OSAKA, Japan & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Takeda (TSE:4502/NYSE:TAK) today announced positive topline results from a Phase 2, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability and efficacy of mezagitamab (TAK-079) in patients with persistent or chronic primary immune thrombocytopenia (ITP).

日本大阪和马萨诸塞州剑桥市。-（商业新闻短讯）--武田（TSE:4502/纽约证券交易所：TAK）今天宣布了一项2期随机双盲安慰剂对照研究的阳性结果，该研究评估了mezagitamab（TAK-079）对持续性或慢性原发性免疫性血小板减少症（ITP）患者的安全性，耐受性和疗效。

Mezagitamab is a fully human immunoglobulin IgG1 monoclonal antibody (mAb) with high affinity for CD38 expressing cells (including plasmablasts, plasma cells, natural killer cells) resulting in their depletion..

Mezagitamab是一种完全人免疫球蛋白IgG1单克隆抗体（mAb），对表达CD38的细胞（包括浆母细胞，浆细胞，自然杀伤细胞）具有高亲和力，导致其耗竭。

The TAK-079-1004 trial (NCT04278924) evaluated three different doses of subcutaneous mezagitamab vs placebo, given once weekly for eight weeks in patients with chronic (more than one year in duration) or persistent (3-12 months in duration) primary ITP. An interim analysis of the ongoing Phase 2 study demonstrated positive safety and efficacy results.

TAK-079-1004试验（NCT04278924）评估了三种不同剂量的皮下美扎吉单抗与安慰剂，对于慢性（持续时间超过一年）或持续性（持续时间为3-12个月）原发性ITP患者，每周一次，持续八周。正在进行的2期研究的中期分析显示了积极的安全性和有效性结果。

Mezagitamab has been generally safe and well tolerated across all three cohorts. All mezagitamab doses tested demonstrated a higher platelet response rate than placebo. The increases in platelet count were dose-dependent with the greatest platelet response observed at the highest dose tested. Platelet response in mezagitamab treated patients occurred rapidly and was maintained post-therapy..

在所有三个队列中，Mezagitamab通常是安全且耐受性良好的。所有测试的mezagitamab剂量均显示出比安慰剂更高的血小板反应率。血小板计数的增加是剂量依赖性的，在测试的最高剂量下观察到最大的血小板反应。美扎吉单抗治疗患者的血小板反应迅速发生，并在治疗后得以维持。

Based on these positive results, and following consultation with global health authorities, Takeda plans to initiate a global Phase 3 trial of mezagitamab in ITP in fiscal year 2024. When mezagitamab advances to Phase 3 in ITP Takeda will have five new molecular entities in Phase 3 development in fiscal year 2024 including TAK-279 for the treatment of psoriasis and psoriatic arthritis, TAK-861 for the treatment of narcolepsy type 1, soticlestat for the treatment of Lennox-Gastaut syndrome and Dravet syndrome, and fazirsiran for the treatment of α1-antitrypsin associated liver disease..

基于这些积极的结果，并在与全球卫生部门协商后，武田计划于2024财政年度在ITP启动mezagitamab的全球3期试验。当mezagitamab进入ITP的第三阶段时，武田将在2024财政年度的第三阶段发展中拥有五个新的分子实体，包括用于治疗牛皮癣和银屑病关节炎的TAK-279，用于治疗发作性睡病1型的TAK-861，用于治疗Lennox-Gastaut综合征和Dravet综合征的soticlestat，和fazirsiran治疗α1-抗胰蛋白酶相关肝病。

ITP is a rare, IgG mediated autoimmune disease caused, in part, by the development of autoantibodies to platelets (and/or megakaryocytes), which are blood cells responsible for preventing or stopping bleeding. ITP is characterized by the accelerated destruction of platelets (with or without impaired production), resulting in a decreased platelet count and an increased risk of bleeding, which can be debilitating (including fatigue and impaired quality of life), and in severe cases may be life-threatening.

ITP是一种罕见的IgG介导的自身免疫性疾病，部分原因是血小板（和/或巨核细胞）自身抗体的发展，血小板是负责预防或止血的血细胞。ITP的特征是血小板的加速破坏（有或没有受损的生产），导致血小板计数减少和出血风险增加，这可能会使人衰弱（包括疲劳和生活质量受损），严重时可能危及生命。

The precedent for approval of new drugs in this indication requires that platelet counts be maintained at 50,000/uL or more for a sustained period. Approximately 20 percent of patients with ITP do not achieve a platelet count above 50,000/uL following treatment with available first- and second-line therapies creating significant patient burden and unmet need for a disease modifying treatment that is also tolerable.1,2.

该适应症中批准新药的先例要求血小板计数持续保持在50000/uL或更高。大约20%的ITP患者在接受可用的一线和二线治疗后，血小板计数未达到50000/uL以上，从而产生了严重的患者负担，并且对疾病缓解治疗的需求也未得到满足。

“These Phase 2 results demonstrate mezagitamab’s compelling disease modifying mechanism of action, which has the potential to achieve disease remission for people with ITP. There remains considerable unmet need among ITP patients who may not respond or have inadequate response to prior treatment. In addition, some patients who do respond to available therapies struggle to manage side effects or relapse despite treatment,” said Chinwe Ukomadu, Head of the Gastrointestinal & Inflammation Therapeutic Area Unit at Takeda.

“这些第二阶段的结果表明，美扎吉单抗具有令人信服的疾病缓解作用机制，有可能为ITP患者缓解疾病。ITP患者对先前治疗可能无反应或反应不足，但仍有大量需求未得到满足。此外，一些对现有治疗有反应的患者难以控制副作用武田胃肠道和炎症治疗区负责人ChinweUkomadu说。

“We look forward to initiating the Phase 3 trial and presenting these data at an upcoming scientific congress.”.

“我们期待着启动第三阶段试验，并在即将举行的科学大会上提交这些数据。”

Therapy with mezagitamab is designed to deliver rapid and sustained improvement in platelet response and generally rapidly restores platelet counts to functional levels. Mezagitamab previously received Orphan Drug Designation for the treatment of ITP from the U.S. Food and Drug Administration and the program recently received Fast Track Designation..

美扎吉单抗治疗旨在快速持续改善血小板反应，并通常迅速将血小板计数恢复到功能水平。Mezagitamab之前从美国食品和药物管理局获得了用于治疗ITP的孤儿药指定，该计划最近获得了快速通道指定。

Results from the Phase 2 trial have no impact on the full year consolidated reported forecast for the fiscal year ending March 31, 2024 (Fiscal Year 2023).

第二阶段试验的结果对截至2024年3月31日财年（2023财年）的全年综合报告预测没有影响。

About Takeda

关于武田

Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience, and vaccines.

武田致力于为人们创造更好的健康，为世界创造更美好的未来。我们的目标是在我们的核心治疗和业务领域发现并提供改变生命的治疗方法，包括胃肠道和炎症、罕见疾病、血浆衍生疗法、肿瘤学、神经科学和疫苗。

Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet.

与我们的合作伙伴一起，我们的目标是通过我们动态多样的渠道改善患者体验，并推进治疗选择的新前沿。作为总部位于日本的领先的基于价值观、研发驱动的生物制药公司，我们以对患者、人民和地球的承诺为指导。

Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries.

我们在大约80个国家和地区的员工是由我们的目标驱动的，并以两个多世纪以来定义我们的价值观为基础。