Pivotal data evaluating the safety and efficacy of the subretinal delivery of ABBV-RGX-314 in patients with

关键数据评估了视网膜下递送ABBV-RGX-314治疗糖尿病患者的安全性和有效性

wet age-related macular degeneration (wet AMD) are expected in 2026

预计2026年将出现湿性年龄相关性黄斑变性（湿性AMD）

AbbVie and REGENXBIO will plan the Phase 3 clinical program of investigational ABBV-RGX-314 in diabetic retinopathy (DR)

AbbVie和REGENXBIO将计划糖尿病视网膜病变（DR）中研究性ABBV-RGX-314的3期临床计划

NORTH CHICAGO, Ill.

伊利诺伊州北芝加哥。

and

和

ROCKVILLE, Md.

马里兰州罗克维尔。

,

,

Jan. 13, 2025

2025年1月13日

/

/

PRNewswire

美通社

/ -- AbbVie (NYSE: ABBV) and REGENXBIO Inc. (Nasdaq: RGNX) today announced updates to the ABBV-RGX-314 clinical program.

/--AbbVie（纽约证券交易所代码：ABBV）和REGENXBIO Inc.（纳斯达克代码：RGNX）今天宣布了对ABBV-RGX-314临床项目的更新。

ABBV-RGX-314 in Wet Age-Related Macular Degeneration (wet AMD), Subretinal Delivery

ABBV-RGX-314在湿性年龄相关性黄斑变性（湿性AMD）视网膜下分娩中的应用

Data from the ATMOSPHERE

来自大气的数据

®

®

and ASCENT

和上升

™

™

pivotal trials evaluating the safety and efficacy of the subretinal delivery of ABBV-RGX-314 in patients with wet AMD are expected in 2026.

预计2026年将进行关键试验，评估视网膜下递送ABBV-RGX-314治疗湿性AMD患者的安全性和有效性。

ABBV-RGX-314 in

ABBV-RGX-314和

Diabetic Retinopathy (DR), Suprachoroidal Delivery

糖尿病视网膜病变（DR），脉络膜上分娩

AbbVie and REGENXBIO will plan a Phase 3 clinical program. The clinical program will utilize the in-office SCS Microinjector

AbbVie和REGENXBIO将计划一项3期临床计划。临床计划将利用办公室SCS微量注射器

®

®

to deliver gene therapy to the suprachoroidal space of the eye.

将基因疗法传递到眼睛的脉络膜上空间。

'Retinal diseases are progressive, with wet AMD and DR among the leading causes of blindness,' said Michael Robinson, M.D., vice president, global head of ophthalmology clinical development, AbbVie. 'More treatment options are needed to help relieve the current treatment burden of chronic, frequent dosing.

“视网膜疾病是进行性的，湿性AMD和DR是导致失明的主要原因之一，”AbbVie眼科临床开发全球负责人、副总裁迈克尔·罗宾逊（MichaelRobinson）医学博士说需要更多的治疗选择来帮助减轻目前慢性频繁给药的治疗负担。

We are excited to continue moving closer to our goal of delivering an additional treatment option to patients with wet AMD and DR in hopes of addressing their significant unmet needs.'.

我们很高兴能够继续朝着为湿性AMD和DR患者提供额外治疗选择的目标迈进，以期解决他们尚未满足的重大需求。”。

'ABBV-RGX-314 has the potential to help millions of people living with wet AMD and DR, globally, who are facing these debilitating diseases,' said

“ABBV-RGX-314有可能帮助全球数百万患有湿性AMD和DR的人，他们正面临这些使人衰弱的疾病，”他说

Curran Simpson

柯兰·辛普森

, president and chief executive officer, REGENXBIO. 'Together with AbbVie, we are excited to continue developing ABBV-RGX-314 as the first potential one-time gene therapy for wet AMD and DR.'

，REGENXBIO总裁兼首席执行官。“我们很高兴与AbbVie一起继续开发ABBV-RGX-314，作为湿性AMD和DR的第一种潜在的一次性基因疗法。”

About ABBV-RGX-314

关于ABBV-RGX-314

ABBV-RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy and potentially other chronic retinal conditions. ABBV-RGX-314 consists of the NAV

正在研究ABBV-RGX-314作为湿性AMD，糖尿病视网膜病变和潜在的其他慢性视网膜疾病的潜在一次性治疗方法。ABBV-RGX-314由导航组成

®

®

AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). ABBV-RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.

AAV8载体，其编码设计用于抑制血管内皮生长因子（VEGF）的抗体片段。据信ABBV-RGX-314抑制VEGF途径，通过该途径，新的渗漏血管生长并促进视网膜中的液体积聚。

1

1

AbbVie and REGENXBIO are advancing the development of two separate routes of administration of ABBV-RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector

AbbVie和REGENXBIO正在通过标准化的视网膜下递送程序以及递送至脉络膜上空间，推进开发两种单独的ABBV-RGX-314眼部给药途径。REGENXBIO已向SCS微量注射器授予了某些专有权

®

®

from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.

由Clearside Biomedical，Inc.提供基因治疗治疗眼脉络膜上空间。

About Wet AMD

关于湿性AMD

Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina.

湿性AMD的特征是由于视网膜中新的渗漏血管形成而导致视力丧失。

2

2

Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to two million people living with wet AMD in these geographies alone.

湿性AMD是美国、欧洲和日本视力丧失的重要原因，仅在这些地区就有多达200万人患有湿性AMD。

3

3

Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients.

目前的抗VEGF疗法已经显着改变了湿性AMD的治疗前景，由于其能够预防大多数患者视力丧失的进展，因此成为标准治疗方法。

4

4

These therapies, however, require life-long repeated intraocular injections, to maintain efficacy.

然而，这些疗法需要终身重复眼内注射才能维持疗效。

5,6

5,6

Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time.

由于治疗负担，随着时间的推移，患者经常会出现视力下降，治疗频率降低。

7

7

About Diabetic Retinopathy

关于糖尿病视网膜病变

Diabetic retinopathy (DR) is the leading cause of vision loss in adults between 24 and 75 years of age worldwide.

糖尿病视网膜病变（DR）是全球24至75岁成年人视力丧失的主要原因。

8

8

DR affects nearly 10 million people in

DR影响了近1000万人

the United States

美国

alone.

独自一人。

9

9

The spectrum of DR severity ranges from non-proliferative diabetic retinopathy (NPDR) to proliferative diabetic retinopathy (PDR).

DR严重程度的范围从非增殖性糖尿病视网膜病变（NPDR）到增殖性糖尿病视网膜病变（PDR）。

4

4

As DR progresses, a large proportion of patients develop vision threatening complications, including diabetic macular edema (DME) and neovascularization that can lead to blindness.

随着DR的进展，大部分患者会出现威胁视力的并发症，包括糖尿病性黄斑水肿（DME）和可能导致失明的新血管形成。

10

10

Current treatment options for patients with NPDR typically include 'watchful waiting' or anti-VEGF treatment. For patients with PDR, current treatment options include anti-VEGF treatment or retinal laser; surgical treatment may be required for advanced PDR.

NPDR患者目前的治疗选择通常包括“观察等待”或抗VEGF治疗。对于PDR患者，目前的治疗选择包括抗VEGF治疗或视网膜激光；晚期PDR可能需要手术治疗。

2

2

About AbbVie

关于AbbVie

AbbVie's mission is to discover and deliver innovative medicines and solutions that solve serious health issues today and address the medical challenges of tomorrow. AbbVie strives to have a remarkable impact on people's lives across several key therapeutic areas – immunology, oncology, neuroscience, and eye care – and products and services in AbbVie's Allergan Aesthetics portfolio.

AbbVie的使命是发现并提供创新药物和解决方案，以解决当今严重的健康问题，并应对未来的医疗挑战。AbbVie致力于在几个关键治疗领域（免疫学、肿瘤学、神经科学和眼部护理）以及AbbVie Allergan美学组合中的产品和服务中对人们的生活产生显着影响。

For more information about AbbVie, please visit us at .

有关AbbVie的更多信息，请访问我们。

www.abbvie.com

www.abbvie.com

. Follow @abbvie on

.关注@abbvie on

LinkedIn,

LinkedIn，

Facebook

脸书

,

,

Instagram

Instagram

,

,

X (formerly Twitter)

X（前推特）

, and

，以及

YouTube.

YouTube。

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 rare and retinal diseases, including RGX-202 for the treatment of Duchenne, ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, 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AAV治疗罕见和视网膜疾病的管道，包括用于治疗Duchenne的RGX-202，用于治疗湿性AMD和糖尿病视网膜病变的ABBV-RGX-314，正在与AbbVie合作开发，以及用于治疗MPS II的RGX 121。

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

数千名患者接受了REGENXBIO的AAV治疗平台的治疗，其中包括诺华的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

用于患有脊髓性肌萎缩症的儿童。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

www.regenxbio.com

.

.

Forward-Looking Statements

前瞻性声明

Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words 'believe,' 'expect,' 'anticipate,' 'project' and similar expressions and uses of future or conditional verbs, generally identify forward-looking statements.

就1995年《私人证券诉讼改革法案》而言，本新闻稿中的一些声明是或可能被视为前瞻性声明。单词“相信”、“期望”、“预期”、“项目”以及类似的将来动词或条件动词的表达和用法通常表示前瞻性陈述。

AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, and changes to laws and regulations applicable to our industry.

AbbVie警告称，这些前瞻性声明存在风险和不确定性，可能导致实际结果与前瞻性声明中明示或暗示的结果存在重大差异。此类风险和不确定性包括但不限于知识产权的挑战、其他产品的竞争、研发过程中固有的困难、不利的诉讼或政府行为以及适用于本行业的法律法规的变化。

Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, 'Risk Factors,' of AbbVie's 2023 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its subsequent Quarterly Reports on Form 10-Q.

有关可能影响艾伯维运营的经济、竞争、政府、技术和其他因素的其他信息，请参见艾伯维2023年年度报告表10-K第1A项“风险因素”，该报告已提交给美国证券交易委员会，并由其随后的10-Q表季度报告更新。

AbbVie undertakes no obligation, and specifically declines, to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law..

除法律要求外，AbbVie没有义务，也明确拒绝因后续事件或事态发展而公开发布对前瞻性声明的任何修订。。

1

1

Penn JS, Madan A, Caldwell RB, et al. Vascular endothelial growth factor in eye disease. Prog Retin Eye Res. 2008;27(4):331-71.

Penn JS，Madan A，Caldwell RB等。血管内皮生长因子在眼病中的作用。Prog Retin Eye Res.2008；27（4）：331-71。

2

2

Carmeliet P. Angiogenesis in life, disease and medicine. Nature. 2005;438:932-6.

Carmeliet P.生命，疾病和医学中的血管生成。自然。2005年；438:932-6年。

3

3

Decision Resources Group, 2019

决策资源小组，2019年

4

4

Alexandru MR,

亚历山大先生，

Alexandra NM

亚历山德拉NM

. Wet age related macular degeneration management and follow-up. Rom J Ophthalmol. 2016;60:9–13.

.湿性年龄相关性黄斑变性的管理和随访。Rom J Ophthalmol。2016年；60:9-13。

5

5

AAO PPP. Preferred Practice Patterns: Age related macular degeneration. American Academy of Ophthalmology. 2019.

AAO PPP。首选实践模式：年龄相关性黄斑变性。美国眼科学会。2019

6

6

Dugel PU, Koh A, Ogura Y, et al. HAWK and HARRIER: phase 3, multicenter, randomized, double-masked trials of brolucizumab for neovascular age-related macular degeneration. Ophthalmology. 2020;127(1):72-84.

Dugel PU，Koh A，Ogura Y等。HAWK和HARRIER：brolucizumab治疗新生血管性年龄相关性黄斑变性的3期多中心随机双盲试验。眼科学。2020年；127（1）：72-84。

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7

Holz FG et al. Br J Ophthalmol. 2015;99:220.

Holz FG等人，Br J Ophthalmol。2015年；99:220。

8

8

Cheung N, Mitchell P, Wong TY. Diabetic retinopathy. Lancet. 2010;376(9735):124–36.

Cheung N，Mitchell P，Wong TY。糖尿病视网膜病变。柳叶刀。2010年；376（9735）：124-36。

9

9

Lundeen EA, Burke-Conte Z, Rein DB, Wittenborn JS, Saaddine J, Lee AY, Flaxman AD. Prevalence of Diabetic Retinopathy in the US in 2021. JAMA Ophthalmology. 2023;141(8):747-754.

Lundeen EA，Burke Conte Z，Rein DB，Wittenborn JS，Saaddine J，Lee AY，Flaxman AD.2021年美国糖尿病视网膜病变的患病率。JAMA眼科。2023年；141（8）：747-754。

10

10

Berrocal MD, Alexandra Acabá. Current Management of Diabetic Retinopathy, 2018

Berrocal MD，Alexandra Acabá。糖尿病视网膜病变的当前管理，2018年

SOURCE AbbVie

来源AbbVie