- Randomized Phase 2 proof-of-concept trial with apitegromab in
patients with obesity on GLP-1 therapies on track to initiate
mid-2024
- Study aims to show the importance of selective myostatin
inhibition in preservation of lean muscle mass as part of healthy,
safe weight loss management
- Scholar Rock is also developing SRK-439, a novel
investigational selective myostatin inhibitor, optimized for the
treatment of obesity
Scholar Rock (NASDAQ: SRRK) (the Company), 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 U.S. Food and
Drug Administration (FDA) has 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 receptor
agonist (GLP-1 RA).
The Phase 2 trial is a randomized, double-blind,
placebo-controlled, multi-center study to evaluate the effect of
apitegromab, a highly selective myostatin inhibitor, to safely
preserve lean muscle mass as an adjunctive therapy in overweight
and obese adults who are 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. The Company plans to file
an IND for SRK-439 in 2025.
“The FDA’s acceptance of our IND application to study
apitegromab in obesity allows us to assess the effect of our highly
selective myostatin inhibitor on preserving lean muscle mass, and
safety and tolerability of our approach when combined with a GLP-1
RA. The IND builds on our encouraging apitegromab clinical and
safety data to date and allows us to inform the development of our
cardiometabolic program with SRK-439, a novel preclinical selective
myostatin inhibitor optimized for development in cardiometabolic
disorders,” said Jay Backstrom, M.D., MPH, President and Chief
Executive Officer of Scholar Rock. “Maintaining lean mass during
weight loss is important to overall metabolic health, and we look
forward to initiating the Phase 2 proof-of-concept trial in
apitegromab to validate our differentiated approach of selectively
targeting only the pro- and latent forms of myostatin to retain
muscle mass.”
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 and enhancing fat mass 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, results and timing of its clinical trials for
apitegromab and its preclinical programs, including SRK-439,
regulatory feedback including with respect to the IND submitted in
connection with the planned Phase 2 trial of apitegromab in
combination with GLP-1 receptor agonists in obesity, 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, 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, that preclinical and clinical data, including
the results from the Phase 2 clinical trial of apitegromab in SMA
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 Phase 2 clinical trial of
apitegromab in obesity; 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/20240123203205/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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