Hemab Therapeutics Unveils HMB-002 Proof-of-Mechanism Data in VWD and Announces Novel Antifibrinolytic HMB-003 at ISTH 2026
核心洞察
HMB-002 demonstrated dose-dependent ≥2.4-fold peak increases in VWF and FVIII with normalization of thrombin generation and APTT, supporting potential monthly subcutaneous dosing in Von Willebrand Disease (搜索).
In the SAD study, 8 of 9 evaluable patients had zero treated bleeds in the 28 days following HMB-002 dosing, with a favorable safety profile and no serious treatment-related adverse events.
Hemab announced HMB-003, a non-hormonal fatty-acid-conjugated peptide plasmin inhibitor with sustained antifibrinolytic activity for approximately one week, initially targeting heavy menstrual bleeding.
Hemab Therapeutics (搜索) presented new clinical data from its HMB-002 program in Von Willebrand Disease (搜索) (VWD) and unveiled HMB-003, a novel antifibrinolytic candidate for heavy menstrual bleeding, at the International Society on Thrombosis and Haemostasis (ISTH) 2026 Congress in Paris, France. The presentations mark a significant step forward for the Cambridge, Mass.- and Copenhagen-based biotechnology company as it builds a franchise of therapies targeting serious bleeding disorders.
"Millions of people living with bleeding disorders including VWD and heavy menstrual bleeding have no reliable way to prevent excessive bleeds," said Benny Sørensen, MD, PhD, CEO of Hemab. "Today, at ISTH 2026 we presented new HMB-002 data demonstrating proof of mechanism in VWD alongside preliminary clinical observations on treated bleeding events."
HMB-002 Demonstrates Proof of Mechanism in VWD
HMB-002 is a monovalent human antibody designed as a first-in-class subcutaneous prophylactic treatment for VWD. Rather than infusing exogenous factor, HMB-002 targets the C-terminal CK domain of Von Willebrand Factor (VWF), shielding the protein from degradation and boosting endogenous levels without compromising function.
Pharmacokinetic analysis from the single ascending dose (SAD) study confirmed a dose-dependent increase in Cmax with prolonged duration. Across all dose cohorts, VWF and Factor VIII (搜索) (FVIII) were elevated in a dose-dependent manner. In cohort A3, which received a 150 mg dose, both VWF and FVIII achieved ≥2.4-fold peak increases, accompanied by restoration of thrombin generation, shortening of activated partial thromboplastin time (APTT), and stable multimer distribution. The pharmacokinetic/pharmacodynamic profile supports the potential for monthly subcutaneous dosing.
"The new HMB-002 data support a non-replacement approach in VWD: a single subcutaneous dose raising both VWF and FVIII more than 2-fold, with a favorable safety profile across every cohort," said Priyanka Raheja, MD, Consultant Haematologist at Barts Health NHS Trust. "Building on 50 years of clinically validated treatment strategy, HMB-002 shows potential to change the VWD treatment paradigm by addressing the root cause of the disease; a durable increase, and for many patients potential normalization, of both VWF and FVIII."
Safety and Preliminary Clinical Observations
The emerging safety profile for HMB-002 was encouraging. 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. Notably, there were no thromboembolic events, no injection site reactions, no thrombocytopenia, and no hypersensitivity reactions.
Although the SAD portion of the study was not designed to measure efficacy, preliminary clinical observations were notable. Across the SAD cohorts, 8 of 9 evaluable patients had zero treated bleeds in the 28 days following HMB-002 dosing, with a mean annualized treated bleed rate (ATBR) of 1.6. Among participants treated with HMB-002, the baseline mean ATBR before treatment was 20.1, reflecting the significant disease burden in Type 1 VWD, based on bleed data collected up to 5.5 months.
HMB-003: A Novel Non-Hormonal Antifibrinolytic
Hemab also announced HMB-003, a subcutaneously administered fatty-acid-conjugated peptide antifibrinolytic designed to stabilize clots and reduce bleeding across multiple settings, beginning with heavy menstrual bleeding.
HMB-003 directly inhibits plasmin at its active site and blocks fibrinolysis across both tissue plasminogen activator (tPA)- and urokinase plasminogen activator (uPA)-driven pathways, while showing no effect on thrombin generation, platelet function, or coagulation in nonclinical studies. In minipigs, a single subcutaneous dose 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.
"We also unveiled HMB-003, a novel antifibrinolytic built on validated fatty-acid conjugated peptide technology, initially targeting heavy menstrual bleeding – an often-ignored vital sign," said Sørensen. "It affects one in three women, causing pain, iron-deficiency anemia, fatigue, stigma, and extensive lost days at school and work, with treatment options largely unchanged in decades. HMB-003 is non-hormonal, potent, and selective, engineered to cover the period, then wash out between cycles."
Addressing Significant Unmet Needs
Von Willebrand Disease (搜索) is the most common inherited bleeding disorder, characterized by quantitative or qualitative defects in VWF, often resulting in frequent mucocutaneous bleeding events and heavy menstrual bleeding. Chronic blood loss frequently leads to iron deficiency anemia, exacerbating disease burden and reducing quality of life. Despite its prevalence, current treatment options primarily focus on managing symptoms rather than addressing the underlying biology of the disease.
Hemab's pipeline also includes sutacimig (搜索) (HMB-001), a bispecific antibody in clinical development for the prophylactic treatment of Glanzmann thrombasthenia and Factor VII deficiency. The company's expanding portfolio reflects a strategic focus on addressing critical gaps across the spectrum of coagulation disorders.