Rett Syndrome patients are seeing promising results from the open-label extension studies LILAC-1™ and LILAC-2™ evaluating the long-term safety and efficacy of DAYBUE™ (trofinetide), as published in the journal Med. Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) announced these findings, which highlight improvements in Rett symptoms as measured by the Rett Syndrome Behaviour Questionnaire (RSBQ). The studies involved patients aged five to 22 years and demonstrated consistent safety profiles with notable improvements in symptoms and quality of life.
The LILAC-1 study, a 40-week extension of the Phase 3 LAVENDER™ trial, evaluated 154 females aged five to 21 years. It followed up on those who had completed the LAVENDER study, which provided double-blind treatment with either DAYBUE or a placebo. The results showed significant improvements in RSBQ scores for those who continued treatment with DAYBUE, regardless of their initial treatment group. Specifically, the mean change from the LAVENDER baseline to week 40 in LILAC-1 was −7.3 for participants who had been treated with DAYBUE and −7.0 for those initially on placebo and then switched to DAYBUE. These findings were consistent across various age groups and severity levels of Rett syndrome, suggesting robust efficacy.
Rett Syndrome Treatment: DAYBUE™ Shows Long-Term Efficacy
In the subsequent LILAC-2 study, 77 participants continued open-label treatment for an additional 104 weeks. The results further supported the long-term benefits of DAYBUE, with 81.8% of participants showing a ≥ 10% improvement in RSBQ scores by week 104. The mean change in RSBQ score from LAVENDER baseline to week 104 of LILAC-2 was −9.8 for those initially treated with DAYBUE and −13.8 for those switched from placebo. Additionally, the Clinical Global Impression–Improvement (CGI-I) scores indicated sustained positive effects. The most common side effects in both studies were diarrhea and vomiting, primarily mild to moderate in severity.
The study included caregiver exit interviews, which provided qualitative insights into the impact of DAYBUE. Caregivers reported improvements in patient engagement, hand use, and eye gaze. Some participants even acquired new sounds or words, indicating potential cognitive and communicative benefits. These anecdotal reports underscore the meaningful improvements in daily living and interaction for those on DAYBUE.
Rett Syndrome Data Reinforces DAYBUE’s Potential
The consistent safety profile observed in LILAC-1 and LILAC-2 aligns with previous findings from the LAVENDER trial. Diarrhea and vomiting were the most commonly reported adverse events, but they were generally mild or moderate. The studies were conducted during the COVID-19 pandemic, which also led to some cases of COVID-19 among participants. However, the overall safety and tolerability of DAYBUE remained favorable.
Dr. Alan Percy, the lead author of the LILAC studies, emphasized the significance of these findings in understanding the long-term impact of DAYBUE for Rett syndrome. Similarly, Dr. Ponni Subbiah from Acadia Pharmaceuticals highlighted the growing body of evidence supporting DAYBUE’s efficacy and safety.
The publication of these studies in Med represents a significant step forward in the treatment landscape for Rett syndrome. With the high unmet need for effective therapies, DAYBUE offers a promising option for patients and their caregivers. As research continues, the goal is to provide robust, evidence-based treatments that improve the quality of life for those affected by this challenging condition.
Resource: Business Wire, July 18, 2024
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