– Supported by evidence from clinical studies
that indicate that zorevunersen may demonstrate substantial
improvement over available therapies –
– Update on the company’s plans for a global,
randomized, controlled Phase 3 registrational study anticipated by
year-end –
Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company
dedicated to restoring protein expression by harnessing the body’s
potential with RNA medicine, today announced that it has received
Breakthrough Therapy Designation from the U.S. Food and Drug
Administration (FDA) for zorevunersen for the treatment of Dravet
syndrome with a confirmed mutation, not associated with
gain-of-function, in the SCN1A gene. Zorevunersen is being
developed as potentially the first disease-modifying medicine for
the treatment of Dravet syndrome.
Clinical data from the Phase 1/2a and open-label extension (OLE)
studies of zorevunersen demonstrated substantial and sustained
reductions in seizure frequency and continuous improvements in
multiple measures of cognition and behavior. These effects were
observed on top of the best available anti-seizure medicines, the
current standard of care. Zorevunersen was generally well tolerated
across the studies. To date, more than 600 doses of zorevunersen
have been administered to patients, some of whom have been on
treatment for more than three years.
Discussions with the FDA and other global regulatory agencies
regarding a global, randomized, controlled Phase 3 registrational
study of zorevunersen continue to progress. The Company plans to
provide an update on its Phase 3 registrational plans by the end of
the year.
“The FDA’s Breakthrough Therapy designation for zorevunersen is
supported by promising clinical data that suggest that zorevunersen
has the potential to demonstrate substantial improvement over
current treatments for Dravet syndrome,” said Shamim Ruff, Chief
Regulatory Affairs Officer, Stoke Therapeutics. “By helping the
body restore naturally occurring NaV1.1 protein levels,
zorevunersen is designed to treat the underlying cause of the
disease. We thank the FDA for their support and look forward to
continuing to work together closely to efficiently advance
zorevunersen into a registrational Phase 3 study.”
“This designation brings new hope to the many patients with
Dravet syndrome who continue to experience treatment-resistant
seizures and a myriad of health and quality of life complications
despite the availability of symptomatic treatments,” said Mary Anne
Meskis, Executive Director, Dravet Syndrome Foundation. “Our
organization has been engaging with the FDA to ensure greater
awareness and understanding of Dravet syndrome. We are encouraged
by the Agency’s shared sense of urgency for the development of
innovative new medicines that could help address the gaps left by
current treatments by treating the underlying cause of the
disease.”
Breakthrough Therapy designation is a process designed to
expedite the development and review of drugs that are intended to
treat a serious condition and preliminary clinical evidence
indicates that the drug may demonstrate substantial improvement
over available therapy on a clinically-significant endpoint(s).
This designation grants zorevunersen access to all Fast Track
designation features, intensive guidance on an efficient drug
development program and an organizational commitment involving
senior FDA managers.
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 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, 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 behavior and cognition at the indicated dosing levels or at all;
and the timing and expected progress of clinical trials, data
readouts, regulatory meetings, regulatory decisions and other
presentations. 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; fast
track or breakthrough designations by the FDA may not lead to
faster development or regulatory review or approval process and do
not increase the likelihood that zorevunersen will receive
marketing approval; 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, including
expectations regarding its collaboration with Acadia
Pharmaceuticals; 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/20241204329451/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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