Thrombasthenia

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, MA, USA I February 27, 2025 I 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. “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 21 st century, and we believe HMB-002 can become a new back-bone therapy for all types of Von Willebrand Disease.” 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 Disease Von 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-002 HMB-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 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 . Follow us on LinkedIn , Facebook , Instagram , and X . SOURCE: Hemab Therapeutics

Interim results demonstrate treatment with HMB-001 resulted in >50% reduction in treated bleeds at all tested dose levels In second oral presentation, Hemab presented first preclinical data for HMB-002, a potential novel treatment for Von Willebrand Disease Results from lived experience of Glanzmann thrombasthenia (GT360) and Von Willebrand Disease (VWD 360) natural history studies were included in two additional oral presentations COPENHAGEN, Denmark and CAMBRIDGE, Mass., Feb. 7, 2025 /PRNewswire/ -- Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today presented interim data from the ongoing evaluation of HMB-001, a novel bispecific antibody in development as first ever prophylactic treatment for the bleeding disorder Glanzmann thrombasthenia (GT). The Phase 2 study consists of a minimum 6-week prospective run-in where participants record bleeds via an electronic bleed diary, followed by 3 months of treatment with HMB-001. Interim efficacy data to date demonstrated >50% reduction in treated bleeds in all 3 tested dose cohorts. Underscoring its potential to address unmet medical needs and to expedite patient access, HMB-001 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration and the UK Medicines and Healthcare products Regulatory Agency has awarded it designation under the Innovative Licensing and Access Pathway. Hemab also unveiled compelling preclinical data for HMB-002, a potential novel treatment for Von Willebrand Disease (VWD). These data were featured as oral presentations at the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) held this week in Milan, Italy. "Glanzmann thrombasthenia is a serious and devastating bleeding disorder where patients often experience in excess of 60-80 treatment-requiring bleeds annually. These interim Phase 2 results signal the potential of a new reality for the Glanzmann thrombasthenia community, bringing us a step closer to a much-needed breakthrough in preventing bleeding," said Kate Madigan, Chief Medical Officer at Hemab. "The preclinical data for HMB-002 highlights its potential to specifically address the underlying patho-etiology and revolutionize care for the Von Willebrand Disease community, offering hope to prevent life-threatening complications and provide patients with a critical new lifeline." Interim pharmacokinetic (PK) data from the Phase 1/2 study of HMB-001 supports the use of weekly or biweekly dosing regimens. Pharmacodynamic (PD) responses are aligned with the mechanism of action, demonstrating a dose-dependent accumulation of activated Factor VII (FVIIa) and enhanced potentiation through TLT-1 binding. Efficacy assessments indicate a reduction of over 50% in treated bleeding episodes at all tested dose levels. In the single ascending dose (SAD) study, doses of 0.2, 0.5, and 1.25 mg/kg were evaluated, while the multiple ascending dose (MAD) study explored biweekly doses of 0.3, 0.6, and 0.9 mg/kg. The findings suggest a safe and effective dosing range of 0.3-0.6 mg/kg, while doses of 0.9 mg/kg and above were associated with elevated D-dimer levels and risk of venous thrombosis. These results provide valuable insights into optimizing dose and regimen for improved outcomes. "Bleeding disorders such as Glanzmann thrombasthenia present significant hardships, including unpredictable and life-altering bleeds, physical pain, social stigma, and emotional isolation. Despite these burdens, preventive treatments have yet to be developed," said Suthesh Sivapalaratnam, MD, PhD, Consultant Hematologist at Barts Health NHS Trust and Associate Professor at Queen Mary University of London. He adds: "With a bleed reduction of greater than 50%, the Phase 2 results highlight the transformative potential of HMB-001, offering people with Glanzmann thrombasthenia and providers a long-awaited breakthrough that could redefine care standards and bring renewed hope to a community in urgent need of innovation." "As a principal investigator in this study, I've seen firsthand how HMB-001 has the potential to significantly improve outcomes for people with Glanzmann thrombasthenia," said Laurent Frenzel, MD, PhD, Head of the Hemophilia Treatment and Research Center at the Necker-Enfants Malades Hospital (Paris Cité). "The Phase 2 data demonstrate promising efficacy and a manageable safety profile, representing a significant advancement in the treatment landscape. I look forward to seeing these results translate into a meaningful impact for those who have long faced limited options and unmet medical needs." Hemab plans to complete Phase 2 study recruitment in the first half of 2025. Hemab presented compelling preclinical data for HMB-002, a monovalent antibody uniquely designed to address VWD by increasing endogenous Von Willebrand Factor (VWF) levels and improving overall hemostasis. Unlike current treatments that often require frequent intravenous infusions or focus on symptom management, HMB-002 has the potential to provide a long-lasting, subcutaneous prophylactic solution that targets the root cause of VWD. Data demonstrate a promising safety and efficacy profile with high-affinity target-selective binding and preservation of key physiological functions of VWF when HMB-002 is bound. Administration of HMB-002 to cynomolgus monkeys resulted in a time-dependent accumulation of VWF, a corresponding rise in VWF activity, and a rise in the amount of another important blood clotting protein called Factor VIII (FVIII). The distribution of different sizes of VWF molecules (multimers) remained largely the same, suggesting that the activity levels of VWF were consistent. These results indicate that HMB-002, by boosting the levels of naturally occurring VWF, has the potential to be an effective, convenient and long-lasting preventive treatment option for people with VWD. The company is actively enrolling participants in VELORA-Discover, a prospective observational study for individuals with Type 1 VWD, to further understand the disease's natural history and support future clinical trials. Hemab's partner Haemnet, a specialist research and communications consultancy in the bleeding disorders community, also presented results from two natural history studies. The Glanzmann's 360 and Von Willebrand Disease 360 studies showcased the unmet needs and burdens the patients face every day. About Glanzmann Thrombasthenia Glanzmann thrombasthenia (GT) is a rare and severe bleeding disorder marked by debilitating, sometimes life-threatening bleeding episodes. Results from an international Glanzmann's 360 (GT360) natural history study revealed the substantial burden of this disease: 88% of the 117 participants reported at least one bleed in the previous week, with 34% of those bleeds requiring medical treatment. These bleeding episodes significantly impact patients' mental health and quality of life, with 67% reporting low mood, 52% reporting emotional problems, and 46% experiencing social isolation. Additionally, 81% of participants reported missing school or work due to bruising or bleeding. To date, there are no effective prophylactic treatment options for GT. About HMB-001 HMB-001 is a subcutaneously administered bispecific antibody that binds and stabilizes endogenous Factor VIIa with one antibody arm and binds to TLT-1 on activated platelets with the other arm. This allows for the accumulation of endogenous Factor VIIa in the body, recruitment of Factor VIIa directly to the surface of the activated platelets, where it is known to facilitate hemostatic plug formation. HMB-001 is designed to be a first-in-class prophylactic treatment for Glanzmann thrombasthenia (GT) with the potential to treat other debilitating rare bleeding disorders. The U.S. Food and Drug Administration granted Fast Track Designation and Orphan Drug Designation to HMB-001 for the treatment of GT while the UK The Medicines and Healthcare products Regulatory Agency has awarded it designation under the Innovative Licensing and Access Pathway (ILAP). For more information, please visit clinicaltrials.gov (NCT06211634). About Von Willebrand Disease Von 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. 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-002 HMB-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 Therapeutics Hemab is a clinical-stage 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, and X. Media Contact: Peg Rusconi [email protected] 617-910-6217 SOURCE Hemab Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

HMB-001 was well tolerated with no drug-related adverse or thromboembolic events; dose-dependent improvements in blood-clotting activity support the potential of HMB-001 as a first-in-class prophylactic treatment for Glanzmann Thrombasthenia and other blood clotting disorders Preclinical HMB-001 findings were published in the February 2024 issue of Nature Cardiovascular Research COPENHAGEN, Denmark and CAMBRIDGE, Mass., Feb. 9, 2024 /PRNewswire/ -- Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today presented initial results from Phase 1 of its ongoing evaluation of HMB-001. A novel bispecific antibody, HMB-001 is in development as a prophylactic treatment for the bleeding disorder Glanzmann Thrombasthenia (Glanzmann). The data were presented as one of 10 abstracts selected for the SLAM oral presentation session at the 17th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) held this week in Frankfurt, Germany. "The first clinical data for HMB-001 in Glanzmann suggest that the demonstrated mechanism is suitable as a new prophylactic treatment for people with neglected blood clotting disorders who face severe, potentially life-threatening bleeds every day," said Joe Vogel, Senior Director and Program Lead for HMB-001 at Hemab. "With Phase 2 already underway, we are committed to advancing clinical evaluation of HMB-001 toward bringing life-changing preventative treatments to improve patients' quality of life." The Phase 1/2 first-in-human open-label study in patients with Glanzmann is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of HMB-001. As part of the UK-based Phase 1 single-ascending dose, seven patients received subcutaneous HMB-001 at 0.2 mg/kg, (n=1), 0.5 mg/kg (n=3) or 1.25 mg/kg (n=3). Over a 56-day observation period, HMB-001 was well tolerated; the most common adverse events (AEs) were mild or moderate in severity. No AEs or serious adverse events were deemed related to HMB-001. There were no dose-limiting toxicities and no thromboembolic events reported. Treatment with HMB-001 resulted in a dose-dependent pharmacodynamic effect of endogenous Factor VIIa accumulation that was associated with decreases in prothrombin time and improvement in exploratory thrombin generation analyses. The pharmacokinetic profile and half-life of HMB-001 support dosing every two weeks or a less-frequent dosing regimen. "People living with bleeding disorders like Glanzmann face devastating bleed events and considerable psychosocial impacts—from severe pain and social stigmatization to depression and isolation—but have no approved preventative treatments to turn to," said Suthesh Sivapalaratnam, MD, PhD, Queen Mary University of London, Barts Health NHS Trust. "These Phase 1 data support further investigation into the promise of HMB-001 for patients and providers across the globe who are awaiting innovation to improve the standard of care." Hemab has begun dosing and is actively enrolling in Phase 2, the multiple-ascending dose of the study, which will be expanded to sites across Europe and the U.S. For more information, visit clinicaltrials.gov (NCT06211634). In addition, Hemab announced that preclinical research findings of HMB-001 are now available online as part of the February 2024 issue of Nature Cardiovascular Research. The study results demonstrate the ability of HMB-001 to accumulate and potentiate the activity of endogenous Factor VIIa selectively on activated platelets, providing a sustained and localized procoagulant activity that may support prophylactic treatment of Glanzmann or other bleeding disorders. Read the paper here. About Glanzmann Thrombasthenia Glanzmann Thrombasthenia (Glanzmann) is a rare and severe bleeding disorder associated with debilitating and sometimes life-threatening bleeding episodes. Initial findings from the international Glanzmann's 360 (GT360) natural history study—Hemab's research initiative in partnership with UK specialist research consultancy Haemnet—found that 87% of the 104 respondents reported experiencing at least one bleed in the previous week, with 37% of those bleeds requiring medical treatment. These bleeding episodes significantly impact the mental health and quality of life of people living with Glanzmann. Low mood, emotional problems, and social isolation were reported by participants (66%, 50%, and 44% respectively), and 80% reported that they missed school or work due to bruising or bleeding. To date, there are no effective prophylactic treatment options for Glanzmann. About HMB-001 HMB-001 is bispecific antibody that binds and stabilizes endogenous Factor VIIa with one antibody arm and binds to TLT-1 on activated platelets with the other arm. This allows for accumulation of endogenous Factor VIIa in the body, recruitment of Factor VIIa directly to the surface of the activated platelets where it is known to facilitate clotting, and avoidance of clotting activity in the absence of tissue damage. HMB-001 is designed to be a first-in-class prophylactic treatment for Glanzmann Thrombasthenia with the potential to treat other debilitating rare bleeding disorders. The U.S. Food and Drug Administration granted Fast Track Designation to HMB-001 for the treatment of Glanzmann. About Hemab Therapeutics Hemab is a clinical-stage 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 monoclonal and bispecific antibody-based therapeutics to transform the treatment paradigm for patients with high unmet need. The company's strategic guidance, Hemab 1-2-5™, targets the development of 5 assets by 2025 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 and X (formerly known as Twitter). SOURCE Hemab Therapeutics