- Phase 3 trial to evaluate apitegromab in
patients with non-ambulatory Type 2 and Type 3 spinal muscular
atrophy (SMA) anticipated to initiate by end of 2021
- DRAGON Phase 1 trial evaluating SRK-181’s
ability to overcome resistance to checkpoint inhibitors continues
to progress and on-track to advance to Part B dose expansion
mid-year 2021
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 second quarter ended June 30, 2021, and
highlighted recent progress and upcoming milestones for its
pipeline programs.
“Scholar Rock has had significant momentum in the first half of
the year with further demonstration of apitegromab’s transformative
potential to improve motor function for patients with SMA, as well
as continued progression of dose escalation in the DRAGON trial
evaluating SRK-181’s potential to overcome resistance to checkpoint
inhibitors in solid tumors,” said Nagesh Mahanthappa, Ph.D.,
Interim CEO of Scholar Rock. “I look forward to working closely
with this exceptional team as we continue to advance our research
and clinical programs to further elucidate the potential of our
scientific platform and help make a difference in the lives of
patients suffering from serious diseases.”
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).
- Positive Top-line Results and Additional Supportive
Exploratory Analyses from the TOPAZ Trial were Presented at the
Cure SMA Annual Conference. In June 2021, positive top-line
data from the TOPAZ Phase 2 trial (NCT03921528) were presented by
the lead principal investigator, Thomas Crawford, M.D. of Johns
Hopkins Medicine. Treatment with apitegromab in conjunction with
nusinersen in patients with Type 2 and 3 SMA led to meaningful
motor function improvements of up to 20 points as measured by
Hammersmith Functional Motor Scale Expanded (HFMSE). The majority
(74%, 23/31) of non-ambulatory patients showed a clinical
improvement, as defined by at least a 1-point increase in HFMSE.
Further evidence that improvements in motor function may be
attributed to apitegromab was supported by a post-hoc analysis that
showed no correlation between change in HFMSE and duration of prior
maintenance nusinersen therapy. In addition, 7/35 non-ambulatory
patients also showed major functional achievements as measured by
World Health Organization (WHO) Motor Development Milestones, with
one patient achieving two and one patient achieving three new WHO
motor milestones.
- Phase 3 Trial Evaluating Apitegromab in Patients with
Non-Ambulatory Type 2 and 3 Patients Anticipated to Initiate by
Year-End 2021. Subject to feedback from regulatory agencies,
Scholar Rock intends to conduct a randomized, double-blind,
placebo-controlled Phase 3 trial to evaluate apitegromab as an
add-on therapy to nusinersen or risdiplam in patients with
non-ambulatory Type 2 and Type 3 SMA. In the TOPAZ Phase 2 trial,
non-ambulatory patients experienced the largest increases in motor
function (HFMSE scores) following treatment with apitegromab as
add-on to chronic maintenance nusinersen. Patients with
non-ambulatory Type 2 and Type 3 SMA are estimated to represent
approximately two-thirds of the overall prevalent SMA patient
population.
- Apitegromab Granted Fast Track Designation by the FDA for
the Treatment of Patients with Spinal Muscular Atrophy. In May
2021, the U.S. Food and Drug Administration (FDA) granted Fast
Track designation for apitegromab, offering Scholar Rock
eligibility to submit a rolling Biologic License Application (BLA)
for apitegromab if relevant criteria are met. Fast Track
designation is intended to facilitate the development and expedite
the review of drugs to treat serious conditions and get new drugs
to patients earlier. In addition to Fast Track designation,
apitegromab had previously received Orphan Drug and Rare Pediatric
Disease designations from the FDA as well as PRIME and Orphan
Medicine Product designations from the European Medicines Agency
for the treatment of SMA, all recognizing the unmet medical needs
of patients with SMA.
- Additional Potential Indications Identified for
Apitegromab. Scholar Rock has identified multiple diseases for
which selectively inhibiting the activation of myostatin may offer
therapeutic benefit, including additional patient populations in
SMA (such as Type 1 SMA and ambulatory Type 3 SMA) and potential
indications outside of SMA.
SRK-181 is a selective
inhibitor of latent TGFβ1 activation being developed with the aim
of overcoming resistance to and increasing the number of patients
who may benefit from checkpoint inhibitor therapy.
- Plan to Advance to Part B Dose Expansion Portion of the
DRAGON Phase 1 Proof-of-Concept Trial Mid-Year. SRK-181 is
being evaluated in the two-part DRAGON trial (NCT04291079) in
patients with locally advanced or metastatic solid tumors
exhibiting primary resistance to anti-PD-(L)1 therapy. Part A dose
escalation, which is evaluating the safety and pharmacokinetics of
SRK-181 as a single-agent and in combination with anti-PD-(L)1
therapy, continues to progress to identify the recommended dose for
Part B of the trial. The Company is on-track to advance to Part B
dose expansion, which will consist of multiple cohorts, including
urothelial carcinoma, cutaneous melanoma, non-small cell lung
cancer, and other solid tumors. Each cohort will enroll up to 40
patients who have demonstrated primary resistance to anti-PD-(L)1
therapy and will be treated with SRK-181 in combination with an
approved anti-PD-(L)1 therapy. An update on dose escalation and
initial clinical data from Part A of the DRAGON trial is
anticipated by year-end.
Second Quarter 2021 Financial Results
For the quarter ended June 30, 2021, net loss was $30.7 million
or $0.84 per share compared to a net loss of $19.3 million or $0.65
per share for the quarter ended June 30, 2020.
- Revenue was $4.6 million for the quarter ended June 30, 2021
compared to $3.9 million for the quarter ended June 30, 2020 and
was related to the Gilead fibrosis-focused collaboration (the
“Gilead Collaboration Agreement”) that was executed in December
2018.
- Research and development expense was $25.6 million for the
quarter ended June 30, 2021 compared to $17.0 million for the
quarter ended June 30, 2020. The increase year-over-year primarily
reflects manufacturing costs associated with apitegromab and higher
personnel and facility-related costs, partially offset by lower
manufacturing costs associated with SRK-181.
- General and administrative expense was $9.3 million for the
quarter ended June 30, 2021 compared to $6.4 million for the
quarter ended June 30, 2020. The increase year-over-year was
primarily attributed to higher personnel and facility-related costs
and professional fees.
“We are executing towards key milestones and continue to work
closely with regulatory authorities to finalize the design of the
Phase 3 trial for apitegromab and to progress the DRAGON trial to
Part B to evaluate SRK-181 across multiple tumor types,” said Ted
Myles, CFO and Head of Business Operations of Scholar Rock. “We
ended the second quarter with approximately $282 million in cash
and cash equivalents, which will allow us to achieve meaningful
milestones as we continue to execute against our plan.”
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
12-month top-line results from the Phase 2 trial of apitegromab,
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, 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 June 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 June
30
Six Months Ended June
30
2021
2020
2021
2020
Revenue
$
4,595
$
3,900
$
9,303
$
8,930
Operating expenses
Research and development
25,603
16,997
48,152
33,899
General and administrative
9,265
6,365
18,631
12,187
Total operating expenses
34,868
23,362
66,783
46,086
Loss from operations
(30,273
)
(19,462
)
(57,480
)
(37,156
)
Other income (expense), net
(434
)
181
(898
)
805
Net loss
$
(30,707
)
$
(19,281
)
$
(58,378
)
$
(36,351
)
Net loss per share, basic and
diluted
$
(0.84
)
$
(0.65
)
$
(1.60
)
$
(1.23
)
Weighted average common shares
outstanding, basic and diluted
36,582,708
29,690,280
36,482,132
29,608,814
Scholar Rock Holding Corporation Condensed
Consolidated Balance Sheets (unaudited) (in thousands)
June 30, 2021 December
31, 2020 Assets Cash, cash equivalents and
marketable securities
$
282,217
$
341,031
Other current assets
8,149
3,373
Total current assets
290,366
344,404
Other assets
41,685
43,901
Total assets
$
332,051
$
388,305
Liabilities and Stockholders' Equity
Current liabilities
$
67,022
$
42,564
Long-term liabilities
48,373
84,971
Total liabilities
115,395
127,535
Total stockholders' equity
216,656
260,770
Total liabilities and stockholders' equity
$
332,051
$
388,305
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210810005957/en/
Scholar Rock: Investors Catherine Hu
chu@scholarrock.com
Media Ariane Lovell Finn Partners
ariane.lovell@finnpartners.com 917-565-2204
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