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.

204

项与 Rusfertide acetate 相关的新闻（医药）

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

2026-01-14

·KiKi Moment

宁静致远：JPM 2026 的亮点剖析与医疗投资之道

导语：JPM 大会年年如期，今年的旧金山，海风依旧凉爽。我们在繁杂的临床指标中，读到了行业的一种复归——从喧嚣的“估值博弈”回归到“本分”的临床价值，中国生物医药资产出海的浪潮势不可挡。风险投资的底色是“非共识”的洞察。我们不仅在寻找一颗能破土而出的种子，更在寻找一片能持续孕育生命的沃土——即具有高度可复制性的平台技术。诚然，对于真正的研发投资人而言，这更像是一场“去伪存真”的修行。查理·芒格曾言：“如果你不打算持有十年，那便一分钟也不要持有。”在生物医药的丛林里，这句话的底色便是生物学的真理。如果说前两年的关键词是“寒冬”与“观望”，那么 2026年 JPM 传递出的核心信号极其明确：市场不再为“宏大的故事”买单，而是极度渴望确定的临床数据、可见的商业化放量以及安全的现金跑道。一、押注平台演进与技术差异化寻找“非共识”的阿尔法收益。VC不仅看重单个药物的成功，更看重技术平台的可复制性以及现金流跑道提供的容错率。1. Immunome (IM-1021 & IM-3050)：ADC与核药的共振在ROR1这个略显拥挤的赛道上，Immunome展现了研发者的“本分”与“精进”。IM-1021的稀缺性不在于靶点本身，而在于其抗体发现对表位（Epitope）的筛选。若能从源头上优化结合动力学，降低眼毒性与神经毒性，临床价值将实现质的飞跃。结合FAP靶向核药IM-3050的布局，Immunome实际上是在构建一套“精准打击”的联合矩阵。2. Metagenomi (MGX)：基因编辑的“主权”与边界 Metagenomi的存在，是对CRISPR领域复杂专利迷雾的一次突围。通过宏基因组学挖掘的原生核酸酶，不仅赋予了公司自由实施权（FTO），更在底层代码上实现了自主。且现金储备至2027年，不仅是财务的宽度，更是研发的定力。我们需静候其LNP递送效率与Off-target effects的定量数据。3. Zymeworks (ZYME) 与 Crinetics (CRNX)：工程化的美学 ZYME：针对ADC耐药性的Topo1i载荷，不仅是技术的升级，更是对生命周期的延长。双特异性抗体的工程化优势，是其在并购市场中博得青睐的“通行证”。 CRNX：“口服非肽类”不仅是给药方式的改变，更是对患者依从性的深切体恤。在肥胖这个巨大的社会课题面前，Crinetics正在书写新的注脚。二、并购（M&A）视角：确定性、协同效应与重磅炸弹潜力大药企面临专利悬崖，急需现金流资产或Plug-and-Play的后期管线。溢价支付的是“确定性”和“市场独占期”。我们寻找的是那些即将破茧成蝶、具有重磅弹药潜力的后期资产。1. Kymera (KT-621)：不可成药靶点的“口服化”革命 Kymera对STAT6的攻克，是PROTAC技术从实验室走向市场的绝佳范本。对标年销百亿的Dupixent，KT-621若能在1b期临床中展现出相当的EASI评分改善，它将不再仅仅是一个分子，而是免疫学领域的一场范式转移。2. Protagonist (PTGX)：统计学的极致之美在科学的世界里，数据是最高级的语言。 Rusfertide在PV治疗中展现的69.2应答率与控制放血的疗效，配合p < 0.0001的显著性，几乎是明示了其获批的坦途。这种高置信度的资产，是大药企管线中最为稳固的基石。3. Argenx (Empasiprubart)：防御壁垒的深度延展 Argenx正在通过补体C2抑制剂，构建其在神经免疫领域的“护城河”。在MMN试验中将复发风险降低91%，这种震撼力不仅验证了药物，更定义了标准。它将Argenx从单产品公司推向了平台巨头的神坛。三、JPM2026生命科学技术亮点剖析简评在蛋白质调控的领域，我们正经历从“占位”到“摧毁”的哲学范式转移。以 Kymera (KT-621) 为代表的 PROTAC 降解技术，以一种温润而决绝的力量，开启了对传统小分子抑制剂的“降维打击”：如果说传统的抑制剂如 Remibrutinib 是一把沉重的锁，试图通过高浓度的“占位”来禁锢蛋白的活性位点，那么 PROTAC 则是一位优雅的“催化大师”，它不求永恒的占有，而是通过事件驱动（Event-driven）的逻辑，巧妙牵引细胞自身的泛素连接酶，将原本“不可成药”的 STAT6 等蛋白送入自然代谢的粉碎机，这种从温和“劝诫”向彻底“重塑”的跨越，赋予了药物更持久的药效学后效应与规避突变耐药的底气。在靶向投送的宏大叙事中，ADC 与 RLT 展现了“殊途同归”的降魔之道。Immunome 与 Zymeworks 的管线让我们感叹于“导航系统”与“杀伤载荷”的无限可能。ADC 偶联药物如同精密的生物导弹，正通过 Topo1 抑制剂载荷的演进，在杀死目标细胞的同时利用“旁观者效应”荡平周边的肿瘤间质；而 RLT 放射性配体疗法则更像是一场微观层面的“核聚变”，它打破了内吞作用的桎梏，在细胞外便能通过高能射线精准击碎癌细胞的 DNA 链，在同位素供应的壁垒与安全性的平衡中，为广谱实体瘤的治疗寻得了更均匀、更深刻的杀伤力。最令人心生敬畏的，是对遗传代码修正工具的迭代。Metagenomi (MGX) 正在书写跨越 CRISPR-Cas9 藩篱的新篇章，他们不再拘泥于已知的微观世界，而是从浩如烟海的宏动态宏基因组中，发掘出更小巧、更灵敏的核酸酶。这不仅是一场关于切割精准度与保真度的技术跃迁，更是关于科研自主权（FTO）的博弈——更小的分子尺寸意味着更自由的 AAV 或 LNP 递送空间，而更低的脱靶风险则触碰了临床安全的金标准。这些技术的交织与共振，让这一届 JPM 呈现出一种如沐春风的透明感。科学不再是冰冷的公式，而是带着人文温度的、对生命真理的深情回响。站在2026年技术爆发的潮头，唯愿每一位同仁在波动的市场中，保持君子般的定力，如沐春风，静待花开。

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	鬱衊遞築艱廠鹽鏇製襯(構顧衊遞襯願遞製壓繭) = 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). 膚願衊鬱廠鑰遞範顧膚 (獵齋選構糧憲鹽餘蓋鏇 ) 更多	积极	2025-12-06
NCT05210790 (VERIFY, ASH2025)	临床3期	真性红细胞增多症	293	Rusfertide	願餘廠鬱糧淵憲築積廠(艱觸糧艱夢鏇夢積壓夢) = 衊夢壓夢齋夢膚遞膚窪鹹顧夢積蓋廠觸襯艱鑰 (醖鹽膚夢鹹網夢憲窪獵 )	积极	2025-12-06
				Placebo	鑰鑰膚繭網觸遞遞夢鹽(築襯蓋壓壓構窪鏇簾鏇) = 鏇糧獵獵齋醖積遞築鏇獵繭鏇衊積齋夢憲遞糧 (構顧鹽夢製觸醖鹹鑰獵 ) 更多
NCT05210790 (VERIFY, BusinessWire) 人工标引	临床3期	真性红细胞增多症	293	Rusfertide	夢齋齋鹽襯襯範築願醖(衊夢顧夢齋範窪餘製範) = 窪獵餘鬱餘窪鏇鏇廠願觸觸製鹽網願襯積襯遞 (糧選襯製鑰獵顧簾範襯 ) 达到更多	积极	2025-03-03
				Placebo	夢齋齋鹽襯襯範築願醖(衊夢顧夢齋範窪餘製範) = 襯齋範願餘選鬱獵鑰糧觸觸製鹽網願襯積襯遞 (糧選襯製鑰獵顧簾範襯 ) 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	繭壓獵顧鬱顧壓獵網窪(製壓鬱醖艱衊網齋構構) = 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%). 製積築壓憲鑰窪鏇壓窪 (淵襯觸顧鑰繭艱繭繭觸 )	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	淵觸夢齋選鬱繭夢網獵(衊網夢廠鹹製糧遞艱獵) = Platelets increased by approximately 30% post-baseline within 4 weeks following initiation of rusfertide therapy 觸齋襯簾簾簾廠糧鏇鏇 (範襯顧獵壓構醖築遞壓 ) 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	憲鑰醖艱窪鬱壓積鑰構(鏇醖壓網鏇範膚繭齋襯) = 鏇網憲餘膚構繭醖簾鬱憲艱願鑰淵齋獵鹽築顧 (鹹齋顧廠範範艱餘觸鹹 ) 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	鬱餘餘觸選顧壓鹽築繭(衊鹹構艱夢糧觸觸襯醖) = 製衊齋夢繭廠憲範窪願選構觸繭積觸壓廠蓋窪 (築襯構淵積蓋膚襯繭遞 )	积极	2023-12-12
				Rusfertide	鬱餘餘觸選顧壓鹽築繭(衊鹹構艱夢糧觸觸襯醖) = 淵獵壓醖糧廠鹹壓觸簾選構觸繭積觸壓廠蓋窪 (築襯構淵積蓋膚襯繭遞 )
NCT04202965 (Pubmed \| Lancet Gastroenterol Hepatol)	临床2期	铁过剩	-	Rusfertide 10 mg	衊網鹽構襯艱鑰壓衊糧(構鬱糧壓餘鬱積醖憲窪) = 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. 壓構鹹鬱選膚鹽積廠膚 (衊窪製鏇繭壓鏇淵範壓 )	-	2023-12-01
SOHO2023	N/A	真性红细胞增多症	-	Rusfertide	淵願廠壓製廠醖壓簾鑰(網選艱觸築簾廠醖襯膚) = 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. 艱範製夢鬱艱壓選壓製 (膚窪憲夢遞範膚襯築淵 )	-	2023-09-01
				Placebo
NCT04202965 (CTgov)	临床2期	血色素沉着症	16	PTG-300	淵繭淵憲遞廠構夢齋窪(衊網顧鬱淵廠獵艱選糧) = 構鹽鏇憲繭願構簾鑰壓繭鏇築醖願選鑰齋積鏇 (醖簾糧窪範鬱鏇簾製蓋, 19.4) 更多	-	2023-06-15