Hemab Therapeutics (COAG) investor relations material

Disease burden and unmet needs

• Patients with neglected blood coagulation disorders face increased mortality, fatigue, depression, cognitive impairment, and significant social stigma, with no prophylactic treatments currently available.

• Existing treatments are burdensome, often involving blood transfusions or risky bone marrow transplants, and abnormal bleeding is normalized in daily life.

• Glanzmann thrombasthenia and Factor VII deficiency patients experience frequent bleeds, hospital visits, and high rates of depression and lost productivity.

• Von Willebrand Disease affects ~120,000 symptomatic patients in key markets, with heavy menstrual bleeding and iron deficiency common among women.

• Heavy menstrual bleeding impacts over 23 million women in the US annually, leading to significant economic and health burdens.

Pipeline and clinical development

• The pipeline includes sutacimig for Glanzmann thrombasthenia and Factor VII deficiency, HMB-002 for Von Willebrand Disease, and HMB-003 for heavy menstrual bleeding.

• Sutacimig has received multiple regulatory designations, including Breakthrough Therapy and Orphan Drug status in the US and EU.

• Phase 3 clinical trial for sutacimig in Glanzmann thrombasthenia is planned for 2H 2026, with a Phase 2B study in Factor VII deficiency ongoing.

• HMB-002 is in Phase 1/2 clinical studies for VWD, with a large natural history study underway.

• HMB-003 is expected to enter clinical development for heavy menstrual bleeding, with initial data anticipated in mid-2027.

Clinical efficacy and safety data

• Sutacimig demonstrated persistent and clinically meaningful reductions in annualized treated bleeding rates (ATBR) across all dose cohorts, with 92% of participants showing improvement.

• High-intensity bleeding events requiring systemic intervention were reduced by 62% in a key subpopulation.

• Sutacimig was generally well tolerated, with most adverse events mild to moderate; venous thrombotic events were observed at higher doses and in patients with multiple risk factors.

• HMB-002 showed dose-dependent increases in VWF antigen and FVIII activity, with favorable safety and no serious adverse events reported.

• Preliminary data for HMB-002 indicated a reduction in treated bleeding events post-dose, with most participants experiencing zero treated bleeds in the observation period.