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The team at Royal London’s Haemophilia Centre has recruited the first patient in the world to the next stage of a clinical trial for Von Willebrand disease, the most common inherited bleeding disorder.

Von Willebrand disease affects the blood's ability to clot properly, and can cause heavy, hard-to-manage bleeding. It most often has an inherited genetic cause and affects 1 in 100-1000 people.

There are three types of the disease which range in severity, but common symptoms include bruising easily, frequent nosebleeds, bleeding gums and bleeding more than usual after an injury or cut.

The impact of Von Willebrand disease, like many bleeding disorders, is especially felt by women as it can often lead to menstrual periods with abnormally heavy or prolonged bleeding, and sometimes difficulties during pregnancy and childbirth.

Treatments are limited, either only targeting some types of the condition or requiring frequent intravenous infusion drips for the patient.

Velora Pioneer is a Phase 1/2 clinical trial being run by Hemab Therapeutics to test the drug HMB-002 for Von Willebrand disease. HMB-002 is an antibody drug designed to increase levels of clotting proteins in the blood, Von Willebrand Factor and Factor VIII, and is being investigated as a long-acting, preventative treatment for individuals with certain types of Von Willebrand disease. It is delivered by injection under the skin.

Recently, Hemab Therapeutics reported that in Part A of the trial , patients given a single dose of the drug saw levels of the two clotting proteins increase in a dose-dependent manner , with most reported side effects being mild to moderate . No blood clots, injection site reactions or allergic reactions were reported.

Part B of the trial, for which the team at Barts Health achieved a global first with the enrolment of the first patient worldwide, is testing multiple doses of the drug and additionally capturing bleeding event data.

Dr Priyanka Raheja, Haemophilia Centre Director and Chief Investigator on the trial at Royal London Hospital, Barts Health NHS Trust, said:

I'm incredibly proud of our team here at the Royal London for enrolling the world's first patient into this part of the trial. This is a real opportunity to investigate a potential new treatment specifically designed to prevent bleeds in Von Willebrand disease. I'm especially excited about what it could mean for women's health. Many women with heavy menstrual bleeding caused by the condition currently have no easily administered , targeted treatment options, so this has potential to make a big difference to their lives.

Benny Sørensen, MD, PhD, Chief Executive Officer of Hemab Therapeutics, said:

People living with bleeding disorders such as Von Willebrand disease and Factor VII deficiency continue to face a significant burden. Reducing it will take close collaboration between patients, clinicians, researchers and industry across the full range of bleeding disorders, and we are grateful to the team at the Royal London and the patients taking part in these trials.

Hemab Therapeutics is additionally running a trial at Barts Health for another rare bleeding disorder, Factor VII deficiency, one of the most common of the rare inherited clotting factor deficiencies . The Royal London Hospital was the first site in the world open for the Phase 2 trial , enrolling its first patient in April of this year. Led by Dr Suthesh Sivapalaratnam, the study aims to evaluate the safety of the investigational antibody drug sutacimig and whether it can increase Factor VII levels in people liv ing with the condition.

Read more about the Royal London Haemophilia Centre .

 

HMB-002 is not approved for the treatment of Von Willebrand disease and is an investigational agent in this indication, with no proven efficacy or safety. Sutacimig is not approved for the treatment of Factor VII deficiency and is an investigational agent in this indication, with no proven efficacy or safety.