Avidity plans to accelerate the initiation of registrational cohorts in FORTITUDE™ trialDelpacibart braxlosiran (AOC 1020), the first investigational therapy to target the underlying cause of FSHD, provided greater than 50% mean reduction across multiple DUX4 regulated gene panels (2 mg/kg at four months)Trends of functional improvement including muscle strength, reachable workspace, and positive trends in patient and clinician reported outcomes demonstrated in people treated with delpacibart braxlosiran 2 mg/kg at four monthsDelpacibart braxlosiran data demonstrate favorable safety and tolerability with all adverse events mild or moderate Volume 9 of virtual investor and analyst series today, Wednesday, June 12 at 8:00 a.m.

Avidity计划加速FORTITUDE™trialDelpacibart-braxlosiran（AOC 1020）注册队列的启动，这是第一种针对FSHD根本原因的研究性治疗方法，在多个DUX4调控的基因组（4个月时为2 mg/kg）中提供了超过50%的平均减少率。功能改善的趋势包括肌肉力量，可到达的工作空间，以及患者和临床医生报告的积极趋势。在4个月时用delpacibart-braxlosiran 2 mg/kg治疗的患者的结果显示，delpacibart-braxlosiran数据显示出良好的安全性和耐受性，所有不良事件轻度或中度的虚拟投资者和分析师系列第9卷今天，星期三6月12日上午8:00。

ETSAN DIEGO, June 12, 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 positive initial AOC 1020 data from the Phase 1/2 FORTITUDE™ trial demonstrating unprecedented and consistent reductions of greater than 50% in DUX4 regulated genes, trends of functional improvement, and favorable safety and tolerability in people living with facioscapulohumeral muscular dystrophy (FSHD).

ETSanDiego，2024年6月12日/PRNewswire/--Avidity Biosciences，Inc.（Nasdaq:RNA），一家致力于提供一类称为抗体寡核苷酸偶联物（AOCs™）的新型RNA治疗剂的生物制药公司，今天宣布了来自1/2期Fortity™试验的阳性初始AOC 1020数据，证明DUX4调节基因前所未有且持续减少50%以上，功能改善趋势以及面肩肱型肌营养不良症（FSHD）患者的良好安全性和耐受性。

Avidity plans to accelerate initiation of registrational cohorts in the FORTITUDE™ study. Avidity also announced delpacibart braxlosiran as the approved international nonproprietary name of AOC 1020, abbreviated as del-brax..

Avidity计划在Fortity™研究中加速注册队列的启动。Avidity还宣布delpacibart braxlosiran为AOC 1020（缩写为del brax）批准的国际非专有名称。。

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(PRNewsfoto/Avidity Biosciences, Inc.)

（PRNewsfoto/Avidity Biosciences，Inc.）

Del-brax is the first investigational therapy designed to treat the underlying cause of FSHD, which is caused by the abnormal expression of a gene called double homeobox 4 or DUX4. FSHD is a rare, hereditary disorder marked by life-long, relentless loss of muscle function, significant pain, fatigue, and progressive disability.

Del brax是第一个旨在治疗FSHD根本原因的研究性疗法，FSHD是由称为双同源框4或DUX4的基因异常表达引起的。FSHD是一种罕见的遗传性疾病，其特征是终身持续的肌肉功能丧失，严重的疼痛，疲劳和进行性残疾。

Currently, there are no approved therapies for the treatment of FSHD..

目前，还没有批准的治疗FSHD的疗法。。

'As the first therapy to directly target DUX4, it is very encouraging to see that the del-brax data demonstrate consistent reductions in DUX4 regulated genes and provided trends of functional improvement in patients with FSHD at the four-month timepoint. These early data would support the notion that del-brax has the potential to change the course of disease for people living with FSHD,' said Jeffrey M.

“作为第一种直接针对DUX4的治疗方法，del brax数据显示DUX4调控基因持续减少，并在四个月的时间点为FSHD患者提供了功能改善的趋势，这是非常令人鼓舞的。JeffreyM。

Statland, M.D., Professor of Neurology, University of Kansas Medical Center, and FORTITUDE™ trial investigator. 'Early data showing trends in del-brax to improve muscle strength and function are very encouraging for patients with FSHD who are in need of treatments to prevent the muscle weakness and disability that is associated with this relentlessly, progressive disease.'.

医学博士斯塔特兰（Statland），堪萨斯大学医学中心神经病学教授，FORTITUDE™试验调查员。”早期数据显示，del brax改善肌肉力量和功能的趋势对于需要治疗以预防与这种无情的进行性疾病相关的肌肉无力和残疾的FSHD患者来说是非常令人鼓舞的。”。

The AOC 1020 initial data will be highlighted in an oral presentation at the 31st Annual FSHD Society International Research Congress, being held June 13-14, 2024, in Denver, Colorado.'With the unprecedented del-brax data from the FORTITUDE trial, we are now focused on accelerating our registrational plans as we understand the urgency to develop a treatment for people living with FSHD who have no treatment options,' said Sarah Boyce, president and chief executive officer at Avidity.

AOC 1020的初始数据将在2024年6月13日至14日于科罗拉多州丹佛举行的第31届FSHD协会国际研究年会上的口头报告中予以强调。”Avidity总裁兼首席执行官莎拉·博伊斯（SarahBoyce）说，凭借Fortity试验中前所未有的del brax数据，我们现在专注于加快注册计划，因为我们了解迫切需要为没有治疗选择的FSHD患者开发治疗方法。

'By directly targeting the root cause of FSHD, we believe that del-brax has the potential to be a first-in-class, best-in-class therapy for people living with FSHD. This is our third rare muscle disease program to show delivery to muscle and target engagement and second therapy to provide signs of functional improvement in patients with rare neuromuscular diseases, further reinforcing the promise of our AOC platform to profoundly improve people's lives by revolutionizing the delivery of RNA therapeutics.'The initial assessment from the randomized, double-blind, placebo-controlled Phase 1/2 FORTITUDE trial of del-brax provides a four-month look at the safety and tolerability for all 39 participants across two dose levels (2 mg/kg and 4 mg/kg).

“通过直接针对FSHD的根本原因，我们相信del brax有可能成为FSHD患者的一流，一流的治疗方法。这是我们的第三个罕见肌肉疾病计划，旨在展示向肌肉和靶标的传递，以及第二种治疗方法，为罕见神经肌肉疾病患者提供功能改善的迹象，进一步加强了我们AOC平台的承诺，通过彻底改变RNA治疗方法的传递来深刻改善人们的生活。”del brax的随机，双盲，安慰剂对照的1/2期坚韧性试验的初步评估为所有39名参与者在两个剂量水平（2 mg/kg和4 mg/kg）下的安全性和耐受性提供了四个月的观察。

For the four-month assessment in the 2 mg/kg cohort, participants received a single-dose of 1 mg/kg del-brax followed by two doses of 2 mg/kg del-brax (siRNA dose), or placebo. Data on DUX4 regulated genes, circulating biomarkers and muscle strength and function were assessed from 12 participants in the 2 mg/kg cohort.In the Phase 1/2 FORTITUDE study, del-brax demonstrated:.

对于2 mg/kg队列的四个月评估，参与者接受单剂量1 mg/kg del-brax，然后接受两剂2 mg/kg del-brax（siRNA剂量）或安慰剂。从2 mg/kg队列的12名参与者中评估了DUX4调控基因，循环生物标志物以及肌肉力量和功能的数据。在1/2期坚韧性研究中，德尔布拉克斯证明了：。

Greater than 50% mean reductions in DUX4 regulated genes across multiple panels for DUX4 regulated gene expression in muscle

肌肉中DUX4调控基因表达的多个面板中DUX4调控基因的平均减少率超过50%

All participants treated with del-brax showed reductions greater than 20% in DUX4 regulated genes

所有接受del-brax治疗的参与者的DUX4调控基因减少了20%以上

Mean reductions of 25% or greater in novel circulating biomarker and creatine kinase

新型循环生物标志物和肌酸激酶的平均减少25%或更高

Trends of functional improvements including increased strength in upper and lower limb muscles, and muscle function as measured by reachable workspace (RWS) compared to placebo and the ReSolve natural history study

与安慰剂和ReSolve自然史研究相比，功能改善的趋势包括上肢和下肢肌肉力量的增加，以及通过可达工作空间（RWS）测量的肌肉功能

Trends of improvement in patient and clinician reported outcomes

患者和临床医生报告结果的改善趋势

Favorable safety and tolerability with all adverse events (AEs) mild or moderate, no serious adverse events and no discontinuations

良好的安全性和耐受性，所有不良事件（AE）轻度或中度，无严重不良事件，无停药

'Data from the FORTITUDE trial are very promising for people living with FSHD as the progression of the disease can make it increasingly difficult to perform critical day-to-day activities, pursue work and can affect many aspects of life, including family and social life,' said Mark Stone, president and chief executive officer of FSHD Society.

FSHD Society总裁兼首席执行官马克·斯通（Mark Stone）说：“坚韧试验的数据对FSHD患者来说非常有希望，因为疾病的进展会使他们越来越难以进行关键的日常活动，从事工作，并可能影响生活的许多方面，包括家庭和社会生活。”。

'FSHD is one of the most prevalent forms of muscular dystrophy and currently, there are no treatment options. The initial del-brax data offers real hope for those living with the disease, their families, and their caregivers, who are desperately waiting for a treatment.'  Video Webcast InformationThe company is hosting Volume 9 of its investor and analyst event series on June 12, 2024, beginning at 8:00 a.m.

FSHD是最常见的肌肉萎缩症之一，目前尚无治疗选择。del brax最初的数据为那些迫切等待治疗的患者、他们的家人和照顾者提供了真正的希望。”视频网络广播信息该公司将于2024年6月12日上午8:00开始主办其投资者和分析师系列活动的第9卷。

ET to discuss the initial data from the FORTITUDE™ trial of del-brax in people living with FSHD. The virtual event will be available via a live video webcast and can be accessed here or from the 'Events and Presentations' page in the 'Investors' section of Avidity's website. A replay of the webcast will be archived on Avidity's website following the event.The management team will be joined by Jeffrey M.

ET讨论了来自患有FSHD的人的del brax FORTITUDE™试验的初始数据。虚拟活动将通过实时视频网络广播提供，可以在此处或从Avidity网站“投资者”部分的“活动和演示”页面访问。活动结束后，网络广播的重播将存档在Avidity的网站上。JeffreyM将加入管理团队。

Statland, M.D., Professor of Neurology, University of Kansas Medical Center, and FORTITUDE™ trial investigator. Dr. Statland is one of the principal investigators in the ReSolve study, an ongoing natural history study being led by the FSHD Clinical Trial Research Network (CTRN).About the Phase 1/2 FORTITUDE™ trialThe FORTITUDE™ trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial designed to evaluate single and multiple doses of delpacibart braxlosiran or del-brax (AOC 1020) in approximately 39 adult participants with facioscapulohumeral muscular dystrophy (FSHD).

医学博士Statland，堪萨斯大学医学中心神经病学教授，FORTITUDE™试验研究员。Statland博士是ReSolve研究的主要研究者之一，ReSolve研究是由FSHD临床试验研究网络（CTRN）领导的一项正在进行的自然史研究。关于1/2期FORTITUDE™试验FORTITUDE™试验是一项随机，安慰剂对照，双盲，1/2期临床试验，旨在评估大约39名面肩肱型肌营养不良症（FSHD）成年参与者的单剂量和多剂量delpacibart-braxlosiran或del-brax（AOC 1020）。

FORTITUDE will evaluate the safety,.

坚韧将评估安全性，。