お知らせ • Jul 12
Hemab Therapeutics Presents New Clinical Data From HMB-002 In Von Willebrand Disease And Introduces HMB-003 For Heavy Menstrual Bleeding
Hemab Therapeutics presented new clinical data from HMB-002 in Von Willebrand disease (VWD) and announced the HMB-003 program at the International Society on Thrombosis and Haemostasis (ISTH) 2026 Congress in Paris, France. HMB-002 clinical data demonstrate proof of mechanism, with dose-dependent, greater than or equal to 2.4-fold peak increases in Von Willebrand Factor (VWF) and Factor VIII (FVIII), normalization of peak thrombin generation and activated partial thromboplastin time (APTT), and durability supporting potential monthly subcutaneous dosing. HMB-003 program announced as non-hormonal direct plasmin inhibitor with potent, selective and extended antifibrinolytic activity, designed to reduce bleeding across multiple settings beginning in heavy menstrual bleeding. HMB-002 is designed to address the underlying biological drivers of VWD through a non-replacement approach—elevating the body's endogenous levels of VWF and FVIII rather than infusing exogenous factor—with early first-in-human data building a compelling case for this paradigm shift. Extended half-life supporting infrequent dosing: Pharmacokinetic (PK) analysis confirmed dose-dependent increase in Cmax with prolonged duration. Dual VWF and FVIII elevation: In all dose cohorts tested, VWF and FVIII were elevated in a dose-dependent manner. In cohort A3 (150 mg), =2.4-fold elevation in both VWF and FVIII was achieved and accompanied by restoration of thrombin generation, shortening of APTT, and stable multimer distribution. The pharmacokinetic/pharmacodynamic (PK/PD) profile supports potential for monthly dosing. Emerging safety profile: Most treatment-emergent adverse events (TEAEs) were mild to moderate in severity, no serious TEAEs occurred, no events were considered related to HMB-002, and no participant discontinued the study due to TEAEs. There were no thromboembolic events, no injection site reactions, no thrombocytopenia, and no hypersensitivity reactions. Preliminary clinical observations: The single ascending dose (SAD) part of the study wasn’t designed to measure efficacy, so clinical observations should be considered descriptive in nature. Across the SAD cohorts, 8 of 9 evaluable patients had zero treated bleeds in the 28 days following HMB-002 dosing, with a mean ATBR of 1.6. Among participants treated with HMB-002, the baseline mean ATBR before HMB-002 treatment was 20.1 reflecting significant disease burden in Type 1 VWD, based on bleed data collected up to 5.5 months. HMB-003 is a novel fatty-acid-conjugated peptide antifibrinolytic, designed to stabilize clots and reduce bleeding across multiple settings, beginning in heavy menstrual bleeding, with preclinical data supporting potent, selective antifibrinolytic activity and extended duration of action. Potent and selective plasmin inhibition: HMB-003 directly inhibits plasmin at its active site and inhibits fibrinolysis across both tPA- and uPA-driven pathways, while showing no effect on thrombin generation, platelet function, or coagulation in nonclinical studies. Extended antifibrinolytic activity: Following a single subcutaneous dose in minipigs, HMB-003 achieved peak plasma levels within hours and sustained antifibrinolytic activity for approximately one week, supporting the potential for cycle-matched dosing in heavy menstrual bleeding. HMB-002 is a monovalent human antibody being developed as the first-in-class subcutaneous prophylactic treatment for Von Willebrand Disease targeting the underlying cause of the disease, a condition driven by a deficiency or defect in Von Willebrand Factor (VWF), a key regulator of hemostasis. By specifically targeting the C-terminal CK domain of VWF, which is distinct from regions critical to its essential interactions, HMB-002 shields the protein from degradation, boosting endogenous levels without compromising its function. Clinical and nonclinical data suggest strong potential for meaningful therapeutic benefit. HMB-003 is a subcutaneously administered peptide-based plasmin inhibitor with a durable half-life — a proven therapeutic target in coagulation medicine — being developed as a novel antifibrinolytic designed to reduce bleeding across multiple settings. Engineered to directly inhibit plasmin at its active site, HMB-003 blocks fibrinolysis independently of the plasminogen activation pathway. HMB-003 is optimized to provide sustained bleed protection across multiple high-unmet-need conditions, ranging from heavy menstrual bleeding and hereditary hemorrhagic telangiectasia to peri-operative bleeding management.