Company to begin enrollment of patients aged 1-3 years

公司将开始招募1-3岁的患者

Expects data from younger cohort to be part of pivotal plans and BLA filing for broad label

预计来自年轻人群的数据将成为关键计划和BLA申请广泛标签的一部分

End-of-Phase II meeting with FDA scheduled for late July to finalize pivotal program design

计划于7月下旬与FDA举行第二阶段会议，以最终确定关键项目设计

Based on recent commercial landscape, confirmed accelerated approval pathway remains available given ongoing unmet need and RGX-202 differentiated design

根据最近的商业前景，鉴于持续未满足的需求和RGX-202的差异化设计，已确认的加速批准途径仍然可用

Remains on track to initiate pivotal trial in late Q3 to early Q4 2024

有望在2024年第三季度末至第四季度初启动关键试验

The next potential therapy to become available for Duchenne patients

Duchenne患者的下一种潜在疗法

ROCKVILLE, Md., June 24, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it initiated enrollment in a new cohort of patients ages 1-3 in its Phase I/II AFFINITY DUCHENNE® trial to evaluate the safety and efficacy of RGX-202 in boys with Duchenne muscular dystrophy (Duchenne).

马里兰州罗克维尔。，2024年6月24日/PRNewswire/--REGENXBIO Inc.（纳斯达克：RGNX）今天宣布，在其I/II期亲和DUCHENNE®试验中，开始招募一组新的1-3岁患者，以评估RGX-202在DUCHENNE肌营养不良症（DUCHENNE）男孩中的安全性和有效性。

RGX-202 is an investigational one-time AAV Therapeutic targeted to deliver a novel microdystrophin, representing the next wave of innovative design in Duchenne gene therapy. RGX-202 is the only gene therapy approved or in development for Duchenne that incorporates the C-Terminal domain, making the RGX-202 transgene the closest to the naturally occurring dystrophin gene..

RGX-202是一种研究性的一次性AAV治疗剂，旨在提供一种新型的微肌营养不良蛋白，代表了杜兴基因治疗的下一波创新设计。RGX-202是唯一被批准或正在开发的Duchenne基因疗法，它包含了C末端结构域，使RGX-202转基因最接近天然存在的肌营养不良蛋白基因。。

With this announcement, REGENXBIO is enrolling ambulatory boys with Duchenne aged 1 to 11 in the AFFINITY DUCHENNE trial. The new cohort is expected to enroll up to five patients aged 1-3 to receive RGX-202 at the pivotal dose level (2x1014 genome copies (GC)/kg body weight).

通过这一宣布，REGENXBIO正在为1至11岁的Duchenne招募非卧床男孩参加AFFINITY Duchenne试验。预计新队列将招募多达5名1-3岁的患者，以关键剂量水平（2x1014个基因组拷贝（GC）/kg体重）接受RGX-202治疗。

Additionally, REGENXBIO has confirmed an end-of-Phase II (EOP2) meeting is scheduled with the FDA at the end of July. This meeting is expected to finalize the AFFINITY DUCHENNE pivotal trial design, with the goal of continuing to expedite the development of RGX-202. The Company anticipates that all patients enrolled at dose level 2 (n=12) will be included in its pivotal trial data set.

此外，REGENXBIO已确认将于7月底与FDA举行第二阶段（EOP2）会议。预计这次会议将最终确定AFFINITY DUCHENNE关键试验设计，目标是继续加快RGX-202的开发。该公司预计，所有以2级剂量（n=12）登记的患者将被纳入其关键试验数据集。

The Company plans to use RGX-202 microdystrophin expression as a surrogate endpoint to support a Biologics License Application (BLA) submission using the accelerated approval pathway with the potential to receive a broad label. REGENXBIO has recently confirmed with the FDA that the pathway can be used given the ongoing high unmet need for differentiated treatment options in the Duchenne community.

该公司计划使用RGX-202微肌营养不良蛋白表达作为替代终点，以支持使用加速批准途径提交生物制剂许可证申请（BLA），并有可能获得广泛的标签。REGENXBIO最近与FDA确认，鉴于杜兴社区对差异化治疗方案的需求持续很高，因此可以使用该途径。

RGX-202 is the most advanced gene therapy enrolling in active clinical trials and is anticipated to be the next gene therapy in a BLA filing for Duchenne.  .

RGX-202是参与积极临床试验的最先进的基因疗法，预计将成为Duchenne BLA申请中的下一种基因疗法。。

'We believe RGX-202 has unique, differentiating features that support its potential to be a best-in-class product and we are pleased to expand its clinical development to reach a wider range of boys with Duchenne in need of treatment options,' said Curran Simpson, Chief Operating Officer of REGENXBIO and President and CEO-elect.

REGENXBIO首席运营官兼总裁兼首席执行官当选人柯兰·辛普森（Curran Simpson）说：“我们相信RGX-202具有独特的、与众不同的功能，有可能成为一流的产品，我们很高兴扩大其临床开发范围，以惠及更多需要治疗选择的杜兴男孩。”。

'Supported by the strong safety profile and positive microdystrophin data demonstrated in the AFFINITY DUCHENNE trial, today's news marks significant steps in rapidly accelerating RGX-202 towards pivotal stage and future commercialization.'.

“在AFFINITY DUCHENNE试验中证明的强大安全性和阳性微肌营养不良蛋白数据的支持下，今天的新闻标志着RGX-202朝着关键阶段和未来商业化的方向快速加速迈出了重要的一步。”。

'The Duchenne community remains in need of differentiated treatment options, and I'm pleased to see the expansion of the AFFINITY DUCHENNE trial to evaluate RGX-202 in younger patients,' said Vamshi K. Rao, M.D., Lurie Children's Hospital, Associate Professor of Pediatrics, Northwestern University Feinberg School of Medicine and trial investigator.

。

'With the wider adoption of newborn screening, there is now an increased opportunity to treat patients earlier, with the hope of impacting disease and preserving muscle.'.

“随着新生儿筛查的广泛采用，现在有更多的机会更早地治疗患者，希望能够影响疾病并保护肌肉。”。

Clinical Program Updates

临床计划更新

The Company continues to rapidly enroll the remaining patients in the ages 4-11 cohort (dose level 2 expansion cohort) and expects to imminently complete the enrollment of up to seven patients at dose level 2 early third quarter of 2024.

。

REGENXBIO expects to share initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial in the second half of 2024. Initiation of the pivotal trial is on track for late third quarter to early fourth quarter of 2024.

REGENXBIO预计将在2024年下半年分享AFFINITY DUCHENNE试验两种剂量水平的初始强度和功能评估数据。关键性试验的启动将于2024年第三季度末至第四季度初进行。

RGX-202 in the AFFINITY DUCHENNE trial is manufactured using REGENXBIO's proprietary, high-yielding NAVXpress™ platform process. This suspension-based manufacturing process has demonstrated robust scalability with consistent yield and product purity and is ready for product commercialization.

AFFINITY DUCHENNE试验中的RGX-202是使用REGENXBIO专有的高产NAVXpress™平台工艺制造的。这种基于悬浮液的制造工艺已证明具有强大的可扩展性，具有一致的产量和产品纯度，并已为产品商业化做好准备。

As of May 3, 2024, RGX-202 has been well tolerated with no drug-related serious adverse events in five patients, aged 4.4 to 12.1. All five patients who completed three-month trial assessments indicate encouraging increases in expression of RGX-202 microdystrophin and reduction from baseline in serum CK levels, supporting evidence of clinical improvement..

截至2024年5月3日，RGX-202在5名年龄在4.4至12.1岁的患者中耐受性良好，没有与药物相关的严重不良事件。完成三个月试验评估的所有五名患者均表明，RGX-202微肌营养不良蛋白的表达令人鼓舞地增加，血清CK水平从基线水平降低，支持了临床改善的证据。。

AFFINITY DUCHENNE Trial Design

AFFINITY DUCHENNE试验设计

The Phase I/II AFFINITY DUCHENNE trial is a multicenter, open-label dose escalation and dose expansion clinical study to evaluate the safety, tolerability and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in patients with Duchenne aged 1-11.

I/II期亲和DUCHENNE试验是一项多中心，开放标签剂量递增和剂量扩展临床研究，旨在评估一次性静脉注射（IV）剂量RGX-202在DUCHENNE患者中的安全性，耐受性和临床疗效1-11岁。

The trial design was informed by the Duchenne community and engagement with key opinion leaders, including a comprehensive, short-term, prophylactic immunosuppression regimen to proactively mitigate potential complement-mediated immunologic responses, and inclusion criteria based on dystrophin gene mutation status, including DMD gene mutations in exons 18 and above.

试验设计由Duchenne社区告知，并与关键意见领袖进行了接触，包括全面，短期，预防性免疫抑制方案，以主动减轻潜在的补体介导的免疫反应，以及基于肌营养不良蛋白基因突变状态的纳入标准，包括外显子18及以上的DMD基因突变。

Trial endpoints include safety, immunogenicity assessments, pharmacodynamic and pharmacokinetic measures of RGX-202, including microdystrophin protein levels in muscle, and strength and functional assessments, including the North Star Ambulatory Assessment (NSAA) and timed function tests..

试验终点包括RGX-202的安全性，免疫原性评估，药效学和药代动力学测量，包括肌肉中的微肌营养不良蛋白水平，以及力量和功能评估，包括北极星动态评估（NSAA）和定时功能测试。。

About RGX-202

RGX-202 has differentiated and important biology most similar to naturally occurring dystrophin that protects from the muscle degradation associated with Duchenne. RGX-202 is designed with advanced science to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin.

RGX-202具有与天然存在的肌营养不良蛋白最相似的分化和重要生物学特性，可防止与杜兴氏相关的肌肉降解。RGX-202是根据高级科学设计的，用于为新型微肌营养不良蛋白提供转基因，该微肌营养不良蛋白包括天然存在的肌营养不良蛋白中C末端（CT）结构域的功能元件。

Presence of the CT domain has been shown in preclinical studies to recruit several key proteins to the muscle cell membrane, leading to improved muscle resistance to contraction-induced muscle damage in dystrophic mice. Additional design features, including codon optimization and reduction of CpG content, may potentially improve gene expression, increase translational efficiency and reduce immunogenicity.

临床前研究表明，CT结构域的存在可以将几种关键蛋白募集到肌肉细胞膜上，从而改善营养不良小鼠对收缩引起的肌肉损伤的肌肉抵抗力。其他设计特征，包括密码子优化和CpG含量降低，可能会改善基因表达，提高翻译效率并降低免疫原性。

RGX-202 is designed to support the delivery and targeted expression of genes throughout skeletal and heart muscle using the NAV AAV8 vector, a vector used in numerous clinical trials, and a well-characterized muscle-specific promoter (Spc5-12)..

RGX-202旨在使用NAV AAV8载体（用于许多临床试验的载体）和特征明确的肌肉特异性启动子（Spc5-12），支持整个骨骼肌和心肌中基因的传递和靶向表达。。

About Duchenne Muscular Dystrophy

关于杜兴氏肌营养不良症

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..

Duchenne是一种严重的，进行性的，退行性的肌肉疾病，影响全世界每年出生的3500至5000名男孩中的1名。Duchenne是由Duchenne基因突变引起的，该基因编码肌营养不良蛋白，肌营养不良蛋白是一种参与肌肉细胞结构和信号通路的蛋白质。没有肌营养不良蛋白，全身肌肉退化并变得虚弱，最终导致运动和独立性丧失，需要呼吸支持，心肌病和过早死亡。。

ABOUT REGENXBIO Inc.

关于REGENXBIO Inc。

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal and rare diseases, including ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, RGX-202 for the treatment of Duchenne and RGX-121 for the treatment of MPS II.

REGENXBIO是一家领先的临床阶段生物技术公司，旨在通过基因治疗的治疗潜力来改善生活。自2009年成立以来，REGENXBIO率先开发了AAV Therapeutics，这是一类创新的基因治疗药物。REGENXBIO正在推进一系列针对视网膜和罕见疾病的AAV治疗方法，包括用于治疗湿性AMD和糖尿病视网膜病变的ABBV-RGX-314，正在与AbbVie，RGX-202合作开发用于治疗Duchenne和RGX-121用于治疗MPS II。

Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA for children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.regenxbio.com..

数千名患者接受了REGENXBIO的AAV治疗平台的治疗，包括诺华的ZOLGENSMA治疗脊髓性肌萎缩症儿童。AAV疗法旨在一次性治疗，有可能改变数百万人的医疗保健方式。有关更多信息，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.

本新闻稿包括经修订的《1933年证券法》第27A节和经修订的《1934年证券交易法》第21E节所指的“前瞻性声明”。这些陈述表达了一种信念、期望或意图，通常伴随着表达预计未来事件或结果的词语，如“相信”、“可能”、“意志”、“估计”、“继续”、“预期”、“假设”、“设计”、“打算”、“期望”、“可能”、“计划”、“潜力”、“预测”、“寻求”、“应该”、“将会”或这些词语的变体或类似表达。

The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations, clinical trials, regulatory plans, 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.

前瞻性报表包括与REGENXBIO未来运营、临床试验、监管计划、成本和现金流等相关的报表。REGENXBIO根据其经验、对历史趋势、现状和预期未来发展的看法，以及REGENXBIO认为在这种情况下合适的其他因素，根据REGENXBIO目前的预期、假设和分析，做出了这些前瞻性陈述。

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 timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges .

然而，实际结果和发展是否符合REGENXBIO的预期和预测，取决于许多风险和不确定性，包括REGENXBIO、其被许可方及其合作伙伴进行的注册、开始和完成的时间以及临床试验的成功，REGENXBIO及其开发合作伙伴进行的临床前研究的开始和完成的时间以及成功，新产品的及时开发和推出，获得和维持候选产品监管批准的能力，获得和维持候选产品知识产权保护的能力以及技术、趋势和挑战。

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

Zolgensma®是诺华基因疗法的注册商标。。

Contacts:

联系人：

Dana Cormack

Dana Cormack

Corporate Communications

公司通信

dcormack@regenxbio.com

dcormack@regenxbio.com

Investors:

投资者：

Chris Brinzey

Chris Brinzey

ICR Westwicke

ICR Westwicke

339-970-2843

339-970-2843

chris.brinzey@westwicke.com

chris.brinzey@westwicke.com

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SOURCE REGENXBIO Inc.

来源REGENXBIO股份有限公司。

Company Codes: NASDAQ-NMS:RGNX

公司代码：NASDAQ-NMS：RGNX