- Topline data from Phase 3 trial expected in Q4 2024
- Company to present encore 36-month efficacy, safety, and
patient-reported outcomes data from the Phase 2 TOPAZ trial
extension at the 2023 World Muscle Society (WMS) Annual
Congress
- Apitegromab is the only muscle-targeted therapy candidate with
clinical data showing proof of concept in spinal muscular
atrophy
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,
announced today that it has completed enrollment of the SAPPHIRE
Phase 3 trial evaluating the safety and efficacy of apitegromab in
nonambulatory patients with Types 2 and 3 spinal muscular atrophy
(SMA) who are receiving SMN therapy (either nusinersen or
risdiplam). The company also announced that it plans to present
encore data from its Phase 2 TOPAZ trial extension period
evaluating patient outcomes in SMA after 36 months of treatment
with apitegromab. Efficacy and safety data at 36 months, and an
analysis of patient-reported outcome measures such as fatigue,
mobility, and activities of daily living, will be presented during
two poster presentations at the 28th Annual Congress of the World
Muscle Society from October 3 – 7 in Charleston, South Carolina,
USA.
“Completing SAPPHIRE enrollment is a critical step on the path
to bringing apitegromab, a potentially transformative therapy, to
individuals living with SMA. SAPPHIRE was designed to build on the
positive TOPAZ results, and we look forward to reporting the
topline results from this registrational study in Q4 next year,”
said Jay Backstrom, M.D., MPH, President and Chief Executive
Officer of Scholar Rock. “The 36-month data from TOPAZ, which
reinforced long-term sustained and clinically meaningful
improvement in motor function on top of SMN therapy, gives us
strong conviction that apitegromab could provide substantial
benefit beyond SMN therapy alone.”
Details of the poster presentations are as follows:
Title: Effect of apitegromab on PEDI-CAT and
PROMIS-fatigue questionnaire at 36 months in patients with spinal
muscular atrophy Presentation type: Poster presentation
Presenter: Thomas O. Crawford, M.D., Professor of Neurology
and Pediatrics, Johns Hopkins University Date and time:
Wednesday, October 4, 2023, 2:30 – 3:30 PM ET Location:
Charleston Area Convention Center, North Charleston, SC; Ballroom A
– C
Title: Effect of apitegromab on motor function at 36
months in patients with nonambulatory spinal muscular atrophy aged
2 – 12 years old Presentation type: Poster presentation
Presenter: Thomas O. Crawford, M.D., Professor of Neurology
and Pediatrics, Johns Hopkins University Date and time:
Wednesday, October 4, 2023, 2:30 – 3:30 PM ET Location:
Charleston Area Convention Center, North Charleston, SC; Ballroom A
– C
For conference information, visit https://www.wms2023.com/.
The presentations will be made available in the Publications
& Posters section of Scholar Rock’s website 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-targeted therapy (either nusinersen
or risdiplam). SAPPHIRE targeted enrolling approximately 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 targeted enrolling up to 48 patients aged 13-21 years old will
also separately be evaluated. These patients were 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, any development plans, strategy and progress for
apitegromab, the timing of its clinical trials, anticipated
clinical data, and therapeutic benefits for apitegromab, and other
product candidates and indication selection and development timing,
the ability of any product candidate to perf,orm in humans in a
manner consistent with earlier nonclinical, preclinical or clinical
trial data, and the potential of apitegromab and its other product
candidates and its 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, 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 SAPPHIRE Phase 3 clinical trial,
information provided or decisions made by regulatory authorities,
Scholar Rock’s dependence on third parties for development and
manufacture of product candidates including, without limitation, to
supply any clinical trials, , as well as those risks more fully
discussed in the section entitled "Risk Factors" in Scholar Rock’s
Annual Report on Form 10-Q for the quarter ended June 30, 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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version on businesswire.com: https://www.businesswire.com/news/home/20230919943390/en/
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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