- Apitegromab is the only muscle-targeted
therapy to demonstrate proof of concept in spinal muscular
atrophy
- Two oral presentations to cover long-term
efficacy, safety, and patient-reported outcome data
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 it will present extension data from the Phase 2
TOPAZ trial evaluating patient outcomes after 36 months of
treatment with apitegromab. Apitegromab is an investigational
antibody inhibiting myostatin activation by selectively binding the
pro- and latent forms of myostatin and has demonstrated clinical
proof of concept in spinal muscular atrophy (SMA). In two
back-to-back oral presentations, Thomas O. Crawford, M.D., will
present 36-month safety and efficacy data, and an analysis of
patient-reported outcome measures such as fatigue, mobility, and
activities of daily living at the Cure SMA Research & Clinical
Care Meeting, taking place June 28-30 in Orlando, Florida.
“As we progress enrollment of our Phase 3 SAPPHIRE trial toward
completion this year, we are excited to share the 36-month data
from the Phase 2 TOPAZ study, the longest efficacy and safety data
on an anti-myostatin in SMA to date,” said Jay Backstrom, M.D.,
M.P.H., President and Chief Executive Officer of Scholar Rock.
“These data continue to deepen our understanding of the essential
role a selective muscle-targeted treatment could play in
transforming care in spinal muscular atrophy, and we look forward
to sharing the 36-month TOPAZ data with the community at the Cure
SMA Annual Conference.”
Details of the presentations 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.
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 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 non-ambulatory 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. In this
subpopulation of older individuals with SMA, the safety and
tolerability of apitegromab will be characterized, and efficacy
will also be evaluated in an exploratory, nonpowered manner. For
more information about SAPPHIRE, visit www.clinicaltrials.gov.
About Apitegromab
Apitegromab is an investigational antibody inhibiting myostatin
activation by selectively binding the pro- and latent forms of
myostatin and has demonstrated 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 promote
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, and often fatal,
genetic disorder that typically manifests in young children. An
estimated 30,000 to 35,000 patients are afflicted with SMA in the
United States and Europe. It 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 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, its cash runway, 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 such as COVID-19 on business operations and expectations,
as well as those risks more fully discussed in the section entitled
"Risk Factors" in Scholar Rock’s Annual Report on Form 10-K for the
year ended December 31, 2022, 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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