Intranasal carbetocin is similar to the naturally-occurring hormone oxytocin, but with greater selectivity for the effect of interest – stimulation of oxytocin receptors.  These receptors are involved in the regulation of both social-emotional and feeding behaviors.

This was a Phase 3 randomized, double-blind study with an 8-week, placebo-controlled period designed to test the effectiveness, safety, and tolerability of investigational LV-101 in participants with PWS.

Effectiveness was measured using both caregiver-reported and clinician-reported measures of hyperphagia (extreme hunger), obsessive and compulsive behaviors, and anxiety. Safety and tolerability was measured by adverse events, laboratory tests, and physical exams.

All participants received active treatment with LV-101 after the 8-week placebo-controlled period, during a long-term follow-up period of 56 weeks. At Week 8, participants who were randomized to placebo in the placebo-controlled period were randomized to one of the two LV-101 doses, administered three times per day before meals.