Phase III LAVENDER trial of Daybue in Rett syndrome published in Nature Medicine
Acadia Pharmaceuticals announced that Nature Medicine published results from the pivotal Phase III LAVENDER trial, a 12-week randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of Daybue (trofinetide) in patients with Rett syndrome five to 20 years of age
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.
See: "Trofinetide for the treatment of Rett syndrome: a randomized phase iii study" Jeffrey L. Neul et al. Nature Medicine (2023) Published: 08 June 2023 https://www.nature.com/articles/s41591-023-02398-1
Related news and insights
Karuna Therapeutics, Inc. a biopharmaceutical company driven to discover, develop, and deliver transformative medicines for people living with psychiatric and neurological conditions, announced the submission of a New Drug Application (NDA) to the FDA for KarXT (xanomeline-trospium) for the treatment of schizophrenia
BrainStorm Cell Therapeutics Inc. announced the outcome of the FDA's Cellular, Tissue and Gene Therapies Advisory Committee meeting to review the Biologics License Application (BLA) for NurOwn, an investigational mesenchymal stem cell therapy for the treatment of amyotrophic lateral sclerosis (ALS)
Merck Inc., known as MSD outside of the United States and Canada, announced that the FDA has accepted for priority review a new Biologics License Application (BLA) for sotatercept, Merck’s novel investigational activin signaling inhibitor, for the treatment of adult patients with pulmonary arterial hypertension (PAH) (WHO Group 1)