On this page of StockholderLetter.com we present the latest annual shareholder letter from Scholar Rock Holding Corp — ticker symbol SRRK. Reading current and past SRRK letters to shareholders can bring important insights into the investment thesis.
Annual Report 2025
transition to strengthen our corporate and operational capabilities, building a highly experienced commercial organization with customer-facing teams in the U.S. and commercial build-out in Europe. In parallel, we enhanced our R&D leadership and clinical pipeline with the advancement of multiple clinical and preclinical programs to broaden our impact for people living with rare, severe, and debilitating neuromuscular diseases. To Our Shareholders: Scholar Rock enters 2026 in a position of strength for what promises to be a year with many important milestones, as we remain focused on obtaining our first regulatory approvals for apitegromab in the U.S. and Europe for people living with spinal muscular atrophy (SMA). I am gratified to have served as Scholar Rock s Board Chairman over the past eight years, and it is a privilege now to lead the organization as Chairman and Chief Executive Officer as we transition toward becoming a global commercialstage company. This past year marked a pivotal chapter for Scholar Rock. As the world leaders in myostatin biology, we are closer than ever to delivering the first and only muscle-targeted treatment to the global SMA community. Additionally, we advanced our industry-leading antimyostatin pipeline and sharpened our strategic focus on rare neuromuscular diseases where muscle is a principal organ affected by the disorder. This focus positions us to meaningfully improve the lives of patients worldwide while providing an opportunity for sustainable growth for Scholar Rock through this decade and well into the next. In the past year, we progressed toward important U.S. and European regulatory milestones for apitegromab for the treatment of children and adults with SMA, advancing our marketing applications through the regulatory process toward an anticipated approval and commercial launch in 2026. In preparation for this next phase, we executed our planned leadership In 2026, our goal is clear: To bring apitegromab to children and adults with SMA as quickly as possible. With the strength of our Phase 3 SAPPHIRE clinical data, we believe we are well-positioned for commercial launches in the U.S. and Europe in 2026. Our teams are working with urgency on behalf of patients, caregivers, and families who have long awaited the world s first muscle-targeted treatment to usher in the next phase of innovation for SMA. In the U.S., following receipt of a Complete Response Letter, we were pleased to hold a constructive and collaborative Type A meeting with the U.S. Food and Drug Administration. Since then, we have resubmitted our Biologics License Application. Global commercial readiness efforts are well underway, positioning us well for U.S. and European launches upon approval, as we expand toward a 50-country operating platform to deliver on our ambition to reach patients with SMA and other rare, severe neuromuscular diseases globally. Alongside our anticipated SMA launch activities, we continue to advance our innovative anti-myostatin pipeline with the vision of reaching all those living with SMA who may benefit. In late 2025, we initiated the Phase 2 OPAL trial to evaluate apitegromab in patients with SMA under two years of age who are receiving treatment with an approved therapy for SMA, including a one-time dose of gene therapy. We have also been working to advance subcutaneous apitegromab, with the completion of a Phase 1 study in healthy volunteers, which represents potential for additional optionality and impact for patients. Notably, we announced plans to develop apitegromab for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD), a rare, progressive neuromuscular disease characterized by muscle atrophy and functional decline, affecting approximately 30,000 individuals across the U.S. and Europe. There are currently no approved treatments for FSHD. The Phase 2 FORGE trial, which we expect will initiate dosing in mid-2026, marks the beginning of the expansion of apitegromab into additional rare, severe, and debilitating neuromuscular diseases where muscle strength and motor function are key unmet needs. We see potential for apitegromab broadly in neuromuscular diseases, and we are actively exploring indications beyond SMA and FSHD. Beyond apitegromab, we continue to leverage our deep expertise in myostatin biology to advance a robust pipeline of anti-myostatin therapies. Last year, our team moved SRK-439, a novel, investigational, subcutaneously administered myostatin inhibitor, into clinical development, dosing the first healthy volunteers in the fourth quarter of 2025. Preclinical data has demonstrated the potential of SRK-439 to potently inhibit myostatin and increase muscle mass, and we look forward to sharing topline results from the Phase 1 study in the second half of 2026. Together, these programs underscore our commitment to sustained innovation in myostatin biology. Thank you to our shareholders for your continued support along this journey as we work with focus, discipline, and urgency to deliver on our opportunity to serve the rare neuromuscular disease community globally. And most of all, thank you to the children and adults living with SMA whose determination, insights, and trust guide and motivate us every day. Sincerely, David Hallal Chairman and Chief Executive Officer Scholar Rock
 • shareholder letter icon 4/22/2026 Letter Continued (Full PDF)
 • stockholder letter icon 5/1/2023 SRRK Stockholder Letter
 • stockholder letter icon 4/29/2024 SRRK Stockholder Letter
 • stockholder letter icon 4/11/2025 SRRK Stockholder Letter
 • stockholder letter icon More "Biotechnology" Category Stockholder Letters
 • Benford's Law Stocks icon SRRK Benford's Law Stock Score = 66


SRRK Shareholder/Stockholder Letter Transcript:

Annual Report
2025

transition to strengthen our corporate
and operational capabilities, building
a highly experienced commercial
organization with customer-facing teams
in the U.S. and commercial build-out
in Europe. In parallel, we enhanced our
R&D leadership and clinical pipeline with
the advancement of multiple clinical
and preclinical programs to broaden our
impact for people living with rare, severe,
and debilitating neuromuscular diseases.
To Our
Shareholders:
Scholar Rock enters 2026 in a position of
strength for what promises to be a year
with many important milestones, as we
remain focused on obtaining our first
regulatory approvals for apitegromab
in the U.S. and Europe for people living
with spinal muscular atrophy (SMA). I am
gratified to have served as Scholar Rock   s
Board Chairman over the past eight
years, and it is a privilege now to lead
the organization as Chairman and
Chief Executive Officer as we transition
toward becoming a global commercialstage company.
This past year marked a pivotal chapter
for Scholar Rock. As the world leaders
in myostatin biology, we are closer
than ever to delivering the first and
only muscle-targeted treatment to the
global SMA community. Additionally, we
advanced our industry-leading antimyostatin pipeline and sharpened our
strategic focus on rare neuromuscular
diseases where muscle is a principal
organ affected by the disorder. This focus
positions us to meaningfully improve
the lives of patients worldwide while
providing an opportunity for sustainable
growth for Scholar Rock through this
decade and well into the next.
In the past year, we progressed
toward important U.S. and European
regulatory milestones for apitegromab
for the treatment of children and adults
with SMA, advancing our marketing
applications through the regulatory
process toward an anticipated approval
and commercial launch in 2026. In
preparation for this next phase, we
executed our planned leadership
In 2026, our goal is clear: To bring
apitegromab to children and adults with
SMA as quickly as possible. With the
strength of our Phase 3 SAPPHIRE clinical
data, we believe we are well-positioned
for commercial launches in the U.S. and
Europe in 2026. Our teams are working
with urgency on behalf of patients,
caregivers, and families who have long
awaited the world   s first muscle-targeted
treatment to usher in the next phase of
innovation for SMA. In the U.S., following
receipt of a Complete Response Letter,
we were pleased to hold a constructive
and collaborative Type A meeting with
the U.S. Food and Drug Administration.
Since then, we have resubmitted our
Biologics License Application. Global
commercial readiness efforts are well
underway, positioning us well for U.S. and
European launches upon approval, as we
expand toward a 50-country operating
platform to deliver on our ambition to
reach patients with SMA and other rare,
severe neuromuscular diseases globally.
Alongside our anticipated SMA launch
activities, we continue to advance our
innovative anti-myostatin pipeline with
the vision of reaching all those living
with SMA who may benefit. In late 2025,
we initiated the Phase 2 OPAL trial to
evaluate apitegromab in patients with
SMA under two years of age who are
receiving treatment with an approved
therapy for SMA, including a one-time
dose of gene therapy. We have also
been working to advance subcutaneous
apitegromab, with the completion of
a Phase 1 study in healthy volunteers,
which represents potential for additional
optionality and impact for patients.
Notably, we announced plans to
develop apitegromab for the treatment
of patients with facioscapulohumeral
muscular dystrophy (FSHD), a rare,
progressive neuromuscular disease
characterized by muscle atrophy
and functional decline, affecting
approximately 30,000 individuals
across the U.S. and Europe. There are
currently no approved treatments for
FSHD. The Phase 2 FORGE trial, which we
expect will initiate dosing in mid-2026,
marks the beginning of the expansion
of apitegromab into additional rare,
severe, and debilitating neuromuscular
diseases where muscle strength and
motor function are key unmet needs. We
see potential for apitegromab broadly
in neuromuscular diseases, and we are
actively exploring indications beyond
SMA and FSHD.
Beyond apitegromab, we continue
to leverage our deep expertise in
myostatin biology to advance a robust
pipeline of anti-myostatin therapies.
Last year, our team moved SRK-439, a
novel, investigational, subcutaneously
administered myostatin inhibitor, into
clinical development, dosing the first
healthy volunteers in the fourth quarter of
2025. Preclinical data has demonstrated
the potential of SRK-439 to potently
inhibit myostatin and increase muscle
mass, and we look forward to sharing
topline results from the Phase 1 study in
the second half of 2026. Together, these
programs underscore our commitment to
sustained innovation in myostatin biology.
Thank you to our shareholders for your
continued support along this journey
as we work with focus, discipline, and
urgency to deliver on our opportunity
to serve the rare neuromuscular disease
community globally. And most of all,
thank you to the children and adults
living with SMA whose determination,
insights, and trust guide and motivate us
every day.
Sincerely,
David Hallal
Chairman and Chief Executive Officer
Scholar Rock


shareholder letter icon 4/22/2026 Letter Continued (Full PDF)
 

SRRK Stockholder/Shareholder Letter (Scholar Rock Holding Corp) | www.StockholderLetter.com
Copyright © 2023 - 2026, All Rights Reserved

Nothing in StockholderLetter.com is intended to be investment advice, nor does it represent the opinion of, counsel from, or recommendations by BNK Invest Inc. or any of its affiliates, subsidiaries or partners. None of the information contained herein constitutes a recommendation that any particular security, portfolio, transaction, or investment strategy is suitable for any specific person. All viewers agree that under no circumstances will BNK Invest, Inc,. its subsidiaries, partners, officers, employees, affiliates, or agents be held liable for any loss or damage caused by your reliance on information obtained. By visiting, using or viewing this site, you agree to the following Full Disclaimer & Terms of Use and Privacy Policy.