Acadia Pharmaceuticals Announces Phase 3 Development Candidate ACP-101 for Prader-Willi Syndrome

Acadia Pharmaceuticals today announced the addition of a new Phase 3 development candidate to its rare disease portfolio, ACP-101 (intranasal carbetocin), for the treatment of hyperphagia (a false and unrelenting state of starvation) in Prader-Willi syndrome (PWS). Acadia acquired worldwide rights to develop and commercialize ACP-101 with the acquisition of Levo Therapeutics in June 2022.

“Acadia’s acquisition of ACP-101 demonstrates our commitment to acquiring and developing novel drug candidates that address significant unmet needs in central nervous system disorders. The addition of this drug candidate to our rare disease portfolio is an important next step in the execution of our business development strategy,” said Steve Davis, Acadia’s President and Chief Executive Officer. “Prader-Willi syndrome is a rare genetic disorder with no approved treatments, characterized by life-threatening hyperphagia, in addition to a broad range of severe metabolic issues and behavioral challenges. We look forward to working with the Prader-Willi community and clinical experts as we continue to advance development of this program.”

Prior to Acadia’s acquisition, Levo conducted a Phase 3 multi-center, randomized, double-blind, 8-week placebo-controlled study evaluating two doses of ACP-101, 3.2 mg and 9.6 mg, versus placebo three times daily with an even randomization (1:1:1). Top-line results showed that ACP-101 was safe and well-tolerated and demonstrated nominally statistically significant efficacy at the 3.2 mg dose.

“We recently met with the FDA and reached alignment to further evaluate the 3.2 milligram dose of ACP-101 in a pivotal Phase 3 study,” said Doug Williamson, Acadia’s Executive Vice President, Head of Research and Development. “If positive, we plan to promptly submit a new drug application for the treatment of hyperphagia in PWS to the FDA.”

Prader-Willi syndrome is a rare, neurobehavioral genetic disorder characterized by severe and life-threatening hyperphagia, metabolic issues, intellectual deficits, and other behavioral problems that are estimated to affect 8,000 to 10,000 patients in the United States. There is no FDA-approved medicine to treat hyperphagia in PWS.