Acadia Presents DAYBUE™ Data in Rett Syndrome at 2024 IRSF Annual Meeting

SAN DIEGO--Acadia Pharmaceuticals, Inc. has announced that interim data from the open-label LOTUS™ study will be showcased at the 2024 International Rett Syndrome Foundation (IRSF) Annual Scientific Meeting in Westminster, Colorado. This ongoing caregiver-reported study evaluates the efficacy and tolerability of DAYBUE™ (trofinetide) in patients with Rett syndrome.

Ponni Subbiah, M.D., M.P.H., Senior Vice President, Global Head of Medical Affairs, and Chief Medical Officer at Acadia, stated that the interim data reinforce the real-world effectiveness of DAYBUE, aligning with results from the DAYBUE Phase 3 clinical trials. These initial findings also highlight caregivers’ strategies for managing gastrointestinal tolerability and emphasize the importance of education and effective diarrhea management to enhance patient experiences.

The study includes 101 Rett syndrome patients aged between two and 60 years. Caregivers of over two-thirds of the participants reported noticeable improvements in at least one symptom category by Month 1, as measured by the Behavioral Improvement Questionnaire (BIQ). The most frequently noted improvements over six months were in nonverbal communication (58.5%), alertness (51.2%), and social interaction/connectedness (40.2%). Gastrointestinal symptoms, such as diarrhea and normal stool formation, were common according to the Gastrointestinal (GI) Health Questionnaire.

Some participants began therapy at doses less than half of the FDA-approved amount, gradually increasing to above 90% of the labeled dose by week 10. However, the study's results are limited due to the absence of a placebo arm, missing data, and its online nature.

Participants are enrolled in the LOTUS study for at least 12 months, with the option to extend for another 12 months. This interim analysis will be presented at the IRSF Scientific Meeting, alongside new findings from health economics and outcomes research in Rett syndrome care, and data from DAYBUE open-label extension and clinical trial programs.

IRSF Poster Presentations on Tuesday, June 18, include:
- Caregiver experiences with trofinetide for Rett syndrome from the LAVENDER™, LILAC-1™, and LILAC-2™ studies
- Real-world benefits and tolerability of trofinetide in the LOTUS study
- Analysis of respiratory clinical outcomes and epilepsy prevalence among Rett syndrome patients in the U.S.
- Long-term safety and efficacy of trofinetide from the open-label LILAC-2 study
- Analysis of nutritional deficiencies, underweight status, and gastrostomy prevalence among Rett syndrome patients

Rett syndrome is a rare, complex neurodevelopmental disorder affecting about 6,000 to 9,000 people in the U.S., with 5,000 currently diagnosed. It typically begins with normal development, followed by a regression phase where communication skills and purposeful hand use are lost. A plateau period may follow, with slight cognitive recovery but severe motor impairments. Most patients experience a lifelong motor deterioration stage. Rett syndrome is usually caused by a mutation in the MECP2 gene, leading to synaptic communication deficits.

Symptoms can also include hand stereotypies like wringing and clapping, and gait abnormalities. Most patients live into adulthood and require constant care.

DAYBUE (trofinetide) is a synthetic analog of a tripeptide of insulin-like growth factor 1. Though its exact therapeutic mechanism is unknown, animal studies show it increases dendritic branching and synaptic plasticity signals.

Common adverse reactions to DAYBUE include diarrhea, vomiting, fever, seizure, anxiety, decreased appetite, fatigue, and nasopharyngitis. Patients should avoid laxatives before starting DAYBUE and monitor for severe diarrhea and significant weight loss.

Acadia Pharmaceuticals is dedicated to advancing neuroscience breakthroughs. They have developed the first FDA-approved drugs for Parkinson’s disease psychosis and Rett syndrome and are working on treatments for Prader-Willi syndrome and Alzheimer’s disease psychosis, among other neuropsychiatric conditions.

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