Scholar Rock Holding Corp
For the period ended Sep 30, 2026. Filed Nov 10, 2026.
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, 2026 · Filed Nov 10, 2026
SRRK$48.00-6.9%Stored close · as of Nov 10, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2026-06-30; filed 2026-08-06.
0001193125-26-336732
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2026-09-30; filed 2026-11-10.
0001193125-26-454434
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 126 previous paragraphs and 141 current paragraphs.
67 unchanged; 50 reworded; 24 added; 8 removed; 1 previous and 0 current paragraphs rearranged.
Tables are not compared: 6 previous and 6 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 (24)
Current report
In September 2026, we commenced commercialization of ISEMBYLD in the U.S. following FDA approval. As a result, we expect to generate product revenue from sales of ISEMBYLD while continuing to incur substantial expenses associated with commercialization activities, including sales, marketing, medical affairs, manufacturing, distribution and post-approval development activities. Although we have begun generating product revenue, we expect to continue to incur significant operating losses for the foreseeable future as we commercialize ISEMBYLD and advance our development pipeline.
Current report
Revenue
Current report
On September 11, 2026, the FDA approved ISEMBYLD for the treatment of SMA in adults and children two years of age and older who are currently receiving SMN2-targeted treatment. We began commercial shipments of ISEMBYLD in the U.S. during the third quarter of 2026 and therefore began recognizing product revenue during the quarter. We sell ISEMBYLD in the U.S. to a specialty distributor, which resells the ISEMBYLD to qualified health care providers, and to specialty pharmacies, which dispenses ISEMBYLD for administration to patients. We record product revenue net of estimated variable consideration, including chargebacks, government and commercial rebates, discounts, distributor and specialty pharmacy service fees, copay assistance and estimated product returns. Product revenue during the launch period reflects sales to our customers, including inventory build within the channel, and may not directly correspond to patient starts, infusions or underlying patient demand during the period.
Current report
Cost of product revenue includes the cost of producing and distributing inventories that are related to product revenue and may also include costs related to excess or obsolete inventory adjustment charges, abnormal costs, unabsorbed manufacturing and overhead costs, or other reasons. Based on our policy to expense costs associated with the manufacturing of our products prior to regulatory approval, certain of the manufacturing costs associated with product shipments of ISEMBYLD recorded during the three months ended September 30, 2026 were expensed prior to FDA approval of ISEMBYLD. This inventory carries a lower cost basis, resulting in lower cost of product revenue and higher gross margin during the initial commercialization period. We had expensed $25.6 million for this inventory prior to receiving approval on September 11, 2026.
Current report
Prior to the regulatory approval of our drug candidates, we incurred expenses for the manufacture of drug product supplies to support clinical development that could potentially be available to support the commercial launch of those drugs. Until the date at which regulatory approval has been received or is otherwise considered probable, we record all such costs as research and development expenses.
Current report
Selling, General and Administrative
Current report
NM - not meaningful
Current report
Product revenue, net
Current report
For the three months ended September 30, 2026, we recorded net product revenue of $0.6 million from sales of ISEMBYLD in the U.S. There was no net product revenue for the three months ended September 30, 2025.
Current report
For the three months ended September 30, 2026, we recorded cost of product revenue of $8,000 related to sales of ISEMBYLD in the United States. Prior to the FDA approval of ISEMBYLD, product costs were expensed as research and development expense.
Current report
$0.3 million increase in costs associated with other early development candidates and unallocated costs;
Current report
Total research and development expenses are expected to continue to be substantial as we support post-approval development activities for ISEMBYLD, including post-marketing requirements from the FDA by ISEMBYLD, ONYX, and OPAL, continue development activities in FSHD through the FORGE trial, advance the development of a subcutaneous formulation of apitegromab and continue development of SRK-439 and our broader pipeline. We expect costs of our SRK-181 program to continue to decrease, as compared to the prior year, as we completed the Phase 1 DRAGON clinical trial in June 2025.
Current report
Selling, general and administrative expense was $52.5 million and $53.1 million for the three months ended September 30, 2026 and 2025, respectively, a decrease of $0.6 million or 1.1%. The total decrease was primarily driven by a decrease in professional fees of $3.2 million, partially offset by an increase of approximately $2.6 million in employee-related costs including salaries, bonus, benefits and payroll taxes related to increased headcount. We expect selling, general and administrative expenses to continue to increase as we continue to invest in building the global infrastructure to support the commercialization of apitegromab.
Current report
Comparison of the Nine Months Ended September 30, 2026 and 2025
Current report
NM - not meaningful
Current report
Product revenue, net
Current report
In September 2026, the U.S. Food and Drug Administration (“FDA”) approved ISEMBYLD for the treatment of SMA in adults and children two years of age and older who are currently receiving a survival motor neuron 2 (“SMN2”)-targeted treatment. For the nine months ended September 30, 2026, we recorded net product revenue of $0.6 million from sales of ISEMBYLD in the U.S. There was no net product revenue for the nine months ended September 30, 2025.
Current report
For the nine months ended September 30, 2026, we recorded cost of product revenue of $8,000 related to sales of ISEMBYLD in the U.S. Prior to the FDA approval of ISEMBYLD, product costs were expensed as research and development expense.
Current report
o
Current report
o
Current report
0.5 million decrease in costs associated with other early development candidates and unallocated costs;
Current report
In connection with the approval of ISEMBYLD, we were issued a Rare Pediatric Disease priority review voucher (“PRV”) by the FDA. The PRV can be used to obtain priority review of a subsequent marketing application or sold or transferred to another sponsor. In November 2026, we entered into an asset purchase agreement with BioNTech US Inc., pursuant to which we agreed to sell our PRV for $205.0 million in cash upon the closing of the sale. The transaction remains subject to customary closing conditions. See Note 16, “Subsequent Events,” to our condensed consolidated financial statements.
Current report
the costs associated with maintaining regulatory approvals for ISEMBYLD, obtaining approvals in additional jurisdictions and pursuing approvals for additional indications;
Current report
the amount of revenue generated from commercial sales of ISEMBYLD and the market acceptance of the product;
Removed paragraphs (8)
Previous report
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 report
Beyond these clinical-stage product candidates, our early-stage pipeline includes additional preclinical programs intended for the treatment of patients with rare, severe, and debilitating neuromuscular diseases.
Previous report
To date, we have not generated any revenue from product sales. If we successfully obtain regulatory approval for apitegromab we may generate revenue in the future from product sales. In addition, if we obtain regulatory approval for apitegromab we have and expect to incur significant expenses related to developing our commercialization capabilities to support product sales, marketing and distribution activities.
Previous report
General and Administrative
Previous report
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.
Previous report
Comparison of the Six Months Ended June 30, 2026 and 2025
Previous report
the costs of seeking marketing approvals for apitegromab;
Previous report
the amount of revenue, if any, received from commercial sales of apitegromab, if approved;
Reworded paragraphs (50)
Previous wording
Our lead product candidates include apitegromab, a subcutaneous formulation of apitegromab, and SRK-439.
Current wording
We have one product approved in the U.S., ISEMBYLD™ (apitegromab-mstn), for the treatment of SMA, and our lead product candidates include a subcutaneous formulation of apitegromab, and SRK-439.
Previous wording
Apitegromab is a novel, investigational, fully human monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. Myostatin is a catabolic agent that functions as a negative regulator of muscle mass, therefore inhibition of myostatin results in increased muscle mass and strength. Apitegromab is in development for the treatment of people with SMA and for the treatment of people with facioscapulohumeral muscular dystrophy (“FSHD”).
Current wording
Apitegromab is a fully human monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. Myostatin is a catabolic agent that functions as a negative regulator of muscle mass; therefore, inhibition of myostatin results in increased muscle mass and strength. On September 11, 2026, the U.S. Food and Drug Administration (the “FDA”) approved ISEMBYLD for the treatment of SMA in adults and children two years of age and older who are currently receiving a survival motor neuron 2 (“SMN2”)-targeted treatment. ISEMBYLD is the first and only muscle-targeted treatment approved for patients with SMA currently receiving SMN2-targeted therapy, and we have initiated the U.S. commercial launch of ISEMBYLD. We plan to seek marketing authorizations for apitegromab for use in SMA in other jurisdictions. In October 2026, we resubmitted our Marketing Authorisation Application (“MAA”) for apitegromab for the treatment of children and adults with SMA to the European Medicines Agency (“EMA”). In November 2026, we submitted our Japanese New Drug Application (“JNDA”) for apitegromab for the treatment of children and adults with SMA to Japan’s Pharmaceuticals and Medicines Devices Agency (“PMDA”).
Previous 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.
Current wording
We continue to evaluate ISEMBYLD in SMA with its ongoing long-term extension ONYX trial and its Phase 2 OPAL trial in SMA patients under two years of age, which was initiated in the third quarter of 2025. Additionally, we are evaluating apitegromab for the treatment of patients with facioscapulohumeral muscular dystrophy (“FSHD”) with our Phase 2 FORGE trial, which 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
Our clinical-stage pipeline also includes SRK-439, a novel, investigational, subcutaneously administered fully human anti-pro/latent myostatin antibody that has high inhibitory potency while maintaining selectivity towards myostatin. SRK-439 is being developed for the treatment of patients with rare, severe, and debilitating neuromuscular diseases. A Phase 1 study of SRK-439 in healthy volunteers is currently underway, with topline data anticipated in the second half of 2026.
Current wording
Our clinical-stage pipeline also includes SRK-439, a novel, investigational, subcutaneously administered fully human anti-pro/latent myostatin antibody that has high inhibitory potency while maintaining selectivity towards myostatin. SRK-439 is being developed for the treatment of patients with rare, severe, and debilitating neuromuscular diseases. A Phase 1 study of SRK-439 in healthy volunteers is currently underway. The company expects to have topline data by year end 2026 and plans to share data from this study in the first quarter of 2027.
Previous 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.
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 debilitating neuromuscular diseases.
Previous wording
As we focus our strategy on rare neuromuscular diseases, we are currently seeking partnerships for our additional programs. These programs include: SRK-181, a Phase 2-ready investigational inhibitor of latent TGFβ1 in development for the treatment of patients with solid tumors that are resistant to anti-PD-(L)1 antibody therapies; SRK-373, an investigational, highly selective inhibitor of the latent TGFβ1 isoform with selective activity in the fibrotic extracellular matrix, in preclinical development for the treatment of fibrotic diseases; and SRK-256, an investigational inhibitor of RGMc, or hemojuvelin, in preclinical development for the treatment of iron-restricted anemias. We are also seeking partners to further evaluate the potential for myostatin inhibition in combination with GLP-1 weight loss approaches following our positive Phase 2 EMBRAZE study, demonstrating proof-of-concept in the ability of apitegromab to drive statistically significant preservation of lean mass during tirzepatide-induced weight loss.
Current wording
As we focus our strategy on rare neuromuscular diseases, we are currently seeking partnerships for our non-neuromuscular disease programs. These programs include: SRK-181, a Phase 2-ready investigational inhibitor of latent TGFβ1 in development for the treatment of patients with solid tumors that are resistant to anti-PD-(L)1 antibody therapies; SRK-373, an investigational, highly selective inhibitor of the latent TGFβ1 isoform with selective activity in the fibrotic extracellular matrix, in nonclinical development for the treatment of fibrotic diseases; and SRK-256, an investigational inhibitor of RGMc, or hemojuvelin, in nonclinical development for the treatment of iron-restricted anemias. We are also seeking partners to further evaluate the potential for myostatin inhibition in combination with GLP-1 weight loss approaches following our positive Phase 2 EMBRAZE study, demonstrating proof-of-concept in the ability of apitegromab to drive statistically significant preservation of lean mass during tirzepatide-induced weight loss.
Previous 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:
Current wording
We have incurred significant operating losses since inception. Our net losses were $322.6 million for the nine months ended September 30, 2026. As of September 30, 2026, we had an accumulated deficit of $1.6 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
continue development activities for apitegromab in SMA, including the ONYX study, our Phase 2 OPAL study for SMA patients under two years of age and the associated drug supply;
Current wording
continue development activities for ISEMBYLD in SMA, including the ONYX study, our Phase 2 OPAL study for SMA patients under two years of age and the associated drug supply;
Previous wording
Research and development expenses consist primarily of costs incurred for our research and development activities, including our product candidate discovery efforts, preclinical studies, manufacturing, and clinical trials under our research programs, which include:
Current wording
Research and development expenses consist primarily of costs incurred for our research and development activities, including our product candidate discovery efforts, nonclinical studies, manufacturing, and clinical trials under our research programs, which include:
Previous wording
expenses incurred under agreements with third parties that conduct research and development and preclinical activities on our behalf;
Current wording
expenses incurred under agreements with third parties that conduct research and development and nonclinical activities on our behalf;
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, 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 for indications other than SMA, 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 for indications other than SMA, 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
the scope, progress, outcome and costs of our preclinical development activities, clinical trials and other research and development activities;
Current wording
the scope, progress, outcome and costs of our nonclinical development activities, clinical trials and other research and development activities;
Previous 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.
Current wording
Any of these variables, or other factors, with respect to the development of apitegromab in indications other than SMA, 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
General and administrative expenses consist primarily of employee-related expenses, including salaries, benefits and equity-based compensation expenses for personnel in executive, finance, business development, investor relations, legal, information technology, human resources and commercial functions. Other significant general and administrative expenses include facility costs not otherwise included in research and development expenses, legal fees relating to patent and corporate matters and fees for accounting, consulting services, professional services and corporate expenses. We expect general and administrative expense to continue to be substantial as we continue to invest in building the infrastructure to support the commercialization of apitegromab.
Current wording
Selling, general and administrative expenses consist primarily of employee-related expenses, including salaries, benefits and equity-based compensation expenses for personnel in executive, finance, business development, investor relations, legal, information technology, human resources and commercial functions. Other significant selling, general and administrative expenses include facility costs not otherwise included in research and development expenses, legal fees relating to patent and corporate matters and fees for accounting, consulting services, professional services and corporate expenses. We expect selling, general and administrative expense to continue to increase as we support the commercial launch and ongoing commercialization of ISEMBYLD in the U.S. and continue to build infrastructure to support potential future launches in other jurisdictions, as well as in support of our research and development activities.
Previous 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):
Current wording
Research and development expense was $54.1 million and $50.5 million for the three months ended September 30, 2026 and 2025, respectively, an increase of $3.6 million or 7.2%. The following table summarizes our research and development expense for the three months ended September 30, 2026 and 2025 (in thousands, except percentages):
Previous wording
The decrease in research and development expense was primarily attributable to the following:
Current wording
The increase in research and development expense was primarily attributable to the following:
Previous wording
A decrease in our external research and development costs of $5.2 million, which primarily consisted of:
Current wording
A decrease in our external research and development costs of $0.1 million, which primarily consisted of:
Previous wording
$5.0 million decrease in costs associated with apitegromab primarily due to a decrease in drug supply manufacturing and a decrease in clinical trial costs as our Phase 3 SAPPHIRE and our Phase 2 EMBRAZE trials are completed, partially offset by an increase in costs related to our Phase 2 OPAL trial in SMA patients under two years of age and our Phase 2 FORGE trial in FSHD patients, which was initiated during the second quarter of 2026;
Current wording
$1.4 million decrease in costs associated with apitegromab primarily due to a decrease in drug supply manufacturing and a decrease in clinical trial costs as our Phase 3 SAPPHIRE and our Phase 2 EMBRAZE trials are completed, partially offset by an increase in costs related to our Phase 2 OPAL trial in SMA patients under two years of age and our Phase 2 FORGE trial in FSHD patients, which was initiated during the second quarter of 2026;
Previous wording
$0.8 million decrease in costs associated with SRK-181, as our Phase 1 DRAGON trial is completed; and
Current wording
$0.3 million decrease in costs associated with SRK-181, as our Phase 1 DRAGON trial is completed;
Previous wording
$1.2 million increase in costs associated with SRK-439 including costs associated with our Phase 1 study in healthy volunteers, partially offset by decreases in preclinical costs and manufacturing development.
Current wording
$1.2 million increase in costs associated with SRK-439 including costs associated with our Phase 1 study in healthy volunteers, partially offset by decreases in nonclinical costs and manufacturing development; and
Previous wording
A $1.1 million increase in internal research and development costs, which was primarily driven by an increase of $1.4 million in employee related costs, including salaries, bonus, benefits and payroll taxes related to increased headcount, an increase in non-cash equity-based compensation expense of $1.6 million related to increased headcount and severance related costs of $0.2 million, partially offset by a decrease in costs associated with our leadership transition in 2025, including $2.0 million in bonuses.
Current wording
A $3.8 million increase in internal research and development costs, which was primarily driven by an increase of $2.1 million in employee related costs, including salaries, bonus, benefits and payroll taxes related to increased headcount, an increase in non-cash equity-based compensation expense of $0.9 million related to increased headcount, as well as an increase of $0.8 million in facilities and other costs to support our internal research and development team.
Previous wording
The following table summarizes our results of operations for the six months ended June 30, 2026 and 2025 (in thousands, except percentages):
Current wording
The following table summarizes our results of operations for the nine months ended September 30, 2026 and 2025 (in thousands, except percentages):
Previous wording
Research and development expense was $110.0 million and $111.1 million for the six months ended June 30, 2026 and 2025, respectively, a decrease of $1.1 million or 0.9%. The following table summarizes our research and development expense for the six months ended June 30, 2026 and 2025 (in thousands, except percentages):
Current wording
Research and development expense was $164.2 million and $161.6 million for the nine months ended September 30, 2026 and 2025, respectively, an increase of $2.6 million or 1.6%. The following table summarizes our research and development expense for the nine months ended September 30, 2026 and 2025 (in thousands, except percentages):
Previous wording
The decrease in research and development expense was primarily attributable to the following:
Current wording
The increase in research and development expense was primarily attributable to the following:
Previous wording
A decrease in our external research and development costs of $8.1 million, which primarily consisted of:
Current wording
A decrease in our external research and development costs of $8.2 million, which primarily consisted of:
Previous wording
$4.0 million decrease in costs associated with apitegromab primarily due to a decrease in drug supply manufacturing and a decrease in clinical trial costs as our Phase 3 SAPPHIRE and our Phase 2 EMBRAZE trials are completed, partially offset by costs associated with our Phase 2 OPAL trial in SMA patients under two years of age and our Phase 2 FORGE trial in FSHD patients, which was initiated during the second quarter of 2026;
Current wording
$5.4 million decrease in costs associated with apitegromab primarily due to a decrease in drug supply manufacturing and a decrease in clinical trial costs as our Phase 3 SAPPHIRE and our Phase 2 EMBRAZE trials are completed, partially offset by costs associated with our Phase 2 OPAL trial in SMA patients under two years of age, our early access program for ISEMBYLD and our Phase 2 FORGE trial in FSHD patients, which was initiated during the second quarter of 2026;
Previous wording
$1.9 million decrease in costs associated with SRK-181, as our Phase 1 DRAGON trial is completed; and
Current wording
$2.2 million decrease in costs associated with SRK-181, as our Phase 1 DRAGON trial is completed;
Previous wording
$1.3 million decrease in preclinical costs and manufacturing development for SRK-439, partially offset by increases in clinical trial costs as we conduct our Phase 1 study in healthy volunteers.
Current wording
$0.1 million decrease in nonclinical costs and manufacturing development for SRK-439, partially offset by increases in clinical trial costs as we conduct our Phase 1 study in healthy volunteers; and
Previous wording
A $7.1 million increase in internal research and development costs, which was primarily driven by an increase of $3.7 million in employee related costs, including salaries, bonus, benefits and payroll taxes, an increase of $4.2 million in non-cash equity-based compensation expense related to increased headcount and an increase in severance costs of $1.0 million, partially offset by a decrease in costs associated with our leadership transition in 2025, including $2.0 million in bonuses.
Current wording
A $10.9 million increase in internal research and development costs, which was primarily driven by an increase of $5.8 million in employee related costs, including salaries, bonus, benefits and payroll taxes, an increase of $5.1 million in non-cash equity-based compensation expense related to increased headcount, an increase in severance costs of $1.0 million, as well as an increase of $1.0 million in facilities and other infrastructure costs, partially offset by a decrease in costs associated with our leadership transition in 2025, including $2.0 million in bonuses.
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 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 as we support post-approval development activities for ISEMBYLD, including ONYX and OPAL, continue development activities in FSHD through the FORGE trial, advance the development of a subcutaneous formulation of apitegromab and continue development of SRK-439 and our broader pipeline. We expect costs of our SRK-181 program to continue to decrease, as compared to the prior year, as we completed the Phase 1 DRAGON clinical trial in June 2025.
Previous wording
General and administrative expense was $100.9 million and $78.1 million for the six months ended June 30, 2026 and 2025, respectively, an increase of $22.8 million or 29.1%. The total increase was primarily driven by investments in infrastructure to support commercial launch readiness for apitegromab, including an increase of approximately $20.9 million in employee-related costs including salaries, bonus, benefits and payroll taxes related to increased headcount, an increase of $8.5 million in equity-based compensation expense, an increase of approximately $8.9 million in professional service fees and an increase of approximately $1.1 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 $12.6 million in non-cash equity-based compensation expense, a decrease of $2.0 million in bonuses, as well as an overall decrease of $2.1 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.
Current wording
Selling, general and administrative expense was $153.4 million and $131.2 million for the nine months ended September 30, 2026 and 2025, respectively, an increase of $22.2 million or 16.9%. The total increase was primarily driven by investments in infrastructure to support commercial launch readiness for apitegromab, including an increase of approximately $24.7 million in employee-related costs including salaries, bonus, benefits and payroll taxes related to increased headcount, an increase of $7.2 million in equity-based compensation expense, an increase of approximately $5.7 million in professional service fees and an increase of approximately $1.1 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 $12.6 million in non-cash equity-based compensation expense, a decrease of $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 global infrastructure to support the commercialization of apitegromab.
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 prior research collaborations, prior issuances of debt and the 2026 Loan Agreement (as defined below) entered into in February 2026 (see Note 11).
Current wording
Since our inception, our funding primarily derived from equity financings, debt financings and collaboration arrangements. Following the FDA approval of ISEMBYLD in September 2026, we began generating product revenue in the U.S.; however, product revenue has not yet been sufficient to fund our operations and we 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 13).
Previous 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.
Current wording
During the nine months ended September 30, 2026, our cash, cash equivalents and marketable securities balance increased by $46.0 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 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.
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 nine months ended September 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 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).
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 13). The additional delayed draw term loan commitment in an aggregate principal amount not to exceed $150.0 million became available on September 11, 2026, on the date of the FDA’s approval for ISEMBYLD in SMA. The additional loan will be available until the date that is the earliest of the full usage thereof, termination thereof and September 30, 2027, which may be borrowed by the Company at its sole option upon satisfying certain customary conditions, including satisfaction of the minimum cash covenant (if applicable).
Previous 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.
Current wording
During the nine months ended September 30, 2026, 6,804,082 of the Company’s pre-funded warrants were exercised. As of September 30, 2026, the Company had 10,558,065 pre-funded warrants outstanding.
Previous 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.
Current wording
During the nine months ended September 30, 2026, 250,000 of the Company’s common warrants were exercised. As of September 30, 2026, the Company did not have any common warrants outstanding.
Previous wording
The following table provides information regarding our cash flows for the six months ended June 30, 2026 and 2025 (in thousands):
Current wording
The following table provides information regarding our cash flows for the nine months ended September 30, 2026 and 2025 (in thousands):
Previous 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.
Current wording
Net cash used in operating activities was $234.2 million for the nine months ended September 30, 2026, and consisted of our net loss of $322.6 million, partially offset by changes in our assets and liabilities of $22.5 million and non-cash adjustments of $65.9 million. The non-cash adjustments are primarily from equity-based compensation.
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.
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Previous report — wording carried elsewhere
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.