拜耳启动Alport综合征患者治疗的IIa期研究-动脉网

MediaBayer starts Phase IIa study for treatment of patients with Alport Syndrome

拜耳等信源发布 2025-12-04 15:40

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December

十二月

2025

08:30 AM

上午 08:30

Europe/Amsterdam

欧洲/阿姆斯特丹

MediaBayer starts Phase IIa study for treatment of patients with Alport Syndrome

拜耳启动Alport综合征患者治疗的IIa期研究

Not intended for UK

不适用于英国

Summary

摘要

BAY 3401016 is an investigational monoclonal antibody with potential to block a protein called Semaphorin 3A (Sema3A), which is thought to be involved in the progression of kidney damage in Alport Syndrome (AS), a rare genetic disorder / Bayer explores opportunity to address high unmet medical need of patients suffering from AS and advances research efforts through close cooperation with AS patient organizations / Derived from the strategic research cooperation with Evotec, advancement of BAY 3401016 into ASSESS Phase IIa clinical study is further strengthening Bayer’s development portfolio / The program has received Fast Track Designation and Orphan Drug Designation from the U.S.

BAY 3401016 是一种研究性单克隆抗体，具有阻断一种名为 Semaphorin 3A（Sema3A）的蛋白质的潜力，该蛋白质被认为与 Alport 综合征（AS）中的肾脏损伤进展有关，Alport 综合征是一种罕见的遗传性疾病 / 拜耳探索满足 AS 患者高度未满足医疗需求的机会，并通过与 AS 患者组织密切合作推进研究工作 / BAY 3401016 源自与 Evotec 的战略研究合作，其进入 ASSESS IIa 期临床研究进一步增强了拜耳的研发产品组合 / 该项目已获得美国授予的快速通道资格和孤儿药资格。

Food and Drug Administration (FDA).

食品和药物管理局（FDA）。

Berlin, December 4, 2025

柏林，2025年12月4日

– Bayer announced today initiation of a Phase IIa clinical trial with BAY 3401016, an investigational monoclonal antibody with potential to block a protein called Semaphorin 3A (Sema3A), which is thought to be involved in the progression of kidney damage in Alport Syndrome (AS), a rare genetic disorder.

– 拜耳今天宣布启动BAY 3401016的IIa期临床试验，这是一种研究性单克隆抗体，具有阻断称为Semaphorin 3A（Sema3A）蛋白的潜力，该蛋白被认为与阿尔波特综合症（AS）中肾脏损伤的进展有关，这是一种罕见的遗传性疾病。

The first-in-patient study, ASSESS, is a randomized, double-blind, placebo-controlled, group-comparison trial (.

首个患者研究 ASSESS 是一项随机、双盲、安慰剂对照的组间比较试验。

NCT07211685

), with an extension phase, which will investigate the efficacy and safety of BAY 3401016 in adult participants with Alport Syndrome.

），包含一个扩展阶段，将研究BAY 3401016在成年Alport综合征患者中的疗效和安全性。

“The initiation of the ASSESS trial represents an important milestone for our investigational BAY 3401016 program,” said Andrea Haegebarth, Ph.D., Global Head of Research and Early Development for Cardiovascular, Renal, and Immunology at Bayer’s Pharmaceuticals Division. “We are collaborating closely with the patient organization community to gain a deeper understanding of the real challenges faced by people living with Alport Syndrome.

“Andrea Haegebarth博士，拜耳制药部门心血管、肾脏和免疫学全球研究与早期开发负责人表示：‘ASSESS试验的启动代表了我们的研究性BAY 3401016项目的一个重要里程碑。’ ‘我们正与患者组织密切合作，以更深入地了解阿尔波特综合征患者面临的真正挑战。’”

We believe BAY 3401016 holds promise as a potential therapeutic approach, and we look forward to assessing its efficacy and safety profile as we advance this important program in our pipeline.”.

我们相信 BAY 3401016 作为一种潜在的治疗方法很有前景，我们期待在推进这一重要项目的过程中评估其疗效和安全性。"

Alport Syndrome is caused by genetic mutations that affect the type IV collagen found in the kidneys, ears, and eyes. It leads to progressive severe proteinuria, loss of kidney function, and early onset of end-stage renal disease. Diagnosis typically occurs in childhood or adulthood (depending on mutation type) via urine tests, kidney biopsy or genetic testing.

Alport综合征是由影响肾脏、耳朵和眼睛中IV型胶原蛋白的基因突变引起的。它会导致进行性严重的蛋白尿、肾功能丧失以及早期终末期肾病的发生。诊断通常在儿童或成年期（取决于突变类型）通过尿液检查、肾活检或基因检测来进行。

Both men and women are affected by Alport Syndrome. Many women may initially have milder symptoms and experience a later onset of disease progression..

Alport综合征会影响男性和女性。许多女性最初可能症状较轻，并且疾病进展的发病时间较晚。

Currently, there is no specific treatment approved for Alport Syndrome and, despite guideline-recommended therapy, patients still experience progressive decline of kidney function resulting in end-stage kidney disease around their fourth decade of life or even earlier.

目前，尚无获批的针对 Alport 综合征的特定治疗方法，尽管有指南推荐的治疗方案，患者仍然会经历肾功能的持续下降，通常在四十岁左右甚至更早发展为终末期肾病。

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The main goal of the ongoing study is to learn how well BAY 3401016 works in slowing down the loss in kidney function in adults with rapidly progressing AS.

正在进行的研究的主要目标是了解 BAY 3401016 在减缓患有快速进展的 AS 的成年人肾功能丧失方面的效果如何。

Investigational BAY 3401016 is derived from Bayer’s strategic research collaboration with Evotec. The program has received Fast Track Designation and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA).

研究性药物BAY 3401016源自拜耳与Evotec的战略研究合作。该计划已获得美国食品药品监督管理局（FDA）的快速通道资格和孤儿药资格。

About Alport Syndrome

关于阿尔波特综合症

Alport Syndrome (AS) is a rare genetic condition that causes kidney disease, hearing loss, and eye abnormalities that occur due to changes in specific genes (COL4A3, COL4A4, and COL4A5) that affect the protein type IV collagen. Type IV collagens are an important component of basement membranes in the kidney, but also other organs, and mutations in these genes result in functional impairment..

Alport综合征（AS）是一种罕见的遗传性疾病，由特定基因（COL4A3、COL4A4和COL4A5）的改变引起，这些基因影响IV型胶原蛋白。IV型胶原蛋白是肾脏和其他器官基底膜的重要组成部分，这些基因的突变会导致功能障碍，并引发肾病、听力损失和眼部异常。

People with AS have a high risk of developing chronic kidney disease (CKD), a condition in which there is progressive loss in kidney function over time resulting in end-stage kidney disease. As a consequence, kidneys have lost their ability to remove waste products from the body properly, leading to the need for kidney replacement therapy, which involves either dialysis or a kidney transplant.

患有 AS 的人患慢性肾病 (CKD) 的风险很高，这是一种肾功能随时间逐渐丧失的疾病，最终会导致终末期肾病。结果，肾脏失去了正确清除体内废物的能力，从而需要进行肾替代治疗，包括透析或肾移植。

A common sign of impaired kidney function is the presence of excess of the protein albumin in the urine that is not usually found with healthy kidneys. This condition is known as albuminuria..

肾功能受损的常见迹象是尿液中存在过量的蛋白质白蛋白，而健康肾脏通常不会出现这种情况。这种状况被称为蛋白尿。

About BAY 3401016

关于BAY 3401016

The study drug BAY 3401016 is a monoclonal antibody that blocks a protein called Semaphorin 3A (Sema3A), which is thought to be involved in the progression of kidney damage in Alport Syndrome (AS). By blocking the action of the Sema3A protein, BAY 3401016 may reduce proteinuria and slow down the loss in kidney function due to AS..

研究药物BAY 3401016是一种单克隆抗体，可阻断一种称为Semaphorin 3A（Sema3A）的蛋白质，该蛋白质被认为与Alport综合征（AS）中肾脏损伤的进展有关。通过阻断Sema3A蛋白的作用，BAY 3401016可能减少蛋白尿并减缓由于AS导致的肾功能丧失。

About the ASSESS study

关于ASSESS研究

The

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本地端口

yndrome

综合征

ema3A

fficacy &

效能与

afety

安全

tudy (ASSESS) is a Phase IIa clinical trial with an investigational drug (BAY 3401016) for patients with Alport Syndrome. The main goal of the ASSESS study is to learn more about how well BAY 3401016 works in slowing down the progression of kidney damage in patients with Alport Syndrome with increased albuminuria. The study will also evaluate the safety of the BAY 3401016 and record any side effects experienced by participants.

ASSESS 研究是一项针对 Alport 综合征患者的 IIa 期临床试验，使用研究性药物 BAY 3401016。ASSESS 研究的主要目标是进一步了解 BAY 3401016 在减缓伴有白蛋白尿增加的 Alport 综合征患者肾脏损伤进展方面的效果。该研究还将评估 BAY 3401016 的安全性，并记录参与者出现的任何副作用。

Further details of the trial can be found at .

有关试验的更多详细信息，请参见。

www.clinicaltrials.gov

(NCT07211685).

(NCT07211685)。

About Bayer

关于拜耳

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population.

拜耳是一家在全球范围内拥有核心竞争力的企业，专注于医疗保健和营养等生命科学领域。秉承“人人健康，无人饥饿”的使命，公司通过产品和服务支持应对全球人口增长和老龄化带来的重大挑战，助力人类与地球共同繁荣。

Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros.

拜耳致力于通过其业务推动可持续发展并产生积极影响。同时，集团旨在通过创新和增长提高盈利能力并创造价值。拜耳品牌在全球范围内代表信任、可靠性和质量。2024财年，集团拥有约93,000名员工，销售额达466亿欧元。

R&D expenses amounted to 6.2 billion euros. For more information, go to .

研发费用达62亿欧元。欲了解更多信息，请访问。

www.bayer.com

。

Find more information at

更多信息请访问

https://pharma.bayer.com

在Facebook上关注我们：

http://www.facebook.com/bayer

Forward-Looking Statements

前瞻性声明

This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here.

本发布可能包含基于拜耳管理层当前假设和预测的前瞻性声明。各种已知和未知的风险、不确定性和其他因素可能导致公司的实际未来结果、财务状况、发展或业绩与这里提供的估计存在重大差异。

These factors include those discussed in Bayer’s public reports which are available on the Bayer website at .

这些因素包括拜耳公开报告中讨论的因素，该报告可在拜耳网站上查阅。

www.bayer.com

. The company assumes no liability whatsoever to update these forward-looking statements or to conform

公司不承担更新这些前瞻性声明或使其符合的任何责任 whatsoever

them to future events or developments.

让他们参与未来的事件或发展。

Chavez E, Goncalves S, Rheault MN, Fornoni A. Alport Syndrome. Adv Kidney Dis Health. 2024 May;31(3):170-179. doi: 10.1053/j.akdh.2024.02.004. PMID: 39004457.

查韦斯 E，贡萨尔维斯 S，雷奥尔特 MN，福尔诺尼 A。阿尔波特综合征。《肾脏疾病与健康进展》。2024年5月；31(3):170-179。doi: 10.1053/j.akdh.2024.02.004。PMID: 39004457。

Torra R, Lipska-Zietkiewicz B, Acke F, Antignac C, Becker JU, Cornec-Le Gall E, van Eerde AM, Feltgen N, Ferrari R, Gale DP, Gear S, Gross O, Haeberle S, Heidet L, Lennon R, Massella L, Pfau K, Pizarro MDPV, Topaloglu R, Wlodkowski T, Zealey H; ERKNet, ERA Genes&Kidney and ESPN Inherited renal disorders working group.

托拉 R、利普斯卡-泽特基维奇 B、阿克 F、安蒂尼 C、贝克尔 JU、科尔内克-勒加尔 E、范埃erde AM、费尔特根 N、法拉利 R、盖尔 DP、吉尔 S、格罗斯 O、海贝尔 S、海德特 L、列侬 R、马塞拉 L、普劳 K、皮萨罗 MDPV、托帕洛格鲁 R、沃洛夫斯基 T、泽利 H；ERKNet，ERA 基因与肾脏及ESPN遗传性肾病工作组。

Diagnosis, management and treatment of the Alport syndrome - 2024 guideline on behalf of ERKNet, ERA and ESPN. Nephrol Dial Transplant. 2025 May 30;40(6):1091-1106. doi: 10.1093/ndt/gfae265. PMID: 39673454; PMCID: PMC12209846..

Alport综合征的诊断、管理和治疗——ERKNet、ERA和ESPN代表发布的2024指南。《肾脏病透析移植》。2025年5月30日；40(6):1091-1106。doi: 10.1093/ndt/gfae265。PMID: 39673454；PMCID: PMC12209846。

Temme J, Peters F, Lange K, Pirson Y, Heidet L, Torra R, Grunfeld JP, Weber M, Licht C, Müller GA, Gross O. Incidence of renal failure and nephroprotection by RAAS inhibition in heterozygous carriers of X-chromosomal and autosomal recessive Alport mutations. Kidney Int. 2012 Apr;81(8):779-83. doi: 10.1038/ki.2011.452.

Temme J、Peters F、Lange K、Pirson Y、Heidet L、Torra R、Grunfeld JP、Weber M、Licht C、Müller GA、Gross O。X染色体和常染色体隐性Alport突变的杂合携带者中肾衰竭的发生率及RAAS抑制对肾脏的保护作用。《国际肾脏杂志》。2012年4月；81（8）：779-83。doi: 10.1038/ki.2011.452。

Epub 2012 Jan 11. PMID: 22237748..

Epub 2012年1月11日。PMID：22237748。

Chavez E, Goncalves S, Rheault MN, Fornoni A. Alport Syndrome. Adv Kidney Dis Health. 2024 May;31(3):170-179. doi: 10.1053/j.akdh.2024.02.004. PMID: 39004457.

Bayer Media Contact:

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Anna Kanmaz, phone +49 1741463541

安娜·坎马兹，电话 +49 1741463541

Email:

电子邮件：

anna.kanmaz@bayer.com

安娜.康马兹@拜耳.com

Contact for media inquiries Bayer U.S.:

媒体查询联系人拜耳美国：

Elaine Colon, phone +1 732 236 1587

伊莱恩·科隆，电话 +1 732 236 1587