Stoke Therapeutics Inc

Previous wording

Our first compound, STK-001, is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Dravet syndrome is characterized by frequent, prolonged and refractory seizures beginning 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 it. Dravet syndrome is one of many diseases caused by a haploinsufficiency, in which a loss of approximately 50% of normal protein levels leads to disease. We have announced end of study data from two Phase 1/2a open-label studies of STK-001, MONARCH in the United States and ADMIRAL in the United Kingdom. We also have two ongoing Open Label Extension (“OLE”) studies of STK-001 for children and adolescents with Dravet syndrome, SWALLOWTAIL in the U.S. and LONGWING in the United Kingdom. Patients who participated in the MONARCH study in the United States or the ADMIRAL study in the United Kingdom and met study entry criteria were eligible to continue treatment in SWALLOWTAIL or LONGWING, respectively, both of which are designed to evaluate the long-term safety and tolerability of repeat doses of STK-001.

Current wording

Our first compound, zorevunersen (STK-001), is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Dravet syndrome is characterized by frequent, prolonged and refractory seizures beginning 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 it. Dravet syndrome is one of many diseases caused by a haploinsufficiency, in which a loss of approximately 50% of normal protein levels leads to disease. We have completed two Phase 1/2a open-label studies of zorevunersen, MONARCH in the United States and ADMIRAL in the United Kingdom. We also have two ongoing Open Label Extension (“OLE”) studies of zorevunersen for children and adolescents with Dravet syndrome, SWALLOWTAIL in the U.S. and LONGWING in the United Kingdom. Patients who participated in the MONARCH study in the United States or the ADMIRAL study in the United Kingdom and met study entry criteria were eligible to continue treatment in SWALLOWTAIL or LONGWING, respectively, both of which are designed to evaluate the long-term safety and tolerability of repeat doses of zorevunersen.

Previous wording

We are also pursuing treatment for a second haploinsufficient disease, autosomal dominant optic atrophy (“ADOA”), the most common inherited optic nerve disorder. STK-002 is our lead clinical candidate for the treatment of ADOA. STK-002 is designed to upregulate OPA1 protein expression by leveraging the non-mutant (wild-type) copy of the OPA1 gene to restore OPA1 protein expression with the aim to stop or slow vision loss in patients with ADOA. We have received authorization in the United Kingdom to proceed with a Phase 1 open-label study (OSPREY) of children and adults ages 6 to 55 who have an established diagnosis of ADOA and have evidence of a genetic mutation in the OPA1gene. We expect the OSPREY study to start in 2024.

Current wording

We are also pursuing treatment for a second haploinsufficient disease, autosomal dominant optic atrophy (“ADOA”), the most common inherited optic nerve disorder. STK-002 is our lead clinical candidate for the treatment of ADOA. STK-002 is designed to upregulate OPA1 protein expression by leveraging the non-mutant (wild-type) copy of the OPA1 gene to restore OPA1 protein expression with the aim to stop or slow vision loss in patients with ADOA. We have received authorization in the United Kingdom to proceed with a Phase 1 open-label study (OSPREY) of children and adults ages 6 to 55 who have an established diagnosis of ADOA and have evidence of a genetic mutation in the OPA1 gene. We expect the OSPREY study to start later this year.

Previous wording

In May 2022, we filed a universal Shelf Registration statement on Form S-3 (the “Registration Statement”) with the SEC. The Registration Statement was declared effective by the SEC on May 31, 2022, and contains two prospectuses: a base prospectus, which covers the offering, issuance and sale by us of up to a maximum aggregate offering price of $400.0 million of our common stock, preferred stock, debt securities, warrants to purchase common stock, preferred stock or debt securities, subscription rights to purchase common stock, preferred stock or debt securities and/or units consisting of some or all of these securities; and a sales agreement prospectus covering the offering, issuance and sale by us of up to a maximum aggregate offering price of $150.0 million of common stock that may be issued and sold under a Controlled Equity Offering Sales Agreement (“Sales Agreement”). The specific terms of any securities to be offered pursuant to the base prospectus will be specified in a prospectus supplement to the base prospectus. The $150.0 million of common stock that may be offered, issued and sold under the sales agreement prospectus is included in the $400.0 million of securities that may be offered, issued and sold by us under the base prospectus. As of March 31, 2024, we had issued approximately 6.5 million shares of common stock pursuant to the Sales Agreement for net proceeds of $53.4 million. We may terminate this at-the-market program at any time, pursuant to its terms. On April 2, 2024, we completed an underwritten public offering of our common stock and issued and sold 5,555,557 shares of common stock at a public offering price of $13.50 per share and issued pre-funded warrants to purchase 3,703,730 shares of common stock at a public offering price of $13.499 per share subject to an exercise price equal to $0.0001. The common stock and warrants sold resulted in net proceeds of $120.3 million after deducting underwriting discounts and commissions.

Current wording

In May 2022, we filed a universal Shelf Registration statement on Form S-3 (the “Registration Statement”) with the SEC. The Registration Statement was declared effective by the SEC on May 31, 2022, and contains two prospectuses: a base prospectus, which covers the offering, issuance and sale by us of up to a maximum aggregate offering price of $400.0 million of our common stock, preferred stock, debt securities, warrants to purchase common stock, preferred stock or debt securities, subscription rights to purchase common stock, preferred stock or debt securities and/or units consisting of some or all of these securities; and a sales agreement prospectus covering the offering, issuance and sale by us of up to a maximum aggregate offering price of $150.0 million of common stock that may be issued and sold under a Controlled Equity Offering Sales Agreement (“Sales Agreement”). The specific terms of any securities to be offered pursuant to the base prospectus will be specified in a prospectus supplement to the base prospectus. The $150.0 million of common stock that may be offered, issued and sold under the sales agreement prospectus is included in the $400.0 million of securities that may be offered, issued and sold by us under the base prospectus.

Previous wording

As of March 31, 2024 and December 31, 2023 we had $178.6 million and $201.4 million, respectively, in cash, cash equivalents and marketable securities.

Current wording

As of June 30, 2024 and December 31, 2023 we had $282.0 million and $201.4 million, respectively, in cash, cash equivalents and marketable securities.

Previous wording

Since inception, we have had operating losses, the majority of which are attributable to research and development activities. Our net losses were $26.4 million and $22.5 million for the three months ended March 31, 2024 and 2023, respectively. As of March 31, 2024, we had an accumulated deficit of $428.2 million.

Current wording

Since inception, we have had operating losses, the majority of which are attributable to research and development activities. Our net losses were $25.7 million and $30.7 million for the three months ended June 30, 2024 and 2023, respectively and $52.1 million and $53.2 million for the six months ended June 30, 2024 and 2023, respectively. As of June 30, 2024, we had an accumulated deficit of $453.9 million.

Previous wording

Based upon our current operating plan, we believe that our existing cash and cash equivalents as of March 31, 2024 together with the $120.3 million of net proceeds from our April 2024 public offering will enable us to fund our operating expenses and capital expenditure requirements at least to the end of 2025. To date, we have not had any products approved for sale and have not generated any product sales. We do not expect to generate any revenues from product sales unless and until we successfully complete development and obtain regulatory approval for one or more of our product candidates, which we expect will take a number of years. If we obtain regulatory approval for any of our product candidates, we expect to incur significant commercialization expenses related to product sales, marketing, manufacturing and distribution. As a result, until such time, if ever, as we can generate substantial product revenue, we expect to finance our cash needs through equity offerings, debt financings or other capital sources, including potentially collaborations, licenses and other similar arrangements. However, we may be unable to raise additional funds or enter into such other arrangements when needed on favorable terms or at all. Any failure to raise capital as and when needed could have a negative impact on our financial condition and on our ability to pursue our business plans and strategies. If we are unable to raise capital, we will need to delay, reduce or terminate planned activities to reduce costs.

Current wording

Based upon our current operating plan, we believe that our existing cash, cash equivalents and marketable securities as of June 30, 2024 will enable us to fund our operating expenses and capital expenditure requirements at least to the end of 2025. To date, we have not had any products approved for sale and have not generated any product sales. We do not expect to generate any revenues from product sales unless and until we successfully complete development and obtain regulatory approval for one or more of our product candidates, which we expect will take a number of years. If we obtain regulatory approval for any of our product candidates, we expect to incur significant commercialization expenses related to product sales, marketing, manufacturing and distribution. As a result, until such time, if ever, as we can generate substantial product revenue, we expect to finance our cash needs through equity offerings, debt financings or other capital sources, including potentially collaborations, licenses and other similar arrangements. However, we may be unable to raise additional funds or enter into such other arrangements when needed on favorable terms or at all. Any failure to raise capital as and when needed could have a negative impact on our financial condition and on our ability to pursue our business plans and strategies. If we are unable to raise capital, we will need to delay, reduce or terminate planned activities to reduce costs.

Previous wording

Patients in the OLE study LONGWING currently receive chronic dosing every four months of 45mg of STK-001, and in March 2024, we announced that the U.S. Food and Drug Administration will allow chronic dosing in OLE study SWALLOWTAIL in the United States to increase to 45mg every four months as well.

Current wording

In August 2024 we announced that the U.S. Food and Drug Administration removed the partial clinical hold on higher doses of zorevunersen in the MONARCH and SWALLOWTAIL studies in the United States. While the MONARCH study has been completed, patients in the SWALLOWTAIL OLE study in the United States currently receive chronic dosing every four months of 45mg of zorevunersen. In the United Kingdom, Patients in the LONGWING OLE study receive chronic dosing every four months of 45mg of zorevunersen as well.

Previous wording

Revenue for the three months ended March 31, 2024 was $4.2 million compared to $5.2 million for the three months ended March 31, 2023, a decrease of $0.9 million. Revenue is generated from satisfying contractual obligations of the collaboration and licensing agreement with Acadia Pharmaceuticals, Inc.

Current wording

Revenue for the three months ended June 30, 2024 was $4.8 million compared to $(2.5) million for the three months ended June 30, 2023, an increase of $7.3 million. Revenue is generated from satisfying contractual obligations of the collaboration and licensing agreement with Acadia Pharmaceuticals, Inc.

Previous wording

Research and development expenses were $22.4 million for the three months ended March 31, 2024 as compared to $19.6 million for the three months ended March 31, 2023, an increase of $2.8 million. The table below summarizes our research and development expenses (in thousands):

Current wording

Research and development expenses were $21.1 million for the three months ended June 30, 2024 as compared to $20.6 million for the three months ended June 30, 2023, an increase of $0.5 million. The table below summarizes our research and development expenses (in thousands):

Previous wording

General and administrative expenses were $10.2 million for the three months ended March 31, 2024 as compared to $10.2 million for the three months ended March 31, 2023.

Current wording

General and administrative expenses were $13.0 million for the three months ended June 30, 2024 as compared to $10.2 million for the three months ended June 30, 2023. The increase of $2.8 million in general and administrative expenses was primarily attributable to an increase of $1.3 million in professional fees, $0.8 million in personnel related expenses, and $0.7 million in facilities and other general and administrative costs.

Previous wording

The increase in research and development expenses were primarily attributable to an increase of $2.5 million in expenses related to our STK-001 program, which is comprised of third-party services and scientific consulting fees, an increase of $0.3 million in external third-party expenses related to SYNGAP1, MECP2, an increase of $0.7 million in facilities and other research and development costs offset by a decrease of $0.4 million in expenses related to our STK-002 program, which is comprised of third-party services and scientific consulting fees, a decrease of $0.3 million and non-project specific consulting and third-party services and a decrease of $0.1 million in personnel costs.

Current wording

The increase in research and development expenses was primarily attributable to an increase of $1.9 million in expenses related to zorevunersen, which is comprised of third-party services and scientific consulting fees, an increase of $0.9 million in facilities and other research and development costs, an increase of $0.5 million in personnel-related expenses, an increase of $0.5 million in external third-party expenses related to SYNGAP1 and MECP2, offset by a decrease of $0.5 million in expenses related to our STK-002 program, which is comprised of third-party services and scientific consulting fees.

Previous wording

Since our inception through March 31, 2024, our operations have been financed by net proceeds of $543.9 million from the sale of convertible notes payable and our convertible preferred stock, our initial public offering, follow-on offering, proceeds from the Sales Agreement and the upfront payment from Acadia. As of March 31, 2024, we had $178.6 million in cash and cash equivalents. Cash in excess of immediate requirements is invested in accordance with our investment policy, primarily with a view to liquidity and capital preservation. On April 2, 2024, we completed an underwritten public offering of our common stock and issued and sold 5,555,557 shares of common stock at a public offering price of $13.50 per share and issued pre-funded warrants to purchase 3,703,730 shares of common stock at a public offering price of $13.499 per share subject to an exercise price equal to $0.0001. The common stock and warrants sold resulted in net proceeds of $120.3 million after deducting underwriting discounts and commissions.

Current wording

Since our inception through June 30, 2024, our operations have been financed by net proceeds of $663.7 million from the sale of convertible notes payable and our convertible preferred stock, our initial public offering ("IPO"), public offerings of common stock and pre-funded warrants, proceeds from the Sales Agreement and the upfront payment from Acadia. As of June 30, 2024, we had $282.0 million in cash, cash equivalents and marketable securities. Cash in excess of immediate requirements is invested in accordance with our investment policy, primarily with a view to liquidity and capital preservation. On April 2, 2024, we completed an underwritten public offering of our common stock and issued and sold 5,555,557 shares of common stock at a public offering price of $13.50 per share and issued pre-funded warrants to purchase 3,703,730 shares of common stock at a public offering price of $13.499 per share subject to an exercise price equal to $0.0001. The common stock and warrants sold resulted in net proceeds of $119.9 million after deducting underwriting discounts, commissions and offering costs. No pre-funded warrants have been exercised.

Previous wording

We have incurred losses since our inception in June 2014 and, as of March 31, 2024, we had an accumulated deficit of $428.2 million. Our primary use of cash is to fund operating expenses, which consist primarily of research and development expenditures, and to a lesser extent, general and administrative expenditures. Cash used to fund operating expenses is impacted by the timing of when we pay these expenses, as reflected in the change in our outstanding accounts payable and accrued expenses.

Current wording

We have incurred losses since our inception in June 2014 and, as of June 30, 2024, we had an accumulated deficit of $453.9 million. Our primary use of cash is to fund operating expenses, which consist primarily of research and development expenditures, and to a lesser extent, general and administrative expenditures. Cash used to fund operating expenses is impacted by the timing of when we pay these expenses, as reflected in the change in our outstanding accounts payable and accrued expenses.

Previous wording

Based upon our current operating plan, we believe that our existing cash and cash equivalents as of March 31, 2024 together with the $120.3 million of net proceeds from our April 2024 public offering will enable us to fund our operating expenses and capital expenditure requirements at least to the end of 2025. We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we currently expect. We will continue to require additional financing to advance our current product candidates through clinical development, to develop, acquire or in-license other potential product candidates and to fund operations for the foreseeable future. We will continue to seek funds through equity offerings, debt financings or other capital sources, including potentially collaborations, licenses and other similar arrangements. However, we may be unable to raise additional funds or enter into such other arrangements when needed on favorable terms or at all. Our ability to raise additional funds may be adversely impacted by potential worsening global macroeconomic conditions, including inflation, changing interest rates and instability in the global banking system, and disruptions to and volatility in the credit and financial markets in the United States and worldwide. If we do raise additional capital through public or private equity offerings, the ownership interest of our existing stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect our stockholders’ rights. If we raise additional capital through debt financing, we may be subject to covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. Any failure to raise capital as and when needed could have a negative impact on our financial condition and on our ability to pursue our business plans and strategies. If we are unable to raise capital, we will need to delay, reduce or terminate planned activities to reduce costs.

Current wording

Based upon our current operating plan, we believe that our existing cash, cash equivalents and marketable securities as of June 30, 2024 will enable us to fund our operating expenses and capital expenditure requirements at least to the end of 2025. We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we currently expect. We will continue to require additional financing to advance our current product candidates through clinical development, to develop, acquire or in-license other potential product candidates and to fund operations for the foreseeable future. We will continue to seek funds through equity offerings, debt financings or other capital sources, including potentially collaborations, licenses and other similar arrangements. However, we may be unable to raise additional funds or enter into such other arrangements when needed on favorable terms or at all. Our ability to raise additional funds may be adversely impacted by potential worsening global macroeconomic conditions, including inflation, changing interest rates and instability in the global banking system, and disruptions to and volatility in the credit and financial markets in the United States and worldwide. If we do raise additional capital through public or private equity offerings, the ownership interest of our existing stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect our stockholders’ rights. If we raise additional capital through debt financing, we may be subject to covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. Any failure to raise capital as and when needed could have a negative impact on our financial condition and on our ability to pursue our business plans and strategies. If we are unable to raise capital, we will need to delay, reduce or terminate planned activities to reduce costs.

Previous wording

During the three months ended March 31, 2024, cash used in operating activities was $24.6 million. This was primarily attributable to a net loss of $26.4 million, a net change of $4.7 million in our net operating assets and liabilities, offset by non-cash charges of $6.5 million for stock-based compensation, depreciation, amortization and accretion of marketable securities, and a reduction in the carrying amount of right of use assets.

Current wording

During the six months ended June 30, 2024, cash used in operating activities was $42.0 million. This was primarily attributable to a net loss of $52.1 million, a net change of $4.6 million in our net operating assets and liabilities, offset by non-cash charges of $14.7 million for stock-based compensation, depreciation, amortization and accretion of marketable securities, and a reduction in the carrying amount of right of use assets.

Previous wording

During the three months ended March 31, 2023, cash used in operating activities was $21.4 million. This was primarily attributable to a net loss of $22.5 million, a net change of $5.9 million in our net operating assets and liabilities, offset by non-cash charges of $7.0 million for share-based compensation, depreciation, amortization and accretion of marketable securities, and a reduction in the carrying amount of right of use assets.

Current wording

During the six months ended June 30, 2023, cash used in operating activities was $43.2 million. This was primarily attributable to a net loss of $53.2 million, a net change of $4.8 million in our net operating assets and liabilities, offset by non-cash charges of $14.8 million for share-based compensation, depreciation, amortization and accretion of marketable securities, and a reduction in the carrying amount of right of use assets.

Previous wording

Our investing activities during the three months ended March 31, 2024 and 2023 have consisted of proceeds from the sales of marketable securities partially offset by purchases of property and equipment.

Current wording

Our investing activities during the six months ended June 30, 2023 consisted of $77.6 million from the sales of marketable securities partially offset by purchases of property and equipment.

Previous wording

Our financing activities during the three months ended March 31, 2024 consisted of $1.3 million of net proceeds from the controlled equity offering sales agreement, $0.2 million from the exercise of stock options and $0.2 million in proceeds from our Employee Stock Purchase Plan (the "ESPP").

Current wording

Our financing activities during the six months ended June 30, 2024 consisted of $119.9 million of net proceeds from a follow-on offering, $1.3 million of net proceeds from the controlled equity offering sales agreement, $1.0 million from the exercise of stock options and $0.2 million in proceeds from our Employee Stock Purchase Plan (the “ESPP”).

Previous wording

Our financing activities during the three months ended March 31, 2023 consisted of $44.7 million of net proceeds from the controlled equity offering sales agreement, $0.2 million from the exercise of stock options and $0.2 million in proceeds from the ESPP.

Current wording

Our financing activities during the six months ended June 30, 2023 consisted of $44.7 million of net proceeds from the controlled equity offering sales agreement, $0.2 million from the exercise of stock options and $0.2 million in proceeds from the ESPP.

Previous wording

We will remain an emerging growth company until the earliest of (i) the last day of our first fiscal year (a) following the fifth anniversary of the completion of our initial public offering ("IPO"), (b) in which we have total annual gross revenues of at least $1.235 billion, or (c) when we are deemed to be a large accelerated filer, which means the market value of our common stock that is held by non-affiliates exceeds $700.0 million as of the prior June 30th, and (ii) the date on which we have issued more than $1.0 billion in non-convertible debt securities during the prior three-year period. We anticipate ceasing to be an emerging growth company as of December 31, 2024, which is the last day of our fiscal year following the fifth anniversary of the completion of our IPO.

Current wording

We will remain an emerging growth company until the earliest of (i) the last day of our first fiscal year (a) following the fifth anniversary of the completion of our IPO, (b) in which we have total annual gross revenues of at least $1.235 billion, or (c) when we are deemed to be a large accelerated filer, which means the market value of our common stock that is held by non-affiliates exceeds $700.0 million as of the prior June 30th, and (ii) the date on which we have issued more than $1.0 billion in non-convertible debt securities during the prior three-year period. We anticipate ceasing to be an emerging growth company as of December 31, 2024, which is the last day of our fiscal year following the fifth anniversary of the completion of our IPO.

Previous wording

In December 2023, the FASB issued ASU 2023-09, “Income Taxes (Topic 740): Improvements to Income Tax Disclosures” (“ASU 2023-09”). ASU 2023-09 requires that an entity disclose specific categories in the effective tax rate reconciliation as well as provide additional information for reconciling items that meet a quantitative threshold and certain disclosures of state versus federal income tax expenses and taxes paid. ASC 2023-09 is effective for fiscal years beginning after December 15, 2024. The Company does not expect the adoption of ASU 2023-09 to have a material impact on its consolidated financial statements and will adopt the standard effective January 1, 2025.

Current wording

In December 2023, the FASB issued ASU 2023-09, “Income Taxes (Topic 740): Improvements to Income Tax Disclosures” (“ASU 2023-09”). ASU 2023-09 requires that an entity disclose specific categories in the effective tax rate reconciliation as well as provide additional information for reconciling items that meet a quantitative threshold and certain disclosures of state versus federal income tax expenses and taxes paid. ASU 2023-09 is effective for fiscal years beginning after December 15, 2024. The Company does not expect the adoption of ASU 2023-09 to have a material impact on its consolidated financial statements and will adopt the standard effective January 1, 2025.