What's Happening?
Acadia Pharmaceuticals has announced the termination of its investigational treatment for hyperphagia in Prader-Willi syndrome following a failed Phase III trial. The trial, known as COMPASS PWS, tested intranasal carbetocin (ACP-101) but showed no significant change in hyperphagia symptoms after 12 weeks. Acadia acquired ACP-101 from Levo Therapeutics in 2022, but the drug failed to meet endpoints in both the COMPASS and previous CARE PWS trials. Despite higher enrollment in COMPASS, the trial did not yield meaningful clinical improvements, leading Acadia to discontinue further investigation of the drug.
Why It's Important?
The discontinuation of ACP-101 highlights the challenges faced by pharmaceutical companies in developing treatments for rare neurological conditions like Prader-Willi syndrome. The failure of the trial impacts Acadia's portfolio and may shift industry focus to other potential treatments for hyperphagia. The decision also underscores the importance of robust clinical trials and the difficulty in addressing complex conditions with limited therapeutic options. Acadia's setback may influence investment and research strategies in the field of rare disease drug development.
What's Next?
Following the trial's failure, Acadia's attention may turn to its Phase II study of ACP-204 for psychosis related to Alzheimer's disease, expected in mid-2026. The company may also explore other avenues for growth and innovation, potentially shifting resources to more promising projects. The broader industry may continue to seek effective treatments for Prader-Willi syndrome, with ongoing research and development efforts aimed at addressing unmet needs in this area.
