Stoke Therapeutics Inc

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 OPA1 gene. We expect the OSPREY study to start later this year.

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. As we continue to focus on zorevunersen as our lead program, we will delay the start of the OSPREY study.

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.

Current wording

In May 2022, we filed a universal Shelf Registration statement on Form S-3 (the “2022 Registration Statement”) with the SEC. The 2022 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 (the “Sales Agreement”).

Previous wording

As of June 30, 2024, we had issued approximately 6.5 million shares of common stock pursuant to the Sales Agreement for net proceeds of $53.4 million. Since June 30, 2024, the Company has issued approximately 0.3 million shares of common stock pursuant to the Sales Agreement for net proceeds of $4.0 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 pre-funded 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 as of June 30, 2024.

Current wording

On April 2, 2024, we completed an underwritten public offering, pursuant to the 2022 Registration Statement, 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 pre-funded 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 as of September 30, 2024.

Previous 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.

Current wording

As of September 30, 2024 and December 31, 2023 we had $269.2 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 $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.

Current 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 $24.5 million for the three months ended September 30, 2024 and 2023, respectively and $78.5 million and $77.7 million for the nine months ended September 30, 2024 and 2023, respectively. As of September 30, 2024, we had an accumulated deficit of $480.3 million.

Previous 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.

Current wording

Revenue for the three months ended September 30, 2024 was $4.9 million compared to $3.3 million for the three months ended September 30, 2023, an increase of $1.6 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 $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):

Current wording

Research and development expenses were $22.2 million for the three months ended September 30, 2024 as compared to $20.3 million for the three months ended September 30, 2023, an increase of $1.9 million. The table below summarizes our research and development expenses (in thousands):

Previous wording

The increase in research and development expenses was primarily attributable to an increase of $0.6 million in personnel-related expenses, $0.2 million in facilities and other research and development costs, an increase of $0.2 million in external third-party expenses related to SYNGAP1 and MECP2 offset by a decrease of $0.5 million in expenses related to zorevunersen and an immaterial change in expense related to our STK-002 program, both of which are comprised of third-party services and scientific consulting fees.

Current wording

The increase in research and development expenses was primarily attributable to an increase of $1.4 million in personnel-related expenses, $0.7 million in expenses related to zorevunersen, which is comprised of third-party services and scientific consulting fees, an increase of $0.4 million in external third-party expenses related to SYNGAP1 and MECP2, offset by a decrease of $0.3 million related to our STK-002 program, which is comprised of third-party services and scientific consulting fees, and a decrease of $0.3 million in facilities and other non-program related costs.

Previous 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.

Current wording

General and administrative expenses were $12.7 million for the three months ended September 30, 2024 as compared to $10.3 million for the three months ended September 30, 2023. The increase of $2.4 million in general and administrative expenses was primarily attributable to an increase of $1.5 million in personnel expenses, $0.7 million in professional fees, and an increase $0.2 million in facilities and other general and administrative costs.

Previous wording

Other income (expense) was $3.6 million for the three months ended June 30, 2024 as compared to $2.6 million for the three months ended June 30, 2023.

Current wording

Other income (expense) was $3.6 million for the three months ended September 30, 2024 as compared to $2.7 million for the three months ended September 30, 2023.

Previous wording

Revenue for the six months ended June 30, 2024 was $9.0 million compared to $2.7 million for the six months ended June 30, 2023, an increase of $6.3 million. Revenue is generated from satisfying contractual obligations of the collaboration and licensing agreement with Acadia Pharmaceuticals, Inc.

Current wording

Revenue for the nine months ended September 30, 2024 was $13.9 million compared to $6.0 million for the nine months ended September 30, 2023, an increase of $7.9 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 $43.5 million for the six months ended June 30, 2024 as compared to $40.2 million for the six months ended June 30, 2023, an increase of $3.3 million. The table below summarizes our research and development expenses (in thousands):

Current wording

Research and development expenses were $65.7 million for the nine months ended September 30, 2024 as compared to $60.5 million for the nine months ended September 30, 2023, an increase of $5.2 million. The table below summarizes our research and development expenses (in thousands):

Previous 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.

Current wording

The increase in research and development expenses was primarily attributable to an increase of $2.7 million in expenses related to zorevunersen, which is comprised of third-party services and scientific consulting fees, an increase of $2.0 million in personnel-related expenses, an increase of $0.9 million in external third-party expenses related to SYNGAP1 and MECP2, an increase of $0.4 million in facilities and other non-program related costs, offset by a decrease of $0.8 million in expenses related to our STK-002 program, which is comprised of third-party services and scientific consulting fees.

Previous wording

General and administrative expenses were $23.3 million for the six months ended June 30, 2024 as compared to $20.4 million for the six months ended June 30, 2023. The increase of $2.9 million in general and administrative expenses was primarily attributable to an increase of $1.1 million in professional fees, $1.3 million in personnel related expenses, and $0.5 million in facilities and other general and administrative costs.

Current wording

General and administrative expenses were $36.0 million for the nine months ended September 30, 2024 as compared to $30.7 million for the nine months ended September 30, 2023. The increase of $5.3 million in general and administrative expenses was primarily attributable to an increase of $2.9 million in personnel related expenses, $1.7 million in professional fees, and $0.7 million in facilities and other general and administrative costs.

Previous 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.

Current wording

Since our inception through September 30, 2024, our operations have been financed by net proceeds of $671.6 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 September 30, 2024, we had $269.2 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 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.

Current wording

We have incurred losses since our inception in June 2014 and, as of September 30, 2024, we had an accumulated deficit of $480.3 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

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.

Current wording

During the nine months ended September 30, 2024, cash used in operating activities was $63.7 million. This was primarily attributable to a net loss of $78.5 million, a net change of $8.0 million in our net operating assets and liabilities, offset by non-cash charges of $22.8 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 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.

Current wording

During the nine months ended September 30, 2023, cash used in operating activities was $60.4 million. This was primarily attributable to a net loss of $77.7 million, a net change of $5.2 million in our net operating assets and liabilities, offset by non-cash charges of $22.5 million for stock-based compensation, depreciation, amortization and accretion of marketable securities, and reduction in the carrying amount of right of use assets.

Previous wording

Our investing activities during the six months ended June 30, 2024 consisted of $88.2 million of purchases of marketable securities offset by $10.0 million from the sales of marketable securities.

Current wording

Our investing activities during the nine months ended September 30, 2024 consisted primarily of $138.1 million of purchases of marketable securities offset by $30.0 million from the sales of marketable securities.

Previous 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.

Current wording

Our investing activities during the nine months ended September 30, 2023 consisted of $92.6 million from the sales of marketable securities partially offset by purchases of property and equipment.

Previous 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”).

Current wording

Our financing activities during the nine months ended September 30, 2024 consisted of $119.9 million of net proceeds from a follow-on offering, $9.0 million of net proceeds from the controlled equity offering sales agreement, $1.2 million from the exercise of stock options and $0.5 million in proceeds from our Employee Stock Purchase Plan (the “ESPP”).

Previous 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.

Current wording

Our financing activities during the nine months ended September 30, 2023 consisted of $44.7 million of net proceeds from the controlled equity offering sales agreement, $0.4 million from the exercise of stock options and $0.6 million in proceeds from the ESPP.