Stoke Therapeutics, Inc.

Previous wording

We are a late-stage clinical-stage company dedicated to addressing the underlying causes of severe diseases by upregulating protein expression with RNA-based medicines. Using our proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, we are developing antisense oligonucleotides (“ASOs”) to selectively restore protein levels.

Current wording

We are a late-stage clinical company dedicated to addressing the underlying causes of severe diseases by upregulating protein expression with RNA-based medicines. Using our proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, we are developing antisense oligonucleotides (“ASOs”) to selectively restore protein levels.

Previous wording

Following discussions with the U.S. Food and Drug Administration (“FDA”), European Medicines Agency (“EMA”), and Japan’s Pharmaceuticals and Medical Devices Agency (“PMDA”), a Phase 3 study, EMPEROR, was initiated in May 2025, with the first patient dosed in August 2025.This trial follows the completion of the Phase 1/2a studies and further evaluates the efficacy and safety of zorevunersen in children and adolescents ages 2 to up to 18 with Dravet syndrome.

Current wording

Following discussions with the U.S. Food and Drug Administration (“FDA”), European Medicines Agency (“EMA”), and Japan’s Pharmaceuticals and Medical Devices Agency (“PMDA”), a Phase 3 study, EMPEROR, was initiated in May 2025, with the first patient dosed in August 2025.This trial follows the completion of two open-label Phase 1/2a studies, MONARCH in the United States and ADMIRAL in the United Kingdom, and further evaluates the efficacy and safety of zorevunersen in children and adolescents ages 2 to up to 18 with Dravet syndrome.

Previous wording

In addition to these studies, we continue to run open-label extension (“OLE”) studies in patients with Dravet syndrome. The Phase 1/2a ADMIRAL and OLE studies have shown substantial and durable reductions in convulsive seizure frequency when administered on top of standard of care anti-seizure medicines. In the Phase 1/2a studies, 85% of patients were taking at least three and 54% were taking at least four medicines to control seizures. Half the patients in the studies were taking concomitant fenfluramine. Ongoing treatment has led to continuous improvements in cognition and behavior through three years. Additional improvements were indicated within the first nine months of treatment among patients in the Phase 1/2a study. These improvements were observed across multiple domains of the Vineland-3 (Vineland Adaptive Behavior Scale, Third Edition), a standardized assessment of behavioral outcomes that is a key secondary endpoint for the Phase 3 study. Zorevunersen has been generally well tolerated across the studies.

Current wording

In addition to the MONARCH and ADMIRAL studies, we continue to run open-label extension (“OLE”) studies in patients who completed the Phase 1/2a studies and met study entry criteria. The four studies have shown substantial and durable reductions in convulsive seizure frequency when administered on top of standard of care anti-seizure medicines. In the Phase 1/2a studies, 85% of patients were taking at least three and 54% were taking at least four medicines to control seizures. Half the patients in the studies were taking concomitant fenfluramine. Ongoing treatment has led to continuous improvements in cognition and behavior through three years. Additional improvements were indicated within the first nine months of treatment among patients in the Phase 1/2a study. These improvements were observed across multiple domains of the Vineland-3 (Vineland Adaptive Behavior Scale, Third Edition), a standardized assessment of behavioral outcomes that is a key secondary endpoint for the Phase 3 study. Zorevunersen has been generally well tolerated across the studies.

Previous wording

In addition to our Dravet program, we are also pursuing treatment for a second haploinsufficient disease, autosomal dominant optic atrophy (“ADOA”), the most common inherited optic nerve disorder for which there are currently no approved treatments. STK-002 is our 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. In a non-human primate (NHP) model of ADOA conducted in collaboration with UC Davis, treatment with STK-002 was well tolerated and helped protect, and possibly improve, eye health. The data suggest that STK-002 may help preserve the function of important vision-related nerve cells, which could potentially improve or maintain vision. We have received authorization in the UK 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 plan to enroll our first patient in the Phase 1 study in the second half of 2025.

Current wording

In addition to our Dravet program, we are also pursuing treatment for a second haploinsufficient disease, autosomal dominant optic atrophy (“ADOA”), the most common inherited optic nerve disorder for which there are currently no approved treatments. STK-002 is our 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. In a non-human primate (NHP) model of ADOA conducted in collaboration with UC Davis, treatment with STK-002 was well tolerated and helped protect, and possibly improve, eye health. The data suggest that STK-002 may help preserve the function of important vision-related nerve cells, which could potentially improve or maintain vision. We received authorization in the UK 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. The trial initiated in August, and we plan to enroll our first patient in the Phase 1 study in the second half of 2025.

Previous wording

In July 2025, we filed a universal Shelf Registration Statement on Form S-3 (the “2025 Registration Statement”) with the SEC. The 2025 Registration Statement was declared effective by the SEC on July 11, 2025. See “Note 12—Subsequent Events” of the notes to our unaudited financial statements included elsewhere in this Quarterly Report on Form 10-Q for additional information related to the 2025 Registration Statement.

Current wording

In July 2025, we filed a universal Shelf Registration Statement on Form S-3 (the “2025 Registration Statement”) with the SEC. The 2025 Registration Statement was declared effective by the SEC on July 11, 2025.

Previous wording

As of June 30, 2025, we had issued approximately 7.0 million shares of common stock pursuant to the Sales Agreement for net proceeds of $61.0 million pursuant to the 2022 Registration Statement. As of June 30, 2025, we had not issued or sold any shares pursuant to the 2024 Registration Statement. We may terminate this at-the-market program at any time, pursuant to its terms.

Current wording

As of September 30, 2025, we had issued approximately 7.0 million shares of common stock pursuant to the Sales Agreement for net proceeds of $61.0 million pursuant to the 2022 Registration Statement. As of September 30, 2025, we had not issued or sold any shares pursuant to the 2024 Registration Statement or 2025 Registration Statement. Since September 30, 2025, through the date of the issuance of these consolidated financial statements, we sold approximately 1.8 million shares of our common stock and received $48.7 million, net of commissions, pursuant to the 2025 Registration Statement. We may terminate this at-the-market program at any time, pursuant to its terms.

Previous wording

As of June 30, 2025 and December 31, 2024 we had $355.0 million and $246.7 million, respectively, in cash, cash equivalents and marketable securities.

Current wording

As of September 30, 2025 and December 31, 2024 we had $328.6 million and $246.7 million, respectively, in cash, cash equivalents and marketable securities.

Previous wording

Since inception, we have primarily had operating losses, which are attributable to research and development activities. Our net losses were $23.5 million and $25.7 million for the three months ended June 30, 2025 and 2024, respectively. For the six months ended June 30, 2025 we had a net income of $89.4 million and for the six months ended June 30, 2024 we had net losses of $52.1 million. As of June 30, 2025, we had an accumulated deficit of $401.4 million.

Current wording

Since inception, we have primarily had operating losses, which are attributable to research and development activities. Our net losses were $38.3 million and $26.4 million for the three months ended September 30, 2025 and 2024, respectively. For the nine months ended September 30, 2025 we had a net income of $51.0 million and for the nine months ended September 30, 2024 we had net losses of $78.5 million. As of September 30, 2025, we had an accumulated deficit of $439.8 million.

Previous wording

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. We expect to continue to incur net losses for the foreseeable future, and we expect our research and development expenses, general and administrative expenses, and capital expenditures will continue to increase. In particular, we expect our expenses and losses to increase as we continue our development of, and seek regulatory approvals for, our product candidates, and begin to commercialize any approved products, as well as hire additional personnel, develop commercial infrastructure, pay fees to outside consultants, lawyers and accountants, and incur increased costs associated with being a public company such as expenses related to services associated with maintaining compliance with Nasdaq listing rules and SEC requirements, insurance and investor relations costs. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and our expenditures on other research and development activities.

Current wording

Our primary use of cash is to fund operating expenses, which consist primarily of research and development expenditures, and to a lesser extent, sales, 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. We expect to continue to incur net losses for the foreseeable future, and we expect our research and development expenses, sales, general and administrative expenses, and capital expenditures will continue to increase. In particular, we expect our expenses and losses to increase as we continue our development of, and seek regulatory approvals for, our product candidates, and begin to commercialize any approved products, as well as hire additional personnel, develop commercial infrastructure, pay fees to outside consultants, lawyers and accountants, and incur increased costs associated with being a public company such as expenses related to services associated with maintaining compliance with Nasdaq listing rules and SEC requirements, insurance and investor relations costs. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and our expenditures on other research and development activities.

Previous wording

Based upon our current operating plan, we believe that our cash, cash equivalents, and marketable securities of approximately $355.0 million as of June 30, 2025, will fund operations to mid-2028. 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 cash, cash equivalents, and marketable securities of approximately $328.6 million as of September 30, 2025, will fund operations to mid-2028. 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

In August, we announced new positive findings from the long-term open-label extension (OLE) studies of zorevunersen in children and adolescents with Dravet syndrome.

Current wording

Presentations of new clinical data for zorevunersen at medical congresses have provided greater understanding of its disease-modifying potential that is highly differentiated from any currently available treatments. In August 2025, we announced new positive findings from the long-term open-label extension (OLE) studies of zorevunersen in children and adolescents with Dravet syndrome.

Previous wording

For the six months ended, June 30, 2025, we recognized revenue related to the Biogen collaboration of $155.6 million.

Current wording

For the nine months ended, September 30, 2025, we recognized revenue related to the Biogen collaboration of $162.3 million.

Previous wording

In January 2022, we entered into a license and collaboration agreement (the “Acadia Agreement”) with Acadia Pharmaceuticals Inc. (“Acadia”) for the discovery, development and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The agreement focuses on the targets SYNGAP1, MECP2 (Rett syndrome), and an undisclosed neurodevelopmental target of mutual interest. In connection with each target, we will collaborate with Acadia to identify potential treatments for further development and commercialization as licensed products. With respect to SYNGAP1, we have agreed with Acadia to co-develop and co-commercialize licensed products for such target globally, and in connection therewith we granted to Acadia worldwide, co-exclusive (with us) licenses for such licensed products. With respect to MECP2 and the neurodevelopmental target, we granted to Acadia worldwide, exclusive licenses to develop and commercialize licensed products for such targets. In May 2025, we received notice that Acadia had elected to discontinue the research programs for MECP2 (Rett Syndrome) and the undisclosed neurodevelopmental target. Under the Acadia Agreement, the licenses granted to Acadia for the discontinued programs will terminate and Acadia’s funding for such programs will wind down. The collaboration with Acadia with respect to SYNGAP1 remains ongoing.

Current wording

In January 2022, we entered into a license and collaboration agreement (the “Acadia Agreement”) with Acadia Pharmaceuticals Inc. (“Acadia”) for the discovery, development and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The Acadia Agreement focused on the targets SYNGAP1, MECP2 (Rett syndrome), and an undisclosed neurodevelopmental target of mutual interest. In connection with each target, we agreed to collaborate with Acadia to identify potential treatments for further development and commercialization as licensed products. With respect to SYNGAP1, we have agreed with Acadia to co-develop and co-commercialize licensed products for such target globally, and in connection therewith we granted to Acadia worldwide, co-exclusive (with us) licenses for such licensed products. With respect to MECP2 and the neurodevelopmental target, we granted to Acadia worldwide, exclusive licenses to develop and commercialize licensed products for such targets. Effective as of September 3, 2025, Acadia terminated the MECP2 and the undisclosed neurodevelopmental programs under the Acadia Agreement (the “Discontinued Acadia Programs”) and rights to these targets returned to us. The collaboration with Acadia with respect to SYNGAP1 remains ongoing.

Previous wording

Pursuant to the terms of the agreement, we received an upfront payment of $60.0 million from Acadia. Acadia agreed to fund the research to identify potential licensed products for MECP2 and the neurodevelopmental target, and we will equally fund with Acadia the research to identify potential licensed products for SYNGAP1. We are eligible to receive up to $907.5 million in potential total milestone payments based upon the achievement of certain development, regulatory, first commercial sales and sales milestone events across the programs for the three targets, assuming each milestone were achieved at least once. With respect to licensed products for MECP2 and the neurodevelopmental target, we are also eligible to receive tiered royalties at percentages ranging from the mid-single digits to the mid-teens on future net sales by Acadia of licensed products worldwide. Royalties payable under the agreement are subject to standard royalty reductions. For SYNGAP1 licensed products that we are co-developing and co-commercializing, we will be responsible for 50% of the development and commercialization costs and will receive 50% of the profits from global commercialization. We are provided with a co-development and co-commercialization opt out option relating to the SYNGAP1 target indication at our discretion. Such opt-out would reduce development and commercialization milestones but would provide us with royalties on an escalating basis attributable to net sales milestones.

Current wording

Pursuant to the terms of the agreement, we received an upfront payment of $60.0 million from Acadia. Acadia agreed to fund the research to identify potential licensed products for the Discontinued Acadia Programs, and we will equally fund with Acadia the research to identify potential licensed products for SYNGAP1. We are eligible to receive up to $245.0 million in potential total milestone payments based upon the achievement of certain development, regulatory, first commercial sales and sales milestone events for the SYNGAP1 program, assuming each milestone were achieved at least once. For SYNGAP1 licensed products that we are co-developing and co-commercializing, we will be responsible for 50% of the development and commercialization costs and will receive 50% of the profits from global commercialization. We are provided with a co-development and co-commercialization opt out option relating to the SYNGAP1 target indication at our discretion. Such opt-out would reduce development and commercialization milestones but would provide us with royalties on an escalating basis attributable to net sales milestones. As a result of the termination of the Discontinued Acadia Programs, the Company is no longer eligible to receive the milestones for those programs of up to $662.5 million or any royalties related thereto.

Previous wording

For the six months ended, June 30, 2025, we recognized revenue related to the Acadia collaboration of $16.8 million.

Current wording

For the nine months ended, September 30, 2025, we recognized revenue related to the Acadia collaboration of $20.7 million.

Previous wording

General and administrative expenses consist primarily of personnel costs, costs related to maintenance and filing of intellectual property, expenses for outside professional services, including legal, human resources, information technology, audit and accounting services, and facilities and other expenses. Personnel costs consist of salaries, benefits and stock-based compensation expense. We expect our general and administrative expenses to increase over the next several years to support our continued research and development activities, manufacturing activities, increased costs of operating as a public company and the potential commercialization of our product candidates. These increases are anticipated to include increased costs related to the hiring of additional personnel, developing commercial infrastructure, fees to outside consultants, lawyers and accountants, and costs associated with being a public company such as expenses related to services associated with maintaining compliance with Nasdaq listing rules and SEC requirements, insurance and investor relations costs.

Current wording

Sales, general and administrative expenses consist primarily of personnel costs, costs related to maintenance and filing of intellectual property, expenses for outside professional services, including legal, human resources, information technology, audit and accounting services, and facilities and other expenses. Personnel costs consist of salaries, benefits and stock-based compensation expense. We expect our sales, general and administrative expenses to increase over the next several years to support our continued research and development activities, manufacturing activities, increased costs of operating as a public company and the potential commercialization of our product candidates. These increases are anticipated to include increased costs related to the hiring of additional personnel, developing commercial infrastructure, fees to outside consultants, lawyers and accountants, and costs associated with being a public company such as expenses related to services associated with maintaining compliance with Nasdaq listing rules and SEC requirements, insurance and investor relations costs.

Previous wording

Revenue for the three months ended June 30, 2025 was $13.8 million compared to $4.8 million for the three months ended June 30, 2024, an increase of $9.0 million. Revenue is generated from satisfying contractual obligations of the collaboration and licensing agreements with Acadia and Biogen. The increase of $9.0 million is driven by $3.2 million for global development activities as part of the Biogen Agreement and $5.8 million related to the Acadia Agreement.

Current wording

Revenue for the three months ended September 30, 2025 was $10.6 million compared to $4.9 million for the three months ended September 30, 2024, an increase of $5.7 million. Revenue is generated from satisfying contractual obligations of the collaboration and licensing agreements with Acadia and Biogen. The increase in revenue is driven by $6.7 million for global development activities as part of the Biogen Agreement offset by a decrease of $1.0 million related to the Acadia Agreement.

Previous wording

Research and development expenses were $25.9 million for the three months ended June 30, 2025 as compared to $21.1 million for the three months ended June 30, 2024, an increase of $4.8 million. The table below summarizes our research and development expenses (in thousands):

Current wording

Research and development expenses were $37.7 million for the three months ended September 30, 2025 as compared to $22.2 million for the three months ended September 30, 2024, an increase of $15.5 million. The table below summarizes our research and development expenses (in thousands):

Previous wording

The increase in research and development expenses was attributable to an increase of $2.7 million in personnel-related expenses, $2.9 million in expenses related to our zorevunersen program, which is comprised of third-party services and scientific consulting fees, an increase of $0.1 million in external third-party expenses related to SYNGAP1 and MECP2, offset by a decrease of $0.6 million in expenses related to our ADOA 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.

Current wording

The increase in research and development expenses was attributable to an increase of $3.2 million in personnel-related expenses, $11.7 million in expenses related to our zorevunersen program, $0.2 million in expenses related to our ADOA program, both of which are comprised of third-party services and scientific consulting fees, an increase of $0.2 million in external third-party expenses related to SYNGAP1 and MECP2, and an increase of $0.2 in facilities and other non-program related costs.

Previous wording

General and administrative expenses were $15.3 million for the three months ended June 30, 2025 as compared to $13.0 million for the three months ended June 30, 2024. The increase of $2.3 million in general and administrative expenses was attributable to an increase of $1.9 million in professional fees, $0.3 million in personnel expenses and an increase $0.1 million in facilities and other general and administrative costs.

Current wording

Sales, general and administrative expenses were $16.0 million for the three months ended September 30, 2025 as compared to $12.7 million for the three months ended September 30, 2024. The increase of $3.3 million in sales, general and administrative expenses was attributable to an increase of $2.5 million in personnel expenses and an increase $0.8 million in facilities and other sales, general and administrative costs.

Previous wording

Other income (expense) was $3.8 million for the three months ended June 30, 2025 as compared to $3.6 million for the three months ended June 30, 2024.

Current wording

Other income (expense) was $3.5 million for the three months ended September 30, 2025 as compared to $3.6 million for the three months ended September 30, 2024.

Previous wording

Revenue for the six months ended June 30, 2025 was $172.4 million compared to $9.0 million for the six months ended June 30, 2024, an increase of $163.4 million. Revenue is generated from satisfying contractual obligations of the collaboration and licensing agreement with Acadia and Biogen. The increase of $163.4 million is primarily driven by $150.8 million related to the IP license performance obligation and $4.9 million for global development activities as part of the Biogen Agreement and $7.7 million related to the Acadia Agreement.

Current wording

Revenue for the nine months ended September 30, 2025 was $183.0 million compared to $13.9 million for the nine months ended September 30, 2024, an increase of $169.1 million. Revenue is generated from satisfying contractual obligations of the collaboration and licensing agreement with Acadia and Biogen. The increase of $169.1 million is primarily driven by $150.8 million related to the IP license performance obligation and $11.5 million for global development activities as part of the Biogen Agreement and $6.8 million related to the Acadia Agreement.

Previous wording

Research and development expenses were $58.5 million for the six months ended June 30, 2025 as compared to $43.5 million for the six months ended June 30, 2024, an increase of $15.0 million. The table below summarizes our research and development expenses (in thousands):

Current wording

Research and development expenses were $96.2 million for the nine months ended September 30, 2025 as compared to $65.7 million for the nine months ended September 30, 2024, an increase of $30.5 million. The table below summarizes our research and development expenses (in thousands):

Previous wording

The increase in research and development expenses was attributable to an increase of $11.3 million in expenses related to zorevunersen, which is comprised of third-party services and scientific consulting fees, an increase of $5.8 million in personnel-related expenses, an increase of $0.1 million in external third-party expenses related to SYNGAP1 and MECP2, offset by a decrease of $1.3 million in expenses related to our ADOA program, which is comprised of third-party services and scientific consulting fees and $0.9 million in facilities and other research and development costs.

Current wording

The increase in research and development expenses was attributable to an increase of $23.0 million in expenses related to zorevunersen, which is comprised of third-party services and scientific consulting fees, an increase of $9.0 million in personnel-related expenses, an increase of $0.3 million in external third-party expenses related to SYNGAP1 and MECP2, offset by a decrease of $1.0 million in expenses related to our ADOA program, which is comprised of third-party services and scientific consulting fees and $0.8 million in facilities and other research and development costs.

Previous wording

General and administrative expenses were $29.9 million for the six months ended June 30, 2025 as compared to $23.3 million for the six months ended June 30, 2024. The increase of $6.6 million in general and administrative expenses was attributable to an increase of $4.2 million in professional fees, $2.2 million in personnel related expenses, and $0.2 million in facilities and other general and administrative costs.

Current wording

Sales, general and administrative expenses were $45.9 million for the nine months ended September 30, 2025 as compared to $36.0 million for the nine months ended September 30, 2024. The increase of $9.9 million in sales, general and administrative expenses was attributable to an increase of $2.4 million in professional fees, $6.7 million in personnel related expenses, and $0.8 million in facilities and other sales, general and administrative costs.

Previous wording

Under U.S. GAAP, the effects of changes in tax laws are recognized in the period in which the new law is enacted. Accordingly, the impact of the OBBBA will be reflected in the Company's financial statements for the three months ending September 30, 2025. The Company is currently assessing the impact of the OBBBA, and an estimate of the financial effect is not yet available.

Current wording

Under U.S. GAAP, the effects of changes in tax laws are recognized in the period in which the new law is enacted. Accordingly, the impact of the OBBBA was reflected as of September 30, 2025 with the reversal of previously recognized income tax expense for both federal and state.

Previous wording

Since our inception through June 30, 2025, our operations have been financed by net proceeds of $836.6 million from the sale of convertible notes payable and our convertible preferred stock, our initial public offering, public offerings of common stock and pre-funded warrants, proceeds from the Sales Agreement and the upfront payments from Acadia and Biogen. As of June 30, 2025, we had $355.0 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, 2025, our operations have been financed by net proceeds of $836.6 million from the sale of convertible notes payable and our convertible preferred stock, our initial public offering, public offerings of common stock and pre-funded warrants, proceeds from the Sales Agreement and the upfront payments from Acadia and Biogen. As of September 30, 2025, we had $328.6 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 primarily incurred losses since our inception in June 2014 and, as of June 30, 2025, we had an accumulated deficit of $401.4 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 primarily incurred losses since our inception in June 2014 and, as of September 30, 2025, we had an accumulated deficit of $439.8 million. Our primary use of cash is to fund operating expenses, which consist primarily of research and development expenditures, and to a lesser extent, sales, 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 cash, cash equivalents, and marketable securities of approximately $355.0 million as of June 30, 2025, will fund operations to mid-2028. 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, tariffs, 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 cash, cash equivalents, and marketable securities of approximately $328.6 million as of September 30, 2025, will fund operations to mid-2028. 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, tariffs, 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 six months ended June 30, 2025, cash provided by operating activities was $106.4 million. This was attributable to a net income of $89.4 million, a net change of $0.5 million in our net operating assets and liabilities and non-cash charges of $16.5 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 nine months ended September 30, 2025, cash provided by operating activities was $76.0 million. This was attributable to a net income of $51.0 million, a net change of $0.6 million in our net operating assets and liabilities and non-cash charges of $25.6 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, 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

Our investing activities during the six months ended June 30, 2025 consisted primarily of $200.6 million of purchases of marketable securities and $0.3 million of purchases of property plant and equipment offset by $66.0 million from the sales of marketable securities.

Current wording

Our investing activities during the nine months ended September 30, 2025 consisted primarily of $224.5 million of purchases of marketable securities and $0.5 million of purchases of property plant and equipment offset by $99.2 million from the sales of marketable securities.

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 financing activities during the three months ended June 30, 2025 consisted of $1.6 million from the exercise of stock options and $0.4 million in proceeds from our Employee Stock Purchase Plan (the “ESPP”).

Current wording

Our financing activities during the nine months ended September 30, 2025 consisted of $4.0 million from the exercise of stock options and $1.0 million in proceeds from our Employee Stock Purchase Plan (the “ESPP”).

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 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 ESPP.

Previous wording

In January 2025, we entered into an agreement to lease 7,581 square feet of space for an initial term of three years and three months. The lease includes two renewal options for an additional three years each. Lease terms commence at $0.7 million per annum, with 2.0% annual base rent increases plus operating expenses, real estate taxes, and utilities. The lease commencement date was July 8, 2025. As we did not have access to the space, a right-of-use asset was not recognized as of June 30, 2025.

Current wording

In January 2025, we entered into an agreement to lease 7,581 square feet of space for an initial term of three years and three months. The lease includes two renewal options for an additional three years each. Lease terms commence at $0.7 million per annum, with 2.0% annual base rent increases plus operating expenses, real estate taxes, and utilities. The lease commencement date was July 8, 2025. In July 2025, we recognized a right-of-use asset and operating lease liability of $1.8 million. In September 2025, we entered into a sublease agreement for the 7,581 square feet of space for a term equal to the initial term of the original lease. Sublease terms commence at $0.4 million per annum with 2% annual rent increases. As a result of this sublease, we recognized a right of use asset impairment of $0.7 million.