sutacimig (HMB-001) / Hemab Therapeutics - LARVOL DELTA

A Phase 2 study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of sutacimig for prophylaxis in glanzmann thrombasthenia: A trial in progress (ASH 2025) - P1/2 | "Participants aged 18-67 years with GT, experiencing an average ≥2 bleeding events per week, and at least one spontaneous or traumatic bleed event within the last 12 months requiring prescribed treatment, medical or surgical procedure were eligible for Part B. This study is ongoing at sites in Belgium, France, Italy, The Netherlands, United Kingdom, and United States. Key outcomes include annualized bleeding rates and annualized treated bleeding rates, and safety and tolerability measures."

Clinical • P2 data • PK/PD data • Hematological Disorders • ITGA2B • ITGB3

Sutacimig, a novel bispecific antibody for prophylactic treatment of glanzmann thrombasthenia: Analysis of a Phase 2 study (ASH 2025) - P1/2 | "In this interim analysis, sutacimig was well tolerated at ongoing dose levels (0.3-0.6 mg/kg)with evidence of clinical activity, with a >50% reduction in median ATBR with sutacimig prophylaxis.Updated safety and efficacy analyses will be conducted following completion of 12-week follow-up for allparticipants. These data are expected to inform dose selection and support planning for a Phase 3 trialevaluating prophylactic sutacimig for GT."

P2 data • Hematological Disorders • Infectious Disease

Enhancing hemostasis in glanzmann thrombasthenia via protein S inhibition (ASH 2025) - "Acute bleeds are primarily managed withplatelet transfusions, and recombinant factor VIIa is used for refractory cases or perioperative support.HMB-001, a novel bispecific antibody, is under investigation as the first prophylactic therapy for GT.Protein S (PS) is a natural anticoagulant that functions as a cofactor for activated protein C, and tissuefactor pathway inhibitor, and independently regulates the prothrombinase and tenase complexes,thereby reducing thrombin generation...Clot retraction was assessed in healthy donor PRP treated with anti-PS antibody, anti-αIIbβ3 antibody (abciximab), both antibodies, or vehicle... A single PS-siRNA injection in αIIbβ3⁻/⁻ mice prevented overt bleeding at necropsy, unlike PBS-treated controls, which showed visible mucocutaneous and GI bleeding. This correlated with a significantincrease in endogenous thrombin potential (ETP): 774 ± 125 nM in PS-siRNA–treated mice vs. 438 ± 81 nMin PBS controls (n = 2–3), indicating that..."

Gastrointestinal Disorder • Hematological Disorders • Hemophilia • Rare Diseases • PROS1 • TINCR

A Phase 1/2, First-in-Human, Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia (ASH 2023) - "Personal or family history of venous or arterial thrombosis or thromboembolic disease. Other risk factors that substantially increase risk of venous or arterial thrombosis Congenital or acquired bleeding disorders other than GT Concurrent disease, treatment, medications, or abnormality in clinical laboratory tests that may pose additional risk Addiction or other diseases that prevent the participant from appropriately assessing the nature and scope of the clinical study or participating in study procedures Participants included in Part B are eligible for Part C following completion of their dosing in Part B."

Clinical • First-in-human • P1/2 data • PK/PD data • Cardiovascular • CNS Disorders • Hematological Disorders • Immunology • Infectious Disease • Inflammation • Psychiatry • Thrombosis

A Phase 1/2 Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia (ASH 2024) - P1/2 | "Recent natural history data reveals a higher disease burden in GT than previously recognized with PwGT experiencing a high frequency of bleeds (1.54 bleeds/day), with nearly half requiring medical intervention with tranexamic acid, recombinant Factor VIIa (FVIIa), or platelet transfusions. Study Population : Male and female participants aged 18 to 65 years with GT. Phase 2 has an inclusion criterion of approximately 2 bleeding events per week on average of any severity and type, and at least 1 spontaneous or traumatic bleed within the last 12 months requiring prescribed treatment, medical or surgical procedure."

Clinical • P1/2 data • PK/PD data • Anemia • Hematological Disorders • ITGA2B • ITGB3

Tranexamic Acid and Desmopressin for Heavy Menstrual Bleeding and Their Impact on Iron Level Tests: The West-Central Mexican Experience (ASH 2024) - "There are other options for HMB (Blood Adv 2023; 7 : 7501-7505) : plasma derived products, recombinant vW factor, bridging therapies as emicizumab, investigational (rondoraptivon pegol, VGA039, HMB-001 and platelet inspired hemostatic nanoparticles), but these have high financial costs or are not available in clinical trial in developing countries. We recommend the use of these drugs to provide either of the treatments, as they are easy to administrate low cost access and have a good security profile. This is an original report from Mexico, no other results were find in PubMed."

Anemia • Hematological Disorders • Hemophilia • Rare Diseases

HMB-001 in Glanzmann thrombasthenia: breakthrough bleed control with reduced platelet and rFVIIa use (ISTH 2025) - "Aims To evaluate the in vitro effects of platelet concentrates (PC), recombinant FVIIa (rFVIIa) and tranexamic acid (TXA) on hemostasis in combination with HMB-001. In presence of HMB-001, FVIIa binding to PC-derived platelets increased 4-fold, and low-grade thrombin generation occurred (ETP 168±43 nM*min) in absence of a platelet agonist. Stimulation of PCs resulted in a 16-fold increase in FVIIa binding and a concomitant rise in TG (407±101 nM*min), indicating that stored platelets are fully activatable and support HMB-001’s mechanism of action."

Hematological Disorders

Interim data from phase 2 study of HMB-001 for prophylactic treatment in Glanzmann thrombasthenia (ISTH 2025) - "PD data demonstrated a dose-dependent increase in endogenous total FVII(a) and FVIIa activity, along with improved thrombin generation comparable to standard rFVIIa dosing, indicating effective coagulation activation. The PK profile supports a convenient, infrequent dosing schedule."

P2 data • Anemia • Hematological Disorders

A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia (clinicaltrials.gov) - P1/2 | N=57 | Active, not recruiting | Sponsor: Hemab ApS | Recruiting ➔ Active, not recruiting

Enrollment closed • Hematological Disorders

Hemab Therapeutics Presents Interim Data from Ongoing Phase 2 Study of HMB-001 as First Ever Prophylactic Treatment in Glanzmann Thrombasthenia…at the 2025 EAHAD Annual Congress (PRNewswire) - P1/2 | N=57 | NCT06211634 | Sponsor: Hemab ApS | "Hemab Therapeutics...today presented interim data from the ongoing evaluation of HMB-001, a novel bispecific antibody in development as first ever prophylactic treatment for the bleeding disorder Glanzmann thrombasthenia (GT). The Phase 2 study consists of a minimum 6-week prospective run-in where participants record bleeds via an electronic bleed diary, followed by 3 months of treatment with HMB-001. Interim efficacy data to date demonstrated >50% reduction in treated bleeds in all 3 tested dose cohorts. Underscoring its potential to address unmet medical needs and to expedite patient access, HMB-001 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration and the UK Medicines and Healthcare products Regulatory Agency has awarded it designation under the Innovative Licensing and Access Pathway...Hemab plans to complete Phase 2 study recruitment in the first half of 2025."

P2 data • Hematological Disorders