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Nature Medicine Publishes Results from Pivotal Phase 3 LAVENDER™ Study Evaluating DAYBUE™ (trofinetide) Efficacy and Safety in Patients with Rett Syndrome
-- Statistically significant differences demonstrated between DAYBUE™ and placebo on efficacy endpoints relevant to Rett syndrome suggest treatment
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[{"type":"text","content":"\n-- Statistically significant differences demonstrated between DAYBUE™ and placebo on efficacy endpoints relevant to Rett syndrome suggest treatment potentially capable of modifying core symptoms consistent with underlying pathophysiology of disease\n\n\n-- Study results provided basis for first FDA-approved treatment for Rett syndrome\n\n\n SAN DIEGO--(BUSINESS WIRE)--\nAcadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that Nature Medicine published results from the pivotal Phase 3 LAVENDER™ trial, a 12-week randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of DAYBUE™ in patients with Rett syndrome five to 20 years of age.\n\n\n“The LAVENDER study was designed to measure the effect of DAYBUE treatment on the range of behavioral, communication, and physical Rett syndrome symptoms that significantly impact the quality of life for patients and their loved ones,” said Jeffrey L. Neul, M.D., Ph.D., Annette Schaffer Eskind Chair and Director, Vanderbilt Kennedy Center, Professor of Pediatrics, Division of Neurology, Pharmacology, and Special Education, Vanderbilt University Medical Center and LAVENDER study lead author. “The publication of the efficacy and safety results for DAYBUE reinforces the significance of this study as a critical advancement in Rett syndrome research, furthering our ability to treat this devastating disease.”\n\n\nIn the study, treatment with DAYBUE (n=93) demonstrated statistically significant improvement compared to placebo (n=94) on both co-primary efficacy endpoints, with the following key findings:\n\n\n\nThe mean change from baseline to week 12 in the Rett Syndrome Behaviour Questionnaire (RSBQ) total score was −5.1 and −1.7 in the DAYBUE and placebo groups, respectively. Based on the mixed-effects model for repeated measures (MMRM) analysis, the least squares mean (LSM) [SE] change from baseline to week 12 in the RSBQ total score was statistically significantly greater with DAYBUE (−4.9 [0.94]) than with placebo (−1.7 [0.90]), with an LSM placebo-subtracted difference of −3.1 [1.30], a 95% confidence interval (CI) of −5.7 to −0.6, a p-value of 0.0175, and a Cohen’s d effect size of 0.37.\n\n\n\n\nChange from baseline for all RSBQ domain subscores were numerically in favor of DAYBUE.\n\n\n\n\nAt week 12 in the DAYBUE and placebo groups, respectively, the mean...