Vadadustat

CAMBRIDGE, Mass., March 19, 2026 (GLOBE NEWSWIRE) -- Akebia Therapeutics®, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, today announced that it will host a virtual R&D Day on Thursday, April 2, 2026 at 10:00 AM EDT. To register for the Akebia virtual R&D Day, click here. The event will feature scientific experts James A. Tumlin, MD (NephroNet), V. Michael Holers, MD (University of Colorado Anschutz) and Jonathan Barratt, MD, PhD, FRCP (University of Leicester) who will join management to discuss Akebia’s robust pipeline of clinical stage kidney diseases programs: Praliciguat, a soluble guanylate cyclase stimulator currently in a Phase 2 clinical trial, targeting Focal Segmental Glomerulosclerosis (FSGS). AKB-097, a next-generation tissue-targeted complement inhibitor planned to enter a Phase 2 basket trial in rare kidney diseases, including IgA nephropathy, lupus nephritis and C3 glomerulopathy, in the second half of 2026. AKB-9090, a hypoxia-inducible factor-prolyl hydroxylase (HIF-PH) inhibitor entering a Phase 1 clinical trial in acute kidney injury (AKI) associated with cardiac surgery, in the first half of 2026. Company management and scientific experts will review the unmet need and current treatment landscape for these rare kidney diseases and discuss the mechanisms of action, supporting data and differentiation of the product candidates. A live question and answer session will follow the formal presentation. A webcast of the presentation can be accessed through the “Investors” section of Akebia’s website at https://ir.akebia.com following the event. About James A. Tumlin, MD Dr. James A. Tumlin is a board-certified nephrologist, clinical researcher, and Professor of Medicine with decades of leadership in kidney disease research and therapeutic development. He serves as Professor of Medicine Emory University School Medicine and Founder and President of NephroNet Clinical Trials Consortium, a nationwide network advancing innovation in renal medicine. Over the course of his career, Dr. Tumlin has held several academic appointments while maintaining an active clinical practice focused on complex kidney disease and dialysis care. Dr. Tumlin has led and participated in numerous NIH-funded and industry-sponsored clinical trials spanning acute kidney injury, glomerular disease, and dialysis technologies, collaborating with major pharmaceutical and biotechnology companies to translate scientific discovery into clinical application. A widely published investigator and editorial board member for leading nephrology journals, he is recognized nationally for advancing therapeutic innovation and improving outcomes for patients with renal disease. His work bridges academic medicine, clinical research, and industry partnership, making him a trusted advisor and key contributor to the development of next-generation kidney therapies. About V. Michael Holers, MD Dr. Holers graduated from Purdue University and Washington University School of Medicine. Following an internship and residency at Barnes Hospital, he was a Rheumatology Fellow at the University of Colorado and then a research postdoctoral fellow with John Atkinson at Washington University. Following rising to Associate Professor with tenure at Washington University, Dr. Holers was recruited in 1993 to the University of Colorado to be the first Smyth Professor of Rheumatology, and then he served as Division Head of Rheumatology and the Scoville Professor of Rheumatology. The historical focus of the Holers’ Laboratory research efforts has been on the structure-function relationships and biologic roles of the complement immune system. In that regard, his research group performed molecular genetic studies in which structure-function relationships of murine complement receptors and membrane regulatory proteins were characterized, essential in vivo roles of these proteins in the development of murine models of human disease were demonstrated, and from these studies first-in-class tissue-directed complement therapeutics were developed. Dr. Holers was a co-founder and CSO of Taligen Therapeutics, a complement therapeutics company which was acquired by Alexion in 2011. More recently, he was a co-founder of Q32 Bio. Dr. Holers has served as the President of the International Complement Society and has been elected a member of several honorary societies, including the American Society for Clinical Investigation (ASCI) and the Association of American Physicians (AAP). Recently he received the singular American College of Rheumatology Presidential Gold Medal. About Jonathan Barratt, MD, PhD, FRCP Professor Barratt's research is focused on a bench to bedside approach to improving our understanding of the pathogenesis of IgA nephropathy a common global cause of kidney failure. He is the IgA nephropathy Rare Disease Group lead for the UK National Registry of Rare Kidney Diseases (RaDaR) and Convener of the International IgA Nephropathy Network. Professor Barratt works closely with pharmaceutical companies interested in new treatments for IgA nephropathy, and is the Chief Investigator for a number of international randomised controlled Phase 2 and 3 clinical trials in IgA nephropathy. He was a member of the FDA and American Society of Nephrology Kidney Health Initiative: Identifying Surrogate Endpoints for Clinical Trials in IgA Nephropathy Work group. Professor Barratt is an Editorial Board member for Kidney International and the American Society of Nephrology journals. Professor Barratt is a co-Chair of the UK Glomerulonephritis Clinical Study Group and the IgA nephropathy lead for the KDIGO Clinical Practice Guidelines for Glomerular Diseases. About Akebia Therapeutics Akebia Therapeutics, Inc. is a fully integrated biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease. Akebia was founded in 2007 and is headquartered in Cambridge, Massachusetts. For more information, please visit our website at www.akebia.com, which does not form a part of this release. Forward-Looking Statements Statements in this press release regarding Akebia Therapeutics, Inc.'s ("Akebia's") strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, and include, but are not limited to, statements regarding: Akebia’s plans and expectations with respect to AKB-097, including its planned Phase 2 basket trial in rare kidney disease and the timing thereof; and Akebia’s plans and expectations with respect to AKB-9090, including its planned Phase 1 clinical trial in acute kidney injury and the timing thereof. The terms "intend," "believe," "plan," "goal," "potential," "anticipate, "estimate," "expect," "future," "will," "continue," derivatives of these words, and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties and other factors, including, but not limited to, risks associated with: the potential therapeutic benefits, safety profile, and effectiveness of Vafseo and Akebia’s development candidates; the results of preclinical and clinical research; Akebia’s ability to initiate and enroll patients in its clinical trials; decisions made by health authorities, such as the FDA, with respect to regulatory filings and other interactions; the potential demand and market potential and acceptance of, as well as coverage and reimbursement related to, Vafseo®, including estimates regarding the potential market opportunity; the competitive landscape for Auryxia® and Vafseo, including generic entrants and the timing thereof; the ability of Akebia to attract and retain qualified personnel; Akebia's ability to achieve and maintain profitability and to maintain operating expenses consistent with its operating plan; manufacturing, supply chain and quality matters and any recalls, write-downs, impairments or other related consequences or potential consequences; early termination of any of Akebia's collaborations; and changes in the geopolitical environment and uncertainty surrounding U.S. trade policy on tariffs. Other risks and uncertainties include those identified under the heading "Risk Factors" in Akebia's Annual Report on Form 10-K for the year ended December 31, 2025, and other filings that Akebia may make with the U.S. Securities and Exchange Commission in the future. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and, except as required by law, Akebia does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release. Akebia Therapeutics®, Auryxia® and Vafseo® are registered trademarks of Akebia Therapeutics, Inc. and its affiliates. Akebia Therapeutics Contact Mercedes Carrasco mcarrasco@akebia.com Source: Akebia Therapeutics, Inc.

这项研究很好地展示了如何通过酶工程对蛋白质分子进行巧妙调整，从而通过生物催化合成推动药物制造。 一种在现代医学中反复出现的单一化学结构，是将羰基连接到氮原子上的酰胺键。它们如此普遍，以至于2023年零售额排名前200的小分子药物中，有117种至少含有一个酰胺键。现在，研究人员发现了一种巧妙的新方法，可以重新设计天然酶，从醛和胺等简单化学品构建酰胺。 研究团队选择了一个自然中丰富的酶家族，即醛脱氢酶（aldehyde dehydrogenase），特别是对羟基苯甲醛脱氢酶，它能有效地将醛转化为酸。团队通过两种主要方式改造其酶的内部口袋，将其转变为一种新型催化剂，称为氧化酰胺酶：使其具有疏水性以防止形成不需要的酸，并使其变大以容纳更大、更多样化的化学部分，从而使它们能够结合在一起。 根据发表在《科学》杂志上的结果，该团队能够通过在一个容器中进行的、两步酶级联反应，直接从市售的醇中获得酰胺。这种方法可以为生产五种主要药物分子开辟更绿色的新方法，其中包括伊马替尼的关键成分，伊马替尼是用于治疗慢性髓性白血病和胃肠道间质肿瘤的基本药物。 通往药物分子骨架的捷径 酰胺键出现在许多用于治疗癌症、心脏病和血液疾病的知名药物中。尽管它们很重要，但这些键的典型形成方式远非理想。大多数传统的酰胺键制造方法依赖高活性化学品、高温或有毒金属，这会产生大量废物，并使药物制造的可持续性降低。 改用基于酶的方法使过程环保，但也有其自身的局限性。例如，脂肪酶，一种常用于将酯转化为酰胺的酶，仅对窄范围的底物有活性。其他酶，如连接酶，则需要非常高的能量输入才能正常运作。 该团队通过利用蛋白质工程的力量，将天然酶转化为高效的合成工具，克服了传统化学的限制。 为了实现这一目标，研究人员重新设计了PHBDD的内部口袋。他们移除了酶中亲水的部分，即两个关键位置R166和E259，并用疏水的甲硫氨酸和亮氨酸替换它们。这种改变改变了反应方向，导致酶使用胺而不是水，并形成酰胺键而不是不需要的酸。 像PHBDD这样的ALDH的另一个问题是，许多重要的药物构建模块太大，无法进入其活性位点。经过几次尝试，研究人员发现，通过突变一个特定点可以扩大通道，将入口通道从6.1 Å拓宽到8.1 Å。 研究人员将他们的新型OxiAm酶与醇脱氢酶配对，创建了一个一锅两步法过程，仅使用空气中的氧气驱动反应，将普通醇直接转化为酰胺。然后，他们用这种方法测试了五种重要药物前体的生产：Vadadustat、Imatinib、Lazabemide、Saruparib和Vorinostat。 这项研究很好地展示了如何通过酶工程对蛋白质分子进行巧妙调整，从而通过生物催化合成推动药物制造。 研究人员认为，这种策略为合成结构多样的酰胺提供了一个绿色且多功能的平台，其应用不仅限于医药领域。（生物谷Bioon.com） 参考文献： Lei Gao et al, Engineered aldehyde dehydrogenases for amide bond formation, Science (2026). DOI: 10.1126/science.adw3365.