Hemab Therapeutics recently presented clinical and preclinical stage data associated with two of their investigational therapies - Sutacimig (formerly HMB-001) and HMB-002. Hemab is a biotechnology company that specializes in the development of therapies that target underserved bleeding and thrombotic disorders. The data were presented last week at the Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Washington, DC.
Sutacimig is a laboratory-engineered bispecific antibody being developed as the first-ever prophylactic treatment for individuals with Glanzmann Thrombasthenia (GT), one of several diseases the company is looking to ultimately target with this investigational, subcutaneous therapy. GT is an ultra-rare inherited bleeding disorder that is characterized by poorly functioning platelets due to a particular protein deficiency – this results in inadequate clotting and greater susceptibility to bleeding. People with GT may experience mild-to-severe bleeding symptoms, some of which can be life threatening if not promptly treated.
Existing therapies employed to treat bleeding associated with GT include antifibrinolytics, platelet transfusions, and recombinant factor VIIa. While these therapies are primarily used to treat bleeding events as they arise, HMB-001 is positioned as a preventive therapy. It binds, stabilizes, and “recruits” the important coagulation protein FVlla to the site of activated platelets at the location of a vascular injury to form a hemostatic plug. This essentially compensates for the body's inability to form healthy clots.
The phase 2 trial data, shared via an abstract presentation at ISTH, indicated that Sutacimig treatment in GT patients demonstrated more than a 50% reduction in median annualized treated bleeding rate (ATBR), with the median ATBR decreasing from 21.2 to 4.61. Sutacimig showed a “favorable” safety profile, with most adverse events (AEs) being mild to moderate in severity, and no reported thromboses or discontinuations due to AEs.
HMB-002 is an investigational subcutaneous therapy for patients with von Willebrand disease (VWD). It is developed with a monovalent antibody to increase levels of both von Willebrand factor and factor VIII. HMB-002 is designed as a prophylactic therapy to prevent bleeding in people will all types of VWD.
The data presented at ISTH is based on the Velora Pioneer trial program which is designed to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of HMB-002 in individuals with VWD. The study demonstrated that an initial single 20mg subcutaneous dose of HMB-002 resulted in consistent and sustained increases in von Willebrand factor (VWF) and factor VIII levels. Within 14 days, mean VWF rose >1.5-fold from the baseline reading. Additional presentations included HMB-002 preclinical trial data relevant to safety, efficacy, and pharmacokinetics. Read the full company press release to learn more.
"The data presented at ISTH underscore the potential for Hemab's impact in addressing the severe unmet needs of people with bleeding disorders," said Kate Madigan, Chief Medical Officer at Hemab. "Our sutacimig and HMB-002 data reveal important progress towards transformative, preventative solutions for these devastating diseases. We're demonstrating the possibilities to meaningfully reduce bleeding and elevate care for people living with Glanzmann thrombasthenia and Von Willebrand disease."
Source: PRNewswire, June 24, 2025