Solid Biosciences Inc.
For the period ended Mar 31, 2026. Filed May 12, 2026.
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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.
Mar 31, 2026 · Filed May 12, 2026
SLDB$6.59+6.6%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-09-30; filed 2025-11-03.
0001193125-25-262818
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2026-03-31; filed 2026-05-12.
0001193125-26-219337
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe 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 143 previous paragraphs and 144 current paragraphs.
95 unchanged; 34 reworded; 12 added; 9 removed; 5 previous and 3 current paragraphs rearranged.
Tables are not compared: 6 previous and 4 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (12)
Current report
On March 11, 2026, we announced positive new interim data from the Phase 1/2 INSPIRE DUCHENNE clinical trial as of a February 23, 2026 data cutoff date. For a full description of the initial results from the INSPIRE DUCHENNE trial, see Part I, Item I, “Business” in our Annual Report on Form 10-K filed on March 19, 2026.
Current report
Enrollment and dosing in the INSPIRE DUCHENNE trial is ongoing and being conducted at 15 clinical sites across the United States, Canada, Italy and the United Kingdom. We believe we have aligned with the FDA on SGT-003's potency assay strategy and will continue commercial-readiness CMC activities, with our process performance qualification manufacturing batches to be completed in 2026.
Current report
In October 2025, we activated the first clinical trial site and began screening participants for IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled trial evaluating SGT-003. In February 2026, we announced positive feedback from a Type C meeting with the FDA where we reached alignment on the IMPACT DUCHENNE trial design, including: the patient population of ambulant participants 7 to <12 years of age, the primary endpoint of change from baseline in Time to Rise (TTR) velocity from supine position evaluated at 18 months and other key secondary endpoints. Clinical trial sites for IMPACT DUCHENNE are currently active in Australia and Canada and additional clinical site activations in the UK and US are expected in the second half of 2026, with sites in the EU anticipated to follow, subject to regulatory clearances. Participant screening is underway and we dosed our first participant in the IMPACT DUCHENNE trial in April 2026.
Current report
We expect to continue discussions with the FDA as we seek guidance on a potential accelerated approval pathway for SGT-003 and we expect to provide an update as regulatory discussions progress.
Current report
Additionally, Solid has received a positive opinion from the European Medicines Agency's Paediatric Committee on its Pediatric Investigation Plan (PIP), establishing alignment on its pediatric development framework for SGT-003 in Europe.
Current report
In March of 2026, we began using the mark POLARIS-101 TMto represent our AAV-SLB101 capsid (“POLARIS-101 TM”). POLARIS‑101 TMis our rationally designed, proprietary capsid used in SGT-003 for Duchenne, which has been generally well tolerated as of May 11, 2026 (N=47) in the INSPIRE DUCHENNE clinical trial, and was also well tolerated in nonclinical NHP and mouse models. We aim to license POLARIS-101 TMbroadly to corporations, institutions and academic labs pursuing neuromuscular and cardiac rare disease research, with more than 50 agreements, including licenses, executed.
Current report
Research and Development Expenses
Current report
General and Administrative Expenses
Current report
Other Income, Net
Current report
Research and Development Expenses
Current report
General and Administrative Expenses
Current report
Other Income, Net
Removed paragraphs (9)
Previous report
On November 3, 2025, we announced positive interim data from the Phase 1/2 INSPIRE DUCHENNE trial as of the data cutoff date of September 29, 2025. Interim biopsy data reported from 10 participants treated with SGT-003 showed a mean microdystrophin expression of 58%, as measured by both western blot and mass spectrometry, and mean microdystrophin positive fibers of 51%, as measured by immunofluorescence. Interim data also demonstrated early signals of cardiac function normalization and improvements in multiple biomarkers that are indicators of muscle integrity, health and resilience. These interim data include favorable reductions across a range of biomarkers of muscle injury and breakdown, including serum creatine kinase (CK), serum alanine transaminase (ALT), serum aspartate aminotransferase (AST), serum lactate dehydrogenase (LDH) and serum titin, through both Day 90 and Day 360 after treatment with SGT-003. Additionally, following treatment with SGT-003, we observed restoration of key components of the dystrophin associated protein complex (DAPC), including beta-sarcoglycan and neuronal nitric oxide synthase (nNOS), and early signals of a potential for cardiac benefit through reduction in troponin I (cTnI) and increased systolic function as measured by LVEF by echocardiography.
Previous report
For a more detailed description of the interim results from the INSPIRE DUCHENNE trial, see our Form 8-K filed November 3, 2025.
Previous report
Enrollment and dosing in the INSPIRE DUCHENNE trial is ongoing, with 30 total participants anticipated to be dosed by early 2026.
Previous report
The INSPIRE DUCHENNE trial currently has 15 active sites across the U.S., Canada, Italy and the United Kingdom, with an additional site expected to be activated by the end of 2025. A meeting with the FDA to discuss potential regulatory pathways, including accelerated approval pathways, for SGT-003 is anticipated to occur in the first half of 2026.
Previous report
Additionally, in October 2025, we activated the first clinical trial site and began screening participants for IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled trial evaluating SGT-003 outside of the United States. IMPACT DUCHENNE has been designed with the aim of supporting potential ex-U.S. regulatory authorizations. We have received regulatory approvals to conduct IMPACT DUCHENNE in both Canada and Australia, and we plan to expand the trial into additional countries, subject to receipt of regulatory approval.
Previous report
AAV-SLB101, Solid's proprietary, rationally designed capsid used in SGT-003, has been generally well tolerated as of October 31, 2025 (N=23), in the INSPIRE DUCHENNE trial as well as in non-human primates ("NHP") and mouse studies.
Previous report
General and administrative expenses were $9.2 million for the three months ended September 30, 2025, compared to $7.9 million for the three months ended September 30, 2024. The increase of $1.3 million was primarily related to a $0.9 million increase in personnel related costs and a $0.4 million increase in legal and consulting fees.
Previous report
Comparison of the Nine Months Ended September 30, 2025 and 2024
Previous report
Other income, net was $5.3 million for the nine months ended September 30, 2025 compared to $7.7 million for the nine months ended September 30, 2024. The decrease of $2.5 million was primarily related to the change in fair value of derivative liabilities.
Reworded paragraphs (34)
Previous wording
The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our unaudited condensed consolidated financial statements and related notes appearing elsewhere in this quarterly report on Form 10-Q and our audited financial statements and related notes for the year ended December 31, 2024 included in our annual report filed on Form 10-K on March 6, 2025.
Current wording
The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our unaudited condensed consolidated financial statements and related notes appearing elsewhere in this quarterly report on Form 10-Q and our audited financial statements and related notes for the year ended December 31, 2025 included in our annual report on Form 10-K filed on March 19, 2026.
Previous wording
We are continuing to advance our pipeline of Candidates. The U.S. Food and Drug Administration (the "FDA") has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations for SGT-003 for Duchenne and SGT-003 has also been awarded an Innovation Passport by the new UK Innovative Licensing and Access Pathway. The FDA has granted Fast Track and Rare Pediatric Disease designations for SGT-212 for the treatment of FA. The FDA has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations to SGT-501 for the treatment of CPVT.
Current wording
We are continuing to advance our pipeline of Candidates. The U.S. Food and Drug Administration (the “FDA”) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations for SGT-003 for Duchenne, and SGT-003 has also been awarded Orphan Drug designation from the European Commission for the treatment of Duchenne, in addition to an Innovation Passport by the new UK Innovative Licensing and Access Pathway, which aims to accelerate time to market and facilitate patient access to new medicines in the United Kingdom. The FDA has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations to SGT-212 for the treatment of FA and SGT-501 for the treatment of CPVT.
Previous wording
Participant 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 participants with Duchenne at a dose of 1E14vg/kg. SGT-003 is administered as a one-time intravenous infusion. In September 2024 and June 2025, 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.
Current wording
Participant 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. Since initiation of the INSPIRE DUCHENNE clinical trial, we amended the clinical trial protocol to increase the anticipated participant enrollment size, expand the participant cohort age groups, and extend the time points of certain secondary objective measurements. In connection with the expanded clinical trial, we have initiated work for additional Good Manufacturing Practices (“GMP”) batches of SGT‑003.
Previous wording
As of October 31, 2025, 23 participants have been dosed in the INSPIRE DUCHENNE trial and SGT-003 has been generally well tolerated as of such date. There has been one treatment-related serious adverse event reported in the INSPIRE DUCHENNE trial. This serious adverse event was identified as a Grade 3 immune-mediated myositis which, importantly, was not associated with muscle pain or weakness, and occurred in a participant who had a large deletion in a region coded for by SGT-003's microdystrophin. The study participant promptly responded to steroid treatment with all clinical symptoms noted at presentation resolving and with muscle biomarkers, including CK, declining well below baseline levels. This SAE was reviewed by the data and safety monitoring board (DSMB) with the recommendation to continue dosing without interruption.
Current wording
SGT-003 has been generally well tolerated in the 47 participants dosed in the Phase 1/2 INSPIRE DUCHENNE clinical trial as of May 11, 2026. The safety and tolerability profile observed in the INSPIRE DUCHENNE trial continued to be promising; SGT-003 is administered using a low-burden, steroid-only prophylactic immunomodulation regimen. As of May 11, 2026, there was one previously disclosed treatment-related serious adverse event reported in the INSPIRE DUCHENNE trial. This serious adverse event was identified as a Grade 3 immune-mediated myositis which, importantly, was not associated with muscle pain or weakness, and occurred in a participant who had a large deletion in a region coded for by SGT-003’s microdystrophin. The trial participant promptly responded to steroid treatment and the event has resolved. This serious adverse event was reviewed by the data and safety monitoring board with the recommendation to continue dosing without interruption.
Previous wording
The FDA has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations for SGT-003 for the treatment of Duchenne. SGT-003 has been awarded an Innovation Passport by the new UK Innovative Licensing and Access Pathway, which aims to accelerate time to market and facilitate patient access to new medicines in the United Kingdom.
Current wording
The FDA has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations for SGT-003 and the European Commission has granted Orphan drug designation for SGT-003 for the treatment of Duchenne. SGT-003 has been awarded an Innovation Passport by the new UK Innovative Licensing and Access Pathway, which aims to accelerate time to market and facilitate patient access to new medicines in the United Kingdom.
Previous wording
In January 2025, we announced that the FDA cleared our investigational new drug ("IND") for SGT-212 for the treatment of FA. In October 2025, we activated the first clinical trial site and began screening participants for FALCON, an open-label, multi-center Phase 1b clinical trial of SGT-212. The trial is expected to enroll non-ambulatory and ambulatory adult participants living with FA in up to three cohorts and is designed to evaluate the safety and tolerability of systemic and bilateral intradentate nucleus administration of SGT-212. The FDA has granted Fast Track and Rare Pediatric Disease designations to SGT-212 for the treatment of FA.
Current wording
The trial is expected to enroll approximately 10 non-ambulatory and ambulatory adult participants (aged 18-40) living with FA in up to three cohorts and is designed to evaluate the safety and tolerability of contemporaneous IDN and systemic IV infusion of SGT-212, with initial data anticipated by the end of 2026, subject to participant enrollment. The FDA has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations to SGT-212 for the treatment of FA.
Previous wording
In July 2025, we announced that the FDA cleared our IND and that Health Canada approved our clinical trial application ("CTA") for SGT-501 for the treatment of CPVT. We anticipate activating the first clinical trial site for ARTEMIS, an open-label, multi-center Phase 1b clinical trial of SGT-501 in adult participants with CPVT in the fourth quarter of 2025. The FDA has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations to SGT-501 for the treatment of CPVT.
Current wording
In July 2025, we announced that the FDA cleared our IND and that Health Canada approved our clinical trial application for SGT-501 for the treatment of CPVT. In January 2026, we announced that clinical trial sites have been activated and participant screening is underway in the ARTEMIS clinical trial, an open-label, multi-center Phase 1b clinical trial evaluating SGT-501 in adult participants with CPVT. The ARTEMIS trial is designed to evaluate the safety and tolerability of a single IV infusion of SGT-501. We anticipate dosing our first participant in the second half of 2026 with initial safety data anticipated in the first half of 2027, subject to participant enrollment.
Previous wording
We are building multiple cardiac and neuromuscular next-generation capsid and promoter libraries with final capsid selection from the first cardiac capsid library anticipated in the first half of 2026.
Current wording
We are building cardiac and neuromuscular next-generation capsid and promoter libraries with capsid selection from the first library anticipated in the second half of 2026.
Previous 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 $45.8 million and $124.5 million for the three and nine months ended September 30, 2025, respectively, and $32.7 million and $82.1 million for the three and nine months ended September 30, 2024, respectively. As of September 30, 2025, we had an accumulated deficit of $908.0 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 $56.7 million and $39.3 million for the three months ended March 31, 2026 and 2025, respectively. As of March 31, 2026, we had an accumulated deficit of $1.0 billion. We expect to incur significant expenses and operating losses for the foreseeable future.
Previous wording
As we seek to develop and commercialize our Candidates, we anticipate that our expenses will increase significantly and that we will need substantial additional funding to support our continuing operations. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through a combination of public or private equity financing, debt financings or other sources, which may include licensing agreements or strategic collaborations. We may be unable to raise additional funds or enter into such agreements or arrangements when needed on favorable terms, if at all. If we fail to raise capital or enter into such agreements as and when needed, we may have to significantly delay, scale back or discontinue the development or commercialization of our Candidates.
Current wording
As we seek to develop and commercialize our Candidates, we anticipate that our expenses will increase significantly and that we will need substantial additional funding to support our continuing operations. Until we can generate significant revenue from product sales, if ever, we expect to finance our operations through a combination of public or private equity financing, debt financings or other sources, which may include licensing agreements or strategic collaborations. We may be unable to raise additional funds or enter into such agreements or arrangements when needed on favorable terms, if at all. If we fail to raise capital or enter into such agreements as and when needed, we may have to significantly delay, scale back or discontinue the development or commercialization of our Candidates.
Previous wording
As of September 30, 2025, we had cash, cash equivalents, and available-for-sale securities of $236.1 million, excluding restricted cash of $1.9 million. We believe that our cash, cash equivalents, and available-for-sale securities as of September 30, 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.
Current wording
As of March 31, 2026, we had cash, cash equivalents, and available-for-sale securities of $380.7 million, excluding restricted cash of $1.3 million. We believe that our cash, cash equivalents, and available-for-sale securities as of March 31, 2026 will enable us to fund our operating expenses and capital expenditure requirements into the first half of 2028. 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
We have not generated any commercial product revenue to date and do not expect to generate any product revenue from the sale of our products for the foreseeable future, if ever. If our development efforts for our Candidates are successful and result in marketing approval, we may generate commercial product revenue in the future.
Current wording
We have not generated any commercial product revenue to date and do not expect to do so for the foreseeable future, if ever. If our development efforts for our Candidates are successful and result in marketing approval, we may generate commercial product revenue in the future.
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 continue to conduct clinical trials for SGT-003 and SGT-212, initiate clinical trials for our other Candidates, including our planned ARTEMIS trial of SGT-501, 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 for most 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 continue to conduct our INSPIRE DUCHENNE, IMPACT DUCHENNE, FALCON and ARTEMIS trials and continue to develop our pipeline and complete ongoing and planned preclinical studies and clinical trials of our Candidates.
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, our IMPACT DUCHENNE trial, our FALCON trial, our planned ARTEMIS trial of SGT-501, and any other 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 IMPACT DUCHENNE trial, our FALCON trial, our ARTEMIS trial and any other planned or future clinical trials for and potential commercialization of our Candidates.
Previous wording
During the nine months ended September 30, 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:
Current wording
During the three months ended March 31, 2026, 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, 2025 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
Research and development expenses for the three months ended September 30, 2025 were $38.9 million, compared to $27.3 million for the three months ended September 30, 2024. The increase of $11.5 million in research and development expenses was primarily due to a $12.8 million increase in costs for SGT-003 primarily related to manufacturing, regulatory, and clinical costs, a $2.7 million increase in personnel related expenses, and a $0.9 million increase in costs for SGT-601 primarily related to manufacturing and research costs, partially offset by a $3.3 million decrease in costs for SGT-212 primarily related to lower license and milestone related costs partially offset by an increase in clinical costs, and a $1.8 million decrease in in costs for SGT‑501 primarily related to lower research and manufacturing costs.
Current wording
Research and development expenses for the three months ended March 31, 2026 were $46.1 million, compared to $30.9 million for the three months ended March 31, 2025. The increase of $15.2 million in research and development expenses was primarily due to a $13.6 million increase in costs for SGT-003 primarily related to manufacturing and clinical costs, a $3.3 million increase in personnel related expenses, a $2.5 million increase in costs for SGT-212 primarily related to clinical costs and license and milestone payments, and a $0.4 million increase in external expenses primarily related to consulting and professional services, partially offset by a $3.1 million decrease in costs for SGT-501 primarily related to lower manufacturing and research costs, and a $1.7 million decrease in costs for SGT-601 related to lower manufacturing and research costs.
Previous wording
General and administrative expenses were $27.6 million for the nine months ended September 30, 2025, compared to $24.2 million for the nine months ended September 30, 2024. The increase of $3.4 million was primarily related to a $3.7 million increase in personnel related costs and a $0.3 million increase in equipment costs, partially offset by a $0.3 million decrease in recruiting fees.
Current wording
General and administrative expenses were $11.2 million for the three months ended March 31, 2026, compared to $9.1 million for the three months ended March 31, 2025. The increase of $2.0 million was primarily related to a $1.5 million increase in personnel related costs, a $0.5 million increase in consulting services, and a $0.4 million increase in equipment costs, partially offset by a $0.5 million decrease in legal fees.
Previous wording
Other income, net was $2.3 million for the three months ended September 30, 2025 compared to $2.5 million for the three months ended September 30, 2024. The decrease of $0.2 million was primarily related to the change in fair value of derivative liabilities.
Current wording
Other income, net was $0.6 million for the three months ended March 31, 2026 compared to $0.8 million for the three months ended March 31, 2025. The decrease of $0.2 million was primarily related to lower interest income from our available-for-sale securities.
Previous 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 September 30, 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 $858.5 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.
Current wording
To date, we have financed our operations primarily through 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, 2026, 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 $1.1 billion 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, 2024, we sold 2,212,937 shares pursuant to the ATM Sales Agreement resulting in net proceeds of $18.4 million. During three and nine months ended September 30, 2025, we sold 236,616 and 298,158 shares pursuant to the ATM Sales Agreement resulting in net proceeds of $1.4 million and $1.7 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, 2025, we sold 891,414 shares pursuant to the ATM Sales Agreement resulting in net proceeds of $5.1 million. During the three months ended March 31, 2026, we sold 1,937,623 shares pursuant to the ATM Sales Agreement resulting in net proceeds of $11.9 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 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.
Current wording
On March 9, 2026, we issued and sold 14,973,257 shares of our common stock at a price of $5.61 per share, and, to certain investors in lieu of shares of common stock, pre-funded warrants to purchase 27,807,482 shares of its common stock at a price of $5.609 per pre-funded warrant, (the “March 2026 Private Placement”). We received approximately $226.3 million of aggregate net proceeds from the March 2026 Private Placement, after deducting offering costs.
Previous wording
As of September 30, 2025, we had cash, cash equivalents, and available-for-sale securities of $236.1 million, excluding restricted cash of $1.9 million, and had no debt outstanding.
Current wording
As of March 31, 2026, we had cash, cash equivalents, and available-for-sale securities of $380.7 million, excluding restricted cash of $1.3 million, and had no debt outstanding.
Previous wording
During the nine months ended September 30, 2025, operating activities used $102.7 million of cash, primarily resulting from our net loss of $124.5 million partially offset by changes in our operating assets and liabilities of $6.4 million and non-cash charges of $15.4 million. Net cash used by changes in our operating assets and liabilities during the nine months ended September 30, 2025 consisted of an increase in prepaid expenses and other assets of $0.3 million, and a decrease in the operating lease liability of $1.3 million, offset by an increase in accounts payable of $4.2 million, and an increase in accrued expenses and other liabilities of $3.9 million. Non-cash activities were driven by equity-based compensation of $10.4 million, a change in the fair value of derivative liabilities of $3.4 million, non-cash lease expense of $1.8 million, depreciation and amortization expense of $1.2 million, partially offset by amortization of available-for-sale securities of $1.1 million.
Current wording
During the three months ended March 31, 2026, operating activities used $47.2 million of cash, primarily resulting from our net loss of $56.7 million partially offset by changes in our operating assets and liabilities of $2.3 million and non-cash charges of $7.2 million. Net cash used by changes in our operating assets and liabilities during the three months ended March 31, 2026 consisted of an increase in accounts payable of $2.3 million and a decrease in prepaid expenses and other assets of $1.0 million, offset by a decrease in accrued expenses and other liabilities of $0.5 million and a decrease in the operating lease liability of $0.5 million. Non-cash activities were driven by equity-based compensation of $5.2 million, a change in the fair value of derivative liabilities of $1.7 million, non-cash lease expense of $0.6 million, and depreciation and amortization expense of $0.4 million, partially offset by amortization of discount on available-for-sale securities of $0.6 million.
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 of 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, 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, 2026, investing activities provided $44.8 million of cash, resulting from the maturities of available-for-sale securities of $128.4 million, partially offset by purchases of available-for sale securities of $83.5 million and the purchases of property plant and equipment of $0.2 million.
Previous wording
During the nine months ended September 30, 2025, investing activities used $105.5 million of cash, resulting from the purchases of available-for sale securities of $212.0 million and the purchases of property plant and equipment of $0.6 million, partially offset by the maturity of available-for sale securities of $107.0 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, 2025, financing activities provided $189.3 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 $189.6 million, and proceeds from the issuance of shares of $0.2 million under our Amended and Restated 2021 Employee Stock Purchase Plan, or ESPP, offset by payments of the principal portion of finance lease obligations of $0.5 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 in the February 2025 Offering, offset by payments of the principal portion of finance lease obligations of $0.1 million.
Previous wording
enroll participants in our INSPIRE DUCHENNE, IMPACT DUCHENNE and FALCON trials and advance clinical development of SGT-003 and SGT-212;
Current wording
enroll participants in our INSPIRE DUCHENNE, IMPACT DUCHENNE, FALCON and ARTEMIS trials and advance clinical development of SGT-003, SGT-212 and SGT-501;
Previous wording
advance our other Candidates into clinical trials, including our planned ARTEMIS trial of SGT-501;
Current wording
advance our other Candidates into clinical trials;
Previous wording
As of September 30, 2025, we had cash, cash equivalents and available-for-sale securities of $236.1 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, 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.
Current wording
As of March 31, 2026, we had cash, cash equivalents and available-for-sale securities of $380.7 million, excluding restricted cash of $1.3 million. Based on our current operating plan, we believe that our cash, cash equivalents and available-for-sale securities as of March 31, 2026 will be sufficient to fund our operating expenses and capital requirements into the first half of 2028. 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
the progress, costs and results of the INSPIRE DUCHENNE, IMPACT DUCHENNE and FALCON trials, our planned ARTEMIS trial of SGT-501, and any future clinical trials of our Candidates;
Current wording
the progress, costs and results of the INSPIRE DUCHENNE, IMPACT DUCHENNE, FALCON and ARTEMIS trials and any future clinical trials of our Candidates;
Previous wording
We are supplying, and expect to continue to supply, our ongoing and future preclinical and clinical development programs with drug product produced at a cGMP compliant facility located at one of our CDMOs. 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 preclinical and clinical development programs with drug product produced at a current GMP compliant facility located at one of our CDMOs. We intend to establish the capability and capacity to supply Candidates at commercial scale from multiple sources.
Previous wording
ownership interest may be diluted. Any debt or preferred equity financing, if available, may involve agreements that include restrictive covenants that may limit our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends, which could adversely impact our ability to conduct our business, and may require the issuance of warrants, which could potentially dilute existing stockholders’ ownership interests.
Current wording
Adequate additional funds may not be available to us on acceptable terms, or at all. We do not currently have any committed external source of funds. To the extent that we raise additional capital through the sale of equity securities, our existing stockholders’ ownership interest may be diluted. Any debt or preferred equity financing, if available, may involve agreements that include restrictive covenants that may limit our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends, which could adversely impact our ability to conduct our business, and may require the issuance of warrants, which could potentially dilute existing stockholders’ ownership interests.
Previous wording
During the nine months ended September 30, 2025, 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, 2024.
Current wording
During the three months ended March 31, 2026, 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, 2025.
Rearranged current paragraphs (3)
Current report — wording carried from elsewhere
In January 2025, we announced that the FDA cleared our IND for SGT-212 for the treatment of FA. In October 2025, we activated the first clinical trial site and began screening participants for FALCON, an open-label, multi-center Phase 1b clinical trial of SGT-212, and in January 2026, we dosed the first participant in the trial. Two participants have been dosed in the FALCON clinical trial and SGT-212 has been well tolerated with no serious adverse events observed as of May 11, 2026.
Current report — wording carried from elsewhere
The FDA has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations to SGT-501 for the treatment of CPVT.
Current report — wording carried from elsewhere
During the three months ended March 31, 2026, financing activities provided $239.0 million of cash, primarily resulting from the net proceeds from the issuance and sale of common stock and pre-funded warrants to purchase shares of common stock in the March 2026 Private Placement.
Rearranged previous paragraphs (5)
Previous report — wording carried elsewhere
The following table summarizes our results of operations for the periods indicated (in thousands, except percentages):
Previous report — wording carried elsewhere
The following table summarizes our research and development expenses by Candidate for the periods indicated (in thousands, except percentages):
Previous report — wording carried elsewhere
Research and development expenses for the nine months ended September 30, 2025 were $102.2 million, compared to $65.7 million for the nine months ended September 30, 2024. The increase of $36.5 million in research and development expenses was primarily due to a $30.1 million increase in costs for SGT-003 primarily related to manufacturing and clinical costs, a $7.0 million increase in personnel related expenses, a $4.9 million increase in costs for SGT-601 primarily related to manufacturing and research costs, partially offset by a $2.3 million decrease in costs for SGT-501 primarily related to lower manufacturing and study costs partially offset by an increase in clinical, regulatory and licensing fees, a $2.1 million decrease in other development programs primarily related to lower research and manufacturing costs, and a $1.5 million decrease in costs for SGT-212 primarily related to lower license and milestone payments partially offset by an increase in clinical, regulatory, and research costs.
Previous report — wording carried elsewhere
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 ESPP, offset by payments of the principal portion of finance lease obligations of $0.3 million.
Previous report — wording carried elsewhere
Adequate additional funds may not be available to us on acceptable terms, or at all. We do not currently have any committed external source of funds. To the extent that we raise additional capital through the sale of equity securities, our existing stockholders’