TargED Announces First Patients Successfully Dosed in Study of TGD001 in Acute Ischemic Stroke
According to the World Stroke Organization, approximately 12 million people suffer a stroke each year, resulting in over six million deaths and a similar number of patients facing permanent disability. Despite advances in care, only a minority of patients receive interventional therapy and even for those fortunate enough to receive therapy, outcomes remain poor.
In the past 30 years incident strokes have increased by 70%, deaths from stroke by 44% and Disability-Adjusted Life Years (DALYs) by 32%. Today, one in four people are likely to have a stroke in their lifetime, translating to global costs of $890B set up double by 2050. Together, these underscore the urgent need for effective, safe and broadly applicable treatment options.
TGD001, TargED’s lead program, is a first-in-class, targeted thrombolytic designed to effectively dissolve blood clots in a broad range of thrombotic indications. By degrading both von Willebrand factor (VWF) and fibrin, TGD001 has the potential to dissolve clots of varying composition and location. At the American Society of Hematology (ASH) Annual Meeting in December 2025, and more recently at the European Stroke Organization Conference (ESOC) 2026, TargED reported positive Phase 1 first-in-human results, demonstrating a favorable safety and tolerability profile, with no spontaneous bleeding events observed. In addition, TGD001 showed potent thrombolytic activity, including rapid plasminogen activation and increased fibrin degradation products (D-dimer).
“Based on the very encouraging results of our healthy volunteer trial, we are advancing TGD001 for evaluation in patients with acute ischemic stroke with a high sense of urgency,” said Josefin-Beate Holz, MD, Chief Medical Officer at TargED Biopharmaceuticals. “Most stroke patients today do not receive optimal treatment in the acute setting addressing their clot burden and consequently face poor outcomes. Even for those who do receive standard of care treatment, outcomes are poor and we believe TGD001’s unique and fast mechanism of action has the potential to reduce the patients disease burden via efficient degradation of clots, transforming outcomes and providing patients and physicians with a new therapeutic option that can reduce severe disability and save lives.”
About the Phase 1b/2a clinical study
The aim of the trial is to establish tolerated doses with signs of pharmacological activity in the Phase 1b dose escalation part and to further establish TGD001’s safety profile and early clinical activity in a randomized, double-blind, placebo-controlled expansion in the Phase 2a part. The trial expects to enroll up to 130 patients with acute ischemic stroke (EU Clinical Trial Identifier: 2025-522455-26-00). The multi-center and multi-regional study is currently open for enrollment at sites in Europe and Serbia with additional regions and sites in activation.
About TargED Biopharmaceuticals
TargED Biopharmaceuticals B.V. is a clinical-stage biotechnology company pioneering targeted enzyme delivery for improved treatment of thrombotic diseases. Founded in 2020 as a spin-off from the University Medical Center Utrecht, the company has raised more than €80 million to advance its pipeline and was awarded a prestigious blended finance grant from the European Innovation Council (EIC) Accelerator Program in 2025. For more information, please visit www.targedbio.com.
About TGD001
TGD001 is a first-in-class, targeted thrombolytic designed to treat a broad range of thrombotic events. As an antibody-enzyme fusion protein, it binds to von Willebrand factor (VWF) and promotes site-specific clot breakdown through the enzyme urokinase. Unlike currently approved therapies, TGD001 leverages the ubiquitous presence of VWF in all clots, enabling broad clot lysis by degrading both VWF and fibrin. TGD001 demonstrated compelling Phase 1 results and is in development for the treatment of acute ischemic stroke (AIS) and thrombotic microangiopathies (TMAs), including immune thrombotic thrombocytopenic purpura (iTTP).
Media Contact: info@targedbio.com