A single ABBV-RGX-314 gene therapy treatment has the potential to become a new standard-of-care option among anti-VEGF treatments by sustaining vision health long term and overcoming the clinical challenges of managing wet AMD due to the treatment burden of chronic anti-VEGF injections

通过长期维持视力健康并克服由于慢性抗VEGF注射的治疗负担而导致的湿性AMD管理的临床挑战，单一的ABBV-RGX-314基因治疗疗法有可能成为抗VEGF治疗中新的护理标准选择

ABBV-RGX-314 continues to be well tolerated in over 100 patients from three dose levels with no drug-related serious adverse events

ABBV-RGX-314在三个剂量水平的100多名患者中仍然具有良好的耐受性，没有与药物相关的严重不良事件

ABBV-RGX-314 in over 50 patients at third dose level demonstrated highest reduction in treatment burden:

在第三剂量水平的50多名患者中，ABBV-RGX-314表现出最高的治疗负担减轻：

80% reduction in annualized injection rate

年注射率降低80%

50% injection-free

50%免注射

Zero cases of intraocular inflammation observed in patients that received short-course prophylactic topical steroid eye drops

在接受短程预防性局部类固醇滴眼液的患者中未观察到眼内炎症病例

Conference call Tuesday, January 16 at 4:30 p.m. EST

美国东部时间1月16日星期二下午4:30召开电话会议

ROCKVILLE, Md., Jan. 16, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced positive interim data from the Phase II AAVIATE® trial of ABBV-RGX-314 for the treatment of wet age-related macular degeneration (wet AMD) using suprachoroidal delivery. Wet AMD is a chronic, life-long disease with available anti-VEGF treatment options that may reduce the risk of blindness, but require frequent injections.

ROCKVILLE，Md.，2024年1月16日/PRNewswire/--REGENXBIO Inc.（纳斯达克：RGNX）今天宣布了来自ABBV-RGX-314的II期AAVIATE®试验的积极中期数据，用于治疗湿性年龄相关性黄斑变性（湿性AMD）使用脉络膜上递送。湿性AMD是一种慢性终身疾病，可用抗VEGF治疗方案可降低失明风险，但需要频繁注射。

Investigational ABBV-RGX-314 using suprachoroidal delivery is designed to be a one-time, in office treatment that has the potential to sustain constant anti-VEGF therapy and stabilize or improve vision long-term for wet AMD patients. The new data presented at the Hawaiian Eye and Retina meeting in Maui, HI by John Pitcher, M.D., Eye Associates of New Mexico, includes 6-month results from two additional dose level 3 cohorts (Cohorts 5 and 6).

使用脉络膜上递送的研究性ABBV-RGX-314被设计为一次性的办公室治疗，有可能维持持续的抗VEGF治疗，并长期稳定或改善湿性AMD患者的视力。新墨西哥州眼科协会的医学博士约翰·皮彻（JohnPitcher）在夏威夷州毛伊岛（Maui）举行的夏威夷眼科和视网膜会议上提供的新数据包括另外两个剂量水平为3的队列（队列5和6）的6个月结果。

ABBV-RGX-314, being developed in collaboration with AbbVie, is also being investigated as a potential one-time gene therapy for the treatment of diabetic retinopathy and other chronic retinal conditions.            .

与AbbVie合作开发的ABBV-RGX-314也正在研究作为治疗糖尿病视网膜病变和其他慢性视网膜疾病的潜在一次性基因疗法。。

'The potential of ABBV-RGX-314 to transform the way chronic retinal conditions are treated has been demonstrated in multiple trials using both subretinal and suprachoroidal delivery, and these latest interim results from the AAVIATE trial continue to support this outlook,' said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO.

REGENXBIO首席医疗官StevePakola医学博士说：“使用视网膜下和脉络膜上分娩的多项试验证明了ABBV-RGX-314改变慢性视网膜疾病治疗方式的潜力，这些来自AAVIATE试验的最新中期结果继续支持这一前景。”。

'One-time, in-office treatment with ABBV-RGX-314 continues to be well tolerated in patients with wet AMD. We are pleased that dose level 3 in this trial has shown the highest reduction in treatment burden with zero cases of inflammation observed in patients who received prophylactic short-course topical steroids.

“有一次，对于湿性AMD患者，使用ABBV-RGX-314进行的办公室治疗仍然具有良好的耐受性。我们很高兴，该试验中的剂量水平3显示出治疗负担的最高降低，在接受预防性短程局部类固醇的患者中观察到零例炎症。

We are encouraged by this data as part of the global ABBV-RGX-314 program.'.

作为全球ABBV-RGX-314计划的一部分，我们对这些数据感到鼓舞。”。

'Real world evidence shows that patients with wet AMD are often undertreated due to the unsustainable treatment burden of frequent injections. As a result, many wet AMD patients experience significant vision loss over time,' said Dr. Pitcher. 'I am encouraged by the safety profile of ABBV-RGX-314 using suprachoroidal delivery and the potential of a one-time, in-office injection that can sustain constant anti-VEGF therapy to address a major unmet need for patients.'.

“现实世界的证据表明，湿性AMD患者往往由于频繁注射造成不可持续的治疗负担而未得到充分治疗。皮彻博士说，结果是，随着时间的推移，许多湿性AMD患者会出现严重的视力下降我对使用脉络膜上递送的ABBV-RGX-314的安全性以及一次性办公室注射的潜力感到鼓舞，该注射可以维持持续的抗VEGF治疗，以解决患者的主要未满足需求。”。

Data and Safety SummaryAAVIATE is a Phase II, multi-center, open-label, randomized, active-controlled, dose-escalation trial that is evaluating the efficacy, safety and tolerability of suprachoroidal delivery of ABBV-RGX-314 in patients with wet AMD. The primary endpoint of the trial is mean change in vision in patients dosed with ABBV-RGX-314, as measured by best corrected visual acuity (BCVA) at Week 40 from baseline, compared to patients receiving monthly injections of ranibizumab.

数据和安全性总结Aaviate是一项II期，多中心，开放标签，随机，主动控制，剂量递增试验，旨在评估脉络膜上递送ABBV-RGX-314对湿性AMD患者的疗效，安全性和耐受性。该试验的主要终点是服用ABBV-RGX-314的患者的平均视力变化，与每月注射雷珠单抗的患者相比，在基线第40周通过最佳矫正视力（BCVA）测量。

Other endpoints include mean change in central retinal thickness (CRT) and number of anti-vascular endothelial growth factor (anti-VEGF) intravitreal injections received following administration of ABBV-RGX-314..

其他终点包括视网膜中央厚度（CRT）的平均变化和服用ABBV-RGX-314后接受的抗血管内皮生长因子（抗VEGF）玻璃体内注射的数量。。

As of November 6, 2023, ABBV-RGX-314 suprachoroidal delivery was well tolerated across 106 patients from three dose levels. No drug-related SAEs were reported. All treatment emergent adverse events (TEAEs) through 6 months in the study eye were mild or moderate and included conjunctival hemorrhage, increased intraocular pressure, episcleritis, and conjunctival hyperemia.

截至2023年11月6日，来自三个剂量水平的106名患者对ABBV-RGX-314脉络膜上递送具有良好的耐受性。没有药物相关SAE的报道。研究眼中6个月内的所有治疗紧急不良事件（TEAE）均为轻度或中度，包括结膜出血，眼压升高，巩膜炎和结膜充血。

Mild intraocular inflammation was reported at similar incidence rate in the first and second dose levels, with mild to moderate intraocular inflammation seen at the third dose level in Cohort 4 and 5. All intraocular inflammation resolved with topical corticosteroids. Notably, there were zero cases of intraocular inflammation in dose level 3, Cohort 6 (n=21), in which patients received a short-course of prophylactic topical steroids following administration of ABBV-RGX-314..

据报道，在第一和第二剂量水平，轻度眼内炎症的发生率相似，在队列4和5的第三剂量水平，轻度至中度眼内炎症可见。所有眼内炎症均用局部皮质类固醇解决。值得注意的是，在剂量水平为3的队列6（n=21）中，没有发生眼内炎症的病例，其中患者在服用ABBV-RGX-314后接受了短期预防性局部类固醇治疗。。

Patients treated with ABBV-RGX-314 continue to demonstrate stable BCVA and CRT at six months. In addition, a meaningful reduction in anti-VEGF treatment burden was observed following administration of ABBV-RGX-314, The highest reduction was seen in dose level 3, demonstrating an 80% reduction in annualized injection rate with 50% of patients remaining injection-free..

接受ABBV-RGX-314治疗的患者在六个月时继续表现出稳定的BCVA和CRT。此外，在服用ABBV-RGX-314后，观察到抗VEGF治疗负担显着降低，剂量水平3的降低幅度最高，表明年注射率降低了80%，50%的患者保持无注射。。

Data presented is available on the 'Publications' section of the REGENXBIO website at www.regenxbio.com.

提供的数据可在REGENXBIO网站的“出版物”部分获得，网址为www.REGENXBIO.com。

Conference CallIn connection with this announcement, REGENXBIO will host a conference call to discuss the Phase II AAVIATE trial data with Dr. Pitcher and Allen Ho, M.D., FACS, FASRS, Co-Director, Wills Eye Retina Service and Director, Retina Research, on Tuesday, January 16 at 4:30 p.m. EST. Listeners can register for the webcast via this link.

电话会议联系此次宣布，REGENXBIO将于美国东部时间1月16日星期二下午4:30与Pitcher博士和医学博士Allen Ho，FACS，FASRS，Wills Eye Retina Service联合总监兼Retina Research总监举行电话会议，讨论第二阶段AAVIATE试验数据。听众可以通过此链接注册网络广播。

Analysts wishing to participate in the question and answer session should use this link. A replay of the webcast will be available via the company's investor website approximately two hours after the call's conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time..

希望参加问答环节的分析师应使用此链接。通话结束大约两小时后，将通过该公司的投资者网站重播网络广播。建议计划参加的人在开始时间前15分钟加入。。

About the AAVIATE® TrialThe multi-center, open-label, randomized, active-controlled, dose-escalation Phase II AAVIATE trial is evaluating the efficacy, safety and tolerability of suprachoroidal delivery of ABBV-RGX-314 in patients with wet AMD using the Clearside SCS Microinjector®. Patients have been dosed across three dose levels: 2.5x1011 (Cohort 1), 5x1011 (Cohorts 2 and 3), and 1x1012 (Cohorts 4-6) genomic copies per eye (GC/eye).

关于AAVIATE®试验多中心，开放标签，随机，主动控制，剂量递增II期AAVIATE试验正在评估使用Clearside SCS Microinjector®在湿性AMD患者中脉络膜上递送ABBV-RGX-314的疗效，安全性和耐受性。患者服用了三种剂量水平：每只眼睛（GC/眼睛）2.5x1011（队列1），5x1011（队列2和3）和1x1012（队列4-6）基因组拷贝。

Cohort 5 is evaluating ABBV-RGX-314 in 20 patients who are NAb positive. Cohort 6 includes a short course of prophylactic steroids following administration of ABBV-RGX-314..

队列5正在评估20名NAb阳性患者的ABBV-RGX-314。队列6包括服用ABBV-RGX-314后的短期预防性类固醇。。

About ABBV-RGX-314ABBV-RGX-314, being developed in collaboration with AbbVie, is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. ABBV-RGX-314 consists of the NAV® AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF).

关于与AbbVie合作开发的ABBV-RGX-314ABBV-RGX-314，正在研究作为湿性AMD，糖尿病视网膜病变和其他慢性视网膜疾病的潜在一次性治疗方法。ABBV-RGX-314由NAV®AAV8载体组成，该载体编码旨在抑制血管内皮生长因子（VEGF）的抗体片段。

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..

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。。

REGENXBIO is advancing research in 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® from Clearside Biomedical, Inc.

REGENXBIO正在通过标准化的视网膜下递送程序以及向脉络膜上空间的递送，推进两种不同途径向眼睛施用ABBV-RGX-314的研究。REGENXBIO已从Clearside Biomedical，Inc.获得SCS Microinjector®的某些专有权。

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

为眼脉络膜上间隙提供基因治疗。。

About Wet AMDWet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. 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.

关于湿性AMD 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。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。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。

These therapies, however, require life-long repeated intraocular injections to maintain efficacy. Due to the burden of treatment, it is difficult for patients to adhere to frequent injections, which can lead to a decline in vision over time..

然而，这些疗法需要终身重复眼内注射才能维持疗效。由于治疗负担，患者很难坚持频繁注射，随着时间的推移，这可能导致视力下降。。

About REGENXBIO Inc.REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8 and AAV9.

关于REGENXBIO Inc.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。REGENXBIO的NAV技术平台是一种专有的腺相关病毒（AAV）基因传递平台，由100多种新型AAV载体的专有权组成，包括AAV7，AAV8和AAV9。

REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a '5x'25' strategy to progress five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025..

REGENXBIO及其第三方导航技术平台许可证持有人正在将导航技术平台应用于开发多个治疗领域的广泛候选项目，包括后期和商业项目。REGENXBIO致力于“5x'25”战略，到2025年，将五种AAV疗法从我们的内部管道和许可计划发展为关键阶段或商业产品。。

Forward-Looking StatementsThis 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.

前瞻性声明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 and clinical trials. 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未来运营和临床试验有关的声明。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 in the busine.

然而，实际结果和发展是否符合REGENXBIO的预期和预测，还存在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临床试验的注册时间、开始和完成以及成功与否，REGENXBIO及其开发合作伙伴进行的临床前研究的开始和完成时间以及取得的成功，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和技术知识产权保护的能力，业务趋势和挑战。

SCS Microinjector® is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.

SCS Microinjector®是Clearside Biomedical，Inc.的商标。此处引用的所有其他商标均为REGENXBIO的注册商标。

Contacts:Dana CormackCorporate Communications[email protected]

联系人：Dana CormackCorporate Communications[受电子邮件保护]

Investors:Chris BrinzeyICR Westwicke339-970-2843[email protected]

投资者：Chris BrinzeyCR Westwick339-970-2843〔email protected〕

SOURCE REGENXBIO Inc.

来源REGENXBIO股份有限公司。