Acadia Pharmaceuticals to Present DAYBUE™ (trofinetide) Clinical Data and Rett Syndrome Real World Evidence at the 2023 American Academy of Neurology (AAN) Annual Meeting
April 20, 2023 at 09:04 AM EDT
Acadia 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 – 27, 2023 in Boston, Mass.
The poster presentations include a secondary analysis reporting on the communication-related endpoints from the pivotal Phase 3 LAVENDER™ study and an interim analysis of the ongoing, open-label DAFFODIL™ 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.
AAN Accepted Posters:
About Rett Syndrome
Rett 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 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’s life.3 Rett syndrome is typically caused by a genetic mutation on the MECP2 gene.5 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
Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities.8 Most Rett patients typically live into adulthood and require round-the-clock care.2,9
About DAYBUE ™ (trofinetide)
Trofinetide 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
Important Safety Information for DAYBUE™ (trofinetide)
Important Safety Information
DAYBUE is available as an oral solution (200mg/mL).
About Acadia Pharmaceuticals
Acadia 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’s disease psychosis and for the treatment of Rett syndrome. Our clinical-stage development efforts are focused on treating the negative symptoms of schizophrenia, Alzheimer’s disease psychosis and neuropsychiatric symptoms in central nervous system disorders. For more information, visit us at www.acadia.com and follow us on LinkedIn and Twitter.
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