Efficacy and Safety of Ropeginterferon Alfa 2b (P1101) for Patients With Polycythemia Vera - A Randomized Open Label Global Multicenter Study (PARADIGM-PV)

This is a randomized, open-label, multicenter, two-arm study to assess the efficacy and safety of ropeginterferon alfa-2b for patients with PV. The entire study period is 60 weeks, including a main treatment phase (32 weeks), an extension treatment phase (24 weeks), and a safety follow-up phase (four weeks). However, the study may be extended for additional period of treatment after Week 60 pending the primary endpoint analysis at Week 32. Approximately 70 patients with PV will be enrolled.

开始日期2026-02-01

申办/合作机构

药华医药股份有限公司

NCT07047885

/ Recruiting临床1期IIT

A Phase I/IB Study of Ropeginterferon in Patients With Cutaneous T-Cell Lymphoma (CTCL)

This is a single-center, phase I/IB study to identify the recommended phase II dose of Ropeginterferon-alfa 2b (P1101) in patients with CTCL who have failed at least two prior lines of skin-directed therapy (Stage IA-IB) or have less than a complete response to phototherapy or extracorporeal photopheresis (ECP) or total skin electron beam therapy (TSET), or stable/progressive disease after at least two lines of topical therapy (Stage IIA-IIIB).

开始日期2025-08-27

申办/合作机构

H. Lee Moffitt Cancer Center & Research Institute, Inc.

[+1]

NCT06468033

/ Recruiting临床3期

A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase III Study to Assess Efficacy and Safety of Ropeginterferon Alfa-2b (P1101) in Adult Patients With Pre-fibrotic/Early Primary Myelofibrosis (PMF) or Overt PMF at Low or Intermediate-1 Risk According to DIPSS Plus (HOPE-PMF): The Core Study and Its Extension Study

This is a phase 3 double-blind clinical trial arm to test Ropeginterferon alfa-2b (P1101) in adult patients with Primary Myelofibrosis (PMF) at early stage or low to medium risk.
Participants will receive the study drug/placebo bi-weekly and have an assessment visit every 4 weeks. The ratio of study drug to placebo group is 2:1.

开始日期2025-07-18

申办/合作机构

药华医药股份有限公司

100 项与罗培干扰素α-2b 相关的临床结果

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100 项与罗培干扰素α-2b 相关的转化医学

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100 项与罗培干扰素α-2b 相关的专利（医药）

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项与罗培干扰素α-2b 相关的文献（医药）

2025-12-01·Current Hematologic Malignancy Reports

Highlights from MPN Asia 2025: Advances in Molecular Pathogenesis and Therapeutic Strategies in Myeloproliferative Neoplasms

Review

作者: Yacoub, Abdulraheem ; Li, Bing ; Mascarenhas, John O ; Duan, Minghui ; Kiladjian, Jean-Jacques ; Hou, Hsin-An ; Hobbs, Gabriela S ; Lee, Sung-Eun ; Bose, Prithviraj ; Rampal, Raajit ; Tashi, Tsewang ; Qin, Albert ; Gill, Harinder ; Huang, Jian ; Hasselbalch, Hans C ; Mesa, Ruben A ; Kirito, Keita ; Yu, Lennex Hsueh-Lin ; Xiao, Zhijian ; Shimoda, Kazuya ; Prchal, Josef T

PURPOSE OF REVIEW:

This report summarizes key insights from the 8th Annual International Symposium on Myeloproliferative Neoplasms (MPN Asia 2025). The symposium brought together global experts to discuss advancements in MPN biology, diagnostics, and therapeutics, with a focus on emerging molecular understanding, novel treatment strategies and real-world data.

RECENT FINDINGS:

Molecular profiling has become essential in MPN risk stratification and therapeutic decision-making. High-risk mutations (e.g., ASXL1, TP53) and inflammatory pathways (e.g., IL-17, NF-κB) were shown to correlate with disease progression and transformation. Interferon-based therapy is increasingly used in younger, low-risk, or treatment-naïve patients, and is also being investigated in myelofibrosis and essential thrombocythemia. Ropeginterferon alfa-2b, a novel interferon-based therapy, demonstrated durable clinical efficacy in polycythemia vera. Its high initial-dose and accelerated titration (HIDAT) regimen led to fast achievement of complete hematologic response, rapid reductions in JAK2V617F allele burden, and high complete molecular response rate. Combination regimens involving ruxolitinib and agents such as pelabresib, selinexor, and interferon showed potential for enhanced efficacy. Population-based studies from Asia contributed regional epidemiological and treatment data, reinforcing the role of real-world evidence. Modern prognostic models such as MIPSS70+ v2.0 and GIPSS were discussed for more precise risk prediction. Preliminary findings also suggest ropeginterferon alfa-2b may be a safe option during pregnancy. MPN Asia 2025 highlighted the growing role of molecular diagnostics and targeted therapeutics in the management of MPNs. Ropeginterferon alfa-2b has emerged as a therapeutic potential across the MPN spectrum. Its early use and personalized strategies are increasingly recognized. Real-world data and regional insights are shaping a more nuanced, globally informed approach to MPN care.

2025-11-01·LEUKEMIA RESEARCH

Low-dose ropeginterferon alfa-2b and peginterferon alfa-2a have comparable efficacy and tolerability in polycythemia vera

Letter

作者: Silver, Richard T ; Rahman, Aqib Abdul ; Erdos, Katie ; Abu-Zeinah, Ghaith ; Scandura, Joseph M ; Lee, Neville

2025-11-01·Lancet Haematology

Ropeginterferon alfa-2b in hydroxyurea-intolerant or hydroxyurea-refractory essential thrombocythaemia (SURPASS ET): a multicentre, open-label, randomised, active-controlled, phase 3 study

Article

作者: Chen, Suning ; Xiao, Zhijian ; Zhang, Sujiang ; Shirane, Shuichi ; Tashi, Tsewang ; Zhang, Lei ; Zuo, Xuelan ; Sato, Toshiaki ; Mascarenhas, John ; Hu, Yu ; Lin, Sheena ; Lim, Ken-Hong ; Lee, Sung-Eun ; Masarova, Lucia ; Gill, Harinder ; Ohishi, Kohshi ; Shih, Lee-Yung ; Maze, Dawn ; Bae, Sung Hwa ; Wang, Yi ; Shimoda, Kazuya ; Teo, Winnie Z Y ; Oh, Stephen T ; Komatsu, Norio ; Takenaka, Katsuto ; Qin, Albert ; Bose, Prithviraj ; Kirito, Keita ; Zagrijtschuk, Oleh ; Ichii, Michiko ; Shih, Weichung Joe ; Jin, Jie ; Xu, Na ; Mesa, Ruben

BACKGROUND:

The initial therapy for high-risk essential thrombocythaemia is usually hydroxyurea, but about a third of patients develop intolerance or resistance. A standard second-line agent has been anagrelide. Ropeginterferon alfa-2b, a new-generation interferon-based therapy, is approved for polycythaemia vera. We aimed to assess efficacy and safety of ropeginterferon alfa-2b compared with anagrelide in patients with essential thrombocythaemia with leukocytosis who are intolerant or resistant to hydroxyurea.

METHODS:

The SURPASS ET open-label, randomised, active-controlled, phase 3 trial was done at 55 clinical sites across China, Japan, Taiwan, Hong Kong, South Korea, the USA, Singapore, and Canada and enrolled patients aged 18 years and older with high-risk (age >60 years with JAK2 Val617Phe or a history of disease-related thrombosis or haemorrhage), hydroxyurea-intolerant or hydroxyurea-resistant essential thrombocythaemia and white blood cell (WBC) count greater than 10 × 10⁹ cells/L. Patients were randomly assigned (1:1) to ropeginterferon alfa-2b or anagrelide, stratified by platelet count, symptom score, and country. Ropeginterferon alfa-2b was subcutaneously dosed every 2 weeks, initially at 250 μg, then titrated to 350 μg at week 2, and to 500 μg from week 4 onward. Anagrelide was orally dosed according to the US Food and Drug Administration-approved prescribing information. The primary endpoint was the rate of response at months 9 and 12, as per modified European LeukemiaNet (ELN) criteria. The main planned analysis for the study was done in the intention-to-treat population. The trial is registered at ClinicalTrials.gov, NCT04285086 and is completed, and an extension study for collecting long-term data is ongoing.

FINDINGS:

Between Aug 25, 2020, and Nov 12, 2024, 245 patients were screened and 174 were randomly assigned (91 to ropeginterferon alfa-2b and 83 to anagrelide). The median follow-up was 12·5 months (IQR 11·5-12·9). At baseline, 47 (52%) of 91 participants in the ropeginterferon alfa-2b group and 44 (53%) of 83 participants in the anagrelide group were female. 167 (96%) of 174 participants were Asian and seven (4%) were White. The trial met its primary endpoint, with 39 (43%) of 91 participants in the ropeginterferon alfa-2b group showing durable modified ELN criteria responses at months 9 and 12, compared with five (6%) of 83 participants in the anagrelide group. This difference (36·5%, 95% CI 25·4-47·7) was significant (p=0·0001). Grade 3 or worse treatment-emergent adverse events occurred in 27 (34%) of 80 patients in the anagrelide group and 21 (23%) of 91 patients in the ropeginterferon alfa-2b group. In the ropeginterferon alfa-2b group, the most common grade 3 or worse adverse events were infections and infestations, occurring eight (9%) of 91 patients, compared with five (6%) of 80 patients in the anagrelide group. In the anagrelide group, the most frequent grade 3 or worse adverse events were nervous system disorders, occurring in six (8%) of 80 patients, compared with one (1%) of 91 patients with ropeginterferon alfa-2b. Serious adverse events occurred in 24 (30%) of 80 participants in the anagrelide group and 13 (14%) of 91 participants in the ropeginterferon alfa-2b group). The most common serious adverse event was cerebral infarction, which occurred in four (5%) of 80 patients in the anagrelide group but was not observed in the ropeginterferon alfa-2b group. There were no treatment-related deaths in either study group.

INTERPRETATION:

Our findings suggest that ropeginterferon alfa-2b could be considered as a second-line treatment option for patients with essential thrombocythaemia and leukocytosis.

FUNDING:

PharmaEssentia.

项与罗培干扰素α-2b 相关的新闻（医药）

2025-11-02

·生命诗筏

解码生物治疗：当人体成为对抗疾病的“智慧工厂”

第一章开场白：当医学突破告别 “一刀切”，生物治疗为何成新希望？在人类与疾病抗争的漫长历史中，手术、化疗、放疗曾是对抗重症的 “三驾马车”，但 “杀敌一千，自损八百” 的局限，让无数患者在治疗中承受着身心双重煎熬。如今，随着生命科学的飞速发展，一种名为 “生物治疗” 的新型疗法正悄然改写医学版图 —— 它不再依赖外力强行清除病灶，而是唤醒人体自身的免疫细胞，或是借助基因工程改造的 “生物武器”，精准狙击病魔。据《自然・医学》2024 年全球生物治疗报告显示，过去五年全球生物治疗市场规模年均增速达 18.7%，2023 年更是突破 4800 亿美元，越来越多的患者正通过这种 “智慧疗法” 重获新生。今天，我们就一起揭开生物治疗的神秘面纱，看看它如何让医学从 “对抗” 走向 “协同”。第二章免疫治疗：唤醒体内 “健康卫士”，让癌细胞无处遁形提及生物治疗，免疫治疗无疑是最受关注的领域之一。我们的免疫系统本就像一支训练有素的 “军队”，免疫细胞便是其中的 “士兵”，负责识别并清除癌细胞等 “入侵者”。但狡猾的癌细胞会通过表达 PD - L1 蛋白等方式，与免疫细胞表面的 PD - 1 受体结合，就像给免疫细胞戴上 “枷锁”，使其失去战斗力。而免疫检查点抑制剂的出现，打破了这一僵局。以帕博利珠单抗为例，《新英格兰医学杂志》2023 年发表的 Ⅲ 期临床试验数据显示，在晚期非小细胞肺癌患者中，接受帕博利珠单抗联合化疗治疗的患者，中位生存期达 24.8 个月，较单纯化疗组延长了 8.3 个月，5 年生存率更是提升至 31.9%，远超传统疗法的 15.6%。除了免疫检查点抑制剂，CAR - T 细胞治疗也展现出惊人潜力。2024 年美国临床肿瘤学会（ASCO）年会上公布的数据显示，针对复发难治性弥漫大 B 细胞淋巴瘤患者，阿基仑赛注射液治疗后的客观缓解率达82.5%，完全缓解率为 58.4%，部分患者甚至实现了长期无瘤生存，真正实现了 “细胞抗癌” 的突破。第三章基因治疗：修复 “生命密码”，攻克遗传病与罕见病如果说免疫治疗是 “唤醒士兵”，那么基因治疗就是直接 “修复生命蓝图”。当人体基因出现缺陷或突变时，就可能引发遗传病、罕见病等疑难病症。基因治疗通过病毒载体、基因编辑等技术，将正常基因导入患者体内，或修正突变基因，从根源上治愈疾病。以脊髓性肌萎缩症（SMA）为例，这是一种因 SMN1 基因缺失导致的罕见遗传病，患儿常因肌肉无力失去行走能力，甚至危及生命。2023 年《柳叶刀・神经病学》发表的长期随访研究显示，接受onasemnogene abeparvovec 基因治疗的 SMA 患儿，在治疗后 5 年，92% 的患儿能够独立行走，83% 的患儿无需依赖呼吸支持，而未接受治疗的传统组患儿，仅12% 能实现独立行走。在基因编辑领域，CRISPR 技术更是让精准治疗成为可能。2024 年《科学》杂志报道，美国研究团队利用 CRISPR - Cas9 技术，成功修正了镰状细胞贫血患者造血干细胞中的基因突变，在接受治疗的 25 名患者中，23 名患者的血红蛋白水平恢复正常，随访 18 个月无严重不良反应，这一成果为遗传病治疗开辟了全新路径。第四章细胞因子与抗体药物：精准靶向病灶，降低治疗副作用除了免疫治疗和基因治疗，细胞因子疗法与单克隆抗体药物也是生物治疗的重要组成部分。细胞因子是免疫系统分泌的 “信号分子”，能调节免疫细胞活性，增强免疫应答。比如干扰素 α - 2b，在慢性乙型肝炎治疗中，《中华肝脏病杂志》2023 年发布的多中心研究显示，使用干扰素 α - 2b 治疗 48 周的患者，乙肝表面抗原清除率达 12.3%，较核苷类似物治疗组提升了 7.8 个百分点，且停药后持续应答率更高，有效降低了肝硬化和肝癌的发生风险。单克隆抗体药物则像 “精准导弹”，能特异性结合病灶部位的抗原，精准杀伤病变细胞。以曲妥珠单抗为例，它专门针对 HER2 阳性乳腺癌细胞，《临床肿瘤学杂志》2024 年的数据显示，HER2 阳性早期乳腺癌患者接受曲妥珠单抗辅助治疗后，复发风险降低 34%，死亡风险降低 26%，10 年无病生存率达 73.7%，显著改善了患者预后。与传统化疗药物相比，这些生物制剂因靶向性强，对正常细胞损伤小，副作用发生率大幅降低，患者生活质量得到明显提升。第五章结尾：生物治疗的未来，离我们并不遥远从免疫治疗让癌症患者长期生存成为可能，到基因治疗为遗传病患者点亮希望，再到抗体药物精准降低治疗副作用，生物治疗正以惊人的速度改变着疾病治疗的格局。当然，它并非完美无缺，部分疗法存在价格较高、适应症有限等问题，但随着技术的不断突破和医保政策的逐步覆盖，这些障碍正在被一一打破。据《全球生物治疗发展白皮书（2024）》预测，到 2030 年，全球将有超过 300 种生物治疗产品上市，覆盖癌症、遗传病、自身免疫病等多个领域，惠及数十亿患者。生物治疗的时代已经来临，它不仅是医学科技的奇迹，更是人类对抗疾病的新希望。如果这篇文章让你对生物治疗有了新的认识，不妨分享给身边的人，让更多人了解这份 “健康守护力量”，共同期待医学带来的更多惊喜。

免疫疗法ASCO会议临床3期基因疗法临床结果

2025-11-02

·今日头条

解码生物治疗：当人体成为对抗疾病的“智慧工厂” 第一章开场白：当医学突破告别 “一刀切”，生物治疗为何成新希望？在人类与疾病抗争的漫长历史中，手术、化疗、放疗曾是对抗重症的 “三驾马车”，但 “杀敌一千，自损八百” 的局限，让无数患者在治疗中承受着身心双重煎熬。如今，随着生命科学的飞速发展，一种

解码生物治疗：当人体成为对抗疾病的“智慧工厂” 第一章开场白：当医学突破告别 “一刀切”，生物治疗为何成新希望？在人类与疾病抗争的漫长历史中，手术、化疗、放疗曾是对抗重症的 “三驾马车”，但 “杀敌一千，自损八百” 的局限，让无数患者在治疗中承受着身心双重煎熬。如今，随着生命科学的飞速发展，一种名为 “生物治疗” 的新型疗法正悄然改写医学版图 —— 它不再依赖外力强行清除病灶，而是唤醒人体自身的免疫细胞，或是借助基因工程改造的 “生物武器”，精准狙击病魔。据《自然・医学》2024 年全球生物治疗报告显示，过去五年全球生物治疗市场规模年均增速达 18.7%，2023 年更是突破 4800 亿美元，越来越多的患者正通过这种 “智慧疗法” 重获新生。今天，我们就一起揭开生物治疗的神秘面纱，看看它如何让医学从 “对抗” 走向 “协同”。第二章免疫治疗：唤醒体内 “健康卫士”，让癌细胞无处遁形提及生物治疗，免疫治疗无疑是最受关注的领域之一。我们的免疫系统本就像一支训练有素的 “军队”，免疫细胞便是其中的 “士兵”，负责识别并清除癌细胞等 “入侵者”。但狡猾的癌细胞会通过表达 PD - L1 蛋白等方式，与免疫细胞表面的 PD - 1 受体结合，就像给免疫细胞戴上 “枷锁”，使其失去战斗力。而免疫检查点抑制剂的出现，打破了这一僵局。以帕博利珠单抗为例，《新英格兰医学杂志》2023 年发表的 Ⅲ 期临床试验数据显示，在晚期非小细胞肺癌患者中，接受帕博利珠单抗联合化疗治疗的患者，中位生存期达 24.8 个月，较单纯化疗组延长了 8.3 个月，5 年生存率更是提升至 31.9%，远超传统疗法的 15.6%。除了免疫检查点抑制剂，CAR - T 细胞治疗也展现出惊人潜力。2024 年美国临床肿瘤学会（ASCO）年会上公布的数据显示，针对复发难治性弥漫大 B 细胞淋巴瘤患者，阿基仑赛注射液治疗后的客观缓解率达 82.5%，完全缓解率为 58.4%，部分患者甚至实现了长期无瘤生存，真正实现了 “细胞抗癌” 的突破。第三章基因治疗：修复 “生命密码”，攻克遗传病与罕见病如果说免疫治疗是 “唤醒士兵”，那么基因治疗就是直接 “修复生命蓝图”。当人体基因出现缺陷或突变时，就可能引发遗传病、罕见病等疑难病症。基因治疗通过病毒载体、基因编辑等技术，将正常基因导入患者体内，或修正突变基因，从根源上治愈疾病。以脊髓性肌萎缩症（SMA）为例，这是一种因 SMN1 基因缺失导致的罕见遗传病，患儿常因肌肉无力失去行走能力，甚至危及生命。2023 年《柳叶刀・神经病学》发表的长期随访研究显示，接受 onasemnogene abeparvovec 基因治疗的 SMA 患儿，在治疗后 5 年，92% 的患儿能够独立行走，83% 的患儿无需依赖呼吸支持，而未接受治疗的传统组患儿，仅 12% 能实现独立行走。在基因编辑领域，CRISPR 技术更是让精准治疗成为可能。2024 年《科学》杂志报道，美国研究团队利用 CRISPR - Cas9 技术，成功修正了镰状细胞贫血患者造血干细胞中的基因突变，在接受治疗的 25 名患者中，23 名患者的血红蛋白水平恢复正常，随访 18 个月无严重不良反应，这一成果为遗传病治疗开辟了全新路径。第四章细胞因子与抗体药物：精准靶向病灶，降低治疗副作用除了免疫治疗和基因治疗，细胞因子疗法与单克隆抗体药物也是生物治疗的重要组成部分。细胞因子是免疫系统分泌的 “信号分子”，能调节免疫细胞活性，增强免疫应答。比如干扰素 α - 2b，在慢性乙型肝炎治疗中，《中华肝脏病杂志》2023 年发布的多中心研究显示，使用干扰素 α - 2b 治疗 48 周的患者，乙肝表面抗原清除率达 12.3%，较核苷类似物治疗组提升了 7.8 个百分点，且停药后持续应答率更高，有效降低了肝硬化和肝癌的发生风险。单克隆抗体药物则像 “精准导弹”，能特异性结合病灶部位的抗原，精准杀伤病变细胞。以曲妥珠单抗为例，它专门针对 HER2 阳性乳腺癌细胞，《临床肿瘤学杂志》2024 年的数据显示，HER2 阳性早期乳腺癌患者接受曲妥珠单抗辅助治疗后，复发风险降低 34%，死亡风险降低 26%，10 年无病生存率达 73.7%，显著改善了患者预后。与传统化疗药物相比，这些生物制剂因靶向性强，对正常细胞损伤小，副作用发生率大幅降低，患者生活质量得到明显提升。第五章结尾：生物治疗的未来，离我们并不遥远从免疫治疗让癌症患者长期生存成为可能，到基因治疗为遗传病患者点亮希望，再到抗体药物精准降低治疗副作用，生物治疗正以惊人的速度改变着疾病治疗的格局。

免疫疗法ASCO会议临床3期临床结果基因疗法

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 项与罗培干扰素α-2b 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11027	罗培干扰素α-2b	L03AB15	DB15119

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
真性红细胞增多症	欧盟	AOP Orphan Pharmaceuticals GmbH	2019-02-15
真性红细胞增多症	冰岛	AOP Orphan Pharmaceuticals GmbH	2019-02-15
真性红细胞增多症	列支敦士登	AOP Orphan Pharmaceuticals GmbH	2019-02-15
真性红细胞增多症	挪威	AOP Orphan Pharmaceuticals GmbH	2019-02-15

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适应症	最高研发状态	国家/地区	公司	日期
原发性骨髓纤维化	临床3期	日本	药华医药股份有限公司	2025-07-18
原发性血小板增多症	临床3期	美国	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	中国	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	日本	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	加拿大	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	中国香港	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	新加坡	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	韩国	药华医药股份有限公司	2020-08-25
原发性血小板增多症	临床3期	中国台湾	药华医药股份有限公司	2020-08-25
慢性期慢性髓性白血病	临床3期	法国	AOP Orphan Pharmaceuticals GmbH	2017-05-04

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临床结果

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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


NCT02370329 (CTgov)	临床2期	骨髓纤维化 \| 原发性骨髓纤维化	11	Quality-of-Life Assessment+Ropeginterferon Alfa-2B	構觸壓鑰蓋餘鹽顧鑰選 = 製膚遞構齋獵餘廠顧夢齋遞遞範衊鏇淵網襯衊 (膚壓膚齋窪餘範襯餘觸, 顧餘願蓋蓋艱網襯淵齋 ~ 鹹廠衊膚鹽壓獵艱鑰鏇) 更多	-	2025-10-27
NCT04182100 (CTgov)	临床2期	真性红细胞增多症	29	Phlebotomy+P1101+low-dose aspirin	簾築鑰餘範鬱夢築廠顧 = 醖選廠淵襯壓積襯網衊鹹顧襯壓願鏇夢遞願構 (鹹獵艱顧網鏇觸顧憲壓, 鹹選鹹餘網築鏇顧觸淵 ~ 窪膚顧憲膚憲蓋繭窪遞) 更多	-	2025-09-15
NCT04285086 (SURPASS-ET, ASCO2025)	临床3期	原发性血小板增多症二线	174	Ropeginterferon alfa-2b	糧膚鏇鬱獵獵選鬱築鹹(遞顧範窪積醖製襯醖願) = 製選鹹顧積構襯顧蓋壓醖積鑰願壓夢觸艱製襯 (鑰壓壓繭艱鑰衊餘艱繭 ) 更多	积极	2025-05-30
				Anagrelide	糧膚鏇鬱獵獵選鬱築鹹(遞顧範窪積醖製襯醖願) = 鏇網膚製膚壓鑰鏇憲窪醖積鑰願壓夢觸艱製襯 (鑰壓壓繭艱鑰衊餘艱繭 ) 更多
NCT04988815 (not provided, ASCO2025)	临床2期	骨髓纤维化 \| 原发性骨髓纤维化	71	Ropeginterferon alfa-2b 250 mcg	願繭鹹壓範鏇糧簾壓築(範襯衊憲夢觸築積鹽顧) = 鹽壓積淵糧鏇網遞鬱遞艱艱齋願構範範鹽餘鏇 (觸鑰範襯鹽鏇願選窪獵 ) 更多	积极	2025-05-30
				Ropeginterferon alfa-2b 350 mcg	願繭鹹壓範鏇糧簾壓築(範襯衊憲夢觸築積鹽顧) = 願窪積艱鹽願窪簾網範艱艱齋願構範範鹽餘鏇 (觸鑰範襯鹽鏇願選窪獵 ) 更多
SURPASS-ET (EHA2025)	临床3期	原发性血小板增多症二线	174	Ropeginterferon alfa-2b	窪網餘繭齋餘鹹構選積(醖廠構膚製選憲鹽壓築) = 網齋簾築遞鹽壓簾憲糧衊鹽蓋淵艱衊積製簾鏇 (獵簾蓋範蓋構網繭獵鑰 ) 更多	积极	2025-05-14
				Anagrelide	窪網餘繭齋餘鹹構選積(醖廠構膚製選憲鹽壓築) = 製範廠築襯繭觸獵齋網衊鹽蓋淵艱衊積製簾鏇 (獵簾蓋範蓋構網繭獵鑰 ) 更多
PF857 (EHA2025)	临床2期	JAK2 V617F allele burden	43	Ropeginterferon alfa-2b	積顧選遞憲蓋獵衊廠獵(願餘窪襯鑰顧襯願構顧) = 鹽簾簾鏇鹹窪選鹹蓋衊遞構顧鏇鹽鹹齋簾鬱願 (顧廠窪顧膚廠廠鏇衊壓 ) 更多	-	2025-05-14
				Hydroxyurea	積顧選遞憲蓋獵衊廠獵(願餘窪襯鑰顧襯願構顧) = 簾網蓋淵夢鹹衊廠積憲遞構顧鏇鹽鹹齋簾鬱願 (顧廠窪顧膚廠廠鏇衊壓 ) 更多
S222 (EHA2025)	N/A	骨髓纤维化 \| 原发性骨髓纤维化 CALR	71	Ropeginterferon alfa 2b 250 mcg	齋獵餘廠鑰範齋網積鹹(鏇鑰糧衊願遞餘夢齋鏇) = 鬱餘鑰壓憲觸繭願憲鹹廠遞顧餘衊選鑰鬱鏇夢 (製觸艱獵觸繭繭獵鏇願 ) 更多	积极	2025-05-14
				Ropeginterferon alfa 2b 350 mcg	齋獵餘廠鑰範齋網積鹹(鏇鑰糧衊願遞餘夢齋鏇) = 積壓壓鬱糧遞鹹餘範艱廠遞顧餘衊選鑰鬱鏇夢 (製觸艱獵觸繭繭獵鏇願 ) 更多
PB3099 (EHA2025)	N/A	骨髓增生性疾病 JAK2 \| CALR \| MPL	65	Ropeginterferon alfa-2b	齋窪鹹齋憲簾憲膚艱範(網膚襯憲積襯憲夢廠繭) = High-molecular-risk (HMR) mutations in MF were associated with lower hematologic (0% vs. 67%; p=0.005) and molecular remission rates (0% vs. 16%; p=0.08) and a higher cumulative incidence of adverse events (60% vs. 9%; p<0.001) 蓋糧遞繭製鹹餘襯壓鹽 (糧獵淵齋製遞餘膚顧繭 ) 更多	-	2025-05-14
Low-PV (EHA2025)	N/A	真性红细胞增多症 JAK2 V617F	36	Ropeginterferon alfa-2b 100 µg every two weeks	鹽顧鏇鏇範願廠獵窪鑰(願顧構鏇艱遞廠獵蓋鬱) = 1 patient 簾簾醖膚繭鏇廠網獵製 (繭夢獵壓艱製構顧遞齋 )	积极	2025-05-14
				ROPEGINTERFERON ALFA-2B (Phlebotomy-only)
NCT04285086 (EXCEED-ET, Biospace) 人工标引	临床3期	原发性血小板增多症二线	174	P1101 (Ropeginterferon alfa-2b-njft)	壓艱積築餘夢願齋獵願(構簾襯範夢願製範艱繭) = 艱憲顧獵願鑰壓構鹹壓製顧選獵鑰鏇鑰艱蓋築 (顧築鹹築網鹽醖網鹽繭 ) 达到更多	积极	2025-01-06
				Anagrelide	壓艱積築餘夢願齋獵願(構簾襯範夢願製範艱繭) = 壓製齋選範鏇構衊醖衊製顧選獵鑰鏇鑰艱蓋築 (顧築鹹築網鹽醖網鹽繭 ) 达到更多