- Enrollment of pivotal Phase 3 SAPPHIRE trial
evaluating apitegromab on track for completion in 2023
- Upcoming presentations of 36-month
apitegromab data from Phase 2 TOPAZ trial at Cure SMA’s Annual
Research & Clinical Conference
- Continued advancement of Phase 1 DRAGON trial
of SRK-181; biomarker and clinical data update planned in the
second half of 2023
- Approximately $275 million in cash, cash
equivalents, and marketable securities as of March 31, 2023,
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
first quarter ended March 31, 2023.
“We are executing across our business and leveraging Scholar
Rock’s differentiated platform, which selectively targets latent
forms of growth factors, such as myostatin and TGFβ1, to develop
highly selective and potentially transformative therapies for
patients who have high unmet medical need,” said Jay Backstrom,
M.D., M.P.H., President & Chief Executive Officer of Scholar
Rock. “We continue to advance our two clinical programs in spinal
muscular atrophy and oncology, and we look forward to sharing
updates on our progress as we approach several key milestones,
including presenting 36-month data on apitegromab from the Phase 2
TOPAZ trial, completing enrollment of our apitegromab Phase 3
SAPPHIRE trial, and providing biomarker and clinical updates from
our SRK-181 Phase 1 DRAGON trial.”
Recent Company Highlights and Upcoming Milestones
Apitegromab is an
investigational antibody inhibiting myostatin activation by
selectively binding the pro- and latent forms of myostatin and is
being developed as the potential first muscle-targeted therapy for
the treatment of spinal muscular atrophy (SMA).
- Continued progress towards completion of enrollment for
Phase 3 SAPPHIRE clinical trial. The randomized double-blind,
placebo-controlled clinical trial evaluating apitegromab for
patients with nonambulatory Types 2 and 3 SMA on either nusinersen
or risdiplam, is actively enrolling SMA patients across sites in
the U.S. and Europe. Enrollment completion is expected 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.
- 36-month data from Phase 2 TOPAZ trial to be presented at
Cure SMA conference in June. The company announced two upcoming
oral presentations at the Cure SMA Research & Clinical
Conference, taking place June 28-30 in Orlando, Florida.
- 24-month data from the TOPAZ trial presented at the 2023
Muscular Dystrophy Association (MDA) Clinical and Scientific
Conference in March. The company shared data evaluating
outcomes after 24 months of treatment with apitegromab,
demonstrating sustained improvements in motor function in patients
with nonambulatory Types 2 and 3 SMA.
SRK-181 is an
investigational selective inhibitor of latent TGFβ1 activation and
is being developed with the aim of overcoming resistance to
checkpoint therapy in patients with advanced cancer.
- Advancing Phase 1 DRAGON proof-of-concept trial. In the
second half of 2023, the company expects to provide biomarker and
clinical updates from Part B of the DRAGON trial. In March 2023,
Scholar Rock presented encore data at the European Society for
Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Congress,
which showed that SRK-181 continued to be generally well tolerated
with early indications of efficacy (as of the data cut-off date of
December 2, 2022).
Corporate
Announced the addition of Richard Brudnick
to the Board of Directors. In April 2023, Mr. Brudnick joined
Scholar Rock’s Board of Directors. As an accomplished biotechnology
executive, he brings an extensive background in corporate
development and strategy, with over 30 years of industry experience
across multiple specialties, stages and therapeutic areas.
First Quarter 2023 Financial Results
For the quarter ended March 31, 2023, net loss was $39.4 million
or $0.49 per share compared to a net loss of $8.0 million or $0.21
per share for the quarter ended March 31, 2022.
- Revenue was $0 for the quarter ended March 31, 2023, compared
to $33.2 million for the quarter ended March 31, 2022. The prior
year revenue was related to the Gilead fibrosis-focused research
collaboration, which was executed in December 2018 and concluded in
December 2021.
- Research and development expense was $29.7 million for the
quarter ended March 31, 2023, compared to $29.4 million for the
quarter ended March 31, 2022. The increase was primarily
attributable to costs associated with clinical trials, including
the Phase 3 SAPPHIRE pivotal trial for apitegromab in SMA, and the
DRAGON trial for SRK-181. These increases were offset by decreases
in employee compensation and benefits costs, resulting from the
restructuring in May 2022.
- General and administrative expense was $10.8 million for the
quarter ended March 31, 2023, compared to $10.8 million for the
quarter ended March 31, 2022.
- As of March 31, 2023, Scholar Rock had cash, cash equivalents,
and marketable securities of approximately $275 million, which is
expected to fund the company’s anticipated operating and capital
expenditure requirements into 2025.
“Scholar Rock is in a strong financial position, and we are
relentlessly focused on achieving our goal of improving the lives
of patients who may potentially benefit from our medicines,” said
Ted Myles, Chief Operating Officer and Chief Financial Officer of
Scholar Rock. “We continue to execute on our plan by advancing our
clinical programs towards key inflection points, while diligently
managing our resources.”
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/).
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, 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, 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 Quarterly Report on Form 10-Q for
the quarter ended March 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
(unaudited)
(in thousands, except share and
per share data)
Three Months Ended March
31
2023
2022
Revenue
$
—
$
33,193
Operating expenses
Research and development
29,735
29,366
General and administrative
10,774
10,760
Total operating expenses
40,509
40,126
Loss from operations
(40,509)
(6,933)
Other income (expense), net
1,130
(1,017)
Net loss
$
(39,379)
$
(7,950)
Net loss per share, basic and diluted
$
(0.49)
$
(0.21)
Weighted average common shares
outstanding, basic and diluted
79,610,059
37,456,574
Scholar Rock Holding Corporation
Condensed Consolidated Balance
Sheets
(unaudited)
(in thousands)
March 31, 2023
December 31, 2022
Assets
Cash, cash equivalents and marketable securities
$
275,284
$
315,361
Other current assets
11,874
12,663
Total current assets
287,158
328,024
Other assets
27,563
30,144
Total assets
$
314,721
$
358,168
Liabilities and Stockholders'
Equity
Current liabilities
$
26,208
$
36,389
Long-term liabilities
59,860
61,544
Total liabilities
86,068
97,933
Total stockholders' equity
228,653
260,235
Total liabilities and stockholders'
equity
$
314,721
$
358,168
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230509005251/en/
Scholar Rock:
Investors & Media Rushmie Nofsinger Scholar
Rock rnofsinger@scholarrock.com ir@scholarrock.com 857-259-5573
Scholar Rock (NASDAQ:SRRK)
Gráfico Histórico do Ativo
De Abr 2024 até Mai 2024
Scholar Rock (NASDAQ:SRRK)
Gráfico Histórico do Ativo
De Mai 2023 até Mai 2024