HMB-002 / Hemab Therapeutics - LARVOL DELTA

Velora pioneer, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of HMB-002 for prophylaxis in von Willebrand disease: A trial in progress (ASH 2025) - P, P1/2 | "Desmopressin provides only transient benefit and is limited by tachyphylaxis, while plasma-derived or recombinant VWF/FVIII concentrates require frequent, burdensome intravenous infusions. Documented residual VWF levels are required, and exclusion criteria include concurrent participation in interventional trials or medical conditions confounding study results. Part B requires participants to have completed the VELORA Discover observational study."

Clinical • P1/2 data • PK/PD data • Hematological Disorders • Hemophilia

A prospective screening study in participants with Von Willebrand Disease: the VELORA Discover study (ISTH 2025) - P | "The findings will inform the design of interventional studies, including VELORA Pioneer, a Phase 1/2 first-in-human trial of HMB-002, an investigational therapy aimed at reducing bleeding severity and frequency in VWD...Results Data collection is ongoing. Results will include detailed disease characteristics, bleed frequency and severity, quality-of-life outcomes, and further insight into menstrual bleeding patterns."

Clinical • Anemia • Hematological Disorders • Hemophilia

Favorable nonclinical safety profile of HMB-002 for prophylactic treatment of Von Willebrand disease (ISTH 2025) - "Furthermore, no HMB-002-related immunotoxicity—such as complement and platelet activation, cytokine release, or Fc gamma receptor binding—was observed in vivo in cynomolgus monkeys, ex vivo in human whole blood, and in human in vitro systems, implying that HMB-002 does not have effects on the immune system. Ex vivo and in vitro safety studies confirmed HMB-002 does not exhibit off-target binding in humans."

Clinical • Anemia • Hematological Disorders • Hemophilia

VELORA Pioneer: first-in-human safety and PK/PD study of HMB-002 in Type 1 Von Willebrand disease (ISTH 2025) - No abstract available

Clinical • Late-breaking abstract • P1 data • PK/PD data • Hemophilia

HMB-002: elevating von Willebrand Factor for prophylactic treatment of Von Willebrand Disease (ISTH 2025) - "Studies in cynomolgus monkeys revealed a time-dependent accumulation of VWF with proportional increases in VWF:RCo and FVIII and a maintained VWF multimer distribution. Administration of rVWF five days post HMB-002 dosing resulted in an approximately 3-fold extension of rVWF half-life and with elevated levels of endogenous VWF maintained after washout of rVWF."

Anemia • Hematological Disorders • Hemophilia

Excitement builds for ISTH 2025 Congress in Washington, D.C. as late-breakthrough abstracts are announced (PRNewswire) - "The International Society on Thrombosis and Haemostasis (ISTH) prepares to welcome nearly 5,000 attendees from the global thrombosis and hemostasis community to the ISTH 2025 Congress in Washington, D.C....A key highlight of the ISTH 2025 Congress, taking place from June 21-25, 2025, is the presentation of late-breakthrough abstracts, offering new data from major clinical trials and breakthrough studies, giving attendees a first look at transformative findings."

Clinical data • Hemophilia A • Immune Thrombocytopenic Purpura • Venous Thromboembolism

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