A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Safety and Efficacy of Fazirsiran in the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease With METAVIR Stage F1 Fibrosis

In some people, the liver makes an abnormal version of the alpha-1 antitrypsin (AAT) protein, called Z-AAT. Making an abnormal version of the AAT protein can result in liver disease as Z-AAT builds up in liver cells, which leads to liver problems such as liver scarring (fibrosis), continuing liver damage (cirrhosis), and eventually endstage liver disease. Fazirsiran is a medicine that reduces the creation of the Z-AAT protein and thus the build-up of this abnormal protein in the liver. People with this type of liver disease who already have mild liver scarring will take part in the study. They will be treated with fazirsiran or a placebo for about 2 years. This study will check the long-term safety of fazirsiran and if participants tolerate the treatment. A liver biopsy, a way of collecting a small tissue sample from the liver, will be taken twice during the study.

开始日期2024-03-01

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

NCT05891158 / Recruiting临床1期

An Open-Label, Phase 1 Study to Evaluate the Pharmacokinetics, Safety, Tolerability, and Pharmacodynamics in Response to a Single Subcutaneous Dose of Fazirsiran (TAK-999) in Subjects With or Without Hepatic Impairment

The main aim of this study is to learn how the body processes fazirsiran (pharmacokinetics [PK]) in people with mild, moderate, or severe liver problems, compared to people with normal liver function.
The study will include participants with liver scarring (cirrhosis) and mild, moderate, or severe liver problems, and participants with normal liver function. All participants will be given 1 injection of fazirsiran and will be followed up for 6 months after the fazirsiran injection.

开始日期2023-10-05

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

NCT05899673 / Enrolling by invitation临床3期

A Phase 3, Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Fazirsiran in Participants With Alpha-1 Antitrypsin Deficiency-Associated Liver Disease

The main aim of this study is to learn if fazirsiran is safe during long-term use in people with liver disease caused by the abnormal Z-alpha-1 antitrypsin (Z-AAT) protein. People who are currently taking part in or have completed previous fazirsiran studies (AROAAT2001 [NCT03945292] or AROAAT2002 [NCT03946449]) can continue to receive fazirsiran in this study. Participants will receive fazirsiran every 3 months for almost 2 years and will then be followed for an additional 6 months. The study may also provide information on whether fazirsiran has a long-term effect in reducing liver fibrosis or slowing down the progression of liver fibrosis in people with liver disease due to the abnormal Z-AAT protein.

开始日期2023-08-08

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

100 项与 Fazirsiran 相关的临床结果

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100 项与 Fazirsiran 相关的转化医学

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100 项与 Fazirsiran 相关的专利（医药）

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项与 Fazirsiran 相关的文献（医药）

2022-09-01·Nature reviews. Gastroenterology & hepatology

Fazirsiran shows promise for liver disease in AAT

Article

作者: Ray, Katrina

2022-08-11·The New England journal of medicine1区 · 医学

Fazirsiran for Liver Disease Associated with Alpha ₁ -Antitrypsin Deficiency

1区 · 医学

Article

作者: Griffiths, William ; Given, Bruce D. ; Trautwein, Christian ; Choudhury, Gourab ; San Martin, Javier ; Hamilton, James C. ; Teckman, Jeffery H. ; Mandorfer, Mattias ; Yi, Min ; Chang, Ting ; Strnad, Pavel ; Loomba, Rohit ; Schluep, Thomas

BACKGROUND:

Alpha₁-antitrypsin (AAT) deficiency results from carriage of a homozygous SERPINA1 "Z" mutation (proteinase inhibitor [PI] ZZ). The Z allele produces a mutant AAT protein called Z-AAT, which accumulates in hepatocytes and can lead to progressive liver disease and fibrosis. This open-label, phase 2 trial investigated the safety and efficacy of fazirsiran, an RNA interference therapeutic, in patients with liver disease associated with AAT deficiency.

METHODS:

We assigned adults with the PI ZZ genotype and liver fibrosis to receive fazirsiran at a dose of 200 mg (cohorts 1 [4 patients] and 2 [8 patients]) or 100 mg (cohort 1b [4 patients]) subcutaneously on day 1 and week 4 and then every 12 weeks. The primary end point was the change from baseline to week 24 (cohorts 1 and 1b) or week 48 (cohort 2) in liver Z-AAT concentrations, which were measured by means of liquid chromatography-mass spectrometry.

RESULTS:

All the patients had reduced accumulation of Z-AAT in the liver (median reduction, 83% at week 24 or 48). The nadir in serum was a reduction of approximately 90%, and treatment was also associated with a reduction in histologic globule burden (from a mean score of 7.4 [scores range from 0 to 9, with higher scores indicating a greater globule burden] at baseline to 2.3 at week 24 or 48). All cohorts had reductions in liver enzyme concentrations. Fibrosis regression was observed in 7 of 15 patients and fibrosis progression in 2 of 15 patients after 24 or 48 weeks. There were no adverse events leading to trial or drug discontinuation. Four serious adverse events (viral myocarditis, diverticulitis, dyspnea, and vestibular neuronitis) resolved.

CONCLUSIONS:

In this small trial, fazirsiran was associated with a strong reduction of Z-AAT concentrations in the serum and liver and concurrent improvements in liver enzyme concentrations. (Funded by Arrowhead Pharmaceuticals; AROAAT-2002 ClinicalTrials.gov number, NCT03946449.).

2020-06-18·JCI insight1区 · 医学

Development of an RNAi therapeutic for alpha-1-antitrypsin liver disease

1区 · 医学

ArticleOA

作者: Peterson, Ryan M ; Branca-Afrazi, Maria F ; Subbotin, Vladimir M ; Gane, Edward ; Teckman, Jeffrey H ; Schwabe, Christian ; Wooddell, Christine I ; Hegge, Julia O ; Hamilton, James ; Christianson, Dawn R ; Kolbe, John ; Lewis, David L ; Hamilton, Holly L ; Chu, Qili ; Blomenkamp, Keith ; Kanner, Steven B ; Given, Bruce D

The autosomal codominant genetic disorder alpha-1 antitrypsin (AAT) deficiency (AATD) causes pulmonary and liver disease. Individuals homozygous for the mutant Z allele accumulate polymers of Z-AAT protein in hepatocytes, where AAT is primarily produced. This accumulation causes endoplasmic reticulum (ER) stress, oxidative stress, damage to mitochondria, and inflammation, leading to fibrosis, cirrhosis, and hepatocellular carcinoma. The magnitude of AAT reduction and duration of response from first-generation intravenously administered RNA interference (RNAi) therapeutic ARC-AAT and then with next-generation subcutaneously administered ARO-AAT were assessed by measuring AAT protein in serum of the PiZ transgenic mouse model and human volunteers. The impact of Z-AAT reduction by RNAi on liver disease phenotypes was evaluated in PiZ mice by measuring polymeric Z-AAT in the liver; expression of genes associated with fibrosis, autophagy, apoptosis, and redox regulation; inflammation; Z-AAT globule parameters; and tumor formation. Ultrastructure of the ER, mitochondria, and autophagosomes in hepatocytes was evaluated by electron microscopy. In mice, sustained RNAi treatment reduced hepatic Z-AAT polymer, restored ER and mitochondrial health, normalized expression of disease-associated genes, reduced inflammation, and prevented tumor formation. RNAi therapy holds promise for the treatment of patients with AATD-associated liver disease. ARO-AAT is currently in phase II/III clinical trials.

项与 Fazirsiran 相关的新闻（医药）

2024-05-31

·BioSpace

Arrowhead Pharmaceuticals to Participate in Upcoming June 2024 Conferences

PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it is scheduled to participate in the following upcoming events: 16th European Glaucoma Society (EGS) Congress – June 1-4, 2024 Title: Development and Characterization of Trabecular Meshwork Targeted RNAi Molecule Platform via Intracameral Administration for Glaucoma Date/Time: June 2, 2024, 10:30 a.m. IST Session: Poster Section Jefferies Global Healthcare Conference – June 5-6, 2024 Type: Fireside Chat Presentation Date/Time: June 5, 2024, 12:30 p.m. EDT European Association for the Study of the Liver (EASL) Congress 2024 – June 5-8, 2024 Title: Proteomic analysis identified fazirsiran treatment-responsive protein biomarkers in patients with alpha-1 antitrypsin deficiency-associated liver disease Date: June 7, 2024 Type: Poster Title: Correlation of non-invasive tests with histological features and intrahepatic Z-alpha-1 antitrypsin burden in patients with alpha-1 antitrypsin deficiency-associated liver disease Date: June 7, 2024 Type: Poster Goldman Sachs 45th Annual Global Healthcare Conference – June 10-13, 2024 Type: Fireside Chat Presentation Date/Time: June 12, 2024, 4:00 p.m. EDT 22nd Asian Pacific Congress of Nephrology (APCN 2024) – June 13-16, 2024 Title: Safety, Tolerability, and Pharmacodynamics of ARO-C3, a Subcutaneously Administered Investigational RNA-interference Therapeutic Targeting Complement C3 in Adult Healthy Volunteers Date: June 13, 2024 Type: Poster American Diabetes Association (ADA) 84th Scientific Sessions – June 21-24, 2024 Title: Liver-specific silencing of INHBE with ARO-INHBE, an siRNA therapeutic, for metabolic diseases Date: June 24, 2024 Type: Poster 6th Annual RNA Therapeutics Conference | Symposium 2024 From Concept to Clinic – June 26-28, 2024 Title: Rank order TRiMTM activity correlates with RISC loading and can be enhanced by cPrp modification Date/Time: June 28, 2024, 1:00-2:30 p.m. EDT Session: Poster Session II Presentation materials may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website. About Arrowhead Pharmaceuticals Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing. For more information, please visit , or follow us on X (formerly Twitter) at @ArrowheadPharma or on LinkedIn. To be added to the Company's email list and receive news directly, please visit . Safe Harbor Statement under the Private Securities Litigation Reform Act: This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline or product candidates, including anticipated regulatory submissions and clinical program results, prospects or benefits of our collaborations with other companies, or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties, including the impact of the ongoing COVID-19 pandemic on our business, the safety and efficacy of our product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances. Source: Arrowhead Pharmaceuticals, Inc.

siRNA

2024-05-02

·BioSpace

Arrowhead Pharmaceuticals Earns $50 Million Milestone from Royalty Pharma

PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced a $50 million milestone payment was received from Royalty Pharma plc (NASDAQ: RPRX). This milestone was triggered after the completion of enrollment of the Phase 3 OCEAN(a) - Outcomes Trial of olpasiran, being conducted by Amgen (NASDAQ: AMGN). Pursuant to its 2016 agreement with Amgen and 2022 agreement with Royalty Pharma, Arrowhead is further eligible to receive up to an additional $375 million from Amgen and $110 million from Royalty Pharma in aggregate development, regulatory, and sales milestone payments associated with olpasiran. “The rapid enrollment of the OCEAN(a) - Outcomes Trial demonstrates the strong interest in olpasiran, developed using Arrowhead’s proprietary TRiMTM technology and licensed to Amgen in 2016. Partnering is an important part of our strategy and we are pleased with all the care and work undertaken to bring this potentially important new therapy closer to patients,” said Christopher Anzalone, Ph.D., Arrowhead’s president and CEO. “Our pipeline of wholly owned or partnered TRiMTM-enabled candidates now includes three programs in Phase 3 - olpasiran, fazirsiran, and plozasiran. Importantly, our lead wholly owned candidate plozasiran, a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of Apolipoprotein C-III (APOC3), is on schedule to complete its first pivotal Phase 3 study this quarter, with a topline readout soon after.” Olpasiran is a small interfering RNA (siRNA) originally developed by Arrowhead using its proprietary Targeted RNAi Molecule (TRiMTM) platform. It is designed to lower levels of lipoprotein(a) (Lp(a)), a genetically determined risk factor for cardiovascular disease. The primary objective of the Phase 3 OCEAN(a) - Outcomes Trial is to compare the effect of treatment with olpasiran, to placebo, on the risk for coronary heart disease death, myocardial infarction, or urgent coronary revascularization in participants with atherosclerotic cardiovascular disease and elevated lipoprotein(a). About Lp(a) Lp(a) is primarily genetically determined1-3 and a presumed independent risk factor for cardiovascular disease (CVD). Although an agreed upon threshold for elevated Lp(a) is not firmly established, approximately 20% of adults have Lp(a) >125 nmol/L (or approximately 50 mg/dL).1 Evidence has emerged from pathophysiological, epidemiologic, and genetic studies on the potential role of elevated Lp(a) in contributing to myocardial infarction, stroke, and peripheral arterial disease.3 About Arrowhead Pharmaceuticals Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing. For more information, please visit , or follow us on X (formerly Twitter) at @ArrowheadPharma or on LinkedIn. To be added to the Company's email list and receive news directly, please visit . Safe Harbor Statement under the Private Securities Litigation Reform Act: This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline or product candidates, including anticipated regulatory submissions and clinical program results, prospects or benefits of our collaborations with other companies, or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties, including the impact of the ongoing COVID-19 pandemic on our business, the safety and efficacy of our product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances. Source: Arrowhead Pharmaceuticals, Inc. ________________ Wilson DP, et al. Clin Lipidol. 2019;13(3):374-92. Reyes-Soffer G, et al. Arterioscler Thromb Vasc Biol. 2022;42(1):e48-e60. Tsimikas S, et al. J Am Coll Cardiol. 2018;71(2): 177–192. View source version on businesswire.com: Contacts Arrowhead Pharmaceuticals, Inc. Vince Anzalone, CFA 626-304-3400 ir@arrowheadpharma.com Investors: LifeSci Advisors, LLC Brian Ritchie 212-915-2578 britchie@lifesciadvisors.com Media: LifeSci Communications, LLC Kendy Guarinoni, Ph.D. 724-910-9389 kguarinoni@lifescicomms.com Source: Arrowhead Pharmaceuticals, Inc. View this news release online at:

临床3期

2024-03-15

·CPHI制药在线

RNAi先驱Arrowhead：百炼成金

关注并星标CPHI制药在线 20世纪90年代，研究人员在线虫转录过程中确定了双链RNA（dsRNA）是线虫中转录后基因沉默（PTGS）的诱因，他们将这种现象称为RNA干扰（RNAi）。 2002年，RNAi被《科学》杂志评为十大科学成就之首。2006年10月，Andrew Z. Fire和Craig C. Mello两位科学家因为发现RNAi现象，获得诺贝尔生理学或医学奖。由于RNAi在沉默致病基因方面表现出极大的潜力，该技术已成为药企争相布局的方向。经过多年发展，业界涌现了一些RNAi领域的领导者，如Alnylam，公司已有5款RNAi疗法获FDA批准上市。除了耳熟能详的Alnylam，在RNAi领域还有一家低调又不容忽视的布局者：Arrowhead Pharmaceuticals。特有技术平台，突破递送难题 RNAi由双链RNA（dsRNA）分子诱发。起初dsRNA被引入细胞，核酸内切酶将dsRNA切割为长约21-23bp的RNA片段（siRNA，小干扰RNA），随后siRNA与RNA诱导的沉默复合物（RISC）相结合，并解旋成单链，其中正义链被降解，反义链则诱导RISC与互补的mRNA相结合，并将其降解，从而导致基因沉默，靶蛋白无法合成。由于单个RNAi可以多次介导mRNA的沉默作用，因此其在正确的触发条件下，能够实现较高的治疗效率。在RNAi领域，递送问题是决胜关键。目前在RNAi领域常用的递送技术是RNAi领头羊Alnylam公司开发的GalNAc。该技术能将N-乙酰半乳糖胺与siRNA连接，肝细胞表面的受体可识别N-乙酰半乳糖胺，从而高度特异性地进入肝细胞，同时限制脱靶效应。但GalNAc无法靶向肝脏以外的组织，这限制了RNAi疗法的应用，使其只能局限于罕见病与肝脏疾病的治疗。 RNAi疗法要想在肿瘤、常见病领域获得更大的发展空间，就必须在递送系统有所突破。Arrowhead的TRiM平台（Targeted RNAi Molecule）采用配体介导递送，形成结构简单的组织特异性靶向系统。TRiM平台根据需要为每个候选药物优化4个组件：具有序列特异性稳定化学性质的高效RNAi触发器；高亲和力靶向配体；各种连接子和化学物质；增强药代动力学的结构。 TRiM平台突破了Alnylam公司的专利限制，基于该平台，Arrowhead打造了肝病、心血管疾病及靶向肺部、肌肉、神经系统的研发产品。 Arrowhead重要研发产品目前Arrowhead拥有16款在研产品，其中3款已进入临床III期：ARO-APOC3、ARO-ANG3、Fazirsiran。 ARO-APOC3 ARO-APOC3（Plozasiran）是一款针对APOC3基因的RNAi药物，用于治疗高甘油三酯血症（HTG）。ARO-APOC3旨在减少载脂蛋白C-III（apoC III）的产生，载脂蛋白是甘油三酯代谢的关键调节因子，通过RNAi抑制肝脏产生apoC III可导致低密度脂蛋白的合成和组装减少。在两项II期临床研究（SHASTA-2和MUIR）中，ARO-APOC3在降低高甘油三酯血症患者的甘油三酯水平、APOC3水平、及非高密度脂蛋白胆固醇方面的疗效显著，而且未发现严重的不良反应。在另一项II期研究ARCHE-2中，ARO-APOC3在降低患者甘油三酯、低密度脂蛋白胆固醇（LDL-C）等相关指标方面表现出强大效果，而且有助于减少患者的肝脂肪分数。 ARO-ANG3 ARO-ANG3是一款靶向血管生成素样蛋白3（ANGPTL3）的RNAi疗法，被开发用于治疗血脂异常、家族性高胆固醇血症（FH）、HTG。ANGPTL3是一种由肝脏合成的脂蛋白酯酶和内皮酯酶抑制剂，抑制ANGPTL3被证实可降低血清低密度脂蛋白（LDL）、血清和肝脏甘油三酯水平，是心血管疾病的一个新治疗靶点。在IIb期ARCHES-2研究中，中期结果显示，ARO-ANG3能够大幅降低混合性血脂异常患者的甘油三酯水平，同时有效降低包括LDL-C在内的致动脉粥样硬化脂蛋白水平。由于ARO-ANG3具有治疗动脉粥样硬化性心血管疾病的潜在价值，因此具有极大的市场空间。根据投资机构Zacks Investment Research预测，ARO-ANG3未来的销售峰值有望达到45亿美元。 Fazirsiran（ARO-AAT） Fazirsiran是Arrowhead研发的一款用于治疗与α-1抗胰蛋白酶缺乏症(AATD)相关肝病的RNAi疗法。2020年，Arrowhead 和武田达成开发fazirsiran的合作和许可协议。武田将获得fazirsiran除美国外的独家商业化许可，Arrowhead有资格获得净销售额20-25%的分级特许权使用费等。 Fazirsiran旨在沉默突变型α-1抗胰蛋白酶(Z-AAT)蛋白的肝脏生成，减少炎性Z-AAT蛋白的产生，阻止肝病的进展并可能使其再生和修复。AATD是一种罕见遗传病，可导致儿童和成人肝病以及成人肺病，主要表现为血清α1-抗胰蛋白酶(AAT)缺乏。在治疗AATD的II期 SEQUOIA研究中，结果显示，fazirsiran对肝病关键标志物具有良好的影响。在治疗第48周时，接受25、100 或 200 mg fazirsiran 的患者的血清Z-AAT分别降低了74%、89%和94%，而接受安慰剂的患者则增加了9%。目前，Arrowhead和武田正在积极推进 fazirsiran的III期 REDWOOD研究。 Arrowhead成立于1989年，2004年在纳斯达克上市。在数十年的发展过程中，Arrowhead经历了濒临退市、大规模裁员、剥离管线、重组等跌宕起伏的历程。最终凭着对RNAi技术的执着，Arrowhead屹立至今。而且从2019年开始，每年面临着高昂的研发费用，Arrowhead通过卖出药品权益支付"管理+研发"费用，再也没有依靠股权融资。在2023年财报中，Arrowhead提出了"20 in 25"发展战略，即力争在2025年拥有20种合作或独资的药物进入临床试验或上市。Arrowhead的技术平台和坚持初心的气魄，是提出这项战略的硬气与底气。主要参考资料： 1、 https://arrowheadpharma.com/. 2、https://ir.arrowheadpharma.com/news-releases/news-release-details/arrowhead-pharmaceuticals-completes-enrollment-phase-3-palisade. 3、https://ir.arrowheadpharma.com/news-releases/news-release-details/results-phase-2-study-fazirsiran-patients-alpha-1-antitrypsin.【智药研习社近期直播预告】来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com▼更多制药资讯，请关注CPHI制药在线▼点击阅读原文，进入智药研习社~

siRNA临床3期临床2期核酸药物上市批准

100 项与 Fazirsiran 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
肝纤维化	临床3期	美国	Takeda Pharmaceutical Co., Ltd.	2024-03-01
肝纤维化	临床3期	意大利	Takeda Pharmaceutical Co., Ltd.	2024-03-01
肝纤维化	临床3期	波兰	Takeda Pharmaceutical Co., Ltd.	2024-03-01
肝纤维化	临床3期	葡萄牙	Takeda Pharmaceutical Co., Ltd.	2024-03-01
α1-抗胰蛋白酶缺乏症	临床3期	美国	Takeda Pharmaceutical Co., Ltd.	2023-03-06
α1-抗胰蛋白酶缺乏症	临床3期	澳大利亚	Takeda Pharmaceutical Co., Ltd.	2023-03-06
α1-抗胰蛋白酶缺乏症	临床3期	奥地利	Takeda Pharmaceutical Co., Ltd.	2023-03-06
α1-抗胰蛋白酶缺乏症	临床3期	比利时	Takeda Pharmaceutical Co., Ltd.	2023-03-06
α1-抗胰蛋白酶缺乏症	临床3期	巴西	Takeda Pharmaceutical Co., Ltd.	2023-03-06
α1-抗胰蛋白酶缺乏症	临床3期	加拿大	Takeda Pharmaceutical Co., Ltd.	2023-03-06

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适应症

分期

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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


NCT03946449 (Corporate Publications) 人工标引	临床2期	肝纤维化	16	ARO-AAT (100 mg)	鹽範鬱鑰範遞簾淵構鏇(壓餘鏇廠築窪廠觸廠醖) = 製膚襯構壓艱範廠遞齋醖選蓋願選齋廠襯齋顧 (繭夢構鏇積糧獵鬱膚選 ) 更多	积极	2021-06-26
NCT03946449 (Corporate Publications) 人工标引	临床2期	肝纤维化	16	ARO-AAT (200 mg)	鹽範鬱鑰範遞簾淵構鏇(壓餘鏇廠築窪廠觸廠醖) = 壓憲淵範鑰餘餘範淵齋醖選蓋願選齋廠襯齋顧 (繭夢構鏇積糧獵鬱膚選 ) 更多	积极	2021-06-26
NCT03946449 (Phase 2 study, EASL2021)	临床2期	肝纤维化 Z-AAT	9	ARO-AAT 200 mg	鏇製鹽襯餘鏇醖醖顧鑰(構餘觸網壓餘構壓廠膚) = 鹹鹹蓋選廠糧製選醖衊繭醖鏇壓築鏇築繭廠網 (繭鑰築壓淵襯範廠鏇糧 ) 更多	-	2021-06-23
NCT03362242 (AROAAT1001, Corporate Publications) 人工标引	临床1期	肝病	40	ARO-AAT	壓構鹹鏇衊齋襯遞蓋繭(願鬱築鑰襯醖鹽鹽積窪) = 2 cases of injection site erythema at 100 mg after 1st dose 顧築選鏇餘願夢製壓鹽 (餘構積憲鬱鹽構餘鹽齋 )	积极	2018-06-29