- Recently presented 36-month apitegromab
extension data from Phase 2 TOPAZ trial demonstrate long-term
substantial and sustained improvement of motor function and further
strengthen conviction in pivotal Phase 3 SAPPHIRE trial
- Pivotal Phase 3 SAPPHIRE trial evaluating
apitegromab on track to complete enrollment in Q3 2023 with
top-line data expected in 2H 2024
- Clinical and biomarker update from the Part B
portion of the Phase 1 DRAGON trial of SRK-181 in solid tumors
anticipated in 2H 2023
- Approximately $249 million in cash, cash
equivalents, and marketable securities as of June 30, 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
second quarter ended June 30, 2023.
“We continue to advance our apitegromab program, notably with
the recent presentation of 36-month data from the Phase 2 TOPAZ
trial which supports apitegromab’s therapeutic potential to
meaningfully improve motor function for patients with SMA,” said
Jay Backstrom, M.D., M.P.H., President and Chief Executive Officer
of Scholar Rock. “The continued success of TOPAZ increases our
conviction in SAPPHIRE and we look forward to completing enrollment
this quarter and sharing a topline data readout in 2024.”
Recent Company Highlights and Upcoming Milestones
Spinal Muscular Atrophy (SMA)
Program
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.
- Presented 36-month extension data from Phase 2 TOPAZ trial
at Cure SMA Research & Clinical Care Meeting in June. The
company shared new data evaluating outcomes after 36 months of
treatment with apitegromab, which showed substantial and sustained
improvement in motor function, as well as improvements in
patient-reported outcome measures in patients with nonambulatory
Types 2 and 3 SMA receiving survival motor neuron (SMN) therapy.
The company also hosted a virtual investor day event in July
featuring key opinion leaders to discuss the current SMA treatment
landscape and apitegromab’s potential to advance the standard of
care.
- Continued progress towards completion of enrollment for
Phase 3 SAPPHIRE clinical trial. The randomized, double-blind,
placebo-controlled Phase 3 clinical trial evaluating the safety and
efficacy of apitegromab in patients with nonambulatory Types 2 and
3 SMA receiving either nusinersen or risdiplam is actively
enrolling SMA patients across sites in the U.S. and Europe.
Enrollment completion is expected in the third quarter of 2023,
with top-line data expected in 2024. If successful and if approved,
the company expects to initiate a commercial product launch in
2025.
- Initiated the ONYX trial, the long-term extension study for
patients from both the TOPAZ and SAPPHIRE studies. As of July
2023, more than 90 percent of patients from TOPAZ receiving
apitegromab and a SMN therapy remained in the extension study.
Immuno-Oncology Program
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. DRAGON
is evaluating SRK-181 in patients with locally advanced or
metastatic solid tumors that exhibit resistance to anti-PD-(L)1
antibodies. The Company will provide biomarker and clinical updates
from Part B of the DRAGON trial in the second half of 2023.
Second Quarter 2023 Financial Results
For the quarter ended June 30, 2023, net loss was $37.9 million
compared to a net loss of $44.0 million for the quarter ended June
30, 2022.
- The Company did not record any revenue for either the quarter
ended June 30, 2023 or June 30, 2022.
- Research and development expense was $26.9 million for the
quarter ended June 30, 2023, compared to $32.1 million for the
quarter ended June 30, 2022. The decrease was primarily
attributable to a decrease in external research and development
costs, including costs for the apitegromab clinical trials and
associated clinical trial material manufacturing costs. In
addition, there was a decrease in employee compensation and
benefits costs, resulting from the restructuring in May 2022.
- General and administrative expense was $12.2 million for the
quarter ended June 30, 2023, compared to $11.1 million for the
quarter ended June 30, 2022.
- As of June 30, 2023, Scholar Rock had cash, cash equivalents,
and marketable securities of approximately $249 million, which is
expected to fund the company’s anticipated operating and capital
expenditure requirements into 2025.
“We are relentlessly focused on execution, and we are encouraged
by the continued strong data from TOPAZ and momentum we’re seeing
in the enrollment of SAPPHIRE. Our overall execution, along with
our spend for the first six months of 2023, is aligned with our
strategic plan,” said Ted Myles, Chief Operating Officer and Chief
Financial Officer of Scholar Rock. “We ended the quarter with $249
million in cash, which we expect to provide runway through a number
of anticipated important milestones.”
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 and SRK-181 and its preclinical programs 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 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 June 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.
Scholar Rock Holding Corporation Condensed Consolidated
Statements of Operations (unaudited) (in thousands, except
share and per share data)
Three Months Ended June
30 Six Months Ended June 30
2023
2022
2023
2022
Revenue
$
—
$
—
$
—
$
33,193
Operating expenses Research and development
26,867
32,073
56,602
61,439
General and administrative
12,215
11,074
22,989
21,834
Total operating expenses
39,082
43,147
79,591
83,273
Loss from operations
(39,082
)
(43,147
)
(79,591
)
(50,080
)
Other income (expense), net
1,157
(853
)
2,287
(1,870
)
Net loss
$
(37,925
)
$
(44,000
)
$
(77,304
)
$
(51,950
)
Net loss per share, basic and diluted
$
(0.47
)
$
(1.06
)
$
(0.97
)
$
(1.31
)
Weighted average common shares outstanding, basic and
diluted
80,117,983
41,622,392
79,865,424
39,550,991
Condensed Consolidated Balance
Sheets
(unaudited)
(in thousands)
June 30, 2023
December 31, 2022
Assets
Cash, cash equivalents and marketable securities
$
248,734
$
315,361
Other current assets
11,076
12,663
Total current assets
259,810
328,024
Other assets
25,090
30,144
Total assets
$
284,900
$
358,168
Liabilities and Stockholders'
Equity
Current liabilities
$
24,270
$
36,389
Long-term liabilities
58,129
61,544
Total liabilities
82,399
97,933
Total stockholders' equity
202,501
260,235
Total liabilities and stockholders'
equity
$
284,900
$
358,168
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230809008048/en/
Scholar Rock: Investors & Media Rushmie
Nofsinger Scholar Rock rnofsinger@scholarrock.com
ir@scholarrock.com 857-259-5573
Scholar Rock (NASDAQ:SRRK)
Gráfica de Acción Histórica
De May 2024 a Jun 2024
Scholar Rock (NASDAQ:SRRK)
Gráfica de Acción Histórica
De Jun 2023 a Jun 2024