– STK-001: Q1 2024 readout expected to include
end of Phase 1/2a study results and longer-term data on the effects
of repeat dosing on seizure frequency, cognition, and behavior from
the ongoing open-label extension (OLE) studies –
– STK-001: Company then plans to meet with
regulators to discuss a Phase 3 study design; Update expected in 1H
2024 –
– 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
reported financial results for the third quarter of 2023 and
provided business updates including those related to STK-001, the
Company’s proprietary antisense oligonucleotide (ASO) which is in
development by Stoke as the first potential medicine to address the
genetic cause of Dravet syndrome.
“Our recent data analyses from studies of STK-001 showed
substantial and sustained reductions in convulsive seizure
frequency among patients with Dravet syndrome who were already
receiving the best available anti-seizure medicines,” said Edward
M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “Even
more striking are the observed improvements in multiple measures of
cognition and behavior, which have not been seen in studies of the
current standard of care. These data, along with encouraging
feedback from clinicians, support our belief that STK-001 is a
disease-modifying approach that is moving treatment beyond seizure
management to address the syndrome. We are on track to complete the
Phase 1/2a studies by year-end and will use those data along with
additional OLE data to support our proposed Phase 3 dosing regimen
for discussion with global regulatory agencies.”
Third Quarter 2023 Business Highlights and Recent
Developments
Dravet Syndrome: STK-001
- In July, the Company shared positive new safety and efficacy
data from the ongoing studies of STK-001 in children and
adolescents with Dravet syndrome that suggest clinical benefit for
patients ages 2 to 18 years old, including reductions in seizures
and improvements in cognition and behavior that support the
potential for disease modification. Single and multiple doses of
STK-001 up to 70mg have been generally well tolerated.
- In September at the 35th International Epilepsy Congress, the
Company presented a new pharmacokinetic (PK) analysis of 61
patients treated in STK-001 clinical trials, demonstrating a
correlation between higher STK-001 drug exposure in brain and
greater reductions in seizure frequency over time.
Upcoming Anticipated Milestones
Dravet Syndrome: STK-001
- The Company plans to present data from the ongoing clinical
studies at the American Epilepsy Society (AES) December 1-5, 2023,
in Orlando, Fla.
- In the first quarter of 2024, the Company plans to report
additional clinical and modeling data from patients treated in the
Phase 1/2a studies MONARCH and ADMIRAL and the two open-label
extension studies (OLE) SWALLOWTAIL and LONGWING of STK-001 in
children and adolescents with Dravet syndrome. These data are
anticipated to help finalize a proposed pivotal study design for
discussion with regulators.
- Following an analysis of these data, the Company plans to meet
with regulators to discuss a Phase 3 study design. An update on
Phase 3 planning is anticipated in the first half of 2024.
Autosomal Dominant Optic Atrophy (ADOA): STK-002
- The Company plans to initiate the Phase 1 study (OSPREY) of
STK-002 in the UK in early 2024.
Third Quarter 2023 and Year-to-Date Financial Results
- As of September 30, 2023, Stoke had $214.7 million in cash,
cash equivalents, and marketable securities, which is anticipated
to fund operations to the end of 2025.
- Revenue recognized for upfront license fees and services
provided from a License and Collaboration Agreement with Acadia
Pharmaceuticals for the three months ended September 30, 2023, was
$3.3 million, compared to $2.9 million for the same period in
2022.
- Net loss for the three months ended September 30, 2023, was
$24.5 million, or $0.55 per share, compared to $26.1 million, or
$0.66 per share, for the same period in 2022.
- Research and development expenses for the three months ended
September 30, 2023, were $20.3 million, compared to $20.1 million
for the same period in 2022.
- General and administrative expenses for the three months ended
September 30, 2023, were $10.3 million, compared to $9.9 million
for the same period in 2022.
- Revenue recognized for upfront license fees and services
provided from a License and Collaboration Agreement for the nine
months ended September 30, 2023, was $6.0 million, compared to $9.1
million for the same period in 2022.
- Net loss for the nine months ended September 30, 2023, was
$77.7 million, or $1.78 per share, compared to $75.4 million, or
$1.95 per share, for the same period in 2022.
- Research and development expenses for the nine months ended
September 30, 2023, were $60.5 million, compared to $56.8 million
for the same period in 2022.
- General and administrative expenses for the nine months ended
September 30, 2023, were $30.7 million, compared to $29.5 million
for the same period in 2022.
- The increase in operating expenses for the three and nine
months ended September 30, 2023 over the same periods in 2022
primarily relate to increases in costs associated with personnel,
third party contracts, consulting, facilities and others associated
with development activities for STK-001 and STK-002, research on
additional therapeutics and growing a public corporation.
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 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.
About STK-002 STK-002 is a proprietary antisense
oligonucleotide (ASO) in preclinical development for the treatment
of Autosomal Dominant Optic Atrophy (ADOA). Approximately 80% of
individuals with ADOA experience symptoms before age 10, typically
beginning between the ages of 4 and 6. Stoke believes that STK-002
has the potential to be the first disease-modifying therapy for
people living with ADOA. An estimated 65% to 90% of cases are
caused by mutations in the OPA1 gene, most of which lead to a
haploinsufficiency resulting in 50% OPA1 protein expression and
disease manifestation. STK-002 is designed to upregulate OPA1
protein expression by leveraging the non-mutant (wild-type) copy of
the OPA1 gene to restore OPA1 protein expression with the aim to
stop or slow vision loss in patients with ADOA. Stoke has generated
preclinical data demonstrating proof-of-mechanism and
proof-of-concept for STK-002. STK-002 has been granted orphan drug
designation by the FDA as a potential new treatment for ADOA and
the company has received authorization of its CTA from the
MHRA.
About the Phase 1 OSPREY Study (United Kingdom) The
OSPREY study is a Phase 1 open-label study of children and adults
ages 6 to 55 who have an established diagnosis of ADOA and have
evidence of a genetic mutation in the OPA1 gene. The primary
objectives for the study are to assess the safety and tolerability
of single ascending doses of STK-002, as well as to determine the
exposure in blood. A secondary objective is to assess efficacy
following intravitreal (IVT) administration of STK-002 in one eye
of each patient as measured by changes in visual function and
ocular structure as well as quality of life in patients with ADOA.
Enrollment and dosing are anticipated to begin in early 2024.
About the FALCON Study FALCON is a multicenter,
prospective natural history study of people ages 8 to 60 who have
an established clinical diagnosis of ADOA that is caused by a
heterozygous OPA1 gene variant. No investigational medications or
other treatments will be provided. The study enrolled 48 patients
across 10 sites in the U.S., U.K., Italy and Denmark. Patients
undergo assessments at baseline, 6 months, 12 months, 18 months,
and 24 months. There will be no additional follow-up period.
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 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
Company’s quarterly results and cash runway; its future operating
results, financial position and liquidity; the ability of STK-001
to treat the underlying causes of Dravet syndrome and reduce
seizures or show improvements in behavior or cognition; the ability
of STK-002 to treat the underlying causes of ADOA; the timing and
expected progress of clinical trials, data readouts and
presentations; the timing or receipt of regulatory interactions or
approvals; the ability of TANGO to design medicines to increase
protein production and the expected benefits thereof. Statements
including words such as “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 its
product candidates, obtain regulatory approval of and ultimately
commercialize its product candidates; the timing and 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 to the end of 2025; the Company’s ability to
protect its intellectual property; 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.
Financial Tables Follow
Stoke Therapeutics, Inc. Consolidated balance sheets
(in thousands, except share and per share amounts)
(unaudited)
September 30,
December 31,
2023
2022
Assets Current assets: Cash and cash equivalents
$
189,977
$
113,556
Marketable securities
24,741
116,039
Prepaid expenses
10,936
10,932
Other current assets
4,269
2,955
Interest receivable
96
588
Total current assets
$
230,019
$
244,070
Restricted cash
569
569
Operating lease right-of-use assets
3,084
4,753
Property and equipment, net
6,217
6,675
Total assets
$
239,889
$
256,067
Liabilities and stockholders’ equity Current liabilities:
Accounts payable
$
3,076
$
766
Accrued and other current liabilities
13,390
15,748
Deferred revenue - current portion
8,735
14,880
Total current liabilities
$
25,201
$
31,394
Deferred revenue - net of current portion
40,730
36,856
Other long term liabilities
959
2,968
Total long term liabilities
41,689
39,824
Total liabilities
$
66,890
$
71,218
Stockholders’ equity Common stock, par value of $0.0001 per share;
300,000,000 shares authorized, 44,293,115 and 39,439,575 shares
issued and outstanding as of September 30, 2023 and December 31,
2022, respectively
4
4
Additional paid-in capital
548,033
483,170
Accumulated other comprehensive loss
(147
)
(1,175
)
Accumulated deficit
(374,891
)
(297,150
)
Total stockholders’ equity
$
172,999
$
184,849
Total liabilities and stockholders’ equity
$
239,889
$
256,067
Stoke Therapeutics, Inc. Consolidated statements of
operations and comprehensive loss (in thousands, except
share and per share amounts) (unaudited)
Three Months Ended September
30,
Nine Months Ended September
30,
2023
2022
2023
2022
Revenue
$
3,308
$
2,905
$
5,978
$
9,137
Operating expenses: Research and development
20,271
20,109
60,453
56,777
General and administrative
10,271
9,944
30,712
29,540
Total operating expenses
30,542
30,053
91,165
86,317
Loss from operations
(27,234
)
(27,148
)
(85,187
)
(77,180
)
Other income: Interest income (expense), net
2,651
995
7,321
1,643
Other income (expense), net
41
42
125
125
Total other income
2,692
1,037
7,446
1,768
Net loss
$
(24,542
)
$
(26,111
)
$
(77,741
)
$
(75,412
)
Net loss per share, basic and diluted
$
(0.55
)
$
(0.66
)
$
(1.78
)
$
(1.95
)
Weighted-average common shares outstanding, basic and diluted
44,266,017
39,420,310
43,669,987
38,716,615
Comprehensive loss: Net loss
$
(24,542
)
$
(26,111
)
$
(77,741
)
$
(75,412
)
Other comprehensive gain (loss): Unrealized gain (loss) on
marketable securities
232
(427
)
1,028
(1,535
)
Total other comprehensive loss
$
232
$
(427
)
$
1,028
$
(1,535
)
Comprehensive loss
$
(24,310
)
$
(26,538
)
$
(76,713
)
$
(76,947
)
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231107639040/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
Stoke Therapeutics (NASDAQ:STOK)
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