CAMBRIDGE, Mass. and COPENHAGEN, Denmark, July 14, 2026
Hemab Therapeutics presented new clinical and preclinical data at the International Society on Thrombosis and Haemostasis (ISTH) 2026 Congress demonstrating sustained efficacy and manageable safety of sutacimig in Glanzmann thrombasthenia (GT) while highlighting its potential as a pan-hemostatic therapy for Factor VII deficiency (FVIID). The company also reported natural history data showing the persistent lifelong bleeding burden and limited use of prophylactic treatment in GT, supporting continued development of preventive therapies. Based on the Phase 2 results, Hemab plans to initiate a Phase 3 trial in the second half of 2026.
Phase 2 Extension Demonstrates Durable Bleed Reduction
Updated long-term extension data from the ongoing Phase 2 study included 34 patients treated for a median of 6.9 months, with some receiving therapy for up to 15.9 months. Results showed that 92% of participants who experienced bleeding before treatment achieved reductions in treated bleeding events, while patients with prior high-intensity bleeding requiring transfusions, recombinant Factor VIIa, or hospitalization experienced a 62% reduction in annualized treated bleeding rates. The weekly low-dose regimen reduced annualized treated bleeding rates by approximately 84%, and three surgical procedures were successfully managed during the study. Safety findings remained manageable, with predominantly mild-to-moderate adverse events and no treatment-related Grade 3 or higher adverse events, supporting the FDA-endorsed weekly Phase 3 dosing regimen.
Preclinical Data Expand Potential into Factor VII Deficiency
Hemab also presented preclinical findings demonstrating that sutacimig restored thrombin generation under disease-mimicking conditions in Factor VII deficiency, supporting its potential beyond GT. The investigational bispecific antibody retained binding across the majority of clinically relevant disease variants, including 22 of 25 tested variants, suggesting broad applicability for patients enrolled in the ongoing Phase 2 study. The data reinforce sutacimig’s mechanism of enhancing endogenous Factor VIIa activity while targeting activated platelets to improve hemostasis across multiple rare bleeding disorders.
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Natural History Studies Highlight Significant Unmet Need
Additional analyses from the GT360 and ATHN Transcends natural history studies demonstrated the substantial burden faced by patients with Glanzmann thrombasthenia. More than 90% of pediatric, adolescent, and young adult patients experienced at least one bleeding episode per week, while 72% of patients aged 40 years or older continued to experience weekly bleeding. Investigators also reported markedly elevated rates of depressive symptoms and found that prophylactic therapy remains significantly underutilized, with only 14% of patients receiving preventive treatment despite persistent disease burden. Hemab stated that the combined clinical, preclinical, and real-world data further support advancing sutacimig toward Phase 3 development as a potential first prophylactic treatment for Glanzmann thrombasthenia while expanding its therapeutic potential to additional rare bleeding disorders.
Source: Hemab Therapeutics,press release
