Solid Biosciences Inc

Previous wording

We are continuing to advance our pipeline of Candidates. The FDA has granted orphan drug designation to SGT-003 for the treatment of Duchenne and the FDA and EMA have granted orphan drug designation to SGT-501 for the treatment of CPVT. We expect to file an IND for 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, 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.

Previous wording

Patient dosing in the Phase 1/2 INSPIRE Duchenne 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 Significant Adverse Events (SAEs) were observed in the patients dosed. Initial safety, expression and certain functional endpoint data is expected in Q4 2024. A Clinical Trial Application (CTA) for INSPIRE Duchenne has been approved in Canada We plan to expand clinical development in the US and Globally and . Subject to clearance, we expect additional sites to become active by Q2 2025 to support current and potential future trials. 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 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.

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 $25.1 million and $49.4 million for the three and six months ended June 30, 2024 and $24.6 million and $54.7 million for the three and six months ended June 30, 2023, respectively. As of June 30, 2024, we had an accumulated deficit of $708.1 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 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.

Previous wording

As of June 30, 2024, we had cash, cash equivalents, and available-for-sale securities of $190.3 million, excluding restricted cash of $1.9 million. We believe that our cash, cash equivalents, and available-for-sale securities as of June 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 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.

Previous wording

During the six months ended June 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 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:

Previous wording

Accrued research and development expenses; and

Current wording

accrued research and development expenses;

Previous wording

Research and development expenses for the three months ended June 30, 2024 were $19.5 million, compared to $19.8 million for the three months ended June 30, 2023. The decrease of $0.3 million in research and development expenses was due to a $3.3 million decrease in costs for SGT-003 primarily related to lower manufacturing and research costs, a $0.3 million decrease in costs for SGT-001 primarily related to lower clinical and research costs due to our decision to deprioritize SGT-001, a $0.4 million decrease in external expenses, offset by a $1.7 million increase in costs for SGT-501 primarily related to manufacturing and research, a $1.7 million increase in other Candidate costs primarily related to BAG3, and a $0.2 million increase in personnel related costs.

Current wording

$5.8 million increase in other development program expenses primarily due to the FA212 asset acquisition and other research costs, a $3.1 million increase in costs for SGT-501 primarily related to manufacturing and research costs, a $1.1 million increase in external expenses, a $0.6 million increase in costs for SGT-003 primarily related to clinical and manufacturing costs, and a $0.1 million increase in personnel related expenses, offset by a $0.1 million decrease in costs for SGT-001 primarily related to lower clinical and research costs due to our decision to deprioritize SGT-001.

Previous wording

General and administrative expenses were $16.3 million for the six months ended June 30, 2024, compared to $14.5 million for the six months ended June 30, 2023. The increase of $1.8 million was primarily related to a $1.4 million increase in legal fees and a $0.2 million increase in software costs, and a $0.2 million increase in audit and tax fees.

Current 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.

Previous wording

Other income, net was $2.7 million for the three months ended June 30, 2024, compared to $2.2 million for the three months ended June 30, 2023. The activity was primarily related to the increase in interest income on available-for-sale securities included within our portfolio due to higher investment balances from proceeds of our amended and restated at-the market offering sales agreement, dated March 13, 2024, by and between us and Jefferies LLC, or Jefferies, or the ATM Sales Agreement, and January 2024 Private Placement.

Current wording

Other income, net was $2.5 million for the three months ended September 30, 2024, compared to $2.1 million for the three months ended September 30, 2023. The activity was primarily related to the increase in interest income on available-for-sale securities included within our portfolio due to higher investment balances from proceeds of our amended and restated at-the market offering sales agreement, dated March 13, 2024, by and between us and Jefferies LLC, or Jefferies, or the ATM Sales Agreement, and January 2024 Private Placement.

Previous wording

Comparison of the six months ended June 30, 2024 and 2023

Current wording

Comparison of the nine months ended September 30, 2024 and 2023

Previous wording

Research and development expenses for the six months ended June 30, 2024 were $38.3 million, compared to $44.4 million for the six months ended June 30, 2023. The decrease of $6.1 million in research and development expenses was primarily due to a $10.6 million decrease for SGT-003 primarily related to manufacturing and research costs, a $2.6 million decrease in costs for SGT-001 due to our decision to deprioritize SGT-001, a $1.0 million decrease in personnel related expenses, offset by a $5.6 million increase in costs for SGT-501 primarily related to manufacturing and research, a $2.0 million increase in other Candidates primarily related to BAG3, and a $0.5 million increase in external expenses.

Current 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.

Previous wording

General and administrative expenses were $8.3 million for the three months ended June 30, 2024, compared to $7.1 million for the three months ended June 30, 2023. The increase of $1.2 million was primarily related to a $0.9 million increase in legal fees and a $0.3 million increase in recruiting and license fees.

Current wording

General and administrative expenses were $24.2 million for the nine months ended September 30, 2024, compared to $20.9 million for the nine months ended September 30, 2023. The increase of $3.2 million was primarily related to a $2.2 million increase in personnel related costs, a $1.3 million increase in legal fees, and a $0.3 million increase in software costs, offset by a $0.6 million decrease in temporary services.

Previous wording

Other income, net was $5.3 million for the six months ended June 30, 2024 compared to other expense of $4.2 million for the six months ended June 30, 2023. The activity was primarily related to the increase in interest income on available-for-sale securities included within our portfolio due to higher investment balances from proceeds of ATM Sales Agreement and January 2024 Private Placement.

Current wording

Other income, net was $7.7 million for the nine months ended September 30, 2024 compared to other expense of $6.3 million for the nine months ended September 30, 2023. The activity was primarily related to the increase in interest income on available‑for‑sale securities included within our portfolio due to higher investment balances from proceeds from our ATM Sales Agreement and the January 2024 Private Placement.

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 June 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 $656.0 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 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.

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 six months ended June 30, 2024, we sold 526,953 and 877,617 shares pursuant to the ATM Sales Agreement resulting in net proceeds of $5.4 million and $8.4 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, 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.

Previous wording

As of June 30, 2024, we had cash, cash equivalents and available-for-sale securities of $190.3 million, excluding restricted cash of $1.9 million, and had no debt outstanding.

Current 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.

Previous wording

During the six months ended June 30, 2024, operating activities used $46.7 million of cash, primarily resulting from our net loss of $49.4 million and changes in our operating assets and liabilities of $2.1 million, partially offset by non-cash charges of $4.8 million. Net cash used by changes in our operating assets and liabilities during the six months ended June 30, 2024 consisted of an increase of $1.6 million in prepaid and other assets, a decrease in the operating lease liability of $0.9 million, a decrease in accrued expenses and other liabilities of $0.2 million, offset by an increase in accounts payable of $0.5 million. Non-cash activities were driven by equity-based compensation of $3.7 million, depreciation and amortization expense of $1.6 million, and non-cash lease expense of $1.2 million, partially offset by amortization on available-for-sale securities of $1.6 million and a gain on lease termination of $0.1 million.

Current 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.

Previous wording

During the six months ended June 30, 2023, operating activities used $55.5 million of cash, primarily resulting from our net loss of $54.7 million and changes in our operating assets and liabilities of $7.6 million, partially offset by non-cash charges of $6.8 million. Net cash used by changes in our operating assets and liabilities during the six months ended June 30, 2023 consisted of a decrease in accrued expenses and other current and non-current liabilities of $6.2 million, a decrease in the operating lease liability of $0.8 million, an increase of $0.6 million in prepaid and other assets. Non-cash activities were driven by equity-based compensation of $4.1 million and depreciation and impairment expense of $1.8 million, and non-cash lease expense of $1.2 million, partially offset by amortization on available-for-sale securities of $0.3 million.

Current 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.

Previous wording

During the six months ended June 30, 2024, investing activities used $43.7 million of cash, resulting from the purchases of available-for sale securities of $103.6 million and the purchases of property plant and equipment of $0.4 million, partially offset by the maturity of available-for sale securities of $60.3 million.

Current 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.

Previous wording

During the six months ended June 30, 2023, investing activities used $11.0 million of cash, resulting from the purchases of available-for sale securities of $68.6 million and the purchases of property plant and equipment of $1.0 million, partially offset by the maturity of available-for sale securities of $58.6 million.

Current 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.

Previous wording

During the six months ended June 30, 2024, financing activities provided $112.2 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 $112.2 million, proceeds from the exercise of common stock options for $0.2 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 $0.2 million.

Current 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.

Previous wording

During the six months ended June 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 our ESPP.

Current 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.

Previous wording

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

Current wording

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

Previous wording

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 Candidates for preclinical and clinical development and potential commercialization;

Previous wording

As of June 30, 2024, we had cash, cash equivalents and available-for-sale securities of $190.3 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 June 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 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.

Previous wording

During the six months ended June 30, 2024, there were no material changes to our contractual obligations and commitments from those described in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our Annual Report on Form 10-K for the fiscal year ended December 31, 2023.

Current wording

During the nine months ended September 30, 2024, other than our acquisition agreement with FA212 to make potential milestone payments and pay royalties based on achievement of specified milestones, there were no material changes to our contractual obligations and commitments from those described in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our Annual Report on Form 10-K for the fiscal year ended December 31, 2023.

Previous wording

Note 1 – Nature of the Business and Basis of Presentation to our condensed consolidated financial statements (See Part I, Item 1 – Financial Statements, of this Quarterly Report on Form 10-Q).

Current wording

A summary of significant recent accounting pronouncements that we expect to adopt is included in Note 1 – Nature of the Business and Basis of Presentation to our condensed consolidated financial statements (See Part I, Item 1 – Financial Statements, of this Quarterly Report on Form 10-Q).