HMB-002

HMB-002 is a monovalent human antibody developed by Hemab Therapeutics as a first-in-class prophylactic treatment for von Willebrand disease (VWD), a bleeding disorder caused by deficiency or dysfunction of von Willebrand Factor (VWF). Unlike current therapies that focus on symptom management or require frequent intravenous infusions, HMB-002 is designed to be administered subcutaneously and targets the root cause of VWD. Its mechanism involves binding to the C-terminal CK domain of VWF, shielding it from degradation and thereby increasing endogenous levels of both VWF and Factor VIII (FVIII) without compromising their physiological functions. This approach aims to provide long-lasting correction or substantial modification of bleeding tendency in people with all types of VWD. Preclinical studies have shown that administration leads to increased levels and activity of both VWF and FVIII, with preservation of normal multimer distribution. The drug is currently being evaluated in Phase 1/2 clinical trials for safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy in individuals with VWD[1][2][4][5][6].