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Acadia Pharmaceuticals Initiates Pivotal Phase 3 Study of Carbetocin (ACP-101) for the Treatment of Hyperphagia in Prader-Willi Syndrome
SAN DIEGO--(BUSINESS WIRE)-- Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced the initiation of the Phase 3 COMPASS PWS study evaluating the
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[{"type":"text","content":" SAN DIEGO--(BUSINESS WIRE)--\nAcadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced the initiation of the Phase 3 COMPASS PWS study evaluating the efficacy and safety of carbetocin nasal spray (ACP-101) for the treatment of hyperphagia in Prader-Willi syndrome (PWS). PWS is a rare, neurobehavioral genetic disorder that is estimated to affect 8,000 to 10,000 patients in the United States.1-4 The most common symptom is hyperphagia, which is an unrelenting lack of satiety. Other defining features of PWS may include altered metabolism, developmental delays, behavioral challenges and moderate cognitive deficits.5\n\n\n“Currently there is no FDA-approved treatment for hyperphagia in PWS, which presents serious challenges for those living with this condition and their families,” said Shawn McCandless, M.D., Chair, Department of Genetics and Metabolism, Children’s Hospital Colorado and COMPASS PWS study investigator. “Essentially all experience hyperphagia, feeling a near-constant state of hunger, as though their body is telling them that they are starving, despite being actually well-nourished. As a result, individuals with PWS often exhibit food-seeking behaviors that require constant supervision to prevent life-threatening risks, including gastric rupture, irregular swallowing and choking. I look forward to the outcome of this Phase 3 clinical trial, and the potential of having a treatment option to offer those living with PWS.”\n\n\nCOMPASS PWS is a 12-week, double-blind, randomized, placebo-controlled global Phase 3 trial evaluating the efficacy and safety of carbetocin nasal spray 3.2 mg three times daily (TID) in approximately 170 children and adults aged five to 30 years with PWS. The primary efficacy endpoint of the study is change from baseline to week 12 on the hyperphagia questionnaire for clinical trials (HQ-CT) score, a caregiver assessment for hyperphagia-related behaviors. Participants who complete the Phase 3 study will be eligible to enroll in a long-term, open-label extension study designed to investigate the safety and tolerability of long-term treatment with ACP-101.\n\n\n“The launch of the Phase 3 COMPASS PWS trial will build on previous Phase 3 clinical trial experience, where carbetocin nasal spray 3.2 mg was observed to reduce hyperphagia-related behaviors,” said Ponni Subbiah, M.D., M.P.H., Senior Vice Pr...