HMB-001 is being evaluated as a prophylactic treatment for Glanzmann thrombasthenia, a bleeding disorder. Credit: Kateryna Kon / Shutterstock.com.

Hemab Therapeutics has released initial positive data from the Phase I portion of its Phase I/II trial of HMB-001, a novel bispecific antibody, for Glanzmann thrombasthenia.

The open-label, first-in-human Phase I/II study is designed to assess the tolerability, safety, pharmacokinetics and pharmacodynamics of HMB-001 as a prophylactic treatment for the bleeding disorder.

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The UK-based Phase I single-ascending dose trial enrolled seven subjects who received subcutaneous HMB-001 at varying doses.

Over the observation period of 56 days, HMB-001 was found to be well tolerated, with the most frequently seen adverse events to be mild or moderate in nature.

Crucially, no adverse events or serious adverse events deemed to be related to HMB-001 were reported in the trial.

Furthermore, there were no dose-limiting toxicities or thromboembolic events reported, indicating a favourable safety profile for the treatment.

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Treatment with HMB-001 also resulted in a dose-dependent pharmacodynamic effect, with endogenous Factor VIIa accumulation linked to decline in prothrombin time and improvement in exploratory thrombin generation assessments.

Its pharmacokinetic profile and half-life showed to support the potential for dosing every two weeks or even less frequently.

The company has commenced dosing and enrolment of subjects in multiple-ascending dose Phase II portion of the study.

Hemab plans to expand the trial to sites across Europe and the US.

In addition to these clinical findings, the company also announced results from preclinical research on HMB-001.

The findings demonstrated the antibody’s ability to selectively accumulate and potentiate the endogenous Factor VIIa activity on activated platelets.

This lasting and confined procoagulant activity is expected to support the prophylactic use of the therapy for Glanzmann thrombasthenia and other bleeding disorders.

Hemab senior director and HMB-001 programme lead Joe Vogel said: “The first clinical data for HMB-001 in Glanzmann suggest that the demonstrated mechanism is suitable as a new prophylactic treatment for people with neglected blood clotting disorders who face severe, potentially life-threatening bleeds every day.

“With Phase II already underway, we are committed to advancing clinical evaluation of HMB-001 toward bringing life-changing preventative treatments to improve patients’ quality of life.”