- Trofinetide met co-primary efficacy endpoints
demonstrating statistically significant improvement over placebo in
the Rett Syndrome Behaviour Questionnaire (RSBQ) (p=0.0175) and the
Clinical Global Impression of Improvement (CGI-I) (p=0.0030)
- Trofinetide met key secondary endpoint
demonstrating statistically significant improvement over placebo in
CSBS-DP-IT–Social (p=0.0064), caregiver scale of ability to
communicate
- Pre-New Drug Application meeting with the
U.S. FDA planned for the first quarter 2022
- Conference call and webcast to be held today
at 4:30 p.m. Eastern Time
Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced
positive top-line results from the pivotal, Phase 3 Lavender™ study
evaluating the efficacy and safety of trofinetide in 187 girls and
young women aged 5-20 years with Rett syndrome. The 12-week
placebo-controlled study demonstrated a statistically significant
improvement over placebo for both co-primary endpoints. On the Rett
Syndrome Behaviour Questionnaire (RSBQ), change from baseline to
week 12 was -5.1 vs. -1.7 (p=0.0175; effect size=0.37). The
Clinical Global Impression–Improvement (CGI-I) score at week 12 was
3.5 vs. 3.8 (p=0.0030; effect size=0.47). 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.
Additionally, trofinetide demonstrated a statistically
significant separation over placebo on the 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 was -0.1 vs.
-1.1 (p=0.0064; effect size=0.43).
“These are encouraging results for patients and families
affected by Rett syndrome. Patients reported improvements in core
symptoms, like being able to respond to a choice when asked by
their parents, or experiencing more freedom from the repetitive
hand movements that create obstacles in other areas of their
lives,” said Jeffrey L. Neul, M.D., Ph.D., Annette Schaffer Eskind
Chair and Director, Vanderbilt Kennedy Center; Professor of
Pediatrics, Division of Neurology, Pharmacology, and Special
Education, Vanderbilt University Medical Center and Lavender study
investigator. “The positive Lavender study results support a
potential treatment for Rett syndrome and represent an important
step forward in addressing this rare and serious neurological
disease.”
Study treatment discontinuation rates related to treatment
emergent adverse events (TEAEs) were 17.2% in the trofinetide group
as compared to 2.1% in the placebo group. The most common adverse
events were diarrhea (80.6% with trofinetide vs. 19.1% with
placebo), of which 97.3% in the trofinetide arm were characterized
as mild-to-moderate, and vomiting (26.9% with trofinetide vs. 9.6%
with placebo), of which 96% in the trofinetide arm were
characterized as mild-to-moderate. Serious adverse events were
observed in 3.2% of study participants in both the trofinetide and
placebo groups. Patients completing the Lavender study had the
opportunity to continue to receive trofinetide in the open-label
Lilac and Lilac-2 extension studies. More than 95% of participants
who completed the Lavender study elected to roll-over to the Lilac
open-label extension study. The results from this study will be
submitted for presentation at upcoming medical meetings.
“The consistent efficacy across primary and key secondary
endpoints in the Lavender study demonstrates the potential of
trofinetide to treat Rett syndrome,” said Kathie Bishop, Ph.D.,
Acadia’s Senior Vice President, Chief Scientific Officer and Head
of Rare Disease. “We want to thank the patients, their caregivers,
study site personnel, physicians and everyone who participated in
the Lavender study for their contribution to making this milestone
a reality. We look forward to continuing this important work and
potentially delivering an FDA-approved treatment for this rare and
devastating disease.”
Acadia is preparing for a pre-NDA meeting with the U.S. Food and
Drug Administration (FDA) in the first quarter of 2022 and plans to
submit a New Drug Application (NDA) around mid-year 2022.
Trofinetide has been granted Fast Track Status and Orphan Drug
Designation for Rett syndrome. Trofinetide has also been granted
Rare Pediatric Disease (RPD) designation by the FDA. An NDA with
Orphan Drug Designation is eligible for priority review. With an
RPD NDA we would expect to be awarded a Priority Review Voucher if
approved, subject to final determination by the FDA.
In 2018, Acadia entered into an exclusive license agreement with
Neuren Pharmaceuticals Limited (ASX: NEU) for the development and
commercialization of trofinetide for Rett syndrome and other
indications in North America.
Conference Call and Webcast Information
Acadia will discuss top-line results from its Lavender study of
trofinetide for the treatment of Rett syndrome via conference call
and webcast today at 4:30 p.m. Eastern Time. The conference call
can be accessed by dialing 855-638-4820 for participants in the
U.S. or Canada and 443-877-4067 for international callers
(reference passcode 7989366). A telephone replay of the conference
call may be accessed through December 20, 2021 by dialing
855-859-2056 for callers in the U.S. or Canada and 404-537-3406 for
international callers (reference passcode 7989366). The conference
call will also be webcast live on Acadia’s website,
www.acadia-pharm.com, in the investors section and archived until
January 3, 2022.
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, debilitating neurological disorder that
occurs primarily in females following apparently normal development
for the first six months of life. Rett syndrome is often
misdiagnosed as autism, cerebral palsy, or non-specific
developmental delay. Rett syndrome is caused by mutations on the X
chromosome on a gene called MECP2. There are more than 200
different mutations found on the MECP2 gene that interfere with its
ability to generate a normal gene product.
Rett syndrome occurs 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. Rett syndrome causes problems in brain
function that are responsible for cognitive, sensory, emotional,
motor and autonomic function. Typically, with symptoms presenting
between six to 18 months of age, patients experience a period of
rapid decline with loss of purposeful hand use (fine motor skills),
development of hand stereotypies, absent or impaired mobility
(gross motor skills), loss of communication skills (including eye
contact) and inability to independently conduct activities of daily
living. Symptoms also include seizures, disorganized breathing
patterns, an abnormal side-to-side curvature of the spine
(scoliosis), and sleep disturbances. 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 shown to
inhibit the production of inflammatory cytokines, inhibit the
overactivation of microglia and astrocytes, and increase the amount
of available IGF-1 that can bind to IGF-1 receptors.
Trofinetide has been granted Fast Track Status and Orphan Drug
Designation for Rett syndrome. Trofinetide has also been granted
Rare Pediatric Disease (RPD) designation by the FDA. Upon FDA
approval of a product with RPD designation, the sponsor can receive
a Priority Review Voucher, which can be used to obtain FDA review
of a New Drug Application for another product in an expedited
period of six months.
About Acadia Pharmaceuticals
Acadia is trailblazing 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 dementia-related psychosis, negative symptoms of
schizophrenia and Rett syndrome, and in early-stage clinical
research we are exploring novel approaches to pain management, and
cognition and neuropsychiatric symptoms in central nervous system
disorders. For more information, visit us at www.acadia-pharm.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, 2020 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20211206005778/en/
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
Acadia Pharmaceuticals (NASDAQ:ACAD)
Historical Stock Chart
From Apr 2024 to May 2024
Acadia Pharmaceuticals (NASDAQ:ACAD)
Historical Stock Chart
From May 2023 to May 2024