- Preclinical data show that SRK-439 increased
lean mass and lowered fat mass gain in mice receiving metformin
- Data support that Scholar Rock’s unique
approach to selective myostatin inhibition could improve body
composition in people living with obesity and on an existing
background treatment
- Scholar Rock’s industry-leading
anti-myostatin portfolio includes SRK-439 and apitegromab, the
first investigational anti-myostatin therapy to show improved motor
function in Spinal Muscular Atrophy (SMA) in a Phase 3 trial
- Obesity program progressing with Phase 2
EMBRAZE trial on track for readout in Q2 2025
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 that support the
potential of SRK-439, a highly selective investigational
antimyostatin antibody, to increase lean mass and lower fat mass
gain when taken with metformin. These data will be presented by
Melissa Fulham, Ph.D., of Scholar Rock, at the ObesityWeek
conference in San Antonio, Texas on November 5.
“These new data build upon a robust body of evidence
demonstrating the potential of selective myostatin inhibition as an
important therapeutic approach,” said Jay Backstrom, M.D., MPH,
President and Chief Executive Officer at Scholar Rock. “These
latest SRK-439 data support our hypothesis that a highly selective
approach to targeting myostatin inhibition could have positive
effects on body composition in people living with obesity and
associated comorbidities like diabetes, and also underscore the
value within Scholar Rock’s platform. We are looking forward to
providing more updates on the exciting advancements in our
cardiometabolic program, including our Phase 2 EMBRAZE trial
topline data in the second quarter of 2025.”
Preclinical study design
The research study tested a murine equivalent of SRK-439 in a
diet-induced obesity (DIO) mouse model. All mice were given a
high-fat diet, followed by either metformin (50 mg/kg daily) or
control (water) for four weeks. Following that four-week period,
mice in metformin and control groups were given either an IgG
control antibody (10 mg/kg weekly) or SRK-439 (10 mg/kg weekly for
younger mice and 3 mg/kg weekly for older mice) for another four
weeks. To assess whether effects were persistent across age ranges,
this study design was repeated in two age groups: both young
(10-week-old) and mature (22-week-old) DIO mice.
Quantitative nuclear magnetic resonance (qNMR) was used to
analyze change in lean mass at baseline, after four weeks of
metformin treatment, and every two weeks following treatment with
SRK-439 or IgG control.
Changes in body composition in the presence or absence of
metformin
The group that received SRK-439 on the background of metformin
showed a significant increase in lean mass versus the group
receiving metformin alone, and this result was consistent across
age groups. Key findings supporting the potential for SRK-439 in
advancing healthier weight management include:
- Young animals treated with SRK-439 and metformin demonstrated a
2-fold increase in lean mass over the duration of the study
compared to the lean mass increase in the metformin group and in
IgG controls (31.6% increase from baseline vs. 15.1%,
p<0.0001).
- In older metformin-treated animals, the increase in lean mass
in the SRK-439 treated animals compared to IgG controls was 50-fold
(10.2% lean mass increase from baseline versus 0.2% in IgG
controls, p<0.0001), owing primarily to the limited lean mass
growth in the IgG control group over the duration of the study due
to the age of the animals.
- In young mice, the combination treatment of SRK-439 and
metformin also resulted in lower fat mass gain (68.2% increase from
baseline) than metformin alone (114% increase from baseline;
p<0.05).
- In older mice, SRK-439 showed a trend toward reduced fat mass
gain when combined with metformin, as compared to metformin alone
(10.6% increase from baseline vs 18.4%; not statistically
significant).
“These new preclinical data show that selectively inhibiting
myostatin in combination with metformin increased lean mass—and
this effect was robust even in the older treatment group that was
more weight stable,” said Mo Qatanani, PhD, Chief Scientific
Officer at Scholar Rock. "In addition, we observed that when we
combined SRK-439 and metformin in younger mice, the effect of
lowering fat mass gain was greater than with either therapy alone.
Taken together, this is compelling evidence suggesting that SRK-439
has the potential to improve body composition and contribute to
healthier weight management in the context of both obesity and type
2 diabetes. These exciting data advance the science of obesity
research and continue to differentiate Scholar Rock’s pipeline and
anti-myostatin programs.”
Poster Presentation Information Title: SRK-439
Selectively Inhibits Myostatin to Promote Healthy Body Composition
During Metformin Therapy Presentation type: Poster
presentation Presenter: Melissa Fulham, Ph.D., Senior
Scientist II, Scholar Rock Date and time: Tuesday, November
5, 2024, 2:30 PM CT Location: Henry B. González Convention
Center, San Antonio, TX
For conference information, visit https://obesityweek.org/.
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. 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.
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
indication selection and development timing, including the timing
of any regulatory submissions, the therapeutic potential, clinical
benefits and safety of any product candidates, expectations
regarding timing, success and data announcements of current ongoing
preclinical and clinical trials, its cash runway, expectations
regarding the achievement of important milestones, 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, whether the results from
the Phase 3 SAPPHIRE trial will be sufficient to support regulatory
approval, that the full results from the Phase 3 SAPPHIRE trial may
differ from the topline data, that preclinical and clinical data,
including the results from the Phase 2 or Phase 3 clinical trial of
apitegromab, 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; 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, and our ability to continue as a
going concern; 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 June 30, 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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