Form 8-K - Current report

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): August 8, 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	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 August 8, 2024, Scholar Rock Holding Corporation (the “Company”) issued a press release announcing its financial and operating results for the quarter ended June 30, 2024. 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 8.01. Other Events.

A copy of the Company’s current corporate slide presentation is being filed herewith as Exhibit 99.2 to this Report on Form 8-K and is incorporated herein by reference. The Company undertakes no obligation to update, supplement or amend the materials attached hereto as Exhibit 99.2.

Item 9.01. Financial Statements and Exhibits.

(d) Exhibits


Exhibit No.			Description

99.1			Press Release issued by the Company on August 8, 2024, furnished hereto.
99.2			Presentation Slide Deck.
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: August 8, 2024	By:	/s/ Junlin Ho
		Junlin Ho
		General Counsel and Corporate Secretary

Exhibit 99.1

Scholar Rock Reports Second Quarter 2024 Financial Results and Highlights Business Progress

Remains on track to report topline data from pivotal Phase 3 SAPPHIRE trial in patients with Spinal Muscular Atrophy (SMA) in 4Q 2024

New data from Phase 2 TOPAZ extension study in patients with nonambulatory SMA showed sustained clinical benefit over 48 months, a continued favorable safety profile with no new safety findings; patient retention rate of over 90%

Phase 2 EMBRAZE proof-of-concept trial enrolling ahead of schedule, topline data expected in 2Q 2025

Presented new SRK-439 preclinical data at American Diabetes Association’s 84^th Scientific Sessions (ADA) supporting the potential to contribute to a favorable body composition; increased lean mass and reduced fat mass regain following withdrawal from GLP-1 receptor agonist treatment

Management to host update call today at 8:15 a.m. ET

CAMBRIDGE, Mass.— (BUSINESS WIRE)— August 8, 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 reported financial results and corporate updates for the second quarter ended June 30, 2024.

“Scholar Rock continues to execute across our portfolio of highly selective myostatin inhibition programs, further cementing our position as the global leader in harnessing the life-changing potential of TGF-beta superfamily biology,” said Jay Backstrom, M.D., MPH, President & Chief Executive Officer of Scholar Rock. “With the only muscle-targeted program to demonstrate clinical proof-of-concept in SMA, our confidence in our lead program apitegromab continues to be supported by the clinical data generated over the past four years. At 48 months, over 90% of TOPAZ patients with nonambulatory SMA remained on apitegromab treatment on top of SMN therapy and we continued to observe sustained clinical benefit. We look forward to reporting topline data from the Phase 3 SAPPHIRE trial of apitegromab in SMA in the fourth quarter of this year.”

Dr. Backstrom continued, “In addition, we are pleased with the progress of our cardiometabolic program. The enrollment of the Phase 2 proof-of-concept EMBRAZE study evaluating apitegromab in obesity has been advancing ahead of schedule and as a result, we are updating our guidance for topline data to the second quarter of 2025. We also presented new preclinical data supporting SRK-439’s potential to help patients retain lean muscle mass at our investor event in May and at the ADA 84^th Scientific Sessions in June. The hallmark of our approach in designing both apitegromab and SRK-439 is the exquisite selectivity for pro- and latent forms of myostatin. Our data in SMA suggest that this selectivity matters for patients, and we are excited to show how SRK-439 can become an integral component of the treatment and management of obesity helping to preserve lean muscle mass for sustainable and healthy weight loss management.”

Company Highlights and Upcoming Milestones

SMA Program

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.

●

On track to report topline data from Phase 3 SAPPHIRE clinical trial in 4Q 2024. If the trial is successful and apitegromab is approved, the Company expects to initiate a commercial product launch in 2025.

●

Reported that long-term apitegromab data continued to show substantial and sustained motor function improvements over 48 months¹. The mean change in Hammersmith Functional Motor Scale (HFMSE) from baseline in nonambulatory patients (ages 2-21) on combination therapy (nusinersen and 20 mg/kg of apitegromab) was 5.3 points (95% CI: 1.5, 9.2; n=23), and for patients 2-12 was 6.4 points (95% CI: 1.8, 11.0, n=19). The mean change in RULM for the 2-21 age group was 3.6 points (95% CI: 2.0, 5.3; N=22) and for the 2-12 age group was 4.5 (95% CI: 2.7, 6.3; n=18). Of the 35 participants in the pooled nonambulatory population, 33 remained in the study over 4 years. The data analysis excluded the scores of 11 patients after undergoing scoliosis surgery, a known confounding factor for motor function assessment. Additional details will be discussed on the conference call this morning.

12-Month Data

24-Month Data

36-Month Data

48-Month Data

Age 2-21 Years Mean Change from Baseline in HFMSE (95% Confidence Interval)

3.6 points

(1.2, 6.0)

n=32

4.2 points

(1.9, 6.6)

n=29

4.0 points

(1.0, 6.9)

n=28

5.3 points

(1.5, 9.2)

n=23

Age 2-12 Years Mean Change from Baseline in HFMSE (95% Confidence Interval)

4.6 points

(1.8, 7.4)

n=26

5.2 points

(2.3, 8.0)

n=23

4.8 points

(1.3, 8.3)

n=23

6.4 points

(1.8, 11.0)

n=19

Age 2-21 Years Mean Change from Baseline in

RULM (95% Confidence Interval)

1.3 points

(0.2, 2.3)

n=31

2.3 points

(1.2, 3.3)

n=31

2.4 points

(1.1, 3.7)

n=27

3.6 points

(2.0, 5.3)

n=22

Age 2-12 Years Mean Change from Baseline in

RULM (95% Confidence Interval)

1.2 points

(0.1, 2.4)

n=25

2.2 points

(1.0, 3.5)

n=25

2.8 points

(1.4, 4.2)

n=22

4.5 points

(2.7, 6.3)

n=18

For the 48-month evaluation, an observed case analysis was conducted using available data by analysis timepoint, censoring any HFMSE and RULM assessments after the patient received scoliosis surgery. The analysis population pooled the nonambulatory patients (Cohorts 2 and 3) and included patients receiving either low dose (2 mg/kg) or high dose (20 mg/kg) apitegromab (inclusive of patients in Cohort 3 who switched from 2 mg/kg to 20 mg/kg in Year 2). A total of 11 patients in the population had scoliosis surgery during the study and their data was excluded from any HFMSE or RULM assessments at 48 months. Visit windows were applied to utilize data from unscheduled or early termination visits if the patient was missing the HFMSE or RULM total score at the scheduled visit.

●

The ONYX open-label, multicenter extension study is ongoing. The extension study is evaluating the long-term safety and efficacy of apitegromab in patients with Type 2 and Type 3 SMA who completed the TOPAZ or SAPPHIRE trials. More than 90 percent of patients on combination therapy in the TOPAZ study have completed 4 years of apitegromab treatment and enrolled into ONYX.

Cardiometabolic Program

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.

●

Presented preclinical data from the SRK-439 program. In May, the Company announced new preclinical data comparing SRK-439 and an anti-activin receptor II (anti-ActRII) antibody that supported SRK-439’s potential as best in class in preserving lean muscle mass in patients on GLP-1 receptor agonists (GLP-1 RAs). In June, the company presented new preclinical data at the American Diabetes Association 84^th Scientific Sessions supporting the potential of SRK-439 to increase lean mass and contribute to a favorable body composition following withdrawal from GLP-1 RA treatment.

●

Initiated Phase 2 EMBRAZE proof-of-concept trial with apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA) in obesity in May. The Phase 2 trial is a randomized, double-blind, placebo-controlled, multi-center study to evaluate the effect of apitegromab, a highly selective investigational myostatin inhibitor, to preserve muscle mass as an adjunctive therapy in overweight and obese adults who are taking a GLP-1 RA. Data are expected in the second quarter of 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.

Other Pipeline Updates

●

New SRK-181 data from the Phase 1 DRAGON proof-of-concept trial presented at the ASCO Annual Meeting in June. SRK-181 is an investigational selective inhibitor of latent TGFβ-1 activation and developed with the aim of overcoming resistance to checkpoint therapy in patients with advanced cancer. Clinical data showed encouraging responses in heavily pretreated and anti-PD-(L)1 resistant patients across multiple tumor types. Enrollment of the DRAGON trial was completed in December 2023, and patients who remain on the study continue to be treated.

●

Published data on SRK-373 was featured on the cover of Science Signaling in July. The article describes the selectivity of SRK-373 for LTBP-presented TGFβ-1, as well as efficacy data from two preclinical models that establish the feasibility of selectively targeting this particular form of TGFβ-1 for the treatment of fibrosis. SRK-373 is an investigational selective inhibitor of matrix associated TGFβ-1 in development for the treatment of fibrosis.

Second Quarter 2024 Financial Results

For the quarter ended June 30, 2024, net loss was $58.5 million or $0.60 per share compared to a net loss of $37.9 million or $0.47 per share for the quarter ended June 30, 2023.

●

The Company did not record any revenue for the quarter ended June 30, 2024 or for the quarter ended June 30, 2023.

●

Research and development expense was $42.4 million for the quarter ended June 30, 2024, compared to $26.9 million for the quarter ended June 30, 2023. The increase was primarily attributable to clinical trial and employee compensation costs.

●

General and administrative expense was $17.1 million for the quarter ended June 30, 2024, compared to $12.2 million for the quarter ended June 30, 2023. The increase was due to employee-related costs.

●

As of June 30, 2024, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $190.5 million, which is expected to fund the Company’s anticipated operating and capital expenditure requirements into the second half of 2025.

“Our year-to-date progress across our pipeline of industry-leading myostatin inhibition programs, combined with our highly experienced and disciplined team, provides us with a robust foundation for growth as we advance towards multiple milestones and our potential evolution into a commercial-stage company,” said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock.

Conference Call Information
Management will provide an update on the Company and discuss second quarter 2024 results via conference call on Thursday, August 8 at 8:15 am ET. To access the live conference call, participants may register here. The live audio webcast of the call will be available under “Events and Presentations” in the Investor Relations 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, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. An archived replay of the webcast will be available on the Company’s website for approximately 90 days.

About Apitegromab

Apitegromab is an investigational fully human monoclonal antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin in the skeletal muscle. It is the first muscle-targeted treatment candidate to demonstrate clinical proof-of-concept in spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. Scholar Rock believes that its highly selective targeting of pro- and latent forms of myostatin with apitegromab may lead to a clinically meaningful improvement in motor function in patients with SMA. The U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. The efficacy and safety of apitegromab have not been established and apitegromab has not been approved for any use by the FDA or any other regulatory agency.

About the Phase 3 SAPPHIRE Trial

SAPPHIRE is an ongoing randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 SMA who are receiving SMN-targeted therapy (either nusinersen or risdiplam). SAPPHIRE targeted enrolling approximately 156 patients aged 2-12 years old in the main efficacy population. These patients were randomized 1:1:1 to receive for 12 months either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks. An exploratory population that targeted enrolling up to 48 patients aged 13-21 years old will also separately be evaluated. These patients were randomized 2:1 to receive either apitegromab 20 mg/kg or placebo. For more information about SAPPHIRE, visit www.clinicaltrials.gov. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established.

About EMBRAZE

EMBRAZE is a randomized, double-blind, placebo-controlled, Phase 2 proof-of-concept trial evaluating the efficacy, safety and pharmacokinetics of apitegromab in adults with a body mass index (BMI) of >27 (overweight) or a BMI of >30 (obese) and taking a GLP-1 RA (tirzepatide or semaglutide). The target enrollment of EMBRAZE is 100 subjects aged 18-65 who are overweight or obese without diabetes. As part of the study design, the treatment period is 24 weeks, and all subjects will receive a GLP-1 RA. In addition, all subjects will be randomized 1:1 to receive either apitegromab or placebo by intravenous (IV) infusion every four weeks during the 24-week treatment period. The primary endpoint is change from baseline at Week 24 in lean mass assessed by dual-energy X-ray absorptiometry. Secondary endpoints include additional weight loss measures, safety and tolerability, and pharmacokinetic outcomes. Exploratory endpoints at Weeks 24 and 32 include cardiometabolic parameters (e.g. HbA1c), body composition, and physical function.

About SRK-439

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 cardiometabolic disorders, including obesity. Based on preclinical data, SRK-439 has the potential to

support healthier weight management by preserving lean mass during weight loss. The efficacy and safety of SRK-439 have not been established and SRK-439 has not been approved for any use by the FDA or any other regulatory agency.

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 and timing of its clinical trials for apitegromab and SRK-181 and its preclinical programs, including SRK-439, 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 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 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; Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials; and 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 our ability to continue as a going concern; 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 June 30, 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.

Scholar Rock Holding Corporation

Condensed Consolidated Statements of Operations

(unaudited)

(in thousands, except share and per share data)


	Three Months Ended June 30,				Six Months Ended June 30,
	2024		2023		2024		2023

Operating expenses
Research and development	$	42,373	$	26,867	$	85,466	$	56,602
General and administrative		17,125		12,215		32,451		22,989
Total operating expenses		59,498		39,082		117,917		79,591
Loss from operations		(59,498)		(39,082)		(117,917)		(79,591)
Other income (expense), net		990		1,157		2,556		2,287
Net loss	$	(58,508)	$	(37,925)	$	(115,361)	$	(77,304)
Net loss per share, basic and diluted	$	(0.60)	$	(0.47)	$	(1.20)	$	(0.97)
Weighted average common shares outstanding, basic and diluted		96,813,116		80,117,983		96,352,858		79,865,424

Scholar Rock Holding Corporation

Condensed Consolidated Balance Sheets

(unaudited)

(in thousands)


	June 30, 2024		December 31, 2023
Assets
Cash, cash equivalents and marketable securities	$	190,494	$	279,938
Other current assets		8,643		8,256
Total current assets		199,137		288,194
Other assets		27,728		22,841
Total assets	$	226,865	$	311,035

Liabilities and Stockholders' Equity
Current liabilities	$	32,987	$	32,741
Long-term liabilities		60,258		53,076
Total liabilities		93,245		85,817
Total stockholders' equity		133,620		225,218
Total liabilities and stockholders' equity	$	226,865	$	311,035

Scholar Rock:

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

Media
Molly MacLeod
Scholar Rock
mmacleod@scholarrock.com
media@scholarrock.com
802-579-5995

Exhibit 99.2

© 2024 Scholar Rock, Inc. All rights reserved. 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 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 our ability to continue as a going concern 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, 2023, and Quarterly Report on Form 10-Q for the quarter ended June 30, 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 and SRK-181 are investigational drug candidates under evaluation. Apitegromab, SRK-181, SRK-373, SRK-256, 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, SRK-373, SRK-256, and SRK-439 have not been established.

© 2024 Scholar Rock, Inc. All rights reserved. Today’s Agenda 4 Topic Speaker  Introduction & Business Update Jay Backstrom, President & Chief Executive Officer  Development Update Jing Marantz, Chief Medical Officer  Pipeline Update Mo Qatanani, Chief Scientific Officer  Upcoming Milestones Jay Backstrom Q&A Session

© 2024 Scholar Rock, Inc. All rights reserved. Selectivity is the Key The hallmark of our differentiated platform is unparalleled selectivity 1 Productive Platform Strong progress & momentum supports SRRK’s scientific approach, capability to grow pipeline and ability to execute 2 Excellent Opportunity SMA and obesity represent high value markets offering significant potential revenue opportunities 3 Advancing Towards Commercialization 5 I N TR O D UC T IO N Next 12 – 24 months of execution is expected to be transformative for Scholar Rock

© 2024 Scholar Rock, Inc. All rights reserved. Traditional Target “mature” active growth factor Scholar Rock’s Target L Latent Growth Factor Complex 6 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 RI GHT TI ME L a t e n t F o r m RI GHT TA RGE T V a l i d a t e d B i o l o g y

© 2024 Scholar Rock, Inc. All rights reserved. Scientific Platform Yielding Growing Pipeline Across High Value Therapeutic Areas 7 *Utilized data from 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. TARGET CANDIDATE DISCOVERY/ PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Latent Myostatin SPINAL MUSCULAR ATROPHY Apitegromab CARDIOMETABOLIC DISORDERS Apitegromab in Obesity* SRK-439 (novel anti-myostatin antibody) Latent TGFβ-1 IMMUNO-ONCOLOGY SRK-181: Selective context-independent, anti-latent TGFβ-1) FIBROSIS SRK-373: Selective context-dependent (LTBP1 & LTBP3) anti-latent TGFβ-1 RGMc ANEMIA SRK-256: Selective anti-RGMc Undisclosed NEUROMUSCULAR DISORDERS

Upcoming SAPPHIRE readout - Q4 2024 Initiate study in SMA patients < 2 years old: Planned for 2025 Exploring additional neuromuscular populations Ph 2 POC EMBRAZE study enrolling: Topline data expected in Q2 2025 Advancing SRK-439 to IND Established PoC with SRK-181 in multiple advanced solid tumors End of Ph 1 meeting planned Advance SRK-373, LTBP, to IND Advancing nonclinical studies in renal and pulmonary fibrosis High Value Growth Opportunities 8 Neuromuscular Disorders Cardiometabolic Disorders Immuno-Oncology Fibrosis Strong Proprietary Platform

© 2024 Scholar Rock, Inc. All rights reserved. 9 Cutting-Edge Research Recognized by Global Scientific Community Promising objective response rates, safety, and novel biomarker data highlighted in oral presentation at ASCO SRK-181 Featured on the cover of Science Signaling and in Focus article: SRK-373 “More Velcro for the TGFB-1 Straightjacket” by Boris Hinz Compelling new preclinical data highlighted in oral presentation at ADA SRK-439

© 2024 Scholar Rock, Inc. All rights reserved. 10 2024 & 2025 Anticipated Milestones Milestones 2024 2025 SRK-181 data at ASCO • Oral presentation June 3 • Developmental Therapeutics-Immunotherapy SRK-439 data at American Diabetes Association • Oral presentation June 23 • New Insights into Therapeutic Strategies for Obesity and Diabetes SRK-439 IND submission EMBRAZE Ph 2 Trial (apitegromab in obesity) • Trial open for enrollment • Topline data expected in Q2 2025 SAPPHIRE Ph 3 Trial (apitegromab in SMA) • Topline readout expected in Q4 2024 Potential SMA launch in Q4 2025, if successful & approved Study in SMA Patients < 2 Years of Age • Study design endorsed by EMA’s paediatric committee • Study initiation planned for 2025

© 2024 Scholar Rock, Inc. All rights reserved. 12 Why We Are Confident A P I T E GR O M A B F O R S P I NA L M U S C U L A R A T RO P H Y Selective muscle-targeting designed to improve motor function while minimizing off-target effects >90% patient retention,1 well tolerated profile supports durability of treatment 1. Muscle Targeting 4. TOPAZ Safety Profile Trial design informed by insights from TOPAZ 3. SAPPHIRE Optimized for Success TOPAZ clinical proof-of-concept with substantial and durable effect across broad SMA patients ages 2-21 2. Clinical Proof-Of-Concept 1 Pooled nonambulatory patients

© 2024 Scholar Rock, Inc. All rights reserved. S PI N AL M U S C U LA R A T R O P HY 13 Finkel RS et al. “Final Safety and Efficacy Data From the SHINE Study in Participants With Infantile-Onset and Later-Onset SMA.” Presented at Cure SMA Annual Conference, July 2024 CHERISH Study Baseline Nusinersen Control N 84 42 Age at Screening 4 years 3 years ▪ Decline in motor function is influenced by scoliosis surgery and contractures (Wolfe 2024; Dunaway 2020; Salazar 2018) ▪ TOPAZ participants had ~2 years of nusinersen treatment at baseline ▪ Blue-shaded area represents similar duration of nusinersen treatment Time Period Similar To TOPAZ study Motor function slowly declines despite following initial increase CHERISH/SHINE study: Long-Term Results of Nusinersen-Treated Patients

© 2024 Scholar Rock, Inc. All rights reserved. 3.4 4.6 5.2 4.8 6.4 0 1 2 3 4 5 6 7 8 9 Baseline 6 months 12 months 24 months 36 months 48 months Apitegromab TOPAZ Clinical Trial: Motor Function Outcomes by HFMSE Over 48 Months Improvements Were Substantial and Sustained 1. For the 48-month evaluation, an observed case analysis was conducted using available data by analysis timepoint, censoring any HFMSE assessments after the patient received scoliosis surgery. The analysis population pooled the nonambulatory patients (Cohorts 2 and 3) and included patients receiving either low dose (2 mg/kg) or high dose (20 mg/kg) apitegromab (inclusive of patients in Cohort 3 who switched from 2 mg/kg to 20 mg/kg in Year 2). A total of 11 patients in the population had scoliosis surgery during the study and their data was excluded from any HFMSE assessments at 48 months. Visit windows were applied to utilize data from unscheduled or early termination visits if the patient was missing the HFMSE total score at the scheduled visit. Error bars represent standard error (SE) and CI represents confidence interval. SMN Rx=SMN therapy. Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established. Pooled Nonambulatory Patients1 Mean Change from Baseline (+SE) Mean Change from Baseline (+SE) S PI N AL M U S C U LA R A T R O P HY 2.8 3.6 4.2 4.0 5.3 0 1 2 3 4 5 6 7 8 9 Baseline 6 months 12 months 24 months 36 months 48 months n= 35 29 32 29 28 23 95% CI= (1.4, 4.1) (1.2, 6.0) (1.9, 6.6) (1.0, 6.9) (1.5, 9.2) n= 29 23 26 23 23 19 95% CI= (1.8, 5.0) (1.8, 7.4) (2.3, 8.0) (1.3, 8.3) (1.8, 11.0) Baseline mean age=7.3 | Time on SMN Rx=24.1m Baseline mean age=5.5 | Time on SMN Rx=24.6m Age 2-21 Years All Doses (N=35) Age 2-12 Years All Doses (N=29) 14

© 2024 Scholar Rock, Inc. All rights reserved. 0.2 1.2 2.2 2.8 4.5 0 1 2 3 4 5 6 Baseline 6 Months 12 Months 24 Months 36 Months 48 Months 0.6 1.3 2.3 2.4 3.6 0 1 2 3 4 5 6 Baseline 6 Months 12 Months 24 Months 36 Months 48 Months Apitegromab TOPAZ Clinical Trial: Motor Function Outcomes by RULM Over 48 Months Improvements Were Substantial and Sustained 1. For the 48-month evaluation, an observed case analysis was conducted using available data by analysis timepoint, censoring any RULM assessments after the patient received scoliosis surgery. The analysis population pooled the nonambulatory patients (Cohorts 2 and 3) and included patients receiving either low dose (2 mg/kg) or high dose (20 mg/kg) apitegromab (inclusive of patients in Cohort 3 who switched from 2 mg/kg to 20 mg/kg in Year 2). A total of 11 patients in the population had scoliosis surgery during the study and their data was excluded from any RULM assessments at 48 months. Visit windows were applied to utilize data from unscheduled or early termination visits if the patient was missing the RULM total score at the scheduled visit. One patient did not have baseline RULM due to young age. Error bars represent standard error (SE) and CI represents confidence interval. SMN Rx=SMN therapy. Apitegromab is an investigational drug candidate being evaluated for the treatment of spinal muscular atrophy. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established. Pooled Nonambulatory Patients1 Mean Change from Baseline (+SE) n= 34 28 31 31 27 22 95% CI= (-0.2, 1.4) (0.2, 2.3) (1.2, 3.3) (1.1, 3.7) (2.0, 5.3) n= 28 22 25 25 22 18 95% CI= (-0.7, 1.1) (0.1, 2.4) (1.0, 3.5) (1.4, 4.2) (2.7, 6.3) S PI N AL M U S C U LA R A T R O P HY Baseline mean age=7.3 | Time on SMN Rx=24.1m Baseline mean age=5.5 | Time on SMN Rx=24.6m Age 2-21 Years All Doses (N=35) Age 2-12 Years All Doses (N=29) 15 Mean Change from Baseline (+SE)

© 2024 Scholar Rock, Inc. All rights reserved. Substantial and Sustained Improvement over 48 MONTHS1 1- A total of 11 patients in the population had scoliosis surgery during the study and their data was excluded from any HFMSE and RULM assessments at 48 months. PRO=Patient Reported Outcome *Pooled non-ambulatory cohorts Data to date has shown substantial clinical benefit that is dose-dependent TOPAZ data suggest that apitegromab has the potential to transform care in SMA by directly addressing progressive muscle weakness Clinical benefit continued to improve or was sustained over 48 months Consistency across functional scales and patient-reported outcomes Well tolerated profile and low discontinuation rate supports durability of treatment >90% of patients remain on therapy* Summary of TOPAZ Data 16

© 2024 Scholar Rock, Inc. All rights reserved. SAPPHIRE Trial Designed for Clinical Success 17 Randomized, double-blind, placebo-controlled, parallel arm design (n=204) Enrolling patients who are on SMN-directed therapy (nusinersen or risdiplam) Completed enrollment in Q3 2023 TREATMENT (52 weeks) Apitegromab (20 mg/kg IV q4w) + SMN-directed therapy Apitegromab (10 mg/kg IV q4w) + SMN-directed therapy Placebo (IV q4w) + SMN-directed therapy SCREENING MAIN POPULATION (n=156) Ages 2-12 With nonambulatory Types 2 and 3 SMA N=52 N=52 N=52 Stratified to ensure balanced allocation R across the three arms: 1. Age at SMN therapy initiation (age < 5 vs. age ≥ 5) 2. SMN therapy (nusinersen vs. risdiplam) ENDPOINTS Primary Efficacy: Mean HFMSE change from baseline at 12 months Additional Efficacy Measures: RULM, WHO, other outcome measures Safety, PK/PD, ADA Additional Data Opportunities Exploratory population (age 13-21), in patients using SMN therapy Focused upon safety & exploratory efficacy (n=48; 2:1 randomization between apitegromab 20 mg/kg vs placebo) Separate open-label extension study (after patients complete 12-month treatment period) Focused upon safety & exploratory long-term efficacy ClinicalTrials.gov Identifier: NCT05156320 HFMSE=Hammersmith Functional Motor Scale Expanded; RULM=Revised Upper Limb Module; R=randomization; SMA=spinal muscular atrophy; SMN=survival motor neuron. S PI N AL M U S C U LA R A T R O P HY

© 2024 Scholar Rock, Inc. All rights reserved. Goals of the EMBRAZE Proof-of-Concept Study C A R D I O M E T A B O L I C D E V E L O P M E NT P R O G R A M IN SI GHT S GA INE D F R OM E MB R A ZE S T UD Y to inform SRK-439 development Study Aims to Demonstrate O B E S I T Y Effect of apitegromab to preserve lean mass in obese or overweight patients receiving a GLP-1 agonist Safety and tolerability data to provide initial support for long-term chronic use Explore the potential effect of apitegromab to improve metabolic profile and physical function Initiated trial in May 2024, ahead of target timeline Strong enrollment momentum Updating guidance for topline data to Q2 2025 18

© 2024 Scholar Rock, Inc. All rights reserved. Enrolling Phase 2 Proof-of-Concept Study of Apitegromab in Obesity 19 E M B R A Z E TR I A L D E S I G N Randomized, double-blind, placebo-controlled (n=100) Enrolling patients who are overweight or obese Enrollment ahead of schedule; topline data expected in Q2 2025 Screening (Up to 4 weeks) • Male or female, age ≥18 and ≤65 years old at the time of informed consent • Stable body weight within 90 days of Screening • BMI ≥30.0 kg/m2 to ≤45.0 kg/m2 or ≥27.0 kg/m2 to <30.0 kg/m2 with the presence of 1 or more weight-related comorbid condition(s) ENDPOINTS Primary Endpoint (Week 24) Change from baseline in lean mass by DEXA scan Secondary Endpoints Additional weight loss measures, safety & tolerability, PK/PD Treatment (24 weeks) Apitegromab Q4W + tirzepatide or semaglutide* QW Placebo Q4W + tirzepatide or semaglutide* QW ADDITIONAL Exploratory Endpoints (Week 24 and 32) Cardiometabolic profile (e.g., HbA1c), body composition, physical function R N=50 N=50 8 weeks (24 weeks) Primary endpoint (32 weeks) Exploratory endpoints *Participating patient will be assigned to either tirzepatide or semaglutide depending on availability. Apitegromab dose regimen will be 10 mg/kg Q4W, based on projected exposure in the obese population comparable to that of 20 mg/kg Q4W in SMA Tirzepatide and semaglutide dose regimen will follow the United States Prescribing Information. O B E S I T Y

© 2024 Scholar Rock, Inc. All rights reserved. Platform and Expertise Drive Success in Clinic 21 Superb Engineering Clinic SRK-439 Cardiometabolic IND planned for 2025 SRK-181 Immuno-oncology T R A N S L A T IO NA L S U C C ES S TO TH E C L I N I C Apitegromab Spinal Muscular Atrophy Translational Models Muscle stimulation (Hz) Muscle strength (torque) Treatment SRK-373 Fibrosis IND planned for 2026 Groups Lean Mass Fibrosis Anti-PD1/ Anti-PD1 SRK-181-mIgG1

© 2024 Scholar Rock, Inc. All rights reserved. 22 O B E S I T Y Preclinical data to date show strong potential to support healthier weight loss in combination with GLP-1 RA: Preservation of lean mass during GLP-1 RA-induced weight loss and improvement in metabolic parameters Increase in lean mass and attenuation of fat mass regain following GLP-1 RA withdrawal Greater potency compared to an anti-ACTRII antibody Give Us Confidence in SRK-439 Strong Scientific Validation and Promising Preclinical Evidence

© 2024 Scholar Rock, Inc. All rights reserved. 23 SRK-439 Increased Lean Mass and Attenuated Regain of Fat Mass After GLP-1 RA Withdrawal in Obesity Mouse Model Study conducted in Diet Induced Obesity (DIO) mouse model utilizing a murine chimera of SRK-439 GLP-1 RA: GLP1 receptor agonist. Day 7 is start of semaglutide and SRK-439 treatment. Day 35 is discontinuation of semaglutide treatment. • Considerable lean mass loss seen with semaglutide treatment as expected • Treatment with SRK-439 led to: • Preservation of lean mass during semaglutide treatment • Significant increase in lean mass upon semaglutide discontinuation • Attenuation of fat mass regain upon semaglutide discontinuation Key Observations SRK-439 Increased Absolute Lean Mass and Attenuated Regain of Absolute Fat Mass O B E S I T Y 0 7 14 21 28 35 42 49 56 63 20 25 30 Absolute Lean Mass Days in study Lean Mass (g) Chow + IgG HFD + IgG HFD + IgG +Sema HFD + 439 + Sema -6 0 7 14 21 28 35 42 49 56 63 0 5 10 15 20 Absolute Fat Mass Days in study Fat Mass (g) Chow + IgG HFD + IgG HFD + IgG +Sema HFD + 439 + Sema -6

© 2024 Scholar Rock, Inc. All rights reserved. 24 SRK-439 Improved Body Composition After GLP 1-RA Withdrawal Study conducted in Diet Induced Obesity (DIO) mouse model utilizing a murine chimera of SRK-439 GLP-1 RA: GLP1 receptor agonist. HFD: High Fat Diet • SRK-439 attenuates regain of fat mass after withdrawal of semaglutide compared to IgG control • SRK-439 leads to higher lean mass proportion after withdrawal of semaglutide compared to IgG control Key Observations SRK-439 Improved Proportion of Lean and Fat Mass to Total Body Weight O B E S I T Y Chow IgG HFD IgG HFD IgG Sema HFD 439 Sema 0 20 40 60 80 Relative Lean Mass Day 63 Lean Mass/BW (%) p < 0.0001 p < 0.0001 p = 0.0004 Chow IgG HFD IgG HFD IgG Sema HFD 439 Sema 0 10 20 30 40 Relative Fat Mass Day 63 Fat Mass/BW (%) p < 0.0001 p = 0.0083 p = 0.0002 p = 0.0023

© 2024 Scholar Rock, Inc. All rights reserved. SRK-439 Is More Potent than Anti-ActRII Antibody at Maintaining Lean Mass During GLP-1 RA-Induced Weight Loss 25 L O W E FF I C A C I O U S D O S E A N D C O M P E T IT I V E P R O F IL E Head-to-Head Comparison to Non-Selective Myostatin Inhibitor in DIO Mouse Model -20 -15 -10 -5 0 Percent Change Lean Mass from Baseline ✱ ✱ ✱✱✱✱ ✱✱✱✱ ✱✱✱✱ ✱✱✱✱ 0.3 1 3 10 0.3 1 3 20 Antibody Treatment (mg/kg) + Semaglutide (0.04mg/kg) SRK-439 Anti-ActRII* p < 0.01 p < 0.0001 p < 0.0001 p < 0.0001 p < 0.0001 •SRK-439 preserved semaglutide-driven lean mass loss dose-dependently and at lower doses than anti-ActRII •Highlights efficacy of SRK-439 and avoids potential liabilities of non-selective approach of anti-ActRII antibodies •Low target dose of SRK-439 supports subcutaneous and potentially best-in-class profile Key Observations *Murine chimera of Bimagrumab Study conducted in Diet Induced Obesity (DIO) mouse model utilizing a murine chimera of SRK-439 O B E S I T Y

© 2024 Scholar Rock, Inc. All rights reserved. Leveraging Apitegromab to Inform Obesity Program 26 P IP E L I N E O V E R VI E W Tests hypothesis of selective anti-myostatin in obese population POC study start* POC data readout Q2* Ph 2 proof-of-concept trial APITEGROMAB + GLP-1 Agonist 2024 2025 IND* SRK-439 + GLP-1 Agonist Novel asset for cardiometabolic indication Phase 1 trial Cardiometabolic Disorders *Expected timelines POC = Proof of Concept O B E S I T Y

© 2024 Scholar Rock, Inc. All rights reserved. 28 Looking Ahead: Upcoming Milestones IND=Investigational new drug; POC=Proof of concept; Q2 2024 ACCOMPLISHMENTS PRESENTED encouraging SRK-181 data at ASCO INITIATED EMBRAZE trial with apitegromab in obesity 2H 2024 focus SAPPHIRE Readout in Q4 Complete EMBRAZE enrollment Advance IND-enabling studies for SRK-439 Prepare for commercialization PRIME pre-submission meeting completed ADVANCED IND-enabling studies for SRK-439

© 2024 Scholar Rock, Inc. All rights reserved. 30 Company Speakers Jay Backstrom, M.D., MPH President & Chief Executive Officer Jing Marantz, M.D., Ph.D. Chief Medical Officer Ted Myles, MBA Chief Operating Officer and Chief Financial Officer Mo Qatanani, Ph.D. Chief Scientific Officer

Document and Entity Information	Aug. 08, 2024
Document and Entity Information [Abstract]
Document Type	8-K
Document Period End Date	Aug. 08, 2024
Entity File Number	001-38501
Entity Registrant Name	Scholar Rock Holding Corporation
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	82-3750435
Entity Address State Or Province	MA
Entity Address, Address Line One	301 Binney Street
Entity Address, Adress Line Two	3rd Floor
Entity Address, City or Town	Cambridge
Entity Address, Postal Zip Code	02142
City Area Code	857
Local Phone Number	259-3860
Written Communications	false
Soliciting Material	false
Pre-commencement Tender Offer	false
Pre-commencement Issuer Tender Offer	false
Title of 12(b) Security	Common Stock, par value $0.001 per share
Trading Symbol	SRRK
Security Exchange Name	NASDAQ
Entity Emerging Growth Company	false
Entity Central Index Key	0001727196
Amendment Flag	false