Sutacimig

Hemab Therapeutics, a biotech headquartered in Cambridge, Massachusetts, and Copenhagen, Denmark, announced receipt of US FDA Breakthrough Therapy Designation (BTD) for sutacimig, a subcutaneously administered bispecific antibody, for the prevention of bleeding episodes in patients with Glanzmann thrombasthenia (GT). Sutacimig, formerly designated HMB-001, binds and stabilizes endogenous Factor VIIa with one antibody arm while binding TLT-1 on activated platelets with the other, facilitating hemostatic plug formation. Hemab Therapeutics is a privately held company with no public stock ticker. BTD provides Hemab Therapeutics with intensive FDA guidance on drug development, organizational commitment from senior FDA managers, and eligibility for rolling review, which permits submission of completed sections of a marketing application before the entire filing is ready. Sutacimig’s clinical progress Sutacimig was originated and developed in-house by Hemab, which was founded in 2019 from academic research in Scandinavian coagulation biology. The BTD was granted on the basis of data from the completed Phase II multiple ascending dose portion of Hemab’s Phase I/II clinical trial (NCT06211634). In that study, the weekly dosing group achieved an estimated 87% reduction in annualized treated bleeding rate, with consistent reductions observed across bleed types, locations, and dosing cohorts. All tested doses produced over a 50% reduction in treated bleeding events. No major safety signals were reported in public disclosures from the Phase II portion. Hemab has stated plans to advance sutacimig into a pivotal Phase III study. Prior to this BTD, sutacimig had already received Fast Track Designation and Orphan Drug Designation from the FDA, orphan medicinal product designation in the European Union, and Innovative Licensing and Access Pathway (ILAP) designation from the UK Medicines and Healthcare products Regulatory Agency. Research context GT is a rare, inherited platelet disorder caused by deficiency or dysfunction of the glycoprotein IIb/IIIa complex, resulting in impaired platelet aggregation and recurrent, sometimes life-threatening bleeding. Data from the international GT360 natural history study reported that 88% of 117 participants experienced at least one bleed in the prior week, and 65% required a bleed-related hospital visit within six months. Over 80% had missed work or school, and more than 50% faced restrictions on social activities and travel. No prophylactic treatment for GT has received regulatory approval. The current standard of care relies on episodic, on-demand interventions, principally recombinant activated Factor VIIa (NovoSeven, Novo Nordisk) and platelet transfusions, neither of which was developed or approved as a prophylactic bleeding disorder treatment for GT. No novel drugs for GT have been approved in the past three years. The competitive landscape for therapies targeting von Willebrand factor or platelet glycoprotein receptors includes caplacizumab (an anti-vWF nanobody for thrombotic thrombocytopenic purpura), vonicog alfa (recombinant vWF for von Willebrand disease), and BT200 (a pegylated RNA aptamer targeting vWF). However, none of these molecules are in development for GT, and none share sutacimig’s bispecific mechanism simultaneously engaging Factor VIIa and activated platelet TLT-1. Unlike agents such as caplacizumab, which block vWF-platelet interactions to prevent pathological clotting, sutacimig is designed to promote hemostasis by concentrating endogenous Factor VIIa at the site of platelet activation — a mechanistically distinct, pro-hemostatic approach. No other bispecific antibody targeting both Factor VIIa and a platelet surface receptor for a pro-hemostatic indication was identified in active clinical development. Hemab’s pipeline also includes HMB-002 for von Willebrand disease and a Phase II trial of sutacimig in congenital Factor VII deficiency (NCT07347249), indicating broader ambitions for the bispecific platform across coagulation disorders. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

创新皮下注射双抗获FDA突破性疗法认定 Hemab Therapeutics今日宣布，美国FDA已授予其在研药物sutacimig突破性疗法认定（BTD），用于预防Glanzmann血小板无力症（GT）患者的出血发作。GT是一种严重的出血性疾病，患者可出现反复且难以控制的出血事件，在部分情况下甚至可能危及生命。此次突破性疗法认定基于一系列临床研究数据，其中包括Hemab正在开展的sutacimig（HMB-001-CL101）1/2期临床试验中已完成的多次递增剂量研究。数据显示，该疗法能够持续且具有临床意义地减少出血事件的发生，包括需要高强度治疗干预的严重出血事件，例如需使用重组凝血因子VIIa、血小板输注、血浆或冷沉淀治疗，或需要进行医疗操作干预的严重出血事件。 Sutacimig是一种通过皮下注射给药的潜在“first-in-class”双特异性抗体，其设计机制为一端抗体臂结合并稳定体内内源性凝血因子VIIa，另一端则与活化血小板表面的TLT-1结合。通过这一作用机制，sutacimig可促进体内内源性凝血因子VIIa的积累，并将其直接招募至活化血小板表面，从而促进血栓形成并增强止血作用。除突破性疗法认定外，sutacimig此前还已获得FDA授予的快速通道资格和孤儿药资格。 突破性寡核苷酸疗法登《新英格兰医学杂志》 Stoke Therapeutics近日宣布，其与渤健（Biogen）共同开发的在研反义寡核苷酸（ASO）药物zorevunersen的研究数据已发表于《新英格兰医学杂志》。该论文汇总了两项已完成的1/2a期临床试验以及正在进行的开放标签延长期研究（OLE）的结果。研究显示，zorevunersen在Dravet综合征患者中首次展现出潜在的疾病修饰作用。数据显示，在标准抗癫痫药物（ASM）治疗基础上联合使用zorevunersen治疗的患者，其癫痫发作频率显著且持续下降，同时在认知和行为等多个评估指标上也出现改善。这些疗效最早在1/2a期治疗阶段显现，并在随后长达三年的延长期研究中持续保持。相关结果也为目前正在开展的全球3期EMPEROR研究提供了支持。 Dravet综合征是一种严重的发育性和癫痫性脑病（DEE），患者通常会出现频繁且严重的癫痫发作，并在大约两岁时出现神经发育停滞。Zorevunersen旨在通过增强SCN1A基因非突变（野生型）拷贝的功能，提升脑细胞中NaV1.1蛋白的表达，从而治疗Dravet综合征的根本病因。Zorevunersen已获得了美国FDA授予的突破性疗法认定、罕见儿科疾病认定，以及FDA和欧洲药品管理局（EMA）授予的孤儿药资格。Stoke公司拥有该药在美国、加拿大和墨西哥的独家权益，渤健则拥有其余国家和地区的商业化权利。 潜在“first-in-class”多肽疗法积极试验结果公布 Parabilis Medicines今日公布了其在研候选药物zolucatetide的初步临床及临床前研究数据，显示该药物在治疗家族性腺瘤性息肉病（FAP）方面具有潜在治疗价值。Zolucatetide是一款在研、潜在“first-in-class”多肽候选药物，可竞争性抑制β-catenin与T细胞因子（TCF）家族转录因子之间的相互作用。通过在细胞内直接靶向β-catenin与TCF之间的蛋白—蛋白相互作用，zolucatetide旨在阻断Wnt信号通路的活性，并能够在不受多种APC或β-catenin突变影响的情况下发挥作用，从而抑制通常驱动相关疾病发生发展的分子机制。根据新闻稿介绍，zolucatetide是首个直接抑制β-catenin与TCF相互作用的抑制剂。 在正在进行的1/2期临床试验中，一名同时患有FAP及相关硬纤维瘤的患者在接受zolucatetide治疗60周后，十二指肠息肉病变出现显著改善。与治疗前评估结果相比，该患者息肉数量和大小均明显减少，疾病分期由Spigelman II期下降至I期。同时，该患者的硬纤维瘤直径减少了52.2%。研究期间未报告与治疗相关的严重不良事件，也未出现因不良事件导致的停药情况。此外，相应的临床前研究显示，在APC突变肿瘤细胞中，zolucatetide能够以剂量依赖方式抑制β-catenin转录活性，并在FAP小鼠模型中减少息肉形成，相关暴露水平与当前临床试验相当。 ▲Mathai Mammen博士在药明康德全球论坛上分享创新药物开发的洞见 值得一提的是，Parabilis Medicines董事长、总裁兼首席执行官Mathai Mammen博士曾参加2026药明康德全球论坛，分享创新药物开发洞见。他表示，企业需要更加系统地利用常规筛查数据，主动识别真正患病的潜在受试者。这一做法不仅有助于改善罕见病患者的诊断路径，也将为相关领域的临床试验推进带来实质性助力。 参考资料： [1] Hemab Therapeutics Receives FDA Breakthrough Therapy Designation for Sutacimig in Glanzmann Thrombasthenia. Retrieved March 5, 2026 from https://www.prnewswire.com/news-releases/hemab-therapeutics-receives-fda-breakthrough-therapy-designation-for-sutacimig-in-glanzmann-thrombasthenia-302704598.html [2] Parabilis Medicines Reports Early Clinical Evidence that Zolucatetide Reduces Polyp Burden in a Patient with Familial Adenomatous Polyposis. Retrieved March 5, 2026 from https://www.businesswire.com/news/home/20260305329916/en/Parabilis-Medicines-Reports-Early-Clinical-Evidence-that-Zolucatetide-Reduces-Polyp-Burden-in-a-Patient-with-Familial-Adenomatous-Polyposis [3] The New England Journal of Medicine Publishes First Data to Demonstrate the Potential for Disease Modification in Dravet Syndrome. Retrieved March 5, 2026 from https://investor.stoketherapeutics.com/news-releases/news-release-details/new-england-journal-medicine-publishes-first-data-demonstrate 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新