1. – Results show that single doses of STK-001 up to 30mg and multiple doses of 20mg were well-tolerated with no safety concerns related to the study drug –

    – A trend toward a reduction in convulsive seizure frequency was observed among patients treated with single doses of STK-001 –

    – >95% of patients anticipated to achieve pharmacologically active levels of STK-001 with three monthly doses of 30mg –

    Complementary Phase 1/2a studies ongoing; First patients dosed in the 30mg multiple ascending dose (MAD) cohorts of MONARCH in the U.S. and ADMIRAL in the UK –

    FDA will allow the evaluation of an additional higher dose level (45mg) in the MONARCH study –

    Management will host a webinar and conference call for analysts and investors at 8:30

    – Results show that single doses of STK-001 up to 30mg and multiple doses of 20mg were well-tolerated with no safety concerns related to the study drug –

    – A trend toward a reduction in convulsive seizure frequency was observed among patients treated with single doses of STK-001 –

    – >95% of patients anticipated to achieve pharmacologically active levels of STK-001 with three monthly doses of 30mg –

    Complementary Phase 1/2a studies ongoing; First patients dosed in the 30mg multiple ascending dose (MAD) cohorts of MONARCH in the U.S. and ADMIRAL in the UK –

    FDA will allow the evaluation of an additional higher dose level (45mg) in the MONARCH study –

    Management will host a webinar and conference call for analysts and investors at 8:30 a.m. Eastern Time today

    Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced positive safety, pharmacokinetic (PK) and cerebrospinal fluid (CSF) exposure data from a planned interim analysis of the multi-center, open-label Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome. STK-001 is an investigational new medicine for the treatment of Dravet syndrome. As part of today's announcement, the company is providing several updates on the clinical progress of STK-001. Management will host a webinar and conference call today at 8:30 a.m. Eastern Time.

    Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures, beginning within the first year of life. Complications of the disease often contribute to poor quality of life for patients and their caregivers. 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.

    "Dravet syndrome is a devastating disease that is difficult to treat. Despite available anti-seizure medicines, seizures are not adequately controlled for more than 90% of patients, contributing to poor quality of life," said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. "The initial positive safety data from MONARCH are highly encouraging and we now have greater clarity on the dose levels that are likely to be pharmacologically active in patients. Although based on an open-label study of a small number of patients, we saw an early trend toward a reduction in convulsive seizure frequency, which is remarkable considering the amount of concomitant medicines used and the relatively low single dose levels of STK-001 evaluated. We look forward to continuing our two ongoing studies as we work to identify a dose level that has the potential to maximize efficacy while minimizing treatment frequency for patients with Dravet syndrome."

    Study Design

    The MONARCH study is a U.S., multi-center, Phase 1/2a open-label study of children and adolescents ages 2 to 18 who have an established diagnosis of Dravet syndrome. The study is designed to evaluate single ascending doses (SAD) and multiple ascending doses (MAD) of STK-001. The primary objectives for the study are to assess the safety and tolerability of STK-001, as well as to characterize blood PK and CSF exposure levels. A secondary objective is to assess the efficacy of STK-001 as measured by the percentage change from baseline in convulsive seizure frequency over a 12-week treatment period.

    The interim analysis is based on data from 21 patients who were treated in the single 10mg (n=5), 20mg (n=4), or 30mg (n=6) dose cohorts of STK-001 and who were followed for at least three months after their last dose. Also included in this analysis were six patients from the 20mg MAD dose cohort, most of whom had received three monthly doses of STK-001.

    Despite concomitant use of multiple anti-seizure medicines, patients enrolled in MONARCH had a high seizure burden. Patients had a median of 17 convulsive seizures during the 4-week screening period leading up to their first dose of STK-001. More than 85% (18/21) of patients were taking three or more concomitant anti-seizure medicines as maintenance therapy and 67% (14/21) were taking four or more concomitant medicines. The most commonly used anti-seizure medicines were clobazam (13/21, 62%) and fenfluramine (10/21, 48%).

    Key findings from the MONARCH study interim analysis include:

    • Single doses of STK-001 up to 30mg and multiple doses of STK-001 at 20mg were found to be well-tolerated with no safety concerns related to the study drug.
    • The most common treatment-emergent adverse events (TEAE) were headache, irritability, vomiting, seizure, and back pain.
    • 3/21 (14%) of patients experienced a TEAE that was related to study drug. None of these patients were in the two higher dose groups (30mg single dose or 20mg multiple dose).
    • 4/21 (19%) of patients had a treatment-emergent serious adverse event (SAE). There were no SAEs related to study drug.
    • A dose proportional increase in study drug exposure was observed in plasma PK.
    • CSF exposure was measurable up to six months following a single intrathecal (IT) dose, indicating sustained exposure of STK-001 in the brain. A dose-proportional increase in CSF concentration was observed from 20mg to 30mg.
    • Preliminary analyses of daily seizure diaries suggested a trend toward a reduction in median percent change from baseline in convulsive seizure frequency among patients treated with single doses of STK-001. This trend was more evident in the 2 to 12 year-old age group.
    • Based on data available from 11 patients in the SAD cohorts (10mg, 20mg, 30mg), 8 out of 11 patients demonstrated a reduction in convulsive seizure frequency.

    No patients discontinued study treatment and, at the time of the analysis, all patients who completed dosing in the SAD portion of MONARCH continued treatment in SWALLOWTAIL, an open-label extension study designed to evaluate the long-term safety and tolerability of repeat doses of STK-001.

    PK Model Findings

    • A population pharmacokinetic model for intrathecal STK-001 was developed using non-human primate data and was scaled and adjusted using clinical data to predict STK-001 concentrations in plasma, CSF and brain in pediatric patients with Dravet syndrome.
    • Data from the MONARCH study showed that STK-001 levels in plasma and CSF in patients treated with STK-001 correlated very well with model predictions, indicating that plasma and CSF levels observed in patients are good predictors of STK-001 brain levels in patients.
    • Estimated pharmacologically active levels of STK-001 in the brain are conservatively defined as those that could result in a 2-fold increase in Nav1.1, which is anticipated to restore normal physiologic levels in a patient's brain cells.
    • Modeling of early clinical data suggests that 95% of patients are predicted to have pharmacologically active STK-001 brain levels following three doses of 30mg administered one month apart. Half of all patients are anticipated to remain at therapeutic levels for approximately three months after the last dose.

    Clinical Progress Updates

    • In September, the first patient was dosed in the 30mg MAD portion of the ongoing Phase 1/2a MONARCH study.
    • In September, the first patient was dosed with STK-001 (30mg) in the Phase 1/2a ADMIRAL study of STK-001 for the treatment of children and adolescents with Dravet syndrome in the United Kingdom. This study complements the Company's ongoing MONARCH study by evaluating multiple doses of STK-001 up to 70mg.
    • 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 (45mg) to the SAD and MAD portions of the MONARCH study.
    • The Company expects to provide greater detail on data from the MONARCH study at the American Epilepsy Society annual meeting December 3-7, 2021 in Chicago.
    • The Company expects to share clinical data from multiple doses of 30mg in the second half of 2022.

    Stoke Webinar and Conference Call for Analysts and Investors

    Stoke will host a webinar and conference call for analysts and investors at 8:30 a.m. Eastern Time on Tuesday, September 21, 2021, to present the interim data from the Phase 1/2a MONARCH study of STK-001. To participate in the call, please dial (866) 996-7187, or (270) 215-9492 for international callers and provide conference ID number 1568363. The webinar will be broadcast live on the Investors & Media section of Stoke's website at https://investor.stoketherapeutics.com/ and can be accessed by following this Link. An archived replay of the webinar will be available for at least 30 days following the event.

    About Dravet Syndrome

    Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures, beginning within the first year of life. Dravet syndrome is difficult to treat and has a poor long-term prognosis. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. The effects of the disease go beyond seizures and often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. There are no approved disease modifying therapies for people living with Dravet syndrome. One out of 16,000 babies are born with Dravet syndrome, which is not concentrated in a particular geographic area or ethnic group.

    About STK-001

    STK-001 is an investigational new medicine for the treatment of Dravet syndrome that is currently being evaluated in ongoing clinical trials. Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. STK-001 is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities. 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 MONARCH Study (United States)

    The MONARCH study is a Phase 1/2a open-label study of children and adolescents ages 2 to 18 who have an established diagnosis of Dravet syndrome and have evidence of a 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 blood 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. 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 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 blood 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. Additional information about the ADMIRAL study can be found at https://www.admiralstudy.com.

    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 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: the ability of STK-101 to treat Dravet syndrome and reduce seizures, the timing and expected progress of clinical trials, data readouts and presentations, and the timing or receipt of regulatory approval. Statements including words such as "plan," "will," "continue," "expect," or "ongoing" and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they 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 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 Company's ability to advance its product candidates, obtain regulatory approval of and ultimately commercialize its product candidates, the timing and results of preclinical and clinical trials, the company's ability to fund development activities and achieve development goals, the company's ability to protect intellectual property and other risks and uncertainties described under the heading "Risk Factors" in documents the company files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the Company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.

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  2. – Dosing up to 30mg complete in the single ascending dose (SAD) portion in the Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome; Enrollment and dosing in the multiple ascending dose (MAD) portion ongoing –

    – Third quarter data readout will include 3-month safety, pharmacokinetic (PK), and cerebrospinal fluid (CSF) drug exposure data from patients in the SAD portion of MONARCH, along with modeling of predicted brain exposure –

    – Company on-track to initiate an additional Phase 1/2a study (ADMIRAL) of STK-001 up to 70mg in patients with Dravet syndrome across multiple sites in the United Kingdom –

    As of June 30, 2021, Company has $251.4 million in cash, cash equivalents, marketable securities, and restricted

    – Dosing up to 30mg complete in the single ascending dose (SAD) portion in the Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome; Enrollment and dosing in the multiple ascending dose (MAD) portion ongoing –

    – Third quarter data readout will include 3-month safety, pharmacokinetic (PK), and cerebrospinal fluid (CSF) drug exposure data from patients in the SAD portion of MONARCH, along with modeling of predicted brain exposure –

    – Company on-track to initiate an additional Phase 1/2a study (ADMIRAL) of STK-001 up to 70mg in patients with Dravet syndrome across multiple sites in the United Kingdom –

    As of June 30, 2021, Company has $251.4 million in cash, cash equivalents, marketable securities, 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 second quarter of 2021 and provided business updates.

    "During the first half of 2021, the Stoke team laid the groundwork for important clinical milestones in the second half of this year," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "With dosing now complete up to 30mg in the single ascending dose portion of MONARCH, we are on track to report preliminary safety, PK and CSF data from a planned interim analysis of this portion of the study in the third quarter. By comparing these first in-human results with our modeling data, we expect to get insight into the predicted brain exposure and projected therapeutic dose range of STK-001 in patients with Dravet syndrome. In the coming months, we also expect to treat the first patient with STK-001 in our ADMIRAL study in the United Kingdom, which together with MONARCH, is anticipated to provide a robust early understanding of STK-001 to inform future development plans."

    Dr. Kaye continued, "We continue to advance our pipeline of TANGO antisense oligonucleotides (ASO) and remain on-track to identify a clinical candidate to address the underlying cause of autosomal dominant optic atrophy (ADOA), a severe and progressive genetic disease that causes irreversible vision loss and for which there are no treatment options, by year end."

    Second Quarter 2021 Business Highlights and Recent Developments

    • Dosing is complete in the 10mg, 20mg, and 30mg SAD portion of the Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome.
    • Enrollment and dosing in the MAD portion of MONARCH is ongoing at the 20mg dose level.
    • Dosing of patients is ongoing 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 May 2021, the Company presented new preclinical efficacy data for TANGO ASO at The Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting and The American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. The new data, which demonstrated TANGO ASO-mediated OPA1 protein upregulation and improved mitochondrial function in human cells derived from ADOA patients with different OPA1 mutations, supports the Company's work to develop the first potential disease modifying approach for the treatment of ADOA, the most common inherited optic nerve disorder.

    Upcoming Anticipated Milestones

    • Enrollment and dosing in the Phase 1/2a ADMIRAL study of STK-001 for the treatment of children and adolescents ages 2 to up to 18 with Dravet syndrome in the United Kingdom is on-track to begin in the second half of 2021.
    • As part of a planned interim analysis, the Company expects to report modeling data and 3-month safety, PK, and CSF drug exposure data from the SAD portion of the Phase 1/2a MONARCH study of STK-001 in the third quarter of 2021.
    • In the second half of 2021, the Company plans to initiate natural history data collection of people living with ADOA to better understand the natural progression of this disease and to support future clinical development of a TANGO ASO for the treatment of ADOA.
    • Lead optimization on track to identify a clinical candidate for the treatment of ADOA by the end of 2021.
    • The Company remains on track to demonstrate in vivo proof of mechanism and safety for a third TANGO ASO program by the end of 2021.

    Second Quarter 2021 and Year-to-Date Financial Results

    • Net loss for the three months ended June 30, 2021 was $22.0 million, or $0.60 per share, compared to $13.0 million, or $0.39 per share, for the same period in 2020.
    • Research and development expenses for the three months ended June 30, 2021 were $14.1 million, compared to $8.0 million for the same period in 2020.
    • General and administrative expenses for the three months ended June 30, 2021 were $8.0 million, compared to $5.0 million for the same period in 2020.
    • Net loss for the six months ended June 30, 2021 was $38.8 million, or $1.06 per share, compared to $24.0 million, or $0.73 per share, for the same period in 2020.
    • Research and development expenses for the six months ended June 30, 2021 were $24.0 million, compared to $15.2 million for the same period in 2020.
    • General and administrative expenses for the six months ended June 30, 2021 were $14.8 million, compared to $9.6 million for the same period in 2020.
    • The increase in expenses for the three and six month periods in 2021 over the same periods in 2020 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, 2021, Stoke had approximately $251.4 million in cash, cash equivalents, marketable securities 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 MONARCH Study (United States)

    The MONARCH study is a Phase 1/2a open-label study of children and adolescents ages 2 to 18 who have an established diagnosis of Dravet syndrome and have evidence of a 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. 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 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. Additional information about the ADMIRAL study can be found at https://www.admiralstudy.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 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. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. There are no approved disease modifying therapies for people living with Dravet syndrome. 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 disease, 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. 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, most of which lead to a haploinsufficiency resulting in 50% OPA1 protein expression and disease manifestation.

    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 quarter-end results and cash runway; timing and expected progress of clinical trials, data readouts and presentations; preclinical data and study results regarding OPA1 and STK-001; our future operating results, financial position and liquidity; 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 and our ability to advance OPA1 as our next preclinical target; 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, including any potential clinical candidate for OPA1; 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 the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations and financial condition; 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 in the United States and abroad; 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,

    2021

    2020

    Assets
    Current assets:
    Cash and cash equivalents

    $

    169,070

     

    $

    287,308

     

    Marketable Securities

     

    82,156

     

     

     

    Prepaid expenses and other current assets

     

    8,612

     

     

    6,435

     

    Restricted cash - short-term

     

    147

     

     

     

    Deferred financing costs

     

    117

     

     

    181

     

    Interest receivable

     

    131

     

     

    6

     

    Total current assets

    $

    260,233

     

    $

    293,930

     

    Restricted cash

     

    75

     

     

    205

     

    Operating lease right-of-use assets

     

    1,258

     

     

    1,115

     

    Property and equipment, net

     

    2,884

     

     

    2,675

     

    Total assets

    $

    264,450

     

    $

    297,925

     

    Liabilities and stockholders' equity
    Current liabilities:
    Accounts payable

    $

    963

     

    $

    1,495

     

    Accrued and other current liabilities

     

    7,234

     

     

    9,930

     

    Total current liabilities

    $

    8,197

     

    $

    11,425

     

    Long term liabilities

     

    1,184

     

     

    422

     

    Total liabilities

    $

    9,381

     

    $

    11,847

     

    Commitments and contingencies (Note 6)
    Stockholders' equity
    Common stock, par value of $0.0001 per share; 300,000,000 shares authorized, 36,722,669 and 36,577,149 shares issued and outstanding as of June 30, 2021 and December 31, 2020, respectively

     

    4

     

     

    4

     

    Additional paid-in capital

     

    404,145

     

     

    396,352

     

    Accumulated other comprehensive loss

     

    (42

    )

     

     

    Accumulated deficit

     

    (149,038

    )

     

    (110,278

    )

    Total stockholders' equity

    $

    255,069

     

    $

    286,078

     

    Total liabilities and stockholders' equity

    $

    264,450

     

    $

    297,925

     

    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,

    2021

    2020

    2021

    2020

    Revenue

    $

     

    $

     

    $

     

    $

     

    Operating expenses:
    Research and development

     

    14,095

     

     

    7,968

     

     

    24,008

     

     

    15,183

     

    General and administrative

     

    7,934

     

     

    5,044

     

     

    14,848

     

     

    9,563

     

    Total operating expenses

     

    22,029

     

     

    13,012

     

     

    38,856

     

     

    24,746

     

    Loss from operations

     

    (22,029

    )

     

    (13,012

    )

     

    (38,856

    )

     

    (24,746

    )

    Other income:
    Interest income (expense), net

     

    34

     

     

    39

     

     

    40

     

     

    704

     

    Other income (expense), net

     

    28

     

     

    14

     

     

    56

     

     

    44

     

    Total other income

     

    62

     

     

    53

     

     

    96

     

     

    748

     

    Net loss

    $

    (21,967

    )

    $

    (12,959

    )

    $

    (38,760

    )

    $

    (23,998

    )

    Net loss per share, basic and diluted

    $

    (0.60

    )

    $

    (0.39

    )

    $

    (1.06

    )

    $

    (0.73

    )

    Weighted-average common shares outstanding, basic and diluted

     

    36,708,188

     

     

    33,054,656

     

     

    36,675,876

     

     

    32,976,026

     

    Comprehensive loss:
    Net loss

    $

    (21,967

    )

    $

    (12,959

    )

    $

    (38,760

    )

    $

    (23,998

    )

    Other comprehensive loss:
    Unrealized loss on marketable securities

     

    (42

    )

     

     

     

    (42

    )

     

     

    Total other comprehensive loss

    $

    (42

    )

    $

     

    $

    (42

    )

    $

     

    Comprehensive loss

    $

    (21,925

    )

    $

    (12,959

    )

    $

    (38,718

    )

    $

    (23,998

    )

     

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  3. 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 in August.

    2021 Wedbush PacGrow Healthcare Virtual Conference

    Date: Wednesday, August 11, 2021

    Time: 11:30 a.m. ET

    Canaccord Genuity 41st Annual Growth Conference

    Date: Thursday, August 12, 2021

    Time: 12:30 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 webcast will be available for 30 days following the…

    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 in August.

    2021 Wedbush PacGrow Healthcare Virtual Conference

    Date: Wednesday, August 11, 2021

    Time: 11:30 a.m. ET

    Canaccord Genuity 41st Annual Growth Conference

    Date: Thursday, August 12, 2021

    Time: 12:30 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 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.

    View Full Article Hide Full Article
  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 UBS Global Healthcare Virtual Conference on Monday, May 24, 2021, at 5:00 p.m. ET.

    A live audio webcast of the presentation, which will be conducted in fireside chat format, 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 chat.

    About Stoke Therapeutics

    Stoke Therapeutics (NASDAQ:STOK) is a biotechnology company dedicated to addressing…

    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 UBS Global Healthcare Virtual Conference on Monday, May 24, 2021, at 5:00 p.m. ET.

    A live audio webcast of the presentation, which will be conducted in fireside chat format, 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 chat.

    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.

    View Full Article Hide Full Article
  5. – Enrollment complete in single ascending dose (SAD) portion (10mg, 20mg, 30mg) of Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome; Interim analysis planned for the third quarter –

    – Enrollment and dosing ongoing in the 20mg multiple ascending dose (MAD) portion of MONARCH –

    – Company on-track to identify a clinical candidate for the treatment of autosomal dominant optic atrophy by year end –

    As of March 31, 2021, Company has $267.7 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…

    – Enrollment complete in single ascending dose (SAD) portion (10mg, 20mg, 30mg) of Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome; Interim analysis planned for the third quarter –

    – Enrollment and dosing ongoing in the 20mg multiple ascending dose (MAD) portion of MONARCH –

    – Company on-track to identify a clinical candidate for the treatment of autosomal dominant optic atrophy by year end –

    As of March 31, 2021, Company has $267.7 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 first quarter of 2021 and provided business updates.

    "The momentum we are seeing with STK-001 in the clinic is driven by high levels of interest from the Dravet community and our shared sense of urgency to advance a potential disease-modifying approach for the treatment of this devastating disease. The single dose portion of our Phase 1/2a MONARCH study in the U.S. is now fully enrolled up to the 30mg dose level and based on our progress, we now anticipate our first data readout from this single dose portion of MONARCH in the third quarter. We are also well underway with preparation to initiate a complementary study of STK-001 in the United Kingdom in the coming months. Together, we believe these studies will provide meaningful data to inform future development of STK-001," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "In addition to clinical progress, we continue to advance our pipeline. We recently presented new preclinical data that provide additional confidence in our ability to upregulate OPA1 protein expression to treat the underlying cause of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. We are on track with lead optimization efforts for the ADOA program and expect to demonstrate proof of mechanism for a third preclinical target using our TANGO approach by year end."

    First Quarter 2021 Business Highlights and Recent Developments

    • The Company announced today, the completion of patient enrollment in the 10mg, 20mg, and 30mg single ascending dose (SAD) portion of the Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome.
    • Enrollment and dosing in the multiple ascending dose (MAD) portion of MONARCH is ongoing.
    • On March 30, 2021, the Company 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 in the United Kingdom. The planned ADMIRAL study complements the ongoing MONARCH study by evaluating multiple doses of up to 70mg of STK-001 and represents a first step in the global clinical development of STK-001.
    • Dosing of patients is ongoing 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.
    • On May 4, 2021, the Company presented new preclinical efficacy data for TANGO antisense oligonucleotides (ASO) at The Association for Research in Vision and Ophthalmology (ARVO). The new data demonstrated, for the first time, both OPA1 protein upregulation and improved mitochondrial function in human cells derived from autosomal dominant optic atrophy (ADOA) patients with different OPA1 mutations. OPA1 protein deficiency is the primary cause of ADOA, the most common inherited optic nerve disorder.

    Upcoming Anticipated Milestones

    • Enrollment and dosing in the Phase 1/2a study (ADMIRAL) of STK-001 for the treatment of Dravet syndrome across multiple sites in the United Kingdom are expected to begin in the second half of 2021.
    • As part of a planned interim analysis, the Company expects to report preliminary safety, pharmacokinetic, and cerebrospinal fluid (CSF) drug exposure data from the SAD portion of the Phase 1/2a MONARCH study of STK-001 in the third quarter of 2021.
    • A presentation of preclinical data supporting the Company's approach to treating ADOA with a TANGO ASO will be presented on May 11, 2021 during the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.
    • In the second half of 2021, the Company plans to initiate natural history data collection of people living with ADOA to better understand the natural progression of this disease and to support future clinical development of a TANGO ASO for the treatment of ADOA.
    • The Company remains on track to identify a clinical candidate for the treatment of ADOA by the end of 2021.
    • The Company expects to demonstrate in vivo proof of mechanism and safety for a third TANGO ASO program by the end of 2021.

    First Quarter 2021 Financial Results

    • Net loss for the quarter ended March 31, 2021 was $16.8 million, or $0.46 per share compared to $11.0 million or $0.34 per share for the same period in 2020.
    • Research and development expenses for the quarter ended March 31, 2021 were $9.9 million, compared to $7.2 million for the same period in 2020.
    • General and administrative expenses for the quarter ended March 31, 2021 were $6.9 million, compared to $4.5 million for the same period in 2020.
    • The increase in expenses for 2021 over the same periods in 2020 primarily relate to increases in costs associated with personnel, third party contracts, consulting, facilities, and other expenses associated with development activities for STK-001 and research on additional therapeutics and growing a public corporation.
    • As of March 31, 2021, Stoke had approximately $267.7 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 MONARCH Study (United States)

    The MONARCH study is a Phase 1/2a open-label study of children and adolescents ages 2 to 18 who have an established diagnosis of Dravet syndrome and have evidence of a 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/.

    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 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 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. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. There are no approved disease modifying therapies for people living with Dravet syndrome. 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 disease, 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. 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, most of which lead to a haploinsufficiency resulting in 50% OPA1 protein expression and disease manifestation.

    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 quarter-end results and cash runway; preclinical data and study results regarding OPA1 and STK-001; 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; 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 and our ability to advance OPA1 as our next preclinical target; 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 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 in the United States and abroad; 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,

     

     

     

    2021

     

     

    2020

     

    Assets

     

     

     

     

     

     

     

     

    Current assets:

     

     

     

     

     

     

     

     

    Cash and cash equivalents

     

    $

    267,514

     

     

    $

    287,308

     

    Prepaid expenses and other current assets

     

     

    7,511

     

     

     

    6,435

     

    Restricted cash - short-term

     

     

    147

     

     

     

     

    Deferred financing costs

     

     

    117

     

     

     

    181

     

    Interest receivable

     

     

    6

     

     

    6

     

    Total current assets

     

    $

    275,295

     

     

    $

    293,930

     

    Restricted cash

     

     

    58

     

     

    205

     

    Operating lease right-of-use assets

     

     

    844

     

     

     

    1,115

     

    Property and equipment, net

     

     

    2,711

     

     

     

    2,675

     

    Total assets

     

    $

    278,908

     

     

    $

    297,925

     

    Liabilities and stockholders' equity

     

     

     

     

     

     

     

     

    Current liabilities:

     

     

     

     

     

     

     

     

    Accounts payable

     

    $

    1,496

     

     

    $

    1,495

     

    Accrued and other current liabilities

     

     

    4,614

     

     

     

    9,930

     

    Total current liabilities

     

    $

    6,110

     

     

    $

    11,425

     

    Long term liabilities

     

     

    333

     

     

     

    422

     

    Total liabilities

     

    $

    6,443

     

     

    $

    11,847

     

    Commitments and contingencies

     

     

     

     

     

     

     

     

    Stockholders' equity

     

     

     

     

     

     

     

     

    Common stock, par value of $0.0001 per share; 300,000,000 shares authorized, 36,697,808 and 36,577,149 shares issued and outstanding as of March 31, 2021 and December 31, 2020, respectively

     

     

    4

     

     

     

    4

     

    Additional paid-in capital

     

     

    399,532

     

     

     

    396,352

     

    Accumulated deficit

     

     

    (127,071

    )

     

     

    (110,278

    )

    Total stockholders' equity

     

    $

    272,465

     

     

    $

    286,078

     

    Total liabilities and stockholders' equity

     

    $

    278,908

     

     

    $

    297,925

     

    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,

     

     

     

     

    2021

     

     

    2020

     

     

    Revenue

     

    $

     

     

    $

     

     

    Operating expenses:

     

     

     

     

     

     

     

     

     

    Research and development

     

     

    9,913

     

     

     

    7,215

     

     

    General and administrative

     

     

    6,914

     

     

     

    4,520

     

     

    Total operating expenses

     

     

    16,827

     

     

     

    11,735

     

     

    Loss from operations

     

     

    (16,827

    )

     

     

    (11,735

    )

     

    Other income:

     

     

     

     

     

     

     

     

     

    Interest income

     

     

    18

     

     

     

    674

     

     

    Other income (expense), net

     

     

    16

     

     

     

    22

     

     

    Total other income

     

     

    34

     

     

     

    696

     

     

    Net loss and comprehensive loss

     

    $

    (16,793

    )

     

    $

    (11,039

    )

     

    Net loss per share, basic and diluted

     

    $

    (0.46

    )

     

    $

    (0.34

    )

     

    Weighted-average common shares outstanding, basic and diluted

     

     

    36,643,205

     

     

     

    32,897,395

     

     

     

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  6. Results presented at ARVO annual meeting confirm earlier preclinical findings and support the Company's work to discover and develop the first potential disease modifying approach for the treatment of ADOA –

    ADOA is the most common inherited optic nerve disorder –

    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 new preclinical data demonstrating in-vitro protein upregulation and improved mitochondrial function in OPA1 protein-deficient fibroblasts derived from patients with ADOA. OPA1 protein deficiency is the primary cause of ADOA and reduced OPA1 levels are associated with impaired…

    Results presented at ARVO annual meeting confirm earlier preclinical findings and support the Company's work to discover and develop the first potential disease modifying approach for the treatment of ADOA –

    ADOA is the most common inherited optic nerve disorder –

    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 new preclinical data demonstrating in-vitro protein upregulation and improved mitochondrial function in OPA1 protein-deficient fibroblasts derived from patients with ADOA. OPA1 protein deficiency is the primary cause of ADOA and reduced OPA1 levels are associated with impaired mitochondrial function. The data will be presented today at The Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting and at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting on Tuesday, May 11, 2021 from 8:00 AM – 10:00 AM Eastern.

    "These findings are important because they offer the first evidence from patient cells that our TANGO approach can address the underlying cause of ADOA by upregulating OPA1 protein production and increasing mitochondrial function," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. "ADOA represents a strong fit for Stoke's science and our strategy: targeting severe genetic diseases that are caused by protein deficiencies and that have limited, if any treatment options. A disease-modifying approach would represent a significant advancement in the treatment of this disease."

    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, most of which lead to a haploinsufficiency resulting in 50% OPA1 protein expression and disease manifestation.

    "By demonstrating an improvement in the mitochondrial function of patient cells across different OPA1 mutations, the data suggest that ASO mediated increase in OPA1 could be sufficient to slow or reduce disease progression in a mutation-independent manner," said Gene Liau, Ph.D., Chief Scientific Officer of Stoke Therapeutics.

    The data presented today provide in-vitro proof-of-concept for TANGO ASOs in ADOA patient fibroblasts. Highlights from today's presentation include new data that demonstrate ADOA patient fibroblast cell lines treated with TANGO ASOs exhibit:

    • Reduced non-productive exon inclusion and increased total OPA1 mRNA expression in three patient fibroblast cell lines with different mutations;
    • Increased expression of multiple OPA1 protein isoforms by ~35% to 47%; and
    • A dose-dependent improvement in mitochondrial bioenergetics.

    Details of today's presentation are as follows:

    Presentation Title: Antisense oligonucleotide mediated increase in OPA1 improves mitochondrial function in fibroblasts derived from patients with autosomal dominant optic atrophy (ADOA)

    Session Date & Time: Tuesday, May 4, 2021; 2:15 p.m. – 3:45 p.m. E.T.

    Session Title: Gene therapy in ocular diseases

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

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

    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 disease, 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. 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, most of which lead to a haploinsufficiency resulting in 50% OPA1 protein expression and disease manifestation.

    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 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: preclinical data and study results regarding OPA1; our ability to develop TANGO ASOs to treat the underlying causes of ADOA and increase mitochondrial function; our ability to advance OPA1 as our next preclinical target; 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 any potential clinical candidate for OPA1; the timing and results of preclinical studies and clinical trials; the risk that positive results in a preclinical trial may not be replicated in subsequent trials or success in early stage preclinical trials may not be predictive of results in later stage trials; 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; 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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  7. – 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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  8. 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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  9. – 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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  10. 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.

    View Full Article Hide Full Article
  11. 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.

    View Full Article Hide Full Article
  12. 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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  13. – 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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  14. 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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  15. 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 prospectus-eq_fi@jpmchase.com; 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 PostSaleManualRequests@broadridge.com; 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 usa.prospectus@credit-suisse.com. 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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  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 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 prospectus-eq_fi@jpmchase.com; 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 PostSaleManualRequests@broadridge.com; 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 usa.prospectus@credit-suisse.com. 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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  17. 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 prospectus-eq_fi@jpmchase.com; 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 PostSaleManualRequests@broadridge.com; 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 usa.prospectus@credit-suisse.com. 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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  18. - 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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  19. 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.

    View Full Article Hide Full Article
  20. 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.

    View Full Article Hide Full Article
  21. - 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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  22. 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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  23. 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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  24. 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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  25. – 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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  26. 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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  27. 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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  28. 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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  29. – 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 ap