- Company plans to expand into cardiometabolic disorders,
leveraging its experience in myostatin inhibition
- Preclinical data support advancing SRK-439, a novel
investigational myostatin inhibitor for the treatment of
cardiometabolic disorders, with an initial focus on obesity,
towards an IND submission in 2025
- To inform the development of SRK-439, Company plans to initiate
a Phase 2 proof-of-concept trial of apitegromab in combination with
a GLP-1 receptor agonist in obesity in 2024 with data readout
expected in mid-2025
- Data from pivotal Phase 3 SAPPHIRE trial in SMA expected in Q4
2024
Scholar Rock (NASDAQ: SRRK), a Phase 3, clinical-stage
biopharmaceutical company focused on the treatment of serious
diseases in which protein growth factors play a fundamental role,
today announced it plans to advance SRK-439, a novel
investigational myostatin inhibitor for the treatment of obesity
towards an investigational new drug application (IND) submission in
2025. As part of the Company’s strategy to advance the development
of SRK-439, it plans to initiate a Phase 2 proof-of-concept trial
with apitegromab in combination with a GLP-1 receptor agonist
(GLP-1 RA) in 2024, subject to IND acceptance. Data from the
clinical trial are expected in mid-2025 and will be used to inform
further clinical development of SRK-439.
“As a long-standing leader in targeting myostatin with a highly
selective antibody platform, we are excited to advance our
cardiometabolic program. We have discovered multiple, highly
selective myostatin inhibitors and see potential with SRK-439 to
retain lean muscle mass, a concern that is commonly associated with
weight loss strategies, including treatments currently on the
market for obesity, a common, serious and costly public health
issue affecting adults and children globally. As apitegromab has
shown to date in spinal muscular atrophy (SMA), muscle-targeted
therapies that are well tolerated have the potential to represent a
new class of treatments across a wide range of diseases,” said Jay
Backstrom, M.D., M.P.H., President and Chief Executive Officer of
Scholar Rock. “While we continue to expand the development of our
pipeline, our top priority remains advancing the apitegromab
program in SMA. We recently completed full enrollment of the
pivotal Phase 3 SAPPHIRE trial of apitegromab in SMA, for which we
expect to read out topline data in Q4 2024.”
Cardiometabolic program
Muscle plays a key role in metabolic functions and energy
homeostasis. Leveraging proven expertise in antimyostatin and its
effect on increasing muscle mass, the Company has been developing
myostatin-selective inhibitors to address cardiometabolic
disorders, including obesity. Scholar Rock’s platform has generated
multiple antibody candidates, including apitegromab, that
selectively target pro- and latent forms of myostatin. The Company
believes the selectivity of these antibody candidates enables a
favorable risk-benefit profile for patients with cardiometabolic
disorders.
SRK-439, a novel antimyostatin antibody candidate developed by
Scholar Rock, has attractive properties, including high in vitro
affinity for pro- and latent myostatin, maintenance of myostatin
specificity (i.e., no GDF11 or Activin-A binding), and robust in
vivo efficacy in preclinical models. Studies in diet-induced obese
mice showed that SRK-439 in combination with GLP-1 receptor agonist
(GLP-1 RA) achieved:
- Dose-dependent reversal of lean mass loss during GLP-1
RA-mediated weight loss; and
- Enhancement of fat mass loss mediated by GLP-1 RA
treatment.
“Cardiometabolic disorders are creating growing global medical
challenges and contribute to the development of serious health
conditions, such as cardiovascular disease, Type 2 diabetes, and
certain cancers,” said Mo Qatanani, Ph.D., Senior Vice President
and Head of Research. “Leveraging our unique scientific platform,
Scholar Rock has developed SRK-439 with a proposed mechanism of
action that aims to safely maintain muscle mass, while enhancing
body fat loss, thereby potentially enhancing metabolic health and
potentially offering a unique opportunity to address a high unmet
need in treating metabolic conditions, including obesity. We look
forward to presenting preclinical data on this new asset from our
pipeline at upcoming scientific conferences.”
About Scholar Rock
Scholar Rock is a clinical-stage biopharmaceutical company
focused on the discovery and development of innovative medicines
for the treatment of serious diseases in which signaling by protein
growth factors plays a fundamental role. Scholar Rock is creating a
pipeline of novel product candidates with the potential to
transform the lives of patients suffering from a wide range of
serious diseases, including neuromuscular disorders, cancer, and
fibrosis. Scholar Rock’s approach to targeting the molecular
mechanisms of growth factor activation enabled it to develop a
proprietary platform for the discovery and development of
monoclonal antibodies that locally and selectively target these
signaling proteins at the cellular level. By developing product
candidates that act in the disease microenvironment, Scholar Rock
intends to avoid the historical challenges associated with
inhibiting growth factors for therapeutic effect. Scholar Rock
believes its focus on biologically validated growth factors may
facilitate a more efficient development path.
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, any development plans, strategy and progress for
apitegromab and SRK-439, the timing of its clinical trials,
anticipated clinical data, and therapeutic benefits for apitegromab
and SRK-439, and other product candidates and indication selection
and development timing, 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
apitegromab, SRK-439 and its other product candidates and its
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,
expectations regarding the therapeutic potential of SRK-439 for the
treatment of obesity, planned timing for an IND submission for
SRK-439, that preclinical and clinical data, including the results
from the Phase 2 clinical trial of apitegromab and preclinical
studies of SRK-439, are not predictive of, may be inconsistent
with, or more favorable than, data generated from future clinical
trials of the same product candidates, including, without
limitation, the Phase 3 clinical trial of apitegromab in SMA and
preclinical studies of SRK-439, 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 SAPPHIRE Phase 3 clinical
trial, information provided or decisions made by regulatory
authorities, Scholar Rock’s dependence on third parties for
development and manufacture of product candidates including,
without limitation, to supply any clinical trials, 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, 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/20231011432164/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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