-ACADIA plans to initiate a Phase 3 study of trofinetide for the
treatment of Rett syndrome, a rare neurodevelopmental congenital
CNS disorder, in the second half of 2019
-Neuren retains all rights to trofinetide outside of North
America
-Neuren to receive US $10 million upfront plus potential
milestones of up to US $455 million and royalties
ACADIA Pharmaceuticals Inc. (NASDAQ: ACAD) and Neuren
Pharmaceuticals Limited (ASX: NEU) announced today that they have
entered into an exclusive North American License Agreement for the
development and commercialization of trofinetide for Rett syndrome
and other indications. Neuren retains rights to develop and
commercialize trofinetide for all indications outside of North
America.
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ACADIA plans to initiate a Phase 3 randomized, double-blind
placebo-controlled study evaluating trofinetide in the second half
of 2019 following completion of additional manufacturing
activities. This study will evaluate trofinetide and placebo in
approximately 180 girls with Rett syndrome and will measure the
Rett Syndrome Behavior Questionnaire (RSBQ), a caregiver
assessment, and the Clinical Global Impression of Improvement
(CGI-I), a physician assessment, as co-primary efficacy
endpoints.
“A potential treatment for Rett syndrome is a perfect fit with
ACADIA’s mission to develop novel therapies to improve the lives of
patients with central nervous system disorders,” said Serge
Stankovic, M.D., M.S.P.H, Executive Vice President, Head of
Research and Development at ACADIA. “Today there are no approved
treatments for the girls and women suffering from Rett syndrome. We
look forward to initiating a Phase 3 study in the second half of
2019 to further explore the potential benefits of trofinetide for
patients and their caregivers.”
Neuren Executive Chairman Dr. Richard Treagus commented, “We are
delighted to be partnering with ACADIA in North America. ACADIA’s
team has a proven record in developing and commercializing
medicines in central nervous system disorders with no approved
therapies and high unmet needs. ACADIA’s additional capabilities
and resources will immediately make a very significant difference,
enabling us to advance our shared goal of developing this novel
treatment option for Rett syndrome patients.”
Steve Kaminsky, Ph.D., Chief Science Officer of
Rettsyndrome.org, commented, “Rettsyndrome.org is grateful to
Neuren for their dedication to the development of trofinetide.
ACADIA’s commitment to advance trofinetide to Phase 3 brings us
closer to the first potential treatment for Rett syndrome with a
drug designed to address the underlying biology and improve the
lives of those suffering from the condition.”
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 disease1,2. Trofinetide has been granted U.S. Food and
Drug Administration (FDA) Fast Track Status and Orphan Drug
Designation in the U.S. and Europe.
Neuren conducted a Phase 2 double-blind placebo-controlled dose
ranging study in girls aged 5 to 15 years with Rett syndrome, in
which statistically significant and clinically meaningful
improvement was demonstrated on the RSBQ and the CGI-I. This
followed positive trends observed in an earlier Phase 2 trial in
adolescents and adults aged 16 to 45 years with Rett syndrome3. In
addition, Neuren has completed an exploratory study in Fragile X
syndrome.
Dr. Daniel Glaze, Medical Director at the Blue Bird Circle Rett
Center, Texas Children’s Hospital commented, “The trofinetide Phase
2 results in Rett syndrome are very promising in terms of both
safety and clinical benefit. For many families, these improvements
would provide meaningful improvement in their child’s quality of
life.”
Under the terms of the License Agreement, Neuren is eligible to
receive US $10 million upfront plus potential milestone payments of
up to US $455 million. In addition, Neuren is eligible to receive
tiered, escalating, double-digit percentage royalties on net sales
of trofinetide in North America and one third of the market value
of any Rare Pediatric Disease Priority Review Voucher, if awarded
by the U.S. FDA upon approval of a New Drug Application for
trofinetide. The potential milestone payments to Neuren consist of
US $105 million subject to achievement of development milestones in
Rett syndrome and Fragile X syndrome and up to US $350 million
subject to achievement of thresholds of annual net sales of
trofinetide in North America. ACADIA will fund and execute the
remaining development for trofinetide in Rett syndrome in North
America, except for the completion by Neuren of certain in-progress
preparatory activities. ACADIA and Neuren will form a Joint
Steering Committee to direct the development of trofinetide in all
indications, including the next clinical trial for Fragile X
syndrome. Any data and regulatory filings generated by ACADIA or
Neuren may be used by either party for the development and
commercialization of trofinetide in their respective territories.
ACADIA has a right of first negotiation to acquire a license to
develop and commercialize trofinetide outside North America. Neuren
has an obligation not to develop a competing product in indications
for which ACADIA develops and commercializes trofinetide.
About TrofinetideTrofinetide 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 disease1,2. Trofinetide has been
granted U.S. FDA Fast Track Status and Orphan Drug Designation in
the U.S. and Europe for both Rett syndrome and Fragile X
syndrome.
About Rett SyndromeRett 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 MeCP24. There are
more than 200 different mutations found on the MeCP2 gene that
interfere with its ability to generate a normal gene product4. Rett
syndrome occurs worldwide in approximately one of every 10,000 to
15,000 female births5 causing problems in brain function that are
responsible for cognitive, sensory, emotional, motor and autonomic
function. Typically, between six to eighteen 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 living5. 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 approved
for the treatment of Rett syndrome1.
About Fragile X syndromeFragile X syndrome is the most common
inherited cause of intellectual disability and the most common
known cause of autism. Fragile X syndrome is caused by a single
gene defect on the X chromosome that impacts the FMRP protein,
which is responsible for regulating the synapses of nerve cells.
One of every 5,000 males and one of every 4,000 to 8,000 females
are estimated to have the full gene mutation6. Generally males are
more severely affected than females. Clinically, Fragile X syndrome
is characterized by intellectual disability, hyperactivity and
attentional problems, autistic symptoms, anxiety, emotional
lability and epilepsy. Currently, there are no medicines approved
for the treatment of Fragile X syndrome.
About ACADIA PharmaceuticalsACADIA 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 and major
depressive disorder. This press release and further information
about ACADIA can be found at: www.acadia-pharm.com.
About Neuren PharmaceuticalsNeuren 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 in
Fragile X syndrome.
About Rettsyndrome.orgRettsyndrome.org is one of the leading
private funders of Rett syndrome research. The organization hosts
the largest global gathering of Rett researchers and clinicians to
establish research direction for the future. The mission of the
organization is to accelerate the full spectrum research to cure
Rett syndrome and empower families with information, knowledge and
connectivity. Further information about Rettsyndrome.org can
be found at: rettsyndrome.org.
Forward-Looking StatementsStatements 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, 2017 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.
This ASX-announcement contains forward-looking statements that
are subject to risks and uncertainties. Such statements involve
known and unknown risks and important factors that may cause the
actual results, performance or achievements of Neuren to be
materially different from the statements in this announcement.
References
1Tropea, D., et al. (2009). "Partial reversal of Rett
Syndrome-like symptoms in MeCP2 mutant mice." Proc Natl Acad Sci U
S A 106(6): 2029-2034.
2Vahdatpour, C., et al. (2016). "Insulin-Like Growth Factor 1
and Related Compounds in the Treatment of Childhood-Onset
Neurodevelopmental Disorders." Front Neurosci 10: 450.
3Glaze, D. G., et al. (2017). "A Double-Blind, Randomized,
Placebo-Controlled Clinical Study of Trofinetide in the Treatment
of Rett Syndrome." Pediatr Neurol 76: 37-46.
4Chahrour, M. and H. Y. Zoghbi (2007). "The story of Rett
syndrome: from clinic to neurobiology." Neuron 56(3): 422-437.
5Ip, J. P. K., et al. (2018). "Rett syndrome: insights into
genetic, molecular and circuit mechanisms." Nat Rev Neurosci 19(6):
368-382.
6Hagerman, R.J., et al. (2017). “Fragile X syndrome.” Nat Rev
Disease Primers 3: 1-19.
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version on businesswire.com: https://www.businesswire.com/news/home/20180806005653/en/
ACADIA Pharmaceuticals Inc.Investor Contact:Elena Ridloff,
CFA(858) 558-2871ir@acadia-pharm.comorMedia Contact:Bob
Laverty(609) 558-5570bob@taftcommunications.comorNeuren
Pharmaceuticals LimitedContact:Dr. Richard TreagusExecutive
Chairman+61 417 520 509rtreagus@neurenpharma.com
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