Prophylactic Therapy for Glanzmann Thrombasthenia Moves Towards Clinical Trials

Glanzmann Thrombasthenia is an ultra-rare inherited bleeding disorder that is characterized by poorly functioning platelets.

The recent Congress of the International Society on Thrombosis and Haemostasis (ISTH) included some noteworthy findings on the rare blood disorders news front. Hemab Therapeutics, which specializes in developing therapies that target serious underserved bleeding and thrombotic disorders, presented new preclinical data on HMB-001.

HMB-001 is a laboratory-engineered bispecific antibody being developed by Hemab as the first-ever prophylactic treatment for individuals with Glanzmann Thrombasthenia (GT), one of several diseases the company is looking to ultimately target with this investigational, subcutaneous therapy.

GT is an ultra-rare inherited bleeding disorder that is characterized by poorly functioning platelets due to a particular protein deficiency – this results in inadequate clotting and greater susceptibility to bleeding. People with GT may experience mild-to-severe bleeding symptoms some of which can be life threatening if not promptly treated.

Existing therapies employed to treat bleeding associated with GT include antifibrinolytics, platelet transfusions, and recombinant factor VIIa. While these therapies are primarily used to treat bleeding events as they arise, HMB-001 is being positioned as a preventive therapy. It binds, stabilizes, and “recruits” the important coagulation protein FVlla to the site of activated platelets at the location of a vascular injury to form a hemostatic  plug. This essentially compensates for the body’s inability to form healthy clots.

The findings, presented at the ISTH Congress July 9-13th in London, demonstrated HMB-001’s ability to generate therapeutic levels of FVIIa in animal (monkey) models. Ex vivo laboratory tests also showed its ability to contribute to platelet aggregation and the forming of viable hemostatic plugs.

Hemab is also targeting other rare blood diseases with this technology, including Hereditary Hemorrhagic Telangiectasia (HHT), factor VII deficiency, Bernard-Soulier syndrome, von Willebrand disease (types 1 and 2), Antithrombin deficiency, and others. While these therapeutic applications are still in the discovery and early preclinical stages of development, it is encouraging that individuals representing additional underserved blood disorder populations may eventually benefit from a new prophylactic therapy.

“For many patients of rare bleeding and thrombosis, zero targeted preventative treatments exist, meaning the standard of care remains decades behind,” said Benny Sorensen, MD, PhD, President, and CEO of Hemab. “We are encouraged by this data showing HMB-001’s promise as the first prophylactic treatment for patients with GT. We are also excited to launch our strategic guidance, Hemab 1-2-5™, aimed at advancing 5 clinical assets by 2025 to help people facing clotting disorders who have been left behind and urgently need innovation.”

In the meantime, the company expects HMB-001 to advance from the preclinical research stage and enter phase 1/2 clinical trials later this year.

Additional Resources:

Read NHF’s new publication Glanzmann’s Thrombasthenia (GT): You Are Not Alone.

Watch this video to learn the “Basics of Glanzmann’s Thrombasthenia and Bernard-Soulier Syndrome.”

Learn more about inherited platelet disorders.

Source: Cision (PRNewswire), July 13, 2022