Acadia Pharmaceuticals Presents DAYBUE™ (trofinetide) Real-World Evidence and Additional Data in Rett Syndrome at the 2024 International Rett Syndrome Foundation (IRSF) Annual Scientific Meeting
SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals, Inc. (Nasdaq: ACAD) today announced that interim data from the open-label real-world LOTUS™ study will be presented at the 2024 International Rett Syndrome Foundation (IRSF) Annual Scientific Meeting, being held this week in Westminster, Colorado. LOTUS is an ongoing, caregiver-reported study evaluating the efficacy and tolerability outcomes in patients with Rett syndrome treated with DAYBUE™ (trofinetide).
“These interim data from the LOTUS study reinforce that the clinical effectiveness of DAYBUE in the real-world setting is consistent with results observed in the DAYBUE Phase 3 clinical trial program and further characterize what Rett syndrome symptom improvements may look like to caregivers,” said Ponni Subbiah, M.D., M.P.H., Senior Vice President, Global Head of Medical Affairs and Chief Medical Officer. “These initial findings also illustrate caregivers’ strategies to manage gastrointestinal tolerability in the real world and underscore the value of education and effective implementation of diarrhea management strategies to help families improve the patient experience.”
Six-month interim findings evaluating DAYBUE in 101 Rett syndrome patients with an age range of two to 60 years old from the Phase 4, observational, prospective study showed caregivers of more than two-thirds of enrolled participants reported improvements at Month 1 in at least one Rett syndrome symptoms category. This was measured using the Behavioral Improvement Questionnaire (BIQ) developed by Acadia in consultation with Rett experts and caregivers. The most consistently reported improvements over six months were nonverbal communication (58.5%), alertness (51.2%) and social interaction/connectedness (40.2%). Additionally, diarrhea and formed/normal stool were both common, as measured by the Gastrointestinal (GI) Health Questionnaire also developed by Acadia for this study. Some participants reported initiating therapy on doses less than half of the FDA approved dose and increasing over several weeks; the majority of patients were on >90% of labeled dose by week 10. The results of this six-month follow-up are limited by lack of placebo arm, missing data 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.
This interim analysis of LOTUS data will be presented at the IRSF Scientific Meeting, as well as new findings from health economics and outcomes research in Rett syndrome care and encore data from DAYBUE open-label extension and clinical trial program studies. IRSF Poster Presentations, Tuesday, June 18, 5pm MDT-7pm MDT:
Assessing experiences with trofinetide for Rett syndrome: interviews with caregivers of patients in LAVENDER™, LILAC-1™, and LILAC-2™ studies
Real-world benefits and tolerability of trofinetide for the treatment of Rett Syndrome: the LOTUS study
Prevalence of respiratory clinical outcomes among patients with Rett syndrome: analysis of real-world data in the United States
Trofinetide for the treatment of Rett syndrome: long-term safety and efficacy results from the open-label LILAC-2 study
Prevalence of epilepsy in individuals with Rett syndrome–a real-world evidence analysis
Prevalence of respiratory comorbidities among patients with Rett syndrome
Prevalence of nutritional deficiency, underweight status and gastrostomy among patients with Rett syndrome: an analysis of electric health record (EHR) data
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