- BLA and MAA submission for apitegromab in SMA
on track for 1Q 2025; if approved, U.S. launch expected in 4Q
2025
- Initiating Phase 2 OPAL clinical trial in SMA
patients under two years old in mid-2025
- Data from Phase 2 EMBRAZE trial evaluating
apitegromab in obesity expected in 2Q 2025
- Filing of IND application for SRK-439 on
track for 3Q 2025
- Presenting at the 43rd Annual J.P. Morgan
Healthcare Conference on Tuesday, January 14, 2025 at 9:45 a.m. PT
(12:45 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 2025.
“We are excited about the potential for apitegromab to help
those living with SMA as the first and only muscle-targeted therapy
for the treatment of this condition and are making excellent
progress in finalizing our regulatory applications and preparing
for commercial launch,” said Jay Backstrom, M.D., MPH, President
and Chief Executive Officer of Scholar Rock. “We remain on track
for BLA and MAA submission in the first quarter of 2025 and are
laser focused on bringing apitegromab to people with SMA as soon as
possible. Building on the success of our leading, highly selective
anti-myostatin approach in SMA, we plan to expand development of
apitegromab into additional rare neuromuscular disorders to serve
more people with unmet medical needs and as the foundation of a
growing neuromuscular franchise.”
Dr. Backstrom continued, “Additionally, we are making excellent
progress to deliver value across our pipeline, including the next
wave of innovation in cardiometabolic disorders. We look forward to
reporting topline data from our Phase 2 EMBRAZE proof-of-concept
trial of apitegromab in combination with a GLP-1 receptor agonist
in obesity and submitting an IND for SRK-439, our highly selective
myostatin inhibitor specifically designed for obesity and
cardiometabolic disorders.”
2025 Strategic Priorities and Upcoming Milestones:
Scholar Rock plans to focus on three strategic priorities in
2025 as it transitions into a commercial-stage biopharmaceutical
company, creating sustainable long-term value opportunities:
- Commercialize apitegromab in SMA.
- Expand the potential benefit of apitegromab.
- Advance Scholar Rock’s anti-myostatin program in
cardiometabolic disorders.
Commercialize apitegromab in
SMA
- On track to submit a biologics license application (BLA) to the
FDA and a marketing authorisation application (MAA) to the European
Medicines Agency in 1Q 2025 for apitegromab in SMA.
- Preparing for U.S. commercial launch in 4Q 2025, and European
launch to follow.
- Presenting detailed results from the pivotal Phase 3 SAPPHIRE
trial at upcoming medical meetings in 2025.
Expand the potential benefit of
apitegromab
- Initiating Phase 2 OPAL clinical trial in SMA patients under
two years of age in mid-2025. The trial will evaluate
apitegromab in patients under two years of age who have been or are
continuing to be treated with any currently approved SMN therapy,
including onasemnogene abeparvovec.
- Building upon the positive Phase 3 SAPPHIRE trial, the Company
is exploring development of apitegromab in other rare neuromuscular
disorders such as Duchenne muscular dystrophy, Becker muscular
dystrophy, Facioscapulohumeral muscular dystrophy and amyotrophic
lateral sclerosis to build a growing neuromuscular franchise.
Advance the anti-myostatin program in
cardiometabolic disorders
- Topline data expected in the second quarter of 2025 from the
Phase 2 EMBRAZE proof-of-concept trial, a randomized, double-blind,
placebo-controlled, multi-center study evaluating the safety and
efficacy of apitegromab, a highly selective investigational
myostatin inhibitor, to preserve muscle mass in overweight and
obese adults who are taking a GLP-1 receptor agonist (GLP-1 RA).
Trial outcomes will be used to guide clinical development of
SRK-439.
- Plan to file an IND for SRK-439 for the treatment of obesity on
the background of standard of care GLP-1 RAs in 3Q 2025.
- Scholar Rock disclosed new preclinical data which showed that
SRK-439 protected against tirzepatide-induced muscle loss in DIO
mice. Lean mass preservation was seen with doses of SRK-439 as low
as 0.3 mg/kg and lean mass gain was observed at higher doses. The
addition of SRK-439 with tirzepatide improved overall body
composition with dose-dependent enhancement of fat mass loss.
Scholar Rock believes that its existing cash, cash equivalents
and marketable securities will be sufficient to fund the Company’s
anticipated operating expenses and capital expenditure requirements
into the fourth quarter of 2026.
“Scholar Rock is funded to scale up and drive forward our key
priorities in 2025, including the commercialization of apitegromab,
the EMBRAZE readout, the initiation of clinical development for
SRK-439, and growing our pipeline,” said Ted Myles, Chief Operating
Officer and Chief Financial Officer of Scholar Rock. “We believe in
the transformative potential of our highly selective
muscle-targeted approach and will advance our programs as we
prepare to transition into a commercial company.”
2024 Highlights and Accomplishments:
- Reported positive topline data from the pivotal SAPPHIRE trial
in October 2024. The trial achieved its primary endpoint,
demonstrating a statistically significant and clinically meaningful
improvement for apitegromab versus placebo in motor function as
measured by the Hammersmith Functional Motor Scale – Expanded
(HFMSE) in SMA patients on chronic dosing of standard of care
therapies (either nusinersen or risdiplam).
- Continued high participation in the ONYX open-label extension
study evaluating the long-term safety and efficacy of apitegromab
in patients who completed the TOPAZ or SAPPHIRE trials. More than
90 percent of patients on combination therapy in the TOPAZ trial
have completed 4 years of apitegromab treatment and enrolled into
ONYX. Following trial completion, 98 percent of SAPPHIRE patients
(185/188) enrolled in the ongoing ONYX open-label expansion
study.
- Initiated Phase 2 EMBRAZE proof-of-concept trial with
apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA)
in obesity in May and completed enrollment in September.
- Presented new SRK-439 preclinical data at multiple conferences:
ObesityWeek, American Diabetes Association’s 84th Scientific
Sessions (ADA) and Keystone Symposia’s Obesity: Causes and
Consequences meeting.
- Successfully completed upsized $345 million public offering in
October to fund planned commercial launch in SMA and continue
advancement of priority programs.
J.P. Morgan Healthcare Conference Presentation and
Webcast
Scholar Rock management will highlight these updates in a
corporate presentation at the 43rd Annual J.P. Morgan Healthcare
Conference on Tuesday, January 14, 2025, at 9:45 a.m. PT (12:45
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 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 the Phase 3 SAPPHIRE Trial
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 EMBRAZE
EMBRAZE is a randomized, double-blind, placebo-controlled, Phase
2 proof-of-concept trial evaluating the efficacy, safety and
pharmacokinetics of apitegromab in adults with a body mass index
(BMI) of >27 (overweight) or a BMI of >30 (obese) and taking
a GLP-1 RA (tirzepatide or semaglutide). The target enrollment of
EMBRAZE is 100 subjects aged 18-65 who are overweight or obese
without diabetes. As part of the study design, the treatment period
is 24 weeks, and all subjects will receive a GLP-1 RA. In addition,
all subjects will be randomized 1:1 to receive either apitegromab
or placebo by intravenous (IV) infusion every four weeks during the
24-week treatment period. The primary endpoint is change from
baseline at Week 24 in lean mass assessed by dual-energy X-ray
absorptiometry. Secondary endpoints include additional weight loss
measures, safety and tolerability, and pharmacokinetic outcomes.
Exploratory endpoints at Weeks 24 and 32 include cardiometabolic
parameters (e.g. HbA1c), body composition, and physical
function.
About SRK-439
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 cardiometabolic disorders, including obesity. Based on
preclinical data, SRK-439 has the potential to support healthier
weight management by preserving lean mass during weight loss. The
efficacy and safety of SRK-439 have not been established and
SRK-439 has not been approved for any use by the FDA or any other
regulatory agency.
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 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.
The efficacy and safety of apitegromab, SRK-181, and SRK-439
have not been established and apitegromab, SRK-181, and SRK-439
have not been approved for any use by the FDA or any other
regulatory agency.
Scholar Rock® is a registered trademark of Scholar Rock,
Inc.
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.
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, its product candidate selection and
development timing, including timing for the initiation of and
reporting results from its preclinical studies and clinical trials
for apitegromab, SRK-439, linavonkibart 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,” “could,”
“might,” “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 for the purposes of the
safe harbor provisions under The Private Securities Litigation
Reform Act of 1995. 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 3 trial of
apitegromab or Part A or Part B of the Phase 1 trial of
linavonkibart, are not predictive of, may be inconsistent with, or
more favorable than, data generated from future or ongoing clinical
trials of the same product candidate; 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, including from the EMBRAZE clinical
trial; 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; the success of
Scholar Rock’s current and potential future collaborations; 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; its ability to establish or maintain strategic business
alliances; its ability to receive priority or expedited regulatory
review or to obtain regulatory approval of apitegromab; its ability
to expand globally and the anticipated commercial launch in the
United States of apitegromab in the fourth quarter of 2025; as well
as those risks more fully discussed in the section entitled "Risk
Factors" in Scholar Rock’s Form 10-K for the year ended December
31, 2023, and Quarterly Report on Form 10-Q for the quarter ended
September 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/20250108353769/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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