Scholar Rock to Present Apitegromab TOPAZ Phase 2 Pharmacologic Data at the 2021 World Congress of Neurology
03 October 2021 - 11:00PM
Business Wire
Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical
company focused on the treatment of serious diseases in which
protein growth factors play a fundamental role, today announced an
e-poster presentation of clinical results from the TOPAZ Phase 2
trial (NCT03921528) that evaluated apitegromab in patients with
Type 2 and Type 3 spinal muscular atrophy (SMA) at the 25th World
Congress of Neurology (WCN) taking place October 3-7, 2021.
Details for the virtual e-poster are as follows:
- Title: Relationship of Pharmacokinetics and
Pharmacodynamics to Apitegromab Efficacy in Patients with
Later-onset Spinal Muscular Atrophy (Types 2 and 3 SMA): Results
from the TOPAZ Study (Poster #EP1150)
- This poster will showcase positive correlations between the
magnitude of target engagement (as measured by serum levels of
latent myostatin) and motor function improvements following
treatment with apitegromab.
- Time: Virtual e-posters are available for viewing for
the duration of the congress (October 3-7, 2021) and will be
available on demand for 3 months post conference.
About Apitegromab
Apitegromab is a selective inhibitor of the activation of
myostatin and is an investigational product candidate for the
treatment of patients with 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 inhibiting myostatin activation with apitegromab may promote a
clinically meaningful improvement in motor function in patients
with SMA. The U.S. Food and Drug Administration (FDA) has granted
Fast Track (FTD), Orphan Drug (ODD) and Rare Pediatric Disease
(RPD) 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 SMA
Spinal muscular atrophy (SMA) is a rare, and often fatal,
genetic disorder that typically manifests in young children. An
estimated 30,000 to 35,000 patients are afflicted with SMA in the
United States and Europe. It is characterized by the loss of motor
neurons, atrophy of the voluntary muscles of the limbs and trunk
and progressive muscle weakness. The underlying pathology of SMA is
caused by insufficient production of the SMN (survival of motor
neuron) protein, essential for the survival of motor neurons, and
is encoded by two genes, SMN1 and SMN2. While there has been
progress in the development of therapeutics that address the
underlying SMA genetic defect, via SMN-dependent pathways, there
continues to be a high unmet need for therapeutics that directly
address muscle function.
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, the Company
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. For more information,
please visit www.ScholarRock.com or follow Scholar Rock on Twitter
(@ScholarRock) and LinkedIn
(https://www.linkedin.com/company/scholar-rock/).
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, SRK-181, and other product candidates and indication
selection and development timing, its cash runway, 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,” “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 preclinical and clinical data, including the
results from the Phase 2 trial of apitegromab, are not predictive
of, are inconsistent with, or more favorable than, data generated
from future clinical trials of the same product candidate,
including the planned Phase 3 trial of apitegromab in SMA, 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, the success of Scholar Rock’s
current and potential future collaborations, including its
collaboration with Gilead, Scholar Rock’s dependence on third
parties for development and manufacture of product candidates
including 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 June 30, 2021,
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/20211003005033/en/
Scholar Rock Contacts: Investors Catherine Hu
chu@scholarrock.com
Media Ariane Lovell Finn Partners
ariane.lovell@finnpartners.com 917-565-2204
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