Waldenstrom's macroglobulinaemia recurrent

在受邀演讲和口头报告环节对百悦泽®（泽布替尼）、BTK CDAC降解剂BGB-16673以及BCL2抑制剂sonrotoclax相关研究进行介绍 美国加州圣马特奥——百济神州有限公司（纳斯达克代码：BGNE；香港联交所代码：06160；上交所代码：688235）是一家全球肿瘤治疗创新公司，今日宣布将在第12届华氏巨球蛋白血症国际研讨会（IWWM）上分享评估百悦泽®（泽布替尼）、布鲁顿氏酪氨酸激酶（BTK）嵌合式降解激活化合物（CDAC）降解剂BGB-16673和B细胞淋巴瘤2（BCL2）抑制剂sonrotoclax用于治疗华氏巨球蛋白血症患者的研究，该研讨会将于10月17日至19日在捷克首都布拉格举行。 百济神州将在2024年IWWM上进行七项展示。百济神州血液学首席医学官Mehrdad Mobasher医学博士及公共卫生硕士将在开幕式上发表 “通过创新实现影响：百济神州如何推进产品和管线以应对华氏巨球蛋白血症及其他疾病”为题的主旨演讲。 华氏巨球蛋白血症是一种罕见且无法治愈的癌症，许多患者在接受初始治疗后再次面临疾病进展，我们的BTK抑制剂百悦泽®已成为这些患者的标准治疗方案。正如我们在IWWM上展示的长期随访数据，该药物在患者中持续取得深度和持久的缓解，并具有良好的安全性。我们的其他展示也表明我们始终致力于开发和评估针对WM患者的新治疗方案，包括我们在BTK CDAC降解剂BGB-16673和BCL2抑制剂sonrotoclax 1期研究中所取得的结果。这些研究结果让我们备受鼓舞，同时我们期待进一步推进这些产品，以帮助更多的WM患者。 在IWWM期间，百济神州将通过两场受邀演讲、三场口头报告和两场海报展示分享研究结果。主要展示内容包括： BTK抑制剂用于WM的治疗I 会议时间：10月18日11:30 a.m.-12:30 p.m.（当地时间，下同） 标题：ASPEN研究长期发现：临床结局 展示类型：口头报告 产品：百悦泽® 主要发现： ASPEN LTE1 是一项长期扩展研究，评估了ASPEN研究中接受伊布替尼治疗的患者转用百悦泽®后的情况。该研究的长期安全性和有效性数据显示，在转用百悦泽®后，伊布替尼治疗引起的不良事件恶化情况很少见，新事件的出现同样少见。研究还发现，该部分患者治疗有效性也得到了维持或改善。 BTK抑制剂不耐受及耐药性疾病 会议时间：10月18日2:30-3:30 p.m. 标题：布鲁顿氏酪氨酸激酶降解剂BGB-16673用于复发或难治性华氏巨球蛋白血症患者治疗的初步有效性和安全性：1期CaDAnCe-101研究结果 展示类型：受邀演讲 产品：BGB-16673 主要发现： 1期CaDAnCe-101研究的初步安全性和有效性数据表明，BGB-16673具有可耐受的安全性特征，并且在既往接受过多线BTK抑制剂治疗的复发/难治性（R/R）WM患者（包括携带BTK和CXCR4突变的患者）中表现出良好的抗肿瘤活性。 标题：泽布替尼用于治疗伊布替尼/阿可替尼不耐受的华氏巨球蛋白血症患者表现出良好耐受性和有效性 展示类型：受邀演讲 产品：百悦泽® 主要发现： 一项2期研究最新结果显示，在对其他BTK抑制剂（包括伊布替尼和阿可替尼）产生不耐受后转为接受百悦泽®治疗的 WM 患者中，大多数导致不耐受的不良事件（AE）没有复发。此外，这些患者的治疗有效性得到维持或改善。 WM海报展示及招待会 会议时间：10月18日4:30-6:30 p.m. 标题：布鲁顿酪氨酸激酶抑制剂用于治疗华氏巨球蛋白血症患者的3期ASPEN研究中相关周围神经病变 展示类型：海报展示 产品：百悦泽® 主要发现： 一项对3期 ASPEN 研究的分析表明，BTK抑制剂治疗后周围神经病变（PN）症状的缓解与疾病缓解的深度相关，对于达到PN缓解的患者，使用百悦泽®治疗后症状缓解速度比使用伊布替尼治疗更快。 标题：提高华氏巨球蛋白血症主动监测质量：一项在英国开展的患者体验问卷调查结果以及英国患者-专家合作建议的主动监测标准方法 展示类型：海报展示 产品：N/A 主要发现： 一项在英国开展的患者体验问卷调查结果表明，无症状WM患者采用“主动监测”（即医疗团队对WM进行主动监测而非立即进行治疗）的情况存在很大差异，超过半数的调查参与者表示他们的体验可以得到改善。 全体会议II 会议时间：10月19日9-10 a.m. 标题：新型BCL2抑制剂sonrotoclax（BGB-11417）用于治疗复发/难治性华氏巨球蛋白血症患者1期研究的安全性和有效性数据 展示类型：口头报告 产品：Sonrotoclax 主要发现： 1期研究的结果显示，sonrotoclax总体耐受性良好，且对于接受过多线治疗的R/R WM患者而言，其初步抗肿瘤活性令人鼓舞。 进行中的WM临床试验 II 会议时间：10月19日4-5 p.m. 标题：BGB-11417-203：一项正在进行的新一代BCL2抑制剂sonrotoclax（BGB-11417）用于华氏巨球蛋白血症患者治疗的2期研究 展示类型：口头报告 产品：Sonrotoclax 主要发现： 试验进展报告概述了一项sonrotoclax 2期研究，该研究受试者是既往接受过BTK抑制剂或含抗CD20抗体的系统性治疗的R/R WM患者。 （上下滑动查看详情） 关于华氏巨球蛋白血症（WM） 华氏巨球蛋白血症是一种罕见的B细胞淋巴瘤，在非霍奇金淋巴瘤患者中占比不到2%1。该疾病通常在老年人中多发，主要见于骨髓，但也可能影响淋巴结和脾脏2。患者通常发病年龄在60至70岁之间。由于尚未查明的原因，WM在男性中的发病率几乎是女性的两倍，在白种人中的发病率高于其他种族群体3。WM 是一种罕见癌症，每年每百万人中只有大约3-5人会患上该癌症2。 关于百悦泽®（泽布替尼） 百悦泽®是一款布鲁顿氏酪氨酸激酶（BTK）小分子抑制剂，其设计主要通过优化生物利用度、半衰期和选择性，实现对BTK蛋白完全、持续的抑制。凭借与其他获批BTK抑制剂存在差异化的药代动力学特征，百悦泽®已被证明能在多个疾病相关组织中抑制恶性B细胞增殖。 关于百济神州 百济神州是一家全球肿瘤治疗创新公司，专注于为全世界的癌症患者研发创新抗肿瘤药物。通过强大的自主研发能力和外部战略合作，我们不断加速开发多元、创新的药物管线和产品组合，致力于为全球更多患者全面提升药物可及性和可负担性。在全球五大洲，我们有超过10,000人的团队。如需了解更多信息，请访问www.beigene.com.cn或关注“百济神州”微信公众号。 前瞻性声明 — 左右滑动查看更多 — 参考文献 1 Buske, C, et al. Treatment and outcome patterns in European patients with Waldenström’s macroglobulinaemia: a large, observational, retrospective chart review. The Lancet Haematology 2018; 5: e0299-309. 2 Lymphoma Research Foundation. Getting the Facts: Waldenström Macroglobulinemia. Accessed March 2022. Available at https://lymphoma.org/wp-content/uploads/2021/12/LRF-Waldenstrom-Macroglobulinemia_Factsheet.pdf 3 IWMF. Frequently Asked Questions – Waldenstrom’s Macroglobulinemia. https://iwmf.com/frequently-asked-questions-waldenstrom-macroglobulinemia/

BeiGene’s Brukinsa (zanubrutinib) has been recommended by the National Institute for Health and Care Excellence (NICE) to treat certain cases of marginal zone lymphoma (MZL). The Bruton’s tyrosine kinase inhibitor, which is the first treatment to be specifically licensed for this form of blood cancer, is now available on the NHS in England as an additional option for patients whose cancer has not responded well to previous treatment. Approximately 2,600 people are diagnosed every year in the UK with MZL, a group of slow-growing non-Hodgkin lymphomas that develop when there is uncontrollable growth in B cells. The disease is often diagnosed at a more advanced stage due to the main symptom being small, painless lumps in lymph nodes, and some patients become resistant to current treatments, including chemotherapy. Brukinsa can be taken at home as either once- or twice-daily capsules, providing an alternative to further rounds of chemotherapy and potentially reducing the inconvenience and side effects of intravenous treatment. NICE’s decision, which is expected to benefit about 470 patients over the next three years, was supported by clinical trial results showing that up to 80% of patients’ cancers responded to BeiGene’s drug. Helen Knight, director of medicines evaluation at NICE, said: “Evidence suggests that this treatment increases how long people have before their condition gets worse and increases how long they live compared with standard care. “People with MZL are typically diagnosed in their 70s, so there is a need for effective and safe treatment options that are convenient for them to take.” Brukinsa is already available to treat two other blood cancers: chronic lymphocytic leukaemia and Waldenstrom’s macroglobulinaemia. Robert Mulrooney, general manager UK and Ireland at BeiGene, said: “We are delighted that NICE has recognised the clinical and economic benefit of Brukinsa for eligible patients across three disease areas.” The recommendation comes just a few months after Brukinsa was granted accelerated approval by the US Food and Drug Administration to treat relapsed or refractory follicular lymphoma. The US regulator specifically authorised the drug for use alongside Roche’s anti-CD20 monoclonal antibody Gazyva (obinutuzumab) in adult patients who have received at least two prior lines of systemic therapy.

Janssen and Pharmacyclics’ ibrutinib has demonstrated a significant and sustained progression-free and overall survival benefit in patients with previously untreated chronic lymphocytic leukaemia (CLL), according to new long-term results shared by the Johnson & Johnson company. Affecting approximately 4.92 per 100,000 people every year in Europe, CLL is typically a slow-growing blood cancer of the white blood cells. Despite patient outcomes improving significantly over the last few decades, CLL is still characterised by consecutive episodes of disease progression and patients are often prescribed multiple lines of therapy as they relapse or become resistant to treatments. Administered orally once daily, ibrutinib is designed to block the BTK protein needed by normal and abnormal B-cells to multiply and spread. The drug already holds approvals to treat treatment-naïve and pretreated CLL, as well as certain patients with mantle cell lymphoma and Waldenström’s macroglobulinaemia. The phase 3 RESONATE-2 trial has been comparing ibrutinib monotherapy against chlorambucil for up to 12 cycles in previously untreated CLL patients aged 65 years and older. The results, presented at this year’s European Hematology Association Congress, showed a median progression-free survival of 8.9 years in the ibrutinib arm versus 1.3 years in the chlorambucil cohort. With up to ten years of follow-up, median overall survival had not been reached with ibrutinib and 27% of patients in the study remained on ibrutinib. The drug was also found to be well tolerated as a long-term treatment and no new safety signals were observed. Commenting on the latest results, clinical study investigator, Alessandra Tedeschi, Niguarda Hospital, said: “When ibrutinib was first introduced more than ten years ago, it changed the course of CLL treatment, and today it remains a central part of the standard of care for patients living with B-cell malignancies. “The final analysis of the RESONATE-2 study confirms the favourable benefit-risk profile of ibrutinib is sustained over time, with the longest follow-up data of any targeted therapy in CLL, and demonstrates its potential to enable patients diagnosed with CLL today to look forward to the possibility of a normalised life expectancy.”