Biohaven推进一类新型精准免疫疗法：首款MoDE细胞外蛋白降解剂BHV-1300启动针对格雷夫斯病的III期关键性临床试验-动脉网

Biohaven Advances a New Class of Precision Immunology Therapies: First MoDE Extracellular Protein Degrader, BHV-1300, Begins Phase 3 Pivotal Trial in Graves' Disease

PR Newswire 等信源发布 2026-06-29 19:30

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BHV-1300 is the first MoDE™ extracellular protein degrader to enter a pivotal trial, pioneering a new class of precision immunology medicines that eliminate the disease-driving antibodies at the root of autoimmune disease.

BHV-1300 是首个进入关键性试验的 MoDE™ 细胞外蛋白降解剂，开创了一类新型精准免疫药物，旨在从根源上清除驱动自身免疫疾病的致病抗体。

BHV-1300 targets the TSHR-IgG1 autoantibody that drives Graves' disease, not the thyroid gland itself. This is a fundamental shift from existing treatment approaches.

BHV-1300 靶向驱动格雷夫斯病的 TSHR-IgG1 自身抗体，而非甲状腺本身。这与现有治疗方法相比是一个根本性的转变。

Exclusively licensed from Yale University, Biohaven's BHV-1300's pivotal trial marks the vanguard of a broader platform: multiple next-generation MoDE and TRAP™ extracellular protein degraders advancing across the pipeline to address immunologic disease.

BHV-1300 的关键性试验获得耶鲁大学的独家授权，标志着更广泛平台的前沿进展：多种下一代 MoDE 和 TRAP™ 细胞外蛋白降解剂正在研发管线中推进，以应对免疫性疾病。

NEW HAVEN, Conn.

康涅狄格州纽黑文

，

June 29, 2026

2026年6月29日

/PRNewswire/ -- Biohaven Ltd. (NYSE:

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

BHVN

) today announced enrollment of the first patient in the pivotal Phase 3 trial of BHV-1300 for Graves' disease. BHV-1300 is the first MoDE extracellular protein degrader, a novel small molecule IgG1, 2 and 4 degrader that harnesses the body's own clearance machinery to eliminate the IgG1 TSHR autoantibody driving Graves' disease.

）今日宣布，其针对格雷夫斯病的BHV-1300关键性III期临床试验已完成首例患者入组。BHV-1300是首款MoDE细胞外蛋白降解剂，作为一种新型小分子IgG1、2和4降解剂，它利用人体自身的清除机制来消除驱动格雷夫斯病的IgG1促甲状腺激素受体（TSHR）自身抗体。

In over 70 years, no new therapy has been approved for Graves' disease. BHV-1300 is designed to change the treatment landscape for this autoimmune disease..

70多年来，尚无新的格雷夫斯病疗法获批。BHV-1300旨在改变这种自身免疫性疾病的治疗格局。

Figure 1

图1

Figure 2

图2

Figure 3

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Beth Emerson, MD, MBA, Executive Medical Director at Biohaven and Lead for the Graves' disease clinical trial, commented, 'The enrollment of the first patient in this pivotal trial marks an important moment for the Graves' disease community. For decades, physicians have relied on treatments that either suppress thyroid function or destroy the gland.

Biohaven公司执行医学总监、格雷夫斯病临床试验负责人Beth Emerson医学博士兼工商管理硕士评论道：“这项关键性试验中首位患者的入组，标志着格雷夫斯病社群的一个重要时刻。数十年来，医生们一直依赖那些要么抑制甲状腺功能、要么破坏甲状腺腺体的治疗方法。”

BHV-1300 represents an opportunity to bring forward a disease modifying therapy, targeting the underlying cause of Graves' disease, thyroid eye disease, and pretibial myxedema rather than addressing the downstream complications.'.

BHV-1300 代表了一种带来疾病修饰疗法的机会，该疗法针对格雷夫斯病、甲状腺眼病和胫前黏液性水肿的根本病因，而非仅处理其下游并发症。

BHV-1300 is the lead molecule from Biohaven's MoDE platform — exclusively licensed from Yale University where the technology originated in the Spiegel Lab and advanced by the Biohaven discovery and clinical teams — which directs disease-driving proteins to the body's own natural clearance pathways for selective elimination.

BHV-1300 是 Biohaven 公司 MoDE 平台的核心分子，该平台技术源自耶鲁大学 Spiegel 实验室，并由 Biohaven 的发现与临床团队推进，Biohaven 已获得其独家许可。该技术能够引导致病蛋白进入人体自身的天然清除途径，从而实现选择性消除。

The Phase 3 program is grounded in Phase 1b data demonstrating:.

III期临床试验方案基于Ib期数据，该数据显示：

Greater than 80% reduction of pathogenic TSHR autoantibodies

致病性TSHR自身抗体减少超过80%

Rapid normalization of free T4 and free T3 in patients with Graves' hyperthyroidism

格雷夫斯病甲亢患者游离T4和游离T3的快速正常化

Improvements in hallmark symptoms of Graves' disease

格雷夫斯病典型症状的改善

Preservation of IgG3, IgA, IgM, and IgE

IgG3、IgA、IgM和IgE的保存

Favorable safety and tolerability

良好的安全性和耐受性

BHV-1300 is the first extracellular degrader to reach a pivotal trial, opening the door to an entirely new therapeutic modality in precision immunology.

BHV-1300 是首个进入关键性临床试验的细胞外降解剂，为精准免疫学中一种全新的治疗模式打开了大门。

Graves' disease is the most common cause of hyperthyroidism, driven by a TSHR-IgG1 autoantibody that overstimulates the TSH receptor. It affects ~1% of the global population, yet no new FDA-approved therapy has emerged in over 70 years. Today's standard of care - antithyroid drugs, radioactive iodine, or surgery - targets only the downstream effects, leaving the autoimmune root cause untreated.

格雷夫斯病是甲状腺功能亢进症最常见的原因，由过度刺激促甲状腺激素（TSH）受体的TSHR-IgG1自身抗体驱动。该病影响全球约1%的人口，然而70多年来尚无新的美国食品药品监督管理局（FDA）批准疗法问世。目前的标准治疗方案——抗甲状腺药物、放射性碘或手术——仅针对下游效应，而未对自身免疫这一根本病因进行治疗。

BHV-1300 targets the disease at its source (see Figure 1)..

BHV-1300 从疾病源头进行靶向治疗（见图 1）。

Figure 1:

图1：

Graves' disease represents a large, long-standing unmet need — affecting approximately 1% of the global population with no new FDA-approved therapy in over seventy years and no approved treatment targeting the root cause. BHV-1300 is now in a pivotal trial designed to treat Graves' disease at its source..

格雷夫斯病（Graves' disease）代表了一个巨大且长期未得到满足的医疗需求——它影响着全球约1%的人口，但在过去七十多年中，没有新的疗法获得美国食品药品监督管理局（FDA）批准，也没有针对其根本病因的获批治疗方法。BHV-1300 目前正处于一项关键性临床试验阶段，旨在从源头上治疗格雷夫斯病。

Tova Gardin, MD, MPP, Chief Translational Officer of Biohaven, commented, 'Enrollment of the first patient in our pivotal Graves' disease trial is a landmark moment for patients and for the future treatment of Graves' disease. With a precision therapeutic designed to be self-administered at home in a patient-friendly autoinjector, BHV-1300 integrates groundbreaking high-science with life-centric solutions.'.

Biohaven首席转化官Tova Gardin医学博士、公共政策硕士表示：“我们关键的格雷夫斯病试验中首位患者的入组，对于患者以及格雷夫斯病的未来治疗而言，都是一个里程碑式的时刻。BHV-1300是一种精准疗法，专为患者友好型自动注射器居家自我给药而设计，将前沿的高科技科学与以患者为中心的生活化解决方案融为一体。”

Every generation of science is defined by breakthroughs that change how physicians think about treating disease. Biohaven believes that MoDE and TRAP extracellular protein degraders have the potential to be one of those breakthroughs, opening the door to a new class of medicines designed to treat disease precisely at its core and offering new possibilities and renewed hope to patients who have long awaited options that address the root of their condition, rather than their symptoms alone..

每一代科学进步都由那些改变医生对疾病治疗思维的突破所定义。Biohaven 相信，MoDE 和 TRAP 细胞外蛋白降解剂有望成为此类突破之一，开启一类全新药物的大门，这些药物旨在从疾病的核心进行精准治疗，为长期期待能够针对病因而非仅缓解症状的治疗方案的患者带来新的可能性和 renewed 希望。

Dr. Gardin added, 'We look forward to investigating BHV-1300 for the treatment of Graves' disease and follow-on IgG-mediated indications. This is more than a single trial or a single molecule; it potentially represents a new paradigm of precision immunology, one in which we move beyond managing disease toward precisely eliminating the proteins that drive it, unlocking this potentially groundbreaking platform.'.

加丁博士补充道：“我们期待开展对BHV-1300用于治疗格雷夫斯病及其他后续IgG介导适应症的深入研究。这不仅仅是一项单一试验或一个单一分子，它可能代表着精准免疫学的新范式——在这一范式中，我们不再局限于疾病管理，而是迈向精确清除驱动疾病的蛋白质，从而释放这一潜在突破性平台的巨大潜力。”

Nearly 200 individuals have been dosed with MoDE and TRAP extracellular protein degraders in Phase 1 testing with favorable tolerability to date - most adverse events were mild and self-resolving. In the Graves' Phase 1 expansion, BHV-1300 produced deep, rapid reductions in TSHR-IgG1 (TRAb) and normalized thyroid hormones, directly linking target engagement to clinical response (see Figure 2)..

在I期临床试验中，近200名受试者接受了MoDE和TRAP细胞外蛋白降解剂的给药，迄今为止耐受性良好——大多数不良事件为轻度且可自行缓解。在Graves病的I期扩展研究中，BHV-1300使TSHR-IgG1（TRAb）水平迅速且显著地降低，并使甲状腺激素恢复正常，直接将靶点参与与临床反应联系起来（见图2）。

Figure 2:

图2：

In a Phase 1 patient expansion study, BHV-1300 (1,000 mg SC weekly) rapidly removed more than 80% of disease-driving TSHR autoantibodies (TRAb) — the root cause of Graves' disease — and normalized free T4 and free T3 in patients with Graves' hyperthyroidism. Preliminary data from an ongoing study (n=3)..

在一项I期患者扩展研究中，BHV-1300（每周皮下注射1,000 mg）迅速清除了超过80%的致病性TSHR自身抗体（TRAb）——即格雷夫斯病的根本病因，并使格雷夫斯病甲亢患者的游离T4和游离T3水平恢复正常。来自一项正在进行的研究（n=3）的初步数据..

The pivotal trial (NCT07661056) is a randomized, double-blind, placebo-controlled study evaluating BHV-1300 in approximately 300 adults with Graves' disease. The trial's primary objective is to assess restoration of normal thyroid function at 26 weeks in the absence of an antithyroid drug. BHV-1300 is administered subcutaneously using a patient-friendly autoinjector designed for self-administration at home.

关键性试验（NCT07661056）是一项随机、双盲、安慰剂对照研究，旨在评估BHV-1300在约300名格雷夫斯病成人患者中的疗效。该试验的主要目的是评估在不使用抗甲状腺药物的情况下，于第26周时恢复正常甲状腺功能的程度。BHV-1300通过皮下注射给药，采用专为患者居家自行注射设计的便捷自动注射器。

Additional information is available at .

更多信息可在以下网址获取：

www.clinicaltrials.gov

。

About Graves' Disease

关于格雷夫斯病

Graves' disease is the most common cause of hyperthyroidism, driven by autoantibodies stimulating the TSH receptor. A relapsing and remitting condition, Graves' disease affects 1% of the global population.

格雷夫斯病是甲状腺功能亢进症最常见的原因，由刺激促甲状腺激素（TSH）受体的自身抗体驱动。这是一种复发与缓解交替的疾病，影响全球1%的人口。

The TSHR-IgG1 autoantibody drives not only hyperthyroidism but also thyroid eye disease, neonatal Graves' disease, and pretibial myxedema. None of today's treatments - antithyroid drugs, radioactive iodine, or thyroidectomy - target this root cause. Among patients on antithyroid drugs, 93% report ongoing symptoms and 72% report five or more.

TSHR-IgG1自身抗体不仅驱动甲状腺功能亢进，还导致甲状腺眼病、新生儿格雷夫斯病和胫前黏液性水肿。目前的治疗方法——抗甲状腺药物、放射性碘或甲状腺切除术——均未针对这一根本病因。在接受抗甲状腺药物治疗的患者中，93%报告存在持续症状，72%报告有五种或更多症状。

With no new therapy approved in over 70 years, the unmet need is profound..

七十多年来没有新的疗法获得批准，未满足的需求极为迫切。

About BHV-1300

关于 BHV-1300

BHV-1300, the first MoDE, is a small-molecule extracellular IgG1,2,4 degrader that leverages the body's natural hepatic clearance pathways to selectively eliminate disease-driving IgG subclasses. Critically differentiated from FcRn inhibitors: BHV-1300 spares IgG3 (which protects against bacteria, viruses, and parasites), does not accelerate clearance of co-administered biologic therapies, and avoids the class effects of cholesterol elevation, albumin reduction, and headache.

BHV-1300 是首款分子降解剂（MoDE），是一种小分子细胞外 IgG1、2、4 降解剂，利用人体天然的肝脏清除途径选择性消除致病性 IgG 亚类。与 FcRn 抑制剂相比，BHV-1300 具有关键性差异：它不影响 IgG3（可抵御细菌、病毒和寄生虫），不会加速合并使用的生物制剂疗法的清除，并避免了胆固醇升高、白蛋白降低和头痛等类别效应。

Delivered via self-administered autoinjector, BHV-1300 degrades the TSHR-IgG1 autoantibody that drives Graves' disease at its source (see Figure 3)..

BHV-1300 通过自我给药的自动注射器递送，从源头上降解驱动格雷夫斯病的 TSHR-IgG1 自身抗体（见图 3）。

Figure 3:

图3：

BHV-1300 degrades the disease-causing TSHR-IgG1 autoantibody that drives Graves' disease, stabilizing thyroid hormone levels and targeting the root cause of Graves' disease and associated TSHR autoantibody-driven conditions, including thyroid eye disease, thyroid dermopathy, and Graves' embryopathy..

BHV-1300 可降解驱动格雷夫斯病的致病性 TSHR-IgG1 自身抗体，从而稳定甲状腺激素水平，并针对格雷夫斯病及相关 TSHR 自身抗体驱动疾病的根本病因，包括甲状腺眼病、甲状腺皮肤病和格雷夫斯胚胎病。

About Biohaven's Extracellular Degrader Platform

关于Biohaven的细胞外降解平台

Biohaven's MoDE and TRAP platforms represent a new class of medicines that selectively remove disease-driving extracellular proteins (such as antibodies) by directing them to the body's natural clearance pathways. Designed to target the cause of disease while preserving healthy immunity, the platform has been evaluated across nearly 200 individuals dosed to date and has demonstrated the potential to deeply, rapidly, and selectively lower the pathogenic antibodies that drive autoimmune disease.

Biohaven 的 MoDE 和 TRAP 平台代表了一类新型药物，它们通过将致病性细胞外蛋白（如抗体）引导至机体的自然清除途径，从而选择性地将其移除。该平台旨在针对疾病病因，同时保留健康的免疫功能；迄今为止，已在近 200 名受试者中进行了评估，并展现出深度、快速且选择性降低驱动自身免疫疾病的致病性抗体的潜力。

BHV-1300 is the lead MoDE degrader advancing in Graves' disease..

BHV-1300 是用于治疗格雷夫斯病的领先 MoDE 降解剂。

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. The Company is advancing its innovative portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms.

Biohaven 是一家生物制药公司，专注于在包括免疫学、神经科学和肿瘤学在内的关键治疗领域发现、开发和商业化具有变革意义的疗法。该公司正凭借其经证实的药物开发经验和多项专有药物开发平台，推进其创新疗法组合。

Biohaven's extensive clinical and preclinical programs include Kv7 ion channel modulation for epilepsy; MoDE and TRAP extracellular protein degradation for immunological diseases; and myostatin inhibition for neuromuscular and metabolic diseases, including obesity. For more information, visit .

Biohaven 广泛的临床和临床前项目包括：用于癫痫治疗的 Kv7 离子通道调节；用于免疫性疾病治疗的 MoDE 和 TRAP 细胞外蛋白降解技术；以及用于神经肌肉疾病和代谢性疾病（包括肥胖症）治疗的肌生成抑制素抑制剂。欲了解更多信息，请访问。

www.biohaven.com

。

Forward-Looking Statements

前瞻性陈述

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', 'potentially', 'potentially groundbreaking' and similar expressions, is intended to identify forward-looking statements.

本新闻稿包含经1995年《私人证券诉讼改革法案》界定的前瞻性陈述。使用某些词语，包括“继续”、“计划”、“将”、“相信”、“可能”、“预期”、“潜在”、“潜在突破性”以及类似表述，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.

提醒投资者注意，任何前瞻性陈述，包括关于候选产品未来开发、时间安排以及潜在监管批准和商业化的陈述，均不构成对未来表现或结果的保证，并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 pivotal study of BHV-1300; 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、发展和事件可能因各种因素而与前瞻性陈述中的内容存在重大差异，这些因素包括：Biohaven 计划中和正在进行的临床试验（包括 BHV-1300 的关键性研究）的预期时间安排、启动及结果；与 FDA 的计划互动和提交文件的时间安排；预期监管申报的时间安排和结果；对适用的美国监管要求的合规情况；Biohaven 候选产品的潜在商业化前景；以及 Biohaven 候选产品的有效性和安全性。

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'.

与前瞻性陈述相关的其他重要因素已在 Biohaven 向美国证券交易委员会提交的文件中予以说明，包括在题为“风险因素”和“管理层对财务状况及经营成果的讨论与分析”的章节中。

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 or otherwise, except a.

前瞻性陈述仅截至本新闻稿发布之日作出，Biohaven 不承担任何义务因新信息、未来事件或其他原因更新任何前瞻性陈述，除非 a。

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：

Jennifer Porcelli

詹妮弗·波切利

Vice President, Investor Relations

投资者关系副总裁

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+1 (201) 248-0741

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