- On track for Phase 2 apitegromab TOPAZ
two-year data by mid-year
- Progressed enrollment for both Phase 3
apitegromab SAPPHIRE clinical trial & Phase 1 SRK-181 DRAGON
clinical trial
- Restructuring reduces operating expenses,
prioritizes R&D activity on completing SAPPHIRE trial and
extends cash runway into the fourth quarter of 2023
- Yung Chyung, MD, has decided to step down as
Chief Medical Officer effective June 30, 2022
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 provided a business update and reported financial results for
the first quarter ended March 31, 2022.
Business Strategy & Update
- Prioritize R&D Activities to Support Progression and
Completion of the Apitegromab Phase 3 SAPPHIRE Trial in Spinal
Muscular Atrophy. Scholar Rock is focusing its expertise in
inhibiting the activation of latent growth factors on completing
the Phase 2 TOPAZ extension clinical trial and the Phase 3 SAPPHIRE
clinical trial, both of which are evaluating the potential of
apitegromab to address motor function impairments in patients with
spinal muscular atrophy (SMA). The SRK-181 program for the
treatment of patients with solid tumors showing primary resistance
to anti-PD-(L)1 therapy remains a priority with strategic value.
While investment in the existing scientific platform continues, the
Company is narrowing the scope of discovery programs it expects to
pursue.
- Focused Strategy to Increase Operational Efficiency, Reduce
Cash Burn and Extend Runway. The Company today announced a
restructuring of its business to prioritize and focus on its
clinical stage assets resulting in a reduction to its workforce by
approximately 25%, in addition to the curtailment of previously
planned hiring. The reduction in operating expenses relates to
general and administrative spend and research activities related to
earlier stage programs. In conjunction with the pipeline
prioritization, these changes are expected to extend the cash
runway into the fourth quarter of 2023.
“We are highly confident in the transformative potential of
apitegromab based upon the Phase 2 TOPAZ data we have released to
date. We look forward to presenting two-year extension trial data
in the coming weeks,” said Nagesh Mahanthappa, Ph.D., Founding
Chief Executive Officer of Scholar Rock. “Based upon the strength
of the data released to date and our commitment to the SMA
community, this narrower focus and associated headcount reduction
is necessary to execute our core mission -- completing the Phase 3
SAPPHIRE trial to bring apitegromab, our highly innovative
therapeutic candidate, to patients suffering with this devastating
disease.”
Dr. Mahanthappa continued, “Parting with employees who have
dedicated their talent and expertise to evolving a scientific
platform into a Phase 3-stage company is very difficult, and we are
extremely grateful for their contributions to our mission to
support patients and help position us to potentially deliver the
next big innovation to patients with SMA.”
Changes to Development Leadership
The Company also announced today that Yung Chyung, M.D. has
decided to step down as Chief Medical Officer (CMO) to explore new
career opportunities, including his interest in global health. Dr.
Chyung will remain with Scholar Rock through June 30 to work with
the executive team and the development organization on a transition
plan, and a retained search for a new CMO is ongoing.
“I am incredibly proud of the progress we’ve achieved to date at
Scholar Rock and am humbled to have served alongside the amazing
and talented employees across the company,” said Dr. Chyung. “I am
enthusiastic about the potential of apitegromab and SRK-181 to
transform the lives of individuals impacted by serious diseases.
With operational momentum continuing to build and the outstanding,
high performing teams in place, I feel this is an appropriate time
for me to explore my other long-term career interests, such as
global health.”
“The Board and I sincerely thank Yung for his ingenuity and
leadership over the years, which have brought the apitegromab
program forward through a successful Phase 2 proof-of-concept study
and now into Phase 3, creating hope for patients and families
suffering from SMA that muscle-directed therapy could have the
potential to make a meaningful impact on their lives,” said Dr.
Mahanthappa. “While the search for a new CMO is a top priority and
we are committed to identifying a medical leader with a strong
track record in late-stage drug development and product launch, the
significant depth of talent in our development team allows us to
continue to build momentum in the SAPPHIRE pivotal and Phase 1
DRAGON proof-of-concept clinical trials without disruption.”
Pipeline Updates
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).
- Enrollment Ongoing for Phase 3 SAPPHIRE Clinical Trial
Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3
SMA. The randomized, double-blind, placebo-controlled Phase 3
SAPPHIRE clinical trial is evaluating apitegromab for patients on
either nusinersen or risdiplam. The clinical trial 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.
- Clinical Data from the Phase 2 TOPAZ 24-Month Extension
Trial to be Presented at Cure SMA Annual Meeting in June.
- Data from the Phase 2 TOPAZ Trial Presented at the Muscular
Dystrophy Association (MDA) Clinical & Scientific Conference
and the American Academy of Neurology Annual Meeting in March, as
well as the European Paediatric Neurology Society Congress in
April.
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.
- Enrollment Ongoing for Part B of the Phase 1 DRAGON POC
Clinical Trial for SRK-181. 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. Initial evidence of
drug activity and safety data are anticipated in 2022.
First Quarter 2022 Financial Results
For the quarter ended March 31, 2022, net loss was $8.0 million
or $0.21 per share compared to a net loss of $27.7 million or $0.76
per share for the quarter ended March 31, 2021.
- Revenue was $33.2 million for the quarter ended March 31, 2022
compared to $4.7 million for the quarter ended March 31, 2021 and
was related to the Gilead fibrosis-focused research collaboration,
which was executed in December 2018 and concluded in December
2021.
- Research and development expense was $29.4 million for the
quarter ended March 31, 2022 compared to $22.5 million for the
quarter ended March 31, 2021. The increase year-over-year primarily
reflects increased clinical costs associated with apitegromab and
higher personnel costs. The Company expects research expenses to
decline beginning in the third quarter of 2022 due to the portfolio
updates and workforce reduction announced today.
- General and administrative expense was $10.8 million for the
quarter ended March 31, 2022 compared to $9.4 million for the
quarter ended March 31, 2021. The increase year-over-year was
primarily attributed to higher personnel costs. As a result of our
reduction in force, we expect our employee-related expenses to
decline in the second half of the year as compared to the first
half of 2022.
- As of March 31, 2022, Scholar Rock had cash, cash equivalents,
and marketable securities of approximately $210 million. The
Company expects that the updates and changes announced today will
fund its anticipated operating and capital expenditure requirements
into the fourth quarter of 2023.
“While we have paused many of our discovery programs, we are
continuing to progress selected pre-clinical programs which best
exemplify the value of our platform. As a platform company,
business development remains an important part of our strategy and
we continue to explore partnerships for these programs which could
be a source of non-dilutive capital in the future,” said Ted Myles,
Chief Operating Officer and Chief Financial Officer at Scholar
Rock. “Our streamlined focus and structure allow us to channel the
majority of our resources to supporting the SAPPHIRE trial and
SRK-181, which we believe are key near- and long-term value
drivers.”
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, additional data generated from clinical trials of
the same product candidate, including, without limitation, the
TOPAZ extension clinical trial and the Phase 3 SAPPHIRE clinical
trial of apitegromab in SMA or Part B of the Phase 1 DRAGON
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 March 31, 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 March
31
2022
2021
Revenue
$
33,193
$
4,708
Operating expenses Research and development
29,366
22,549
General and administrative
10,760
9,366
Total operating expenses
40,126
31,915
Loss from operations
(6,933
)
(27,207
)
Other income (expense), net
(1,017
)
(464
)
Net loss
$
(7,950
)
$
(27,671
)
Net loss per share, basic and diluted
$
(0.21
)
$
(0.76
)
Weighted average common shares outstanding, basic and
diluted
37,456,574
36,380,438
Scholar Rock Holding
Corporation
Condensed Consolidated Balance
Sheets
(unaudited)
(in thousands)
March 31, 2022
December 31, 2021
Assets Cash, cash equivalents and marketable securities
$
210,211
$
252,994
Other current assets
18,375
12,325
Total current assets
228,586
265,319
Other assets
37,391
39,126
Total assets
$
265,977
$
304,445
Liabilities and Stockholders' Equity Current
liabilities
$
33,373
$
64,297
Long-term liabilities
61,288
68,074
Total liabilities
94,661
132,371
Total stockholders' equity
171,316
172,074
Total liabilities and stockholders' equity
$
265,977
$
304,445
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220516005286/en/
Scholar Rock Contacts: Investors Rushmie Nofsinger
Scholar Rock 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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