Acadia Pharmaceuticals to Present DAYBUE™ (trofinetide) Clinical Data and Rett Syndrome Real World Evidence at the 2023 American Academy of Neurology (AAN) Annual Meeting

Rhea-AI Summary

Acadia Pharmaceuticals (NASDAQ: ACAD) announced the presentation of four posters at the American Academy of Neurology (AAN) annual meeting from April 22 to 27, 2023, in Boston, Mass. The posters include a secondary analysis of communication-related endpoints from the Phase 3 LAVENDER™ study and an interim analysis of the DAFFODIL™ study. Notable presentations involve real-world physician surveys and an analysis of healthcare resource utilization in Rett syndrome patients. The poster P13 highlights the benefits of Trofinetide compared to placebo in communication abilities among individuals with Rett syndrome, thus showcasing the drug's potential. Rett syndrome is a rare neurological disorder affecting approximately 6,000 to 9,000 patients in the U.S., with Trofinetide being a key treatment option.

Positive

• Presentation at a major conference enhances visibility and credibility.
• Trofinetide's interim results suggest benefits compared to placebo in communication abilities for Rett syndrome patients.

Negative

• None.

SAN DIEGO--(BUSINESS WIRE)-- 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:

• P12 - Poster Session 12/P12.4-002: Clinical Management of Rett Syndrome in the Real World: A Physician Survey. Online and in person, Wednesday, April 26, 5:30 – 6:30 p.m. ET
• P13 - Poster Session 13/P.13.9-004: Elevated Healthcare Resources Utilization and Costs Among Individuals Diagnosed with Rett Syndrome. Online and in person, Thursday, April 27, 8:00 – 9:00 a.m. ET
• P13 - Poster Session 13/P13.9-005: Trofinetide for the Treatment of Rett Syndrome: An Open-Label Study in Girls Aged 2 to 4 Years. Online and in person, Thursday, April 27, 8:00 – 9:00 a.m. ET
• P13 - Poster Session 13/P13.9-006: Treatment with Trofinetide Shows Benefit Compared with Placebo in the Ability to Communicate in Individuals with Rett syndrome. Online and in person, Thursday, April 27, 8:00 – 9:00 a.m. ET

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

• Warnings and Precautions
  • Diarrhea: In a 12-week study and in long-term studies, an aggregate of 85% of patients treated with DAYBUE experienced diarrhea. In those treated with DAYBUE, 49% either had persistent diarrhea or recurrence after resolution despite dose interruptions, reductions, or concomitant antidiarrheal therapy. Diarrhea severity was of mild or moderate severity in 96% of cases. In the 12-week study, antidiarrheal medication was used in 51% of patients treated with DAYBUE.
    
    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.
    
    
  • Weight Loss: In the 12-week study, 12% of patients treated with DAYBUE experienced weight loss of greater than 7% from baseline, compared to 4% of patients who received placebo. In long-term studies, 2.2% of patients discontinued treatment with DAYBUE due to weight loss. Monitor weight and interrupt, reduce dose, or discontinue DAYBUE if significant weight loss occurs.

• Adverse Reactions: The common adverse reactions (≥5% for DAYBUE-treated patients and at least 2% greater than in placebo) reported in the 12-week study were diarrhea (82% vs 20%), vomiting (29% vs 12%), fever (9% vs 4%), seizure (9% vs 6%), anxiety (8% vs 1%), decreased appetite (8% vs 2%), fatigue (8% vs 2%), and nasopharyngitis (5% vs 1%).

• Drug Interactions: Effect of DAYBUE on other Drugs
  • DAYBUE is a weak CYP3A4 inhibitor; therefore, plasma concentrations of CYP3A4 substrates may be increased if given concomitantly with DAYBUE. Closely monitor when DAYBUE is used in combination with orally administered CYP3A4 sensitive substrates for which a small change in substrate plasma concentration may lead to serious toxicities.
  • Plasma concentrations of OATP1B1 and OATP1B3 substrates may be increased if given concomitantly with DAYBUE. Avoid the concomitant use of DAYBUE with OATP1B1 and OATP1B3 substrates for which a small change in substrate plasma concentration may lead to serious toxicities.

• Use in Specific Population: Renal Impairment
  • DAYBUE is not recommended for patients with moderate or severe renal impairment.

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

Forward-Looking Statements

Statements 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’s annual report on Form 10-K for the year ended December 31, 2022, as well as Acadia’s 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.

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		Tropea D, Giacometti E, Wilson NR, et al. Partial reversal of Rett Syndrome-like symptoms in MeCP2 mutant mice. Proc Natl Acad Sci USA. 2009; 106(6): 2029-2034.
11		Acadia Pharmaceuticals Inc, Data on file. Study Report 2566-026. 2010.

 

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Media Contact:

Acadia Pharmaceuticals Inc.

Deb Kazenelson

(818) 395-3043

media@acadia-pharm.com



Investor Contact:

Acadia Pharmaceuticals Inc.

Mark Johnson, CFA

(858) 261-2771

ir@acadia-pharm.com

Source: Acadia Pharmaceuticals Inc.

FAQ

What are the key findings from the recent Acadia Pharmaceuticals press release regarding Trofinetide?

The press release highlights that Trofinetide shows benefits over placebo in communication abilities for individuals with Rett syndrome, based on ongoing studies.

When and where will the presentations about Rett syndrome take place?

The presentations will occur at the American Academy of Neurology annual meeting from April 22 to 27, 2023, in Boston, Mass.

What is the significance of the LAVENDER™ study results presented by Acadia Pharmaceuticals?

The LAVENDER™ study provides secondary analysis on communication-related endpoints that could support the efficacy of Trofinetide in treating Rett syndrome.

How many patients are affected by Rett syndrome in the U.S. according to Acadia Pharmaceuticals?

Approximately 6,000 to 9,000 patients are affected by Rett syndrome in the U.S.