Scholar Rock Holding Corp
For the period ended Sep 30, 2025. Filed Nov 14, 2025.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Sep 30, 2025 · Filed Nov 14, 2025
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-06-30; filed 2025-08-06.
0001558370-25-010400
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-09-30; filed 2025-11-14.
0001104659-25-111935
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 89 previous paragraphs and 83 current paragraphs.
49 unchanged; 27 reworded; 4 added; 12 removed; 1 previous and 3 current paragraphs rearranged.
Tables are not compared: 65 previous and 64 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 (4)
Current report
In addition to advancing development of apitegromab in neuromuscular diseases, we continue to advance our efforts for a second anti-myostatin product candidate and explore therapeutic benefit beyond neuromuscular disorders. In October 2023, we announced an expansion of our anti-myostatin program with SRK-439, a novel, fully human anti-myostatin
Current report
monoclonal antibody and in September 2025, an investigational new drug application (“IND”) for SRK-439 was cleared by the FDA. The Company plans to initiate dosing of SRK-439 in healthy volunteers in the fourth quarter of 2025.
Current report
Comparison of the Nine Months Ended September 30, 2025 and 2024
Current report
Research and development expense was $161.6 million and $134.2 million for the nine months ended September 30, 2025 and 2024, respectively, an increase of $27.4 million or 20.4%. The following table summarizes our
Removed paragraphs (12)
Previous report
Recent Developments
Previous report
Management Transition
Previous report
Effective April 27, 2025, our board of directors appointed David Hallal as our Chief Executive Officer of the Company following Jay T. Backstrom’s transition from his role as our Chief Executive Officer and President to Senior Advisor. Dr. Backstrom is expected to serve as Senior Advisor to us through October 31, 2025 as part of the planned transition. Our board of directors also appointed Akshay Vaishnaw, a current member of our board of directors, as our President of Research & Development, R. Keith Woods as our Chief Operating Officer and Vikas Sinha as our Chief Financial Officer, in each case effective April 27, 2025.
Previous report
Phase 2 EMBRAZE Trial Top-line Data
Previous report
On June 18, 2025, we announced positive top-line data from our Phase 2 EMBRAZE clinical trial evaluating apitegromab, an investigational muscle-targeted treatment, with tirzepatide to preserve lean mass during tirzepatide-induced weight loss.
Previous report
The EMBRAZE trial was designed to assess the ability to preserve lean body mass associated with tirzepatide-induced weight loss in patients with obesity (BMI of ≥30.0 kg/m 2) or overweight (BMI≥27.0 kg/m 2 with one or more weight-related co-morbidities). Treatment was administered over a 24-week period, and patients were randomized into two treatment arms: apitegromab with tirzepatide and placebo with tirzepatide.
Previous report
Topline results successfully demonstrated proof-of-concept for a highly selective, anti-myostatin antibody to preserve lean mass, with the potential to improve the quality of weight loss with tirzepatide therapy. The 24-week data demonstrated the following:
Previous report
Consistent with prior apitegromab studies, the EMBRAZE trial demonstrated a well tolerated and encouraging safety profile. The incidence of adverse events was generally similar between apitegromab and placebo, with adverse events observed consistent with the known safety profile of tirzepatide. No subjects experienced serious adverse events or discontinuations considered to be related to apitegromab treatment, and there were no deaths.
Previous report
Participants in both arms completed treatment at 24 weeks and the follow-up period is ongoing. Additional data from the trial will be presented at upcoming medical congresses.
Previous report
Comparison of the Six Months Ended June 30, 2025 and 2024
Previous report
Research and development expense was $111.1 million and $85.5 million for the six months ended June 30, 2025 and 2024, respectively, an increase of $25.6 million or 30.0%. The following table summarizes our research and development expense for the six months ended June 30, 2025 and 2024 (in thousands, except percentages):
Previous report
TOPAZ and SAPPHIRE studies, our Phase 2 OPAL trial in SMA patients under the age of two and the costs and timing of conducting future preclinical studies and clinical trials for SRK-439, SRK-373, SRK-256 or any other product candidates;
Reworded paragraphs (27)
Previous wording
We are a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for patients with SMA and other severe and debilitating neuromuscular diseases. As a global leader in the biology of TGFβ superfamily, our novel understanding of the molecular mechanisms of growth factor activation enabled us to develop a proprietary platform for the discovery and development of monoclonal antibodies that locally and selectively target the precursor, or latent, forms of growth factors. By targeting the signaling proteins at the cellular level and acting in the disease microenvironment, we believe we may avoid the historical dose-limiting safety challenges associated with inhibiting growth factors for therapeutic effect. We believe our focus on biologically validated growth factors may facilitate a more efficient development path.
Current wording
We are a global biopharmaceutical company dedicated to dramatically improving the lives of children and adults with SMA and additional rare, severe and debilitating neuromuscular diseases. As a global leader in the biology of TGFβ superfamily, our novel understanding of the molecular mechanisms of growth factor activation enabled us to develop a proprietary platform for the discovery and development of monoclonal antibodies that locally and selectively target the precursor, or latent, forms of growth factors. By targeting the signaling proteins at the cellular level and acting in the disease microenvironment, we believe we may avoid the historical dose-limiting safety challenges associated with inhibiting growth factors for therapeutic effect. We believe our focus on biologically validated growth factors may facilitate a more efficient development path.
Previous wording
Our first product candidate, apitegromab, is a highly selective, fully human, monoclonal antibody, with a unique mechanism of action that results in inhibition of the activation of the growth factor, myostatin, in skeletal muscle. Apitegromab is being developed as a potential first muscle-targeted treatment for SMA. In October 2024, we announced positive top-line results in SAPPHIRE, a pivotal Phase 3 clinical trial to evaluate the efficacy and safety of apitegromab in patients with non-ambulatory Type 2 and Type 3 SMA (which is estimated to represent the majority of the current prevalent SMA patient population in the U.S. and Europe). The study achieved its primary endpoint. At the March 2025 Muscular Dystrophy Association Clinical & Scientific Conference, we presented additional data related to secondary endpoint analyses in which apitegromab demonstrated a clinically meaningful and consistent benefit in motor function across pre-specified patient subgroups. We submitted a U.S. Biologics License Application to the FDA in January 2025, which is now under priority review with a PDUFA target action date of September 22, 2025. Priority review designation conveys that the FDA has determined that if apitegromab is approved, it could offer significant improvement in the safety or effectiveness of treatment of the serious condition of SMA. In March 2025 we submitted to the European Medicines Agency (“EMA”) and received validation of our marketing authorisation application (“MAA”) for apitegromab for the treatment of SMA. Validation confirms that the application includes the essential regulatory elements required for scientific assessment of the MAA and the scientific evaluation process by the EMA’s Committee for Medicinal Products for Human Use can begin. If apitegromab is approved on or around its PDUFA action date, we expect to initiate a commercial product launch in the fourth quarter of 2025 in the United States, with a commercial launch of apitegromab in Europe anticipated to follow in 2026.
Current wording
Our first product candidate, apitegromab, is a highly selective, fully human, monoclonal antibody, with a unique mechanism of action that results in inhibition of the activation of the growth factor, myostatin, in skeletal muscle. Apitegromab is being developed as a potential first muscle-targeted treatment for SMA. In October 2024, we announced positive top-line results in SAPPHIRE, a pivotal Phase 3 clinical trial to evaluate the efficacy and safety of apitegromab in patients with non-ambulatory Type 2 and Type 3 SMA (which is estimated to represent the majority of the current prevalent SMA patient population in the U.S. and Europe). The study achieved its primary endpoint. At the March 2025 Muscular Dystrophy Association Clinical & Scientific Conference, we presented additional data related to secondary endpoint analyses in which apitegromab demonstrated a clinically meaningful and consistent benefit in motor function across pre-specified patient subgroups. We submitted a U.S. Biologics License Application to the FDA in January 2025 and the BLA was granted priority review designation. Priority review designation conveys that the FDA has determined that if apitegromab is approved, it could offer significant improvement in the safety or effectiveness of treatment of the serious condition of SMA. In September 2025, we received a Complete Response Letter (“CRL”) from the FDA related to observations identified during a routine general site inspection of a third-party fill-finish facility. The facility was issued a Form 483 by the FDA in July 2025 and the inspection classification of this facility is “official action indicated” (OAI). The observations were site-related and not specific to apitegromab. The CRL did not cite any other approvability concerns, including apitegromab’s efficacy and safety data or the third-party drug substance manufacturer. In November 2025, we completed a constructive in-person Type A meeting with the FDA that included participation of representatives from the third-party fill-finish facility. We plan to resubmit the apitegromab BLA at such time as the facility has been reclassified by the FDA. In March 2025 we submitted to the European Medicines Agency (“EMA”) and received validation of our marketing authorisation application (“MAA”) for apitegromab for the treatment of SMA. Validation confirms that the application includes the essential regulatory elements required for scientific assessment of the MAA and the scientific evaluation process by the EMA’s Committee for Medicinal Products for Human Use can begin. If apitegromab is approved by the FDA or EMA, we expect to initiate a commercial product launch in the applicable jurisdictions upon approval.
Previous wording
We believe that apitegromab has the potential to be the first muscle-targeted treatment that is aimed at improving motor function in patients with SMA who are receiving an SMN-targeted therapy. We also have identified multiple other diseases for which the selective inhibition of the activation of myostatin may offer therapeutic benefit, including additional patient populations in SMA (such as patients with SMA under two years of age) and indications for other neuromuscular disorders beyond SMA. We plan to initiate the Phase 2 OPAL trial in SMA patients under two years of age in the third quarter of 2025.
Current wording
We believe that apitegromab has the potential to be the first muscle-targeted treatment that is aimed at improving motor function in patients with SMA who are receiving an SMN-targeted therapy. We also have identified opportunities to potentially expand the therapeutic benefit of apitegromab, including additional patient populations in SMA (such as patients with SMA under two years of age) and multiple other diseases beyond SMA where selective inhibition of myostatin activation may offer therapeutic benefit. We initiated the Phase 2 OPAL trial in SMA patients under two years of age in the third quarter of 2025.
Previous wording
In October 2023, we announced an expansion of our anti-myostatin program with SRK-439, a novel, fully human anti-myostatin monoclonal antibody. We are developing SRK-439 towards a potential investigational new drug application (“IND”) submission in the second half of 2025. In 2024, we presented preclinical data at scientific conferences which support the potential of SRK-439 to increase lean mass and contribute to a favorable body composition in conjunction with GLP-1 receptor agonist (“GLP-1 RA”) treatment. In June 2025, the Company announced positive top-line results from the Phase 2 EMBRAZE proof-of-concept trial, designed to assess the safety and efficacy of apitegromab to preserve lean mass in individuals living with obesity and on background therapy of a GLP-1 RA (tirzepatide). The trial demonstrated that patients receiving apitegromab with tirzepatide over 24 weeks showed a 54.9% preservation of lean mass (+4.2 lbs of lean mass) versus tirzepatide alone (p=0.001).
Current wording
In 2024, we presented preclinical data at scientific conferences which support the potential of SRK-439 to increase lean mass and contribute to a favorable body composition in conjunction with GLP-1 receptor agonist (“GLP-1 RA”) treatment. In June 2025, the Company announced positive top-line results from the Phase 2 EMBRAZE proof-of-concept trial, designed to assess the safety and efficacy of apitegromab to preserve lean mass in individuals living with obesity and on background therapy of a GLP-1 RA (tirzepatide). The trial demonstrated that patients receiving apitegromab with tirzepatide over 24 weeks showed a 54.9% preservation of lean mass (+4.2 lbs of lean mass) versus tirzepatide alone (p=0.001).
Previous wording
We have incurred significant operating losses since inception. Our net losses were $184.8 million for the six months ended June 30, 2025. As of June 30, 2025, we had an accumulated deficit of $1,107.5 million. We expect to continue to incur significant expenses and operating losses for the foreseeable future in performing our ongoing activities, as we:
Current wording
We have incurred significant operating losses since inception. Our net losses were $287.0 million for the nine months ended September 30, 2025. As of September 30, 2025, we had an accumulated deficit of $1.2 billion. We expect to continue to incur significant expenses and operating losses for the foreseeable future in performing our ongoing activities, as we:
Previous wording
The successful development of apitegromab, SRK-181, SRK-439, SRK-373, SRK-256 and any future product candidates is uncertain. Accordingly, at this time, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete the remainder of the development of apitegromab, SRK-181, SRK-439, SRK-373, SRK-256 and any future product candidates. We are also unable to predict when, if ever,
Current wording
The successful development of apitegromab, SRK-181, SRK-439, SRK-373, SRK-256 and any future product candidates is uncertain. Accordingly, at this time, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete the remainder of the development of apitegromab, SRK-
Previous wording
material net cash inflows will commence from the sale of our product candidates, if approved. This is due to the numerous risks and uncertainties associated with developing product candidates, including the uncertainty of:
Current wording
181, SRK-439, SRK-373, SRK-256 and any future product candidates. We are also unable to predict when, if ever, material net cash inflows will commence from the sale of our product candidates, if approved. This is due to the numerous risks and uncertainties associated with developing product candidates, including the uncertainty of:
Previous wording
Research and development expense was $62.4 million and $42.4 million for the three months ended June 30, 2025 and 2024, respectively, an increase of $20.0 million or 47.3%. The following table summarizes our research and development expense for the three months ended June 30, 2025 and 2024 (in thousands, except percentages):
Current wording
Research and development expense was $50.5 million and $48.7 million for the three months ended September 30, 2025 and 2024, respectively, an increase of $1.8 million or 3.6%. The following table summarizes our research and development expense for the three months ended September 30, 2025 and 2024 (in thousands, except percentages):
Previous wording
Total research and development expenses are expected to continue to be substantial, driven by employee compensation costs and development costs associated with our manufacture of drug supply, as well as development of our clinical stage programs as we continue development activities for apitegromab in SMA, the conduct of ONYX, our initiation of our Phase 2 OPAL trial in SMA patients under the age of two, as well as costs associated with supporting our anti-myostatin program, including our preclinical program, SRK-439. Additionally, we will continue to invest in our pipeline. We expect costs of our SRK-181 program to decrease, as we completed the Phase 1 DRAGON clinical trial in June 2025.
Current wording
Total research and development expenses are expected to continue to be substantial, driven by employee compensation costs and development costs associated with our manufacture of drug supply, as well as development of our clinical stage programs as we continue development activities for apitegromab in SMA, the conduct of ONYX, the conduct of our Phase 2 OPAL trial in SMA patients under the age of two, as well as costs associated with supporting our anti-myostatin program, including SRK-439. Additionally, we will continue to invest in our pipeline. We expect costs of our SRK-181 program to decrease, as we completed the Phase 1 DRAGON clinical trial in June 2025.
Previous wording
General and administrative expense was $78.1 million and $32.5 million for the six months ended June 30, 2025 and 2024, respectively, an increase of $45.6 million or 140.7%. The total increase was primarily associated with an approximately $9.6 million increase in employee-related costs including salaries, bonus, benefits and payroll taxes related to increased headcount, an increase of $4.6 million in one-time severance and other costs associated with our leadership change, an increase of $5.5 million in non-cash equity-based compensation expense related to increased
Current wording
General and administrative expense was $53.1 million and $16.1 million for the three months ended September 30, 2025 and 2024, respectively, an increase of $37.0 million or 230.4%. The total increase was primarily driven by investments in infrastructure to support launch readiness for apitegromab, including an increase of approximately $13.2 million in employee-related costs including salaries, bonus, benefits and payroll taxes related to increased headcount, an increase of $7.6 million in non-cash equity-based compensation expense related to increased headcount, severance related costs of $1.1 million and an increase of approximately $14.3 million in professional service fees. The increase in headcount is partially associated with the hiring of our commercial and field-facing teams. We expect general and administrative expenses to continue to be substantial as we continue to invest in building the infrastructure to support the commercialization of apitegromab.
Previous wording
The following table summarizes our results of operations for the six months ended June 30, 2025 and 2024 (in thousands, except percentages):
Current wording
The following table summarizes our results of operations for the nine months ended September 30, 2025 and 2024 (in thousands, except percentages):
Previous wording
Total research and development expenses are expected to continue to be substantial, driven by employee compensation costs and development costs associated with manufacture of drug supply, as well as our clinical stage programs as we continue development activities for apitegromab in SMA, the conduct of ONYX, our initiation of our Phase 2 OPAL trial in SMA patients under the age of two, as well as costs associated with supporting our anti-myostatin program, including our preclinical program, SRK-439. Additionally, we will continue to invest in our pipeline. We expect costs of our SRK-181 program to decrease, as we completed the Phase 1 DRAGON clinical trial in June 2025.
Current wording
Total research and development expenses are expected to continue to be substantial, driven by employee compensation costs and development costs associated with manufacture of drug supply, as well as our clinical stage programs as we continue development activities for apitegromab in SMA, the conduct of ONYX, our initiation of our Phase 2 OPAL trial in SMA patients under the age of two, as well as costs associated with supporting our anti-myostatin program, including SRK-439. Additionally, we will continue to invest in our pipeline. We expect costs of our SRK-181 program to decrease, as we completed the Phase 1 DRAGON clinical trial in June 2025.
Previous wording
General and administrative expense was $49.7 million and $17.1 million for the three months ended June 30, 2025 and 2024, respectively, an increase of $32.6 million or 190.3%. The total increase was primarily associated with an approximately $5.6 million increase in employee-related costs including salaries, bonus, benefits and payroll taxes related to increased headcount, an increase of $4.4 million in one-time severance and other costs associated with our leadership change, an increase of $4.7 million in non-cash equity-based compensation expense related to increased headcount, an increase in one-time charges in non-cash equity-based compensation expense of $8.6 million related to the modification of certain equity awards and an increase of approximately $8.8 million in professional service fees. We expect general and administrative expenses, excluding the $13.0 million in one-time charges associated with our leadership change, to continue to be substantial as we continue to invest in building the infrastructure to support the commercialization of apitegromab.
Current wording
General and administrative expense was $131.2 million and $48.5 million for the nine months ended September 30, 2025 and 2024, respectively, an increase of $82.7 million or 170.4%. The total increase was primarily driven by investments in infrastructure to support launch readiness for apitegromab, including an increase of approximately $22.8 million in employee-related costs including salaries, bonus, benefits and payroll taxes related to increased headcount, an increase of $5.8 million in severance and other costs associated with our leadership change, an increase of $13.1 million in non-cash equity-based compensation expense related to increased headcount, an increase in one-time charges in non-cash equity-based compensation expense of $12.6 million related to the modification of certain equity awards and an increase of approximately $27.1 million in professional service fees. The increase in headcount is partially associated with the hiring of our commercial and field-facing teams. We expect general and administrative expense, excluding the $18.4 million in charges associated with the leadership change, to continue to be substantial as we continue to invest in building the infrastructure to support the commercialization of apitegromab.
Previous wording
During the six months ended June 30, 2025, our cash, cash equivalents and marketable securities balance decreased by $142.3 million. The change was primarily due to cash used to operate our business, including payments related to, among other things, research and development and general and administrative expenses as we continued to invest in our product candidates and supported our internal research and development efforts and made interest payments on our debt, partially offset by proceeds from the exercises of stock options.
Current wording
During the nine months ended September 30, 2025, our cash, cash equivalents and marketable securities balance decreased by $67.6 million. The change was primarily due to cash used to operate our business, including payments related to, among other things, research and development and general and administrative expenses as we continued to invest in our product candidates and supported our internal research and development efforts and made interest payments on our debt, partially offset by proceeds from our debt facility, sales under our ATM program and the exercises of stock options.
Previous wording
Our current ATM program, established in November 2022, allows for the sale of shares of our common stock having an aggregate offering price of up to $100 million. As of June 30, 2025, the Company has sold 619,290 shares of our common stock, generating net proceeds of $5.2 million, under the ATM program. No sales were made under the ATM program during the six months ended June 30, 2025.
Current wording
Our current ATM program with Jefferies, established in November 2022, allows for the sale of shares of our common stock from time to time in “at-the-market” offerings through Jefferies as the Company’s sales agent. As of September 30, 2025, the Company has sold 3,386,290 shares of our common stock, generating net proceeds of $96.9 million, under the ATM program. Of this amount, we sold 2,767,000 shares of our common stock under the ATM program during the nine months ended September 30, 2025, generating net proceeds of $91.7 million.
Previous wording
On February 10, 2025, we entered into the Amended and Restated Loan and Security Agreement with Oxford for up to $200 million of which $25.0 million from Tranche 1 was received in October 2020 and $25.0 million from Tranche 2 was received in December 2021 (see Note 11). The Amended and Restated Loan and Security Agreement consolidates the existing outstanding loan tranches solely with Oxford.
Current wording
In February 2025, we entered into the Amended and Restated Loan and Security Agreement with Oxford for up to $200 million of which $25.0 million from Tranche 1 was received in October 2020, $25.0 million from Tranche 2 was received in December 2021 and $50.0 million from Tranche 3 was received in September 2025, bringing the total outstanding balance under the Term Loans to $100.0 million (see Note 11). The Amended and Restated Loan and Security Agreement consolidates the existing outstanding loan tranches solely with Oxford.
Previous wording
During the six months ended June 30, 2025, none of the Company’s pre-funded warrants were exercised. As of June 30, 2025, the Company had 17,362,147 pre-funded warrants outstanding.
Current wording
During the nine months ended September 30, 2025, none of the Company’s pre-funded warrants were exercised. As of September 30, 2025, the Company had 17,362,147 pre-funded warrants outstanding.
Previous wording
During the six months ended June 30, 2025, 458,044 of the Company’s common warrants were exercised, generating proceeds of $3.4 million. As of June 30, 2025, the Company had 8,220,620 common warrants outstanding.
Current wording
During the nine months ended September 30, 2025, 458,044 of the Company’s common warrants were exercised, generating proceeds of $3.4 million. As of September 30, 2025, the Company had 8,220,620 common warrants outstanding with an exercise price of $7.35, which expire on December 31, 2025.
Previous wording
The following table provides information regarding our cash flows for the six months ended June 30, 2025 and 2024 (in thousands):
Current wording
The following table provides information regarding our cash flows for the nine months ended September 30, 2025 and 2024 (in thousands):
Previous wording
Net cash used in operating activities was $155.6 million for the six months ended June 30, 2025, and consisted of our net loss of $184.8 million and changes in our assets and liabilities of $9.2 million, partially offset by non-cash adjustments of $38.4 million. The non-cash adjustments are primarily from equity-based compensation.
Current wording
Net cash used in operating activities was $224.5 million for the nine months ended September 30, 2025, and consisted of our net loss of $287.0 million, partially offset by changes in our assets and liabilities of $4.6 million and non-cash adjustments of $57.8 million. The non-cash adjustments are primarily from equity-based compensation.
Previous wording
Net cash used in operating activities was $99.0 million for the six months ended June 30, 2024, and consisted of our net loss of $115.4 million and changes in our assets and liabilities of $2.0 million, partially offset by non-cash adjustments of $18.4 million. The non-cash adjustments are primarily from equity-based compensation.
Current wording
Net cash used in operating activities was $151.4 million for the nine months ended September 30, 2024, and consisted of our net loss of $179.8 million and changes in our assets and liabilities of $0.6 million, partially offset by non-cash adjustments of $29.0 million. The non-cash adjustments are primarily from equity-based compensation.
Previous wording
Net cash provided by investing activities was $120.0 million for the six months ended June 30, 2025 compared to net cash provided by investing activities of $84.0 million for the six months ended June 30, 2024. Net cash provided by investing activities for both periods was primarily associated with transactions involving our marketable securities.
Current wording
Net cash provided by investing activities was $179.7 million for the nine months ended September 30, 2025 compared to net cash provided by investing activities of $119.3 million for the nine months ended September 30, 2024. Net cash provided by investing activities for both periods was primarily associated with transactions involving our marketable securities.
Previous wording
Net cash provided by financing activities was $10.6 million for the six months ended June 30, 2025 compared to $6.5 million for the six months ended June 30, 2024. Net cash provided by financing activities for the six months ended June 30, 2025 was primarily attributable to proceeds from the exercise of common warrants and stock options, partially offset by the net impact of our debt refinancing. Net cash provided by financing activities for the six months ended June 30, 2024 was primarily attributable to proceeds from the exercise of warrants.
Current wording
Net cash provided by financing activities was $155.1 million for the nine months ended September 30, 2025 compared to $6.7 million for the nine months ended September 30, 2024. Net cash provided by financing activities for the nine months ended September 30, 2025 was primarily attributable to $91.7 million in proceeds from the sale of common shares under our ATM program, $50.0 million in proceeds from our debt facility and $3.4 million from the exercise of common warrants and $10.9 million from the exercise of stock options, partially offset by the net impact of our debt refinancing. Net cash provided by financing activities for the nine months ended September 30, 2024 was primarily attributable to proceeds from the exercise of warrants.
Previous wording
We expect our expenses to be substantial as we continue the research and development of apitegromab in SMA. In addition, we are seeking marketing approval for apitegromab, and we expect to incur significant commercialization expenses related to product sales, marketing, manufacturing and distribution. We expect to continue to incur apitegromab development costs as we invest in trials to support other SMA patient populations, such as our Phase 2 OPAL clinical trial. We expect to incur costs to support our anti-myostatin program, including the close out activities for our Phase 2 EMBRAZE proof-of-concept trial of apitegromab and our preclinical program, SRK-439. Additionally, we will support the development of our pipeline and any other preclinical programs. Furthermore, we expect to continue to incur costs associated with operating as a public company.
Current wording
We expect our expenses to be substantial as we continue the research and development of apitegromab in SMA. In addition, we are seeking marketing approval for apitegromab, and we expect to incur significant commercialization expenses related to product sales, marketing, manufacturing and distribution. We expect to continue to incur apitegromab development costs as we invest in trials to support other SMA patient populations, such as our Phase 2 OPAL clinical trial. We expect to incur costs to support our anti-myostatin program, including the close out activities for our Phase 2 EMBRAZE proof-of-concept trial of apitegromab and our Phase 1 trial for SRK-439. Additionally, we will support the development of our pipeline and any other preclinical programs. Furthermore, we expect to continue to incur costs associated with operating as a public company.
Previous wording
Based on our current operating model, which includes planned revenues, we expect that our existing cash, cash equivalents, marketable securities and cash available to us will enable us to fund our operating expenses and capital expenditure requirements into 2027. However, we will require additional capital in order to complete clinical development and commercialization for each of our current programs. We have based this estimate on assumptions that may prove to be wrong, and we may use our available capital resources sooner than we currently expect. Our future capital requirements will depend on many factors, including:
Current wording
Based on our current operating model, we expect that our existing cash, cash equivalents, marketable securities and cash available to us will enable us to fund our operating expenses and capital expenditure requirements into 2027. However, we will require additional capital in order to complete clinical development and commercialization for each of our current programs. We have based this estimate on assumptions that may prove to be wrong, and we may use our
Previous wording
If we raise funds through collaborations, strategic alliances or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or to grant licenses on terms that may not be favorable to us. Market volatility or other factors could also adversely impact our ability to access capital as and when needed. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Current wording
If we raise funds through collaborations, strategic alliances or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or to grant licenses on terms that may not be favorable to us. Market volatility or other factors could also adversely impact our ability to access capital as and when needed. If we are unable to raise additional funds through equity or debt financings
Previous wording
Based on the market value of our common stock held by our non-affiliates as of June 30, 2024, we were considered a “non-accelerated filer” and a “smaller reporting company,” each effective as of December 31, 2024. Based on the market value of our common stock held by our non-affiliates as of June 30, 2025, we will no longer be a smaller reporting company and will be considered a “large accelerated filer” effective as of December 31, 2025, and thus will be subject to additional disclosure and compliance requirements, accelerated filing deadlines, as well as the requirements of Section 404(b) of the Sarbanes-Oxley Act of 2002.
Current wording
Based on the market value of our common stock held by our non-affiliates as of June 30, 2024, we were considered a “smaller reporting company,” effective as of December 31, 2024. Based on the market value of our common stock held by our non-affiliates as of June 30, 2025, we will no longer be a smaller reporting company. Accordingly, we will cease to be eligible to use the requirements for a smaller reporting company beginning with our Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, and will thus be subject to additional disclosure and compliance requirements. Because we remain eligible to use the requirements for smaller reporting companies through December 31, 2025, we will continue to be a non-accelerated filer as of December 31, 2025 and will remain a “non-accelerated filer” through December 31, 2026.
Rearranged current paragraphs (3)
Current report — wording carried from elsewhere
research and development expense for the nine months ended September 30, 2025 and 2024 (in thousands, except percentages):
Current report — wording carried from elsewhere
available capital resources sooner than we currently expect. Our future capital requirements will depend on many factors, including:
Current report — wording carried from elsewhere
when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Rearranged previous paragraphs (1)
Previous report — wording carried elsewhere
headcount, an increase in one-time charges in non-cash equity-based compensation expense of $12.6 million related to the modification of certain equity awards and an increase of approximately $12.8 million in professional service fees. We expect general and administrative expense, excluding the $17.2 million in one-time charges associated with the leadership change, to continue to be substantial as we continue to invest in building the infrastructure to support the commercialization of apitegromab.