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

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.

Shares of the company tumbled 11% in the pre-market session. On Stocktwits, retail sentiment around ACAD stock stayed within the ‘neutral’ territory over the past 24 hours, while message volume stayed at ‘normal’ levels.

Acadia’s Head of Research and Development, Elizabeth HZ Thompson, said that the company is “disappointed” by these findings. “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,” Thompson added.

CEO Catherine Owen Adams highlighted that the firm’s approved products are projected to generate over $1 billion in net sales in 2025 despite the disappointment with the trial. The company’s drugs for the treatment of Rett syndrome and for treating hallucinations and delusions associated with Parkinson’s disease psychosis are approved by the U.S. Food and Drug Administration.

“Looking ahead, we anticipate seven Phase 2 or 3 study starts through 2026 and four data readouts by the end of 2027,” the CEO added.

ACAD stock is up 29% this year and approximately 49% over the past 12 months. 

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