- Enrollment of pivotal Phase 3 SAPPHIRE trial
evaluating apitegromab on track for completion in 2023, and
36-month extension data from Phase 2 TOPAZ trial expected in
mid-2023
- Phase 1 DRAGON trial of SRK-181 continues to
advance with biomarker and clinical data updates planned in
2023
- Company expanded commercial experience with
recent appointment of Chief Commercial Officer
- Ended 2022 with approximately $315 million in
cash, cash equivalents, and marketable securities, which is
expected to fund operations into 2025
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,
today reported financial results and corporate updates for the full
year ended December 31, 2022.
“2023 is off to a great start as we continue to build upon our
momentum from last year. Importantly, we are on track to complete
the enrollment of our pivotal SAPPHIRE trial of apitegromab and are
looking forward to providing 36-month extension data from the Phase
2 TOPAZ trial of apitegromab in the coming months. Additionally, we
look forward to sharing biomarker and clinical updates throughout
the year from the SRK-181 Phase 1 DRAGON trial in patients with
relapsed and/or refractory cancers resistant to checkpoint
inhibitor therapy,” said Jay Backstrom, M.D., M.P.H., President
& Chief Executive Officer of Scholar Rock.
Company Highlights and Upcoming Milestones
Apitegromab is a selective
inhibitor of latent myostatin activation being developed as the
potential first muscle-targeted therapy for the treatment of spinal
muscular atrophy (SMA).
- Continue to progress and complete enrollment of Phase 3
SAPPHIRE clinical trial. The last patient is expected to be
enrolled in SAPPHIRE in 2023, with the top-line data readout
expected in 2024. If successful, and if approved, the company
expects to initiate a commercial product launch in 2025. SAPPHIRE
is a randomized, double-blind, placebo-controlled Phase 3 clinical
trial evaluating apitegromab in patients with nonambulatory Types 2
and 3 SMA on either nusinersen or risdiplam. 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
designations to apitegromab for the treatment of SMA.
- 36-month extension data from Phase 2 TOPAZ trial to be
presented in mid-2023. As of December 31, 2022, approximately
90 percent of patients who entered the trial’s long-term extension
study (51/57) remained on study. At the Cure SMA Research &
Clinical Care Meeting in June 2022, the company presented 24-month
extension data from the TOPAZ trial which demonstrated sustained
gains in Hammersmith Functional Motor Scale Expanded (HFMSE),
increased Revised Upper Limb Module (RULM) scores, and positive
trends in quality-of-life data for nonambulatory patients with
Types 2 and 3 SMA receiving an SMN therapy.
SRK-181 is an
investigational selective inhibitor of latent TGFβ-1 activation and
is being developed with the aim of overcoming resistance to
checkpoint therapy in patients with advanced cancer.
- Continue to progress Phase 1 DRAGON trial. The company
expects to provide biomarker and clinical updates from the DRAGON
proof-of-concept trial throughout 2023. In November 2022, Scholar
Rock presented data from DRAGON at the Society for Immunotherapy of
Cancer’s (SITC) Annual Meeting that showed SRK-181 continued to be
generally well tolerated with early indications of efficacy (as of
the data cut-off date of August 29, 2022).
- Encore data from DRAGON will be presented at the European
Society for Medical Oncology Targeted Anticancer Therapies (ESMO
TAT) Congress in Paris, France on March 7 at 11:05-12:30 p.m.
EST. The presentation is titled “Safety and efficacy results of
SRK-181 (a latent TGFβ1 inhibitor) from a phase 1 trial (DRAGON
trial).”
Corporate
- Appointed Tracey M. Sacco as Chief Commercial Officer.
In February 2023, Ms. Sacco joined Scholar Rock to build and lead
all commercial functions, including sales, marketing, market
access, and commercial operations. She will help the company
prepare for the potential commercial launch of apitegromab, if
approved. Ms. Sacco brings extensive biopharmaceutical industry
experience, including commercial strategy and global launch
experience of rare disease products.
Full Year 2022 Financial Results
For the full year ended December 31, 2022, net loss was $134.5
million or $2.26 per share compared to a net loss of $131.8 million
or $3.59 per share for the full year ended December 31, 2021.
- Revenue was $33.2 million for the year ended December 31, 2022,
compared to $18.8 million for the year ended December 31, 2021.
Revenue recognized during both periods was associated with the
Gilead collaboration which ended in 2022.
- Research and development expense was $124.4 million for the
year ended December 31, 2022, compared to $108.5 million for the
year ended December 31, 2021. The increase was primarily
attributable to costs associated with running the company’s Phase 3
SAPPHIRE pivotal trial for apitegromab in SMA, including clinical
trials costs as well as costs associated with employee compensation
and benefits.
- General and administrative expense was $43.1 million for the
year ended December 31, 2022, compared to $40.3 million for the
year ended December 31, 2021. The increase was due to an increase
in employee compensation and benefits, primarily associated with
equity-based compensation and professional fees.
- As of December 31, 2022, Scholar Rock had cash, cash
equivalents, and marketable securities of approximately $315
million, which is expected to fund the Company’s anticipated
operating and capital expenditure requirements into 2025.
“As Scholar Rock works to develop transformative therapies for
patients with our highly differentiated platform, we are entering
2023 with an experienced team, a strong balance sheet, and a clear
line of sight to important anticipated milestones, all of which are
expected to help advance the next phase of growth for the company,”
said Ted Myles, Chief Operating Officer and Chief Financial Officer
of Scholar Rock.
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,
fibrosis and anemia. 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/). 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, SRK-181, 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, or Part A of the Phase 1 clinical trial of
SRK-181, 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 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, 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.
Scholar Rock Holding Corporation Condensed Consolidated
Statements of Operations (unaudited) (in thousands, except
share and per share data)
Years Ended December
31
2022
2021
Revenue
$
33,193
$
18,816
Operating expenses Research and development
124,444
108,468
General and administrative
43,119
40,269
Total operating expenses
167,563
148,737
Loss from operations
(134,370
)
(129,921
)
Other income (expense), net
(132
)
(1,878
)
Net loss
$
(134,502
)
$
(131,799
)
Net loss per share, basic and diluted
$
(2.26
)
$
(3.59
)
Weighted average common shares outstanding, basic and
diluted
59,611,656
36,711,833
Scholar Rock Holding Corporation Condensed Consolidated
Balance Sheets (unaudited) (in thousands)
December
31, 2022 December 31, 2021 Assets Cash, cash
equivalents and marketable securities
$
315,361
$
252,994
Other current assets
12,663
12,325
Total current assets
328,024
265,319
Other assets
30,144
39,126
Total assets
$
358,168
$
304,445
Liabilities and Stockholders' Equity Current
liabilities
$
36,389
$
64,297
Long-term liabilities
61,544
68,074
Total liabilities
97,933
132,371
Total stockholders' equity
260,235
172,074
Total liabilities and stockholders' equity
$
358,168
$
304,445
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230307005330/en/
Scholar Rock:
Investors Rushmie Nofsinger Scholar Rock
rnofsinger@scholarrock.com ir@scholarrock.com 857-259-5573
Media Ariane Lovell Finn Partners
ariane.lovell@finnpartners.com media@scholarrock.com
917-565-2204
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