- Preclinical findings presented at Keystone
Symposia show lean mass preservation, fat mass loss, and improved
glucose metabolism
- Scholar Rock’s novel myostatin inhibitor,
SRK-439, is part of its growing, industry-leading portfolio of
innovative, highly selective anti-myostatin treatments under
development
Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical
company focused on advancing innovative treatments for spinal
muscular atrophy (SMA), cardiometabolic disorders, and other
serious diseases where protein growth factors play a fundamental
role, today announced new preclinical data showing the potential of
SRK-439 to preserve lean mass and improve metabolic health as part
of healthy weight loss. These data showed that SRK-439 maintained
lean mass and improved fat mass loss when used in combination with
a GLP-1 receptor agonist (GLP-1 RA; in separate experiments with
semaglutide and liraglutide) in diet-induced obesity (DIO) mice.
SRK-439 treatment also led to incremental lowering of fasting
glucose beyond the levels seen with semaglutide alone. Detailed
results were presented by Melissa Fulham, PhD, of Scholar Rock, at
the Keystone Symposia’s Obesity: Causes and Consequences meeting in
Vancouver, BC, Canada on February 5.
“These preclinical data showing that SRK-439 preserves lean mass
and improves fat mass loss provide compelling scientific rationale
to study SRK-439 in combination with GLP-1 RA therapies for healthy
weight loss management,” said Jay Backstrom, M.D., MPH, President
and CEO of Scholar Rock. “We believe that preserving lean muscle
mass through our highly selective approach to myostatin inhibition
in combination with GLP-1 RA therapy has the potential to transform
the management of weight loss. We look forward to initiating our
proof-of-concept study with apitegromab in combination with GLP-1
RA therapy in obesity as we also continue to advance SRK-439 to
clinic.”
In January, Scholar Rock announced that the U.S. Food and Drug
Administration cleared the company’s Investigational New Drug (IND)
application for its Phase 2 proof-of-concept trial of apitegromab
to treat obesity in patients taking a GLP-1 RA. Trial initiation is
on track for mid-2024, and data from the apitegromab Phase 2 trial
are expected in mid-2025. In parallel, Scholar Rock is developing
SRK-439, a novel investigational selective myostatin inhibitor,
optimized for the treatment of obesity.
Selectivity and affinity: SRK-439 works by selectively
binding to the pro- and latent forms of myostatin, as confirmed
through in vitro ELISA testing that shows SRK-439 does not bind to
closely related TGFβ family members GDF11 and Activin A, both of
which, if inhibited, have potentially detrimental effects outside
of the muscle. The studies confirmed that SRK-439 binds to latent
myostatin with 0.579 nM affinity. This selectivity and affinity,
along with favorable developability characteristics and durable
pharmacokinetics suggests that SRK-439 could be suitable for dosing
in a subcutaneous formulation and in a low dose volume in a
population of adults with obesity.
Changes in body weight, lean mass, and fat mass:
Quantitative nuclear magnetic resonance (qNMR) was used to analyze
body composition in DIO mice in two separate experiments. In the
first, mice received either liraglutide, 0.06 mg/kg daily, or
liraglutide, at the same dose, plus SRK-439 in either a 0.3, 1.0,
or 3.0 mg/kg weekly dose. In the second experiment, DIO mice
received either semaglutide, 0.04 mg/kg daily or semaglutide, at
the same dose, plus SRK-439 in either a 0.1, 0.3, 1.0, or 3.0 mg/kg
weekly dose. Total body weight, lean mass, and fat mass were
assessed. As expected, both liraglutide and semaglutide reduced
body weight in DIO mice compared to baseline. SRK-439 diminished
the GLP-1 RA-driven lean mass loss in combination with semaglutide
(–7.55% to –1.43% change from baseline in lean mass) and with
liraglutide (1.98% to 5.55% from baseline). These results were
dose-dependent: lean mass was preserved more as the dose of SRK-439
increased. SRK-439 also improved fat mass loss (–36.60% to –46.32%
from baseline with semaglutide; –17.31% to –19.04% from baseline
with liraglutide).
Results below are shown for the 3 mg/kg dose group of SRK-439 as
compared to the IgG control group in each experiment (IgG +
semaglutide or IgG + liraglutide), with the full results available
on the poster in the Posters and Presentations section of the
Scholar Rock website.
Experiment 1: Liraglutide
Experiment 2: Semaglutide
IgG Control
SRK-439 (3mg/kg)
IgG Control
SRK-439 (3mg/kg)
Change in Lean Mass from Baseline
–4.5% (n=8; p<0.0001)
5.6% (n=8; p<0.0001)
–11.3% (n=8; P<0.0001)
–1.4% (n=8; p<0.0001)
Change in Fat Mass from Baseline
0.4% (n=8; p<0.001)
–18.6% (n=8; ns)
–36.6% (n=8; ns)
–37.99 (n=8; ns)
Changes in fasting glucose Fasting glucose was measured
on Day 18 of the study in DIO mice that had received treatment with
either semaglutide or semaglutide in combination with SRK-439. Mean
fasting glucose levels were lower in mice receiving SRK-439, with
results for 0.3, 1.0, and 3.0 mg/kg reaching significance compared
to semaglutide + IgG control alone (103.0 – 107.8 mg/dL;
p<0.05).
For conference information, visit
https://www.keystonesymposia.org/.
The poster is available in the Publications & Posters
section of Scholar Rock’s website.
About SRK-439
SRK-439 is a novel, preclinical, investigational myostatin
inhibitor that has high in vitro affinity for pro- and latent
myostatin and maintains myostatin specificity (i.e., no GDF11 or
Activin-A binding), and is initially being developed for the
treatment of obesity. Based on preclinical data, SRK-439 has the
potential to support healthier weight management by preserving lean
mass. The efficacy and safety of SRK-439 have not been established
and SRK-439 has not been approved for any use by the FDA or any
other regulatory agency.
About Scholar Rock
Scholar Rock is a biopharmaceutical company that discovers,
develops, and delivers life-changing therapies for people with
serious diseases that have high unmet need. As a global leader in
the biology of the transforming growth factor beta (TGFβ)
superfamily of cell proteins and named for the visual resemblance
of a scholar rock to protein structures, the clinical-stage company
is focused on advancing innovative treatments where protein growth
factors are fundamental. Over the past decade, Scholar Rock has
created a pipeline with the potential to advance the standard of
care for neuromuscular disease, cardiometabolic disorders, cancer,
and other conditions where growth factor-targeted drugs can play a
transformational role.
Scholar Rock is the only company to show clinical
proof-of-concept for a muscle-targeted treatment in spinal muscular
atrophy (SMA). This commitment to unlocking fundamentally different
therapeutic approaches is powered by broad application of a
proprietary platform, which has developed novel monoclonal
antibodies to modulate protein growth factors with extraordinary
selectivity. By harnessing cutting-edge science in disease spaces
that are historically under-addressed through traditional
therapies, Scholar Rock works every day to create new possibilities
for patients. Learn more about our approach at ScholarRock.com and
follow @ScholarRock and on LinkedIn.
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 Twitter and
LinkedIn. The information that we post on our website or on 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. Scholar Rock® is a registered trademark of
Scholar Rock, Inc.
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 expectations regarding its growth,
strategy, progress, results and timing of its clinical trials for
apitegromab and its preclinical programs, including SRK-439, 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, that preclinical
data 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
provide the financial support, resources and expertise necessary to
identify and develop product candidates on their expected
timelines; the data generated from Scholar Rock’s nonclinical and
preclinical studies and clinical trials; 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; 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 manage expenses and to obtain additional funding when
needed to support its business activities and establish and
maintain strategic business alliances and new business initiatives,
and the impacts of public health pandemics such as COVID-19 on
business operations and expectations, 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
September 30, 2023, 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240206102641/en/
Scholar Rock: Investors Rushmie Nofsinger Scholar
Rock rnofsinger@scholarrock.com ir@scholarrock.com 857-259-5573
Media Molly MacLeod Scholar Rock mmacleod@scholarrock.com
media@scholarrock.com 802-579-5995
Grafico Azioni Scholar Rock (NASDAQ:SRRK)
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Da Dic 2024 a Gen 2025
Grafico Azioni Scholar Rock (NASDAQ:SRRK)
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