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Stoke Therapeutics, Inc.

For the period ended Jun 30, 2026. Filed Aug 3, 2026.

View on SEC.gov

About this filing

A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.

Jun 30, 2026 · Filed Aug 3, 2026

STOK$26.54+1.8%Stored close · as of Nov 6, 2026

What changed in Management’s Discussion and Analysis?

Read the current filing

Previous report: 10-Q

Period ended 2026-03-31; filed 2026-05-07.

0001193125-26-211874

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

Current report: 10-Q

Period ended 2026-06-30; filed 2026-08-03.

0001193125-26-330557

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.

Compared 125 previous paragraphs and 132 current paragraphs.

85 unchanged; 29 reworded; 12 added; 11 removed; 0 previous and 6 current paragraphs rearranged.

Tables are not compared: 5 previous and 7 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.

A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.

Added paragraphs (12)

Current report

Presentations of new clinical data for zorevunersen at medical congresses have contributed to greater understanding of its disease-modifying potential that is highly differentiated from any currently available treatments. The Company will continue its educational efforts at major neurology and epilepsy congresses during the second half of 2026. Analyses of data from the ongoing OLEs will support efforts to increase clinician understanding of zorevunersen while the Phase 3 EMPEROR study advances toward completion and the Company prepares for a potential U.S. approval and launch. The Company will also continue discussions with the FDA to update the Agency on its long-term data for zorevunersen.

Current report

An additional cohort of approximately 30 patients is currently being enrolled in Europe, where the sham control is administered via a needle prick (NP). Screening for this cohort has now closed, and enrollment is expected to be completed in August 2026.

Current report

Site activation and screening are also underway in China, where zorevunersen has been granted Breakthrough Therapy Status. Enrollment is anticipated to complete in the second half of 2026.

Current report

In August 2026, we announced that Stoke intends to participate in a pre-NDA meeting with the FDA during the second half of 2026 in preparation for the rolling NDA submission planned to initiate in the first quarter of 2027. Among other items, this meeting is intended to achieve alignment on the data package and statistical analysis plan for the rolling NDA

Current report

On July 13, 2026, we announced the appointment of Thomas McCauley, Ph.D., as the Company’s Chief Scientific Officer. In this new role, Dr. McCauley will be responsible for leading Stoke’s scientific strategy and leveraging the Company’s proprietary RNA medicines platform to expand and advance its pipeline of potential medicines for severe genetic diseases.

Current report

Other income (expense) was $3.8 million for the three months ended June 30, 2026 as compared to $3.8 million for the three months ended June 30, 2025.

Current report

Results of operations for the six months ended June 30, 2026 and 2025

Current report

Research and development expenses were $89.2 million for the six months ended June 30, 2026 as compared to $58.5 million for the six months ended June 30, 2025, an increase of $30.7 million. The table below summarizes our research and development expenses (in thousands):

Current report

Other income (expense) was $7.2 million for the six months ended June 30, 2026 as compared to $6.7 million for the six months ended June 30, 2025.
Removed paragraphs (11)

Previous report

Presentations of new clinical data for zorevunersen at medical congresses have contributed to greater understanding of its disease-modifying potential that is highly differentiated from any currently available treatments. In May 2026, we announced new findings from four-year longitudinal data from the ongoing Phase 1/2 open-label extension (OLE) studies of zorevunersen in children and adolescents with Dravet syndrome. 93% (75/81) of eligible patients continued treatment in the OLE studies, providing a longitudinal dataset to assess long-term outcomes. As of February 2026, 77% (58/75) of patients remain in these studies.

Previous report

As shown in Exhibit 1, durable reductions in major motor seizure frequency on top of standard-of-care anti-seizure medicines were observed at year four of zorevunersen treatment. The data also demonstrate continuing improvements in cognition and behavior over the treatment period, as shown in Exhibit 2. Zorevunersen continues to be generally well tolerated, with some patients treated for more than five years in the Phase 1/2a and ongoing OLE studies in which more than 850 doses have been administered. Elevated CSF protein lab values occurred in approximately 94% of patients of which 59% have been classified as a treatment-emergent adverse event. No serious or severe clinical manifestations have been associated with CSF protein elevations. No new safety findings have emerged.

Previous report

Other income (expense) was $3.4 million for the three months ended March 31, 2026 as compared to $2.9 million for the three months ended March 31, 2025.
Reworded paragraphs (29)

Previous 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.

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 major motor 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 four 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 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. In February 2026, we dosed the first patient with STK-002 in the OSPREY Phase 1 study for the treatment of ADOA.

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 are studying STK-002 in a Phase 1 open-label study (OSPREY) of children and adults ages 6 to 55 who have an established diagnosis of ADOA and evidence of a genetic mutation in the OPA1 gene. In February 2026, we dosed the first patient with STK-002 in the OSPREY Phase 1 study for the treatment of ADOA. Dose escalation is continuing, and the study is actively recruiting in the United Kingdom and the European Union.

Previous wording

As of March 31, 2026, we had issued approximately 3.2 million shares of common stock pursuant to the 2025 Registration Statement for net proceeds of $87.8 million. In addition, as of March 31, 2026, we had issued approximately 2.6 million shares of common stock pursuant to the Sales Agreement and the 2026 Registration Statement for net proceeds of $80.7 million. We may terminate this at-the-market program under the Sales Agreement at any time, pursuant to its terms.

Current wording

As of December 31, 2025, we had issued approximately 3.2 million shares of common stock pursuant to the 2025 Registration Statement for net proceeds of $87.8 million. In addition, as of June 30, 2026, we had issued approximately 2.6 million shares of common stock pursuant to the Sales Agreement and the 2026 Registration Statement for net proceeds of $80.7 million. Since June 30, 2026, through the issuance of these condensed consolidated financial statements, we sold approximately 2.1 million shares of our common stock pursuant to the Sales Agreement and the 2026 Registration Statement for net proceeds of $65.7 million after deducting commissions. We may terminate this at-the-market program under the Sales Agreement at any time, pursuant to its terms.

Previous wording

As of March 31, 2026 and December 31, 2025 we had $411.0 million and $390.9 million, respectively, in cash, cash equivalents and marketable securities.

Current wording

As of June 30, 2026 and December 31, 2025 we had $354.3 million and $390.9 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 $50.0 million for the three months ended March 31, 2026 and our net income for the three months ended March 31, 2025 was $112.9 million. As of March 31, 2026, we had an accumulated deficit of $547.7 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 $61.6 million and $23.5 million for the three months ended June 30, 2026 and 2025, respectively. For the six months ended June 30, 2026 we had a net loss of $111.6 million and for the six months ended June 30, 2025 we had net income of $89.4 million. As of June 30, 2026, we had an accumulated deficit of $609.3 million.

Previous wording

Based upon our current operating plan, we believe that our cash, cash equivalents, and marketable securities of approximately $411.0 million as of March 31, 2026, will fund operations into 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. 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 on our current operating plan, we believe that our cash, cash equivalents, and marketable securities of approximately $354.3 million as of June 30, 2026 and additional proceeds from the ATM sale of $65.7 million, will fund operations through to potential U.S. commercialization of zorevunersen in early 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. 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

EMPEROR is a global, pivotal Phase 3 study. New patient entry into screening for the U.S., UK, and Japan cohort is closed and patients currently in screening are progressing through the 8-week screening period. Following successful completion of screening, these patients will be randomized to treatment with zorevunersen or a sham control administered via lumbar puncture (LP). The final patient is expected to be randomized in June 2026. Data from this LP cohort of approximately 150 patients are anticipated in mid-2027 and are expected to be the final data required for completion of the planned rolling U.S. NDA submission. Commercial preparation is underway to support a potential U.S. commercial launch as early as late 2027.

Current wording

EMPEROR is a global, pivotal Phase 3 study. In June 2026, we announced completion of enrollment of 162 patients in the U.S., UK, and Japan, where patients are randomized to treatment with zorevunersen or a sham control administered via lumbar puncture (LP). A Phase 3 data readout is anticipated in the third quarter of 2027 to complete the planned rolling U.S. NDA submission in the second half of 2027. Commercial preparation is underway to support a potential U.S. commercial launch of zorevunersen in early 2028.

Previous wording

In Europe, sites are active and patient screening is underway for an additional cohort of at least 20 patients. Enrollment in this cohort is expected to be completed in the third quarter of 2026. Data from the European cohort are not planned for inclusion in the U.S. NDA submission.

Current wording

Data from the additional patients in Europe and China are not planned for inclusion in the U.S. NDA submission to the FDA.

Previous wording

We are also pursuing treatment for Autosomal Dominant Optic Atrophy (ADOA), the most common inherited optic nerve disorder, with STK-002. In February 2026, the first patient was dosed in the Phase 1 OSPREY study of STK-002. As of May 2026, two patients have been dosed and recruitment is ongoing at sites in the United Kingdom, Germany, Austria and Denmark. Dose escalation across initial four cohorts is expected to continue through 2026 and into early 2027, pending safety and tolerability assessments.

Current wording

We are also pursuing treatment for Autosomal Dominant Optic Atrophy (ADOA), the most common inherited optic nerve disorder, with STK-002. In February 2026, the first patient was dosed in the Phase 1 OSPREY study of STK-002. As of August 2026, all eight planned clinical trial sites are active in the United Kingdom, Germany, Austria, Italy and Denmark, and dosing of the first cohort of patients (n=3) is complete with no serious safety events observed to date. Dosing of the second cohort is expected to begin in August 2026. Subject to ongoing safety assessments, dosing in the third and fourth dose cohorts are expected to follow with a readout of safety and efficacy results anticipated in the first half of 2027.

Previous wording

For the three months ended March 31, 2026, we recognized revenue related to the Acadia collaboration of $2.2 million and for the three months ended March 31, 2025, we recognized revenue related to the Acadia collaboration of $6.1 million.

Current wording

Revenue recognized under the Acadia collaboration was $2.8 million and $10.7 million for the three months ended June 30, 2026 and 2025, respectively. For the six months ended June 30, 2026 and 2025, the Company recognized $5.0 million and $16.8 million, respectively, in revenue related to the Acadia Agreement.

Previous wording

On February 14, 2025, we entered into a License and Collaboration Agreement (the “Biogen Agreement”) with Biogen International GmbH (“Biogen”) for the joint development and commercialization of zorevunersen and other compounds targeting the SCN1A gene (the “Licensed Product”). Under the terms of the Biogen Agreement, we granted Biogen an exclusive, royalty-bearing license to develop, manufacture, and export licensed products in all territories outside the U.S., Canada, and Mexico (the “Biogen Territory”). In addition, Biogen has the option to exercise an exclusive, royalty-bearing, sublicensable and transferable license for certain future follow-on ASOs directed to SCN1A controlled by us. Under the Biogen Agreement, the parties are jointly developing zorevunersen, and we are leading global development. We and Biogen share global development costs based on an agreed budget. We are responsible for 70% of these development costs and Biogen is responsible for the remaining 30% of these development costs.

Current wording

Under the terms of the Biogen Agreement, we granted Biogen an exclusive, royalty bearing license to develop, manufacture and commercialize licensed products outside the United States, Canada and Mexico (the “Biogen Territory”). Biogen also has an option to exercise an exclusive, royalty-bearing, sublicensable and transferable license to certain future follow on antisense oligonucleotides (“ASOs”) directed to SCN1A controlled by the Company. The parties are jointly developing zorevunersen, with us leading global development, and development costs are shared pursuant to an agreed budget, with us responsible for 70% and Biogen responsible for 30%.

Previous wording

In connection with the closing of this transaction in February 2025, we received an upfront payment of $165.0 million and are eligible to receive development and commercial milestone payments that could total up to approximately $50.0 million and $335.0 million, respectively, if all the specified milestones set forth in this collaboration are achieved. In addition, we are eligible to receive tiered, double-digit royalties ranging from the low double-digit to high teen percentages of future net sales on the licensed products. As of March 31, 2026, no milestones have been met. Royalties payable under the Biogen Agreement are subject to standard royalty reductions.

Current wording

We received an upfront payment of $165.0 million and are eligible to receive development and commercial milestones that could total up to approximately $50.0 million and $335.0 million, respectively if all the specified milestones set forth in the collaboration arrangement are achieved. We are also eligible to receive tiered, double-digit royalties ranging from the low double digits to the high teen percentages on future net sales in the Licensed Product territory. As of June 30, 2026, no milestones have been achieved.

Previous wording

For the three months ended March 31, 2026, we recognized revenue related to the Biogen collaboration of $4.0 million and for the three months ended March 31, 2025, we recognized revenue related to the Biogen collaboration of $152.5 million.

Current wording

Revenue recognized under the Biogen collaboration was $6.5 million and $3.2 million for the three months ended June 30, 2026 and 2025, respectively. For the six months ended June 30, 2026 and 2025, the Company recognized $10.6 million and $155.6 million, respectively, in revenue related to the Biogen Agreement.

Previous wording

See Note 7—License and Collaboration Agreementsof the notes to our unaudited condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q.

Current wording

For more information see Note 7—License and Collaboration Agreementsof the notes to our unaudited condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q.

Previous wording

Research and development expenses were $39.7 million for the three months ended March 31, 2026 as compared to $32.7 million for the three months ended March 31, 2025, an increase of $7.0 million. The table below summarizes our research and development expenses (in thousands):

Current wording

Research and development expenses were $49.5 million for the three months ended June 30, 2026 as compared to $25.9 million for the three months ended June 30, 2025, an increase of $23.6 million. The table below summarizes our research and development expenses (in thousands):

Previous wording

Sales, general and administrative expenses were $20.0 million for the three months ended March 31, 2026 as compared to $14.7 million for the three months ended March 31, 2025. The increase of $5.3 million in sales, general and administrative expenses was attributable to an increase of $3.4 million in facilities and other sales, general and administrative costs for commercial readiness, and $1.9 million in personnel-related expenses.

Current wording

Sales, general and administrative expenses were $25.3 million for the three months ended June 30, 2026 as compared to $15.3 million for the three months ended June 30, 2025. The increase of $10.0 million in sales, general and administrative expenses was attributable to an increase of $6.6 million in facilities and other sales, general and administrative costs for commercial readiness, and $3.4 million in personnel-related expenses.

Previous wording

Revenue for the three months ended March 31, 2026 was $6.2 million compared to $158.6 million for the three months ended March 31, 2025, a decrease of $152.4 million. Revenue is generated from satisfying contractual obligations of the collaboration and licensing agreements with Acadia and Biogen. The decrease in revenue is primarily driven by the recognition of $150.8 million related to the IP license performance obligation during the year ended December 31, 2025.

Current wording

Revenue for the six months ended June 30, 2026 was $15.6 million compared to $172.4 million for the six months ended June 30, 2025, a decrease of $156.8 million. Revenue is generated from satisfying contractual obligations of the collaboration and licensing agreements with Acadia and Biogen. The decrease in revenue is primarily driven by the recognition of $150.8 million related to the Biogen IP license performance obligation during the six months ended June 30, 2025.

Previous wording

The increase in research and development expenses was attributable to an increase of $5.1 million in personnel-related expenses, $0.7 million in expenses related to our ADOA program, which are comprised of third-party services and scientific consulting fees, an increase of $0.4 million related to SYNGAP1 offset by a decrease of $0.1 million related to MECP2 in external third-party expenses related to SYNGAP1 and MECP2, and an increase of $1.4 million in other non-program related costs offset by a decrease of $0.5 million in expenses related to our zorevunersen program.

Current wording

The increase in research and development expenses was attributable to an increase of $12.3 million in expenses related to zorevunersen, which are comprised of third-party services and scientific consulting fees, $11.7 million in personnel-related expenses, $5.1 million in other external program costs, $2.2 million in expenses related to our ADOA program, which are comprised of third-party services and scientific consulting fees, an increase of $0.6 million in external third-party expenses related to SYNGAP1, offset by a decrease of $1.2 million related to other non-program costs.

Previous wording

Since our inception through March 31, 2026, our operations have been financed by net proceeds of $1.0 billion 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 March 31, 2026, we had $411.0 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

$354.3 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 March 31, 2026, we had an accumulated deficit of $547.7 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.

Current wording

We have primarily incurred losses since our inception in June 2014 and, as of June 30, 2026, we had an accumulated deficit of $609.3 million. Our primary use of cash is to fund operating expenses, which consist primarily of research and development expenditures, and to a lesser extent, 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 $411.0 million as of March 31, 2026, will fund operations into 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 on our current operating plan, we believe that our cash, cash equivalents, and marketable securities of approximately $354.3 million as of June 30, 2026 and additional proceeds from the ATM sale of $65.7 million, will fund operations through to potential U.S. commercialization of zorevunersen in early 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, military conflicts 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, 2026, cash used in operating activities was $60.6 million. This was attributable to a net loss of $50.0 million, a net change of $20.1 million in our net operating assets and liabilities offset by non-cash charges of $9.5 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, 2026, cash used in operating activities was $117.1 million. This was attributable to a net loss of $111.6 million, a net change of $26.6 million in our net operating assets and liabilities offset by non-cash charges of $21.0 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, 2025, cash provided by operating activities was $131.8 million. This was primarily attributable to a net income of $112.9 million, a net change of $11.5 million in our net operating assets and liabilities and non-cash charges of $7.3 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, 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.

Previous wording

Our investing activities during the three months ended March 31, 2026 consisted primarily of $60.9 million from the maturity of marketable securities offset by $9.9 million of purchases of marketable securities and $0.4 million of purchases of property plant and equipment.

Current wording

Our investing activities during the six months ended June 30, 2026 consisted primarily of $108.0 million from the maturity of marketable securities offset by $46.0 million of purchases of marketable securities and $2.3 million of purchases of property plant and equipment.

Previous wording

Our investing activities during the three months ended March 31, 2025 consisted primarily of $32.8 million from the maturity of marketable securities offset by $19.0 million of purchases of marketable securities.

Current 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.

Previous wording

Our financing activities during the three months ended March 31, 2026 consisted primarily of $80.7 million in proceeds from the controlled equity offering sales agreement, $3.2 million from the exercise of stock options and $0.5 million in proceeds from our Employee Stock Purchase Plan (the “ESPP”).

Current wording

Our financing activities during the six months ended June 30, 2026 consisted primarily of $80.7 million in proceeds from the controlled equity offering sales agreement, $5.1 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 three months ended March 31, 2025 consisted of $1.0 million from the exercise of stock options and $0.4 million in proceeds from the ESPP.

Current wording

Our financing activities during the six months ended June 30, 2025 consisted of $1.6 million from the exercise of stock options and $0.4 million in proceeds from our ESPP.

Previous wording

On January 21, 2026, we entered into a new lease for our corporate headquarters and laboratory space with NWALP PHOP Property Owner LLC, located at 245 Fifth Avenue, Waltham, Massachusetts (the “Lease Agreement”), in order to expand our office and laboratory space. The Lease Agreement premises include approximately 98,500 square feet. The Lease Agreement will expire on March 31, 2038, and includes two options to further extend the Lease for an additional five years at the then-prevailing market rate. The aggregate estimated base rent payments due over the term of the Lease Agreement is approximately $86.6 million. We paid a security deposit of $3.0 million in the form of an irrevocable standby letter of credit, which may be reduced over time in accordance with the terms of the Lease Agreement. We expect to occupy the space late in 2026.

Current wording

On January 21, 2026, we entered into a new lease for our corporate headquarters and laboratory space with NWALP PHOP Property Owner LLC, located at 245 Fifth Avenue, Waltham, Massachusetts (the “Lease Agreement”), in order to expand our office and laboratory space. The Lease Agreement premises include approximately 98,500 square feet. The Lease Agreement will expire on March 31, 2038, and includes two options to further extend the Lease Agreement for an additional five years at the then-prevailing market rate. The aggregate estimated base rent payments due over the term of the Lease Agreement is approximately $86.6 million. We paid a security deposit of $3.0 million in the form of an irrevocable standby letter of credit, which may be reduced over time in accordance with the terms of the Lease Agreement. We expect to occupy the space late in 2026.

Previous wording

We are a “smaller reporting company,” meaning that our annual revenue is less than $100.0 million during the last completed fiscal year or the market value of our stock held by non-affiliates was less than $700.0 million as of the end of that fiscal year’s second fiscal quarter. We will continue to be a smaller reporting company until the first quarter of 2027. As a smaller reporting company we may choose to present only the two most recent fiscal years of audited financial statements in our Annual Report on Form 10-K and smaller reporting companies have reduced disclosure obligations regarding executive compensation.

Current wording

We are a “smaller reporting company,” meaning that our annual revenue is less than $100.0 million during the last completed fiscal year or the market value of our stock held by non-affiliates was less than $700.0 million as of the end of that fiscal year’s second fiscal quarter. We expect to continue to be a smaller reporting company until the first quarter of 2027. As a smaller reporting company we may choose to present only the two most recent fiscal years of audited financial statements in our Annual Report on Form 10-K and smaller reporting companies have reduced disclosure obligations regarding executive compensation.
Rearranged current paragraphs (6)

Current report — wording carried from elsewhere

On February 14, 2025, we entered into a License and Collaboration Agreement (the “Biogen Agreement”) with Biogen International GmbH (“Biogen”) for the joint development and commercialization of zorevunersen and other compounds targeting the SCN1A gene (the “Licensed Product”).

Current report — wording carried from elsewhere

Revenue for the three months ended June 30, 2026 was $9.3 million compared to $13.8 million for the three months ended June 30, 2025, a decrease of $4.5 million. Revenue is generated from satisfying contractual obligations of the collaboration and licensing agreements with Acadia and Biogen.

Current report — wording carried from elsewhere

The increase in research and development expenses was attributable to an increase of $12.8 million of expenses related to zorevunersen, which are comprised of third-party services and scientific consulting fees, $6.6 million in personnel-related expenses, $3.4 million in other external program costs, $1.5 million in expenses related to our ADOA program, which are comprised of third-party services and scientific consulting fees, and an increase of $0.3 million in external third-party expenses related to SYNGAP1, offset by a decrease of $1.2 million related to other non-program costs, and $0.2 million in external third-party expenses related to MECP2.

Current report — wording carried from elsewhere

The following table sets forth our results of operations:

Current report — wording carried from elsewhere

Sales, general and administrative expenses were $45.2 million for the six months ended June 30, 2026 as compared to $29.9 million for the six months ended June 30, 2025. The increase of $15.3 million in sales, general and administrative expenses was attributable to an increase of $10.1 million in facilities and other sales, general and administrative costs for commercial readiness, and $5.2 million in personnel-related expenses.

Current report — wording carried from elsewhere

Since our inception through June 30, 2026, our operations have been financed by net proceeds of $1.0 billion 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, 2026, we had