Form 8-K

8-K — Scholar Rock Holding Corp

Accession: 0001104659-26-107273

Filed: 2026-09-14

Period: 2026-09-11

CIK: 0001727196

SIC: 2836 (BIOLOGICAL PRODUCTS (NO DIAGNOSTIC SUBSTANCES))

Item: Regulation FD Disclosure

Item: Other Events

Item: Financial Statements and Exhibits

Documents

8-K — tm2625362d1_8k.htm (Primary)

EX-99.1 — EXHIBIT 99.1 (tm2625362d1_ex99-1.htm)

EX-99.2 — EXHIBIT 99.2 (tm2625362d1_ex99-2.htm)

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0001727196

2026-09-14

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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): September 11, 2026

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 7.01. Regulation FD Disclosure.

On September 11, 2026, Scholar Rock Holding Corporation (the

“Company”) issued a press release announcing that the U.S. Food and Drug Administration (“FDA”) has approved

ISEMBYLD™ (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 years of age and

older who are currently receiving a survival motor neuron 2 (SMN2) targeted treatment. A copy of the press release is furnished as

Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference. A copy of the Company’s

presentation materials in connection with the announcement is furnished as Exhibit 99.2 to this Current Report on Form 8-K

and is incorporated herein by reference. The Company will host a webcast and conference call on September 14, 2026 at 8:00

a.m. Eastern Time to discuss the approval, during which management will review the presentation materials furnished as

Exhibit 99.2.

The information in Item 7.01 of this Form 8-K, including Exhibits

99.1 and 99.2, 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 a filing.

Item 8.01. Other Events.

On September 11, 2026, the Company announced that the FDA

has approved ISEMBYLD™ (apitegromab-mstn) for the treatment of SMA in adults and children 2 years of age and older who are

currently receiving a survival SMN2 targeted treatment.

Item 9.01. Financial Statements and Exhibits.

(d) Exhibits

Exhibit

No.

Description

99.1

Press Release issued by the Company on September 11,

2026, furnished hereto.

99.2

Presentation Slides, furnished hereto.

104

Cover Page Interactive Data File (embedded within

the Inline XBRL document)

SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934,

the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

Scholar Rock Holding Corporation

Date: September 14, 2026

By:

/s/ Junlin Ho

Junlin Ho

General Counsel & Corporate Secretary

EX-99.1 — EXHIBIT 99.1

EX-99.1

Filename: tm2625362d1_ex99-1.htm · Sequence: 2

Exhibit 99.1

Scholar Rock Announces

FDA Approval of ISEMBYLD™ (apitegromab-mstn), the First and Only Muscle-Targeted Treatment for Children and Adults with Spinal

Muscular Atrophy (SMA)

September 11, 2026

· ISEMBYLD

is approved for use in all adults and children ≥2 years of age with SMA who are currently

receiving a survival motor neuron 2 (SMN2)-targeted

treatment

· ISEMBYLD

recommended dose of 10 mg/kg showed a robust, clinically meaningful 2.2-point improvement

in motor function as measured by the gold-standard Hammersmith Functional Motor Scale-Expanded

(HFMSE) compared to placebo, with all patients receiving SMN2-targeted background therapy

(p = 0.0121*)

· ISEMBYLD

showed a ≥ 3-point increase in HFMSE in 34.2% of patients compared to 13.5% of patients

on placebo (odds ratio 3.8; p = 0.0125*)

· ISEMBYLD

U.S. commercial launch underway with product available to ship in the coming days; Scholar

Rock Supports™ dedicated support team now available to assist patients and caregivers

· Management

to host investor call Monday, September 14, 2026, at 8:00 a.m. ET

* nominal p-value

CAMBRIDGE, Mass.--(BUSINESS

WIRE)--Sep. 11, 2026-- Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of patients with

rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology, today announced that the

FDA has approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA) in adults and children two years of age

and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment.

SMA is a rare, severe

neuromuscular disease that results in irreversible loss of motor neurons and progressive muscle wasting, causing continuous motor function

decline throughout life and diminishing the independence of both adults and children.

ISEMBYLD is the first

and only muscle-targeted treatment to demonstrate motor function improvement in individuals with SMA currently receiving an SMN2-targeted

treatment. In the Phase 3 randomized, placebo-controlled SAPPHIRE study, individuals receiving ISEMBYLD demonstrated a robust, clinically

meaningful improvement in motor function after one year of treatment while those on an SMN2-targeted treatment alone experienced a loss

of motor function.

“Today’s

FDA approval of ISEMBYLD marks a defining moment for the SMA community as we now launch the world’s first-ever muscle targeted

treatment for children and adults living with SMA in the U.S.,” said David L. Hallal, Chairman and Chief Executive Officer of Scholar

Rock. “After decades of failed industry-wide efforts to unlock the potential of myostatin inhibition, Scholar Rock has delivered

a therapeutic breakthrough with ISEMBYLD. Our U.S. commercial team is now engaging physicians, SMA care teams, and payers on behalf of

the SMA community and our Scholar Rock Supports™ team stands ready to provide dedicated, comprehensive assistance to patients and

caregivers. I would like to extend my deepest thanks to our clinical study investigators, Cure SMA, and other patient advocacy groups

for their dedication and support on this journey. Above all, I want to express my heartfelt gratitude to the patients and families affected

by SMA who participated in our clinical trials for their trust in Scholar Rock and unwavering resilience every step of the way.”

“The

approval of ISEMBYLD as the first-ever treatment to directly target the muscular component of SMA is a significant turning point for

adults and children who have been waiting for innovative therapeutic options to improve motor function,” said Kenneth Hobby, President

of Cure SMA. “We appreciate the FDA’s recognition, as reflected in this approval that supports access for a broad population

within the SMA community, that improving motor function is a significant unmet need that must be addressed with urgency. Such improvements

are fundamental to maintaining independence and to enabling participation in important activities of daily living from self-care to work

and social interactions.”

With the approval of

ISEMBYLD, Scholar Rock was awarded a Rare Pediatric Disease Priority Review Voucher, which may be used to obtain priority review for

a future marketing application.

ISEMBYLD Robust Clinical

Efficacy and Safety Profile from Phase 3 SAPPHIRE Study

The

approval of ISEMBYLD was based on positive results from the Phase 3 pivotal, randomized, double-blind, placebo-controlled SAPPHIRE

study. The SAPPHIRE study met its primary endpoint, and demonstrated a robust, clinically meaningful 2.2-point improvement in the

gold-standard Hammersmith Functional Motor Scale-Expanded (HFMSE) in patients receiving ISEMBYLD 10 mg/kg and an SMN2-targeted

treatment compared to patients receiving an SMN2-targeted treatment alone at one year (nominal p= 0.0121; main efficacy population 2

- 12 years of age, n = 103). Additionally, 34.2% of ISEMBYLD-treated patients showed a ≥ 3-point increase in HFMSE compared to

13.5% of placebo-treated patients (odds ratio of 3.8; nominal p = 0.0125).

ISEMBYLD has a well-characterized

safety profile. The safety database includes more than 500 individuals across all apitegromab clinical studies globally, some of whom

have been on treatment for more than 7 years. Ninety-eight percent of participants treated in SAPPHIRE elected to continue in the ONYX

long-term extension study. In the SAPPHIRE study, the most common adverse reactions were upper respiratory tract infections, vomiting,

cough, other viral infections, headache, gastroenteritis, pharyngitis, and hypersensitivity. Fractures occurred in 9% of patients treated

with ISEMBYLD 10 mg/kg vs. 2% in placebo.

For further information, see

Important Safety Information below.

“Today’s

approval of ISEMBYLD marks a new era for the treatment of SMA,” said Dr. Basil Darras, M.D., Associate Neurologist-in-Chief, Director

of the Neuromuscular Center and Spinal Muscular Atrophy Program at Boston Children’s Hospital, and a principal investigator in

the SAPPHIRE study. “As neurologists, families consistently tell us that their top priority is gaining motor function, and we are

now able to directly target the muscle, not just the motor neuron, for people living with SMA.”

ISEMBYLD Commercial Availability

and Patient Access

Scholar Rock Supports is

now available to assist patients who have been prescribed ISEMBYLD. This program is designed to provide personalized support for patients

and families, and includes help to understand insurance coverage, financial assistance programs for eligible patients, and disease and

treatment education. Scholar Rock Supports will also help patients and families navigate their site of care options, as well as ongoing

infusion logistics. Based on eligibility, infusions can be given at convenient locations including hospital, home, or infusion center.

ISEMBYLD will be available to ship in the coming days.

Scholar Rock is working

closely with top commercial and government payers to establish reliable, broad access to ISEMBYLD for appropriate patients.

Healthcare providers and

patients can learn more about Scholar Rock’s patient support services by visiting www.ScholarRockSupports.com or calling

833-777-5444 (833-SRRK-444). For more information about ISEMBYLD and U.S. Prescribing Information, visit www.ISEMBYLD.com.

Conference Call Information

Scholar Rock will host

a conference call and webcast on Monday, September 14, 2026, at 8:00 a.m. ET. To access the live audio webcast, please go to “Events

and Presentations” in the Investors section of the Scholar Rock website at https://investors.scholarrock.com.

To participate via telephone,

please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how

to join the conference call.

A replay of the webcast will

be available on the Company’s website for approximately 90 days.

About ISEMBYLD

ISEMBYLD is a fully human

monoclonal IgG4 antibody that binds to promyostatin and latent myostatin and inhibits the activation of myostatin, blocking myostatin

signaling. ISEMBYLD is approved in the United States for the treatment of spinal muscular atrophy (SMA) in adults and pediatric patients

2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment.

For more information,

visit www.ISEMBYLD.com.

Indication and Important Safety Information

What is ISEMBYLD (apitegromab-mstn)?

ISEMBYLD

is a prescription medicine used to treat spinal muscular atrophy (SMA) in adults and children 2 years of age and older who are

currently receiving a survival motor neuron 2 (SMN2)-targeted treatment.

IMPORTANT SAFETY INFORMATION

Before taking ISEMBYLD, tell

your healthcare provider about all of your medical conditions, including if you:

· have

a history of low bone density or bone fractures

· are

pregnant or plan to become pregnant. If you are pregnant or are planning to become pregnant,

ask your healthcare provider for advice before taking this medicine. It is not known if ISEMBYLD

will harm your unborn baby. Tell your healthcare provider right away if you become pregnant

during treatment with ISEMBYLD

· are

breastfeeding or plan to breastfeed. It is not known if ISEMBYLD passes into breast milk.

Talk to your healthcare provider about the best way to feed your baby while on treatment

with ISEMBYLD

Tell your healthcare

provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Keep a list of them to show your healthcare provider, including your pharmacist, when you get a new medicine.

What are the possible side

effects of ISEMBYLD?

· Fractures.

Treatment with ISEMBYLD may increase the risk of bone fractures, including serious fractures.

Bone fractures may happen with or without a fall or other injury. Your healthcare provider

may consider stopping treatment with ISEMBYLD if you experience a bone fracture during treatment.

The most common side effects

of ISEMBYLD include:

· upper

respiratory tract infections

· vomiting

· cough

· viral

infections

· headache

· stomach

flu (gastroenteritis)

· sore

throat (pharyngitis)

· hypersensitivity

These are not all of the possible

side effects of ISEMBYLD. Call your healthcare provider for medical advice about side effects.

You are encouraged to

report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see full Prescribing

Information and Patient Information.

About the SAPPHIRE Clinical

Trial

SAPPHIRE (NCT05156320)

was a global, multi-national, randomized, double-blind, placebo-controlled Phase 3 clinical trial that evaluated the safety and efficacy

of ISEMBYLD (apitegromab-mstn) in a total of 188 patients in 9 countries with a diagnosis of 5q SMA who were 2 to 21 years of age. Patients

were randomized in a 1:1:1 ratio to receive ISEMBYLD 20 mg/kg (2 times the recommended dosage), ISEMBYLD 10 mg/kg (the recommended dosage),

or placebo, respectively, via intravenous infusion once every 4 weeks for approximately 1 year. All patients enrolled in this trial were

receiving an approved SMN2-targeted treatment (either nusinersen or risdiplam).

About SMA

Spinal muscular atrophy

(SMA) is a rare, severe, genetic neuromuscular disease. The disease is characterized by the irreversible loss of motor neurons, atrophy

of the voluntary muscles of the limbs and trunk, and progressive muscle wasting that causes continuous motor function decline throughout

life and can diminish the independence of both children and adults. Motor function decline in SMA patients is affected by motor neuron

health and muscle responsiveness. SMN-targeted treatments are designed to prevent motor neuron loss but do not directly address muscle.

It is estimated that approximately 35,000 SMA patients globally have been treated with an SMN-targeted treatment.

About Scholar Rock

Scholar Rock is delivering

muscle-targeted breakthroughs to transform the treatment of spinal muscular atrophy (SMA) and other rare neuromuscular diseases where

muscle atrophy remains a critical unmet need. Scholar Rock intends to commercialize ISEMBYLD (apitegromab-mstn) globally, beginning in

the U.S. for individuals living with SMA who are 2 years of age and older and currently receiving a survival motor neuron 2 (SMN2)-targeted

treatment.

a global leader in myostatin biology, a field focused on proteins that regulate muscle mass, the biopharmaceutical company is named

for the visual resemblance of a scholar rock to protein structures. Our commitment to unlock fundamentally different treatment

approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate

protein growth factors with extraordinary selectivity. Scholar Rock works every day to create new possibilities for patients through

its highly innovative anti-myostatin program, including opportunities in additional rare neuromuscular diseases. Learn more about

our neuromuscular franchise at ScholarRock.com and follow @ScholarRock on X and on LinkedIn.

Scholar Rock® is a

registered trademark and ISEMBYLD ™is a trademark of Scholar Rock, Inc.

Availability of Other Information

About Scholar Rock

Investors and others should

note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited

to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference

call transcripts and webcast transcripts, as well as on X (formerly known as Twitter) and LinkedIn. The information that we post on our

website or on X (formerly known as 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.

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: Scholar Rock’s expectations regarding the U.S. commercial launch of ISEMBYLD, ISEMBYLD’s

ability to support a broad patient population, the potential approval and launch of apitegromab in other geographies,

ISEMBYLD’s ability to change patient lives, the anticipated benefits of ISEMBYLD for patients with SMA, and the

Company’s business strategy, plans and prospects. The words “may,” “might,” “will,”

“could,” “would,” “should,” “expect,” “anticipate,” “plan,”

“believe,” “intend,” “estimate,” “potential,” “continue,”

“target,” “goal,” and similar expressions are intended to identify forward-looking statements, although not

all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on

the Company’s current expectations, intentions, and beliefs regarding future events, and are subject to risks and

uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. These risks

and uncertainties include, without limitation: the Company’s ability to successfully commercialize ISEMBYLD in the U.S.; the

Company’s ability to obtain and maintain regulatory approval of apitegromab in other jurisdictions; risks related to market

acceptance, competition, pricing, reimbursement and access; manufacturing and supply chain risks; Scholar Rock’s ability to

obtain, maintain and protect its intellectual property; and Scholar Rock’s dependence on third parties for development and

manufacture of ISEMBYLD, 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, 2026, as well as discussions of potential risks,

uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission.

Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing

its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock

undertakes no duty to update this information unless required by law.

Multimedia Assets:

https://scholarrock.com/media-kit/ISEMBYLD.jpg

https://scholarrock.com/media-kit/ISEMBYLD_logo.jpg

https://scholarrock.com/media-kit/Scholar_Rock_logo.png

https://scholarrock.com/media-kit/Scholar_Rock_Supports.jpg

View source version on businesswire.com:

https://www.businesswire.com/news/home/20260911564629/en/

Scholar Rock:

Investors

Laura Ekas, Ph.D.

ir@scholarrock.com

917-439-0374

Media

Jeff Smith

682-401-8428

Molly MacLeod, Ph.D.

802-579-5995

media@scholarrock.com

Source: Scholar Rock

EX-99.2 — EXHIBIT 99.2

EX-99.2

Filename: tm2625362d1_ex99-2.htm · Sequence: 3

Exhibit

99.2

ISEMBYLD

(apitegromab-mstn)

FDA Approval

Conference Call

September 14, 2026

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 the U.S. commercial launch of ISEMBYLD, the potential approval and launch of apitegromab in Europe and

other geographies, the anticipated benefits of ISEMBYLD for patients with SMA, the Company's business strategy, plans and prospects, its growth, strategy, cash runway, progress and

timing of its clinical trials and development programs for apitegromab, including its subcutaneous formulation, including the timing of any regulatory submissions, decisions and

anticipated approvals, the therapeutic potential, clinical benefits and safety of ISEMBYLD and any product candidates; expectations regarding commercial launch timing, and the

achievement of important milestones, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the

potential of its product candidates and proprietary platform. The use of words such as “may,” “could,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,”

“project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements for the purposes of the safe harbor

provisions under The Private Securities Litigation Reform Act of 1995. All such forward-looking statements are based on management's current expectations of future events and are

subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements.

These risks and uncertainties include, without limitation, the potential therapeutic benefit of ISEMBYLD; the Company's ability to successfully commercialize ISEMBYLD in the U.S.; the

Company's ability to obtain and maintain regulatory approval of apitegromab in other jurisdictions; risks related to market acceptance, competition, pricing, reimbursement and access;

manufacturing and supply chain risks; that preclinical and clinical data, including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, data from any ongoing or future

trials of apitegromab, 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;

Scholar Rock’s ability to manage expenses or provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline;

information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain

and protect its intellectual property; the success of Scholar Rock’s current and potential future collaborations; Scholar Rock’s dependence on third parties for development and

manufacture of product candidates including, without

limitation, to supply any clinical trials; Scholar Rock’s ability to obtain additional funding when needed to support its business activities; its ability to receive priority or expedited

regulatory review or to obtain regulatory approval of apitegromab; its ability to expand globally and the anticipated commercial launch in the United States of apitegromab in 2026; as

well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as well as

discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking

statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this presentation is as

of the date of this presentation, and Scholar Rock undertakes no duty to update this information unless required by law.

This presentation may also contain estimates and other statistical data made by independent parties and by us relating to market size and growth and other data about our industry. This

data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. In addition, projections, assumptions, and estimates of our

future performance and the future performance of the markets in which we compete are necessarily subject to a high degree of uncertainty and risk.

Apitegromab has not been approved for any use by any regulatory agency other than the FDA.

ISEMBYLD FDA Approval

David L. Hallal

Chairman & Chief Executive Officer

4 SMA, Spinal Muscular Atrophy.

FIRST AND ONLY

muscle-targeted therapy

for adults and children ≥2

years of age with SMA

FDA

APPROVED

NOW

Ushering in the next phase of innovation for the

SMA community

1. ISEMBYLD prescribing information. HMFSE, Hammersmith Functional Motor Scale-Expanded.

*Nominal p-value

ISEMBYLD (apitegromab-mstn) is

indicated for the treatment of

spinal muscular atrophy (SMA) in

adults and children 2 years of

age and older who are currently

receiving a survival motor neuron 2

(SMN2)-targeted treatment1

U.S. LAUNCH NOW UNDERWAY

LOSS OF

MOTOR

FUNCTION

GAIN OF

MOTOR

FUNCTION

+2.2 POINT

IMPROVEMENT IN HMFSE (p=0.0121*)

WORKING TO BRING

TO SMA PATIENTS GLOBALLY

Building a 50-country operating platform

to serve patients with rare, severe neuromuscular diseases

UNITED

STATES EUROPE REST OF

WORLD

JAPAN

ISEMBYLD FDA Approval

and Clinical Program

Akshay Vaishnaw, M.D., Ph.D.

President of R&D

FIRST AND ONLY

FDA-approved

myostatin inhibitor FDA

APPROVED

NOW

Motor neuron loss and muscle atrophy in SMA lead to progressive

muscle weakness

SMA, Spinal muscular atrophy; SMN, Survival motor neuron.

Figure adapted from: SMA Foundation Overview. http://www.smafoundation.org/wp-content/uploads/2012/03/SMA-Overview.pdf.

HEALTHY MOTOR UNIT MOTOR UNIT IN SMA

Healthy

Motor Neuron

Latent

Myostatin

Inhibition

SMN2-TARGETED

TREATMENT

MUSCLE-TARGETED

THERAPY

Degenerating

Motor Neuron

Muscle

Atrophy

Healthy

Muscle

SMA, Spinal Muscular Atrophy; SMN, Survival Motor Neuron. 1. Barrett D, et al. Adv Ther 2021;38;3203-3222. 2. Crawford TO, et al. Front Neurol 2024: 15:1419791. 3. Crawford TO, et al.

Presented at World Muscle Society, Oct 8-12, 2024. 4. Crawford, TO, et al. Lancet Neurol. 2025 Sep; 24(9):727-739. 5. ISEMBYLD Prescribing Information.

ISEMBYLD has been rigorously studied in children and adults with SMA

Apitegromab clinical development program

ISEMBYLD showed a gain in motor function while placebo was associated with a loss in motor function

PARTICIPANTS

Healthy volunteers, N=40

PURPOSE

Dose-finding in

healthy volunteers

KEY FINDING

Showed robust target

engagement and

acceptable safety1

PARTICIPANTS

Ambulatory and non-ambulatory SMA, N=58, ages

2-21

PURPOSE

Characterize safety

experience over 4 years

KEY FINDING

Showed preliminary

evidence of efficacy with an

SMN-targeted treatment2,3

PARTICIPANTS

Non-ambulatory SMA,

N=188, ages 2-21

PURPOSE

Placebo-controlled pivotal

study to assess:

• Efficacy

• Safety

KEY FINDING

Demonstrated statistically

significant motor function

improvement over placebo

with an SMN-targeted

treatment4,5

PARTICIPANTS

TOPAZ & SAPPHIRE patients

PURPOSE

Open-label Phase 3

Extension, capturing

additional:

• Efficacy

• Safety

ONGOING

PARTICIPANTS

Children <2 years with SMA

PURPOSE

To determine safety and

efficacy in young children,

including those treated with

SMN1-targeted treatment

(Zolgensma )

ONGOING

PHASE 1 PHASE 2 PHASE 3 EXTENSION PHASE 2

Positive Phase 3 trial demonstrates robust, clinically meaningful benefit

of ISEMBYLD in children and adults living with SMA

HFMSE, Hammersmith Functional Motor Scale-Expanded; SMA, Spinal Muscular Atrophy; SMN, Survival Motor Neuron. *Nominal p-value; Based on ISEMBYLD 10 mg/kg + SMN-targeted

therapy (nusinersen or risdiplam) versus placebo + SMN-targeted therapy. Change from baseline in HFMSE total score at 1 year with +1.0 change from baseline for ISEMBYLD 10 mg/kg vs. -

Percentage of patients experiencing a ≥3 point improvement in

HFMSE total score at 52 weeks

P=0.0125 (nominal)*

Placebo with

SMN2-targeted treatment (n=50)

13.5%

ISEMBYLD (10mg/kg) with

SMN2-targeted treatment (n=53)

34.2%

Percentage

≥3 point

increase

in HFMSE

HFMSE IMPROVEMENT

with ISEMBYLD and an SMN2-

targeted treatment compared

to SMN2-targeted treatment

alone (n=103)

2.2 POINT (p=0.0121*)

*Occurring in ≥20% of ISEMBYLD-treated patients and more frequently than in placebo.

An increased risk of fractures, including serious fractures, was observed with ISEMBYLD.

All AEs were coded using the MedDRA (Medical Dictionary for Regulatory Activities Terminology) version 26.1.

ISEMBYLD well-established safety profile

MOST COMMON

ADVERSE REACTIONS* (AEs)

ISEMBYLD 10 MG/KG

(n = 53)

PLACEBO

(n = 50)

Upper respiratory tract infections 66 54

Vomiting 30 16

Cough 28 22

Other viral infections 26 16

Headache 23 16

Gastroenteritis 23 8

Pharyngitis 21 14

Hypersensitivity 21 16

Children and adults with SMA

have been studied

246

Total time on ISEMBYLD for

longest treated patients

MORE THAN 7YEARS

Patient-years of safety data

in SMA

900+

Participants in TOPAZ and

SAPPHIRE entered the ONYX

long-term extension study

99%

ISEMBYLD U.S. Launch

Keith Woods

Chief Operating Officer

Persistent and progressive muscle atrophy is a critical unmet

need in SMA despite SMN-targeted treatments

Continue to experience

persistent and progressive

muscle atrophy that limits

both function and

independence1

~95%

OF PATIENTS

THE FUTURE OF SMA TREATMENT

TARGETS BOTH

MOTOR NEURON + MUSCLE

LOSS OF

MOTOR

FUNCTION

GAIN OF

MOTOR

FUNCTION

U.S. LAUNCH

UNDERWAY

6,600

Children and adults with

SMA in the U.S. receiving

an SMN2-targeted treatment

2,600

PRESCRIBING

PHYSICIANS 140

TREATMENT

CENTERS

COMPELLING CLINICAL BENEFIT

EXPECT STEADY AND CONSISTENT ISEMBYLD

NEW PATIENT STARTS OVER TIME

Scholar Rock Supports patient support program designed to offer

personalized, comprehensive services to patients and caregivers

Disease &

Treatment

Education

Insurance

Navigation

Infusion Day

Logistics

Caregiver

Support

Financial

Assistance*

Establishing broad and reliable access to ISEMBYLD

SMA, Spinal Muscular Atrophy. SMN, Survival Motor Neuron.

COMMERCIAL

GOVERNMENT

~50%

EXTREME RARITY: ~6,600 patients with SMA on

an SMN2-targeted treatment in the U.S.

SEVERITY: devastating and progressive

disease, despite the use of chronic

SMN-targeted treatments

ISEMBYLD COMPELLING CLINICAL BENEFIT:

Loss of motor function to gain of motor function

demonstrated in Phase 3 SAPPHIRE study

EXPECTED

PAYER MIX

CONSIDERATIONS IN PRICING ISEMBYLD

Ready to serve the SMA community in the U.S. with ISEMBYLD

Available for infusion

in coming days

Centers of Excellence,

infusion centers,

or the patient’s home

COMMERCIAL

SUPPLY

SITE OF

CARE

Optionality for patients,

caregivers, providers,

and payers

DISTRIBUTION

NETWORK

Financial Update

Vikas Sinha

Chief Financial Officer

Strong financial position

Additional $150 million available under

debt facility 1

Rare Pediatric Disease Priority Review Voucher

Scholar Rock intends to monetize 2

3 Begin to recognize revenue from ISEMBYLD

$492M

IN CASH, CASH

EQUIVALENTS, AND

MARKETABLE SECURITIES

AS OF JUNE 30, 2026

PRIORITIZED

INVESTMENTS

ISEMBYLD launch

in U.S.

Strengthening

supply chain

Launch readiness

in Europe and

Japan

Highly innovative

clinical programs

21 SMA, Spinal Muscular Atrophy.

FIRST AND ONLY

muscle-targeted therapy

for adults and children ≥2

years of age with SMA

FDA

APPROVED

NOW

ir@scholarrock.com media@scholarrock.com www.scholarrock.com

THANK YOU

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