A Phase 1/2, First-in-Human, Single and Multiple Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants With Glanzmann Thrombasthenia

Glanzmann Thrombasthenia

1. Part A • Safety as assessed by the incidence of treatment emergent adverse events (AEs) and changes in physical examinations, vital signs, clinical laboratory assessments, and electrocardiogram (ECG) parameters., 2. Part B and Part C • Safety as assessed by the incidence of treatment emergent AEs and changes in physical examinations, vital signs, clinical laboratory assessments, and ECG parameters., 3. Part B and Part C • Preliminary prophylactic effect of HMB-001 as assessed via: − Bleed frequency: annualized treated bleed rate (ATBR) and annualized bleed rate (ABR)

1. Part A • Plasma concentrations of HMB-001 • PK parameters including, but not limited to: − Maximum observed plasma concentration (Cmax) − Area under the curve from time zero to last quantifiable concentration (AUClast) − Area under the curve from time zero to extrapolated infinite time (AUCinf) − Time to reach maximum observed plasma concentration (Tmax), 2. Part A • Anti-drug antibody (ADA) formation, 3. Part A • PD parameters including, but not limited to: − Maximum, mean increase in FVII from baseline − Maximum, mean decrease from baseline in prothrombin time (PT) − Maximum, mean decrease from baseline in activated partial thromboplastin time (aPTT)., 4. Part B and Part C • Plasma concentrations of HMB-001 • PK parameters including, but not limited to: − Cmax − AUClast − AUCinf − Tmax., 5. Part B and Part C • Preliminary prophylactic effect of HMB-001 as assessed by: - Subcategories of ABR, including, but not limited to: o Sponstaneous and impactful − Bleed Severity o Occurrence of severe bleeding events, 6. Part B and Part C • Preliminary prophylactic effect of HMB-001 as assessed by: − Requirement for Treatment o Volume of transfusion product use: platelets, fresh frozen plasma, cryoprecipitate o Volume of Factor Concentration use: rFVIIa o Volume of red blood cell (RBC) transfusion − Iron Status o Mean change in hemoglobin from baseline o Mean change in ferritin and total iron from baseline, 7. Part B and Part C • ADA formation., 8. Part B and Part C • Changes from baseline in Euro Quality of life 5- Dimensions 5-Level (EQ-5D-5L), Patient-Reported Outcomes Measurement Information System (PROMIS)-29, and Work Productivity and Activity Impairment (WPAI) scores.

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