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Dyne Therapeutics, Inc.

For the period ended Jun 30, 2026. Filed Jul 29, 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 Jul 29, 2026

DYN$18.07+4.1%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 2026-05-11.

0001193125-26-215702

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

Current report: 10-Q

Period ended 2026-06-30; filed 2026-07-29.

0001193125-26-323639

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 135 previous paragraphs and 147 current paragraphs.

105 unchanged; 20 reworded; 15 added; 10 removed; 0 previous and 7 current paragraphs rearranged.

Tables are not compared: 4 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 (15)

Current report

Z-rostudirsen is being evaluated in the long-term extension portion of the DELIVER trial, a global Phase 1/2 clinical trial designed to be registrational, and the FORZETTO trial, a global Phase 3 clinical trial designed to be confirmatory. We have aligned with the FDA on the FORZETTO trial design and protocol. The registrational expansion cohort of the global Phase 1/2 DELIVER clinical trial of z-rostudirsen met its primary endpoint. Data from the DELIVER trial served as the basis for a biologics license application, or BLA, for potential U.S. Accelerated Approval.

Current report

In May 2026, we initiated our Phase 3 FORZETTO clinical trial, a global, randomized, placebo-controlled, double-blind, confirmatory Phase 3 trial designed to assess the efficacy, safety, and tolerability of z-rostudirsen administered intravenously to ambulatory male participants with DMD amenable to exon 51 skipping. The trial will enroll approximately 90 participants 4 to 18 years of age who will be randomized 1:1 to receive 20 mg/kg of z-rostudirsen or placebo every four weeks (Q4W). The primary endpoint is the change from baseline in rise from floor (RFF) velocity at Week 73. Secondary endpoints include changes from baseline in stride velocity 95th centile (SV95C), North Star Ambulatory Assessment (NSAA) total score, 10-meter walk/run (10MWR) velocity, four-stair climb (4SC) velocity and forced vital capacity percent predicted (FVC%p), as well as additional functional and patient-reported outcome measures. Following the 72-week double-blind placebo-controlled treatment period, participants will be eligible to enroll in a 96-week open-label long-term extension. We have aligned with the FDA on the FORZETTO Phase 3 trial design and protocol. FORZETTO is intended to serve as a confirmatory trial to support the potential conversion of Accelerated Approval to traditional approval in the United States and to support ex-U.S. marketing applications.

Current report

In March 2026, we initiated our Phase 3 HARMONIA clinical trial, a global, randomized, placebo controlled, double-blind, confirmatory Phase 3 trial designed to assess the multi-system efficacy, safety, and tolerability of z-basivarsen administered intravenously to individuals with DM1. We began dosing patients in July 2026. The trial will enroll approximately 150 participants age 16 and older who will be randomized 1:1 to receive 6.8 mg/kg of z-basivarsen or placebo every eight weeks (Q8W). The primary endpoint is the change from baseline in the five times sit to stand (5xSTS) test at week 49. Secondary endpoints include video hand opening time, quantitative muscle testing, the 10-Meter Walk/Run test, the Myotonic Dystrophy Health Index, and additional patient- and clinician-reported outcomes. The trial also includes a broad set of exploratory endpoints designed to assess multiple domains of DM1 CNS impact. Following the 48-week double-blind placebo-controlled treatment period, patients will be eligible to enroll in a 24-week long-term extension.

Current report

In July 2026, we announced that we received clearance from the FDA for our investigational new drug, or IND, application to initiate a Phase 1 clinical trial for DYNE-302 in FSHD. DYNE-302 leverages the same FORCE platform as our first two clinical programs, z-rostudirsen in DMD amenable to exon 51 skipping and z-basivarsen in DM1. We plan to evaluate DYNE-302 in a Phase 1 randomized, placebo-controlled, double-blind, multiple ascending dose clinical trial in ambulatory adult individuals with FSHD with a primary endpoint of safety and tolerability. The trial will also assess pharmacokinetics and pharmacodynamics, including change from baseline in muscle DUX4 transcriptome and plasma KHDC1L levels.

Current report

In the first cohort, nine participants will receive three intravenous doses administered every four weeks (Q4W), randomized 2:1 to DYNE-302 1.5 mg/kg (approximate siRNA dose) or placebo. Following the completion of this cohort, we intend to evaluate higher dosing and less frequent administration. Participants who complete the placebo-controlled period may enter an open-label long-term extension and receive DYNE-302 for up to an additional 96 weeks. We intend to pursue a traditional approval pathway in the U.S. for DYNE-302.

Current report

In July 2026, we completed an underwritten public offering, pursuant to which we issued and sold 21,045,000 shares of our common stock, which included 2,745,000 shares issued upon the exercise in full by the underwriters of their option to purchase additional shares of common stock in the offering, which we refer to as the July 2026 offering. We estimate that the net proceeds from the offering were approximately $405.0 million, after deducting underwriting discounts and commissions and offering expenses payable by us.

Current report

Interest expense for the three months ended June 30, 2026 was $4.6 million due to our Loan Agreement with Hercules and interest owed on our vendor financing arrangement. Interest expense for the three months ended June 30, 2025 was less than $0.1 million due to our Loan Agreement with Hercules.

Current report

The following table summarizes our research and development expenses for the six months ended June 30, 2026 and 2025:

Current report

Interest expense for the six months ended June 30, 2026 was $8.8 million due to our Loan Agreement with Hercules and interest owed on our vendor financing arrangement. Interest expense for the six months ended June 30, 2025 was less than $0.1 million due to our Loan Agreement with Hercules.

Current report

Other expense for the six months ended June 30, 2026 and 2025 was $0.2 million and $1.7 million, respectively, due to realized foreign currency losses.

Current report

In July 2026, we completed the July 2026 offering, pursuant to which we issued and sold 21,045,000 shares of our common stock. We estimate that the net proceeds from the offering were approximately $405.0 million, after deducting underwriting discounts and commissions and offering expenses payable by us.
Removed paragraphs (10)

Previous report

Interest expense for the three months ended March 31, 2026 was $4.2 million due to our entry into the Loan Agreement with Hercules in June 2025. No interest expense was incurred in the three months ended March 31, 2025.
Reworded paragraphs (20)

Previous wording

Z-rostudirsen is currently being evaluated in the DELIVER trial, a global Phase 1/2 clinical trial which is designed to be registrational. Following a positive pre-BLA meeting with the FDA, we continue to plan to submit a biologics license application, or BLA, to the FDA for U.S. Accelerated Approval in the second quarter of 2026 based on dystrophin as a surrogate endpoint. We continue to expect a potential U.S. launch of z-rostudirsen in the first quarter of 2027, assuming FDA grants priority review and FDA approval is received on the anticipated timeline. Further, we plan to initiate a global confirmatory Phase 3 clinical trial of z-rostudirsen in the second quarter of 2026, and we have aligned with the FDA on the Phase 3 trial design and protocol. We continue to pursue approval pathways outside of the United States for z-rostudirsen.

Current wording

In July 2026, we announced that the FDA accepted for review our BLA for z-rostudirsen for the treatment of individuals with DMD amenable to exon 51 skipping. The FDA has granted the BLA priority review and assigned a Prescription Drug User Fee Act, or PDUFA, target action date of January 21, 2027. We continue to expect a potential U.S. launch of z-rostudirsen in the first quarter of 2027, assuming FDA approval is received on the anticipated timeline. We continue to pursue approval pathways outside of the United States for z-rostudirsen.

Previous wording

Z-basivarsen is being evaluated in the ACHIEVE trial, a global Phase 1/2 clinical trial designed to be registrational, and the HARMONIA trial, a global Phase 3 clinical trial designed to be confirmatory, which was initiated in March 2026. We have aligned with the FDA on the HARMONIA trial design and protocol. We have also reached our enrollment target of 60 participants in the registrational expansion cohort, or REC, of the ACHIEVE trial. We plan to allow any participants currently in screening to enroll if they meet all eligibility criteria. As a result, we expect to complete enrollment of more than 60 participants in the second quarter of 2026. We plan to announce data from the REC in the first quarter of 2027 to support a potential BLA submission to the FDA for U.S. Accelerated Approval early in the third quarter of 2027. We anticipate a potential U.S. launch of z-basivarsen in the first quarter of 2028, assuming we receive favorable data from the REC, priority review is granted, and FDA approval is received on the anticipated timeline. We continue to pursue approval pathways outside of the United States for z-basivarsen.

Current wording

In June 2026, we completed enrollment of 71 participants in the registrational expansion cohort, or REC, of the ACHIEVE trial. We plan to announce data from the REC in the first quarter of 2027 to support a potential BLA submission to the FDA for U.S. Accelerated Approval in the third quarter of 2027. We anticipate a potential U.S. launch of z-basivarsen in the first half of 2028, assuming we receive favorable data from the REC, priority review is granted, and FDA approval is received on the anticipated timeline. We continue to pursue approval pathways outside of the United States for z-basivarsen.

Previous wording

We continue to explore additional applications for our proprietary FORCE platform, along with further iterations. To that end, we have identified two compounds, which we refer to as Conjugate 1 and Conjugate 2, utilizing a Fab conjugated to microtubule associated protein tau (MAPT) siRNA designed to downregulate expression of all MAPT isoforms. Conjugate 1 utilizes our FORCE platform with the same Fab as our other programs. Conjugate 2 utilizes a modified form of the FORCE TfR1-binding Fab that has been further optimized for enhanced central nervous system delivery and potential use in neurological indications. In preclinical studies, both conjugates achieved robust MAPT RNA knockdown (approximately 75% for Conjugate 2) in both mice and nonhuman primates, with widespread and consistent delivery across brain regions, including the deep brain. Subcutaneous administration in mice achieved an equivalent reduction in MAPT RNA as compared to intravenous administration in mice.

Current wording

We continue to explore additional applications for our proprietary FORCE platform, along with further iterations. To that end, we have identified two compounds, which we refer to as Conjugate 1 and Conjugate 2, utilizing a Fab conjugated to microtubule associated protein tau, or MAPT, siRNA designed to downregulate expression of all MAPT isoforms. Conjugate 1 utilizes our FORCE platform with the same Fab as our other programs. Conjugate 2 utilizes a modified form of the FORCE TfR1-binding Fab that has been further optimized for enhanced central nervous system delivery and potential use in neurological indications. In preclinical studies, both conjugates achieved robust MAPT RNA knockdown (approximately 75% for Conjugate 2) in both mice and nonhuman primates, with widespread and consistent delivery across brain regions, including the deep brain. Subcutaneous administration in mice achieved an equivalent reduction in MAPT RNA as compared to intravenous administration in mice.

Previous wording

Since our inception, we have incurred significant operating losses. Our ability to generate any product revenue or product revenue sufficient to achieve profitability will depend on the successful development and eventual commercialization of one or more product candidates. For the three months ended March 31, 2026 and 2025, we reported net losses of $120.9 million and $115.4 million, respectively. As of March 31, 2026, we had an accumulated deficit of $1.5 billion.

Current wording

Since our inception, we have incurred significant operating losses. Our ability to generate any product revenue or product revenue sufficient to achieve profitability will depend on the successful development and eventual commercialization of one or more product candidates. For the six months ended June 30, 2026 and 2025, we reported net losses of $299.4 million and $226.2 million, respectively. As of June 30, 2026, we had an accumulated deficit of $1.7 billion.

Previous wording

We believe that our existing cash, cash equivalents and marketable securities will enable us to fund our operating expenses, debt service obligations and capital expenditure requirements into the first quarter of 2028.

Current wording

We believe that our cash, cash equivalents and marketable securities as of June 30, 2026, as well as the approximately $405.0 in net proceeds from the July 2026 offering, will enable us to fund our operating expenses, debt service obligations and capital expenditure requirements into the second quarter of 2028.

Previous wording

The U.S. administration has announced or imposed multiple series of tariffs on U.S. trading partners, and there is uncertainty as to whether, when, or to what extent they may apply to our supply chain. In response, several countries have threatened or imposed retaliatory measures, and multiple states have challenged these tariffs in court. While we have not experienced, and do not currently expect to experience, any significant direct impact from these tariffs or retaliatory measures, we could experience a negative impact on our costs of materials and production processes and supply chain disruptions, and the uncertainty surrounding the future of U.S. trade policy may itself negatively impact our supply chain operations and the costs of materials and production processes. Supply chain disruptions may impact the development, testing and clinical trials of our product candidates, and regulatory approval or commercial launch of any resulting product may be delayed or not obtained, which could significantly harm our business. The full extent of the future impact of these and other threatened measures remains uncertain. We continue to monitor these tariffs, retaliatory measures, and related litigation, and their possible effects on our business. See Part II, Item 1A. “Risk Factors—Risks related to regulatory approval and other regulatory and legal compliance matters” in this Quarterly Report for additional risks associated with current U.S. trade policy.

Current wording

The U.S. administration has announced or imposed multiple series of tariffs on U.S. trading partners. In response, several countries have threatened or imposed retaliatory measures, and multiple states have challenged these tariffs in court. While we have not experienced, and do not currently expect to experience, significant direct impact from these tariffs or retaliatory measures, the uncertainty surrounding the future of U.S. trade policy may negatively impact our supply chain operations and the costs of materials and production processes. Supply chain disruptions may impact the development, testing and clinical trials of our product candidates, and regulatory approval or commercial launch of any resulting product may be delayed or not obtained, which could significantly harm our business. The full extent of the future impact of these and other threatened measures remains uncertain. We continue to monitor these tariffs, retaliatory measures, and related litigation, and their possible effects on our business. See Part II, Item 1A. “Risk Factors—Risks related to regulatory approval and other regulatory and legal compliance matters” in this Quarterly Report for additional risks associated with current U.S. trade policy.

Previous wording

The increase in platform and external research and development expenses in the three months ended March 31, 2026 compared to the three months ended March 31, 2025 was due to increased external research activity associated with our preclinical programs and product candidates, primarily DYNE-302 and DYNE-401. The increase in personnel-related expenses was primarily due to increased headcount in our research and development function of 37 employees and higher stock-based compensation expense for awards granted to new hires and existing employees. The increase in facility-related and other expenses was primarily due to the increased costs of supporting a larger number of research and development personnel.

Current wording

The increase in platform and external research and development expenses in the three months ended June 30, 2026 compared to the three months ended June 30, 2025 was due to increased external research activity associated with our preclinical programs and product candidates. The increase in personnel-related expenses was primarily due to the increase of 39 employees in our research and development headcount and higher stock-based compensation expense for awards granted to new hires and existing employees. The increase in facility-related and other expenses was primarily due to the increased costs of supporting a larger number of research and development personnel.

Previous wording

The increase in personnel-related and stock-based compensation expenses in the three months ended March 31, 2026 compared to the three months ended March 31, 2025 was due to increased headcount in our general and administrative function of 34 employees. Professional and consulting fees increased due to the higher consulting costs for preparation activities for the potential launch of z-rostudirsen and the overall growth of the organization in the three months ended March 31, 2026. Facility-related and other expenses increased due to higher costs of supporting a larger number of general and administrative personnel in the three months ended March 31, 2026.

Current wording

The increase in personnel-related and stock-based compensation expenses in the three months ended June 30, 2026 compared to the three months ended June 30, 2025 was due to the increase of 44 employees in our general and administrative headcount. Professional and consulting fees increased due to higher consulting costs for the preparation activities for the potential launch of z-rostudirsen and the overall growth of the organization in the three months ended June 30, 2026. Facility-related and other expenses increased due to higher costs of supporting a larger number of general and administrative personnel in the three months ended June 30, 2026.

Previous wording

Interest income for the three months ended March 31, 2026 and 2025 was $8.8 million and $7.1 million, respectively, due to interest earned on invested cash balances. The increase in interest income was due to increased cash, cash equivalents and marketable securities balances in the three months ended March 31, 2026 compared to the three months ended March 31, 2025.

Current wording

Interest income for the three months ended June 30, 2026 and 2025 was $7.8 million and $6.6 million, respectively, due to interest earned on invested cash balances. The increase in interest income was due to increased cash, cash equivalents and marketable securities balances in the three months ended June 30, 2026 compared to the three months ended June 30, 2025.

Previous wording

Other income for the three months ended March 31, 2026 and 2025 was $0.1 million in each quarter due to realized foreign currency gains.

Current wording

Other expense, net, for the three months ended June 30, 2026 and 2025 was $0.1 million and $1.6 million, respectively, in each quarter due to realized foreign currency losses.

Previous wording

Expenses related to z-rostudirsen increased in the three months ended March 31, 2026 compared to the three months ended March 31, 2025. This was attributable to higher manufacturing activity in the first quarter of 2026 for drug substance conjugation primarily for clinical supply for the long-term extension of the DELIVER trial and the anticipated commencement of the global confirmatory Phase 3 clinical trial of z-rostudirsen in the second quarter of 2026. Additionally, higher clinical costs were incurred in the first quarter of 2026 due to z-rostudirsen Phase 3 start-up costs. Expenses related to z-basivarsen decreased in the three months ended March 31, 2026 compared to the three months ended March 31, 2025. This was primarily attributable to higher manufacturing activity in the first quarter of 2025 for process performance qualification batches of oligonucleotide payload and the timing of drug substance conjugation activities, partially offset by higher clinical trial costs due to the ongoing enrollment of the registrational expansion cohort of the ACHIEVE trial and the commencement of the HARMONIA trial in March 2026.

Current wording

Expenses related to z-rostudirsen increased in the six months ended June 30, 2026 compared to the six months ended June 30, 2025. This was attributable to increased manufacturing activity of drug substance and components in the six months ended June 30, 2026, primarily for clinical supply for the long-term extension of the DELIVER trial and the global confirmatory Phase 3 clinical trial of z-rostudirsen, FORZETTO, that commenced in the second quarter of 2026. Additionally, higher clinical costs were incurred in the six months ended June 30, 2026 due to the commencement of the FORZETTO trial. Expenses related to z-basivarsen decreased in the six months ended June 30, 2026 compared to the six months ended June 30, 2025. This was primarily attributable to higher manufacturing activity for process performance qualification batches of oligonucleotide payload and the timing of drug substance conjugation activities in the six months ended June 30, 2025, which were partially offset by higher clinical costs in the six months ended June 30, 2026 due to the completion of enrollment of the registrational expansion cohort of the ACHIEVE trial in the second quarter of 2026 and increased enrollment in the HARMONIA trial.

Previous wording

Since our inception, we have incurred significant operating losses. We expect to incur significant expenses and operating losses for the foreseeable future as we support our continued research activities and development of our product candidates and platform. We have not yet commercialized any product candidates, and we do not expect to generate revenue from sales of any product candidates at least until 2027, if at all. To date, we have funded our operations primarily with proceeds from sales of equity securities and our borrowing under the Loan Agreement with Hercules. As of March 31, 2026 we had cash, cash equivalents and marketable securities of $972.2 million.

Current wording

Since our inception, we have incurred significant operating losses. We expect to incur significant expenses and operating losses for the foreseeable future as we support our continued research activities and development of our product candidates and platform. We have not yet commercialized any product candidates, and we do not expect to generate revenue from sales of any product candidates at least until 2027, if at all. To date, we have funded our operations primarily with proceeds from sales of equity securities and our borrowing under the Loan Agreement with Hercules. As of June 30, 2026 we had cash, cash equivalents and marketable securities of $898.5 million.

Previous wording

In November 2021, we entered into an Open Market Sale Agreement SM, or the Sales Agreement, with Jefferies LLC, or Jefferies. On March 5, 2024, we filed a universal shelf registration statement on Form S-3, or the 2024 Shelf Registration Statement, and included a prospectus relating to the Sales Agreement. Under the 2024 Shelf Registration Statement, we may offer and sell debt securities, common stock, preferred stock, units and/or warrants from time to time at an indeterminate aggregate offering price in one or more offerings. In November 2024, we filed a prospectus supplement relating to the Sales Agreement, pursuant to which, in accordance with the Sales Agreement, we may offer and sell shares of our common stock having an aggregate offering price of up to $300.0 million, which we refer to as our at-the-market offering program. Sales of common stock under the Sales Agreement through Jefferies may be made by any method that is deemed an “at-the-market” offering as defined in Rule 415(a)(4) under the Securities Act. During the three months ended March 31, 2026, we did not issue or sell any shares of common stock pursuant to the Sales Agreement.

Current wording

In November 2021, we entered into an Open Market Sale Agreement SM, or the Sales Agreement, with Jefferies LLC, or Jefferies. On March 5, 2024, we filed a universal shelf registration statement on Form S-3, or the 2024 Shelf Registration Statement, and included a prospectus relating to the Sales Agreement. Under the 2024 Shelf Registration Statement, we may offer and sell debt securities, common stock, preferred stock, units and/or warrants from time to time at an indeterminate aggregate offering price in one or more offerings. In November 2024, we filed a prospectus supplement relating to the Sales Agreement, pursuant to which, in accordance with the Sales Agreement, we may offer and sell shares of our common stock having an aggregate offering price of up to $300.0 million, which we refer to as our at-the-market offering program. Sales of common stock under the Sales Agreement through Jefferies may be made by any method that is deemed an “at-the-market” offering as defined in Rule 415(a)(4) under the Securities Act. During the six months ended June 30, 2026, we did not issue or sell any shares of common stock pursuant to the Sales Agreement.

Previous wording

In June 2025, we entered into the Loan Agreement with Hercules, in its capacity as administrative agent and collateral agent and as a lender, and certain other financial institutions that from time to time become parties to the Loan Agreement as lenders, which we refer to collectively as the Lenders. In December 2025, we entered into the First Amendment to the Loan Agreement with Hercules. The Loan Agreement, as amended by the First Amendment, provides for term loans in an aggregate principal amount of up to $275.0 million under multiple tranches, available as follows: (i) an initial term loan tranche funded on the closing date of the Loan Agreement in aggregate principal amount of $100.0 million; (ii) subject to the achievement of specified clinical, regulatory and commercial milestones, and after the borrowing of the second term loan tranche of $50.0 million in December 2025, two additional term loan tranches totaling up to $75.0 million; and (iii) subject to approval by the Lenders’ investment committee in their discretion, a final term loan tranche of up to $50.0 million. At March 31, 2026, the principal term loan balance was $150.0 million. Refer to Note 7, “Debt” in the accompanying notes to the condensed consolidated financial statements for a discussion of the Loan Agreement with Hercules.

Current wording

In June 2025, we entered into the Loan Agreement with Hercules, in its capacity as administrative agent and collateral agent and as a lender, and certain other financial institutions that from time to time become parties to the Loan Agreement as lenders, which we refer to collectively as the Lenders. In December 2025, we entered into the First Amendment to the Loan Agreement with Hercules. In June 2026, we entered into the Second Amendment to the Loan Agreement with Hercules. The Loan Agreement, as amended by the First and the Second Amendment, provides for term loans in an aggregate principal amount of up to $400.0 million under multiple tranches, available as follows: (i) an initial term loan tranche funded on the closing date of the Loan Agreement in aggregate principal amount of $100.0 million; (ii) subject to the achievement of specified clinical, regulatory and commercial milestones, and after the borrowing of the second term loan tranche of $50.0 million in December 2025 and third term loan tranche of $50.0 million in June 2026, three additional term loan tranches totaling up to $125.0 million; and (iii) subject to approval by the Lenders’ investment committee in their discretion, a final term loan tranche of up to $75.0 million. At June 30, 2026, the principal term loan balance was $200.0 million. Refer to Note 7, “Debt” in the accompanying notes to the condensed consolidated financial statements for a discussion of the Loan Agreement with Hercules.

Previous wording

During the three months ended March 31, 2026, operating activities used $144.9 million of cash, due to our net loss of $120.9 million and net cash used by changes in our operating assets and liabilities of $37.6 million, partially offset by non-cash charges of $13.6 million. Net cash used in changes in our operating assets and liabilities primarily consisted of a $32.8 million increase in prepaid expenses and other current assets and a $4.8 million decrease in accounts payable and other liabilities. During the three months ended March 31, 2025, operating activities used $105.9 million of cash, due to our net loss of $115.4 million and net cash used by changes in our operating assets and liabilities of $3.6 million, partially offset by non-cash charges of $13.1 million. Net cash used by changes in our operating assets and liabilities primarily consisted of a $8.6 million decrease in accounts payable and other liabilities and a $5.0 million decrease in prepaid expenses and other current assets. Changes in our operating assets and liabilities during these periods were generally due to the growth of our business, increased clinical trial activity, increased manufacturing activities, advancement of our product candidates, the timing of vendor invoices and payments and annual bonus payments.

Current wording

During the six months ended June 30, 2026, operating activities used $275.2 million of cash, due to our net loss of $299.4 million and net cash used by changes in our operating assets and liabilities of $4.8 million, partially offset by non-cash charges of $29.0 million. Net cash used in changes in our operating assets and liabilities primarily consisted of a $23.4 million increase in prepaid expenses and other current assets, partially offset by a $14.2 million increase in accounts payable and other liabilities and a $4.4 million decrease in other non-current assets. During the six months ended June 30, 2025, operating activities used $200.6 million of cash, due to our net loss of $226.2 million, partially offset by non-cash charges of $23.6 million and net cash provided by changes in our operating assets and liabilities of $2.0 million. Net cash provided by changes in our operating assets and liabilities primarily consisted of a $2.8 million decrease in prepaid expenses and other current assets, partially offset by a $0.8 million decrease in accounts payable and other liabilities. Changes in our operating assets and liabilities during these periods were generally due to the growth of our business, increased clinical trial activity, increased manufacturing activities, advancement of our product candidates and the timing of vendor invoices and payments.

Previous wording

During the three months ended March 31, 2026, net cash used in investing activities was $2.4 million due to purchases of marketable securities of $64.3 million and purchases of property and equipment of $0.2 million, partially offset by maturities of marketable securities of $33.5 million and sales of marketable securities of $28.6 million. During the three months ended March 31, 2025, net cash provided by investing activities was $1.5 million due to maturities of marketable securities of $65.4 million and sales of marketable securities of $3.0 million, partially offset by purchases of marketable securities of $66.0 million and purchases of property and equipment of $1.0 million.

Current wording

During the six months ended June 30, 2026, net cash used in investing activities was $7.2 million due to purchases of marketable securities of $107.1 million and purchases of property and equipment of $1.7 million, partially offset by maturities of marketable securities of $58.2 million and sales of marketable securities of $43.4 million. During the six months ended June 30, 2025, net cash provided by investing activities was $8.2 million due to maturities of marketable securities of $107.7 million and sales of marketable securities of $8.5 million, partially offset by purchases of marketable securities of $106.9 million and purchases of property and equipment of $1.0 million.

Previous wording

During the three months ended March 31, 2026, net cash provided by financing activities was $7.1 million, including $6.8 million in proceeds from our vendor financing arrangement that we entered into during the quarter and $1.3 million in proceeds received from stock option exercises. These cash inflows were partially offset by $0.6 million in repayments related to our vendor financing arrangement and the payment of $0.4 million of issuance costs from the follow-on public offering we completed in December 2025, pursuant to which we issued and sold 21,827,549 shares of our common stock and from which we received net proceeds of $377.7 million, after deducting underwriting discounts and commissions and offering expenses paid by us. During the three months ended March 31, 2025, net cash provided by financing activities was $141.2 million, consisting of $140.6 million in aggregate net proceeds from sales of our common stock under our at-the-market offering program and $0.6 million in net proceeds received from stock option exercises.

Current wording

During the six months ended June 30, 2026, net cash provided by financing activities was $65.2 million, including $49.5 million in net proceeds from the third term loan tranche under our Loan Agreement with Hercules, $18.0 million in proceeds from our vendor financing arrangement and $2.0 million in proceeds received from stock option exercises. These cash inflows were partially offset by $3.9 million in repayments related to our vendor financing arrangement and the payment of $0.4 million of issuance costs from the follow-on public offering we completed in December 2025, pursuant to which we issued and sold 21,827,549 shares of our common stock and from which we received net proceeds of $377.7 million, after deducting underwriting discounts and commissions and offering expenses paid by us. During the six months ended June 30, 2025, net cash provided by financing activities was $242.0 million, consisting of $140.6 million in aggregate net proceeds from sales of our common stock under our at-the-market offering program, $98.8 million in net proceeds from the initial term loan tranche under the Loan Agreement with Hercules and $2.6 million in net proceeds received from stock option exercises.

Previous wording

We believe that our existing cash, cash equivalents and marketable securities will enable us to fund our operating expenses, debt service obligations, and capital expenditure requirements into the first quarter of 2028. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect.

Current wording

We believe that our cash, cash equivalents and marketable securities as of June 30, 2026, as well as the approximately $405.0 in net proceeds from the July 2026 offering, will enable us to fund our operating expenses, debt service obligations, and capital expenditure requirements into the second quarter of 2028. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect.

Previous wording

On January 15, 2025, we entered into a master manufacturing services agreement with a CMO which secures capacity at the CMO's manufacturing facilities for certain of our product candidates and components thereof. As of March 31, 2026, we have paid $27.7 million towards current assets and $31.2 million towards non-current assets under this agreement and pursuant to a mutually agreed rolling forecast we have committed to pay an additional $107.6 million in fees through March 2028. In specified termination circumstances, the agreement requires us to pay the CMO for services completed, the cost of the CMO's raw materials that cannot be repurposed and specified cancellation fees. This agreement formalizes and supersedes a letter agreement that we entered into with the CMO on July 18, 2024.

Current wording

On January 15, 2025, we entered into a master manufacturing services agreement with a CMO which secures capacity at the CMO's manufacturing facilities for certain of our product candidates and components thereof. As of June 30, 2026, we have paid $26.9 million towards non-current assets under this agreement and pursuant to a mutually agreed rolling forecast we have committed to pay an additional $99.7 million in fees through June 2028. In specified termination circumstances, the agreement requires us to pay the CMO for services completed, the cost of the CMO's raw materials that cannot be repurposed and specified cancellation fees. This agreement formalizes and supersedes a letter agreement that we entered into with the CMO on July 18, 2024.

Previous wording

On October 31, 2025, we entered into another master manufacturing services agreement with a CMO which also secures capacity at the CMO's manufacturing facilities for certain of our product candidate components. The agreement obligates us to compensate the CMO for producing certain of our product candidate components pursuant to a mutually agreed rolling forecast, pursuant to which, as of March 31, 2026, we have committed to pay an additional $52.7 million in fees through September 2027. In specified termination circumstances, the agreement requires us to pay the CMO for services completed, the cost of the CMO's raw materials that cannot be repurposed, capital equipment and certain manufacturing activities previously committed to.

Current wording

On October 31, 2025, we entered into another master manufacturing services agreement with a CMO which also secures capacity at the CMO's manufacturing facilities for certain of our product candidate components. The agreement obligates us to compensate the CMO for producing certain of our product candidate components pursuant to a mutually agreed rolling forecast, pursuant to which, as of June 30, 2026, we have committed to pay an additional $59.8 million in fees through December 2027. In specified termination circumstances, the agreement requires us to pay the CMO for services completed, the cost of the CMO's raw materials that cannot be repurposed, capital equipment and certain manufacturing activities previously committed to.
Rearranged current paragraphs (7)

Current report — wording carried from elsewhere

Z-basivarsen is being evaluated in the ACHIEVE trial, a global Phase 1/2 clinical trial designed to be registrational, and the HARMONIA trial, a global Phase 3 clinical trial designed to be confirmatory. We have aligned with the FDA on the HARMONIA trial design and protocol.

Current report — wording carried from elsewhere

Expenses related to z-rostudirsen increased in the three months ended June 30, 2026 compared to the three months ended June 30, 2025. This was attributable to increased manufacturing activity in the second quarter of 2026 of drug components primarily for clinical supply for the long-term extension of the DELIVER trial and the global confirmatory Phase 3 clinical trial of z-rostudirsen, FORZETTO, that commenced in the second quarter of 2026. Additionally, higher clinical costs were incurred in the second quarter of 2026 due to the commencement of the FORZETTO trial. Expenses related to z-basivarsen increased in the three months ended June 30, 2026 compared to the three months ended June 30, 2025. This was primarily attributable to increased manufacturing activity in the second quarter of 2026 of drug substance and components primarily for clinical supply for the registrational expansion cohort of the ACHIEVE trial and the global confirmatory Phase 3 clinical trial of z-basivarsen, HARMONIA, that commenced in the first quarter of 2026. Additionally, higher clinical costs were incurred in the second quarter of 2026 due to the completion of enrollment of the registrational expansion cohort of the ACHIEVE trial in the second quarter of 2026 and increased enrollment in the HARMONIA trial.

Current report — wording carried from elsewhere

The following table summarizes our results of operations for the six months ended June 30, 2026 and 2025:

Current report — wording carried from elsewhere

The increase in platform and external research and development expenses in the six months ended June 30, 2026 compared to the six months ended June 30, 2025 was primarily due to increased external research activity associated with our preclinical programs and product candidates. The increase in personnel-related expenses was primarily due to the increase of 39 employees in our research and development headcount and higher stock-based compensation expense for awards granted to new hires and existing employees. The increase in facility-related and other expenses was primarily due to the increased costs of supporting a larger number of research and development personnel.

Current report — wording carried from elsewhere

The following table summarizes our general and administrative expenses for the six months ended June 30, 2026 and 2025:

Current report — wording carried from elsewhere

The increase in personnel-related and stock-based compensation expenses in the six months ended June 30, 2026 compared to the six months ended June 30, 2025 was due to the increase of 44 employees in our general and administrative headcount. Professional and consulting fees increased due to higher consulting costs for the preparation activities for the potential launch of z-rostudirsen and the overall growth of the organization in the six months ended June 30, 2026. Facility-related and other expenses increased due to higher costs of supporting a larger number of general and administrative personnel in the six months ended June 30, 2026.

Current report — wording carried from elsewhere

Interest income for the six months ended June 30, 2026 and 2025 was $16.5 million and $13.7 million, respectively, due to interest earned on invested cash balances. The increase in interest income was due to an increased cash, cash equivalents and marketable securities balance throughout the six months ended June 30, 2026 compared to the six months ended June 30, 2025.