Acadia Pharmaceuticals publishes interim LOTUS study data in journal

Acadia Pharmaceuticals (ACAD) announced that the journal Developmental Medicine and Child Neurology published interim results from the caregiver-reported observational online, open-label, ongoing LOTUS study evaluating effectiveness and tolerability outcomes in patients with Rett syndrome who are prescribed Daybue under routine clinical care in the U.S. Findings included reported improvements in symptoms of Rett syndrome, with early insights into managing gastrointestinal symptoms in the real world among patients receiving up to 12 months of treatment with DAYBUE. 71-90% of those who filled out the Behavioral Improvement questionnaire reported at least one behavioral symptom improvement that were new or maintained when compared with before DAYBUE treatment. The BIQ was developed by Acadia specifically for this study to document caregiver observations. Consistent with the previously reported six-month interim analysis, the most frequent caregiver-reported improvements were nonverbal communication, alertness and social interaction/connectedness, as measured by the BIQ. Additionally, the median change from baseline in Quality-of-Life Inventory-Disability Questionnaire total score was 4.6 for those patients who had both baseline and 12-month assessments. The incidence of diarrhea reported by caregivers varied from weeks 1 to 12 and months 4 to 12. Most reports of diarrhea were contained inside the patient’s diaper, as measured by the Gastrointestinal Health Questionnaire, also developed by Acadia specifically for this study in consultation with Rett experts and caregivers. The most commonly reported diarrhea management strategies included avoiding constipation medications, increasing fluid intake, consuming supplementary fiber, and using antidiarrheal medication. Caregivers reported normal stools over the last 3 days immediately prior to completing the GI assessment from weeks 1 to 12 and months 4 to 12. The median dose reported at Week 1 was 36% of the target weight-banded FDA approved dose; by Week 10 onwards, the median dose was at least 80% of labeled dose. The results of this 12-month follow-up are limited by the number of patients who had reached later time points, which resulted in the data being restricted to 12 months, lack of a placebo arm, missing data, lack of validation of BIQ and GI questionnaires, reliance solely on caregiver reports, the use of descriptive statistics and the online nature of this study. Participants are being enrolled in the LOTUS study for at least 12 months from initiation of DAYBUE treatment, with the option to extend participation for an additional 12 months.