BioCentury
ARTICLE | Distillery Therapeutics

Therapeutics: Liver X receptor (LXR)

April 14, 2016 7:00 AM UTC

Mouse and patient sample studies suggest promoting LXR expression with 2-hydroxypropyl-β-cyclodextrin (HPBCD) could help treat atherosclerosis. In primary mouse macrophages and carotid atherosclerotic plaque samples from patients, HPBCD increased expression of LXR target genes and cholesterol efflux compared with vehicle. In two mouse models of diet-induced atherosclerosis, subcutaneous injection of HPBCD decreased plaque size in aortic root, cholesterol content of plaques, aortic production of reactive oxygen species (ROS) and levels of proinflammatory cytokines in plasma. Next steps include identifying partners for clinical trials.

Vtesse Inc. has HPBCD (VTS-270), which targets cholesterol and sphingolipid storage, in Phase II/III testing to treat Niemann-Pick disease type C1 (NPC1)...