Drug intelligence / Profile preview

urokinase plasminogen activator (LTI-01)

Development stage
Phase 2
Lead developer
Rein Therapeutics
Modality
Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
Administration
Intrapleural
01

Overview

LTI-01 is an injectable, fibrinolytic drug designed to treat empyema and complicated parapneumonic effusions (CPE), conditions characterized by fibrinous scarring that prevents fluid drainage in the pleural space. It is a lyophilized formulation of single-chain urokinase plasminogen activator (scuPA), which acts as a proenzyme or zymogen and becomes activated in the pleural space. Its mechanism involves enhancing scar removal around the lungs to improve fluid drainage while minimizing bleeding risk. LTI-01 is relatively resistant to inhibition by plasminogen activator inhibitor type 1 (PAI-1) and increases local fibrinolytic activity without causing systemic fibrinogenolysis. The drug has received orphan drug designation from both the US FDA and EU for empyema, reflecting its development for rare diseases with significant unmet need[1][2][3][4][5][7].

Other names
single-chain urokinase plasminogen activatorscuPA
02

Targets

PAI-1 (Plasminogen activator inhibitor type 1)PLG (Plasminogen)

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