Acadia Pharmaceuticals, Inc. (Nasdaq: ACAD) today announced that
interim data from the open-label real-world LOTUS™ study will be
presented at the 2024 International Rett Syndrome Foundation (IRSF)
Annual Scientific Meeting, being held this week in Westminster,
Colorado. LOTUS is an ongoing, caregiver-reported study evaluating
the efficacy and tolerability outcomes in patients with Rett
syndrome treated with DAYBUE™ (trofinetide).
“These interim data from the LOTUS study reinforce that the
clinical effectiveness of DAYBUE in the real-world setting is
consistent with results observed in the DAYBUE Phase 3 clinical
trial program and further characterize what Rett syndrome symptom
improvements may look like to caregivers,” said Ponni Subbiah,
M.D., M.P.H., Senior Vice President, Global Head of Medical Affairs
and Chief Medical Officer. “These initial findings also illustrate
caregivers’ strategies to manage gastrointestinal tolerability in
the real world and underscore the value of education and effective
implementation of diarrhea management strategies to help families
improve the patient experience.”
Six-month interim findings evaluating DAYBUE in 101 Rett
syndrome patients with an age range of two to 60 years old from the
Phase 4, observational, prospective study showed caregivers of more
than two-thirds of enrolled participants reported improvements at
Month 1 in at least one Rett syndrome symptoms category. This was
measured using the Behavioral Improvement Questionnaire (BIQ)
developed by Acadia in consultation with Rett experts and
caregivers. The most consistently reported improvements over six
months were nonverbal communication (58.5%), alertness (51.2%) and
social interaction/connectedness (40.2%). Additionally, diarrhea
and formed/normal stool were both common, as measured by the
Gastrointestinal (GI) Health Questionnaire also developed by Acadia
for this study. Some participants reported initiating therapy on
doses less than half of the FDA approved dose and increasing over
several weeks; the majority of patients were on >90% of labeled
dose by week 10. The results of this six-month follow-up are
limited by lack of placebo arm, missing data and the online nature
of this study.
Participants are being enrolled in the LOTUS study for at least
12 months from initiation of DAYBUE treatment, with the option to
extend participation for an additional 12 months.
This interim analysis of LOTUS data will be presented at the
IRSF Scientific Meeting, as well as new findings from health
economics and outcomes research in Rett syndrome care and encore
data from DAYBUE open-label extension and clinical trial program
studies.
IRSF Poster Presentations, Tuesday, June 18, 5pm MDT-7pm
MDT:
- Assessing experiences with trofinetide for Rett syndrome:
interviews with caregivers of patients in LAVENDER™, LILAC-1™, and
LILAC-2™ studies
- Real-world benefits and tolerability of trofinetide for the
treatment of Rett Syndrome: the LOTUS study
- Prevalence of respiratory clinical outcomes among patients with
Rett syndrome: analysis of real-world data in the United
States
- Trofinetide for the treatment of Rett syndrome: long-term
safety and efficacy results from the open-label LILAC-2 study
- Prevalence of epilepsy in individuals with Rett syndrome–a
real-world evidence analysis
- Prevalence of respiratory comorbidities among patients with
Rett syndrome
- Prevalence of nutritional deficiency, underweight status and
gastrostomy among patients with Rett syndrome: an analysis of
electric health record (EHR) data
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.3 Rett syndrome is typically caused by a genetic
mutation on the MECP2 gene.5 In preclinical studies, deficiency in
MeCP2 is thought 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 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. In animal studies, trofinetide has been shown to increase
branching of dendrites and synaptic plasticity signals.10
Important Safety Information for DAYBUE™
(trofinetide)
- 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 (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.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. Forward-looking statements include all statements other than
statements of historical fact and can be identified by terms such
as “intends,” “may,” “will,” “should,” “could,” “would,” “expects,”
“plans,” “anticipates,” “believes,” “estimates,” “projects,”
“predicts,” “potential” and similar expressions (including the
negative thereof) intended to identify forward-looking statements.
Forward-looking statements contained in this press release,
include, but are not limited to, statements about: (i) the clinical
benefits of DAYBUE and continued statistically significant efficacy
observed in the DAYBUE Phase 3 clinical trial program, (ii) the
safety and tolerability profile of DAYBUE and anticipated Rett
syndrome symptom improvements, and (iii) the timing and outcome of
future results from, and continued enrollment and possible
participation extensions in, the real world, observational LOTUS
study. Forward-looking statements are subject to known and unknown
risks, uncertainties, assumptions and other factors that may cause
our actual results, performance or achievements to differ
materially and adversely from those anticipated or implied by our
forward-looking statements. Such risks, uncertainties, assumptions
and other factors include, but are not limited to: our ability to
continue to successfully commercialize DAYBUE, the timing,
enrollment and results of ongoing and future clinical trials and
our ability to continue to stay in compliance with applicable laws
and regulations. Given the risks and uncertainties, you should not
place undue reliance on these forward-looking statements. For a
discussion of these and other risks, uncertainties, assumptions and
other factors that may cause our actual results, performance or
achievements to differ, please refer to our quarterly report on
Form 10-Q for the period ended March 31, 2024 filed with the
Securities and Exchange Commission on May 9, 2024, as well as our
subsequent filings with the Securities and Exchange Commission from
time to time. The forward-looking statements contained herein are
made as of the date hereof, and we undertake no obligation to
update them after this date, 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 Acadia Pharmaceuticals Inc., Data on file.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240618895011/en/
Investor Contact: Acadia Pharmaceuticals Inc. Al Kildani (858)
261-2872 ir@acadia-pharm.com Acadia Pharmaceuticals Inc. Jessica
Tieszen (858) 261-2950 ir@acadia-pharm.com Media Contact: Acadia
Pharmaceuticals Inc. Deb Kazenelson (818) 395-3043
media@acadia-pharm.com
Acadia Pharmaceuticals (NASDAQ:ACAD)
過去 株価チャート
から 11 2024 まで 12 2024
Acadia Pharmaceuticals (NASDAQ:ACAD)
過去 株価チャート
から 12 2023 まで 12 2024
