- Advancing industry-leading antimyostatin
pipeline, comprised of multiple, novel assets with unparalleled
selectivity, to treat spinal muscular atrophy (SMA) and
cardiometabolic disorders
- Completed enrollment for apitegromab pivotal
Phase 3 SAPPHIRE trial in patients with SMA; topline data
anticipated in 4Q 2024
- Apitegromab Phase 2 proof-of-concept trial in
obesity expected to commence in mid-2024
- Presenting new preclinical data on SRK-439, a
novel investigational myostatin inhibitor for the treatment of
obesity, at Keystone Symposia in February
- Scholar Rock reports year-end cash and cash
equivalents of approximately $280 million
- Presenting at the 42nd Annual J.P. Morgan
Healthcare Conference on Tuesday, January 9, 2024 at 1:30 p.m. PT
(4:30 p.m. 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 provided recent corporate updates and highlighted
upcoming priorities for 2024.
“We were pleased with our progress in 2023 as we advanced our
industry leading antimyostatin pipeline with apitegromab and
SRK-439 and have great momentum heading into 2024. With enrollment
completed for our Phase 3 SAPPHIRE trial for patients with spinal
muscular atrophy, we expect topline data in Q4 this year,” said Jay
Backstrom, M.D., MPH, President and Chief Executive Officer of
Scholar Rock. “Additionally, we are thrilled with the planned
initiation in mid-2024 of an apitegromab Phase 2 proof-of-concept
trial in patients with obesity and on GLP-1 therapies that aims to
establish the importance of selective, safe myostatin inhibition to
preserve lean muscle mass as part of healthy weight loss.”
2024 Priorities:
SPINAL MUSCULAR ATROPHY
Apitegromab is an investigational, fully human monoclonal
antibody that inhibits myostatin activation by selectively binding
the pro- and latent forms of myostatin in skeletal muscle and is
being developed as a potential first muscle-targeted therapy for
the treatment of SMA. Apitegromab is the only muscle-targeted
therapy to show clinical proof-of-concept in SMA.
- Planning to announce Phase 3 SAPPHIRE clinical trial topline
data in 4Q 2024. SAPPHIRE is a randomized, double-blind,
placebo-controlled clinical trial evaluating apitegromab in
patients with nonambulatory Types 2 and 3 SMA on either nusinersen
or risdiplam. If the trial is successful and apitegromab is
approved, the Company expects to initiate a commercial product
launch in 2025.
- Continue to progress the ONYX long-term extension study for
patients from both the TOPAZ and SAPPHIRE studies.
CARDIOMETABOLIC
DISORDERS
SRK-439 is a novel, preclinical, investigational
myostatin inhibitor that has high in vitro affinity for pro- and
latent myostatin and maintains myostatin specificity (i.e., no
GDF11 or Activin-A binding), and is initially being developed for
the treatment of obesity.
- Initiating a Phase 2 proof-of-concept trial with apitegromab
in combination with a GLP-1 receptor agonist (GLP-1 RA) in obesity
in mid-2024. As part of the Company’s strategy to advance the
development of SRK-439, it plans to initiate a Phase 2
proof-of-concept trial with apitegromab in combination with a GLP-1
RA, subject to IND clearance. Data from the clinical trial are
expected in mid-2025 and will be used to guide clinical development
of SRK-439. The Company plans to file an IND for SRK-439 for the
treatment of obesity in 2025.
- Presenting preclinical SRK-439 data in a poster presentation
at Keystone Symposia at the Obesity: Causes and Consequences
meeting on February 5, 2024, in Vancouver, BC, Canada.
2023 Highlights:
- Completed enrollment of apitegromab pivotal Phase 3 SAPPHIRE
trial.
- Presented TOPAZ 36-month extension trial data at the Cure SMA
Research & Clinical Care Meeting in June, and at the 28th
Annual Congress of the World Muscle Society in October, which
showed long-term substantial and sustained improvements in motor
function and patient-reported outcome measures in patients with
nonambulatory Types 2 and 3 SMA receiving survival motor neuron
(SMN) therapy.
- Initiated the ONYX trial, a long-term extension study for
patients from both the TOPAZ and SAPPHIRE studies, which remains
ongoing.
- Announced plans to expand into cardiometabolic disorders with
SRK-439, starting with a Phase 2 proof-of-concept trial evaluating
apitegromab in obesity to inform development of SRK-439.
- Presented SRK-181 Phase 1 DRAGON trial clinical and biomarker
data at the SITC 38th Annual Meeting, which showed favorable
tolerability and promising anti-tumor activity in heavily
pretreated patients with clear cell renal cell carcinoma (ccRCC)
resistant to anti-PD-1. The Company believes these data support
proof-of-concept and completed enrollment of the DRAGON trial in
December. The Company will provide additional clinical data updates
as they become available in 2024.
- Completed an equity financing of $98 million in October. As of
December 31, 2023, Scholar Rock reported cash, cash equivalents,
and marketable securities of approximately $280 million, which is
projected to fund the Company’s operations into the second half of
2025.
“We are excited to enter 2024 with several important near-term
milestones ahead of us. As the leader in selective myostatin
inhibition, we believe we are well positioned to deliver
significant value to patients living with spinal muscular atrophy
and the millions of people suffering from the wide array of health
challenges stemming from cardiometabolic and obesity disorders. We
are highly encouraged by our potential to advance the standard of
care where muscle-targeted therapies can play a role in addressing
unmet patient needs,” said Ted Myles, Chief Operating Officer and
Chief Financial Officer.
J.P. Morgan Healthcare Conference Presentation and
Webcast
Scholar Rock management will highlight these updates in a
corporate presentation at the 42nd Annual J.P. Morgan Healthcare
Conference on Tuesday, January 9, 2024, at 1:30 p.m. PT (4:30 p.m.
ET). A live webcast of the presentation may be accessed by visiting
the Investors & Media section of the Scholar Rock website at
http://investors.scholarrock.com. An archived replay of the webcast
will be available on the Company’s website for approximately 90
days following the presentation.
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.
Scholar Rock is the only company to show clinical
proof-of-concept for a muscle-targeted treatment in spinal muscular
atrophy (SMA). 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, results and timing of its clinical trials for
apitegromab and SRK-181 and its preclinical programs, including
SRK-439, regulatory feedback including with respect to the IND
submitted in connection with the planned Phase 2 trial of SRK-439
in combination with GLP-1 RAs in obesity, and indication selection
and development timing, including the therapeutic potential,
clinical benefits and safety thereof, expectations regarding
timing, success and data announcements of current ongoing
preclinical and clinical trials, its expected cash and cash
equivalents as of December 31, 2023 and 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, that preclinical and
clinical data, including the results from the Phase 2 clinical
trial of apitegromab, or Part A or Part B of the Phase 1 clinical
trial of SRK-181, 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, including, without
limitation, the Phase 3 clinical trial of apitegromab in SMA or
Part B of the Phase 1 clinical trial of SRK-181; Scholar Rock’s
ability to provide the financial support, resources and expertise
necessary to identify and develop product candidates on their
expected timelines; 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 Quarterly Report on Form 10-Q for the
quarter ended September 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/20240104939112/en/
Investors & Media Rushmie Nofsinger Scholar Rock
rnofsinger@scholarrock.com ir@scholarrock.com 857-259-5573
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