Experimental VWD treatment boosts clotting factors in clinical trial
Enrolling continues to test higher and repeated doses of HMB-002
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HMB-002, an experimental antibody-based treatment from Hemab Therapeutics for von Willebrand disease (VWD), increased levels of von Willebrand factor (VWF) and improved measures of blood clotting in a clinical trial.
That’s according to initial data from the Phase 1/2 Velora Pioneer study (NCT06754852), which showed that a single dose of the treatment increased the levels of VWF and factor VIII , two proteins involved in blood clotting, by more than two times.
The results were presented at this year’s International Society on Thrombosis and Haemostasis (ISTH) Congress, held in July in Paris. The company expects to report additional data from the trial later this year or in early 2027. The trial is still enrolling people with VWD at multiple locations in the U.S., U.K., and Australia.
“We had a significant presence at ISTH 2026, presenting data from all our programs. The strong interest we saw in that data underscores both the significant unmet need in bleeding disorders and the potential for our clinical and preclinical pipeline to redefine the standard of care in this space,” Benny Sørensen, MD, PhD, Hemab’s CEO, said in a company press release.
HMB-002 designed to bind to and stabilize VWF
VWD is caused by mutations in the gene that encodes VWF, resulting in low levels or activity of this protein. As a result, the body cannot properly form blood clots, leading to symptoms such as easy bruising or prolonged bleeding.
HMB-002 is designed to bind to and stabilize VWF, with the goal of increasing VWF levels and helping to restore normal blood clotting. Preclinical studies demonstrated that the treatment can bind to the VWF protein tightly without interfering with its binding to other proteins needed for blood clotting. It also allowed the protein to stay in the body for longer.
The Velora Pioneer study is assessing HMB-002’s safety, tolerability, pharmacological properties, and efficacy at single or multiple doses. More than 100 patients, ages 16 to 69, are expected to enroll. The results now available refer to 13 patients (mean age 35 years) who received a single dose of the therapy, at 20 mg, 50,mg, and 150 mg, via subcutaneous (under the skin) injections.
Among these 13 participants, most (85%) had VWD type 1, the most common disease type characterized by low levels of VWF, and two had VWD type 1C, in which VWF is produced normally but is cleared from the blood too quickly.
HMB-002 was generally safe, with no serious treatment-emergent adverse events reported. Participants experienced a dose-dependent increase in HMB-002 blood levels, which peaked six to nine days after administration. The therapy remained detectable for almost two months in participants who received the highest dose, supporting the potential for monthly dosing, according to the company.
Improvements seen in measures of blood clotting
Results also showed increases in VWF levels and FVIII activity, along with improvements in measures of blood clotting. These included normalized production of thrombin, a key enzyme in blood clotting, and a reduction in activated partial thromboplastin time, a measure of how quickly blood clots.
The annualized bleeding rate was also lower among participants who received HMB-002 than among those in a natural history study called Velora Discover (NCT06610201). The mean annualized bleeding rate was 1.6 events among HMB-002-treated participants, compared with 20.1 events in the natural history group.
While two of 10 patients in Velora Discover had no reported bleeding events during approximately 5.5 months of follow-up, eight of nine participants in Velora Pioneer reported no bleeding events over four weeks.
The trial is currently enrolling patients into an additional group receiving a single 300 mg dose of HMB-002, a multiple-dose part testing 150 mg doses given every four weeks, and a third part evaluating HMB-002 in combination with clotting factor concentrates.
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