Scholar Rock Holding Corp

Previous wording

We caution readers not to place undue reliance upon any such forward-looking statements, which speak only as of the date they are made. We disclaim any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in our expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.

Current wording

We caution readers against placing undue reliance upon any such forward-looking statements, which speak only as of the date they are made. We disclaim any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in our expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.

Previous 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.

Current wording

We are a global biopharmaceutical company dedicated to improving the lives of children and adults with spinal muscular atrophy (“SMA”) and additional rare, severe and debilitating neuromuscular diseases. As a leader in the biology of transforming growth factor beta (“TGFβ”) superfamily, our novel understanding of the molecular mechanisms of growth factor activation enabled the development of a proprietary platform for the discovery and development of monoclonal antibodies that locally and selectively target the precursor, or latent, forms of growth factors. Based on our innovative, proprietary, and scalable technology platform, we are building a world-leading anti-myostatin pipeline.

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 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.

Current wording

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 from secondary endpoint analyses in which apitegromab demonstrated a clinically meaningful and consistent benefit in motor function across pre-specified patient subgroups. We submitted a BLA 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 CRL from the FDA related to observations identified during an FDA 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 related to the facility and were 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 an in-person Type A meeting with the FDA that included participation of representatives from the third-party

Previous wording

Using our innovative approach and proprietary platform, we are creating a pipeline of novel product candidates that selectively modulate the activation of growth factors implicated in a variety of serious diseases with high unmet need, including neuromuscular disorders, cardiometabolic disorders, cancer, and other serious diseases where growth factor-targeted therapies can play a transformational role. Our proprietary platform is designed to generate highly selective antibodies that target the growth factor’s latent precursor form prior to its activation within the disease microenvironment, or tissue where it is localized. Our structural insights and unique antibody discovery capabilities can also be applied to other protein classes beyond growth factors, with an aim of generating differentiated candidates targeting cell surface receptors such as immune cell receptors or G-protein coupled receptors, where selectivity remains challenging.

Current wording

Using our innovative approach and proprietary platform, we are creating a pipeline of novel product candidates that selectively modulate growth factor activation implicated in rare, severe, and devastating neuromuscular diseases.

Previous 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:

Current wording

We have incurred significant operating losses since inception. Our net losses were $105.5 million for the three months ended March 31, 2026. As of March 31, 2026, we had an accumulated deficit of $1.4 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

Research and development activities are central to our business model. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials, as well as the associated clinical trial material requirements. We expect research and development costs for our product candidates to continue to be substantial for the foreseeable future as the development programs progress. However, we do not believe that it is possible at this time to accurately project total program-specific expenses through commercialization. There are numerous factors associated with the successful commercialization of any of our product candidates, including future trial design and various regulatory requirements, many of which cannot be determined with accuracy at this time based on our stage of development. Additionally, future commercial and regulatory factors beyond our control will impact our clinical development programs and plans.

Current wording

Research and development activities are central to our business model. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials, as well as the associated clinical trial material requirements. We expect research and development costs for our product candidates to continue to be substantial for the foreseeable future as the development programs progress. However, we do not believe that it is possible at this time to accurately project total program-specific expenses through commercialization. There are numerous factors associated with the successful commercialization of any of our product candidates, including future trial design and various regulatory requirements, many of which cannot be determined with accuracy at this time based on our stage of

Previous 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:

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-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

A change in the outcome of any of these variables with respect to the development of apitegromab, SRK-181, SRK-439, SRK-373, SRK-256 or any of our future product candidates could significantly change the costs and timing associated with the development of that product candidate.

Current wording

Any of these variables, or other factors, with respect to the development of apitegromab, SRK-181, SRK-439, SRK-373, SRK-256 or any of our future product candidates could significantly change the costs and timing associated with the development of that product candidate.

Previous 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):

Current wording

Research and development expense was $51.8 million and $48.7 million for the three months ended March 31, 2026 and 2025, respectively, an increase of $3.1 million or 6.4%. The following table summarizes our research and development expense for the three months ended March 31, 2026 and 2025 (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, 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.

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, the conduct of our planned Phase 2 FORGE trial in FSHD patients, 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 continue to decrease, as we completed the Phase 1 DRAGON clinical trial in June 2025.

Previous 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.

Current wording

General and administrative expense was $50.2 million and $28.4 million for the three months ended March 31, 2026 and 2025, respectively, an increase of $21.8 million or 76.7%. The total increase was primarily driven by investments in infrastructure to support launch readiness for apitegromab, including an increase of approximately $11.5 million in employee-related costs including salaries, bonus, benefits and payroll taxes related to increased headcount, an increase of $2.3 million in non-cash equity-based compensation expense related to increased headcount, an increase of approximately $7.2 million in professional service fees and an increase of approximately $0.8 million in facility and other costs. 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 change in other income (expense), net was primarily attributable to an increase in interest income earned due to higher balances in our cash, cash equivalents and marketable securities.

Current wording

The change in other income (expense), net was primarily attributable to our loss on extinguishment of debt, partially offset by an increase in interest income earned due to higher average balances in our cash, cash equivalents and marketable securities.

Previous wording

Since our inception, we have not generated any product revenue and have incurred significant operating losses and negative cash flows from our operations. We have funded our operations to date primarily with proceeds from the sale of our convertible preferred stock and units in private placements before our initial public offering (“IPO”), and issuance of our common stock through our IPO in 2018, to Gilead in an exempt private placement, through multiple secondary public offerings and through at-the-market (“ATM”) sales, as well as payments from our research collaborations and the Loan and Security Agreement entered into in October 2020 and subsequently amended (see Note 11).

Current wording

Since our inception, we have not generated any product revenue and have incurred significant operating losses and negative cash flows from our operations. We have funded our operations to date primarily with proceeds from the sale of our convertible preferred stock and units in private placements before our initial public offering (“IPO”), and issuance of our common stock through our IPO in 2018, to Gilead in an exempt private placement, through multiple secondary public offerings and through “at-the-market offerings” (“ATM”) sales, as well as payments from our research collaborations and the 2026 Loan Agreement (as defined below) entered into in February 2026 (see Note 11).

Previous 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.

Current wording

During the three months ended March 31, 2026, our cash, cash equivalents and marketable securities balance increased by $112.4 million. The change was primarily due to proceeds from our debt facility, sales under our ATM program and the exercises of stock options, partially offset by 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.

Previous 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.

Current wording

Our current sales agreement with Jefferies, entered into in November 2022, allows for the sale of shares of our common stock from time to time in ATM offerings through Jefferies as the Company’s sales agent. During the three months ended March 31, 2026, we sold 2,576,299 shares of our common stock under the ATM program, generating net proceeds

Previous 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.

Current wording

In February 2025, we entered into an Amended and Restated Loan and Security Agreement with Oxford Finance LLC (“Oxford”) and Silicon Valley Bank (“SVB”) (such agreement, as amended, the “Existing Loan Agreement”) 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. This debt facility was paid off in February 2026 (see Note 11).

Previous 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.

Current wording

During the three months ended March 31, 2026, 6,804,082 of the Company’s pre-funded warrants were exercised. As of March 31, 2026, the Company had 10,558,065 pre-funded warrants outstanding.

Previous 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.

Current wording

During the three months ended March 31, 2026, none of the Company’s common warrants were exercised. As of March 31, 2026, the Company had 250,000 common warrants outstanding.

Previous wording

The following table provides information regarding our cash flows for the nine months ended September 30, 2025 and 2024 (in thousands):

Current wording

The following table provides information regarding our cash flows for the three months ended March 31, 2026 and 2025 (in thousands):

Previous 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.

Current wording

Net cash used in operating activities was $82.1 million for the three months ended March 31, 2026, and consisted of our net loss of $105.5 million, partially offset by changes in our assets and liabilities of $0.1 million and non-cash adjustments of $23.3 million. The non-cash adjustments are primarily from equity-based compensation.

Previous 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.

Current wording

Net cash used in operating activities was $78.7 million for the three months ended March 31, 2025, and consisted of our net loss of $74.7 million and changes in our assets and liabilities of $17.6 million, partially offset by non-cash adjustments of $13.6 million. The non-cash adjustments are primarily from equity-based compensation.

Previous 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.

Current wording

Net cash used in investing activities was $5.3 million for the three months ended March 31, 2026 compared to net cash provided by investing activities of $34.6 million for the three months ended March 31, 2025. Net cash provided by and used in investing activities for both periods was primarily associated with transactions involving our marketable securities.

Previous 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.

Current wording

Net cash provided by financing activities was $193.8 million for the three months ended March 31, 2026 compared to $4.2 million for the three months ended March 31, 2025. Net cash provided by financing activities for the three months ended March 31, 2026 was primarily attributable to $98.0 million in proceeds from the sale of common shares under our ATM program, $196.7 million in proceeds from our debt facility and $3.5 million from the exercise of stock options, partially offset by $103.1 million from the repayment of our Existing Loan Agreement with Oxford and $1.3 million of debt issuance costs related to our Financing Agreement with Blue Owl. Net cash provided by financing activities for the three months ended March 31, 2025 was primarily attributable to proceeds from the exercise of stock options and the proceeds from our debt refinancing, partially offset by the long-term debt extinguishment.

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 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.

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, global 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, and for programs in multiple other diseases beyond SMA where selective inhibition of myostatin activation may offer therapeutic benefit, such as our planned Phase 2 FORGE 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, 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

Current wording

Based on our current operating model, we expect that our existing cash, cash equivalents and marketable securities 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:

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

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 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.

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 “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.

Current wording

Based on the market value of our common stock held by our non-affiliates as of June 30, 2025, we are no longer a smaller reporting company. Accordingly, we ceased 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 are thus subject to additional disclosure and compliance requirements. Because we remained eligible to use the requirements for smaller reporting companies through December 31, 2025, we will remain a “non-accelerated filer” through December 31, 2026.