Clinical Data from Open-Label Extension LILAC-1™ and LILAC-2™ Studies Evaluating Long-Term Safety and Efficacy of DAYBUE™ (trofinetide) in Patients with Rett Syndrome Published in Med

— Continued Improvements in RSBQ and CGI-I scores seen with long-term DAYBUE treatment in Phase 3 LAVENDER™ and LILAC studies

— DAYBUE safety profile was consistent with findings from the LAVENDER trial

Excerpt from the Press Release:

SAN DIEGO–(BUSINESS WIRE)–Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the journal Med published results from two open-label extension studies, LILAC-1™ and LILAC-2™, which showed that patients treated with DAYBUE^™ who completed these studies experienced improvement in Rett symptoms as measured by the Rett Syndrome Behaviour Questionnaire (RSBQ). LILAC-1 was a 40-week, open-label extension study of the 12-week Phase 3 LAVENDER™ trial, evaluating the long-term safety and efficacy of DAYBUE in patients with Rett syndrome five to 21 years of age. LILAC-2 was a 32-month open-label extension study, evaluating the long-term safety and efficacy of DAYBUE in females aged five to 22 years who completed LILAC-1. The most common side effects reported in these studies were diarrhea and vomiting. Results from both studies also showed DAYBUE’s safety profile was consistent with results from the LAVENDER trial.

“These data from the open-label extension LILAC studies provide deeper insight into the long-term safety and potential benefit of DAYBUE for patients with Rett syndrome,” said Alan Percy, M.D., Professor of Pediatrics, Neurology, Neurobiology, Genetics, and Psychology at University of Alabama, Birmingham and lead author for the LILAC-1 and LILAC-2 studies. “These publications add to the growing body of research on clinical experience with DAYBUE and its potential ongoing impact for those living with this condition.”

“These findings from LILAC-1 and LILAC-2 add to the results from the pivotal Phase 3 LAVENDER trial and the ongoing LOTUS real-world evidence study and include patients who have been on treatment for over two years, contributing to a robust and growing portfolio of impactful data furthering our understanding of DAYBUE for the treatment of Rett syndrome,” said Ponni Subbiah, M.D., M.P.H., Acadia’s Senior Vice President, Global Head of Medical Affairs and Chief Medical Officer.

About the data:

• In LILAC-1, 154 females with Rett syndrome five to 21 years of age received open-label treatment with DAYBUE for 40 weeks following double-blind treatment with DAYBUE (n = 69) or placebo (n = 85) in the 12-week LAVENDER study.

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