– Establishes co-development and
co-commercialization agreement for Stoke’s SYNGAP1 preclinical
program
– Acadia receives exclusive worldwide licenses
for two additional preclinical programs: Rett syndrome (MECP2) and
undisclosed neurodevelopmental target
– Combines Stoke’s TANGO research platform with
Acadia’s expertise in neurology drug development and
commercialization
– Stoke receives a $60 million upfront payment
and potential milestone payments of up to $907 million and
royalties on future sales
Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) and Stoke
Therapeutics, Inc. (Nasdaq: STOK) announced today that the
companies have entered a collaboration to discover, develop and
commercialize novel RNA-based medicines for the potential treatment
of severe and rare genetic neurodevelopmental diseases of the
central nervous system (CNS). The collaboration includes SYNGAP1
syndrome, Rett syndrome (MECP2), and an undisclosed
neurodevelopmental target of mutual interest.
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the full release here:
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“Stoke’s RNA-based approach to upregulating healthy proteins
offers very exciting new possibilities for the treatment of rare,
neurodevelopmental diseases like Rett syndrome,” said Steve Davis,
Chief Executive Officer of Acadia Pharmaceuticals. “Combining
Stoke’s capabilities with Acadia’s extensive expertise in
neuroscience drug development and commercialization enables us to
push harder and faster in exploring some of the new frontiers in
rare central nervous system disorders. We are excited to have the
opportunity to further build our Rett syndrome franchise and pursue
treatments in SYNGAP1 syndrome and other neurodevelopmental
disorders.”
“Rett syndrome and SYNGAP1 syndrome are two severe, intractable
diseases of the central nervous system and both are associated with
developmental delays, motor function loss, autism, seizures and
other comorbidities that impact quality of life for patients and
their families,” said Edward M. Kaye, M.D., Chief Executive Officer
of Stoke Therapeutics. “Acadia shares our deep commitment to
improving outcomes for people living with neurodevelopmental
disorders. We believe their late-stage development and
commercialization capabilities, in addition to their neuroscience
expertise, complement our discovery research efforts and clinical
learnings from our work in Dravet syndrome. Together, we believe we
have a significant opportunity to improve treatment options by
delivering new disease-modifying medicines to people who need
them.”
Terms of Collaboration Under the terms of the agreement,
Stoke will receive an upfront payment of $60 million from Acadia
and is eligible to receive up to $907 million in milestones as well
as royalties on future sales.
For the SYNGAP1 program, the two companies will jointly share
global research, development and commercialization responsibilities
and share 50/50 in all worldwide costs and future profits. In
addition, Stoke is eligible to receive potential development,
regulatory, first commercial sales and sales milestones.
For the Rett syndrome (MECP2) and the undisclosed
neurodevelopmental program, Stoke will lead research and
pre-clinical development activities, while Acadia will lead
clinical development and commercialization activities. Acadia will
fully fund the research and pre-clinical development activities
related to these two targets and Stoke is eligible to receive
potential development, regulatory, first commercial sales and sales
milestones as well as tiered royalty payments on worldwide sales
starting in the mid-single digit range and escalating to the
mid-teens based on revenue levels.
About SYNGAP1 Syndrome SYNGAP1 syndrome is a rare
neurological disorder characterized by moderate to severe
intellectual disability that is evident in early childhood.
Mutations in the SYNGAP1 gene (which produces the SynGAP protein)
were first identified in 2009 and since then, an increasing number
of children with SYNGAP1 syndrome have been identified. Normal
levels of SynGAP protein are essential for proper brain function
and development. Mutations in the SYNGAP1 gene also play an
important role in the development of epileptic encephalopathies
(DEEs). The severity and onset of symptoms can vary from patient to
patient. SYNGAP1 syndrome is characterized by developmental delay
or intellectual disability, generalized epilepsy, and autism
spectrum disorder (ASD) and other behavioral abnormalities. More
than 80% of cases of SYNGAP1 syndrome are caused by a
haploinsufficiency of the SYNGAP1 gene. SYNGAP1 syndrome is
estimated to account for 1% to 2% of all intellectual disability
cases. There are currently no approved treatments for SYNGAP1
syndrome.
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 6
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 TANGO TANGO (Targeted Augmentation of Nuclear Gene
Output) is Stoke’s proprietary research platform. Stoke’s initial
application for this technology are diseases in which one copy of a
gene functions normally and the other is mutated, also called
haploinsufficiencies. In these cases, the mutated gene does not
produce its share of protein, resulting in disease. Using the TANGO
approach and a deep understanding of RNA science, Stoke researchers
design antisense oligonucleotides (ASOs) that bind to pre-mRNA and
help the functional (or wild-type) genes produce more protein.
TANGO aims to restore missing proteins by increasing – or stoking –
protein output from healthy genes, thus compensating for the mutant
copy of the gene.
About Stoke Therapeutics Stoke Therapeutics (Nasdaq:
STOK), is a biotechnology company dedicated to addressing the
underlying cause of severe diseases by upregulating protein
expression with RNA-based medicines. Using Stoke’s proprietary
TANGO (Targeted Augmentation of Nuclear Gene Output) approach,
Stoke is developing antisense oligonucleotides (ASOs) to
selectively restore protein levels. Stoke’s first compound,
STK-001, is in clinical testing for the treatment of Dravet
syndrome, a severe and progressive genetic epilepsy. Dravet
syndrome is one of many diseases caused by a haploinsufficiency, in
which a loss of ~50% of normal protein levels leads to disease.
Stoke is pursuing treatment for a second haploinsufficient disease,
autosomal dominant optic atrophy (ADOA), the most common inherited
optic nerve disorder. Stoke’s initial focus is haploinsufficiencies
and diseases of the central nervous system and the eye, although
proof of concept has been demonstrated in other organs, tissues,
and systems, supporting its belief in the broad potential for its
proprietary approach. Stoke is headquartered in Bedford,
Massachusetts with offices in Cambridge, Massachusetts. For more
information, visit https://www.stoketherapeutics.com/ or follow
Stoke on Twitter at @StokeTx.
About Acadia Pharmaceuticals Acadia is advancing
breakthroughs in neuroscience to elevate life. For more than 25
years Acadia has been working at the forefront of healthcare to
bring vital solutions to people who need them most. Acadia
developed and commercialized the first and only approved therapy
for hallucinations and delusions associated with Parkinson’s
disease psychosis. Acadia’s late-stage development efforts are
focused on treating psychosis in patients with dementia, the
negative symptoms of schizophrenia and Rett syndrome. Acadia’s
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-pharm.com and follow us on LinkedIn and Twitter.
Stoke’s Cautionary Note Regarding Forward-Looking
Statements This press release contains forward-looking
statements within the meaning of the "safe harbor" provisions of
the Private Securities Litigation Reform Act of 1995, including,
but not limited to, statements regarding: receipt of upfront
payments; receipt of potential milestone payments under the SYNGAP1
collaboration; receipt of potential milestones and royalty payments
under the MECP2 program and the third program; the ability to
develop new treatments for neurodevelopmental disorders; and
expectations regarding the proposed transaction with Acadia.
Statements including words such as “believe,” “will,” “eligible,”
or “potential,” and any other statements in the future tense are
forward-looking statements. These forward-looking statements
involve risks and uncertainties, as well as assumptions, which, if
they do not fully materialize or prove incorrect, could cause our
results to differ materially from those expressed or implied by
such forward-looking statements. Forward-looking statements are
subject to risks and uncertainties that may cause the company’s
actual activities or results to differ significantly from those
expressed in any forward-looking statement, including risks and
uncertainties related to: Stoke’s ability to develop and identify
potential product candidates; that if Acadia were to breach or
terminate the collaboration, Stoke would not obtain the anticipated
financial or other benefits; the possibility that Stoke and Acadia
may not be successful in their development efforts under any of the
collaborations and that, even if successful, Stoke and Acadia may
be unable to successfully commercialize any resulting product
candidates; and other risks and uncertainties described under the
heading “Risk Factors” in documents Stoke files from time to time
with the Securities and Exchange Commission. These forward-looking
statements speak only as of the date of this press release and
Stoke undertakes no obligation to revise or update any
forward-looking statements to reflect events or circumstances after
the date hereof.
Acadia’s Forward-Looking Statement 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.
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Acadia Media & Investor Contacts 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
Stoke Media & Investor Contacts Media Contact: Stoke
Therapeutics, Inc. Dawn Kalmar Chief Communications Officer
781-303-8302 dkalmar@stoketherapeutics.com
Investor Contact: Stoke Therapeutics, Inc. Eric Rojas Vice
President, Investor Relations 617-312-2754
IR@stoketherapeutics.com
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