Hemab Therapeutics Launches Velora Pioneer Trial for Von Willebrand Disease

COPENHAGEN, Denmark and CAMBRIDGE, MA, USA — February 27, 2025

Hemab Therapeutics, a biotechnology company focused on developing innovative therapies for bleeding and thrombotic disorders, has announced a milestone in its clinical trials. The first participant with Von Willebrand Disease (VWD) has been dosed in the Velora Pioneer clinical study, which is a Phase 1/2 trial examining HMB-002. This therapy is a potential breakthrough in subcutaneous treatments for VWD.

Dr. Catherine Rea, Vice President of Clinical Development at Hemab, expressed enthusiasm about the progress. She highlighted that HMB-002 represents the first fixed-dose, antibody-based therapy administered subcutaneously with the aim of transforming patient care. The therapy is designed to enhance the levels of von Willebrand Factor (VWF) and Factor VIII (FVIII), providing a long-term preventative solution for those with VWD.

HMB-002 is a monovalent antibody specifically developed to boost endogenous VWF and FVIII levels. It seeks to offer a long-acting, subcutaneous prophylactic treatment for various forms of VWD. The initial findings, presented at the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), revealed its potential to address the root causes of VWD by stabilizing VWF and aiding in hemostatic correction.

Dr. Priyanka Raheja from the Royal London Hospital emphasized the significance of HMB-002. VWD is characterized by deficiencies in VWF activity and, at times, reduced FVIII levels, both crucial for managing severe bleeding. HMB-002 works by increasing VWF and FVIII, potentially offering comprehensive disease correction or significant reduction in bleeding tendencies for patients. Dr. Raheja is actively involved as an investigator in the Phase 1/2 trial.

The trial aims to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of HMB-002 in VWD patients. The initial phase involves controlled single ascending doses of HMB-002. The first participant was treated at Richmond Pharmacology in London, with Dr. Ulrike Lorch as the Principal Investigator. Participant recruitment is ongoing, and interim results are expected later in the year.

Dr. Benny Sorensen, CEO of Hemab, commented on the significance of this clinical program. The initiation of the HMB-002 trial signifies Hemab's commitment to revolutionizing blood clotting treatments for underserved bleeding disorders. Despite being recognized for about a century, current VWD treatments remain limited and outdated. Dr. Sorensen believes HMB-002 could become a cornerstone therapy for all VWD types.

In addition to the Velora Pioneer trial, Hemab is conducting the Velora Discover study. This prospective natural history study gathers data on bleeding events, treatments, and quality of life for those with VWD. Hemab collaborates with Haemnet to carry out VWD 360, the largest natural history study focusing on all VWD types based on patients' reported experiences. Initial findings were recently shared at EAHAD 2025.

Von Willebrand Disease is the most prevalent inherited bleeding disorder, marked by issues with VWF, often leading to frequent bleeding episodes and heavy menstrual bleeding in women. The severity ranges from minor bleeding to life-threatening hemorrhages. Chronic blood loss often results in iron deficiency anemia, worsening the disease's impact and reducing quality of life, particularly for those with mild subtypes. Current treatments mainly manage symptoms without addressing the underlying VWF issue.

HMB-002 is a pioneering prophylactic therapy targeting the root cause of VWD, which is driven by deficiencies or defects in VWF. By targeting the C-terminal CK domain of VWF, HMB-002 prevents protein degradation, increasing VWF levels without affecting its function. Preclinical data indicates significant therapeutic potential for patients with VWD.