Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015
May 31 2018 - 7:00AM
Scholar Rock (NASDAQ:SRRK), a 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, today announced that it
has completed dosing of the first cohort and is continuing
enrollment of healthy volunteers in its Phase 1 clinical trial of
SRK-015, the Company’s lead antibody product candidate. SRK-015 is
a selective inhibitor of the activation of myostatin, which Scholar
Rock believes has the potential to be the first muscle-directed
therapy to reverse or prevent further muscle atrophy in patients
with spinal muscular atrophy (SMA) and could be used as a
monotherapy or in conjunction with the current standard of care.
“The initiation of this Phase 1 clinical trial of SRK-015 is a
momentous milestone for Scholar Rock and we are one step closer to
potentially bringing the first muscle-directed therapy to patients
suffering from SMA,” said Nagesh Mahanthappa, PhD, President and
Chief Executive Officer of Scholar Rock. “We are also excited to be
building and progressing a pipeline of novel product candidates
focused on addressing other neuromuscular disorders, cancer,
fibrosis, and anemia.”
The placebo-controlled, double-blind Phase 1 clinical trial will
evaluate the safety, tolerability, pharmacokinetics and
pharmacodynamics of single- and multiple-ascending doses of
intravenous SRK-015 in healthy adult volunteers. Assuming the
successful completion of the Phase 1 trial, the Company plans to
initiate a Phase 2 proof-of-concept trial in the first quarter of
2019 to evaluate the efficacy and safety of SRK-015 in patients
with later-onset SMA as a monotherapy or in conjunction with an
approved SMN upregulator therapy as background standard of
care.
About SRK-015SRK-015 is a selective inhibitor
of the activation of myostatin and is an investigational therapy
for the treatment of patients with spinal muscular atrophy (SMA).
Myostatin, a member of the TGF-beta superfamily of growth factors,
is expressed primarily in 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 U.S.
Food and Drug Administration (FDA) has granted Orphan Drug
Designation (ODD) for 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 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 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 RockScholar Rock is a
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.
Safe Harbor StatementVarious statements in this
presentation concerning Scholar Rock’s future expectations, plans
and prospects, including without limitation, Scholar Rock’s
expectations regarding its strategy, its product candidate
selection and development timing, including timing for the
initiation of and reporting results from its clinical trials for
its product candidates, its disease indication selection timing,
its management team capabilities, and the ability of SRK-015 to
affect the treatment of patients suffering from SMA either as a
monotherapy or in conjunction with the current standard of care,
constitute forward-looking statements for the purposes of the safe
harbor provisions under The Private Securities Litigation Reform
Act of 1995. 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. Actual results may differ materially
from those indicated by these forward-looking statements as a
result of various important factors, including, without limitation,
Scholar Rock’s ability to provide the financial support and
resources necessary to identify and develop product candidates on
the expected timeline, competition from others 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 to supply any clinical trials, and 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 as well
as those risks more fully discussed in the section entitled "Risk
Factors" in the final prospectus related to Scholar Rock’s initial
public offering filed with the Securities and Exchange Commission
pursuant to Rule 424(b) of the Securities Act, 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. Scholar Rock
explicitly disclaims any obligation to update any forward-looking
statements unless required by law.
Scholar Rock Contact:Investors/MediaCatherine
Huchu@scholarrock.com917-601-1649
Media Contact:The Yates NetworkKathryn
Morriskathryn@theyatesnetwork.com914-204-6412
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