Full Press Release Details
Scholar Rock Reports Full Year 2023 Financial
Results and Highlights Business Progress
Phase 3 SAPPHIRE trial in patients with Spinal Muscular Atrophy on track to report topline data in 4Q 2024
FDA clearance of IND application to initiate Phase 2 proof-of-concept trial with apitegromab to treat obesity; expected to commence in
preclinical data supporting potential benefit of SRK-439 to preserve lean muscle mass as part of healthier weight management at Keystone
2023 with approximately $280 million in cash, cash equivalents, and marketable securities, which is expected to fund operations into 2H
Mass.--(BUSINESS WIRE)- March 19, 2024-- 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 reported financial results and corporate updates for the full year ended December 31,
"In 2023, Scholar Rock made significant progress across our industry-leading
antimyostatin pipeline, and we are poised for a transformational year ahead. We remain on track to release topline data for our Phase
3 SAPPHIRE trial of apitegromab in spinal muscular atrophy in the fourth quarter of 2024, which if successful, will be the foundation
of our neuromuscular franchise. Additionally, we remain on track to initiate a Phase 2 proof-of-concept trial with apitegromab in combination
with a GLP-1 RA in mid-2024, as we continue to move SRK-439, our novel preclinical myostatin inhibitor for obesity, toward IND,"
said Jay Backstrom, M.D., M.P.H., President & Chief Executive Officer of Scholar Rock. "As the only company to show clinical
proof of concept in SMA with our selective antimyostatin approach, we are well positioned to further extend our leadership with a potential
launch in SMA and clinical data in obesity anticipated in 2025."
Company Highlights and Upcoming Milestones
Spinal Muscular Atrophy (SMA) Program
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 candidate to show clinical proof-of-concept in SMA.
Cardiometabolic Program
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.
Full Year 2023 Financial Results
For the full year ended December 31, 2023, net loss was $165.8
million or $1.99 per share compared to a net loss of $134.5 million or $2.26 per share for the full year ended December 31, 2022.
"We executed across our portfolio in 2023 and leveraged our world-leading
expertise in myostatin inhibition with the formal launch of our cardiometabolic program. Additionally, we managed spend carefully throughout
the year, and the capital raise in the fourth quarter extended our cash runway," said Ted Myles, Chief Operating Officer and Chief
Financial Officer of Scholar Rock. "We are off to a strong start in 2024 and are well positioned for a pivotal year, looking to
the SAPPHIRE data read-out as a critical inflection point for the company."
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. Apitegromab has not been approved for any use by the US FDA or any other
health authority, and its safety and efficacy have not been established.
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. Based on preclinical data, SRK-439 has the potential to support healthier
weight management by preserving lean mass. 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.
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 (TGF ) 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,
Scholar Rock is a registered trademark of Scholar
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 SRK-181 and its preclinical programs, including SRK-439, 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 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 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; and 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; 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, 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.
Scholar Rock Holding Corporation
Condensed Consolidated Statements of Operations
(in thousands, except share and per share data)