要成为推动全球人类健康改善的驱动力。诺和诺德成立于1923年。在走过的第一百年中，诺和诺德的核心贡献体现在行业领先的研发成果上，其疗法惠及了全球超过4000万患有严重慢性疾病的患者。其中，针对II型糖尿病和肥胖症未满足的需求日益增长，GLP-1为基础的疗法需求激增，促使诺和诺德达到了历史性的患者触及数量，北美及国际业务的销售额实现了强劲增长。诺和诺德努力成为一家可持续发展的公司，为社会和我们的未来事业创造价值。我们以对财务、环境和社会负责的方式开展业务，并践行诺和诺德之道。通过取得成功，我们将为慢性病患者、员工、合作伙伴、股东和社会创造长期价值。诺和诺德基金会通过诺和控股公司持有诺和诺德公司 77.1% 的投票权和 28.1% 的股份。诺和诺德基金会在三个战略领域提供资助：健康、可持续性和生命科学生态系统。2023 年，诺和诺德基金会提供超过 90 亿丹麦克朗的资助。诺和诺德在产品管线方面取得了显著进展。SELECT试验显示，司美格鲁肽2.4 mg 可将肥胖患者发生重大心血管事件的风险降低 20%，为此公司积极寻求对 Wegovy® 标签的更新；FLOW肾脏试验提前结束，基于 semaglutide 的新组合疗法 CagriSema 已进入II型糖尿病和肥胖症的三期临床开发阶段；潜在全球首款每周一次的基础胰岛素——icodec胰岛素正在等待监管批准。此外，诺和诺德还通过收购拓展了在心血管疾病领域的布局，如在罕见血液病领域推进 Mim8 和 etavopivat 的三期临床试验。在财务表现方面，诺和诺德展现了卓越的商业实力和社会责任。2023年度，公司实现了高达232.261亿丹麦克朗的净销售额，运营利润达到102.574亿丹麦克朗，全球员工人数增至64,319名，并在80个国家设有分支机构，研发投入超过450亿丹麦克朗，同比增长35%。同时，公司不断减少环境足迹，相较于2019年，运营和运输过程中的二氧化碳当量排放减少了34%，并且协助供应商转向100%可再生能源供电。为提升健康公平性，诺和诺德继续强化全球范围内的预防工作。新近与南非阿斯彭制药建立的生产合作关系将大幅提高非洲大陆平价胰岛素的供应量，而长期运行的“改变糖尿病”儿童项目以及“获取胰岛素承诺”等举措则持续支持低收入和中等收入国家的脆弱群体。在美国，更多患者受益于诺和诺德提供的包括非品牌生物制剂、低成本人胰岛素和患者援助计划在内的更广泛可负担方案。诺和诺德同样重视研发领域的投入与创新技术平台的合作，首个RNA干扰衍生疗法Rivfloza™已在美国获批，这标志着公司在新型疗法领域取得突破。加强波士顿地区研发中心的建设，有望打开更多的合作机会，加速药物发现与开发进程。诺和诺德将继续拓宽并深化产品管线，依托内部世界级研发能力和对外部创新及业务发展的聚焦，进军诸如代谢功能障碍相关脂肪性肝炎（MASH）、慢性肾病（CKD）和阿尔茨海默病（AD）等新兴治疗领域，追求疾病修饰和治愈性疗法。材料中也提到了诺和诺德公司所销售产品的专利到期日期的说明，并列出了这些产品在美国、中国、日本及欧洲地区，关于活性成分专利的具体到期日期，包括多款糖尿病、肥胖和罕见病产品。公司在糖尿病治疗领域的全球领导地位，通过GLP-1（胰高血糖素样肽-1）为基础的药物产品强劲增长而得到进一步巩固。在过去的一年里，诺和诺德凭借其GLP-1类药物如Ozempic®（司美格鲁肽）和Rybelsus®（口服司美格鲁肽）在全球范围内实现了显著的增长，尤其是在北美市场和国际业务中，这两大产品的快速接纳和广泛使用极大地推动了公司价值市场份额的提升。在肥胖市场方面，公司针对市场领先肥胖症疗法采取了谨慎与控制性上市策略。Wegovy®基于GLP-1受体激动剂原理开发，该产品建立在公司首款用于肥胖治疗的GLP-1药物Saxenda®的成功基础上，使得诺和诺德在市场上占据了明显的优势地位。Wegovy®的需求急剧增长，但受限于全球供应短缺以及前所未有的媒体关注带来的挑战。为确保疗法能够到达最需要的患者手中，并考虑到产能限制问题，公司在包括美国在内的各个主要市场采取了有计划、有限度的推广方式。在美国市场，Wegovy®的市场需求依然强劲，而在其他国家如英国，这款药品通过国家卫生服务系统内的专科体重管理服务提供给符合严格标准的患者使用，同时也可以通过私人注册医疗机构获取。诺和诺德充分意识到肥胖症在全球范围内的严重性和日益增长的趋势，预计到2030年将有超过12亿成年患者受到肥胖困扰。由于肥胖症与多种疾病的发生息息相关，例如糖尿病、心脏病和肝脏疾病、癌症等，因此，越来越多的医疗专业人员和社会公众认识到有效治疗肥胖症对于改善公共卫生状况和降低长期医疗成本的重要性。为了应对高需求和供应压力，诺和诺德不仅在供应链上加大投资以扩大生产规模，还调整了产品推出和分销模式，确保负责任地向现有患者群体提供适当治疗选择的同时，也为无力承担自费购药费用的患者预留了一定比例的产品供应，尤其是在新上市市场中，彰显了其社会责任感和对健康公平性的承诺。此外，通过监测和评估新市场的反应及患者的反馈，诺和诺德持续优化治疗方案和服务，致力于帮助更多患者实现长期健康改善的目标。在罕见病治疗领域，公司也展开了积极的战略布局，寻求可持续增长。尽管该部门在过去的一年中面临挑战，特别是由于生产限制导致核心产品 Norditropin®（用于生长激素缺乏症的长期疗法）销售额下降15%，但公司仍通过新推出的每周一次治疗方案Sogroya®来开拓市场机会。Sogroya®凭借其减少患者每日注射负担、并展现疗效优势的特点，为生长激素缺乏症患者带来了新的希望。面对供应问题对满足不断增长的产品需求带来的阻碍，诺和诺德将解决供应链瓶颈作为首要任务。为了减轻一次性注射装置供应的压力，公司采取了推出药物的一周制剂而非日制剂的形式，并且正在寻找降低对此类装置依赖的方法。同时，在罕见血液疾病方面，尽管NovoSeven®和NovoEight®销售下滑，但新产品如 Esperoct® 和 Refixia® 的强劲需求带动了血友病A和B治疗领域的整体销售额增长1%。公司已投入超过750亿丹麦克朗的资金用于全球生产网络的扩建，包括丹麦卡尔乌尔布洛普、法国沙特尔以及爱尔兰阿斯隆等地的主要设施扩张与收购棕色地带开发及生产场地。公司努力确保现有工厂24小时不间断地运营，以最大限度提高现有生产基础的产量，从而向全球范围内的更多患者提供所需的药品。最后，材料中小结了2023年业绩亮点：糖尿病治疗业务：诺和诺德继续巩固其在糖尿病治疗领域的领导地位，销售额以丹麦克朗计算增长了24%，按固定汇率计算增长了29%。特别是GLP-1为基础的产品销售增长强劲，推动全球价值市场份额上升至33.8%。肥胖症治疗市场：公司在肥胖症市场的表现显著，销售额按固定汇率（CER）计算增长了154%，达到约416亿丹麦克朗，展现出该领域产品的巨大市场需求和潜力。罕见病管线：尽管面临供应限制导致罕见病产品销售额下降15%（CER），但公司仍致力于解决供应链瓶颈问题，并批准了Somapacitan在美国、欧盟和日本用于儿童生长激素缺乏症的治疗，为罕见病业务恢复增长打下了基础。财务表现：全年实现总销售额增长36%（CER），运营利润增长44%（CER）。通过优化价值链操作效率，增强了公司的盈利能力，确保能够投资于未来的增长资产。可持续发展与社会责任：公司在环保方面取得进步，如碳排放减少；同时扩大了对糖尿病患者的医疗支持，在非洲地区通过合作伙伴生产平价胰岛素，以提高药品可及性。研发进展：完成了一系列关键药物的研发里程碑，包括口服司美格鲁肽不同剂量的三期临床试验成功、CagriSema在II型糖尿病中的三期临床试验启动以及心血管结局研究的积极结果等。2024年展望稀有疾病领域：预计通过加强现有管线并推进新疗法的开发，实现罕见病业务的持续增长前景。心血管疾病和其他新兴治疗领域：计划进一步拓展心血管疾病（CVD）、代谢功能障碍相关脂肪性肝炎（MASH）和慢性肾病（CKD）等新兴治疗领域，通过启动细胞疗法临床试验、收购高血压治疗药物以及开展针对特定靶点抗体的I期临床试验等方式来建立业务。糖尿病领导力强化：目标是在全球价值市场上继续保持领先地位，争取超过三分之一的市场份额。肥胖症领域：期望到2025年实现超过250亿丹麦克朗的销售收入，继续利用已有的优势产品及即将上市的新药推动销售增长。诺和诺德正在研发中的产品管线，涵盖了糖尿病、肥胖症、罕见病以及心血管疾病共四个治疗领域。糖尿病方向：Oral Semaglutide (NN9924) 描述：一款长效GLP-1类似物，25mg 和 50mg 剂量，设计为每日一次口服给药适应症：II型糖尿病阶段：已上市或处于后期开发阶段 Icodec NN1436 描述：一种长效基础胰岛素类似物，旨在每周一次皮下注射适应症：I型糖尿病和II型糖尿病阶段：已上市或处于后期开发阶段 CagriSema NN9388 描述：一款 Amylin 类似物 cagrilintide 和 GLP-1 类似物 semaglutide 的复方制剂，用于每周一次的皮下注射治疗。适应症：II型糖尿病和肥胖症阶段：临床3期开发阶段 OW GLP-1 GIP NN9541 描述：这是一种GLP-1和GIP双重激动剂，设计为每周一次皮下注射适应症：肥胖症和II型糖尿病阶段：临床1期开发阶段 DNA Immunotherapy NN9041描述：一种编码前胰岛素和前胰岛素的新型质粒，用于保护β细胞功能。适应症：I型糖尿病阶段：临床1期开发阶段肥胖方向：Oral Semaglutide NN9932 描述：一款长效GLP-1类似物，设计为每日一次口服给药。阶段：临床3期开发阶段 CagriSema NN9838 描述：一款 Amylin 类似物 cagrilintide 和 GLP-1 类似物 semaglutide 的复方制剂，用于每周一次的皮下注射治疗。Amycretin NN9487 描述：这是一款 GLP-1 受体和 amylin 受体的双重激动剂 (co-agonist) , 设计为每周一次皮下注射或一日一次口服阶段：临床1期开发阶段罕见病方向：Concizumab (NN7415)描述：一种针对组织因子途径抑制剂（TFPI）的单克隆抗体适应症：用于血友病A或B患者的预防性治疗，尤其是那些伴有抑制物（inhibitors）的患者阶段：已上市或处于后期开发阶段Nedosiran (NN7022) 描述：一种小干扰RNA药物，靶向乳酸脱氢酶A (LDHA) 适应症：原发性高草酸尿症1型（PH1）阶段：已上市或处于后期开发阶段 Mim8 (NN7769) 描述：下一代FVIIIa模拟双特异性抗体适应症：血友病A阶段：临床3期开发阶段Etavopivat NN7535 描述：第二代PKR-激活剂小分子药物适应症：镰状细胞病（SCD）阶段：临床2期开发阶段Etavopivat NN7536描述：同为第二代PKR激活剂小分子药物，与NN7535具有相似作用机制适应症：地中海贫血阶段：临床2期开发阶段NDec NN7533 描述：是一种针对镰状细胞病和/或地中海贫血的疗法，该药物是一种口服组合疗法，由去甲基化剂 decitabine 和 tetrahydrouridine 组成。这种联合用药旨在改善患者的疾病状况，并可能通过改变骨髓中血红细胞生成的过程来减轻症状和并发症。适应症：镰状细胞病和地中海贫血阶段：临床2期开发阶段心血管代谢方向：Ziltivekimab (NN6018) 描述：一款针对IL-6活性的单克隆抗体适应症：慢性肾脏病（CKD）、动脉粥样硬化性心血管疾病（ASCVD）以及射血分数保留型心力衰竭 (HFpEF)阶段：临床3期开发阶段Ocedurenone NN6023描述：非甾体类选择性盐皮质激素受体拮抗剂（nsMRA），作为口服疗法用于心血管疾病（CVD）的治疗。适应症：心血管疾病阶段：临床3期开发阶段ATTR-CM NN6019描述：一种用于静脉注射的抗淀粉样蛋白免疫疗法。适应症：心血管疾病阶段：临床2期开发阶段CM4HF NN9003描述：一种用于恢复慢性心力衰竭患者心脏功能的试验性细胞疗法。适应症：心血管疾病阶段：临床2期开发阶段Anti-ANGPTL3 mAb NN6491描述：一种 ANGPTL3 中和扫抗体，用于每月一次的皮下注射治疗。适应症：心血管疾病阶段：临床2期开发阶段Semaglutide NN6535 ｜NN9931适应症：阿尔茨海默氏症和代谢功能障碍相关性脂肪性肝炎CagriSema NN9588 适应症：代谢功能障碍相关性脂肪性肝炎FGF21 NN9500描述：一种长效 FGF21 类似物，用于每周一次的皮下注射治疗。适应症：代谢功能障碍相关性脂肪性肝炎阶段：临床2期开发阶段LXRa NN6582描述：一种靶向 LXRa 的 siRNA，用于每月一次的皮下注射治疗。适应症：代谢功能障碍相关性脂肪性肝炎阶段：临床1期开发阶段MARC1 NN6581描述：一种靶向 MARC1 的 siRNA，用于每月一次的皮下注射治疗。适应症：代谢功能障碍相关性脂肪性肝炎阶段：临床1期开发阶段VAP-1i NN6561描述：一种用于每日一次口服治疗的 VAP-1 抑制剂。适应症：代谢功能障碍相关性脂肪性肝炎阶段：临床1期开发阶段SC4PD NN9001描述：一种用于改变疾病治疗的低温保存细胞疗法。适应症：帕金森病阶段：临床1期开发阶段STAT3 NN4002描述：GalXC 衍生脂质共轭物一次性皮下注射疗法。适应症：癌症阶段：临床1期开发阶段以上文章来源于GPCR drug discovery，作者段思研本文仅作信息交流之目的，文中观点不代表相关疾病治疗立场，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。

临床3期并购申请上市临床2期

2023-06-21

·Pharmaceutical Technology

CSL doses first patient with haemophilia B gene therapy Hemgenix

Aidan McNamee CSL’s Hemgenix uses an engineered adeno-associated virus (AAV) to make the body produce blood clotting proteins. Source: Shutterstock / Kateryna Kon. CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. At $3.5m per dose, it is the most expensive single-use gene therapy in the US. The high price tag may be hard to defend , but with lifetime treatment costs for haemophilia B reaching as high as $23m for some, the single-dose treatment could prove far more cost-effective. Despite this, the Institute for Clinical and Economic Review (ICER) claimed that a fairer price would be in the region of $2.9m. Recommended Reports Reports LOA and PTSR Model - (Decitabine + Tetrahydrouridine) in B-Cell Non-Hodgkin Lymphoma GlobalData Reports LOA and PTSR Model - Gene Therapy to Target CD22 for Relapsed and Refractory B-Cell Non-Hodgkin L... GlobalData View all Companies Intelligence UniQure NV Cytiva CSL Behring LLC Medicines and Healthcare products Regulatory Agency View all In the past year, Hemgenix has been granted approval by the US Food and Drug Administration (FDA) and has received conditional marketing authorisation from the European Commission (EC) and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA). This followed positive results from CSL’s Phase III HOPE-B trial (NCT03569891). In the study, Hemgenix reduced or eliminated the need for prophylactic treatment in 94% of patients. In a 20 June press release , CSL Behring’s North America general manager and senior vice-president Bob Lojewski said that the company was “thrilled” by this latest milestone and looked forward to more patients benefitting from the therapy. Hemgenix works by dosing a patient with an engineered adeno-associated virus (AAV), containing the gene responsible for producing a protein called factor IX. This protein helps to form blood clots and stop bleeding, with haemophilia B arising when the body is partially or completely unable to produce it. Once administered, the body will then be able to produce factor IX and prevent severe bleeds. Already a big player in haemophilia treatments, CSL licensed exclusive global rights to Hemgenix from gene therapy developers UniQure back in 2021. UniQure announced that it had received $100m from this first commercial sale milestone, on top of a $450m upfront payment. It is eligible for up to $1.6bn based on future milestones, as well as royalties amounting to more than 20% of net product sales. Haemophilia B is a rare condition. It predominantly affects males, with the CDC estimating 33,000 cases in males across the US. Despite the limited market, the price tags of haemophilia make it potentially lucrative. GlobalData forecasts predict that the global market for haemophilia A and haemophilia B treatments will grow to $14.7bn by 2030, up from $9.3bn in 2020 – an annual growth of nearly 5%. Gene therapies have long been predicted as the next big thing in haemophilia treatment, and are likely to drive this growth. GlobalData is the parent company of Pharmaceutical Technology. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

基因疗法临床3期临床结果上市批准细胞疗法

2022-09-01

·MedCity News

Novo Nordisk to buy sickle cell disease biotech Forma Therapeutics in $1.1B deal

Novo Nordisk announced a big purchase on Thursday to augment its rare disease portfolio. The Danish pharmaceutical giant is shelling out $20 per share in cash to acquire clinical-stage biotech Forma Therapeutics, which focuses on rare hematologic diseases. The deal represents a total equity value of $1.1 billion, about $425 million more than Forma’s current market cap. The transaction is expected to close before the end of the year. Forma made its market debut in 2020 with a $278 million initial public offering. The Massachusetts-based company’s lead drug candidate is etavopivat, a once-daily selective pyruvate kinase-R activator designed to improve anemia and red blood cell health in patients with sickle cell disease (SCD). SCD is an inherited disease that causes hemoglobin, the oxygen-carrying protein in red blood cells, to become rigid and misshapen. This makes normally round and flexible red blood cells rigid, and they take on a sickle shape. The clogging of these sickle-shaped blood cells impedes blood flow, as well as the delivery of oxygen to tissues. The condition leads to complications including vaso-occlusive crisis, which occurs when sickled red blood cells block blood flow to tissues thereby depriving them of oxygen. In a Phase 1 trial, etavopivat was shown to increase hemoglobin in SCD patients, improve their red blood cell health and decrease vaso-occlusive crises. The drug is currently being studied in a global Phase 2/3 trial involving patients with SCD, as well as a Phase 2 involving patients with transfusion-dependent SCD or another inherited hemoglobinopathy called thalassemia. The first interim data release for the Phase 2/3 is slated to take place before the end of the year. The acquisition is “by no means out of the blue,” Ludovic Helfgott, Novo’s executive vice president and head of rare disease, said in an interview. The deal signals Novo’s commitment to developing more products to treat hematological disorders, which Helfgott identified as an area with a large unmet need. Novo was attracted to etavopivat for a few reasons. The drug has demonstrated a good safety profile and efficacy during its clinical testing, and it is convenient for patients given that it needs to be taken only once daily. Novo also liked how flexible the drug can be — it can be used alone or in combination with other treatments, and it is currently being developed for use in pediatric patients as well as adults, according to Helfgott. This is not the first time Novo has made an acquisition to strengthen its SCD portfolio. In 2018, the company paid $400 million for Ohio-based EpiDestiny’s early stage SCD program. Together, Novo and EpiDestiny have built NDec — an oral, fixed-dose formulation of decitabine and tetrahydrouridine for the treatment of SCD. The compound is currently in clinical development, Helfgott said. Novo’s acquisition of Forma is the second billion-dollar SCD deal the pharmaceutical industry has seen in the last 30 days. Pfizer forked over $5.4 billion in August to buy Global Blood Therapeutics, a company with one commercialized therapy for sickle cell disease and a pipeline of additional drugs in various stages of development for the blood disorder.

IPO并购

100 项与四氢尿苷相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
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研发状态

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
恶性上皮肿瘤	临床2期	美国	National Cancer Institute	2018-04-11
食管癌	临床2期	美国	National Cancer Institute	2018-04-11
恶性胸膜间皮瘤	临床2期	美国	National Cancer Institute	2018-04-11
转移性非小细胞肺癌	临床2期	美国	The Case Comprehensive Cancer Center	2017-06-06
晚期非小细胞肺癌	临床2期	美国	National Cancer Institute	2009-08-20
膀胱癌	临床2期	美国	National Cancer Institute	2009-08-20
乳腺癌	临床2期	美国	National Cancer Institute	2009-08-20
头颈部肿瘤	临床2期	美国	National Cancer Institute	2009-08-20
肺癌	临床2期	美国	National Cancer Institute	2009-08-20
急性髓性白血病	临床2期	-	City of Hope National Medical Center	-

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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


NCT03233724 (CTgov)	临床1/2期	恶性胸膜间皮瘤 \| 食管癌 \| 转移性非小细胞肺癌 ... 更多	9	Decitabine+Tetrahydrouridine (Decitabine 0.2mg/kg + Tetrahydrouridine 10mg/kg Sunday, Monday, Tuesday (SuMT))	鏇繭鹽齋鏇積獵範遞獵 = 夢蓋繭鹽鹹餘壓觸願餘積淵膚糧觸積選鏇觸願 (獵憲選遞遞襯築齋窪簾, 範淵網憲衊蓋醖鹹鹹鹽 ~ 範衊簾壓鹹顧夢糧艱壓) 更多	-	2023-05-31
NCT03233724 (CTgov)	临床1/2期	恶性胸膜间皮瘤 \| 食管癌 \| 转移性非小细胞肺癌 ... 更多	9	Decitabine+Tetrahydrouridine (Decitabine 0.2mg/kg + Tetrahydrouridine 10mg/kg Monday-Tuesday (MT))	鏇繭鹽齋鏇積獵範遞獵 = 顧鑰製鑰選繭衊夢鹽齋積淵膚糧觸積選鏇觸願 (獵憲選遞遞襯築齋窪簾, 壓糧鏇艱鹹顧網襯夢夢 ~ 鏇膚獵鹽糧鬱鬱艱壓糧) 更多	-	2023-05-31
NCT02664181 (PRECISE \| P295, CTgov)	临床2期	非小细胞肺癌 \| 转移性非小细胞肺癌	13	Nivolumab (Nivolumab)	廠廠醖遞廠鹽夢繭製糧: P-Value = 0.05 更多	-	2020-12-10
NCT02664181 (PRECISE \| P295, CTgov)	临床2期	非小细胞肺癌 \| 转移性非小细胞肺癌	13	Nivolumab+Oral decitabine+Tetrahydrouridine (Oral THU/Decitabine + Nivolumab)	廠廠醖遞廠鹽夢繭製糧: P-Value = 0.05 更多	-	2020-12-10
NCT01479348 (CTgov)	早期临床1期	肺癌 \| 乳腺癌 \| 膀胱癌 ... 更多	5	Positron emission tomography (PET)/Computed tomography (CT)+Tetrahydrouridine intravenous (IV)+[F-18]-5-FLUORO-2'-DEOXYCYTIDINE	憲獵艱壓鹹膚襯廠鑰夢 = 廠齋鬱醖鹽壓鹹窪獵網艱築選齋鏇網衊獵窪顧 (窪鑰築積遞獵衊鹽構鏇, 衊積製網獵遞鹹窪窪選 ~ 齋範顧齋鏇鹹膚遞網願) 更多	-	2020-05-22
NCT00978250 (CTgov)	临床2期	肺癌 \| 乳腺癌 \| 非小细胞肺癌 ... 更多	95	Tetrahydrouridine (THU)+5-Fluoro-2'-Deoxycytidine (FdCyd)	願鹹廠範鹹餘繭築廠網 = 鹹醖範獵網願鏇醖壓獵範糧選廠鑰醖網簾襯網 (顧襯顧襯膚願膚夢糧廠, 製窪選鹽鬱膚衊構獵鏇 ~ 膚衊憲鏇衊襯鹽網願繭) 更多	-	2019-12-27
NCT00359606 (Pubmed \| Cancer Chemother Pharmacol) 人工标引	临床1期	实体瘤	-	tetrahydrouridine+FdCyd	顧夢醖範鹹觸蓋鹹積鏇(網構鏇範鏇遞觸鬱膚衊) = 100 mg/m2/day FdCyd with 350 mg/m2/day THU 艱築壓夢獵觸鹹淵網醖 (繭餘鬱窪獵鹹夢艱糧觸 ) 更多	积极	2015-03-01