1. – New ADMIRAL study complements ongoing U.S. studies and marks first step in global expansion of clinical development of STK-001 –

    – Study will evaluate safety and PK of multiple doses of STK-001 starting at 30mg; Enrollment and dosing anticipated to begin in 2H 2021 –

    – STK-001 has the potential to be the first disease-modifying therapy to address the root cause of Dravet syndrome, a severe and progressive genetic epilepsy –

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom Medicines and Healthcare products…

    – New ADMIRAL study complements ongoing U.S. studies and marks first step in global expansion of clinical development of STK-001 –

    – Study will evaluate safety and PK of multiple doses of STK-001 starting at 30mg; Enrollment and dosing anticipated to begin in 2H 2021 –

    – STK-001 has the potential to be the first disease-modifying therapy to address the root cause of Dravet syndrome, a severe and progressive genetic epilepsy –

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2a study (ADMIRAL) of STK-001 for the treatment of Dravet syndrome.

    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, a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. Dravet syndrome is classified as a developmental and epileptic encephalopathy resulting in developmental delay and cognitive impairment, in addition to seizure activity, that arise from the genetic mutation that causes the disease.

    "We are making excellent progress with our ongoing studies of STK-001 in the U.S. The high level of interest from the Dravet community underscores the urgent need for new treatment options that go beyond seizure control," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "The ADMIRAL study complements our U.S.-based MONARCH study, enabling the evaluation of higher dose levels of STK-001 and representing an initial step in the expansion of our global clinical development efforts. Together, we anticipate that these studies will generate a comprehensive set of data that will inform our future development plans. We look forward to working with the U.K. Dravet community – patients, families and healthcare providers – to add to our understanding of the potential for STK-001 to be the first disease-modifying therapy for Dravet syndrome."

    ADMIRAL is an open-label, multi-center, Phase 1/2a study designed to assess the safety and tolerability of multiple doses of up to 70mg of STK-001, as well as to characterize human pharmacokinetics. Secondary endpoints include change in seizure frequency and quality of life measures. The study is expected to enroll approximately 22 children and adolescents with Dravet syndrome across multiple clinical sites in the United Kingdom. Patient enrollment and dosing are expected to start in the second half of 2021.

    "Current treatments for Dravet syndrome help us manage seizures, but some of the most devastating effects of the disease such as developmental and intellectual disabilities are not addressed by current therapies," said Professor Helen Cross, Honorary Consultant in Paediatric Neurology at Great Ormond Street Hospital for Children NHS Foundation Trust. "There is great hope that a new approach, such as STK-001, that targets the root cause of the disease, may address the seizures as well as the myriad of devastating comorbidities that have impacts on patients and their families. I'm pleased to be helping lead the effort to bridge the treatment gap by conducting the first study of STK-001 in the U.K. as the lead investigator for the ADMIRAL study."

    MONARCH Phase 1/2a Clinical Trial is Ongoing in the United States

    Enrollment of children and adolescents in the first two single ascending dose (SAD) cohorts (10mg and 20mg) is complete and enrollment and dosing in the third (30mg) SAD cohort is underway. Dosing above 30mg in this study remains on partial clinical hold with the U.S. Food and Drug Administration (FDA). Preliminary safety and pharmacokinetic data from the SAD portion of the MONARCH study are expected in the second half of 2021.

    In addition, enrollment and dosing in the multiple ascending dose (MAD) portion of the MONARCH study is underway at the 20mg dose level. Patients who participated in the MONARCH study 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. Enrollment and dosing in SWALLOWTAIL are underway.

    About STK-001

    STK-001 is an investigational new medicine for the treatment of Dravet syndrome and is being evaluated in a Phase 1/2a clinical trial. 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 up-regulate 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 the Phase 1/2a MONARCH Clinical 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 characterize human pharmacokinetics. A secondary objective is to assess the effect 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. Enrollment and dosing are ongoing in MONARCH and Stoke plans to enroll approximately 48 patients in the study across 20 sites in the United States.

    Additional information about the MONARCH study can be found at https://www.monarchstudy.com/.

    Patients who participated in the MONARCH study 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. Enrollment and dosing in SWALLOWTAIL are underway.

    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 characterize human pharmacokinetics. 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 over a 24-week treatment period. Stoke also intends to measure non-seizure aspects of the disease, such as overall clinical status and quality of life, as secondary endpoints. Stoke plans to enroll approximately 22 patients in the study across multiple sites in the United Kingdom.

    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. 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. Approximately 85% of those diagnosed with Dravet syndrome have a mutation of the SCN1A gene. 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 dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines. Using the company's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels. The company'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. The company is pursuing treatment for a second haploinsufficient disease, 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 the company's 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 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: enrollment in the ADMIRAL study and the study's ability to support our clinical development plans; our expectation about timing and execution of anticipated milestones; our ability to use ADMIRAL or MONARCH study data to advance the development of STK-001; the ability of STK-001 to treat the underlying causes of Dravet syndrome and the expected benefits thereof; 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, any potential clinical candidate for OPA1 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 and activities in international jurisdictions; 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; the occurrence of adverse safety events; failure to protect and enforce our intellectual property and other proprietary rights; 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; failure to comply with legal and regulatory requirements in the United States and abroad; 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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  2. Stoke Therapeutics, Inc., (NASDAQ:STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today announced that Chief Executive Officer Edward M. Kaye, M.D., will present at two upcoming investor conferences, which will each be conducted in a fireside chat format:

    Stifel 3rd Annual CNS Day
    Date:
    Wednesday, March 31, 2021
    Time: 2:00 p.m. ET

    20th Annual Needham Virtual Healthcare Conference
    Date:
    Thursday, April 15, 2021
    Time: 12:45 p.m. ET

    A live audio webcast of each fireside chat will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30…

    Stoke Therapeutics, Inc., (NASDAQ:STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today announced that Chief Executive Officer Edward M. Kaye, M.D., will present at two upcoming investor conferences, which will each be conducted in a fireside chat format:

    Stifel 3rd Annual CNS Day

    Date:
    Wednesday, March 31, 2021

    Time: 2:00 p.m. ET

    20th Annual Needham Virtual Healthcare Conference

    Date:
    Thursday, April 15, 2021

    Time: 12:45 p.m. ET

    A live audio webcast of each fireside chat will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30 days following the fireside chats.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines. Using the company's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels. The company'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. The company is pursuing treatment for a second haploinsufficient disease, 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 the company's 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 the company on Twitter at @StokeTx.

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  3. – First patient dosed in the multiple ascending dose portion of Phase 1/2a MONARCH study of STK-001 in Dravet syndrome –

    – Preliminary safety and PK data from the single ascending dose portion of MONARCH still expected in H2 2021 –

    As of December 31, 2020, Company had $287.5 million in cash, cash equivalents and restricted cash, anticipated to fund operations into 2024

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today reported financial results for the full year ended December 31, 2020 and provided business updates.

    "During the last year, the power and potential of RNA medicines became clear…

    – First patient dosed in the multiple ascending dose portion of Phase 1/2a MONARCH study of STK-001 in Dravet syndrome –

    – Preliminary safety and PK data from the single ascending dose portion of MONARCH still expected in H2 2021 –

    As of December 31, 2020, Company had $287.5 million in cash, cash equivalents and restricted cash, anticipated to fund operations into 2024

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today reported financial results for the full year ended December 31, 2020 and provided business updates.

    "During the last year, the power and potential of RNA medicines became clear to the world. Simultaneously, the Stoke team advanced our efforts to discover and develop RNA-based medicines that aim to address the underlying cause of severe genetic diseases by selectively boosting protein production," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "Our Phase 1/2a MONARCH study in children and adolescents with Dravet syndrome is well underway and patients are now being treated with STK-001 in both the single and multiple ascending dose portions of this study. We also recently initiated an open-label extension study called SWALLOWTAIL that will provide ongoing treatment with STK-001 for patients who complete MONARCH. We expect that SWALLOWTAIL and the MAD portion of MONARCH will provide important information on the potential effects of repeat doses of STK-001."

    Dr. Kaye continued, "Looking ahead to 2021, we are on track to report preliminary safety and PK data from the SAD portion of MONARCH in the second half of the year. Our pipeline continues to advance as well. Lead optimization efforts are underway to identify a clinical candidate for OPA1 protein deficiency, the primary cause of autosomal dominant optic atrophy, and our research team is interrogating several targets of interest with the goal of demonstrating in vivo proof of mechanism and safety for another program by year end."

    Fourth Quarter 2020 Business Highlights and Recent Developments

    • The Company announced today continued clinical progress with the Phase 1/2a MONARCH study of children and adolescents with Dravet syndrome. Enrollment of patients in the first two single ascending dose (SAD) cohorts (10mg and 20mg) is now complete. Enrollment and dosing in the third (30mg) SAD cohort is underway.
    • Also announced today was the start of the multiple ascending dose (MAD) portion of the MONARCH study with the first patient enrolled and dosed in February at the 20mg dose level.
    • In January 2021, the Company began dosing patients in SWALLOWTAIL, an open label extension (OLE) study designed to evaluate the long-term safety and tolerability of repeat doses of STK-001 in patients who participated in the Phase 1/2a MONARCH study.
    • In January 2021, the Company announced completion of enrollment (n=36) in the BUTTERFLY observational study of children and adolescents ages 2 to 18 years old with Dravet syndrome. The study is ongoing.
    • In December 2020, the Company presented four abstracts related to its work in Dravet syndrome at the American Epilepsy Society (AES) 2020 Virtual Annual Meeting. Highlights from these presentations include baseline data from children and adolescents with Dravet syndrome enrolled in the BUTTERFLY observational study, which provide initial support of use of standard measures of cognition (BSID-III and WPPSI-IV) in evaluating non-seizure comorbidities in people with Dravet syndrome and new preclinical data demonstrating the ability of TANGO ASOs to help restore normal function of nerve cells, which correlate to reductions in seizure frequency and extended survival. In addition, data showed that a no-cost epilepsy genetic testing program co-sponsored by Stoke can diagnose genetic epilepsies earlier.
    • On November 24, 2020, the Company closed an underwritten public offering of 2,875,000 shares of its common stock at a price to the public of $39.00 per share. The net proceeds from the offering were approximately $104.9 million, after deducting underwriting discounts, commissions and offering expenses.

    Upcoming Anticipated Milestones

    • Preliminary safety and pharmacokinetic data from the SAD portion of the Phase 1/2a MONARCH study are expected in the second half of 2021.
    • In the second half of 2021, the Company plans to initiate natural history data collection of people living with autosomal dominant optic atrophy (ADOA) to better understand the natural progression of this disease, which is the most common inherited optic nerve disorder. The Company hopes to use the data to support future clinical development plans for the Company's OPA1 program.
    • Lead optimization is underway to potentially identify a clinical candidate for OPA1, the Company's next preclinical target, by the end of 2021.
    • Also, by the end of 2021, the Company expects to demonstrate in vivo proof of mechanism and safety for a third TANGO ASO program.

    Year End 2020 Financial Results

    • Net loss for the year ended December 31, 2020 was $52.3 million, or $1.56 per share compared to $32.3 million or $1.80 per share for the same period in 2019.
    • Research and development expenses for the year ended December 31, 2020 were $32.2 million, compared to $23.8 million for the same period in 2019.
    • General and administrative expenses for the year ended December 31, 2020 were $20.8 million, compared to $11.9 million for the same period in 2019.
    • The increase in expenses for the 2020 periods 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 December 31, 2020, Stoke had approximately $287.5 million in cash, cash equivalents and restricted cash, which is anticipated to fund operations into 2024.

    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, so the body does not function normally. 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 target genes produce more protein. TANGO aims to restore missing proteins by increasing – or stoking – protein output from healthy genes, thus compensating for the non-functioning copy of the gene.

    About STK-001

    STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in a Phase 1/2a clinical trial. 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 are to assess the safety and tolerability of STK-001, as well as to characterize human pharmacokinetics. 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 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. Enrollment and dosing are ongoing in MONARCH and Stoke plans to enroll approximately 48 patients in the study across 20 sites in the United States.

    Additional information about the MONARCH study can be found at https://www.monarchstudy.com/.

    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. 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. 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 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. Symptoms typically begin between the ages of 4 and 6 years old, affecting males and females equally. The severity of the condition by adolescence reflects the overall level of visual function to be expected throughout most of the individual's adult life. Roughly half of people with ADOA fail driving standards and up to 46% are registered as legally blind. ADOA is considered a haploinsufficiency, as most people living with ADOA have genetic mutations in the OPA1 gene that result in only half the necessary OPA1 protein being produced. More than 400 OPA1 mutations have been reported in people diagnosed with ADOA. Currently there is no approved treatment for people living with ADOA.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines. Using the company's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels. The company'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. The company is pursuing treatment for a second haploinsufficient disease, 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 the company's 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 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 year-end results and cash runway; our expectation about timing and execution of anticipated milestones, responses to regulatory authorities, expected nomination of future product candidates and programs and timing thereof; preclinical data and study results regarding OPA1 and STK-001, 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, any potential clinical candidate for OPA1 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 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; 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)

     
     

     

     

    As of December 31,

     

     

     

    2020

     

     

    2019

     

    Assets

     

     

     

     

     

     

     

     

    Current assets:

     

     

     

     

     

     

     

     

    Cash and cash equivalents

     

    $

    287,308

     

     

    $

    222,471

     

    Prepaid expenses and other current assets

     

     

    6,435

     

     

     

    3,281

     

    Deferred financing costs

     

     

    181

     

     

     

     

    Interest receivable

     

     

    6

     

     

     

    281

     

    Total current assets

     

     

    293,930

     

     

     

    226,033

     

    Restricted cash

     

     

    205

     

     

     

    205

     

    Operating lease right-of-use assets

     

     

    1,115

     

     

     

     

    Property and equipment, net

     

     

    2,675

     

     

     

    2,512

     

    Total assets

     

    $

    297,925

     

     

    $

    228,750

     

    Liabilities and stockholders' equity

     

     

     

     

     

     

     

     

    Current liabilities:

     

     

     

     

     

     

     

     

    Accounts payable

     

    $

    1,495

     

     

    $

    751

     

    Accrued and other current liabilities

     

     

    9,930

     

     

     

    3,350

     

    Total current liabilities

     

     

    11,425

     

     

     

    4,101

     

    Long term liabilities

     

     

    422

     

     

     

    221

     

    Total liabilities

     

     

    11,847

     

     

     

    4,322

     

    Commitments and contingencies

     

     

     

     

     

     

     

     

    Stockholders' equity

     

     

     

     

     

     

     

     

    Common stock, par value of $0.0001 per share; 300,000,000 shares authorized, 36,577,149 and 32,861,842 shares issued and outstanding as of December 31, 2020 and 2019, respectively

     

     

    4

     

     

     

    3

     

    Additional paid-in capital

     

     

    396,352

     

     

     

    282,460

     

    Accumulated deficit

     

     

    (110,278

    )

     

     

    (58,035

    )

    Total stockholders' equity

     

     

    286,078

     

     

     

    224,428

     

    Total liabilities and stockholders' equity

     

    $

    297,925

     

     

    $

    228,750

     

     

    Stoke Therapeutics, Inc.

    Condensed consolidated statements of operations and comprehensive loss

    (in thousands, except share and per share amounts)

    (unaudited)

     

     

     

    Year Ended

    December 31,

     

     

     

    2020

     

     

    2019

     

    Revenue

     

    $

     

     

    $

     

    Operating expenses:

     

     

     

     

     

     

     

     

    Research and development

     

     

    32,197

     

     

     

    23,764

     

    General and administrative

     

     

    20,847

     

     

     

    11,914

     

    Total operating expenses

     

     

    53,044

     

     

     

    35,678

     

    Loss from operations

     

     

    (53,044

    )

     

     

    (35,678

    )

    Other income (expense):

     

     

     

     

     

     

     

     

    Interest income

     

     

    744

     

     

     

    3,351

     

    Other income, net

     

     

    57

     

     

     

    2

     

    Total other income (expense)

     

     

    801

     

     

     

    3,353

     

    Net loss and comprehensive loss

     

     

    (52,243

    )

     

     

    (32,325

    )

    Net loss per share —basic and diluted

     

    $

    (1.56

    )

     

    $

    (1.80

    )

    Weighted average common shares outstanding—basic and diluted

     

     

    33,488,456

     

     

     

    17,971,443

     

     

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  4. Stoke Therapeutics, Inc., (NASDAQ:STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today announced that Chief Executive Officer Edward M. Kaye, M.D., will present at the Barclays Global Healthcare Conference on Thursday, March 11, 2021, at 3:35 p.m. ET.

    A live audio webcast of the presentation will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30 days following the presentations.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases…

    Stoke Therapeutics, Inc., (NASDAQ:STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today announced that Chief Executive Officer Edward M. Kaye, M.D., will present at the Barclays Global Healthcare Conference on Thursday, March 11, 2021, at 3:35 p.m. ET.

    A live audio webcast of the presentation will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30 days following the presentations.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines. Using the company's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels. The company'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. The company is pursuing treatment for a second haploinsufficient disease, 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 the company's 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 the company on Twitter at @StokeTx.

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  5. Stoke Therapeutics, Inc., (NASDAQ:STOK), a clinical-stage biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced that Chief Executive Officer Edward M. Kaye, M.D., will present at the 39th Annual J.P. Morgan Healthcare Conference on Monday, January 11, 2021, at 4:30 p.m. ET.

    A live audio webcast of the presentation will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30 days following the presentations.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a clinical-stage biotechnology company pioneering a new way to treat the…

    Stoke Therapeutics, Inc., (NASDAQ:STOK), a clinical-stage biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced that Chief Executive Officer Edward M. Kaye, M.D., will present at the 39th Annual J.P. Morgan Healthcare Conference on Monday, January 11, 2021, at 4:30 p.m. ET.

    A live audio webcast of the presentation will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30 days following the presentations.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a clinical-stage 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.

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  6. Stoke Therapeutics, Inc., (NASDAQ:STOK), a clinical-stage biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced that Chief Executive Officer Edward M. Kaye, M.D., will participate in a fireside chat at the Needham Virtual Epilepsy & Pain – Specialty Therapeutics Conference on Thursday, December 10, 2020, at 12:45 p.m. ET.

    A live audio webcast of the fireside chat will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30 days following the presentations.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a clinical-stage biotechnology…

    Stoke Therapeutics, Inc., (NASDAQ:STOK), a clinical-stage biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced that Chief Executive Officer Edward M. Kaye, M.D., will participate in a fireside chat at the Needham Virtual Epilepsy & Pain – Specialty Therapeutics Conference on Thursday, December 10, 2020, at 12:45 p.m. ET.

    A live audio webcast of the fireside chat will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30 days following the presentations.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a clinical-stage 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.

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  7. – Baseline data from the BUTTERFLY observational study provide initial validation of standard measures of cognition (BSID-III and WPPSI-IV) for use in evaluating non-seizure comorbidities in people with Dravet syndrome –

    – The current design for the ongoing MONARCH Phase 1/2a study to assess both single and multiple ascending doses of STK-001 in children and adolescents with Dravet syndrome will be presented –

    – New data in a mouse model of Dravet syndrome demonstrate that TANGO ASOs can help restore normal function of nerve cells which correlate to reductions in seizure frequency and extended survival –

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases…

    – Baseline data from the BUTTERFLY observational study provide initial validation of standard measures of cognition (BSID-III and WPPSI-IV) for use in evaluating non-seizure comorbidities in people with Dravet syndrome –

    – The current design for the ongoing MONARCH Phase 1/2a study to assess both single and multiple ascending doses of STK-001 in children and adolescents with Dravet syndrome will be presented –

    – New data in a mouse model of Dravet syndrome demonstrate that TANGO ASOs can help restore normal function of nerve cells which correlate to reductions in seizure frequency and extended survival –

    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 highlights from presentations being made at the American Epilepsy Society (AES) 2020 Virtual Annual Meeting December 4-8, 2020 related to the Company's work to advance STK-001, the first potential new medicine to target the underlying cause of Dravet syndrome. Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease. Highlights from the Company's presentations include:

    BUTTERFLY Observational Study – Baseline Analysis

    Abst. 81. Observational Study to Investigate Cognition and Quality of Life in Children and Adolescents with Dravet Syndrome: Baseline Analysis of the BUTTERFLY Study

    December 5, 2020 9:00 AM – 10:30 AM; Track: 4. Clinical Epilepsy / 4B. Clinical Diagnosis

    22 children and adolescents with Dravet syndrome were enrolled in the Company's BUTTERFLY observational study and included in a baseline analysis.

    All study participants evaluated are representative of patients with Dravet syndrome. The majority of patients were able to complete commonly used cognition assessments including either the BSID-III (Bayley Scales of Infant and Toddler Development) or the WPPSI-IV (Wechsler Preschool and Primary Scale of Intelligence Fourth Edition), indicating that these measures are valid and appropriate for use in patients with Dravet syndrome. An initial analysis of data from 17/22 patients who completed one of these assessments showed substantially decreased neurocognitive abilities compared to children of the same age level despite the use of multiple anti-epileptic therapies. The assessment also demonstrated an apparent widening in overall intellectual development that increases with age. In addition, a gap in adaptive functioning was noted using the Vineland Adaptive Behavior Scales.

    "We have long known that Dravet syndrome is far more than seizures and the BUTTERFLY study is providing quantifiable information on cognition and quality of life that fill gaps in our understanding of this disease and its progression as children age," said Joseph Sullivan, M.D., Professor of Neurology at UCSF Benioff Children's Hospitals and Director of the UCSF Pediatric Epilepsy Center of Excellence. "Initial results from the study show significantly diminished neurocognitive abilities in children with Dravet syndrome such that 10 year-olds are functioning below the level of a healthy 1 year-old even while they are being treated with available anti-epileptic medicines. The fact that the majority of patients in this study were able to complete standard assessments of neurocognition gives us confidence that we can objectively assess whether potential new disease-modifying medicines have an effect on the non-seizure comorbidities that make Dravet so devastating."

    "Dravet syndrome is characterized by an array of effects that go beyond seizures and include motor and speech impairment, intellectual and developmental disabilities, behavioral deficits and abnormal sleep patterns," said Barry Ticho, M.D., Ph D., Chief Medical Officer of Stoke Therapeutics. "We continue to generate strong preclinical evidence supporting the potential for STK-001 to address the underlying cause of Dravet syndrome and reduce seizure frequency. Our goal is to develop a medicine that goes beyond seizure control to address many of the other comorbidities associated with Dravet syndrome. As we look to future clinical studies of STK-001, we are encouraged by data showing the ability to diagnose children earlier as well as the validation of tools that will help us measure the potential impact of STK-001 on cognition and quality of life."

    MONARCH Phase 1/2a Current Study Design

    Abst. 344. Safety and Pharmacokinetics of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome: Single and Multiple Ascending Dose Design for the Open-Label Phase 1/2a MONARCH Study

    December 6, 2020 12:00 PM – 1:30 PM; Track: 7. Antiepileptic Drugs / 7B. Clinical Trials

    A review of the study design for MONARCH, the Company's ongoing Phase 1/2a study of STK-001 in children and adolescents with Dravet syndrome will be presented during a poster session by Linda Laux, M.D., Associate Professor of Pediatrics (Neurology and Epilepsy), Northwestern University Feinberg School of Medicine. Following a recent protocol amendment, MONARCH is designed to evaluate both single and multiple ascending doses of up to 30mg of STK-001 in children and adolescents with Dravet syndrome. The primary endpoints are safety, tolerability and pharmacokinetic (PK) profile of STK-001 in Dravet syndrome patients. The impact of STK-001 on frequency of convulsive seizures and quality of life are secondary endpoints of this study. Patient enrollment and dosing in MONARCH is ongoing and preliminary safety and PK data are anticipated in 2021.

    Restoration of Interneuron Firing Frequency in a Dravet Mouse Model

    Abst. 236. Targeted Augmentation of Nuclear Gene Output (TANGO) of SCN1A Reduces Seizures and Rescues Parvalbumin Positive Interneuron Firing Frequency in a Mouse Model of Dravet Syndrome

    December 5, 2:00 PM – 3:30 PM; Platform A: Translational Research / Genetics

    December 6, 12:00 PM – 1:30 PM; Track: 2. Translational Research / 2B. Devices, Technologies, Stem Cells

    New preclinical data provide additional evidence of the potential for TANGO antisense oligonucleotides (ASOs) to provide a gene-specific, disease-modifying treatment for Dravet syndrome. In this study, Dravet syndrome mice treated with a TANGO ASO had significantly decreased seizure frequency and increased survival. Data presented by Eric Wengert, graduate student at the University of Virginia, show that 100% of mice treated with a TANGO ASO were seizure free between postnatal day 16 and postnatal day 19 compared to 50% of vehicle-treated control mice. The data also provide evidence that decreases in seizures and mortality are, in part, due to restoration of excitability of parvalbumin (PV) expressing interneurons. PV interneurons are commonly hypoexcitable in Dravet syndrome. In this study, treatment with a TANGO ASO restored Dravet syndrome mouse PV interneuron firing frequency to that of wild-type mice.

    Additional Work in a Dravet Mouse Model

    Antisense Oligonucleotides Increase Scn1a Expression and Reduce Seizures and SUDEP Incidence in a Mouse Model of Dravet Syndrome

    December 8, 1:30 PM – 4:00 PM; Annual Fundamentals Symposium: Fundamentally New Ideas in Epilepsy Treatment and Research

    Lori Isom, Ph.D., Maurice H. Seevers Professor and Chair of Pharmacology, University of Michigan Medical School, will present a review of data from experiments conducted with TANGO ASOs in a mouse model of Dravet syndrome.

    Early Diagnosis of Dravet Syndrome

    Abst. 392. Reducing the Time to Diagnosis and Increasing the Detection of Individuals with SCN1A-related Disease Through a Sponsored Epilepsy Genetic Testing Program

    December 6, 12:00- 1:30 PM; Track: 12. Genetics / 12A. Human Studies

    Data from an analysis of 6,874 children who participated in a no-cost epilepsy genetic testing program co-sponsored by Stoke showed that 152 had a positive molecular diagnosis related to the SCN1A gene, accounting for 2.2% of all patients. Results demonstrated a substantial decrease in the time to diagnosis from >6 years of age (2011-2015) to <2 years of age (2019-2020).

    About STK-001

    STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in a Phase 1/2a clinical trial. 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 the BUTTERFLY Observational Study

    BUTTERFLY study, an ongoing, multicenter, longitudinal, prospective 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 designed to evaluate neurodevelopmental status and change from baseline to 24 months. Secondary and exploratory endpoints in the study will evaluate changes in other disease measures, including seizures and additional non-seizure comorbidities. No investigational medications or other treatments will be provided. Participants continue to receive their usual care, and will be observed by a team of doctors and nurses over time for up to two years. The study is being conducted at approximately 20 sites in the United States.

    About the 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. Enrollment and dosing are ongoing in MONARCH and Stoke plans to enroll approximately 48 patients in the study across 20 sites in the United States.

    Additional information about the MONARCH study can be found at https://www.monarchstudy.com/.

    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 clinical-stage 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: preclinical data and study results regarding STK-001 and Dravet syndrome, including from the BUTTERFLY study; our 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 future product candidates 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, OPA1 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; 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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  8. 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 four abstracts related to the Company's work in Dravet syndrome have been selected for presentation at the upcoming American Epilepsy Society (AES) 2020 Virtual Annual Meeting, taking place December 4 – 8, 2020. Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease.

    Highlights…

    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 four abstracts related to the Company's work in Dravet syndrome have been selected for presentation at the upcoming American Epilepsy Society (AES) 2020 Virtual Annual Meeting, taking place December 4 – 8, 2020. Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease.

    Highlights from the Company's presentations include:

    • Baseline data from children and adolescents with Dravet syndrome enrolled in the Company's BUTTERFLY study, an ongoing, multicenter, longitudinal, prospective study designed to evaluate neurodevelopmental status using several scales assessing cognition. Secondary and exploratory endpoints in the study will evaluate changes in disease measures including seizures and additional non-seizure comorbidities. Patients enrolled to date are representative of patients with Dravet syndrome, and data collected indicate that the selected cognition measures are valid and appropriate for use in patients with Dravet syndrome.
    • A review of the study design for MONARCH, the Company's ongoing Phase 1/2a study of STK-001, the first potential disease-modifying therapy to address the genetic cause of Dravet syndrome. Following a recent protocol amendment, the study is designed to evaluate both single ascending dose levels and multiple ascending doses of up to 30 mg of STK-001 in children and adolescents with Dravet syndrome. The primary endpoints are safety, tolerability and pharmacokinetic profile of STK-001 in Dravet syndrome patients. The impact of STK-001 on frequency of convulsive seizures and quality of life are secondary endpoints of this study. Preliminary safety and PK data are anticipated in 2021.
    • New preclinical data further support the ability of a TANGO antisense oligonucleotides (ASO) to decrease seizures and death in a Dravet syndrome mouse model and, for the first time, provide evidence this is likely due to restoration of excitability of parvalbumin (PV) expressing interneurons. In this study, 50% of control animals developed seizures between P16-19 while ASO-treated mice were seizure free. Consistent with a previous study of STK-001, the ASO also greatly decreased seizure frequency and death post-weaning. Associated with this, ASO-treatment restored Dravet syndrome mouse PV interneurons firing frequency to that of wild type mice PV interneurons.
    • Data generated from a no-cost epilepsy genetic testing program co-sponsored by Stoke demonstrate that this program can reduce the time to diagnosis and increase the detection of individuals with SCN1A-related disease.

    "Our understanding of the diagnosis, progression and effects of Dravet syndrome continues to expand and everything we are learning reinforces the urgent need for a medicine that treats the underlying cause of the disease," said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. "The data from our BUTTERFLY study give us a strong foundational understanding of the course of the disease and validate the applicability of several measures of cognition and other non-seizure comorbidities that will be helpful in evaluating potential disease-modifying medicines like STK-001."

    Details for the AES presentations are as follows:

    Title: Observational Study to Investigate Cognition and Other Non-seizure Comorbidities in Children and Adolescents with Dravet Syndrome: Patient Analysis of the BUTTERFLY Study

    Session Date & Time: Available on-demand December 4

    Presenter: Joseph Sullivan, M.D., Professor of Neurology at the University of California San Francisco and Director of the Benioff Children's Hospital Pediatric Epilepsy Center of Excellence

    Abstract Number: 81

    Title: Reducing the Time to Diagnosis and Increasing the Detection of Individuals with SCN1A-Related Disease Through a No-cost, Sponsored Epilepsy Genetic Testing Program

    Session Date & Time: Available on-demand December 4

    Presenter: Dianalee McKnight,Ph.D., Director, Medical Affairs, Invitae

    Abstract Number: 392

    Title: Safety and Pharmacokinetics of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome: Single Ascending Dose Design for the Open-Label Phase 1/2a MONARCH Study

    Session Date & Time: Available on-demand December 4

    Presenter: Linda Laux, M.D, Associate Professor of Pediatrics (Neurology and Epilepsy) at Northwestern University Feinberg School of Medicine and Attending Physician, Neurology and Epilepsy Center, Ann & Robert H. Lurie Children's Hospital of Chicago

    Abstract Number: 344

    Title: Targeted Augmentation of Nuclear Gene Output (TANGO) of SCN1A Reduces Seizures and Rescues Parvalbumin Positive Interneuron Firing Frequency in a Mouse Model of Dravet Syndrome

    Session Date & Time: Available on-demand December 4

    Presenter: Eric Wengert, Researcher, Department of Anesthesiology, University of Virginia

    Abstract Number: 236

    About STK-001

    STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in a Phase 1/2a clinical trial. 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. Enrollment and dosing are ongoing in MONARCH and Stoke plans to enroll approximately 48 patients in the study across 20 sites in the United States.

    Additional information about the MONARCH study can be found at https://www.monarchstudy.com/.

    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 clinical-stage 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: preclinical data and study results regarding OPA1, 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 future product candidates and timing thereof, our ability to complete lead optimization of ASOs for ADOA, the timing and results of ADOA preclinical studies, our ability to develop ASOs treat the underlying causes of ADOA, our ability to advance OPA1 as our next preclinical target, 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, OPA1 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.

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  9. 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 closing of its underwritten public offering of 2,875,000 shares of its common stock, including 375,000 shares sold upon full exercise of the underwriters' option to purchase additional shares, at a price to the public of $39.00 per share. The net proceeds from the offering were approximately $105.1 million, after deducting underwriting discounts and commissions and estimated offering expenses.

    J.P. Morgan Securities LLC, Cowen and Company, LLC, and Credit Suisse Securities (USA) LLC acted as joint book-running managers in the offering. Canaccord…

    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 closing of its underwritten public offering of 2,875,000 shares of its common stock, including 375,000 shares sold upon full exercise of the underwriters' option to purchase additional shares, at a price to the public of $39.00 per share. The net proceeds from the offering were approximately $105.1 million, after deducting underwriting discounts and commissions and estimated offering expenses.

    J.P. Morgan Securities LLC, Cowen and Company, LLC, and Credit Suisse Securities (USA) LLC acted as joint book-running managers in the offering. Canaccord Genuity LLC and Cantor Fitzgerald & Co. acted as passive bookrunners in the offering.

    Stoke intends to use the net proceeds from the proposed offering, together with its existing cash and cash equivalents, to fund research, clinical and process development and manufacturing of its product candidates, including late stage development of STK-001, clinical development of its next target for the treatment of Autosomal Dominant Optic Atrophy, developing additional product candidates, working capital, capital expenditures and other general corporate purposes.

    The public offering was made pursuant to a registration statement on Form S-3 previously filed and declared effective by the Securities and Exchange Commission (the "SEC"). This offering was made solely by means of a prospectus supplement and accompanying prospectus relating to and describing the terms of the offering, copies of which may be obtained from: J.P. Morgan Securities LLC, c/o Broadridge Financial Services, Attention: Prospectus Department, 1155 Long Island Avenue, Edgewood, New York 11717, or by telephone: (866) 803-9204, or by emailing ; from Cowen and Company, LLC c/o Broadridge Financial Solutions, Attention: Prospectus Department, 1155 Long Island Avenue, Edgewood, New York 11717, or by telephone: (833) 297-2926, or by emailing ; or from Credit Suisse Securities (USA) LLC, Attention: Prospectus Department, 6933 Louis Stephens Drive, Morrisville, NC 27560, by telephone at (800) 221-1037, or by email at . Electronic copies of the prospectus supplement and accompanying prospectus are available on the website of the SEC at http://www.sec.gov.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy any securities of Stoke, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    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.

    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. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. Examples of forward-looking statements include, among others, statements the Company makes regarding its expectation of market conditions, use of proceeds and Stoke's plan to develop its precision medicine platform. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although Stoke believes that the expectations reflected in such forward-looking statements are reasonable, Stoke cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the Company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the impact of the COVID-19 pandemic on the Company's business, clinical trial sites, supply chain and manufacturing facilities, market conditions, the satisfaction of customary closing conditions related to the proposed offering, as well as other risks and uncertainties described under the heading "Risk Factors" in documents Stoke files from time to time with the SEC. These forward-looking statements speak only as of the date hereof and Stoke specifically disclaims any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise, except as required by law.

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  10. 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 pricing of an underwritten public offering of 2,500,000 shares of its common stock at a price to the public of $39.00 per share. The gross proceeds from this offering are expected to be $97.5 million, before deducting underwriting discounts and commissions and other offering expenses payable by Stoke. The offering is expected to close on or about November 24, 2020, subject to the satisfaction of customary closing conditions. Stoke has also granted the underwriters a 30-day option to purchase up to an additional 375,000 shares of common stock in…

    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 pricing of an underwritten public offering of 2,500,000 shares of its common stock at a price to the public of $39.00 per share. The gross proceeds from this offering are expected to be $97.5 million, before deducting underwriting discounts and commissions and other offering expenses payable by Stoke. The offering is expected to close on or about November 24, 2020, subject to the satisfaction of customary closing conditions. Stoke has also granted the underwriters a 30-day option to purchase up to an additional 375,000 shares of common stock in connection with the public offering. All of the shares of common stock are being offered by Stoke.

    J.P. Morgan Securities LLC, Cowen and Company, LLC, and Credit Suisse Securities (USA) LLC are acting as joint book-running managers in the offering. Canaccord Genuity LLC and Cantor Fitzgerald & Co. are acting as passive bookrunners in the offering.

    Stoke intends to use the net proceeds from the proposed offering, together with its existing cash and cash equivalents, to fund research, clinical and process development and manufacturing of its product candidates, including late stage development of STK-001, clinical development of its next target for the treatment of Autosomal Dominant Optic Atrophy, developing additional product candidates, working capital, capital expenditures and other general corporate purposes.

    The shares are being offered by Stoke pursuant to a registration statement on Form S-3 previously filed and declared effective by the Securities and Exchange Commission (the "SEC"). A preliminary prospectus supplement and accompanying prospectus relating to this offering have been filed with the SEC. Copies of the preliminary prospectus supplement and the accompanying prospectus relating to this offering, and when available, the final prospectus supplement, may be obtained from: J.P. Morgan Securities LLC, c/o Broadridge Financial Services, Attention: Prospectus Department, 1155 Long Island Avenue, Edgewood, New York 11717, or by telephone: (866) 803-9204, or by emailing ; from Cowen and Company, LLC c/o Broadridge Financial Solutions, Attention: Prospectus Department, 1155 Long Island Avenue, Edgewood, New York 11717, or by telephone: (833) 297-2926, or by emailing ; or from Credit Suisse Securities (USA) LLC, Attention: Prospectus Department, 6933 Louis Stephens Drive, Morrisville, NC 27560, by telephone at (800) 221-1037, or by email at . Electronic copies of the preliminary prospectus supplement and accompanying prospectus will also be available on the website of the SEC at http://www.sec.gov.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy any securities of Stoke, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    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.

    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. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. Examples of forward-looking statements include, among others, statements the Company makes regarding its expectation of market conditions and the satisfaction of customary closing conditions related to the offering, its ability to complete the offering and expected use of proceeds, Stoke's plan to develop its precision medicine platform, anticipated preclinical and clinical development activities, potential benefits of Stoke's product candidates and potential market opportunities for Stoke's product candidates. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although Stoke believes that the expectations reflected in such forward-looking statements are reasonable, Stoke cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the Company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the impact of the COVID-19 pandemic on the Company's business, clinical trial sites, supply chain and manufacturing facilities, market conditions, the satisfaction of customary closing conditions related to the proposed offering, as well as other risks and uncertainties described under the heading "Risk Factors" in documents Stoke files from time to time with the SEC. These forward-looking statements speak only as of the date hereof and Stoke specifically disclaims any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise, except as required by law.

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  11. 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 a proposed underwritten public offering in which it intends to offer and sell, subject to market and other conditions, up to 2,500,000 shares of its common stock. In addition, Stoke intends to grant the underwriters a 30-day option to purchase up to an additional 375,000 shares of common stock. All of the shares of common stock are being offered by Stoke. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    J.P…

    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 a proposed underwritten public offering in which it intends to offer and sell, subject to market and other conditions, up to 2,500,000 shares of its common stock. In addition, Stoke intends to grant the underwriters a 30-day option to purchase up to an additional 375,000 shares of common stock. All of the shares of common stock are being offered by Stoke. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    J.P. Morgan Securities LLC, Cowen and Company, LLC, and Credit Suisse Securities (USA) LLC are acting as joint book-running managers in the offering. Canaccord Genuity LLC and Cantor Fitzgerald & Co. are acting as passive bookrunners in the offering.

    Stoke intends to use the net proceeds from the proposed offering, together with its existing cash and cash equivalents, to fund research, clinical and process development and manufacturing of its product candidates, including late stage development of STK-001, clinical development of its next target for the treatment of Autosomal Dominant Optic Atrophy, developing additional product candidates, working capital, capital expenditures and other general corporate purposes.

    The shares are being offered by Stoke pursuant to a registration statement on Form S-3 previously filed and declared effective by the Securities and Exchange Commission (the "SEC"). A preliminary prospectus supplement and accompanying prospectus relating to this offering will be filed with the SEC. When available, copies of the preliminary prospectus supplement and the accompanying prospectus relating to this offering may be obtained from: J.P. Morgan Securities LLC, c/o Broadridge Financial Services, Attention: Prospectus Department, 1155 Long Island Avenue, Edgewood, New York 11717, or by telephone: (866) 803-9204, or by emailing ; from Cowen and Company, LLC c/o Broadridge Financial Solutions, Attention: Prospectus Department, 1155 Long Island Avenue, Edgewood, New York 11717, or by telephone: (833) 297-2926, or by emailing ; or from Credit Suisse Securities (USA) LLC, Attention: Prospectus Department, 6933 Louis Stephens Drive, Morrisville, NC 27560, by telephone at (800) 221-1037, or by email at . Electronic copies of the preliminary prospectus supplement and accompanying prospectus will also be available on the website of the SEC at http://www.sec.gov.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy any securities of Stoke, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

    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.

    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. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. Examples of forward-looking statements include, among others, statements the Company makes regarding its intention to conduct an offering and sale of securities, the grant of the option to purchase additional shares, the ability to complete the offering and expected use of proceeds, Stoke's plan to develop its precision medicine platform, anticipated preclinical and clinical development activities, potential benefits of Stoke's product candidates and potential market opportunities for Stoke's product candidates. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although Stoke believes that the expectations reflected in such forward-looking statements are reasonable, Stoke cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the Company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the impact of the COVID-19 pandemic on the Company's business, clinical trial sites, supply chain and manufacturing facilities, market conditions, the satisfaction of customary closing conditions related to the proposed offering, as well as other risks and uncertainties described under the heading "Risk Factors" in documents Stoke files from time to time with the SEC. These forward-looking statements speak only as of the date hereof and Stoke specifically disclaims any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise, except as required by law.

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  12. - Company nominates OPA1 as the next preclinical target for its proprietary TANGO approach to treating the underlying cause of severe genetic diseases –

    - OPA1 protein deficiency is the leading cause of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder –

    - Enrollment and dosing in Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome is ongoing; Preliminary data still anticipated in 2021 –

    As of September 30, 2020, Company has $191.7 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…

    - Company nominates OPA1 as the next preclinical target for its proprietary TANGO approach to treating the underlying cause of severe genetic diseases –

    - OPA1 protein deficiency is the leading cause of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder –

    - Enrollment and dosing in Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome is ongoing; Preliminary data still anticipated in 2021 –

    As of September 30, 2020, Company has $191.7 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 third quarter of 2020 and provided business updates.

    "We have made important progress in recent months that includes the continued enrollment and dosing of children and adolescents in our MONARCH study, reaching agreement with the FDA to evaluate an additional higher dose of STK-001 in this study and submitting a plan to the Agency that also would allow us to evaluate multiple ascending doses. We remain on track for preliminary data from this Phase 1/2a study in 2021," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "In addition, based on new preclinical data, we are announcing today the expansion of our pipeline with the nomination of OPA1 as our next preclinical target. Consistent with our strategy, we believe our approach has the potential to be a first-in-class, disease modifying treatment for autosomal dominant optic atrophy, the most common inherited optic nerve disorder. There are currently no treatments available for this disease, which causes progressive and irreversible vision loss in both eyes starting in the first decade of life."

    The nomination of OPA1 as the Company's next preclinical target is supported by preclinical data that demonstrated in vitro and in vivo target engagement and protein upregulation in OPA1 protein-deficient cells. In these studies, TANGO antisense oligonucleotides (ASOs) demonstrated:

    • Dose-dependent decreases in non-productive OPA1 mRNA and increases in OPA1 protein expression in vitro and in vivo.
    • An increase in OPA1 protein expression to approximately 75% of wild-type levels in an OPA1 haploinsufficient (OPA1 +/-) cell line.
    • In vivo increases in OPA1 protein levels in the retina of wild-type rabbits that correlated with increases in the level of the test ASO.
    • The test ASO was well tolerated for up to 29 days (maximum days evaluated) after intravitreal injection.

    Recently completed preclinical studies have now demonstrated the ability of TANGO ASOs targeting the OPA1 gene to upregulate adenosine triphosphate production (ATP) levels in the mitochondria. These new data showed that in haploinsufficient cells where half the amount of OPA1 is present and mitochondrial function is impaired, our ASOs demonstrated an ability to increase OPA1 protein levels and also partially restore mitochondrial function as measured by an increase in ATP production. OPA1 expression is essential to retinal ganglion cell survival and visual signal transmission. Retinal ganglion cells have high energy needs making them particularly susceptible to losses in ATP production due to OPA1 haploinsufficiency.

    "The ATP finding is significant because in patients with autosomal dominant optic atrophy (ADOA), the retinal ganglion cells are not producing enough ATP and have defective mitochondrial function, which leads to cell death and progressive vision loss. These new data suggest that our ASO approach can restore mitochondrial function to potentially address the underlying cause of autosomal dominant optic atrophy," said Gene Liau Ph.D., Executive Vice President, Head of Research and Preclinical Development of Stoke Therapeutics. "Our goal is to advance an ASO that would delay, or potentially even prevent, vision loss for people living with ADOA. We aim to complete our lead optimization studies by the end of 2021 so that we can advance the most promising potential new medicine into human studies."

    OPA1 protein deficiency is the primary cause of ADOA, the most common inherited optic nerve disorder. ADOA typically presents in the first decade of life and affects approximately one in 30,000 people globally with a higher incidence in Denmark of one in 10,000 due to a founder effect. An estimated 65% to 90% of cases are caused by loss of function mutations in one allele (haploinsufficiency) in the OPA1 gene. There are over 400 different mutations reported to date in ADOA patients. Similar to Stoke's Dravet syndrome program, Stoke's approach for ADOA leverages upregulation of the wild-type allele and can potentially be used to treat ADOA due to loss of OPA1 activity in a mutation-independent manner.

    Third Quarter 2020 Business Highlights and Recent Developments

    • On October 7, the Company announced plans to move forward with dosing of STK-001 in its ongoing Phase 1/2a MONARCH study for Dravet syndrome. The FDA will allow the Company to add an additional higher dose level to the single ascending dose portion of the study, which will now include a total of three dose levels (10 mg, 20 mg and 30 mg). In addition, the Company has submitted an amendment to the MONARCH protocol to add a multiple ascending dose portion to the study, pending FDA review.
    • On August 26, the journal Science Translation Medicine published preclinical data from studies of STK-001 that demonstrated significant improvements in survival and reductions in seizure frequency in a mouse model of Dravet syndrome.
    • On August 17, the Company appointed Gary E. Menzel, Ph.D., to both its Board of Directors and Compensation Committee. Dr. Menzel brings more than 25 years of executive management experience in the global healthcare sector and currently serves as President and Chief Executive Officer of TCR2 Therapeutics Inc.
    • On July 9, the journal Nature Communications published data that support the Company's proprietary 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 continues, 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

    • Preliminary safety and pharmacokinetic data from the MONARCH study are still expected in 2021.
    • Several abstracts related to Stoke's work in Dravet syndrome have been accepted for presentation at the American Epilepsy Society (AES) Annual Meeting, December 4-8, 2020.
    • The Company expects to complete lead optimization for TANGO ASOs directed at OPA1 in 2021.

    Third Quarter and Year-to-Date Results

    • Net loss for the three months ended September 30, 2020 was $13.7 million, or $0.41 per share compared to $8.6 million or $0.26 per share for the same period in 2019.
    • Research and development expenses for the three months ended September 30, 2020 were $8.1 million, compared to $6.5 million for the same period in 2019.
    • General and administrative expenses for the three months ended September 30, 2020 were $5.6 million, compared to $3.3 million for the same period in 2019.
    • Net loss for the first nine months of 2020 was $37.7 million or $1.14 per share, compared to net loss of $22.2 million or $1.71 per share for the same period in 2019.
    • Research and development expenses for the nine months ended September 30, 2020 were $23.3 million, compared to $16.7 million for the same period in 2019.
    • General and administrative expenses for the nine months ended September 30, 2020 were $15.2 million, compared to $7.9 million for the same period in 2019.
    • The increase in expenses for the three and nine 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 September 30, 2020, Stoke had approximately $191.7 million in cash, cash equivalents and restricted cash, which is anticipated to fund operations into 2023.

    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, so the body does not function normally. 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 target genes produce more protein. TANGO aims to restore missing proteins by increasing – or stoking – protein output from healthy genes, thus compensating for the non-functioning copy of the gene.

    About STK-001

    STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in a Phase 1/2a clinical trial. 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. Enrollment and dosing are ongoing in MONARCH and Stoke plans to enroll approximately 48 patients in the study across 20 sites in the United States. Additional information about the MONARCH study can be found at https://www.monarchstudy.com/.

    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 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. Symptoms typically begin between the ages of 4 and 6 years old, affecting males and females equally. The severity of the condition by adolescence reflects the overall level of visual function to be expected throughout most of the individual's adult life. Roughly half of people with ADOA fail driving standards and up to 46% are registered as legally blind. ADOA is considered a haploinsufficiency, as most people living with ADOA have genetic mutations in the OPA1 gene that result in only half the necessary OPA1 protein being produced. More than 400 OPA1 mutations have been reported in people diagnosed with ADOA. Currently there is no approved treatment for people living with ADOA.

    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: preclinical data and study results regarding OPA1, 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 future product candidates and timing thereof, our ability to complete lead optimization of ASOs for ADOA, the timing and results of ADOA preclinical studies, our ability to develop ASOs treat the underlying causes of ADOA, our ability to advance OPA1 as our next preclinical target, 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, OPA1 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)

     

     

    September 30,

     

     

    December 31,

     

     

     

    2020

     

     

    2019

     

    Assets

     

     

     

     

     

     

     

     

    Current assets:

     

     

     

     

     

     

     

     

    Cash and cash equivalents

     

    $

    191,461

     

     

    $

    222,471

     

    Prepaid expenses and other current assets

     

     

    3,615

     

     

     

    3,281

     

    Deferred financing costs

     

     

    378

     

     

     

     

    Interest receivable

     

     

    2

     

     

    281

     

    Total current assets

     

    $

    195,456

     

     

    $

    226,033

     

    Restricted cash

     

     

    205

     

     

    205

     

    Operating lease right-of-use assets

     

     

    1,381

     

     

     

     

    Property and equipment, net

     

     

    2,893

     

     

     

    2,512

     

    Total assets

     

    $

    199,935

     

     

    $

    228,750

     

    Liabilities and stockholders' equity

     

     

     

     

     

     

     

     

    Current liabilities:

     

     

     

     

     

     

     

     

    Accounts payable

     

    $

    1,095

     

     

    $

    751

     

    Accrued and other current liabilities

     

     

    5,640

     

     

     

    3,350

     

    Total current liabilities

     

    $

    6,735

     

     

    $

    4,101

     

    Long term liabilities

     

     

    665

     

     

     

    221

     

    Total liabilities

     

    $

    7,400

     

     

    $

    4,322

     

    Commitments and contingencies

     

     

     

     

     

     

     

     

    Stockholders' equity

     

     

     

     

     

     

     

     

    Common stock, par value of $0.0001 per share; 300,000,000 shares

    authorized, 33,361,188 and 32,861,842 shares issued and outstanding as

    of September 30, 2020 and December 31, 2019, respectively

     

     

    3

     

     

     

    3

     

    Additional paid-in capital

     

     

    288,249

     

     

     

    282,460

     

    Accumulated deficit

     

     

    (95,717

    )

     

     

    (58,035

    )

    Total stockholders' equity

     

    $

    192,535

     

     

    $

    224,428

     

    Total liabilities and stockholders' equity

     

    $

    199,935

     

     

    $

    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

    September 30,

     

     

    Nine Months Ended

    September 30,

     

     

     

    2020

     

     

    2019

     

     

    2020

     

     

    2019

     

    Revenue

     

    $

     

     

    $

     

     

    $

     

     

    $

     

    Operating expenses:

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    Research and development

     

     

    8,109

     

     

     

    6,518

     

     

     

    23,293

     

     

     

    16,675

     

    General and administrative

     

     

    5,602

     

     

     

    3,324

     

     

     

    15,165

     

     

     

    7,935

     

    Total operating expenses

     

     

    13,711

     

     

     

    9,842

     

     

     

    38,458

     

     

     

    24,610

     

    Loss from operations

     

     

    (13,711

    )

     

     

    (9,842

    )

     

     

    (38,458

    )

     

     

    (24,610

    )

    Other income:

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    Interest income

     

     

    11

     

     

     

    1,236

     

     

     

    734

     

     

     

    2,447

     

    Other income (expense), net

     

     

    16

     

     

     

    2

     

     

     

    42

     

     

     

    (2

    )

    Total other income

     

     

    27

     

     

     

    1,238

     

     

     

    776

     

     

     

    2,445

     

    Net loss and comprehensive loss

     

    $

    (13,684

    )

     

    $

    (8,604

    )

     

    $

    (37,682

    )

     

    $

    (22,165

    )

    Net loss per share attributable to common stockholders, basic

    and diluted

     

    $

    (0.41

    )

     

    $

    (0.26

    )

     

    $

    (1.14

    )

     

    $

    (1.71

    )

    Weighted-average common shares outstanding, basic and diluted

     

     

    33,273,597

     

     

     

    32,707,647

     

     

     

    32,954,727

     

     

     

    12,991,672

     

     

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  13. Stoke Therapeutics, Inc., (NASDAQ:STOK), a clinical-stage biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced that Chief Executive Officer Edward M. Kaye, M.D., will present at the Stifel 2020 Virtual Healthcare Conference on Monday, November 16, 2020, at 3:20 p.m. ET.

    A live audio webcast of the presentation will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30 days following the presentations.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a clinical-stage biotechnology company pioneering a new way to treat the underlying…

    Stoke Therapeutics, Inc., (NASDAQ:STOK), a clinical-stage biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced that Chief Executive Officer Edward M. Kaye, M.D., will present at the Stifel 2020 Virtual Healthcare Conference on Monday, November 16, 2020, at 3:20 p.m. ET.

    A live audio webcast of the presentation will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30 days following the presentations.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a clinical-stage 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.

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  14. Stoke Therapeutics, Inc., (NASDAQ:STOK), a clinical-stage biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced that Chief Executive Officer Edward M. Kaye, M.D., will participate in a fireside chat at the 29th Annual Credit Suisse Virtual Healthcare Conference on Monday, November 9, 2020, at 10:15 a.m. ET.

    A live audio webcast of the fireside chat will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30 days following the presentation.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a clinical-stage biotechnology company…

    Stoke Therapeutics, Inc., (NASDAQ:STOK), a clinical-stage biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced that Chief Executive Officer Edward M. Kaye, M.D., will participate in a fireside chat at the 29th Annual Credit Suisse Virtual Healthcare Conference on Monday, November 9, 2020, at 10:15 a.m. ET.

    A live audio webcast of the fireside chat will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30 days following the presentation.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a clinical-stage 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.

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  15. - FDA to Allow an Additional Higher Dose Level in the Single Ascending Dose Portion (Formerly, Part A) of the Study –

    - New Preclinical Repeat-Dose Data Support Company Plans to Evaluate Multiple Ascending Dose Levels in MONARCH, Subject to FDA Review -

    - Enrollment and Dosing in MONARCH is Ongoing; Preliminary Data Still Anticipated in 2021 -

    Stoke Therapeutics, Inc., (NASDAQ:STOK), a clinical-stage biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced plans to move forward with dosing of STK-001 in children and adolescents in its ongoing Phase 1/2a MONARCH study for Dravet syndrome. Following recent interactions with the U.S. Food and…

    - FDA to Allow an Additional Higher Dose Level in the Single Ascending Dose Portion (Formerly, Part A) of the Study –

    - New Preclinical Repeat-Dose Data Support Company Plans to Evaluate Multiple Ascending Dose Levels in MONARCH, Subject to FDA Review -

    - Enrollment and Dosing in MONARCH is Ongoing; Preliminary Data Still Anticipated in 2021 -

    Stoke Therapeutics, Inc., (NASDAQ:STOK), a clinical-stage biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced plans to move forward with dosing of STK-001 in children and adolescents in its ongoing Phase 1/2a MONARCH study for Dravet syndrome. Following recent interactions with the U.S. Food and Drug Administration (FDA) related to the partial clinical hold on higher dose levels in the MONARCH study, the FDA will allow the Company to add an additional higher dose level to the single ascending dose (SAD) portion of the study (previously Part A). A total of three dose levels will now be evaluated in this portion of the study: 10 mg, 20 mg and 30 mg. Dosing above 30 mg in this study remains on FDA partial clinical hold.

    In addition, subject to FDA review, the Company is preparing to add a multiple ascending dose (MAD) portion to the MONARCH study, replacing Part B. This decision is based on new preclinical repeat-dose toxicology data, which were reviewed by the FDA as part of ongoing discussions with the Company. There were no adverse effects observed in the non-human primate (NHP) repeat dose study. The Company plans to submit a protocol amendment to the FDA, which will reflect these changes to the SAD and MAD portions of the study.

    "There is an urgent need for more effective medicines for people who are living with Dravet syndrome, so we are pleased to be moving ahead quickly with our plans to continue dosing children and adolescents in this important Phase 1/2a study of STK-001," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "We appreciate the FDA's timely review of our additional data and look forward to evaluating a total of three individual dose levels in the single ascending dose portion of the study. In addition, we are encouraged by preclinical data that demonstrated the ability to safely achieve greater exposure levels with multiple doses of STK-001. Based on these data, we plan to also evaluate multiple ascending doses of up to 30 mg in this ongoing study. Our team is working diligently to submit a revised protocol to the FDA in the coming days."

    In March 2020, the Company announced the FDA had placed a partial clinical hold on higher doses of STK-001 in the MONARCH study, pending additional preclinical testing to determine the safety profile of doses higher than the current no observed adverse effect level (NOAEL). When intrathecal doses above the NOAEL were administered to NHPs, adverse hind limb paresis was observed. This finding is known to occur following intrathecal delivery of antisense oligonucleotides (ASOs) to NHPs and is not known to translate to the human experience. When extremely high dose levels were administered, acute convulsions were observed immediately following STK-001 administration. The dose levels were well above the range of corresponding human doses that would ever be administered in the clinic, and were delivered in a formulation that was at a higher concentration than would be administered in the clinic. There is no apparent correlation of these acute adverse events with the mechanism of action of STK-001.

    Enrollment and dosing in MONARCH is ongoing. Preliminary safety and pharmacokinetic data are still anticipated in 2021.

    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 48 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 intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, chronic infections, disruptions of the autonomic nervous system and mood disorders. 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 clinical-stage 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 expectation about the dosing, timing and execution of our Phase 1/2a MONARCH study of STK-001, including our ability to include a multiple ascending dose portion in the study and the partial clinical hold on Part B of the study; the expansion of our pipeline and the use of the TANGO platform to treat other genetic diseases; 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.

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  16. 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 Chief Executive Officer Edward M. Kaye, M.D., will present at the Cantor Virtual Global Healthcare Conference on Tuesday, September 15, 2020, at 9:20 a.m. ET.

    A live webcast of the presentation will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30 days following the presentations.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a clinical-stage biotechnology company pioneering a new way to treat the underlying causes…

    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 Chief Executive Officer Edward M. Kaye, M.D., will present at the Cantor Virtual Global Healthcare Conference on Tuesday, September 15, 2020, at 9:20 a.m. ET.

    A live webcast of the presentation will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast will be available for 30 days following the presentations.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a clinical-stage 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.

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  17. STK-001 has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome

    A Phase 1/2a study of STK-001 in children and adolescents ages 2 to 18 years old is now underway

    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 publication of preclinical data from studies of STK-001 that demonstrated significant improvements in survival and reductions in seizure frequency in a mouse model of Dravet syndrome. Data published today in the journal Science Translational Medicine also showed that STK-001 achieved target engagement, pharmacologic activity and…

    STK-001 has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome

    A Phase 1/2a study of STK-001 in children and adolescents ages 2 to 18 years old is now underway

    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 publication of preclinical data from studies of STK-001 that demonstrated significant improvements in survival and reductions in seizure frequency in a mouse model of Dravet syndrome. Data published today in the journal Science Translational Medicine also showed that STK-001 achieved target engagement, pharmacologic activity and efficacy by selectively increasing Scn1a gene and Nav1.1 protein expression. STK-001 is an antisense oligonucleotide (ASO) that was created using Stoke's proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) approach. The company announced on August 10, 2020 that the first patient was dosed in Part A of its Phase 1/2a study of STK-001.

    "These preclinical studies are foundational to our understanding of STK-001 and belief in its potential to be the first disease-modifying treatment for Dravet syndrome by precisely targeting the SCN1A gene to increase protein production," said Gene Liau, Ph.D., Executive Vice President, Head of Research and Preclinical Development at Stoke Therapeutics and senior Stoke author on the paper. "We recently advanced STK-001 into the clinic with the dosing of the first patient in Part A of our Phase 1/2a MONARCH study and we look forward to learning more about the translatability of our preclinical findings to the human experience."

    "Dravet syndrome is usually caused by insufficient Nav1.1 protein levels in the brain, which leads to intractable seizures, cognitive impairments and a high risk of sudden death," said Lori I. Isom, Ph.D., Chair, Department of Pharmacology and Professor of Molecular and Integrative Physiology and Neurology at the University of Michigan Medical School and corresponding author on the paper. "In these studies, we observed that when Dravet syndrome mice were treated with a single dose of STK-001 at postnatal Day 2, 97 percent survived to Day 90 compared to 23 percent in the placebo treated group. In contrast to other emerging gene-based treatments, the ASO approach is designed to be precise, reversible and not limited by the size of a target gene, which makes it particularly promising as a future treatment for this devastating disease."

    "Antisense Oligonucleotides Increase Scn1a Expression and Reduce Seizures and SUDEP Incidence in a Mouse Model of Dravet Syndrome," is now available online at: https://stm.sciencemag.org/lookup/doi/10.1126/scitranslmed.aaz6100.

    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 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 cognitive regression or developmental stagnation, ataxia, speech impairment and sleep disturbances. 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 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, so the body does not function normally. 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 target genes produce more protein. TANGO aims to restore missing proteins by increasing – or stoking – protein output from healthy genes, thus compensating for the non-functioning copy of the gene.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK), is a clinical-stage 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 expectation about timing and execution of anticipated milestones and timing thereof, the expansion of our pipeline and the use of the TANGO platform to treat other genetic diseases; 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.

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  18. Dr. Menzel brings significant leadership in the global healthcare sector

    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 Garry E. Menzel, Ph.D., to both its Board of Directors and Compensation Committee.

    "Garry brings more than 25 years of executive management experience in the global healthcare sector, including senior leadership roles at TCR2 Therapeutics, DaVita Healthcare, and Regulus Therapeutics. His training as a scientist along with his deep financial and operational expertise in life sciences will make him an important resource for the Stoke team, especially now…

    Dr. Menzel brings significant leadership in the global healthcare sector

    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 Garry E. Menzel, Ph.D., to both its Board of Directors and Compensation Committee.

    "Garry brings more than 25 years of executive management experience in the global healthcare sector, including senior leadership roles at TCR2 Therapeutics, DaVita Healthcare, and Regulus Therapeutics. His training as a scientist along with his deep financial and operational expertise in life sciences will make him an important resource for the Stoke team, especially now that we are a clinical stage company following the start of our Phase 1/2a clinical trial in Dravet syndrome and as we work to expand the pipeline using our TANGO platform," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics.

    "As a long-serving board member of the Epilepsy Foundation, I have learned how challenging epilepsy can be, particularly for people who are living with severe genetic epilepsies like Dravet syndrome and their families," said Dr. Menzel. "The pace of discovery and innovation in this space is gathering momentum and patients are in dire need of creative therapies such as those being developed by Stoke. I look forward to working with Ed and his team to further their efforts not only with Dravet syndrome but also expanding use of TANGO to other severe genetic diseases."

    Dr. Menzel currently serves as President and Chief Executive Officer of TCR2 Therapeutics Inc. (NASDAQ:TCRR), a clinical stage oncology company with three novel immunotherapies for solid tumors and hematological malignancies. He is a co-founder of Black Diamond Therapeutics, Inc. (NASDAQ:BDTX), a clinical stage oncology company, where he continues to serve on the Board of Directors. Dr. Menzel previously served as Chief Financial Officer of DaVita Healthcare Partners (NYSE:DVA), which at the time operated one of the largest networks of kidney dialysis centers and primary care physician practices in the United States. Prior to that, Dr. Menzel served as Chief Operating Officer at microRNA therapy company Regulus Therapeutics, Inc. (NASDAQ:RGLS). He began his career in the banking industry, starting as a consultant at Bain & Company and subsequently held global leadership roles running the biotechnology practices at Goldman Sachs and Credit Suisse where he advised on more than $100 billion in strategic transactions.

    Outside of his corporate board positions, Dr. Menzel serves on the National Board of the Epilepsy Foundation, and previously served on the Board of Directors of the Institute for Systems Biology and the Board of Directors of the University of California, San Francisco (UCSF) School of Pharmacy. Dr. Menzel holds a Ph.D. in Biochemistry and Molecular Biology from St. John's College, University of Cambridge, a Master of Business Administration from Stanford University and a Bachelor of Science in Biochemistry from the Imperial College of Science and Technology.

    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.

    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 expectation about timing and execution of anticipated milestones and timing thereof; the expansion of our pipeline and the use of the TANGO platform to other genetic diseases 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.

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  19. – 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…

    – 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, respectively

     

                    3

     

     

                          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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  20. 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. ET

    BTIG Virtual Biotechnology Conference
    Date:
    Tuesday, August 11, 2020
    Time: 2:00 p.m. ET

    Canaccord Genuity 40th Annual Growth Conference
    Date:
    Wednesday, August 12, 2020
    Time: 2:30 p.m. ET

    A 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…

    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. ET

    BTIG Virtual Biotechnology Conference

    Date:
    Tuesday, August 11, 2020

    Time: 2:00 p.m. ET

    Canaccord Genuity 40th Annual Growth Conference

    Date:
    Wednesday, August 12, 2020

    Time: 2:30 p.m. ET

    A 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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  21. 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).

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200709005385/en/

    TANGO (Targeted Augmentation of Nuclear Gene Output)

    TANGO (Targeted Augmentation…

    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).

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200709005385/en/

    TANGO (Targeted Augmentation of Nuclear Gene Output)

    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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  22. 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…

    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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  23. – 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…

    – 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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  24. First in-vivo proof-of-concept for TANGO antisense oligonucleotides in an ocular disease

    Results presented at the American Society of Gene and Cell Therapy Annual Meeting further validate the company's mutation-independent approach to amplifying protein expression to treat severe 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 new preclinical data demonstrating in-vitro and in-vivo target engagement and protein upregulation in OPA1 protein-deficient cells. OPA1 protein deficiency is the primary cause of autosomal dominant optic atrophy (ADOA), the most common inherited optic…

    First in-vivo proof-of-concept for TANGO antisense oligonucleotides in an ocular disease

    Results presented at the American Society of Gene and Cell Therapy Annual Meeting further validate the company's mutation-independent approach to amplifying protein expression to treat severe 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 new preclinical data demonstrating in-vitro and in-vivo target engagement and protein upregulation in OPA1 protein-deficient cells. OPA1 protein deficiency is the primary cause of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. This is the first proof-of-concept data for TANGO antisense oligonucleotides (ASOs) in an ocular disease. The results further validate the company's mutation-independent approach to amplifying protein expression to treat severe genetic diseases. These data will be presented today in a virtual poster session at the American Society of Gene and Cell Therapy (ASGCT) 2020 Annual Meeting.

    "These data provide early evidence of the potential to address the underlying cause of autosomal dominant optic atrophy, an optic nerve disorder that causes progressive and irreversible vision loss starting in the first decade of a child's life. There are currently no approved treatments for ADOA," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "Our TANGO technology represents a unique, mutation-independent approach to treating the underlying cause of a variety of genetic diseases, particularly in the central nervous system and the eye. The ADOA program is one of several under consideration for future prioritization, and we look forward to nominating a second product candidate later this year."

    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. An estimated 65% to 90% of cases are caused by mutations in the OPA1 gene.

    The data presented today demonstrate in-vitro and in-vivo proof-of-concept for TANGO ASOs in an ocular disease. Highlights from today's presentation include:

    • Dose-dependent decreases in non-productive OPA1 mRNA and increases in OPA1 protein expression were observed in-vitro and in-vivo.
    • An increase in OPA1 protein expression to approximately 75% of wild-type levels was observed in an OPA1 haploinsufficient (OPA1 +/-) cell line.
    • In-vivo increases in OPA1protein levels in the retina of wild-type rabbits were observed and the test ASO was well tolerated for up to 15 days after intravitreal injection.

    Details of today's presentation are as follows:

    Presentation Title: Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for treatment of autosomal dominant optic atrophy

    Session Date & Time: Tuesday, May 12, 2020; 5:30 p.m. – 6:30 p.m. E.T.

    Session Title: Oligonucleotide Therapeutics

    Presenter: Aditya Venkatesh, Ph.D., Senior Scientist, Stoke Therapeutics

    Poster Number: 1593

    The poster presented at ASGCT is now available online on the Events and Presentations section of Stoke's website at https://investor.stoketherapeutics.com/.

    About Autosomal Dominant Optic Atrophy

    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. Symptoms typically begin between the ages of 4 and 6 years old, affecting males and females equally. The severity of the condition by adolescence reflects the overall level of visual function to be expected throughout most of the individual's adult life. Roughly half of people with ADOA fail driving standards and up to 46% are registered as legally blind. ADOA is considered a haploinsufficiency, as most people living with ADOA have genetic mutations in the OPA1 gene that result in only half the necessary OPA1 protein being produced. More than 400 OPA1 mutations have been reported in people diagnosed with ADOA. Currently there is no approved treatment for people living with ADOA.

    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, so the body does not function normally. 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 target genes produce more protein. TANGO aims to restore missing proteins by increasing – or stoking – protein output from healthy genes, thus compensating for the non-functioning copy of the gene.

    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 caused by 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 ability to precisely upregulate protein expression in OPA1 protein-deficient cells; Stoke's ability to treat the underlying cause of ADOA; and Stoke's ability to use preclinical data to advance the development of TANGO ASOs to treat ocular disease. Statements including words such as "plan," "continue," "expect," "target," 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 without limitation risks and uncertainties related to Stoke's ability to develop, obtain regulatory approval for and commercialize TANGO ASOs to treat ocular disease; the impact of the COVID-19 pandemic on Stoke's operations and the U.S. and world economies; the timing and results of preclinical studies; the timing for nominating a second product candidate; risks associated with potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; risks associated with current and potential future healthcare reforms; Stoke's ability to protect its intellectual property; and other risks set forth in our filings with the Securities and Exchange Commission, including the risks set forth in our quarterly report on Form 10-Q for the quarter ended March 31, 2020. These forward-looking statements are based on our current beliefs and expectations and speak only as of the date hereof and Stoke specifically disclaims any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise, except as required by law.

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  25. Company receives FDA clearance to begin dosing STK-001 in Part A of Phase 1/2a "Monarch" clinical trial in Dravet syndrome; enrollment and dosing expected to start in 2H 2020 –

    Part B of study, designed to evaluate higher doses of STK-001, on partial clinical hold pending preclinical data to more fully characterize the safety profile of STK-001 at doses higher than the current NOAEL; partial clinical hold not due to any identified manufacturing or safety issue –

    Additional toxicology studies now underway; preliminary data from Monarch study still anticipated in 2021 –

    As of December 31, 2019, company has approximately $222.7 million in cash, cash equivalents and restricted cash, which is anticipated to fund operations into 2023

    Company receives FDA clearance to begin dosing STK-001 in Part A of Phase 1/2a "Monarch" clinical trial in Dravet syndrome; enrollment and dosing expected to start in 2H 2020 –

    Part B of study, designed to evaluate higher doses of STK-001, on partial clinical hold pending preclinical data to more fully characterize the safety profile of STK-001 at doses higher than the current NOAEL; partial clinical hold not due to any identified manufacturing or safety issue –

    Additional toxicology studies now underway; preliminary data from Monarch study still anticipated in 2021 –

    As of December 31, 2019, company has approximately $222.7 million in cash, cash equivalents and restricted cash, which is 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 full year ended December 31, 2019, and provided business updates.

    "In 2019 we advanced our understanding of the potential applications of our TANGO technology to a variety of genetic targets, and generated preclinical data that underscore our confidence in STK-001 as a promising potential disease-modifying treatment for Dravet syndrome," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "As we look ahead to 2020, we are poised to become a clinical-stage company with the anticipated start of our first in-human study of STK-001, and a growing portfolio as we work toward identifying our next preclinical product candidate later this year."

    Today, the company also provided an update on its lead product candidate, STK-001, for Dravet syndrome. In the first quarter of 2020, Stoke received communication from the U.S. Food and Drug Administration (FDA) confirming that it may proceed with clinical 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.

    As part of that communication, the FDA also informed the company that it has placed a partial clinical hold on Part B of the study, which is designed to evaluate higher doses of STK-001, pending additional preclinical toxicology data. This partial clinical hold is not due to any identified manufacturing or safety issue. Rather, the FDA is requesting additional preclinical testing to determine the safety profile of doses higher than the current no observed adverse effect level (NOAEL).

    The NOAEL was determined using data from a pivotal non-human primate study that evaluated intrathecal delivery of single dose levels of STK-001. The highest dose administered in this study was equivalent to a human dose that is higher than what Stoke plans to administer in Part B of its Phase 1/2a clinical study and did not demonstrate effects that were considered adverse. Stoke has initiated single-dose toxicology studies to more fully characterize the safety profile of STK-001 at higher doses, in order to facilitate the removal of the partial clinical hold and proceed to Part B of the study. At the completion of dosing in Part A, and upon FDA clearance, Stoke will proceed with the higher-dosing cohorts planned in Part B of the study. Stoke still anticipates preliminary data from the study in 2021 and is working to minimize any potential delay to continued clinical testing of STK-001.

    "We are encouraged by the preclinical STK-001 safety and efficacy data generated to date," said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. "We look forward to moving ahead with our plans to enroll and dose patients with Dravet syndrome in Part A of the Phase 1/2a study later this year. To facilitate removal of the partial clinical hold on Part B of the study, we have begun additional single-dose toxicology studies and will work closely with the FDA to address their request and move forward as soon as possible with Part B."

    Fourth Quarter 2019 Business Highlights:

    • Presented new preclinical data supporting the planned clinical development of STK-001 at the American Epilepsy Society meeting in Baltimore, December 6 to 8, 2019. Highlights from these data included significant improvements in survival and reductions in seizure frequency as measured by electroencephalogram (EEG) in a mouse model of Dravet syndrome. Data from studies in non-human primates showed distribution throughout the brain, target engagement and increased Na v1.1 protein expression throughout the cortex after a single intrathecal injection. Safety findings showed STK-001 to be well-tolerated at both dose levels studied.
    • Added to the Nasdaq Biotechnology Index on December 23, 2019.
    • Submitted an Investigational New Drug (IND) application for STK-001 to the FDA in late 2019.

    Upcoming Anticipated Milestones:

    • An abstract detailing the use of TANGO antisense oligonucleotides (ASO) to address the underlying OPA1 protein deficiency that causes autosomal dominant optic atrophy, a rare syndrome that causes vision loss, was previously accepted for presentation at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting in May 2020. Following cancellation of the meeting due to COVID-19, Stoke is planning to submit the abstract for review and presentation at a later meeting.
    • 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 in the second half of 2020.
    • Nomination of a second product candidate for the treatment of an additional genetic disease expected in the second half of 2020.

    Year End 2019 Results

    • Net loss for the year ended December 31, 2019 was $32.3 million, compared to net loss of $12.5 million for the same period in 2018.
    • Research and development expenses for the year ended December 31, 2019 were $23.8 million, compared to $8.4 million for the same period in 2018.
    • General and administrative expenses for the year ended December 31, 2019 were $11.9 million, compared to $4.4 million for the same period in 2018.
    • The increases in expenses for the 2019 periods over the same periods in 2018 primarily relate to increases in costs associated with personnel costs, third-party contracts, consulting, facilities and other costs associated with development activities for STK-001, research on additional therapeutics and growing a public corporation.
    • As of December 31, 2019, Stoke had approximately $222.7 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 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 year end results; our expectation about timing and execution of anticipated milestones, including our IND submission; the planned initiation of 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)

     

     

     

    As of December 31,

     

     

     

    2019

     

     

    2018

     

    Assets

     

     

     

     

     

     

     

     

    Current assets:

     

     

     

     

     

     

     

     

    Cash and cash equivalents

     

    $

    222,471

     

     

    $

    105,399

     

    Prepaid expenses and other current assets

     

     

    3,281

     

     

     

    548

     

    Interest receivable

     

     

    281

     

     

     

    196

     

    Total current assets

     

     

    226,033

     

     

     

    106,143

     

    Restricted cash

     

     

    205

     

     

     

    204

     

    Property and equipment, net

     

     

    2,512

     

     

     

    1,192

     

    Total assets

     

    $

    228,750

     

     

    $

    107,539

     

    Liabilities and stockholders' equity

     

     

     

     

     

     

     

     

    Current liabilities:

     

     

     

     

     

     

     

     

    Accounts payable

     

    $

    751

     

     

    $

    1,071

     

    Accrued and other current liabilities

     

     

    3,350

     

     

     

    1,396

     

    Total current liabilities

     

     

    4,101

     

     

     

    2,467

     

    Long term liabilities

     

     

    221

     

     

     

    4

     

    Total liabilities

     

     

    4,322

     

     

     

    2,471

     

    Commitments and contingencies

     

     

     

     

     

     

     

     

    Stockholders' equity

     

     

     

     

     

     

     

     

    Preferred Stock, par value of $0.0001 per share; 10,000,000 shares authorized, none issued and outstanding as of December 31, 2019; and no shares authorized, issued or outstanding as of December 31, 2018

     

     

     

     

     

     

    Convertible Preferred Stock, par value of $0.0001 per share; no shares authorized, issued or outstanding at December 31, 2019; 225,584,874 shares authorized, 22,677,585 shares issued and outstanding as of December 31, 2018, aggregate liquidation preference of $130,850 at December 31, 2018

     

     

     

     

     

    2

     

    Common stock, par value of $0.0001 per share; 300,000,000 and 278,527,249 shares authorized, 32,861,842 and 727,413 shares issued and outstanding as of December 31, 2019 and 2018, respectively

     

     

    3

     

     

     

     

    Additional paid-in capital

     

     

    282,460

     

     

     

    130,776

     

    Accumulated deficit

     

     

    (58,035

    )

     

     

    (25,710

    )

    Total stockholders' equity

     

     

    224,428

     

     

     

    105,068

     

    Total liabilities and stockholders' equity

     

    $

    228,750

     

     

    $

    107,539

     

    Stoke Therapeutics, Inc.

    Condensed consolidated statements of operations and comprehensive loss

    (in thousands, except share and per share amounts)

    (unaudited)

     

     

     

    Year Ended

    December 31,

     

     

     

    2019

     

     

    2018

     

    Revenue

     

    $

     

     

    $

     

    Operating expenses:

     

     

     

     

     

     

     

     

    Research and development

     

     

    23,764

     

     

     

    8,371

     

    General and administrative

     

     

    11,914

     

     

     

    4,410

     

    Total operating expenses

     

     

    35,678

     

     

     

    12,781

     

    Loss from operations

     

     

    (35,678

    )

     

     

    (12,781

    )

    Other income (expense):

     

     

     

     

     

     

     

     

    Interest income

     

     

    3,351

     

     

     

    270

     

    Other income, net

     

     

    2

     

     

     

    (10

    )

    Total other income (expense)

     

     

    3,353

     

     

     

    260

     

    Net loss and comprehensive loss

     

     

    (32,325

    )

     

     

    (12,521

    )

    Net loss per share attributable to common stockholders—basic and diluted

     

    $

    (1.80

    )

     

    $

    (17.65

    )

    Weighted average common shares outstanding—basic and diluted

     

     

    17,971,443

     

     

     

    709,336

     

     

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  26. Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today announced that Chief Executive Officer Edward M. Kaye, M.D., will present at the 40th Annual Cowen Health Care Conference on Monday, March 2, 2020 at 3:30 p.m. ET in Boston.

    A live audio webcast of the presentation will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast 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…

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today announced that Chief Executive Officer Edward M. Kaye, M.D., will present at the 40th Annual Cowen Health Care Conference on Monday, March 2, 2020 at 3:30 p.m. ET in Boston.

    A live audio webcast of the presentation will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast 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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  27. SAN FRANCISCO, Jan. 8, 2020 /PRNewswire/ -- Today BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) and Invitae Corporation (NYSE:NVTA) announced that Biogen (NASDAQ:BIIB), Encoded Therapeutics, Neurogene, Praxis Precision Medicines and PTC Therapeutics joined Behind the Seizure®, an innovative, cross-company collaboration that aims to provide faster diagnosis for young children with epilepsy. The program will also be expanded to make no-charge testing available for healthcare providers to order for any child under the age of eight who has an unprovoked seizure.

    www.invitae.com (PRNewsFoto/Invitae Corporation)" alt="Invitae's (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare…

    SAN FRANCISCO, Jan. 8, 2020 /PRNewswire/ -- Today BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) and Invitae Corporation (NYSE:NVTA) announced that Biogen (NASDAQ:BIIB), Encoded Therapeutics, Neurogene, Praxis Precision Medicines and PTC Therapeutics joined Behind the Seizure®, an innovative, cross-company collaboration that aims to provide faster diagnosis for young children with epilepsy. The program will also be expanded to make no-charge testing available for healthcare providers to order for any child under the age of eight who has an unprovoked seizure.

    www.invitae.com (PRNewsFoto/Invitae Corporation)" alt="Invitae's (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)">

    "Behind the Seizure is one of the longest-running cross-company collaborations aimed at increasing access to genetic testing. It has been shown to decrease time to diagnosis for children experiencing unprovoked seizures by one to two years from reported averages, and as more companies have joined the program, more children have been helped," said Robert Nussbaum, chief medical officer of Invitae. "Earlier diagnosis enables clinicians to focus on providing disease-specific care sooner, which is particularly important in neurodegenerative diseases. We applaud these companies for their commitment to expanding this unique effort to help children."

    Previously the program was available to children under age five. The newest companies to join Behind the Seizure include:

    • Biogen: Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies.
    • Encoded Therapeutics: Encoded Therapeutics, Inc. is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders including epilepsies with a genetic etiology. Our mission is to realize the potential of genomics-driven precision medicine by overcoming key limitations of viral gene therapy. We are advancing our lead asset, ETX101, for the treatment of SCN1A-positive Dravet Syndrome.
    • Neurogene: Neurogene is committed to the rare neurological disease community, which includes children living with CLN5 and CLN7, two types of Batten disease involving recurrent seizures, or epilepsy. Expanding the age range of disease identification via Behind the Seizure will provide answers for many more children with unexplained seizures, including those with a later symptom onset, and will help advance important research in pursuit of developing life-changing medicines for patients.
    • Praxis Precision Medicines: Praxis is leveraging important breakthroughs in the genetics of epilepsy to develop novel precision medicines addressing the underlying cause of multiple pediatric genetic epilepsies.
    • PTC Therapeutics: PTC Therapeutics is a global biotech that is working toward bringing to market the first gene therapy indicated for Aromatic L-amino acid decarboxylase (AADC) deficiency, a devastating rare disease that often can be misdiagnosed as epilepsy. AADC deficiency is fatal and there are no approved therapies that address the underlying cause.

    Behind the Seizure is supported by eight sponsors in all, including Stoke Therapeutics and Xenon Pharmaceuticals who joined in 2019.

    Since the program began, thousands of children have received genetic testing through Behind the Seizure and research has shown that participants in the program were diagnosed one to two years sooner than historic averages.1 Companies that participate in the program sponsor the cost of testing using the Invitae Epilepsy Panel, which includes more than 180 genes associated with both syndromic and non-syndromic causes of epilepsy, including neurodegenerative conditions. With the expansion of the program, healthcare providers now can order the test for patients under the age of eight with unprovoked seizures. Test results are available quickly (14 days on average).

    More than half of epilepsies are based in genetics. When a child presents with seizures, genetic testing can help identify more than 100 underlying, often rare conditions. Early genetic testing may be the most cost-effective, direct and accurate diagnostic tool for children, shortening years-long diagnostic odysseys. Delays in diagnosis can be devastating for children, as some genetic epilepsies are neurodegenerative and early symptoms may be subtle and easy to misdiagnose.

    Participating companies provide financial support for this program, which includes testing and services performed by Invitae. Healthcare professionals must confirm that patients meet certain criteria to use the program. Third parties and commercial organizations may receive de-identified patient data and contact information for healthcare providers who use this program, but at no time do they receive patient identifiable information. Genetic testing and counseling are available in the US and Canada. Healthcare professionals and patients who participate in this program have no obligation to recommend, purchase, order, prescribe, promote, administer, use or support any other products or services from Invitae or from third parties or commercial organizations.

    About Behind the Seizure®
    Behind the Seizure is an innovative, cross-company collaboration designed to increase access to genetic testing for children who experience unprovoked seizures in childhood in the United States and Canada. More than half of epilepsies have some genetic basis, and are often associated with rare, neurodegenerative conditions with non-specific symptoms. Early genetic testing may be the most direct, cost-effective, and accurate diagnostic tool. Participants in the Behind the Seizure program are diagnosed one to two years sooner than reported averages. The program was established by BioMarin and Invitae and now includes: Biogen, Encoded Therapeutics, Neurogene Inc., Praxis Precision Medicines, PTC Therapeutics, Stoke Therapeutics and Xenon Pharmaceuticals. To learn more about the Behind the Seizure program please visit https://www.invitae.com/en/behindtheseizure/.

    About BioMarin
    BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com.

    About Invitae
    Invitae Corporation (NYSE:NVTA) is a leading genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website at invitae.com.

    About Encoded Therapeutics
    Encoded Therapeutics, Inc. is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. Our mission is to realize the potential of genomics-driven precision medicine by overcoming key limitations of viral gene therapy. We focus on delivering life-changing advances that move away from disease management and towards lasting disease modification. For more information, please visit www.Encoded.com.

    About Neurogene Inc.
    Neurogene was founded to bring life-changing medicines to patients and families affected by rare neurological disorders. We partner with leading academic researchers, patient advocacy organizations and caregivers to bring to patients therapies that address the underlying genetic cause of a broad spectrum of neurological diseases where no effective treatment options exist today. Our lead programs use AAV-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Neurogene is also investing in novel technology to develop treatments for diseases not well served by gene therapy. For more information, visit www.neurogene.com.

    About Praxis Precision Medicines
    Praxis Precision Medicines is a clinical-stage genetic neuroscience company developing high-impact therapies for patients and families affected by complex and debilitating brain disorders, including rare pediatric epilepsies and neuropsychiatric disorders. These two disease areas share overlapping disease biology and genetic targets, as well as a profound need for new therapeutic options that target the underlying cause of the disease. Praxis is advancing a pipeline of breakthrough medicines with the potential to more precisely treat complex brain disorders. For more information, please visit www.praxismedicines.com.

    About PTC Therapeutics
    PTC Therapeutics is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.

    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.

    About Xenon Pharmaceuticals
    Xenon Pharmaceuticals is a clinical stage biopharmaceutical company committed to developing innovative therapeutics to improve the lives of patients with neurological disorders, including rare central nervous system (CNS) conditions. We are advancing a novel product pipeline of neurology therapies to address areas of high unmet medical need, with a focus on epilepsy. For more information, please visit www.xenon-pharma.com.

    Safe Harbor Statements
    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the benefits of genetic testing and the Behind the Seizure program, including shortening the time to diagnosis and improved treatment outcomes for patients. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: the company's ability to use rapidly changing genetic data to interpret test results accurately and consistently; the ability of genetic testing to result in faster or more accurate diagnosis; laws and regulations applicable to the company's business; and the other risks set forth in Invitae's filings with the Securities and Exchange Commission, including the risks set forth in its Quarterly Report on Form 10-Q for the quarter ended September 30, 2019. These forward-looking statements speak only as of the date hereof, and Invitae Corporation disclaims any obligation to update these forward-looking statements.

    Contact:
    Laura D'Angelo

    (628) 213-3283

    1 Miller, Nicole, et al, "Behind the Seizure: A No-Cost 125-gene Epilepsy Panel for Pediatric Seizure Onset Between 2–4 Years". Presented at the American Society of Human Genetics Meeting: October 16–20, 2018, San Diego, CA.

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/behind-the-seizure-program-further-expands-access-to-genetic-testing-for-children-to-speed-the-diagnosis-of-genetic-epilepsy-300983302.html

    SOURCE Invitae Corporation

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  28. Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company
    pioneering a new way to treat the underlying cause of severe genetic
    diseases by precisely upregulating protein expression, today
    announced that Chief Executive Officer Edward M. Kaye, M.D., will
    present at the 38th Annual J.P. Morgan Healthcare Conference
    on Tuesday, January 14, 2020 at 2:30 p.m. PT (5:30 p.m. ET) in San
    Francisco.

    A live audio webcast of the presentation will be available on the
    Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/.
    A replay of the webcast will be available for 30 days following the
    presentations.

    About Stoke Therapeutics
    Stoke…

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company
    pioneering a new way to treat the underlying cause of severe genetic
    diseases by precisely upregulating protein expression, today
    announced that Chief Executive Officer Edward M. Kaye, M.D., will
    present at the 38th Annual J.P. Morgan Healthcare Conference
    on Tuesday, January 14, 2020 at 2:30 p.m. PT (5:30 p.m. ET) in San
    Francisco.

    A live audio webcast of the presentation will be available on the
    Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/.
    A replay of the webcast 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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  29. Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today announced that it has been selected for addition to the NASDAQ Biotechnology Index (NASDAQ:NBI). This addition is effective prior to market open today.

    The NASDAQ Biotechnology Index tracks the performance of a set of NASDAQ-listed securities that are classified as either biotechnology or pharmaceutical according to the Industry Classification Benchmark. Selected companies must meet eligibility requirements, including minimum market capitalization, average daily trading volume and seasoning as a public company, among other criteria. The NASDAQ Biotechnology…

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today announced that it has been selected for addition to the NASDAQ Biotechnology Index (NASDAQ:NBI). This addition is effective prior to market open today.

    The NASDAQ Biotechnology Index tracks the performance of a set of NASDAQ-listed securities that are classified as either biotechnology or pharmaceutical according to the Industry Classification Benchmark. Selected companies must meet eligibility requirements, including minimum market capitalization, average daily trading volume and seasoning as a public company, among other criteria. The NASDAQ Biotechnology Index is re-ranked annually and forms the basis for a number of Exchange Traded Funds (ETFs), including the iShares NASDAQ Biotechnology ETF. For more information about the NASDAQ Biotechnology Index visit https://indexes.nasdaqomx.com/Index/Overview/NBI.

    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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  30. STK-001 showed distribution throughout the brain after a single intrathecal injection; Target engagement and increased Nav1.1 protein expression were observed throughout the cortex

    Safety findings showed STK-001 to be well-tolerated at both dose levels studied

    IND submission on track for early 2020; Phase 1/2 study anticipated to start in 1H 2020

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today presented new preclinical data on STK-001, a potential new disease-modifying medicine for the treatment of Dravet syndrome. Data from studies in non-human primates (NHP) showed STK-001 distributed throughout…

    STK-001 showed distribution throughout the brain after a single intrathecal injection; Target engagement and increased Nav1.1 protein expression were observed throughout the cortex

    Safety findings showed STK-001 to be well-tolerated at both dose levels studied

    IND submission on track for early 2020; Phase 1/2 study anticipated to start in 1H 2020

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today presented new preclinical data on STK-001, a potential new disease-modifying medicine for the treatment of Dravet syndrome. Data from studies in non-human primates (NHP) showed STK-001 distributed throughout the brain and achieved target engagement and increased Nav1.1 protein expression throughout the cortex after a single intrathecal injection. Safety findings showed STK-001 to be well-tolerated at the two intrathecal dose levels studied. These data were presented today in a poster session at the American Epilepsy Society (AES) Annual Meeting in Baltimore.

    "The effects of Dravet syndrome go beyond seizures and often include cognitive regression or developmental stagnation, ataxia, speech impairment and sleep disturbances. The disease is believed to affect multiple areas of the brain, with the cerebral cortex playing a particularly important role," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "These new data are encouraging because they show the ability of STK-001 to broadly distribute in the brain and to elicit target engagement and increased Nav1.1 throughout the cortex. These results will be included in our planned IND submission and provide additional confidence in our clinical plans for STK-001."

    Dravet syndrome is a severe and progressive form of genetic epilepsy that affects approximately 35,000 people in the United States, Canada, Japan, Germany, France and the United Kingdom. Approximately 85% of Dravet syndrome cases are caused by spontaneous, heterozygous mutations in the SCN1A gene, resulting in 50% of normal Nav1.1 protein expression.

    Stoke selected two dose levels of STK-001 for this non-GLP study in order to evaluate safety, brain biodistribution, target engagement and Nav1.1 protein expression. On day 1, treatment-naïve cynomolgus monkeys were administered a single, bolus intrathecal lumbar (IT-L) injection at one of two dose levels of STK-001. After dosing, the animals underwent standard clinical and neurological observation, and blood samples were collected. STK-001 concentration level, gene expression, and protein expression were assessed in the brain on day 3 and on day 29.

    The following are highlights from today's poster presentation.

    • Brain tissue exposure to STK-001 was observed on day 3 and day 29. In the high dose group, exposure of STK-001 was observed in all brain regions examined, except pons and thalamus. STK-001 levels in cortical brain regions were generally higher than in deeper structures and were also increased from day 3 to day 29.
    • Nav1.1 protein levels were observed to increase up to 3-fold in some regions of the cortex on day 29 in the high dose group. No or marginal changes in Nav1.1 protein levels were observed on day 29 in the low dose group, or on day 3 in either dose group.
    • Significant target engagement (SCN1A expression) was observed on day 29 throughout the cortex and the limbic lobe in the high dose group. No or marginal change in SCN1A levels in brain tissues were observed at the low dose of STK-001, or on day 3 in either dose group.
    • A favorable safety profile was demonstrated for STK-001 at both dose levels with no change in neurological or physical measures, even in animals that overexpressed Nav1.1 protein above wild type levels.

    Stoke plans to submit an investigational new drug (IND) application to the U.S. Food and Drug Administration in early 2020 and, subject to acceptance of the IND, plans to initiate a Phase 1/2 single-ascending dose study in children and adolescents with Dravet syndrome in the first half of 2020.

    Details of today's presentation are as follows:

    Presentation Title: TANGO Oligonucleotides for the Treatment of Dravet Syndrome: Safety, Biodistribution and Pharmacology in the Non-Human Primate

    Session Date & Time: Sunday, December 8, 2019, 10:00 a.m. – 4:00 p.m. ET

    Session Title: Poster Session 2

    Presenter: Anne Christiansen, Ph.D., Associate Director, Neuroscience, Stoke Therapeutics

    Poster Number: 2.195

    Location: The Baltimore Convention Center, Hall E

    Data from preclinical studies of STK-001 in a Dravet syndrome mouse model were presented at AES on Saturday, December 7, 2019. (Poster Number 1.116)

    The posters presented at AES are now available online on the Events and Presentations section of Stoke's website at https://investor.stoketherapeutics.com/.

    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 U.S. Food and Drug Administration as a potential new treatment for Dravet syndrome. Stoke plans to submit an investigational new drug (IND) application to the U.S. Food and Drug Administration in early 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 cognitive regression or developmental stagnation, ataxia, speech impairment and sleep disturbances. 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, 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: the ability of STK-001 to improve survival and reduce seizure frequency in mice, as well as its biodistribution, target engagement and ability to increase protein expression in non-human primates; 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. 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 our actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company'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, the company's ability to protect intellectual property; and other risks set forth in our filings with the Securities and Exchange Commission, including the risks set forth in our quarterly report on Form 10-Q for the three months ended September 30, 2019. These forward-looking statements speak only as of the date hereof and we specifically disclaim any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise, except as required by law.

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  31. New data from electroencephalography (EEG) recordings showed 76% of Dravet syndrome (DS) mice treated with STK-001 were seizure free compared to 48% of placebo-treated mice

    An 80% reduction in the average number of spontaneous seizures was also observed among treated DS mice

    Data support the clinical development of STK-001 as a potential new medicine for Dravet syndrome, a severe and progressive genetic epilepsy

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today announced preclinical data from studies of STK-001 that showed significant improvements in survival and reductions in seizure frequency…

    New data from electroencephalography (EEG) recordings showed 76% of Dravet syndrome (DS) mice treated with STK-001 were seizure free compared to 48% of placebo-treated mice

    An 80% reduction in the average number of spontaneous seizures was also observed among treated DS mice

    Data support the clinical development of STK-001 as a potential new medicine for Dravet syndrome, a severe and progressive genetic epilepsy

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today announced preclinical data from studies of STK-001 that showed significant improvements in survival and reductions in seizure frequency in a mouse model of Dravet syndrome (DS). New data from electroencephalography (EEG) recordings showed 76% (16/21) of DS mice treated with STK-001 were seizure free compared to 48% (10/21) that were treated with a placebo. An 80% reduction in the average number of spontaneous seizures (3 seizures vs 16 seizures) was also observed among treated DS mice compared to placebo. EEG is a highly sensitive measure of seizure activity, which enables the detection of seizures that may not be otherwise visible. These data were presented in a poster session at the American Epilepsy Society (AES) Annual Meeting in Baltimore.

    "The data on STK-001 from this mouse model give us confidence in our approach to treating the underlying cause of Dravet syndrome by restoring Nav1.1 protein expression to near normal levels," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "The reductions in mortality previously observed with STK-001 were compelling and the new EEG data provide further evidence of the potential for STK-001 to impact Dravet syndrome by reducing seizure frequency and possibly preventing seizures entirely. What is particularly remarkable is that these data were generated from a spontaneous seizure model, which we believe accurately reflects the clinical situation in people with Dravet syndrome."

    Dravet syndrome is a severe and progressive genetic epilepsy that begins within the first year of life. The effects of the disease go beyond seizures and often include cognitive regression or developmental stagnation, ataxia, speech impairment and sleep disturbances. Approximately 85% of Dravet syndrome cases are caused by spontaneous, heterozygous variants in the SCN1A gene. This gene encodes the voltage-gated sodium channel α subunit, NaV1.1. STK-001 is a proprietary antisense oligonucleotide (ASO) designed to increase – or upregulate – protein production by manipulating the cell's RNA splicing process.

    In the studies presented in today's poster, the efficacy of STK-001 was evaluated in a Scn1a-linked mouse model of Dravet syndrome that results in Nav1.1 haploinsufficiency. Seizures and Sudden Unexpected Death in Epilepsy (SUDEP) in these mice occurred spontaneously and were not provoked by temperature changes or other manipulation. Mice were administered a single intracerebroventricular (ICV) injection of STK-001 or placebo at postnatal day 2 or postnatal day 14 and monitored to postnatal day 90. The data showed improvements among mice treated with STK-001 compared to placebo, including:

    • Significant increases in Scn1a mRNA and Nav1.1 protein expression in the brain at day 90;
    • Significant improvements in survival among DS mice treated at postnatal day 2 or postnatal day 14 compared to placebo;
      • 97% (33/34) mice survived to day 90 after treatment on postnatal day 2, compared to 23% (14/62) mice in the placebo-treated group (p<0.0001).
      • 85% (45/53) mice survived to day 90 after treatment at postnatal day 14, compared to 64% (47/74) mice in the placebo-treated group (p<0.005).
    • An increase in the number of DS mice that experienced no seizures following administration of STK-001 at postnatal day 2, as measured by EEG. Between postnatal day 22 and postnatal day 46, 76% (16/21) of DS mice treated with STK-001 were seizure free compared to 48% (10/21) in the placebo-treated group;
    • An 80% reduction in the average number of spontaneous seizures (3 seizures vs 16 seizures) detected between postnatal day 22 and postnatal day 46 in DS mice after treatment with STK-001 compared to placebo (p<0.05).

    "Although we cannot make a direct correlation, these data are encouraging because they suggest a potential for STK-001 to reduce seizure frequency in Dravet syndrome patients. Showing a 50% improvement in the number of DS mice that had no detectable seizures is a significant finding, particularly considering the high seizure burden associated with Dravet syndrome," said Lori Isom, Ph.D., Maurice H. Seevers Professor and Chair of Pharmacology, University of Michigan Medical School. "Across all measures, the data showed a consistent benefit for DS mice treated with STK-001 compared to placebo, making the totality of these data compelling."

    Details of the presentation are as follows:

    Presentation Title: Targeted Augmentation of Nuclear Gene Output (TANGO) of Scn1a Prevents SUDEP in a Mouse Model of Dravet Syndrome

    Session Date & Time: Saturday, December 7, 2019, 12:00 p.m. – 6:00 p.m. ET

    Session Title: Poster Session 1

    Presenter: Lori Isom, Ph.D., Maurice H. Seevers Professor and Chair of Pharmacology, University of Michigan Medical School

    Location: The Baltimore Convention Center, Hall E

    Additional preclinical data will be presented on Sunday, December 8, 2019 showing biodistribution, target engagement, pharmacodynamics, safety and tolerability in non-human primates treated with STK-001:

    Presentation Title: TANGO Oligonucleotides for the Treatment of Dravet Syndrome: Safety, Biodistribution and Pharmacology in the Non-Human Primate

    Session Date & Time: Sunday, December 8, 2019, 10:00 a.m. – 4:00 p.m. ET

    Session Title: Poster Session 2

    Presenter: Anne Christiansen, Ph.D., Associate Director, Neuroscience, Stoke Therapeutics

    Poster Number: 2.195

    Location: The Baltimore Convention Center, Hall E

    The posters for these studies will be made available online upon presentation at the meeting on the Events and Presentations section of Stoke's website at https://www.stoketherapeutics.com/.

    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 U.S. Food and Drug Administration as a potential new treatment for Dravet syndrome. Stoke plans to submit an investigational new drug application (IND) to the U.S. Food and Drug Administration in early 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 cognitive regression or developmental stagnation, ataxia, speech impairment and sleep disturbances. 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, 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: the ability of STK-001 to improve survival and reduce seizure frequency in mice, as well as its biodistribution, target engagement and ability to increase protein expression in non-human primates; 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. 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 our actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company'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, the company's ability to protect intellectual property; and other risks set forth in our filings with the Securities and Exchange Commission, including the risks set forth in our quarterly report on Form 10-Q for the three months ended September 30, 2019. These forward-looking statements speak only as of the date hereof and we specifically disclaim any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise, except as required by law.

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  32. Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today announced that Chief Executive Officer Edward M. Kaye, M.D., will present at the Piper Jaffray 31st Annual Healthcare Conference on Wednesday, December 4, 2019 at 11:50 a.m. ET in New York, NY.

    A live audio webcast of the presentation will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast 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…

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today announced that Chief Executive Officer Edward M. Kaye, M.D., will present at the Piper Jaffray 31st Annual Healthcare Conference on Wednesday, December 4, 2019 at 11:50 a.m. ET in New York, NY.

    A live audio webcast of the presentation will be available on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/. A replay of the webcast 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.

    View Full Article Hide Full Article
  33. -- STK-001 is a potential new disease-modifying medicine for the treatment of Dravet syndrome, a severe and progressive form of genetic epilepsy --

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today announced that it will present new preclinical data on STK-001, a potential new disease-modifying medicine for the treatment of Dravet syndrome, at the American Epilepsy Society (AES) Annual Meeting, taking place December 6-10, 2019 in Baltimore.

    Data will be presented from preclinical studies demonstrating the effects of STK-001, a proprietary antisense oligonucleotide (ASO), in the Scn1a-linked Dravet…

    -- STK-001 is a potential new disease-modifying medicine for the treatment of Dravet syndrome, a severe and progressive form of genetic epilepsy --

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, today announced that it will present new preclinical data on STK-001, a potential new disease-modifying medicine for the treatment of Dravet syndrome, at the American Epilepsy Society (AES) Annual Meeting, taking place December 6-10, 2019 in Baltimore.

    Data will be presented from preclinical studies demonstrating the effects of STK-001, a proprietary antisense oligonucleotide (ASO), in the Scn1a-linked Dravet syndrome mouse model and in non-human primates. New results of EEG recordings used to measure the frequency of seizures in Dravet syndrome mice treated with STK-001 compared to placebo will be presented, as well as data on STK-001 biodistribution, target engagement, pharmacodynamics, safety and tolerability in non-human primates.

    Dravet syndrome is a severe and progressive form of genetic epilepsy that affects approximately 35,000 people in the United States, Canada, Japan, Germany, France and the United Kingdom. Approximately 85% of Dravet syndrome cases are caused by spontaneous, heterozygous loss of function mutations in the SCN1A gene, resulting in 50% Nav1.1 protein expression.

    "These data support our belief that by restoring the Nav1.1 protein to physiological levels, STK-001 has the potential to provide a gene-specific, disease-modifying therapy for people living with Dravet syndrome," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "We look forward to continuing to advance STK-001 toward the clinic and, in the meantime, to sharing and discussing these important new data with the Dravet community at AES."

    Details on the presentations are as follows:

    Presentation Title: Targeted Augmentation of Nuclear Gene Output (TANGO) of SCN1A Prevents SUDEP in a Mouse Model of Dravet Syndrome

    Session Date & Time: Saturday, December 7, 2019, 12:00 p.m. – 6:00 p.m. ET

    Session Title: Poster Session 1

    Presenter: Lori Isom, Ph.D., Maurice H. Seevers Professor and Chair of Pharmacology, University of Michigan Medical School

    Poster Number: 1.116

    Location: The Baltimore Convention Center, Hall E

    Presentation Title: TANGO Oligonucleotides for the Treatment of Dravet Syndrome: Safety, Biodistribution and Pharmacology in the Non-Human Primate

    Session Date & Time: Sunday, December 8, 2019, 10:00 a.m. – 4:00 p.m. ET

    Session Title: Poster Session 2

    Presenter: Anne Christiansen, Ph.D., Associate Director, Neuroscience, Stoke Therapeutics

    Poster Number: 2.195

    Location: The Baltimore Convention Center, Hall E

    The abstracts for these presentations are now available online on the Events and Presentations section of Stoke's website at https://investor.stoketherapeutics.com/ or through the AES 2019 Annual Meeting mobile application.

    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 for STK-001. STK-001 has been granted orphan drug designation by the U.S. Food and Drug Administration as a potential new treatment for Dravet syndrome.

    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 cognitive regression or developmental stagnation, ataxia, speech impairment and sleep disturbances. 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, 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: Stoke's 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. 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 the company'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, the company's ability to protect intellectual property; and other risks set forth in our filings with the Securities and Exchange Commission, including the risks set forth in our quarterly report on Form 10-Q for the quarter and nine months ended September 30, 2019. These forward-looking statements speak only as of the date hereof and Stoke specifically disclaims any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise, except as required by law.

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  34. 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 third quarter of 2019.

    "Our successful IPO has put us in a strong financial position to rapidly advance STK-001 toward the clinic, invest in research to help us identify the most promising new genetic targets for our TANGO platform and continue to hire the talented team we need to get the job done," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "In August we initiated BUTTERFLY, our observational study of children and adolescents living with Dravet syndrome, which is designed to provide information…

    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 third quarter of 2019.

    "Our successful IPO has put us in a strong financial position to rapidly advance STK-001 toward the clinic, invest in research to help us identify the most promising new genetic targets for our TANGO platform and continue to hire the talented team we need to get the job done," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "In August we initiated BUTTERFLY, our observational study of children and adolescents living with Dravet syndrome, which is designed to provide information about the natural course of the disease – including a range of effects beyond just seizures – that impact patient health and quality of life. We remain on track with our clinical plans for STK-001, our investigational new medicine for the treatment of Dravet syndrome. We have completed the toxicology studies for our investigational new drug application and have made good progress on the protocol for our planned Phase 1/2 study."

    Third Quarter 2019 Business Highlights:

    • On August 6, 2019, we announced that the U.S Food and Drug Administration (FDA) granted orphan drug designation to our lead product candidate, STK-001, an investigational new medicine for Dravet syndrome, a severe and progressive form of epilepsy.
    • On August 20, 2019, we announced first patient enrollment in BUTTERFLY, an observational study of children and adolescents ages 2 to 18 with Dravet syndrome. The two-year study is designed to evaluate non-seizure comorbidities associated with the disease, including cognitive development, behavior, movement, communication skills, seizure frequency and sleep pattern. Data from the study will help inform clinical development plans for STK-001.
    • On September 11, 2019, Stoke announced the company has been added to the Russell 2000® and 3000® indexes effective September 20, 2019. Stoke's addition is based on FTSE Russell's quarterly update.

    Recent Developments:

    • In October 2019, the company expanded to more than 50 employees and announced the appointments of two new members of the senior leadership team: Robin A. Walker, J.D., as Senior Vice President and Chief Legal Officer, and Joan Wood as Head of Human Resources.

    Upcoming Anticipated Milestones:

    • New preclinical data on STK-001 to be presented at the American Epilepsy Society meeting in Baltimore, December 6-8, 2019.
    • Submission of the investigational new drug (IND) application for STK-001 to the FDA in early 2020.
    • Initiation of Phase 1/2 single-ascending dose study of STK-001 in children and adolescents with Dravet syndrome expected in the first half of 2020.
    • Nomination of second product candidate for the treatment of an additional genetic disease expected by the first half of 2020.

    Third Quarter and Year to Date 2019 Results

    • Net loss for the third quarter of 2019 was $8.6 million, compared to net loss of $3.3 million for the same period in 2018. Net loss for the nine months ended September 30, 2019 was $22.2 million, compared to net loss of $8.2 million for the same period in 2018.
    • Research and development expenses for the third quarter of 2019 was $6.5 million, compared to $2.2 million for the same period in 2018. Research and development expense for the nine months ended September 30, 2019 was $16.7 million, compared to $5.4 million for the same period in 2018.
    • General and administrative expenses for the three months ended September 30, 2019 were $3.3 million, compared to $1.1 million for the same period in 2018. General and administrative expenses for the nine months ended September 30, 2019 were $7.9 million, compared to $2.9 million for the same period in 2018.
    • The increases in expenses for the 2019 periods over the same periods in 2018 primarily relate to increases in costs associated with personnel costs, third party contracts, consulting, facilities and other costs associated with development activities for STK-001, research on additional therapeutics and growing a public corporation.
    • As of September 30, 2019, Stoke had approximately $233.2 million in cash, cash equivalents and restricted cash, which is anticipated to fund operations into 2023.

    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 second quarter results; Stoke's expectation about timing and execution of anticipated milestones, including IND submission; the planned initiation of Stoke's BUTTERFLY study and subsequent Phase 1/2 trial, and Stoke's 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. 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 the company'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, the company's ability to protect intellectual property; and other risks set forth in our filings with the Securities and Exchange Commission, including the risks set forth in our quarterly report on Form 10-Q for the quarter and nine months ended September 30, 2019. These forward-looking statements speak only as of the date hereof and Stoke specifically disclaims any obligation to update these forward-looking statements or reasons why actual results might differ, whether as a result of new information, future events or otherwise, except as required by law.

    Stoke Therapeutics, the Stoke logo and all product names are trademarks of Stoke Therapeutics, Inc.

    Financial Tables Follow

    Stoke Therapeutics, Inc.

    Condensed consolidated balance sheets

    (in thousands, except share and per share amounts)

    (unaudited)

     

     

     

     

     

     

     

     

     

     

     

    September 30,

     

     

    December 31,

     

     

     

    2019

     

     

    2018

     

    Assets

     

     

     

     

     

     

     

     

    Current assets:

     

     

     

     

     

     

     

     

    Cash and cash equivalents

     

    $

    233,049

     

     

    $

    105,399

     

    Prepaid expenses and other current assets

     

     

    3,255

     

     

     

    548