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 4, 2024
 
Scholar Rock Holding Corporation
(Exact Name of Registrant as Specified in Charter)
 
Delaware
001-38501
82-3750435
(State or Other Jurisdiction of 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:
 
   Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
   Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
   Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
   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.
 
On January 4, 2024, Scholar Rock Holding Corporation (the “Company”) issued a press release which contained information regarding the Company’s preliminary, unaudited estimate of cash and cash equivalents of approximately $280 million as of December 31, 2023, which is projected to fund the Company’s operations into the second half of 2025. This information is preliminary and unaudited. The Company expects to report its audited cash, cash equivalents and marketable securities, as well as other information necessary for a complete understanding of its financial position, in its Annual Report on Form 10-K for the year ended December 31, 2023.
 
The information in this Item 2.02 is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934 (the “Exchange Act”) or otherwise subject to the liabilities of that section.  It may only be incorporated by reference in another filing under the Exchange Act or the Securities Act of 1933, as amended, if such subsequent filing specifically references the information furnished pursuant to Item 7.01 and Exhibit 99.1 of this Current Report on Form 8-K.
 
Item 7.01. Regulation FD Disclosure.

On January 4, 2024, the Company announced that management will present at the 42nd Annual J.P. Morgan Healthcare Conference on Tuesday, January 9, 2024 at 1:30 p.m. PT (4:30 p.m. ET).  A copy of the presentation slide deck that will be presented is being furnished as Exhibit 99.1 to this report on Form 8-K.  A live webcast of the presentation may be accessed by visiting the Investors & Media section of the Scholar Rock website at http://investors.scholarrock.com.
 
The information in this Item 7.01 and Exhibit 99.1 attached hereto is 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.  It may only be incorporated by reference in another filing under the Exchange Act or the Securities Act of 1933, as amended, if such subsequent filing specifically references the information furnished pursuant to Item 7.01 and Exhibit 99.1 of this Current Report on Form 8-K.
 
Item 8.01. Other Events.

On January 4, 2024, the Company issued a press release announcing a corporate update and highlighting priorities for 2024. A copy of this press release is being filed as Exhibit 99.2 to this Current Report on Form 8-K and is incorporated by reference herein.
 
Item 9.01. Financial Statements and Exhibits. 
 
(d) Exhibits

Exhibit
No.
 
Description
 
 
 
 
 
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
 
 
 
 
 
 
Date: January 8, 2024
By:
/s/ Junlin Ho
 
 
Junlin Ho
 
 
General Counsel & Corporate Secretary

Exhibit 99.1

 © 2024 Scholar Rock, Inc. All rights reserved.  Advancing  New Possibilities for Patients  42nd ANNUAL J.P. MORGAN  HEALTHCARE CONFERENCE  JANUARY 9, 2024 
 

 © Scholar Rock, Inc. All rights reserved. 2024.  Forward-Looking Statements  Various statements in this presentation concerning the future expectations, plans and prospects of Scholar Rock, Inc. (“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 SRK-439, apitegromab, SRK-181, and other product candidates and indication selection and development timing, its cash runway, 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 trial of apitegromab or Part A or Part B of the Phase 1 trial of SRK-181, 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, including the Phase 3 clinical trial of apitegromab in SMA and Part B of the Phase 1 clinical trial of SRK-181, respectively, 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, 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 and establish and maintain strategic business alliances and new business initiatives, and the impacts of current macroeconomic and geopolitical events, hostilities in Ukraine, increasing rates of inflation and rising interest rates, on business operations and expectations, 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, 2022, and Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, 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 and SRK-181 are investigational drug candidates under evaluation. Apitegromab, SRK-181, 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-181 and SRK-439 have not been established. 
 

 3  TGFβ=Transforming growth factor-beta. SMA=Spinal muscular atrophy  *Christopher is a participant in the TOPAZ and ONYX clinical trials.  OUR MISSION  To discover, develop, and deliver life-changing therapies by harnessing cutting-edge science to create new possibilities for people living with serious diseases  We are a global leader in harnessing the life-changing potential of TGFβ biology  Emily & Christopher  living with SMA*  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 4  Building a Fully Integrated Therapeutics Company  SMA=Spinal muscular atrophy; EMA=European Medicines Agency; FDA=United States Food and Drug Administration; R&D=research and development.  Apitegromab in SMA  Potential therapy in Ph 3 designed to improve motor function to help address remaining unmet need after receipt of existing SMA therapies  SRK- 439 in Obesity  Novel antimyostatin antibody with the potential to support healthier weight management by preserving lean muscle  SRK-181 in Immuno-Oncology  In Ph 1 development to overcome resistance to checkpoint inhibitors in multiple tumor types  Revolutionary Scientific Platform  Pioneers in unparalleled  selective targeting of the latent forms  of growth factors  Robust pipeline of novel assets including two clinical programs and a growing portfolio of preclinical programs  Transformative Therapeutics  in Development  Experienced and Focused  Seasoned team with track record of  clinical and commercial success  Deep rare disease, R&D, FDA/EMA  approval & launch experience  Focused, efficient approach to scaling the organization  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 Traditional Target  “mature” active growth factor  Scholar Rock’s Target  Latent Growth Factor  Our Approach  S e l e c t i v i t y D r i v e s S u c c e s s  Industry-leading antibody design and protein engineering to selectively target latent growth factors  Optimized for efficacy and mitigates off-target effects  Deep structural insights to validated targets  RIGHT  TIME  L a t e n t  F o r m  RIGHT  TARGE T  V a l i d a t e d B i o l o g y  5  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 Latent  Myostatin  SPINAL MUSCULAR ATROPHY  Apitegromab  CARDIOMETABOLIC DISORDERS  Apitegromab in Obesity*  SRK-439 (novel antimyostatin antibody)  Latent  TGFβ-1  IMMUNO-ONCOLOGY  SRK-181 (selective context-independent,  anti-latent TGFβ-1)  FIBROSIS  Selective context-dependent (LTBP1 & LTBP3) anti-latent TGFβ-1  RGMc  ANEMIA  Selective anti-RGMc  TARGET  DISCOVERY/  PRECLINICAL PHASE 1 PHASE 2 PHASE 3  Potential to transform the lives of people living with a wide range of serious diseases, including neuromuscular disorders, cardiometabolic disorders, oncology, and fibrosis  Advancing a Robust Pipeline with Our Differentiated Platform  * Subject to receipt of regulatory authority approval. We plan to utilize data from a previously completed Ph 1 study in healthy volunteers and initiate a Ph 2 POC trial in 2024.  LTBP1=Latent transforming growth factor beta binding protein 1; LTBP3=Latent transforming growth factor beta binding protein 3; POC=Proof of concept; RGMc=Repulsive guidance molecule C; TGFβ-1=Transforming growth factor beta-1.  6  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 © 2024 Scholar Rock, Inc. All rights reserved.  Antimyostatin Program: Apitegromab in Spinal Muscular Atrophy 
 

 8  Hallmarks of SMA  Motor Neuron Loss and Muscle Atrophy Leads to Progressive Muscle Weakness  SMA=Spinal muscular atrophy; SMN=Survival motor neuron.  Hua Y, et al. Nature. 2011;478(7367):123-6.  Figure adapted from: SMA Foundation Overview. http://www.smafoundation.org/wp-content/uploads/2012/03/SMA-Overview.pdf.; Accessed April 18, 2021.  S P I N A L M U S C U L A R A T R O P H Y  SMN therapies  slow further degeneration of motor neurons1  ...but do not directly address muscle atrophy  Motor neuron degeneration2  Muscle fiber  atrophy  Spinal Muscular Atrophy  Motor neuron impairment and loss due to SMN genetic deficiency leads to muscle atrophy and weakness  There is further potential to  regain vital muscle function by also addressing the progressive muscle atrophy and associated weakness of SMA  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 SMA Leads to Deterioration in Essential Muscle Function  Quotes are from patient advocates who participated in 2022 Cure SMA FDA Patient-Led Listening Session and not from the pictured individuals. Summary of the listening session can be found on the FDA website at https://www.curesma.org/cure-sma-holds-patient-led-listening-session-with-fda/  9  S P I N A L M U S C U L A R A T R O P H Y  Despite significant advancements, progressive muscle weakness remains an unmet need in SMA  Small tasks are huge success in my life. If I could lift that 1L bottle of water at work instead of having to find a graduate student to move it for me…things don’t take a ton more muscle, but they are all muscle I still don’t have.  What may seem like minimal gains in strength actually translate to exponential gains in functional abilities.  I often have to choose between taking a shower and doing homework because I don't have the energy to do both.  Muscle weakness can lead to deterioration in mobility, swallowing, breathing and cause debilitating fatigue  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 10  SMA Today: More Patients Screened and Treated  CHF=Swiss franc; LTM=last twelve months; SMA=Spinal muscular atrophy; SMN=Survival motor neuron; WW=worldwide.  Cure SMA 2022 Report: 9042022_State-of-SMA_vweb.pdf (curesma.org)  Lally et al. Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States. Orphanet J Rare Dis. 2017 Nov 28;12(1):175. doi: 10.1186/s13023-017-0724-z.  Revenue as of Biogen 3Q23 financial update; includes patients treated worldwide in post-marketing setting, expanded access program, and clinical trials as of May 2022.  Revenue as of Roche 3Q23 financial update; includes patients treated worldwide as of July 2023.  S P I N A L M U S C U L A R A T R O P H Y  GLOBAL DISEASE:  >20,000 affected  in US and Europe1, 2  Three treatments to address SMN loss  >13,000 patients  treated WW  $1.8 billion  annual revenue (LTM)3  > 11,000 patients  treated WW  ~CHF1.4 billion  annual revenue (LTM)4  > 3,500 patients  treated WW  ~$1.2 billion  in revenues (LTM)5  Established market dynamics support Scholar Rock’s first potential commercial launch  5. Revenue as of Novartis 3Q23 financial update; includes patients treated worldwide including clinical trials, commercially, and managed access programs as of August 2023.  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 11  Muscle-Targeted Therapy: A New Treatment Frontier  *Percentages represent percent of patients who named these unmet needs when asked, “What are your most significant current unmet needs that you hope new therapies would address?”  HFMSE=Hammersmith Functional Motor Scale-Expanded.  1. Mercuri E et al.; N Engl J Med 2018; 378:625-635; DOI: 10.1056/NEJMoa1710504; cherish trial results; 2. 2022 Community Update Survey, Cure SMA.  S P I N A L M U S C U L A R A T R O P H Y  0  Mean improvement in HFMSE experienced by patients in nusinersen Phase 3 CHERISH trial1  20  40  60  80  3.9-point increase  in HFMSE from nusinersen  (4.9 point increase relative to sham control)  HFMSE Score at Month 15  Total Possible  HFMSE Score of 66  Unmet need remains substantial  Patients and caregivers want new therapies to address the following unmet needs2 :  INCREASE  muscle strength  This third-party information is provided for background only and is not intended to convey or imply a comparison to the TOPAZ clinical trial results.  © 2024 Scholar Rock, Inc. All rights reserved.  IMPROVE  daily activities  STABILIZE  or GAIN new motor function  REDUCE  fatigue 
 

 12  Apitegromab Offers Significant Potential to Address Unmet Needs  * Based on Animal Model Data; 1. Long KK, et al. Hum Mol Genet. 2019;28(7):1077-1088; 2. Pirruccello-Straub M, et al. Sci Reports. 2018;8(1):2292. doi:10.1038/s41598-018-20524-9 3.  S P I N A L M U S C U L A R A T R O P H Y  Apitegromab is a selective MUSCLE-TARGETED APPROACH  designed to improve motor function*1,2  Myostatin is a negative modulator of muscle growth  Strong clinical and preclinical evidence indicates upstream targeting of structurally differentiated pro- and latent myostatin avoids undesirable off-target effects  Apitegromab specifically and only inhibits myostatin and has the potential to build muscle and strength  to improve patient outcomes  Apitegromab  Muscle fiber  atrophy  Motor neuron  degeneration3  Figure adapted from: SMA Foundation Overview. http://www.smafoundation.org/wp-content/uploads/2012/03/SMA-Overview.pdf.; Accessed April 18, 2021. For illustrative purposes only.  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 © 2024 Scholar Rock, Inc. All rights reserved.  Time (weeks)  No. of observations  N = 35; Baseline mean age=7.3 |Time on SMN Rx=24.1m  PRO=patient reported outcomes; HFMSE=Hammersmith Functional Motor Scale Expanded; OC=observed case; PEDI-CAT=Pediatric Evaluation of Disability Inventory Computer Adaptive Test; PROMIS=Patient Reported Outcome Measurement Information System; RULM=Revised upper limb module; SE=standard error of the mean. Pooled nonambulatory patients, age 2-21, all doses. Crawford et al., Cure SMA 2023  32  32 31  27  27  27  Change from baseline (±SE)  Year 1  IMPROVEMENT  8  7  6  5  4  3  2  1  0  -1  -2  0  136  156  Year 1  IMPROVEMENT  2  1  0  -1  -2  -3  -4  -5  -6  -7  -8  0  PROMIS Fatigue (Proxy)  Year 2 Year 3  IMPROVEMENT  28  27  Change from baseline (±SE)  5  4  3  2  1  0  -1  -2  0  Time (weeks) 0 8 16 24 32 40 52 68 84 104 120 136 156 0 8 16 24 32 40 52 68 84 104 120 136 156  No. of observations 31 29 30 21 23 25 25 27 22 23 16 15 17 25 23 22 19 19 22 16 18 19 19 16 14 14  0 8 16 24 32 40 52 68 84 104 120  35 35 34 29 28 29 32 32 32 29 27  PEDI-CAT Daily Activities  Year 1 Year 2 Year 3  Year 3  Year 2 Year 3 Year 1 Year 2  -2  -1  0  1  2  3  4  0  0 8 16 24 32 40 52  34 34 34 28 27 29 31  68  84 104  120  136  156  IMPROVEMENT  HFMSE RULM  13  S P I N A L M U S C U L A R A T R O P H Y  TOPAZ Over 36 Months  Sustained Functional and PRO Improvements Beyond SMN Treatment 
 

 14  TOPAZ Over 36 Months  Well Tolerated Safety Profile & Low Discontinuation Rate  S P I N A L M U S C U L A R A T R O P H Y  RIGHT TIME  L a t e n t F o r m  M y o s t a t i n  RIGHT  TARGE T  Crawford et al., Cure SMA 2023  * Excludes patients on monotherapy  >90% of patients on combination therapy remained in extension  study*  Treatment-emergent adverse events (TEAEs) were consistent with previous reports with no new findings after 198 patient- years of exposure  – Most frequently reported TEAEs included headache, pyrexia,  COVID-19, nasopharyngitis, & upper respiratory tract infection  – TEAEs were mostly mild to moderate and generally consistent with the underlying patient population and nusinersen therapy  No treatment-related serious AEs or hypersensitivity reactions  No report of positive apitegromab antibodies (ADA)  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 15  SAPPHIRE Phase 3 Design is Optimized by Insights from TOPAZ  HFMSE=Hammersmith Functional Motor Scale Expanded.  TOPAZ Learnings  SAPPHIRE Design Elements  Phase 3 SAPPHIRE Trial Registrational trial with topline 12-month data readout expected in Q4 2024  S T U D Y P O P U L A T I O N  Substantial HFMSE gains observed in the nonambulatory Type 2/3 SMA cohorts  A G E  Exploratory age 2-12 analysis in nonambulatory  Type 2/3 showed transformative potential  D U R A T I O N  HFMSE gains substantial by 12 months of treatment  D O S E  Dose response seen (greater effect observed with 20 mg/kg over 2 mg/kg)  S T U D Y P O P U L A T I O N  Nonambulatory Type 2/3 SMA  Primary efficacy endpoint: HFMSE  A G E  Age 2-12 main efficacy population  Age 13-21 exploratory population  D U R A T I O N  12-month treatment duration  D O S E  20 mg/kg apitegromab dose  10 mg/kg apitegromab dose  S P I N A L M U S C U L A R A T R O P H Y  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 16  Apitegromab: Potential to Maximize Outcomes for People Living with Spinal Muscular Atrophy (SMA)  Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy (SMA). Apitegromab has not been approved for any use by the FDA or any other regulatory agency, and its safety and efficacy have not been established.  S P I N A L M U S C U L A R A T R O P H Y  Phase 2 TOPAZ Trial Demonstrated substantial and sustained functional improvements in Type 2 and nonambulatory Type 3 SMA patients  First and only muscle-targeted investigational treatment  to demonstrate clinical proof-of-concept in SMA  © 2024 Scholar Rock, Inc. All rights reserved.  Phase 3 SAPPHIRE Trial  Registrational trial with topline 12-month data expected in Q4 2024  ONYX Open-Label  Extension Study  Evaluating the long-term safety and efficacy of apitegromab in patients who have completed TOPAZ or SAPPHIRE  Transformative Potential to Change the Standard of Care 
 

 17  Expanding to Benefit More People Living with SMA  *Subject to regulatory approval.  SMA=Spinal muscular atrophy; Sub-Q=Subcutaneous  S P I N A L M U S C U L A R A T R O P H Y  WHERE WE ARE  WHERE WE WERE  WHERE WE ARE GOING  P H A S E 2  12-Month  COMPLETE  24-Month EXTENSION COMPLETE  36-Month EXTENSION COMPLETE  12-Month  Q4 2024  data  P H A S E 3  Scale for Commercial Launch (2025)*  Under 2 Years of Age  (including those treated with gene therapy)  Ambulatory Population  Sub-Q Formulation  Long-term  EXTENSION for  TOPAZ and SAPPHIRE studies  C O M M E R C I A L I Z A T I O N  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 © 2024 Scholar Rock, Inc. All rights reserved.  Next Horizon  Antimyostatin Program: Cardiometabolic Disorders 
 

 Obesity is Recognized as a Top Global Public Health Issue  1. The World Obesity Foundation, World Obesity Atlas 2022; 2. Ward ZJ, Bleich SN, Long MW, Gortmaker SL (2021) Association of body mass index with health care expenditures in the United States by age and sex. PLoS ONE 16(3): e0247307.  BY 2030, OBESITY WILL AFFECT:  >1 BILLION  adults  >250 MILLION  children and  adolescents1  Obesity can increase  the risk of comorbidities, such as some cancers, heart disease, and  type 2 diabetes  Adult obesity associated with more than $170 billion  in excess costs  annually in the U.S.2  In the US,  1 in 5 children and  more than 1 in 3  adults are obese  Obesity is a common, serious, and costly chronic disease affecting adults and children worldwide  19  O B E S I T Y  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 20  Loss of Lean Muscle Significant with GLP-1 RA Therapy  Lean muscle is essential to healthy metabolic function  GLP-1 RA=Glucagon-like peptide-1 receptor agonists.  1. Muller TD, et al Anti-obesity drug discovery: advances and challenges. Nature Reviews Drug Discovery 2022; 21, 201–223; 2. Wilding JPH, Batterham RL, Calanna S, et al. Once-Weekly Semaglutide in Adults with Overweight or Obesity. N Engl J Med. 2021;384(11):989-1002; 3. Jastreboff AM, et al Tirzepatide Once Weekly for the Treatment of Obesity. NEJM 2022; 387 (3): 205-216; 4. Cava et al. Preserving healthy muscle during weight loss. Adv Nutr 2017;8:511-19; 5. Lundgren JR et al. Healthy Weight Loss Maintenance with Exercise, Liraglutide or Both Combined. NEJM 2021;384:1719-30;  6. Beal JW et al. Dietary weight loss-induced improvements in metabolic function are enhanced by exercise in people with obesity and prediabetes. Nat Metab. 2022;5(7):1221-1235; 7. Dulloo AG, et al How dieting makes some fatter: from a perspective of human body composition autoregulation. Proc Nutr Soc. 2012 Aug;71(3):379-89.  O B E S I T Y  Significant proportion of weight loss due to loss of lean muscle mass     Recently approved GLP-1 RAs are highly effective in weight loss & experiencing rapid uptake  But 25%-40% of total body weight loss mediated by GLP-1 RA therapy may be attributed to loss of lean muscle mass2,3  Preserving lean muscle mass is important to promote  long-term metabolic benefits, sustainable weight management and health outcomes4-7  Current Weight Loss Strategies Challenged by:  Tolerability  Lack of durability  Significant  muscle loss1-3  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 21  Maintaining Muscle is Important for Healthy Weight Loss  GLP-1 RA=Glucagon-like peptide-1 receptor agonist.  1. Aristizabal JC, Freidenreich DJ, Volk BM, et al. Effect of resistance training on resting metabolic rate and its estimation by a dual-energy X-ray absorptiometry metabolic map. Eur J Clin Nutr.2015; 69, 831–836. https://doi.org/10.1038/ejcn.2014.216; 2. Lindegaard B, Hansen T, Hvid T, et al. The effect of strength and endurance training on insulin sensitivity and fat distribution in human immunodeficiency virus-infected patients with lipodystrophy. J Clin Endocrinol Metab. 2008; 93:3860–9; 3. Srikanthan P, Karlamangla AS. Relative muscle mass is inversely associated with insulin resistance and prediabetes. Findings from the third National Health and Nutrition Examination Survey. J Clin Endocrinol Metab. 2011; 96:2898–903. doi: 10.1210/jc.2011-0435; 4. Wewege MA, Desai I, Honey C, et al. The effect of resistance training in healthy adults on Body fat percentage, fat mass and visceral fat: A systematic review and meta-analysis. Sports Med. 2022(Feb);52(2):287-300. doi: 10.1007/s40279-021-01562-2; 5. Zurlo, F., Larson, K., Bogardus, C., et al. Skeletal muscle metabolism is a major determinant of resting energy expenditure. J Clin Invest. 1990;86(5), 1423-1427; 6. Fukushima Y, Kurose S, Shinno H, et al. Importance of lean muscle maintenance to improve insulin resistance by body weight reduction in female patients with obesity. Diabetes Metab J. 2016;40: 147-153; 7. Roh E, Choi KM. Health consequences  The preservation of lean mass has many benefits for overall health:  Improved strength  Improved insulin sensitivity  Increased basal metabolic rate  Improved metabolic profile  Reduced total body fat  O B E S I T Y  Better insulin sensitivity  and lower risk of prediabetes3  Reduced visceral fat4  Increased bone density, strength, function, and longevity and decreased risk of injury, and disability6-8  Increased caloric expenditure post-exercise5  Enhanced glucose homeostasis2  Increased basal metabolic rate (BMR)1  of sarcopenic obesity: a narrative review. Front. Endocrinol. 2020;11: 332; 8. Volpi E, Nazemi R, Fujita S. Muscle tissue changes with aging. Curr Opin Clin Nutr Metab Care. 2004;7(4): 405-410.  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 22  Our Antibodies Selectively Inhibit Activation of Myostatin  ActRIIB=Activin Receptor IIB; FSH=Follicle stimulating hormone; GDF-11=Growth and differentiation factor 11; TGF-β=Transforming growth factor-beta.  1. Oh SP & Li E. Genes Dev. 1997 Jul 15;11(14):1812-26; 2. Garito T, et al. Clin Endocrinol (Oxf). 2018 Jun;88(6):908-919; 3. McPherron AC et al Nat Genet 1999, 22(3):260-264.; 4. Joonho Suha et al  O B E S I T Y  ActRIIB  Myostatin  Activin A  GDF-11  Selective Targeting of Proforms of Myostatin  Promyostatin Pro-GDF-11 Proactivin  SRRK antibodies do not bind to mature myostatin or any form of GDF11, Activin A, or other TGF-β family members  Selectivity is critical to avoid  safety concerns  Apitegromab  and  SRK-439  LATENT FORMS  MATURE FORMS  Broad inhibition of ActRIIb signaling may be problematic:  ActRIIB knockout animals die shortly after birth with developmental defects in respiratory and cardiac organs1  Activins are critical in reproductive biology, and inhibition was shown to reduce FSH levels in women2  GDF11 loss leads to embryonic lethality, skeletal and kidney formation defects3  GDF11 signaling inhibition may have negative impacts on bone4, 5  Proc Natl Acad Sci U S A . 2020 Mar 3;117(9):4910-4920; 5, Ravenscroft TA et al. Genet Med 2021 Oct;23 (10):1889-1900).  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 23  SRK-439: Novel Myostatin Inhibitor  Preclinical candidate in development with potential to address muscle loss associated with weight loss  mTLL2 IC50: Inhibitory concentration at 50% as measure of in vitro potency of the antibody in its ability to inhibit the activation of latent myostatin by its activating enzyme mammalian tolloid like protease 2 | KD: Equilibrium dissociation constant as a measure of binding affinity of the antibody to latent myostatin.  O B E S I T Y  SRK-439  Promyostatin  Myostatin  Attractive Properties  High in vitro affinity  for pro- and  latent myostatin  Optimized for subcutaneous formulation and dosing  Maintained myostatin specificity (No GDF-11 or  Activin-A binding)  Maintained  good developability  profile  GDF-11=Growth and differentiation factor 11.  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 24  SRK-439 Reversed Lean Mass Loss and  Enhanced Fat Mass Loss Induced by Semaglutide Treatment†  † In Mouse Diet Induced Obesity (DIO) Model.  Figure showed the effects of increasing doses of SRK-439 in combination with semaglutide on lean mass (left panel) and fat mass (right panel) in DIO mouse model as measured by qNMR; statistical analysis was done using one-way ANOVA (Dunnett’s multiple comparison test).  O B E S I T Y  -20  -10  0  10  20  Additional Fat Mass Loss vs Semaglutide Alone  Dose-dependent Preservation of Lean Mass with effects seen as low as 0.3mg/kg  One-way ANOVA  * P < 0.05  ** P < 0.005  *** P < 0.0005  **** P < 0.0001  % Change – Fat Mass (qNMR)  % Change – Lean Mass (qNMR)  % Change  *  ****  0.1  3.0  IgG  Semaglutide 0.04 mg/kg  SRK-439 IgG  0.3 1.0  SRK-439 (mg/kg)  SRK-439  0.06 mg/kg Lira  -80  -60  -40  -20  0  20  % Change  Semaglutide 0.04 mg/kg  0.1  3.0  SRK-439  IgG  IgG  0.3 1.0  SRK-439 (mg/kg)  ANOVA=Analysis of variance; IgG=Immunoglobulin G; qNMR=Quantitative nuclear magnetic resonance.  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 25  Opportunity for Safe, Durable Weight Loss: Add Highly Selective Antimyostatin to GLP-1 RA to Preserve Lean Muscle  GLP-1 RA=Glucagon-like peptide-1 receptor agonist.  1. Pirruccello-Straub M et al. Blocking extracellular activation of myostatin as a strategy for treating muscle wasting. Sci Reports 2017;8:2922; 2. Welsh BT et al. Preclinical safety assessment and toxicokinetics of apitegromab, an antibody targeting proforms of myostatin for the treatment of muscle-atrophying disease. Int J Tox 2021;40(4):322-336; 3. Barrett D et al. A randomized phase 1 safety, pharmacokinetic and pharmacodynamic study of a novel myostatin inhibitor apitegromab (SRK-015): A potential treatment for spinal muscular atrophy. Adv Ther 2021;38:3203-3222. 4.Yang  O B E S I T Y  Myostatin Inhibition  Preclinical models demonstrated: increased muscle mass  Beneficial metabolic effects  (insulin sensitivity, basal metabolic  rate, reduction in fat mass)4  Exquisite Selectivity  Only inhibits myostatin  Avoids undesirable off-target  effects 1-3  Lean Muscle Retention  Inhibition of myostatin in combination with GLP-1 RA-driven weight loss may lead to retention of lean muscle mass and combat the counter-regulatory metabolic effects of weight loss  M et al. Myostatin: A potential therapeutic target for metabolic syndrome. Frontiers in Endocrinology 2023;14:1181913;  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 26  Expedite Cardiometabolic Program with  Ph2 Proof-of-Concept Study of Apitegromab in Obesity  * Anticipated milestones.  Creates additional anticipated milestones in next 18-24 months  O B E S I T Y  2024  2025  Spinal Muscular Atrophy  Apitegromab  SAPPHIRE  Topline Q4*  Potential Commercial Launch*  Cardiometabolic Disorders  POC study start*  POC data readout*  Apitegromab POC  APITEGROMAB + GLP-1 RA  Ph 2 proof-concept-trial  IND*  SRK-439  SRK-439 + GLP-1 RA  Novel asset for  cardiometabolic indication  GLP-1 RA=Glucagon-like peptide-1 receptor agonist; IND=Investigational new drug; POC=Proof of concept.  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 Building on this success,  in 2024  we are focused on  27  Key Accomplishments and 2024 Strategic Priorities  2023  ACCOMPLISHMENTS  COMPLETED  SAPPHIRE enrollment  EXPANDED  antimyostatin program into cardiometabolic disorders  SUCCESSFUL  $98M public offering, extending projected runway into second half of 2025  SAPPHIRE  Readout in Q4  Initiate Ph 2 POC trial with apitegromab in obesity  Advance  IND-enabling studies for SRK-439  Prepare for commercialization  IND=Investigational new drug; POC=Proof of concept;  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 © 2024 Scholar Rock, Inc. All rights reserved.  Appendix 
 

 29  Proven Expertise in Anti-Latent TFGB & Antimyostatin Inhibition  * Contingent upon receipt of regulatory approval.  IND=Investigational new drug; SMA=Spinal muscular atrophy; LTBP1=Latent transforming growth factor beta binding protein 1; LTBP3=Latent transforming growth factor beta binding protein 3; TGFβ-1=Transforming growth factor beta-1.  1.Pirruccello-Straub M et al. Blocking extracellular activation of myostatin as a strategy for treating muscle wasting. Sci Reports 2017;8:2922; 2. Welsh BT et al. Preclinical safety assessment and toxicokinetics of apitegromab, an antibody targeting proforms of myostatin for the treatment of muscle-atrophying disease. Int J Tox 2021;40(4):322-336; 3. Barrett D et al. A randomized phase 1 safety, pharmacokinetic and pharmacodynamic study of a novel myostatin inhibitor apitegromab (SRK-015): A potential treatment for spinal muscular atrophy. Adv Ther 2021;38:3203-3222.  Anti-Latent TGFβ-1  Inhibition  Antimyostatin Inhibition  Antibody discovery technology and deep structural insights  FOUNDATION FOR SUCCESS  SRK-439 in Obesity  Novel highly selective antimyostatin to preserve lean muscle & avoid undesirable off-target effects1-3  IND-enabling studies in 2024  File IND in 2025  Apitegromab in SMA  In Ph 3 with potential to be first muscle-targeted treatment to advance the standard of care  SAPPHIRE data in Q4 2024  Commercial launch 2025*  SRK-181 in Immuno-oncology  Recent for SRK-181 data supports proof of concept and validates scientific hypothesis of selective targeting  Fibrosis  Selective context-dependent (LTBP1 & LTBP3) anti-latent TGFβ-1  NEXT HORIZON  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 -10  -5  0  5  10  -60  -40  -20  0  20  40  60  30  SRK-439 Maintained Lean Mass When Combined with GLP-1 RA Therapy†  ANOVA=Analysis of variance; GLP-1 RA=Glucagon-like peptide-1 receptor agonist; IgG=Immunoglobulin G; qNMR=Quantitative nuclear magnetic resonance.  † In Mouse Diet Induced Obesity (DIO) Model.  Figure shows the effects of increasing doses of SRK-439 in combination with liraglutide on lean mass (left panel) and fat mass (right panel) in a 28-day DIO mouse model as measured by qNMR; statistical analysis was done using one-way ANOVA (Dunnett’s multiple comparison test).  O B E S I T Y  Improved Fat Mass Loss vs GLP-1 RA Alone  Increased Lean Mass Gain vs GLP-1 RA Alone  % Change Fat Mass from Baseline (qNMR)  % Change Lean Mass from Baseline (qNMR)  % Change  IgG  0.3  1  3  SRK-439  IgG  0.06 mg/kg Lira  % Change  IgG  0.3  1  3  SRK-439  IgG  0.06 mg/kg Lira  © 2024 Scholar Rock, Inc. All rights reserved.  ****  ****  ****  One-way ANOVA  * P < 0.05  ** P < 0.005  *** P < 0.0005  **** P < 0.0001 
 

 SRK-181: Targeting Latent TGFB1 to Overcome Immunotherapy Resistance  PD-1=Programmed cell death ligand 1; TGFβ=Transforming growth factor-beta; ccRCC=Clear cell renal cell carcinoma  1.Sumanta Kumar Pal et al. Atezolizumab plus cabozantinib versus cabozantinib monotherapy for patients with renal cell carcinoma after progression with previous immune checkpoint inhibitor treatment (CONTACT-03): a multicentre, randomised, open-label, phase 3 trial. The Lancet, Volume 402, Issue 10397, 2023, Pages 185-195, https://doi.org/10.1016/S0140-6736(23)00922-4  S R K - 181   Enrollment completed December 2023  NEXT  STEPS  Present ongoing emerging data at future medical meetings  Conduct an end of Phase 1 meeting with regulatory authorities to inform next steps  Differentiation  Monoclonal antibody selectively targeting latent and context-independent binding to TGFβ1  Novel and highly selective inhibition of  TGFβ-1 targeting latent form  Offers potential to avoid toxicity and dose-limiting challenges of non-selective TGFβ inhibition approaches  Ph1 DRAGON Demonstrated Proof- of-Concept in ccRCC patients  Showed objective, durable clinical responses above  what is expected from continuing PD-1 alone1  Biomarker data supports proof-of-mechanism in multiple tumor types  31  PD-1/PD-L1)  © 2024 Scholar Rock, Inc. All rights reserved. 
 

 32  Promising Anti-tumor Activity in Heavily Pretreated ccRCC Patients;  Biomarker Data Supports Proof of Mechanism Across Multiple Tumor Types  ccRCC=Clear cell renal cell carcinoma  Data cutoff August 29, 2023  CD8 Stain – Melanoma  Pre-Treatment  Continued Tolerability &  Promising Anti-Tumor Activity in ccRCC Patients*  Post-Treatment  Best Response in Target Lesions  100  80  60  40  20  0  -20  -40  -60  -80  -100  Percent Change from Baseline  Pt #6  Pt #3  Pt #5  Pt #4  Pt #1  Pt #2  Change in Tumor Volume Over Time  100  80  60  40  20  0  -20  Percent Change from Baseline  -40  -60  -80  -100  0  2 4 6 8 10 12 14  Months on SRK-181 + Pembrolizumab Treatment  Pt #1  Pt #2  Pt #4  Pt #5  Pt #3  Pt #6  S R K - 181   *28 patients  © 2024 Scholar Rock, Inc. All rights reserved. 
 
Exhibit 99.2

Scholar Rock Provides Corporate Update and Highlights Priorities for 2024

- Advancing industry-leading antimyostatin pipeline, comprised of multiple, novel assets with unparalleled selectivity, to treat spinal muscular atrophy (SMA) and cardiometabolic disorders

- Completed enrollment for apitegromab pivotal Phase 3 SAPPHIRE trial in patients with SMA; topline data anticipated in 4Q 2024

- Apitegromab Phase 2 proof-of-concept trial in obesity expected to commence in mid-2024

- Presenting new preclinical data on SRK-439, a novel investigational myostatin inhibitor for the treatment of obesity, at Keystone Symposia in February

- Scholar Rock reports year-end cash and cash equivalents of approximately $280 million

- Presenting at the 42nd Annual J.P. Morgan Healthcare Conference on Tuesday, January 9, 2024 at 1:30 p.m. PT (4:30 p.m. ET)

CAMBRIDGE, Mass.--(BUSINESS WIRE)--January 4, 2024--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today provided recent corporate updates and highlighted upcoming priorities for 2024.

“We were pleased with our progress in 2023 as we advanced our industry leading antimyostatin pipeline with apitegromab and SRK-439 and have great momentum heading into 2024. With enrollment completed for our Phase 3 SAPPHIRE trial for patients with spinal muscular atrophy, we expect topline data in Q4 this year,” said Jay Backstrom, M.D., MPH, President and Chief Executive Officer of Scholar Rock. “Additionally, we are thrilled with the planned initiation in mid-2024 of an apitegromab Phase 2 proof-of-concept trial in patients with obesity and on GLP-1 therapies that aims to establish the importance of selective, safe myostatin inhibition to preserve lean muscle mass as part of healthy weight loss.”

2024 Priorities:

SPINAL MUSCULAR ATROPHY

Apitegromab is an investigational, fully human monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle and is being developed as a potential first muscle-targeted therapy for the treatment of SMA. Apitegromab is the only muscle-targeted therapy to show clinical proof-of-concept in SMA.

  • Planning to announce Phase 3 SAPPHIRE clinical trial topline data in 4Q 2024. SAPPHIRE is a randomized, double-blind, placebo-controlled clinical trial evaluating apitegromab in patients with nonambulatory Types 2 and 3 SMA on either nusinersen or risdiplam. If the trial is successful and apitegromab is approved, the Company expects to initiate a commercial product launch in 2025.
  • Continue to progress the ONYX long-term extension study for patients from both the TOPAZ and SAPPHIRE studies.

CARDIOMETABOLIC DISORDERS

SRK-439 is a novel, preclinical, investigational myostatin inhibitor that has high in vitro affinity for pro- and latent myostatin and maintains myostatin specificity (i.e., no GDF11 or Activin-A binding), and is initially being developed for the treatment of obesity.

  • Initiating a Phase 2 proof-of-concept trial with apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA) in obesity in mid-2024. As part of the Company’s strategy to advance the development of SRK-439, it plans to initiate a Phase 2 proof-of-concept trial with apitegromab in combination with a GLP-1 RA, subject to IND clearance. Data from the clinical trial are expected in mid-2025 and will be used to guide clinical development of SRK-439. The Company plans to file an IND for SRK-439 for the treatment of obesity in 2025.
  • Presenting preclinical SRK-439 data in a poster presentation at Keystone Symposia at the Obesity: Causes and Consequences meeting on February 5, 2024, in Vancouver, BC, Canada.

2023 Highlights:

  • Completed enrollment of apitegromab pivotal Phase 3 SAPPHIRE trial.
  • Presented TOPAZ 36-month extension trial data at the Cure SMA Research & Clinical Care Meeting in June, and at the 28th Annual Congress of the World Muscle Society in October, which showed long-term substantial and sustained improvements in motor function and patient-reported outcome measures in patients with nonambulatory Types 2 and 3 SMA receiving survival motor neuron (SMN) therapy.
  • Initiated the ONYX trial, a long-term extension study for patients from both the TOPAZ and SAPPHIRE studies, which remains ongoing.
  • Announced plans to expand into cardiometabolic disorders with SRK-439, starting with a Phase 2 proof-of-concept trial evaluating apitegromab in obesity to inform development of SRK-439.
  • Presented SRK-181 Phase 1 DRAGON trial clinical and biomarker data at the SITC 38th Annual Meeting, which showed favorable tolerability and promising anti-tumor activity in heavily pretreated patients with clear cell renal cell carcinoma (ccRCC) resistant to anti-PD-1. The Company believes these data support proof-of-concept and completed enrollment of the DRAGON trial in December. The Company will provide additional clinical data updates as they become available in 2024.
  • Completed an equity financing of $98 million in October. As of December 31, 2023, Scholar Rock reported cash, cash equivalents, and marketable securities of approximately $280 million, which is projected to fund the Company’s operations into the second half of 2025.

“We are excited to enter 2024 with several important near-term milestones ahead of us. As the leader in selective myostatin inhibition, we believe we are well positioned to deliver significant value to patients living with spinal muscular atrophy and the millions of people suffering from the wide array of health challenges stemming from cardiometabolic and obesity disorders. We are highly encouraged by our potential to advance the standard of care where muscle-targeted therapies can play a role in addressing unmet patient needs,” said Ted Myles, Chief Operating Officer and Chief Financial Officer.

J.P. Morgan Healthcare Conference Presentation and Webcast

Scholar Rock management will highlight these updates in a corporate presentation at the 42nd Annual J.P. Morgan Healthcare Conference on Tuesday, January 9, 2024, at 1:30 p.m. PT (4:30 p.m. ET). A live webcast of the presentation may be accessed by visiting the Investors & Media section of the Scholar Rock website at http://investors.scholarrock.com. An archived replay of the webcast will be available on the Company’s website for approximately 90 days following the presentation.

About Scholar Rock

Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need. As a global leader in the biology of the transforming growth factor beta (TGFβ) superfamily of cell proteins and named for the visual resemblance of a scholar rock to protein structures, the clinical-stage company is focused on advancing innovative treatments where protein growth factors are fundamental. Over the past decade, Scholar Rock has created a pipeline with the potential to advance the standard of care for neuromuscular disease, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted drugs can play a transformational role.

Scholar Rock is the only company to show clinical proof-of-concept for a muscle-targeted treatment in spinal muscular atrophy (SMA). This commitment to unlocking fundamentally different therapeutic approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. By harnessing cutting-edge science in disease spaces that are historically under-addressed through traditional therapies, Scholar Rock works every day to create new possibilities for patients. Learn more about our approach at ScholarRock.com and follow @ScholarRock and on LinkedIn.

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 Twitter and LinkedIn. The information that we post on our website or on Twitter 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.

Scholar Rock® is a registered trademark of Scholar Rock, Inc.

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, results and timing of its clinical trials for apitegromab and SRK-181 and its preclinical programs, including SRK-439, regulatory feedback including with respect to the IND submitted in connection with the planned Phase 2 trial of SRK-439 in combination with GLP-1 RAs in obesity, and indication selection and development timing, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, its expected cash and cash equivalents as of December 31, 2023 and cash runway, expectations regarding 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,” “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, that preclinical and clinical data, including the results from the Phase 2 clinical trial of apitegromab, or Part A or Part B of the Phase 1 clinical trial of SRK-181, 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, including, without limitation, the Phase 3 clinical trial of apitegromab in SMA or Part B of the Phase 1 clinical trial of SRK-181; Scholar Rock’s ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on their expected timelines; the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials; 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; 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 and establish and maintain strategic business alliances and new business initiatives, and the impacts of public health pandemics such as COVID-19 on business operations and expectations, as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, 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.

Contacts

Investors & Media
Rushmie Nofsinger
Scholar Rock
rnofsinger@scholarrock.com
ir@scholarrock.com
857-259-5573