Abstracts - 2022 - Haemophilia
Hemab Therapeutics Announces Start Of Velora Pioneer, A Phase 1/2 Clinical Trial Investigating HMB-002 For The Treatment Of Von Willebrand Disease
First participant with Von Willebrand Disease dosed with HMB-002, a novel subcutaneous therapy designed to increase endogenous von Willebrand Factor and Factor VIII levels
COPENHAGEN, Denmark and CAMBRIDGE, Mass., Feb. 27, 2025 /PRNewswire/ -- Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today announced the first participant with Von Willebrand Disease (VWD) has been dosed in Velora Pioneer, a Phase 1/2 clinical trial investigating HMB-002, a potential first-in-class subcutaneous therapy for VWD.
Hemab Therapeutics (PRNewsfoto/Hemab Therapeutics)"We are excited to advance the clinical development of HMB-002 for Von Willebrand Disease," said Catherine Rea, MD, PhD, Vice President of Clinical Development at Hemab. "HMB-002 is the first fixed-dose subcutaneously administered antibody-based preventative therapy aiming to transform daily life for patients."
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 target the underlying patho-etiology of VWD by stabilizing VWF and promoting sustained hemostatic correction.
"Von Willebrand Disease is characterized by a deficiency in VWF activity and sometimes associated with lowered levels of FVIII – both essential to control severe bleeding," said Dr. Priyanka Raheja, the Royal London Hospital, Barts Health NHS Trust. "HMB-002 functions by increasing VWF and FVIII and could offer a complete disease correction for some and substantial modification for bleeding tendency for others. I am excited to be a contributing investigator on the Phase 1/2 trial."
The Phase 1/2 study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of HMB-002 in individuals with VWD. The initial part of the study will evaluate single ascending doses of HMB-002 in a controlled setting. The first participant was dosed at Richmond Pharmacology in London under the supervision of Principal Investigator, Dr. Ulrike Lorch. The study is actively enrolling participants, with interim data anticipated later this year.
"The initiation of the HMB-002 clinical program establishes Hemab as a multi-asset clinical-stage biotech and underscores our unwavering commitment to reimagine blood clotting therapies for people living with a variety of underserved bleeding disorders," said Benny Sorensen, MD, PhD, CEO of Hemab. "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."
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In addition to Velora Pioneer, Hemab is also conducting Velora Discover, a prospective natural history study collecting data on bleeds, treatments, and quality-of-life elements in people with VWD. Hemab is partnered with Haemnet, a specialist research and communications consultancy in the bleeding disorders community, to conduct VWD 360, the largest-ever natural history study in all types of VWD based on people's detailed reported lived experience. Recent initial results were shared at EAHAD 2025.
About Von Willebrand DiseaseVon Willebrand Disease (VWD) is the most common inherited bleeding disorder, characterized by quantitative or qualitative defects in Von Willebrand Factor (VWF), often resulting in frequent mucocutaneous bleeding events and heavy menstrual bleeding in women. The severity of bleeding ranges from low-volume events to potentially life-threatening hemorrhages. Chronic blood loss frequently leads to iron deficiency anemia, exacerbating the disease burden and reducing quality of life, particularly for those with clinically understated subtypes. Despite its prevalence, current treatment options for VWD primarily focus on managing symptoms rather than addressing the underlying defect in VWF production or function.
About HMB-002HMB-002 is a monovalent human antibody developed as the first-in-class prophylactic treatment for Von Willebrand Disease targeting the underlying root cause of the disease, a condition driven by a deficiency or defect in Von Willebrand Factor (VWF), a key regulator of hemostasis. By specifically targeting the C-terminal CK domain of VWF, which is distinct from regions critical to its essential interactions, HMB-002 shields the protein from degradation, boosting endogenous levels without compromising its function. Preclinical data suggest strong potential for meaningful therapeutic benefit. For more information, please visit clinicaltrials.Gov (NCT06610201).
About Hemab TherapeuticsHemab 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 1-2-5TM, 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. Follow us on LinkedIn, Facebook, Instagram, and X.
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SOURCE Hemab Therapeutics
Humate-p
Humate-p Generic Name & Formulations General DescriptionAntihemophilic Factor VIII/Von Willebrand Factor Complex (human) 250 IU FVIII + 600 IU VWF, 500 IU FVIII + 1200 IU VWF, 1000 IU FVIII + 2400 IU VWF; per vial; lyophilized pwd for IV infusion after reconstitution; contains albumin.
Pharmacological ClassCoagulation factor complex.
How SuppliedSingle-use vials—1 (w. Diluent, supplies)
Humate-p Indications IndicationsTreatment and prevention of bleeding in adults with Hemophilia A. Treatment of spontaneous and trauma-induced bleeding, and prevention of excessive bleeding during and after surgery in adults and children with von Willebrand disease (VWD).
Humate-p Dosage and Administration AdultMax injection rate: 4mL/min. Hemophilia A: Minor bleed: 15 IU FVIII/kg (obtain 30% FVIII increase) once; if needed, may give ½ dose once or twice daily for 1–2 days. Moderate bleed: initially 25 IU FVIII/kg (obtain 50% FVIII increase), then 15 IU FVIII/kg (maintain 30% FVIII increase) every 8–12hrs for 1–2 days, then repeat dose for 1–2 times daily for a total of 7 days or until healed. Severe bleed: initially 40–50 IU FVIII/kg, then 20–25 IU FVIII/kg every 8hrs (maintain 80–100% FVIII increase) for 7 days, then repeat dose for 1–2 times daily for additional 7 days (maintain 30–50% FVIII increase). VWD: Type 1 (Mild): major bleed: initially 40–60 IU/kg, then 40–50 IU/kg every 8–12hrs for 3 days, then once daily for a total of 7 days. Type 1 (Moderate or severe): minor bleed: 40–50 IU/kg for 1–2 doses; major bleed: initially 50–75 IU/kg, then 40–60 IU/kg every 8–12hrs for 3 days, then once daily for a total of 7 days. Types 2 and 3: minor bleed: 40–50 IU/kg for 1–2 doses; major bleed: initially 60–80 IU/kg, then 40–60 IU/kg every 8–12hrs for 3 days, then once daily for a total of 7 days. For dosing in surgery: see literature.
ChildrenMax injection rate: 4mL/min. VWD: Type 1 (Mild): major bleed: initially 40–60 IU/kg, then 40–50 IU/kg every 8–12hrs for 3 days, then once daily for a total of 7 days. Type 1 (Moderate or severe): minor bleed: 40–50 IU/kg for 1–2 doses; major bleed: initially 50–75 IU/kg, then 40–60 IU/kg every 8–12hrs for 3 days, then once daily for a total of 7 days. Types 2 and 3: minor bleed: 40–50 IU/kg for 1–2 doses; major bleed: initially 60–80 IU/kg, then 40–60 IU/kg every 8–12hrs for 3 days, then once daily for a total of 7 days. For dosing in surgery: see literature.
Humate-p Contraindications ContraindicationsPrevious anaphylactic or severe systemic response to antihemophilic factor or von Willebrand factor preparations.
Humate-p Boxed WarningsNot Applicable
Humate-p Warnings/Precautions Warnings/PrecautionsConfirm Factor VIII or von Willebrand factor deficiency prior to treatment. Increased risk of thromboembolic events in VWD. Contains human plasma; monitor for possible infection transmission. Large or frequent doses: monitor hematocrit for signs of hemolytic anemia. Monitor for development of inhibitors. Pregnancy (Cat.C).
Humate-p PharmacokineticsSee Literature
Humate-p InteractionsNot Applicable
Humate-p Adverse Reactions Adverse ReactionsAllergic reaction, GI upset, inj site reactions, mild vasodilation, pruritus, paresthesia, peripheral edema, antibody formation; anaphylaxis, thrombosis.
Humate-p Clinical TrialsSee Literature
Humate-p Note NotesReport all infections suspected to be transmitted by Humate-P to (800) 504–5434.
Humate-p Patient CounselingSee Literature
NICE Recommends Altuvoct As NHS Treatment Option For Hemophilia A
The U.K.'s National Institute for Health and Care Excellence (NICE) has recommended Altuvoct (efanesoctocog alfa) be available through the National Health Service (NHS), England's public healthcare system, as a treatment option to manage and prevent bleeds in people with severe hemophilia A, ages 2 and older.
This decision came shortly after the treatment's approval last month by the country's Medicines and Healthcare products Regulatory Agency. The therapy already was approved in the European Union under the same brand name. It's also been approved in the U.S., where it's marketed as Altuviiio.
"[This] decision … shows how, by collaborating with system partners we can quickly recommend treatments that deliver tangible benefits for patients and the NHS alongside value-for-money for taxpayers," Helen Knight, director of medicines evaluation at NICE, said in an institute press release.
According to NICE, Altuvoct, with its weekly dosing regimen, offers patients an alternative to other available therapies that are given several times per week.
"Current factor VIII replacements can be difficult to manage due to the need for frequent dosing to prevent potentially life-threatening and debilitating bleeding episodes. … [Altuvoct] only has to be taken once a week," Knight said. "Combined with its effective bleeding control, it has the potential to have a significant positive impact for some people with severe [hemophilia] A."
Altuvoct to be available through NHS as on-demand and preventive treatmentJames Palmer, NHS England's medical director for specialized services, called the decision "an important step forward in treatment for patients," saying it "ensures they continue to have access to the latest advances in care on the NHS to help prevent bleeding episodes."
Hemophilia A is caused by mutations in the F8 gene that lead to a lack or dysfunction of a protein called factor VIII, or FVIII, which is crucial in the blood clotting process. This typically results in episodes of excessive bleeding and easy bruising that may occur spontaneously or as a result of injury or trauma.
Standard treatment generally involves replacement therapies, which supply a version of the missing or dysfunctional FVIII to patients to treat or prevent bleeds.
Altuvoct is a new type of hemophilia A replacement therapy that contains a lab-made version of FVIII fused to a fragment of von Willebrand factor — another clotting protein that helps stabilize and prevent FVIII degradation. In addition, the therapy has two other modifications that help it stay active in the body for longer periods of time.
Together, these features enable Altuvoct to have a more sustained presence in the bloodstream, and therefore be administered less frequently when used as a prophylactic treatment, or to prevent bleeds.
The therapy, when used as a prophylaxis, is recommended to be given once weekly at a dose of 50 international units per kilogram of body weight, via an intravenous, or into-the-vein, injection. It also may be used as an on-demand therapy for active bleeds, including those occurring during surgery.
It's fantastic news that this time-saving therapy will now be available to help improve patients' lives, reinforcing the NHS' commitment to providing innovative treatments at value for the taxpayer.
The therapy's approval in the U.K. Was based on data from the Phase 3 XTEND-1 trial (NCT04161495), which enrolled 159 people with severe hemophilia A. Two U.K. Sites were among the study's 47 locations.
The results showed that 65% of patients who received weekly injections of Altuvoct had no bleeding episodes over the course of one year of treatment. Altuvoct also eased pain and effectively treated bleeding episodes when used on demand.
Similar results were seen in the Phase 3 XTEND-Kids trial (NCT04759131), which also included the two U.K. Study sites. That trial enrolled children younger than 12 with severe hemophilia A.
"This condition can have really debilitating and painful impacts and it's fantastic news that this time-saving therapy will now be available to help improve patients' lives, reinforcing the NHS' commitment to providing innovative treatments at value for the taxpayer," Palmer said.
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