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

Acadia Pharmaceuticals Inc. announced the addition of a new candidate in Phase 3 development to its rare disease treatment system, ACP-101, for the treatment of hyperphagia in Prader-Willi syndrome (PWS). Acadia acquired the worldwide development and commercialization rights to 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,”

“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.”

Steve Davis, Acadia’s President and Chief Executive Officer.

Prior to the acquisition of Acadia, Levo conducted an 8-week, multicenter, randomized, double-blind, placebo-controlled, Phase 3 study that evaluated two doses of ACP-101, 3.2 mg and 9.6 mg, compared with placebo three times daily with equal randomization. Preliminary results showed that ACP-101 was safe, well tolerated, and demonstrated a nominally statistically significant effect 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,”

“If positive, we plan to promptly submit a new drug application for the treatment of hyperphagia in PWS to the FDA.”

Doug Williamson, Acadia’s Executive Vice President, Head of Research and Development.

Prader-Willi syndrome is a rare inherited neurobehavioral disorder characterized by life-threatening overeating, metabolic problems, intellectual disability, and mental problems. Other behaviors are estimated to affect 8,000 to 10,000 patients in the United States. There are no FDA-approved medications to treat overeating in PWS.