- Phase 3 pivotal trial evaluating apitegromab
in patients with non-ambulatory Type 2 and Type 3 SMA on track to
initiate by year-end 2021
- Update from Part A of the DRAGON Phase 1
trial evaluating SRK-181’s ability to overcome primary resistance
to checkpoint inhibitors being presented at the Society for
Immunotherapy of Cancer (SITC) 36th Annual Meeting
- Initiated Part B dose expansion portion of
the DRAGON trial
- U.S. patent issued providing additional
product protection for SRK-181 to May 2040
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 third quarter ended September 30, 2021,
and highlighted recent progress and upcoming milestones for its
pipeline programs.
“The DRAGON trial addresses two key questions; first, can the
selectivity of SRK-181 for TGFβ1 increase the therapeutic window
for inhibition of this signaling pathway thereby enabling higher
doses safely, and second, does SRK-181 exhibit anti-tumor activity
effective in overcoming resistance to checkpoint inhibitors in
solid tumors. We are pleased with the results of Part A, as will be
provided this week at the Society for Immunotherapy of Cancer
(SITC) Annual Meeting, and we look forward to exploring the
efficacy dimension of our therapeutic hypothesis in Part B,” said
Nagesh Mahanthappa, Ph.D., Interim CEO of Scholar Rock. “Building
on the momentum from the first half of this year, we are also
excited about the progress with apitegromab and are on track to
initiate by year-end the Phase 3 pivotal trial evaluating its
efficacy in patients with non-ambulatory Type 2 and Type 3 spinal
muscular atrophy (SMA).”
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).
- Additional Exploratory Responder Analyses and Pharmacologic
Data from the TOPAZ Phase 2 Trial were Presented at Various Medical
Congresses. In September 2021, two posters were presented at
the World Muscle Society Virtual Congress, including a late-breaker
poster featuring an exploratory analysis evaluating time to
achieving various thresholds of improvement in Hammersmith
Functional Motor Scale Expanded (HFMSE) scores, which are
consistent with the observed dose response in clinical efficacy. In
October 2021, a poster presented at the 25th World Congress of
Neurology (WCN) described the positive correlation between the
magnitude of target engagement and motor function improvements
following apitegromab treatment. At the 50th Child Neurology
Society Annual Meeting, additional exploratory responder analyses
on Hammersmith scale scores were presented, including time to
achieve different thresholds of improvement in HFMSE scores.
- Phase 3 Trial Evaluating Apitegromab in Patients with
Non-Ambulatory Type 2 and 3 Patients Remains on Track to Initiate
by Year-End 2021. Scholar Rock is preparing to announce the
design of the Phase 3 pivotal study later this month. The company
is on track to initiate by the end of 2021 the randomized,
double-blind, placebo-controlled Phase 3 trial evaluating
apitegromab as add-on therapy for patients on either nusinersen or
risdiplam with non-ambulatory Type 2 and Type 3 SMA. This patient
population comprises an estimated two-thirds of the overall
prevalence of SMA, and the greatest improvements in motor function
(as measured by HFMSE) observed in the TOPAZ Phase 2 trial were in
patients with non-ambulatory Type 2 and Type 3 SMA receiving
apitegromab as add-on therapy to background nusinersen.
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.
- Update from Part A of the DRAGON Phase 1 Trial and Part B
Dose to be Presented at the Society for Immunotherapy of Cancer
(SITC) 36th Annual Meeting. A poster titled, “First-in-Human
Phase 1 Trial of SRK-181: A Latent TGFβ1 inhibitor, Alone or in
Combination with Anti-PD-(L)1 Treatment in Patients with Advanced
Solid Tumors (DRAGON trial)” will be presented at the SITC meeting
on November 12, 2021. The poster will include initial clinical data
from Part A of the DRAGON trial as well as the rationale for the
identified Part B dose. The Company has initiated the Part B dose
expansion portion of the trial, which consists of multiple cohorts,
each enrolling up to 40 patients with various solid tumors who have
demonstrated primary resistance to anti-PD-(L)1 therapy.
- U.S. Patent Issued Providing Composition of Matter Product
Protection for SRK-181. In September 2021, the United States
Patent and Trademark Office (USPTO) issued U.S. Patent No.
11,130,803 with an expiry of May 2040, including 313 days of Patent
Term Adjustment (PTA). The European counterpart has also been
granted.
Third Quarter 2021 Financial Results
For the quarter ended September 30, 2021, net loss was $37.5
million or $1.02 per share compared to a net loss of $23.6 million
or $0.79 per share for the quarter ended September 30, 2020.
- Revenue was $5.5 million for the quarter ended September 30,
2021, compared to $3.0 million for the quarter ended September 30,
2020 and was related to the Gilead Collaboration Agreement that was
executed in December 2018.
- Research and development expense was $31.3 million for the
quarter ended September 30, 2021, compared to $18.4 million for the
quarter ended September 30, 2020. The increase year-over-year
primarily reflects manufacturing costs associated with apitegromab,
clinical trial costs for SRK-181, and additional personnel and
facility-related costs.
- General and administrative expense was $11.3 million for the
quarter ended September 30, 2021, compared to $8.3 million for the
quarter ended September 30, 2020. The increase year-over-year was
primarily attributed to additional personnel, professional fees,
and facility-related costs.
“As we approach the end of 2021, I am incredibly proud of the
execution by the entire Scholar Rock team this year. Not only have
we initiated Part B of the DRAGON trial, but we’re also very close
to initiating our Phase 3 pivotal trial for apitegromab,” said Ted
Myles, CFO and Head of Business Operations of Scholar Rock. “We
ended the third quarter with approximately $246 million in cash and
cash equivalents and are well-funded to continue executing on our
development programs while continuing to invest in our robust
discovery platform.”
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/).
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,” “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 preclinical and clinical data, including the
results from the Phase 2 trial of apitegromab or Part A of the
Phase 1 trial of SRK-181, are not predictive of, are inconsistent
with, or more favorable than, data generated from future clinical
trials of the same product candidate, including the planned Phase 3
trial of apitegromab in SMA and Part B of the Phase 1 trial of
SRK-181, respectively, 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, the success
of Scholar Rock’s current and potential future collaborations,
including its collaboration with Gilead, Scholar Rock’s dependence
on third parties for development and manufacture of product
candidates including 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
September 30, 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)
Three Months Ended September
30
Nine Months Ended September
30
2021
2020
2021
2020
Revenue
$
5,464
$
3,037
$
14,767
$
11,967
Operating expenses Research and development
31,265
18,383
79,417
52,282
General and administrative
11,276
8,272
29,907
20,459
Total operating expenses
42,541
26,655
109,324
72,741
Loss from operations
(37,077
)
(23,618
)
(94,557
)
(60,774
)
Other income (expense), net
(430
)
57
(1,328
)
862
Net loss
$
(37,507
)
$
(23,561
)
$
(95,885
)
$
(59,912
)
Net loss per share, basic and diluted
$
(1.02
)
$
(0.79
)
$
(2.62
)
$
(2.02
)
Weighted average common shares outstanding, basic and
diluted
36,683,026
29,779,114
36,549,833
29,665,995
Scholar Rock Holding
Corporation
Condensed Consolidated Balance
Sheets
(unaudited)
(in thousands)
September 30, 2021 December 31, 2020
Assets Cash, cash equivalents and marketable securities
$
246,435
$
341,031
Other current assets
11,721
3,373
Total current assets
258,156
344,404
Other assets
40,233
43,901
Total assets
$
298,389
$
388,305
Liabilities and Stockholders' Equity Current
liabilities
$
64,601
$
42,564
Long-term liabilities
46,528
84,971
Total liabilities
111,129
127,535
Total stockholders' equity
187,260
260,770
Total liabilities and stockholders' equity
$
298,389
$
388,305
View source
version on businesswire.com: https://www.businesswire.com/news/home/20211109005546/en/
Scholar Rock: Investors Stephanie Ascher Stern
Investor Relations, Inc. Stephanie.Ascher@sternir.com
212-362-1200
Media Ariane Lovell Finn Partners
ariane.lovell@finnpartners.com 917-565-2204
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