CSTI-500

CSTI-500 is a first-in-class, orally administered small molecule drug classified as a Triple Monoamine Reuptake Inhibitor (TRI). It simultaneously inhibits the reuptake of serotonin, dopamine, and norepinephrine, thereby increasing their levels in the synaptic clefts of neurons. This mechanism is designed to address neurotransmitter deficiencies implicated in Prader-Willi Syndrome (PWS), particularly targeting symptoms such as hyperphagia and neurobehavioral challenges. The drug has demonstrated central nervous system target engagement via PET imaging and has shown a favorable safety profile in Phase 1 clinical trials involving both healthy volunteers and PWS patients. CSTI-500 is being developed by ConSynance Therapeutics and has received Rare Pediatric Disease Designation from the FDA for PWS. It is poised to enter Phase 2 clinical development for this indication[1][3][4][5][6][7].

Other names

4-(3,4-dichlorophenyl)-7-((1,2,4)triazolo(1,5-a)pyridin-6-yl)-1,2,3,4-tetrahydroisoquinoline