Acadia Pharmaceuticals Presents Real-World Data on DAYBUE™ (trofinetide) for Rett Syndrome

Interim data from the open-label real-world LOTUS™ study, presented by Acadia Pharmaceuticals Inc., showcases the effectiveness and tolerability of DAYBUE™ (trofinetide) in treating Rett syndrome. The study, involving 101 patients aged two to 60 years, revealed that caregivers of over two-thirds of participants reported improvements in at least one Rett syndrome symptom category within the first month of treatment. Significant improvements were noted in nonverbal communication, alertness, and social interaction/connectedness over six months. The study also addressed the management of gastrointestinal tolerability, emphasizing the importance of education and effective diarrhea management strategies. Despite the promising results, the study's findings are limited by the absence of a placebo arm, missing data, and its online nature. The LOTUS study continues to enroll participants for at least 12 months, with an option to extend for an additional 12 months. This research underscores the potential of DAYBUE™ in improving the quality of life for individuals with Rett syndrome and their caregivers.