EX-99.2 3 srrk-20250227xex99d2.htm EX-99.2
Exhibit 99.2

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Fourth Quarter 2024 Business Update February 27, 2025 © 2025 Scholar Rock, Inc. All rights reserved.

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Forward-Looking Statements © 2025 Scholar Rock, Inc. All rights reserved. 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 strategy, its product candidate selection and development timing, including timing for the initiation of and reporting results from its preclinical studies and clinical trials for apitegromab, SRK-439 and other product candidates and indication selection and development timing, its cash runway, its ability to use its expanded credit facility, 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 3 trial of apitegromab, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidate; Scholar Rock’s ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials, including from the EMBRAZE clinical trial; 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 manage expenses and to obtain additional funding 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 to obtain regulatory approval of apitegromab; its ability to expand globally and the anticipated commercial launch in the United States of apitegromab in the fourth quarter of 2025; as well as 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, 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. 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 is an 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 SRK-439 have not been established. 2

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Agenda © 2025 Scholar Rock, Inc. All rights reserved. 3 TO P I C S P E A K E R Apitegromab in SMA SAPPHIRE Phase 3 Pivotal Trial Results and Regulatory Milestones Jay Backstrom, President & Chief Executive Officer Apitegromab in SMA Planning for a 2025 Launch to Serve Patients Tracey Sacco, Chief Commercial Officer Scholar Rock Innovative Myostatin Platform Ambition to Transform Current GLP-1 Treatment Paradigm Jay Backstrom, President & Chief Executive Officer Q&A Session

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2025 ANTICIPATED MILESTONES MAA submission apitegromab Q1 EMBRAZE Ph2 POC data readout Q2 OPAL study initiation Q3 SRK-439 IND filing Q3 US launch apitegromab* Q4 Strong Momentum in 2024 Sets the Stage for a Transformative Year Ahead © 2025 Scholar Rock, Inc. All rights reserved. 4 RECENT ACCOMPLISHMENTS Positive Phase 3 SAPPHIRE data BLA Submitted for apitegromab in SMA SAPPHIRE data and DMD poster accepted for presentation at MDA * Assuming priority review granted and regulatory approval received. SMA Cardiometabolic 2025

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S E L E C T I V I T Y D R I V E S S U C C E S S Positive Phase 3 Trial Using HFMSE, Gold Standard SMA Scale © 2025 Scholar Rock, Inc. All rights reserved. 5 * Based on apitegromab combined dose (10 mg/kg and 20 mg/kg) + SOC versus placebo + SOC (Hochberg multiplicity adjustment). † 12.5% of patients on placebo + SOC achieved a ≥3-point improvement in HFMSE Apitegromab is subject to regulatory approval. SOC=Standard of care (i.e., nusinersen or risdiplam); HFMSE=Hammersmith Functional Motor Scale–Expanded. The Only Muscle-Targeted Therapy with Over 4 Years of Clinical Experience in >200 Patients Apitegromab has the potential to transform the standard of care in Spinal Muscular Atrophy (SMA) 30% vs.12.5% ACHIEVED ≥3 POINT IMPROVEMENT IN HFMSE† apitegromab + SOC patients vs. placebo + SOC CONSISTENT clinically meaningful benefit observed across all age groups (2‒21) FAVORABLE SAFETY profile consistent with >48 months experience in Phase 2 TOPAZ trial 1.8 POINT IMPROVEMENT (p=0.0192) in HFMSE* vs. placebo

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Apitegromab in SMA: Planning for a 2025 Launch to Serve Patients © 2025 Scholar Rock, Inc. All rights reserved. 6 Tracey Sacco, MBA Chief Commercial Officer

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C O M M E R C I A L P R E P A R E D N E S S Progressive, Debilitating Muscle Atrophy Remains a Critical Unmet Need in SMA Despite SMN-Targeted Therapies © 2025 Scholar Rock, Inc. All rights reserved. 7 1. Cure SMA State of SMA 2023 Report. https://www.curesma.org/wp-content/uploads/2024/06/9042024_State-of-SMA_vWeb.pdf; 2. Scholar Rock Internal Market Research (US Neurologists), 2024. The SMA community is calling for a treatment to address progressive muscle weakness Muscle atrophy and loss of strength is a key issue in these patients. Increasing a patients’ HFMSE score is really important. It’s measurable and meaningful. Pediatric Neurologist (UK) Personal hygiene, using the toilet and the shower on my own would be huge. My four-year-old can do it on her own. It’s degrading. US Patient >80% of neurologists agree efforts to preserve muscle should start as early as possible in SMA2 97% of patients seek improving muscle strength from a new treatment in SMA1

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C O M M E R C I A L P R E P A R E D N E S S The Future of SMA is in Treating the Muscle and the Motor Neuron © 2025 Scholar Rock, Inc. All rights reserved. 8 1. Cure SMA State of SMA 2023 Report. https://www.curesma.org/wp-content/uploads/2024/06/9042024_State-of-SMA_vWeb.pdf; 2. Biogen Q4 2023 Report; 3. Roche Q3 2024 report; 4. Novartis Q4 2024 Report;5. Scholar Rock internal market research (US Neurologists), 2024; 6. Cure SMA, 2025. Approaches to Drug Development. https://www.curesma.org/approaches-to-drug-development/ WORLDWIDE ~35,000 patients have received an approved SMN-targeted therapy2,3,4 IN THE US ~10,000 living with SMA1 2/3 SMA patients received an SMN therapy1 SMA Today 74% of neurologists agree that in the future, multiple modalities are necessary to treat SMA5 Many researchers believe that it will take a combination of SMN-dependent and SMN-independent treatments to provide the most benefit for those with SMA – Cure SMA6

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C O M M E R C I A L P R E P A R E D N E S S Preparing the Market for a Potential New Treatment Paradigm in SMA © 2025 Scholar Rock, Inc. All rights reserved. 9 Disease Education • Amplifying the patient voice in first muscle-focused SMA disease education campaign HCP Engagement • MSL team engaged 100% of Cure SMA-identified sites • Key HCPs, including doctors, nurses, PTs and OTs engaged since SAPPHIRE topline Patient Advocacy Partnership • Long-tenured relationship and strong partnerships across initiatives on education and access

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C O M M E R C I A L P R E P A R E D N E S S Significant Rare Disease Experience to Drive Successful Apitegromab Launch © 2025 Scholar Rock, Inc. All rights reserved. 1 0 ATTRACTING TOP TALENT To build world-class commercial organization RARE DISEASE LAUNCH EXPERIENCE Among Commercial, Medical Affairs, and Patient Advocacy teams SEASONED COMMERCIAL LEADERSHIP Commercial leadership team averaging 20+ years of industry experience INTERNAL COMMERCIAL EXPERTISE Across market access, marketing, sales, and operations

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C O M M E R C I A L P R E P A R E D N E S S Scholar Rock is Positioned for a Successful Launch in SMA © 2025 Scholar Rock, Inc. All rights reserved. 1 1 * Assuming priority review granted and regulatory approval received. Initiating payer outreach to top US commercial and federal payers in Q1 Ongoing disease education and SMA stakeholder engagement Building a customer-facing team of sales, reimbursement, and patient support personnel in Q2/Q3 Preparing patient services and home infusion offering for a Q4 launch*

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Scholar Rock Innovative Myostatin Platform: Ambition to Transform Current GLP-1 Treatment Paradigm © 2025 Scholar Rock, Inc. All rights reserved. Jay Backstrom, M.D., MPH 1 2

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C A R D I O M E T A B O L I C P R O G R A M GLP-1 RAs are Transforming Weight Loss for Millions of People © 2025 Scholar Rock, Inc. All rights reserved. 1 3 Source: UBS Bank, GLP-1: A medication worth $126 billion in sales by 2029? https://www.ubs.com/global/en/investment-bank/insights-and-data/2024/glp-1-a-medication.html. GLP-1 RA=GLP-1 receptor agonist. RECENTLY APPROVED GLP-1 RAs are highly effective in weight loss & experiencing rapid uptake BY 2029... 40 million people on GLP-1 RAs $126 billion in sales*

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C A R D I O M E T A B O L I C P R O G R A M However, Patients Want Healthier Weight Loss* © 2025 Scholar Rock, Inc. All rights reserved. 1 4 *Source: Scholar Rock market research, completed December 2024. GLP-1 RA=GLP-1 receptor agonist. WEAKNESS is a Concern Patients complain of reduced strength after GLP-1 RA treatment Patients hope for a combination treatment approach to address this need Improved LEAN MASS Significant Weight REGAIN In patients after stopping GLP-1 RA treatment, with fat regain > lean mass Scholar Rock’s unique highly selective approach to targeting latent myostatin has the potential to address these patient needs Patients feel good about the number on the scale, but there are issues with muscle loss … they complain of weakness or reduced strength. Obesity Clinician

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C A R D I O M E T A B O L I C P R O G R A M Significant Muscle Loss Accompanies Tirzepatide-Induced Weight Loss © 2025 Scholar Rock, Inc. All rights reserved. 1 5 Figure from Jastreboff, A.M. et al. N Engl J Med. 2022;387(3):205-216. * Data from Phase 3 clinical trial of tirzepatide in adults with obesity. Tirzepatide Percent Change in Body Weight -24 -20 -16 -12 -8 -4 0 -3.1 -15.0 -19.5 -20.9 0 4 8 12 16 20 24 36 48 60 72 TRE Weeks Since Randomization -2.4 -16.0 -21.4 -22.5 Tirzepatide, 5 mg Tirzepatide, 10 mg Tirzepatide, 15 mg Placebo Percent Change in Body Weight by Week (efficacy estimand) Time period of EMBRAZE Overall mean baseline weight = 104.8 kg DECREASE 14-16% body weight (week 24) ~25% of weight lost is lean mass

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C A R D I O M E T A B O L I C P R O G R A M Every Kilogram Matters: Significant Cost of Losing Lean Mass © 2025 Scholar Rock, Inc. All rights reserved. 1 6 *Marlou L Dirks, Benjamin T Wall, et al; Diabetes 2016; 65:2862-2875 Mitchell W.K, Williams J, et al Front. Physiol. 2012; 3: 260 | Gustafsson T and Ulfhake B; Int. J. Molecular Sciences. 2024; 25: 1-16 | Siparsky P.N., Kirkendall D.T et al. Sports Health 2024; 6 (1): 36-40 | Macaluso, A. and De Vito, G. Eur. J. Appl. Physiol. 2004; 91(4):450-472. Loss of ~1 kg lean mass resulted in decreased leg strength and increased insulin resistance* LEAN MASS Loss of~1% per year Adults >60 (M>F) STRENGTH Loss of 10‒15% per decade ~7% decrease in leg strength ~6% reduction in exercise capacity ~30% decrease in insulin sensitivity

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C A R D I O M E T A B O L I C P R O G R A M Goals of the EMBRAZE Proof-of-Concept Study © 2025 Scholar Rock, Inc. All rights reserved. 1 7 COMPARABLE WEIGHT LOSS REDUCE FAT REGAIN PRESERVE LEAN MASS COMPARABLE SAFETY (no added toxicities) INFORMING THE DEVELOPMENT OF SRK-439 Highly selective muscle-targeted therapy in obesity Exploratory: Potential to improve METABOLIC PROFILE (e.g., HbA1c) and PHYSICAL FUNCTION

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C A R D I O M E T A B O L I C P R O G R A M SRK-439: Best in Class Potential © 2025 Scholar Rock, Inc. All rights reserved. 1 8 GLP-1 RA: GLP1 receptor agonist 1. Nunn E, et al., Mol Metab 2024; 2. Schang G., et al, J. Endoc Soc 2023; 3. Ackerman, P, et al. Obesity Week 2023 Poster 211; 4. 1. Barrett et al. Adv Therapy. 2021; 5. Crawford T et al. Neurology. 2024; 6. Garito T et al. Clin Endocrinol (Oxf). 2018; 7. Amato AA et al. Neurology. 2021; 8. Heymsfield SB et al. JAMA. 2021; 9. Vanhoutte F et al. J Clin Pharmacol. 2020; 10. Attie KM et al. Muscle Nerve. 2013; 11. Attie KM et al. Am J Hematol. 2014; 12. Campbell C et al. Muscle Nerve. 2017; 13. Hoeper MM et al. N Engl J Med. 2023; 14. Ruckle J et al. J Bone Miner Res. 2009; 15. Sherman ML et al. J Clin Pharmacol. 2013; 16. Muntoni F et al. Neurol Ther. 2024. 17. Di Rocco M et al. Nat Med. 2023. SRK-439 ActRII Ab Ligand Trap Adnectin Selectivity for myostatin Action limited to muscle Combination GLP-1 RA data in obesity preclinical models1-3 Low efficacious dose in preclinical obesity models1-3 Potential lower risk of undesirable effects in clinic4-17

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C A R D I O M E T A B O L I C P R O G R A M Industry-Leading Anti-Myostatin Platform: Leveraging Apitegromab’s Success to Advance the Obesity Program 19 *Expected timelines POC=Proof of Concept; GLP-1 RA=GLP-1 receptor agonist. POC data readout Q2* Ph 2 proof-of-concept trial APITEGROMAB + tirzepatide 2025 IND submission Q3* SRK-439 + GLP-1 RA Novel asset for cardiometabolic indication Phase 1 trial Cardiometabolic Disorders • EMBRAZE POC readout in Q2 2025 • SRK-439 IND submission in Q3 2025 Testing hypothesis of selective anti-myostatin antibody in obese population © 2025 Scholar Rock, Inc. All rights reserved.

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Conclusions © 2025 Scholar Rock, Inc. All rights reserved. 2 0

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2 1 © 2025 Scholar Rock, Inc. All rights reserved. Scholar Rock is Moving with a Sense of Urgency To Bring Transformative Medicines to Patients TO D AY ’ S F O C U S *Pending regulatory approval. EXPAND Apitegromab Development Program: Building a Pipeline in a Product COMMERCIALIZE Execute a Successful Commercial Launch* ADVANCE Anti-myostatin Program into Cardiometabolic Indications 1 2 3 BLA submitted for apitegromab in SMA and potential US launch in 4Q 2025 $2B+ opportunity in SMA MAA on track for 1Q 2025 submission Reaching more patients for apitegromab in additional rare neuromuscular diseases 2Q 2025 EMBRAZE Phase 2 study results expected IND submission on track for 3Q 2025 for SRK-439, a highly innovative myostatin inhibitor Earlier treatment in SMA on track to initiate OPAL study for patients under 2 years of age with SMA in 3Q 2025

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Q&A © 2025 Scholar Rock, Inc. All rights reserved. 2 2

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Q&A: Scholar Rock Management © 2025 Scholar Rock, Inc. All rights reserved. 2 3 Ted Myles, MBA Chief Operating Officer and Chief Financial Officer Jay Backstrom, M.D., MPH President & Chief Executive Officer Tracey Sacco, MBA Chief Commercial Officer Mo Qatanani, PhD Chief Scientific Officer