- Positive 24-month data from ongoing Phase 2
TOPAZ trial extension supports potential benefit of apitegromab for
patients with Types 2 and 3 Spinal Muscular Atrophy (SMA)
- Actively enrolling in pivotal Phase 3
SAPPHIRE clinical trial for apitegromab
- Discovery pipeline advancements made for
LTBP-TGFβ1 target to treat a wide range of fibrotic diseases
- Significantly strengthened balance sheet with
the completion of $205 million registered direct offering
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, 2022.
“We are pleased to have reported important progress with our two
clinical programs during the second quarter. Enrollment continues
to advance in our pivotal Phase 3 SAPPHIRE trial for apitegromab in
SMA. We also presented new data from our Phase 2 TOPAZ trial
highlighting sizeable and sustained improvement in Hammersmith
Functional Motor Scale-Expanded (HFMSE) scores at 24 months for
non-ambulatory patients, reinforcing our belief in the unique
potential of apitegromab as a muscle-targeted therapy to treat SMA,
and we continue to evaluate these patients in their third year of
treatment,” said Nagesh Mahanthappa, Founding Chief Executive
Officer & President of Scholar Rock. “In our SRK-181 program,
enrollment is ongoing in the Phase 1 DRAGON trial Part B dose
expansion. We look forward to providing updates on DRAGON as data
become available. Further, our focused research continues to make
excellent progress as we develop next-generation programs to build
out our future pipeline.”
Company Highlights and Upcoming Milestones
Apitegromab is a selective inhibitor of myostatin
activation being developed as the first muscle-targeted therapy
with the potential to treat spinal muscular atrophy (SMA).
- Positive 24-Month Phase 2 TOPAZ Extension Data Support
Potential Benefit for Patients. Scholar Rock announced positive
data at the Cure SMA Research & Clinical Care Meeting in June,
demonstrating sizable and sustained motor function improvement at
24 months with apitegromab as measured by Hammersmith Functional
Motor Scale-Expanded (HFMSE) scores for non-ambulatory patients
with Types 2 and 3 SMA on nusinersen. Results also showed a
substantial increase in Revised Upper Limb Module (RULM) scores,
with no serious safety risks identified over 24 months of
apitegromab treatment. Of the 55 patients who completed the
24-month extension period, 54 opted to continue into the 36-month
extension period.
- Enrollment Ongoing for Phase 3 SAPPHIRE Clinical Trial
Evaluating Apitegromab in Non-Ambulatory Patients with Types 2 and
3 SMA. The randomized, double-blind, placebo-controlled Phase 3
clinical trial is evaluating apitegromab for patients on either
nusinersen or risdiplam. Approximately 156 non-ambulatory patients
aged 2-12 years old with Types 2 and 3 SMA are planned to be
enrolled in the main efficacy population. Patients will be
randomized 1:1:1 to receive for 12 months either apitegromab 20
mg/kg, apitegromab 10 mg/kg, or placebo by intravenous (IV)
infusion every 4 weeks. SAPPHIRE is expected to be conducted across
55 sites in the U.S. and Europe. Scholar Rock presented on the
trial design at the 17th International Congress on Neuromuscular
Diseases (ICNMD 2022) in July 2022, along with publication of the
abstract in the peer-reviewed Journal of Neuromuscular
Diseases.
SRK-181 is a selective inhibitor of latent TGFβ1
activation being developed with the aim of overcoming primary
resistance to and increasing the number of patients who may benefit
from checkpoint inhibitor therapy.
- Advancing Enrollment for Part B of the Phase 1 DRAGON
Proof-of-Concept Clinical Trial for SRK-181. Part B of the
Phase 1 DRAGON trial consists of multiple proof-of-concept cohorts
focused on evaluating the ability of SRK-181 to overcome primary
resistance to anti-PD-(L)1 therapy in patients with solid tumors.
The biomarker strategy for DRAGON explores early signs of SRK-181
activity, including target engagement and pathway modulation. It
includes measuring effects on both circulating and tumor immune
contexture, such as CD8+ T cell infiltration and reductions in
myeloid-derived suppressor cell (MDSC) populations, as well as
analysis of TGFβ-related pathway signaling. Initial evidence of
drug activity and safety data are anticipated in 2022.
Scholar Rock’s preclinical discovery pipeline includes a highly
selective, context-dependent TGFβ1 antibody that inhibits proTGFβ1
activation selectively in the extracellular matrix via targeting
the covalent complexes of proTGFβ1 with latent TGFβ binding
proteins 1 and 3 (“LTBPs”— the targets are collectively referred to
as “LTBP-TGFβ1”).
- Preclinical Data Supports Selective Targeting of
Matrix-Associated TGFβ1 as an Attractive Approach for Anti-Fibrotic
Therapy. Scholar Rock recently presented data on its targeted
approach to LTBP-TGFβ1 that show reduction of TGFβ1 signaling and
reduction of fibrosis in relevant preclinical in vivo models.
Second Quarter 2022 Financial Results
For the quarter ended June 30, 2022, net loss was $44.0 million
or $1.06 per share compared to a net loss of $30.7 million or $0.84
per share for the quarter ended June 30, 2021.
- Revenue was $0 for the quarter ended June 30, 2022, compared to
$4.6 million for the quarter ended June 30, 2021.
- Research and development expense was $32.1 million for the
quarter ended June 30, 2022, compared to $25.6 million for the
quarter ended June 30, 2021. The increase was primarily
attributable to increased clinical costs associated with
apitegromab and higher personnel costs, including severance
expenses associated with the recent restructuring.
- General and administrative expense was $11.1 million for the
quarter ended June 30, 2022, compared to $9.3 million for the
quarter ended June 30, 2021. The increase was primarily due to
higher personnel costs, including severance expenses associated
with the recent restructuring.
- As of June 30, 2022, Scholar Rock had cash, cash equivalents,
and marketable securities of approximately $371 million, which is
expected to fund the Company’s anticipated operating and capital
expenditure requirements into 2025.
“We were thrilled to announce the completion of a $205 million
registered direct offering during the quarter. This financing
included several high-quality, long-term oriented,
fundamentals-based biotechnology investors who saw the promise of
our platform and our programs. Importantly, this capital puts us in
a strong financial position to fully fund the Phase 3 SAPPHIRE
trial, continue advancing Part B of the Phase 1 DRAGON trial for
SRK-181, while investing in selected early-stage programs that
exemplify the power of our scientific platform,” 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, and
fibrosis. 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, are not predictive of, may be inconsistent with, or more
favorable than, data generated from future clinical trials of the
same product candidate, 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, 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)
Three Months Ended June
30 Six Months Ended June 30
2022
2021
2022
2021
Revenue
$
—
$
4,595
$
33,193
$
9,303
Operating expenses Research and development
32,073
25,603
61,439
48,152
General and administrative
11,074
9,265
21,834
18,631
Total operating expenses
43,147
34,868
83,273
66,783
Loss from operations
(43,147
)
(30,273
)
(50,080
)
(57,480
)
Other income (expense), net
(853
)
(434
)
(1,870
)
(898
)
Net loss
$
(44,000
)
$
(30,707
)
$
(51,950
)
$
(58,378
)
Net loss per share, basic and diluted
$
(1.06
)
$
(0.84
)
$
(1.31
)
$
(1.60
)
Weighted average common shares outstanding, basic and
diluted
41,622,392
36,582,708
39,550,991
36,482,132
Scholar Rock Holding Corporation Condensed Consolidated
Balance Sheets (unaudited) (in thousands)
June 30,
2022 December 31, 2021 Assets Cash, cash
equivalents and marketable securities
$
370,688
$
252,994
Other current assets
18,451
12,325
Total current assets
389,139
265,319
Other assets
34,989
39,126
Total assets
$
424,128
$
304,445
Liabilities and Stockholders' Equity Current
liabilities
$
40,162
$
64,297
Long-term liabilities
54,459
68,074
Total liabilities
94,621
132,371
Total stockholders' equity
329,507
172,074
Total liabilities and stockholders' equity
$
424,128
$
304,445
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220808005487/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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