# EDGAR Filing Document

**Accession Number:** 0001727196
**File Stem:** 0001104659-25-092317
**Filing Date:** 2025-9
**Character Count:** 15209
**Document Hash:** 2286a38ede3d31fa4eab03b18e204c76
**Contains OCR:** False
**Source Format:** 

## Filing Content

## Filing Summary
**0001104659-25-092317.hdr.sgml**: 20250923

**ACCESSION NUMBER**: 0001104659-25-092317

**CONFORMED SUBMISSION TYPE**: 8-K

**PUBLIC DOCUMENT COUNT**: 13

**CONFORMED PERIOD OF REPORT**: 20250923

**ITEM INFORMATION**: Regulation FD Disclosure

**ITEM INFORMATION**: Other Events

**ITEM INFORMATION**: Financial Statements and Exhibits

**FILED AS OF DATE**: 20250923

**DATE AS OF CHANGE**: 20250923

**FILER**: 

**COMPANY DATA:**
- **COMPANY CONFORMED NAME:** Scholar Rock Holding Corp
- **CENTRAL INDEX KEY:** 0001727196
- **STANDARD INDUSTRIAL CLASSIFICATION:** BIOLOGICAL PRODUCTS (NO DIAGNOSTIC SUBSTANCES) [2836]
- **ORGANIZATION NAME:** 03 Life Sciences
- **EIN:** 823750435
- **STATE OF INCORPORATION:** DE
- **FISCAL YEAR END:** 1231

**FILING VALUES:**
- **FORM TYPE:** 8-K
- **SEC ACT:** 1934 Act
- **SEC FILE NUMBER:** 001-38501
- **FILM NUMBER:** 251331715

**BUSINESS ADDRESS:**
- **STREET 1:** 301 BINNEY STREET
- **STREET 2:** 3RD FLOOR
- **CITY:** CAMBRIDGE
- **STATE:** MA
- **ZIP:** 02142
- **BUSINESS PHONE:** 857-259-3860

**MAIL ADDRESS:**
- **STREET 1:** 301 BINNEY STREET
- **STREET 2:** 3RD FLOOR
- **CITY:** CAMBRIDGE
- **STATE:** MA
- **ZIP:** 02142

?xml version='1.0' encoding='ASCII'?

**UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549**

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**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 23, 2025

**Scholar Rock Holding Corporation**

(Exact Name of Registrant as Specified in Charter)

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| | | |
|:---|:---|:---|
| **Delaware** | **001-38501** | **82-3750435** |
| (State or Other Jurisdiction of<br> Incorporation) | (Commission File Number) | (I.R.S. Employer Identification Number) |
| **301 Binney Street, 3rd Floor, Cambridge, MA 02142** | **301 Binney Street, 3rd Floor, Cambridge, MA 02142** | **301 Binney Street, 3rd Floor, Cambridge, MA 02142** |
| (Address of Principal Executive Offices) (Zip Code) | (Address of Principal Executive Offices) (Zip Code) | (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:

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| |
|:---|
| 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:

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| | | |
|:---|:---|:---|
| **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 23, 2025, Scholar Rock Holding Corporation (the "Company") issued a press release announcing that the U.S. Food and Drug Administration ("FDA") has issued a Complete Response Letter ("CRL") regarding the Biologics License Application ("BLA") for apitegromab for the treatment of spinal muscular atrophy ("SMA"). 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.

The information in Item 7.01 of this Form 8-K, including Exhibit 99.1, 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 23, 2025, the Company announced that the FDA has issued a CRL regarding the BLA for apitegromab for the treatment of SMA.

**Item 9.01. Financial Statements and Exhibits.**

(d) Exhibits

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| | |
|:---|:---|
| **Exhibit<br> No.** | **Description** |
| [99.1](tm2526733d1_ex99-1.htm) | [Press Release issued by the Company on September 23, 2025, furnished hereto.](tm2526733d1_ex99-1.htm) |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) |

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**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** | **Scholar Rock Holding Corporation** |
| Date: September 23, 2025 | By: | /s/ Junlin Ho |
|  |  | Junlin Ho |
|  |  | General Counsel & Corporate Secretary |

---

## Exhibit 99.1

**Exhibit 99.1**

**FDA Issues Complete Response Letter (CRL) for Apitegromab** **as a Treatment for<br> Patients with Spinal Muscular Atrophy (SMA) Solely Related to Observations Identified at <br> Catalent Indiana LLC Fill-Finish Facility**

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;· *No other approvability issues cited in CRL* 

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;· *Scholar Rock intends to resubmit the apitegromab Biologics License Application (BLA) upon resolution of Catalent Indiana LLC-related observations* 

CAMBRIDGE, Mass. — September 23, 2025 — Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to dramatically improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe and debilitating neuromuscular diseases by applying its leading platform in myostatin biology to advance musculoskeletal health, today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the apitegromab Biologics License Application (BLA) for the treatment of patients with spinal muscular atrophy (SMA). The CRL is related to observations identified during a routine general site inspection of Catalent Indiana LLC, a third-party fill-finish facility which was acquired by Novo Nordisk A/S in December 2024. The observations are not specific to apitegromab. The CRL did not cite any other approvability concerns, including apitegromab's efficacy and safety data or the third-party drug substance manufacturer.

The Catalent Indiana observations were discussed during Scholar Rock's second quarter 2025 earnings announcement. Catalent Indiana submitted a comprehensive response in early August 2025 to address the observations noted by the FDA. Following that submission, Catalent Indiana has continued to work rapidly to take corrective action and has kept the FDA apprised of that progress.

Upon Catalent Indiana's successful remediation of the FDA observations, Scholar Rock will resubmit the apitegromab BLA. Scholar Rock believes that the FDA will be able to act expeditiously on the application once the manufacturing issues have been resolved.

"We are continuing to work closely with Catalent Indiana on the FDA's manufacturing observations so that we can resubmit the apitegromab BLA as soon as possible," said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. "We remain focused on working hand-in-hand with the FDA to pursue approval of the first and only muscle-targeted treatment for people living with SMA."

"While we are disappointed that the availability of a muscle-targeted treatment approach for patients with SMA has been delayed, we remain enthusiastic about the transformative potential of apitegromab," said Kenneth Hobby, President of Cure SMA. "Muscle strength and motor function are significant unmet needs for many in the SMA community and are fundamental to independence. A gain in motor function can allow someone to participate in important activities of daily living from self-care to work and social interactions, and as such, we urgently await the availability of the first-ever treatment with the potential to address the muscular component of SMA."

Apitegromab was granted Orphan Drug Designation, Rare Pediatric Disease Designation, Priority Review, and Fast Track designation, which recognizes the potential to treat a serious condition and fill an unmet medical need.

Outside of the U.S., the apitegromab marketing authorisation application (MAA) is under review by the European Medicines Agency and a decision is anticipated near mid-2026. European launch is anticipated in the second half of 2026, with Germany expected to be the first European market with patient access.

**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 in spinal muscular atrophy (SMA) to demonstrate clinical success in a pivotal Phase 3 clinical trial. 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. Apitegromab has not been approved for any use by the FDA or any other regulatory agency.

**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 a registered trademark of Scholar Rock, Inc.

**Availability of Other Information About Scholar Rock**

Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X (formerly known as Twitter) and LinkedIn. The information that we post on our website or on X (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, statements regarding Scholar Rock's future expectations, plans and prospects, including without limitation, the timing of any regulatory responses and anticipated approvals, the therapeutic potential, clinical benefits and safety of 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," "might," "could," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "project," "intend," "future," "potential," or "continue," and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, whether preclinical and clinical data, including the results from the Phase 3 SAPPHIRE trial, will be sufficient to support regulatory approval, whether the FDA observations related to Catalent Indiana are resolvable in a timely manner or at all, whether Scholar Rock will be able to resubmit its BLA in a timely manner and whether the updated BLA will be sufficient to support regulatory approval; information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock's ability to obtain, maintain and protect its intellectual property; and Scholar Rock's dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock's Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, 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:**

**Investors**

Laura Ekas, Ph.D.

**ir@scholarrock.com**

917-439-0374

**Media**

Molly MacLeod

**media@scholarrock.com**

802-579-5995