Acadia Pharmaceuticals announces COMPASS PWS trial misses endpoints

Acadia Pharmaceuticals (ACAD) announced top-line results from the Phase 3 COMPASS PWS trial evaluating the efficacy and safety of intranasal carbetocin in patients with hyperphagia in Prader-Willi syndrome. Intranasal carbetocin did not demonstrate a statistically significant improvement over placebo on the study’s primary endpoint, change from baseline to Week 12 on the Hyperphagia Questionnaire for Clinical Trials, nor was there separation from placebo on any secondary endpoint. The safety and tolerability profile of intranasal carbetocin was consistent with previous clinical trials, showing a low rate of adverse events. “We are disappointed by these findings, especially for Prader-Willi syndrome patients, their families and the entire community,” said Elizabeth H.Z. Thompson, Ph.D., Acadia’s Head of Research and Development. “I want to thank the many patients, families, study site personnel, and physicians who participated in the COMPASS PWS study as well as the intranasal carbetocin clinical development program, for their dedication and contributions in this important study. We are committed to sharing a summary of the data in the future to ensure learning for the PWS community; however, given these results, we do not intend to investigate intranasal carbetocin any further.” COMPASS PWS was a 12-week, double-blind, randomized, placebo-controlled global Phase 3 trial evaluating the efficacy and safety of intranasal carbetocin 3.2 mg three times daily in 175 enrolled children and adults aged five to 30 years with PWS.