Alnylam将在2025年心力衰竭大会上分享其转甲状腺素蛋白淀粉样变性领域的进展，包括HELIOS三期研究结果的更多分析-动脉网

Alnylam to Share Progress Across its Transthyretin Amyloidosis Franchise Including Additional Analyses of the HELIOS Phase 3 Study Results at Heart Failure 2025 Congress

阿里拉姆制药等信源发布 2025-05-12 19:11

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CAMBRIDGE, Mass.

马萨诸塞州剑桥市

--(BUSINESS WIRE)--May 12, 2025--

--（商业资讯）--2025年5月12日--

Alnylam Pharmaceuticals, Inc.

阿尔尼拉姆制药公司

(Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Company will present the latest data from its flagship transthyretin amyloidosis (TTR) franchise at the upcoming Heart Failure 2025

(Nasdaq: ALNY)，领先的RNAi治疗公司，今天宣布公司将在于即将到来的心力衰竭2025会议上展示其旗舰转甲状腺素蛋白淀粉样变性（TTR）专营的最新数据。

Congress

国会

, a scientific congress of the

，一个科学大会的

European Society of Cardiology

欧洲心脏病学会

, taking place

，发生

May 17-20

5月17日至20日

in Belgrade,

在贝尔格莱德，

Serbia

塞尔维亚

。

The latest analyses of the HELIOS-B Phase 3 study of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM), including further outcomes data on cardiovascular hospitalizations and urgent heart failure visits up to 42-months, will be presented as a late-breaking abstract in the “Hottest Trials and Trial Updates 1” session. .

Vutrisiran在伴有心肌病的转甲状腺素蛋白淀粉样变性（ATTR-CM）患者中的HELIOS-B III期研究的最新分析，包括长达42个月的心血管住院和紧急心衰就诊的更多结果数据，将会在“最热门试验及试验更新1”环节中以最新突破性摘要的形式展示。

Data from the HELIOS-B study supported the recent approvals of AMVUTTRA

HELIOS-B 研究的数据支持了 AMVUTTRA 的近期获批。

(vutrisiran) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults in the

(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性的心肌病。

U.S.

美国

and

和

Brazil

巴西

. These data also supported the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the

这些数据也支持了人用药品委员会 (CHMP) 的积极意见。

European Medicines Agency

欧洲药品管理局

(EMA) recommending approval of vutrisiran for the same indication. 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.

欧洲药品管理局（EMA）建议批准vutrisiran用于相同适应症。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，从源头上治疗疾病，每年需进行四次皮下注射。

Additional updates to be presented include the design and rationale of the TRITON-CM Phase 3 study of nucresiran (ALN-TTRsc04), an investigational next-generation TTR silencer, in patients with ATTR-CM, as well as an additional analysis from the HELIOS-B study of vutrisiran in patients with ATTR-CM who experienced disease progression while being treated with tafamidis. .

其他将要介绍的更新包括针对ATTR-CM患者的nucresiran（ALN-TTRsc04）——一种研究中的下一代TTR沉默剂——的TRITON-CM III期研究的设计和基本原理，以及来自HELIOS-B研究的另一项分析，该分析针对的是在使用tafamidis治疗期间出现疾病进展的ATTR-CM患者中的vutrisiran。

Presentation Details

演示详情

Vutrisiran Reduces All-Cause Mortality, Cardiovascular Mortality, and Cardiovascular Events in Patients with Transthyretin Amyloid Cardiomyopathy: Analysis from the HELIOS-B Trial

Vutrisiran降低转甲状腺素蛋白淀粉样变心肌病患者的所有原因死亡率、心血管死亡率和心血管事件：来自HELIOS-B试验的分析

Session: Hottest Trials and Trial Updates 1

会议：最热门的试验和试验更新 1

Saturday, May 17

5月17日，星期六

, 11:50 –

，11:50 –

11:58 CEST

11:58 中欧夏令时间

, 5:50 –

，5:50 –

5:58 A.M. EST

早上5点58分（东部标准时间）

Presenting Author:

报告作者：

Marianna Fontana

玛丽安娜·丰塔纳

，

United Kingdom

英国

Clinical Presentation and Treatment Landscape of Patients with Transthyretin Amyloidosis With Cardiomyopathy: A Real-world Study in Five European Countries and

淀粉样转甲状腺素蛋白心肌病患者的临床表现及治疗前景：欧洲五国真实世界研究和

Japan

日本

Session: Novel Insights into Heart Failure Therapeutics

会议：心力衰竭治疗的新见解

Sunday, May 18

5月18日，星期日

, 13:00 –

，13:00 –

13:45 CEST

13:45 中欧夏令时间

, 7:00 –

，7:00 –

7:45 A.M. EST

早上7点45分（东部标准时间）

Presenter:

主持人：

Caroline Morbach

卡罗琳·莫拉赫

，

Germany

德国

Utility of Genetic Testing For Diagnosing hATTR Patients: Results from a European and Middle East Genetic Testing Program

遗传检测在诊断hATTR患者中的应用：来自欧洲和中东遗传检测项目的结果

Session: Novel Insights into Heart Failure Therapeutics

会议：心力衰竭治疗的新见解

Monday, May 19

5月19日，星期一

, 9:00 –

，9:00 –

10:00 CEST

10:00 中欧夏令时间

, 3:00 –

，3:00 –

4:00 A.M. EST

凌晨4点（东部标准时间）

Presenter:

主持人：

Antoine Bondue

安托万·邦迪

，

Belgium

比利时

Design and Rationale of a Phase 3 Study to Evaluate Efficacy and Safety of Nucresiran (ALN-TTRsc04) in Patients with Transthyretin Amyloidosis with Cardiomyopathy

评估Nucresiran（ALN-TTRsc04）在转甲状腺素蛋白淀粉样变心肌病患者中的疗效和安全性的III期研究设计与原理

Session: Research Methodology

会议：研究方法论

Monday, May 19

5月19日，星期一

, 14:00 –

，14:00 –

15:00 CEST

15:00 中欧夏令时间

, 8:00 –

，8:00 –

9:00 A.M. EST

上午9:00（东部标准时间）

Presenter:

主持人：

Marianna Fontana

玛丽安娜·丰塔纳

，

United Kingdom

英国

Vutrisiran In Patients With Transthyretin Amyloidosis with Cardiomyopathy In HELIOS-B Who Had Progressed On Tafamidis

Vutrisiran 在 HEIOS-B 中用于治疗转甲状腺素蛋白淀粉样变性心肌病患者，这些患者在使用 Tafamidis 后病情已进展。

Session: Evolving Treatment Paradigms in Heart Failure: SGLT2 Inhibition to TTR Stabilisation and Beyond

会议：心力衰竭治疗模式的演变：从SGLT2抑制到TTR稳定化及更多

Tuesday, May 20

5月20日，星期二

, 9:21 –

，9:21 –

9:30 CEST

9:30 中欧夏令时间

, 3:21 –

，3:21 –

3:30 A.M. EST

凌晨3点30分（东部标准时间）

Presenter:

主持人：

Jose Gonzalez-Costello

何塞·冈萨雷斯-科斯特洛

，

Spain

西班牙

About AMVUTTRA

关于AMVUTTRA

(vutrisiran)

（维特利森）

AMVUTTRA

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

(vutrisiran) 是一种 RNAi 治疗药物，能够快速减少变异型和野生型转甲状腺素蛋白 (TTR)，针对转甲状腺素蛋白 (ATTR) 淀粉样变性的根本原因。vutrisiran 通过皮下注射每季度给药一次，已在超过 15 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)。

In .

在。

Europe

欧洲

, it is administered as a subcutaneous injection once every three months, either by a healthcare professional, or self-administered by patients or their caregivers. Vutrisiran is also in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), which encompasses both wild-type and hereditary forms of the disease. .

，它每三个月通过皮下注射给药一次，可以由医疗专业人员进行注射，也可以由患者或其护理人员自行注射。Vutrisiran 也正在开发用于治疗伴有心肌病的转甲状腺素蛋白淀粉样变性 (ATTR-CM)，涵盖该疾病的野生型和遗传型两种形式。

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、致残且致命的疾病，由错误折叠的转甲状腺素蛋白（TTR）引起，这些蛋白在身体的各个部位（包括神经、心脏和胃肠道）积累为淀粉样沉积物。患者可能会表现为多发性神经病、心肌病或这两种疾病的表现。

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）是一种天然的基因沉默细胞过程，代表了当今生物学和药物开发中最有前途且进展最快的前沿领域之一。它的发现被誉为“每隔十年左右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，并构成了Alnylam的RNAi治疗平台，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. .

这是一项具有变革患者遗传病及其他疾病治疗潜力的革命性方法。

About

关于

Alnylam Pharmaceuticals

阿尼兰制药公司

Alnylam

阿尔尼拉姆

(Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of 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，有望改变那些患有罕见和普遍但未满足需求的疾病患者的生活。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，能够产生变革性的药物。

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.

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

阿尔尼拉姆

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

拥有一个包含多个处于后期开发阶段的产品候选药物的深入研究药物管道。

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，造就了领先的生物技术形象。”

Alnylam

阿尔尼拉姆

is headquartered in

总部位于

Cambridge, MA.

马萨诸塞州剑桥市。

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

霍金斯 PN，安藤 Y，迪斯彭泽里 A，等。

Ann Med

医学年鉴

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