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
that it is presenting encore data in an e-poster at the American
Academy of Neurology (AAN) 2022 Annual Meeting, taking place in
Seattle, Washington (April 2-7) and virtually (April 24-26).
Details of the poster are as follows:
Title: Apitegromab in Spinal Muscular Atrophy (SMA): An
Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial
(P15.005)
- The poster will present multiple efficacy endpoints, including
Hammersmith scales and Revised Upper Limb Module (RULM) function,
from the 12- month TOPAZ Phase 2 trial evaluating apitegromab in
patients with Types 2 and 3 Spinal Muscular Atrophy (SMA).
Authors: T.O. Crawford, B.T. Darras, J.W. Day, et al. On
behalf of the TOPAZ Study Team Poster Session: P15: Child
Neurology: SMA/DMD/Muscle 1 Date/Time: Wednesday, April 6,
5:30 - 6:30 p.m. PDT (Poster session with Authors)
The e-poster is available live, April 2-7 and virtually from
April 24-26 and on-demand thereafter until May 14. These data were
first presented at the World Muscle Society 2021 Virtual Congress
in September.
For conference information, visit https://www.aan.com/events
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/). 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 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 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 are not
predictive of, may be inconsistent with, or more favorable than,
data generated from future clinical trials of the same product
candidate, including, without limitation, the Phase 3 clinical
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, 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 Annual Report on Form 10-K for the
year ended December 31, 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/20220322005157/en/
Scholar Rock: Investors Rushmie Nofsinger Scholar
Rock rnofsinger@scholarrock.com ir@scholarrock.com 857-259-5573
Media Ariane Lovell Finn Partners
ariane.lovell@finnpartners.com media@scholarrock.com
917-565-2204
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