TargED Biopharmaceuticals begins Phase 1 trial of TGD001 for AIS and TTP

TargED Biopharmaceuticals, a biotechnology company based in the Netherlands, has begun the clinical development of TGD001, an innovative drug designed to enhance treatment options for thrombotic diseases. This development was marked by the successful administration of the first dose to a participant in Germany as part of a Phase 1 study. TGD001 aims to present a more effective approach in managing blood clotting disorders, particularly targeting a wide array of clot sizes and compositions, ranging from large clots seen in conditions like acute ischemic stroke (AIS) to the micro-clots involved in immune-mediated thrombotic thrombocytopenic purpura (iTTP).

The Phase 1 study, a randomized, double-blind, placebo-controlled trial, aims to assess the safety, tolerability, and pharmacokinetics of TGD001 in healthy volunteers. This study is expected to offer preliminary safety data by mid-2025. Following this, TargED plans to continue its clinical development by initiating two proof-of-concept trials in the latter half of 2025. These will include a Phase 2a trial targeting AIS patients and a Phase 1b trial for iTTP patients, with the latter having already received EU Orphan Drug Designation from the European Commission.

TGD001 is hailed as a first-in-class 'fusion protein' drug exhibiting a distinctive two-step mechanism. The drug targets and binds to the von Willebrand Factor (VWF) found in all thrombi using an antibody fragment. This action facilitates the activation of an endogenous enzyme system that breaks down VWF and fibrin, the substances forming the structural bond in thrombi. This approach enables rapid and efficient clot breakdown without triggering the systemic lysis cascade, thereby minimizing bleeding risks. TGD001's administration as a short injection makes it suitable for emergency and in-patient scenarios.

Kristof Vercruysse, CEO and co-founder of TargED, emphasized the potential of TGD001 to revolutionize thrombotic disorder treatments. The drug addresses significant limitations associated with current thrombolytics, which, despite their effectiveness, pose substantial safety risks and limit their broader clinical utility. He highlighted that TGD001's design aims to rapidly dissolve clots without increasing the risk of bleeding, thereby offering clinical benefits to a diverse patient population.

Steven de Maat, TargED's Chief Scientific Officer and co-founder, pointed out the urgent need for improved treatments in AIS and iTTP. Current thrombolytics like intravenous alteplase (tPA) serve only a fraction of AIS patients due to their constraints. In the case of iTTP, despite treatment progress, a significant mortality rate and long-term health issues persist among survivors. TGD001 offers potential breakthroughs in these areas by improving patient outcomes in both AIS and iTTP, and possibly extending benefits to other thrombotic conditions.

The statistics underline the gravity of these conditions. AIS accounts for roughly 90% of all stroke cases, with millions affected annually worldwide, leading to deaths and permanent disabilities. Prompt treatment is crucial to prevent brain damage, but current thrombolytics are accessible to only a minority of patients. On the other hand, iTTP, though rarer, presents severe risks with a high fatality rate without treatment. Treatment complexities and risks associated with traditional methods underscore the need for safer and more effective alternatives like TGD001.

TargED Biopharmaceuticals, established in 2020 as a University Medical Center Utrecht spin-off, focuses on developing innovative biological drugs to enhance thrombosis treatment. The company has attracted over €45 million in funding, supporting its transition to a clinical-stage entity, with TGD001 poised to significantly advance the field of thrombolytic therapy.