– Data anticipated in Q1 2024 from studies of
STK-001 for the treatment of Dravet syndrome –
– End of Phase 1/2a study results and OLE data
from patients receiving ongoing treatment; Analyses to include
effects of STK-001 on seizure frequency, cognition and behavior
–
– Pending Q1 data, Company to request Phase 3
planning meetings with regulators –
– As of September 30, 2023, Company had $214.7
million in cash, cash equivalents and marketable securities,
anticipated to fund operations to the end of 2025 –
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 strategic priorities and anticipated milestones for
2024.
“Over the last three years we have generated a comprehensive set
of data from 81 patients that support STK-001 as potentially the
first disease-modifying medicine for Dravet syndrome. 2024 is all
about advancing this potential new medicine toward a registrational
study,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke
Therapeutics. “The recent 2-year data from our BUTTERFLY natural
history study make it clear that even the best available
anti-seizure medicines are inadequate. These data show continued
high seizure burden and stagnation of neurodevelopment, which are
in stark contrast to the improvements we see across the studies of
STK-001. Results from clinical studies anticipated in the first
quarter are expected to give us the data necessary to request
meetings with regulators to discuss our Phase 3 plans.”
Updates and Anticipated Milestones
STK-001: Dravet Syndrome
- In December, at the American Epilepsy Society annual meeting,
the Company presented data supporting the continued advancement of
STK-001 as a disease-modifying potential new medicine for the
treatment of Dravet syndrome. Two-year natural history study data
showed a lack of improvement among patients who are taking the best
available anti-seizure medicines. In contrast, substantial and
sustained reductions in seizure frequency and improvements in
cognition and behavior were observed in clinical studies of
STK-001. Modeling data suggest that higher STK-001 drug exposure in
brain leads to greater seizure reductions. Single and multiple
doses of STK-001 up to 70mg have been generally well tolerated to
date.
- In the first quarter of 2024, the Company plans to report
additional clinical and modeling data from 81 patients treated in
the Phase 1/2a studies of STK-001 (MONARCH and ADMIRAL) and the two
ongoing open-label extension studies (OLE) (SWALLOWTAIL and
LONGWING), including:
- Safety, pharmacokinetic (PK) modeling, and cerebrospinal fluid
(CSF) results;
- Seizure frequency data from ~20 patients who received 1, 2, or
3 initial doses of 70mg of STK-001 and were followed for six
months;
- The effects of repeat doses of STK-001 (30mg, 45mg) on seizure
frequency and cognition and behavior from patients treated in the
SWALLOWTAIL and LONGWING OLE studies.
- Pending the results of the Q1 data readout, the Company plans
to proceed with Phase 3 preparation activities, including
discussions with global regulatory agencies, availability of
chronic toxicology data, preparation of the investigator brochure,
submission of a final protocol to regulatory agencies and
institutional review boards (IRB).
Pipeline
- STK-002: Autosomal Dominant Optic Atrophy (ADOA): FALCON
natural history study is fully enrolled. Phase 1 study (OSPREY) of
STK-002 is expected to start in the UK in 2024.
- The Company’s collaboration with Acadia Pharmaceuticals 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) is
ongoing. The collaboration includes Rett syndrome (MECP2), SYNGAP1,
and an undisclosed neurodevelopmental target of mutual
interest.
Cash Position and Financial Guidance
- Stoke ended the third quarter of 2023 with $214.7 million in
cash, cash equivalents and marketable securities, anticipated to
fund operations to the end of 2025.
Stoke’s Presentation at the 42nd Annual J.P. Morgan
Healthcare Conference
Dr. Edward Kaye will discuss Stoke’s strategic priorities and
anticipated milestones in a presentation at the 42nd Annual J.P.
Morgan Healthcare Conference on Wednesday, January 10, 2024, at
6:45 p.m. ET (3:45 p.m. PT).
A live audio webcast of the presentation will be available on
the Investors & News section of Stoke’s website at
https://investor.stoketherapeutics.com/ and can be accessed by
following this Link. A replay of the webcast will be available for
30 days following the presentation.
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.
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.
Dosing in SWALLOWTAIL is ongoing.
About the Phase 1/2a ADMIRAL Study (United Kingdom)
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 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 STK-001 as an adjunctive antiepileptic
treatment with respect to the percentage change from baseline in
convulsive seizure frequency. Stoke also measured 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 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 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.
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 observational 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 by a team of doctors and nurses over
time for up to two years. The study was conducted at approximately
20 sites in the United States.
About Autosomal Dominant Optic Atrophy (ADOA)
Autosomal dominant optic atrophy (ADOA) is the most common
inherited optic nerve disorder. It is a rare disease that causes
progressive and irreversible vision loss in both eyes starting in
the first decade of life. Severity can vary and the rate of vision
loss can be difficult to predict. Roughly half of people with ADOA
fail driving standards and up to 46% are registered as legally
blind. More than 400 OPA1 mutations have been reported in people
diagnosed with ADOA. Currently there is no approved treatment for
people living with ADOA. ADOA affects approximately one in 30,000
people globally with a higher incidence in Denmark of one in 10,000
due to a founder effect. For more information about ADOA, visit
https://www.stoketherapeutics.com/disease-areas/adoa/.
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/.
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
behavior or cognition at the indicated dosing levels or at all; the
timing and expected progress of clinical trials, data readouts and
presentations for STK-001 and STK-002; the timing of regulatory
interactions or the outcome thereof; and the Company’s cash runway.
Statements including words such as “anticipate,” “plan,” “will,”
“continue,” “expect,” 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; the timing of data readouts
and interim and final results of preclinical and clinical trials;
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; preliminary
interim data readouts of ongoing trials may show results that
change when such trials are completed; the Company’s ability to
fund development activities and achieve development goals into
2025; 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, 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240108010230/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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