F4 Pharma GmbH is developing FX06 in orphan diseases associated with capillary leak and high medical need. Capillary leak can be observed in patients with acute respiratory distress syndrome (ARDS), sepsis, septic shock, Clarkson disease, vasoplegic syndrome or several hemorrhagic fevers (Ebola, Hanta, Dengue etc.). Capillary leak is also a major concern in ICU patients under ECMO.
FX06 is a platform molecule with a novel mode of action. FX06 binds to the so-called adherence junctions between the endothelial cells which are forming the inner layer of blood vessels. In doing so, the drug preserves the integrity of the endothelium and prevents the leakage of fluid from the blood vessel into the surrounding tissue. In addition, FX06 also has anti-inflammatory properties. The drug was already investigated in another - mechanistically related - indication and has demonstrated convincing efficacy in a clinical study with 236 patients. FX06 is very well tolerated and exhibits a benign safety profile.