ACADIA Pharmaceuticals Inc. (Nasdaq: ACAD), a biopharmaceutical
company focused on the development and commercialization of
innovative medicines to address unmet medical needs in central
nervous system (CNS) disorders, today announced that it has
initiated the Phase 3 LAVENDER placebo-controlled study to evaluate
the efficacy and safety of trofinetide for girls and young women
with Rett syndrome. Rett syndrome is a serious and rare
neurodevelopmental congenital CNS disorder with symptoms that
typically present between six to 18 months of age, and lead to
problems with cognitive, sensory, motor, and autonomic
function.
“There is no approved treatment for Rett syndrome, which is a
rare neurological disease that impacts nearly every aspect of a
child’s life, resulting in loss of speech, difficulty breathing,
lack of motor control, loss of muscle tone and mobility, seizures,
and more,” 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 Kennedy Center, Vanderbilt University Medical
Center and LAVENDER study investigator. “I look forward to the
outcomes of this clinical program evaluating trofinetide as a
potentially new treatment for Rett syndrome.”
The LAVENDER Phase 3 study is a 12-week, double-blind,
randomized, placebo-controlled study evaluating the efficacy and
safety of trofinetide in approximately 180 girls and young women 5
to 20 years of age with Rett syndrome. Half of study participants
will receive trofinetide and half will receive placebo. Co-primary
efficacy endpoints of the study will measure symptom improvement
using the Rett Syndrome Behavior Questionnaire (RSBQ), a caregiver
assessment, and the Clinical Global Impression Scale-Improvement
(CGI-I), a clinician assessment.
“For patients living with this debilitating disease, and the
families whose dedication to their care inspires us, the LAVENDER
study is an important next step in what we hope will result in the
first FDA-approved treatment for Rett syndrome,” said Serge
Stankovic, M.D., M.S.P.H., ACADIA’s President. “We are grateful to
study participants and their families, investigators,
Rettsyndrome.org, and Neuren Pharmaceuticals who have played
instrumental roles in advancing trofinetide to this stage of
clinical development and look forward to building upon this work to
further evaluate trofinetide in the Phase 3 LAVENDER study.”
The LAVENDER study will be followed by LILAC, a nine-month
extension study in which all participants, including those on
placebo in the LAVENDER study, will be eligible to receive
trofinetide. All LILAC participants will be followed to evaluate
long-term tolerability, safety, and effectiveness of trofinetide. A
second extension study, LILAC-2, will follow in which eligible
patients who complete the LILAC trial will continue to receive
trofinetide.
“The start of the trofinetide study has been highly anticipated
by the Rett community, which currently has no approved treatment
for Rett syndrome,” said Melissa Kennedy, Executive Director of
RettSyndrome.org (RSO). “We are hopeful for what this study means
for patients and their families as it potentially brings us closer
to improving the lives of many living with Rett syndrome.”
In 2018, ACADIA entered into an exclusive North American license
agreement with Neuren for the development and commercialization of
trofinetide for Rett syndrome and other indications. In an
end-of-Phase 2 meeting, the FDA confirmed that positive results
from a pivotal Phase 3 study for trofinetide in Rett syndrome and
the extension study could be the basis of a New Drug Application
(NDA) submission.
More information about the LAVENDER study is available at
www.rettsyndromestudies.com.
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. 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 in the U.S. and Orphan Drug Designation in Europe for
both Rett syndrome and Fragile X syndrome.
About Rett Syndrome
Rett syndrome is a debilitating neurological disorder that
occurs primarily in females following apparently normal development
for the first six months of life. Rett syndrome has been most 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 causing problems in brain function that are responsible for
cognitive, sensory, emotional, motor and autonomic function.
Typically, between six to 18 months of age, patients experience a
period of rapid decline with loss of purposeful hand use and spoken
communication 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 ACADIA Pharmaceuticals
ACADIA is a biopharmaceutical company focused on the development
and commercialization of innovative medicines to address unmet
medical needs in central nervous system disorders. ACADIA has
developed and commercialized a treatment for hallucinations and
delusions associated with Parkinson's disease psychosis. In
addition, ACADIA has ongoing clinical development efforts in
additional areas with significant unmet need, including
dementia-related psychosis, schizophrenia-negative symptoms, major
depressive disorder, and Rett syndrome. This press release and
further information about ACADIA can be found at
www.acadia-pharm.com.
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 the commencement of the Phase 3 clinical trial evaluating
trofinetide; the likelihood of success of such clinical trial; the
prospects for FDA approval of trofinetide for Rett syndrome and
other indications; and the success of any efforts to commercialize
trofinetide in North America. 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 discovery, 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, 2018 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20191030005944/en/
Investor Contact: ACADIA Pharmaceuticals Inc. Mark Johnson, CFA
(858) 261-2771 ir@acadia-pharm.com
Media Contact: ACADIA Pharmaceuticals Inc. Maurissa Messier
(858) 768-6068 media@acadia-pharm.com
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