Scholar Rock (NASDAQ: SRRK), a Phase 3, clinical-stage
biopharmaceutical company focused on the treatment of serious
diseases in which protein growth factors play a fundamental role,
today announced that it will be presenting additional data from the
Phase 2 TOPAZ trial extension period evaluating patient outcomes
after 24-months of treatment with apitegromab, an investigational
selective inhibitor of the activation of myostatin for spinal
muscular atrophy (SMA). An analysis of multiple efficacy endpoints,
including patient reported outcomes, from the study will be
presented as a poster at the World Muscle Society (WMS) Hybrid
Congress in Halifax, Canada on October 11-15, and a podium
presentation at the International Scientific Congress on Spinal
Muscular Atrophy in Barcelona, Spain on October 21-23.
“These findings provide further evidence of the potential of
apitegromab, which is the first and only muscle-directed myostatin
therapy to show positive proof of concept in SMA,” said Nagesh
Mahanthappa, Founding and Interim Chief Executive Officer of
Scholar Rock. “We look forward to sharing the latest information
from our clinical development program with the medical community,
in particular new data on multiple efficacy endpoints from our
TOPAZ study.”
Details of the presentations are as follows:
WMS Hybrid Congress:
Title: Apitegromab in SMA: An analysis of multiple efficacy
endpoints in the TOPAZ extension study (P.102) Presentation
Type: Poster Session 2 Presenter: Thomas Crawford, M.D.,
Professor of Neurology and Pediatrics, Johns Hopkins University
Date and Time: Wednesday, October 12, 4:00-5:30 p.m. AST
International Scientific Congress on
SMA: Title: Apitegromab in SMA: Efficacy, Safety
and PK/PD Assessments in 24 Months of TOPAZ Presentation
Type: Oral Presenter: Basil Darras, M.D., Associate
Neurologist-in-Chief, Boston Children’s Hospital; and Professor of
Neurology, Harvard Medical School Date and Time: Saturday,
October 22, Session 4 – Therapy Data Update, 11:20-12:50 p.m.
CEST
The presentations will be made available in the Publications
& Posters section of Scholar Rock’s website following the
conferences.
For conference information, visit
https://www.wms2022.com/page/programme and
https://barcelona2022.sma-europe.eu/.
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, including
humans. Scholar Rock believes that inhibiting myostatin activation
with apitegromab may result in a clinically meaningful improvement
in motor function in patients with SMA. The U.S. Food and Drug
Administration (FDA) has granted Fast Track, Orphan Drug and Rare
Pediatric Disease designations, and the European Medicines Agency
(EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal
Product designations, to apitegromab for the treatment of SMA. The
efficacy 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 SMA Spinal 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, via SMN-dependent
pathways, there continues to be a high unmet need for therapeutics
that directly address muscle function.
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, and fibrosis. 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/). Investors and
others should note that we communicate with our investors and the
public using our company website www.scholarrock.com, including,
but not limited to, company disclosures, investor presentations and
FAQs, Securities and Exchange Commission filings, press releases,
public conference call transcripts and webcast transcripts, as well
as on Twitter and LinkedIn. The information that we post on our
website or on Twitter or LinkedIn could be deemed to be material
information. As a result, we encourage investors, the media and
others interested to review the information that we post there on a
regular basis. The contents of our website or social media shall
not be deemed incorporated by reference in any filing under the
Securities Act of 1933, as amended.
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, and
progress and indication selection and development timing, 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,” “could,” “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, without limitation, 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, and
Scholar Rock’s ability to manage expenses and to obtain additional
funding when needed to support its business activities, 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, 2022, 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.
Source: Scholar Rock
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version on businesswire.com: https://www.businesswire.com/news/home/20221004005305/en/
Scholar Rock: Investors Rushmie Nofsinger Scholar Rock
rnofsinger@scholarrock.com ir@scholarrock.com 857-259-5573
Media Ariane Lovell Finn Partners ariane.lovell@finnpartners.com
media@scholarrock.com 917-565-2204
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