A Phase 2 Trial Investigating the Addition of Pacritinib to a Hypomethylating Agent as a Bridge to Allogeneic Hematopoietic Stem Cell Transplant in Patients With Accelerated and Blast Phase Myeloproliferative Neoplasms

This phase II trial tests if adding pacritinib to standard of care azacitidine or decitabine increases the number of patients able to proceed to hematopoietic stem cell transplantation (bridging) for patients with accelerated and blast phase myeloproliferative neoplasms. Pacritinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Azacitidine and decitabine are in a class of medications called hypomethylation agents. They work by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Adding pacritinib to standard of care azacitidine or decitabine may increase the number of patients able to proceed to hematopoietic stem cell transplantation for patients with accelerated and blast phase myeloproliferative neoplasms.

开始日期2026-02-01

申办/合作机构

University of Washington

[+1]

NCT07033598

/ Not yet recruiting临床2期

A Randomized Phase 2 Study of Pacritinib vs. Hydroxyurea in Patients With Advanced Proliferative Chronic Myelomonocytic Leukemia

The goal of this clinical trial is to learn if pacritinib works better than hydroxyurea to treat advanced proliferative chronic myelomonocytic leukemia in adults. The main questions it aims to answer are:
* Does pacritinib improve disease control compared to hydroxyurea?
* What medical problems do participants have when taking pacritinib or hydroxyurea? Researchers will compare pacritinib to hydroxyurea to see if pacritinib is more effective and better tolerated in people with advanced proliferative chronic myelomonocytic leukemia.
Participants will be randomly assigned to receive either pacritinib twice a day or hydroxyurea for up to 48 weeks.
After treatment ends, participants will be followed for up to one year.

开始日期2025-12-01

申办/合作机构

Theradex (Europe) Ltd.

[+2]

NCT06675123

/ Recruiting临床1期IIT

A Pilot Study of Pacritinib Combined With a BTK Inhibitor in Patients With Relapsed/Refractory Mantle Cell Lymphoma

This phase I trial tests the safety and side effects of pacritinib in combination with a Bruton's tyrosine kinase (BTK) inhibitor and how well it works in treating patients with mantle cell lymphoma that has come back after a period of improvement (relapsed) or that has not responded to previous treatment (refractory). Pacritinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. BTK inhibitors block a protein called BTK which is present on B-cell (a type of white blood cell) cancers such as mantle cell lymphoma at abnormal levels. This may help keep tumor cells from growing and spreading. Giving pacritinib in combination with a BTK inhibitor may be safe, tolerable and/or effective in treating patients with relapsed or refractory mantle cell lymphoma.

开始日期2025-11-01

申办/合作机构

City of Hope National Medical Center

[+1]

100 项与帕克替尼相关的临床结果

登录后查看更多信息

100 项与帕克替尼相关的转化医学

登录后查看更多信息

100 项与帕克替尼相关的专利（医药）

登录后查看更多信息

244

项与帕克替尼相关的文献（医药）

2025-12-01·Current Hematologic Malignancy Reports

Emerging Therapeutic Approaches for Anemia in Myelofibrosis

Review

作者: Harrington, Patrick ; Palumbo, Giuseppe A ; Chifotides, Helen T ; Duminuco, Andrea ; Bose, Prithviraj ; Torre, Elena ; Vetro, Calogero

PURPOSE OF REVIEW:

In this review, we highlight conventional agents and novel emerging therapeutic strategies to treat anemia in MF.

RECENT FINDINGS:

Anemia is a common and challenging feature of myelofibrosis (MF). The pathobiology of anemia is multifactorial, including progressive bone marrow fibrosis, decreased erythropoiesis due to high hepcidin levels leading to iron sequestration in the reticuloendothelial system, hypersplenism, erythropoiesis inhibition by myelosuppressive JAK inhibitors (ruxolitinib, fedratinib), and others. MF-associated anemia has a negative impact on survival. Conventional agents to manage anemia include erythropoiesis-stimulating agents, danazol, corticosteroids, and immunomodulatory agents, but responses are infrequent and lack durability. Notable advancements have emerged in developing novel treatments for anemia in MF, including the regulatory approval of momelotinib (ACVR1/JAK1/2 inhibitor) in 2023 and development of novel promising agents targeting hemojuvelin and activins. Momelotinib and pacritinib (ACVR1/JAK2 inhibitor) are the preferred JAK inhibitors for patients with cytopenias (anemia, thrombocytopenia). Luspatercept and elritercept are activin receptor ligand traps, promoting erythroid maturation and late-stage erythropoiesis. Currently, luspatercept is being evaluated in a phase 3 trial (INDEPENDENCE™) for anemia in MF patients who are on a JAK2 inhibitor and require transfusions, and in a phase 2 trial (ODYSSEY) in combination with momelotinib in MF patients who are transfusion dependent, whether or not on a JAK inhibitor. Interim results of the RESTORE trial demonstrated that elritercept significantly decreased transfusions in MF patients. DISC-0974 is a first-in-class anti-hemojuvelin (positive hepcidin regulator) monoclonal antibody that decreased hepcidin expression, increased serum iron, and enhanced erythropoiesis in anemic patients with MF in a phase 1b/2 study. Burgeoning studies of novel anemia-targeted agents and combinations are significantly improving the quality of life and outcomes of patients with MF. The recent approval of momelotinib to treat MF with anemia and the emerging novel anemia-directed strategies in early and advanced clinical development have ushered in a new era in the treatment of MF-related anemia.

2025-10-01·Chinese Journal of Chromatography

Determination of 13 janus kinase inhibitors in anti-alopecia cosmetics by ultra-high performance liquid chromatography-tandem triple quadrupole composite linear ion trap mass spectrometry

Article

作者: Chen, Mao-Qin ; Wang, Hai-Bo ; Fan, Lei-Lei ; Yang, Qiu-Hong

According to the Safety and Technical Specification for Cosmetics （2015）， the addition of chemical drugs to cosmetics is strictly prohibited. Anti-alopecia cosmetics are often found to contain illegal additions of prohibited drugs such as minoxidil， finasteride and other substances. Ultra-high performance liquid chromatography-tandem triple quadrupole-linear ion trap mass spectrometry （UHPLC-Q-TRAP/MS） has become a powerful technology for the simultaneous detection of illegal ingredients in cosmetics due to its advantages of rapid analysis， high sensitivity， high throughput and high selectivity. An ultra-high performance liquid chromatography-multiple reaction monitoring-information dependent acquisition-enhanced production scanning （UHPLC-MRM-IDA-EPI） method was developed to determine 13 JAK inhibitors in anti-alopecia cosmetics， including baricitinib， tofacitinib， ritlecitinib， peficitinib， abrocitinib， upadacitinib， ivarmacitinib， fedratinib， filgotinib， ruxolitinib， momelotinib， pacritinib and bozitinib. The influence of extraction solvents was investigated. Approximately 0.2 g of the sample was weighed and placed in a 50 mL graduated centrifuge tube with a cap. Then， 2 mL of 0.1% （v/v） aqueous formic acid and 10 mL of acetonitrile were added， followed by vortexing for 1 min. Subsequently， the mixture was sonicated in an ultrasonic bath for 15 min. Added 0.5 g of sodium chloride， and the mixture was centrifuged at 8 000 r/min for 8 min at 5 °C. The supernatant was transferred to another 50 mL graduated centrifuge tube with a cap. The residue was added with 10 mL of acetonitrile and the extraction procedure was repeated. After high-speed centrifugation， the supernatant was combined. The combined supernatant was added with 0.2 g of sodium chloride， and the mixture was frozen at ‒20 °C for 1 h. It was centrifuged at 8 000 r/min for 5 min at 5 °C. The acetonitrile layer was collected and filtered through a 0.22 μm organic filter membrane. The initial filtrate was discarded， and the subsequent filtrate was collected as the sample solution. A C₁₈ column was used for chromatographic separation， enabling the successful separation of the analytes within 10 min. The mobile phase consisted of 0.1% （v/v） aqueous formic acid and acetonitrile， with gradient elution applied to optimize separation efficiency. The flow rate was set at 0.3 mL/min， and the column temperature was maintained at 40 ℃ to ensure consistent performance. A fixed injection volume of 2 μL was used to guarantee reproducibility and accuracy in the analysis. This configuration enabled the rapid and reliable separation of the target compounds. For the analysis of the 13 kinds of JAK inhibitors， an ESI source operating in positive ion mode was used with MRM-IDA-EPI detection. Quantification was performed by the external standard method using matrix-matched standard solutions. Good separation of the 13 JAK inhibitors was achieved under the optimized chromatographic conditions. Calibration curves were constructed by plotting the peak area of quantitative ions against the compound mass concentration. These curves exhibited excellent linearity within the investigated concentration range， with r all exceeding 0.996. The limits of detection （LODs） and limits of quantification （LOQs） of the 13 JAK inhibitors were 1.5-1.7 ng/g and 9.2-10.9 ng/g. To validate accuracy and precision， two cosmetic matrices （water-soluble and cream） were tested at three levels： LOQ， 2×LOQ and 10×LOQ. Recoveries of the 13 JAK inhibitors ranged from 94.7% to 102.2% for the water-soluble matrix and from 92.4% to 99.2% for the cream matrix， with relative standard deviations （RSDs） ≤8.8%. This method is characterized by high efficiency， rapidity， accuracy， sensitivity and simplicity， making it a powerful tool for rapid risk screening and simultaneous quantitative analysis of JAK inhibitors in anti-alopecia cosmetics.

2025-08-28·JOURNAL OF MEDICINAL CHEMISTRY

Discovery of Highly Selective and Potent Macrocyclic JAK2 Inhibitors for the Treatment of MPNs

Article

作者: Wu, Rui ; Yang, Tao ; Tian, Yang ; Cen, Xiaobo ; Chen, Lijuan ; Qin, Songhui ; Tang, Minghai ; Yan, Wei

The development of selective JAK2 inhibitors represents a promising therapeutic strategy for MPNs. Building upon the foundation of our first-generation JAK2 inhibitor, we employed macrocyclization-driven structural optimization to address its off-target limitations. This strategy yielded a novel class of macrocyclic JAK2 inhibitors, with compound 15au emerging as a standout candidate through systematic SAR studies. Compound 15au exhibits potent JAK2 inhibition (IC₅₀ = 2 nM), coupled with enhanced kinase selectivity, demonstrating 89.5-, 80.5-, and 51-fold selectivity over JAK1, JAK3, and TYK2, respectively. Mechanistic studies confirm its ability to suppress the JAK2/STAT5 signaling pathway. 15au demonstrates good pharmacokinetic properties, achieving high C_max and AUC_0-_t. These properties enable remarkable in vivo efficacy, where 15au outperforms both pacritinib and fedratinib in MPN models at lower doses. This work validates macrocyclization as an effective strategy for refining JAK2 inhibitors, simultaneously enhancing kinase selectivity, metabolic stability, and efficacy.

117

项与帕克替尼相关的新闻（医药）

2025-10-15

·智药邦

Commun Chem｜华东师范大学李洪林等：基于深度学习的大环化学空间探索新策略

2025年10月7日，华东理工大学药学院和华东师范大学药学院的李洪林教授/刁妍妍副研究员/朱丽丽教授/张凯教授研究团队在Nature旗下期刊《Communications Chemistry》发表最新成果（论文题目：Exploring the macrocyclic chemical space for heuristic drug design with deep learning models）。团队提出了创新性的大环分子生成模型CycleGPT，并结合逐级迁移学习策略与新型采样方法HyperTemp，首次将“大环结构修饰”转化为“大环化学空间探索”，显著缓解了长期困扰该领域的数据稀缺问题。在基于CycleGPT与CyclePred(JAK2活性预测模型)的前瞻性药物设计中，研究团队进一步验证了该方法的实用性，成功发现了一种兼具良好活性与激酶选择性的新型JAK2抑制剂候选物。在动物实验中，该分子在红细胞增多症模型中展现出显著的治疗潜力。研究背景大环类化合物通常含有十二元环及以上的环状结构，其结构介于小分子和生物抗体药类大分子之间，这些化学结构能够与蛋白质形成较大的接触界面，相比分子量相当的直链化合物，其往往具有更高的结合亲和力和更好的选择性。同时，它们的变色龙效应可提高药物的稳定性，并赋予其有利的药代动力学特征，因而近年来逐渐成为药物研发的热点。然而，大环药物的理性设计并不容易。其设计过程往往包括两个关键步骤：1.直链化合物大环化；2.大环结构修饰。目前已有若干计算方法针对线性化合物进行大环化，但在“如何基于已有活性大环进行结构修饰，以提升成药性或扩展临床候选分子库”这一问题上，仍缺乏有效手段。数据库中大环分子的比例极低（如ChEMBL中仅占1–2%），大环化学空间几乎是一片“未被照亮的领域”，研究者更多依赖药化经验与试错，这也进一步制约着大环药物的研发进程。研究内容 1.大环生成模型——CycleGPT CycleGPT是首个基于先导大环分子进行化学空间扩展，实现大环结构修饰的生成模型。通过引入一种渐进式迁移学习策略，模型能够从线性分子逐步迁移到具体靶点活性大环的学习任务，有效利用稀疏的大环数据，生成合理且多样的候选结构（图1）。图1 CycleGPT示意图 2.创新的采样策略HyperTemp 在分子生成领域中，采样算法和网络架构共同决定了生成SMILES的质量。本研究提出的HyperTemp sampling在Tempered sampling的基础上进一步优化，对生成token的概率分布进行更精细调整，在提高新颖性的同时保持生成大环的有效性。此外，本研究引入另外13种采样策略进行对比，这是首次针对大环分子生成采样方法的系统性探讨。 3.药物设计实例验证在JAK2靶点药物设计中，研究团队利用CycleGPT结合HyperTemp sampling以及JAK2预测模型CyclePred（图2），探索已知JAK2大环抑制剂的化学空间，获得具有高活性高选择性以及体内药效的备选大环化合物，优于上市药物。图2 CyclePred示意图研究结果 1.生成效果研究人员首先评价了大环有效性与新颖性方面的指标，包括Validity、Macrocycle_ratio以及novel_unique_macrocycles。novel_unique_macrocycles为综合指标，用于衡量生成的有效，独一且新颖的大环比例。结果可以看到，在生成novel_unique_macrocycles指标上，CycleGPT结合HyperTemp sampling(CycleGPT-HyperTemp)相比其他模型(同样在大环数据集上训练后进行分子生成)具备显著优势（表1）。表1 CycleGPT-HyperTemp与其他模型的对比 2.采样机制为了更全面地了解HyperTemp采样方法的特点和性能，研究人员以CycleGPT作为基础模型，并将HyperTemp替换为13种不同的采样策略，在novel_unique_macrocycles这一指标方面，HyperTemp采样方法在所有采样算法中表现最佳。此外，研究人员进一步分析了HyperTemp采样算法对生成token的影响。发现在更精细的概率调整后，HyperTemp相比Tempered sampling进一步减少了对最优token的偏好，并增强了对次优token的探索，从而提高了token采样的多样性并增强了新颖性（图3）。图3 HyperTemp采样性能 3.结构修饰能力为了评估方法在下游靶点应用中的能力，研究人员使用CycleGPT-HyperTemp方法扩展了大环化合物Loratinib的化学空间（图4）。可以看出，生成的大环化合物正确迁移到了Loratinib附近的化学空间。此外，从生成的示例分子可以看出，CycleGPT实现了(1)大环骨架跃迁，以及(2)大环取代基修饰。这与药物化学中的常见结构修饰方法一致，体现了CycleGPT方法所实现的结构修饰实用性。图4 Loratinib结构修饰大环的UMAP可视化示意图 4.药物验证在本研究中，研究人员利用CycleGPT从已知的3个JAK2大环化合物出发，在其周围的化学空间进行探索修饰，获得5000多个候选大环分子。通过CyclePred进行JAK2活性预测，以及对接模拟，最终挑选了6个备选分子进行合成验证。6个大环分子中有3个对JAK2的抑制活性达到了nM级别。活性最强的化合物2，IC50达到了1.17 nM，优于上市药物Fedratinib（表2）。表2 化合物1-6的结构及体外活性在激酶谱测试中，化合物2仅仅对JAK2在内的17种野生型激酶具备一定的活性，而Pacritinib和Fedratinib抑制的野生型激酶的数量为分别为55和34（图5）。这种更优的激酶选择性对控制药物的脱靶毒性来说，意义非凡。图5 激酶谱选择性测试此外，研究人员对化合物2进行动物实验验证。在红细胞增多症小鼠模型中，它的药效甚至优于更高剂量的对照药物。总结本研究提出的CycleGPT+HyperTemp+CyclePred框架，为大环化合物的结构修饰提供了全新思路： 1.方法学层面：CycleGPT引入渐进式迁移学习策略，将结构修饰问题转化为化学空间探索，创新性地缓解了长期困扰该领域的数据稀缺问题。HyperTemp采样算法提升了生成大环的新颖性，并可迁移应用于其他需要提升新颖性的序列生成任务中(例如RNA序列生成，蛋白序列生成，开放式文本生成等)。 2.工具价值：CycleGPT模型生成结果相比先导大环的修饰部分与药物化学常见修饰策略吻合，包括了大环骨架跃迁以及大环取代基修饰，展示了其较强的实用性。 3.应用前景：通过CycleGPT发现了具有优越活性与选择性的JAK2抑制剂候选物，并验证了其在体内红细胞增多症治疗中的潜在价值，体现了该方法在药物设计中的广阔应用前景。该工作不仅为大环类药物设计提供了可行路径，也为人工智能驱动的药物研发拓展了新方向。未来，随着方法进一步优化和扩展，AI有望成为点亮大环化学空间的关键引擎，加速创新药物的发现与发展。华东理工大学博士生胡枫、华东理工大学硕士生贾晓彤和华东理工大学硕士生廖文杰为本文共同第一作者，华东理工大学/华东师范大学药学院李洪林教授、刁妍妍副研究员、朱丽丽教授、张凯教授为该文章的通讯作者。该工作得到了国家重点研发计划（2022YFC3400501）、国家自然科学基金（82425104/82404517）等项目的资助。参考文献 Hu F, Jia X, Liao W. et al. Exploring the macrocyclic chemical space for heuristic drug design with deep learning models. Communications Chemistry. 2025, 8: 299. https://www.nature.com/articles/s42004-025-01686-w --------- End --------- 感兴趣的读者，可以添加小邦微信加入读者实名讨论微信群。添加时请主动注明姓名-企业-职位/岗位或姓名-学校-职务/研究方向。

2025-09-16

·医学新视点

命运给了他第二次机会，这一次，他让新药顺利上市！

编者按：血液癌症是一类起源于造血系统或淋巴系统的恶性肿瘤，包括白血病、淋巴瘤和多发性骨髓瘤等多个亚型，严重威胁全球公共健康。GLOBOCAN的统计数据显示，2022年全球血液系统恶性肿瘤新发病例超过120万例，死亡人数超过67万。过去十余年间，随着靶向疗法、免疫疗法和细胞疗法的不断涌现，血液癌症的治疗版图持续刷新，让越来越多患者在延长生命的同时获得更好的生活质量。疗法进步的背后，是科研人员的不懈探索与产业伙伴的高效协作。长期以来，药明康德始终凭借“一体化、端到端”的CRDMO平台，持续赋能合作伙伴在血液癌症新药研发中不断突破，加速创新成果落地，惠及全球患者。在这个血液癌症宣传月，本文将带您走进一款血液癌症创新疗法的开发故事，这是科学与产业合力的结晶，也是无数创新者为患者点亮希望的缩影。为患者带来拯救生命的新药，是每一位研发人员的梦想。约翰·胡德（John Hood）博士曾与这一梦想仅一步之遥。多年之后，他终于笑到了最后。故事要从十多年前说起。当时的约翰担任TargeGen公司的研究负责人。在他的带领下，公司发现了多款小分子激酶抑制剂，其中一款名为fedratinib的新药在临床试验中表现亮眼，显著改善了患者的生活。 ▲John Hood博士，现为Endeavor Biomedicines的首席执行官特蕾莎·布兰达（Theresa Blanda）女士便是受益者之一。她与白血病抗争多年，却在2008年的一次检查中被告知病情恶化，并出现了骨髓纤维化。那时，疤痕组织在她的骨髓中疯狂增生，严重影响了血细胞的生成。当时的最佳治疗方案是骨髓移植，但这需要找到合适的供体。在这方面，特蕾莎没有足够的运气。主治医生卡崔奥娜·贾米森（Catriona Jamieson）博士建议她参加fedratinib的临床试验。卡崔奥娜介绍说，这款新药能抑制人体内突变的JAK2蛋白激酶，从而阻断肿瘤细胞的生长。她的实验室先前进行的不少研究，已经证实了这一治疗思路的可行性。 “我正处在白血病全面爆发的边缘。”特蕾莎回忆道。白血病将她的造血干细胞挤出了骨髓外，引起的髓外造血让她的膝盖肿胀不堪。谁也没有想到，fedratinib带来了奇迹：在短短几个月，特蕾莎的病情出现了极大改善。“我的脾脏、肝脏、一切都恢复了正常，”她说：“我还重新开始了工作”。参加临床试验的5年里，特蕾莎的生命重新燃起了希望。她开始畅想未来，甚至萌生了与高中恋人再续前缘的念头。然而，2013年的一次突发变故，让这一切戛然而止。神秘的并发症 2010年，赛诺菲（Sanofi）看中了fedratinib的潜力，收购了TargeGen公司并将fedratinib推进至后期临床试验。特蕾莎参与的临床试验，正是其关键临床项目之一。新药研发人员很欣慰地看到，像特蕾莎这样的患者能够从白血病的魔爪下逃脱，重新回到正常的生活。乐观的分析师们也认为，这款新药离上市或许已经不远了。然而，后期数据却揭示了潜在风险：部分接受fedratinib治疗的患者出现韦尼克脑病（Wernicke’s encephalopathy）。这是一种与维生素B1缺乏相关的罕见神经疾病，患者大脑内的多个部分会出现病变，带来共济失调、精神异常等症状。 ▲Fedratinib的分子结构式（图片来源：PubChem）考虑到这一疾病的风险，美国FDA在2013年暂停了该临床试验的进行。赛诺菲仅隔一个周末就很快做出了回应，并决定终止7项正在进行的临床试验。像特蕾莎这样原本病情已得到控制的患者，不得不重新寻找新的治疗方法。许多人并没有找到解决方案。特蕾莎的病情很快出现复发和恶化。2016年，她离开了人世。卡崔奥娜医生说，许多在研药物都使用了类似的机制，但它们不如fedratinib有效。无数患者请求继续提供这款新药，好让他们的生命延续。重新出发患者的呼声传到了约翰耳中。作为fedratinib的共同发明人之一，约翰在赛诺菲收购TargeGen前就离开了这家公司。与几名朋友一道，他共同创立了一家名为Samumed的生物技术新锐并担任首席科学官，带领团队将多款候选新药推进至晚期临床。然而，他一直没有忘记曾经为fedratinib倾注的心血，而从特蕾莎与卡崔奥娜那里听到的故事也让他难过。“在2013年终止开发fedratinib的决定让许多患者心碎。当时，他们在临床试验中取得了非常积极的缓解。对这些患者来说，能够使用的治疗方案非常有限。对于大部分患者来说，除了fedratinib之外，几乎没有其他有效的药物。”约翰说道。思虑再三，约翰辞去了在Samumed的职位，选择重新出发。与卡崔奥娜一道，他们创立了一家名为Impact Biomedicines的新锐，目标是取回fedratinib的开发权，将它推进上市。与赛诺菲的沟通非常顺利。Impact获得了fedratinib的全球开发与商业化权益，赛诺菲没有向Impact收取任何先期付款。为了继续开发fedratinib，Impact聘请了一批具有丰富经验的新药研发人员，其中包括了来自于TargeGen的研发骨干。 ▲Fedratinib的研发里程碑（图片来源：参考资料[5]）约翰和他的团队同时也获得了fedratinib的更新临床数据，这包括了18项不同的研究，涉及877名患者。在一项名为JAKARTA-1的全球性3期关键临床试验中，fedratinib在24周的治疗后，显著减少了患者脾脏尺寸。在36%的受试者中，减少的幅度超过了35%（p<0.0001），达到了主要临床终点和次要临床终点。在随后进行的一项名为JAKARTA-2的临床试验里，研究人员进一步分析了对现有疗法产生耐受的患者，是否也能从fedratinib的治疗中受益。结果表明，fedratinib同样让55%的患者出现了脾脏尺寸缩小。另一方面，卡崔奥娜也在分析为何患者会出现韦尼克脑病这样怪异的不良事件。研究人员发现，在接受治疗时，癌症患者常因病情压力导致营养不良，而这是韦尼克脑病出现的原因。之前，研究人员没有检查患者的营养情况，因此导致了一系列不良反应的产生。若在治疗中关注营养补充，即可大幅降低风险。基于详尽的数据审查，FDA允许临床试验恢复。2018年1月，新基（Celgene）公司宣布以最高12.5亿美元的数额收购Impact，将fedratinib纳入其研发管线。最终，这款药物在2019年顺利通过FDA审评，成功上市。对于约翰来说，他终于成功实现了他的梦想——为患者带来了拯救生命的新药！创新永不止步约翰·胡德博士的故事，是药物研发人员将科学突破转化为创新疗法的缩影。过去20多年中，美国FDA已批准了至少86款血液肿瘤新疗法，涵盖小分子药物、抗体、多肽等多种药物类型。目前，全球血液肿瘤领域有超过1500款在研药物处于不同的临床开发阶段，其中小分子化学药物占比达三到四成；3期临床项目中，更有多款寡核苷酸和多肽偶联药物及靶向蛋白降解剂等创新治疗模式的身影，有望造福更多病患。作为全球医药创新的赋能者，药明康德很高兴能助力合作伙伴，加速多款治疗血液肿瘤的创新疗法问世，造福病患。长期以来，药明康德都在支持全球合作伙伴从药物研究（R）、开发（D）到商业化生产（M）各个阶段的需求，通过独特的一体化、端到端CRDMO模式，助力更多药物加速从实验室来到患者身边。以血液肿瘤的小分子药物为例，药明康德化学服务平台能支持各种化学药物的分子形式及类别，满足从药物发现到商业化生产各个阶段、各个规模的各种物料需求。药明康德生物学业务平台作为综合性的早期发现和转化生物学赋能平台，能为包括血液肿瘤在内的多个疾病领域的新药研发提供从早期发现到临床研究阶段的生物学解决方案。药明康德测试业务平台可为包括血液肿瘤在内多个疾病领域的药物提供全生命周期的一体化研发测试服务及全方位的临床研究服务，助力合作伙伴的药物成功申报IND、NDA以及通过核查上市。在这个血液癌症宣传月，我们致敬像约翰·胡德博士这样以坚持和智慧改变患者命运的科学家，也向所有奋战在临床一线的医生与研究人员致以敬意。血液癌症防治之路依旧充满挑战，但正是这些不懈努力，让越来越多的患者看到希望的曙光。药明康德将继续依托其“一体化、端到端”的CRDMO平台，加速合作伙伴的更多创新疗法问世，让科学的力量惠及每一位患者。 Accelerating the Fight Against Myelofibrosis: WuXi AppTec’s End-to-End Support for Next-Generation Therapies Myelofibrosis (MF), a rare and debilitating blood cancer, continues to impose severe burdens on patients worldwide. Although targeted therapies have advanced care in recent years, many patients still struggle with persistent symptoms, limited treatment durability, and few curative options. In this challenging landscape, WuXi AppTec partners with pharmaceutical and biotech companies around the globe to accelerate the discovery, development, and manufacturing of transformative therapies. With its fully integrated, end-to-end CRDMO (Contract Research, Development, and Manufacturing Organization) model, WuXi AppTec delivers seamless support across small molecules, immunotherapies, and emerging modalities—helping innovators move from early research through clinical and commercial development with speed and confidence. Unraveling the Burden of Myelofibrosis Myelofibrosis is a chronic myeloproliferative neoplasm characterized by progressive bone marrow fibrosis, impaired hematopoiesis, and a heavy symptom burden. Patients often experience fatigue, severe splenomegaly, weight loss, anemia, and night sweats, all of which significantly reduce quality of life. At the molecular level, mutations in genes such as JAK2, CALR, and MPL drive abnormal blood cell production and fuel chronic inflammation, creating a vicious cycle that accelerates disease progression. Despite therapeutic advances, myelofibrosis remains life-limiting. Approved JAK inhibitors—including ruxolitinib, fedratinib, pacritinib, and momelotinib—can shrink spleens and relieve symptoms, but they rarely alter the underlying disease biology. Over time, many patients develop resistance or intolerance, leaving them with few alternatives. Stem cell transplantation remains the only potentially curative option, yet it is often inaccessible due to age, comorbidities, and procedural risks. This reality underscores the urgent need for more effective therapies. Patients and caregivers seek treatments that not only provide symptom relief but also deliver durable benefits by addressing the root causes of the disease and meaningfully extending survival. Driving Innovation Through Science and Collaboration As a trusted partner to innovators worldwide, WuXi AppTec plays a vital role in advancing the next generation of therapies for hematologic malignancies, including myelofibrosis, through its integrated CRDMO platform. By combining scientific expertise with integrated global infrastructure, WuXi AppTec enables faster, more efficient progress across the entire drug development lifecycle. For small-molecule innovation, WuXi AppTec’s Chemistry Services Platform supports all categories of chemical compounds and production scales. This infrastructure helps partners meet material needs from lead optimization to clinical supply and beyond. Meanwhile, WuXi AppTec’s Biology Business Unit offers an integrated platform for early discovery and translational biology, empowering researchers to evaluate new targets and mechanisms in hematologic cancers with precision and speed. WuXi AppTec’s Testing Division enhances these capabilities with comprehensive R&D testing and clinical research services. From preclinical safety assessments to IND/NDA-enabling studies, the team helps streamline regulatory submissions and approval processes. This integrated model not only accelerates timelines but also enhances efficiency—helping transform innovative ideas into life-saving therapies. Shaping the Future of Myelofibrosis Treatment The treatment landscape for myelofibrosis is entering a period of rapid innovation. Beyond JAK inhibition, new approaches such as BET inhibitors, BCL-2 inhibitors, telomerase inhibitors, and antifibrotic agents are gaining momentum. Early clinical results suggest that combination strategies—pairing JAK inhibitors with epigenetic or antifibrotic therapies—may achieve deeper, more durable responses, offering the potential to alter the natural course of disease. Globally, many novel therapies are advancing through clinical pipelines, with many showing encouraging improvements in symptom control, spleen volume reduction, and even reversal of bone marrow fibrosis. These developments reflect a growing shift from palliative management toward true disease modification—and ultimately, the pursuit of curative solutions. WuXi AppTec is proud to stand alongside its global partners at the forefront of this dynamic landscape. By providing integrated, end-to-end CRDMO solutions, the company continues to empower the development of transformative therapies across a wide range of drug modalities. As we mark Blood Cancer Awareness Month this September, WuXi AppTec reaffirms its commitment to advancing innovative solutions that bring hope to patients with hematologic malignancies—working toward a future where these diseases are no longer life-limiting, but manageable, and ultimately curable. 题图来源：123RF 参考资料（可上下滑动查看） [1] Abandoned blood cancer drug resurrected by San Diego startup. Retrieved August 16, 2019, from https://www.sandiegouniontribune.com/business/biotech/sd-me-impact-fedratinib-20171013-story.html [2] Four years after it imploded at Sanofi, John Hood is resurrecting the myelofibrosis drug fedratinib. Retrieved August 16, 2019, from https://endpts.com/four-years-after-it-imploded-at-sanofi-john-hood-is-resurrecting-the-myelofibrosis-drug-fedratinib/ [3] Impact bags $22M to save ex-Sanofi drug from trial limbo. Retrieved August 16, 2019, from https://www.fiercebiotech.com/biotech/impact-bags-22m-to-save-ex-sanofi-drug-from-trial-limbo [4] Bray et al., (2024). Global cancer statistics 2022: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA: A Cancer Journal for Clinicians, https://doi.org/10.3322/caac.21834 [5] Mullally et al., (2000). Fedratinib in myelofibrosis. Blood Adv, https://doi.org/10.1182/bloodadvances.2019000954 [6] Sun et al., (2025). Global landscape and trends in lifetime risks of haematologic malignancies in 185 countries: population-based estimates from GLOBOCAN 2022. eClinicalMedicine, DOI: 10.1016/j.eclinm.2025.103193 [7] Loscocco and Guglielmelli. (2025). Targeted Therapies in Myelofibrosis: Present Landscape, Ongoing Studies, and Future Perspectives. Am J Hematol., doi: 10.1002/ajh.27658 免责声明：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：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，传递医学新知

免疫疗法并购细胞疗法

2025-09-03

·PRNewswire

Myelofibrosis Market is Going to Grow at a CAGR of 9% in the Next Ten Years (2025-2034) | DelveInsight

The launch of emerging therapies like BESREMi (PharmaEssentia and AOP Orphan Pharmaceuticals), INCB057643 (Incyte), XPOVIO (Karyopharm Therapeutics), RYTELO (Geron), REBLOZYL (Bristol Myers Squibb), Navtemadlin (Kartos Therapeutics), Pelabresib (Novartis), and others is going to shift the myelofibrosis market. LAS VEGAS, Sept. 3, 2025 /PRNewswire/ -- DelveInsight's Myelofibrosis Market Insights report includes a comprehensive understanding of current treatment practices, emerging myelofibrosis drugs, market share of individual therapies, and current and forecasted myelofibrosis market size from 2020 to 2034, segmented into leading markets (the US, EU4, UK, and Japan). Myelofibrosis Market Summary The total myelofibrosis treatment market size in the leading markets (the US, EU4, UK, and Japan) was USD 2.2 billion in 2024. The United States accounts for the largest market size of myelofibrosis, in comparison to EU4 (Germany, Italy, France, and Spain) and the UK, and Japan. Based on DelveInsight's assessment in 2024, the 7MM had approximately 56K patient pool (prevalent cases) of myelofibrosis. Key myelofibrosis companies, including PharmaEssentia, AOP Orphan Pharmaceuticals, Incyte, Karyopharm Therapeutics, Geron, Bristol Myers Squibb, Kartos Therapeutics, Novartis, Merck, Telios Pharma, Ryvu Therapeutics, Sumitomo Pharma, Syntara, Disc Medicine, Menarini Group, and others, are actively working on innovative myelofibrosis drugs. Some of the key myelofibrosis therapies in clinical trials include BESREMi (ropeginterferon alfa-2b-njft/P-1101), INCB057643, XPOVIO (NEXPOVIO/selinexor/KPT-330), RYTELO (imetelstat), REBLOZYL (luspatercept/ACE-536), Navtemadlin (KRT-232), Pelabresib (DAK539), Bomedemstat (IMG-7289/MK-3543), TL-895, RVU120 ( SEL-120), TP-3654 (nuvisertib), SNT-5505 (PXS-5505), DISC-0974, ELZONRIS (tagraxofusp/SL-401), and others. These novel myelofibrosis therapies are anticipated to enter the myelofibrosis market in the forecast period and are expected to change the market. By 2034, among all the therapies, the highest revenue is expected to be generated by OJJAARA/OMJJARA. Discover which myelofibrosis therapies are expected to grab the largest market share @ Myelofibrosis Market Report Key Factors Driving the Growth of the Myelofibrosis Market Novel Therapies Drive Myelofibrosis Treatment Advancements Currently, four JAK inhibitors have been approved by the US FDA for the treatment of myelofibrosis, including JAKAFI (ruxolitinib), INREBIC (fedratinib), VONJO (pacritinib), and OJJAARA (momelotinib). No drug therapy can cure myelofibrosis. As myelofibrosis primarily affects older adults, stem cell transplantation is not a treatment option for most myelofibrosis patients. The launch of another JAK inhibitor, BESREMi, will further change the dynamics of the myelofibrosis market. Rich clinical pipeline and emerging non-JAK approaches driving the myelofibrosis landscape Beyond JAK inhibitors, active clinical development includes BET inhibitors (Incyte's INCB057643; Novartis' Pelabresib), XPO1 inhibitor (Karyopharm Therapeutics' XPOVIO), Telomerase inhibitor (Geron's RYTELO), MDM2 protein inhibitor (Kartos Therapeutics' Navtemadlin), Tyrosine kinase inhibitors (Telios Pharma's TL-895), PIM1 kinase inhibitor (Sumitomo Pharma's TP-3654), LOX inhibitor (Syntara's SNT-5505), and others, raising market potential by promising new label expansions, second-line options, and higher-value therapies should any prove to be disease-modifying. An aging population & increasing incidence with better survival Myelofibrosis primarily affects older adults; population aging increases the absolute number of patients. As treatments improve symptoms and (in some cases) survival, prevalent patient pools grow, supporting longer-term market demand. In the US in 2024, the 70+ years of age group had the highest number of cases, accounting for approximately 60% of the total prevalent cases, while the ≤39 years of age group accounted for just ~2%. Diagnostic & genomic testing improvements are driving myelofibrosis patient pool, leading to the growth of myelofibrosis market Wider use of molecular profiling (JAK2, CALR, MPL, and others) enables earlier and more accurate MF diagnosis and better patient stratification for targeted therapies. This both expands the diagnosed population and helps match patients to appropriate, often higher-value, treatments. Myelofibrosis Market Analysis JAK inhibitors have become the cornerstone of treatment for patients with myelofibrosis, offering significant benefits such as spleen reduction, symptom relief, and improved quality of life, which can also extend survival in those with advanced disease. All approved JAK inhibitors primarily target JAK2, particularly the wild-type form, but they differ in their activity against other JAK family members. For instance, JAKAFI myelofibrosis inhibits both JAK1 and JAK2; INREBIC selectively inhibits JAK2 while sparing JAK1 and also affects FLT3 and other targets; VONJO myelofibrosis inhibits JAK2 while sparing JAK1 but additionally impacts FLT3, IRAK, and ACVR1; and OJJAARA, approved through a different pathway, targets JAK1/JAK2 and ACVR1, mainly for myelofibrosis patients with anemia. These varying mechanisms lead to distinct patient outcomes. JAKAFI myelofibrosis continues to see strong demand and is expected to grow further, maintaining its position as the standard of care in myelofibrosis. Myelofibrosis will remain the largest segment of JAKAFI's patient population until polycythemia vera cases eventually increase. However, market growth may be limited due to patent expirations of key therapies, with JAKAFI patents set to expire in 2027 for Novartis and 2028 for Incyte, presenting potential opportunities for competitors. In response, Incyte is exploring combination trials with novel drugs to extend JAKAFI's therapeutic lifespan. Learn more about the treatment options for myelofibrosis @ Myelofibrosis Therapy The myelofibrosis treatment landscape offers significant opportunities for innovation, focusing on several key areas: Treating Lower-Risk Patients: Developing safe and effective therapies for patients with lower-risk myelofibrosis, allowing for earlier and more favorable interventions. Enhancing First-Line Treatments: Improving existing first-line therapies for intermediate- to high-risk patients through novel drugs or combination strategies. Managing Cytopenia: Developing targeted therapies to address cytopenia, a significant unmet need in patient care. Myelofibrosis Competitive Landscape The myelofibrosis clinical trial pipeline includes several drugs in mid- and late-stage development that are expected to enter the market during the forecast period. The emerging landscape offers a diverse range of therapeutic alternatives for treatment, including XPOVIO (Karyopharm Therapeutics), Elitracet (Takeda/Keros Therapeutics), Navtemadlin (Kartos Therapeutics), Pelabresib (Novartis), INCB057643 (Incyte), Bomedemstat (Merck), and others, all of which are used in various lines of treatment. The expected launch of these therapies is expected to have a further positive impact on the market. INCB57643 is an orally administered small molecule. Bromodomain and extra-terminal (BET) proteins act as epigenetic readers that control the expression of key oncoproteins implicated in the development of myelofibrosis and other hematologic malignancies, including B-lymphoma-2, nuclear factor kappa, and c-Myc. In a prior Phase I/II clinical trial, the oral BET inhibitor INCB057643, tested both as a monotherapy and in combination with ruxolitinib, demonstrated favorable tolerability and promising clinical activity in patients with advanced cancers. In its Q2 2025 financial report, the company announced that the combination of INCB057643 with ruxolitinib and INCB57643 (a JAK1/JAK2 and BET inhibitor) is currently being evaluated in a Phase II trial for myelofibrosis. RYTELO (imetelstat) is an investigational telomerase inhibitor that targets telomerase, selectively eliminating malignant stem and progenitor cells in the bone marrow, which drive diseases like myelodysplastic syndromes (MDS) and myelofibrosis. By blocking the proliferation of these malignant cells, imetelstat supports the recovery of healthy bone marrow and blood cell production and has shown disease-modifying effects and clinical benefits in Phase III trials for myelofibrosis. This mechanism sets imetelstat apart from other approved or investigational therapies for these blood cancers. In January 2025, Geron reported achieving 75% enrollment in the Phase III IMpactMF trial, which is comparing imetelstat to Best Available Therapy (BAT) in intermediate-2 or high-risk myelofibrosis patients who have relapsed or are refractory to JAK inhibitor treatment. Elritercept is a late-stage investigational activin inhibitor aimed at treating anemia associated with hematologic malignancies, including MDS and myelofibrosis. It is currently undergoing Phase II evaluation in patients with myelofibrosis. In December 2024, Keros Therapeutics presented updated data from this ongoing Phase II trial at ASH 2024. Additionally, in December 2024, Takeda announced an exclusive licensing agreement with Keros to further develop, manufacture, and commercialize elritercept globally, excluding mainland China, Hong Kong, and Macau. The anticipated launch of these emerging myelofibrosis therapies are poised to transform the myelofibrosis market landscape in the coming years. As these cutting-edge myelofibrosis therapies continue to mature and gain regulatory approval, they are expected to reshape the myelofibrosis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about new myelofibrosis treatment, visit @ Myelofibrosis Treatment Market Recent Developments in the Myelofibrosis Market In July 2025, Incyte announced that the Phase I data in patients with myelofibrosis as monotherapy and in combination with ruxolitinib are anticipated in the second half of 2025. In June 2025, QIAGEN and Incyte announced a new global collaboration to develop a novel diagnostic panel to support Incyte's extensive portfolio of investigational therapies for patients with myeloproliferative neoplasms (MPNs), including Incyte's monoclonal antibody INCA033989. In January 2025, Karyopharm Therapeutics stated that it expects to report top-line results from the Phase III SENTRY trial in the second half of 2025, which could represent a potentially transformative opportunity to establish a new treatment paradigm in myelofibrosis. What is Myelofibrosis? Myelofibrosis is a rare blood cancer marked by the accumulation of scar tissue, or 'fibrosis', in the bone marrow. This excess scar tissue prevents the bone marrow from producing enough healthy blood cells. It belongs to a group of blood cancers called 'myeloproliferative neoplasms (MPNs)', in which the blood cells produced by the bone marrow grow and function abnormally. When myelofibrosis arises independently, without being caused by another bone marrow disorder, it is referred to as primary myelofibrosis. In other cases, it can develop from another MPN, such as polycythemia vera or essential thrombocythemia. When this occurs, it is called secondary myelofibrosis, sometimes specifically termed post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. Myelofibrosis Epidemiology Segmentation The myelofibrosis epidemiology section provides insights into the historical and current myelofibrosis patient pool and forecasted trends for the leading markets (the US, EU4, UK, and Japan). It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The myelofibrosis market report proffers epidemiological analysis for the study period 2020–2034 in the leading markets (the US, EU4, UK, and Japan) segmented into: Total Prevalent Cases of Myelofibrosis Type-specific Cases of Myelofibrosis Myelofibrosis Cases Based on Risk Stratification Age-specific Prevalent Cases of Myelofibrosis Myelofibrosis Cases Based on Molecular Alterations Download the report to understand which factors are driving myelofibrosis epidemiology trends @ Myelofibrosis Treatment Drugs Scope of the Myelofibrosis Market Report Myelofibrosis Therapeutic Assessment: Myelofibrosis current marketed and emerging therapies Myelofibrosis Market Dynamics: Conjoint Analysis of Emerging Myelofibrosis Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Myelofibrosis Market Unmet Needs, KOL's views, Analyst's views, Myelofibrosis Market Access and Reimbursement Discover more about myelofibrosis drugs in development @ Myelofibrosis Clinical Trials Table of Contents Related Reports JAK Inhibitors Market JAK Inhibitors Market Size, Target Population, Competitive Landscape, and Market Forecast – 2034 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key JAK inhibitors companies, including Pfizer, AbbVie, Galapagos, Sierra Oncology, Theravance Biopharma, Dizal Pharmaceutical, Aclaris Therapeutics, Celon Pharma, Incyte Corporation, Gilead Sciences, Reistone Biopharma, Jiangsu Hengrui Medicine Co., MaxiNovel Pharmaceuticals, among others. Myelofibrosis Clinical Trial Analysis Myelofibrosis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myelofibrosis companies, including Geron Corporation, Merck, Ryvu Therapeutics SA, Morphic Therapeutic, iOnctura, Pharmaxis, Nippon Shinyaku, Active Biotech, Incyte Corporation, Sumitomo Pharma America, Inc., Cellenkos, Disc Medicine, among others. Polycythemia Vera Market Polycythemia Vera Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key polycythemia vera companies, including Incyte, Novartis, PharmaEssentia, AOP Orphan Pharmaceuticals, Protagonist Therapeutics, Merck (Imago BioSciences), Italfarmaco, Ionis Pharmaceutical, Silence Therapeutics, Perseus Proteomics, AbbVie, Johnson & Johnson Innovative Medicine, Mabwell (Shanghai) Bioscience, Disc Medicine, GluBio Therapeutics, among others. Multiple Myeloma Market Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key multiple myeloma companies, including Sanofi, Karyopharm Therapeutics, AbbVie, Takeda Pharmaceutical, Celgene, Bristol-Myers Squibb, RAPA Therapeutics, Pfizer, Array Biopharma, Cellectar Biosciences, BioLineRx, Celgene, Aduro Biotech, ExCellThera, Janssen Pharmaceutical, Precision BioSciences, Takeda, Glenmark (Ichnos Sciences SA), Poseida Therapeutics, Molecular Partners AG, Chipscreen Biosciences, AbbVie, Genentech (Roche), Janssen Biotech, Nanjing Legend Biotech, Merck Sharp & Dohme Corp., among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Connect with us on LinkedIn | Facebook | Twitter Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床2期引进/卖出临床1期上市批准临床结果

100 项与帕克替尼相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D11768	帕克替尼	-	DB11697

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
原发性骨髓纤维化	美国	Sobi, Inc.	2022-02-28
血小板减少症	美国	Sobi, Inc.	2022-02-28

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
原发性血小板增多症后骨髓纤维化	临床3期	比利时	CTI BioPharma Corp.	2013-01-01
原发性血小板增多症后骨髓纤维化	临床3期	德国	CTI BioPharma Corp.	2013-01-01
原发性血小板增多症后骨髓纤维化	临床3期	荷兰	CTI BioPharma Corp.	2013-01-01
原发性血小板增多症后骨髓纤维化	临床3期	新西兰	CTI BioPharma Corp.	2013-01-01
真性红细胞增多症后骨髓纤维化	临床3期	比利时	CTI BioPharma Corp.	2013-01-01
真性红细胞增多症后骨髓纤维化	临床3期	德国	CTI BioPharma Corp.	2013-01-01
真性红细胞增多症后骨髓纤维化	临床3期	荷兰	CTI BioPharma Corp.	2013-01-01
真性红细胞增多症后骨髓纤维化	临床3期	新西兰	CTI BioPharma Corp.	2013-01-01
华氏巨球蛋白血症难治	临床2期	美国	Sobi, Inc.	2025-11-01
VEXAS 综合征	临床2期	美国	Swedish Orphan Biovitrum AB	2025-05-28

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ASCO2025	N/A	骨髓纤维化	148	Pacritinib	網齋蓋襯窪遞顧壓壓窪(糧鏇蓋網製簾願窪壓積) = 簾鑰構廠齋顧醖選衊鹽顧鑰鑰遞顧鹽積選窪艱 (襯襯網製襯糧鑰壓膚鏇, 27 ~ 84) 更多	积极	2025-05-30
NCT02055781 (PERSIST-2, ASCO2025)	临床3期	骨髓纤维化	-	Pacritinib	構淵築製餘築膚鹽選齋(範範齋壓窪網餘鏇選鹽) = 膚網簾鏇鑰鏇築網製觸構顧鑰廠廠範範鹹蓋壓 (製蓋願襯淵窪鹹網壓構 ) 更多	积极	2025-05-30
NCT02055781 (PERSIST-2, ASCO2025)	临床3期	骨髓纤维化	-	Best Available Therapy (BAT)	構淵築製餘築膚鹽選齋(範範齋壓窪網餘鏇選鹽) = 襯襯鬱簾願繭繭餘憲襯構顧鑰廠廠範範鹹蓋壓 (製蓋願襯淵窪鹹網壓構 ) 更多	积极	2025-05-30
ASCO2025	N/A	骨髓纤维化一线 \| 二线	212	Pacritinib	鑰廠夢鏇廠壓糧憲齋觸(膚鏇遞築襯糧觸觸簾網) = 壓醖獵衊製夢鬱鬱築觸構鏇繭製艱醖淵遞鹽鹹 (鹹蓋壓壓襯繭衊積選選, 61.5 ~ 75.3) 更多	积极	2025-05-30
PS1827 (EHA2025)	N/A	贫血 \| 血小板减少症 \| 骨髓纤维化一线	179	Pacritinib	繭構憲廠淵範齋鬱遞膚(範積鬱膚醖獵艱範蓋遞) = 簾範鏇衊繭夢築鬱鬱壓憲製獵鏇獵餘鏇網簾廠 (蓋艱積選範膚構壓壓構, 6.9 ~ 7.6) 更多	积极	2025-05-14
MY-PAC STUDY (EHA2025)	N/A	骨髓纤维化	35	Pacritinib (PAC)	淵繭製選積糧築築遞鏇(餘襯糧餘鑰網獵餘膚淵) = 鹹衊膚鏇構網艱淵選淵齋糧遞構簾觸願襯醖鏇 (鑰衊遞鏇窪鹹醖餘艱齋, -1.1% ~ 12.5%) 更多	-	2025-05-14
EHA2025	N/A	骨髓纤维化	90	Pacritinib	糧積憲製觸簾艱鏇窪餘(夢衊醖構簾繭鏇鏇鬱夢) = 憲構淵網願繭網鹹鏇簾餘淵醖選鹹膚齋憲窪範 (製鏇廠願憲選蓋壓構範, 7.9 ~ 10.6) 更多	-	2025-05-14
PF1242 (EHA2025)	临床3期	骨髓纤维化	-	Pacritinib 200 mg BID	窪廠蓋鬱鑰糧獵窪觸夢(廠衊繭廠蓋壓繭鹹衊鹽) = The MAIC did not indicate statistically significant differences between PAC and MMB for the outcomes evaluated, though all endpoints favoured pacritinib 築網鏇獵觸範餘壓積餘 (範願鏇選鹹壓鏇憲糧構 ) 更多	-	2025-05-14
PF1242 (EHA2025)	临床3期	骨髓纤维化	-	Momelotinib 200 mg QD		-	2025-05-14
NCT04635059 (BLAST, CTgov)	临床2期	前列腺癌 \| 非转移性前列腺癌 \| 前列腺腺癌	6	Pacritinib	鏇齋餘繭糧構構壓窪製 = 鹹鑰壓製廠鏇築鑰壓窪觸範廠膚願淵網選獵齋 (醖艱壓憲製繭鏇齋襯獵, 艱鏇積壓繭鑰鏇醖齋淵 ~ 觸艱積膚憲遞醖糧遞夢) 更多	-	2024-12-17
NCT01773187 \| NCT02055781 \| NCT04884191 (PERSIST-1, PERSIST-2, PAC203, ASH2024)	临床2/3期	serum albumin \| cholesterol	484	Pacritinib 400 mg QD	鏇網繭選襯網選顧醖餘(夢鏇構餘網鬱壓築齋顧) = 顧衊壓醖衊獵廠淵憲範鬱獵積醖膚鹽襯蓋膚顧 (製膚夢獵廠鹽鏇夢鬱範, -1.3 ~ +3.14)	积极	2024-12-08
ASH2024	N/A	骨髓纤维化	-	Pacritinib	遞獵壓願獵獵觸夢製遞(齋願廠願襯醖觸網範鏇) = 選夢簾製鑰餘夢夢觸淵觸淵製積鏇顧膚獵遞鏇 (遞糧鹹遞築觸觸憲蓋壓, 63.2 ~ 99.0) 更多	-	2024-12-08