Scholar Rock Announces that SRK-015 has Received Rare Pediatric Disease Designation from U.S. FDA for the Treatment of Spinal...
August 13 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
that the U.S. Food and Drug Administration (FDA) has granted Rare
Pediatric Disease (RPD) designation for SRK-015 for the treatment
of Spinal Muscular Atrophy (SMA), a progressive, rare genetic
disease that leads to motor function impairments. Scholar Rock is
on track to report 6-month interim efficacy and safety data from
the ongoing TOPAZ Phase 2 clinical trial of SRK-015 in patients
with Type 2 and Type 3 SMA in the fourth quarter of 2020. Top-line
data for the 12-month treatment period are expected in the first
half of 2021.
“This Rare Pediatric Disease designation along with the
previously granted Orphan Drug Designation highlights the FDA’s
recognition of the unmet medical needs of patients with SMA. We
continue to aim to establish SRK-015 as the potential first
muscle-directed therapy to address motor function deficits that
persist despite the availability of SMN upregulators,” said Yung
Chyung, M.D., Chief Medical Officer of Scholar Rock. “We are
encouraged by the progress of the TOPAZ Phase 2 trial and look
forward to the interim read-out next quarter that will provide
important insights into the potential of SRK-015 in patients with
SMA.”
The FDA grants Rare Pediatric Disease designation for serious
and life-threatening diseases that primarily affect children ages
18 years or younger and fewer than 200,000 individuals in the
United States. If a biologics license application (BLA) for SRK-015
for the treatment of SMA is approved by the FDA, Scholar Rock may
be eligible to receive a priority review voucher, which may be
redeemed to obtain priority review for any subsequent marketing
application or be sold or transferred.
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 mass and strength in multiple animal
species. Scholar Rock believes the inhibition of the activation of
myostatin with SRK-015 may promote a clinically meaningful increase
in muscle strength. A Phase 2 clinical trial in patients with Type
2 and Type 3 SMA is ongoing. 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 SRK-015 for the
treatment of SMA. The effectiveness and safety of SRK-015 have not
been established and SRK-015 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 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 SRK-015,
SRK-181, 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 nonclinical or preclinical
study data, the potential of its proprietary platform, and the
impact of COVID-19 on its clinical trials and its business and
operations in general. 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 Quarterly Report on Form 10-Q for the quarter ended June 30,
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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Scholar Rock Contact: Investors/Media Catherine Hu
chu@scholarrock.com 917-601-1649
Media Contact: The Yates Network Kathryn Morris
kathryn@theyatesnetwork.com 914-204-6412
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