APOL1 small molecule inhibitor

APOL1 small molecule inhibitors represent an emerging class of targeted therapies designed to treat APOL1-mediated kidney disease (AMKD), a genetically driven, progressive form of chronic kidney disease that predominantly affects individuals of West African ancestry carrying high-risk G1 and G2 variants of the apolipoprotein L1 (APOL1) gene. Under inflammatory conditions, these toxic gain-of-function variants form pores or channels in the membranes of podocytes, leading to cellular injury, glomerular filtration barrier disruption, and severe proteinuria. By binding directly to APOL1 and blocking its pore-forming and channel activities, small molecule inhibitors prevent podocyte damage, reduce albuminuria, and potentially slow or halt progression to end-stage kidney disease. Key clinical-stage candidates in this class include Vertex Pharmaceuticals' inaxaplin (VX-147), which is in Phase 2/3 development, and Maze Therapeutics' MZE829 (alcedaplin), which is in Phase 2 development.

Other names

APOL1 channel blockerAPOL-1 channel blockerAPOL 1 channel blockerAPOL1 channel inhibitorAPOL-1 channel inhibitorAPOL 1 channel inhibitorAPOL1 inhibitorAPOL-1 inhibitorAPOL 1 inhibitorAPOL1 pore inhibitorAPOL-1 pore inhibitorAPOL 1 pore inhibitorAPOL1 pore-function inhibitorAPOL-1 pore-function inhibitorAPOL 1 pore-function inhibitor