PRINCETON, N.J. & TOKYO & WALTHAM, Mass.--(BUSINESS WIRE)--Otsuka Pharmaceutical Development & Commercialization, Inc., (OPDC); Otsuka Pharmaceutical, Co. Ltd. (Otsuka); and Visterra, Inc., an Otsuka group company announce that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for the investigational drug sibeprenlimab for the treatment of immunoglobulin A nephropathy (IgAN; Berger’s disease)..

新泽西州普林斯顿和马萨诸塞州东京和沃尔瑟姆（BUSINESS WIRE）--大冢制药开发与商业化公司（OPDC）；大冢制药有限公司（大冢）；大冢集团公司Visterra，Inc.宣布，美国食品和药物管理局（FDA）已批准用于治疗免疫球蛋白A肾病（IgAN；伯格氏病）的研究药物西贝普瑞单抗的突破性治疗指定。。

Sibeprenlimab is a humanized monoclonal antibody that blocks the action of the B-cell growth factor, APRIL (an acronym for a proliferation-inducing ligand), which plays a key role in the development and progression of IgAN.1,2,3 Otsuka and Visterra announced positive results of the Phase 2 ENVISION trial (NCT04287985) for IgAN in November 2023.

Sibeprenlimab是一种人源化单克隆抗体，可阻断B细胞生长因子APRIL（增殖诱导配体的缩写）的作用，APRIL在IgAN的发展和进展中起着关键作用。1,2,3大冢和Visterra于2023年11月宣布了IgAN的2期ENVISION试验（NCT04287985）的阳性结果。

The trial results were also published in The New England Journal of Medicine.1 Breakthrough therapy designation is granted by the FDA for a drug intended to treat a serious condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on at least one clinically significant endpoint.

该试验结果也发表在《新英格兰医学杂志》上。当初步临床证据表明该药物可能在至少一个临床上显示出比现有疗法的显着改善时，FDA批准了一种用于治疗严重疾病的药物的突破性治疗指标。重要的终点。

Breakthrough therapy designation allows the agency to prioritize and focus resources on the most promising products for which no satisfactory treatment option exists..

突破性治疗指定使该机构能够优先考虑并将资源集中在没有令人满意的治疗选择的最有前景的产品上。。

“We are encouraged by the FDA’s decision to grant breakthrough therapy status for the sibeprenlimab program,” said John Kraus, M.D., Ph.D., executive vice president and chief medical officer at Otsuka. “This is an important milestone that recognizes the potential value that this investigational candidate may have in the future for people living with one of the most common causes of kidney failure.

大冢执行副总裁兼首席医疗官、医学博士约翰·克劳斯（JohnKraus）表示：“FDA决定授予西贝普瑞单抗项目突破性治疗地位，我们对此感到鼓舞。“这是一个重要的里程碑，认识到这位研究候选人在未来可能对患有肾衰竭最常见原因之一的人具有潜在价值。

Otsuka and Visterra are committed to advancing the Phase 3 trial for sibeprenlimab and we are deeply appreciative to all of the study participants, their caregivers, and the investigators who have contributed so much to this research.”.

大冢和维斯特拉致力于推进西贝普瑞单抗的3期临床试验，我们非常感谢所有研究参与者，他们的护理人员以及为这项研究做出如此巨大贡献的研究人员。”。

Immunoglobulin A nephropathy is the most common form of primary glomerulonephritis worldwide and is the most common cause of kidney failure in young adults.4,5 The disease is associated with a reduction in life expectancy of 10 years,5 with at least 30 percent of affected patients progressing to kidney failure within 20 to 30 years, despite optimized standard of care therapy.6,7 Current standard of care management is based on renin-angiotensin aldosterone system (RAAS) blockers and adequate blood pressure control, but the risk of kidney failure remains high.8.

免疫球蛋白A肾病是全世界最常见的原发性肾小球肾炎，也是年轻人肾衰竭的最常见原因。4,5该疾病与预期寿命缩短10岁有关，5至少有30%的受影响患者在20至30年内发展为肾衰竭，尽管优化了护理治疗标准[6,7]。目前的护理管理标准是基于肾素-血管紧张素-醛固酮系统（RAAS）阻滞剂和适当的血压控制，但肾衰竭的风险仍然很高。

“Sibeprenlimab is the first of several precision therapy biologics designed and engineered by Visterra to treat chronic kidney disease patients with major unmet medical needs,” said Brian J. G. Pereira, M.D., chief executive officer of Visterra Inc. “The results of the Phase 2 trial in patients with IgA nephropathy have been encouraging, and we look forward to completion of the Phase 3 trial and submission of the Biological Licensing Application (BLA) to the US FDA and other global regulatory authorities.”.

医学博士布莱恩·佩雷拉（BrianJ.G.Pereira）说：“西贝普瑞单抗是Visterra设计和设计的几种精密治疗生物制剂中的第一种，用于治疗主要医疗需求未得到满足的慢性肾病患者。”。，Visterra Inc.首席执行官：“IgA肾病患者的2期试验结果令人鼓舞，我们期待着完成3期试验并向美国FDA和其他全球监管机构提交生物许可申请（BLA）。”。

About Sibeprenlimab

关于Sibepenlimab

Sibeprenlimab (formerly VIS649) is an investigational humanized IgG2 monoclonal antibody that reduces the production of Gd-IgA1 by binding to a specific signaling molecule called APRIL (short for “a proliferation-inducing ligand”), which has been demonstrated to be a driver of IgA and Gd-IgA1 production.

Sibeprenlimab（以前称为VIS649）是一种研究性人源化IgG2单克隆抗体，通过与称为APRIL的特定信号分子（简称“增殖诱导配体”）结合来减少Gd-IgA1的产生，该分子已被证明是IgA和Gd-IgA1产生的驱动因素。

By binding and neutralizing APRIL, sibeprenlimab may reduce the amount of IgA and Gd-IgA1. Lower levels of Gd-IgA1 may then result in reduced auto-antibody production, which in turn may result in fewer immune complexes, decreased immune complex deposits in kidney, and reduced kidney inflammation. By reducing the production of Gd-IgA1, sibeprenlimab is believed to prevent further kidney damage and the progression to end stage kidney disease.1,2,3.

通过结合和中和APRIL，sibeprenimab可以减少IgA和Gd-IgA1的量。较低水平的Gd-IgA1可能导致自身抗体产生减少，进而可能导致免疫复合物减少，肾脏免疫复合物沉积减少，肾脏炎症减少。通过减少Gd-IgA1的产生，sibeprenlimab被认为可以预防进一步的肾损伤和终末期肾病的进展[1,2,3]。

About APRIL (A Proliferation-Inducing Ligand)

关于APRIL（一种增殖诱导配体）

APRIL is a tumor necrosis factor (TNF) superfamily cytokine involved in B-cell signaling. Its function in driving IgA class switch recombination and production and survival of IgA-secreting plasma cells has become increasingly acknowledged as a cytokine likely to play a key role in pathogenesis of IgAN.

APRIL是参与B细胞信号传导的肿瘤坏死因子（TNF）超家族细胞因子。它在驱动IgA类开关重组以及分泌IgA的浆细胞的产生和存活中的功能越来越被认为是一种细胞因子，可能在IgAN的发病机理中起关键作用。

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About Otsuka

关于大冢

Otsuka Pharmaceutical Co., Ltd. is a global healthcare company with the corporate philosophy: Otsuka–people creating new products for better health worldwide. Otsuka researches, develops, manufactures, and markets innovative products, with a focus on pharmaceutical products to meet unmet medical needs and nutraceutical products for the maintenance of everyday health..

大冢制药有限公司是一家全球性的医疗保健公司，其企业理念是：大冢-人们创造新产品以改善全球健康。大冢研究、开发、制造和销售创新产品，重点是满足未满足医疗需求的医药产品和维持日常健康的营养保健产品。。

In pharmaceuticals, Otsuka is a leader in the challenging areas of mental, renal, and cardiovascular health and has additional research programs in oncology and on several under-addressed diseases including tuberculosis, a significant global public health issue. These commitments illustrate how Otsuka is a “big venture” company at heart, applying a youthful spirit of creativity in everything it does..

在制药领域，大冢在精神、肾脏和心血管健康等具有挑战性的领域处于领先地位，并在肿瘤学和包括结核病（一个重要的全球公共卫生问题）在内的一些未得到充分解决的疾病方面拥有额外的研究项目。这些承诺说明大冢在本质上是一家“大风险”公司，在其所做的每件事中都运用了年轻的创造力。。

Otsuka established a presence in the U.S. in 1973 and today its U.S. affiliates include Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC) and Otsuka America Pharmaceutical, Inc. (OAPI). These two companies’ 2,000 employees in the U.S. develop and commercialize medicines in the areas of mental health and nephrology, using cutting-edge technology to address unmet healthcare needs..

大冢于1973年在美国成立了分支机构，如今其美国分支机构包括大冢制药开发与商业化公司（OPDC）和大冢美国制药公司（OAPI）。这两家公司在美国的2000名员工利用尖端技术开发心理健康和肾脏病领域的药物并将其商业化，以解决未满足的医疗保健需求。。

OPDC and OAPI are indirect subsidiaries of Otsuka Pharmaceutical Company, Ltd., which is a subsidiary of Otsuka Holdings Co., Ltd. headquartered in Tokyo, Japan. The Otsuka group of companies employed 47,000 people worldwide and had consolidated sales of approximately USD 13.1 billion in 2022.

OPDC和OAPI是大冢制药有限公司的间接子公司，大冢制药有限公司是大冢控股有限公司的子公司，总部位于日本东京。大冢集团在全球拥有47000名员工，2022年的合并销售额约为131亿美元。

All Otsuka stories start by taking the road less traveled. Learn more about Otsuka in the U.S. at www.otsuka-us.com and connect with us on LinkedIn and Twitter at @OtsukaUS. Otsuka Pharmaceutical Co., Ltd.’s global website is accessible at https://www.otsuka.co.jp/en/.

所有大冢的故事都是从走人迹罕至的路开始的。有关大冢在美国的更多信息，请访问www.Otsuka-us.com，并通过LinkedIn和Twitter@OtsukaUS与我们联系。大冢制药有限公司的全球网站可访问https://www.otsuka.co.jp/en/.

About Visterra, Inc.

关于Visterra，Inc。

Visterra is a clinical-stage biotechnology company and a wholly owned subsidiary of Otsuka America, Inc. committed to developing innovative antibody-based therapies for the treatment of patients with kidney diseases and other hard-to-treat autoimmune diseases.

Visterra是一家临床阶段生物技术公司，是大冢美国公司的全资子公司，致力于开发基于抗体的创新疗法，用于治疗肾脏疾病和其他难以治疗的自身免疫性疾病。

Visterra’s proprietary Hierotope® platform enables the design and engineering of precision biologics-based product candidates that specifically bind to, and modulate, key disease targets that are not adequately addressed by traditional therapeutic approaches. The platform also includes Fc engineering capabilities for half-life extension, bispecific antibodies and antibody-drug conjugates (ADCs)..

Visterra专有的Hierotope®平台能够设计和工程化基于精密生物制剂的候选产品，这些候选产品专门结合并调节传统治疗方法无法充分解决的关键疾病目标。该平台还包括用于延长半衰期的Fc工程能力，双特异性抗体和抗体-药物偶联物（ADC）。。

Visterra’s pipeline includes programs targeting IgA nephropathy and other kidney diseases, immunologically-driven diseases and infectious diseases. For more information, visit www.visterrainc.com.

Visterra的项目包括针对IgA肾病和其他肾脏疾病、免疫驱动疾病和传染病的项目。有关更多信息，请访问www.visterrainc.com。

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