Acadia Pharmaceuticals’ Investigational Drug For Rare Genetic Disorder Fails In Late-Stage Study

The company noted that the safety and tolerability of intranasal Carbetocin were consistent with those observed in previous clinical trials.

Acadia Pharmaceuticals Inc. (ACAD) announced on Tuesday that its late-stage intranasal Carbetocin trial in Prader-Willi syndrome (PWS) patients did not achieve a statistically significant improvement in hyperphagia.

The study enrolled 175 children and adults aged five to 30 years with PWS, a rare genetic disorder characterized by an insatiable hunger or hyperphagia and a constant need for food, leading to obesity. However, intranasal Carbetocin did not demonstrate a statistically significant improvement over placebo to week 12 on the Hyperphagia Questionnaire for Clinical Trials, the company said.

The firm noted that the safety and tolerability of intranasal Carbetocin were consistent with those observed in previous clinical trials, with a low rate of adverse events. 

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