Scholar Rock Holding Corp

Previous 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

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 fill-finish facility. Also in November 2025, the third-party fill-finish facility received a Warning Letter from the FDA. We resubmitted the apitegromab BLA in March 2026 with two third-party fill-finish facilities included, which was accepted by the FDA with a September 30, 2026 Prescription Drug User Fee Act (“PDUFA”) action date. 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. If apitegromab is approved by the FDA and/or the EC, we expect to initiate a commercial product launch in the applicable jurisdictions upon approval.

Previous wording

A Phase 2 study evaluating apitegromab in patients with FSHD is expected to initiate in mid-2026. We see potential for apitegromab broadly in additional rare, severe, and debilitating neuromuscular diseases where muscle atrophy is a key component of disease pathogenesis, and we are actively exploring indications beyond SMA and FSHD.

Current wording

A Phase 2 study evaluating apitegromab in patients with FSHD was initiated in the second quarter of 2026. We see potential for apitegromab broadly in additional rare, severe, and debilitating neuromuscular diseases where muscle atrophy is a key component of disease pathogenesis, and we are actively exploring indications beyond SMA and FSHD.

Previous 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:

Current wording

We have incurred significant operating losses since inception. Our net losses were $215.4 million for the six months ended June 30, 2026. As of June 30, 2026, we had an accumulated deficit of $1.5 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

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 development. Additionally, future commercial and regulatory factors beyond our control will impact our clinical development programs and plans.

Previous wording

Other income (expense), net consists primarily of interest income earned on our cash, cash equivalents and marketable securities, partially offset by interest expense incurred on our debt facility, including amortization of debt discount and debt issuance costs.

Current wording

Other income (expense), net consists primarily of interest income earned on our cash, cash equivalents and marketable securities, offset by interest expense incurred on our debt facility, including amortization of debt discount and debt issuance costs.

Previous 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):

Current wording

Research and development expense was $58.2 million and $62.4 million for the three months ended June 30, 2026 and 2025, respectively, a decrease of $4.2 million or 6.7%. The following table summarizes our research and development expense for the three months ended June 30, 2026 and 2025 (in thousands, except percentages):

Previous wording

The increase in research and development expense was primarily attributable to the following:

Current wording

The decrease in research and development expense was primarily attributable to the following:

Previous 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.

Current wording

General and administrative expense was $50.7 million and $49.7 million for the three months ended June 30, 2026 and 2025, respectively, an increase of $1.0 million or 1.9%. The total increase was primarily driven by investments in infrastructure to support commercial launch readiness for apitegromab, including an increase of approximately $9.3 million in employee-related costs including salaries, bonus, benefits and payroll taxes related to increased headcount, an increase of $2.2 million in non-cash equity based compensation expense, an increase of approximately $1.7 million in professional service fees and an increase of approximately $0.3 million in facility and other costs. The increase in headcount is partially associated with the hiring of our commercial and field-facing teams. These increases were partially offset by a decrease in costs associated with our leadership transition in 2025, including $8.6 million in non-cash equity-based compensation expense and $2.0 million in bonuses, as well as an overall decrease of $1.9 million in severance related costs. 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 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.

Current wording

The change in other income (expense), net was primarily attributable to interest expense incurred on our debt facility, including amortization of debt discount and debt issuance costs, partially offset by an increase in interest income earned due to higher average balances in our cash, cash equivalents and marketable securities.

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, 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.

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 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

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).

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 prior research collaborations, prior issuances of debt and the 2026 Loan Agreement (as defined below) entered into in February 2026 (see Note 11).

Previous 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.

Current wording

During the six months ended June 30, 2026, our cash, cash equivalents and marketable securities balance increased by $124.5 million. The change was primarily due to proceeds from our debt facility, sales under our ATM program and the exercises of stock options and warrants, 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, invested in building the infrastructure to support the commercialization of apitegromab and made interest payments on our debt.

Previous 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

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 six months ended June 30, 2026, we sold 3,543,692 shares of our common stock under the ATM program, generating net proceeds of $160.8 million.

Previous 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).

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 “Oxford 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

In February 2026, we entered into a Financing Agreement (the “Financing Agreement”) and a Pledge and Security Agreement (the “Security Agreement,” together with the Financing Agreement, the “2026 Loan Agreement”) with certain funds managed by Blue Owl Capital Corporation (“Blue Owl”) for up to $350.0 million, of which $100.0 million of the initial term loan (the “Initial Term Loan”) was received in February 2026. In addition, the Financing Agreement includes an uncommitted incremental term loan facility that requires the consent of Blue Owl in an aggregate principal amount not to exceed $200.0 million. We used the proceeds of the Initial Term Loan upon the closing under the 2026 Loan Agreement to repay all outstanding obligations, totaling $103.7 million, under the Existing Loan Agreement with Oxford and upon such repayment, terminated the Existing Loan Agreement. The amount repaid by the Company included $100.0 million of outstanding indebtedness plus accrued and unpaid interest as of February 27, 2026 and fees. We recognized a loss on extinguishment of debt of $3.3 million in connection with the termination of the Existing Loan Agreement. As a result of the termination, all credit commitments under the Existing Loan Agreement were terminated and all security interests and guarantees executed in connection with the Existing Loan Agreement were released. On March 31, 2026, the Company received $100.0 million from the next available tranche under the 2026 Loan Agreement, bringing the outstanding balance to $200.0 million (see Note 11).

Current wording

In February 2026, we entered into a Financing Agreement (the “Financing Agreement”) and a Pledge and Security Agreement (the “Security Agreement,” together with the Financing Agreement, the “2026 Loan Agreement”) with certain funds managed by Blue Owl Capital Corporation (“Blue Owl”) for up to $350.0 million, of which $100.0 million of the initial term loan (the “Initial Term Loan”) was received in February 2026. In addition, the Financing Agreement includes an uncommitted incremental term loan facility that requires the consent of Blue Owl in an aggregate principal amount not to exceed $200.0 million. We used the proceeds of the Initial Term Loan upon the closing under the 2026 Loan Agreement to repay all outstanding obligations, totaling $103.7 million, under the Oxford Loan Agreement with Oxford and upon such repayment, terminated the Oxford Loan Agreement. The amount repaid by the Company included $100.0 million of outstanding indebtedness plus accrued and unpaid interest as of February 27, 2026 and fees. We recognized a loss on extinguishment of debt of $3.3 million in connection with the termination of the Oxford Loan Agreement. As a result of the termination, all credit commitments under the Oxford Loan Agreement were terminated and all security interests and guarantees executed in connection with the Oxford Loan Agreement were released. On March 31, 2026, the Company received $100.0 million from the next available tranche under the 2026 Loan Agreement, bringing the outstanding balance to $200.0 million (see Note 11).

Previous 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.

Current wording

During the six months ended June 30, 2026, 6,804,082 of the Company’s pre-funded warrants were exercised. As of June 30, 2026, the Company had 10,558,065 pre-funded warrants outstanding.

Previous 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.

Current wording

During the six months ended June 30, 2026, 250,000 of the Company’s common warrants were exercised. As of June 30, 2026, the Company did not have any common warrants outstanding.

Previous wording

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

Current wording

The following table provides information regarding our cash flows for the six months ended June 30, 2026 and 2025 (in thousands):

Previous 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.

Current wording

Net cash used in operating activities was $152.4 million for the six months ended June 30, 2026, and consisted of our net loss of $215.4 million, partially offset by changes in our assets and liabilities of $17.0 million and non-cash adjustments of $46.0 million. The non-cash adjustments are primarily from equity-based compensation.

Previous 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.

Current 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.

Previous 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.

Current wording

Net cash used in investing activities was $10.8 million for the six months ended June 30, 2026 compared to net cash provided by investing activities of $120.0 million for the six months ended June 30, 2025.

Previous 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.

Current wording

Net cash provided by financing activities was $276.9 million for the six months ended June 30, 2026 compared to $10.6 million for the six months ended June 30, 2025. Net cash provided by financing activities for the six months ended June 30, 2026 was primarily attributable to $160.8 million in proceeds from the sale of common shares under our ATM program, $197.7 million in proceeds from our debt facility and $16.8 million from the exercise of stock options, partially offset by $103.1 million from the repayment of our Oxford Loan Agreement with Oxford and $2.5 million of debt issuance costs related to our Financing Agreement with Blue Owl.

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, 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.

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 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 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:

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 the second half of 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: