– First presentation of landmark data from
Phase 1/2a and OLE studies of zorevunersen that showed substantial
reductions in seizures and continued improvements in multiple
measures of cognition and behavior –
Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company
dedicated to restoring protein expression by harnessing the body’s
potential with RNA medicine, today announced it will present data
from its Dravet syndrome clinical program at the 15th European
Epilepsy Congress (EEC) taking place September 7 – 11, in Rome,
Italy.
The Company is advancing zorevunersen (STK-001), a proprietary
antisense oligonucleotide (ASO), as the first potential medicine to
address the genetic cause of Dravet syndrome. Presentations include
a platform session as well as two poster presentations showcasing a
comprehensive dataset from the Company’s Dravet syndrome
studies.
“Our natural history study makes a compelling case that current
treatments are insufficient to address the needs of patients with
Dravet syndrome because patients still have high seizure rates and
plateau in their neurodevelopment,” said Barry Ticho, M.D., Ph.D.,
Chief Medical Officer of Stoke Therapeutics. “In this context, and
based on the data we have seen from our clinical studies of
zorevunersen, we believe we have the potential to change the way
this disease is treated by addressing the root cause of the
disease, not just the symptoms. We look forward to continuing to
work with Dravet syndrome leaders around the world to prepare for a
Phase 3 registrational study of zorevunersen.”
Details for the Company’s presentations at EEC are as
follows:
- Title: MONARCH and ADMIRAL: Open-label, Phase 1/2a
studies in USA and UK investigating safety, drug exposure, and
clinical effect of zorevunersen, an antisense oligonucleotide
(ASO), in children and adolescents with Dravet syndrome
Session: Platform Session – Paediatric Epileptology
Event Type: Platform Session Presentation Date &
Time: Tuesday, September 10, 12:22 – 12:30 PM CEST
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
- Title: 24-month analysis of BUTTERFLY: A prospective,
observational study to investigate seizures and comorbidities in
children and adolescents with Dravet syndrome (DS) Event
Type: In-Person Posters Presentation Date & Time:
Sunday, September 8, 2:30 PM - 2:33 PM CEST 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:
P788
- Title: SWALLOWTAIL and LONGWING: Open-Label Extension
(OLE) studies for children and adolescents with Dravet syndrome who
previously participated in a study of Antisense Oligonucleotide
zorevunersen Event Type: In-Person Posters Presentation
Date & Time: Sunday, September 8 - Tuesday, September 10;
Q&A from 1:30 - 3:00 PM CEST each day Presenter: Andreas
Brunklaus, MD, Consultant Paediatric Neurologist at the Royal
Hospital for Children, Glasgow, Honorary Professor at the
University of Glasgow, member of Dravet Syndrome UK's Medical
Advisory Board Poster Number: P875
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 Zorevunersen (STK-001)
Zorevunersen is an investigational new medicine for the
treatment of Dravet syndrome currently being evaluated in ongoing
clinical trials. Stoke believes that zorevunersen, a proprietary
antisense oligonucleotide (ASO), has the potential to be the first
disease-modifying therapy to address the genetic cause of Dravet
syndrome. Zorevunersen 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. Zorevunersen 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 U.S. Studies: MONARCH (Phase 1/2a) and SWALLOWTAIL
(OLE)
The MONARCH study was 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 were to assess the
safety and tolerability of zorevunersen (STK-001), as well as to
determine the pharmacokinetics in plasma and exposure in
cerebrospinal fluid. A secondary objective was to assess the
efficacy as an adjunctive antiepileptic treatment with respect to
the percentage change from baseline in convulsive seizure
frequency.
Following completion of MONARCH, patients who met study entry
criteria were 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 zorevunersen. The study
is also evaluating the long-term effects of zorevunersen on
convulsive seizure frequency and on behavior, cognition and overall
quality of life. Dosing in SWALLOWTAIL is ongoing.
About the UK Studies: ADMIRAL (Phase 1/2a) and LONGWING
(OLE)
The ADMIRAL study was 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 were to assess the
safety and tolerability of multiple doses of zorevunersen
(STK-001), as well as to determine the pharmacokinetics in plasma
and exposure in cerebrospinal fluid. A secondary objective was to
assess the effect of multiple doses of zorevunersen as an
adjunctive antiepileptic treatment with respect to the percentage
change from baseline in convulsive seizure frequency. Overall
clinical status and quality of life were secondary endpoints of
ADMIRAL.
Following completion of ADMIRAL, patients who met study entry
criteria were 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 zorevunersen. The study
is also evaluating the long-term effects of zorevunersen on
convulsive seizure frequency and on behavior, cognition and overall
quality of life. Dosing in LONGWING is ongoing.
About the BUTTERFLY Observational Study
The BUTTERFLY study was 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 study was designed to evaluate
neurodevelopmental status and change from baseline to 24 months.
Secondary and exploratory endpoints in the study evaluated changes
in other disease measures, including seizures and additional
non-seizure comorbidities. No investigational medications or other
treatments were provided. Participants continued to receive their
usual care, including anti-seizure medications, and were observed
for up to two years. The study was conducted at approximately 20
sites in the United States. Two-year results were presented at the
American Epilepsy Society in December 2023 and showed that, on
average, patients experienced no meaningful improvement in
convulsive seizure frequency and exhibited widening gaps in
cognition and behavior compared to neurotypical peers, despite
treatment with the best available anti-seizure medicines.
About Stoke Therapeutics
Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company
dedicated to restoring protein expression by harnessing the body’s
potential with RNA medicine. 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, zorevunersen (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/.
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 zorevunersen to treat the underlying
causes of Dravet syndrome and reduce seizures or show improvements
in non-seizure comorbidities, the timing and expected progress of
clinical trials, regulatory meetings and regulatory decisions, and
the participation of scientists associated with the Company making
presentations at EEC and the presentation of data at EEC.
Statements including words such as “expect,” “plan,” “will,”
“continue,” or “ongoing” 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 prove incorrect or do not fully materialize, could cause our
results to differ materially from those expressed or implied by
such forward-looking statements, including, but not limited to,
risks and uncertainties related to: the Company’s ability to
advance, obtain regulatory approval of, and ultimately
commercialize its product candidates, including zorevunersen; the
timing of data readouts and interim and final results of
preclinical and clinical trials; the receipt and timing of
potential regulatory decisions; positive results in a clinical
trial may not be replicated in subsequent trials or successes in
early stage clinical trials may not be predictive of results in
later stage trials; the Company’s ability to fund development
activities and achieve development goals; the Company’s ability to
protect its intellectual property; the direct or indirect impact of
global business, political and macroeconomic conditions, including
inflation, interest rate volatility, cybersecurity events,
uncertainty with respect to the federal budget, instability in the
global banking system and volatile market conditions, and global
events, including public health crises, and ongoing geopolitical
conflicts, such as the conflicts in Ukraine and the Middle East;
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, 2023, its quarterly reports on Form 10-Q, and
the other documents it 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/20240903430091/en/
Stoke Media & Investor Contacts: Dawn Kalmar Chief
Communications Officer dkalmar@stoketherapeutics.com
781-303-8302
Doug Snow Director, Communications & Investor Relations
IR@stoketherapeutics.com 508-642-6485
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