- Positive interim analysis data from the TOPAZ
Phase 2 trial demonstrated proof-of-concept for apitegromab
(SRK-015) in patients with Type 2 and Type 3 Spinal Muscular
Atrophy
- Progress update on dose escalation in Part A
of the SRK-181 DRAGON Phase 1 trial to be presented at the Society
for Immunotherapy of Cancer (SITC) Congress; no dose-limiting
toxicities observed as of the data cutoff
- Raised $230 million in gross proceeds through
a public offering to advance apitegromab, SRK-181 and preclinical
programs
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, 2020
and highlighted recent progress and upcoming milestones for its
pipeline programs.
“We achieved a significant milestone with the TOPAZ interim
analysis proof-of-concept results. The data support apitegromab’s
potential to improve motor function for patients with Type 2 and
Type 3 SMA and importantly, highlight the therapeutic potential of
our platform targeting the latent forms of growth factors, which
includes SRK-181 in immuno-oncology,” said Tony Kingsley, President
and CEO of Scholar Rock. “As we look to the remainder of 2020 and
into 2021, I am very excited for the potential of the company as we
continue to advance and expand our pipeline across a wide range of
serious diseases where growth factors may play a role, including
other neuromuscular disorders, cancer, and fibrosis.”
Company Updates and Upcoming Milestones
Apitegromab (SRK-015) Program for
Spinal Muscular Atrophy (SMA):
Apitegromab is a highly selective inhibitor of latent myostatin
activation being developed as the potential first muscle-directed
therapy for the treatment of SMA.
- Six-Month Interim Data from TOPAZ Demonstrate Meaningful
Potential for Apitegromab to Improve Motor Function for Patients
with SMA. In October 2020, Scholar Rock announced positive
proof-of-concept data from a pre-planned six-month interim analysis
of the TOPAZ Phase 2 trial, which enrolled 58 patients with Type 2
and Type 3 SMA across three parallel cohorts. Key findings
included:
- Treatment with apitegromab led to improvements in the
Hammersmith scale scores (primary efficacy endpoint that measures
motor function) in all three cohorts of patients with Type 2 and
Type 3 SMA.
- Substantial proportion of patients in each cohort attained
≥3-point improvement in Hammersmith scores.
- Dose response in the primary efficacy endpoint was observed in
the randomized, double-blind Cohort 3, with apitegromab high dose
(20 mg/kg) attaining a 5.6 point mean improvement at six-months
over baseline compared to low dose (2 mg/kg) attaining a 2.4 point
mean improvement over baseline.
- Pharmacokinetic and pharmacodynamic data supported the
clinically observed dose response; apitegromab high dose (20 mg/kg)
yielded higher levels of drug exposure and target engagement than
low dose (2 mg/kg).
- No safety signals were identified from the interim analysis.
The five most frequently reported treatment-emergent adverse events
(TEAEs) were headache, upper respiratory tract infection, pyrexia,
nasopharyngitis, and cough.
- Detailed interim analysis results from the TOPAZ trial were
issued on October 27, 2020: Scholar Rock Announces Positive
Proof-of-Concept Data from TOPAZ Phase 2 Trial Interim Analysis of
SRK-015 in Patients with Type 2 and Type 3 Spinal Muscular
Atrophy.
Top-line data for the 12-month treatment
period are expected in the second quarter of 2021. Twelve-month
results could provide additional insights on the potential
durability of effect and the potential for further improvements in
motor function, as well as additional safety data. There may be
impacts on the timing of future doses and assessments for patients
in the trial as the effects of the COVID-19 pandemic continue to
evolve.
As of November 5, 2020, 45 of 45 patients who
have completed the 12-month study have opted into the extension
period.
- Product candidate, SRK-015, now referred to as
apitegromab. The United States Adopted Names (USAN) Council has
approved the use of the non-proprietary name apitegromab for
SRK-015, the Company’s product candidate for the treatment of
patients with SMA. The USAN Council is responsible for selecting
simple, informative and unique non-proprietary (generic) drug names
and establishes logical nomenclature classifications based on
pharmacological and/or chemical relationships. The council consists
of a Food and Drug Administration (FDA) liaison, one
member-at-large, and one representative from each of the following:
The American Medical Association (AMA), United States Pharmacopeia
(USP) and the American Pharmacists Association (APhA).
SRK-181 Program for
Immuno-Oncology:
SRK-181 is a potent and highly selective inhibitor of latent
TGFβ1 activation being developed towards an aim of overcoming
resistance to and increasing the number of patients who may benefit
from checkpoint inhibitor therapy.
- Progress Update on Dose Escalation in Part A of the DRAGON
Phase 1 Trial to be Presented at the Society for Immunotherapy of
Cancer (SITC) Annual Meeting. A poster presentation titled:
“DRAGON: Phase 1 trial of SRK-181, a latent TGFβ1 inhibitor in
combination with anti-PD-(L)1 inhibitors for patients with solid
tumors unresponsive to anti-PD-(L)1 therapy alone,” is being
presented at the SITC congress being held November 9-14, 2020. The
poster outlines the dose escalation scheme and provides an update
on progress of patient enrollment and advancement in dose levels.
As of October 1, 2020, eight patients have been dosed in Part A1 of
the trial, which evaluates SRK-181 as a monotherapy. No
dose-limiting toxicities had been observed up to the 800 mg dose
and the 1600 mg dose is currently under evaluation. Part A2 of the
trial, which evaluates SRK-181 in combination with an approved
anti-PD-(L)1 therapy, has enrolled two patients at the 240 mg dose
as of October 1, 2020. The two-part DRAGON Phase 1 clinical trial
consists of a dose escalation portion (Part A) and a dose expansion
portion (Part B) to evaluate SRK-181 in combination with an
approved anti-PD-(L)1 therapy in patients with locally advanced or
metastatic solid tumors exhibiting primary resistance to that
anti-PD-(L)1 therapy. Part B will encompass multiple cohorts that
are expected to include urothelial carcinoma, cutaneous melanoma,
non-small cell lung cancer, and other solid tumors. Scholar Rock
anticipates several updates from the DRAGON trial over the next
year, including updates on dose escalation, an advancement to Part
B that is expected in the first quarter of 2021, and clinical
response and safety data in the second half of 2021. There may be
impacts on the enrollment rate and dosing of patients in the trial
as the effects of the COVID-19 pandemic continue to evolve.
- Presentation of Preclinical TGFβ1 Data at the Festival of
Biologics, World Immunotherapy Congress 2020. On November 5,
2020, Scholar Rock presented previously announced preclinical data
at the virtual Festival of Biologics. The presentation titled:
“Selective inhibition of TGFβ1 activation overcomes primary
resistance to checkpoint inhibition therapy” detailed the potential
of a potent and select inhibitor of latent TGFβ1 activation, such
as SRK-181, in overcoming the dose-limiting toxicities associated
with non-selective approaches to targeting TGFβ. In mouse tumor
models that recapitulate key features of primary resistance to
checkpoint inhibitor therapy, combination treatment with
SRK-181-mIgG1 (murine version of SRK-181) and an anti-PD-1 therapy
resulted in tumor regression or control and survival benefit.
Third Quarter 2020 Financial Results
For the quarter ended September 30, 2020, net loss was $23.6
million or $0.79 per share compared to a net loss of $16.1 million
or $0.55 per share for the quarter ended September 30, 2019.
- Revenue was $3.0 million for the quarter ended September 30,
2020 compared to $4.8 million for the quarter ended September 30,
2019. Revenue was related to the Gilead fibrosis-focused
collaboration that was executed in December 2018.
- Research and development expense was $18.4 million for the
quarter ended September 30, 2020 compared to $15.7 million for the
quarter ended September 30, 2019. The increase year-over-year is
primarily attributable to costs associated with the apitegromab
TOPAZ Phase 2 clinical trial, including clinical drug supply
manufacturing, as well as higher personnel-related costs.
- General and administrative expense was $8.3 million for the
quarter ended September 30, 2020 compared to $6.2 million for the
quarter ended September 30, 2019. The increase year-over-year was
primarily attributable to higher personnel-related costs.
As of September 30, 2020, Scholar Rock had cash, cash
equivalents, and marketable securities of $116.3 million. On
October 19, 2020, Scholar Rock announced the closing of a $50
million debt facility, of which $25 million was funded at closing.
On November 2, 2020, Scholar Rock announced the closing of a public
offering of common stock and prefunded warrants and full exercise
of the underwriters' option to purchase additional shares of common
stock. The aggregate gross proceeds to Scholar Rock from this
offering were $230 million, before deducting underwriting discounts
and commissions and other offering expenses.
“With the recent public offering and the addition of the debt
facility, we have significantly strengthened our balance sheet and
extended our cash runway into 2023,” said Ted Myles, CFO and Head
of Business Operations of Scholar Rock. “We are fully investing in
our clinical product candidates, apitegromab and SRK-181, as well
as our preclinical programs and the underlying scientific platform
to develop important therapies for patients in need.”
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/).
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 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, 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 June 30,
2020, 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
2020
2019
2020
2019
Revenue
$
3,037
$
4,774
$
11,967
$
12,919
Operating expenses Research and development
18,383
15,699
52,282
40,153
General and administrative
8,272
6,181
20,459
14,961
Total operating expenses
26,655
21,880
72,741
55,114
Loss from operations
(23,618
)
(17,106
)
(60,774
)
(42,195
)
Other income (expense), net
57
959
862
2,768
Net loss
$
(23,561
)
$
(16,147
)
$
(59,912
)
$
(39,427
)
Net loss per share, basic and diluted
$
(0.79
)
$
(0.55
)
$
(2.02
)
$
(1.46
)
Weighted average common shares outstanding, basic and
diluted
29,779,114
29,232,158
29,665,995
26,929,215
Scholar Rock Holding Corporation
Condensed Consolidated Balance
Sheets
(unaudited)
(in thousands)
September 30, 2020
December 31, 2019
Assets Cash, cash equivalents and marketable securities
$
116,264
$
157,448
Other current assets
3,117
27,719
Total current assets
119,381
185,167
Other assets
44,040
11,214
Total assets
$
163,421
$
196,381
Liabilities and Stockholders' Equity Current
liabilities
$
32,869
$
32,814
Long-term liabilities
67,575
50,666
Total liabilities
100,444
83,480
Total stockholders' equity
62,977
112,901
Total liabilities and stockholders' equity
$
163,421
$
196,381
View source
version on businesswire.com: https://www.businesswire.com/news/home/20201109005132/en/
Scholar Rock Contact: Investors/Media Catherine Hu
chu@scholarrock.com 917-601-1649
Media Contact: The Yates Network Kathryn Morris
kathryn@theyatesnetwork.com 914-204-6412
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