– New data include 24-month results from the
largest and longest prospective natural history study of children
and adolescents with Dravet syndrome, which showed a lack of
improvement despite treatment with available medicines –
– Additional presentations include data that
support advancement of STK-001 as potentially the first
disease-modifying new medicine for Dravet syndrome, including data
showing substantial reductions in seizures and improvements in
cognition and behavior as well as pharmacokinetic modeling –
Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company
dedicated to addressing the underlying cause of severe diseases by
upregulating protein expression with RNA-based medicines, today
announced that presentations related to the Company’s work in
Dravet syndrome will be presented at the American Epilepsy Society
(AES) 2023 Annual Meeting, taking place December 1 – 5, in Orlando,
Florida. The company is advancing STK-001 as potentially the first
medicine to address the genetic cause of Dravet syndrome.
“People living with Dravet syndrome typically experience high
rates of seizures and debilitating effects on neurodevelopment,
including behavioral and developmental delays, movement and balance
issues, and delayed language and speech, among other life-altering
challenges, which current treatments do not adequately address,”
said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke
Therapeutics. “We look forward to sharing the findings from more
than 3 years of clinical studies, including important new data from
the longest and largest prospective natural history study of
patients with Dravet syndrome, which showed that despite treatment
with multiple anti-seizure medicines, most patients continued to
experience convulsive seizures over 24 months and that gaps in
neurodevelopment persist between children with Dravet syndrome and
their neurotypical peers. These data, together with the initial
clinical findings from our Phase 1/2a and open label extension
studies, as well as pharmacokinetic data to inform dosing, are
giving us a very good understanding of how STK-001 works and
highlight the critical need for a disease-modifying new medicine
for Dravet syndrome.”
Details for the Company’s poster presentations at AES are as
follows:
- Title: 24-Month Analysis of BUTTERFLY: A Prospective,
Observational Study to Investigate Cognition and Other
Comorbidities in Children & Adolescents with Dravet Syndrome
(DS) Session Date & Time: Saturday, December 2 at 12:00
PM EST Oral Presentation Date & Time: Monday, December 4
at 3:15 PM EST Presenter: Joseph Sullivan, M.D., FAES,
Professor of Neurology and Pediatrics and Director of the Pediatric
Epilepsy Center of Excellence at the University of California San
Francisco Poster Number: 1.233
- Title: MONARCH & ADMIRAL: Phase 1/2a Studies in US
& UK Investigating Safety and Drug Exposure of STK-001, an
Antisense Oligonucleotide (ASO), in Children & Adolescents with
Dravet Syndrome (DS) Session Date & Time: Saturday,
December 2 at 12:00 PM EST Presenter: Helen Cross, MB ChB,
Ph.D., Professor, The Prince of Wales’s Chair of Childhood Epilepsy
and Head of the Developmental Neuroscience Programme at University
College London Great Ormond Street Institute of Child Health,
Honorary Consultant in Paediatric Neurology, President of the
International League Against Epilepsy Poster Number:
1.276
- Title: SWALLOWTAIL & LONGWING: Open-Label Extension
(OLE) Studies for Children and Adolescents with Dravet Syndrome
(DS) who Previously Participated in a Study of Antisense
Oligonucleotide (ASO) STK-001 Session Date & Time:
Saturday, December 2 at 12:00 PM EST Presenter: Archana
Desurkar M.D., Consultant Paediatric Neurologist at Sheffield
Children’s Hospital National Health Service Foundation Trust
Poster Number: 1.279
- Title: Utilization of a Pharmacokinetic (PK) Model for
STK-001 in Patients with Dravet Syndrome (DS) To Support Selection
of Dosing Regimens in Clinic Session Date & Time:
Monday, December 4 at 12:00 PM EST Presenter: Meena, Ph.D.,
Senior Vice President of Translational DMPK and Clinical
Pharmacology at Stoke Therapeutics Poster Number: 3.110
About Dravet Syndrome
Dravet syndrome is a severe and progressive genetic epilepsy
characterized by frequent, prolonged and refractory seizures,
beginning within the first year of life. Dravet syndrome is
difficult to treat and has a poor long-term prognosis.
Complications of the disease often contribute to a poor quality of
life for patients and their caregivers. The effects of the disease
go beyond seizures and often include intellectual disability,
developmental delays, movement and balance issues, language and
speech disturbances, growth defects, sleep abnormalities,
disruptions of the autonomic nervous system and mood disorders. The
disease is classified as a developmental and epileptic
encephalopathy due to the developmental delays and cognitive
impairment associated with the disease. Compared with the general
epilepsy population, people living with Dravet syndrome have a
higher risk of sudden unexpected death in epilepsy, or SUDEP. There
are no approved disease-modifying therapies for people living with
Dravet syndrome. One out of 16,000 babies are born with Dravet
syndrome, which is not concentrated in a particular geographic area
or ethnic group.
About STK-001
STK-001 is an investigational new medicine for the treatment of
Dravet syndrome currently being evaluated in ongoing clinical
trials. Stoke believes that STK-001, a proprietary antisense
oligonucleotide (ASO), has the potential to be the first
disease-modifying therapy to address the genetic cause of Dravet
syndrome. STK-001 is designed to upregulate NaV1.1 protein
expression by leveraging the non-mutant (wild-type) copy of the
SCN1A gene to restore physiological NaV1.1 levels, thereby reducing
both occurrence of seizures and significant non-seizure
comorbidities. STK-001 has been granted orphan drug designation by
the FDA and the EMA, and rare pediatric disease designation by the
FDA as a potential new treatment for Dravet syndrome.
About the Phase 1/2a MONARCH Study (United States)
The MONARCH study is a Phase 1/2a open-label study of children
and adolescents ages 2 to 18 who have an established diagnosis of
Dravet syndrome and have evidence of a genetic mutation in the
SCN1A gene. The primary objectives for the study are to assess the
safety and tolerability of STK-001, as well as to determine the
pharmacokinetics in plasma and exposure in cerebrospinal fluid. A
secondary objective is to assess the efficacy as an adjunctive
antiepileptic treatment with respect to the percentage change from
baseline in convulsive seizure frequency. Stoke also intends to
measure non-seizure aspects of the disease, such as quality of
life, as secondary endpoints. Additional information about the
MONARCH study can be found at https://www.monarchstudy.com/.
Patients who participated in the MONARCH study and meet study
entry criteria are eligible to continue treatment in SWALLOWTAIL,
an open-label extension (OLE) study designed to evaluate the
long-term safety and tolerability of repeat doses of STK-001. We
expect that SWALLOWTAIL will also provide valuable information on
the preliminary effects of STK-001 on seizures along with
non-seizure aspects of the disease, such as quality of life and
cognition.
Enrollment and dosing in SWALLOWTAIL are ongoing.
About the Phase 1/2a ADMIRAL Study (United Kingdom)
The ADMIRAL study is a Phase 1/2a open-label study of children
and adolescents ages 2 to <18 who have an established diagnosis
of Dravet syndrome and have evidence of a genetic mutation in the
SCN1A gene. The primary objectives for the study are to assess the
safety and tolerability of multiple doses of STK-001, as well as to
determine the pharmacokinetics in plasma and exposure in
cerebrospinal fluid. A secondary objective is to assess the effect
of multiple doses of STK-001 as an adjunctive antiepileptic
treatment with respect to the percentage change from baseline in
convulsive seizure frequency. Stoke also intends to measure
non-seizure aspects of the disease, such as overall clinical status
and quality of life, as secondary endpoints.
Patients who participated in the ADMIRAL study and meet study
entry criteria are eligible to continue treatment in LONGWING, an
open-label extension (OLE) study designed to evaluate the long-term
safety and tolerability of repeat doses of STK-001. We expect that
LONGWING will also provide valuable information on the preliminary
effects of STK-001 on seizures along with non-seizure aspects of
the disease, such as quality of life and cognition.
Enrollment and dosing in LONGWING are ongoing.
About the BUTTERFLY Observational Study
The BUTTERFLY study is a multicenter, longitudinal, prospective,
observational study of children and adolescents ages 2 to 18 who
have been diagnosed with Dravet syndrome as a result of an SCN1A
gene mutation. This observational study is designed to evaluate
neurodevelopmental status and change from baseline to 24 months.
Secondary and exploratory endpoints in the study will evaluate
changes in other disease measures, including seizures and
additional non-seizure comorbidities. No investigational
medications or other treatments will be provided. Participants
continue to receive their usual care, including anti-seizure
medications, and will be observed by a team of doctors and nurses
over time for up to two years. The study is being conducted at
approximately 20 sites in the United States.
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 the development of STK-002 for the treatment of
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 X @StokeTx.
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, the ability of STK-001 to treat the underlying causes
of Dravet syndrome and reduce seizures or show improvements in
non-seizure comorbidities, the participation of scientists
associated with Stoke making presentations at AES 2023 and the
presentation of data at AES 2023, and the ability of TANGO to
design medicines to increase protein production and the expected
benefit thereof. Statements including words such as “plan,”
“potential,” “will,” “continue,” “expect,” or similar words and
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 advance its product candidates, obtain regulatory
approval of and ultimately commercialize its product candidates,
the timing and results of preclinical and clinical trials, the risk
that positive results in a clinical trial may not be replicated in
subsequent trials or success in early stage clinical trials may not
be predictive of results in later stage trials and preliminary
interim data readouts of ongoing trials may show results that
change when such trials are completed, Stoke’s ability to fund
development activities and achieve development goals, Stoke’s
ability to protect intellectual property, the risks associated with
the direct and indirect impacts of the ongoing COVID-19 pandemic on
our business, and other risks and uncertainties described under the
heading “Risk Factors” in the Company’s Annual Report on Form 10-K
for the year ended December 31, 2022, its Quarterly Reports on Form
10-Q, and the other documents the Company 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 the
Company undertakes no obligation to revise or update any
forward-looking statements to reflect events or circumstances after
the date hereof.
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version on businesswire.com: https://www.businesswire.com/news/home/20231121061395/en/
Stoke Media & Investor Contacts: Dawn Kalmar Chief
Communications Officer dkalmar@stoketherapeutics.com
781-303-8302
Eric Rojas Vice President, Investor Relations
IR@stoketherapeutics.com 617-312-2754
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