– Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, “Astellas”) today announced the submission of a New Drug Application (NDA) to Japan’s Ministry of Health, Labour and Welfare (MHLW) for Conditional Approval of avacincaptad pegol intravitreal solution (ACP), a synthetic aptamer that inhibits the complement C5 protein, for the treatment of GA secondary to AMD.

– 安斯泰来制药公司（TSE: 4503，总裁兼首席执行官：冈村直树，“安斯泰来”）今日宣布，已向日本厚生劳动省（MHLW）提交了一份新药申请（NDA），申请有条件批准avacincaptad pegol玻璃体内注射液（ACP），这是一种抑制补体C5蛋白的合成适配体，用于治疗继发于AMD的地图样萎缩（GA）。

If approved, ACP has the potential to become the first and only GA treatment available in Japan..

如果获得批准，ACP有望成为日本首个也是唯一可用的GA治疗方法。

GA is a progressive form of AMD that can cause irreversible vision loss, with no treatments currently approved outside the US or Australia.

GA是一种进展性的AMD（年龄相关性黄斑变性），可能导致不可逆的视力丧失，目前在美国或澳大利亚以外尚无获批的治疗方法。

Globally, over five million people are estimated to have GA and, without timely treatment, an estimated 66% of people living with GA may become legally blind or severely visually impaired.

全球估计有超过500万人患有GA，如果不及时治疗，约66%的GA患者可能会成为法定盲人或严重视力受损。

As a result, GA secondary to AMD has a substantial impact on patients’ daily lives and psychological wellbeing.

因此，继发于AMD的GA对患者的日常生活和心理健康产生了重大影响。

Marci English, Vice President, Head of BioPharma and Ophthalmology Development, Astellas

安斯泰来生物制药和眼科开发副总裁 Marci English

“Today’s submission comes as good news to people in Japan living with geographic atrophy who have no approved treatment options for this devastating disease. If approved, avacincaptad pegol has the potential to be the first and only treatment to slow disease progression for eligible patients in Japan.

“今天的提交对日本患有地理萎缩的患者来说是个好消息，因为目前对于这种毁灭性疾病尚无获批的治疗方案。如果获得批准，avacincaptad pegol有望成为日本符合条件的患者中首个也是唯一一个能够减缓疾病进展的治疗方法。”

As such, we are committed to working with regulatory authorities in Japan to ensure that patients can benefit from this vital new treatment.”.

因此，我们致力于与日本监管机构合作，确保患者能够从这一重要的新疗法中获益。

The NDA submission is based on results of overseas clinical trials, including the GATHER1 and GATHER2 randomized, sham-controlled clinical trials, which evaluated the safety and efficacy of monthly 2mg intravitreal administration of ACP in patients with GA secondary to AMD.

NDA提交基于海外临床试验的结果，包括GATHER1和GATHER2随机、假对照临床试验，这些试验评估了每月2mg玻璃体内注射ACP对继发于AMD的地理萎缩患者的安全性和有效性。

The data from both trials demonstrates that ACP slows GA lesion growth and has a favorable safety profile.

两项试验的数据表明，ACP减缓了GA病变的增长，并具有良好的安全性。

Sustained efficacy of ACP, as shown in the restriction of lesion growth over time, was observed over a follow up of two years in GATHER1 and GATHER2 studies.

在GATHER1和GATHER2研究中，通过两年的随访观察到ACP的持续疗效，表现为随时间推移对病变生长的限制。

This submission will have no impact on the financial forecasts of the current fiscal year ending March 31, 2025.

本次提交对截至2025年3月31日的本财年财务预测无影响。

About avacincaptad pegol

关于阿瓦西普他培

Avacincaptad pegol (ACP) is an investigational drug for treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) that has been submitted for Conditional Approval in Japan. ACP is approved in the U.S. as IZERVAY for the treatment of GA secondary to AMD.

Avacincaptad pegol (ACP) 是一种研究性药物，用于治疗继发于年龄相关性黄斑变性（AMD）的地图样萎缩（GA），已提交日本进行有条件批准。ACP 在美国作为 IZERVAY 获批用于治疗继发于 AMD 的 GA。

ACP is a synthetic aptamer that inhibits the complement C5 protein.

ACP是一种抑制补体C5蛋白的合成适配体。

Overactivity of the complement system and the C5 protein play a critical role in the development and growth of scarring and vision loss associated with GA secondary to AMD.

补体系统和C5蛋白的过度活跃在与AMD继发的GA相关的瘢痕形成和视力丧失的发展和增长中起着关键作用。

By targeting C5, ACP is considered to decrease activity of the complement system known to cause the degeneration of retinal cells and thus slow the progression of GA.

通过靶向C5，ACP被认为可以减少已知导致视网膜细胞退化的补体系统活性，从而减缓GA的进展。

About Geographic Atrophy

关于地理性萎缩

Age-related macular degeneration (AMD) is the major cause of moderate and severe loss of central vision in aging adults, affecting both eyes in the majority of patients.

年龄相关性黄斑变性（AMD）是导致中老年人中度和重度中央视力丧失的主要原因，大多数患者双眼均会受到影响。

The macula is a small area in the central portion of the retina responsible for central vision. As AMD progresses, the loss of retinal cells and the underlying blood vessels in the macula results in marked thinning and/or atrophy of retinal tissue.

黄斑是视网膜中央的一小块区域，负责中央视力。随着年龄相关性黄斑变性（AMD）的进展，黄斑中的视网膜细胞和底层血管的丧失导致视网膜组织显著变薄和/或萎缩。

Geographic atrophy (GA), associated with AMD, leads to further irreversible loss of vision in these patients.

地理萎缩（GA）与AMD相关，会导致这些患者的视力进一步不可逆地丧失。

About the GATHER2 Clinical Trial

关于GATHER2临床试验

GATHER2 (NCT04435366) was a randomized, double-masked, sham-controlled, multicenter Phase 3 clinical trial to evaluate the safety and efficacy of intravitreal administration of avacincaptad pegol (ACP) in 448 enrolled patients with GA secondary to AMD.

GATHER2（NCT04435366）是一项随机、双盲、假对照、多中心的3期临床试验，旨在评估玻璃体内注射阿维卡普塔德聚乙二醇（ACP）在448名因年龄相关性黄斑变性（AMD）继发的地理萎缩（GA）患者中的安全性和有效性。

ACP met its primary objective at 12 months, for which patients were randomized to receive either ACP or sham procedure monthly.

ACP在12个月时达到了其主要目标，患者被随机分配每月接受ACP或假手术。

In year 2 of the study, patients treated with ACP in year 1 were re-randomized to receive either ACP dosed monthly (EM, n=96) or every other month (EOM, n=93); patients who received sham in year 1 continued to receive sham in year 2 (n=203).

在研究第2年，第1年接受ACP治疗的患者被重新随机分配，每月接受一次ACP剂量（EM，n=96）或每两个月接受一次（EOM，n=93）；第1年接受假治疗的患者在第2年继续接受假治疗（n=203）。

IZERVAY is continuing to be evaluated in an open-label extension study.

IZERVAY正在一项开放标签扩展研究中继续接受评估。

About Astellas

关于安斯泰来

Astellas is a global life sciences company committed to turning innovative science into VALUE for patients. We provide transformative therapies in disease areas that include oncology, ophthalmology, urology, immunology and women's health. Through our research and development programs, we are pioneering new healthcare solutions for diseases with high unmet medical need.

安斯泰来是一家全球生命科学公司，致力于将创新科学转化为患者的价值。我们在肿瘤学、眼科、泌尿科、免疫学和女性健康等疾病领域提供变革性疗法。通过我们的研发项目，我们正在为医疗需求高度未满足的疾病开创新的医疗解决方案。