- Progressing Phase 3 SAPPHIRE clinical trial
evaluating apitegromab in patients with non-ambulatory Type 2 and 3
Spinal Muscular Atrophy (SMA)
- Two-year data from open label extension of
TOPAZ Phase 2 trial in SMA expected by mid-2022
- Data from DRAGON Part B proof-of-concept
trial evaluating SRK-181 to overcome checkpoint inhibitor
resistance in cancer patients anticipated in 2022
- Ended 2021 with approximately $253 million in
cash, cash equivalents, and marketable securities
Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical
company focused on the treatment of serious diseases in which
protein growth factors play a fundamental role, today reported
financial results for the full year ended December 31, 2021, and
highlighted recent progress and upcoming milestones for its
pipeline programs.
“We are excited by the momentum and engagement around our
ongoing Phase 3 SAPPHIRE trial of apitegromab in spinal muscular
atrophy, as well as our DRAGON Phase 1 trial of SRK-181 in solid
tumors. These programs have significant potential to address unmet
patient need in a differentiated way, as well as demonstrate the
tremendous potential of Scholar Rock’s platform, which focuses on
inhibiting the activation of latent growth factors,” said Nagesh
Mahanthappa, Interim Chief Executive Officer of Scholar Rock.
“While we progress these clinical programs, we also continue to
build upon our world-leading expertise in selectively targeting the
activation mechanisms of the TGFβ-superfamily growth factors and
advance our preclinical pipeline focused on diseases such as
fibrosis and regulation of iron metabolism. We look forward to
providing more details about these programs at scientific meetings
and through publications in the coming months.”
Company Updates and Upcoming Milestones
Apitegromab is a selective inhibitor of myostatin
activation being developed as the potential first muscle-directed
therapy for the treatment of spinal muscular atrophy (SMA).
- Conducting SAPPHIRE Phase 3 Clinical Trial Evaluating
Apitegromab in Patients with Non-Ambulatory Type 2 and 3 SMA.
The randomized, double-blind, placebo-controlled Phase 3 clinical
trial is evaluating apitegromab as add-on therapy for patients on
either nusinersen or risdiplam. The study design plans for
approximately 156 patients aged 2-12 years old with non-ambulatory
Type 2 and 3 SMA 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 in addition to background
SMN treatment.
- Data from TOPAZ Phase 2 Open Label Extension Trial Expected
to be Presented by mid-2022. As of February 28, 2022, 55 of 57
patients remain enrolled in the long-term extension trial of
apitegromab in Type 2 and Type 3 SMA.
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 Part B of the DRAGON Phase 1 POC Trial for
SRK-181. Based on the safety and pharmacokinetic data from Part
A of the DRAGON Phase 1 trial, Scholar Rock is conducting the Part
B dose expansion portion of the trial, in which SRK-181 is dosed at
1500 mg every three weeks (Q3W) in patients receiving an approved
anti-PD-(L)1 therapy dosed Q3W, or 1000 mg every two weeks (Q2W) in
patients receiving an approved anti-PD-(L)1 therapy dosed Q2W. Part
B consists of multiple proof-of-concept cohorts focused upon
evaluating the ability of SRK-181 to overcome primary resistance to
anti-PD-(L)1 therapy. Each cohort will enroll up to 40 patients
with various solid tumors, including urothelial carcinoma (UC),
cutaneous melanoma (MEL), non-small cell lung cancer (NSCLC), clear
cell renal cell carcinoma (ccRCC), and other solid tumors. The
biomarker strategy in part B of DRAGON will explore early signs of
SRK-181 activity, including target engagement and pathway
modulation. This will include 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. Early efficacy
and safety data are anticipated in 2022.
Full Year 2021 Financial Results
For the full year ended December 31, 2021, net loss was $131.8
million or $3.59 per share compared to a net loss of $86.5.0
million or $2.81 per share for the year ended December 31,
2020.
- Revenue was $18.8 million for the year ended December 31, 2021
compared to $15.4 million for the year ended December 31, 2020.
Revenue was related to the Gilead fibrosis-focused research
collaboration, which concluded in December 2021.
- Research and development expense was $108.5 million for the
year ended December 31, 2021 compared to $74.1 million for the year
ended December 31, 2020. The increase year-over-year was primarily
attributable to planned spend associated with apitegromab
development, including costs associated with clinical drug supply
manufacturing and costs associated with our SAPPHIRE trial, as well
as higher personnel-related costs.
- General and administrative expense was $40.3 million for the
year ended December 31, 2021 compared to $28.2 million for the year
ended December 31, 2020. The increase year-over-year was primarily
attributable to professional services and higher personnel-related
costs, including filling key positions essential to progressing
research, development and pre-commercial activities.
As of December 31, 2021, Scholar Rock had cash, cash
equivalents, and marketable securities of approximately $253.0
million, which is expected to fund the Company’s operations into
mid-2023. “Continued operational excellence is an important
priority for us in 2022 as we advance several programs across the
continuum of drug development. As we continue to progress our SMA
program with our Phase 3 SAPPHIRE trial, we are also encouraged
that so many of the patients who participated in the TOPAZ study
are still opting to receive apitegromab. We look forward to
obtaining further insights into longer-term treatment outcomes and
sharing that data in the coming months. In our immuno-oncology
program, the DRAGON study has entered Part B and could produce
meaningful data later this year and our research colleagues are
pushing forward on several fronts as we continue to generate
exciting programs from our scientific platform,” said Ted Myles,
COO and CFO 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 Annual Report on Form 10-K for the
year ended December 31, 2021, 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
2021
2020
Revenue
$
18,816
$
15,403
Operating expenses
Research and development
108,468
74,062
General and administrative
40,269
28,219
Total operating expenses
148,737
102,281
Loss from operations
(129,921
)
(86,878
)
Other income (expense), net
(1,878
)
395
Net loss
$
(131,799
)
$
(86,483
)
Net loss per share, basic and diluted
$
(3.59
)
$
(2.81
)
Weighted average common shares
outstanding, basic and diluted
36,711,833
30,734,109
Scholar Rock Holding
Corporation
Condensed Consolidated Balance
Sheets
(unaudited)
(in thousands)
December 31, 2021
December 31, 2020
Assets
Cash, cash equivalents and marketable
securities
$
252,994
$
341,031
Other current assets
12,325
3,373
Total current assets
265,319
344,404
Other assets
39,126
43,901
Total assets
$
304,445
$
388,305
Liabilities and Stockholders'
Equity
Current liabilities
$
64,297
$
42,564
Long-term liabilities
68,074
84,971
Total liabilities
132,371
127,535
Total stockholders' equity
172,074
260,770
Total liabilities and stockholders'
equity
$
304,445
$
388,305
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220307005079/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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