Scholar Rock agrees to $205M voucher sale to BioNTech
A $205 million voucher sale remains subject to closing conditions while ISEMBYLD begins U.S. commercialization and regulatory reviews advance overseas.
Sentiment and the balance of points
Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.
Rhea-AI Filing Summary
Scholar Rock Holding Corporation reported $570,000 in U.S. ISEMBYLD net product revenue for Q3 2026 and a net loss of $107.239 million, compared with $102.220 million in Q3 2025; net loss per share was $0.81 versus $0.90. Cash, cash equivalents and marketable securities were $413.599 million as of September 30, 2026.
Its wholly owned subsidiary Scholar Rock Inc. agreed to sell a Rare Pediatric Disease Priority Review Voucher to BioNTech US Inc. for $205 million in cash upon closing, subject to customary closing conditions, including expiration or termination of the applicable HSR waiting period. FDA approved ISEMBYLD on September 11, 2026, and the U.S. launch is underway, with initial patients infused in October 2026. Europe's apitegromab application was resubmitted and validated in October, with a CHMP opinion expected in H1 2027; Japan accepted the application under priority review. In the Phase 3 SAPPHIRE study, the company reported a +2.2-point HFMSE result at one year for ISEMBYLD plus SMN2-targeted therapy versus placebo plus therapy.
How this balance works
Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.
It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.
Rhea-AI Sentiment measures something else, the tone of the wording.
Hollow bars mark forward-looking points. How the balance works
Positive
- Moderate point. Forward-looking: it has not happened yet and may not happen.The agreed voucher sale is for $205 million in cash upon closing, subject to customary closing conditions. 3.3% of market cap
Negative
- None.
Filing Explained
The presentation reports
8-K Event Classification
Key Figures
Key Terms
Rare Pediatric Disease Priority Review Voucher regulatory
Marketing Authorisation Application regulatory
Hart-Scott-Rodino Antitrust Improvements Act regulatory
Hammersmith Functional Motor Scale-Expanded medical
Earnings Snapshot
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
How much is SRRK selling its Priority Review Voucher for?
What were SRRK's Q3 2026 financial results?
What is the status of SRRK's apitegromab applications in Europe and Japan?
AI-generated analysis. How Rhea-AI works. Not financial advice.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
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Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
Securities registered pursuant to Section 12(b) of the Act:
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item 1.01 Entry into a Material Definitive Agreement.
On November 9, 2026, Scholar Rock Holding Corporation’s (the “Company”) wholly owned subsidiary Scholar Rock Inc. (“Scholar Rock”) entered into an asset purchase agreement (the “PRV Asset Purchase Agreement”) with BioNTech US Inc. (“Buyer”) pursuant to which Scholar Rock agreed to sell a Rare Pediatric Disease Priority Review Voucher (“PRV”) to Buyer. Scholar Rock was awarded the voucher by the U.S. Food and Drug Administration (“FDA”) on September 11, 2026, upon approval of Scholar Rock’s biologics license application for ISEMBYLD™ (apitegromab-mstn). Pursuant to the PRV Asset Purchase Agreement, Buyer agreed to pay Scholar Rock $205 million, payable in cash, upon the closing of the sale.
The PRV Asset Purchase Agreement contains customary representations, warranties, covenants, and indemnification provisions subject to certain limitations. The transaction remains subject to customary closing conditions, including the expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976.
The foregoing description of the PRV Asset Purchase Agreement does not purport to be complete and is qualified in its entirety by the full text of the PRV Asset Purchase Agreement, a copy of which will be filed with the Company’s Annual Report on Form 10-K for the year ended December 31, 2026.
Item 2.02 Results of Operations and Financial Condition.
On November 10, 2026, Scholar Rock Holding Corporation (the “Company”) issued a press release announcing its financial and operating results for the quarter ended September 30, 2026. A copy of the press release is being furnished as Exhibit 99.1 to this Report on Form 8-K.
The information in this Current Report on Form 8-K and Exhibit 99.1 attached hereto is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 7.01. Regulation FD Disclosure.
The Company will utilize slides during its conference call scheduled for 8:00 am ET on November 10, 2026 to announce its financial and operating results for the fiscal quarter ended September 30, 2026 and to provide a business update. A copy of the slides is furnished as Exhibit 99.2 to this Current Report on Form 8-K and is incorporated by reference into this Item 7.01 as if fully set forth herein.
The information set forth in the immediately preceding paragraph in this Current Report on Form 8-K and Exhibit 99.2 attached hereto are intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Exchange Act, or otherwise subject to the liabilities of that section, nor shall they be deemed incorporated by reference in any filing under the Securities Act or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits
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Description |
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99.1 |
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Press Release issued by the Company on November 10, 2026, furnished hereto. |
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Scholar Rock Holding Corporation Corporate Presentation dated November 10, 2026, furnished hereto. |
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Cover Page Interactive Data File (embedded within the Inline XBRL document). |
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SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
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Scholar Rock Holding Corporation |
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Date: November 10, 2026 |
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/s/ Junlin Ho |
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Junlin Ho |
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General Counsel and Corporate Secretary |
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Exhibit 99.1
Scholar Rock Reports Third Quarter 2026 Financial Results and Recent Business Highlights
CAMBRIDGE, Mass.--(BUSINESS WIRE)— November 10, 2026 — Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of patients with rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology, today reported financial results for the third quarter ended September 30, 2026, and provided an update on recent company developments.
“The third quarter was a defining period for Scholar Rock, marked by the FDA approval and U.S. launch of ISEMBYLD, the world’s first and only muscle-targeted therapy for children and adults living with SMA,” said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. “We are very encouraged by the early ISEMBYLD launch dynamics in the U.S., where the breadth of early demand across SMA treatment centers, physicians, and patients reinforces the significant unmet need in the SMA community. With the U.S. launch underway, our regulatory applications advancing in Europe and Japan, important progress across our world-leading anti-myostatin pipeline, and our strengthened financial position, we are building a foundation to redefine the treatment of neuromuscular disease globally and drive sustainable growth for years to come.”
Business Highlights and Upcoming Milestones
Apitegromab Spinal Muscular Atrophy (SMA) Program
Apitegromab Facioscapulohumeral Muscular Dystrophy (FSHD) Program
SRK-439 Program
Financial Update
Third Quarter 2026 Financial Results
Scholar Rock reported a net loss of $107.2 million, including stock-based compensation of $17.9 million, for the quarter ended September 30, 2026, compared to a net loss of $102.2 million, including stock-based compensation of $18.3 million, for the quarter ended September 30, 2025. Net loss per common share was $0.81 for the quarter ended September 30, 2026, compared to $0.90 per common share for the quarter ended September 30, 2025.
Conference Call Information
Scholar Rock will host a conference call and webcast today, Tuesday, November 10, at 8:00 a.m. ET to review its third quarter 2026 financial results and discuss recent business updates. To access the live audio webcast, please go to “Events and Presentations” in the Investors section of the Scholar Rock website at http://investors.scholarrock.com.
To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call.
A replay of the webcast will be available on the Company’s website for approximately 90 days.
About Scholar Rock
Scholar Rock is delivering muscle-targeted breakthroughs to transform the treatment of spinal muscular atrophy (SMA) and other rare neuromuscular diseases where muscle atrophy remains a critical unmet need. Following FDA approval in September 2026, Scholar Rock is commercializing ISEMBLYD (apitegromab-mstn) in the U.S. for people living with SMA who are 2 years of age and older and currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. The Company plans to expand access to ISEMBYLD globally, in up to 50 countries worldwide, subject to regulatory approvals.
As a global leader in myostatin biology, a field focused on proteins that regulate muscle mass, the biopharmaceutical company is named for the visual resemblance of a scholar rock to protein structures. Our commitment to unlock fundamentally different treatment approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. Scholar Rock works every day to create new possibilities for patients through its highly innovative anti-myostatin program, including opportunities in additional rare neuromuscular diseases. Learn more about our neuromuscular franchise at ScholarRock.com and follow @ScholarRock on X and on LinkedIn.
Scholar Rock® is a registered trademark and ISEMBYLD™ is a trademark of Scholar Rock, Inc.
Availability of Other Information About Scholar Rock
Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X (formerly known as Twitter) and LinkedIn. The information that we post on our website, X, or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding the U.S. commercial launch and commercial performance of ISEMBYLD, including patient and physician
demand, launch dynamics, patient enrollment trends, market acceptance, coverage and reimbursement, and the timing of an ISEMBYLD-specific J-code; expectations regarding its growth, strategy, progress and timing of its clinical trials and development programs for apitegromab, including its subcutaneous formulation, the potential characteristics and benefits of such formulation, and its engagement with regulators regarding the development path for such formulation, as well as the ongoing Phase 2 OPAL and FORGE trials, and for SRK-439, including the expected timing of topline data from its Phase 1 clinical trial and the timing of public disclosure of such data, the timing of any regulatory submissions, decisions and anticipated approvals, and the therapeutic potential, clinical benefits and safety of any product candidates; expectations regarding the timing and outcome of EMA review of the resubmitted MAA for apitegromab, including the expected timing of a CHMP opinion in the first half of 2027; expectations regarding the timing and outcome of PMDA review of the JNDA for apitegromab under priority review; expectations regarding the availability and timing of commercial supply of ISEMBYLD and apitegromab, including from Scholar Rock’s alternate fill-finish facility; expectations regarding the closing of the sale of Scholar Rock’s PRV, including the satisfaction of closing conditions, including the expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act, and the anticipated receipt and use of proceeds therefrom; plans to expand access to ISEMBYLD globally, including in up to 50 countries worldwide, and expectations regarding commercial launch timing in additional jurisdictions, and the achievement of important milestones, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical or clinical data; and the potential of its product candidates and proprietary platform. The use of words such as “may,” “might,” “could,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, whether nonclinical and clinical data, including the results from the Phase 3 SAPPHIRE trial and any results from ongoing or future clinical trials, including the Phase 2 OPAL clinical trial, the Phase 2 FORGE trial and the Phase 1 clinical trial of SRK-439, will be sufficient to support regulatory approval or further development; that nonclinical and clinical data, including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, data from any ongoing or future trials of apitegromab or data for SRK-439, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidates; whether the resubmitted MAA and the JNDA will be sufficient to support regulatory approval of apitegromab in the European Union and Japan, respectively, on the expected timelines or at all; Scholar Rock’s ability to successfully commercialize ISEMBYLD, including achieving market acceptance among patients, physicians and payors and obtaining adequate coverage and reimbursement; whether early patient and physician demand for ISEMBYLD translates into sustained patient treatment and product revenue; Scholar Rock’s ability to obtain regulatory approval for, and to successfully expand access to, ISEMBYLD in additional countries; Scholar Rock’s ability to maintain sufficient commercial supply of ISEMBYLD, including through its alternate fill-finish facility; whether the conditions to the closing of the PRV sale will be satisfied on the expected timeline or at all; Scholar Rock’s need for additional capital and its ability to obtain it on acceptable terms; Scholar Rock’s ability to manage expenses or provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; information provided or decisions made by regulatory authorities, including regarding the development path for subcutaneous apitegromab; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; and Scholar Rock’s dependence on third parties for development and manufacture of apitegromab, ISEMBYLD and other product candidates including,
without limitation, to supply commercial demand for ISEMBYLD and any clinical trials as well as those risks more fully discussed in the section entitled “Risk Factors” in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2026, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law.
Scholar Rock Holding Corporation
Condensed Consolidated Statements of Operations
(unaudited)
(in thousands, except share and per share data)
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Three Months Ended September 30, |
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Nine Months Ended September 30, |
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2026 |
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2025 |
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2026 |
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2025 |
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Revenue: |
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Product revenue, net |
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$ |
570 |
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$ |
— |
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$ |
570 |
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$ |
— |
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Operating expenses: |
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Cost of revenue |
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8 |
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— |
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8 |
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- |
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Research and development |
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54,128 |
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50,486 |
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164,176 |
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161,565 |
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Selling, general and administrative |
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52,495 |
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53,064 |
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153,364 |
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131,184 |
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Total operating expenses |
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106,631 |
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103,550 |
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317,548 |
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292,749 |
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Loss from operations |
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(106,061 |
) |
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(103,550 |
) |
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(316,978 |
) |
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(292,749 |
) |
Other income (expense), net |
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(1,178 |
) |
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1,330 |
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(5,667 |
) |
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5,775 |
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Net loss |
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$ |
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(107,239 |
) |
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$ |
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(102,220 |
) |
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$ |
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(322,645 |
) |
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$ |
(286,974 |
) |
Net loss per share, basic and diluted |
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$ |
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(0.81 |
) |
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$ |
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(0.90 |
) |
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$ |
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(2.48 |
) |
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$ |
(2.54 |
) |
Weighted average common shares outstanding, basic and diluted |
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132,372,289 |
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113,729,850 |
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130,106,052 |
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112,763,974 |
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Scholar Rock Holding Corporation
Condensed Consolidated Balance Sheets
(unaudited)
(in thousands)
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September 30, 2026 |
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December 31, 2025 |
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Assets |
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Cash, cash equivalents and marketable securities |
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$ |
413,599 |
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$ |
367,563 |
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Other current assets |
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19,830 |
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17,584 |
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Total current assets |
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433,429 |
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385,147 |
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Other assets |
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12,543 |
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19,125 |
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Total assets |
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$ |
445,972 |
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$ |
404,272 |
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Liabilities and Stockholders' Equity |
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Current liabilities |
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$ |
80,124 |
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$ |
55,419 |
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Long-term liabilities |
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197,172 |
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103,365 |
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Total liabilities |
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277,296 |
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158,784 |
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Total stockholders' equity |
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168,676 |
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245,488 |
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Total liabilities and stockholders' equity |
|
$ |
445,972 |
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|
$ |
404,272 |
|
Investors
Laura Ekas, Ph.D.
917-439-0374
ir@scholarrock.com
Media
Jeff Smith
682-401-8428
Molly MacLeod, Ph.D.
802-579-5995
media@scholarrock.com
Q3 2026 Earnings Call November 10, 2026 © 2026 Scholar Rock, Inc. All rights reserved.
SMA, Spinal Muscular Atrophy. © 2026 Scholar Rock, Inc. All rights reserved. Q3 2026 Earnings Call 01 02 03 04 05 BUSINESS OVERVIEW David L. Hallal Chairman & Chief Executive Officer COMMERCIAL UPDATE Keith Woods Chief Operating Officer R&D PROGRESS Akshay Vaishnaw, M.D., Ph.D. President of R&D FINANCIAL RESULTS Vikas Sinha Chief Financial Officer Q&A SESSION
Forward-Looking Statements © 2026 Scholar Rock, Inc. All rights reserved. This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding the U.S. commercial launch and commercial performance of ISEMBYLD, including patient and physician demand, launch dynamics, patient enrollment trends, market acceptance, coverage and reimbursement, and the timing of an ISEMBYLD-specific J-code; expectations regarding its growth, strategy, progress and timing of its clinical trials and development programs for apitegromab, including its subcutaneous formulation, the potential characteristics and benefits of such formulation, and its engagement with regulators regarding the development path for such formulation, as well as the ongoing Phase 2 OPAL and FORGE trials, and for SRK-439, including the expected timing of topline data from its Phase 1 clinical trial and the timing of public disclosure of such data, the timing of any regulatory submissions, decisions and anticipated approvals, and the therapeutic potential, clinical benefits and safety of any product candidates; expectations regarding the timing and outcome of EMA review of the resubmitted MAA for apitegromab, including the expected timing of a CHMP opinion in the first half of 2027; expectations regarding the timing and outcome of PMDA review of the JNDA for apitegromab under priority review; expectations regarding the availability and timing of commercial supply of ISEMBYLD and apitegromab, including from Scholar Rock’s alternate fill-finish facility; expectations regarding the closing of the sale of Scholar Rock’s PRV, including the satisfaction of closing conditions, including the expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act, and the anticipated receipt and use of proceeds therefrom; plans to expand access to ISEMBYLD globally, including in up to 50 countries worldwide, and expectations regarding commercial launch timing in additional jurisdictions (including Europe and Japan), and the achievement of important milestones, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, or clinical data; and the potential of its product candidates and proprietary platform. The use of words such as “may,” “might,” “could,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, whether nonclinical and clinical data, including the results from the Phase 3 SAPPHIRE trial and any results from ongoing or future clinical trials, including the Phase 2 OPAL clinical trial, the Phase 2 FORGE trial and the Phase 1 clinical trial of SRK-439, will be sufficient to support regulatory approval or further development; that nonclinical and clinical data, including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, data from any ongoing or future trials of apitegromab or data for SRK-439, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidates; whether the resubmitted MAA and the JNDA will be sufficient to support regulatory approval of apitegromab in the European Union and Japan, respectively, on the expected timelines or at all; Scholar Rock’s ability to successfully commercialize ISEMBYLD, including achieving market acceptance among patients, physicians and payors and obtaining adequate coverage and reimbursement; whether early patient and physician demand for ISEMBYLD translates into sustained patient treatment and product revenue; Scholar Rock’s ability to obtain regulatory approval for, and to successfully expand access to, ISEMBYLD in additional countries; Scholar Rock’s ability to successfully launch ISEMBYLD in Europe and Japan on the expected timelines, or at all; Scholar Rock’s ability to maintain sufficient commercial supply of ISEMBYLD, including through its alternate fill-finish facility; whether the conditions to the closing of the PRV sale will be satisfied on the expected timeline or at all; Scholar Rock’s need for additional capital and its ability to obtain it on acceptable terms; Scholar Rock’s ability to manage expenses or provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; information provided or decisions made by regulatory authorities, including regarding the development path for subcutaneous apitegromab; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; and Scholar Rock’s dependence on third parties for development and manufacture of apitegromab, ISEMBYLD and other product candidates including, without limitation, to supply commercial demand for ISEMBYLD and any clinical trials as well as those risks more fully discussed in the section entitled “Risk Factors” in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2026, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law.
Business Overview David L. Hallal Chairman & Chief Executive Officer © 2026 Scholar Rock, Inc. All rights reserved.
© 2026 Scholar Rock, Inc. All rights reserved. BUILDING THE NEXT GLOBAL BIOTECH POWERHOUSE FDA APPROVED NOW First and only muscle-targeted therapy for adults and children ≥2 years of age with SMA U.S. Launch Underway Apitegromab MAA in Europe: Resubmitted MAA for treatment of children and adults with SMA; CHMP opinion expected in H1 2027 Apitegromab JNDA in Japan: Submitted JNDA for treatment of children and adults with SMA; accepted under priority review; launch anticipated near YE 2027 FORGE Phase 2 Study in FSHD: Commenced participant dosing; program granted Fast Track and Orphan Drug Designations by FDA Agreement to monetize PRV: Entered into definitive agreement to monetize PRV for $205M
ISEMBYLD™ is ushering in next phase of innovation in SMA © 2026 Scholar Rock, Inc. All rights reserved. 93% OF PATIENTS said gaining muscle strength is a significant unmet need in a 2026 Cure SMA survey1 ~3/4 recognize the future of treatment targets the motor neuron AND the muscle2 OF NEUROLOGISTS Motor unit consists of motor neuron and muscle MUSCLE MOTOR NEURON SMN-Targeted Therapies Muscle-Targeted Therapy INITIAL PHASE OF INNOVATION: MOTOR NEURON NEXT PHASE OF INNOVATION: MUSCLE 1. Cure SMA State of SMA 2025 Report; 2. Scholar Rock internal market research (US Neurologists). 2016 2026 2020 2019 2025
© 2026 Scholar Rock, Inc. All rights reserved. ISEMBYLD™ (apitegromab-mstn) is indicated for the treatment of spinal muscular atrophy (SMA) in adults and children 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment1 U.S. LAUNCH NOW UNDERWAY LOSS OF MOTOR FUNCTION GAIN OF MOTOR FUNCTION +2.2 POINT IMPROVEMENT IN HMFSE (p=0.0121*) Supported by global Phase 3 SAPPHIRE study, FDA-approved ISEMBYLD label provides a substantial opportunity to serve SMA patients in the U.S. HFMSE, Hammersmith Functional Motor Scale-Expanded; SMA, Spinal Muscular Atrophy; SMN, Survival Motor Neuron. *Nominal p-value; Based on ISEMBYLD™ 10 mg/kg + SMN-targeted therapy (nusinersen or risdiplam) versus placebo + SMN-targeted therapy. Change from baseline in HFMSE total score at 1 year with +1.0 change from baseline for ISEMBYLD™ 10 mg/kg vs. -1.2 change from baseline for the placebo. Population aged 2 – 12 years. ISEMBYLD prescribing information.
Broad-based demand in the earliest phase of launch as measured by volume and breadth of prescribers and patients As of October 31, 2026. © 2026 Scholar Rock, Inc. All rights reserved. SITES PRESCRIBING ISEMBYLD of Top 140 SMA treatment centers enrolled initial patients >1/3 all geographies nationwide BALANCED across Significant number of patients enrolled PATIENTS ENROLLED IN SCHOLAR ROCK SUPPORTS ISEMBYLD-treatment naïve Background therapy of Evrysdi, Spinraza, HD Spinraza, including some who previously received Zolgensma Transitioning from clinical trial and EAP Children and adults All trajectories of motor function Steady and consistent pace of demand in initial weeks of ISEMBYLD™ launch from sites OUTSIDE TOP 140
9 Opportunity to serve a steady and consistent number of new patients for quarters and years to come.
Commercial Update Keith Woods Chief Operating Officer © 2026 Scholar Rock, Inc. All rights reserved.
1. Data provided by Cure SMA U.S. SMA OPPORTUNITY FDA APPROVED NOW ISEMBYLD™ positioned to redefine the standard-of-care in SMA ~6,600 patients have received an approved SMN2-targeted therapy1 © 2026 Scholar Rock, Inc. All rights reserved.
© 2026 Scholar Rock, Inc. All rights reserved. Executed key ISEMBYLD™ launch priorities in September upon FDA approval ISEMBYLD vials labeled, packaged, and shipped to specialty network within one week Scholar Rock Supports immediately began working with physicians and families for initial patients J-code application filed for ISEMBYLD-specific code; expected to be available on April 1, 2027
Patient and caregiver awareness and engagement is high Registered for ISEMBYLD webinars ~1,000 PATIENTS & CAREGIVERS Email opt-ins from patients, caregivers and HCPs for ISEMBYLD communications 10,000 © 2026 Scholar Rock, Inc. All rights reserved. Extensive field team reach to Top 140 centers in U.S. >95% of Top 140 SMA Treatment Centers with Scholar Rock engagements through October HCP, Healthcare Professional.
Broad-based demand in the earliest phase of launch as measured by volume and breadth of prescribers and patients As of October 31, 2026. © 2026 Scholar Rock, Inc. All rights reserved. SITES PRESCRIBING ISEMBYLD of Top 140 SMA treatment centers enrolled initial patients >1/3 all geographies nationwide BALANCED across Significant number of patients enrolled PATIENTS ENROLLED IN SCHOLAR ROCK SUPPORTS ISEMBYLD-treatment naïve Background therapy of Evrysdi, Spinraza, HD Spinraza, including some who previously received Zolgensma Transitioning from clinical trial and EAP Children and adults All trajectories of motor function Steady and consistent pace of demand in initial weeks of ISEMBYLD™ launch from sites OUTSIDE TOP 140
Facilitating a clear path from prescription to infusion Broad and reliable access to ISEMBYLD™ to be established over time © 2026 Scholar Rock, Inc. All rights reserved. ISEMBYLD-SPECIFIC J-CODE PAYER POLICIES ESTABLISHED HOSPITAL FORMULARY APPROVALS PRIOR AUTHORIZATION PROCESS Key Factors Required to Reduce Time from Prescription to Infusion
98% of patients are enrolling in Scholar Rock Supports to assist with the treatment journey Source: Scholar Rock Supports. ROCC, Rx Onboarding and Care Coordinator. © 2026 Scholar Rock, Inc. All rights reserved. Providing personalized, dedicated support from prescription to infusion, which can take more than 60 to 90 days on average at this early stage of launch
WORKING TO BRING TO SMA PATIENTS GLOBALLY Building a 50-country operating platform to serve patients with rare, severe neuromuscular diseases UNITED STATES EUROPE REST OF WORLD Pending regulatory approvals outside the United States. © 2026 Scholar Rock, Inc. All rights reserved. JAPAN 17
R&D Progress Akshay Vaishnaw, M.D., Ph.D. President of R&D © 2026 Scholar Rock, Inc. All rights reserved.
Scholar Rock R&D priorities © 2026 Scholar Rock, Inc. All rights reserved. To enable treatment for children and adults with SMA in up to 50 countries worldwide SECURE GLOBAL APITEGROMAB REGULATORY APPROVALS Subcutaneous apitegromab, SMA patients <2 years of age, and new indications ADVANCE APITEGROMAB CLINICAL DEVELOPMENT SRK-439 is a high potency, investigational, subcutaneously administered myostatin inhibitor PROGRESS NEXT-GENERATION ANTI-MYOSTATIN PLATFORM 01 02 03
Securing regulatory approvals to bring apitegromab to children and adults with SMA in up to 50 countries globally MAA, Marketing Authorisation Application; EMA, European Medicines Agency; JNDA, Japanese New Drug Application; PMDA, Japan Pharmaceuticals and Medical Devices Agency. Europe Japan MAA resubmitted and validated by EMA Includes alternate fill-finish facility CHMP opinion anticipated H1 2027 JNDA submitted to PMDA, accepted under priority review Program granted Orphan Drug Designation © 2026 Scholar Rock, Inc. All rights reserved.
Advancing apitegromab clinical development programs Subcutaneous apitegromab and apitegromab have not been approved for the indications and uses discussed on this slide by the FDA or any other regulatory agency. © 2026 Scholar Rock, Inc. All rights reserved. PHASE 1 STUDY COMPLETE Subcutaneous Apitegromab Innovating for the SMA community to provide optionality as a self- or caregiver-administered anti-myostatin antibody Initiating engagements with regulators on the development path forward PHASE 2 STUDY Evaluating apitegromab in people living with FSHD in a randomized, double-blind, placebo-controlled trial expected to enroll 60 patients Patient dosing commenced in September Program granted Fast Track and Orphan Drug Designations by FDA Addressing the needs of children under 2 years of age with SMA including those who received SMN1-targeted treatment Participant enrollment and dosing ongoing PHASE 2 STUDY
© 2026 Scholar Rock, Inc. All rights reserved. Progressing SRK-439, our next generation anti-myostatin program PHASE 1 STUDY SRK-439 High potency, high affinity, investigational, subcutaneously administered myostatin inhibitor Enrollment and dosing ongoing On track to report topline data in early 2027 Single Ascending Dose PART A Cohorts 1 - 5 SRK-439 Placebo Multiple Ascending Dose PART B Cohorts 6 - 8 Repeated dosing per protocol schedule Repeated dosing per protocol schedule N ≈ 76 Healthy Adult Participants SRK-439 Placebo R R
Financial Update Vikas Sinha Chief Financial Officer © 2026 Scholar Rock, Inc. All rights reserved.
Q3 2026 Financial Results: Operating from a position of strength © 2026 Scholar Rock, Inc. All rights reserved. Reported $0.6 million in Q3, reflecting initial ISEMBYLD inventory shipments 1 Definitive agreement signed to sell Priority Review Voucher for additional $205 million* 2 Additional $150 million available under existing debt facility 3 $413.6M IN CASH, CASH EQUIVALENTS, AND MARKETABLE SECURITIES AS OF SEPTEMBER 30, 2026 PRIORITIZED INVESTMENTS ISEMBYLD launch in U.S. Strengthening supply chain Launch readiness in Europe and Japan Highly innovative clinical programs *Cash to be received upon closing of transaction.
Closing Remarks David L. Hallal Chairman & Chief Executive Officer © 2026 Scholar Rock, Inc. All rights reserved.
Q&A © 2026 Scholar Rock, Inc. All rights reserved.
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