– STK-001: Additional data from patients
treated with single and multiple doses (70mg) along with data from
open-label extension studies (30mg, 45mg) anticipated in Q1 2024
–
– STK-001: Company plans to share an update on
Phase 3 planning in 1H 2024 –
– STK-002: Company received authorization to
initiate a Phase 1 study in the UK for the treatment of Autosomal
Dominant Optic Atrophy (ADOA) –
– As of June 30, 2023, Company had $231.4
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 second quarter of 2023 and
provided business updates including those related to STK-001, the
company’s proprietary antisense oligonucleotide (ASO) being
developed by Stoke as the first potential new medicine to address
the genetic cause of Dravet syndrome.
“We recently shared positive new data supporting the potential
for STK-001 as the first disease-modifying treatment for Dravet
syndrome. The data showed substantial and sustained reductions in
convulsive seizure frequency on top of the current standard of
care, along with improvements in multiple measures of cognition and
behavior, a first in the treatment of this disease,” said Edward M.
Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “The
response from clinicians to the data has been highly encouraging
and we look forward to spending more time sharing it with them at
the International Epilepsy Congress in September. We are continuing
our Phase 3 preparations as we gather additional data for analysis
in Q1 2024 to inform a pivotal study design.”
Second Quarter 2023 Business Highlights and Recent
Developments
Dravet Syndrome
- In July, the Company announced that dosing is complete in the
Phase 1/2a MONARCH and ADMIRAL studies.
- 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 from 10mg up to 70mg have been generally well
tolerated.
Autosomal Dominant Optic Atrophy (ADOA)
- In June, the Company completed enrollment (n=48) in the FALCON
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. The study is ongoing.
- In April, the Company received authorization of its Clinical
Trial Application (CTA) by the United Kingdom Medicines and
Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1
study (OSPREY) of STK-002 for the treatment of autosomal dominant
optic atrophy (ADOA), the most common inherited optic nerve
disorder. OSPREY is a 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.
Upcoming Anticipated Milestones
Dravet Syndrome
- The Company plans to provide more detail on data from the
ongoing clinical studies at the 35th International Epilepsy
Congress September 2-6, 2023 in Dublin, Ireland and also at the
American Epilepsy Society (AES) December 1-5, 2023 in Orlando,
Fla.
- The Company is on track to complete the Phase 1/2a MONARCH and
ADMIRAL studies by year-end.
- The Company anticipates additional data, including the end of
study data from MONARCH (including patients treated with a single
dose of 70mg) and ADMIRAL as well as additional data from the
SWALLOWTAIL and LONGWING open label extension studies (OLEs) in the
first quarter of 2024. These data are anticipated to inform dose
level and dosing regimen for the planned Phase 3 study.
- The Company plans to share an update on Phase 3 planning for
STK-001 in the first half of 2024.
Autosomal Dominant Optic Atrophy (ADOA)
- The Company plans to initiate the Phase 1 study (OSPREY) of
STK-002 in the UK in early 2024.
Second Quarter 2023 and Year-to-Date Financial
Results
- As of June 30, 2023, Stoke had approximately $231.4 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 June 30, 2023, was
$(2.5) million, compared to $3.2 million for the same period in
2022.
- During the quarter ended June 30, 2023, Stoke updated its
estimate of the total effort it expected to expend to satisfy its
performance obligations under the Acadia collaboration. As a
result, Stoke recorded a cumulative catch-up adjustment of $(5.3)
million which resulted in a reversal of revenue during the quarter
ended June 30, 2023. The adjustment was recorded because the total
cost to complete the work associated with the three targets
increased.
- Net loss for the three months ended June 30, 2023, was $30.7
million, or $0.69 per share, compared to $24.7 million, or $0.63
per share, for the same period in 2022.
- Research and development expenses for the three months ended
June 30, 2023, were $20.6 million, compared to $18.4 million for
the same period in 2022.
- General and administrative expenses for the three months ended
June 30, 2023, were $10.2 million, compared to $10.1 million for
the same period in 2022.
- Revenue recognized for upfront license fees and services
provided from a License and Collaboration Agreement for the six
months ended June 30, 2023, was $2.7 million, compared to $6.2
million for the same period in 2022.
- Net loss for the six months ended June 30, 2023, was $53.2
million, or $1.23 per share, compared to $49.3 million, or $1.29
per share, for the same period in 2022.
- Research and development expenses for the six months ended June
30, 2023, were $40.2 million, compared to $36.7 million for the
same period in 2022.
- General and administrative expenses for the six months ended
June 30, 2023, were $20.4 million, compared to $19.6 million for
the same period in 2022.
- The increase in expenses for the three and six month periods
ending June 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 Twitter at @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
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; the direct and indirect impacts
of public health crises, including the COVID-19 pandemic, on the
Company’s 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.
Financial Tables Follow
Stoke Therapeutics, Inc. Consolidated balance sheets
(in thousands, except share and per share amounts)
(unaudited) June 30, December 31,
2023
2022
Assets Current assets: Cash and cash equivalents
$
192,060
$
113,556
Marketable securities
39,387
116,039
Prepaid expenses
10,950
10,932
Other current assets
3,699
2,955
Interest receivable
136
588
Total current assets
$
246,232
$
244,070
Restricted cash
569
569
Operating lease right-of-use assets
3,646
4,753
Property and equipment, net
6,472
6,675
Total assets
$
256,919
$
256,067
Liabilities and stockholders’ equity Current liabilities:
Accounts payable
$
1,556
$
766
Accrued and other current liabilities
12,222
15,748
Deferred revenue - current portion
8,059
14,880
Total current liabilities
$
21,837
$
31,394
Deferred revenue - net of current portion
43,258
36,856
Other long term liabilities
1,629
2,968
Total long term liabilities
44,887
39,824
Total liabilities
$
66,724
$
71,218
Commitments and contingencies Stockholders’ equity Common stock,
par value of $0.0001 per share; 300,000,000 sharesauthorized,
44,202,997 and 39,439,575 shares issued and outstandingas of June
30, 2023 and December 31, 2022, respectively
4
4
Additional paid-in capital
540,919
483,170
Accumulated other comprehensive loss
(379
)
(1,175
)
Accumulated deficit
(350,349
)
(297,150
)
Total stockholders’ equity
$
190,195
$
184,849
Total liabilities and stockholders’ equity
$
256,919
$
256,067
Stoke Therapeutics, Inc. Consolidated statements
of operations and comprehensive loss (in thousands, except
share and per share amounts) (unaudited) Three
months ended June 30, Six Months Ended June 30,
2023
2022
2023
2022
Revenue
$
(2,481
)
$
3,231
$
2,671
$
6,232
Operating expenses: Research and development
20,551
18,358
40,182
36,668
General and administrative
10,230
10,111
20,442
19,596
Total operating expenses
30,781
28,469
60,624
56,264
Loss from operations
(33,262
)
(25,238
)
(57,953
)
(50,032
)
Other income: Interest income (expense), net
2,567
544
4,670
648
Other income (expense), net
41
42
84
83
Total other income
2,608
586
4,754
731
Net loss
$
(30,654
)
$
(24,652
)
$
(53,199
)
$
(49,301
)
Net loss per share, basic and diluted
$
(0.69
)
$
(0.63
)
$
(1.23
)
$
(1.29
)
Weighted-average common shares outstanding, basic and diluted
44,188,464
39,258,358
43,367,032
38,358,936
Comprehensive loss: Net loss
$
(30,654
)
$
(24,652
)
$
(53,199
)
$
(49,301
)
Other comprehensive gain (loss): Unrealized gain (loss) on
marketable securities
219
(592
)
796
(1,108
)
Total other comprehensive loss
$
219
$
(592
)
$
796
$
(1,108
)
Comprehensive loss
$
(30,435
)
$
(25,244
)
$
(52,403
)
$
(50,409
)
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230807835130/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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