Scholar Rock Reports Second Quarter 2019 Financial Results and Highlights Business Progress
14 August 2019 - 9:30PM
Scholar Rock Holding Corporation (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 quarter ended June 30,
2019 and highlighted recent progress and upcoming milestones for
its pipeline programs.
“Throughout the first half of 2019, we made significant progress
both clinically and financially as we advanced SRK-015 into a Phase
2 trial in SMA, SRK-181 towards the initiation of a Phase 1 trial
in cancer immunotherapy in mid-2020, as well as successfully
completed a follow-on offering to ensure that we are
well-capitalized to support our portfolio of product candidates and
overall company growth,” said Nagesh Mahanthappa, Ph.D., President
and CEO of Scholar Rock. “We look forward to building upon
this momentum as we continue to progress our broad pipeline, with a
number of important milestones at the end of this year and into
2020.”
Company Highlights and
Upcoming Milestones
SRK-015 Program:
- Positive Final Results from the Phase 1 Clinical Trial
of SRK-015 in Healthy Volunteers Presented at the Cure SMA Annual
Conference. In June 2019, Scholar Rock presented positive
detailed results from the Phase 1 trial of SRK-015, which consisted
of data from both the single-ascending and multiple-ascending dose
portions of the trial. SRK-015 was observed to be
well-tolerated with no dose-limiting toxicities identified in up to
the highest evaluated dose of 30 mg/kg. The pharmacologic
profile showed robust engagement of latent myostatin, which was
saturated and sustained up to at least Day 140 after three doses of
SRK-015 given once every two weeks at 20 mg/kg or 30 mg/kg.
In contrast, no meaningful change was observed in the latent
myostatin biomarker concentrations in subjects who received
placebo.
- Preliminary Pharmacokinetic (PK) and Pharmacodynamic
(PD) Data from TOPAZ Phase 2 Trial Expected by End
of 2019. Patient enrollment is underway in the TOPAZ
Phase 2 proof-of-concept trial to assess the safety and efficacy of
SRK-015 in patients with Type 2 and Type 3 Spinal Muscular Atrophy
(SMA). Approximately 55 patients are anticipated to be
enrolled in the U.S., Canada, and Europe across three distinct and
parallel cohorts and treated with SRK-015, either as a monotherapy
or in conjunction with an approved survival motor neuron (SMN)
upregulator therapy. Scholar Rock is on track to report preliminary
PK/PD data for a subset of patients in the Phase 2 TOPAZ trial by
the end of 2019, which will provide initial observations on target
engagement of latent myostatin in patients with Type 2 and Type 3
SMA. These preliminary PK/PD results will be followed by
interim safety and efficacy results for a subset of patients with
six months of treatment exposure expected in the first half of 2020
and top-line data for the full 12-month treatment period expected
beginning in the fourth quarter of 2020.
- Details of Scholar Rock-Developed Biomarker Assay that
Quantifies Serum Latent Myostatin Published in Peer-Reviewed
Journal. In July 2019, Scholar Rock published
details of its immunoassay used to measure serum latent myostatin
in peer-reviewed journal Society of Laboratory Automation and
Screening Discovery. The publication, “A Sensitive and
Selective Immunoassay for the Quantitation of Serum Latent
Myostatin After In-Vivo Administration of SRK-015, a Selective
Inhibitor of Myostatin Activation,”(1) highlights the ability of
the immunoassay to quantify serum latent myostatin without
cross-reactivity to promyostatin, mature myostatin, or closely
related members of the TGFβ superfamily and the establishment of
the robustness, reproducibility, and stability of the
immunoassay.Through use of this biomarker assay, robust and
sustained target engagement of latent myostatin has been observed
in both preclinical studies and the Phase 1 clinical trial in
healthy volunteers, establishing proof-of-mechanism of Scholar
Rock’s therapeutic approach of targeting the latent form of growth
factors.
- Identification of Second Indication for SRK-015 Planned
for 2020. Scholar Rock continues to evaluate
multiple potential opportunities for which SRK-015 could offer
clinical benefit and is assessing additional potential clinical
settings in which the selective inhibition of the activation of
myostatin may offer therapeutic benefit.
SRK-181 Program:
- Initiation of the Phase 1 Clinical Trial of SRK-181 in
Patients with Solid Tumors Anticipated in Mid-2020.
In March 2019, Scholar Rock selected SRK-181, a highly specific
inhibitor of TGFβ1 activation, as the first product candidate in
its TGFβ1 cancer immunotherapy program based on the strength of
preclinical data and human translational insights. Scholar
Rock intends to develop SRK-181 for the treatment of tumors
resistant to checkpoint blockade therapies (CBTs), such as
anti-PD(L)1 antibodies, and believes there is significant
opportunity to expand the number of patients who can benefit from
CBTs. Scholar Rock plans to initiate a Phase 1 trial in
patients with primary resistance to approved checkpoint blockade
therapies in mid-2020 with initial clinical data anticipated by the
end of 2021.
RGMc Program:
- Nomination of a Product Candidate from the RGMc Program
Planned in First Half of 2020. For its third product
candidate, Scholar Rock is evaluating a number of highly specific
inhibitors of repulsive guidance molecule C (RGMc) and plans to
nominate an antibody in the first half of 2020. RGMc is a
co-receptor of bone morphogenetic protein 6 (BMP6), a member of the
TGFβ superfamily that plays an important role in iron metabolism.
RGMc’s known function is localized to hepatocytes and the
identification of RGMc selective-antibodies may offer the potential
for liver-specific modulation of BMP6 signaling to address
iron-restricted anemias.
Corporate Update:
- Public Offering of Common Stock Completed in
June/July 2019. Scholar Rock successfully
completed a public offering of 3,450,000 shares of common stock,
inclusive of the full exercise of the over-allotment option by the
underwriters, raising gross proceeds of approximately $51.8
million.
Second Quarter 2019 Financial Results
For the quarter ended June 30, 2019, net loss was $12.5 million
or $0.48 per share compared to a net loss of $14.7 million or $1.39
per share for the quarter ended June 30, 2018.
- Research and development expense was $13.7 million for the
quarter ended June 30, 2019 compared to $11.4 million for the
quarter ended June 30, 2018. The increase year-over-year
reflects preclinical and manufacturing costs for SRK-181 and higher
personnel-related costs, partially offset by a decrease
year-over-year in manufacturing costs associated with
SRK-015.
- General and administrative expense was $4.7 million for the
quarter ended June 30, 2019 compared to $3.5 million for the
quarter ended June 30, 2018. The increase year-over-year was
primarily attributable to increased headcount, stock compensation,
and higher operational fees associated with operating as a public
company.
As of June 30, 2019, Scholar Rock had cash, cash equivalents,
and marketable securities of $185.1 million, compared to $175.6
million as of December 31, 2018. The cash balance as of June 30,
2019 is inclusive of the approximately $42.2 million in net
proceeds from the public offering of 3,000,000 shares of common
stock and exclusive of $6.3 million in net proceeds from the
underwriter’s purchase of an additional 450,000 shares of common
stock that was settled in July 2019.
- Cote, S. M., Jackson, J., Pirruccello-Straub, M., Carven, G.
J., & Wawersik, S. (2019). A Sensitive and Selective
Immunoassay for the Quantitation of Serum Latent Myostatin after In
Vivo Administration of SRK-015, a Selective Inhibitor of Myostatin
Activation. SLAS DISCOVERY: Advancing Life Sciences R&D.
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 newly elucidated
understanding of 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.Scholar Rock®
is a registered trademark of Scholar Rock,
Inc.Forward-Looking StatementsThis 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 SRK-015, SRK-181,
and other product candidate and indication selection and
development timing, and the ability of any product candidate to
perform in humans in a manner consistent with nonclinical or
preclinical study data. 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, and 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
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, 2019, 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
Contact: Investors/Media Catherine Hu chu@scholarrock.com
917-601-1649Media Contact: The Yates Network
Kathryn Morris kathryn@theyatesnetwork.com 914-204-6412
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, |
|
|
|
2019 |
|
|
|
2018 |
|
|
|
2019 |
|
|
|
2018 |
|
|
|
|
|
|
|
|
|
|
Revenue |
$ |
5,039 |
|
|
$ |
- |
|
|
$ |
8,145 |
|
|
$ |
- |
|
|
|
|
|
|
|
|
|
|
Operating
expenses |
|
|
|
|
|
|
|
|
Research and development |
|
13,715 |
|
|
|
11,424 |
|
|
|
24,454 |
|
|
|
18,125 |
|
|
General and
administrative |
|
4,710 |
|
|
|
3,460 |
|
|
|
8,780 |
|
|
|
5,775 |
|
Total operating
expenses |
|
18,425 |
|
|
|
14,884 |
|
|
|
33,234 |
|
|
|
23,900 |
|
Loss from
operations |
|
(13,386 |
) |
|
|
(14,884 |
) |
|
|
(25,089 |
) |
|
|
(23,900 |
) |
Other income
(expense), net |
|
861 |
|
|
|
177 |
|
|
|
1,809 |
|
|
|
301 |
|
Net loss |
$ |
(12,525 |
) |
|
$ |
(14,707 |
) |
|
$ |
(23,280 |
) |
|
$ |
(23,599 |
) |
|
|
|
|
|
|
|
|
|
Net loss per share, basic and diluted |
$ |
(0.48 |
) |
|
$ |
(1.39 |
) |
|
$ |
(0.90 |
) |
|
$ |
(3.51 |
) |
|
|
|
|
|
|
|
|
|
Weighted-average common shares outstanding, basic and diluted |
|
25,922,833 |
|
|
|
10,593,987 |
|
|
|
25,758,658 |
|
|
|
6,716,283 |
|
|
Scholar Rock Holding Corporation |
|
Condensed Consolidated Balance
Sheets |
|
(unaudited) |
|
(in thousands) |
|
|
|
|
|
|
|
June 30, 2019 |
|
December 31, 2018 |
Assets |
|
|
|
|
Cash, cash equivalents and marketable securities |
$ |
185,131 |
|
$ |
175,645 |
|
Other current assets |
|
3,247 |
|
|
2,296 |
|
Total current assets |
|
188,378 |
|
|
177,941 |
|
Other assets |
|
8,533 |
|
|
3,395 |
Total assets |
$ |
196,911 |
|
$ |
181,336 |
|
|
|
|
|
Liabilities and Stockholders' Equity |
|
|
|
|
Current liabilities |
$ |
32,049 |
|
$ |
31,123 |
|
Long-term liabilities |
|
35,907 |
|
|
43,590 |
|
Total liabilities |
|
67,956 |
|
|
74,713 |
|
Total stockholders' equity |
|
128,955 |
|
|
106,623 |
Total liabilities and stockholders' equity |
$ |
196,911 |
|
$ |
181,336 |
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