\nAcadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that four posters in Rett syndrome will be presented at the upcoming American Academy of Neurology (AAN) annual meeting, being held April 22 \u2013 27, 2023 in Boston, Mass.<\/p>\n
\nThe poster presentations include a secondary analysis reporting on the communication-related endpoints from the pivotal Phase 3 LAVENDER\u2122 study and an interim analysis of the ongoing, open-label DAFFODIL\u2122 study. Additional poster presentations include physician survey data to illustrate the current state of Rett syndrome treatment and healthcare resource utilization in children with Rett syndrome.<\/p>\n
AAN Accepted Posters:<\/b><\/p>\n About Rett Syndrome<\/i><\/p>\n \nRett syndrome is a rare, complex, neurodevelopmental disorder that may occur over four stages and affects approximately 6,000 to 9,000 patients in the U.S., with approximately 4,500 patients currently diagnosed according to an analysis of healthcare claims data.1-4<\/sup> A child with Rett syndrome exhibits an early period of apparently normal development until six to 18 months, when their skills seem to slow down or stagnate. This is typically followed by a duration of regression when the child loses acquired communication skills and purposeful hand use. The child may then experience a plateau period in which they show mild recovery in cognitive interests, but body movements remain severely diminished. As they age, those living with Rett may continue to experience a stage of motor deterioration which can last the rest of the patient\u2019s life.3<\/sup> Rett syndrome is typically caused by a genetic mutation on the MECP2 gene.5<\/sup> In preclinical studies, deficiency in MeCP2 function has been shown to lead to impairment in synaptic communication, and the deficits in synaptic function may be associated with Rett manifestations.5-7<\/sup><\/p>\n \nSymptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities.8<\/sup> Most Rett patients typically live into adulthood and require round-the-clock care.2,9<\/sup><\/p>\n About DAYBUE \u2122 <\/sup>(trofinetide)<\/i><\/p>\n \nTrofinetide is a synthetic version of a naturally occurring molecule known as the tripeptide glycine-proline-glutamate (GPE). The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. In animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals.10,11<\/sup><\/p>\n Important Safety Information for DAYBUE\u2122 (trofinetide)<\/b><\/p>\n Important Safety Information<\/b><\/p>\n Patients should stop taking laxatives before starting DAYBUE. If diarrhea occurs, patients should notify their healthcare provider, consider starting antidiarrheal treatment, and monitor hydration status and increase oral fluids, if needed. Interrupt, reduce dose, or discontinue DAYBUE if severe diarrhea occurs or if dehydration is suspected.<\/p>\n<\/li>\n \nDAYBUE is available as an oral solution (200mg\/mL).<\/p>\n \nPlease read the accompanying full Prescribing Information<\/a>, also available at DAYBUE.com<\/a><\/p>\n About Acadia Pharmaceuticals<\/i><\/p>\n \nAcadia is advancing breakthroughs in neuroscience to elevate life. For almost 30 years we have been working at the forefront of healthcare to bring vital solutions to people who need them most. We developed and commercialized the first and only approved therapies for hallucinations and delusions associated with Parkinson\u2019s disease psychosis and for the treatment of Rett syndrome. Our clinical-stage development efforts are focused on treating the negative symptoms of schizophrenia, Alzheimer\u2019s disease psychosis and neuropsychiatric symptoms in central nervous system disorders. For more information, visit us at www.acadia.com<\/a> and follow us on LinkedIn<\/a> and Twitter<\/a>.<\/p>\n Forward-Looking Statements<\/i><\/p>\n \nStatements in this press release that are not strictly historical in nature are forward-looking statements. These statements include but are not limited to statements regarding the timing of future events. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks and uncertainties inherent in drug development, approval and commercialization. For a discussion of these and other factors, please refer to Acadia\u2019s annual report on Form 10-K for the year ended December 31, 2022, as well as Acadia\u2019s subsequent filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All forward-looking statements are qualified in their entirety by this cautionary statement and Acadia undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof, except as required by law.<\/p>\n 1<\/sup><\/p>\n<\/td>\n \nAcadia Pharmaceuticals Inc, Data on file. RTT US Prevalence. March 2022.<\/p>\n<\/td>\n<\/tr>\n 2<\/sup><\/p>\n<\/td>\n \nFu C, Armstrong D, Marsh E, et al. Consensus guidelines on managing Rett syndrome across the lifespan. BMJ Paediatrics Open<\/i>. 2020; 4: 1-14.<\/p>\n<\/td>\n<\/tr>\n 3<\/sup><\/p>\n<\/td>\n \nKyle SM, Vashi N, Justice MJ. Rett syndrome: a neurological disorder with metabolic components. Open Biol<\/i>. 2018; 8: 170216.<\/p>\n<\/td>\n<\/tr>\n 4<\/sup><\/p>\n<\/td>\n \nAcadia Pharmaceuticals Inc, Data on file.<\/p>\n<\/td>\n<\/tr>\n 5<\/sup><\/p>\n<\/td>\n \nAmir RE, Van den Veyver IB, Wan M, et al. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2. Nat Genet<\/i>. 1999; 23(2): 185-188.<\/p>\n<\/td>\n<\/tr>\n 6<\/sup><\/p>\n<\/td>\n \nFukuda T, Itoh M, Ichikawa T, et al. Delayed maturation of neuronal architecture and synaptogenesis in cerebral cortex of Mecp2-deficient mice. J Neuropathol Exp Neurol<\/i>. 2005; 64(6): 537-544.<\/p>\n<\/td>\n<\/tr>\n 7<\/sup><\/p>\n<\/td>\n \nAsaka Y, Jugloff DG, Zhang L, et al. Hippocampal synaptic plasticity is impaired in the Mecp2-null mouse model of Rett syndrome. Neurobiol Dis<\/i>. 2006; 21(1): 217-227.<\/p>\n<\/td>\n<\/tr>\n 8<\/sup><\/p>\n<\/td>\n \nNeul JL, Kaufmann WE, Glaze DG, et al. Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol<\/i>. 2010; 68(6): 944-950.<\/p>\n<\/td>\n<\/tr>\n 9<\/sup><\/p>\n<\/td>\n \nDaniel C, Tarquinio DO, Hou W, et al. The changing face of survival in Rett syndrome and MECP2-related disorders. Pediatr Neurol<\/i>. 2015; 53(5): 402-411.<\/p>\n<\/td>\n<\/tr>\n 10<\/sup><\/p>\n<\/td>\n \nTropea D, Giacometti E, Wilson NR, et al. Partial reversal of Rett Syndrome-like symptoms in MeCP2 mutant mice. Proc Natl Acad Sci USA<\/i>. 2009; 106(6): 2029-2034.<\/p>\n<\/td>\n<\/tr>\n 11<\/sup><\/p>\n<\/td>\n \nAcadia Pharmaceuticals Inc, Data on file. Study Report 2566-026. 2010.<\/p>\n<\/td>\n<\/tr>\n<\/table>\n \n\u00a0<\/p>\n View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20230420005361\/en\/<\/a><\/span><\/p>\n Media Contact: Investor Contact: KEYWORDS:<\/b> California United States North America<\/p>\n INDUSTRY KEYWORDS:<\/b> Science Biotechnology Research Pharmaceutical Health Mental Health Clinical Trials Other Health<\/p>\n\n
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Acadia Pharmaceuticals Inc.
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Deb Kazenelson
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(818) 395-3043
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media@acadia-pharm.com
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Acadia Pharmaceuticals Inc.
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Mark Johnson, CFA
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(858) 261-2771
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ir@acadia-pharm.com<\/a><\/p>\n
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