- Improvements in patient-reported outcomes
consistent with gains in motor function scores
- Safety profile at 36 months consistent with
previous reports with no new safety findings; more than 90 percent
of nonambulatory patients remained on study
- Enrollment progressing in pivotal Phase 3
SAPPHIRE registrational trial, anticipated completion in Q3
2023
- Scholar Rock to host virtual investor event
on July 12 at 9:00 AM EST to discuss the current SMA treatment
landscape and apitegromab’s potential to advance the standard of
care
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 new data from the Phase 2 TOPAZ trial extension
period evaluating patient outcomes at 36 months of treatment with
apitegromab. These data showed that continued treatment with
apitegromab over the extended treatment period was associated with
substantial and sustained improvement in motor function, as well as
improvements in patient-reported outcome measures in patients with
nonambulatory Types 2 and 3 spinal muscular atrophy (SMA) receiving
survival motor neuron (SMN)-targeted therapy. Detailed results were
presented today by Thomas Crawford, M.D., of Johns Hopkins
Medicine, and the lead principal investigator of the TOPAZ trial,
during two podium presentations at the Cure SMA Research &
Clinical Care Meeting in Orlando, Florida.
“These promising long-term data highlight the therapeutic
potential of muscle-targeted therapies, such as apitegromab, to
help those with SMA address persistent weakness,” said Dr.
Crawford. “While SMN-targeted therapies play an important role in
preventing further loss of motor neurons, many people still
experience persistent or progressive symptoms due to preexisting
motor neuron degeneration. Incorporating a muscle-targeted therapy
with apitegromab’s clinical profile into the treatment paradigm
could allow patients to sustain or potentially achieve new gains in
motor functioning.”
“We are excited to share these new Phase 2 data that support
apitegromab’s long-term durability of effect and consistent
tolerability and safety profile. The results further strengthen our
confidence in apitegromab’s therapeutic potential for patients with
SMA, as well as validate Scholar Rock’s unique approach to
selectively inhibiting the pro and latent forms of myostatin,” said
Jay Backstrom, M.D., MPH, President and Chief Executive Officer of
Scholar Rock. “In addition to the sustained benefit observed with
consistent HFMSE scores, we saw continued improvement in RULM
scores, and reductions in fatigue as reported by patients—all of
which can be important factors in performing activities of daily
living. We remain committed to advancing the standard of care for
people with SMA, and we look forward to sharing updates on our
pivotal Phase 3 SAPPHIRE trial, which we anticipate will complete
enrollment in the third quarter of 2023.”
Substantial and Sustained Gains in Motor Function Observed
Over the Extended Treatment Period: Nonambulatory patients
(ages 2-21) experienced substantial and sustained gains in
Hammersmith Functional Motor Scale-Expanded (HFMSE) and Revised
Upper Limb Module (RULM) scores over the 36-month extended
treatment period from baseline:
12-Month Data
24-Month Data
36-Month Data
Mean Change from Baseline in
HFMSE (95% Confidence Interval)
3.6 points
(1.2, 6.0)
N=32
4.2 points
(1.9, 6.6)
N=29
4.0 points
(1.0, 6.9)
N=28
Mean Change from Baseline
in RULM (95% Confidence Interval)
1.3 points
(0.2, 2.3)
N=31
2.3 points
(1.2, 3.3)
N=31
2.4 points
(1.1, 3.7)
N=27
For the 36-month data, an observed case analysis was conducted,
which pooled data for all nonambulatory patients (including those
patients on 20 mg/kg of apitegromab for the full duration of the
trial, and those who switched from 2 mg/kg to 20 mg/kg at various
time intervals in year 2) and was based upon the available data.
These analyses exclude data for patients post scoliosis
surgery.
Improvement in Patient-Reported Outcomes Consistent with
Improvements in Motor Function: Nonambulatory patients (ages
2-21) had improvements in PEDI-CAT (measure of activities of daily
living) and PROMIS-Fatigue (a patient-reported outcome tool
measuring fatigue) that were consistent and sustained at 36 months.
The mean change in PEDI-CAT daily activity domain from baseline at
36 months was 2.2 (95% CI: –0.1, 4.5; N=17), indicating an
improvement in the ability to perform daily activities. The mean
change in PROMIS-Fatigue from baseline at 36 months was –4.6 (95%
CI: –8.7, –0.5; N=14), indicating a decline in fatigue. These
improvements in PEDI-CAT and PROMIS-Fatigue were generally
consistent with improvements in motor function across the 36 months
of the study period.
Consistent Safety Data: Treatment-emergent adverse events
(TEAEs) at 36 months were consistent with previous reports at 12
and 24 months, with no new findings after an aggregate of 198
patient-years of exposure. TEAEs were mostly mild-to-moderate in
severity, and generally consistent with the underlying patient
population and background therapy. The five most common TEAEs were
headache, pyrexia, COVID-19, nasopharyngitis, and upper respiratory
tract infection. No deaths or suspected unexpected serious adverse
reactions or hypersensitivity reactions were observed with
apitegromab at 36 months. A total of 21 serious TEAEs were reported
over the 36-month treatment period. No patients displayed positive
titers for apitegromab antibodies (ADA).
More than 90 percent of nonambulatory patients remained on
treatment in the extension study.
Details of the podium presentations at SMA Research &
Clinical Care Meeting are as follows:
Title: Effect of apitegromab on PEDI-CAT and
PROMIS-Fatigue questionnaire at 36 months in patients with Type 2
and nonambulatory Type 3 spinal muscular atrophy Presentation
type: Oral presentation Presenter: Thomas O. Crawford,
M.D., Professor of Neurology and Pediatrics, Johns Hopkins
University Date and time: Friday, June 30, 2023, 10:40 AM
EST Location: Disney Swan and Dolphin Hotels, Orlando,
FL
Title: Effect of apitegromab on motor function at 36
months in patients with Type 2 and nonambulatory Type 3 spinal
muscular atrophy Presentation type: Oral presentation
Presenter: Thomas O. Crawford, M.D., Professor of Neurology
and Pediatrics, Johns Hopkins University Date and time:
Friday, June 30, 2023, 11:00 AM EST Location: Disney Swan
and Dolphin Hotels, Orlando, Florida For conference information,
visit https://www.researchandclinicalcaremeeting.com/
The presentations will be made available in the Publications
& Posters section of Scholar Rock’s website following the
presentation.
Conference Call/Webcast:
Scholar Rock will host a virtual investor event on July 12 at
9:00 AM EST to discuss the current SMA treatment landscape and
apitegromab’s potential to advance the standard of care for
patients with nonambulatory Types 2 and 3 SMA. Click here to
register and listen to the webcast. A link to the webcast of this
event is also available on the Investors & Media section of the
Scholar Rock website at http://investors.scholarrock.com.
An archived replay of the webcast will be available on Scholar
Rock’s website at: https://scholarrock.com/ for approximately 90
days following the presentation.
About the Phase 2 TOPAZ Trial
The TOPAZ trial is an ongoing proof-of-concept, open-label Phase
2 trial evaluating the safety and efficacy of apitegromab in
patients with Types 2 and 3 SMA. In the main treatment period,
patients were dosed intravenously every four weeks as monotherapy
or with nusinersen, an approved SMN-targeted therapy. The trial
enrolled 58 patients in the U.S. and Europe. The primary efficacy
endpoints were mean change from baseline in Revised Hammersmith
Scale (RHS) score at 12 months for the ambulatory population
(Cohort 1), and mean change from baseline in HFMSE score at 12
months for the nonambulatory population (Cohorts 2 and 3). The
trial also includes multiple 12-month extension periods designed to
evaluate longer-term patient outcomes.
About the Phase 3 SAPPHIRE Trial
SAPPHIRE is an ongoing randomized, double-blind,
placebo-controlled, phase 3 clinical trial evaluating the safety
and efficacy of apitegromab in nonambulatory patients with Types 2
and 3 SMA who are receiving SMN therapy (either nusinersen or
risdiplam). Approximately 156 patients aged 2-12 years old are
anticipated to be enrolled in the main efficacy population. These
patients will be 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
of approximately 48 patients aged 13-21 years old will also
separately be evaluated. These patients will be randomized 2:1 to
receive either apitegromab 20 mg/kg or placebo. For more
information about SAPPHIRE, visit www.clinicaltrials.gov.
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 our 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. 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, 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 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/).
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 other product candidates 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, that preclinical and clinical data, including
the results from the Phase 2 clinical trial of apitegromab, and are
not predictive of, may be inconsistent with, or more favorable
than, data generated from future clinical trials of the same
product candidates, including, without limitation, the Phase 3
clinical trial of apitegromab in SMA , 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 any
clinical trials, 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 the impacts of public health
pandemics 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, 2023, 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:
Investors Rushmie Nofsinger Scholar Rock
rnofsinger@scholarrock.com ir@scholarrock.com 857-259-5573
Media Teagan White Finn Partners
teagan.white@finnpartners.com media@scholarrock.com
650-766-3955
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