STOK Stoke Therapeutics Inc.
Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
|
STK-001 - MONARCH
Dravet syndrome
|
Phase 1/2
Phase 1/2
|
Phase 1/2 trial has been initiated.
|
Latest News
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– First patient dosed with STK-001 in Part A of Phase 1/2a MONARCH clinical trial for Dravet syndrome –
– Company on track to identify an additional pre-clinical candidate derived from its TANGO platform for the treatment of an additional genetic disease in 2H 2020 –
– As of June 30, 2020, company has $202.1 million in cash, cash equivalents and restricted cash, anticipated to fund operations into 2023 –
Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today reported financial results for the second quarter of 2020 and provided business updates.
"Today we are announcing that the first patient has been dosed with STK-001, which we believe has the potential to be the first-disease modifying medicine for Dravet syndrome, a severe and progressive genetic epilepsy that is characterized by developmental delays and cognitive impairment, in addition to seizure activity," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "The start of MONARCH also marks Stoke's official transition to a clinical-stage biotech company. We enter this new stage in a strong financial position to execute on our plans for STK-001 in Dravet syndrome and continue to advance the potential of our TANGO platform for additional genetic diseases."
Second Quarter 2020 Business Highlights and Recent Developments
- Stoke announced today that the first patient was enrolled and has been dosed with STK-001 in Part A of the Phase 1/2a MONARCH study of children and adolescents ages 2 to 18 years old with Dravet syndrome. Part A of the study is designed to evaluate two dose cohorts of STK-001. The U.S. FDA placed a partial clinical hold on Part B of the study, which is designed to evaluate higher doses of STK-001. Stoke has generated additional data and is in the process of preparing its response to the FDA.
- On June 12, Stoke presented additional data on the use of Stoke's TANGO technology to address OPA1 protein deficiency at the Association for Research in Vision and Ophthalmology (ARVO). OPA1 protein deficiency is the primary cause of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder.
- On July 9, the journal Nature Communications published data supporting Stoke's Targeted Augmentation of Nuclear Gene Output (TANGO) approach to addressing severe genetic diseases by precisely upregulating protein expression.
- The BUTTERFLY observational study is ongoing. Despite experiencing a slowing in new patient enrollment earlier this year due to the impact of COVID-19, new patient enrollment has resumed and we believe we have achieved sufficient participation in the study to provide informative data about the natural progression of Dravet syndrome.
Upcoming Anticipated Milestones
- Nomination of a second product candidate for the treatment of an additional genetic disease is expected in the second half of 2020.
Second Quarter and Year-to-Date Results
- Net loss for the three months ended June 30, 2020 were $13.0 million, or $0.39 per share compared to $7.8 million or $1.54 per share for the same period in 2019.
- Research and development expenses for the three months ended June 30, 2020 were $8.0 million, compared to $6.0 million for the same period in 2019.
- General and administrative expenses for the three months ended June 30, 2020 were $5.0 million, compared to $2.4 million for the same period in 2019.
- Net loss for the first six months of 2020 was $24.0 million or $0.73 per share, compared to net loss of $13.6 million or $4.57 per share for the same period in 2019.
- Research and development expenses for the six months ended June 30, 2020 were $15.2 million, compared to $10.2 million for the same period in 2019.
- General and administrative expenses for the six months ended June 30, 2020 were $9.6 million, compared to $4.6 million for the same period in 2019.
- The increase in expenses for the three and six month periods in 2020 over the same periods in 2019 primarily relate to increases in costs associated with personnel, third party contracts, consulting, facilities and others associated with development activities for STK-001, research on additional therapeutics and growing a public corporation.
- As of June 30, 2020, Stoke had approximately $202.1 million in cash, cash equivalents and restricted cash, which is anticipated to fund operations into 2023.
About STK-001
STK-001 is an investigational new medicine for the treatment of Dravet syndrome. 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. Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-001. STK-001 has been granted orphan drug designation by the FDA as a potential new treatment for Dravet syndrome.
About Phase 1/2a Clinical Study (MONARCH)
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 pathogenic genetic mutation in the SCN1A gene. The primary objectives for the study will be to assess the safety and tolerability of STK-001, as well as to characterize human pharmacokinetics. A secondary objective will be to assess the efficacy as an adjunctive antiepileptic treatment with respect to the percentage change from baseline in convulsive seizure frequency over a 12-week treatment period. Stoke also intends to measure non-seizure aspects of the disease, such as quality of life as secondary endpoints. Stoke plans to enroll approximately 40 patients across 20 sites in the United States.
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 severe intellectual disabilities, severe developmental disabilities, motor impairment, speech impairment, autism, behavioral difficulties and sleep abnormalities. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome affects approximately 35,000 people in the United States, Canada, Japan, Germany, France and the United Kingdom, and it is not concentrated in a particular geographic area or ethnic group.
About Stoke Therapeutics
Stoke Therapeutics (NASDAQ:STOK) is a biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression to restore target proteins to near normal levels. Stoke aims to develop the first precision medicine platform to target the underlying cause of a broad spectrum of genetic diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50% of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ or follow the company 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: future operating results, financial position and liquidity, the direct and indirect impact of COVID-19 on our business, financial condition and operations, including on our expenses, supply chain, strategic partners, research and development costs, clinical trials and employees; our expectation about timing and execution of anticipated milestones, responses to regulatory authorities, expected nomination of a second product candidate and timing thereof, and our ability to use study data to advance the development of STK-001; the ability of STK-001 to treat the underlying causes of Dravet syndrome; and the ability of TANGO to design medicines to increase protein production and the expected benefits thereof. These forward-looking statements may be accompanied by such words as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "might," "plan," "potential," "possible," "will," "would," and other words and terms of similar meaning. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop, obtain regulatory approval for and commercialize STK-001 and future product candidates; the timing and results of preclinical studies and clinical trials; the risk that positive results in a clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; failure to protect and enforce our intellectual property, and other proprietary rights; failure to successfully execute or realize the anticipated benefits of our strategic and growth initiatives; risks relating to technology failures or breaches; our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; risks associated with current and potential future healthcare reforms; risks relating to attracting and retaining key personnel; failure to comply with legal and regulatory requirements; risks relating to access to capital and credit markets; environmental risks; risks relating to the use of social media for our business; and the other risks and uncertainties that are described in the Risk Factors section of our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this press release. We do not undertake any obligation to publicly update any forward-looking statements.
Financial Tables Follow
Stoke Therapeutics, Inc. Condensed consolidated balance sheets (in thousands, except share and per share amounts) (unaudited) June 30, December 31, 2020
2019
Assets Current assets: Cash and cash equivalents $
201,930
$
222,471
Prepaid expenses and other current assets 3,528
3,281
Deferred financing costs 77
—
Interest receivable 9
281
Total current assets $
205,544
$
226,033
Restricted cash 205
205
Operating lease right-of-use assets 1,642
—
Property and equipment, net 2,823
2,512
Total assets $
210,214
$
228,750
Liabilities and stockholders' equity Current liabilities: Accounts payable $
904
$
751
Accrued and other current liabilities 4,901
3,350
Total current liabilities $
5,805
$
4,101
Long term liabilities 1,009
221
Total liabilities $
6,814
$
4,322
Commitments and contingencies (Note 5) Stockholders' equity Common stock, par value of $0.0001 per share; 300,000,000 shares
authorized, 33,212,544 and 32,861,842 shares issued and outstanding as
of June 30, 2020 and December 31, 2019, respectively3
3
Additional paid-in capital 285,430
282,460
Accumulated deficit (82,033
)
(58,035
)
Total stockholders' equity $
203,400
$
224,428
Total liabilities and stockholders' equity $
210,214
$
228,750
Stoke Therapeutics, Inc. Condensed 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, 2020
2019
2020
2019
Revenue $
—
$
—
$
—
$
—
Operating expenses: Research and development 7,968
6,023
15,183
10,156
General and administrative 5,044
2,422
9,563
4,611
Total operating expenses 13,012
8,445
24,746
14,767
Loss from operations (13,012
)
(8,445
)
(24,746
)
(14,767
)
Other income: Interest income 50
629
723
1,210
Other income (expense), net 3
(3
)
25
(4
)
Total other income 53
626
748
1,206
Net loss and comprehensive loss $
(12,959
)
$
(7,819
)
$
(23,998
)
$
(13,561
)
Net loss per share attributable to common stockholders, basic and diluted $
(0.39
)
$
(1.54
)
$
(0.73
)
$
(4.57
)
Weighted-average common shares outstanding, basic and diluted 33,054,656
5,083,620
32,976,026
2,970,292
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Stoke Therapeutics, Inc., (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced that members of management will present at three upcoming investor conferences in August:
2020 Wedbush PacGrow Healthcare Virtual Conference
Date: Tuesday, August 11, 2020
Time: 11:30 a.m. ETBTIG Virtual Biotechnology Conference
Date: Tuesday, August 11, 2020
Time: 2:00 p.m. ETCanaccord Genuity 40th Annual Growth Conference
Date: Wednesday, August 12, 2020
Time: 2:30 p.m. ETA live audio webcast of each presentation will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcasts will be available for 30 days following the presentations.
About Stoke Therapeutics
Stoke Therapeutics, Inc. (NASDAQ:STOK), is a biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression to restore target proteins to near normal levels. Stoke aims to develop the first precision medicine platform to target the underlying cause of a broad spectrum of genetic diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50% of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies. The company's lead investigational new medicine is STK-001, a proprietary antisense oligonucleotide (ASO) that has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ or follow the company on Twitter at @StokeTx.
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Stoke's TANGO antisense oligonucleotides showed dose-dependent reductions in non-productive mRNA and increases in both productive mRNA and protein levels from genes of diverse size, type and function
Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases, today announced the publication of data in the journal Nature Communications that support the company's proprietary approach to precisely upregulate protein expression using TANGO (Targeted Augmentation of Nuclear Gene Output) antisense oligonucleotides (ASOs).
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TANGO (Targeted Augmentation of Nuclear Gene Output)
"Stoke was founded on the idea that we could use unique insights in RNA biology to do something that has never been done before," said Isabel Aznarez, Ph.D., Co-Founder and Vice President, Head of Biology of Stoke Therapeutics and the corresponding author on the paper. "Rather than address genetic diseases by replacing, repairing or editing faulty genes, we set out to increase – or stoke – protein output from healthy genes. These data show that we can increase full-length, fully functional protein expression from a variety of healthy genes, which supports our hypothesis and may lead to a new way of treating severe genetic diseases."
To evaluate the approach broadly, Stoke selected four gene targets that vary in type and abundance of non-productive splicing events, gene size and protein function: PCCA (propionic acidemia); SYNGAP1 (autosomal dominant mental retardation 5); CD274 (autoimmune diseases, including uveitis); and SCN1A (Dravet syndrome). Stoke designed TANGO ASOs to target the non-productive splicing events in these genes and their activity was evaluated. Dose-dependent reductions of non-productive mRNA were observed to lead to increases in both productive mRNA and protein levels for each of the target genes.
More than 10,000 genetic diseases are caused by mutations in a single gene, however, current therapeutic approaches address as few as 5% of these diseases. In the experiments published today, a proprietary bioinformatics analysis of RNA sequencing datasets was used to identify a variety of non-productive alternative-splicing events that lead to mRNA degradation and limit protein production. Stoke found 7,757 unique genes that contained at least one non-productive event, of which 16% (1,246) were associated with causing a specific disease.
A link to the publication, "Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression," can be found here: https://www.nature.com/articles/s41467-020-17093-9
Pre-mRNA Splicing and TANGO
Human cells naturally regulate protein production to maintain health. Pre-mRNA splicing, including alternative splicing, is an important mechanism used to regulate how much protein and which protein variant is produced. During splicing, introns are removed and exons are joined together to generate the mRNA template that carries the code to synthesize proteins. More than one third of alternative splicing events in mammals do not produce functional proteins and lead to mRNA degradation through nonsense-mediated mRNA decay (NMD). TANGO ASOs act at the pre-mRNA level and prevent non-productive alternative splicing so that the body produces more protein-coding mRNA and thus more protein. This approach is particularly applicable to diseases that are caused by insufficient protein production.
About Stoke Therapeutics
Stoke Therapeutics, Inc. (NASDAQ:STOK), is a biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression to restore target proteins to near normal levels. Stoke aims to develop the first precision medicine platform to target the underlying cause of a broad spectrum of genetic diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50% of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies. The company's lead investigational new medicine is STK-001, a proprietary antisense oligonucleotide (ASO) that has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ or follow the company on Twitter at @StokeTx.
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: our expectations about Company's proprietary approach to precisely upregulate protein expression using TANGO ASOs and the potential benefits thereof. These forward-looking statements may be accompanied by such words as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "might," "plan," "potential," "possible," "will," "would," and other words and terms of similar meaning. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: risks related to the direct and indirect impact of COVID-19; our ability to develop, obtain regulatory approval for and commercialize current and future product candidates; the timing and results of preclinical studies and clinical trials; the risk that positive results in a clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials; the risk that regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; risks related to the occurrence of adverse safety events; risks related to failure to protect and enforce our intellectual property, and other proprietary rights; risks related to failure to successfully execute or realize the anticipated benefits of our strategic and growth initiatives; risks relating to technology failures or breaches; our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; risks associated with current and potential future healthcare reforms; risks relating to attracting and retaining key personnel; failure to comply with legal and regulatory requirements; risks relating to access to capital and credit markets; environmental risks; risks relating to the use of social media for our business; and the other risks and uncertainties that are described in the Risk Factors section of our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this press release. We do not undertake any obligation to publicly update any forward-looking statements.
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Ms. Smith brings significant experience in biotech leadership and genetic diseases
Stoke Therapeutics, Inc., (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced the appointment of Julie Anne Smith to its Board of Directors. Ms. Smith has also been appointed to the Compensation Committee of the Board of Directors. Ms. Smith will replace Samuel Hall, Ph.D., whose term on the Board of Directors expired.
"Julie brings more than two decades of experience in the life sciences industry, with a strong track record of successfully developing and commercializing medicines for rare and inherited diseases. Her expertise in drug development for neurodegenerative diseases will be particularly valued as we advance STK-001 for Dravet syndrome into the clinic later this year," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "We thank Sam for his many important contributions to Stoke from our inception and as we matured through our successful IPO to become a public company prepared to enter the clinic with STK-001, the first potential medicine developed using our TANGO platform. We welcome Julie to the Board and look forward to her insights and contributions."
"This is an exciting time for Stoke as it transitions to a clinical stage company and looks to the future," said Ms. Smith. "I am pleased to work with the Board members and the executive leadership team as they advance their work in Dravet and expand the pipeline to help people who are living with severe genetic diseases and realize the potential of the TANGO platform."
Ms. Smith currently serves as President and CEO of ESCAPE Bio, Inc., a biotechnology company developing precisely targeted therapeutics for genetic forms of neurodegenerative disease. She previously served as President and CEO of Nuredis, Inc., a biotechnology company developing small-molecule therapies for nucleotide repeat disorders such as Huntington's disease. In 2014, Ms. Smith was appointed President and CEO at Raptor Pharmaceuticals, a public biotechnology company with two commercial medicines for orphan diseases, where she served until its acquisition in 2016 (by Horizon Pharmaceuticals, Inc.). Prior to joining Raptor, Ms. Smith served as the Chief Commercial Officer at Enobia Pharmaceuticals (acquired by Alexion Pharmaceuticals, Inc.). Earlier in her career, she held positions of increasing responsibility at Jazz Pharmaceuticals plc, Genzyme, Novazyme and Bristol-Myers Squibb Company.
Ms. Smith previously served on the board of directors of Audentes Therapeutics, Inc., a genetic medicines company, and as a director on the Health and Emerging Companies Section Governing Boards of the Biotechnology Industry Organization (BIO). She currently serves on the board of directors of Exelixis, Inc., a public genomics-based drug discovery company. Ms. Smith holds a B.S. in biological and nutritional sciences from Cornell University.
About Stoke Therapeutics
Stoke Therapeutics (NASDAQ:STOK), is a biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression to restore target proteins to near normal levels. Stoke aims to develop the first precision medicine platform to target the underlying cause of a broad spectrum of genetic diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50% of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ or follow the company 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: Stoke's expectation about timing and execution of anticipated milestones with respect to STK-001, including advancement of STK-001 to the clinical stage, and expansion of the Company's pipeline. Statements including words such as "plan," "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 do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. Forward-looking statements are subject to risks and uncertainties that may cause Stoke's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to Stoke's ability to develop, obtain regulatory approval for and commercialize STK-001 and its future product candidates, the timing and results of preclinical studies and clinical trials, Stoke's ability to protect intellectual property; and other risks set forth in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These forward-looking statements are based on our current believes and expectations and speak only as of the date of this press release. We do not undertake any obligation to publicly update any forward-looking statements.
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– Company on track to begin enrollment and dosing of STK-001 in Part A of Phase 1/2a "Monarch" clinical trial in children and adolescents with Dravet syndrome in 2H 2020 –
– Research activities ongoing to identify an additional preclinical candidate derived from the company's TANGO technology platform for the treatment of an additional genetic disease in 2H 2020–
– As of March 31, 2020, company has $211.5 million in cash, cash equivalents and restricted cash, anticipated to fund operations into 2023 –
Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today reported financial results for the first quarter of 2020 and provided business updates.
"I am incredibly gratified by the focus and determination of our employees during these challenging times. Thanks to their unwavering commitment to patients, we are continuing to make progress with STK-001 and are on track to enroll and dose the first children and adolescents with Dravet syndrome in the Phase 1/2a Monarch study later this year," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "Our understanding of the potential for our TANGO technology in additional genetic diseases has continued to advance and we are generating data that we believe will support the nomination of a second preclinical candidate in the second half of 2020."
First Quarter 2020 Business Highlights and Recent Developments
- As previously announced, Stoke received U.S. Food and Drug Administration (FDA) clearance to begin dosing in Part A of its planned Phase 1/2a "Monarch" study of STK-001 in children and adolescents ages 2 to 18 years old with Dravet syndrome. Part A of the study is designed to evaluate two dose cohorts of STK-001. There is a partial clinical hold on Part B of the study, which is designed to evaluate higher doses. Stoke has initiated preclinical studies to address the FDA's request to more fully characterize the safety profile of STK-001 at doses higher than the current no observed adverse effect level. This partial clinical hold is not due to any identified manufacturing or safety issue.
- On May 12, 2020 Stoke presented the first in-vivo proof-of-concept data for TANGO antisense oligonucleotides (ASO) in an ocular disease at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting. The preclinical data showed in-vitro and in-vivo target engagement and protein upregulation in OPA1 protein deficiency, which is the primary cause of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder.
- Patients currently enrolled in the BUTTERFLY observational study are continuing to be followed. We have experienced a slowing of new patient enrollment in this study due to the impact of COVID-19 on clinical trial sites.
Upcoming Anticipated Milestones
- Data on the use of Stoke's TANGO technology to address OPA1 protein deficiency are planned for virtual presentation at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in June.
- Enrollment and dosing of patients in Part A of the Phase 1/2a Monarch single-ascending dose study of STK-001 in children and adolescents with Dravet syndrome is still expected to begin in the second half of 2020. Currently, we have not experienced any delay in initiating Monarch. To help mitigate the impact of COVID-19 to our clinical trial, we are pursuing innovative approaches such as remote monitoring and remote patient visits. We continue to anticipate preliminary data from the study in 2021.
- Nomination of a second product candidate for the treatment of an additional genetic disease is expected in the second half of 2020.
First Quarter and Year-to-Date Results
- Net loss for the first three months of 2020 was $11.0 million, compared to net loss of $5.7 million for the same period in 2019.
- Research and development expenses for the three months ended March 31, 2020 were $7.2 million, compared to $4.1 million for the same period in 2019.
- General and administrative expenses for the three months ended March 31, 2020 were $4.5 million, compared to $2.2 million for the same period in 2019.
- The increase in expenses for the 2020 period over the same period in 2019 primarily relate to increases in costs associated with personnel, third party contracts, consulting, facilities and others associated with development activities for STK-001, research on additional therapeutics and growing a public corporation.
- As of March 31, 2020, Stoke had approximately $211.5 million in cash, cash equivalents and restricted cash, which is anticipated to fund operations into 2023.
About STK-001
STK-001 is an investigational new medicine for the treatment of Dravet syndrome. 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. Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-001. STK-001 has been granted orphan drug designation by the FDA as a potential new treatment for Dravet syndrome.
About Phase 1/2a Clinical Study (Monarch)
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 pathogenic genetic mutation in the SCN1A gene. The primary objectives for the study will be to assess the safety and tolerability of STK-001, as well as to characterize human pharmacokinetics. A secondary objective will be to assess the efficacy as an adjunctive antiepileptic treatment with respect to the percentage change from baseline in convulsive seizure frequency over a 12-week treatment period. Stoke also intends to measure non-seizure aspects of the disease, such as quality of life as secondary endpoints. Stoke plans to enroll approximately 40 patients at 20 sites in the United States. Enrollment and dosing are expected to begin in the second half of 2020.
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 severe intellectual disabilities, severe developmental disabilities, motor impairment, speech impairment, autism, behavioral difficulties and sleep abnormalities. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome affects approximately 35,000 people in the United States, Canada, Japan, Germany, France and the United Kingdom, and it is not concentrated in a particular geographic area or ethnic group.
About Stoke Therapeutics
Stoke Therapeutics (NASDAQ:STOK) is a biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression to restore target proteins to near normal levels. Stoke aims to develop the first precision medicine platform to target the underlying cause of a broad spectrum of genetic diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50% of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ or follow the company 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: our first quarter results; the direct and indirect impact of COVID-19 on our business, financial condition and operations, including on our, expenses, supply chain, strategic partners, research and development costs, clinical trials and employees; our expectation about timing and execution of anticipated milestones, including enrollment in Part A of our Phase 1/2a Monarch clinical trial in Dravet syndrome, and our ability to use study data to advance the development of STK-001; the ability of STK-001 to treat the underlying causes of Dravet syndrome; and the ability of TANGO to design medicines to increase protein production. These forward-looking statements may be accompanied by such words as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "might," "plan," "potential," "possible," "will," "would," and other words and terms of similar meaning. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop, obtain regulatory approval for and commercialize STK-001 and future product candidates; the timing and results of preclinical studies and clinical trials; the risk that positive results in a clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; failure to protect and enforce our intellectual property, and other proprietary rights; failure to successfully execute or realize the anticipated benefits of our strategic and growth initiatives; risks relating to technology failures or breaches; our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; risks associated with current and potential future healthcare reforms; risks relating to attracting and retaining key personnel; failure to comply with legal and regulatory requirements; risks relating to access to capital and credit markets; environmental risks; risks relating to the use of social media for our business; and the other risks and uncertainties that are described in the Risk Factors section of our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this press release. We do not undertake any obligation to publicly update any forward-looking statements.
Financial Tables Follow
Stoke Therapeutics, Inc.
Condensed consolidated balance sheets
(in thousands, except share and per share amounts)
(unaudited)March 31,
December 31,
2020
2019
Assets
Current assets:
Cash and cash equivalents
$
211,288
$
222,471
Prepaid expenses and other current assets
4,342
3,281
Interest receivable
144
281
Total current assets
$
215,774
$
226,033
Restricted cash
205
205
Operating lease right-of-use assets
1,900
—
Property and equipment, net
2,962
2,512
Total assets
$
220,841
$
228,750
Liabilities and stockholders' equity
Current liabilities:
Accounts payable
$
1,779
$
751
Accrued and other current liabilities
3,676
3,350
Total current liabilities
$
5,455
$
4,101
Long term liabilities
1,044
221
Total liabilities
$
6,499
$
4,322
Commitments and contingencies
Stockholders' equity
Common stock, par value of $0.0001 per share; 300,000,000 shares authorized, 32,967,350 and 32,861,842 shares issued and outstanding as of March 31, 2020 and December 31, 2019, respectively
3
3
Additional paid-in capital
283,413
282,460
Accumulated deficit
(69,074
)
(58,035
)
Total stockholders' equity
$
214,342
$
224,428
Total liabilities and stockholders' equity
$
220,841
$
228,750
Stoke Therapeutics, Inc.
Condensed consolidated statements of operations and comprehensive loss
(in thousands, except share and per share amounts)
(unaudited)Three Months Ended March 31,
2020
2019
Revenue
$
—
$
—
Operating expenses:
Research and development
7,215
4,133
General and administrative
4,520
2,189
Total operating expenses
11,735
6,322
Loss from operations
(11,735
)
(6,322
)
Other income:
Interest income
674
580
Other income, net
22
—
Total other income
696
580
Net loss and comprehensive loss
$
(11,039
)
$
(5,742
)
Net loss per share attributable to common stockholders, basic and diluted
$
(0.34
)
$
(6.89
)
Weighted-average common shares outstanding, basic and diluted
32,897,395
833,469
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