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.

 •  4/11/2025 Letter Continued (Full PDF)
 •  5/1/2023 SRRK Stockholder Letter
 •  4/29/2024 SRRK Stockholder Letter
 •  More "Biotechnology" Category Stockholder Letters
 •  SRRK Benford's Law Stock Score = 71

SRRK Shareholder/Stockholder Letter Transcript:

Annual
Report
2024

specific scale (Hammersmith Functional
Motor Scale Expanded), demonstrating
its potential to transform the lives of
people with SMA who are receiving
SMN-targeted therapies. Apitegromab
is now the first and only anti-myostatin
therapeutic candidate to demonstrate
a statistically significant functional
improvement in a pivotal Phase 3 study.
These positive results from SAPPHIRE
bring us closer to offering a much-needed
treatment option and the first muscletargeted therapy for SMA.
To Our
Shareholders:
2024 was a transformational year 2024
was a transformational year for Scholar
Rock. With the success of SAPPHIRE, our
pivotal Phase 3 trial evaluating the safety
and efficacy of apitegromab in spinal
muscular atrophy (SMA), we are on our
way to fulfilling our mission to deliver
life changing therapies and create new
possibilities for people living with serious
diseases starting with those living with
SMA. We entered 2025 on the threshold
of commercialization of apitegromab
for SMA, which if approved, will mark
our transition into a fully-integrated
commercial-stage biotechnology
company. Our achievements in the past
year have set the foundation for continued
success, giving our team tremendous
momentum to potentially launch our
first product and expand our pipeline of
clinical programs, and brought us closer to
making a meaningful difference in the lives
of people with serious disease.
SMA is a progressive neuromuscular
disorder. Despite the innovation and
improvements achieved with the current
standard of care treatments, all of which
address the SMN protein deficiency,
muscle weakness remains a core unmet
need. Those living with SMA need and
want more: more gain in motor function,
more muscle strength.
We designed and conducted SAPPHIRE
to see if we could help address the
unmet need and create new treatment
possibilities for those living with SMA. We
were thrilled to report that SAPPHIRE
achieved its primary endpoint of
statistically significant and clinically
meaningful improvement in motor
function using a gold standard, SMA-
To support the potential
commercialization of apitegromab, we
completed an upsized public offering.
We also prepared and submitted the
biologics license application (BLA) in
the United States and the marketing
authorization application (MAA) in
Europe allowing us to be on track for a
U.S. commercial launch upon approval in
2025, with European launch anticipated
in 2026. In preparation, we are engaging
with the SMA community, leading the first
ever muscle focused disease education
in SMA, building a top-tier commercial
and medical affairs organization, and
establishing the infrastructure needed to
deliver apitegromab to SMA patients if
it is approved. SMA has benefited from
three SMN-targeted therapies and the
global market has grown to $4.5 billion
annually. Our team is making excellent
progress, and we are well positioned for
a successful commercial launch. We
believe we have the right medicine and
the right market and we are executing
the right plan.
Building on the success of SAPPHIRE,
we plan to initiate the Phase 2 OPAL
clinical trial in the third quarter of 2025 to
evaluate apitegromab in SMA patients
under two years of age who have been
or are continuing to be treated with any
currently approved SMN therapy. This
trial will expand our efforts to provide
treatment options for SMA as early as
possible for people with SMA. As part
of our efforts to serve a growing number
of patients living with devastating
neuromuscular diseases, we are also
exploring development of apitegromab
in neuromuscular conditions where
progressive muscle weakness is present,
such as Duchenne muscular dystrophy
(DMD), Becker muscular dystrophy,
facioscapulohumeral muscular dystrophy
(FSHD), and amyotrophic lateral
sclerosis (ALS).
Last year, we also entered the next
frontier of our highly selective antimyostatin approach by leveraging our
platform to expand into cardiometabolic
disorders including obesity. We were
excited to initiate the Phase 2 EMBRAZE
trial exploring the combination of
apitegromab and GLP-1 receptor
agonists for people living with obesity.
Enrollment for EMBRAZE was completed
in September, and we expect to report
topline data from the trial in the second
quarter of 2025. These outcomes will
inform and guide our clinical development
plans for SRK-439, our highly selective
anti-myostatin program designed for
obesity and cardiometabolic disease,
for which we presented preclinical data
at multiple medical conferences. We
plan to submit an Investigational New
Drug (IND) Application for SRK-439
in the third quarter of 2025. We look
forward to demonstrating the potential
of our industry-leading approach to
selective myostatin inhibition to address
the ongoing challenges that patients
encounter with muscle loss and weakness
when being treated with GLP-1 receptor
agonist therapies for obesity.
As Scholar Rock evolves into a fully
integrated biotechnology company
and we prepare for our first anticipated
drug approval, I would like to express
my gratitude to our shareholders for
their support and trust in our vision, and
to recognize our employees for their
exceptional dedication to our mission of
creating possibilities for patients. I would
also like to sincerely thank the many
patients, families, and investigators who
participated, or are participating, in our
clinical programs.
With the potential for our first FDAapproved medicine on the horizon, and
as we continue to advance and expand
our promising clinical programs, Scholar
Rock is well-positioned to accelerate
breakthroughs and deliver potentially
life-changing therapies for patients.
Sincerely,
Jay T. Backstrom, MD, MPH
President & Chief Executive Officer



 4/11/2025 Letter Continued (Full PDF)