- Completed enrollment for pivotal Phase 3 SAPPHIRE trial
evaluating apitegromab; topline data expected in 4Q 2024
- Expanding into cardiometabolic disorders with SRK-439, a novel
investigational myostatin inhibitor
- Presented new data supporting proof of concept for SRK-181 in
heavily pretreated patients with resistant metastatic ccRCC;
completing enrollment of the Phase 1 DRAGON trial in December
2023
- Completed $98 million public offering, extending projected cash
runway into second half of 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
third quarter ended September 30, 2023.
“We have made significant progress across our pipeline over the
last quarter, including completing enrollment of our Phase 3
SAPPHIRE trial of apitegromab, which was designed to build on the
positive Phase 2 TOPAZ results. Based on the sustained improvement
in motor function that was observed after 36 months of treatment in
the TOPAZ trial, the favorable safety and tolerability profile, and
the high continued participation rate in our long-term extension
study, we believe apitegromab has the potential to be a
transformative therapy for SMA patients,” said Jay Backstrom, M.D.,
MPH, President and Chief Executive Officer of Scholar Rock.
“Further, we are excited to leverage our expertise in selective
myostatin inhibition and expand into cardiometabolic disorders,
including obesity. We believe our highly selective myostatin
inhibitor SRK-439 has the potential to help patients retain lean
muscle mass, which has been a key obstacle for many on GLP-1
receptor agonist therapy, and we plan to file an IND in 2025 to
initiate clinical testing of SRK-439 in combination with GLP-1
receptor agonists.”
“Additionally, we recently presented new clinical and biomarker
data from the Phase 1 DRAGON trial that demonstrated the
therapeutic potential of SRK-181 in heavily pretreated patients
with anti-PD-1 resistant clear cell renal cell carcinoma. We
believe that the DRAGON trial has achieved the objective of
establishing proof of concept that our highly selective approach to
blocking latent TGFβ1 can restore sensitivity to a checkpoint
inhibitor, most notably in those with anti-PD-1 resistant ccRCC and
supports further development of SRK-181,” he added.
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.
- Completed enrollment of its pivotal Phase 3 SAPPHIRE
clinical trial. The Company announced that it completed
enrollment in September 2023, with topline data expected in the
fourth quarter of 2024. If successful and if approved, the Company
expects to initiate a commercial product launch in 2025.
- Presented encore 36-month extension data from the Phase 2
TOPAZ trial at the 28th Annual Congress of the World Muscle Society
in October. The Company presented encore data from its Phase 2
TOPAZ trial extension period evaluating patient outcomes in SMA
after 36 months of treatment of apitegromab, which showed long-term
sustained improvements in motor function and in patient-reported
outcome measures in patients with nonambulatory Types 2 and 3 SMA
receiving survival motor neuron (SMN) therapy.
- ONYX long-term extension study for patients from both the
TOPAZ and SAPPHIRE studies remains ongoing.
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.
- Presented clinical and biomarker data from Phase 1 DRAGON
proof-of-concept trial at the SITC 38th Annual Meeting. The
Company presented new data from its Phase 1 DRAGON proof-of-concept
trial. As of the August 29, 2023 data cutoff, SRK-181 data showed
favorable tolerability and promising anti-tumor activity in heavily
pretreated patients with clear cell renal cell carcinoma (ccRCC)
resistant to anti-PD-1. Of 28 evaluable patients in the ccRCC
cohort, six patients had confirmed partial responses and achieved a
best tumor reduction of 33% to 93%. The objective response rate
(ORR) was 21.4% and the disease control rate was 57%. Circulating
granulocytic myeloid-derived suppressor cell (gMDSC) levels
correlated with better clinical responsiveness in ccRCC patients
treated with SRK-181 in combination with pembrolizumab. The Company
believes these data from the Phase 1 DRAGON trial supports proof of
concept and that the trial has met its primary objectives. The
Company is completing enrollment of the trial in December 2023,
while continuing to treat patients who remain on study.
Cardiometabolic Program
SRK-439 is a novel, preclinical investigational myostatin
inhibitor that has high in vitro affinity for pro- and latent
myostatin, maintenance of myostatin specificity (i.e., no GDF11 or
Activin-A binding), and is initially being developed for the
treatment of obesity.
- Plans to initiate a Phase 2 proof-of-concept trial with
apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA)
in obesity in 2024. As part of the Company’s strategy to
advance the development of SRK-439, it plans to initiate a Phase 2
proof-of-concept trial with apitegromab in combination with a GLP-1
RA in 2024, subject to IND clearance. Data from the clinical trial
are expected in mid-2025 and will be used to inform further
clinical development of SRK-439. The Company plans to file an IND
for SRK-439 for the treatment of obesity in 2025.
Third Quarter 2023 Financial Results
For the quarter ended September 30, 2023, net loss was $42.4
million compared to a net loss of $43.3 million for the quarter
ended September 30, 2022.
- The Company did not record any revenue for either the quarter
ended September 30, 2023 or September 30, 2022.
- Research and development expense was $30.3 million for the
quarter ended September 30, 2023, compared to $33.4 million for the
quarter ended September 30, 2022.
- General and administrative expense was $13.3 million for the
quarter ended September 30, 2023, compared to $10.5 million for the
quarter ended September 30, 2022.
- As of September 30, 2023, Scholar Rock had cash, cash
equivalents, and marketable securities of approximately $218.6
million, which in addition to approximately $92.5 million of net
proceeds from the October 2023 equity offering, is projected to
fund the Company’s anticipated operating and capital expenditure
requirements into the second half of 2025.
“Our upsized public offering achieved two key objectives: it
enables us to bring our expertise to the cardiometabolic and
obesity space with SRK-439, a highly selective myostatin inhibitor,
and it extends our cash runway well past our upcoming SAPPHIRE
Phase 3 data read out in Q4 2024,” said Ted Myles, Chief Operating
Officer and Chief Financial Officer of Scholar Rock. “We are
executing against our plan and we are well positioned going into
2024.”
Conference Call and Webcast The Company’s earnings
conference call for the third quarter will be held at 8:00 a.m. ET
on November 7, 2023. To access the conference call by phone,
participants may register here to receive the dial-in number and
unique PIN. A live webcast of the conference call will be available
on the Investors & Media section of the Scholar Rock website at
http://investors.scholarrock.com. An archived replay of the webcast
will be available for approximately 90 days following the call.
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.
About SRK-181 & the Phase 1 DRAGON Proof-of-Concept
Trial SRK-181 is a selective inhibitor of TGFβ1 activation
being developed to overcome primary resistance to checkpoint
inhibitor therapy, such as anti-PD-(L)1 antibodies, in advanced
cancer. TGFβ1 is the predominant TGFβ isoform expressed in many
human tumor types. Based on analyses of various human tumors that
are resistant to anti-PD-(L)1 therapy, data suggest that TGFβ1 is a
key contributor to the immunosuppressive tumor microenvironment,
excluding and preventing entry of cytotoxic T cells into the tumor,
thereby inhibiting anti-tumor immunity. 1
SRK-181 specifically targets the latent TGFβ1 isoform in a
context-independent manner, designed to enable complete inhibition
of TGFβ1 in all compartments within the tumor microenvironment.
Scholar Rock believes that SRK-181 has the potential to overcome
this immune cell exclusion and induce tumor regression when
administered in combination with anti-PD-(L)1 therapy while
potentially avoiding toxicities associated with non-selective TGFβ
inhibition. The Phase 1 DRAGON proof-of-concept clinical trial
(NCT04291079) in patients with locally advanced or metastatic solid
tumors is ongoing. The trial is currently enrolling and dosing
patients in multiple proof of concept cohorts conducted in
parallel, including urothelial carcinoma (UC), cutaneous melanoma
(MEL), non-small cell lung cancer (NSCLC), head and neck squamous
cell carcinoma (HNSCC), and clear cell renal cell carcinoma
(ccRCC). SRK-181 is an investigational product candidate, and its
efficacy and safety have not been established. SRK-181 has not been
approved for any use by the FDA or any other regulatory agency.
1Martin et al., Sci. Transl. Med. 12: 25
March 2020
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 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 the 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, 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, 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;
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 September
30
Nine Months Ended September
30
2023
2022
2023
2022
Revenue
$
—
$
—
$
—
$
33,193
Operating expenses
Research and development
30,337
33,392
86,939
94,831
General and administrative
13,335
10,470
36,324
32,304
Total operating expenses
43,672
43,862
123,263
127,135
Loss from operations
(43,672
)
(43,862
)
(123,263
)
(93,942
)
Other income (expense), net
1,313
565
3,600
(1,305
)
Net loss
$
(42,359
)
$
(43,297
)
$
(119,663
)
$
(95,247
)
Net loss per share, basic and
diluted
$
(0.53
)
$
(0.55
)
$
(1.49
)
$
(1.80
)
Weighted average common shares
outstanding, basic and diluted
80,606,438
79,336,161
80,115,143
52,958,447
Scholar Rock Holding Corporation Condensed Consolidated
Balance Sheets (unaudited) (in thousands)
September 30, 2023 December 31, 2022
Assets Cash, cash equivalents and marketable
securities
$
218,635
$
315,361
Other current assets
9,830
12,663
Total current assets
228,465
328,024
Other assets
22,224
30,144
Total assets
$
250,689
$
358,168
Liabilities and Stockholders' Equity
Current liabilities
$
26,730
$
36,389
Long-term liabilities
56,294
61,544
Total liabilities
83,024
97,933
Total stockholders' equity
167,665
260,235
Total liabilities and stockholders' equity
$
250,689
$
358,168
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231107339655/en/
Investors & Media Rushmie Nofsinger Scholar Rock
rnofsinger@scholarrock.com ir@scholarrock.com 857-259-5573
Scholar Rock (NASDAQ:SRRK)
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