- SRK-439, a myostatin inhibitor, is part of
Scholar Rock’s industry-leading anti-myostatin portfolio
- Obesity program continues to progress, with
first participants dosed in Phase 2 EMBRAZE trial of apitegromab in
obesity
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 that the first participants were dosed in the
Phase 2 EMBRAZE proof-of-concept trial, designed to assess the
safety and efficacy of apitegromab, an investigational, highly
selective myostatin inhibitor, to preserve lean muscle mass in
individuals living with obesity and on background therapy of a
GLP-1 receptor agonist (GLP-1 RA). The trial will also evaluate the
effects of apitegromab on the durability of weight loss upon
withdrawal of GLP-1 RA therapy. The results from this trial will
inform the development of SRK-439, a novel investigational
selective myostatin inhibitor optimized for the treatment of
cardiometabolic disorders, including obesity.
The Company also presented new preclinical data that support the
potential of SRK-439 to increase lean mass and contribute to a
favorable body composition following withdrawal from GLP-1 RA
treatment. These data were presented by Melissa Fulham, PhD, of
Scholar Rock, at the American Diabetes Association’s 84th
Scientific Sessions on June 23rd in Orlando, Florida.
“We are happy to share the exciting news that we’ve dosed the
first participants in our EMBRAZE clinical trial ahead of schedule
and to have new preclinical data with SRK-439, our highly selective
anti-myostatin, featured at the American Diabetes Association
Scientific Sessions,” said Jay Backstrom, M.D., MPH, President and
Chief Executive Officer at Scholar Rock. “SRK-439 preclinical data
to date have demonstrated preservation of lean mass with GLP-1
RA-induced weight loss, attenuation of fat mass regain following
GLP-1 RA withdrawal, and greater potency compared to an anti-ACTRII
antibody. Together, these data continue to support a best-in-class
potential for healthy weight loss management and could be
transformative for the management of weight loss. We are looking
forward to providing additional updates on our cardiometabolic
program as we advance SRK-439, as well as the EMBRAZE trial.”
Preclinical experimental design
For the preclinical research study, the Company tested a murine
equivalent of SRK-439 in a diet-induced obesity (DIO) mouse model.
Mice were given either a high-fat diet plus semaglutide (0.04
mg/kg, daily) and an IgG control antibody (weekly, 10 mg/kg), or a
high-fat diet plus semaglutide (0.04 mg/kg, daily) in combination
with weekly injections of SRK-439 (10 mg/kg). Following four weeks
of treatment, semaglutide was withdrawn from both treatment groups
and mice remained on either the IgG control antibody weekly or on
SRK-439. Treatment continued for another four weeks, for a total of
eight weeks in the study. Quantitative nuclear magnetic resonance
(qNMR) was used to analyze change in lean mass at two weeks and
again at four weeks of semaglutide treatment, and every two weeks
after that until the end of the subsequent four-week withdrawal
period.
Changes in body composition after semaglutide
withdrawal
The group that received SRK-439 maintained more favorable body
composition than the group receiving IgG antibody. Key findings
supporting the potential for SRK-439 in advancing healthier weight
management include:
- Administration with SRK-439 attenuated the loss of lean mass
during semaglutide treatment and significantly increased lean mass
after semaglutide discontinuation as compared to IgG control;
- SRK-439 administration also attenuated the fat mass rebound
after semaglutide discontinuation as compared to that in IgG
control + semaglutide mice; and
- Body composition, i.e. proportion of lean mass or fat mass to
total body weight, was more favorable in mice receiving SRK-439 as
compared to IgG control: Mice administered SRK-439 had higher
relative lean mass (65.8%) and lower relative fat mass (18.0%) at
the end of the withdrawal period compared to IgG control (57.1%
lean mass and 28.7% fat mass).
Shown below are results for body composition at baseline (6 days
before semaglutide treatment), the end of semaglutide treatment (at
4 weeks), and at the end of the semaglutide withdrawal period (at 8
weeks):
Endpoint (units)
IgG control +
semaglutide
SRK-439 + semaglutide
P value
Absolute lean mass (g) at
baseline
24.8
25.5
n.s.
Absolute lean mass (g) at 4
weeks
22.3
26.4
P<0.001
Absolute lean mass (g) at 8
weeks
25.1
29.4
P<0.0001
Absolute fat mass (g) at
baseline
11.8
10.3
n.s.
Absolute fat mass (g) at 4
weeks
5.9
3.8
n.s.
Absolute fat mass (g) at 8
weeks
12.7
8.3
n.s.
Relative lean mass (%) at 8
weeks
57.1%
65.8%
P<0.001
Relative fat mass (%) at 8
weeks
28.7%
18.0%
P<0.01
“These new preclinical data provide compelling evidence that
SRK-439 contributed to lean muscle preservation during GLP-1
RA-induced weight loss and attenuated fat mass rebound following
discontinuation of semaglutide,” said Mo Qatanani, PhD, Chief
Scientific Officer at Scholar Rock. “Mice receiving SRK-439
treatment had significantly more lean mass at the end of the
semaglutide withdrawal period. These exciting data continue to
support the differentiated profile of SRK-439 and its potential to
contribute to healthier weight management and long-term metabolic
benefits during and after GLP-1 RA treatment.”
For conference information, visit
https://professional.diabetes.org/scientific-sessions.
The slides from the presentation are available in the
Publications & Posters section of Scholar Rock’s website.
About EMBRAZE
EMBRAZE is a randomized, double-blind, placebo-controlled, Phase
2 proof-of-concept trial evaluating the efficacy, safety and
pharmacokinetics of apitegromab in adults with a body mass index
(BMI) of >27 (overweight) or a BMI of >30 (obese) and taking
a GLP-1 RA (tirzepatide or semaglutide). The target enrollment of
EMBRAZE is 100 subjects aged 18-65 who are overweight or obese
without diabetes. As part of the study design, the treatment period
is 24 weeks, and all subjects will receive a GLP-1 RA. In addition,
all subjects will be randomized 1:1 to receive either apitegromab
or placebo by intravenous (IV) infusion every four weeks during the
24-week treatment period. The primary endpoint is change from
baseline at Week 24 in lean mass assessed by dual-energy X-ray
absorptiometry. Secondary endpoints include additional weight loss
measures, safety and tolerability, and pharmacokinetic outcomes.
Exploratory endpoints at Weeks 24 and 32 include cardiometabolic
parameters (e.g. HbA1c), body composition, and physical
function.
About SRK-439
SRK-439 is a novel, preclinical, investigational myostatin
inhibitor that binds to pro- and latent myostatin with high
affinity and is selective for myostatin (i.e., no GDF11 or
Activin-A binding), and is initially being developed for the
treatment of cardiometabolic disorders, including obesity. Based on
preclinical data, SRK-439 has the potential to support healthier
weight management by preserving lean mass during weight loss. 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 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 to demonstrate
clinical proof-of-concept in spinal muscular atrophy (SMA).
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. The efficacy and safety of apitegromab have not been
established and apitegromab 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 and timing of its clinical trials for
apitegromab and its preclinical programs, including SRK-439, and
indication selection and development timing, including the
therapeutic potential, clinical benefits and safety thereof,
expectations regarding timing, success and data announcements of
current ongoing preclinical and clinical trials, 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 and
clinical data, including the results from the Phase 2a clinical
trial of apitegromab, or its preclinical data with respect to
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, including, without
limitation, the Phase 3 clinical trial of apitegromab in SMA or the
Phase 2a EMBRAZE clinical trial; Scholar Rock’s ability to provide
the financial support, resources and expertise necessary to
identify and develop product candidates on the expected timeline;
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; and 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; 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 March 31, 2024,
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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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
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