utreloxastat

Utreloxastat (PTC857) is an orally bioavailable small molecule inhibitor of 15-lipoxygenase (15-LO) originally developed by BioElectron Technology Corporation and later acquired by PTC Therapeutics. It functions as a redox-active inhibitor of ferroptosis, an iron-dependent oxidative mode of cell death, by reducing oxidative stress and preventing the depletion of reduced glutathione. The drug was primarily investigated for the treatment of amyotrophic lateral sclerosis (ALS) and GBA-associated Parkinson's disease. In November 2024, PTC Therapeutics announced that the Phase 2 CardinALS trial failed to meet its primary endpoint of slowing disease progression in ALS patients, as well as its secondary efficacy endpoints. Consequently, further development of utreloxastat for ALS has been discontinued.

Other names

alkyl-substituted cyclohexadienedione