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Stoke Therapeutics, Inc.

For the period ended Sep 30, 2026. Filed Nov 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.

Sep 30, 2026 · Filed Nov 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 2026-06-30; filed 2026-08-03.

0001193125-26-330557

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

Current report: 10-Q

Period ended 2026-09-30; filed 2026-11-03.

0001193125-26-445326

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 132 previous paragraphs and 126 current paragraphs.

90 unchanged; 30 reworded; 6 added; 11 removed; 1 previous and 0 current paragraphs rearranged.

Tables are not compared: 7 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 (6)

Current report

An additional cohort of 34 patients in Europe completed enrollment in August 2026 and an additional cohort of 5 patients in China completed enrollment in October 2026. Global enrollment in the clinical trial is now closed.

Current report

In October 2026, we announced the completion of a successful meeting with the FDA intended to align on key aspects of the planned submission of an NDA for zorevunersen. Following the discussion, we plan to use a multicomponent assessment of four prespecified Vineland-3 subdomains to evaluate changes in cognition and behavior in the Phase 3 EMPEROR study. On October 29, 2026, we presented a new exploratory analysis of Vineland-3 data from the Phase 1/2a and open-label extension (OLE) studies of zorevunersen using the multicomponent assessment.

Current report

On September 23, 2026, we announced the appointment of Bo Cumbo to our Board of Directors. Mr. Cumbo brings deep experience commercializing new medicines for people with rare genetic diseases. Concurrent with Mr. Cumbo’s appointment, Edward Kaye, M.D., retired from his role as a Director on our Board of Directors.

Current report

Other income (expense) was $3.7 million for the three months ended September 30, 2026 as compared to $3.5 million for the three months ended September 30, 2025.

Current report

Results of operations for the nine months ended September 30, 2026 and 2025

Current report

Other income (expense) was $10.9 million for the nine months ended September 30, 2026 as compared to $10.2 million for the nine months ended September 30, 2025.
Removed paragraphs (11)

Previous 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.

Previous 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.

Previous report

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

Previous 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

Previous 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.

Previous 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.

Previous report

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

Previous 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.

Previous report

Our commitments primarily consist of obligations under our agreement with the University of Southampton. As of June 30, 2026, we were unable to estimate the timing or likelihood of achieving the milestones or making future product sales. For additional information regarding our agreements, see Note 6—Commitments and Contingencies of the notes to our condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q.

Previous report

Additionally, we have entered into agreements with third-party contract manufacturers for the manufacture and processing of certain of our product candidates for preclinical testing purposes, and we have entered and will enter into other contracts in the normal course of business with contract research organizations for clinical trials and other vendors for other services and products for operating purposes. These agreements generally provide for termination or cancellation, other than for costs already incurred.
Reworded paragraphs (30)

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 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.

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, with the first two dose cohorts now complete, and the study is actively recruiting in the United Kingdom and the European Union.

Previous wording

In March 2026, we filed an automatic universal Shelf Registration Statement on Form S-3 (the “2026 Registration Statement”) with the SEC. The 2026 Registration Statement contains two prospectuses: a base prospectus, which covers the offering, issuance and sale by us 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 our common stock that may be issued and sold under the 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. We intend to use the 2026 Registration Statement for any future issuances and sales of common stock.

Current wording

In March 2026, we filed an automatic universal Shelf Registration Statement on Form S-3 (the “2026 Registration Statement”) with the SEC. The 2026 Registration Statement originally contained two prospectuses: a base prospectus, which covers the offering, issuance and sale by us 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 (the “Initial Sales Agreement Prospectus”) which covered the offering, issuance and sale by us of up to a maximum aggregate offering price of $150.0 million of our common stock that may be issued and sold under the Sales Agreement. In August 2026, we filed a prospectus supplement to the 2026 Registration Statement (the “August 2026 Sales Agreement Prospectus”), which superseded and replaced the Initial Sales Agreement Prospectus and covers the offering, issuance and sale by us of up to an additional maximum aggregate offering price of $200.0 million of our common stock that may be issued and sold under the 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. We intend to use the 2026 Registration Statement for any future issuances and sales of common stock.

Previous 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.

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 September 30, 2026, we issued approximately 4.6 million shares of common stock pursuant to the Sales Agreement and the Initial Sales Agreement Prospectus for net proceeds of $146.4 million. As of September 30, 2026, we had not sold any shares of common stock under the August 2026 Sales Agreement Prospectus. We may terminate this at-the-market program under the Sales Agreement at any time, pursuant to its terms.

Previous 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.

Current wording

As of September 30, 2026 and December 31, 2025 we had $358.8 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 $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.

Current wording

Since inception, we have had operating losses, the majority of which are attributable to research and development activities. Our net losses were $73.6 million and $38.3 million for the three months ended September 30, 2026 and 2025, respectively. For the nine months ended September 30, 2026 we had a net loss of $185.3 million and for the nine months ended September 30, 2025 we had net income of $51.0 million. As of September 30, 2026, we had an accumulated deficit of $683.0 million.

Previous 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.

Current wording

Based on our current operating plan, we believe that our cash, cash equivalents, and marketable securities of approximately $358.8 million as of September 30, 2026, will fund operations into 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

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 wording

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. We, together with Biogen, recently presented long-term clinical data at the 16th European Epilepsy Congress (EEC) and the 55th Child Neurology Society (CNS) Annual Meeting. These data included a new exploratory sub-analysis that showed substantial reductions in the most severe seizure types, which are the leading risk factor for sudden unexpected death in epilepsy (SUDEP). We will continue our educational efforts at major neurology and epilepsy congresses through the remainder of 2026 and into 2027. 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 we prepare for a potential U.S. approval and launch in early 2028.

Previous 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.

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 U.S. NDA submission in the second half of 2027.

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 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.

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 October 2026, all eight planned clinical trial sites are active in the United Kingdom, Germany, Austria, Italy and Denmark, and dosing of the first two cohorts of patients is complete. Dosing of the third cohort recently initiated and is expected to complete by the end of 2026. Subject to ongoing safety assessments, dosing in the fourth dose cohort is expected to follow in early 2027. Cohorts three and four have the potential to reach therapeutic levels of dosing, with initial safety and efficacy data from these latter cohorts anticipated in the first half of 2027. Presentations of data for STK-002 were recently delivered at the American Academy of Ophthalmology (AAO) 2026 Annual Meeting on October 11, 2026, supporting the ongoing Phase 1 OSPREY study and the disease-modifying potential of STK-002 in ADOA.

Previous 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.

Current wording

Revenue recognized under the Acadia collaboration was $2.5 million and $3.9 million for the three months ended September 30, 2026 and 2025, respectively. For the nine months ended September 30, 2026 and 2025, the Company recognized $7.5 million and $20.7 million, respectively, in revenue related to the Acadia Agreement.

Previous 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.

Current wording

Revenue recognized under the Biogen collaboration was $8.8 million and $6.7 million for the three months ended September 30, 2026 and 2025, respectively. For the nine months ended September 30, 2026 and 2025, the Company recognized $19.3 million and $162.3 million, respectively, in revenue related to the Biogen Agreement.

Previous wording

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 wording

Revenue for the three months ended September 30, 2026 was $11.3 million compared to $10.6 million for the three months ended September 30, 2025, an increase of $0.7 million. Revenue is generated from satisfying contractual obligations of the collaboration and licensing agreements with Acadia and Biogen.

Previous 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):

Current wording

Research and development expenses were $59.8 million for the three months ended September 30, 2026 as compared to $37.7 million for the three months ended September 30, 2025, an increase of $22.1 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 $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 wording

The increase in research and development expenses was attributable to an increase of $9.7 million of expenses related to zorevunersen, which are comprised of third-party services and scientific consulting fees, $7.0 million in personnel-related expenses, $3.6 million in other external program costs, $2.0 million in expenses related to our ADOA program, which are comprised of third-party services and scientific consulting fees, and an increase of $0.4 million related to other non-program costs, offset by a decrease of $0.6 million related in external third-party expenses related to SYNGAP1.

Previous 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.

Current wording

Sales, general and administrative expenses were $28.7 million for the three months ended September 30, 2026 as compared to $16.0 million for the three months ended September 30, 2025. The increase of $12.7 million in sales, general and administrative expenses was attributable to an increase of $7.9 million in other sales, general and administrative costs for commercial readiness, and $4.8 million in personnel-related expenses.

Previous 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.

Current wording

Revenue for the nine months ended September 30, 2026 was $26.8 million compared to $183.0 million for the nine months ended September 30, 2025, a decrease of $156.2 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 nine months ended September 30, 2025.

Previous wording

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 wording

Research and development expenses were $149.0 million for the nine months ended September 30, 2026 as compared to $96.2 million for the nine months ended September 30, 2025, an increase of $52.8 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 $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.

Current wording

The increase in research and development expenses was attributable to an increase of $22.0 million in expenses related to zorevunersen, which are comprised of third-party services and scientific consulting fees, $18.7 million in personnel-related expenses, $9.5 million in other external program costs, $4.2 million in expenses related to our ADOA program, which are comprised of third-party services and scientific consulting fees, an increase of $0.1 million in external third-party expenses related to SYNGAP1, offset by a decrease of $1.8 million related to other non-program costs.

Previous wording

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 wording

Sales, general and administrative expenses were $74.0 million for the nine months ended September 30, 2026 as compared to $45.9 million for the nine months ended September 30, 2025. The increase of $28.1 million in sales, general and administrative expenses was attributable to an increase of $17.7 million in other sales, general and administrative costs for commercial readiness, and $10.3 million in personnel-related expenses.

Previous 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.

Current wording

Since our inception through September 30, 2026, our operations have been financed by net proceeds of $1.08 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 September 30, 2026, we had $358.8 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, 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.

Current wording

We have primarily incurred losses since our inception in June 2014 and, as of September 30, 2026, we had an accumulated deficit of $683.0 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 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.

Current wording

Based on our current operating plan, we believe that our cash, cash equivalents, and marketable securities of approximately $358.8 million as of September 30, 2026, will fund operations into 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 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.

Current wording

During the nine months ended September 30, 2026, cash used in operating activities was $179.9 million. This was attributable to a net loss of $185.3 million, a net change of $27.4 million in our net operating assets and liabilities offset by non-cash charges of $32.8 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, 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

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.

Current wording

Our investing activities during the nine months ended September 30, 2026 consisted primarily of $158.7 million from the maturity of marketable securities offset by $104.0 million of purchases of marketable securities and $5.2 million of purchases of property plant and equipment.

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 $0.4 million of purchases of property plant and equipment offset by $99.2 million from the maturity of marketable securities.

Previous 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”).

Current wording

Our financing activities during the nine months ended September 30, 2026 consisted primarily of $146.4 million in proceeds from the controlled equity offering sales agreement, $8.6 million from the exercise of stock options and $1.4 million in proceeds from our Employee Stock Purchase Plan (the “ESPP”).

Previous 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.

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 ESPP.

Previous wording

In December 2023, we entered into an agreement to extend the term of the 38,000 square foot lease for a period of two years commencing on January 1, 2025 and ending on December 31, 2026. In December 2023, we recognized a right-of-use asset and operating lease liability of $4.1 million.

Current wording

In December 2023, we entered into an agreement to extend the term of the 38,000 square foot lease for a period of two years commencing on January 1, 2025 and ending on December 31, 2026. In December 2023, we recognized a right-of-use asset and operating lease liability of $4.1 million. In August 2026, we further amended the term of the lease for a period of two months commencing on January 1, 2027, and ending on February 28, 2027.

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 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.

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 and 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.
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Previous report — wording carried 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