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2025-01-01

申办/合作机构

药华医药股份有限公司

NCT06770842

/ Not yet recruiting临床2期

Safety and Efficacy of Ropeginterferon Alfa-2b in Combination With Ruxolitinib in Patients With Myelofibrosis Demonstrating Suboptimal Response to Ruxolitinib Monotherapy

In this open-label single arm phase 2 study, approximately 20 patients with MF demonstrating suboptimal response to ruxolitinib monotherapy will be enrolled. Patients will continue to receive ruxolitinib at a stable dose and ropeginterferon alfa 2b will be added to the regimen.

开始日期2025-01-01

申办/合作机构

香港大学

NCT06468033

/ Not yet 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

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2024-12-01

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）

2025-06-01·Pharmacology Research & Perspectives

Population Pharmacokinetics‐Pharmacodynamics and Exposure‐Response of Ropeginterferon Alfa‐2b in Chinese and Japanese Patients With Polycythemia Vera

Article

作者: Wu, Lei ; Suo, Shanshan ; Komatsu, Norio ; Sato, Toshiaki ; Liao, Jason ; Li, Yaning ; Shen, Weihong ; Shimoda, Kazuya ; Chen, Haoqi ; Su, Xia ; Zhang, Lei ; Wu, Daoxiang ; Fu, Rongfeng ; Jin, Jie ; Kirito, Keita ; Gao, Yucheng ; Qin, Albert ; Wang, Wei ; Zhang, Jingjing

ABSTRACT:

Ropeginterferon alfa‐2b (ropeg) represents a new‐generation interferon‐based therapy approved for polycythaemia vera (PV) treatment. This study aimed to elucidate its population pharmacokinetics‐pharmacodynamics (PopPK‐PD) and exposure‐response (E‐R) relationships. A PopPK model was developed using pooled data from four clinical studies, including two Phase I studies in healthy volunteers (n = 48) and two Phase II studies in Chinese or Japanese patients with PV (n = 78). Sequential modeling was used to analyze pharmacokinetics‐pharmacodynamics (PK‐PD) regarding hematological parameters, including hematocrit, platelet, and white blood cell counts. Hematological changes were simulated using fast‐ and slow‐dose titration regimens. Individual exposure values were used to analyze the E‐R relationships regarding complete hematologic response (CHR), driver mutation, JAK2V617F allele burden, and safety. In this study, we developed a target‐mediated drug disposition model. Sigmoid indirect effects elucidated the PK‐PD in terms of hematological changes. Simulations showed that the fast‐titration regimen significantly accelerated hematocrit reduction. Logistic regression models showed that the probability of achieving CHR increased with exposure at Week 24 but not at Week 52. In contrast, JAK2V617F allele reductions correlated with exposure at both Weeks 24 and 52. Exposure‐safety analysis revealed a manageable risk of adverse events associated with transaminase increases. This study established a robust framework for ropeg PK‐PD, providing insights into its E‐R relationships and disease‐modifying action.Trial Registration: A17‐102, A19‐201, and A20‐202 are registered at ClinicalTrials.gov. The registration numbers are as follows: A17‐102, NCT03546465; A19‐201, NCT04182100; and A20‐202, NCT05485948. A17‐101 is registered at www.chinadrugtrials.org.cn. The registration number is CTR20190451

2025-05-01·HemaSphere

Event‐free survival in early polycythemia vera patients correlates with molecular response to ropeginterferon alfa‐2b or hydroxyurea/best available therapy (PROUD‐PV/CONTINUATION‐PV)

Letter

作者: Illes, Arpad ; Empson, Victoria ; Krochmalczyk, Dorota ; Dulicek, Petr ; Egyed, Miklos ; Hasselbalch, Hans C. ; Gercheva‐Kyuchukova, Liana ; Sivcheva, Lylia ; Kiladjian, Jean‐Jacques ; Gisslinger, Heinz ; Kralovics, Robert ; Klade, Christoph ; Georgiev, Pencho ; Pylypenko, Halyna ; Krejcy, Kurt ; Mayer, Jiří ; Yablokova, Vera

2025-05-01·Therapeutic Advances in Hematology

Management of common autoimmune diseases in patients with myeloproliferative neoplasms treated with pegylated interferon alfa—case report, review of the literature and multidisciplinary clinical practice recommendations

Article

作者: Zeerleder, Sacha S. ; Odermatt, Urs ; Brand, Christoph ; de Gottardi, Andrea ; Melzer, Ralph ; Fischli, Stefan ; Rüfer, Axel ; Wuillemin, Walter A.

Pegylated interferons alfa are increasingly used in patients with myeloproliferative neoplasms (MPN) due to their potential disease-modifying effect. Ropeginterferon alfa-2b has been approved for patients with polycythemia vera (PV) with no symptomatic splenomegaly as first-line cytoreductive therapy, based on the results of the PROUD-PV/CONTINUATION-PV studies, documenting significantly higher rates of complete hematologic response (CHR) compared with hydroxyurea from 2-year timepoint onward. Although safety profile of pegylated interferons is overall good, interferon-related toxicities can occur. Focus of this article is on interferon-mediated autoimmune diseases. We describe two patients with PV treated with pegylated interferons alfa, one patient developed a cutaneous, paranasal sarcoidosis without any systemic symptoms and was successfully treated with topical steroids. The other patient developed widespread psoriatric skin lesions, which were treated with moderate effect with topical therapy with calcipotriene and betamethasone dipropionate foam, antidry calm lotion and in the course of the disease with ultraviolet light therapy (UVB). Only with methotrexate she achieved a nearly complete remission of the psoriasis. In both patients pegylated interferon was continued in view of the CHR and therefore the beneficial effect on MPN. We review the pertinent literature on the management of interferon-mediated autoimmune diseases in patients with MPN. As there is little published evidence on that topic, we propose multidisciplinary clinical practice recommendations based on available evidence and clinical experience in the management of patients with MPN treated with pegylated interferon alfa.

项与罗培干扰素α-2b 相关的新闻（医药）

2025-05-20

·Business Wire

PharmaEssentia to Present Phase 3 SURPASS-ET Data in Oral Session at 2025 ASCO Annual Meeting

BURLINGTON, Mass.--(BUSINESS WIRE)--PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced it will present results from the Phase 3 SURPASS-ET clinical trial in an oral session at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place May 30-June 3 in Chicago. SURPASS-ET (NCT04285086) is evaluating ropeginterferon alfa-2b-njft as a second-line treatment for patients with essential thrombocythemia (ET). Earlier this year, PharmaEssentia announced positive topline Phase 3 results, with ropeginterferon alfa-2b-njft demonstrating a significantly higher durable clinical response rate compared to anagrelide (42.9% vs. 6.0%; p=0.0001), along with a favorable safety profile and a greater reduction in JAK2 V617F allelic burden over 12 months. Ropeginterferon alfa-2b-njft is currently FDA-approved and marketed as BESREMi® for the treatment of adults with polycythemia vera (PV). BESREMi® has been recognized by the National Comprehensive Cancer Network® (NCCN®) as a preferred first-line cytoreductive therapy for adults with symptomatic, low-risk PV and the only preferred therapeutic option for both high-risk and low-risk (symptomatic) patients, regardless of treatment history. “Current options for patients with ET are limited. Standard therapies like hydroxyurea have notable drawbacks and do not target the underlying biology of the disease, while anagrelide has been associated with toxicity concerns and limited efficacy,” said Ruben Mesa, M.D., co-principal investigator, presenting author, and President of Atrium Health Levine Cancer Institute, the largest cancer program in the Carolinas which includes the Comprehensive Cancer Center at Wake Forest Baptist. “This marks the first registrational Phase 3 trial of a long-acting interferon in ET, demonstrating not only well-tolerated blood count control but also a measurable reduction in JAK2 mutation allele burden. These findings support further investigation of ropeginterferon as a second-line option for patients with ET who are seeking additional treatment approaches.” “The ASCO meeting is an important opportunity to share detailed findings of our positive data from the SURPASS-ET study with the medical community,” said Albert Qin, M.D., Ph.D., Chief Medical Officer of PharmaEssentia USA. “These data highlight a significant advance in the treatment of essential thrombocythemia and reinforce our commitment to delivering innovative, non-chemotherapy options for patients living with myeloproliferative neoplasms.“ Presentation Details Title: Ropeginterferon alfa-2b versus anagrelide for the treatment of essential thrombocythemia: Topline results of the phase 3 SURPASS-ET trial Abstract Number: 6500 Presenter: Dr. Ruben Mesa Session: Hematologic Malignancies — Leukemia, Myelodysplastic Syndromes, and Allotransplant Date: Monday, June 2, 2025 Time: 3:00 p.m. - 6:00 p.m. CDT About Essential Thrombocythemia Essential thrombocythemia is a chronic, rare blood disorder that is the most common type of myeloproliferative neoplasm. Essential thrombocythemia is most often caused by genetic mutations that cause the bone marrow to produce too many platelets, which can obstruct blood flow and cause a stroke, heart attack or pulmonary embolism. About BESREMi® (ropeginterferon alfa-2b-njft) in polycythemia vera (PV) BESREMi is an innovative monopegylated, long-acting interferon. With its unique pegylation technology, BESREMi has a long duration of activity in the body and is aimed to be administered once every two weeks (or every four weeks with hematological stability for at least one year), allowing flexible dosing that helps meet the individual needs of patients. BESREMi has orphan drug designation for the treatment of polycythemia vera (PV) in adults in the United States. BESREMi has been approved in more than 40 countries, with approval from the European Medicines Agency (EMA) in 2019, by the US Food and Drug Administration (FDA) in 2021, and by the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan in 2023. It was invented by PharmaEssentia and is manufactured in the company’s Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018. PharmaEssentia retains full global intellectual property rights for the product in all indications. BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders. Please see full Prescribing Information, including Boxed Warning. About PharmaEssentia PharmaEssentia USA Corporation, located in Burlington, Massachusetts, is a subsidiary of PharmaEssentia Corporation (TWSE: 6446). PharmaEssentia Corporation headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology, oncology, and immunology with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan. For more information about PharmaEssentia USA, visit the website, LinkedIn or X (formerly Twitter).

上市批准临床3期临床结果孤儿药ASCO会议

2025-05-16

·ioncology

2025 EHA大会摘要公布！30项中国研究入选口头报告，于“时尚之都”米兰奏响中国最强音

编者按第30届欧洲血液学会年会（EHA 2025）将于6月12日至15日在意大利米兰盛大召开。作为全球血液学领域的顶尖学术盛会之一，本届大会将全面聚焦血液学及其各亚专科的最新进展，深入探讨循证诊疗新方法，发布临床与转化研究的重磅成果，呈现创新技术、精准诊断工具与风险评估策略的最新突破。日前，大会官网正式公布了本次年会的入选摘要（不含LBA）。《肿瘤瞭望-血液时讯》第一时间汇总整理了中国学者入选的口头报告名单（如有遗漏，欢迎留言补充），让我们一同见证东方智慧在“时尚之都”米兰的精彩绽放。全体大会摘要（Plenary Abstract Session）01摘要号：S102英文标题：BETTER SAFETY AND EFFICACY WITH ROPEGINTERFERON ALFA-2B OVER ANAGRELIDE AS SECOND-LINE TREATMENT OF ESSENTIAL THROMBOCYTHEMIA IN THE TOPLINE RESULTS OF THE RANDOMIZED PHASE 3 SURPASS-ET TRIAL中文标题：随机Ⅲ期SURPASS-ET试验的初步结果显示，罗培干扰素α-2b相较阿那格雷二线治疗原发性血小板增多症的安全性和疗效更佳类型：口头报告专场名称：全体大会摘要汇报者及单位：Harry Gill，中国香港大学李嘉诚医学院口头报告（Oral Presentations）急性髓系白血病——生物学与转化研究Acute myeloid leukemia - Biology & translational research01摘要号：S134英文标题：DE NOVO DESIGN OF A CHROMATIN READER ZMYND8-TARGETING PEPTIDE PROTAC FOR ACUTE MYELOID LEUKEMIA THERAPY中文标题：靶向染色质识别蛋白ZMYND8的肽类PROTAC的重新设计：用于急性髓系白血病的治疗类型：口头报告专场名称：AML发病机制中的染色质和基因调控(Chromatin and gene regulation in AML pathogenesis)汇报者及单位：Hui Feng，西安交通大学第一附属医院02摘要号：S125英文标题：CELL-INTRINSIC INFLAMMATION SHAPES CELL-STATE HETEROGENEITY AND DRIVES THERAPEUTIC RESISTANCE IN HUMAN ACUTE MYELOID LEUKEMIA中文标题：细胞内在炎症塑造人类急性髓系白血病细胞状态异质性并驱动治疗耐药性类型：口头报告专场名称：AML免疫治疗的新进展（Novel advances in AML immunotherapy）汇报者及单位：Lina Liu，浙江大学医学院附属第一医院骨髓移植中心急性髓系白血病——临床Acute myeloid leukemia - Clinical01摘要号：S143英文标题：LONG-TERM FOLLOW-UP OF PREDOMINANTLY ASIAN PATIENTS WITH RELAPSED/REFRACTORY FLT3-MUTATED ACUTE MYELOID LEUKEMIA TREATED WITH GILTERITINIB VERSUS SALVAGE CHEMOTHERAPY IN THE PHASE 3 COMMODORE TRIAL中文标题：Ⅲ期COMMODRE试验中接受吉瑞替尼与挽救化疗治疗的复发/难治性FLT3突变急性髓系白血病患者（以亚洲患者为主）的长期随访类型：口头报告专场名称：AML的新疗法与FLT3抑制剂（Novel therapies and FLT3 inhibition for AML）汇报者及单位：王建祥，中国医学科学院血液病医院（中国医学科学院血液学研究所）侵袭性非霍奇金淋巴瘤——临床Aggressive Non-Hodgkin lymphoma - Clinical01摘要号：S246英文标题：A PIVOTAL STUDY OF SHR2554, AN ORAL INHIBITOR AGAINST ENHANCER OF ZESTE HOMOLOG 2 (EZH2), IN RELAPSED OR REFRACTORY (R/R) PERIPHERAL T-CELL LYMPHOMA (PTCL)中文标题：SHR2554[一种口服的增强子同源物2（EZH2）抑制剂）]用于复发或难治性（R/R）外周T细胞淋巴瘤（PTCL）的治疗：一项关键研究类型：口头报告专场名称：侵袭性非霍奇金淋巴瘤——临床（前瞻性）[Aggressive Non-Hodgkin lymphoma - Clinical (Prospective)]汇报者及单位：宋玉琴，北京大学肿瘤医院出血性疾病（先天性和获得性）Bleeding disorders (congenital and acquired)01摘要号：S315英文标题：BLASTS- AND MODIFIED EASIX-GUIDED RISK STRATIFICATION FOR CAR-T-ASSOCIATED COAGULOPATHY AND OUTCOMES中文标题：BLASTS和改良EASIX指导的CAR-T相关凝血病及预后的风险分层类型：口头报告专场名称：出血性疾病（先天性和获得性）[Bleeding disorders (congenital and acquired)]汇报者及单位：Yingying Li，华中科技大学同济医学院附属协和医院骨髓衰竭综合征（包括PNH）——临床Bone marrow failure syndromes incl.PNH-Clinical01摘要号：S184英文标题：EFFICACY AND SAFETY OF CYCLOSPORINE PLUS LUSPATERCEPT VERSUS CYCLOSPORINE IN NEWLY DIAGNOSED NON-TRANSFUSION-DEPENDENT NON-SEVERE APLASTIC ANEMIA: A PROSPECTIVE RANDOMIZED TRIAL中文标题：环孢素联合罗特西普 vs. 环孢素治疗新诊断非输血依赖型非重型再生障碍性贫血的疗效和安全性：一项前瞻性随机试验类型：口头报告专场名称：骨髓衰竭综合征(Bone marrow failure syndromes)汇报者及单位：Zhuxin Zhang，中国医学科学院北京协和医院慢性髓系白血病——生物学及转化研究Chronic myeloid leukemia-Biology & translational research01摘要号：S162英文标题：INTEGRATING GENOMIC AND TRANSCRIPTOMIC INSIGHTS FOR PREDICTING RESPONSES AND OUTCOMES IN PATIENTS WITH CML RECEIVING 3RD-GENERATION TKI THERAPY中文标题：整合基因组与转录组见解以预测接受第三代TKI治疗的CML患者的反应和预后类型：口头报告专场名称：CML治疗反应与耐药的生物学(Biology of CML treatment response and resistance)汇报者及单位：张小帅，北京大学人民医院基因治疗、细胞免疫治疗和疫苗接种——生物学和转化研究Gene therapy,cellular immunotherapy and vaccination-Biology & translational research01摘要号：S277英文标题：TYPE I INTERFERON IMPAIRS CAR-T CELL FUNCTION BY DISRUPTING IMMUNE SYNAPSE ASSEMBLY中文标题：I型干扰素通过破坏免疫突触组装削弱CAR-T细胞功能类型：口头报告专场名称：基因治疗、细胞免疫治疗和疫苗接种——生物学和转化研究（Gene therapy, cellular immunotherapy and vaccination - Biology & translational research）汇报者及单位：Meijuan Huang，华中科技大学同济医学院附属同济医院基因治疗、细胞免疫治疗和疫苗接种——临床Gene therapy,cellular immunotherapy and vaccination-Clinical01摘要号：S283英文标题：CTD402: A UNIVERSAL ANTI-CD7 CHIMERIC ANTIGEN RECEPTOR T-CELL THERAPY FOR PATIENTS WITH RELAPSED OR REFRACTORY (R/R) T-CELL ACUTE LYMPHOBLASTIC LEUKEMIA/LYMPHOBLASTIC LYMPHOMA (T-ALL/LBL)中文标题：CTD402：一种针对复发或难治性（R/R）T细胞急性淋巴细胞白血病/淋巴母细胞淋巴瘤（T-ALL/LBL）患者的通用抗CD7嵌合抗原受体T细胞疗法类型：口头报告专场名称：基因治疗、细胞免疫治疗和疫苗接种——临床（Gene therapy, cellular immunotherapy and vaccination - Clinical）汇报者及单位：Jiangtao Ren，Bioheng Therapeutics Co., Ltd,, Nanjing, China造血、干细胞和微环境Hematopoiesis,stem cells and microenvironment01摘要号：S272英文标题：ENHANCE THE THERAPEUTIC EFFICACY OF HUMAN UMBILICAL CORD-DERIVED MESENCHYMAL STEM CELLS IN PREVENTION OF ACUTE GRAFT-VERSUS-HOST DISEASE THROUGH CRISPLD2 MODULATION中文标题：通过调控CRISPLD2增强人脐带间充质干细胞在预防急性移植物抗宿主病中的治疗效果类型：口头报告专场名称：造血干细胞及其微环境(Hematopoietic stem cells and their niche)汇报者及单位：Xu Qing，陆军军医大学第二附属医院（新桥医院）02摘要号：S273英文标题：TGF-ß1-TRIGGERED MALADAPTIVE BONE MARROW ENDOTHELIUM IMPEDES HEMATOPOIETIC RECOVERY中文标题：TGF-β1诱导的骨髓内皮功能失调阻碍造血恢复类型：口头报告专场名称：造血干细胞及其微环境(Hematopoietic stem cells and their niche)汇报者及单位：Zhong-Shi Lyu，北京大学人民医院淋巴瘤生物学与转化研究Lymphoma biology & translational research01摘要号：S251英文标题：MULTI-OMICS DISSECTION OF EBV-DRIVEN HETEROGENEITY, METABOLIC REMODELING, AND TUMOR CELLULAR LANDSCAPE IN ENKTL PROGRESSION中文标题：EB病毒驱动的ENKTL进展中的异质性、代谢重塑及肿瘤细胞图谱的多组学解析类型：口头报告专场名称：淋巴瘤生物学与转化研究(Lymphoma biology & translational research)汇报者及单位：梁金花，南京医科大学第一附属医院（江苏省人民医院）02摘要号：S252英文标题：PTPN2 INHIBITION DISRUPTS MITOCHONDRIAL RENEWAL AND BLOCKS TFRC-MEDIATED PINK1-PRKN-DEPENDENT MITOPHAGY TO EXERT ANTI-TUMOR ACTIVITIES IN ALK-POSITIVE ANAPLASTIC LARGE CELL LYMPHOMA中文标题：PTPN2抑制通过破坏线粒体更新并阻断TFRC介导的PINK1-PRKN依赖性线粒体自噬，在ALK阳性间变性大细胞淋巴瘤中发挥抗肿瘤作用类型：口头报告专场名称：淋巴瘤生物学与转化研究(Lymphoma biology & translational research)汇报者及单位：梁金花，南京医科大学第一附属医院（江苏省人民医院）骨髓增生异常综合征——生物学与转化研究Myelodysplastic syndromes-Biology & translational research01摘要号：S170英文标题：SINGLE-CELL ATLAS OF PEDIATRIC NRAS-MUTANT MYELOID NEOPLASMS REVEALS MYELOID LINEAGE BIAS AND IDENTIFIES POTENTIAL THERAPEUTIC TARGETS中文标题：儿童NRAS突变髓系肿瘤的单细胞图谱揭示髓系谱系偏倚并识别潜在治疗靶点类型：口头报告专场名称：MDS cellular and molecular therapeutic targeting汇报者及单位：Suyu Zong，中国医学科学院血液病医院（中国医学科学院血液学研究所）骨髓瘤及其他单克隆免疫球蛋白病——生物学与转化研究Myeloma and other monoclonal gammopathies -Biology & translational research01摘要号：S188英文标题：ENHANCER EXTRACHROMOSOMAL CIRCULAR DNA ANKRD28 DRIVES DRUG RESISTANCE VIA POU2F2-MEDIATED TRANSCRIPTIONAL REWIRING IN MULTIPLE MYELOMA中文标题：增强子染色体外环状DNA ANKRD28通过POU2F2介导的转录重编程驱动多发性骨髓瘤的药物耐药类型：口头报告专场名称：骨髓瘤及其他单克隆免疫球蛋白病——生物学与转化研究（Myeloma and other monoclonal gammopathies - Biology & translational research）汇报者及单位：Binzhen Chen，上海交通大学医学院附属仁济医院骨髓增生性肿瘤——临床Myeloproliferative neoplasms-Clinical01摘要号：S222英文标题：ROPEGINTERFERON ALFA-2B FOR PRE-FIBROTIC PRIMARY MYELOFIBROSIS AND DIPSS LOW/INTERMEDIATE-RISK MYELOFIBROSIS中文标题：罗培干扰素α-2b治疗纤维化前原发性骨髓纤维化及DIPSS低/中风险骨髓纤维化类型：口头报告专场名称：Innovative treatment approaches in MPN汇报者及单位：Harry Gill，中国香港大学李嘉诚医学院血液学中的新技术、新方法及数字分析工具Novel technologies,techniques and digital analytical tools in hematology01摘要号：S332英文标题：STEM CELL AGING CLOCK: A DEEP LEARNING DRIVEN FRAMEWORK FOR PREDICTING STEM CELL AGE中文标题：干细胞衰老时钟：基于深度学习的干细胞年龄预测框架类型：口头报告专场名称：计算机与人工智能辅助血液学的新进展（New developments in computational and AI-assisted hematology）汇报者及单位：Guanlin Wang，复旦大学代谢与整合生物学研究院血小板疾病Platelet disorders01摘要号：S313英文标题：BARICITINIB CORRECTS AP2-NAK MEDIATED C-MPL ENDOCYTIC DYSREGULATION TO RESTORE MEGAKARYOCYTES TPO-RA SENSITIVITY IN IMMUNE THROMBOCYTOPENIA中文标题：巴瑞替尼纠正AP2-NAK介导的c-MPL内吞调控异常，恢复免疫性血小板减少症中巨核细胞对TPO-RA的敏感性类型：口头报告专场名称：血小板疾病（Platelet disorders）汇报者及单位：安倬玉，北京大学人民医院02摘要号：S318英文标题：TARGETED SUPPRESSION OF EFFECTOR T CELLS BY GPIBα CAAR-TREG CELLS IN REFRACTORY/RELAPSED IMMUNE THROMBOCYTOPENIA中文标题：GPIBα CAAR-Treg细胞对难治/复发性免疫性血小板减少症效应T细胞的靶向抑制类型：口头报告专场名称：出血性疾病（先天性与获得性）[Bleeding disorders (congenital and acquired)]汇报者及单位：Jinhui Shu，华中科技大学同济医学院附属协和医院干细胞移植——临床Stem cell transplantation-Clinical01摘要号：S261英文标题：AUTONOMOUS AI-DRIVEN INTENSIFIED PROPHYLAXIS OF SEVERE ACUTE GRAFT-VERSUS-HOST DISEASE IN HAPLOIDENTICAL TRANSPLANTS: A SINGLE-CENTRE, PROOF-OF-CONCEPT, PHASE 2 TRIAL中文标题：自主人工智能驱动的加强预防严重急性移植物抗宿主病在半相合移植中的应用：一项单中心概念验证Ⅱ期试验类型：口头报告专场名称：干细胞移植——专场2(Stem cell transplantation - Session 2)汇报者及单位：Yigeng Cao，中国医学科学院血液病医院（中国医学科学院血液学研究所）02摘要号：S262英文标题：ANTI-CD25 ANTIBODY AS AN ALTERNATIVE TO METHOTREXATE FOR GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS RESULTED IN BETTER OUTCOMES IN PATIENT OF CHIMERIC ANTIGEN RECEPTOR-T CELL THERAPY BRIDGING TO TRANSPLANT中文标题：抗CD25抗体作为替代甲氨蝶呤的移植物抗宿主病预防方案，在嵌合抗原受体T细胞治疗桥接移植患者中取得了更好的疗效类型：口头报告专场名称：干细胞移植——专场2（Stem cell transplantation - Session 2）汇报者及单位：李智慧，北京高博博仁医院03摘要号：S263英文标题：PHARMACOKINETIC-GUIDED ATG DOSING OPTIMIZES OUTCOMES IN MYELOABLATIVE HAPLO-CORD HCT中文标题：基于药代动力学指导的ATG给药优化改善了清髓性半相合脐带血干细胞移植的治疗效果类型：口头报告专场名称：干细胞移植——专场2（Stem cell transplantation - Session 2）汇报者及单位：Qingya cui,苏州大学附属第一医院04摘要号：S256英文标题：TARGETED DOSING OF ANTI-THYMOCYTE GLOBULIN VERSUS FIXED DOSING STRATEGY IN PATIENTS UNDERGOING UNMANIPULATED HAPLOIDENTICAL HAEMATOPOIETIC STEM-CELL TRANSPLANTATION: A RANDOMIZED, MULTICENTER, PHASE 3 CLINICAL TRIAL中文标题：抗胸腺细胞球蛋白的目标给药与固定给药策略在接受未处理半相合造血干细胞移植患者中的比较：一项随机、多中心、Ⅲ期临床试验类型：口头报告专场名称：干细胞移植——专场1（Stem cell transplantation - Session 1）汇报者及单位：刘代红，解放军总医院第五医学中心05摘要号：S258英文标题：WHO IS THE BEST ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANT DONOR FOR SEVERE APLASTIC ANEMIA? A MULTICENTER RETROSPECTIVE EXPERIENCE FROM CHINA中文标题：重型再生障碍性贫血的最佳异基因造血干细胞移植供者是谁？来自中国的多中心回顾性研究经验类型：口头报告专场名称：干细胞移植——专场1（Stem cell transplantation - Session 1）汇报者及单位：Xiao-Di Ma，北京大学人民医院06摘要号：S259英文标题：COMPARABLE SURVIVAL OUTCOMES IN HLA-MATCHED AND HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR SEVERE APLASTIC ANEMIA PATIENTS AGED 40-50: A CBMTR REGISTRY-BASED PSM ANALYSIS OVER THE LAST DECADE中文标题：接受HLA匹配与半相合造血干细胞移植40-50岁重型再生障碍性贫血患者的生存结局相当：过去十年基于CBMTR登记的PSM分析类型：口头报告专场名称：干细胞移植——专场1（Stem cell transplantation - Session 1）汇报者及单位：许兰平，北京大学人民医院干细胞移植——实验Stem cell transplantation-Experimental01摘要号：S255英文标题：JAK/ROCK INHIBITION WITH ROVADICITINIB SUPPRESSES MURINE AND HUMAN ACUTE GRAFT-VERSUS-HOST DISEASE: THE RESULTS OF PRECLINICAL AND PHASE 1B STUDY中文标题：JAK/ROCK抑制剂罗伐昔替尼抑制小鼠及人类急性移植物抗宿主病：临床前及Ⅰb期研究结果类型：口头报告专场名称：干细胞移植——专场1（Stem cell transplantation - Session 1）汇报者及单位：赵妍敏，浙江大学医学院附属第一医院骨髓移植中心地中海贫血Thalassemias01摘要号：S290英文标题：EFFICACY AND SAFETY OF BRL-101 IN TDT AND SCD中文标题：BRL-101 在输血依赖型地中海贫血（TDT）和镰状细胞病（SCD）中的疗效与安全性类型：口头报告专场名称：地中海贫血和红细胞生成（Thalassemia and erythopoiesis）汇报者及单位：Tan Qian，Shanghai tech immunology department, Shanghai, China02摘要号：S291英文标题：TREATMENT OF PATIENTS WITH SEVERE TRANSFUSION-DEPENDENT ß-THALASSEMIA WITH CS-101, AN AUTOLOGOUS, EX VIVO EDITED, CD34+ HEMATOPOIETIC STEM CELL PRODUCT USING INNOVATIVE TRANSFORMER BASE EDITOR (TBE)中文标题：采用创新型Transformer基础编辑器（TBE）对自体CD34+造血干细胞进行体外编辑制备的CS-101，用于治疗重型输血依赖型β-地中海贫血患者类型：口头报告专场名称：地中海贫血和红细胞生成（Thalassemia and erythopoiesis）汇报者及单位：陈佳，上海科技大学生命科学与技术学院基因编辑中心03摘要号：S292英文标题：FIRST-IN-HUMAN STUDY OF AUTOLOGOUS HBA2-EDITED CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS IN ALPHA-THALASSEMIA WITH CONSTANT SPRING MUTATION中文标题：首次人体研究：自体HBA2基因编辑的CD34+造血干/祖细胞用于Constant Spring突变型α-地中海贫血治疗类型：口头报告专场名称：地中海贫血和红细胞生成（Thalassemia and erythopoiesis）汇报者及单位：Hui Xu，Reforgene Medicine, Guangzhou, China（来源：《肿瘤瞭望–血液时讯》编辑部）声明凡署名原创的文章版权属《肿瘤瞭望》所有，欢迎分享、转载。本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。

蛋白降解靶向嵌合体临床结果

2025-05-15

·抗体圈

大肠杆菌生产药物的关键技术与挑战

摘要：大肠杆菌凭借重组 DNA 技术成为生物制药领域的核心工具，从首个重组胰岛素的生产至今，已在激素、酶、疫苗、抗体等多个领域展现重要价值。尽管存在翻译后修饰不足和毒性等局限，但其低成本、高产量和成熟的生产体系使其仍是首选宿主。本文系统梳理了大肠杆菌生产的各类生物药物，包括已获批的激素、酶、肽类、疫苗及融合蛋白等，并探讨其临床应用、优势与未来发展方向，展现其在生物制药产业中的关键地位与潜力。一、引言大肠杆菌在重组 DNA 技术的推动下，成为生物制药的重要宿主。自 1980 年代首个重组胰岛素获批以来，其凭借快速生长、低成本和高产量等优势，广泛应用于多种生物药物生产。尽管面临复杂蛋白折叠困难和缺乏糖基化修饰等挑战，但其在激素、酶、疫苗等领域的成果显著，已成为全球生物制药产业的基石之一（参考图 1: General method for producing recombinant biopharmaceuticals in Escherichia coli，了解大肠杆菌生产重组生物药物的通用流程）。二、大肠杆菌生产的生物药物类型2.1激素类药物大肠杆菌是生产重组激素的主力，涵盖胰岛素、生长激素、干扰素等。例如，首个重组人胰岛素Humulin由礼来公司开发，1982 年获 FDA 批准，彻底改变糖尿病治疗模式。随后，长效胰岛素类似物Lantus、生长激素Sogroya等相继问世，显著提升患者生活质量（表1: Approved recombinant hormones produced in Escherichia coli and their mechanism of action列出了主要激素药物及其作用机制）。干扰素 α-2b（Intron A）则用于肝炎、癌症治疗，其改良型Besremi对真性红细胞增多症疗效显著。2.2酶类药物酶类药物在代谢疾病和癌症治疗中至关重要。Krystexxa（聚乙二醇化尿酸酶）用于慢性痛风，通过降解尿酸缓解症状；Voraxaze（葡糖羧肽酶）可快速清除化疗药物甲氨蝶呤，降低肾损伤风险；Asparlas（聚乙二醇天冬酰胺酶）则用于白血病治疗，延长患者生存期（表2: Approved recombinant enzymes produced in Escherichia coli and their mechanism of action详细展示了相关酶类药物）。2.3肽类药物肽类药物在心血管和代谢疾病中应用广泛。Natrecor（奈西立肽）用于急性心力衰竭，通过扩张血管改善症状；Voxzogo（vosoritide）针对软骨发育不全，通过调节信号通路促进骨骼生长；Oxervate（神经生长因子）则用于治疗神经萎缩性角膜炎，修复角膜损伤（表3: Approved recombinant peptides produced in Escherichia coli and their mechanism of action列举了代表性肽类药物）。2.4疫苗类药物大肠杆菌在疫苗生产中表现突出，尤其在病毒样颗粒（VLP）疫苗领域。Hecolin是首个基于大肠杆菌的戊肝疫苗，在中国获批上市；Cecolin（HPV 疫苗）有效预防生殖器病变；兽用疫苗如Strangvac（马链球菌疫苗）和Leucogen（猫白血病疫苗）也展现重要价值（表4: Approved recombinant vaccines produced in Escherichia coli总结了人用及兽用疫苗）。2.5融合蛋白与抗体片段融合蛋白和抗体片段是癌症与免疫疾病的关键疗法。Lucentis（雷珠单抗）靶向 VEGF，用于湿性黄斑变性；Kimmtrak（ Tebentafusp）通过双特异性结合 T 细胞和肿瘤抗原，治疗转移性葡萄膜黑色素瘤；Cimzia（ certolizumab pegol）作为 TNF-α 抑制剂，有效缓解类风湿关节炎（表5: Approved recombinant antibodies and fusion proteins produced in Escherichia coli列出了主要抗体及融合蛋白药物）。2.6其他生物药物包括粒细胞集落刺激因子Neupogen（治疗中性粒细胞减少症）、瘦素类似物Myalept（治疗脂肪营养不良）等，均通过大肠杆菌生产，满足罕见病和免疫疾病治疗需求。三、大肠杆菌的优势与挑战优势：遗传背景清晰：便于基因操作和表达系统优化，如利用已知的克隆技术和表达载体高效生产目标蛋白。生产成本低：培养基简单（仅需基础营养物质），适合大规模发酵，例如 mRNA 新冠疫苗的低成本生产模式可复制至其他生物药。生产周期短：生长繁殖速度快，可在短时间内实现蛋白大量表达，如重组胰岛素从基因设计到生产仅需数周。挑战：翻译后修饰局限：无法进行复杂糖基化、二硫键形成等修饰，影响部分蛋白（如全长单克隆抗体）的功能，需依赖哺乳动物细胞补位（参考表5中部分抗体需哺乳动物细胞生产的限制）。内毒素风险：细胞壁成分可能引发免疫反应，需额外纯化工艺去除内毒素，增加生产成本（如疫苗生产中的严格纯化流程）。蛋白折叠问题：复杂蛋白易形成包涵体，需优化表达条件或采用共表达伴侣蛋白辅助折叠（如图1中纯化步骤的重要性）。四、未来发展方向技术优化：通过基因工程改造大肠杆菌，引入糖基化通路或共表达折叠辅助因子，提升复杂蛋白生产能力。例如，利用合成生物学技术构建 “设计型” 大肠杆菌，实现更接近天然蛋白的修饰。联合疗法拓展：大肠杆菌生产的疫苗与免疫检查点抑制剂（ICIs）联用，如mRNA 肿瘤疫苗 + PD-1 单抗的协同效应，已在黑色素瘤等适应症中展现潜力（参考前文KEYNOTE-942 研究数据）。兽用与罕见病药物：针对动物传染病（如马链球菌疫苗Strangvac）和罕见病（如脂肪营养不良药物Myalept），开发高性价比的大肠杆菌生产药物，填补市场空白。可持续生产：探索无血清培养基和连续生产工艺，降低环境负担，符合绿色制药趋势。五、结论大肠杆菌凭借低成本、高可及性和成熟的工业化体系，在生物制药领域占据不可替代的地位。尽管面临修饰能力有限等挑战，但其在激素、酶、疫苗等领域的成功案例（如表1-5中多款 FDA/EMA 批准药物）及技术优化潜力，使其持续成为创新药物开发的核心平台。未来，随着合成生物学和联合疗法的突破，大肠杆菌将进一步推动生物制药向高效、经济、普惠方向发展。识别微信二维码，添加抗体圈小编，符合条件者即可加入抗体圈微信群！请注明：姓名+研究方向！本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。

疫苗免疫疗法微生物疗法

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

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
原发性骨髓纤维化	临床3期	-	药华医药股份有限公司	2024-12-01
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	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
慢性期慢性髓性白血病	临床3期	德国	Aop Orphan Pharmaceuticals GmbH	2017-05-04

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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


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)	壓鑰願構艱構醖選膚鹹(獵鏇鏇願製構顧獵餘製) = 淵壓廠繭製繭餘齋膚鏇築壓糧遞壓鑰醖壓積艱 (壓鹹繭廠憲鹹獵襯製襯 ) 达到更多	积极	2025-01-06
NCT04285086 (EXCEED-ET, Biospace) 人工标引	临床3期	原发性血小板增多症二线	174	Anagrelide	壓鑰願構艱構醖選膚鹹(獵鏇鏇願製構顧獵餘製) = 網憲觸襯鬱憲構餘遞獵築壓糧遞壓鑰醖壓積艱 (壓鹹繭廠憲鹹獵襯製襯 ) 达到更多	积极	2025-01-06
EHA2024 人工标引	临床2期	真性红细胞增多症 JAK2V617F VAF \| IFNL4	114	Ropeginterferon alfa-2b (Ropeg)	糧鹽糧夢夢醖鏇窪廠鏇(願鬱觸衊築選膚蓋憲餘) = 積構壓繭觸獵選網醖簾鑰淵顧製顧網獵襯憲繭 (鹽艱齋艱願衊築獵願夢 )	积极	2024-05-14
EHA2024 人工标引	临床2期	真性红细胞增多症 JAK2V617F VAF \| IFNL4	114	Ropeginterferon alfa-2b (Ropeg)py, STD)	糧鹽糧夢夢醖鏇窪廠鏇(願鬱觸衊築選膚蓋憲餘) = 醖選製壓醖網蓋鬱廠獵鑰淵顧製顧網獵襯憲繭 (鹽艱齋艱願衊築獵願夢 )	积极	2024-05-14
EUCTR2012-005259-18-CZ \| EUCTR2014-001357-17-PL (PROUD-PV, EHA2024) 人工标引	临床3期	真性红细胞增多症 JAK2V617F allele burden	169	ropeginterferon alfa-2b	鏇艱網壓鏇願蓋蓋顧淵(糧廠製網鹹糧範遞鏇衊) = 鏇繭範願膚範範餘鑰鹽醖淵構憲簾襯簾壓憲餘 (蓋壓齋壓窪鹹淵積襯構 ) 更多	积极	2024-05-14
	临床3期	真性红细胞增多症 JAK2V617F allele burden	169	HU/BAT	鏇艱網壓鏇願蓋蓋顧淵(糧廠製網鹹糧範遞鏇衊) = 簾醖積衊糧簾壓襯夢廠醖淵構憲簾襯簾壓憲餘 (蓋壓齋壓窪鹹淵積襯構 ) 更多	积极	2024-05-14
EHA2024	临床2期	真性红细胞增多症 JAK2 V617F allele burden	27	Ropeginterferon alfa-2b	艱蓋衊獵網獵網築鹽鑰(鹽遞獵觸願築觸鬱繭顧) = Although most patients (25/27 [92.6%]) experienced adverse events (AEs), ropeginterferon alfa-2b was generally well tolerated. The most common AEs were WBC count decreased (7/27 [25.9%]) and urine beta-2 microglobulin increased (6/27 [22.2%]) 遞積壓鑰顧網蓋糧鹽願 (憲艱糧製襯願艱鬱餘膚 ) 更多	积极	2024-05-14
EHA2024	临床3期	真性红细胞增多症	34	Ropeginterferon alfa-2b	淵夢鹹醖齋構製糧願遞(繭壓鹽夢膚糧鬱鏇壓願) = One patient developed autoimmune thyroiditis without the need to suspend treatment 觸選襯壓選鏇觸範築夢 (簾壓鏇鹽衊憲膚積觸蓋 ) 更多	积极	2024-05-14
PROUD-PV/CONTINUATION (EHA2024)	临床3期	二线 JAK2 mutated	12	Ropeginterferon alfa-2b	鬱築獵築繭願糧簾範範(繭製艱糧衊鬱鑰遞窪鹹) = One withdrew therapy due to intolerance (flushing and eczema, grade 1) after 9 months of treatment 簾齋艱膚夢鏇夢憲蓋醖 (衊齋醖積壓積鏇鹹糧齋 )	积极	2024-05-14
P1101MF (ASH2023)	临床2期	原发性骨髓纤维化	-	Ropeginterferon alfa 2b	觸衊選淵鏇製衊鹹襯觸(觸繭蓋蓋壓壓鹽範顧繭) = 襯衊網網鑰鬱餘積夢壓製夢鹹鹹鏇構積鬱襯簾 (醖醖遞夢齋衊糧淵遞壓 ) 更多	-	2023-12-11
ASH2023	N/A	真性红细胞增多症	99	P1101	膚壓構觸網廠壓築膚鹹(壓築蓋簾夢獵觸顧糧襯) = 窪窪鹽積廠艱餘構餘廠齋餘簾艱衊鏇膚繭醖夢 (蓋蓋鏇夢衊鏇願餘鏇網 ) 更多	-	2023-12-11
PROUD-PV/CONTINUATION-PV (ASH2023)	N/A	真性红细胞增多症	-	Ropeginterferon alfa-2b	淵壓選鏇襯簾鬱製淵襯(淵窪糧築蓋遞鏇構觸憲) = 觸積艱淵簾鬱夢鬱壓構獵顧蓋範齋鏇艱壓簾遞 (襯艱構選網顧醖遞齋窪 )	-	2023-12-11
CTR20211664 \| NCT05485948 (Pubmed 1 \| Pubmed 2 \| Exp Hematol Oncol) 人工标引	临床2期	真性红细胞增多症	49	ropeginterferon alfa-2b	鹽鏇觸憲糧窪艱衊糧窪(獵築觸獵製製廠廠膚齋) = 獵夢憲築壓簾廠積糧築鬱獵憲簾簾夢襯築壓壓 (網窪築餘鏇鏇範齋簾壓 ) 更多	积极	2023-06-21