- Apitegromab met primary endpoint with statistically significant
and clinically meaningful improvement in motor function as measured
by the gold standard Hammersmith Functional Motor Scale Expanded
(HFMSE) for patients with SMA receiving apitegromab versus placebo
(current standard of care) at week 52
- 30.4% of patients receiving apitegromab had >3 point improvement in HFMSE versus 12.5% of
patients on placebo
- Patients receiving apitegromab demonstrated early motor
function improvement compared to placebo from the first measured
time point at 8 weeks, benefit observed at 52 weeks as measured by
HFMSE
- Patients receiving apitegromab experienced clinically
meaningful benefit in motor function across all age groups (ages
2-21)
- Favorable safety profile in SAPPHIRE consistent with
apitegromab’s long-term safety profile observed in the Phase 2
TOPAZ trial with >48 months of treatment experience in SMA
patients
- Scholar Rock plans to submit a U.S. Biologics License
Application and European Union marketing authorisation application
in Q1 2025
- Scholar Rock to host Investor Call today at 8:00 AM ET
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 positive topline results from the Phase 3
SAPPHIRE clinical trial (NCT05156320) evaluating the efficacy and
safety of apitegromab, an investigational muscle-targeted therapy,
in patients with SMA.
The study achieved its primary endpoint demonstrating a
statistically significant and clinically meaningful improvement for
apitegromab versus placebo in motor function as measured by the
gold standard HFMSE in SMA patients on chronic dosing of standard
of care therapies (either nusinersen or risdiplam). Based upon the
similar pharmacological profile of the 20 mg/kg and 10 mg/kg doses
of apitegromab, the statistical analysis plan was prespecified to
analyze both the combined dose (10 mg/kg and 20 mg/kg) compared to
placebo, and 20 mg/kg dose each compared to placebo. Statistical
significance is achieved per the prespecified statistical analysis
plan (Hochberg multiplicity adjustment) where the p-value (≤0.025)
is more rigorous if only one prespecified analysis crosses the
statistical significance boundary of ≤ 0.05.
- In the main efficacy population (ages 2-12), the mean
difference in change from baseline in HFMSE was 1.8 points
(p=0.0192) for all patients receiving apitegromab 10 mg/kg and 20
mg/kg (n=106) compared to placebo (n=50). Patients receiving 20
mg/kg of apitegromab (n=53) showed a 1.4 point mean difference
compared to placebo (p=0.1149).
- The prespecified analysis of the 10 mg/kg dose showed that
patients receiving 10 mg/kg of apitegromab (n=53) showed an
improvement of 2.2 points (nominal p=0.0121) compared to
placebo.
- Based upon PK/PD data from the SAPPHIRE trial, similar levels
of target engagement were observed for the 10 mg/kg and 20 mg/kg
dose groups.
Motor function outcomes were meaningful and consistent across
the main efficacy population and in the ages 13-21 exploratory
population, favored apitegromab (n=22) compared to placebo (n=10).
Thirty percent of patients receiving apitegromab had >3 point improvement in HFMSE versus 12.5% of
patients on placebo. Patients receiving apitegromab demonstrated
early motor function improvement compared to placebo from the first
measured time point at 8 weeks, benefit expanded at 52 weeks as
measured by HFMSE. Following trial completion, 98 percent of
SAPPHIRE patients (185/188) enrolled in the ongoing ONYX open-label
expansion study.
“We are thrilled that apitegromab met the primary endpoint in
our Phase 3 SAPPHIRE clinical study. The results clearly
demonstrate robust and clinically meaningful improvement in motor
function in patients with SMA,” said Jay Backstrom, M.D., MPH,
President and Chief Executive Officer of Scholar Rock. “At Scholar
Rock, we are working with urgency to deliver the potentially
transformative benefits of apitegromab to children and adults with
SMA in the US, Europe, and around the world.”
Treatment with apitegromab was well-tolerated across all age
groups. There were no clinically relevant differences in the
adverse event profile by dose, 10 mg/kg versus 20 mg/kg. No new
safety findings were observed in the SAPPHIRE clinical trial; the
profile was consistent with that observed in the Phase 2 TOPAZ
clinical trial, including an extension study which had over four
years of treatment as of the cut-off date. Serious adverse events
(SAEs) were consistent with the underlying disease and current
standard of care received by patients; no SAEs were assessed as
related to apitegromab. There were no study drug discontinuations
due to adverse events.
“We are grateful to the families and investigators who
participated in our trials. The positive Phase 3 SAPPHIRE trial,
along with over 4 years of TOPAZ clinical trial data, clearly
demonstrate the potentially transformative benefit of apitegromab
to drive clinically meaningful improvements in motor function as
measured by HFMSE in a broad SMA population, where motor function
would normally be expected to generally decline over time,” said
Jing Marantz, M.D., Ph.D., Chief Medical Officer at Scholar Rock.
“We look forward to submitting our applications to the FDA and the
EMA in Q1 2025.”
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 Company plans to submit a U.S.
Biologics License Application (BLA) and a European Union marketing
authorisation application (MAA) in Q1 2025.
“It’s a great day for people living with SMA and their families.
These encouraging trial results mark a critical milestone for the
SMA community,” said Kenneth Hobby, President of Cure SMA.
“Declining motor function and hopes for reversing losses associated
with muscle weakness are significant unmet needs, impacting
activities of daily living, from breathing, eating, self-care, to
working and social interactions. We need an approved therapy that
can support motor function and further improve daily activities for
people with SMA.”
Analyses of the full Phase 3 SAPPHIRE data are ongoing, and
Scholar Rock plans to present detailed results at an upcoming
medical conference in early 2025. Preliminary baseline
characteristics from the trial will be presented during a poster
presentation at the upcoming 29th Annual Congress of the World
Muscle Society on Friday, October 11, 2024, being held in Prague,
Czech Republic.
Conference Call Information
Scholar Rock will hold an investor conference call today,
October 7 at 8:00 am ET. To access the live conference call,
participants may register here. The live audio webcast of the call
will be available under “Events and Presentations” in the Investor
Relations section of the Scholar Rock website at
http://investors.scholarrock.com. To participate via telephone,
please register here. Upon registration, all telephone participants
will receive a confirmation email detailing how to join the
conference call, including the dial-in number along with a unique
passcode and registrant ID that can be used to access the call. An
archived replay of the webcast will be available on the Company’s
website for approximately 90 days.
Presentation at Annual Congress of the World Muscle
Society
Scholar Rock will present baseline characteristics from the
SAPPHIRE trial in a poster presentation at the 29th Annual Congress
of the World Muscle Society. Details of the presentation are as
follows:
Title: Apitegromab in Spinal Muscular Atrophy: baseline
characteristics of participants enrolled in the phase 3 SAPPHIRE
study Presentation type: Poster presentation
Presenter: Thomas O. Crawford, M.D., Professor of Neurology
and Pediatrics, Johns Hopkins University Date and time:
Friday, October 11, 2024, 3:45 PM CET Location: Prague,
Czech Republic
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
clinical proof-of-concept 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. Scholar Rock believes
that its highly selective targeting of pro- and latent forms of
myostatin with apitegromab may lead to 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. Apitegromab has not been approved for any use by the FDA or
any other regulatory agency.
About SAPPHIRE
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
are 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 for 12 months either apitegromab 10 mg/kg, apitegromab
20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks. An
exploratory population that enrolled 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.
About SMA
Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular
disease that afflicts an estimated 30,000 to 35,000 people in the
United States and Europe. The disease is characterized by the loss
of motor neurons, atrophy of the voluntary muscles of the limbs and
trunk, and progressive muscle weakness. While there has been
progress in the development of therapeutics that address the loss
of motor neurons, there continues to be a high unmet need for
therapies that directly address the progressive muscle weakness
that leads to loss of motor function in SMA.
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 of cell proteins 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.
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.
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, progress and timing of its clinical trials for
apitegromab, and indication selection and development timing,
including the timing of any regulatory submissions, the therapeutic
potential, clinical benefits and safety of any product candidates,
expectations regarding timing, success and data announcements of
current ongoing preclinical and clinical trials, its cash runway,
expectations regarding the achievement of important milestones, 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, whether the results
from the Phase 3 SAPPHIRE trial will be sufficient to support
regulatory approval, that the full results from the Phase 3
SAPPHIRE trial may differ from the topline data, that preclinical
and clinical data, including the results from the Phase 2 or 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;
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 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, and our ability to continue as a
going concern; 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, 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/20241007599288/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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