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 delivering a podium presentation at the 14th European
Paediatric Neurology Society (EPNS) Congress, taking place in
Glasgow, UK from April 28 - May 2, 2022.
Details of the podium presentation are as follows:
Title: Apitegromab in Spinal Muscular Atrophy (SMA): An
Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial
- The presentation will feature an analysis of 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 SMA.
Presenter: Dr. Thomas Crawford Session: Parallel
Session 15 – Neuromuscular (PA15) Date/Time: May 1, 10:30
a.m. – 12:15 p.m. BST Location: Gaylord Scottish Event
Campus (SEC) in Glasgow, United Kingdom
The Scholar Rock booth will be located in the Exhibition Area,
Ground Floor, Hall 1 & 2 (#08) and available virtually.
For conference information, visit https://epns-congress.com/
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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220421005002/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
Scholar Rock (NASDAQ:SRRK)
Historical Stock Chart
From Mar 2024 to Apr 2024
Scholar Rock (NASDAQ:SRRK)
Historical Stock Chart
From Apr 2023 to Apr 2024