apilimod

Apilimod is a small molecule drug initially developed as an oral inhibitor of interleukin (IL)-12 and IL-23 for the treatment of autoimmune diseases such as Crohn's disease and rheumatoid arthritis, though clinical trials did not demonstrate efficacy for these indications[1][3][4]. It was later discovered to be a potent and highly selective inhibitor of the lipid kinase PIKfyve (phosphatidylinositol 3-phosphate 5-kinase), with nanomolar potency[1][4][7]. PIKfyve regulates endosomal trafficking, cytoskeletal rearrangement, autophagy, and lysosome function. Inhibition of PIKfyve by apilimod disrupts synthesis of phosphoinositides such as PI(3,5)P2 and PI5P, leading to effects on endomembrane homeostasis including cytoplasmic vacuolization[6][8]. Apilimod has been repurposed for potential use in non-Hodgkin lymphoma (especially B-cell types), viral infections (including Ebola virus disease, Lassa fever, COVID‑19), and neurodegenerative diseases; it is under clinical development by AI Therapeutics[1][4].

Brand names

Apilimod

Other names

apilimod [INN]541550-19-0