Scholar Rock updates March 31 2026 quarter results
Scholar Rock Holding Corporation furnished an update on its financial and operating results for the quarter ended March 31, 2026.
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Rhea-AI Filing Summary
Scholar Rock Holding Corporation furnished an update on its financial and operating results for the quarter ended March 31, 2026. The company issued a press release, provided as Exhibit 99.1, and prepared a corporate presentation, furnished as Exhibit 99.2, in connection with these results.
The slides are being used during a conference call scheduled for 8:00 am ET on May 7, 2026 to discuss the quarter and provide a business update. The press release and slides are designated as furnished, not filed, under the Exchange Act, limiting their use for certain legal purposes.
8-K Event Classification
Key Figures
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Regulation FD Disclosure regulatory
Emerging growth company regulatory
Exchange Act regulatory
Inline XBRL document technical
Press Release financial
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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
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:
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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 (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.
On May 7, 2026, Scholar Rock Holding Corporation (the “Company”) issued a press release announcing its financial and operating results for the quarter ended March 31, 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 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 May 7, 2026 to announce its financial and operating results for the fiscal quarter ended March 31, 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 in this Current Report on Form 8-K and Exhibits 99.1 and 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
Exhibit | | Description | ||
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99.1 |
| Press Release issued by the Company on May 7, 2026, furnished hereto. | ||
99.2 | Scholar Rock Holding Corporation Corporate Presentation dated May 7, 2026, furnished hereto. | |||
104 |
| Cover Page Interactive Data File (embedded within the Inline XBRL document). | ||
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: May 7, 2026 | By: | /s/ Junlin Ho |
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| Junlin Ho |
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| General Counsel & Corporate Secretary |
Exhibit 99.1
| Exhibit 99.1 Scholar Rock Reports First Quarter 2026 Financial Results and Recent Business Highlights • FDA accepted apitegromab Biologics License Application (BLA) for treatment of children and adults with spinal muscular atrophy (SMA) with September 30, 2026 Prescription Drug User Fee Act (PDUFA) action date • Accepted apitegromab BLA includes two fill-finish facilities, Catalent Indiana LLC (part of Novo Nordisk), and a second U.S.-based facility • FDA has completed reinspection of Catalent Indiana; classification of facility expected within 90 days following reinspection, in accordance with FDA guidelines • Second fill-finish facility on track to have commercial apitegromab supply in early Q3 2026 • Scholar Rock is prepared for U.S. apitegromab launch immediately upon FDA approval, which may be granted at any time through September 30, 2026 • Cash, cash equivalents, and marketable securities of $480 million as of March 31, 2026; includes an additional $100 million in debt and $98 million in net cash proceeds from the Company’s at-the-market (ATM) program • Management to host a conference call today at 8:00 a.m. ET CAMBRIDGE, Mass.--(BUSINESS WIRE)— May 7, 2026 — Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology to advance musculoskeletal health, today reported financial results for the first quarter ended March 31, 2026, and provided an update on recent company developments. “With the FDA’s acceptance of our apitegromab BLA, we have achieved another critical milestone as we work with urgency to deliver on our mission to bring the world’s first muscle-targeted treatment to the SMA community,” said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. “We are grateful for the FDA’s continued high level of engagement, and we are pleased that important progress continues to be made at both of our fill-finish facilities. Our U.S. commercial team stands ready to launch apitegromab on or at any time prior to the September 30th PDUFA date.” Mr. Hallal continued, “Our balance sheet is strong, our clinical-stage pipeline continues to advance, and we are poised, now more than ever, to usher in the next phase of innovation for patients with SMA.” Business Highlights and Upcoming Milestones Apitegromab Apitegromab is an investigational fully human monoclonal antibody designed to inhibit myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. It is the first and only muscle-targeted therapeutic candidate in SMA to demonstrate a statistically significant and clinically meaningful benefit in a pivotal Phase 3 clinical trial (SAPPHIRE). SMA Program • Apitegromab BLA accepted by FDA with September 30, 2026 PDUFA action date. The accepted BLA includes two fill-finish facilities, Catalent Indiana and a second U.S.-based fill-finish facility. |
| • FDA completed reinspection of Catalent Indiana. Following the FDA’s acceptance of the apitegromab BLA, the Agency completed reinspection of Catalent Indiana. In accordance with FDA guidelines, classification of the facility is anticipated within 90 days following reinspection. • Significant progress continues at second fill-finish facility. Apitegromab commercial supply from this facility is expected to be available early in the third quarter of 2026. • Preparations ongoing for U.S. commercial launch. The Commercial team continues to expand its reach and engagement with key stakeholders, including a significant presence at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, which was held March 8 – 11, 2026 in Orlando, FL and at the upcoming 2026 Annual SMA Conference, which is being held June 25 – 28, 2026 in Orlando, FL. The U.S. commercial team is prepared to launch apitegromab immediately upon FDA approval. • European Medicines Agency (EMA) regulatory review ongoing. A Committee for Medicinal Products for Human Use (CHMP) opinion for the apitegromab Marketing Authorisation Application (MAA) is anticipated near mid-2026. The Scholar Rock team in Europe continues to engage with key stakeholders on SMA disease awareness and education initiatives, including at the 5th International Scientific Congress on SMA, which was held March 11 – 14, 2026 in Budapest, Hungary. The Company is planning for an apitegromab launch in Europe in the second half of 2026, beginning with Germany. • Enrollment progressing in Phase 2 OPAL clinical trial. Patients continue to be enrolled and dosed in the Phase 2 OPAL study (NCT07047144). The trial is evaluating apitegromab in infants and toddlers with SMA under two years of age who have received an approved SMN1-targeted gene therapy or who are receiving ongoing treatment with an approved SMN2-targeted therapy. • Subcutaneous apitegromab development continues to progress. Scholar Rock is advancing a subcutaneous formulation of apitegromab intended to provide optionality for patients as a small volume, self- or caregiver-administered anti-myostatin antibody suitable for an autoinjector. A Phase 1 study in healthy volunteers has been completed. Further development activities are ongoing, including anticipated FDA and EMA regulatory engagements following apitegromab approvals. FSHD Program • Phase 2 FORGE trial on track for initiation in mid-2026. Scholar Rock is developing apitegromab for the treatment of people with facioscapulohumeral muscular dystrophy (FSHD). FSHD is a rare, progressive neuromuscular disease characterized by muscle atrophy and functional decline, affecting approximately 30,000 individuals across the U.S. and Europe. Initiation of a Phase 2 randomized, double-blind, placebo-controlled trial, called FORGE, is expected in mid-2026. SRK-439 |
| SRK-439 is a novel, investigational, subcutaneously administered myostatin inhibitor that binds to pro-and latent myostatin with high affinity and selectivity (i.e., no GDF11 or Activin A binding). Based on preclinical data, SRK-439 has the potential to potently inhibit myostatin and increase muscle mass. • Phase 1 healthy volunteer study ongoing. A Phase 1 study evaluating SRK-439 in healthy volunteers is underway, with topline data expected in the second half of 2026. First Quarter 2026 Financial Results Scholar Rock reported a net loss of $105.5 million, including stock-based compensation of $18.2 million, for the quarter ended March 31, 2026, compared to a net loss of $74.7 million, including stock-based compensation of $13.4 million, for the quarter ended March 31, 2025. Net loss per common share was $0.83 for the quarter ended March 31, 2026, compared to $0.67 per common share for the quarter ended March 31, 2025. • The Company did not record any revenue for the quarters ended March 31, 2026 and 2025. • Research and development expense was $51.8 million, including $6.5 million in stock-based compensation, for the quarter ended March 31, 2026, compared to $48.7 million, including $4.0 million in stock-based compensation, for the quarter ended March 31, 2025. • General and administrative expense was $50.2 million, including $11.7 million in stock-based compensation, for the quarter ended March 31, 2026, compared to $28.4 million, including $9.4 million in stock-based compensation, for the quarter ended March 31, 2025. • As of March 31, 2026, Scholar Rock had cash, cash equivalents, and marketable securities of $479.9 million. This reflects a drawdown of $100.0 million from the Company’s debt facility and net cash proceeds of $98.0 million from the Company’s at-the-market (ATM) program. Conference Call Information Scholar Rock will host a conference call and webcast today, Thursday, May 7, at 8:00 a.m. ET to review its first 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 a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for children and adults with spinal muscular atrophy (SMA) and other rare, severe and debilitating neuromuscular diseases. 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 at ScholarRock.com and follow @ScholarRock on X and on LinkedIn. Scholar Rock® is a registered 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 or on 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 its growth, strategy, progress and timing of its clinical trials and development programs for apitegromab, including its subcutaneous formulation, SRK-439 and its preclinical programs, and indication selection and development timing, including the timing of any regulatory submissions, decisions 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, expectations regarding actions by the FDA after its reinspection of the Catalent Indiana facility; the expected timing and outcome of FDA review of the accepted BLA for apitegromab, including the September 30, 2026 PDUFA action date; expectations regarding the availability and timing of commercial supply of apitegromab from Catalent Indiana and a second U.S.-based fill-finish facility, including expected supply from the second fill-finish facility; expectations regarding commercial launch timing, 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, 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 preclinical 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 preclinical 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 FDA will accept the remediations to the Catalent Indiana fill finish facility in response to the FDA Observations, whether the updated BLA will be sufficient to support regulatory approval, 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; 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 product candidates including, without limitation, to supply 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 March 31, 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. 2026 2025 Operating expenses Research and development $ 51,814 $ 48,678 General and administrative 50,202 28,412 Total operating expenses 102,016 77,090 Loss from operations (102,016) (77,090) Other income (expense), net (3,494) 2,367 Net loss $ (105,510) $ (74,723) $ (0.83) $ (0.67) 127,277,144 111,838,272 Net loss per share, basic and diluted Three Months Ended March 31, Scholar Rock Holding Corporation Condensed Consolidated Statements of Operations (unaudited) (in thousands, except share and per share data) Weighted average common shares outstanding, basic and diluted |
| Investor Contact Laura Ekas, Ph.D. ir@scholarrock.com 917-439-0374 Media Contact Molly MacLeod, Ph.D. media@scholarrock.com 802-579-5995 March 31, 2026 December 31, 2025 Cash, cash equivalents and marketable securities $ 479,944 $ 367,563 Other current assets 35,340 17,584 Total current assets 515,284 385,147 Other assets 19,970 19,125 $ 535,254 $ 404,272 Current liabilities $ 59,643 $ 55,419 Long-term liabilities 199,598 103,365 Total liabilities 259,241 158,784 Total stockholders' equity 276,013 245,488 $ 535,254 $ 404,272 Assets Scholar Rock Holding Corporation Condensed Consolidated Balance Sheets (unaudited) Total liabilities and stockholders' equity Total assets Liabilities and Stockholders' Equity (in thousands) |
Exhibit 99.2
| © 2026 Scholar Rock, Inc. All rights reserved. Q1 2026 Earnings Call MAY 7, 2026 Exhibit 99.2 |
| © 2026 Scholar Rock, Inc. All rights reserved. 2 TOPIC SPEAKER BUSINESS UPDATE David L. Hallal Chairman & Chief Executive Officer R&D PROGRESS Akshay Vaishnaw, M.D., Ph.D. President of R&D COMMERCIAL READINESS Keith Woods Chief Operating Officer COMPANY FINANCIALS Vikas Sinha Chief Financial Officer 5 Q&A SESSION 1 2 3 4 Q1 2026 EARNINGS CALL |
| © 2026 Scholar Rock, Inc. All rights reserved. 3 Forward-Looking Statements Various statements in this presentation concerning the future expectations, plans and prospects of Scholar Rock Holding Corporation and Scholar Rock, Inc. (collectively, “Scholar Rock”), including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of its clinical trials and development programs for apitegromab, including its subcutaneous formulation, SRK-439 and its preclinical programs, and indication selection and development timing, including the timing of any regulatory submissions, decisions 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, expectations regarding actions by the FDA after its reinspection of the Catalent Indiana facility; the expected timing and outcome of FDA review of the accepted BLA for apitegromab; expectations regarding the availability and timing of commercial supply of apitegromab from third-party U.S.-based fill-finish facility, including expected supply from the second fill-finish facility; expectations regarding commercial launch timing, 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. The use of words such as “may,” “could,” “might,” “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 for the purposes of the 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 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, that preclinical 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 FDA will accept the remediations to the Catalent Indiana fill finish facility in response to the FDA Observations, whether the updated BLA will be sufficient to support regulatory approval, 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; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain 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 and manufacture of product candidates including, without limitation, to supply any clinical trials; Scholar Rock’s ability to obtain additional funding when needed to support its business activities; its ability to receive priority or expedited regulatory review or 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 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 March 31, 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 presentation is as of the date of this presentation, and Scholar Rock undertakes no duty to update this information unless required by law. 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 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, 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 uncertainty and risk. 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 regulatory agency and the safety and efficacy of apitegromab and SRK-439 have not been established. |
| © 2026 Scholar Rock, Inc. All rights reserved. Business Update David L. Hallal Chairman and Chief Executive Officer 4 |
| © 2026 Scholar Rock, Inc. All rights reserved. 5 Scholar Rock is positioned for a pivotal year ahead Our top priority: Bringing apitegromab to children and adults living with SMA BLA, Biologics License Application; PDUFA, Prescription Drug User Fee Act. Included in BLA resubmission, providing two independent paths to FDA approval Two Fill-Finish Facilities Apitegromab BLA Accepted by FDA Steady and rapid progress continues in collaboration with the FDA U.S. Commercial team is ready for launch immediately upon approval, which may be granted at any time through September 30th PDUFA Action Date of September 30th |
| © 2026 Scholar Rock, Inc. All rights reserved. 6 Two independent paths to apitegromab FDA approval by September 30th PDUFA date BLA, Biologics License Application; PDUFA, Prescription Drug User Fee Act. U.S. Commercial team is ready to launch apitegromab immediately upon FDA approval FDA Completes Reinspection Drug Product Vialed To support FDA review FDA/Catalent Indiana Meeting Early Q1 2026 Type C Meeting with FDA Within 90 days of end of reinspection, per FDA guidelines FDA Classifies Facility Commercial Apitegromab Supply Available Early Q3 2026 CATALENT INDIANA FACILITY SECOND FILL-FINISH FACILITY Apitegromab BLA Accepted Submitted late March; includes two fill-finish facilities POTENTIAL FDA APPROVAL Early Q2 2026 Early March 2026 |
| © 2026 Scholar Rock, Inc. All rights reserved. SHAPING THE FUTURE OF TREATMENT FOR PATIENTS LIVING WITH RARE NEUROMUSCULAR DISEASES INITIATE PHASE 2 STUDY for apitegromab in facioscapulohumeral muscular dystrophy SRK-439 PHASE 1 STUDY topline data in healthy volunteers MID - 2026 H2 2026 APITEGROMAB FOR PATIENTS WITH SMA Myostatin inhibitor with a successful Phase 3 study Only muscle-targeted treatment to show a statistically significant, clinically meaningful benefit in SMA ON TRACK BLA resubmission accepted by FDA; PDUFA action date of September 30th CHMP opinion anticipated near mid-2026 ~35,000 have received an SMN-targeted therapy1-3 $2B+ opportunity to serve patients with SMA alone $480M in cash and cash equivalents as of March 31, 2026 7 Scholar Rock is poised for a transformative year SMA, Spinal Muscular Atrophy; PDUFA, Prescription Drug User Fee Act; CHMP, Committee for Medicinal Products for Human Use; SC, Subcutaneous. 1. Biogen Q4 2023 Report; 2. Roche Q3 2024 report; 3. Novartis Q4 2024 Report. PHASE 2 OPAL STUDY for apitegromab in infants and toddlers with SMA UNDERWAY SC APITEGROMAB development activities ONGOING |
| © 2026 Scholar Rock, Inc. All rights reserved. R&D Progress Akshay Vaishnaw, M.D., Ph.D. President of R&D 8 |
| © 2026 Scholar Rock, Inc. All rights reserved. 9 Advancing Scholar Rock’s world-leading anti-myostatin pipeline *Following FDA and EMA regulatory approval of IV apitegromab; YoA, Years of Age; PDUFA, Prescription Drug User Fee Act; CHMP, Committee for Medicinal Products for Human Use. Program Indication Milestone Apitegromab Spinal Muscular Atrophy ≥2 YoA PDUFA action date of September 30 CHMP opinion mid-2026 Facioscapulohumeral muscular dystrophy Phase 2 FORGE study initiation in mid-2026 SRK-439 Healthy Volunteers Phase 1 study topline results expected in H2 2026 Subcutaneous Apitegromab Development activities ongoing, with anticipated FDA and EMA regulatory engagements on clinical path forward* Spinal Muscular Atrophy Phase 2 OPAL study in infants and toddlers ongoing Spinal Muscular Atrophy <2 YoA |
| © 2026 Scholar Rock, Inc. All rights reserved. Evaluating PK, PD, efficacy, safety, and tolerability of apitegromab over 48 weeks 1 0 Furthering our commitment to broad SMA community Ongoing Phase 2 OPAL study evaluating apitegromab in infants and toddlers with SMA PK, Pharmacokinetic; PD, Pharmacodynamic. Patient enrollment and dosing underway in Phase 2 OPAL study including evaluation of apitegromab in patients who received SMN1-targeted gene therapy Expanding our potential impact to reach patients earlier Focused on addressing the needs of children <2 years of age with SMA seeking to address the motor neuron and muscle in youngest patients Time is muscle |
| © 2026 Scholar Rock, Inc. All rights reserved. 1 1 FSHD: Rare, devastating NMD with significant unmet need >30,000 patients diagnosed in U.S. and Europe1-3; no approved therapies FLExD, FLExDUX4.Cre; FSHD, facioscapulohumeral muscular dystrophy; NMD, Neuromuscular Disease; WT, wild type; Ab, antibody; 6MWD, 6-minute walk distance. 1.Scholar Rock. Data on file. 2. Tihaya MS, et al. Nat Rev Neurol. 2023;19(2):91-108. 3. Attarian S, et al. J Neurol. 2024;271(9):5778-5803. 4. Fogel A. Presented at: FSHD Society International Research Congress; Jun 12- 13, 2025; Amsterdam, Netherlands. 5. Bankole, LC, et al. Medicine. 2016; 95(31). 6. Andersen, G, et al. Neurology. 2015; 85:396-403. 7. Heatwole, CR, et al. Neurol Genet. 2025;11:e200292. Support for apitegromab therapeutic hypothesis in FSHD Randomized studies of exercise programs suggest muscle has capacity to show functional benefit5,6 Study of anabolic agents suggests increase in lean mass and muscle function7 Increase in Lean Body Mass Improvement in 6MWD FSHD FLExDUX4 Mouse Model Robust Increase in Muscle Mass (28 Days)4 WT Control Ab muSRK-015P 0 20 40 60 TA Weight (mg) 11% Phase 2 FORGE study on track to initiate mid-2026 |
| © 2026 Scholar Rock, Inc. All rights reserved. 1 2 Advancing innovation with subcutaneous apitegromab and SRK-439 SRK-439 is a novel, investigational myostatin inhibitor. Leveraging world-leading expertise to drive continued innovation Subcutaneous Apitegromab Phase 1 study comparing intravenous (IV) and subcutaneous (SC) apitegromab in healthy volunteers At 800mg, SC and IV apitegromab produced overlapping PD responses (total latent myostatin) Further development activities ongoing, including anticipated FDA and EMA regulatory engagements following apitegromab approvals SRK-439 Novel, highly potent myostatin inhibitor Phase 1 study in healthy volunteers in progress; topline data expected in H2 2026 Optimized for subcutaneous administration Strong preclinical data demonstrating favorable muscle mass preservation |
| © 2026 Scholar Rock, Inc. All rights reserved. Commercial Readiness Keith Woods Chief Operating Officer 1 3 |
| © 2026 Scholar Rock, Inc. All rights reserved. 1 4 Muscle strength and motor function remain the top unmet need in children and adults living with SMA SMA, Spinal Muscular Atrophy. Scholar Rock’s disease education program continues to focus on a broader understanding of SMA as a disease of the motor unit— which consists of both the motor neuron and the muscle MUSCLE MOTOR NEURON OF PATIENTS continue to experience persistent and progressive 95% muscle atrophy that limits function and independence OF PEOPLE LIVING WITH SMA IN THE U.S. have received two or more FDA approved SMA treatments, either sequentially or in combination ~1/3 |
| © 2026 Scholar Rock, Inc. All rights reserved. 1 5 U.S commercial team operating with urgency to prepare for launch SMA, Spinal Muscular Atrophy. SMA Treatment Centers, SMA Prescribing Physicians, Multi-Disciplinary Care Teams TARGETING >2,600 SMA Prescribers TARGETING 140 SMA Centers Patient Engagement & Community Activation National & Regional Payers Medicare & Medicaid |
| © 2026 Scholar Rock, Inc. All rights reserved. 1 6 Strong momentum with apitegromab launch readiness in Europe in advance of mid-2026 CHMP opinion SMA, Spinal Muscular Atrophy; CHMP, Committee for Medicinal Products for Human Use. 1 Building world-class team 2 Engaging the SMA community 3 Establishing access |
| © 2026 Scholar Rock, Inc. All rights reserved. ~35,000 SMA patients have received an approved SMN-targeted therapy1-3 Powering Scholar Rock through the end of this decade and into the next 1 7 Global apitegromab opportunity in SMA alone offers potential for many years of sustainable growth 1. Biogen Q4 2023 Report; 2. Roche Q3 2024 report; 3. Novartis Q4 2024 Report. SMN, survival motor neuron. |
| © 2026 Scholar Rock, Inc. All rights reserved. Company Financials Vikas Sinha Chief Financial Officer 1 8 |
| © 2026 Scholar Rock, Inc. All rights reserved. 1 9 Operating with financial discipline to achieve our ambitions as of March 31, 2026 $480M in cash and equivalents Prioritized investments focused on: Apitegromab commercial launch readiness in the U.S. and Europe 1 Strengthening supply chain to support expanding pipeline and anticipated growing global commercial demand for apitegromab over time 2 3 Advancing highly innovative clinical programs |
| © 2026 Scholar Rock, Inc. All rights reserved. Closing Remarks David L. Hallal Chairman and Chief Executive Officer 2 0 |
| © 2026 Scholar Rock, Inc. All rights reserved. Q&A 2 1 |
| © 2026 Scholar Rock, Inc. All rights reserved. For more information, please contact: ir@scholarrock.com media@scholarrock.com Or visit us at www.scholarrock.com 2 2 |
