PTC Therapeutics announced that its oral small molecule, utreloxastat, failed to meet its primary endpoint of slowing disease progression in amyotrophic lateral sclerosis (ALS) in a global phase 2 study.
According to PTC, while there was modest numerical benefit recorded on the primary endpoint and correlation of favorable clinical effect with lowering of plasma neurofilament light chain, a biomarker of neuronal damage, statistical significance was not achieved in the placebo-controlled CardinALS study. In addition, significance was not achieved on the secondary efficacy endpoints
Due to the lack of efficacy and biomarker signal in the CardinALS study, PTC says it will halt development of utreloxastat.
Utreloxastat is a 15-lipoxygenase inhibitor with a unique mechanism of action that targets enzymatic activity in the lipoxygenase pathway. It was thought that reducing the activity of this enzyme in cells may reduce the damage to and death of motor neurons.
Earlier this month, PTC was granted accelerated approval of its gene replacement therapy for the treatment of AADC deficiency, marking the first-ever gene therapy approved in the U.S. that is directly administered to the brain. Branded Kebilidi, eladocagene exuparvovec-tneq is indicated for the treatment of children and adults with AADC deficiency — a rare, severe, genetic neurometabolic disorder — including the full spectrum of disease severity.