The goal of this clinical trial is to assess long-term safety and efficacy of rusfertide in subjects with polycythemia vera. Subjects who complete dosing with rusfertide until the end-of-treatment visit of a Phase 2 rusfertide study and meet the inclusion/exclusion criteria for this study, are eligible to participate in this open-label study and continue treatment with rusfertide.

开始日期2024-01-22

申办/合作机构

Protagonist Therapeutics, Inc.

NCT05210790

/ Active, not recruiting临床3期

A Phase 3 Study of the Hepcidin Mimetic Rusfertide (PTG-300) in Patients With Polycythemia Vera

The study is designed to evaluate the safety and efficacy of rusfertide in subjects with polycythemia vera (PV) in maintaining hematocrit control and in improving symptoms of PV.

开始日期2022-04-01

申办/合作机构

Protagonist Therapeutics, Inc.

NCT04767802

/ Completed临床2期

A Phase 2 Study of the Hepcidin Mimetic PTG-300 in Patients With Polycythemia Vera and Elevated Hematocrit (PACIFIC)

This is an open label, single arm trial of PTG-300 in subjects with PV who are newly diagnosed or for whom current therapy is not sufficient to control their hematocrit and have hematocrit >48% prior to dosing. The PTG-300 dose and schedule may be adjusted every 2 to 4 weeks to maintain hematocrit <45% with a target of <43%. Subjects may receive PTG-300 treatment for up to 52 weeks.

开始日期2021-02-02

申办/合作机构

Protagonist Therapeutics, Inc.

100 项与 Rusfertide acetate 相关的临床结果

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100 项与 Rusfertide acetate 相关的转化医学

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100 项与 Rusfertide acetate 相关的专利（医药）

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项与 Rusfertide acetate 相关的文献（医药）

2025-12-01·LEUKEMIA RESEARCH

Rusfertide rapidly decreases hematocrit in patients with suboptimally controlled polycythemia vera

Article

作者: Kirubamoorthy, Kamini ; Molina, Arturo ; Valone, Frank ; Khanna, Sarita ; Modi, Nishit B ; Chew, Lee Ping ; Lee, Sung-Eun ; Dinh, Phillip ; Lee, Jae Hoon ; Ginzburg, Yelena Z ; Gupta, Suneel

Polycythemia vera (PV) is characterized by overproduction of erythrocytes, leukocytes, and platelets. Rusfertide is a hepcidin mimetic polypeptide that binds to ferroportin and decreases iron delivery to bone marrow, reducing the production of red blood cells. The primary objective of this study was to evaluate the efficacy and safety of rusfertide in patients with PV and elevated (>48 %) baseline hematocrit. Patients were treated with 40 mg subcutaneous rusfertide twice weekly until their hematocrit reached < 45 %; once achieved, patients received weekly rusfertide dosing. Of the 20 patients enrolled, 15 (75 %) completed 24 weeks of treatment, 13 (65 %) completed 52 weeks, and 7 discontinued rusfertide therapy. Mean baseline hematocrit was 51.6 %. By Week 8, 17 (85 %) patients achieved hematocrit < 45 %, and 19 (95 %) achieved ≥ 5 % absolute reduction from baseline. Median time to first hematocrit < 45 % was 4.9 weeks. Mean hematocrit was < 45 % at Week 4 and subsequently remained < 45 % throughout the study. No patients received phlebotomy during rusfertide treatment. Rusfertide led to a significant reduction in erythrocyte count (5.98 ×10¹²/L to 4.79 ×10¹²/L; nominal p-value 0.0036) and an improvement in iron deficiency markers (eg, ferritin and mean corpuscular volume) relative to baseline. Seventeen patients (85 %) experienced treatment-emergent adverse events, most of which were Grade 1/2. In this open-label phase 2 study, rusfertide resulted in a rapid and sustained improvement in hematocrit and was generally well tolerated. Rapid and sustained control of hematocrit reduces the need for therapeutic phlebotomy and may help reduce thrombotic and cardiovascular events over the long term in patients with PV.

2025-11-01·CLINICAL THERAPEUTICS

Evaluation of Rusfertide, a Hepcidin Mimetic, on Cardiac Repolarization: A Randomized, Placebo- and Positive-Controlled Crossover Thorough QT Study in Healthy Participants

Article

作者: Modi, Nishit B ; Darpo, Borje ; Dinh, Phillip ; Xue, Hongqi

PURPOSE:

Rusfertide, a hepcidin mimetic, is in clinical development for the treatment of polycythemia vera. This study evaluated the effect of rusfertide on cardiac repolarization.

METHODS:

A positive-controlled, randomized, double-blind, crossover study was conducted in healthy adults. Subjects received subcutaneous rusfertide 90 mg, matching subcutaneous placebo, and oral moxifloxacin. Subjects underwent continuous 12-lead Holter recording during each period. A central laboratory extracted up to 10 replicate electrocardiograms (ECGs) at each time point prior to and following treatment, paired with pharmacokinetic sampling. The primary analysis for Fridericia corrected QT interval (QTcF) was a mixed model for repeated measures with change from baseline QTcF (ΔQTcF) as the dependent variable; period, sequence, time, treatment, and time-by-treatment interaction as fixed effects; and baseline QTcF as a covariate. The relationship between rusfertide and metabolite concentrations and change-from-baseline QTcF was quantified using linear mixed-effects modeling.

FINDINGS:

Mean age and body mass index were 40.1 years and 25.4 mg/m², respectively, with a slight majority of female (57%) and White (60%) subjects. Mean rusfertide plasma concentrations increased rapidly, with median peak of 4 hours. The geometric mean rusfertide peak concentration (C_max) was 1100 ng/mL. Rusfertide did not have a clinically relevant effect on heart rate (HR) or cardiac conduction (PR and QRS interval). The least squares (LS) mean ΔQTcF on rusfertide closely followed the placebo pattern, and LS mean placebo-corrected ΔQTcF (ΔΔQTcF) varied from -2.0 to 1.8 ms. After 400 mg oral moxifloxacin, an increase of LS mean ΔΔQTcF was observed with a peak of 12.4, 12.4, and 11.2 ms at 2, 3, and 4 hours, respectively, with all three lower bounds of the 2-sided 90% confidence interval greater than 5 ms. Estimated population slope of the rusfertide concentration-QTc relationship was not statistically significant: 0.00042 ms per ng/mL (90% confidence interval [CI]: -0.001172 to 0.002013; P = 0.6624). Predicted ΔΔQTcF at the geometric mean C_max was 0.04 ms (90% CI: -1.21 to 1.29). Overall, 24 of 60 subjects (40.0%) experienced treatment-emergent adverse events (TEAEs). All TEAEs were mild. TEAEs noted in ≥5% subjects were headache, nausea, injection site erythema, injection site pain, and influenza-like illness. There were no clinically significant changes in clinical laboratory, vital signs, ECG, or physical examination findings.

IMPLICATIONS:

Rusfertide had no clinically relevant effect on HR, cardiac conduction, or QTc. Rusfertide was well tolerated. An effect on ΔΔQTcF exceeding 10 ms could be excluded within the observed rusfertide plasma concentrations up to approximately 2130 ng/mL.

2025-04-01·Clinical Pharmacology in Drug Development

Multiple‐Dose Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Subcutaneous Rusfertide, a Hepcidin Mimetic, in Healthy Subjects

Article

作者: Ifode Ajari ; Nishit B. Modi ; Phillip Dinh

Abstract:

Rusfertide, a peptide hepcidin mimetic, has shown efficacy in polycythemia vera. This trial investigated the multiple‐dose pharmacokinetics, pharmacodynamics, and safety of once‐weekly rusfertide 60 mg for 5 weeks in healthy subjects. Subjects were randomized to subcutaneous injection in the abdomen, upper arm, or thigh. Eighteen subjects were enrolled, and 15 completed the study. Geometric mean peak rusfertide plasma concentrations (Cmax) following the first dose were 547, 387, and 560 ng/mL following injection in the abdomen, thigh, and arm, respectively (P = .0054). There was no difference between injection sites in the rusfertide area under the plasma concentration‐time curve (AUC) following the first dose (P ≥ .179) or in the Cmax or AUC during the dosing interval following the last dose (P ≥ .238). Geometric mean accumulation (Dose 5/Dose 1) for AUC and Cmax was 1.5 and 1.2, respectively, and similar across injection sites. Mechanism‐based decreases in serum iron, transferrin‐iron saturation, hemoglobin, and hematocrit were noted following multiple doses. There were no differences between injection sites in the pharmacodynamic effect as measured by change from baseline in hematocrit values. There were no serious adverse events. Treatment‐emergent adverse events in 2 or more subjects were injection‐site reactions (erythema, induration, pruritus), fatigue, and headache. There were no clinically relevant findings in the safety laboratory parameters, vital signs, electrocardiograms, or physical examination. While a higher incidence of treatment‐emergent adverse events was noted in these healthy participants following multiple doses of 60 mg, rusfertide was generally well tolerated. There were no clinically meaningful differences in rusfertide pharmacokinetics or pharmacodynamics between injection sites.

205

项与 Rusfertide acetate 相关的新闻（医药）

2026-01-23

·药咖荟

J.P.摩根 week：礼来、AZ、默沙东、罗氏巨头战略布局全景图

每年年初的JP摩根医疗健康大会（JPM Healthcare Conference）被誉为全球生物医药行业的“风向标”，不仅是企业展示年度成绩与未来蓝图的关键舞台，更是洞察产业趋势、资本动向与研发重心的窗口。本文将梳理多家领军企业在本次大会中释放的核心信号，勾勒出制药行业在新时代下的竞争图景。礼来 AI赋能研发，构建“减肥王国” PART.1 礼来在肥胖及相关并发症治疗领域已建立起显著优势，但其目光已投向更广阔的“帮助更多患者”的生态构建。公司宣布在全球有13个生产基地正在建设中，以保障其代谢明星产品的全球供应。更为重要的是，礼来正积极向“全方位健康解决方案提供者”演进。公司展示了其超越药物开发的创新：1月12日，礼来与英伟达合作建设行业领先的超级计算机以加速研发；通过风险投资支持超过180家生物科技公司，构建外部创新网络；以及直接服务患者，在2025年服务患者超过100万人。在研发层面，礼来正快速推进抗体偶联药物、放射性配体疗法、siRNA和基因治疗等新平台，并通过积极的并购（2025年完成39项交易，部署超40亿美元资本）将前沿科学快速纳入自身体系，旨在从药物发现、开发到患者支持的整个价值链建立竞争优势。阿斯利康剑指2030年800亿美元营收 PART.2 阿斯利康在其展示中重申了“到2030年实现800亿美元总收入”的雄心。公司2025年前九个月表现强劲，总收入增长11%，核心每股收益增长15%。其战略核心在于五大重点治疗领域（肿瘤、心血管、肾脏与代谢、呼吸与免疫、疫苗与免疫疗法）的均衡发展，并通过全球布局与多元化业务来源支撑增长。值得关注的是，阿斯利康将人工智能定位为贯穿价值链的核心驱动力。其介绍的“AI开发代理（AIDA）”系统旨在变革端到端的合成药物开发流程，有望将CMC开发时间缩短50%。此外，定量连续评分（QCS）技术应用于计算病理学，以更精准识别治疗响应人群。在潜力技术管线方面，公司重点展示了其在体重和心血管风险管理、抗体偶联药物（ADC）、双特异性抗体、细胞疗法等前沿领域的领先布局，例如rilvegostomig在非小细胞肺癌中的数据，以及AZD0120在多发性骨髓瘤中的潜力。百时美施贵宝通过BD引领企业增长 PART.3 百时美施贵宝（BMS）提出“构建一家财务强劲、在2030年代及以后实现行业领先可持续增长的公司”的目标。公司2025年的增长产品组合销售额增长了17%，并通过战略性业务发展增强了管线。 BMS着重介绍了其具有数十亿美元潜力的管线资产，包括神经科学领域的Cobenfy（KarXT）、心血管领域的milvexian、免疫领域的admilparant、肿瘤领域的pumitamig（PD-L1 x VEGF双抗）以及血液学领域的iberdomide/mezigdomide等新型蛋白降解剂。公司预计到2030年将有超过10个新产品上市机会，并强调严格的研发执行和资本配置纪律。其资本部署策略包括投资业务、通过股息和回购回报股东，以及战略性业务开发，近期与BioNTech、SystImmune等公司的合作即是例证。默沙东构建“后K药时代”的增长支柱 PART.4 默沙东的核心战略是“用下一波创新产品组合实现转型”。公司目前拥有约80项正在进行的三期研究，预计将有超过20个新药上市，其中绝大多数具备重磅炸弹潜力。其新增长驱动力的中期商业机会已增至超过700亿美元，显著高于市场对K药在2028年销售额的共识。这一增长主要来源于通过积极的“科学驱动型业务开发”所构建的丰富管线。公司正通过外部合作与自主研发，系统性地填补管线。例如，与第一三共合作的B7-H3 ADC（I-DXd）和HER3 ADC（P-DXd），与Kelun合作的TROP2 ADC（sac-TMT），以及自身在心血管代谢（如口服Lp(a)抑制剂）、感染性疾病（如长效HIV预防药MK-8527）和免疫学（TL1A抗体tulisokibart）等领域的布局。默沙东强调，这些在临床上已降低风险的产品，其收入机会几乎能完全抵消KEYTRUDA专利到期的影响，为公司平稳过渡至2030年代奠定了坚实基础。安进六大增长引擎，新秀减肥管线 PART.5 安进强调其通过“六大关键增长驱动因素”实现长期可持续增长的战略，涵盖心血管（Repatha）、骨骼健康（EVENITY）、呼吸（TEZSPIRE）、罕见病、创新肿瘤学和生物类似药。其中，罕见病组合年化销售额已接近50亿美元，而生物类似药业务累计销售额已达约130亿美元。本次大会中，安进着重推介了其肥胖症治疗候选药物Marifide。公司公布了其在2型糖尿病和慢性体重管理方面的二期研究积极结果，强调其作为“首个月度给药型2型糖尿病疗法”的潜力，以及其在维持期治疗中展现出的良好耐受性。六项全球三期研究正在进行中，标志着安进正式加入竞争日益激烈的代谢疾病战场。诺华手握多个重磅新药 PART.6 诺华首席执行官Vas Narasimhan展示了公司在剥离山德士后的强劲增长轨迹。公司战略聚焦于心血管-肾脏-代谢、免疫学、神经科学和肿瘤学四大核心治疗领域，以及化学、生物治疗、xRNA、放射性配体疗法和基因与细胞疗法五大技术平台。公司预计2025-2030年销售额复合年增长率（CER）为5-6%，并在2029年将核心利润率恢复至40%以上。诺华在未来两年拥有超过15项可能支持申报的临床数据读出，管线中拥有30多个高价值新分子实体。近期在心血管领域新增的abelacimab（FXI抑制剂）、pacibektug（抗IL-6单抗）和farabursen（miR-17抑制剂）等项目，进一步丰富了其产品线。罗氏聚焦关键上市产品 PART.7 罗氏制药首席执行官Teresa Graham指出，公司正致力于“交付下一个创新周期”。短期战略围绕最大化在售产品组合、交付关键上市产品（如Gazyva、giredestrant、fenebrutinib）以及推进后期管线。罗氏强调其超过60%的研发管线和制药销售额涉及合作伙伴关系，展现了开放创新的模式。在肿瘤领域，下一代选择性雌激素受体降解剂（SERD）giredestrant有潜力成为ER+乳腺癌的 endocrine therapy 骨干。在神经科学领域，fenebrutinib作为唯一在复发型和原发进展型多发性硬化症三期试验中取得阳性结果的BTK抑制剂，备受关注。此外，罗氏明确表达了在肥胖症领域成为TOP3玩家的决心，预计2026年将公布五款新分子实体的三期启用数据。拜耳聚焦心血管肾病 PART.8 拜耳制药展示了其“焕新收入、提升管线价值、利用新运营模式”三大战略支柱已取得切实成果。尽管面临Xarelto的销售下滑，但新产品Nubeqa（前列腺癌）和Kerendia（心肾疾病）的持续增长势头有效地弥补了缺口。下一代产品，如用于继发性卒中预防的因子XIa抑制剂asundexian（已取得首个阳性三期结果）、用于血管舒缩症状的神经激肽拮抗剂Lynkuet，以及用于转甲状腺素蛋白淀粉样变性心肌病的Beyontra，构成了公司的下一波增长动力。拜耳特别强调了其“独特创新引擎”的重塑。新的、更聚焦的研发模型在2025年取得了五项新适应症或产品批准，完成了16个临床项目的推进。公司通过战略性交易（如从BridgeBio引进Beyontra）和投资细胞与基因治疗等平台公司，弥补了近期管线缺口，并为长远发展储备了项目。拜耳的目标是，最迟在2027年恢复中个位数增长，并在2030年将利润率提升至30%左右。赛诺菲专注创新药，重塑增长路径 PART.9 在本次JPM大会上，赛诺菲明确展示了其向纯粹“研发驱动、AI赋能”生物制药公司转型的战略定力。在完成消费者健康业务剥离后，公司正将资源全面聚焦于具备长期增长潜力的核心制药与疫苗创新。 2025年的业绩初步验证了这一路径。通过积极的“买与卖”，公司在收购Blueprint、Dynavax等强化管线的同时，完成了大规模股份回购。前九个月销售额增长8.7%，12款新产品贡献了39亿欧元收入，其中血友病A疗法ALTUVIIO已成为新的重磅炸弹。公司的未来增长高度依赖于其聚焦免疫炎症、呼吸、罕见病及疫苗的中后期管线。核心资产包括：在特应性皮炎领域已进入三期的OX40L单抗amlitelimab、在化脓性汗腺炎显示潜力的IL-17A/F双抗brivekimig，以及与阿斯利康合作、市场表现强劲的RSV预防药物nirsevimab。赛诺菲强调，2026年将是管线价值验证的关键一年，多项重要临床数据即将读出。其战略核心在于，通过彻底的业务聚焦和资本配置，将科学潜力转化为一系列成功的上市产品，重塑其作为领先创新药企的增长轨迹。武田三款重磅产品重塑增长曲线 PART.10 武田将其发展周期划分为两个阶段：FY2021-2025是管理专利悬崖、聚焦研发和建设数字化能力的“蓄力期”；而FY2026-2030+则是凭借变革性后期管线驱动增长的“收获期”。公司明确指出，未来18个月内有望上市的三款新药——用于发作性睡病1型的orexin受体2激动剂TAK-994、用于真性红细胞增多症的hepcidin模拟物rusfertide、以及用于银屑病的高选择性TYK2抑制剂zasocitinib——将把公司带入全新的增长轨道。每款产品都瞄准了存在显著未满足需求的领域，并具备成为标准疗法的潜力。例如，rusfertide在三期研究中展现出持久且稳定的血细胞比容控制能力，能大幅降低患者放血治疗需求，有望革新真性红细胞增多症的全程管理。武田强调，这八款具有数十亿美元潜力的在研产品，加上在2030年前有限的仿制药冲击，将支持公司在投资新药上市和研发的同时，实现利润率的改善。第一三共坚定迈向全球肿瘤领域领导者 PART.11 第一三共明确宣示其目标：“顶级ADC公司，成为全球肿瘤领导者”。公司凭借其独创的DXd ADC技术平台，已衍生出7款在研药物，其中ENHERTU®（trastuzumab deruxtecan）已成为过去五年肿瘤领域最成功的上市产品之一，累计销售额超过1.5万亿日元。DATROWAY®（datopotamab deruxtecan）也展现出快速增长的势头。公司计划到2030年在至少4款ADC药物中推出多项新适应症，覆盖患者数预计将增长近六倍。此外，第一三共正积极布局免疫肿瘤学（IO）及其他新模态（如多特异性抗体、基因治疗等），构建超越ADC的多元化技术矩阵，以确保持续创新。吉利德 HIV领导者与多元化管线并进 PART.12 吉利德的核心信息围绕三大支柱展开：延续至2040年代的HIV治疗与预防领导地位、公司历史上最强劲的临床与上市管线，以及对运营开支和并购的积极主动且 disciplined 的策略。其HIV业务预计2025年收入将超过200亿美元，比卡替拉韦/恩曲他滨/丙酚替诺福韦（Biktarvy）市场地位稳固，而长效注射剂Yeztugo（lenacapavir）用于HIV暴露前预防（PrEP）的上市正开辟全新格局。在肿瘤领域，Trodelvy在转移性三阴性乳腺癌一线治疗的数据，以及BCMA CAR-T疗法anito-cel在复发/难治性多发性骨髓瘤中的潜力，预示着吉利德在肿瘤领域的增长动能。公司预计2026年将有最多10款产品处于上市或即将上市阶段。渤健神经与免疫管线接棒增长 PART.13 渤健正致力于讲述一个“创新与增长新篇章”的故事。公司通过“Fit for Growth”计划完成了成本重组（节省10亿美元），并重建了研发管线，将资源集中于高确信度的项目。目前，其上市新产品（如阿尔茨海默病药物LEQEMBI、弗里德赖希共济失调药物SKYCLARYS等）的收入增长已完全抵消了多发性硬化症产品线的下滑。公司的长期战略依赖于三波增长动力：当前的增长产品、后期注册管线（如狼疮药物litifilimab、IgA肾病药物felzartamab等）以及早期高风险/高回报项目。2026年被渤健视为转型的关键一年，将迎来litifilimab的两项三期数据读出、LEQEMBI皮下制剂的监管决定以及多个早期项目的重要进展。渤健正努力从一个依赖成熟产品的公司，转型为一个由新上市产品和丰富管线驱动的增长型生物科技企业。结语纵观本届JPM大会，制药巨头的战略共性凸显：首先，AI和数字化技术渗透至研发、生产及商业化全链条，以提升效率与精准性；其次，肿瘤、代谢疾病、神经科学成为管线竞争焦点，ADC、细胞疗法等前沿平台屡见突破；最后，资本配置更趋理性，企业通过股息、回购与选择性并购平衡短期收益与长期创新。在患者需求与政策变革的双重驱动下，行业正迈向一个更高效、更个性化的新纪元。参考资料： 1. 2026年摩根大通医疗健康大会官网 END △ 聚焦“国之大者”CBIITA 联合体荣获“中国产学研合作十大好平台” 更多优质内容，欢迎关注媒体合作投稿转载/资料领取/加入社群请添加药小咖与/智/者/同/行为/创/新/赋/能

2026-01-16

·医药地理

2026JPM巨头亮剑！揭秘MNC王牌布局（附PPT)

2026年1月，第44届JPM医疗健康大会如期而至。全球顶尖跨国药企齐聚旧金山，在这场被誉为“医药界风向标”的盛会上，纷纷亮出未来五至十年的战略底牌。后疫情时代，行业竞争已从单一产品比拼，升级为平台技术、管线布局、全球运营与生态构建的全方位较量。本文将深度解析各大巨头在2026年的战略重心与王牌布局，揭示下一轮增长背后的逻辑与野心。默沙东（Merck & Co.）通过“科学驱动型业务发展”持续加码ADC。与第一三共合作开发的I-DXd（B7-H3 ADC）和P-DXd（HER3 ADC）已进入Ⅲ期临床，同时与科伦博泰合作开发的芦康沙妥珠单抗（TROP2 ADC）也在探索新适应症。默沙东的策略是：以KEYTRUDA为基石，通过ADC、双抗等多元技术构建肿瘤免疫联合治疗矩阵，打造超过700亿美元的商业机会。 Pharma ONE药物研发大数据平台显示，2025年芦康沙妥珠单抗（MK-2870/Sac-TMT）在全球范围内针对三阴性乳腺癌、HR阳性/HER2阴性乳腺癌、子宫内膜癌及卵巢癌等多种实体瘤，启动了多项国际多中心III期临床试验，评估其单药或与帕博利珠单抗等联合治疗的疗效与安全性。芦康沙妥珠单抗2025年关键III期临床试验一览来源：Pharma ONE药物研发大数据平台，中国医药工业信息中心点击“阅读原文”获取更多数据第一三共（Daiichi Sankyo）明确提出“成为顶级ADC公司，迈向全球肿瘤领导者”。其DXd-ADC技术平台已催生7款候选药物，ENHERTU（HER2 ADC）累计销售额突破1.5万亿日元，DATROWAY（TROP2 ADC）亦快速放量。公司计划到2030年在至少4款ADC上推出多个新适应症，覆盖患者数预计增长近6倍。第一三共的野心不仅在于输出产品，更在于确立ADC技术标准与平台领导力。诺华（Novartis）诺华聚焦四大核心治疗领域：心血管-肾-代谢、免疫、神经科学及肿瘤，依托“2+3”技术平台（化学与生物治疗为基础，xRNA、放射性配体、基因与细胞治疗为前沿），深耕美、中、德、日四大市场，通过内生研发与外部并购（如Avidity）驱动增长。其重磅管线实力雄厚：心血管领域布局abelacimab（FXI抑制剂）、pacibektug（抗IL-6单抗）及farabursen（miR-17抑制剂）三个潜在重磅药物；免疫学拥有CAR-T疗法YTB323与高亲和力IL-15抗体GIA632；神经科学重点推进remibrutinib（MS）及RNA疗法（DM1/FSHD）；肿瘤学则深化放射性配体治疗（RLT）平台，拓展Pluvicto在前列腺癌中的应用并探索新型组合。未来两年预计超15项关键临床数据提交，支撑其中长期销售中高个位数增长及2029年核心利润率重返40%以上的目标。礼来（Eli Lilly）作为全球首家“万亿俱乐部”的药企，礼来正进入“治疗肥胖及相关并发症的新时代”，战略上持续扩大产能（全球13个在建基地）并加速研发效率——其从发现到首发的周期比行业平均快3.5年，2015–2025年间共推出24个新分子实体（NMEs）。管线方面，公司重点布局抗体药物偶联物（ADC）、放射性配体疗法、siRNA、基因医学等新一代技术平台，以解决未满足的临床需求。此外，礼来积极通过外部合作与收购增强研发实力，2025年完成39笔交易，投入资本超40亿美元，并携手NVIDIA打造行业顶级超算平台，支持生物科技创新。整体战略围绕“超越分子”的创新，整合疗法、数字化与合作生态，旨在服务更广泛患者群体。罗氏（Roche）公司正着力于“下一个创新周期”，其战略核心是通过商业卓越执行、关键产品上市和管线焕新来维持增长势头。罗氏计划最大化现有产品组合（拥有17个增长强劲的重磅药物），并积极推进如giredestrant（乳腺癌）、fenebrutinib（多发性硬化症）及Gazvya（自免疾病）等关键产品的上市与拓展。公司明确目标成为全球肥胖治疗领域前三的企业，计划在2026年公布5款肥胖新药的III期启用数据。此外，罗氏高度重视合作与收购，其60%的研发管线及药品销售均涉及合作伙伴，以此加速创新。管线方面，公司布局了多达19款具备上市潜力的新分子实体，覆盖阿尔茨海默病、肿瘤、免疫等多个领域，其中多款首创/最佳（FIC/BIC）药物预计自2027年起陆续读出关键数据，旨在持续推出具有变革潜力的疗法。辉瑞（Pfizer）公司2026年的战略布局围绕四大重点展开：最大化关键交易价值（如对Seagen等的整合）、实现关键研发里程碑、投资布局2028年后增长，并规模化应用人工智能以提升整体运营效率。在肿瘤领域，多项重要资产如PADCEV（肌层浸润性膀胱癌）、ELREXFIO（多发性骨髓瘤）及PD-1xVEGF双抗（结直肠癌、肺癌等）将迎来重大进展；在免疫领域，LITFULO（利特昔替尼）将拓展至白癜风及中重度斑秃；在疫苗与预防领域，莱姆病候选疫苗及下一代肺炎球菌疫苗PCV25将推进关键研究；尤为值得注意的是其在代谢疾病的深入布局，超长效GLP-1单药及GLP-1+胰淀素复方制剂已进入中后期临床，显示出其进军肥胖治疗市场的强大决心。辉瑞正通过强化内部研发与外部收购，构建一个覆盖肿瘤、免疫、疫苗和代谢等核心治疗领域的多元化、具有持续增长潜力的产品组合。拜耳（Bayer）公司战略围绕“三支柱”展开：更新产品线、提升管线价值以及利用新运营模式。当前增长主要由前列腺癌药物Nubeqa、心肾保护药物Kerendia及眼科产品Eylea驱动，同时下一波增长将依托新上市产品Beyontra（ATTR-CM）、Lynkuet（更年期症状）及在研重磅药物Asundexian。Asundexian作为首个完成III期研究的Factor XIa抑制剂，在预防中风复发中显示出显著疗效且未增加大出血风险，具备成为变革性疗法的潜力。拜耳正通过内部研发、平台公司（如细胞与基因治疗资产）及外部合作（如与BridgeBio、Kumquat等的交易）持续补充管线，并将在2026年迎来多项关键里程碑，包括Asundexian的数据展示及Kerendia在非糖尿病慢性肾病中的III期结果。整体战略旨在重返增长轨道，并为长期发展奠定创新与效率基础。赛诺菲（Sanofi）一家 “由研发驱动、人工智能赋能的生物制药公司” ，正积极通过外部收购（如Blueprint、Dynavax）与内部研发强化管线，并持续优化产品组合与资本配置。当前增长主要由新上市产品驱动，其中血友病药物ALTUVIIIO已成为新的重磅产品。在免疫炎症、罕见病及疫苗领域，多项资产在2025年取得关键进展，包括 amiltelimab（特应性皮炎）的III期积极数据、brivekimig（化脓性汗腺炎）的II期数据，以及针对流感、狂犬病、RSV的疫苗项目。公司预计2026年将继续迎来密集的管线进展，包括lunsekimig、duvakitug（炎症性肠病）及黄热病疫苗等项目的推进，为其长期增长奠定基础。武田（Takeda）正处于增长的关键拐点，其战略核心是成为一家以创新为导向的全球生物制药企业，聚焦于胃肠道与炎症、神经科学、肿瘤三大核心治疗领域，并全面嵌入数据、数字与技术（DD&T）以提升研发与运营效率。武田计划在2026至2030年间推出多达8款具有重磅潜力的创新药物，并在未来18个月内上市三款变革性药物：overpextron（发作性睡病1型）作为首款提交NDA的食欲素激动剂，有望重塑该领域治疗格局；rusfertide（真性红细胞增多症）作为一种铁调素模拟物，能持久控制血细胞比容，满足显著未满足的临床需求；以及zasocitinib（银屑病）作为高选择性TYK2抑制剂，凭借其口服便利性和优异疗效，旨在拓展并变革高级口服疗法市场。百济神州（BeOne）致力于成为“全球领先的肿瘤公司”，以核心产品BRUKINSA（泽布替尼）为基础，通过固定疗程组合疗法ZS 拓展一线CLL市场，旨在提供兼具疗效、安全性与便利性的新一代治疗方案。在管线方面，百济神州着力推进新一代BCL-2抑制剂Sonrotoclax（已获FDA突破性疗法认定）及首创的BTK靶向蛋白降解剂（BTK CDAC）如BGB-16673，以解决耐药患者未满足的需求。同时，公司积极布局多特异性抗体、细胞疗法等新模态，计划在2026年将多款新分子实体推进至临床阶段，持续构建涵盖CLL全线治疗的领导地位，并向更广泛的肿瘤领域拓展。药明生物（WuXi Biologics）以“让天下没有难做的生物药”为愿景，打造全球领先的大规模、一体化CRDMO（合同研究、开发与生产）平台，通过持续强化在美国、新加坡及中东（如卡塔尔）等关键区域的产能与服务体系，构建覆盖生物药全生命周期的全球化、端到端服务网络。公司以双特异性/多特异性抗体、抗体偶联药物（ADC）及复杂蛋白为主要增长引擎，其中双多抗业务已成为增速最快、盈利能力最强的板块，贡献集团近20%的收入，同时依托差异化技术平台（如CD3 TCE、高剂量给药、数字化系统）和大规模生产经验，持续赋能合作伙伴推进管线。未来五年，医药行业的竞争将更加多维和立体。技术领先者将凭借平台优势持续输出新产品；疾病领域专家将在细分赛道建立护城河；生态构建者则通过诊断、治疗、服务的一体化方案赢得患者与支付方。在这场没有硝烟的战争中，唯有那些战略清晰、执行坚决、持续创新的企业，才能穿越周期，赢得未来。 2026，巨头已亮剑，战局正酣。对此您有什么看法，欢迎在评论区留言分享。关注「医药地理数据库」并回复“2026JPM”，即可获取完整PPT END 👇欢迎扫码入群 ①权威报告获取 ②医药行业情报 ③专属交流平台如需获取更多数据洞察信息或公众号内容合作，请联系医药地理小助手微信号：pharmadl001 戳“阅读原文”试用数据库

2026-01-16

·BioSpace

JPM26: Protagonist Has a $400M Decision To Make. It’s a No-Brainer

iStock, SIphotography Following rusfertide’s triumphant Phase III trial last year, Protagonist must decide how involved to be in future development. Hundreds of millions of dollars are on the line. Protagonist CEO Dinesh Patel has a big decision to make. It could be worth as much as $400 million—and much more later on. The submission of a new drug application for rare blood cancer therapy rusfertide by partner Takeda at the end of December triggered a three-month countdown for Protagonist to decide whether to opt in or out on 50-50 co-development. If Protagonist opts out, the company would be eligible for $400 million in opt-out payments, larger milestone payments and 14%–29% tiered royalties on worldwide sales. “Now the clock is ticking,” Patel told BioSpace on the sidelines of the J.P. Morgan Healthcare Conference in San Francisco on Wednesday. Opting out would also allow Protagonist to leave the development and commercial process to Takeda, clearing the way for an increased focus on the biotech’s wholly owned pipeline. “What we have started expressing is we are strongly leaning towards opting out,” Patel said. “And the reason for that is like the economics around opting out are just amazing.” That’s because rusfertide aced its clinical program. The drug is being developed for polycythemia vera, a type of rare and incurable cancer that causes the bone marrow to produce too many red blood cells. Patients are currently treated by removing blood from the body to manage elevated hemocrit levels—a procedure that can cause fatigue, visual disturbances and other complications. In the Phase III VERIFY trial that read out last year , rusfertide reduced the need for blood withdrawals and lowered hemocrit levels as compared to placebo. The secondary endpoints, which looked at symptoms and treatment improvements, were also met. “All of a sudden it’s like, oh, this could be a big drug,” Patel said. The results put the 29% royalty in sight, Patel said. If the trial had not been so successful, opting in would have been a better option with lower potential royalties expected. But now, for any sales beyond $1.5 billion, Protagonist will receive 29% royalties. Meanwhile, Takeda will be doing the API sourcing, manufacturing, erecting the sales force to drive prescriptions and setting up direct-to-consumer engagement. “Without lifting a finger, I’m getting such a big share,” Patel said. The FDA has granted priority review for rusfertide, and a decision is expected in the next few months or by August, according to Patel. Phase III Takeda-Protagonist Drug for Incurable Cancer Reduces Need for Blood Withdrawals in Phase III Trial Protagonist Therapeutics notches a milestone in its pact with Takeda for rusfertide. New data show that many patients with a chronic blood cancer taking the drug didn’t need to have their blood removed to bring down dangerously high hemocrit levels. March 3, 2025 · 2 min read · Annalee Armstrong Gold Nuggets Rusfertide is not Protagonist’s only partnership—or its only new drug application before the FDA. The company is also working with Johnson & Johnson on icotrokinra, an IL-23 receptor blocker under development for multiple immunology and inflammatory diseases. The drug has been heralded as a “new standard of treatment” in plaque psoriasis after Phase III results were released in March 2025 . The NDA was filed in July last year, with a decision expected in the middle of the year. Meanwhile, J&J—and Takeda—are working hard to launch Protagonist’s therapies right away. “That’s a very nice setup,” Patel said. J&J was rumored to be interested in acquiring Protagonist over the fall. A deal has yet to materialize, and Patel declined to comment. But he did say that Protagonist has no interest in becoming a commercial company. The focus will remain on developing new treatments, including a handful of oral obesity assets that will go up against Novo Nordisk and Eli Lilly. The partnership structure the team has built means Protagonist has a solid financial outlook, with milestones to support along the way. “We are laser-focused on the future,” Patel said. “All the nuggets we have in the next wave of R&D.”

临床3期优先审批申请上市

100 项与 Rusfertide acetate 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D12064	-	-	-

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
真性红细胞增多症	申请上市	美国	Takeda Pharmaceutical Co., Ltd.	2026-01-05
血色素沉着症	临床2期	美国	Protagonist Therapeutics, Inc.	2020-03-19
血色素沉着症	临床2期	加拿大	Protagonist Therapeutics, Inc.	2020-03-19
β地中海贫血	临床2期	美国	Protagonist Therapeutics, Inc.	2018-12-19
β地中海贫血	临床2期	希腊	Protagonist Therapeutics, Inc.	2018-12-19
β地中海贫血	临床2期	意大利	Protagonist Therapeutics, Inc.	2018-12-19
β地中海贫血	临床2期	黎巴嫩	Protagonist Therapeutics, Inc.	2018-12-19
β地中海贫血	临床2期	马来西亚	Protagonist Therapeutics, Inc.	2018-12-19
β地中海贫血	临床2期	泰国	Protagonist Therapeutics, Inc.	2018-12-19
β地中海贫血	临床2期	突尼斯	Protagonist Therapeutics, Inc.	2018-12-19

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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


NCT06033586 (THRIVE, ASH2025)	临床2期	真性红细胞增多症	46	Rusfertide	積繭醖鹽窪構夢鏇鹹憲(製獵觸衊憲廠製製襯簾) = the most common AEs in the pts who participated in REVIVE and THRIVE were Grade 1-2 injection site reactions (87%), fatigue (59%), COVID-19 (50%), pruritus (44%), arthralgia (39%), dizziness (35%), anemia (30%), paresthesia (28%), headache (26%), abdominal pain and nausea (24% each), and diarrhea, dyspnea, and upper respiratory tract infection (22% each). 憲製艱壓獵製蓋構艱製 (壓夢糧鬱襯膚窪積蓋網 ) 更多	积极	2025-12-06
NCT05210790 (VERIFY, ASH2025)	临床3期	真性红细胞增多症	293	Rusfertide	選範壓廠觸窪獵憲簾網(廠鬱顧遞醖獵醖蓋鏇網) = 餘夢顧獵構廠築衊願獵蓋憲簾艱鹹鏇選選積繭 (製夢築願範製衊壓餘繭 )	积极	2025-12-06
				Placebo	鬱構壓膚廠鹽餘繭獵繭(觸壓觸繭積餘鹹積衊糧) = 鏇構構積觸築製廠簾構願壓壓鑰鬱廠範憲鹽繭 (鬱鹽夢鏇衊鏇積鹽觸範 ) 更多
NCT05210790 (VERIFY, BusinessWire) 人工标引	临床3期	真性红细胞增多症	293	Rusfertide	獵鹹廠壓願鑰繭觸製觸(簾鏇獵衊製膚網觸膚衊) = 鬱鹹鏇簾壓憲鹽憲願糧艱構築觸積積網糧鹹鑰 (衊鹽鑰構壓製積選選範 ) 达到更多	积极	2025-03-03
				Placebo	獵鹹廠壓願鑰繭觸製觸(簾鏇獵衊製膚網觸膚衊) = 築積憲鬱壓築網憲築憲艱構築觸積積網糧鹹鑰 (衊鹽鑰構壓製積選選範 ) 达到更多
NCT04057040 (REVIVE, ASH2024) 人工标引	临床2期	真性红细胞增多症	70	rusfertide	襯獵壓壓壓築鏇壓選鹽(繭齋鏇鹽築憲糧醖鏇鏇) = The most common (≥20%) treatment-emergent adverse events (TEAEs) were injection site reactions (85.7%), fatigue (38.6%), COVID-19 (32.9%), pruritus (34.3%), arthralgia (30.0%), dizziness (30.0%), nausea (24.3%), headache (24.3%), and anemia (21.4%). 廠鏇壓積鏇壓齋窪鑰繭 (顧夢淵積遞蓋構顧壓積 )	积极	2024-12-09
NCT04057040 (REVIVE, EHA 12024 \| EHA 22024) 人工标引	临床2期	真性红细胞增多症	70	Rusfertide	願醖蓋選襯膚鑰壓築遞(糧繭膚簾顧範夢繭鹹憲) = Platelets increased by approximately 30% post-baseline within 4 weeks following initiation of rusfertide therapy 醖糧築齋繭醖鬱獵壓遞 (廠範夢選憲範襯淵繭積 ) 更多	积极	2024-05-14
				Placebo
NCT04057040 (REVIVE, Literature) 人工标引	临床2期	真性红细胞增多症	70	Rusfertide	鏇鏇鹹製夢築壓鬱製構(蓋鹽衊鹹選憲窪膚壓鏇) = 獵廠選鬱衊膚繭鹹壓製衊糧鏇觸醖膚鹹鹽憲餘 (醖構餘鹹廠願齋餘襯壓 ) 达到	积极	2024-02-22
NCT04767802 (PACIFIC \| REVIVE, Biospace) 人工标引	临床2期	真性红细胞增多症	70	Rusfertide	窪膚網餘襯淵網餘壓糧(願鹹艱鑰選構鬱製齋壓) = 艱積鹹壓醖窪遞窪廠繭觸鑰餘糧廠繭襯範選鹹 (蓋網獵鬱艱範蓋積憲遞 )	积极	2023-12-12
				Rusfertide	窪膚網餘襯淵網餘壓糧(願鹹艱鑰選構鬱製齋壓) = 構鏇遞糧膚製築顧選鹽觸鑰餘糧廠繭襯範選鹹 (蓋網獵鬱艱範蓋積憲遞 )
NCT04202965 (Pubmed \| Lancet Gastroenterol Hepatol)	临床2期	铁过剩	-	Rusfertide 10 mg	網淵鏇願襯夢艱構願鹽(構鹽廠繭製醖鹹夢艱淵) = 12 (75%) patients had at least one TEAE, the most common of which was injection site pain (five [31%] patients). All TEAEs were mild or moderate in severity, except for a serious adverse event of pancreatic adenocarcinoma, which was considered severe and unrelated to treatment and was pre-existing and diagnosed 21 days after starting rusfertide treatment. 構艱餘簾鏇艱範憲顧壓 (選夢簾觸鹹網築製窪選 )	-	2023-12-01
SOHO2023	N/A	真性红细胞增多症	-	Rusfertide	窪鏇構構壓範願繭網膚(糧築製夢獵鹹壓淵網製) = 83% of treatment-emergent adverse events (TEAEs) were grade 1-2, 17% were grade 3, with none grade 4 or 5. Most common TEAEs were localized grade 1-2 injection site reactions. 鑰獵膚鏇膚顧鏇鹹鹽鹹 (窪構夢選窪觸衊繭襯鑰 )	-	2023-09-01
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NCT04202965 (CTgov)	临床2期	血色素沉着症	16	PTG-300	選遞鹽積顧顧築鏇壓壓(淵夢壓繭齋蓋醖鏇觸構) = 廠廠夢壓糧窪選鏇壓鹽鹹鹹遞鬱醖遞淵鏇餘壓 (壓窪鏇鏇網齋憲餘遞膚, 19.4) 更多	-	2023-06-15