Clinical Read2026-07-13

HMB-002 - (VELORA Discover)

Hemab Therapeutics Holdings Inc.COAG
Indication
Von Willebrand Disease (VWD)
Stage
phase1
Event
Initial Data
Details

July 12, 2026: Hemab Therapeutics (NASDAQ: COAG) presented new clinical data for HMB-002 in von Willebrand disease (VWD) and announced its HMB-003 program at the ISTH 2026 Congress (Paris).

HMB-002 (VWD; first-in-human SAD): The company reported proof-of-mechanism data showing dose-dependent increases in endogenous von Willebrand factor (VWF) and Factor VIII (FVIII). In cohort A3 (150 mg), peak VWF and FVIII increased ≥2.4-fold, with normalization/restoration of thrombin generation and shortening of activated partial thromboplastin time (APTT), and stable multimer distribution. PK/PD supported potential monthly subcutaneous dosing. Safety was described as favorable across cohorts: mostly mild-to-moderate TEAEs, no serious TEAEs, no discontinuations, and no thromboembolic events, injection site reactions, thrombocytopenia, or hypersensitivity reactions. Descriptive clinical observations (not efficacy endpoints): 8/9 evaluable patients had zero treated bleeds in the 28 days post-dose; mean annualized treated bleed rate (ATBR) 1.6 vs baseline mean ATBR 20.1 (based on bleed data collected up to 5.5 months).

HMB-003 (program announcement; heavy menstrual bleeding initial focus): Introduced as a non-hormonal, fatty-acid-conjugated peptide direct plasmin inhibitor antifibrinolytic. Preclinical data indicated potent, selective inhibition of plasmin and no observed effects on thrombin generation, platelet function, or coagulation in nonclinical studies; in minipigs, a single subcutaneous dose sustained antifibrinolytic activity for ~1 week.