Hemab Therapeutics Secures $157 Million Series C to Advance First-in-Class Treatments for Rare Bleeding Disorders
核心洞察
Hemab Therapeutics (搜索) completed a $157 million Series C financing led by Sofinnova Partners (搜索) to advance breakthrough treatments for underserved bleeding disorders including Glanzmann thrombasthenia (搜索) and Von Willebrand disease (搜索).
The funding enables advancement of sutacimig (搜索), the first-ever prophylactic treatment for Glanzmann thrombasthenia (搜索), to registration studies in 2026 following successful Phase 2 completion.
HMB-002, targeting Von Willebrand disease (搜索)'s underlying pathophysiology by increasing both Von Willebrand Factor and Factor VIII (搜索) levels, will also advance toward registration studies.
Hemab Therapeutics (搜索) announced the completion of a multifold oversubscribed $157 million Series C financing round to advance novel prophylactic treatments for serious, underserved bleeding disorders. The round was led by Sofinnova Partners (搜索) with substantial participation from a large long-only global asset management company, a large global sovereign wealth fund, and Avoro Capital Advisors, alongside existing investors including RA Capital Management (搜索), Novo Holdings (搜索), and others.
The Cambridge, Massachusetts and Copenhagen-based clinical-stage biotechnology company is developing breakthrough treatments for rare bleeding disorders that have historically lacked effective prophylactic options. The financing positions Hemab to advance multiple drug candidates through registration studies and expand its clinical pipeline.
Advancing First-in-Class Treatment for Glanzmann Thrombasthenia
The funding follows successful completion of sutacimig (搜索)'s Phase 2 study in Glanzmann thrombasthenia (搜索) (GT), with planned advancement to a registration study in 2026. Sutacimig represents the first-ever prophylactic treatment for GT, a severe bleeding disorder marked by debilitating, sometimes life-threatening bleeding episodes.
Results from Hemab's international 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. The 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.
"As a mother of two boys with GT, I live with a constant fear, knowing from experience that a simple fall or injury can lead to a life-threatening bleed," said Alexandra Sullivan, patient advocate and mother. "We feel so limited by the lack of effective treatments in the event of an emergency and cannot stress enough how urgently we feel the need for breakthrough treatments."
Sutacimig (搜索) is a subcutaneously administered bispecific antibody that binds and stabilizes endogenous Factor VIIa with one antibody arm and binds to TLT-1 (搜索) on activated platelets with the other arm. This mechanism allows for the accumulation of endogenous Factor VIIa in the body and recruitment of Factor VIIa directly to the surface of activated platelets, where it facilitates hemostatic plug formation. The U.S. Food and Drug Administration granted Fast Track Designation and Orphan Drug Designation to sutacimig for GT treatment, while the UK Medicines and Healthcare products Regulatory Agency awarded it designation under the Innovative Licensing and Access Pathway.
Targeting Von Willebrand Disease's Root Cause
The financing also supports advancing HMB-002 towards a registration study for Von Willebrand Disease (搜索) (VWD), the most common inherited bleeding disorder. HMB-002 is Hemab's antibody-based treatment with Phase 1 proof of mechanism data showing that it directly targets the underlying pathophysiology of VWD by increasing both Von Willebrand Factor and Factor VIII (搜索) levels.
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. 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. Despite its prevalence, current treatment options for VWD primarily focus on managing symptoms rather than addressing the underlying defect in VWF production or function.
HMB-002 is a monovalent human antibody being 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, which is distinct from regions critical to its essential interactions, HMB-002 shields the protein from degradation, boosting endogenous levels without compromising its function.
"For too long, people living with von Willebrand Disease (搜索) have watched from the sidelines as breakthrough prophylactic therapies transformed hemophilia care," said Jeanette Cesta, Executive Director, VWD Connect Foundation. "HMB-002 represents hope: in its potential to be the kind of innovative, patient-centered treatment our community has been waiting for."
Pipeline Expansion and Strategic Vision
The additional capital enables Hemab to advance additional novel drug candidates into clinical development, with HMB-003 expected to be announced in H1 2026. From inception, Hemab's program development has been shaped by a portfolio of groundbreaking natural history studies including GT360, FVIID360 and VWD360, which have challenged historical assumptions and uncovered the overlooked burden of these diseases.
"The quality of our investor syndicate and this significant financing validate our approach and enable us to continue building what we believe will become the ultimate clotting company," said Benny Sorensen, MD, PhD, CEO of Hemab. "We carefully listen to people living with clotting diseases and collaborate closely with patients and families affected by these conditions to deliver 21st century treatment options."
Joe Anderson, PhD, Partner at Sofinnova Partners (搜索), who will join the Hemab Board in connection with this financing, commented: "Hemab's focus on patients and an innovative approach to treating neglected bleeding disorders, combined with their deep scientific excellence and strong clinical execution, makes it a standout company in the biotech landscape."
The company's strategic guidance, Hemab 1-2-5™, targets building a pipeline of development programs to deliver long-awaited innovation for people with high unmet need diseases like Glanzmann thrombasthenia (搜索), Factor VII Deficiency (搜索), Von Willebrand Disease (搜索), and others.