Ono and Vertex have entered into an exclusive collaboration and license agreement for povetacicept in Japan and South Korea

ONO与Vertex就povetacicept在日本和韩国的独家合作和许可协议达成一致。

Povetacicept is a therapeutic in development for multiple serious B cell-mediated diseases including IgA nephropathy and primary membranous nephropathy

Povetacicept 是一种正在开发的治疗多种严重 B 细胞介导疾病的药物，包括 IgA 肾病和原发性膜性肾病。

Povetacicept is a recombinant fusion protein and a dual antagonist of the BAFF and APRIL cytokines

Povetacicept 是一种重组融合蛋白，是 BAFF 和 APRIL 细胞因子的双重拮抗剂。

OSAKA, Japan & BOSTON, MA -- June 23, 2025 -- Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; “Ono”) and Vertex Pharmaceuticals Incorporated (Headquarters: Boston, MA, U.S.; CEO: Reshma Kewalramani, M.D.; “Vertex”) today announced an exclusive collaboration and license agreement for the development and commercialization of Vertex’s povetacicept in Japan and South Korea..

日本大阪 & 美国马萨诸塞州波士顿 -- 2025年6月23日 -- 小野药品工业株式会社（总部：日本大阪；总裁兼首席运营官：泷野俊一；“小野”）与福泰制药公司（总部：美国马萨诸塞州波士顿；首席执行官：Reshma Kewalramani博士；“福泰”）今日宣布达成一项独家合作和许可协议，将在日本和韩国开发并商业化福泰的povetacicept药物。

Povetacicept is a recombinant fusion protein therapeutic and dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines with best-in-class potential being studied for the treatment of immunoglobulin A nephropathy (IgAN), primary membranous nephropathy (pMN) and other serious B cell-mediated diseases..

Povetacicept是一种重组融合蛋白治疗药物，是BAFF（B细胞活化因子）和APRIL（增殖诱导配体）细胞因子的双重拮抗剂，具有同类最佳潜力，正在研究用于治疗免疫球蛋白A肾病（IgAN）、原发性膜性肾病（pMN）和其他严重的B细胞介导疾病。

Under the terms of the agreement, Ono will pay Vertex an upfront payment, as well as certain regulatory and commercial milestone payments and tiered royalties. Ono will utilize its extensive development expertise to help advance Vertex’s clinical trials for povetacicept and will be responsible for obtaining marketing authorizations in Japan and South Korea.

根据协议条款，小野制药将向福泰制药支付首付款，以及一定的监管和商业里程碑付款和阶梯式特许权使用费。小野制药将利用其广泛的开发专业知识，帮助推进福泰制药关于povetacicept的临床试验，并将负责在日本和韩国获得上市许可。

Following approval, Ono will be solely responsible for commercializing povetacicept in these regions. The agreement includes povetacicept for both IgAN and pMN, with the potential to add other indications..

在获得批准后，小野将全权负责在这些地区将povetacicept商业化。该协议包括用于IgAN和pMN的povetacicept，并有可能增加其他适应症。

“Vertex has a strong track record of developing innovative therapies for serious diseases. Through this strategic partnership, we can strengthen our late-stage pipeline in the immunology field, which is a key focus area for Ono,” said Toichi Takino, Representative Director, President and Chief Operating Officer of Ono.

“Vertex在开发严重疾病创新疗法方面有着良好的记录。通过这一战略合作，我们可以加强我们在免疫学领域的后期研发管线，这是Ono的一个重点聚焦领域，”Ono代表董事、总裁兼首席运营官Toichi Takino表示。

“We look forward to collaborating with Vertex to provide this new therapeutic option for patients with IgAN and other autoimmune diseases in Japan and South Korea, and to maximize the value of this treatment.”.

“我们期待与Vertex合作，为日本和韩国的IgAN患者及其他自身免疫疾病患者提供这种新的治疗选择，并最大限度地发挥这一疗法的价值。”

“Ono is a proven leader in Japan and South Korea, bringing established local relationships, infrastructure, and nephrology expertise that make them a perfect partner for Vertex as we look to deliver povetacicept to the thousands of potential patients in these countries,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex.

“小野制药是日本和韩国的知名领导者，带来了成熟的本地关系、基础设施和肾病学专业知识，这使他们成为我们向这些国家成千上万名潜在患者提供povetacicept的理想合作伙伴，”Vertex首席执行官兼总裁Reshma Kewalramani博士表示。

“We are very pleased to partner with Ono and look forward to close collaboration as we continue to advance this potentially best-in-class treatment for IgAN, pMN and other serious B cell-mediated diseases.”.

“我们非常高兴能与ONO合作，并期待在我们继续推进这一潜在的针对IgAN、pMN和其他严重B细胞介导疾病的同类最佳治疗时，保持紧密的合作。”

About Povetacicept

关于Povetacicept

Povetacicept is a recombinant fusion protein therapeutic and a dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines, which play key roles in pathogenesis of multiple autoimmune diseases via their roles in the activation, differentiation, and/or survival of B cells, T cells, and innate immune cells.

Povetacicept是一种重组融合蛋白治疗剂，同时也是BAFF（B细胞活化因子）和APRIL（增殖诱导配体）细胞因子的双重拮抗剂，这些因子通过在B细胞、T细胞和先天免疫细胞的激活、分化和/或存活中发挥作用，在多种自身免疫疾病的发病机制中起关键作用。

Based upon an engineered TACI (transmembrane activator and calcium modulator ligand interactor) domain, povetacicept has higher binding affinity and greater potency in preclinical studies versus other inhibitors of BAFF and/or APRIL alone and has demonstrated potential best-in-class efficacy in a clinical trial in patients with IgA nephropathy and primary membranous nephropathy.

基于工程化的TACI（跨膜激活剂和钙调制配体相互作用因子）结构域，povetacicept在临床前研究中相较于其他单独抑制BAFF和/或APRIL的抑制剂表现出更高的结合亲和力和更强的效力，并在IgA肾病和原发性膜性肾病患者的临床试验中展示了潜在的最佳疗效。

Povetacicept is also in development for multiple serious B cell-mediated diseases including other autoimmune kidney diseases and autoimmune cytopenias..

Povetacicept 还在开发中，用于治疗多种严重的B细胞介导的疾病，包括其他自身免疫性肾病和自身免疫性血细胞减少症。

About IgA Nephropathy (IgAN)

关于IgA肾病（IgAN）

IgAN is a serious, progressive, life-threatening, B cell-mediated chronic kidney disease that is the most common cause of primary (idiopathic) glomerulonephritis, affecting approximately 300,000 people in the United States and Europe. It is estimated that there are approximately 33,000 diagnosed patients in Japan.

IgAN 是一种严重的、进行性的、威胁生命的、B细胞介导的慢性肾病，是原发性（特发性）肾小球肾炎最常见的病因，影响美国和欧洲约30万人。据估计，日本约有33,000名确诊患者。

IgAN results from deposition of circulating immune complexes consisting of immunoglobulins and galactose-deficient immunoglobulin A (Gd-IgA1) in the renal glomerular mesangium, triggering kidney injury and fibrosis. Up to 72% of adult IgAN patients progress to end-stage renal disease within 20 years.

IgAN是由于循环免疫复合物（由免疫球蛋白和半乳糖缺乏的免疫球蛋白A（Gd-IgA1）组成）在肾小球系膜沉积，引发肾脏损伤和纤维化。高达72%的成年IgAN患者在20年内进展为终末期肾病。

There are no approved therapies that specifically target the underlying cause of IgAN..

目前尚无获批的专门针对IgAN根本原因的疗法。

About Primary Membranous Nephropathy (pMN)

关于原发性膜性肾病 (pMN)

Primary membranous nephropathy is a serious, progressive, life-threatening B cell-mediated chronic kidney disease affecting people worldwide, with approximately 150,000 people diagnosed in the U.S. and Europe. It is estimated that there are approximately 6,000 diagnosed patients with pMN in Japan. pMN is a rare glomerular disease that occurs when the body generates an abnormal immune response, including autoantibodies, against proteins that are part of the kidney.

原发性膜性肾病是一种严重的、进展性的、威胁生命的B细胞介导的慢性肾脏疾病，影响着全球人群，其中美国和欧洲约有15万人被诊断出患有此病。据估计，日本大约有6000名确诊的pMN患者。pMN是一种罕见的肾小球疾病，当身体产生异常的免疫反应（包括自身抗体）对抗作为肾脏组成部分的蛋白质时，就会发生这种疾病。

Autoantibodies trigger damage and inflammation, especially within the glomeruli (the parts of the kidney that filter blood), impairing the kidneys’ ability to properly filter waste and fluid, eventually causing progressive loss of kidney function. There are no approved therapies that specifically target the underlying cause of pMN..

自身抗体引发损害和炎症，尤其是在肾小球（肾脏中过滤血液的部分），削弱了肾脏正常过滤废物和液体的能力，最终导致肾功能的逐步丧失。目前尚无获批的疗法能够专门针对原发性膜性肾病 (pMN) 的根本病因。

About RAINIER

关于RAINIER

RAINIER is a global Phase 3 pivotal trial of povetacicept 80 mg vs. placebo on top of standard of care in approximately 480 people with IgAN. The study is designed to have a pre-planned interim analysis evaluating urine protein to creatinine ratio (UPCR) for the povetacicept arm versus placebo after a certain number of patients reach 36 weeks of treatment.

RAINIER 是一项全球 III 期关键试验，针对约 480 名 IgAN 患者，在标准治疗基础上比较 povetacicept 80 mg 与安慰剂的效果。该研究设计包含一项预先计划的中期分析，在一定数量的患者完成 36 周治疗后，评估 povetacicept 组与安慰剂组的尿蛋白肌酐比值 (UPCR)。

If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval in the U.S. Final analysis will occur at two years of treatment, with a primary endpoint of total eGFR slope through Week 104..

如果结果积极，中期分析可能作为Vertex在美国寻求加速批准的依据。最终分析将在治疗两年时进行，主要终点为通过第104周的总eGFR斜率。

The Phase 3 clinical trial is underway in multiple regions, including the U.S., EU and Asia. Specifically, Japanese and South Korean regulatory authorities have approved the Clinical Trial Application (CTA) for RAINIER, where the Phase 3 trial is underway.

第三阶段临床试验正在包括美国、欧盟和亚洲在内的多个地区进行。具体而言，日本和韩国的监管机构已经批准了RAINIER的临床试验申请（CTA），第三阶段试验正在进行中。

About RUBY-3

关于 RUBY-3

RUBY-3 is an ongoing, multiple ascending dose, multi-cohort, open label, Phase 1/2 basket study of povetacicept in autoimmune glomerulonephritis, including IgAN, primary membranous nephropathy, lupus nephritis, and ANCA-associated vasculitis with glomerulonephritis where povetacicept is being administered subcutaneously for up to 104 weeks..

RUBY-3 是一项正在进行的、多剂量递增、多队列、开放标签的 1/2 期篮式研究，评估 Povetacicept 在自身免疫性肾小球肾炎（包括 IgAN、原发性膜性肾病、狼疮性肾炎以及伴有肾小球肾炎的 ANCA 相关性血管炎）中的应用，Povetacicept 通过皮下注射给药，最长持续时间为 104 周。

About Ono Pharmaceutical Co., Ltd

关于小野制药株式会社

Ono Pharmaceutical Co., Ltd. delivers innovative therapies for patients worldwide. Upholding its philosophy of “Dedicated to the Fight against Disease and Pain,” Ono targets areas with unmet medical needs including oncology, immunology, and neurology, and fosters partnerships with academic and biotech organizations to accelerate drug discovery.

日本小野药品工业株式会社为全球患者提供创新疗法。秉承“致力于抗击疾病和痛苦”的理念，小野专注于包括肿瘤学、免疫学和神经学在内的未满足医疗需求的领域，并与学术和生物技术组织建立合作伙伴关系以加速药物研发。

Through its affiliate, Deciphera Pharmaceuticals, Ono is accelerating clinical development and commercial operations in the US and Europe to drive global business expansion and further its commitment to patient care. For more information, please visit the company's website at .

通过其附属公司Deciphera Pharmaceuticals，Ono正在加速在美国和欧洲的临床开发和商业运营，以推动全球业务扩展，并进一步履行对患者护理的承诺。欲了解更多信息，请访问公司网站。

https://www.ono-pharma.com/en

https://www.ono-pharma.com/cn

.

。

About Vertex

关于顶点

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas.

Vertex是一家全球生物技术公司，投资于科学创新，为患有严重疾病和病症的人群创造变革性药物。该公司已获批的疗法涵盖囊性纤维化、镰状细胞病、依赖输血的β-地中海贫血和急性疼痛，并继续在这些领域推进临床和研究项目。

Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1..

Vertex公司还在其他一些严重疾病中拥有强大的临床研究疗法管道，这些疾病涉及多种治疗模式，并且公司对因果人类生物学有深刻见解，包括神经性疼痛、APOL1介导的肾病、IgA肾病、原发性膜性肾病、常染色体显性多囊肾病、1型糖尿病和1型肌强直性营养不良。

Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For.

Vertex成立于1989年，其全球总部位于波士顿，在伦敦设有国际总部。此外，该公司在北美、欧洲、澳大利亚、拉丁美洲和中东地区拥有研发基地和商业办公室。Vertex连续15年被评为《科学》杂志最佳雇主之一，并被《财富》评为100家最佳工作公司之一，始终被公认为行业内的顶级工作场所之一。

For company updates and to learn more about Vertex’s history of innovation, visit .

如需了解公司更新信息以及更多关于 Vertex 创新历史的内容，请访问。

www.vrtx.com

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Ono Forward-Looking Statements

向前看的陈述

In this press release, statements made with respect to current plans, estimates, strategies and beliefs, and other statements that are not historical facts are forward-looking statements about the future performance of the company. These statements are based on current assumptions and beliefs in light of the information currently available and involve known and unknown risks and uncertainties.

在本新闻稿中，关于当前计划、估计、策略和信念的陈述，以及其他非历史事实的陈述，是关于公司未来业绩的前瞻性声明。这些声明基于当前的假设和信念，并考虑了目前可获得的信息，涉及已知和未知的风险与不确定性。

A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in the business environment in the pharmaceutical market and amendments to relevant laws and regulations, (ii) disruptions to product supply due to stagnation or delays in production caused by natural disasters, fires, etc., (iii) the possibility that sales activities for new and existing products may not achieve the expected results, (iv) the emergence of new side effects in post-marketing drugs, and (v) infringements of intellectual property rights by third parties.

可能导致实际结果与前瞻性陈述中讨论的结果存在重大差异的因素包括但不限于：(i) 医药市场商业环境的变化及有关法律法规的修订，(ii) 因自然灾害、火灾等导致生产停滞或延迟而对产品供应造成的中断，(iii) 新产品和现有产品的销售活动可能无法达到预期效果的可能性，(iv) 上市后药物出现新的副作用，以及 (v) 第三方对知识产权的侵权行为。

Information about pharmaceutical products included in this press release is not intended to constitute an advertisement or medical advice..

本新闻稿中包含的有关药品的信息并不构成广告或医疗建议。

Vertex Forward-Looking Statements

顶点前瞻性声明

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Reshma Kewalramani M.D., and Toichi Takino, in this press release, and statements about the terms of and expectations for Vertex’s collaboration with Ono, statements about potential benefits and results that may be achieved through the collaboration, statements regarding the future activities of the parties pursuant to the collaboration, including Ono’s help to advance clinical trials and Ono’s responsibility to obtain marketing authorizations in Japan and South Korea and to commercialize povetacicept in the regions, statements regarding upfront and milestone payments, and potential royalties on future products, and statements about Vertex’s plans and expectations for the RAINIER and RUBY-3 clinical trials and potential plans to seek accelerated approval in the U.S.

本新闻稿包含1995年《私人证券诉讼改革法案》（经修订）定义的前瞻性声明，包括但不限于Reshma Kewalramani博士和Toichi Takino在本新闻稿中的声明，以及关于Vertex与Ono合作条款及预期的声明、通过合作可能实现的潜在利益和结果的声明、关于各方未来活动的声明，包括Ono协助推进临床试验、Ono负责在日本和韩国获得市场授权并在该地区商业化povetacicept的声明、关于预付款和里程碑付款以及未来产品潜在版税的声明，以及Vertex对RAINIER和RUBY-3临床试验的计划与预期，以及在美国寻求加速批准的潜在计划的声明。

based on interim analysis from the RAINIER trial. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company’s beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements.

基于RAINIER试验的中期分析。虽然Vertex认为本新闻稿中包含的前瞻性陈述是准确的，但这些前瞻性陈述仅代表公司截至本新闻稿发布日期的观点，并且存在许多可能导致实际事件或结果与这些前瞻性陈述所表达或暗示的内容存在重大差异的风险和不确定性。

Those risks and uncertainties include, among other things, that the anticipated benefits and potential of the collaboration between Vertex and Ono may not be achieved on the anticipated timeline, or at all, that data may not support further development of the therapies subject to the collaboration due to safety, efficacy, or other reasons, and other risks listed under the heading “Risk Factors” in Verte.

这些风险和不确定性包括但不限于：Vertex与Ono之间的合作可能无法在预期的时间内实现预期的利益和潜力，或者根本无法实现；由于安全性、有效性或其他原因，数据可能不支持进一步开发合作涉及的疗法；以及Vertex公司“风险因素”标题下列出的其他风险。

www.vrtx.com

www.vrtx.com

and on the SEC’s website at

并在SEC的网站上

www.sec.gov

www.sec.gov

. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

您不应过分依赖这些声明。Vertex不对因新信息出现而更新本新闻稿中包含的信息承担任何义务。