Drug intelligence / Profile preview

fosmanogepix

Development stage
Phase 3
Lead developer
Basilea Pharmaceutica
Modality
Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
Administration
Oral, Intravenous
01

Overview

Fosmanogepix is a first-in-class, broad-spectrum antifungal prodrug developed for the treatment of invasive fungal infections. It is converted in vivo to its active form, manogepix, which inhibits the fungal enzyme Glycosylphosphatidylinositol-anchored Wall protein Transfer 1 (Gwt1). This enzyme is essential for the biosynthesis of glycosylphosphatidylinositol anchors that are critical for trafficking and anchoring mannoproteins to the fungal cell wall and membrane. By inhibiting Gwt1, fosmanogepix disrupts cell wall integrity and impairs pathogenicity in fungi. The drug demonstrates potent activity against a wide range of yeasts and molds—including Candida species (including multidrug-resistant strains like Candida auris), Aspergillus species, Fusarium spp., Scedosporium spp., Coccidioides immitis, Lomentospora prolificans, and some Mucorales—with efficacy even against strains resistant to other antifungal classes. Fosmanogepix can be administered orally or intravenously with high bioavailability and favorable pharmacokinetics. It has received Fast Track and Orphan Drug designations from the FDA for multiple indications[1][2][4][6][8].

Other names
manogepix (active moiety)APX001A (active moiety)E1210 (active moiety)
02

Targets

GWT1 (Glycosylphosphatidylinositol biosynthesis protein Gwt1)

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