HMB-002 / Hemab Therapeutics - LARVOL DELTA

A Study Assessing HMB-002 in Participants With Von Willebrand Disease (clinicaltrials.gov) - P1/2 | N=108 | Recruiting | Sponsor: Hemab ApS | Not yet recruiting ➔ Recruiting

Enrollment open • Hemophilia

Hemab Therapeutics Announces Start of Velora Pioneer, a Phase 1/2 Clinical Trial Investigating HMB-002 for the Treatment of Von Willebrand Disease (PRNewswire) - "Hemab Therapeutics...announced the first participant with Von Willebrand Disease (VWD) has been dosed in Velora Pioneer, a Phase 1/2 clinical trial investigating HMB-002, a potential first-in-class subcutaneous therapy for VWD....The Phase 1/2 study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of HMB-002 in individuals with VWD. The initial part of the study will evaluate single ascending doses of HMB-002 in a controlled setting. The first participant was dosed at Richmond Pharmacology in London under the supervision of Principal Investigator, Dr. Ulrike Lorch. The study is actively enrolling participants, with interim data anticipated later this year."

P1/2 data • Trial status • Hematological Disorders

Hemab Therapeutics Presents…reclinical Data from HMB-002 in Von Willebrand Disease at the 2025 EAHAD Annual Congress (PRNewswire) - "Data demonstrate a promising safety and efficacy profile with high-affinity target-selective binding and preservation of key physiological functions of VWF when HMB-002 is bound. Administration of HMB-002 to cynomolgus monkeys resulted in a time-dependent accumulation of VWF, a corresponding rise in VWF activity, and a rise in the amount of another important blood clotting protein called Factor VIII (FVIII). The distribution of different sizes of VWF molecules (multimers) remained largely the same, suggesting that the activity levels of VWF were consistent. These results indicate that HMB-002, by boosting the levels of naturally occurring VWF, has the potential to be an effective, convenient and long-lasting preventive treatment option for people with VWD."

Preclinical • Hematological Disorders