Avidity Biosciences宣布完成HARBOR™的入组，这是Delpacibart Etedesiran（del-desiran）用于治疗DM1的全球首个三期临床试验，并提供关于监管提交的指导-动脉网

Avidity Biosciences Announces Completion of Enrollment for HARBOR™, the First Global Phase 3 Trial of Delpacibart Etedesiran (del-desiran) for Treatment of DM1 and Provides Guidance on Regulatory Submission

CISION 等信源发布 2025-07-28 21:00

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可切换为仅中文

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-- Topline data readout from HARBOR study anticipated in Q2 2026 --

-- 预计 2026 年第二季度从 HARBOR 研究中获得顶线数据读数 --

-- Marketing application submissions for del-desiran including in U.S., EU and

-- 包括在美国、欧盟的del-desiran营销应用提交

Japan

日本

anticipated to start in H2 2026; on track to potentially be the first globally approved drug for DM1--

预计将在2026年下半年开始；有望成为全球首个获批的DM1药物——

-- On track to share updates from ongoing MARINA-OLE™ trial of del-desiran including long-term 4 mg/kg efficacy and safety data in Q4 2025 --

-- 有望在2025年第四季度分享MARINA-OLE™试验中del-desiran的最新进展，包括长期4 mg/kg的疗效和安全性数据 --

SAN DIEGO

圣地亚哥

，

July 28, 2025

2025年7月28日

/PRNewswire/ --

Avidity Biosciences, Inc.

Avidity 生物科学公司

(Nasdaq:

（纳斯达克：

RNA

), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced the completion of enrollment in the ongoing global Phase 3 HARBOR™ clinical trial of delpacibart etedesiran (del-desiran) for people living with myotonic dystrophy type 1 (DM1).

)，一家致力于提供一类新型RNA疗法（称为抗体寡核苷酸偶联物（AOCs™））的生物制药公司，今天宣布已完成正在进行的全球3期HARBOR™临床试验的入组工作，该试验涉及delpacibart etedesiran（del-desiran），用于治疗1型肌强直性营养不良（DM1）患者。

Topline data from HARBOR, the first global Phase 3 clinical trial in DM1, are anticipated in the second quarter of 2026..

HARBOR 是首个针对 DM1 的全球 III 期临床试验，其初步数据预计将在 2026 年第二季度公布。

Prior to initiation of the HARBOR trial, Avidity aligned with global regulators, including FDA, on the registrational path for del-desiran. The Company plans to submit marketing applications beginning in the second half of 2026 including in

在HARBOR试验启动之前，Avidity与包括FDA在内的全球监管机构就del-desiran的注册路径达成了一致。公司计划从2026年下半年开始提交上市申请，包括在

the United States

美国

, European Union and

，欧盟和

Japan

日本

。

'Completing enrollment in our Phase 3 HARBOR study for del-desiran marks a significant step toward bringing the first potentially approved drug for DM1 to people around the world who are living with this devastating rare neuromuscular disease,' said

“完成我们在HARBOR三期临床试验中的受试者入组，标志着我们朝着为全球患有这种毁灭性罕见神经肌肉疾病的DM1患者带来首个潜在获批药物迈出了重要一步，”

Steve Hughes

史蒂夫·休斯

, M.D., chief medical officer at Avidity. 'The growing body of data from clinical studies to date support the promise of del-desiran, including durable improvements in multiple clinical endpoints compared to natural history data and evidence that del-desiran is addressing the underlying genetic cause of disease progression.

Avidity首席医学官M.D.表示：“迄今为止，临床研究中越来越多的数据支持了del-desiran的潜力，包括在多个临床终点上相较于自然病史数据的持久改善，以及del-desiran正在解决疾病进展的根本遗传原因的证据。”

These encouraging results suggest that this potential therapy may be transformational for patients who urgently need treatment options. We extend our deepest appreciation to the DM1 community for their ongoing support of our research and development program, especially the patients, families, global advocacy groups, healthcare providers and staff who are part of the HARBOR and MARINA-OLE™ studies.'.

这些令人鼓舞的结果表明，这种潜在的疗法可能对迫切需要治疗选择的患者具有变革性意义。我们向DM1社区持续支持我们的研发计划表示最深切的感谢，特别是参与HARBOR和MARINA-OLE™研究的患者、家属、全球倡导团体、医疗保健提供者和工作人员。

Del-desiran is an investigational treatment designed to address the underlying genetic cause of DM1, a rare, hereditary, progressive neuromuscular disease that shortens life expectancy and requires life-long care. DM1 is characterized by multisystemic manifestations including myotonia and progressive muscle weakening and may be underrecognized because it presents heterogeneously across skeletal, cardiac, and smooth muscles, leading to impairment of the cardiovascular, gastrointestinal, respiratory, ocular, and/or endocrine systems.

德尔德西兰是一种研究性治疗方法，旨在解决导致DM1的潜在遗传原因。DM1是一种罕见的、遗传性的、进展性的神经肌肉疾病，会缩短预期寿命并需要终身护理。DM1的特点是多系统表现，包括肌强直和进行性肌肉无力，可能由于其在骨骼肌、心肌和平滑肌中表现异质性而被低估，从而导致心血管、胃肠、呼吸、眼部和/或内分泌系统的损害。

Currently, there are no approved drugs for people living with DM1..

目前，尚无批准用于治疗DM1患者的药物。

Del-desiran has received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA). Del-desiran was also the first investigational treatment for DM1 to receive Orphan Drug designation in .

德尔德西兰已获得美国食品和药物管理局（FDA）授予的突破性疗法、孤儿药和快速通道资格，并获得了欧洲药品管理局（EMA）授予的孤儿药资格。德尔德西兰也是首个获得孤儿药资格的DM1研究性治疗方法。

Japan

日本

。

About the Phase 3 HARBOR™ Trial

关于第三阶段HARBOR™试验

The global Phase 3 HARBOR™ trial is a randomized, placebo-controlled, double blind pivotal study designed to evaluate del-desiran in approximately 150 people (age 16 and older) living with DM1. The trial is being conducted at approximately 40 sites globally. Patients are administered either del-desiran or placebo (1:1) every eight weeks.

全球三期HARBOR™试验是一项随机、安慰剂对照、双盲关键研究，旨在评估约150名16岁及以上患有DM1的患者使用del-desiran的效果。该试验在全球大约40个地点进行。患者每八周接受一次del-desiran或安慰剂（1:1）治疗。

HARBOR is designed to assess multiple key functional aspects of DM1. The primary endpoint is video hand opening time (vHOT), a measurement of myotonia, which is a hallmark symptom of DM1. Key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ.

HARBOR 旨在评估 DM1 的多个关键功能方面。主要终点是视频手张开时间 (vHOT)，这是肌强直的测量值，而肌强直是 DM1 的标志性症状。关键的次要终点包括通过握力和定量肌肉测试 (QMT) 总分测量的肌肉力量，以及通过 DM1-Activ 测量的日常生活活动能力。

All study participants, regardless of whether they receive active treatment or placebo, have the option to enroll into an open-label extension trial that is expected to begin in Q3 2025. For more information about the HARBOR trial, visit the .

所有研究参与者，不论他们接受的是活性治疗还是安慰剂，都有选择加入预计在2025年第三季度开始的开放标签扩展试验。欲了解有关HARBOR试验的更多信息，请访问。

HARBOR study website

HARBOR研究网站

or visit

或访问

http://www.clinicaltrials.gov

and search for NCT06411288.

搜索NCT06411288。

About Myotonic Dystrophy Type 1

关于一型肌强直性营养不良

Myotonic dystrophy type 1 (DM1) is a rare, hereditary (autosomal dominant), progressive neuromuscular disease that shortens life expectancy and requires life-long care caused by a triplet-repeat in the DMPK gene, resulting in a toxic gain of function mRNA. The disease is highly variable with respect to severity, presentation and age of onset, however, all forms of DM1 are associated with high levels of disease burden.

1型肌强直性营养不良（DM1）是一种罕见的、遗传性的（常染色体显性）、进行性神经肌肉疾病，因DMPK基因中的三核苷酸重复序列导致毒性功能获得性mRNA，从而缩短预期寿命并需要终身护理。该病在严重程度、表现形式和发病年龄上具有高度变异性，但所有类型的DM1均与高水平的疾病负担相关。

DM1 is characterized by multisystemic manifestations including myotonia and progressive muscle weakening and may be underrecognized because it presents heterogeneously across skeletal, cardiac, and smooth muscles, leading to impairment of the cardiovascular, gastrointestinal, respiratory, ocular, and/or endocrine systems.

DM1 的特点是多系统表现，包括肌强直和进行性肌肉无力，可能由于其在骨骼肌、心肌和平滑肌上的异质性表现而被低估，导致心血管、胃肠道、呼吸系统、眼部和/或内分泌系统的功能障碍。

Currently, there are no approved drugs for people living with DM1..

目前，还没有针对DM1患者的获批药物。

About del-desiran

关于 del-desiran

Del-desiran, utilizing Avidity's AOC platform technology, is designed to address the genetic cause of DM1 by reducing levels of toxic DMPK mRNA. Del-desiran consists of a proprietary monoclonal antibody that binds to transferrin receptor 1 (TfR1) and is conjugated to a siRNA that targets DMPK mRNA. Del-desiran is currently being assessed in the global .

德尔德西兰利用Avidity的AOC平台技术，旨在通过减少有毒DMPK mRNA的水平来解决DM1的遗传原因。德尔德西兰由一种专有的单克隆抗体组成，该抗体结合转铁蛋白受体1（TfR1），并与靶向DMPK mRNA的siRNA结合。德尔德西兰目前正在全球范围内进行评估。

Phase 3 HARBOR™ trial

第三阶段HARBOR™试验

and in the ongoing MARINA-OLE™ trial in people with DM1. Long-term data from the MARINA-OLE trial showed reversal of disease progression in people living with DM1 across multiple endpoints including video hand opening time (vHOT) as a measure of hand function and myotonia, muscle strength and activities of daily living when compared to END-DM1 natural history data.

并且在正在进行的针对DM1患者的MARINA-OLE™试验中。MARINA-OLE试验的长期数据显示，与END-DM1自然病史数据相比，跨多个终点观察到DM1患者疾病进展的逆转，这些终点包括作为手功能和肌强直衡量标准的视频手张开时间（vHOT）、肌肉力量以及日常生活活动能力。

德尔德西兰已获得美国食品药品监督管理局（FDA）的突破性疗法、孤儿药和快速通道资格认定，并获得了欧洲药品管理局（EMA）的孤儿药资格认定。德尔德西兰也是首个获得孤儿药资格认定的DM1研究性治疗药物。

Japan

日本

。

About Avidity

关于Avidity

Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies.

Avidity Biosciences, Inc. 的使命是通过提供一类新型的 RNA 治疗药物——抗体寡核苷酸偶联物 (AOCs™)，显著改善人们的生活。Avidity 正在利用其专有的 AOC 技术革新 RNA 领域，该技术旨在将单克隆抗体的特异性与寡核苷酸疗法的精准性相结合，从而针对现有 RNA 疗法无法触及的靶点和疾病。

Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD).

利用其专有的AOC平台，Avidity成功展示了首个将RNA靶向递送到肌肉的案例，并在针对三种罕见肌肉疾病的临床开发项目中处于领先地位：1型肌强直性营养不良（DM1）、杜氏肌营养不良（DMD）和面肩肱型肌营养不良（FSHD）。

Avidity is also advancing two wholly-owned precision cardiology development candidates addressing rare genetic cardiomyopathies. In addition, Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit .

Avidity还在推进两个全资拥有的精准心脏病学开发候选药物，针对罕见的遗传性心肌病。此外，Avidity正在通过关键合作伙伴关系，扩大和推进其在心脏病学和免疫学领域的产品线，从而进一步拓展AOC的应用范围。Avidity总部位于加利福尼亚州圣地亚哥。如需了解更多关于我们的AOC平台、临床开发管线和团队的信息，请访问。

www.aviditybiosciences.com

and engage with us on

并联系我们

领英

and

和

。

Forward-Looking Statements

前瞻性声明

Avidity cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company's current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: the characterization of data associated with del-desiran, and the impact of such data on the advancement of the del-desiran; .

Avidity提醒读者，本新闻稿中关于非历史事实的陈述属于前瞻性声明。这些声明基于公司当前的信念和期望。此类前瞻性声明包括但不限于：与del-desiran相关的数据特征，以及此类数据对del-desiran进展的影响。

the status of the global Phase 3 HARBOR

全球第三阶段HARBOR的状态

™

trial, including but not limited to enrollment, design and goals;

试验，包括但不限于注册、设计和目标；

the regulatory status of a registrational path for del-desiran; Avidity's plans to release topline data from the HARBOR trial and the timing thereof; and Avidity's plans to submit marketing applications for del-desiran and the timing thereof

del-desiran注册路径的监管状态；Avidity公司计划发布HARBOR试验的顶线数据及其时间安排；以及Avidity公司计划提交del-desiran的上市申请及其时间安排。

。

The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of these plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: preliminary results of a clinical trial are not necessarily indicative of final results; further analysis of existing clinical data and analysis of new data may lead to conclusions different from those established as of the respective data cutoff dates in Avidity's clinical study of del-desiran, and such data may not meet Avidity's expectations; unexpected adverse side effects to, or inadequate efficacy of, del-desiran that may delay or limit its development, regulatory approval and/or commercialization; later developments with the FDA and other global regulators that could be inconsistent with the feedback received to date regarding del-desiran and could affect the timing or likelihood of its potential approval; Avidity's approach to the discovery and development of product candidates based on its AOC™ platform is unproven; potential delays in the data readouts and completion of the HARBOR trial; Avidity's dependence on third parties in connection with clinical testing and product manufacturing; legislative, judicial and regulatory developments in .

前瞻性陈述的包含不应被视为Avidity对其中任何计划将实现的承诺。由于Avidity业务中固有的风险和不确定性以及其无法控制的因素，实际结果可能与此新闻稿中的陈述有所不同，这些因素包括但不限于：临床试验的初步结果不一定反映最终结果；对现有临床数据的进一步分析及新数据的分析可能导致与Avidity的del-desiran临床研究中截至各自数据截止日期得出的结论不同，并且这些数据可能未达到Avidity的预期；del-desiran可能出现意外的不良副作用或疗效不足，从而可能延迟或限制其开发、监管批准和/或商业化；FDA及其他全球监管机构的后续发展可能与迄今为止收到的关于del-desiran的反馈不一致，可能影响其潜在批准的时间或可能性；Avidity基于其AOC™平台进行产品候选物发现和开发的方法尚未得到验证；HARBOR试验的数据读出和完成可能存在潜在延迟；Avidity在临床测试和产品制造方面对第三方的依赖；立法、司法和监管的发展等。

the United States

美国

and foreign countries; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended

以及外国国家；以及Avidity年度报告中描述的其他风险，该报告为截至某财政年度末的Form 10-K。

December 31, 2024

2024年12月31日

and subsequent filings with the SEC. Avidity cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date hereof.

并向美国证券交易委员会提交了后续文件。Avidity提醒读者不要过分依赖这些前瞻性声明，这些声明仅截至本日期有效，公司没有义务更新这些声明以反映本日期之后发生的事件或出现的情况。

All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995..

所有前瞻性声明均受此警示性声明的全面约束，该警示性声明是在1995年《私人证券诉讼改革法案》的安全港条款下作出的。

Investor Contact:

投资者联系方式：

Kat Lange

凯特·兰格

(619) 837-5014

investors@aviditybio.com

投资者@aviditybio.com

Media Contact:

媒体联系人：

Kristina Coppola

克里斯蒂娜·科波拉