Hemab Therapeutics Presents New Data on HMB-001 for the Treatment of Glanzmann Thrombasthenia and Other Bleeding Disorders at 2022 ISTH Congress

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Jul 13, 2022, 05:45 ET

Data suggest HMB-001 potentiates endogenous factor Vlla-dependent fibrin formation on platelets in Glanzmann Thrombasthenia and accumulates FVIIa to levels considered therapeutically effective

Hemab's strategic guidance, Hemab 1-2-5™, aims to advance 5 clinical assets by 2025 to address significant unmet need across a wide range of bleeding and thrombotic disorders

COPENHAGEN, Denmark and BOSTON, July 13, 2022 /PRNewswire/ -- Hemab Therapeutics, a biotechnology company developing next-generation therapeutics for serious, underserved bleeding and thrombotic disorders, today presented new pre-clinical data on HMB-001, a novel bispecific antibody poised to become the first-ever prophylactic treatment option for Glanzmann Thrombasthenia (GT) and other rare bleeding conditions. Data show HMB-001 potentiates endogenous factor Vlla (FVIIa)-dependent fibrin formation on platelets in GT and accumulates FVIIa to levels that are considered therapeutically effective. The company highlighted findings in two presentations at the 2022 International Society on Thrombosis and Haemostasis (ISTH) Congress held this week in London.

"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."

Expected to enter Phase 1/2 clinical trials in late 2022, HMB-001 binds, stabilizes, and recruits FVlla to the site of vascular injury to overcome the body's inability to form healthy clots—building on the clinically validated mechanisms of action of recombinant FVlla with the benefit of prophylaxis.

Hemab's oral presentation described compelling data showing HMB-001 binds FVIIa in a neutral manner, preserving native biological functions, such as autoactivation of FVII to FVIIa, FVIIa–mediated activation of Factor X, and inhibition of FVIIa by AT3 and TFPI. In studies of cynomolgus monkeys, HMB-001 administration resulted in dose-dependent endogenous FVIIa accumulation to therapeutic levels, supporting the use of HMB-001 as a prophylactic treatment for GT in weekly, biweekly, or monthly dosing.

Ex vivo data shared in a poster presentation suggest that HMB-001 further potentiates fibrin formation and fibrin-dependent platelet aggregation of GT platelets. This assay was performed in a microfluidics chamber, mimicking flowing blood to simulate in vivo conditions for clot formation.

The company also debuted its strategic guidance, Hemab 1-2-5™, targeting development of 5 clinical assets by 2025 to transform treatment for rare bleeding and thrombotic disorders with high unmet need, including Factor VII Deficiency, Bernard Soulier Syndrome, Von Willebrand Disease, Hereditary Hemorrhagic Telangiectasia (or Osler-Weber-Rendu disease), Congenital Antithrombin III Deficiency, and others.

Presentations at ISTH Congress:

Title: HMB-001 – a novel bispecific antibody accumulating and targeting endogenous FVIIa to activated platelets for subcutaneous prophylaxis in multiple bleeding disorders including Glanzmann Thrombasthenia

Session Theme: Hemophilia and Rare Bleeding Disorders/Rare Bleeding Disorders
Abstract Number: OC 78.3
Presenter: Henrik Østergaard, PhD, MSc, Scientific Director, Hemab

Title: The novel bispecific antibody HMB-001 enhances the haemostatic response in models of Glanzmann Thrombasthenia by targeting FVIIa to activated platelets

Session Theme: Hemophilia and Rare Bleeding Disorders/Rare Bleeding Disorders
Abstract Number: PB0719
Presenter: Minka Zivkovic, MSc, PhD Candidate in Thrombosis and Haemostasis, UMC Utrecht

About Hemab Therapeutics
Hemab is a biotech company developing next generation therapeutics for serious, underserved bleeding and thrombosis disorders. The company announced an oversubscribed $55 million Series A led by RA Capital Management, Novo Holdings and HealthCap in July 2021. Based in Denmark and the US, Hemab aims to progress its pipeline of monoclonal and bispecific antibody-based therapeutics with the vision of transforming the treatment paradigm for patients with bleeding and thrombotic disorders with high unmet need. Learn more at hemab.com.

SOURCE Hemab Therapeutics