Trial Launched of SQ Monovalent Human Antibody Prophylaxis for All Types of Von Willebrand Disease

The first patient enrolled in the Velora Pioneer phase 1/2 clinical trial of HMB-002 for von Willebrand disease (VWD) has been administered the test agent. This was announced in a press release by Hemab Therapeutics, the international biotech company developing HMB-002.

HMB-002 is a first-in-class, monovalent human antibody that operates against VWD by increasing levels of von Willebrand factor and factor VIII in patients. Hemab has designed this agent to be a long-acting, subcutaneously administered prophylactic treatment for use in all types of the disease.

The Velora Pioneer trial is a clinical evaluation of HMB-002’s pharmacologic activity, efficacy, safety, and tolerability in patients with VWD, beginning with an assessment of single ascending doses of the agent in a controlled setting. Enrollment is ongoing, and interim data from the trial are expected to be reported later in 2025.

“Von Willebrand Disease has been documented for about 100 years. However, the current standard of care is sparse, ancient, and inconvenient. It’s time to leapfrog treatment into the 21st century, and we believe HMB-002 can become a new back-bone therapy for all types of Von Willebrand Disease,” commented Hemab chief executive officer Benny Sorensen, MD, PhD.¹

Regarding other clinical investigations Hemab is conducting into VWD, active recruitment is under way for its Velora Discover prospective study to gather data on bleeding, quality of life, and the treatments received among patients with VWD type 1, 2, or 3.² The company has also teamed up with bleeding disorders consultancy Haemnet Ltd. to perform the VWD 360 natural history study, compiling patients’ reports of life with the disease.¹

References

1. Hemab Therapeutics announces start of Velora Pioneer, a phase 1/2 clinical trial investigating HMB-002 for the treatment of von Willebrand disease. News release. PR Newswire. February 27, 2025. Accessed March 17, 2025. https://www.prnewswire.com/news-releases/hemab-therapeutics-announces-start-of-velora-pioneer-a-phase-12-clinical-trial-investigating-hmb-002-for-the-treatment-of-von-willebrand-disease-302387425.html
2. ClinicalTrials.gov. A study of bleeding and treatment in participants with von Willebrand disease. ID# NCT06610201. Last updated October 18, 2024. Accessed March 17, 2025. https://clinicaltrials.gov/study/NCT06610201