Le LÚzard
Classified in: Health, Science and technology
Subjects: Annual Meeting, Business Update

Acadia Pharmaceuticals Presents New DAYBUEtm (trofinetide) Clinical Data at the 2024 American Academy of Neurology (AAN) Annual Meeting


Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that two oral presentations featuring DAYBUEtm (trofinetide) findings in Rett syndrome will be presented at the American Academy of Neurology (AAN) 2024 annual meeting, being held this week in Denver, CO.

The presentations include outcomes from the DAFFODILtm, a Phase 2/3 open-label study evaluating the safety, tolerability and exploratory efficacy of DAYBUE in girls aged two to four living with Rett syndrome, as well as findings from exit interviews with caregivers of individuals living with Rett syndrome who participated in the LAVENDERtm, LILACtm, LILAC-2tm and DAFFODIL studies.

AAN Oral Presentations:

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 5,000 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.5 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 DAYBUEtm (trofinetide)

Trofinetide is a synthetic analog of the N-terminal tripeptide of insulin-like growth factor 1. The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. Trofinetide is thought to enhance neuronal synaptic function and morphology. This hypothesis is supported by findings from studies of trofinetide in a methyl-CpG-binding protein 2 gene (Mecp2) mouse model of Rett syndrome, in which increased branching of the dendrites that form synapses and synaptic plasticity signals were observed.10

Important Safety Information for DAYBUEtm (trofinetide)

DAYBUE is available as an oral solution (200 mg/mL).

Please read the accompanying full Prescribing Information, also available at DAYBUE.com

About Acadia Pharmaceuticals

Acadia is advancing breakthroughs in neuroscience to elevate life. For 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 FDA-approved drug to treat hallucinations and delusions associated with Parkinson's disease psychosis and the first and only FDA-approved drug for the treatment of Rett syndrome. Our clinical-stage development efforts are focused on Prader-Willi syndrome, Alzheimer's disease psychosis and multiple other programs targeting neuropsychiatric symptoms in central nervous system disorders. For more information, visit us at Acadia.com and follow us on LinkedIn and Twitter.

References

1 Acadia Pharmaceuticals Inc., Data on file. RTT US Prevalence. March 2022.
2 Fu C, Armstrong D, Marsh E, et al. Consensus guidelines on managing Rett syndrome across the lifespan. BMJ Paediatrics Open. 2020; 4: 1-14.
3 Kyle SM, Vashi N, Justice MJ. Rett syndrome: a neurological disorder with metabolic components. Open Biol. 2018; 8: 170216.
4 Acadia Pharmaceuticals Inc., Data on file.
5 Amir 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. 1999; 23(2): 185-188.
6 Fukuda 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. 2005; 64(6): 537-544.
7 Asaka Y, Jugloff DG, Zhang L, et al. Hippocampal synaptic plasticity is impaired in the Mecp2-null mouse model of Rett syndrome. Neurobiol Dis. 2006; 21(1): 217-227.
8 Neul JL, Kaufmann WE, Glaze DG, et al. Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol. 2010; 68(6): 944-950.
9 Daniel C, Tarquinio DO, Hou W, et al. The changing face of survival in Rett syndrome and MECP2-related disorders. Pediatr Neurol. 2015; 53(5): 402-411.
10 Acadia Pharmaceuticals Inc., Data on file.


These press releases may also interest you

at 06:51
82% (n=9/11) of patients on felzartamab experienced resolution of antibody-mediated rejection per Banff criteria on biopsy at 24 weeks versus 20% (n=2/10) who received placebo, and patients treated with felzartamab showed reductions in disease-linked...

at 06:15
In the ALIGN study, atrasentan, in addition to supportive care with a renin-angiotensin system (RAS) inhibitor, demonstrated a statistically significant 36.1% proteinuria (protein in urine) reduction vs. placebo + supportive care at 36...

at 02:32
The buzz surrounding Live4Well's successful Genesis NFT membership launch on May 23 has captivated both traditional and web3 communities. Combining the power of AI technology andádecentralized physical infrastructure (DePin) concept, Live4Well has...

at 00:22
Built after China's reform and opening up,áRizhao Port in east China's Shandong Province is a vital global hub for the transshipment of energy and bulk raw materials. Through recent technological innovation, the port has been upgraded into a modern...

at 00:20
The buzz surrounding Live4Well's successful Genesis NFT membership launch on May 23 has captivated both traditional and web3 communities. Combining the power of AI technology andádecentralized physical infrastructure (DePin) concept, Live4Well has...

at 00:15
Africa is an amazingly beautiful continent with a powerful young generation who have the inherent right to an inclusive and continued quality education. Yet ? and especially in Sub-Saharan Africa ? they have been left furthest behind for far too...



News published on and distributed by: