8-K
Scholar Rock Holding Corp (SRRK)
8-K
2026-01-12
For: 2026-01-12
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April 07, 2026
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
Date of Report (Date of earliest event Reported): January 12, 2026
Scholar Rock Holding Corporation
(Exact Name of Registrant as Specified in Charter)
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| Delaware | | 001-38501 | | 82-3750435 |
| (State or Other Jurisdiction of<br>Incorporation) | | (Commission File Number) | | (I.R.S. Employer Identification Number) |
301 Binney Street , 3rd Floor , Cambridge , MA **** 02142
(Address of Principal Executive Offices) (Zip Code)
( 857 ) 259-3860
(Registrant’s telephone number, including area code)
(Former name or former address, if changed since last report)
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:
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| ☐ | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
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| ☐ | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
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| ☐ | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
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| ☐ | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which registered |
|---|---|---|
| Common Stock, par value $0.001 per share | SRRK | The Nasdaq Global Select Market |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (17 CFR §230.405) or Rule 12b-2 of the Securities Exchange Act of 1934 (17 CFR §240.12b-2). 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 2.02 | Results of Operations and Financial Condition. |
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On January 12, 2026, Scholar Rock Holding Corporation (the “Company”) issued a press release announcing its preliminary cash balance as of December 31, 2025, a business update and further details on its 2026 strategic priorities.
Although it has not finalized its full financial results for the fourth quarter and fiscal year ended December 31, 2025, the Company announced on January 12, 2026, that it estimates it had approximately $365 million (unaudited) of cash, cash equivalents and marketable securities as of December 31, 2025.
This financial information is unaudited and preliminary and does not present all information necessary for an understanding of the Company’s financial condition as of December 31, 2025 and its results of operations for the fourth quarter and fiscal year ended December 31, 2025. The audit of the Company’s consolidated financial statements for the year ended December 31, 2025 is ongoing and could result in changes to the information set forth above.
The information contained in Item 2.02 of this Current Report on Form 8-K is being 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 (the “Securities Act”), or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 7.01. Regulation FD Disclosure.
On January 12, 2026, the Company issued a press release announcing business updates and further details on its 2026 strategic priorities. A copy of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K.
The information contained in Item 7.01 and Exhibit 99.1 of this Current Report on Form 8-K is being 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 it 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 a filing.
Item 8.01. Other Events.
The Company will be conducting meetings with participants attending the 44th Annual J.P. Morgan Healthcare Conference (the “Conference”) during the week of January 12, 2026. A copy of the slides to be presented by the Company at the Conference is filed as Exhibit 99.2 to this Current Report on Form 8-K. The Company undertakes no obligation to update, supplement or amend the materials attached hereto as Exhibit 99.2.
Forward-Looking Statements
This Current Report on Form 8-K contains “forward-looking statements” within the meaning of the federal securities laws, including Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. In this context, forward-looking statements often address expected future business and financial performance and financial condition, and often contain words such as “anticipate,” “believe,” “expect,” “intend,” “plan,” “seek,” “see,” “will,” “would,” “may,” “target,” and similar expressions and variations or negatives of these words. Forward-looking statements by their nature address matters that are, to different degrees, uncertain, such as statements regarding the Company’s expectations regarding its growth, strategy, progress and timing of its clinical trials for apitegromab, SRK-439 and its preclinical programs, and indication selection and development timing, including the timing of any regulatory submissions and anticipated approvals, the therapeutic potential, clinical benefits and safety of any product candidates, its ability to address the observations identified in the complete response letter, its cash runway into 2027, expectations regarding commercial launch timing in the US and in Europe, expectations regarding a new fill finish facility and the achievement of important milestones, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of its product candidates and proprietary platform. These and other forward-looking statements are not guarantees of future results and are subject to risks, uncertainties and assumptions that could cause actual results to differ materially from those expressed in any forward-looking statements. Important risk factors that may cause such a difference include, but are not limited to the risks described in the Company’s most recent quarterly report on Form 10-Q for the fiscal quarter ended September 30, 2025. We undertake no obligation to update any forward-looking statements as a result of new information, events or
circumstances or other factors arising or coming to our attention after the date hereof. The provision of the information in this report shall not be deemed an admission as to the materiality of any of the information contained herein.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits
| Exhibit<br><br>No. | Description | ||
|---|---|---|---|
| 99.1<br><br> | | Press release issued by Scholar Rock Holding Corporation on January 12, 2026, furnished herewith. | |
| 99.2 | | Corporate Presentation, dated January 12, 2026. | |
| 104 | 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.
| Scholar Rock Holding Corporation | ||
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| Date: January 12, 2026 | By: | /s/ Junlin Ho |
| Junlin Ho | ||
| General Counsel & Corporate Secretary |
| Exhibit 99.1<br>Scholar Rock Highlights 2026 Strategic Priorities<br>• Apitegromab Biologics License Application (BLA) resubmission and U.S. launch, following<br>FDA approval, are anticipated in 2026 for the treatment of children and adults with spinal<br>muscular atrophy (SMA)<br>• FDA has scheduled meeting with Catalent Indiana, LLC (part of Novo Nordisk) in early 2026<br>to discuss progress of remediation efforts<br>• Apitegromab Investigational New Drug (IND) application cleared for facioscapulohumeral<br>muscular dystrophy (FSHD); Phase 2 study initiation and patient dosing expected mid-2026<br>• Subcutaneous apitegromab advancing; Phase 1 study demonstrated favorable<br>bioavailability, with pharmacodynamic profile comparable to IV administration<br>• SRK-439 Phase 1 trial ongoing in healthy volunteers; topline data expected in H2 2026<br>• Approximately $365 million1 in cash as of December 31, 2025; cash runway expected to<br>support operations into 2027<br>CAMBRIDGE, Mass. — January 12, 2026 — Scholar Rock (NASDAQ: SRRK), a global<br>biopharmaceutical company dedicated to dramatically improving the lives of children and adults<br>with spinal muscular atrophy (SMA) and additional rare, severe, and debilitating neuromuscular<br>diseases by applying its leading platform in myostatin biology to advance musculoskeletal health,<br>today provided a corporate update and highlighted its strategic priorities for 2026.<br>“We believe 2026 will be a transformative year for Scholar Rock. We are executing with urgency,<br>and we have strong momentum across our rare, severe neuromuscular disease programs as we<br>build our foundation to become the next global biotech powerhouse,” said David L. Hallal,<br>Chairman and Chief Executive Officer of Scholar Rock. “Our foremost objective remains bringing<br>apitegromab to children and adults with SMA as quickly as possible, and we are confident in the<br>steps we are taking to support a BLA resubmission and a U.S. commercial launch, following<br>approval, this year.”<br>2026 Strategic Priorities<br>Scholar Rock is focused on three core strategic priorities in 2026 to create sustainable long-term<br>value and maximize the potential of its innovative anti-myostatin platform to serve patients:<br>• FDA and European Medicines Agency (EMA) regulatory approvals and commercialization of<br>apitegromab for children and adults with SMA.<br>• Develop apitegromab for patients with SMA (<2 years old) and for additional rare, severe<br>neuromuscular diseases.<br>1 Unaudited, estimated cash, cash equivalents and marketable securities as of December 31,<br>2025. |
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| Exhibit 99.1<br>• Advance world-leading anti-myostatin pipeline with SRK-439 in addition to apitegromab.<br>FDA and EMA regulatory approvals and commercialization of apitegromab for children and<br>adults with SMA<br>Scholar Rock remains focused on bringing apitegromab, the world’s first and only muscle-targeted<br>treatment candidate to improve motor function in patients with SMA, to patients as quickly as<br>possible.<br>• BLA resubmission and U.S. launch, following FDA approval, are anticipated in 2026.<br>Scholar Rock continues to work closely with the FDA and Catalent Indiana LLC, part of<br>Novo Nordisk, to progress remediation activities at the Bloomington, Indiana fill-finish<br>facility to support BLA resubmission as rapidly as possible.<br>• EMA regulatory review ongoing. The Company continues to expect a decision on its<br>apitegromab Marketing Authorisation Application (MAA) in mid-2026.<br>• U.S. and European launch preparations ongoing. U.S. and European customer-facing<br>teams are in the field and continue to engage with key stakeholders on SMA disease<br>awareness and education initiatives, including the unmet need in SMA and the importance<br>of targeting muscle.<br>• Second U.S. fill-finish facility commercial capacity reserved beginning Q1 2026.<br>Scholar Rock is advancing activities at a second U.S.-based fill-finish facility to strengthen<br>supply continuity and support future commercial demand. This facility manufactures<br>commercially available, FDA-approved products and has a strong regulatory inspection<br>track record. The Company expects to submit a supplemental BLA (sBLA) with this second<br>site later in 2026.<br>Develop apitegromab for patients with SMA (<2 years old) and for additional rare, severe<br>neuromuscular diseases<br>As part of Scholar Rock’s long-term strategy, the Company is developing apitegromab for<br>patients with SMA under two years of age and for additional rare, severe, and debilitating<br>neuromuscular diseases.<br>• Phase 2 OPAL clinical trial underway. The Phase 2 OPAL trial is designed to evaluate<br>apitegromab in infants and toddlers with SMA under two years of age who have received an<br>approved SMN1-targeted gene therapy or who are receiving ongoing treatment with an<br>approved SMN2-targeted therapy. The trial is enrolling participants and patient dosing is<br>underway.<br>• Phase 2 FORGE trial on track to initiate in mid-2026. Scholar Rock is developing<br>apitegromab for the treatment of people living with facioscapulohumeral muscular<br>dystrophy (FSHD). FSHD is a rare, progressive neuromuscular disease characterized by<br>muscle atrophy and functional decline, affecting approximately 30,000 individuals across<br>the U.S. and Europe. The IND application is cleared, and the Company plans to initiate a<br>Phase 2 randomized, double-blind, placebo-controlled trial, called FORGE, in mid-2026.<br>Advance world-leading anti-myostatin pipeline with SRK-439 in addition to apitegromab |
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| Exhibit 99.1<br>As the first and only company to deliver a successful Phase 3 study with a myostatin inhibitor,<br>Scholar Rock is building a robust pipeline of anti-myostatin product candidates to serve patients by<br>addressing a broad range of rare, severe, and debilitating neuromuscular diseases.<br>• Subcutaneous apitegromab Phase 1 study complete. Scholar Rock is advancing a<br>subcutaneous presentation of apitegromab. This formulation is intended to provide<br>optionality for patients as a small volume, self- or caregiver-administered anti-myostatin<br>antibody suitable for an autoinjector. A Phase 1 study in healthy volunteers has been<br>completed, and further development activities are ongoing, including planned FDA and<br>EMA regulatory engagements.<br>• Dosing underway in SRK-439 Phase 1 healthy volunteer study. SRK-439 is a novel,<br>investigational, subcutaneously administered myostatin inhibitor that binds to pro- and<br>latent myostatin with high affinity and selectivity (i.e., no GDF11 or Activin A binding). SRK-439 has demonstrated the potential to potently inhibit myostatin and increase muscle<br>mass in preclinical studies. A Phase 1 study in healthy volunteers is underway, and topline<br>data are expected in the second half of 2026.<br>Corporate Updates<br>As of December 31, 2025, Scholar Rock had cash, cash equivalents, and marketable securities of<br>approximately $365 million. This cash is expected to fund operations into 2027.<br>2025 Highlights and Accomplishments<br>• Progressed U.S. and European regulatory activities for apitegromab for the treatment<br>of children and adults with SMA. Scholar Rock submitted a BLA to the FDA in January<br>2025, received Priority Review designation in March 2025, and achieved acceptance of the<br>EMA MAA in March 2025, representing significant regulatory milestones toward<br>commercialization. The Company completed a constructive and collaborative in-person<br>Type A meeting with the FDA in November 2025, following the receipt of a Complete<br>Response Letter (CRL) from FDA on September 22, 2025.<br>• Transformed leadership team. Appointed 8-year Board Chairman, David Hallal, as Chief<br>Executive Officer; Akshay Vaishnaw, M.D., Ph.D. as President of R&D; R. Keith Woods as<br>Chief Operating Officer; Vikas Sinha as Chief Financial Officer; and Rebecca McLeod as<br>Chief Brand Officer and U.S. General Manager, strengthening the Company’s leadership as<br>it transitions toward a global commercial-stage organization.<br>• Initiated U.S. and European build-out of commercial organization. Established a lean,<br>experienced U.S. customer-facing team of approximately 50 professionals with deep<br>expertise in neurology and rare disease. Initiated commercial build-out in Europe to<br>support the planned European launch, starting with Germany.<br>• Advanced industry-leading anti-myostatin pipeline. Advanced Scholar Rock’s strategic<br>plan to build a robust pipeline of therapies for the treatment of people living with rare,<br>severe, and debilitating neuromuscular diseases. |
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| Exhibit 99.1<br>o Initiated dosing in the Phase 2 OPAL study evaluating apitegromab in infants and<br>toddlers under 2 years of age with SMA<br>o Advanced preclinical development of apitegromab for FSHD and filed an IND<br>application to support the initiation of a Phase 2 study<br>o Completed a subcutaneous apitegromab Phase 1 study in healthy volunteers<br>o Initiated dosing in a Phase 1 study evaluating SRK-439 study in healthy volunteers<br>o Continued the ongoing ONYX open-label extension study evaluating the long-term<br>safety and efficacy of apitegromab in patients with SMA who participated in the<br>TOPAZ and SAPPHIRE clinical programs<br>o Completed the Phase 2 EMBRAZE study, demonstrating proof-of-concept in the<br>ability of apitegromab to drive statistically significant preservation of lean mass<br>during tirzepatide-induced weight loss<br>J.P. Morgan Healthcare Conference Presentation and Webcast<br>Scholar Rock management will discuss these updates in a corporate presentation at the 44th<br>Annual J.P. Morgan Healthcare Conference on Monday, January 12, 2026 at 7:30 a.m. PST (10:30<br>a.m. EST). A live webcast of the presentation can be accessed by visiting the Investors & Media<br>section of the Scholar Rock website at http://investors.scholarrock.com. An archived replay of<br>the webcast will be available on the Company’s website for approximately 90 days following the<br>presentation.<br>About Scholar Rock<br>Scholar Rock is a late-stage biopharmaceutical company focused on developing and<br>commercializing apitegromab for children and adults with spinal muscular atrophy (SMA) and other<br>rare, severe, and debilitating neuromuscular diseases. As a global leader in myostatin biology, a<br>field focused on proteins that regulate muscle mass, the biopharmaceutical company is named for<br>the visual resemblance of a scholar rock to protein structures. Our commitment to unlock<br>fundamentally different treatment approaches is powered by broad application of a proprietary<br>platform, which has developed novel monoclonal antibodies to modulate protein growth factors<br>with extraordinary selectivity. Scholar Rock works every day to create new possibilities for patients<br>through its highly innovative anti-myostatin program, including opportunities in additional rare<br>neuromuscular diseases. Learn more at ScholarRock.com and follow @ScholarRock on X and on<br>LinkedIn.<br>Scholar Rock® is a registered trademark of Scholar Rock, Inc.<br>Availability of Other Information About Scholar Rock<br>Investors and others should note that we communicate with our investors and the public using our<br>company website www.scholarrock.com, including, but not limited to, company disclosures,<br>investor presentations and FAQs, Securities and Exchange Commission filings, press releases,<br>public conference call transcripts and webcast transcripts, as well as on X (formerly known as |
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| Exhibit 99.1<br>Twitter) and LinkedIn. The information that we post on our website or on X (formerly known as<br>Twitter) or LinkedIn could be deemed to be material information. As a result, we encourage<br>investors, the media and others interested to review the information that we post there on a regular<br>basis. The contents of our website or social media shall not be deemed incorporated by reference<br>in any filing under the Securities Act of 1933, as amended.<br>Forward-Looking Statements<br>This press release contains "forward-looking statements" within the meaning of the Private<br>Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar<br>Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s<br>expectations regarding its growth, strategy, progress and timing of its clinical trials for<br>apitegromab, SRK-439 and its preclinical programs, and indication selection and development<br>timing, including the timing of any regulatory submissions and anticipated approvals, the<br>therapeutic potential, clinical benefits and safety of any product candidates, its ability to address<br>the observations identified in the complete response letter, its cash runway into 2027,<br>expectations regarding commercial launch timing in the US and in Europe, expectations regarding<br>a new fill finish facility and the achievement of important milestones, the ability of any product<br>candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or<br>clinical trial data, and the potential of its product candidates and proprietary platform. The use of<br>words such as “may,” “might,” “could,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,”<br>“estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions<br>are intended to identify such forward-looking statements. All such forward-looking statements are<br>based on management's current expectations of future events and are subject to a number of risks<br>and uncertainties that could cause actual results to differ materially and adversely from those set<br>forth in or implied by such forward-looking statements. These risks and uncertainties include,<br>without limitation, whether preclinical and clinical data, including the results from the Phase 3<br>SAPPHIRE trial, will be sufficient to support regulatory approval, that preclinical and clinical data,<br>including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, or the preclinical<br>data for SRK-439, are not predictive of, may be inconsistent with, or more favorable than, data<br>generated from future or ongoing clinical trials of the same product candidates; whether the FDA<br>will accept the remediations to the Novo Nordisk Bloomington Indiana fill finish facility in response<br>to the FDA Observations, whether Scholar Rock will be able to resubmit its BLA in a timely manner<br>and whether the updated BLA will be sufficient to support regulatory approval, Scholar Rock’s<br>ability to manage expenses or provide the financial support, resources and expertise necessary to<br>identify and develop product candidates on the expected timeline; information provided or<br>decisions made by regulatory authorities; competition from third parties that are developing<br>products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual<br>property; and Scholar Rock’s dependence on third parties for development and manufacture of<br>product candidates including, without limitation, to supply any clinical trials as well as those risks<br>more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on<br>Form 10-Q for the quarter ended September 30, 2025, as well as discussions of potential risks,<br>uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities<br>and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only |
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| Exhibit 99.1<br>as of today and should not be relied upon as representing its views as of any subsequent date. All<br>information in this press release is as of the date of the release, and Scholar Rock undertakes no<br>duty to update this information unless required by law.<br>Scholar Rock Contacts<br>Investors<br>Laura Ekas, Ph.D.<br>ir@scholarrock.com<br>917-439-0374<br>Media<br>Molly MacLeod, Ph.D.<br>media@scholarrock.com<br>802-579-5995 |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>44th Annual J.P. Morgan<br>Healthcare Conference<br>Scholar Rock Investor Presentation<br>JANUARY 12, 2026<br>Exhibit 99.2 |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>2<br>Forward-Looking Statements<br>Various statements in this presentation concerning the future expectations, plans and prospects of Scholar Rock Holding Corporation and Scholar Rock, Inc. (collectively,<br>“Scholar Rock”), including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of its clinical trials for apitegromab, SRK-439<br>and its preclinical programs, and indication selection and development timing, including the timing of any regulatory submissions and anticipated approvals, the therapeutic<br>potential, clinical benefits and safety of any product candidates, its ability to address the observations identified in the complete response letter, its cash runway into 2027,<br>expectations regarding commercial launch timing in the US and in Europe, expectations regarding a new fill finish facility and the achievement of important milestones, the<br>ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of its product<br>candidates and proprietary platform. The use of words such as “may,” “could,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,”<br>“project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements for the purposes of the<br>safe harbor provisions under The Private Securities Litigation Reform Act of 1995. All such forward-looking statements are based on management's current expectations of<br>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<br>such forward-looking statements. These risks and uncertainties include, without limitation, that preclinical and clinical data are not predictive of, may be inconsistent with,<br>or more favorable than, data generated from future or ongoing clinical trials of the same product candidate; the results from the Phase 3 SAPPHIRE trial will be sufficient to<br>support regulatory approval; Scholar Rock’s ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the<br>expected timeline; the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials, including from the EMBRAZE clinical trial; information<br>provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain<br>and protect its intellectual property; the success of Scholar Rock’s current and potential future collaborations; Scholar Rock’s dependence on third parties for development<br>and manufacture of product candidates including, without limitation, to supply any clinical trials; Scholar Rock’s ability to manage expenses and to obtain additional funding<br>when needed to support its business activities; its ability to establish or maintain strategic business alliances; its ability to receive priority or expedited regulatory review or<br>to obtain regulatory approval of apitegromab; its ability to expand globally and the anticipated commercial launch in the United States of apitegromab in 2026; as well as<br>those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Form 10-K for the year ended December 31, 2024, and Quarterly Report on Form 10-Q<br>for the quarter ended September 30, 2025 as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the<br>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<br>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<br>required by law.<br>This presentation may also contain estimates and other statistical data made by independent parties and by us relating to market size and growth and other data about our<br>industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. In addition, projections,<br>assumptions, and estimates of our future performance and the future performance of the markets in which we compete are necessarily subject to a high degree of<br>uncertainty and risk.<br>Apitegromab and SRK-439 are investigational drug candidates under evaluation. Apitegromab and SRK-439 have not been approved for any use by the FDA or any other<br>regulatory agency and the safety and efficacy of apitegromab and SRK-439 have not been established. |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>SHAPING THE FUTURE OF TREATMENT FOR<br>PATIENTS LIVING WITH RARE NEUROMUSCULAR DISEASES (NMDs)<br>INITIATE PHASE 2<br>STUDY for apitegromab<br>in facioscapulohumeral<br>muscular dystrophy<br>SRK-439 DOSING<br>COMMENCED in<br>Phase 1 healthy<br>volunteer study<br>ONGOING MID - 2026<br>APITEGROMAB FOR PATIENTS WITH SMA<br>Myostatin inhibitor with a successful Phase 3 study<br>Only muscle-targeted treatment to demonstrate<br>clinically meaningful benefit in SMA<br>ON TRACK<br>BLA resubmission and U.S. launch anticipated1<br>EMA decision expected in mid-2026<br>~35,000 have received an SMN-targeted therapy<br>$2B opportunity to serve patients with SMA alone<br>$365M<br>in cash and cash<br>equivalents as of<br>Dec 31, 20252<br>CASH RUNWAY INTO 2027<br>APPROXIMATELY<br>3<br>Scholar Rock is in a position of strength entering 2026<br>1. Subject to U.S. FDA approval. 2. Financial information as of December 31, 2025 has not been audited and has been prepared by, and is the responsibility of management. This information<br>could change as a result of further review. SMA, Spinal Muscular Atrophy; BLA, Biologics License Application; EMA, European Medicines Agency; SMN, survival motor neuron. |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>INITIAL PHASE OF INNOVATION: MOTOR NEURON NEXT PHASE OF INNOVATION: MUSCLE<br>4<br>Apitegromab poised to usher in next phase of innovation in SMA<br>Targeting muscle ‒ the principal organ affected in SMA<br>NOTE: Apitegromab and SRK-439 launch expectations following regulatory approval(s). SMN, survival motor neuron.<br>Motor unit consists of<br>motor neuron and muscle<br>2016 2019 2020 2025 2026<br>MUSCLE<br>MOTOR<br>NEURON<br>SMN-Targeted Therapies<br>Apitegromab U.S. launch<br>SRK-439 global launch<br>2030+<br>Advancing Muscle-Targeted<br>Treatments<br>Addressing progressive muscle<br>atrophy with potential to improve<br>motor function<br>Subcutaneous apitegromab global launch<br>Apitegromab European launch<br>Apitegromab global expansion |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>~35,000<br>SMA patients have received an<br>approved SMN-targeted therapy1-3<br>Powering Scholar Rock through the end of this decade and into next<br>High SMA diagnosis rates and accelerated time to<br>treatment<br>5<br>Global apitegromab opportunity in SMA alone offers potential<br>for many years of sustainable growth<br>1. Biogen Q4 2023 Report; 2. Roche Q3 2024 report; 3. Novartis Q4 2024 Report. SMN, survival motor neuron.<br>1<br>Increasing number of patients receiving SMN-targeted therapies for >10 years 2<br>Apitegromab has potential to be world’s first and only<br>muscle-targeted treatment for patients with SMA 3 |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>6<br>Poised for 2026 U.S. launch of apitegromab, followed by<br>EMA approval and launch in Europe, beginning with Germany<br>NOTE: Following regulatory approval(s).<br>Building a 50-country platform to serve patients<br>with rare, severe neuromuscular diseases<br>United States<br>Europe<br>Expansion to<br>Asia Pacific,<br>LATAM Countries |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>7<br>Shaping the future of treatment for patients living with rare<br>neuromuscular disease<br>IV, Intravenously; SC, Subcutaneously.<br>THERAPEUTIC TARGET INDICATION PRECLINICAL PHASE 1 PHASE 2 PHASE 3 COMMERCIAL<br>APITEGROMAB<br>Selective anti-latent<br>myostatin antibody<br>IV administered<br>SPINAL MUSCULAR ATROPHY<br>≥2 YEARS<br>SPINAL MUSCULAR ATROPHY<br><2 YEARS<br>FACIOSCAPULOHUMERAL<br>MUSCULAR DYSTROPHY<br>NEUROMUSCULAR DISEASE<br>INDICATION #3<br>NEUROMUSCULAR DISEASE<br>INDICATION #4<br>NEUROMUSCULAR DISEASE<br>INDICATION #5<br>SC administered<br>SPINAL MUSCULAR ATROPHY,<br>ADDITIONAL NEUROMUSCULAR<br>DISEASES<br>SRK-439 Novel anti-latent myostatin<br>antibody SC administered<br>RARE NEUROMUSCULAR<br>DISEASES<br>UNDISCLOSED Novel target RARE NEUROMUSCULAR<br>DISEASES |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>8<br>Scholar Rock 2026 priorities<br>1. Subject to regulatory approval(s). NMDs, neuromuscular diseases.<br>Focused execution and financial discipline<br>1 COMMERCIALIZE<br>Commercialize apitegromab for<br>treatment of patients with SMA1<br>BLA resubmission and U.S.<br>launch, following FDA approval, for<br>children and adults with SMA<br>Advance launch readiness in U.S.<br>and Europe<br>2 EXPAND<br>Develop apitegromab for patients with<br>SMA <2 years and for additional rare,<br>severe NMDs<br>Initiate apitegromab Phase 2<br>FORGE study in patients with FSHD<br>in mid-2026<br>Progress Phase 2 OPAL study for<br>patients with SMA <2 years of age<br>EMA decision expected mid-2026,<br>initial launch planned in Germany<br>3 ADVANCE<br>Advance world-leading anti-myostatin<br>pipeline<br>Progress subcutaneous apitegromab;<br>Phase 1 study complete<br>Advance Phase 1 study for SRK-439,<br>a novel, subcutaneously<br>administered myostatin inhibitor |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>Commercialize Apitegromab<br>for Treatment of Patients<br>with SMA<br>Transforming the treatment of SMA<br>with muscle-targeted therapy<br>9 |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>...but do not target muscle<br>SMN-Targeted Therapies<br>slow further degeneration<br>of motor neurons1<br>Apitegromab has the potential to directly<br>target progressive muscle atrophy and<br>weakness<br>Muscle-Targeted Treatment<br>Spinal Muscular<br>Atrophy<br>Muscle atrophy and<br>weakness can<br>lead to deterioration in<br>mobility, swallowing,<br>and breathing, and can<br>cause debilitating<br>fatigue<br>1 0<br>SMA causes motor neuron loss leading to muscle atrophy and<br>progressive weakness<br>SMA, Spinal muscular atrophy; SMN, Survival motor neuron.<br>1. Hua Y, et al. Nature; 2011;478(7367):123-6; 2. Figure adapted from: SMA Foundation Overview. http://www.smafoundation.org/wp-content/uploads/2012/03/SMA-Overview.pdf;<br>Accessed April 18, 2021.<br>Despite significant advancements, progressive muscle weakness remains #1 unmet need in SMA<br>HALLMARKS OF SMA<br>MUSCLE<br>MOTOR<br>NEURON<br>APITEGROMAB |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>~35,000<br>patients have received an<br>approved SMN-targeted<br>therapy1-3<br>~7,000<br>patients have received an<br>approved SMN-targeted<br>therapy4<br>GLOBAL SMA OPPORTUNITY 90%<br>of patients with SMA<br>rate muscle strength and motor<br>function as top unmet needs5<br>1 1<br>Addressing progressive muscle weakness: apitegromab positioned<br>to be future standard-of-care with ongoing SMN-targeted therapy<br>1. Biogen Q4 2023 Report; 2. Roche Q3 2024 report; 3. Novartis Q4 2024 Report; 4. Cure SMA State of SMA 2023 Report; 5. Cure SMA State of SMA 2024 Report. https://www.curesma.org/wp-content/uploads/2025/04/State-of-SMA-Report2024_vWeb.pdf; 6. Scholar Rock internal market research (US Neurologists), 2024. Approaches to Drug Development. SMN, survival motor neuron.<br>WORLDWIDE IN THE U.S.<br>74%<br>of neurologists<br>agree that multiple modalities<br>are necessary to treat SMA6 |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>1 2<br>Apitegromab successful in Phase 3 SMA study<br>First and only myostatin inhibitor with a positive, statistically significant Phase 3 outcome<br>Sources: Long KK, et al. Hum Mol Genet. 2019;28:1076-1089; Pirruccello-Straub M, et al. Sci Rep. 2018;8:2292. HFMSE, Hammersmith Functional Motor Scale ‒ Expanded<br>P-value reflects data from Primary Endpoint for SMA treatment population ages 2‒12. SOC, standard-of-care;<br>GAIN of motor function<br>apitegromab + SOC<br>-2<br>-1<br>0<br>1<br>2<br>Placebo + SOC (N=50) Apitegromab combined dose + SOC (N=106)<br>0 8 16 24 32 40<br>Time (weeks)<br>52<br>LS Mean Change from Baseline<br>in HFMSE<br>LOSS of motor function<br>Placebo + SOC<br>HFMSE Total Score by Visit (MITT Set) in Patients on SMN-Targeted Therapy<br>p=0.0192<br>Phase 3 randomized, placebo-controlled study in SMA patients (N=188) on standard-of-care (nusinersen or risdiplam)<br>GAIN of motor function<br>apitegromab + SOC |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>Apitegromab has potential to be the FIRST and ONLY muscle-targeted<br>treatment to improve motor function in SMA<br>30.0% vs 12.5%<br>30.0% of apitegromab patients<br>ACHIEVED ≥3PT<br>IMPROVEMENT IN HFMSE1<br>compared to 12.5% on SMN2-<br>targeted treatment alone<br>+1.8 HFMSE points2 (p=0.0192)<br>vs. SMN2-targeted treatment alone<br>Consistent clinically meaningful benefit<br>across all age groups (2-21 yrs)<br>Encouraging safety profile<br>consistent with >48 months experience in<br>Phase 2 TOPAZ trial<br>1 3<br>Statistically significant, clinically meaningful benefits underscore<br>apitegromab’s potential to impact broad SMA patient population<br>1. 12.5% of patients on placebo + SOC achieved a ≥3-point improvement in HFMSE; SOC=Standard of care (i.e., nusinersen or risdiplam); HFMSE, Hammersmith Functional Motor Scale–<br>Expanded; 2. Based on apitegromab combined dose (10 mg/kg and 20 mg/kg; n=106) + SOC versus placebo + SOC (n=50) (Hochberg multiplicity adjustment). |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>1 4<br>Apitegromab BLA resubmission and U.S. launch, following<br>approval, anticipated in 2026<br>BLA, Biologics License Application; OAI, Official Action Indicated<br>Tech transfer ongoing at second fill-finish facility; commercial capacity reserved beginning in Q1 2026<br>Submitted BLA to FDA<br>January 2025<br>Catalent Indiana<br>receives OAI<br>October 2025<br>2025 2026<br>BLA accepted under<br>priority review<br>March 2025<br>Catalent Indiana, LLC (part of Novo<br>Nordisk) receives 483s from FDA<br>July 2025<br>Received CRL<br>September 22, 2025<br>Catalent Indiana Warning Letter<br>November 2025<br>FDA meeting scheduled with<br>Catalent Indiana<br>in Early 2026<br>Constructive, collaborative<br>in-person Type A FDA meeting<br>November 12, 2025<br>Catalent Indiana response to<br>Warning Letter<br>December 2025<br>BLA reviewed, late-stage label discussions<br>March ‒ September 2025<br>Catalent Indiana reinspection<br>and apitegromab BLA<br>resubmission |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>>2,600<br>SMA Prescribers<br>Prescribers and SMA<br>Treatment Centers<br>1 5<br>U.S. commercial field team focused on engagement,<br>disease education across SMA stakeholder landscape<br>Broaden and deepen<br>engagement<br>Patient Advocacy National & Regional<br>Payers<br>Educating on unmet need<br>and potential benefit of<br>apitegromab<br>Market access team<br>engaging with national and<br>regional payers<br>Building lasting relationships<br>one patient, one caregiver,<br>one family at a time<br>Strong collaboration with<br>advocacy groups<br>140<br>SMA Centers<br>Understand patient journey<br>and roles of treatment team |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>1 6<br>Strong momentum with apitegromab launch readiness in Europe<br>in advance of mid-2026 EMA decision<br>EMA, European Medicines Agency; KOL, Key Opinion Leader.<br>Large opportunity to serve SMA patients in Europe<br>Key leadership hires<br>made and additional<br>offers extended<br>Distribution model in<br>place<br>Expanded physical<br>presence in Zug and<br>Dublin<br>Building World-Class Team<br>Engaging SMA<br>Community<br>Expanded engagement<br>with KOLs and SMA<br>Centers of Excellence<br>Deepening relationships<br>with multiple country<br>patient advocacy groups<br>Global Medical Affairs<br>team focused on SMA<br>disease education<br>Establishing<br>Access<br>Multiple reimbursement<br>dossiers on track for<br>submission<br>Compassionate use<br>program enrolling in<br>Germany<br>Finalizing plans with<br>international partner<br>markets |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>Apitegromab in SMA represents a large global opportunity to<br>serve patients<br>1. Revenue as of Biogen 3Q25 financial update, Roche 3Q25 financial update, and Novartis 3Q25 financial update; 2. Scholar Rock internal estimates as of December 2025. SMN, survival motor<br>neuron; SMA, spinal muscular atrophy.<br>SMA community demanding the first and only muscle-targeted therapy<br>~$5B1<br>muscle-targeted treatment to<br>show clinical benefit in SMA<br>Apitegromab global<br>revenue potential<br>$2B+2<br>Estimated 2025 global revenue<br>for SMN-targeted therapies<br>1st & Only<br>1 7 |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>Develop Apitegromab for<br>Patients with SMA Under 2 and<br>for Additional Neuromuscular<br>Diseases (NMDs)<br>Building a pipeline<br>-in<br>-<br>a<br>-product to<br>reach more patients with SMA and<br>with additional rare, severe NMDs<br>1 8 |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>Evaluating PK, PD, efficacy,<br>safety, and tolerability of<br>apitegromab over 48 weeks<br>1 9<br>Furthering our commitment to broad SMA community<br>Ongoing Phase 2 OPAL study evaluating apitegromab in infants and toddlers with SMA<br>PK, Pharmacokinetic, PD, Pharmacodynamic.<br>Patient enrollment and dosing underway in Phase 2 OPAL study<br>including evaluation of apitegromab in patients<br>who received SMN1-targeted gene therapy<br>Expanding our potential impact<br>to reach patients earlier<br>Addressing the needs of children<br>under 2 years of age with SMA<br>seeking to address the motor neuron and muscle<br>in youngest patients<br>Time is muscle |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>2 0<br>FSHD: Rare, devastating NMD with significant unmet need<br>>30,000 patients diagnosed in U.S. and Europe; no approved therapies<br>1. Tawil R, et al. Skelet Muscle. 2014;4:12; 2. Hubregtse L, et al. Neuromuscul Disord. 2024;36:6-15; 3. Tihaya MS, et al. Nat Rev Neurol. 2023;19(2):91-108; 4. FSHD Society. Accessed Oct 7,<br>2025. https://www.fshdsociety.org/wp-content/uploads/2020/11/Voice-of-the-Patient-Report-FINAL.pdf; 5. Bankole, LC, et al. Medicine. 2016; 95(31); 6. Andersen, G, et al. Neurology.<br>2015; 85:396-403; 7. Heatwole, CR, et al. Neurol Genet. 2025;11:e200292. 6MWD, 6-minute walk distance; FSHD, facioscapulohumeral muscular dystrophy.<br>FSHD results in progressive muscle atrophy<br>leading to cumulative loss of function and<br>loss of independence<br>Symptoms often begin age 15 ‒ 303<br>Caused by myotoxic effects from abnormal<br>expression of DUX41,2<br>Standard-of-care (e.g., physical therapy) only<br>addresses symptoms, not underlying disease<br>>80% of patients report moderate/severe<br>impact on activities involving arms, core,<br>and/or legs; ~20% will become wheelchair<br>dependent3,4<br>Support for apitegromab therapeutic hypothesis in FSHD<br>Randomized studies of exercise programs suggest muscle<br>has capacity to show functional benefit5,6<br>Study of anabolic agents suggests increase in lean mass<br>and muscle function7<br>Increase in Lean Body Mass Improvement in 6MWD |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>2 1<br>Preclinical studies provide mechanistic rationale for apitegromab in FSHD<br>Increased muscle mass, strength, and endurance in gold standard FLExDUX4 mouse model of FSHD<br>*Muscle mass=weight of tibialis anterior muscle; muSRK-015P is a murine version of apitegromab.<br>FLExD, FLExDUX4.Cre; FSHD, facioscapulohumeral muscular dystrophy; WT, wild type; Ab, antibody. Fogel A. Presented at: FSHD Society International Research Congress; Jun 12-13, 2025;<br>Amsterdam, Netherlands. Oral presentation.<br>FSHD FLExDUX4 Mouse Model<br>Robust Increase in Muscle<br>Mass (28 Days)<br>WT<br>Control Ab<br>muSRK-015P<br>0<br>20<br>40<br>60<br>TA Weight (mg)<br>11%<br>Significant Improvements in<br>Muscle Force (28 Days)<br>0 25 50 75 100 125 150<br>0<br>10<br>20<br>30<br>40<br>Stimulation Frequency (Hz)<br>Total Force (mN)<br>Control Ab<br>muSRK-015P<br>* * **<br>WT<br>Consistent Gains in Endurance<br>(28 Days)<br>WT<br>Control Ab<br>muSRK-015P<br>0<br>200<br>400<br>600<br>Treadmill Distance (meters)<br>✱<br>*Data from 8-month-old mice; similar results observed in<br>younger mice.<br>37% |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>KEY ELIGIBILITY CRITERIA<br>• Aged 18-60 years<br>• Genetically confirmed FSHD1 or<br>FSHD2<br>• Clinical severity score of 1.5 to 3.0<br>• Baseline 10 MWRT time ≤5 seconds<br>Apitegromab (10mg/kg IV Q4W)<br>Placebo (IV Q4W)<br>PRIMARY ENDPOINT:<br>Mean lean muscle volume (LMV) change<br>from baseline at 12 months<br>SECONDARY & OTHER ENDPOINTS:<br>•Mean LMV change from baseline<br>at 6 months<br>•Mean change from baseline in additional<br>muscle parameters (6 and 12 months)<br>•Quantitative myometry testing (QMT)<br>• Safety, PK/PD, ADA<br>R<br>1<br>1<br>2 2<br>Phase 2 trial evaluating apitegromab in patients with FSHD<br>10 MWRT, 10-Meter Walk/Run Test; ADA, antidrug antibody; FSHD, facioscapulohumeral muscular dystrophy; IV, intravenous; LMV, lean muscle volume; N, total number of participants;<br>PD, pharmacodynamics; PK pharmacokinetics; Q4W, every 4 weeks; QMT, quantitative muscle testing; R, randomization.<br>SCREENING TREATMENT<br>IND application cleared; on track to initiate dosing in Phase 2 FORGE study in mid-2026<br>4 Weeks 52 Weeks<br>Randomized, double-blind, placebo-controlled, multicenter study (N≈60) |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>2 3<br>Unlocking pipeline-in-a-product with apitegromab in additional rare<br>NMDs, beginning with FSHD<br>Broad landscape of potential indications supports significant opportunity for muscle-targeted therapies<br>Scholar Rock deploying innovative, world-leading anti-myostatin pipeline to<br>potentially address a range of rare, severe neuromuscular diseases<br>Spinal muscular atrophy<br>(SMA)<br>Amyotrophic Lateral<br>Sclerosis (ALS)<br>Peripheral neuropathies<br>GENETIC MYOPATHIES<br>Muscular Dystrophies:<br>- Facioscapulohumeral dystrophy (FSHD)<br>- Duchenne muscular dystrophy (DMD)<br>- limb girdle muscular dystrophy (LGMD)<br>Congenital Myopathies, e.g.,<br>Myotubular<br>Metabolic Myopathies, e.g.,<br>Pompe disease<br>ACQUIRED MYOPATHIES<br>Inflammatory Myopathies:<br>- Inclusion myositis<br>Toxic Myopathies:<br>- Drug-induced myopathy<br>Endocrine Myopathies,<br>e.g., Cushing’s disease<br>Amyotrophic Lateral<br>Sclerosis (ALS)<br>Myasthenia gravis (MG)<br>Guillain-Barré syndrome<br>Inflammatory neuropathies<br>ACQUIRED NEUROPATHIC<br>DISEASES<br>© 2026 Scholar Rock, Inc. All rights reserved.<br>2 3<br>GENETIC NEUROPATHIC<br>DISEASES<br>NMDs, neuromuscular diseases. |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>2 4<br>NEXT PHASE OF INNOVATION: MUSCLE<br>2026<br>Apitegromab U.S. launch<br>SRK-439 global launch<br>2030+<br>SC Apitegromab global launch<br>Apitegromab European launch<br>Apitegromab global expansion<br>Advance World-Leading<br>Anti-Myostatin Pipeline<br>Driving continued innovation<br>for treatment of patients<br>living with rare, severe NMDs<br>Note: Apitegromab and SRK-439 launch expectations following regulatory approval(s). SC, subcutaneous. |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>2 5<br>Subcutaneous apitegromab demonstrated favorable bioavailability,<br>with pharmacodynamic profile comparable to IV administration<br>Source: Scholar Rock internal data. PD, pharmacodynamic.<br>Phase 1 study comparing<br>intravenous (IV) and<br>subcutaneous (SC)<br>apitegromab in healthy<br>volunteers<br>Further development<br>activities ongoing, including<br>planned FDA and EMA<br>regulatory engagements<br>At 800mg, SC and IV<br>apitegromab produced<br>overlapping PD responses<br>(total latent myostatin)<br>Phase 1 study<br>0<br>200<br>400<br>600<br>800<br>1000<br>1200<br>1 3 5 7 9 1113151719212325272931333537394143454749515355575961636567697173757779818385878991939597991010310510710911113<br>Apitegromab 800 mg IV (N=15)<br>Apitegromab 800 mg SC (N=15)<br>Apitegromab 100 mg SC (N=15)<br>Serum Total Latent Myostatin<br>Concentration (ug/L)<br>1 8 15 22 29 43 57 85 113<br>Time in Days<br>Overlapping Pharmacodynamic Profiles for IV and SC Apitegromab (800mg) |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>2 6<br>Advancing Scholar Rock’s innovative anti-myostatin platform<br>with SRK-439<br>SRK-439 is a novel, investigational myostatin inhibitor. muSRK-015P is a murine version of apitegromab.<br>Leveraging world-leading expertise to drive continued innovation<br>0.0001 0.01 1 100 10000<br>0.0<br>0.5<br>1.0<br>1.5<br>2.0<br>2.5<br>Antibody (huIgG4) Concentration (nM)<br>OD 450nM<br>SRK-439<br>SRK-015P<br>Isotype Control<br>Inhibition of Tolloid Cleavage of<br>Latent Myostatin by SRK-015P and SRK-439<br>Change in Whole Body Lean Mass<br>from Baseline (NHP)<br>OD 450nM<br>Antibody (hulgG4) Concentration (nM)<br>Latent Myostatin Potency Assay<br>Comparing SRK-015P and SRK-439<br>* ** *<br>Vehicle<br>Control<br>0.3 3.0 10.0<br>SRK-439 (mg/kg)<br>-5<br>0<br>5<br>10<br>15<br>20<br>25<br>% Change from Baseline % Change from Baseline<br>SRK-439<br>SRK-015P<br>Control Ab<br>Novel, highly potent<br>myostatin inhibitor<br>Optimized for subcutaneous<br>administration<br>Preclinical data demonstrated<br>favorable muscle mass preservation<br>Strong scientific validation<br>Increasing potency |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>N≈76 Healthy Adult Participants<br>PART A<br>PART B<br>2 7<br>SRK-439 Phase 1 study assessing<br>safety, tolerability, and PK/PD profile SRK-439 program key milestones<br>2 7<br>IND cleared in November 2025<br>Dosing commenced in Phase 1 study<br>in December 2025<br>On track to report topline Phase 1<br>data in H2 2026<br>SINGLE ASCENDING DOSE<br>MULTIPLE ASCENDING DOSE<br>PK/PD, pharmacokinetic/pharmacodynamic. |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>Financials &<br>Upcoming Milestones<br>2 8 |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>2 9<br>Fortified balance sheet in 2025, strong cash position entering 2026<br>Operating with financial discipline to achieve Scholar Rock’s ambitions<br>Financial information as of December 31, 2025 has not been audited and has been prepared by, and is the responsibility of management. This information could change as a result of further<br>review.<br>Thoughtful<br>capital allocation<br>to advance<br>clinical pipeline<br>Strategic<br>investments<br>to support<br>commercial<br>readiness<br>Cash runway<br>to support<br>operations into<br>2027<br>as of December 31, 2025<br>$365M<br>in cash and equivalents<br>APPROXIMATELY |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>3 0<br>Scholar Rock 2026 Priorities<br>Poised to be next global biotech powerhouse<br>1. Upon U.S. FDA approval. NMDs, neuromuscular diseases.<br>Driving value through focused execution and financial discipline<br>1 COMMERCIALIZE<br>Commercialize apitegromab for<br>treatment of patients with SMA1<br>BLA resubmission and U.S. launch<br>following FDA approval for<br>children and adults with SMA<br>Advance launch readiness in U.S.<br>and Europe<br>2 EXPAND<br>Develop apitegromab for patients with<br>SMA <2 years and for additional rare,<br>severe NMDs<br>Initiate apitegromab Phase 2<br>FORGE study in patients with FSHD<br>in mid-2026<br>Progress Phase 2 OPAL study for<br>patients with SMA <2 years of age<br>EMA decision mid-2026, initial<br>launch planned in Germany<br>3 ADVANCE<br>Advance world-leading anti-myostatin<br>pipeline<br>Progress subcutaneous apitegromab;<br>Phase 1 study complete<br>Advance Phase 1 study for SRK-439,<br>with topline data expected in<br>H2 2026 |
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| © 2026 Scholar Rock, Inc. All rights reserved.<br>For more information, please contact:<br>ir@scholarrock.com<br>media@scholarrock.com<br>Or visit us at www.scholarrock.com<br>3 1 |
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