137 0 Kommentare Nature Medicine Publishes Results from Pivotal Phase 3 LAVENDER Study Evaluating DAYBUE (trofinetide) Efficacy and Safety in Patients with Rett Syndrome

Acadia 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.

“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.”

In 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:

The 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.

Change from baseline for all RSBQ domain subscores were numerically in favor of DAYBUE.

At week 12 in the DAYBUE and placebo groups, respectively, the mean [SE] Clinical Global Impression–Improvement (CGI-I) scores were 3.5 [0.08] and 3.8 [0.06]. MMRM analysis showed a statistically significant improvement with DAYBUE compared with placebo at week 12, with a LSM [SE] difference of −0.3 [0.10], a 95% CI of −0.5 to −0.1, a p-value of 0.0030 and a Cohen’s d effect size of 0.47.

A subgroup analysis showed a similar benefit with DAYBUE over placebo irrespective of age, baseline RSBQ severity, and category of documented disease-causing MECP2 mutation severity.

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“We are pleased that Nature Medicine has published these important results from the pivotal LAVENDER trial,” said Kathie Bishop, Acadia’s Senior Vice President, Chief Scientific Officer and Head of Rare Disease. “The positive findings from this study were integral to the FDA’s approval of DAYBUE for Rett syndrome, ushering in the first available treatment option approved by the agency to address a multitude of challenging symptoms in those living with Rett syndrome with the potential to create significant impact for patients and their families.”

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