Beacon Therapeutics为laru-zova（AGTC-501）获得FDA再生医学高级疗法（RMAT）认证-动脉网

Beacon Therapeutics Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for laru-zova (AGTC-501)

CISION 等信源发布 2025-01-28 21:00

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RMAT designation provides benefits including enhanced communications with FDA throughout development

RMAT指定提供了一些好处，包括在整个开发过程中加强与FDA的沟通

Recognizes preliminary clinical evidence from the Phase 2 DAWN and SKYLINE trials of laru-zova in X-linked retinitis pigmentosa (XLRP)

认识到laru zova在X连锁性视网膜色素变性（XLRP）中的2期DAWN和SKYLINE试验的初步临床证据

Laru-zova also holds Fast Track designation in US, Priority Medicines (PRIME) designation in EU and Innovative Licensing and Access Pathway (ILAP) designation in UK

拉鲁·佐瓦（Laru zova）还拥有美国的快速通道认证、欧盟的优先药物（PRIME）认证以及英国的创新许可和获取途径（ILAP）认证

LONDON

伦敦

and

和

CAMBRIDGE, Mass.

马萨诸塞州剑桥。

Jan. 28, 2025

2025年1月28日

/PRNewswire/ --

美通社

Beacon Therapeutics Holdings Limited

Beacon Therapeutics控股有限公司

('Beacon Therapeutics' or 'the Company'), a leading ophthalmic gene therapy company with a mission to save and restore the vision of patients with blinding retinal diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to laru-zova (laruparetigene zovaparvovec) for the treatment of X-linked retinitis pigmentosa (XLRP)..

（“Beacon Therapeutics”或“the Company”）是一家领先的眼科基因治疗公司，其使命是挽救和恢复致盲性视网膜疾病患者的视力，今天宣布，美国食品和药物管理局（FDA）已授予laru zova（laruparetigene zovaparvovec）再生医学高级疗法（RMAT）称号，用于治疗X连锁性视网膜色素变性（XLRP）。。

RMAT designation was created to expedite the development and review of regenerative medicine therapies that have the potential to address unmet need in serious or life-threatening diseases, based on preliminary clinical evidence. RMAT designation provides benefits including interactive communications with the FDA throughout development, the opportunity to discuss appropriate endpoints to support accelerated approval, and eligibility for expedited programs such as priority review..

根据初步的临床证据，创建RMAT指定是为了加快再生医学疗法的开发和审查，这些疗法有可能解决严重或危及生命的疾病中未满足的需求。。。

The FDA's RMAT designation for laru-zova recognizes preliminary clinical evidence from the Phase 2 DAWN and SKYLINE trials evaluating the efficacy, safety and tolerability of laru-zova in patients with XLRP caused by mutations in the

FDA对laru zova的RMAT指定认可了第二阶段DAWN和SKYLINE试验的初步临床证据，该试验评估了laru zova对XLRP患者的疗效，安全性和耐受性，这些患者是由XLRP突变引起的

RPGR

gene. This evidence includes emerging data from DAWN showing improvements in low luminance visual acuity (LLVA), which provides support for the measure as a clinically meaningful endpoint in XLRP.

基因。这一证据包括来自黎明的新兴数据，显示低亮度视力（LLVA）有所改善，这为该测量作为XLRP的临床意义终点提供了支持。

'The FDA's decision to grant RMAT designation to laru-zova is a significant milestone for the XLRP patient community, and underscores our promising data and the potential for laru-zova to significantly improve the lives of patients who suffer from XLRP,' said

“FDA决定授予laru zova RMAT称号是XLRP患者社区的一个重要里程碑，并强调了我们有希望的数据以及laru zova显着改善XLRP患者生活的潜力，”他说

Lance Baldo

兰斯·巴尔多

, MD, chief executive officer of Beacon Therapeutics. 'We look forward to working closely with the FDA on continued development activities to support an expedited pathway for laru-zova.'

，医学博士，Beacon Therapeutics首席执行官。”我们期待着与FDA密切合作，继续开展开发活动，以支持拉鲁·佐瓦的快速发展。”

Laru-zova also holds several additional regulatory designations, including Fast Track designation from the FDA, PRIME designation from the European Medicines Agency, and ILAP designation from the Medicines and Healthcare products Regulatory Agency in the UK.

拉鲁·佐瓦（Laru zova）还拥有其他几项监管指定，包括美国食品和药物管理局（FDA）的快速通道指定、欧洲药品管理局（European Medicines Agency）的主要指定以及英国药品和保健品监管局（Medicines and Healthcare products Regulation Agency）的ILAP指定。

Beacon Therapeutics recently presented updates on several studies of laru-zova, including positive interim 3-month data in the

Beacon Therapeutics最近介绍了laru zova的几项研究的最新情况，包括

Phase 2 DAWN trial,

第二阶段DAWN试验，

positive interim 24-month data from the

来自

Phase 2 SKYLINE trial

第二阶段SKYLINE试验

, and ongoing enrollment in the

，以及正在进行的注册

Phase 2/3 VISTA trial

VISTA 2/3期试验

About laru-zova

关于laru-zova

Laru-zova is a gene therapy currently being investigated for the treatment of patients with XLRP. Laru-zova expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss, representing a potential best-in-class treatment for progressive vision loss in patients with XLRP..

Laru zova是目前正在研究的一种基因疗法，用于治疗XLRP患者。Laru zova表达全长RPGR蛋白，从而解决了XLRP引起的光感受器损伤的全部补体，包括视杆细胞和视锥细胞的损失，代表了XLRP患者进行性视力丧失的潜在最佳治疗方法。。

About the SKYLINE, DAWN and VISTA Studies

关于天际线、黎明和远景研究

SKYLINE is an ongoing, fully enrolled, Phase 2, randomized, controlled study evaluating the safety, efficacy and tolerability of laru-zova in 14 male patients with XLRP caused by mutations in the

SKYLINE是一项正在进行的，完全登记的2期随机对照研究，评估了laru zova对14名男性XLRP患者的安全性，有效性和耐受性，这些患者是由XLRP突变引起的

RPGR

gene. The study's primary endpoint is the proportion of response by microperimetry between the study and fellow eye at Month 12.

基因。该研究的主要终点是在第12个月时，研究和对侧眼睛之间通过显微视野检查的反应比例。

NCT06333249

DAWN is an ongoing Phase 2, non-randomized, open-label study of laru-zova in the fellow eye of male participants with XLRP who have previously been treated with an AAV vector-based gene therapy delivering the full-length RPGR protein. The objective of DAWN is to assess two different dose levels of laru-zova for efficacy, safety and tolerability in the target population.

DAWN是一项正在进行的laru zova第二阶段非随机开放标签研究，研究对象是XLRP男性参与者，他们之前曾接受过基于AAV载体的基因治疗，可提供全长RPGR蛋白。DAWN的目的是评估两种不同剂量水平的laru zova在目标人群中的疗效，安全性和耐受性。

DAWN is also evaluating the changes in visual function and functional vision, and is the first trial in the laru-zova clinical development program that is collecting and evaluating LLVA data. .

DAWN还正在评估视觉功能和功能视力的变化，这是laru zova临床开发计划中收集和评估LLVA数据的首次试验。。

NCT06275620

型号：NCT06275620

VISTA is a currently enrolling, Phase 2/3, randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety and tolerability of laru-zova in two study groups compared to an untreated control group. VISTA is currently enrolling.

VISTA是一项目前正在进行的2/3期随机，对照，掩蔽，多中心关键研究，评估两个研究组与未治疗对照组相比laru zova的疗效，安全性和耐受性。VISTA当前正在注册。

NCT04850118

编号：NCT04850118

About XLRP

关于XLRP

XLRP is a severe, aggressive, inherited retinal disease that often leads to blindness by middle age, with no treatment options available. XLRP is predominantly caused by mutations in the

XLRP是一种严重的，侵袭性的遗传性视网膜疾病，通常会在中年时导致失明，没有可用的治疗选择。XLRP主要是由突变引起的

retinitis pigmentosa GTPase regulator (RPGR)

色素性视网膜炎GTP酶调节剂（RPGR）

gene. XLRP primarily affects young males, with an estimated prevalence of 1 in 25,000 males in the US,

基因。XLRP主要影响年轻男性，估计在美国每25000名男性中就有1名患病，

Europe

欧洲

and

和

Australia

澳大利亚

having XLRP with

具有XLRP

RPGR

mutations.

突变。

About Beacon Therapeutics

关于Beacon Therapeutics

Beacon Therapeutics is an ophthalmic gene therapy company founded to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. The Company has an established scientific foundation that includes a late-stage development candidate to treat XLRP and two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting an inherited cone-rod dystrophy (CRD)..

Beacon Therapeutics是一家眼科基因治疗公司，旨在挽救和恢复患有一系列导致失明的普遍和罕见视网膜疾病的患者的视力。该公司拥有一个既定的科学基础，其中包括治疗XLRP的晚期开发候选人和两个临床前项目，一个针对干性年龄相关性黄斑变性（AMD），另一个针对遗传性视锥细胞营养不良（CRD）。。

Beacon is supported by funds from Syncona Limited, Forbion, Oxford Science Enterprises, TCGX, Advent Life Sciences and additional investors. Find out more about Beacon Therapeutics at

Beacon得到了Syncona Limited、Forbion、Oxford Science Enterprises、TCGX、Advent Life Sciences和其他投资者的资金支持。有关Beacon Therapeutics的更多信息，请访问

beacontx.com

Contact:

联系人：

info@beacontx.com

Media:

媒体：

beacontherapeutics@edelman.com

SOURCE Beacon Therapeutics

SOURCE Beacon Therapeutics公司

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