Acadia Pharmaceuticals Submits New Drug Application to the U.S. FDA for Trofinetide for the Treatment of Rett Syndrome
July 18 2022 - 3:07PM
Business Wire
Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that
the company has submitted a New Drug Application (NDA) to the U.S.
Food and Drug Administration (FDA) for trofinetide for the
treatment of Rett syndrome in adults and pediatric patients two
years of age and older.
“This is an important step forward for members of the Rett
community who face a devastating disease with no approved
therapies,” said Steve Davis, Chief Executive Officer. “We are
grateful to the patients, their families and the physicians who
have participated in the trofinetide clinical studies, including
our pivotal Phase 3 Lavender study. We look forward to working with
the FDA as it evaluates the NDA.”
The NDA submission is supported by results from the pivotal
Phase 3 Lavender™ study evaluating the efficacy and safety of
trofinetide versus placebo in 187 girls and young women aged 5-20
years with Rett syndrome. The study demonstrated a statistically
significant improvement over placebo on the co-primary endpoints,
the Rett Syndrome Behaviour Questionnaire (RSBQ) change from
baseline to 12 weeks (p=0.0175; effect size=0.37) and the Clinical
Global Impression-Improvement (CGI-I) scale score (p=0.0030; effect
size=0.47) at 12 weeks. The RSBQ is a caregiver assessment of the
core symptoms of Rett syndrome and the CGI-I is a global physician
assessment of worsening or improving of Rett syndrome. In addition,
the study also met its key secondary endpoint, the Communication
and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler
Checklist–Social composite score (CSBS-DP-IT–Social) change from
baseline to week 12 (p=0.0064; effect size=0.43), a caregiver scale
of the ability to communicate.
In 2018, Acadia entered into an exclusive license agreement with
Neuren Pharmaceuticals Limited (ASX: NEU) for the development and
commercialization of trofinetide for the treatment of Rett syndrome
and other indications in North America. Trofinetide has been
granted Fast Track Status and Orphan Drug Designation for the
treatment of Rett syndrome in the United States. An NDA with Orphan
Drug Designation is eligible for priority review. Trofinetide has
also been granted Rare Pediatric Disease (RPD) designation by the
FDA. With such designation, Acadia expects to be awarded a Priority
Review Voucher if the NDA is approved.
About Lavender™ The Lavender study was a Phase 3, 12-week,
double-blind, randomized, placebo-controlled study of trofinetide
in 187 girls and young women aged 5-20 years with Rett syndrome,
designed to evaluate its efficacy and safety. The co-primary
endpoints of Lavender included both a caregiver (Rett Syndrome
Behaviour Questionnaire [RSBQ]) and physician (Clinical Global
Impression–Improvement [CGI-I]) assessment. The key secondary
endpoint was also a caregiver assessment designed to evaluate
communication skills, the Communication and Symbolic Behavior
Scales Developmental Profile™ Infant-Toddler Checklist – Social
Composite Score (CSBS-DP-IT–Social).
About Rett Syndrome Rett syndrome is a rare genetic
neurodevelopmental disorder that occurs primarily in females
following a near normal development in the first two years of
life.1,2 It is caused by mutations on the X chromosome on a gene
called MECP2.3 Occurring worldwide in approximately one of every
10,000 to 15,000 female births and in the United States impacts
6,000 to 9,000 patients.4 Children with Rett syndrome experience a
period of developmental regression between 18-30 months of age,
which is typically followed by a plateau period lasting years to
decades.1,2,5 Rett syndrome is diagnosed based on clinical
evaluation, typically by about three years of age.2,6
A complex and multisystem disorder, Rett syndrome causes
profound impairment to central nervous system (CNS) function,
including loss of communication skills, purposeful hand use, gait
abnormalities, and stereotypic hand movements such as hand
wringing/squeezing, clapping/tapping, mouthing and washing/rubbing
automatisms.2 People living with Rett syndrome may also experience
a range of additional symptoms, such as gastrointestinal
complications, skeletal abnormalities, neuroendocrine
abnormalities, disruptive and anxiety-like behaviors, as well as
mood dysregulation and sleep disturbances.1 Currently, there are no
FDA-approved medicines for the treatment of Rett syndrome.
About Trofinetide Trofinetide is an investigational drug. It is
a novel synthetic analog of the amino‐terminal tripeptide of IGF-1
designed to treat the core symptoms of Rett syndrome by potentially
reducing neuroinflammation and supporting synaptic function.
Trofinetide is thought to stimulate synaptic maturation and
overcome the synaptic and neuronal immaturities that are
characteristic of Rett syndrome pathophysiology. In the central
nervous system, IGF-1 is produced by both of the major types of
brain cells – neurons and glia. IGF-1 in the brain is critical for
both normal development and for response to injury and disease.
Trofinetide has been granted Fast Track Status and Orphan Drug
Designation for Rett syndrome and has also been granted Rare
Pediatric Disease (RPD) designation by the FDA.
About Acadia Pharmaceuticals Acadia is advancing breakthroughs
in neuroscience to elevate life. For more than 25 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 therapy for
hallucinations and delusions associated with Parkinson’s disease
psychosis. Our late-stage development efforts are focused on
treating psychosis in patients with dementia, the negative symptoms
of schizophrenia and Rett syndrome. Our early-stage development
efforts are focused on novel approaches to pain management,
cognition 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, 2021 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 Fu et al. Consensus guidelines on managing Rett
syndrome across the lifespan. BMJ Paediatrics Open. 2020;4:1-14. 2
Neul JL, Kaufmann WE, Glaze DG, et al. Rett syndrome: revised
diagnostic criteria and nomenclature. Ann Neurol.
2010;68(6):944-950. 3 Amir RE, et al. Rett syndrome is caused by
mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2.
Nat Genet. 1999;23:185-188. 4 Acadia Pharmaceuticals Inc. Data on
file. RTT US Prevalence. March 2022. 5 NINDS. Rett Syndrome Fact
Sheet. Retrieved from
https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Rett-Syndrome-Fact-Sheet.
Accessed February 2022. 6 Tarquinio. Age of Diagnosis in Rett
Syndrome: Patterns of Recognition Among Diagnosticians and Risk
Factors for Late Diagnosis. Pediatric Neurology.
2015;52:585-591.
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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
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