Biohaven在研发日强调了其MoDE和TRAP降解平台的创新与进展，宣布了TRAP降解剂的积极数据，实现了超过80%的半乳糖缺乏IgA1（Gd-IgA1）持续降低，并介绍了潜在的同类首创药物BHV-1400用于治疗IgA肾病（IgAN）-动脉网

Biohaven Highlights Innovation and Advancement Across MoDE and TRAP Degrader Platform at R&D Day, Announcing Positive TRAP Degrader Data Achieving > 80% Sustained Reductions in Galactose-Deficient IgA1 (Gd-IgA1) with Potential First-in-Class BHV-1400 for I

CISION 等信源发布 2025-05-29 03:00

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可切换为仅中文

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Optimized subcutaneous (SC) administration of BHV-1400 achieved rapid, deep, selective, and sustained lowering of Gd-IgA1, differentiating Biohaven's leading TRAP degrader for IgAN from the complement and BLyS/APRIL inhibitor competition.

优化的BHV-1400皮下（SC）给药实现了快速、深度、选择性和持续的Gd-IgA1降低，使Biohaven领先的IgAN TRAP降解剂区别于补体和BLyS/APRIL抑制剂竞争产品。

Up to 81% reduction of Gd-IgA1 was observed, with reductions from baseline sustained for weeks after a single SC dose administration.

观察到Gd-IgA1最多减少了81%，单次皮下注射后的数周内，减幅持续保持。

BHV-1400 brings precision immunology to the treatment landscape of IgAN as it was rationally designed to selectively remove galactose-deficient IgA1 (Gd-IgA1), the pathogenic antibody driver of the disease while sparing healthy antibodies IgA, IgG, IgE, and IgM.

BHV-1400将精准免疫学引入IgAN的治疗领域，因为它经过合理设计，能够选择性去除缺乏半乳糖的IgA1（Gd-IgA1），这种致病抗体是该疾病的关键驱动因素，同时保留健康的抗体IgA、IgG、IgE和IgM。

Preservation of immunoglobulins, the complement system, and cell-mediated and humoral immunity offers key differentiation against immunosuppressive BLyS/APRIL inhibitors, complement inhibitors, and budesonide.

保留免疫球蛋白、补体系统以及细胞介导和体液免疫，提供了与免疫抑制性BLyS/APRIL抑制剂、补体抑制剂和布地奈德的关键区别。

Based upon the rapid and deep reductions of Gd-IgA1 observed with subcutaneous BHV-1400, Biohaven plans to study BHV-1400 in patients with IgAN, initiating a pivotal trial in 2026 using urine protein-creatinine ratio (UPCR) as a surrogate endpoint for accelerated approval.

基于皮下注射BHV-1400后观察到的Gd-IgA1快速且显著的减少，Biohaven计划在IgAN患者中研究BHV-1400，并于2026年启动一项关键试验，使用尿蛋白肌酐比值（UPCR）作为加速审批的替代终点。

Optimized SC administration of BHV-1300 has continued to demonstrate rapid, deep, and sustained lowering of total IgG in Phase 1 studies.

优化的BHV-1300皮下注射在1期研究中继续显示出总IgG的快速、深度和持续降低。

Recently generated clinical data shows that BHV-1300 achieved rapid, deep, and sustained lowering of total IgG, with reductions up to 87%.

最近产生的临床数据显示，BHV-1300 实现了快速、深度且持续的总 IgG 降低，降幅高达 87%。

Median maximum reductions of 83% were observed within 18 days of the first subcutaneous dose.

在第一次皮下注射后的18天内，观察到中位最大减少了83%。

The range of IgG reductions possible with varying dosing paradigms of BHV-1300 allows for tunable dosing regimens tailored for acute and chronic disease management, with higher doses for acute disease management and lower, less frequent dosing for chronic disease management.

BHV-1300的不同剂量方案可以实现不同程度的IgG降低，从而允许针对急性和慢性疾病管理进行可调的剂量方案，急性疾病管理采用较高剂量，而慢性疾病管理则采用较低且频率较少的剂量。

Based upon the rapid and deep reductions of IgG and favorable tolerability profile observed with BHV-1300, Biohaven plans to study BHV-1300 in patients with Graves' Disease and initiate pivotal trials in 2H 2025.

基于BHV-1300观察到的IgG快速深度降低和良好的耐受性，Biohaven计划在格雷夫斯病患者中研究BHV-1300，并于2025年下半年启动关键试验。

NEW HAVEN, Conn.

纽黑文，康涅狄格州

，

May 28, 2025

2025年5月28日

/PRNewswire/ -- Biohaven Ltd. (NYSE:

/PRNewswire/ -- Biohaven Ltd.（纽约证券交易所：

BHVN

) ('Biohaven'), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases, today highlighted the success of its first MoDE™ and TRAP™ degraders in achieving key target pharmacodynamic endpoints and announced plans to initiate pivotal trials in Graves' Disease and in IgA nephropathy in 2H 2025 and 1H 2026, respectively at Biohaven's 2025 R&D Day, held concurrently with the Yale Innovation Summit in .

）（'Biohaven'），一家全球临床阶段的生物制药公司，专注于发现、开发和商业化改变生命的疗法，以治疗多种罕见和常见疾病，今天重点介绍了其首个MoDE™和TRAP™降解剂在实现关键目标药效学终点方面的成功，并宣布计划分别在2025年下半年和2026年上半年启动针对格雷夫斯病和IgA肾病的关键试验，这是在2025年Biohaven研发日上宣布的，该活动与耶鲁创新峰会同时举行。

New Haven, Connecticut

康涅狄格州纽黑文市

. The presentation slides from Biohaven's R&D day for the TRAP and MoDE degraders and its other platforms will be available on the

Biohaven公司研发日关于TRAP和MoDE降解剂及其他平台的演示幻灯片将在

Events and Presentations page

事件和展示页面

of the Biohaven website just prior to their presentations.

Biohaven网站在他们演讲之前。

继续阅读

Figure 1

图1

Figure 2

图2

BHV-1400, the Company's potential first-in-class galactose-deficient IgA1 (Gd-IgA1) TRAP degrader for the treatment of IgA nephropathy (IgAN) achieved deep, rapid, and sustained reductions in Gd-IgA1. In the Phase 1 study, a single dose of BHV-1400 was subcutaneously administered at a dose of 500 mg and achieved rapid, deep and sustained reductions in Gd-IgA1 of up to 81%, with a median reduction of 66% (Figure 1).

BHV-1400 是公司潜在的首创半乳糖缺乏型 IgA1（Gd-IgA1）TRAP 降解剂，用于治疗 IgA 肾病（IgAN），实现了深度、快速且持续的 Gd-IgA1 降低。在 1 期研究中，单剂量 500 mg 的 BHV-1400 通过皮下注射给药，实现了高达 81% 的快速、深度和持续的 Gd-IgA1 降低，中位降低幅度为 66%（图 1）。

Reductions occurred within hours of each dose, were progressive, and were sustained for weeks after a single dose administration. Effects were selective, with no significant reductions observed in other immunoglobulins: IgA, IgG, IgE, or IgM..

每次给药后数小时内即出现减少，且呈渐进式，并在单次给药后持续数周。效果具有选择性，其他免疫球蛋白（IgA、IgG、IgE 或 IgM）未观察到显著减少。

Dr.

博士

Jonathan Barratt

乔纳森·巴雷特

, the Mayer Professor of Renal Medicine at University of Leicester and leading expert in the treatment of IgAN, commented on the new Phase 1 data, 'The reason I am excited about BHV-1400 is because it specifically targets the fundamental abnormality in IgA nephropathy while leaving the rest of the immune system untouched.

莱斯特大学肾病医学教授、IgAN治疗领域的权威专家Mayer评论了新的第一阶段数据：“我对BHV-1400感到兴奋的原因是它专门针对IgA肾病的根本异常，而不会影响免疫系统的其他部分。”

It has the potential to take away the major driver for immune complex formation while leaving other antibodies completely unaffected, which means it has efficacy with unrivaled safety.'.

它有可能在不影响其他抗体的情况下消除免疫复合物形成的主要驱动因素，这意味着它具有无与伦比的安全性和有效性。

IgA nephropathy is the leading cause of glomerular disease globally and is commonly diagnosed in individuals in their second and third decades of life, with most individuals progressing to renal failure over the ensuing 10-15 years. As a disease of the immune system, IgA nephropathy frequently returns even after renal transplant.

IgA肾病是全球肾小球疾病的首要病因，常在青少年和年轻成人中确诊，多数患者在随后的10至15年内进展为肾衰竭。作为一种免疫系统疾病，IgA肾病即使在肾移植后也经常复发。

While the 2021 Kidney Disease Improving Global Outcomes (KDIGO) treatment guidelines recommended only standard chronic kidney disease treatments, the 2024 draft guidelines emphasize the importance of treating the underlying immune disease by removing aberrant forms of IgA. 'Galactose deficient IgA1 is the fundamental abnormality in IgA nephropathy,' Dr.

2021年的《改善全球肾脏病预后组织（KDIGO）治疗指南》仅推荐了标准的慢性肾病治疗方法，而2024年草案指南则强调通过去除异常形式的IgA来治疗潜在的免疫疾病的重要性。Dr. 表示：“缺乏半乳糖的IgA1是IgA肾病的基本异常。”

Barratt explained, 'It's a group of IgA molecules that have changes to the sugars on the IgA1 hinge region that fundamentally change the way this antibody behaves. It promotes immune complex formation and it's these immune complexes that cause glomerular injury and damage and promote loss of kidney function.'.

巴雷特解释说：“这是一组IgA分子，它们在IgA1铰链区的糖分发生了变化，这些变化从根本上改变了这种抗体的行为。它促进免疫复合物的形成，正是这些免疫复合物导致肾小球损伤和破坏，并促使肾功能丧失。”

BHV-1400's selective approach has the potential to offer an improved safety profile compared to broadly immunosuppressive agents. BHV-1400 has been safe and well-tolerated across the ongoing Phase 1 study. Most adverse events (AEs) were mild and self-resolving, there were no discontinuations due to AEs related to study drug, and there were no serious or severe AEs related to drug.

BHV-1400的选择性方法相比广谱免疫抑制剂有可能提供更高的安全性。在正在进行的1期研究中，BHV-1400已被证明安全且耐受性良好。大多数不良事件（AEs）为轻度并能自行缓解，没有因与研究药物相关的AE导致的退出，也没有发生与药物相关的严重或重度AE。

There were no clinically significant increases in ALT, AST or bilirubin, no clinically significant reductions in albumin and no clinically significant increases in cholesterol relative to placebo over the 4-week dosing period. There were no clinically significant reductions in other immunoglobulins including IgG, IgA, IgE, or IgM relative to baseline..

在为期4周的给药期间，ALT、AST或胆红素没有临床显著升高，白蛋白没有临床显著降低，胆固醇相对于安慰剂没有临床显著升高。与其他免疫球蛋白（包括IgG、IgA、IgE或IgM）相比，相对于基线没有临床显著降低。

With regards to the applicability of Phase 1 data in healthy volunteers to patients with IgAN, Dr. Barratt added, 'It's not that the Gd-IgA1 is subtly different, it's the same in both (healthy volunteers and patients with IgAN), but in patients, they just have an excess quantity. And so, what I believe is that because you're seeing a suppressed Gd-IgA1 in healthy subjects you are going to see the same in patients with IgA nephropathy, and indeed that's what some of the drugs targeting BAFF and APRIL have shown.

关于第一阶段健康志愿者数据对IgAN患者的适用性，Barratt博士补充道：“并不是Gd-IgA1有细微的不同，它在健康志愿者和IgAN患者中是相同的，只是患者体内的数量过多。因此，我相信，由于在健康受试者中观察到Gd-IgA1受到抑制，在IgA肾病患者中也会看到同样的效果，而这正是某些针对BAFF和APRIL的药物所显示的结果。”

They presented data in their healthy volunteer population that was precisely replicated when they went in to patients.'.

他们在健康志愿者群体中展示的数据在转向患者时被精确复制。

BHV-1400 fundamentally differentiates from alternative approaches by virtue of its precision. While agents targeting the glucocorticoid receptors may have steroid-like side effects, those targeting complement require vaccination for encapsulated bacterial infections, and B-cell-directing therapies cause reductions in all isotypes of immunoglobulin, potentially increasing long-term infection risk. .

BHV-1400 以其精确性从根本上区别于其他替代方法。虽然针对糖皮质激素受体的药物可能会有类固醇样的副作用，那些针对补体系统的需要对包裹细菌感染进行疫苗接种，而导向 B 细胞的疗法会导致所有类型的免疫球蛋白减少，可能增加长期感染风险。

Based upon the rapid and deep reductions of Gd-IgA1 observed with SC dosing of BHV-1400, Biohaven plans to study BHV-1400 in patients with IgAN and initiate pivotal trials in 2026 using UPCR surrogate endpoint via the accelerated approval pathway.

基于在使用BHV-1400进行皮下注射给药时观察到的Gd-IgA1快速且显著的减少，Biohaven计划在IgAN患者中研究BHV-1400，并计划于2026年通过加速审批途径，使用UPCR替代终点启动关键试验。

At its R&D Day, Biohaven also released new positive data from its completing Phase 1 study of its leading MoDE degrader, BHV-1300, that targets IgG for removal. In the Phase 1 multiple-dose study, SC administered BHV-1300 achieved IgG reductions up to 87%. Median maximum reductions of 83% were achieved within 18 days (Figure 2).

在研发日上，Biohaven还发布了其领先的MoDE降解剂BHV-1300的I期研究完成后的新的积极数据，该药物旨在去除IgG。在I期多剂量研究中，皮下注射的BHV-1300实现了高达87%的IgG减少。在18天内达到了中位最大减少83%（图2）。

Biohaven recently reported the 1000 mg weekly dose achieved rapid, deep and sustained reductions in total IgG of up to 84%, with a median reduction of 80% by Week 4 (Figure 2). Reductions at all doses occurred within hours of administration, were progressive, and effects were durable between dosing intervals.

Biohaven最近报告称，每周1000毫克的剂量在总IgG上实现了快速、深度且持续的降低，最高降幅达84%，到第4周时中位降幅为80%（图2）。所有剂量组的IgG水平均在给药后数小时内开始下降，呈渐进式降低，且在两次给药间隔期间效果持久。

The range of IgG lowering enabled by different dose levels of BHV-1300 offers tunability and flexibility in dosing paradigm, with higher doses planned for management of acute conditions, and lower, less frequent dosing planned for the management of chronic disease..

不同剂量水平的BHV-1300可实现不同程度的IgG降低，这为给药模式提供了可调性和灵活性，高剂量计划用于急性病症的管理，而低剂量、较低频率的给药计划用于慢性病的管理。

BHV-1300 has been safe and well-tolerated in subcutaneous doses up to 2000 mg with no clinically significant increases in ALT, AST, or bilirubin, no clinically significant reductions in albumin, and no clinically significant increases in cholesterol over the four-week dosing period relative to placebo.

BHV-1300在高达2000毫克的皮下注射剂量下已被证明是安全且耐受性良好，与安慰剂相比，在四周的给药期间，ALT、AST或胆红素没有临床显著升高，白蛋白没有临床显著降低，胆固醇没有临床显著升高。

There were no clinically significant reductions in IgG3, IgA, IgE, or IgM relative to baseline. Most AEs were mild and self-resolving, and there were no serious or severe AEs..

与基线相比，IgG3、IgA、IgE 或 IgM 没有临床显著减少。大多数不良事件为轻度且能自行缓解，没有严重或重度不良事件。

BHV-1300 is a potential first-in-class novel small molecule MoDE degrader in development for the treatment of IgG-mediated diseases, such as Graves' Disease. It is designed for self-administration via an easy-to-use and patient-friendly autoinjector. Biohaven plans to study BHV-1300 in patients with Graves' Disease and initiate a pivotal trial in 2H 2025..

BHV-1300 是一种潜在的首创新型小分子MoDE降解剂，正在开发用于治疗IgG介导的疾病，如格雷夫斯病。它被设计为通过易于使用且对患者友好的自动注射器进行自我给药。Biohaven计划在格雷夫斯病患者中研究BHV-1300，并于2025年下半年启动一项关键试验。

Tova Gardin

托娃·加丁

, MD, MPP, Chief Translational Officer at Biohaven, commented, 'Our lead MoDE and TRAP degraders, BHV-1300 and BHV-1400, deliver on the promise of ground-breaking chemistry, demonstrating unrivaled selectivity, and profound depletion of potentially disease-causing proteins. With the selectivity of the platform and the initial profile observed in Phase 1, we aim to bring precision immunology to patients with safe and effective treatments that target the core of disease biology.

医学博士、公共政策硕士、Biohaven首席转化官评论道：“我们的主要MoDE和TRAP降解剂BHV-1300和BHV-1400实现了突破性化学的承诺，展示了无与伦比的选择性和对潜在致病蛋白的深度清除。凭借该平台的选择性以及在第一阶段观察到的初步特征，我们旨在为患者带来精准免疫学治疗，安全有效地针对疾病生物学核心。”

We are incredibly proud of our innovative, agile, and dynamic team that has catalyzed a first of its kind extracellular degrader technology from bench to the clinic. Driven by patient need and leading-edge, mechanistically precise science, Biohaven remains focused on delivering the promise of precision therapies to patients with immune-mediated disease.'.

我们为我们的创新、敏捷和充满活力的团队感到无比自豪，这个团队已经将一种前所未有的细胞外降解技术从实验室成功推进到临床。Biohaven 始终以患者需求为导向，依托前沿且机制精确的科学，致力于为免疫介导疾病的患者实现精准治疗的承诺。

About BHV-1400

关于BHV-1400

BHV-1400 is a potential first-in-class Gd-IgA1 TRAP degrader, rationally designed to leverage the body's natural hepatic clearance mechanisms to selectively target and remove Gd-IgA1, the underlying cause of the IgA nephropathy and IgA vasculitis. BHV-1400 spares IgG, IgA, IgE, and IgM to preserve patient immune protection against bacteria, viruses and parasites.

BHV-1400 是一种潜在的首创 Gd-IgA1 TRAP 降解剂，通过合理设计利用人体天然的肝脏清除机制，选择性靶向并去除 Gd-IgA1——这是导致 IgA 肾病和 IgA 血管炎的根本原因。BHV-1400 不影响 IgG、IgA、IgE 和 IgM，以保留患者对细菌、病毒和寄生虫的免疫保护。

The results of the ongoing Phase 1 study confirm that BHV-1400 produces deep reductions in Gd-IgA1 within hours, is selective, sparing other immunoglobulins, is tunable, and is safe and well-tolerated..

正在进行的1期研究结果证实，BHV-1400 在数小时内显著降低 Gd-IgA1，具有选择性，不影响其他免疫球蛋白，且可调节，并安全且耐受性良好。

About BHV-1300

关于BHV-1300

BHV-1300 is a small molecule and potential first-in-class extracellular IgG degrader, rationally designed to leverage the body's natural hepatic clearance mechanisms to selectively target and remove IgG1, IgG2, and IgG4, the underlying cause of many immune-mediated diseases. BHV-1300 spares IgG3 to preserve patient immune protection against bacteria, viruses and parasites.

BHV-1300是一种小分子，也是潜在的首创类细胞外IgG降解剂，通过合理设计利用人体天然的肝脏清除机制，选择性靶向并去除IgG1、IgG2和IgG4，这些是许多免疫介导疾病的根本原因。BHV-1300保留了IgG3，以维持患者对细菌、病毒和寄生虫的免疫保护。

The results of ongoing Phase 1 study confirm that BHV-1300 produces deep reductions in total IgG, is selective, sparing IgG3, is tunable, and is safe and well-tolerated..

正在进行的1期研究结果证实，BHV-1300可显著减少总IgG，具有选择性，不会影响IgG3，且其效果可调节，安全性和耐受性良好。

About Biohaven

关于Biohaven

Biohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. Biohaven is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven's extensive clinical and preclinical programs include Kv7 ion channel modulation for epilepsy and mood disorders; MoDE™ and TRAP™ extracellular protein degradation for immunological diseases; TRPM3 antagonism for migraine and neuropathic pain; TYK2/JAK1 inhibition for neuroinflammatory disorders; glutamate modulation for OCD and SCA; myostatin inhibition for neuromuscular and metabolic diseases, including SMA and obesity; antibody recruiting bispecific molecules; and antibody drug conjugates for cancer.

Biohaven是一家生物制药公司，专注于在关键治疗领域（包括免疫学、神经科学和肿瘤学）中发现、开发和商业化改变生命的治疗方法。Biohaven依托其经过验证的药物开发经验及多个专有的药物开发平台，正在推进其创新的治疗产品组合。Biohaven广泛的临床和临床前项目包括：Kv7离子通道调节用于癫痫和情绪障碍；MoDE™和TRAP™细胞外蛋白降解技术用于免疫疾病；TRPM3拮抗作用用于偏头痛和神经性疼痛；TYK2/JAK1抑制用于神经炎症性疾病；谷氨酸调节用于强迫症和脊髓小脑共济失调；肌肉生长抑制素抑制用于神经肌肉和代谢疾病，包括脊髓性肌萎缩症和肥胖症；双特异性抗体招募分子；以及用于癌症的抗体药物偶联物。

For more information, visit .

欲了解更多信息，请访问。

www.biohaven.com

。

Forward-looking Statements

前瞻性声明

This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The use of certain words, including 'continue', 'plan', 'will', 'believe', 'may', 'expect', 'potential first-in-class', 'potentially', 'groundbreaking' and similar expressions, is intended to identify forward-looking statements.

本新闻稿包含1995年《私人证券诉讼改革法案》所指的前瞻性陈述。某些词语的使用，包括“继续”、“计划”、“将”、“相信”、“可能”、“预期”、“潜在的首创”、“可能地”、“开创性”及类似表述，旨在识别前瞻性陈述。

Investors are cautioned that any forward-looking statements, including statements regarding the future development, timing and potential marketing approval and commercialization of development candidates, are not guarantees of future performance or results and involve substantial risks and uncertainties.

投资者应注意，任何前瞻性陈述，包括有关未来开发、时机以及开发候选产品潜在的市场批准和商业化的陈述，并不能保证未来的业绩或结果，且涉及重大风险和不确定性。

Actual results, developments and events may differ materially from those in the forward-looking statements as a result of various factors including: the expected timing, commencement and outcomes of Biohaven's planned and ongoing clinical trials, including the studies of BHV-1300 and BHV-1400; the timing of planned interactions and filings with the FDA; the timing and outcome of expected regulatory filings; complying with applicable US regulatory requirements; the potential commercialization of Biohaven's product candidates; and the effectiveness and safety of Biohaven's product candidates.

实际结果、发展和事件可能因多种因素而与前瞻性声明中的内容存在重大差异，这些因素包括：Biohaven计划和正在进行的临床试验的预期时间、启动和结果，包括BHV-1300和BHV-1400的研究；与FDA计划互动和提交文件的时间；预期监管文件的时间和结果；遵守适用的美国监管要求；Biohaven产品候选物的潜在商业化；以及Biohaven产品候选物的有效性和安全性。

Additional important factors to be considered in connection with forward-looking statements are described in Biohaven's filings with the Securities and Exchange Commission, including within the sections titled 'Risk Factors' and 'Management's Discussion and Analysis of Financial Condition and Results of Operations'.

与前瞻性声明相关的其他重要因素在Biohaven向证券交易委员会提交的文件中有所描述，包括标题为“风险因素”和“管理层对财务状况和经营结果的讨论与分析”的部分。

The forward-looking statements are made as of the date of this news release, and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events.

本前瞻性声明是截至本新闻稿发布之日作出的，Biohaven不承担更新任何前瞻性声明的义务，无论是由于新信息还是未来事件。

MoDE and TRAP are trademarks of Biohaven Therapeutics Ltd.

MoDE 和 TRAP 是 Biohaven Therapeutics Ltd. 的商标。

Investor Contact:

投资者联系人:

Jennifer Porcelli

詹妮弗·波切利

Vice President, Investor Relations

副总裁，投资者关系

jennifer.porcelli@biohavenpharma.com

+1 (201) 248-0741

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