- Phase 2 EMBRAZE proof-of-concept trial designed to assess
apitegromab's ability to safely preserve lean muscle mass in
individuals on GLP-1 receptor agonist therapy for obesity
- New preclinical head-to-head comparison shows that SRK-439 is
more potent than an anti-ActRII antibody in maintaining lean mass
in diet-induced obesity (DIO) mice; lean mass loss with SRK-439,
1mg/kg dose was equivalent to anti-ActRII antibody, 20mg/kg
dose
- Company hosting Investor Event today in New York City focusing
on its selective latent myostatin inhibition programs in Spinal
Muscular Atrophy and obesity with presentations from executive
leadership and key experts
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 the initiation of the Phase 2 EMBRAZE
proof-of-concept trial, designed to assess the safety and efficacy
of apitegromab, a 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
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 announced new preclinical data from a
head-to-head comparison of SRK-439 and an anti-activin receptor II
(anti-ActRII) antibody, which demonstrate SRK-439’s potential as
best in class in preserving lean mass in patients on GLP-1 RAs.
This data will be presented by Mo Qatanani, Ph.D., Chief Scientific
Officer, at Scholar Rock’s Investor Event, which begins today at
8:30 a.m. ET and is being held in New York City.
“We are thrilled to have initiated the EMBRAZE clinical trial
ahead of schedule and to share new data from our SRK-439 program,
which we believe further support our hypothesis that selective
latent myostatin inhibition has advantages over less selective
approaches to safely and effectively maintain lean muscle mass,”
said Jay Backstrom, M.D., MPH, President and Chief Executive
Officer at Scholar Rock. “Selectivity is key in mitigating
potential safety concerns for this patient population and we look
forward to sharing additional preclinical data at the American
Diabetes Association 84th Scientific Sessions in June to support
the best-in-class potential of SRK-439.”
New SRK-439 Data
For the head-to-head preclinical research study, the Company
generated and tested an anti-ActRII antibody (a murine equivalent
of bimagrumab), along with a murine equivalent of SRK-439 in a
weight-stable diet induced obesity (DIO) mouse model. Mice were
given either semaglutide (0.04mg/kg, daily) with an IgG control
antibody (weekly, 20mg/kg) or semaglutide (0.04mg/kg, daily) in
combination with weekly injections of either SRK-439 (0.3-10mg/kg)
or of the anti-ActRII antibody (0.3-20mg/kg). Quantitative nuclear
magnetic resonance (qNMR) was used to analyze change in lean mass
after four weeks of treatment.
Lean mass differences were significant in all doses of SRK-439
tested, supporting the hypothesis that SRK-439 could be an
important therapy to aid in lean mass preservation and is suitable
for subcutaneous dosing in a population of adults with obesity.
- SRK-439 attenuated the GLP-1 RA-driven lean mass loss in
combination with semaglutide at a dose as low as 0.3 mg/kg (8.3%
lean mass loss from baseline).
- Maximal effects observed at all doses over 1 mg/kg (4.2% lean
mass loss from baseline at 10mg/kg), as compared to an IgG control
+ semaglutide (14.1% lean mass loss from baseline).
- Superiority to anti-ActRII antibody was shown in all equivalent
doses tested:
- 3 mg/kg: -4.7% SRK-439 vs. -12.0% for anti-ActRII
- 1 mg/kg: -5.0% SRK-439 vs. -12.6% for anti-ActRII
- 0.3 mg/kg: -8.3% SRK-439 vs. -15.4% for anti-ActRII
- Equivalent lean mass preservation was seen at the highest dose
tested for both drugs; 10 mg/kg SRK-439 (-4.2%) and 20 mg/kg
anti-ActRII (-4.3%).
“The data from this head-to-head comparison show that
selectively inhibiting myostatin alone is sufficient to preserve
lean mass on a background of GLP-1 RA, in preclinical models.
Combined with the positive data observed at low doses of SRK-439,
we believe that our selective myostatin approach is the right
potential solution to preserve lean muscle mass while avoiding the
potential off-target effects observed in less selective programs
for this indication. We view this as strong evidence that SRK-439
could have a more favorable benefit/risk profile,” said Mo
Qatanani, Ph.D., Chief Scientific Officer at Scholar Rock.
Phase 2 EMBRAZE Trial Design
Scholar Rock announced in January that the U.S. Food and Drug
Administration cleared the Company’s Investigational New Drug (IND)
application for EMBRAZE, 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.
Primary data from EMBRAZE are expected in mid-2025 and will
inform Scholar Rock’s development of SRK-439 towards an anticipated
IND filing in 2025.
The presentation from Scholar Rock’s Investor Day will be
available in the Investors and Media section of Scholar Rock’s
website. Live webcast of the event may be accessed by visiting the
Investors & Media section of the Scholar Rock website at
http://investors.scholarrock.com. An archived replay of the webcast
will be available on the Company’s website for approximately 90
days following the presentations.
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 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240522236452/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
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