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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): March 31, 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 March 31, 2026, Scholar Rock Holding Corporation
(the “Company”) issued a press release announcing it resubmitted its biologics license application (“BLA”) to
the U.S. Food and Drug Administration (the “FDA”) for apitegromab for the treatment of children and adults with spinal muscular
atrophy (“SMA”). A copy of the press release is being furnished as Exhibit 99.1 to this Report on Form 8-K. The Company will
hold a conference call scheduled for 8:00 am ET on March 31, 2026 to discuss this update.
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.
On March 31, 2026, the Company announced that
it resubmitted its BLA to the FDA for apitegromab for the treatment of children and adults with SMA. The Company anticipates FDA
acceptance within 30 days and a review period of up to six months from the date of resubmission, with a Prescription Drug User Fee
Act (“PDUFA”) action date expected in late September 2026.
Cautionary Notes on Forward-Looking Statements
This Current Report on Form 8-K contains “forward-looking
statements” within the meaning of the federal securities laws, including Section 27A of the Securities Act of 1933, as amended,
and Section 21E of the Securities Exchange Act of 1934, as amended. In this context, forward-looking statements often address expected
future business and financial performance and financial condition, and often contain words such as “anticipate,” “believe,”
“expect,” “intend,” “plan,” “seek,” “see,” “will,” “would,”
“may,” “target,” and similar expressions and variations or negatives of these words. Forward-looking statements
by their nature address matters that are, to different degrees, uncertain, such as statements regarding timing of any regulatory submissions
and anticipated approvals, the therapeutic potential, clinical benefits and safety of any product candidates, the Company’s ability
to address the observations identified in the complete response letter, expectations regarding resubmission and timing of its BLA for
apitegromab upon the successful FDA reinspection of Catalent Indiana, expectations regarding commercial launch timing in the U.S. and
in Europe, expectations regarding a new fill finish facility and the achievement of important milestones, and the potential of its product
candidates and proprietary platform. These and other forward-looking statements are not guarantees of future results and are subject to
risks, uncertainties and assumptions that could cause actual results to differ materially from those expressed in any forward-looking
statements. Important risk factors that may cause such a difference include, but are not limited to the risks described in the Company’s
most recent annual report on Form 10-K for the year ended December 31, 2025. We undertake no obligation to update any forward-looking
statements as a result of new information, events or circumstances or other factors arising or coming to our attention after the date
hereof. The provision of the information in this report shall not be deemed an admission as to the materiality of any of the information
contained herein.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits
Exhibit
No. |
|
Description |
| |
|
|
| 99.1 |
|
Press Release issued by the Company on March 31, 2026, 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: March 31, 2026 |
By: |
/s/ Junlin Ho |
| |
|
Junlin Ho |
| |
|
General Counsel & Corporate Secretary |
Exhibit 99.1
Scholar Rock Resubmits Biologics License Application
(BLA) to FDA for Apitegromab for Treatment of Children and Adults with Spinal Muscular Atrophy (SMA)
| · | Apitegromab BLA resubmission includes Catalent Indiana LLC (part of Novo Nordisk) and second U.S.-based fill-finish facility, aligned
with FDA guidance from March 3, 2026 Type C meeting |
| · | FDA and Novo Nordisk Q1 2026 interactions resulted in Scholar Rock’s alignment with FDA to resubmit apitegromab BLA prior
to FDA reinspection of Catalent Indiana facility |
| · | FDA and Scholar Rock Q1 2026 interactions regarding accelerated progress in qualifying a second fill-finish facility resulted in
alignment with FDA to include facility in BLA resubmission |
| · | Company anticipates FDA acceptance of BLA within 30 days and a review period of up to 6 months from date of resubmission, with
PDUFA action date expected in late September 2026 |
| · | Management to host a conference call today at 8:00am ET |
CAMBRIDGE, Mass.—(BUSINESS WIRE)—March 31, 2026-- Scholar
Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to 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 it has resubmitted the Biologics License Application (BLA) for apitegromab for the treatment of
children and adults with SMA to the U.S. Food and Drug Administration (FDA).
Apitegromab is the first
and only muscle-targeted therapy to demonstrate statistically significant and clinically meaningful improvements in motor function in
a pivotal Phase 3 clinical trial in patients with SMA receiving treatment with an SMN-targeted therapy.
“Our apitegromab BLA resubmission marks an important step forward
in our mission to bring the world’s first muscle-targeted therapy to children and adults living with SMA,” said David L. Hallal,
Chairman and Chief Executive Officer of Scholar Rock. “We continue to be encouraged by the FDA’s engagement and shared sense
of urgency as we work relentlessly for the SMA community. As we execute our plans for both Catalent Indiana and our second fill-finish
facility, we look forward to anticipated apitegromab approvals and launches in both the U.S. and Europe this year.”
In September 2025, Scholar Rock
received a Complete Response Letter (CRL) from the FDA for apitegromab related to observations identified during a routine general site
inspection of Catalent Indiana, LLC (part of Novo Nordisk). The observations were not specific to apitegromab and the CRL did not cite
any other approvability concerns.
Since that time, Scholar Rock, Catalent Indiana, and the FDA have engaged
in several constructive and collaborative interactions, including an in-person Type A meeting in November 2025 and a meeting between Catalent
Indiana and the FDA early in the first quarter of 2026. During the meeting and an FDA site visit that followed, no additional corrective
actions were requested by the FDA to Novo Nordisk’s remediation plan. The decision to resubmit the apitegromab BLA prior to FDA
reinspection of Catalent Indiana was made in alignment with the FDA.
The apitegromab BLA resubmission also includes a second U.S.-based
fill-finish facility to strengthen the apitegromab supply chain and support future growing demand across the planned global commercial
footprint. Following a positive Type C meeting on March 3, 2026, Scholar Rock aligned with the FDA to include the additional facility
in the BLA resubmission based on the Company’s accelerated commercial fill-finish timelines.
Scholar Rock anticipates FDA acceptance of the BLA within 30 days of
resubmission and a review period of up to six months from the date of resubmission. A PDUFA action date is expected in late September
2026.
Updates to the apitegromab
BLA for the resubmission were limited in scope and primarily composed of a standard
safety update.
In Europe, the European Medicines Agency (EMA) review of the Marketing
Authorisation Application (MAA) for apitegromab is progressing well, with a decision anticipated mid-2026.
The FDA has granted Fast Track, Orphan Drug, Priority Review, and Rare
Pediatric Disease designations to apitegromab for the treatment of SMA. The European Medicines Agency (EMA) has granted Priority
Medicines (PRIME) and Orphan Medicinal Product designations.
Conference Call Information
Scholar Rock will host a conference call and webcast today, Tuesday,
March 31, 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 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. A 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 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 Scholar Rock
Scholar Rock is a late-stage biopharmaceutical company focused
on developing and commercializing apitegromab for children and adults with spinal muscular atrophy (SMA) and other rare, severe, and
debilitating neuromuscular diseases. As 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 at ScholarRock.com and follow @ScholarRock on X and on LinkedIn.
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 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, Scholar Rock’s timing of any regulatory submissions
and anticipated approvals, the therapeutic potential, clinical benefits and safety of any product candidates, its ability to address the
observations identified in the complete response letter, expectations regarding resubmission and timing of review of its BLA for apitegromab,
expectations regarding approval and commercial launch timing in the U.S. and in Europe, expectations regarding a new fill-finish facility
and the achievement of important milestones, 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, that preclinical and clinical data, including the results
from the Phase 2 or Phase 3 clinical trial of apitegromab, or the preclinical data for SRK-439, 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; whether the FDA will
accept the remediations to the Catalent Indiana fill-finish facility in response to the FDA observations, and whether the updated BLA
will be sufficient to support regulatory approval, risks related to delays in obtaining or failure to obtain FDA clearances or approvals
and noncompliance with FDA regulations, information provided or decisions made by regulatory authorities; 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 Annual Report on Form
10-K for the year ended December 31, 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.
Investor Contact
Laura Ekas, Ph.D.
ir@scholarrock.com
917-439-0374
Media Contact
Molly MacLeod, Ph.D.
media@scholarrock.com
802-579-5995
