- Scholar Rock’s BLA submission is based on the Phase 3 SAPPHIRE
trial that demonstrated a statistically significant improvement in
motor function for patients receiving apitegromab compared to
placebo, as measured by the Hammersmith Functional Motor
Scale-Expanded at week 52
- The FDA has granted apitegromab Fast Track, Orphan Drug and
Rare Pediatric Disease Designations in SMA
- Scholar Rock remains on track to submit the apitegromab
Marketing Authorisation Application (MAA) to the European Medicines
Agency in 1Q 2025, with PRIME and Orphan Medicinal Product
Designations
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, today announced the submission of a Biologics License
Application to the U.S. Food and Drug Administration (FDA) for
apitegromab, a 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.
The Company remains on track to file a Marketing Authorisation
Application to the European Medicines Agency in 1Q 2025.
“We are gratified that in patients already on a SMN-targeted
treatment, the SAPPHIRE trial met its primary endpoint for the main
efficacy population showing a statistically significant 1.8-point
improvement for patients receiving apitegromab compared to placebo,
as measured by the Hammersmith Functional Motor Scale-Expanded at
week 52,” said Jing Marantz, M.D., Ph.D., Chief Medical Officer of
Scholar Rock. “With the strength of our Phase 3 data as the
foundation of our submission, we look forward to continuing to work
closely with the FDA through the review of our BLA on behalf of
patients and families living with SMA.”
The review of the apitegromab BLA submission will be conducted
by the Division of Neurology Products in FDA’s Center for Drug
Evaluation and Research. The submission is supported by data from
the Phase 3 SAPPHIRE trial and the Phase 2 TOPAZ trial. The Company
shared positive topline data from the SAPPHIRE trial in 2024, and
additional data will be presented at the 2025 MDA Clinical &
Scientific Conference being held March 16-19 in Dallas, Texas.
Scholar Rock has requested Priority Review which, if granted,
would shorten the FDA’s review time to six months from the date of
filing acceptance. The 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.
Scholar Rock plans to also study apitegromab in SMA patients
under two years of age in the Phase 2 OPAL trial, with a planned
initiation in mid-2025. The trial will evaluate apitegromab in
patients who have been or are continuing to be treated with any
currently approved SMN therapy (nusinersen, risdiplam and
onasemnogene abeparvovec).
About Apitegromab
Apitegromab is an investigational fully human monoclonal
antibody inhibiting 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 the
absence of its gene 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
(SMN-targeted treatments), 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 progress and
plans for apitegromab. The use of words such as “may,” “could,”
“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 for the purposes of the
safe harbor provisions under The Private Securities Litigation
Reform Act of 1995. 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 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 candidate; 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; the success
of Scholar Rock’s current and potential future collaborations;
Scholar Rock’s dependence on third parties for development and
manufacture of product candidates including, without limitation, to
supply any clinical trials; Scholar Rock’s ability to manage
expenses and to obtain additional funding when needed to support
its business activities; its ability to establish or maintain
strategic business alliances; its ability to receive priority or
expedited regulatory review or to obtain regulatory approval of
apitegromab; its ability to expand globally and the anticipated
commercial launch in the United States of apitegromab in the fourth
quarter of 2025; as well as those risks more fully discussed in the
section entitled "Risk Factors" in Scholar Rock’s Form 10-K for the
year ended December 31, 2023, and 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/20250129052122/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
Scholar Rock (NASDAQ:SRRK)
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