Scholar Rock Announces Positive Opinion by the European Medicines Agency on Orphan Drug Designation for SRK-015 for the Treat...
December 03 2018 - 3:05PM
Scholar Rock Holding Corporation (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 European Medicines Agency’s (EMA’s)
Committee for Orphan Medicinal Products (COMP) has adopted a
positive opinion recommending SRK-015 (human anti-promyostatin
monoclonal antibody) for designation as an orphan medicinal product
for the treatment of Spinal Muscular Atrophy (SMA). SRK-015
was previously granted orphan drug designation by the U.S. Food and
Drug Administration (FDA) in March 2018.
“We are delighted with the EMA COMP’s adoption of a positive
opinion for SRK-015 orphan drug designation, which represents
another important milestone for this clinical program,” said Nagesh
Mahanthappa, PhD, President and CEO of Scholar Rock. “We
believe SRK-015 has the potential to be the first muscle-directed
therapy to improve muscle function in patients with SMA and look
forward to working with the EMA as we progress the program,
including our intention to initiate a Phase 2 proof-of-concept
study in the first quarter of 2019.”
Orphan Medicinal Product Designation by the European Commission
is available to companies developing products intended to treat a
life-threatening or chronically debilitating condition that affects
fewer than five in 10,000 people in the EU, and has the potential
to be of significant benefit. This designation could allow
for a number of incentives, including protocol assistance, access
to the centralized authorization procedure, reduced regulatory
fees, and a 10-year period of marketing exclusivity in the EU after
product approval.
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 TGF-beta
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 mass and strength. A Phase 1
clinical trial in healthy volunteers is ongoing. The
effectiveness and safety of SRK-015 have not been established and
SRK-015 has not been approved by the FDA or any other
regulatory agency.
About SMASpinal 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, there
continues to be a high unmet need for therapeutics that directly
address muscle atrophy.
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 newly elucidated
understanding of 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 StatementsThis 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 potential of SRK-015 as a
therapy in SMA and the timeline for and progress in developing
SRK-015. 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 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 September 30, 2018, 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.
Scholar Rock Contact:
Investors/Media
Catherine Hu, 917-601-1649
chu@scholarrock.com
Media Contact:
The Yates Network
Kathryn Morris, 914-204-6412
kathryn@theyatesnetwork.com
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