Scholar Rock Presents Clinical and Preclinical Data for SRK-015 at the 2020 Virtual Cure SMA Research and Clinical Care Meeting
June 12 2020 - 6:30AM
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 two
presentations as part of the 2020 Virtual Cure SMA Research and
Clinical Care Meeting. The two presentations will review previously
presented data from preclinical studies, the Phase 1 healthy
volunteer trial, as well as preliminary pharmacokinetic and
biomarker data and baseline demographics from the TOPAZ Phase 2
proof-of-concept trial evaluating SRK-015 for the treatment of
patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA).
Details for the two virtual presentations at the meeting are as
follows:
- Title: Clinical Development of SRK-015, a Fully Human
Anti-proMyostatin Monoclonal Antibody, for the Treatment of
Later-Onset Spinal Muscular Atrophy
- Title: A Phase 2 Study to Evaluate the Efficacy and
Safety of SRK-015 in Patients with Later-Onset Spinal Muscular
Atrophy (TOPAZ): An Introduction
- Clinical Drug Development Session: Virtual presentations
on June 12, 2020 at 12:00-3:15pm CST.
About SRK-015
SRK-015 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 strength in multiple animal species(1).
Preclinical SMA mouse model studies have demonstrated that
selective inhibition of myostatin may have therapeutic potential in
improving muscle strength(2). Scholar Rock believes by targeting
the precursor forms of myostatin, and preventing the release of the
mature growth factor, SRK-015 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 the European Commission (EC) has granted Orphan
Medicinal Product Designation, to SRK-015 for the treatment of SMA.
The effectiveness and safety of SRK-015 have not been established.
SRK-015 has not been approved for any use by the FDA or any other
regulatory agency.
(1)
Han, H.Q., Zhou, Xiaolan, Goldberg, A. Myostatin/activin pathway
antagonism: Molecular basis and therapeutic potential.
International Journal of Biochemistry & Cell Biology, 2013,
45(10):2119-2348
(2)
Long, K., O’Shea, K., Khairallah, R., et al. Specific Inhibition
of Myostatin Activation is Beneficial in Mouse Models of SMA
Therapy. 2018. Human Molecular Genetics, ddy382
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 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 the timing of its
clinical trials for SRK-015; the potential of SRK-015 to address
certain patient unmet needs; and the ability of any product
candidate to perform in humans in a manner consistent with
nonclinical or preclinical study data. 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;
preclinical data and results may not be predictive of clinical
results; Scholar Rock’s dependence on third parties for development
and manufacture of product candidates including to supply any
clinical trials; and 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 March 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/20200612005062/en/
Scholar Rock: Investors/Media Catherine Hu
chu@scholarrock.com 917-601-1649 Media: The Yates Network
Kathryn Morris kathryn@theyatesnetwork.com 914-204-6412
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