TargED Biopharmaceuticals has today announced that it has commenced clinical development with the dosing of the first participant with TGD001—a “groundbreaking” thrombolytic designed to break down clots of all sizes and compositions, from large clots that cause acute ischaemic stroke to ‘micro-clots’ that cause conditions like the rare, life-threatening disorder immune-mediated thrombotic thrombocytopenic purpura (iTTP).
The first study participant was successfully dosed on 18 December at a Phase 1 clinic in Germany, according to TargED. The study—a randomised, double-blind, placebo-controlled single-ascending dose study—is evaluating TGD001’s safety, tolerability and pharmacokinetics in healthy volunteers.
First safety read-out from the Phase 1 study is expected by mid-2025. TargED plans to continue clinical development with two proof-of-concept trials in the second half of 2025: a Phase 2a trial in acute ischaemic stroke patients and a Phase 1b trial in iTTP patients, for which the company received EU orphan drug designation from the European Commission in July 2024.
Kristof Vercruysse, chief executive officer (CEO) and co-founder of TargED, said: “The dosing of the first participant with our lead compound TGD001 marks a watershed in the treatment of thrombotic disorders. Rapid treatment with highly effective thrombolytics that are well tolerated and don’t exaggerate the risk of bleeding is essential to reduce the risk of serious disability or even death from thrombotic disorders. Today’s thrombolytics show efficacy but their significant drawbacks greatly limit their safety and effectiveness, and result in restricted utility. TGD001 is specifically designed to address these shortcomings. Its unique mode of action allows for blood clots of all sizes and compositions to be targeted and rapidly dissolved without increasing bleeding risk, resulting in clinical benefit for a broad population of patients.”
Steven de Maat, chief scientific officer and co-founder of TargED, added: “Acute ischaemic stroke is one of the biggest causes of preventable death and disability in the world today, but up to 80% of acute ischaemic stroke patients cannot be treated with currently available thrombolytics, such as intravenous alteplase (tPA). Meanwhile, there is significant room for improvement in the treatment of iTTP. Despite recent advances, 15% of iTTP patients who receive treatment do not survive, and half of those who live still suffer long-term health impairment. TGD001 has the potential to greatly improve outcomes in both these indications and, we believe, in other thrombotic indications as well.”
TGD001 is a first-in-class ‘fusion protein’ drug with a unique, two-step mode of action, TargED claims. It targets clots via an antibody fragment that binds to a non-functional domain of von Willebrand factor (VWF)—a protein present in all types of thrombi—and, in the immediate vicinity of the thrombus, activates an endogenous enzyme system that degrades both VWF and fibrin—two key clotting ingredients that form the structural bond in the thrombus.
TargED’s release notes that this mode of action enables rapid and effective thrombolysis without systemic activation of the lysis cascade, and the resulting bleeding risk. According to the company, TGD001 is administered as a ‘short’ or bolus injection, making it a suitable therapeutic for emergency, in-patient and first-response settings.
