Alnylam在美国心脏病学会2025年科学年会上公布了Vutrisiran在ATTR淀粉样变心肌病（ATTR-CM）患者中的HELIOS三期研究的新数据-动脉网

Alnylam Presents New Data from the HELIOS Phase 3 Study of Vutrisiran in Patients with ATTR Amyloidosis with Cardiomyopathy (ATTR-CM) at the American College of Cardiology’s Annual Scientific Session 2025

阿里拉姆制药等信源发布 2025-03-29 01:01

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可切换为仅中文

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CAMBRIDGE, Mass.

马萨诸塞州剑桥市

--(BUSINESS WIRE)--Mar. 29, 2025--

--（商业资讯）--2025年3月29日--

Alnylam Pharmaceuticals, Inc.

Alnylam制药公司

(Nasdaq: ALNY), the leading RNAi therapeutics company, today announced moderated poster presentations of new data from the landmark HELIOS-B Phase 3 clinical trial, which evaluated vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM) in a population representative of today’s patients.

(Nasdaq: ALNY)，领先的RNAi治疗公司，今天宣布了来自具有里程碑意义的HELIOS-B第三阶段临床试验的新数据的海报展示，该试验评估了vutrisiran用于治疗伴有心肌病的转甲状腺素蛋白淀粉样变性（ATTR-CM）在代表当前患者群体中的应用。

Data were presented at the .

数据在会议上展示。

American College

美国学院

of Cardiology’s Annual Scientific Session (ACC.25) held in

心脏病学年度科学会议（ACC.25）在举行

Chicago, Illinois

伊利诺伊州芝加哥市

. These data build upon the body of clinical evidence which supported the recent

这些数据建立在支持近期临床证据的基础上。

U.S. Food and Drug Administration

美国食品药品监督管理局

(FDA) approval of AMVUTTRA

(FDA) 批准 AMVUTTRA

(vutrisiran) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. AMVUTTRA is an RNAi therapeutic that works upstream to deliver rapid knockdown of transthyretin, addressing the disease at its source, with four subcutaneous doses per year. .

(vutrisiran) 用于治疗成人野生型或遗传性转甲状腺素蛋白介导的淀粉样变心肌病，以降低心血管死亡率、心血管住院率和紧急心力衰竭就诊率。AMVUTTRA 是一种 RNAi 疗法，通过上游作用实现转甲状腺素蛋白的快速敲低，从源头上解决疾病，每年需进行四次皮下注射。

“The HELIOS-B study continues to deliver a robust data package showcasing the unique, differentiated value of vutrisiran as a first-line treatment option that can enable patients with ATTR-CM to live longer, better, and healthier lives,” said

“HELIOS-B研究继续提供强有力的数据包，展示出vutrisiran作为一线治疗选择的独特价值，可以使ATTR-CM患者活得更长、更好、更健康，”

Pushkal Garg

普什卡尔·加格

, M.D., Chief Medical Officer of

，医学博士，首席医疗官

Alnylam

阿尔尼拉姆

. “Data showing beneficial effects on cardiac systolic and diastolic function are novel and indicate the direct impact of vutrisiran in cardiac structure and function of patients living with ATTR-CM. These findings, together with the observed improvements in functional capacity, health status and quality of life, as well as the reductions in all-cause mortality and cardiovascular events, demonstrate the rapid, broad, and sustained impacts of vutrisiran on ATTR-CM and underscore the importance of early intervention for this progressive and ultimately fatal disease.” .

“数据显示对心脏收缩和舒张功能的有益影响是新颖的，表明了vutrisiran对ATTR-CM患者心脏结构和功能的直接影响。这些发现，加上观察到的功能能力、健康状况和生活质量的改善，以及全因死亡率和心血管事件的减少，展示了vutrisiran对ATTR-CM的快速、广泛和持续的影响，并强调了对这种进展性且最终致命的疾病进行早期干预的重要性。”

Moderated Poster Presentations:

适度的海报展示：

The Relationship Between Cardiac Structure, Function, and Clinical Outcomes and the Impact of Vutrisiran from the HELIOS-B Trial

HELIOS-B试验中心脏结构、功能与临床结果之间的关系及Vutrisiran的影响

New echocardiographic data from the HELIOS-B Phase 3 clinical trial, the largest systematic echocardiographic study in a pivotal trial, demonstrated that treatment with vutrisiran improved echocardiographic cardiac function. Vutrisiran treatment led to significant improvements in diastolic function and attenuation of declines in left ventricular (LV) and right ventricular (RV) systolic function at Month 18, compared to placebo.

HELIOS-B 三期临床试验中的新超声心动图数据，作为关键试验中规模最大的系统性超声心动图研究，显示了 vutrisiran 治疗改善了超声心动图上的心脏功能。与安慰剂相比，vutrisiran 治疗在第 18 个月显著改善了舒张功能，并减缓了左心室（LV）和右心室（RV）收缩功能的下降。

Baseline measures of LV and RV systolic function, as well as indices of diastolic function, were shown to provide important prognostic information beyond clinical characteristics and biomarker-based staging systems. Notably, worsening LV and RV systolic function over 18 months was significantly associated with an increased risk of subsequent all-cause death, highlighting the importance of these parameters in assessing disease progression.

左心室和右心室收缩功能的基线测量值，以及舒张功能的指标，被证明提供了超出临床特征和基于生物标志物分期系统的重要的预后信息。值得注意的是，18个月内左心室和右心室收缩功能的恶化与随后全因死亡风险的增加显著相关，突显了这些参数在评估疾病进展中的重要性。

The improved clinical outcomes of patients receiving vutrisiran in HELIOS-B may be mediated by its impact on cardiac function. .

HELIOS-B试验中接受vutrisiran治疗的患者临床结果改善可能是由其对心脏功能的影响所介导的。

Impact of Baseline Heart Failure Severity on Efficacy of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy in the HELIOS-B Trial: A Subgroup Analysis

HELIOS-B试验中基线心力衰竭严重程度对Vutrisiran在转甲状腺素蛋白淀粉样变心肌病患者中疗效的影响：亚组分析

An exploratory subgroup analysis demonstrated that vutrisiran reduced all-cause mortality and recurrent cardiovascular events across a range of baseline heart failure severities in patients with ATTR-CM. The greatest benefit was observed in patients with earlier, less severe disease, underscoring the need for timely diagnosis and early intervention.

一项探索性亚组分析表明，vutrisiran 在 ATTR-CM 患者中，跨越各种基线心力衰竭严重程度的情况下，减少了全因死亡率和心血管事件的复发。最大的获益见于疾病较早期且不那么严重的患者，突显了及时诊断和早期干预的重要性。

Similar effects were seen in the monotherapy population and across additional endpoints, including functional capacity and cardiac biomarkers, reinforcing the efficacy of vutrisiran regardless of disease stage. These results were recently published in .

在单药治疗人群和包括功能能力和心脏生物标志物在内的其他终点中也观察到了类似的效果，这进一步证明了vutrisiran无论在疾病哪个阶段都具有疗效。这些结果最近发表在。

JACC

。

Maintenance or Improvement of Functional Capacity, Health Status, and Quality of Life with Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy: Data from the HELIOS-B Study

维特立森治疗伴有心肌病的转甲状腺素蛋白淀粉样变性患者的功能能力、健康状况和生活质量的维持或改善：来自HELIOS-B研究的数据

A separate analysis confirmed that vutrisiran significantly maintained or improved functional capacity, and patient-reported health status and quality of life, compared to placebo over 30 months. Greater proportions of patients treated with vutrisiran preserved or improved 6-minute walk test distance and Kansas City Cardiomyopathy Questionnaire scores at clinically meaningful thresholds.

一项单独的分析证实，与安慰剂相比，维特利西兰在30个月内显著保持或改善了患者的功能能力、患者报告的健康状况和生活质量。在接受维特利西兰治疗的患者中，有更高比例的患者在临床意义上保持或提高了6分钟步行测试距离和堪萨斯城心肌病问卷评分。

These findings provide further evidence of vutrisiran’s ability to deliver meaningful benefits beyond reducing mortality and cardiovascular events. These results were recently published in .

这些发现进一步证明了vutrisiran除了降低死亡率和心血管事件外，还能够带来有意义的益处。这些结果最近发表在。

JACC

。

An additional Flatboard Poster presentation, “Real-World Persistency on Tafamidis: An Analysis of

此外，Flatboard Poster 展示了题为“Tafamidis 在现实世界中的持久性：一项分析”的报告，

U.S.

美国

Insurance Claims Data” will be presented on

保险索赔数据”将被展示在

Monday, March 31

3月31日，星期一

在

10:30 am (CDT)

上午10点30分（中部夏令时间）

，

11:30 am (EDT)

上午11点30分（东部夏令时间）

。

To view the results presented at ACC.25, please visit

要查看在 ACC.25 上展示的结果，请访问

Capella

北极星

。

About AMVUTTRA

关于AMVUTTRA

(vutrisiran)

（维特利森）

AMVUTTRA

(vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection by a healthcare professional, AMVUTTRA is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and is approved in the .

(vutrisiran) 是一种 RNAi 治疗药物，能够快速降低转甲状腺素蛋白 (TTR)，针对转甲状腺素蛋白淀粉样变性 (ATTR) 的根本病因。AMVUTTRA 通过医疗专业人员每季度进行皮下注射给药，已在 15 多个国家获批并上市，用于治疗成人遗传性转甲状腺素蛋白介导的淀粉样变性多发性神经病 (hATTR-PN)，并且在。

U.S.

美国

for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. For more information about AMVUTTRA, including the full

用于治疗野生型或遗传性转甲状腺素蛋白介导的淀粉样变心肌病（ATTR-CM）成年患者，以减少心血管死亡、心血管住院和紧急心力衰竭就诊。有关AMVUTTRA的更多信息，包括完整信息，请访问相关网站。

U.S.

美国

Prescribing Information, visit

处方信息，请访问

AMVUTTRA.com

。

About ATTR

关于ATTR

Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease.

转甲状腺素蛋白淀粉样变性 (ATTR) 是一种诊断不足、进展迅速、使人衰弱且致命的疾病，由错误折叠的转甲状腺素蛋白 (TTR) 引发，这些蛋白在身体的各个部位（包括神经、心脏和胃肠道）以淀粉样沉积物的形式积累。患者可能表现为多发性神经病、心肌病或两种症状同时出现。

There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide..

ATTR有两种不同的形式——遗传性ATTR（hATTR），由TTR基因变异引起，全球约有50,000人受到影响；以及野生型ATTR（wtATTR），它在没有TTR基因变异的情况下发生，全球估计有200,000至300,000人受到影响。

1-4

About RNAi

关于RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine.

RNAi（RNA干扰）是一种天然的细胞基因沉默过程，代表了当今生物学和药物开发领域最有前途且进展最快的前沿之一。它的发现被誉为“大约每十年才会出现一次的重大科学突破”，并因此获得了2006年诺贝尔生理学或医学奖。

By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made.

通过利用我们细胞中发生的天然生物过程RNAi，一类被称为RNAi治疗药物的新药已经成为现实。小干扰RNA（siRNA）是介导RNAi的分子，也是Alnylam公司RNAi治疗平台的组成部分，它通过有效沉默信使RNA（mRNA）——即编码致病或疾病通路蛋白的遗传前体——从而阻止这些蛋白的生成，在当今药物的上游发挥作用。

This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases. .

这是一项具有革命性的方法，有望改变对遗传病及其他疾病患者的护理方式。

About

关于

Alnylam Pharmaceuticals

阿尔尼拉姆制药公司

Alnylam Pharmaceuticals

Alnylam制药公司

(Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines for people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines.

(Nasdaq: ALNY) 已将RNA干扰（RNAi）技术转化为一类全新的创新药物，为患有罕见和普遍疾病但需求未得到满足的患者带来了希望。基于诺贝尔奖获奖科学，RNAi疗法代表了一种强大且经过临床验证的方法，能够带来变革性的药物。

Since its founding in 2002, .

自 2002 年成立以来，。

Alnylam

阿尔尼拉姆

has led the RNAi revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products include ONPATTRO

引领了RNAi革命，并继续致力于实现将科学可能性变为现实的大胆愿景。Alnylam的商用RNAi治疗产品包括ONPATTRO。

(patisiran), AMVUTTRA

帕蒂西兰，AMVUTTRA

(vutrisiran), GIVLAARI

(vutrisiran)，GIVLAARI

(givosiran), and OXLUMO

（吉沃西兰）和OXLUMO

(lumasiran), which are being developed and commercialized by

（lumasiran），这些药物正在被开发和商业化

Alnylam

阿尔尼拉姆

, and Leqvio

，以及Leqvio

(inclisiran) and Qfitlia

英克司兰和Qfitlia

™

(fitusiran), which are being developed and commercialized by Alnylam’s partners, Novartis and Sanofi, respectively.

（fitusiran），它们分别由Alnylam的合作伙伴诺华和赛诺菲开发和商业化。

Alnylam

阿尔尼拉姆

has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development.

拥有一个深入的在研药物管道，包括多个处于后期开发阶段的产品候选物。

Alnylam

阿尔尼拉姆

is executing on its “

正在执行其“

Alnylam P

阿尼拉姆 P

x25

” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile.

“通过可持续创新和卓越的财务表现，为全球患者提供罕见病和常见病的变革性药物的战略，造就领先的生物技术企业形象。

Alnylam

阿里拉姆

is headquartered in

总部位于

Cambridge, MA.

马萨诸塞州剑桥市。

For more information about our people, science and pipeline, please visit

有关我们的人才、科学和研发管线的更多信息，请访问

www.alnylam.com

and engage with us on X (formerly Twitter) at

并在X（前身为Twitter）上与我们互动

@Alnylam

, or on

，或者在

领英

，

Facebook

脸书

, or

，或者

Instagram

图享

。

Alnylam Forward-Looking Statements

Alnylam前瞻性声明

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, Alnylam’s expectations regarding the safety and efficacy of vutrisiran for the treatment of ATTR-CM, including the ability of vutrisiran to reduce mortality and cardiovascular events in ATTR-CM patients; the potential of vutrisiran to become a first-line therapy for ATTR-CM; the potential of vutrisiran to enable ATTR-CM patients to live longer, better and healthier lives; and the potential for early treatment with vutrisiran to have beneficial effects in ATTR-CM patients should be considered forward-looking statements. .

本新闻稿包含1933年《证券法》第27A条和1934年《证券交易法》第21E条所指的前瞻性陈述。所有关于Alnylam的预期、信念、目标、计划或前景的非历史事实的陈述，包括但不限于：Alnylam对vutrisiran治疗ATTR-CM的安全性和有效性的预期，包括vutrisiran降低ATTR-CM患者死亡率和心血管事件的能力；vutrisiran成为ATTR-CM一线治疗的潜力；vutrisiran使ATTR-CM患者更长寿、更健康生活的可能性；以及早期使用vutrisiran治疗可能对ATTR-CM患者产生有益效果的潜力，均应被视为前瞻性陈述。

Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to Alnylam’s ability to successfully execute on its “.

实际结果和未来计划可能与这些前瞻性声明所表明的内容存在重大差异，原因在于各种重要的风险、不确定性和其他因素，包括但不限于与Alnylam成功执行其“计划相关”的风险和不确定性。

Alnylam P

阿尼拉姆 P

x25

” goals; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam’s 2024 Annual Report on Form 10-K filed with the .

目标；Alnylam发现和开发新型候选药物及递送方法，并成功证明其候选产品的有效性和安全性的能力；Alnylam候选产品的临床前和临床结果；监管机构的行动或建议，以及Alnylam为其候选产品获得并维持监管批准的能力，同时确保有利的定价和报销；在全球范围内成功推出、营销和销售Alnylam的已批准产品；Alnylam候选产品或其上市产品的生产与供应中的延迟、中断或失败；获取、维护和保护知识产权；Alnylam通过在运营上的审慎投资来管理其增长和运营开支的能力，以及未来实现财务自给自足的能力；Alnylam保持战略性业务合作的能力；Alnylam对第三方在某些产品的开发和商业化方面的依赖；诉讼的结果；未来政府调查的潜在风险；以及意外支出；还包括在Alnylam提交的2024年Form 10-K年度报告中“风险因素”部分更全面讨论的那些风险。

Securities and Exchange Commission

证券交易委员会

(SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that

证券交易委员会（SEC），可能会在Alnylam公司随后的10-Q季度报告中不时更新，并在其他文件中有所记载。

Alnylam

阿尔尼拉姆

makes with the

与...一起制作

SEC

证券交易委员会

. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing Alnylam’s views as of any subsequent date.

此外，任何前瞻性声明仅代表Alnylam截至今日的观点，不应被视为代表Alnylam在任何后续日期的观点。

Alnylam

阿尔尼拉姆

explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

明确否认任何义务，除非法律要求，否则不会更新任何前瞻性声明。

Hawkins PN, Ando Y, Dispenzeri A, et al.

霍金斯 PN、安藤 Y、迪斯潘泽里 A 等。

Ann Med

医学年鉴

. 2015;47(8):625-638.

Gertz MA.

格茨 M.A.