Solid Biosciences Inc

Previous wording

Some of the statements contained in this discussion and analysis or set forth elsewhere in this quarterly report on Form 10-Q, including information with respect to our plans and strategy for our business, constitute forward looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, or the Securities Act, and Section 21E of the Securities Exchange Act of 1934, as amended. We have based these forward-looking statements on our current expectations and projections about future events. The following information and any forward-looking statements should be considered in light of factors discussed elsewhere in this quarterly report on Form 10-Q particularly including those risks identified in Part II, Item 1A “Risk Factors” and our other filings with the Securities and Exchange Commission, or the SEC.

Current wording

Some of the statements contained in this discussion and analysis or set forth elsewhere in this quarterly report on Form 10-Q, including information with respect to our plans and strategy for our business, constitute forward looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (the "Securities Act"), and Section 21E of the Securities Exchange Act of 1934, as amended (the "Exchange Act"). We have based these forward-looking statements on our current expectations and projections about future events. The following information and any forward-looking statements should be considered in light of factors discussed elsewhere in this quarterly report on Form 10-Q particularly including those risks identified in Part II, Item 1A “Risk Factors” and our other filings with the Securities and Exchange Commission (the "SEC").

Previous wording

Solid Biosciences Inc. (we, us, our, the Company, or Solid) is a life sciences company focused on advancing a portfolio of current and future gene therapy candidates, which we refer to collectively as our Candidates, including SGT-003 for the treatment of Duchenne muscular dystrophy, or Duchenne, SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia, or CPVT, and additional assets for the treatment of cardiac and other diseases, at different stages of development, with varying levels of investment. We are advancing our diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management and care. Patient-focused and founded by those directly impacted by Duchenne, our mission is to improve the daily lives of patients living with these devastating diseases.

Current wording

We are a life sciences company focused on advancing a portfolio of current and future gene therapy candidates, which we refer to collectively as our Candidates, including SGT-003 for the treatment of Duchenne muscular dystrophy ("Duchenne"), SGT-212 for the treatment of Friedreich's ataxia ("FA"), SGT-501 for the treatment of Catecholaminergic polymorphic ventricular tachycardia ("CPVT"), SGT-601 for the treatment of TNNT2-mediated dilated cardiomyopathy ("TNNT2 DCM"), and additional assets for the treatment of genetic cardiac and neuromuscular diseases, at different stages of development, with varying levels of investment. We are advancing our diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management and care. Patient-focused and founded by those directly impacted by Duchenne, our mission is to improve the daily lives of patients living with these devastating diseases.

Previous wording

Solid was purpose-built to advance the best science and accelerate the discovery and development of treatments that may benefit all patients with Duchenne. As Solid expands to bring meaningful treatments to patients living with other neuromuscular and cardiac diseases, the values and guiding principles that drive us continue. Our corporate vision is to build an innovation platform enabling the discovery and development of high-value genetic medicines for neuromuscular and cardiac diseases by integrating internal capabilities, including a vector core, use of validated animal models, optimized expression cassettes, novel capsids and regulatory expertise, and collaborations with leaders in related clinical and research fields. Our mission, which guides our operations, is to treat and change the course of neuromuscular and cardiac diseases at all stages. Underscoring this mission, our disease-focused business model is founded on the following fundamental principles:

Current wording

Solid was purpose-built to advance the best science and accelerate the discovery and development of treatments that may benefit all patients with Duchenne. As we expand to bring meaningful treatments to patients living with other neuromuscular and cardiac diseases, the values and guiding principles that drive us continue. Our corporate vision is to build an innovation platform enabling the discovery and development of high-value genetic medicines for neuromuscular and cardiac diseases by integrating internal capabilities, including a vector core, use of validated animal models, optimized expression cassettes, novel capsids and regulatory expertise, and collaborations with leaders in related clinical and research fields. Our mission, which guides our operations, is to treat and change the course of neuromuscular and cardiac diseases at all stages. Underscoring this mission, our disease-focused business model is founded on the following fundamental principles:

Previous wording

identify and develop meaningful therapies for patients with neuromuscular and cardiac diseases;

Current wording

identify and develop meaningful therapies for underserved patients with sometimes fatal neuromuscular and cardiac diseases;

Previous wording

We are continuing to advance our pipeline of Candidates. The U.S. Food and Drug Administration, or the FDA, has granted orphan drug designation to SGT-003 for the treatment of Duchenne, and the FDA and the European Medicines Agency, or EMA, have granted orphan drug designation to SGT-501 for the treatment of CPVT. We expect to submit an Investigational New Drug, or IND, application for SGT-501 for the treatment of CPVT in the first half of 2025. The FDA has also granted Rare Pediatric Disease and fast track designation for SGT-003.

Current wording

We are continuing to advance our pipeline of Candidates. The U.S. Food and Drug Administration (the "FDA") has granted orphan drug designation, Rare Pediatric Disease designation and Fast Track designation for SGT-003 for Duchenne. The FDA has granted Fast Track Designation for SGT-212 for the treatment of FA. The FDA and the European Medicines Agency (the "EMA") have granted orphan drug designation to SGT-501 for the treatment of CPVT and the FDA has granted Rare Pediatric Disease Designation for SGT-501.

Previous wording

Patient dosing in the Phase 1/2 INSPIRE DUCHENNE multicenter clinical trial of SGT-003 began in the second quarter of 2024, and at the time of filing, SGT-003 has been well tolerated and no Serious Adverse Events (SAEs) were observed in the patients dosed. In September 2024, we amended the INSPIRE DUCHENNE clinical trial protocol to increase the anticipated participant enrollment size, expand the participant cohort age groups, and extend the timepoints of certain secondary objective measurements. In the first quarter of 2025, we anticipate reporting initial safety, expression and biomarker data from the first patients dosed, and provide a trial update, in first quarter of 2025 following the completion and collective assessment of 90-day muscle biopsies. We plan to expand clinical development of SGT-003 in the United States and in Canada and Europe. A Clinical Trial Application for INSPIRE DUCHENNE was authorized in Canada in the second quarter of 2024. In anticipation of an expanded clinical trial, we have initiated work for additional GMP batches of SGT-003.

Current wording

Patient dosing in the Phase 1/2 INSPIRE DUCHENNE trial of SGT-003 began in the second quarter of 2024. The INSPIRE DUCHENNE trial is a Phase 1/2 first-in-human, open-label, single-dose, multicenter trial designed to evaluate the safety, tolerability and efficacy of SGT-003 in pediatric patients with Duchenne at a dose of 1E14vg/kg. SGT-003 is administered as a one-time intravenous infusion. In September 2024, we amended the INSPIRE DUCHENNE clinical trial protocol to increase the anticipated participant enrollment size, expand the participant cohort age groups, and extend the timepoints of certain secondary objective measurements. In connection with the expanded clinical trial, we have initiated work for additional GMP batches of SGT-003.

Previous wording

AAV-SLB101, Solid’s proprietary capsid used in SGT-003, was well tolerated in the first patients dosed in the INSPIRE DUCHENNE trial, and was well tolerated in NHP and mouse studies. We have also transitioned our BAG3 and TNNT2 cardiac programs to AAV-SLB101.

Current wording

Solid's proprietary capsid used in SGT-003, AAV-SLB101, was well tolerated in the first patients dosed in the INSPIRE DUCHENNE trial as well as in non-human primates ("NHP") and mouse studies.

Previous wording

We are focused on developing transformative treatments to improve the lives of patients with rare neuromuscular and cardiac diseases. Our current programs are all designed to treat these diseases with gene transfer products. Gene transfer, a type of gene therapy, is designed to address diseases caused by mutated genes through the delivery of functional versions of genes, called transgenes. The transgenes are then utilized by the body to produce desired proteins to compensate for mutated genes, potentially offering long-lasting clinical benefit. In addition to a transgene, our gene transfer Candidates include a viral capsid or vector (a protein shell utilized as a vehicle to deliver a transgene to cells in the body) and a promoter (a specialized DNA sequence that directs cells to produce the protein in specific tissues). The capsid is modified to no longer self-replicate yet still retain its ability to introduce new genetic material directly into patients’ cells. Adeno-associated virus, or AAV, capsids have been approved for use to deliver transgenes to patients, including via systemic delivery. The use of AAV capsids to deliver gene therapies has also been extensively studied by third parties in human clinical trials for multiple disease indications, and in certain of these trials AAV was delivered systemically to the patient.

Current wording

We are focused on developing transformative treatments to improve the lives of patients with rare neuromuscular and cardiac diseases. The majority of our current programs are designed to treat these diseases with gene transfer products. Gene transfer, a type of gene therapy, is designed to address diseases caused by mutated genes through the delivery of functional versions of genes, called transgenes. The transgenes are then utilized by the body to produce desired proteins that act therapeutically to treat the condition. In addition to a transgene, our gene transfer Candidates include a viral capsid or vector (a protein shell utilized as a vehicle to deliver a transgene to cells in the body) and a promoter (a specialized DNA sequence that directs cells to produce the protein in specific tissues). The capsid is modified to no longer self-replicate yet still retain its ability to introduce new genetic material directly into patients’ cells. Adeno-associated virus ("AAV") capsids have been approved for use to deliver transgenes to patients, including via systemic delivery as well as stereotactic neurosurgical administration to the brain. The use of AAV capsids to deliver gene therapies has also been extensively studied by third parties in human clinical trials for multiple disease indications, and in certain of these trials AAV was delivered systemically to the patient.

Previous wording

Due to our significant research and development expenditure, licensing and patent investment, and general administrative costs associated with our operations, we have generated substantial operating losses in each period since our inception. Our net losses were $32.7 million and $82.1 million for the three and nine months ended September 30, 2024 and $21.0 million and $75.7 million for the three and nine months ended September 30, 2023, respectively. As of September 30, 2024, we had an accumulated deficit of $740.9 million. We expect to incur significant expenses and operating losses for the foreseeable future.

Current wording

Due to our significant research and development expenditure, licensing and patent investment, and general and administrative costs associated with our operations, we have generated substantial operating losses in each period since our inception. Our net losses were $39.3 million and $24.3 million for the three months ended March 31, 2025 and 2024, respectively. As of March 31, 2025, we had an accumulated deficit of $822.7 million. We expect to incur significant expenses and operating losses for the foreseeable future.

Previous wording

Because of the numerous risks and uncertainties associated with product development, we are unable to predict the timing or amount of increased expenses or determine when or if we will be able to achieve or maintain profitability. Even if we are able to generate revenue from product sales, we may not become profitable. If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be unable to continue our operations at planned levels and be forced to reduce or terminate our operations.

Current wording

Because of the numerous risks and uncertainties associated with product development, we are unable to predict the timing or amount of increased expenses or determine when or if we will be able to achieve or maintain profitability. Even if we are able to generate revenue from product sales, we may not become profitable. If we fail to become profitable or are unable to sustain profitability on a continuing basis, we may be unable to continue our operations at planned levels and be forced to reduce or terminate our operations.

Previous wording

As of September 30, 2024, we had cash, cash equivalents, and available-for-sale securities of $171.1 million, excluding restricted cash of $1.9 million. We believe that our cash, cash equivalents, and available-for-sale securities as of September 30, 2024 will enable us to fund our operating expenses and capital expenditure requirements into 2026. We have based this estimate on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently anticipate.

Current wording

As of March 31, 2025, we had cash, cash equivalents, and available-for-sale securities of $306.9 million, excluding restricted cash of $2.0 million. We believe that our cash, cash equivalents, and available-for-sale securities as of March 31, 2025 will enable us to fund our operating expenses and capital expenditure requirements into the first half of 2027. We have based this estimate on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently anticipate.

Previous wording

expenses incurred under agreements with third parties, including contract research organizations, or CROs, that conduct research, preclinical and clinical activities on our behalf, as well as contract manufacturing organizations, or CMOs, that manufacture SGT-003, SGT-501, and other Candidates for use in our preclinical studies and clinical trials;

Current wording

expenses incurred under agreements with third parties, including contract research organizations ("CROs") that conduct research, preclinical and clinical activities on our behalf, as well as contract manufacturing organizations ("CMOs") that manufacture SGT-003, SGT-501, SGT-212, and other Candidates for use in our preclinical studies and clinical trials;

Previous wording

Research and development activities are central to our business model. We are still in the early stages of development of our Candidates. Candidates in later stages of clinical development generally have higher development costs than those in preclinical development or in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. We expect that our research and development expenses will continue to increase for the foreseeable future if and as we conduct clinical trials for SGT-003, initiate clinical trials for our other Candidates, and continue to identify and develop additional Candidates.

Current wording

Research and development activities are central to our business model. We are still in the early stages of development of our Candidates. Candidates in later stages of clinical development generally have higher development costs than those in preclinical development or in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. We expect that our research and development expenses will continue to increase for the foreseeable future if and as we conduct clinical trials for SGT-003, initiate clinical trials for our other Candidates, including our planned Phase 1b clinical trial of SGT-212, and continue to identify and develop additional Candidates.

Previous wording

We cannot determine with certainty the duration, costs, and timing of clinical trials of SGT-003, SGT-501, or our other Candidates, or if, when, or to what extent we will generate revenue from the commercialization and sale of any of our Candidates for which we obtain marketing approval or our other research and development expenses. We may never succeed in obtaining marketing approval for any of our Candidates. The duration, costs, and timing of clinical trials and development of our Candidates will depend on a variety of factors, including:

Current wording

We cannot determine with certainty the duration, costs, and timing of ongoing and planned clinical trials of SGT-003, SGT-212, SGT-501, or our other Candidates, or if, when, or to what extent we will generate revenue from the commercialization and sale of any of our Candidates for which we obtain marketing approval or our other research and development expenses. We may never succeed in obtaining marketing approval for any of our Candidates. The duration, costs, and timing of clinical trials and development of our Candidates will depend on a variety of factors, including:

Previous wording

We expect that our general and administrative expenses will increase in the future as we support our research and development activities, and activities related to our INSPIRE DUCHENNE trial, and any planned or future clinical trials for, and potential commercialization of, our Candidates.

Current wording

We expect that our general and administrative expenses will increase in the future as we support our research and development activities and activities related to our INSPIRE DUCHENNE trial, our planned Phase 1b clinical trial of SGT-212, and any other planned or future clinical trials for and potential commercialization of our Candidates.

Previous wording

Other income, net consists primarily of interest income. Interest income consists of income earned on our cash and cash equivalents, available-for-sale securities, and restricted cash equivalents.

Current wording

Other income, net consists primarily of interest income. Interest income consists of income earned on our cash and cash equivalents, available-for-sale securities, and restricted cash.

Previous wording

Our discussion and analysis of our financial condition and results of operations is based on our condensed consolidated financial statements, which have been prepared in accordance with generally accepted accounting principles in the United States. The preparation of our condensed consolidated financial statements and related disclosures requires us to make estimates and judgments that affect the reported amounts of assets and liabilities, costs and expenses and the disclosure of contingent assets and liabilities in our condensed consolidated financial statements. We base our estimates on historical experience, known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. We evaluate our estimates and assumptions on an ongoing basis. Our actual results may differ materially from these estimates.

Current wording

Our management's discussion and analysis of our financial condition and results of operations is based on our condensed consolidated financial statements, which have been prepared in accordance with generally accepted accounting principles in the United States. The preparation of our condensed consolidated financial statements and related disclosures requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities, costs and expenses and the disclosure of contingent assets and liabilities in our condensed consolidated financial statements. We base our estimates on historical experience, known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. We evaluate our estimates and assumptions on an ongoing basis. Our actual results may differ materially from these estimates.

Previous wording

During the nine months ended September 30, 2024, there were no material changes to our critical accounting policies, except as noted in Note 1 – Nature of the Business and Basis of Presentation. Our critical accounting policies are described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Critical accounting policies and use of estimates” in our Annual Report on Form 10-K for the year ended December 31, 2023 and the notes to the unaudited condensed consolidated financial statements included in Part I, Item 1, “Financial Statements (unaudited),” of this quarterly report on Form 10-Q. We believe that of our critical accounting policies, the following accounting policies involve the most judgment and complexity:

Current wording

During the three months ended March 31, 2025, there were no material changes to our critical accounting policies. Our critical accounting policies are described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Critical Accounting Policies and Use of Estimates” in our Annual Report on Form 10-K for the year ended December 31, 2024 and the notes to the unaudited condensed consolidated financial statements included in Part I, Item 1, “Financial Statements (unaudited),” of this Quarterly Report on Form 10-Q. We believe that of our critical accounting policies, the following accounting policies involve the most judgment and complexity:

Previous wording

The following table summarizes our research and development expenses by Candidate for the respective periods (in thousands, except percentages):

Current wording

The following table summarizes our research and development expenses by Candidate for the periods indicated (in thousands, except percentages):

Previous wording

Research and development expenses for the nine months ended September 30, 2024 were $65.7 million, compared to $61.1 million for the nine months ended September 30, 2023. The increase of $4.6 million in research and development expenses was primarily due to an $8.7 million increase in costs for SGT-501 primarily related to manufacturing and research costs, a $7.8 million increase in other development program expenses primarily due to the FA212 asset acquisition and other research costs, and a $1.6 million increase in external expenses, offset by a $10.0 million decrease for SGT-003 primarily related to manufacturing and research costs, a $2.7 million decrease in costs for SGT-001 due to our decision to deprioritize SGT-001, and a $0.9 million decrease in personnel related expenses.

Current wording

Research and development expenses for the three months ended March 31, 2025 were $30.9 million, compared to $18.9 million for the three months ended March 31, 2024. The increase of $12.0 million in research and development expenses was primarily due to a $7.4 million increase in costs for SGT-003 primarily related to manufacturing, regulatory, and clinical costs, a $1.9 million increase in personnel related expenses, a $1.3 million increase in other development program expenses primarily due to manufacturing costs, a $0.8 million increase in costs for SGT-212 primarily related to regulatory and clinical costs, and a $0.7 million increase in costs for SGT-501 primarily related to research, regulatory, and clinical costs, offset by a $0.1 million decrease for SGT-001 due to our decision to deprioritize SGT-001.

Previous wording

General and administrative expenses were $7.9 million for the three months ended September 30, 2024, compared to $6.4 million for the three months ended September 30, 2023. The increase of $1.4 million was primarily related to a $1.5 million increase in personnel related costs, and a $0.2 million increase in consulting fees, offset by a $0.3 million decrease in temporary services.

Current wording

General and administrative expenses were $9.1 million for the three months ended March 31, 2025, compared to $8.0 million for the three months ended March 31, 2024. The increase of $1.1 million was primarily related to a $1.7 million increase in personnel related costs, partially offset by a $0.2 million decrease in legal fees, and a $0.2 million decrease in finance costs.

Previous wording

To date, we have financed our operations primarily through the sale of redeemable preferred units and member units, the sale of common stock and pre-funded warrants to purchase shares of our common stock in private placements and the sale of common stock in our initial public offering and a follow-on public offering and sales of common stock under our ATM Sales Agreement. Through September 30, 2024, we raised an aggregate of $144.6 million of gross proceeds from our sales of preferred units prior to the completion of our initial public offering, and an aggregate of $662.0 million of net proceeds from the sale of our common stock through public offerings, including our IPO and a follow-on public offering, private placements, the ATM Sales Agreement, and pursuant to the stock purchase agreement with Ultragenyx, as detailed in the following paragraphs.

Current wording

To date, we have financed our operations primarily through the sale of redeemable preferred units and member units, the sale of securities in private placements and follow-on offerings, the sale of common stock in our initial public offering, and sales of common stock under our "at-the-market offering" sales agreement with Jefferies LLC ("Jefferies") (the "ATM Sales Agreement"). Through March 31, 2025, we raised an aggregate of $144.6 million of gross proceeds from our sales of preferred units prior to the completion of our initial public offering, and an aggregate of $857.1 million of net proceeds from the sale of our common stock through public offerings, including our IPO and follow-on public offerings, private placements, the ATM Sales Agreement, and pursuant to the stock purchase agreements.

Previous wording

On March 13, 2019, we entered into the ATM Sales Agreement, which was amended and restated in March 2024, under which we may offer and sell, from time to time, shares of our common stock through Jefferies as sales agent. Any such sales being made by any method that is deemed an “at-the-market offering” as defined in Rule 415 promulgated under the Securities Act. We will pay Jefferies a commission of up to 3% of the gross proceeds of any sales of common stock pursuant to the ATM Sales Agreement. During the year ended December 31, 2023, we sold 602,030 shares pursuant to the ATM Sales Agreement resulting in net proceeds of $3.0 million. During the three and nine months ended September 30, 2024, we sold 330,670 and 1,208,287 shares pursuant to the ATM Sales Agreement resulting in net proceeds of $3.1 million and $11.5 million, respectively.

Current wording

On March 13, 2019, we entered into the ATM Sales Agreement, which was amended and restated in March 2024, under which we may offer and sell, from time to time, shares of our common stock through Jefferies as sales agent. Any such sales being made by any method that is deemed an “at-the-market offering” as defined in Rule 415 promulgated under the Securities Act. We will pay Jefferies a commission of up to 3% of the gross proceeds of any sales of common stock pursuant to the ATM Sales Agreement. During the year ended December 31, 2024, we sold 2,212,937 shares pursuant to the ATM Sales Agreement resulting in net proceeds of $18.4 million. During the three months ended March 31, 2025, we sold 61,542 shares pursuant to the ATM Sales Agreement resulting in net proceeds of $0.2 million.

Previous wording

On January 11, 2024, we issued and sold 16,973,103 shares of our common stock at a price per share of $5.53 and, to one investor in lieu of shares of common stock, pre-funded warrants to purchase 2,712,478 shares of common stock at a price of $5.529 per pre-funded warrant, in the January 2024 Private Placement. We received $103.7 million of net proceeds from the January 2024 Private Placement after deducting offering costs.

Current wording

On January 11, 2024, we issued and sold 16,973,103 shares of our common stock at a price per share of $5.53 and, to one investor in lieu of shares of common stock, pre-funded warrants to purchase 2,712,478 shares of common stock at a price of $5.529 per pre-funded warrant, in a private placement we refer to as the January 2024 Private Placement. We received $103.7 million of net proceeds from the January 2024 Private Placement after deducting offering costs.

Previous wording

As of September 30, 2024, we had cash, cash equivalents and available-for-sale securities of $171.1 million, excluding restricted cash of $1.9 million, and had no debt outstanding.

Current wording

As of March 31, 2025, we had cash, cash equivalents, and available-for-sale securities of $306.9 million, excluding restricted cash of $2.0 million, and had no debt outstanding.

Previous wording

During the nine months ended September 30, 2024, operating activities used $70.4 million of cash, primarily resulting from our net loss of $82.1 million partially offset by changes in our operating assets and liabilities of $0.7 million and non-cash charges of $11.0 million. Net cash used by changes in our operating assets and liabilities during the nine months ended September 30, 2024 consisted of an increase of $2.5 million in prepaid and other assets, and a decrease in the operating lease liability of $1.3 million, offset by an increase in accrued expenses and other liabilities of $3.1 million, and an increase in accounts payable of $1.4 million. Non-cash activities were driven by equity-based compensation of $6.7 million, depreciation and amortization expense of $2.0 million, non-cash lease expense of $1.8 million, and a change in the fair value of derivative liabilities of $3.4 million, partially offset by amortization on available-for-sale securities of $2.8 million.

Current wording

During the three months ended March 31, 2025, operating activities used $31.9 million of cash, primarily resulting from our net loss of $39.3 million partially offset by changes in our operating assets and liabilities of $1.8 million and non-cash charges of $5.6 million. Net cash used by changes in our operating assets and liabilities during the three months ended March 31, 2025 consisted of a decrease of $2.0 million in prepaid and other assets and an increase in accounts payable of $0.5 million, offset by a decrease in the operating lease liability of $0.4 million and a decrease in accrued expenses and other liabilities of $0.3 million. Non-cash activities were driven by equity-based compensation of $3.3 million, depreciation and amortization expense of $0.4 million, non-cash lease expense of $0.6 million, and a change in the fair value of derivative liabilities of $1.7 million, partially offset by amortization on available-for-sale securities of $0.3 million.

Previous wording

During the nine months ended September 30, 2023, operating activities used $73.4 million of cash, primarily resulting from our net loss of $75.7 million and changes in our operating assets and liabilities of $6.4 million, partially offset by non-cash charges of $8.7 million. Net cash used by changes in our operating assets and liabilities during the nine months ended September 30, 2023 consisted of a decrease in accrued expenses and other current and non-current liabilities of $5.3 million, a decrease in the operating lease liability of $1.3 million, and a decrease in accounts payable of $0.9 million, partially offset by a decrease in prepaid and other assets of $1.1 million. Non-cash activities were driven by equity-based compensation of $5.7 million, depreciation and impairment expense of $2.4 million, and non-cash lease expense of $1.9 million, partially offset by amortization on available-for-sale securities of $1.3 million.

Current wording

During the three months ended March 31, 2024, operating activities used $25.2 million of cash, primarily resulting from our net loss of $24.3 million and changes in our operating assets and liabilities of $2.9 million, partially offset by non-cash charges of $2.0 million. Net cash used by changes in our operating assets and liabilities during the three months ended March 31, 2024 consisted of an increase of $2.0 million in prepaid and other assets, a decrease in accounts payable of $0.6 million, and a decrease in the operating lease liability of $0.5 million, offset by an increase in accrued expenses and other liabilities of $0.2 million. Non-cash activities were driven by equity-based compensation of $1.6 million, non-cash lease expense of $0.6 million and depreciation expense of $0.3 million, partially offset by amortization on available-for-sale securities of $0.5 million and a gain on lease termination of $0.1 million.

Previous wording

During the nine months ended September 30, 2024, investing activities used $54.7 million of cash, resulting from the purchases of available-for sale securities of $189.0 million and the purchases of property plant and equipment of $0.5 million, partially offset by the maturity of available-for sale securities of $134.8 million.

Current wording

During the three months ended March 31, 2025, investing activities used $27.5 million of cash, resulting from the purchases of available-for sale securities of $65.3 million and the purchases of property plant and equipment of $0.1 million, partially offset by the maturity of available-for sale securities of $38.0 million.

Previous wording

During the nine months ended September 30, 2023, investing activities used $35.6 million of cash, resulting from the purchases of available-for sale securities of $102.8 million and the purchases of property plant and equipment of $1.4 million, partially offset by the maturity of available-for sale securities of $68.6 million.

Current wording

During the three months ended March 31, 2024, investing activities used $38.5 million of cash, resulting from the purchases of available-for sale securities of $83.3 million and the purchases of property plant and equipment of $0.2 million, partially offset by the maturity of available-for sale securities of $45.0 million.

Previous wording

During the nine months ended September 30, 2024, financing activities provided $115.5 million of cash, resulting from the net proceeds from the issuance of common stock and pre-funded warrants to purchase shares of common stock of $115.3 million, proceeds from the exercise of common stock options for $0.4 million, and proceeds from the issuance of shares of $0.1 million under the Company's 2021 Employee Stock Purchase Plan, or ESPP, offset by payments of the principal portion of finance lease obligations of $0.3 million.

Current wording

During the three months ended March 31, 2025, financing activities provided $189.7 million of cash, resulting from the net proceeds from the issuance and sale of common stock and pre-funded warrants to purchase shares of common stock of $189.9 million, offset by payments of the principal portion of finance lease obligations of $0.1 million.

Previous wording

During the nine months ended September 30, 2023, financing activities provided $2.6 million of cash, resulting from the proceeds from the issuance of common stock of $2.5 million and purchases of shares under the ESPP.

Current wording

During the three months ended March 31, 2024, financing activities provided $107.2 million of cash, resulting from the proceeds from the issuance of common stock and pre-funded warrants to purchase shares of common stock of $107.1 million and proceeds from the exercise of common stock options for $0.1 million.

Previous wording

continue to enroll patients in our expanded INSPIRE DUCHENNE trial and advance clinical development of SGT-003;

Current wording

continue to enroll patients in our INSPIRE DUCHENNE trial and advance clinical development of SGT-003;

Previous wording

advance our other Candidates into clinical trials;

Current wording

advance our other Candidates into clinical trials, including our planned Phase 1b clinical trial of SGT-212;

Previous wording

engage in regulatory interactions with the United States Food and Drug Administration, or FDA, and other regulatory authorities;

Current wording

engage in regulatory interactions with the FDA and other regulatory authorities;

Previous wording

scale up our manufacturing processes and arrange manufacturing for larger quantities of our Candidates for preclinical and clinical development and potential commercialization;

Current wording

scale up our manufacturing processes and arrange manufacturing for larger quantities of our product candidates for preclinical and clinical development and potential commercialization;

Previous wording

As of September 30, 2024, we had cash, cash equivalents and available-for-sale securities of $171.1 million, excluding restricted cash of $1.9 million. Based on our current operating plan, we believe that our cash, cash equivalents, and available-for-sale securities as of September 30, 2024 will be sufficient to fund our operating expenses and capital requirements into 2026. As a result, in order to continue to operate our business beyond that time, we will need to raise additional funds. However, there can be no assurance that we will be able to generate funds on terms acceptable to us, on a timely basis, or at all. In addition, we have based this estimate on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently anticipate.

Current wording

As of March 31, 2025, we had cash, cash equivalents and available-for-sale securities of $306.9 million, excluding restricted cash of $2.0 million. Based on our current operating plan, we believe that our cash, cash equivalents and available-for-sale securities as of March 31, 2025 will be sufficient to fund our operating expenses and capital requirements into the first half of 2027. As a result, in order to continue to operate our business beyond that time, we will need to raise additional funds. However, there can be no assurance that we will be able to generate funds on terms acceptable to us, on a timely basis, or at all. In addition, we have based this estimate on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently anticipate.

Previous wording

Because of the numerous risks and uncertainties associated with the development of our Candidates and because the extent to which we may enter collaborations with third parties for development of our Candidates is unknown, we are unable to estimate the timing and amounts of increased capital outlays and operating expenses associated with completing the research and development of our Candidates. Our future capital requirements will depend on many factors, including:

Current wording

Due to the numerous risks and uncertainties associated with the development of our Candidates and because the extent to which we may enter collaborations with third parties for development of our Candidates is unknown, we are unable to estimate the timing and amounts of increased capital outlays and operating expenses associated with completing the research and development of our Candidates. Our future capital requirements will depend on many factors, including:

Previous wording

the progress, costs and results of the INSPIRE DUCHENNE trial and any future clinical trials of our Candidates;

Current wording

the progress, costs and results of the INSPIRE DUCHENNE trial, our planned clinical trial of SGT-212, and any future clinical trials of our Candidates;

Previous wording

if and as we need to adapt our business in response to public health emergencies or pandemics, such as the recent COVID–19 pandemic, and collateral consequences related thereto.

Current wording

if and as we need to adapt our business in response to public health emergencies or pandemics and collateral consequences related thereto.

Previous wording

We are supplying, and expect to continue to supply, our ongoing and future clinical development programs with drug produced at a facility compliant with the current Good Manufacturing Practice regulations enforced by the FDA, or cGMPs, located at one of our CMOs. We intend to establish the capability and capacity to supply Candidates at commercial scale from multiple sources.

Current wording

We are supplying, and expect to continue to supply, our ongoing and future pre-clinical and clinical development programs with drug product produced at a cGMP compliant facility located at one of our CMOs. We intend to establish the capability and capacity to supply Candidates at commercial scale from multiple sources.