Scholar Rock to Present Apitegromab Clinical Data from the TOPAZ Phase 2 Trial in Patients with Type 2 & 3 Spinal Muscular At...
07 June 2021 - 9:30PM
Business Wire
- Lead principal investigator, Dr. Thomas
Crawford, to present TOPAZ results showing apitegromab’s
therapeutic potential to improve motor function in patients with
Spinal Muscular Atrophy (SMA)
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 an
oral presentation of apitegromab TOPAZ Phase 2 clinical trial
results at the 2021 Virtual SMA Research and Clinical Care Meeting,
being held in conjunction with Cure SMA’s Annual SMA Conference
June 7-11, 2021.
Details for the virtual Cure SMA oral presentation are as
follows:
- Title: TOPAZ: A Phase 2 Study to Evaluate the Efficacy
and Safety of Apitegromab (SRK-015) in Patients with Later-Onset
Spinal Muscular Atrophy (Type 2 and Type 3 SMA): Topline
Results
- Presenter: Thomas Crawford, M.D., Co-Director, Muscular
Dystrophy Association Clinic and Professor of Neurology, Johns
Hopkins Medicine (Lead TOPAZ Principal Investigator)
- Clinical Drug Development Session: Virtual oral
presentation on June 11, 2021 at 2:00pm CST
Scholar Rock recently announced positive 12-month top-line data
from the TOPAZ Phase 2 trial (NCT03921528), which evaluated
apitegromab, a selective inhibitor of myostatin activation, in
patients with Type 2 and 3 SMA. The results show the transformative
potential of apitegromab to further improve motor function in
patients with non-ambulatory Type 2 and 3 SMA who were already
receiving chronic maintenance therapy with a background SMN
upregulator (nusinersen). A Phase 3 registrational trial in
patients with non-ambulatory Type 2 and Type 3 SMA is anticipated
to initiate by the end of 2021.
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 promote a
clinically meaningful improvement in motor function in patients
with SMA. The U.S. Food and Drug Administration (FDA) has granted
Fast Track (FTD), Orphan Drug (ODD) and Rare Pediatric Disease
(RPD) 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,
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/).
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 expectations regarding its growth, strategy, progress and
timing of its clinical trials and the ability of any product
candidate to perform in humans in a manner consistent with earlier
nonclinical, preclinical or clinical trial 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, the data generated from Scholar Rock’s
nonclinical and preclinical studies and clinical trials 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 March 31, 2021,
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 Contacts: Investor Contact: Catherine
Hu chu@scholarrock.com Media Contact: Ariane Lovell Finn
Partners ariane.lovell@finnpartners.com 917-565-2204
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