Richmond Pharmacology completes first cohort dosing in Hemab Therapeutics Phase1/2 Clinical Trial Investigating HMB-002 for Von Willebrand disease

Announces enrolment underway for next trial phases

Richmond Pharmacology, one of the UK’s leading first-in-human clinical trials companies, has just completed the dosing in the first cohort in Velora Pioneer, a phase 1/2 clinical trial investigating Hemab Therapeutics’ HMB-002. This investigational product is a potential first-in-class subcutaneous therapy for Von Willebrand disease (VWD). The company also announced today that people with VWD are invited to register for the next parts of this clinical trial.

Von Willebrand disease is the most common inherited bleeding disorder, affecting over 12,000 people in the UK. The condition involves bleeding episodes that can range from frequent, low volume bleeds that impact daily living to potentially life-threatening events. Chronic blood loss frequently leads to iron deficiency anaemia, exacerbating the disease burden and reducing quality of life. Despite its prevalence, current treatment options for VWD primarily focus on reactively managing symptoms rather than addressing the underlying condition proactively.

HMB-002 is a monovalent antibody uniquely designed to increase levels of Von Willebrand Factor (VWF) and Factor VIII and provide a long-acting, subcutaneous prophylactic treatment for individuals with all types of VWD. Preclinical data for this investigational therapy were first presented at the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), showcasing its potential to stabilise VWF and promote a sustained correction of the blood clotting process.

The Phase 1/2 study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of HMB-002 in individuals with VWD. The first patient cohort has now been dosed at Richmond Pharmacology’s facilities in London under the supervision of Principal Investigator, Dr. Ulrike Lorch, and the study is actively enrolling further participants.

Dr Lorch said: “Von Willebrand affects over 12,000 people in the UK. We are delighted to be involved in a trial which may offer hope, not just for future generations of patients, but for those currently experiencing the day-to-day impacts of this often-debilitating condition. Anyone with VWD who is interested in taking part in our continuing trials can find more information on our Trials4Us website.”

Benny Sorensen, CEO of Hemab Therapeutics, said: “The HMB-002 clinical program underscores our unwavering commitment to reimagining blood clotting therapies for people living with a variety of underserved bleeding disorders. While Von Willebrand disease has been documented for about 100 years, the current standard of care is sparse, ancient, and inconvenient. It’s time to leapfrog treatment into the 21^st century, and we believe HMB-002 can become a new back-bone therapy for all types of Von Willebrand disease.”

Patient Enrolment Information

Patients interested in registering for the trial should go to Trials4Us

More information about this trial can be found at: https://clinicaltrials.gov/study/NCT06754852

The Hemab Therapeutics press release announcing the start of the trial can be found here.

-Ends-

Notes to Editor: 

About Von Willebrand Disease

Von Willebrand disease (VWD) is characterized by quantitative or qualitative defects in von Willebrand Factor (VWF), often resulting in frequent mucocutaneous bleeding events and heavy menstrual bleeding in women. Despite being the most common inherited bleeding disorder, current treatment options focus primarily on managing symptoms rather than addressing the underlying defect in VWF production or function.

About HMB-002

HMB-002 is a monovalent human antibody developed as the first-in-class prophylactic treatment for VWD, targeting the underlying root cause of the disease. By specifically targeting the C-terminal CK domain of VWF, HMB-002 shields the protein from degradation, boosting endogenous levels without compromising its function.

About Richmond 

Richmond is a leading UK contract research organisation, founded in 2001, specialising in first-in-human studies and adaptive clinical trials for gene editing and gene silencing therapies. In 2021 it was first in the world to treat a patient with the Nobel Prize-winning CRISPR-cas9 gene editing tool and currently has more patients undergoing cutting-edge genetic trials than any other clinic in the world. It has completed more than 500 early phase studies and helped deliver 30 medicines to market - including drugs for heart failure, hypertension and cancer. Clients include top 10 pharma, genetic engineering and biotech companies from around the world, especially Europe, the USA and Japan. In 2020, Richmond founded the Richmond Research Institute to advance understanding of under-researched diseases. 

About Hemab Therapeutics

Hemab is a multiple clinical-asset biotech company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in Cambridge, MA, and Copenhagen, Denmark, Hemab is progressing a pipeline of innovative therapeutic solutions, leveraging a variety of cutting-edge technologies and approaches to transform the treatment paradigm for patients with high unmet need. The company’s strategic guidance, Hemab 1-2-5^TM, targets building a pipeline of multiple development programs to deliver long-awaited innovation for patients with high unmet need blood-clotting disorders like Glanzmann thrombasthenia, Factor VII Deficiency, Von Willebrand disease, and others. Learn more at hemab.com.

For more information, please contact Linda Rose: linda@denhams.digital 07788 592842