-- Statistically Significant Improvement
Indicates Trofinetide’s Potential for Treating Rett Syndrome--
Trofinetide for Rett Syndrome has Fast Track Status and Orphan Drug
Designation in the U.S. and Orphan Drug Designation in Europe
ACADIA Pharmaceuticals Inc. (NASDAQ: ACAD), Neuren
Pharmaceuticals Limited (ASX: NEU), and Rettsyndrome.org (RSO)
announced today that the positive results from a Phase 2 study
conducted by Neuren, which evaluated the safety and efficacy of
trofinetide in females with Rett syndrome (RTT), have been
published in Neurology®, the medical journal of the American
Academy of Neurology.
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The study, “Double-Blind, Randomized, Placebo-Controlled Study
of Trofinetide in Pediatric Rett Syndrome” has been published
online and will appear in the April 16, 2019 issue of Neurology.
The study evaluated 82 females with Rett syndrome aged 5 to 15
years and found at the highest dose (200 mg/kg twice daily or BID)
trofinetide achieved statistically significant improvement compared
with placebo on three of five syndrome-specific efficacy measures:
the Rett Syndrome Behaviour Questionnaire (RSBQ), a caregiver
assessment (p=0.042), the Clinical Global Impression
Scale-Improvement (CGI-I), a clinician assessment of the
improvement of Rett syndrome (p=0.029), and the RTT-Clinician
Domain Specific Concerns-Visual Analog Scale (RTT-DSC), a clinician
assessment (p=0.025). Results of the study also showed trofinetide
was well-tolerated at all dose levels (50 mg/kg BID, 100 mg/kg BID,
and 200 mg/kg BID).
“Disease burden is severe for Rett patients and their families,
and the impact of the disorder is life-long,” said Daniel Glaze,
M.D., study author, Baylor College of Medicine, Department of
Pediatrics and Neurology and Director at the Blue Bird Circle Rett
Center, Texas Children’s Hospital. “The data reported in this study
show that females treated with trofinetide experienced lessened
neurobehavioral impairments including social communication
deficits, anxiety-like behavior, and mood dysregulation. These are
very promising data for the Rett community that is currently
without any U.S. FDA-approved treatment option.”
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.
“Rett syndrome is a condition that leads to severe neurological
impairments and is not only debilitating for the person with the
disease, but also very hard on the families and caregivers of the
children, mostly females, who are often unable to speak, walk, eat,
and even breathe normally,” said Steve Kaminsky, Ph.D., Chief
Science Officer of RSO. “These results are very encouraging because
they provide strong evidence that trofinetide may be a potential
treatment for Rett syndrome.”
ACADIA plans to initiate a 12-week Phase 3 double-blind,
randomized, placebo-controlled study evaluating trofinetide in the
second half of 2019 following completion of additional
manufacturing scale-up activities. This study will evaluate
efficacy and safety of trofinetide and placebo in approximately 180
females ages 5 to 20 years with Rett syndrome. Half of the study
participants will receive trofinetide and half will receive
placebo. The study will use the RSBQ and the CGI-I syndrome
specific efficacy measures as co-primary efficacy endpoints. The
Phase 3 study will be followed by a nine month open label extension
study in which all participants, including those on placebo in the
Phase 3 study, will be eligible to receive trofinetide. In the open
label extension study, all participants will be followed to
evaluate long term tolerability and safety of trofinetide.
“Neuren has successfully led the clinical development of
trofinetide to date, which allows us to further evaluate
trofinetide as a potentially important treatment option for Rett
syndrome,” said Serge Stankovic, M.D., M.S.P.H., ACADIA’s
President. “In addition to achieving successful outcomes from
early-stage clinical research with trofinetide for Rett syndrome,
Neuren has fostered strong and enduring relationships with the Rett
community. We will build on these efforts as we continue to study
trofinetide for this unmet medical need.”
Phase 2 Study Design
In the Phase 2 study, 82 females were enrolled and randomized to
receive liquid trofinetide or placebo orally or via gastrostomy
tube for six weeks: 24 subjects to placebo BID, 15 subjects to 50
mg/kg of trofinetide BID, 16 subjects to 100 mg/kg of trofinetide
BID, and 27 subjects to 200 mg/kg of trofinetide BID. Safety and
tolerability assessments included adverse events (AEs), clinical
laboratory tests, physical examinations, and concomitant
medications.
The five core efficacy endpoints included three
clinician-completed measures and two caregiver-completed
measures.
- The RSBQ, a rating scale in which the
subject’s caregiver rates the frequency of symptoms.
- The CGI-I, in which the clinician rates
how much the subject’s overall illness has improved or worsened,
relative to baseline.
- The RTT-DSC, in which the clinician
assesses - on a visual analog scale - the severity of concerns
identified for each subject on an individual basis.
- The Motor Behavioral Assessment, a
rating scale in which the clinician rates the subject’s current
level of function.
- The Caregiver Top 3 Concerns, in which
the subject’s caregiver assesses - on a visual analog scale - the
severity of concerns identified for each subject on an individual
basis.
About Trofinetide
Trofinetide is a novel synthetic analog of the amino‐terminal
tripeptide of IGF-1 designed to treat the core symptoms of Rett
syndrome by 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
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 is commercializing the first and only medicine
approved for the treatment of 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 inadequate response, 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.
About Neuren Pharmaceuticals
Neuren Pharmaceuticals Limited (Neuren) is a biopharmaceutical
company developing new therapies for brain injury,
neurodevelopmental and neurodegenerative disorders. Neuren has
completed Phase 2 development of trofinetide for Rett syndrome and
has completed a Phase 2 clinical trial of trofinetide in Fragile X
syndrome. In addition, Neuren is advancing the pre-clinical
development of its second drug candidate NNZ-2591. Further
information about Neuren can be found at: www.neurenpharma.com.
About Rettsyndrome.org
Rettsyndrome.org (RSO) is one of the leading private funders of
Rett syndrome research, investing over $46 million to date. The
mission of the organization is to accelerate full spectrum research
to cure Rett syndrome and empower families with information,
knowledge and connectivity. Rettsyndrome.org recently earned
Charity Navigator’s prestigious 4-star rating for its strong
financial health and commitment to accountability and transparency.
Further information about Rettsyndrome.org can be found at:
www.rettsyndrome.org.
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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ACADIA Pharmaceuticals Inc.Investor Contact:Mark
Johnson, CFA(858) 261-2771ir@acadia-pharm.com
Media Contact:Maurissa Messier(858)
768-6068media@acadia-pharm.com
Neuren PharmaceuticalsContact:Jon Pilcher, Chief
Financial Officer+61 438 422 271jpilcher@neurenpharma.com
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