Canbridge Pharmaceuticals, Inc. (United States)

3月17日，致力于研究、开发和商业化治疗罕见疾病和肿瘤的全球生物制药公司CANbridge Pharmaceuticals宣布，其自研药物CAN008（asunercept）配伍替莫唑胺/放疗（TMZ/RT）就治疗多形性胶质母细胞瘤（glioblastoma multiforme，GBM）患者的1/2期研究的长期后续数据显示，在试验结束三年后，受试者五年的长期生存率达到67%。 胶质母细胞瘤是中枢神经系统最常见的恶性肿瘤，占颅内肿瘤的35.26％–60.9％。根据最新的颅内肿瘤分级标准，世界卫生组织（WHO）将其分类为WHO IV级。GBM具有高度浸润性和侵袭性，因此通常无法通过手术将其彻底清除，从而导致肿瘤的快速复发。尽管GBM的治疗在手术切除、放疗和化学治疗等领域一直在不断改进与尝试，但其预后并未得到实质性改善。中位生存时间仍然只有14.6个月。因此，迫切需要开发针对GBM的新型疗法。 CAN008是一种CD95-Fc融合蛋白，其通过与CD95L的结合，干扰CD95/CD95L的信号传导，从而阻断CD95/CD95L通路。CAN008具有独特的双重作用机制，不但可以抑制肿瘤细胞的生长和迁移，还可以抑制由Caspase介导的T细胞死亡，修复免疫功能。既往临床试验数据表明，CAN008具有良好的安全性，可以有效提高GBM患者的生存质量，并可以大幅度延长患者的生存期。基于此，欧洲药品管理局（EMA）将CAN008纳入优先药物（PRIME）计划。 在CAN008配伍标准化放疗，以治疗新诊断的多形性胶质母细胞瘤患者的I/II期研究的长期随访中，研究人员报告，参与CAN008 1/2期试验的9名新诊断GBM患者在五年随访，即试验完成三年后，仍有四名患者存活。所有四名存活的患者都属于试验的高剂量组，即在标准的替莫唑胺/放疗（TMZ/RT）之外接受400mg的CAN008给药。高剂量CAN008组两年的总生存率为83%，三年、四年和五年为67%。相比之下，GBM数据库（n=218）记录的总生存率为两年34.3%，三年19.5%，四年16.1%，五年8.2%。 此外，高剂量CAN008队列的中位无进展生存期为17.95个月。而据报道，接受标准护理（TMZ/RT）的GBM患者的中位无进展生存期仅为6.9个月。研究人员还报告，CAN008对高肿瘤突变负担和DNAH家族基因突变的患者有良好的治疗效果。 该研究在临床研究地点台湾桃园的长庚纪念医院进行。主要研究者是Wei Kuo-Chen博士。试验数据将在3月20-22日在瑞士卢加诺举行的欧洲肿瘤内科医师协会（ESMO）肉瘤和罕见癌症年度大会上以海报形式公布。目前，CAN008正在中国进行就治疗GBM的第二阶段临床试验。预计将在2023年中旬进行中期数据分析。 CANbridge首席医疗官兼首席开发战略师、医学博士Gerry Cox评论："多形性胶质母细胞瘤是最致命的癌症之一，其生存率不到15个月，治疗进展很少，未满足的医疗需求很大。我们很高兴看到CAN008治疗的GBM患者的中位无进展生存期达到17.95个月，是标准护理GBM患者PFS的两倍多，而且67%的CAN008高剂量治疗患者五年后仍然存活。" 参考资料： https://www.canbridgepharma.com/en/media-news/press-release/20230317/

BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- CANbridge Pharmaceuticals, Inc. (1228.HK), a China and U.S.-based global biopharmaceutical company committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, announced that it will be presenting data from the CAN106 Phase 1 trial in healthy volunteers at two conferences. CAN106 is under development for paroxysmal nocturnal hemoglobinuria (PNH) and other complement-mediated diseases and is currently in a Phase 1b/2 study in patients with PNH in China. The conferences are: 17th National Conference on Hematology (Shanghai) Bing Han, MD, Chief Physician and Professor in the Department of Hematology, at the Peking Union Medical College Hospital, and principal investigator of the ongoing CAN106 Phase 1b/2 trial in PNH in China, will present CAN106 Phase 1 data. Presentation on Saturday, September 24, 2022, at 4:05 PM CST 6th Annual Complement Based Drug Development Summit 2022 (Boston) Gerry Cox, MD, Interim Chief Medical Officer, Chief Development Strategist, CANbridge Pharmaceuticals, will present CAN106 Phase 1 data. Presentation on Wednesday, September 28, 2022, at 11:40 AM EST About CANbridge Pharmaceuticals Inc. CANbridge Pharmaceuticals Inc. (HKEX:1228) is a China and U.S.-based global biopharmaceutical company committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology. CANbridge has a differentiated drug portfolio, with three approved drugs and a pipeline of 10 assets, targeting prevalent rare disease and rare oncology indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases, as well as glioblastoma multiforme. CANbridge is also building next-generation gene therapy development capability through a combination of collaboration with world-leading researchers and biotech companies and internal capacity. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the UMass Chan Medical School, the University of Washington School of Medicine, Scriptr Global and LogicBio.