REGENXBIO报告了RGX-202基因治疗在AFFINITY DUCHENNE®试验中的积极生物标志物数据-动脉网

REGENXBIO REPORTS POSITIVE BIOMARKER DATA FROM AFFINITY DUCHENNE® TRIAL OF RGX-202 GENE THERAPY

CISION 等信源发布 2025-03-19 21:15

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可切换为仅中文

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Positive biomarker data in patient aged 1-3 add to consistent, robust microdystrophin and transduction levels across all treated ages

1-3岁患者中的阳性生物标志物数据进一步增加了所有治疗年龄段一致且强大的微抗肌萎缩蛋白和转导水平

Patient aged 3 years at dosing had expression level at 122.3% compared to control

患者在给药时年龄为3岁，其表达水平为对照组的122.3%。

With a differentiated novel construct and proactive short course immune modulation regimen, RGX-202 continues to demonstrate encouraging safety profile with no SAEs or AESIs

凭借差异化的新型结构和积极的短期免疫调节方案，RGX-202继续表现出令人鼓舞的安全性，无严重不良事件（SAEs）或特别关注的不良事件（AESIs）。

Phase III portion of AFFINITY DUCHENNE

AFFINITY DUCHENNE 的 III 期部分

trial enrolling ambulatory patients aged 1 and above, on track for BLA submission mid-2026

招募1岁及以上可步行患者的试验，预计将于2026年年中提交BLA。

ROCKVILLE, Md.

罗克维尔，马里兰州

，

March 19, 2025

2025年3月19日

/PRNewswire/ -- REGENXBIO Inc. (Nasdaq:

/PRNewswire/ -- REGENXBIO Inc.（纳斯达克：

RGNX

) today reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE

今天报道了AFFINITY DUCHENNE项目第一/二阶段部分中另外两名患者的最新积极中期数据。

trial of RGX-202, a differentiated investigational gene therapy for Duchenne muscular dystrophy (Duchenne). Results were presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.

RGX-202 的临床试验，这是一种用于治疗杜氏肌营养不良症（Duchenne）的差异化研究性基因疗法。结果在 2025 年肌肉萎缩症协会 (MDA) 临床与科学会议上公布。

'RGX-202 is the only next generation gene therapy for Duchenne in a pivotal phase trial. The new data from the age 1-3 cohort builds on the favorable safety and efficacy profile seen in ages 4 and older and reinforces the potential of RGX-202 to serve a wide age range of patients,' said

“RGX-202 是唯一进入关键阶段试验的下一代杜氏肌营养不良基因疗法。1至3岁年龄组的新数据进一步巩固了在4岁及以上患者中观察到的良好安全性和有效性，并加强了 RGX-202 治疗广泛年龄段患者的潜力，”

Steve Pakola

史蒂夫·帕科拉

, M.D., Chief Medical Officer of REGENXBIO. 'The consistent, robust microdystrophin levels seen across the age range as well as the functional improvements previously reported support RGX-202's potential to alter the course of this devastating disease. We look forward to sharing additional Phase I/II functional data in the first half of 2025.

医学博士，REGENXBIO首席医疗官表示：“在各个年龄层中观察到的持续且强劲的微抗肌萎缩蛋白水平，以及先前报告的功能改善，支持了RGX-202改变这种毁灭性疾病进程的潜力。我们期待在2025年上半年分享更多的I/II期功能数据。”

We also continue to rapidly advance the pivotal trial towards completing enrollment this year and BLA submission mid-2026.'.

我们还将继续快速推进关键试验，目标是在今年完成入组，并于2026年年中提交BLA。

'Patients with Duchenne continue to be in need of treatment options that could meaningfully impact the course of disease,' said

“杜氏肌营养不良患者仍然需要能够对病程产生有意义影响的治疗选择，”他说。

Carolina Tesi-Rocha

卡罗琳娜·特西-罗查

, M.D., Stanford Children's Hospital. 'The microdystrophin expression and biomarker data presented represent key indicators of potential therapeutic effect. Combined with the safety and functional data to date, I am highly encouraged by the profile of RGX-202.'

，医学博士，斯坦福儿童医院。“所展示的微营养不良蛋白表达和生物标志物数据代表了潜在治疗效果的关键指标。结合迄今为止的安全性和功能性数据，我对RGX-202的特性感到非常鼓舞。”

AFFINITY DUCHENNE Phase I/II Interim Data Updates

AFFINITY DUCHENNE 第一/二期中期数据更新

Biomarker Data

生物标志物数据

New biomarker data from two patients who received the pivotal dose of RGX-202 were presented at MDA and continue to support consistent, robust expression and transduction of RGX-202 microdystrophin across all ages.

在MDA上展示了两名接受关键剂量RGX-202治疗的患者的新型生物标志物数据，这些数据继续支持RGX-202微抗肌萎缩蛋白在所有年龄段中的一致且强劲的表达和转导。

In a patient aged 3 at dosing, microdystrophin expression was measured to be 122.3% compared to control. Patients under 4 years old have no access to gene therapy, and REGENXBIO is the only gene therapy sponsor recruiting patients in this age group in the U.S.

在一名3岁接受治疗的患者中，微量肌营养蛋白表达被测量为对照组的122.3%。4岁以下的患者无法接受基因治疗，而REGENXBIO是美国唯一一家招募该年龄段患者的基因治疗赞助商。

In a patient aged 7 years old, RGX-202 microdystrophin expression was measured to be 31.5% compared to control.

在一名7岁患者中，RGX-202微营养不良蛋白的表达量测量为对照组的31.5%。

In all patients, RGX-202 was appropriately localized to the sarcolemma, demonstrating the differentiated construct with the CT-Domain is appropriately targeting the muscle. RGX-202 microdystrophin expression results in ambulatory patients aged 8+ are the highest reported microdystrophin levels across approved or investigational gene therapies.

在所有患者中，RGX-202 正确定位于肌膜，证明了带有 CT 结构域的差异化构建正确靶向肌肉。在 8 岁及以上可行走患者中，RGX-202 微抗肌萎缩蛋白表达结果是目前已批准或研究中的基因疗法中报告的最高微抗肌萎缩蛋白水平。

To support a Biologics License Application (BLA) using the accelerated approval pathway, the primary endpoint in the pivotal phase of AFFINITY DUCHENNE is the proportion of participants whose RGX-202 microdystrophin expression is ≥10% at Week 12..

为了通过加速审批途径支持生物制品许可申请（BLA），在AFFINITY DUCHENNE的关键阶段，主要终点是第12周时RGX-202微抗肌萎缩蛋白表达量≥10%的参与者比例。

RGX-202 also continues to demonstrate the highest reported vector genome copies (4.9-55.4) measured by qPCR across approved or investigational gene therapies.

RGX-202 还继续展示了通过 qPCR 测量的已批准或研究中的基因疗法中报告的最高载体基因组拷贝数（4.9-55.4）。

Safety and Tolerability Data

安全性与耐受性数据

As of

截至

February 21, 2025

2025年2月21日

, RGX-202 was well tolerated with no serious adverse events (

，RGX-202 耐受性良好，无严重不良事件 (

SAEs

严重不良事件

) and no

）并且没有

AEs

不良事件

of special interest (

特别感兴趣（

AESIs

). Common drug-related

). 常见的药物相关

AEs

不良事件

included nausea, vomiting and fatigue. All resolved and are typically anticipated with gene therapy administration. A thorough, proactive, short-course immune modulation regimen in combination with industry-leading product purity levels of more than 80% full

包括恶心、呕吐和疲劳。所有症状均得到缓解，并且通常是基因治疗过程中预期会出现的反应。结合超过80%的全行业领先产品纯度，进行了一套全面、主动、短期的免疫调节方案。

capsids

衣壳

may contribute to the favorable safety profile seen in patients receiving RGX-202 to date.

可能有助于迄今为止接受 RGX-202 治疗的患者所表现出的良好安全性。

RGX-202 Treatment

RGX-202 治疗

Emergent Adverse Events

紧急不良事件

Dose Level 1

剂量水平1

Dose Evaluation

剂量评估

(1x10

GC/kg)

每千克垃圾收集（GC/kg）

Dose Level 2

剂量水平2

Younger Boys

小男孩

(2x10

GC/kg)

GC/千克）

Dose Level 2

剂量水平2

Dose Evaluation /

剂量评估 /

Expansion

扩展

(2x10

GC/kg)

GC/千克）

Total

总计

n=11

Age Range

年龄范围

(number dosed)

（给药数量）

4-11

(n=3)

1-3

(n=1)

4-11

(n=7)

All Ages

所有年龄段

SAE

AESI

Central or peripheral neurotoxicity

中枢或外周神经毒性

Drug-induced liver injury

药物性肝损伤

Thrombocytopenia

血小板减少症

Myocarditis

心肌炎

Myositis

肌炎

As reported in

据报道

November 2024

2024年11月

, the first five participants in the Phase I/II portion of the AFFINITY DUCHENNE trial all showed functional improvements that exceeded external natural history controls, demonstrating evidence of RGX-202 positively impacting disease trajectory. (

，AFFINITY DUCHENNE试验第一/二期的前五名参与者均表现出超过外部自然病史对照组的功能改善，证明了RGX-202对疾病进程的积极影响。（

press release

新闻稿

). Patients demonstrated stable or improved function on the North Star Ambulatory Assessment (NSAA) and timed function tests. REGENXBIO plans to share additional interim functional data in the first half of 2025.

）。患者在北极星移动评估（NSAA）和定时功能测试中表现出稳定或改善的功能。REGENXBIO计划在2025年上半年分享更多的中期功能数据。

About RGX-202

关于RGX-202

RGX-202 is a potential best-in-class investigational gene therapy designed for improved function and outcomes in Duchenne. RGX-202 is the only gene therapy approved or in late-stage development for Duchenne with a differentiated microdystrophin construct that encodes key regions of naturally occurring dystrophin, including the C-Terminal (CT) domain.

RGX-202 是一种潜在的同类最佳研究性基因疗法，旨在改善杜氏肌营养不良症的功能和治疗效果。 RGX-202 是唯一获准或处于杜氏肌营养不良症晚期开发阶段的基因疗法，其独特的微抗肌萎缩蛋白构建体编码天然存在的抗肌萎缩蛋白的关键区域，包括 C 端 (CT) 结构域。

In preclinical studies, the CT domain has been shown to protect the muscle from contraction-induced stress and improve its ability to repair itself..

在临床前研究中，CT结构域已被证明可以保护肌肉免受收缩引起的应力，并提高其自我修复能力。

Additional design features may potentially improve gene expression, increase protein translation efficiency and reduce immunogenicity. RGX-202 is designed to support the delivery and targeted expression of microdystrophin throughout skeletal and heart muscle using the NAV

额外的设计特征可能有助于提高基因表达、增加蛋白质翻译效率并降低免疫原性。RGX-202旨在通过NAV支持微营养不良蛋白在整个骨骼肌和心肌中的递送和靶向表达。

AAV8 vector and a well-characterized muscle-specific promoter (Spc5-12). RGX-202 is manufactured using REGENXBIO's proprietary, high-yielding NAVXpress™ suspension-based platform process.

AAV8载体和一个特征明确的肌肉特异性启动子（Spc5-12）。RGX-202是使用REGENXBIO专有的高产量NAVXpress™悬浮平台工艺制造的。

About Duchenne Muscular Dystrophy

关于杜氏肌营养不良症

Duchenne is a severe, progressive, degenerative muscle disease, affecting 1 in 3,500 to 5,000 boys born each year worldwide. Duchenne is caused by mutations in the Duchenne gene which encodes for dystrophin, a protein involved in muscle cell structure and signaling pathways. Without dystrophin, muscles throughout the body degenerate and become weak, eventually leading to loss of movement and independence, required support for breathing, cardiomyopathy and premature death..

杜氏肌营养不良症是一种严重的、进行性的、退行性肌肉疾病，每年全世界每3500至5000名新生男婴中就有一人受到影响。杜氏肌营养不良症是由杜氏基因突变引起的，该基因编码一种参与肌肉细胞结构和信号通路的蛋白质——抗肌萎缩蛋白。没有抗肌萎缩蛋白，全身的肌肉会退化并变得无力，最终导致运动和独立性的丧失，需要呼吸支持，出现心肌病并早逝。

ABOUT REGENXBIO Inc.

关于REGENXBIO公司

REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie.

REGENXBIO是一家生物技术公司，致力于通过基因治疗的治愈潜力改善生活。自2009年成立以来，REGENXBIO一直是AAV基因治疗领域的先驱。REGENXBIO正在推进一系列针对罕见病和视网膜疾病的晚期一次性治疗管线，包括用于治疗杜氏肌营养不良的RGX-202；与日本新药株式会社合作开发的用于治疗MPS II的clemidsogene lanparvovec（RGX-121）和用于治疗MPS I的RGX-111；以及与艾伯维合作开发的用于治疗湿性AMD和糖尿病视网膜病变的surabgene lomparvovec（ABBV-RGX-314）。

Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA.

数千名患者已经通过 REGENXBIO 的 AAV 平台接受了治疗，其中包括接受诺华公司 ZOLGENSMA 治疗的患者。

. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit

REGENXBIO的在研基因疗法有可能改变数百万人的医疗保健方式。欲了解更多信息，请访问

www.REGENXBIO.com

。

FORWARD-LOOKING STATEMENTS

前瞻性声明

This press release includes 'forward-looking statements,' within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as 'believe,' 'may,' 'will,' 'estimate,' 'continue,' 'anticipate,' 'assume,' 'design,' 'intend,' 'expect,' 'could,' 'plan,' 'potential,' 'predict,' 'seek,' 'should,' 'would' or by variations of such words or by similar expressions.

本新闻稿包含《1933年证券法》第27A条（经修订）和《1934年证券交易法》第21E条（经修订）所指的“前瞻性陈述”。这些陈述表达了一种信念、期望或意图，并通常伴随着传达预计未来事件或结果的词语，例如“相信”、“可能”、“将”、“估计”、“继续”、“预期”、“假设”、“设计”、“打算”、“预计”、“能够”、“计划”、“潜力”、“预测”、“寻求”、“应该”、“会”，或是这些词语的变体或类似表述。

The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations, clinical trials, costs and cash flow. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances.

前瞻性声明包括与REGENXBIO未来运营、临床试验、成本和现金流等相关的内容。REGENXBIO基于其当前的预期、假设以及根据其经验、对历史趋势、当前状况和预期未来发展的看法进行的分析，以及其他REGENXBIO认为在相关情况下适当的因素，作出了这些前瞻性声明。

However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timing or likelihood of payments from AbbVie or Nippon Shinyaku, the monetization of any priority review voucher, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and mainta.

然而，实际结果和发展是否符合REGENXBIO的预期和预测，仍受多种风险和不确定性的影响，包括REGENXBIO及其被许可方和合作伙伴进行的临床试验的入组、启动和完成时间及成功与否，REGENXBIO及其开发合作伙伴进行的临床前研究的启动和完成时间及成功与否，AbbVie或Nippon Shinyaku付款的时间或可能性，任何优先审查券的货币化，新产品的及时开发和上市，获得并维持候选产品监管批准的能力，以及获得并维持。

December 31, 2024

2024年12月31日

, which will be filed with the U.S. Securities and Exchange Commission (SEC) in the first quarter of 2025, and comparable 'risk factors' sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at

，该报告将于2025年第一季度提交给美国证券交易委员会（SEC），以及REGENXBIO的季度报告10-Q表格和其他已提交给SEC的文件中类似“风险因素”部分，这些文件已在美国证券交易委员会网站上公布，网址为

WWW.SEC.GOV

. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations.

本新闻稿中所有的前瞻性声明均明确受到本文所述或提及的警示性声明的限制。预期的实际结果或发展可能无法实现，或者即使基本实现，它们也可能不会对REGENXBIO及其业务或运营产生预期的后果或影响。

Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release.

此类声明并非对未来业绩的保证，实际结果或发展可能与前瞻性声明中预计的情况有重大差异。读者被提醒不要过分依赖本新闻稿中包含的前瞻性声明。这些前瞻性声明仅适用于本新闻稿发布之日。

Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise..

除非法律要求，REGENXBIO不承担任何义务，并明确拒绝任何更新或修改前瞻性声明的义务，无论是否因新信息、未来事件或其他原因。

Zolgensma

诺西那生钠

is a registered trademark of Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO.

是诺华基因疗法的注册商标。本文提及的所有其他商标均为 REGENXBIO 的注册商标。

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联系人：

Dana

达娜

Cormack

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