Triple Monoamine Reuptake Inhibitor for the treatment of hyperphagia in Prader-Willi Syndrome.
CSTI-500 is First-in-Class, orally administered New Chemical Entity (NCE), Triple Monoamine Reuptake Inhibitor (TRI). By simultaneously inhibiting the reuptake of serotonin, dopamine, and norepinephrine, CSTI-500 can address the often-reduced levels of these neurotransmitters in PWS patients. It is an optimally balanced TRI with unique pharmacological features that allow for predictable and safe titration to an efficacious dose tailored to the individual patient.
CSTI-500 was generally safe and well-tolerated and has a half-life of 50 hours in Phase I clinical trials. Also importantly, it demonstrated CNS target engagement via positron emission tomography (PET) that predicts efficacy in patients. CSTI-500 is now posed for Phase 2 development.
CSTI-500 has the potential to provide a transformative therapy, not just for addressing hyperphagia in PWS, but also for tackling many of the associated psychiatric co-morbidities.