Avidity initiating global Phase 3 HARBOR™ study for delpacibart etedesiran this quarter

Avidity本季度为delpacibart etedesiran启动全球第三阶段HARBOR™研究

Delpacibart etedesiran data from MARINA-OLE™ showed reversal of disease progression in multiple functional measures in DM1 compared to END-DM1 natural history data

来自MARINA-OLE™的Delpacibart-Etedesrian数据显示，与END-DM1自然史数据相比，DM1中多种功能指标的疾病进展逆转

SAN DIEGO, May 8, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company's lead clinical development program, for the treatment of myotonic dystrophy type 1 (DM1).

圣地亚哥，2024年5月8日/PRNewswire/--Avidity Biosciences，Inc.（纳斯达克：RNA），一家致力于提供一类称为抗体寡核苷酸缀合物（AOCs™）的新型RNA治疗剂的生物制药公司，今天宣布，美国食品和药物管理局（FDA）已授予该公司领先的临床开发计划delpacibart Etedesrian（AOC 1001）突破性治疗指定，用于治疗强直性营养不良1型（DM1）。

Delpacibart etedesiran, abbreviated as del-desiran, is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies..

Delpacibart etedesiran，缩写为del desiran，是一种研究性治疗方法，旨在解决DM1的根本原因，DM1是一种未被认可的，进行性的，通常致命的神经肌肉疾病，没有批准的治疗方法。。

'We are pleased that the FDA has granted Breakthrough Therapy designation to del-desiran for myotonic dystrophy type 1, underscoring the potential of del-desiran to be an effective treatment and the urgency of bringing this treatment to people living with DM1,' said Sarah Boyce, president and chief executive officer at Avidity.

Avidity总裁兼首席执行官莎拉·博伊斯（SarahBoyce）说：“我们很高兴美国食品和药物管理局（FDA）授予德尔·迪西兰（del desiran）突破性治疗1型强直性肌营养不良的称号，强调了德尔·迪西兰（del desiran）作为一种有效治疗方法的潜力，以及将这种治疗方法带给DM1患者的紧迫性。”。

'Initiation is underway for our global Phase 3 HARBOR™ study as we focus on rapidly advancing del-desiran for people living with DM1, who currently have no treatment options to address the underlying cause of this devastating rare muscle disease.'.

“我们的全球3期HARBOR™研究正在启动中，因为我们专注于为DM1患者快速推进del desiran，目前他们没有治疗选择来解决这种毁灭性罕见肌肉疾病的根本原因。”。

Avidity is initiating the global pivotal HARBOR™ study of del-desiran this quarter. The primary endpoint in the Phase 3 HARBOR trial is video hand opening time (vHOT) and 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.

Avidity将于本季度启动del desiran的global pivotal HARBOR™研究。第三阶段HARBOR试验的主要终点是视频开门时间（vHOT），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。

Avidity recently reported positive long-term MARINA-OLE™ data demonstrating reversal of disease progression in adults living with DM1 across multiple endpoints including vHOT, muscle strength and DM1-Activ when compared to natural history data..

Avidity最近报道了积极的长期MARINA-OLE™数据，表明与自然史数据相比，患有DM1的成年人在多个终点（包括vHOT，肌肉力量和DM1活动）的疾病进展逆转。。

In addition to receiving FDA Breakthrough Therapy designation, del-desiran has previously been granted Orphan Drug and Fast Track designations by the FDA and Orphan designation by the European Medicines Agency (EMA) for the treatment of DM1.

除了获得FDA突破性治疗指定之外，del desiran之前还被FDA授予孤儿药和快速通道指定，并被欧洲药品管理局（EMA）授予孤儿指定用于治疗DM1。

About the Phase 2 MARINA-OLE™ Study

关于第二阶段MARINA-OLE™研究

MARINA-OLE™ is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of del-desiran (AOC 1001) in participants with DM1 who were previously enrolled in the MARINA® Phase 1/2 trial. This trial will continue to evaluate the safety, tolerability, PK, PD, and efficacy of del-desiran in participants enrolled in the randomized, placebo-controlled, Phase 1/2 MARINA clinical trial.

MARINA-OLE™是一项开放标签的多中心试验，旨在评估先前参加MARINA®1/2期试验的DM1参与者的del desiran（AOC 1001）的长期安全性和耐受性。该试验将继续评估del desiran在随机，安慰剂对照，1/2期MARINA临床试验参与者中的安全性，耐受性，PK，PD和疗效。

Participants enrolled in the MARINA-OLE study receive quarterly doses of del-desiran regardless of whether they received active treatment or placebo in the MARINA study. The total duration of active treatment with del-desiran in the MARINA-OLE study is approximately 24 months. Once patients have completed active treatment, there will be a nine-month safety follow-up period.

参加MARINA-OLE研究的参与者每季度接受一次del desrian剂量，无论他们在MARINA研究中是否接受了积极治疗或安慰剂。在MARINA-OLE研究中，del desiran积极治疗的总持续时间约为24个月。一旦患者完成积极治疗，将有9个月的安全随访期。

Avidity may extend active treatment beyond 24 months at a future timepoint. For more information on this study click here or visit http://www.clinicaltrials.gov and search for NCT05479981..

在未来的时间点，亲和力可能会将积极治疗延长到24个月以上。有关这项研究的更多信息，请单击此处或访问http://www.clinicaltrials.gov并搜索NCT05479981。。

About Del-desiran (AOC 1001)

关于Del desiran（AOC 1001）

Del-desiran (AOC 1001), Avidity's lead product candidate utilizing its AOC platform, is designed to address the root cause of DM1 by reducing levels of a disease-related mRNA called DMPK. Del-desiran consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA.

Del desiran（AOC 1001）是Avidity利用其AOC平台的主要候选产品，旨在通过降低称为DMPK的疾病相关mRNA的水平来解决DM1的根本原因。Del desiran由专有的单克隆抗体组成，该抗体与转铁蛋白受体1（TfR1）结合，并与靶向DMPK mRNA的siRNA结合。

In preclinical studies, del-desiran successfully delivered siRNAs to muscle cells, resulting in durable, dose-dependent reductions of DMPK RNA across a broad range of muscles including skeletal, cardiac, and smooth muscles. Del-desiran is currently in Phase 1/2 development with the completed MARINA® trial and the ongoing MARINA-OLE™ trial in adults with DM1.

在临床前研究中，del desiran成功地将siRNA递送至肌肉细胞，从而在包括骨骼肌，心肌和平滑肌在内的广泛肌肉中持久，剂量依赖性地减少DMPK RNA。Del desiran目前正处于1/2期开发阶段，已完成MARINA®试验和正在进行的MARINA-OLE™成人DM1试验。

The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted Orphan Designation for del-desiran and the FDA has granted del-desiran Fast Track Designation..

美国食品和药物管理局（FDA）和欧洲药品管理局（EMA）已授予del desiran孤儿称号，FDA已授予del desiran快速通道称号。。

About Myotonic Dystrophy Type 1

关于1型强直性营养不良

Myotonic dystrophy type 1 (DM1) is an underrecognized, progressive and often fatal disease 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 and may cause premature mortality.

1型肌强直性营养不良（DM1）是一种未被充分认识的进行性致命疾病，由DMPK基因的三联体重复序列引起，导致功能性mRNA的毒性增加。该疾病的严重程度，表现和发病年龄差异很大，但所有形式的DM1都与高水平的疾病负担有关，并可能导致过早死亡。

DM1 primarily affects skeletal and cardiac muscle, however patients can suffer from a constellation of manifestations including myotonia and muscle weakness, respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioral impairment.

DM1主要影响骨骼肌和心肌，但是患者可能会出现一系列表现，包括肌强直和肌肉无力，呼吸系统问题，疲劳，嗜睡，心脏异常，严重的胃肠道并发症以及认知和行为障碍。

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

目前，尚无针对DM1患者的批准治疗方法。。

About Avidity

关于亲合力

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的RNA治疗剂-抗体寡核苷酸偶联物（AOCs™），深刻改善人们的生活。Avidity凭借其专有的AOC正在彻底改变RNA领域，AOC旨在将单克隆抗体的特异性与寡核苷酸疗法的精确度相结合，以解决以前现有RNA疗法无法达到的靶标和疾病。

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).

Avidity利用其专有的AOC平台，首次成功地将RNA靶向递送到肌肉中，并在三种罕见肌肉疾病的临床开发计划领域处于领先地位：1型强直性肌营养不良症（DM1），杜兴氏肌营养不良症（DMD）和面肩肱型肌营养不良症（FSHD）。

Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through internal discovery efforts and key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit www.aviditybiosciences.com and engage with us on LinkedIn and X..

Avidity通过其不断推进和扩大的渠道，包括通过内部发现工作和关键合作伙伴关系的心脏病学和免疫学项目，扩大了AOC的覆盖面。Avidity总部位于加利福尼亚州圣地亚哥。有关我们AOC平台、临床开发渠道和人员的更多信息，请访问www.aviditybiosciences.com，并通过LinkedIn和X与我们联系。。

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 implications of Breakthrough Therapy designation for the advancement of del-desiran; the global pivotal Phase 3 HARBOR™ trial for del-desiran, the timing of its initiation and key endpoints to be used therein; the MARINA-OLE™ study, its design, goals, dosage amounts and timelines; the potential of Avidity's product candidates, including del-desiran, to treat rare diseases and Avidity's efforts to bring them to people suffering from applicable diseases; the potential of AOCs to target a range of different cells and tissues beyond the liver, and to treat cardiac and immunological diseases; and Avidity's plans to expand its AOC platform and to invest in its pipeline programs.

Avidity提醒读者，本新闻稿中关于非历史事实的声明是前瞻性声明。这些陈述基于公司目前的信念和期望。此类前瞻性声明包括但不限于以下声明：突破性治疗指定对del desiran进步的影响；del desiran的全球关键3期HARBOR™试验，启动时间和其中使用的关键终点；MARINA-OLE™研究，其设计，目标，剂量和时间表；Avidity的候选产品（包括del desiran）治疗罕见疾病的潜力，以及Avidity努力将其带给患有适用疾病的人；AOC靶向肝脏以外的一系列不同细胞和组织，并治疗心脏和免疫疾病的潜力；以及Avidity计划扩展AOC平台并投资其管道项目。

This press release also contains estimates and other statistical data made by independent parties and by us. This data involves a number of assumptions and limitations, and the reader is cautioned not to give undue weight to such estimates..

本新闻稿还包含独立方和我们所做的估计和其他统计数据。这些数据涉及一些假设和限制，请读者不要过分重视这些估计。。

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, including, without limitation: Avidity may not be able to resolve the partial clinical hold related to the serious adverse event which occurred in the Phase 1/2 MARINA trial; additional participant data related to del-desiran that continues to become available may be inconsistent with the data produced as of the date hereof, and further analysis of existing data and analysis of new data may lead to conclusions different from those established as of the date hereof; unexpected adverse side effects to, or inadequate efficacy of, Avidity's product candidates that may delay or limit their development, regulatory approval and/or commercialization, or may result in additional clinical holds which may not be timely lifted, recalls or product liability claims; Avidity is early in its development efforts; Avidity's approach to the discovery and development of product candidates based on its AOC platform is unproven, and the company does not know whether it will be able to develop any products of commercial value; potential delays in the commencement, enrollment, data readouts and completion of preclinical studies or clinical trials; Avidity's dependence on third parties in connection with preclinical and clinical testing and product manufacturing; regulatory developments in the United States and foreign countries; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, filed with the Securities and Exchange Commission (SEC) on February 28, 2024, and in s.

纳入前瞻性声明不应被视为Avidity表示将实现任何这些计划。由于Avidity业务固有的风险和不确定性，实际结果可能与本新闻稿中规定的结果不同，包括但不限于：Avidity可能无法解决与1/2期MARINA试验中发生的严重不良事件相关的部分临床暂停；继续可用的与del desiran相关的其他参与者数据可能与截至本协议日期产生的数据不一致，对现有数据的进一步分析和对新数据的分析可能会得出与截至本协议日期确定的结论不同的结论；Avidity候选产品的意外不良副作用或功效不足，可能会延迟或限制其开发，监管批准和/或商业化，或可能导致额外的临床扣留，可能无法及时解除，召回或产品责任索赔；Avidity是其开发工作的早期阶段；Avidity基于其AOC平台发现和开发候选产品的方法尚未得到证实，该公司不知道是否能够开发任何具有商业价值的产品；临床前研究或临床试验的开始，登记，数据读取和完成的潜在延迟；Avidity在临床前和临床试验以及产品制造方面对第三方的依赖；美国和外国的监管发展；以及Avidity于2024年2月28日向美国证券交易委员会（SEC）提交的截至2023年12月31日的财政年度10-K表年度报告和s。

Investor Contact:

投资者联系人：

Geoffrey Grande, CFA

杰弗里·格兰德，CFA

(619) 837-5014

(619) 837-5014

investors@aviditybio.com

investors@aviditybio.com

Media Contact:

媒体联系人：

Navjot Rai

纳夫乔特·雷

(619) 837-5016

(619) 837-5016

media@aviditybio.com

media@aviditybio.com

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SOURCE Avidity Biosciences, Inc.

来源Avidity Biosciences，Inc。

Company Codes: NASDAQ-NMS:RNA

公司代码：NASDAQ-NMS：RNA