- Oral presentation to include additional efficacy data from the
Phase 3 SAPPHIRE trial of apitegromab in patients with spinal
muscular atrophy (SMA)
- Poster presentations include preclinical data on a combination
treatment approach for building muscle mass and strength in a
Duchenne muscular dystrophy (DMD) model
Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical
company focused on advancing innovative treatments for spinal
muscular atrophy (SMA), cardiometabolic disorders, and other
serious diseases where protein growth factors play a fundamental
role, announced today that it will present additional data from its
Phase 3 SAPPHIRE clinical trial (NCT05156320) at the 2025 Muscular
Dystrophy Association (MDA) Clinical & Scientific Conference
being held March 16-19 in Dallas, Texas.
SAPPHIRE evaluated the safety and efficacy of apitegromab, an
investigational muscle-targeted therapy designed and developed to
provide clinically meaningful improvement in motor function for
people living with SMA who are receiving SMN-targeted treatments.
Scholar Rock previously shared positive topline data from the trial
in October 2024 and announced the submission of a Biologics License
Application (BLA) to the U.S. Food and Drug Administration (FDA)
for apitegromab in January 2025. A detailed analysis of the study
will be presented as an oral presentation by Thomas O. Crawford,
M.D., Professor of Neurology and Pediatrics at Johns Hopkins
University on March 19th, as well as through an accompanying
poster.
In addition, Scholar Rock will present a poster sharing
preclinical data on an investigational approach combining a
preclinical version of apitegromab with a dystrophin corrector for
addressing muscle defects in a Duchenne muscular dystrophy (DMD)
model. That poster will be presented by Adam I. Fogel, Ph.D.,
Director of Discovery Biology at Scholar Rock.
“We look forward to the data from our positive Phase 3 SAPPHIRE
trial of apitegromab in patients with spinal muscular atrophy being
featured at the upcoming Muscular Dystrophy Association Clinical
and Scientific Conference, along with promising preclinical data
that show the therapeutic potential for apitegromab in DMD," said
Jay Backstrom, M.D., MPH, President and Chief Executive Officer of
Scholar Rock. “Building on the success of apitegromab in SMA, we
are actively working to enhance our understanding of the role that
apitegromab can play in treating other rare neuromuscular disorders
such as DMD with the goal to reach more people living with these
serious conditions.”
Details of the presentations are as follows:
Title: Efficacy and safety of apitegromab in individuals
with type 2 and type 3 spinal muscular atrophy evaluated in the
phase 3 SAPPHIRE trial Presentation type: Oral presentation
Presenter: Thomas O. Crawford, M.D., Professor of Neurology
and Pediatrics, Johns Hopkins University Location: Hilton
Anatole Dallas, Coronado ABCD Date and time: Wednesday,
March 19, 2025, 10:45-11:00 AM CT
Title: Efficacy and safety of apitegromab in individuals
with type 2 and type 3 spinal muscular atrophy evaluated in the
phase 3 SAPPHIRE trial Presentation type: Poster
presentation (Poster #O284) Presenter: Thomas O. Crawford,
M.D., Professor of Neurology and Pediatrics, Johns Hopkins
University Location: Trinity Exhibit Hall; Hilton Anatole
Dallas Date and time: Sunday, March 16 – Tuesday, March 18,
6:00 p.m. - 8:00 p.m. CT
Title: muSRK-015 builds muscle mass and strength in
combination with dystrophin upregulation in a mouse model of DMD
Presentation type: Poster presentation (Poster #P160)
Presenter: Adam I. Fogel, Ph.D., Director, Discovery
Biology, Scholar Rock Location: Trinity Exhibit Hall; Hilton
Anatole Dallas Date and time: Sunday, March 16 – Tuesday,
March 18, 6:00 p.m. - 8:00 p.m. CT
The abstracts for these presentations are available on MDA’s
website:
https://www.mdaconference.org/abstracts/2025-abstract-library/
The presentations will be made available in the Publications
& Posters section of Scholar Rock’s website following the
conference.
For conference information, visit
https://www.mdaconference.org.
About Apitegromab
Apitegromab is an investigational fully human monoclonal
antibody, which inhibits myostatin activation by selectively
binding the pro- and latent forms of myostatin in the skeletal
muscle. It is the first muscle-targeted treatment candidate to
demonstrate clinically meaningful and statistically significant
motor function improvement in a pivotal Phase 3 trial in spinal
muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily
of growth factors, is expressed primarily by skeletal muscle cells,
and its absence is associated with an increase in muscle mass and
strength in multiple animal species, including humans. 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. Apitegromab has not been approved for any use by
the FDA or any other regulatory agency.
About the Phase 3 SAPPHIRE Trial
SAPPHIRE was a randomized, double-blind, placebo-controlled
Phase 3 clinical trial that evaluated the safety and efficacy of
apitegromab in nonambulatory patients with Types 2 and 3 SMA who
were receiving current standard of care (either nusinersen or
risdiplam). SAPPHIRE enrolled 156 patients aged 2-12 years old in
the main efficacy population. These patients were randomized 1:1:1
to receive either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or
placebo by intravenous (IV) infusion every 4 weeks for 12 months.
An exploratory population including 32 patients aged 13-21 years
old was also evaluated. These patients were randomized 2:1 to
receive either apitegromab 20 mg/kg or placebo every 4 weeks for 12
months.
The SAPPHIRE trial met its primary endpoint for the main
efficacy population with a statistically significant 1.8-point
improvement for all patients receiving apitegromab 10 mg/kg and 20
mg/kg (with an SMN-targeted treatment) compared to placebo (with an
SMN-targeted treatment), as measured by the Hammersmith Functional
Motor Scale-Expanded at week 52.
About Scholar Rock
Scholar Rock is a biopharmaceutical company that discovers,
develops, and delivers life-changing therapies for people with
serious diseases that have high unmet need. As a global leader in
the biology of the transforming growth factor beta (TGFβ)
superfamily and named for the visual resemblance of a scholar rock
to protein structures, the clinical-stage company is focused on
advancing innovative treatments where protein growth factors are
fundamental. Over the past decade, Scholar Rock has created a
pipeline with the potential to advance the standard of care for
neuromuscular disease, cardiometabolic disorders, cancer, and other
conditions where growth factor-targeted drugs can play a
transformational role.
This commitment to unlocking fundamentally different therapeutic
approaches is powered by broad application of a proprietary
platform, which has developed novel monoclonal antibodies to
modulate protein growth factors with extraordinary selectivity. By
harnessing cutting-edge science in disease spaces that are
historically under-addressed through traditional therapies, Scholar
Rock works every day to create new possibilities for patients.
Learn more about our approach at ScholarRock.com and follow
@ScholarRock and on LinkedIn.
Scholar Rock® is a registered trademark of Scholar Rock,
Inc.
Availability of Other Information About 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.
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 for apitegromab, including the therapeutic
potential, clinical benefits and safety thereof, expectations
regarding timing, success and data announcements of current ongoing
clinical trials, 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, that
preclinical and clinical data, including the results from the Phase
3 clinical trial of apitegromab, are not predictive of, may be
inconsistent with, or more favorable than, data generated from
future or ongoing clinical trials of the same product candidates,
and may not be sufficient for regulatory approval; 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;
information provided or decisions made by regulatory authorities;
competition from third parties that are 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, without limitation, to supply for apitegromab; 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 Scholar Rock’s Quarterly
Report on Form 10-Q for the quarter ended September 30, 2024, 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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version on businesswire.com: https://www.businesswire.com/news/home/20250220821289/en/
Scholar Rock: Investors Rushmie Nofsinger Scholar
Rock rnofsinger@scholarrock.com ir@scholarrock.com 857-259-5573
Media Molly MacLeod Scholar Rock mmacleod@scholarrock.com
media@scholarrock.com 802-579-5995
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