Scholar Rock Presents TOPAZ Interim Analysis Data for Apitegromab in Spinal Muscular Atrophy at the 2021 Muscular Dystrophy A...
15 March 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 its
oral and poster presentations at the 2021 Muscular Dystrophy
Association (MDA) Virtual Clinical & Scientific Conference,
being held March 15-18.
Both presentations will include six-month interim analysis data
from the TOPAZ Phase 2 clinical trial evaluating the potential of
apitegromab, a highly selective inhibitor of latent myostatin, in
improving motor function in patients with Type 2 and Type 3 Spinal
Muscular Atrophy (SMA). Top-line clinical data from TOPAZ for the
full 12-month treatment period are expected in the second quarter
of 2021.
Oral presentation details:
- Title: Apitegromab, a Novel High-Affinity
Anti-proMyostatin Monoclonal Antibody for Treating Spinal Muscular
Atrophy: Results of a Phase 2 Interim Analysis
- Date and Time: Thursday, March 18, 2021, 2:00-2:15 pm
EST
- Presenter: Amy Place, PhD, MBA, MS, RD, CLT on behalf of
the apitegromab development team
Poster presentation details:
- Title: Apitegromab, a Novel High-Affinity
Anti-Promyostatin Monoclonal Antibody for Treating Spinal Muscular
Atrophy: Results of a Phase 2 Interim Analysis (Poster #51)
- Dates: Virtual poster presentations are available to
view on the virtual conference platform from March 15-18, 2021
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. Scholar Rock believes the inhibition of
the activation of myostatin with apitegromab may promote a
clinically meaningful improvement in motor function.
A Phase 2 clinical trial in patients with Type 2 and Type 3 SMA
is ongoing (NCT03921528). The U.S. Food and Drug Administration
(FDA) has granted Orphan Drug Designation (ODD) and Rare Pediatric
Disease (RPD) designation, and the European Commission (EC) has
granted Orphan Medicinal Product Designation, to apitegromab for
the treatment of SMA. The effectiveness 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 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,
fibrosis and anemia. 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 https://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 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, 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 Annual Report on Form 10-K for the year ended December 31,
2020, 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/20210315005127/en/
Scholar Rock: Investors/Media Catherine Hu
chu@scholarrock.com Media: Ariane Lovell Finn Partners
ariane.lovell@finnpartners.com 917-565-2204
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