Pharma Pioneer

HMB-001 Phase 1 Success in Glanzmann Thrombasthenia Showcased at EAHAD 2024

29 May 2024
2 min read

HMB-001, a novel bispecific antibody developed by Hemab Therapeutics, has shown promising results in Phase 1 clinical trials for treating Glanzmann Thrombasthenia, a severe bleeding disorder. The drug demonstrated a good safety profile with no drug-related adverse effects or thromboembolic incidents. The study, which included patients receiving various doses of HMB-001, revealed dose-dependent improvements in clotting activity, suggesting its potential as a first-in-class prophylactic treatment for Glanzmann Thrombasthenia and other clotting disorders.

The positive outcomes were presented at the 17th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) in Frankfurt, Germany, and are part of ongoing clinical evaluations. Hemab Therapeutics is already conducting Phase 2 trials, which are set to expand across Europe and the U.S. for further assessment.

The preclinical findings of HMB-001 were published in the February 2024 issue of "Nature Cardiovascular Research," highlighting its ability to selectively accumulate and enhance the activity of endogenous Factor VIIa on activated platelets, which could be instrumental in the prophylactic treatment of Glanzmann Thrombasthenia and related bleeding conditions.

Glanzmann Thrombasthenia is characterized by frequent and potentially life-threatening bleeding episodes, which have a profound impact on the mental health and quality of life of affected individuals. Currently, there are no approved preventative treatments for this condition.

HMB-001 works by binding to endogenous Factor VIIa and TLT-1 on activated platelets, leading to a localized increase in clotting activity without causing unwanted clotting elsewhere in the body. The U.S. Food and Drug Administration has granted Fast Track Designation to HMB-001 for the treatment of Glanzmann Thrombasthenia.

Hemab Therapeutics, with locations in Cambridge, MA, and Copenhagen, Denmark, is focused on creating innovative therapeutics for serious and underserved bleeding and thrombotic disorders. The company is working towards its strategic goal, Hemab 1-2-5™, which includes the development of five assets by 2025 to address high unmet needs in conditions like Glanzmann Thrombasthenia, Factor VII Deficiency, and Von Willebrand Disease.

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