— Company Advances Clinical and Pre-Clinical
Pipeline Designed to Bring Therapies to Patients With Unmet Medical
Needs —
Exicure, Inc.® (NASDAQ: XCUR), a pioneer in gene regulatory and
immunotherapeutic drugs utilizing spherical nucleic acid (SNA™)
technology, today reported financial results for the quarter ended
March 31, 2021 and provided an update on corporate progress.
“Exicure is off to a strong start in 2021, as we continue to
advance our clinical and pre-clinical programs and add depth to our
Board of Directors and management team,” commented Dr. David
Giljohann, Exicure’s Chief Executive Officer. “In the first
quarter, cavrotolimod (AST-008) was granted two Fast Track
designations and an Orphan Drug designation from the U.S. Food and
Drug Administration. We also strengthened our Board of Directors
with the appointments of Mr. James Sulat, Dr. Elizabeth Garofalo,
and Mr. Andrew Sassine, and added expertise to our management team
with the additions of Ms. Rebecca Peterson, VP, Investor Relations
& Corporate Communications and Ms. Sarah Longoria, VP, Human
Resources. We believe we are well positioned to execute on upcoming
clinical milestones and operational goals for the remainder of
2021.”
Pipeline Highlights
Immuno-Oncology – Cavrotolimod (AST-008)
- The U.S. Food and Drug Administration (FDA) granted two Fast
Track designations for cavrotolimod in January 2021:
- Cavrotolimod in combination with anti-programmed death (PD)-1
or anti-PD ligand 1 (PD-L1) therapy for the treatment of patients
with locally advanced or metastatic Merkel cell carcinoma (MCC),
refractory to prior anti-PD-(L)1 blockade.
- Cavrotolimod in combination with anti-PD-1 therapy for the
treatment of patients with locally advanced or metastatic cutaneous
squamous cell carcinoma (CSCC), refractory to prior anti-PD-1
blockade.
- The FDA granted Orphan Drug designation for cavrotolimod for
the treatment of patients with MCC in March 2021.
- The Phase 1b/2 clinical trial of intra-tumoral cavrotolimod in
combination with approved checkpoint inhibitors pembrolizumab or
cemiplimab, for the treatment of patients with advanced or
metastatic MCC or CSCC, is open and actively enrolling patients:
- In the first quarter of 2021, the Company continued enrolling
patients in the Phase 2 dose expansion stage of the Phase 1b/2
clinical trial.
- As of April 22, 2021, 20 clinical trial sites are open for
enrollment and seven additional sites are pending activation. The
Company plans to open up to 30 sites for the Phase 2 stage of the
clinical trial.
- The Company expects to provide interim results from the Phase 2
portion of the clinical trial in mid-2021, and we expect to report
overall response rate (ORR) results in the first half of 2022.
Neurology – XCUR-FXN
- The Company hosted a virtual R&D Day in January 2021 to
discuss progress within its neurology pipeline, which included an
overview of the Company’s XCUR-FXN path to clinical validation for
the treatment of Friedreich’s Ataxia (FA).
- During the first quarter of 2021, the Company continued to
advance preclinical and IND-enabling studies of XCUR-FXN in FA and
disclosed encouraging preclinical mouse data, demonstrating
significant upregulation of XCUR-FXN components in the brain and
spinal cord. The Company believes these data support the potential
for XCUR-FXN as a disease-modifying treatment for the disease.
- The Company is on track with prior guidance to submit an IND
for XCUR-FXN in FA by year-end 2021 and expects to initiate a
first-in-patient Phase 1b clinical trial of XCUR-FXN in FA in the
first half of 2022.
First Quarter Financial Results and Financial
Guidance
Cash Position: Cash, cash equivalents and short-term
investments were $67.4 million as of March 31, 2021 compared to
$82.1 million as of December 31, 2020.
Research and Development (R&D) Expenses: R&D
expenses were $10.3 million for the quarter ended March 31, 2021,
compared to $6.1 million for the quarter ended March 31, 2020. The
Company has increased full-time headcount in R&D from 36 at
March 31, 2020 to 54 at March 31, 2021. The increase in R&D
expense reflects this increased headcount and the related increase
in R&D activities, in addition to increased clinical trial
activities.
General and Administrative Expenses: General and
administrative expenses were $2.9 million for the quarter ended
March 31, 2021, compared to $2.6 million for the quarter ended
March 31, 2020. This increase is primarily due to costs related to
new hires needed to grow the Company as it evolves.
Net Loss: Exicure had a net loss of $12.5 million for the
quarter ended March 31, 2021 compared to net income of $1.2 million
for the quarter ended March 31, 2020. The increase in net loss was
primarily driven by lower revenue associated with Exicure’s
collaboration with AbbVie as well as higher R&D costs to
advance our pipeline.
Cash Runway Guidance: The Company believes that, based on
its current operating plans and estimates of future expenses, as of
the date of this press release, its existing cash, cash equivalents
and short-term investments will be sufficient to fund its
operations for at least the next 12 months.
About Friedreich’s Ataxia (FA)
FA is a rare, degenerative, life-shortening neuro-muscular
disorder that affects children and adults, and involves the loss of
strength and coordination usually leading to wheelchair use,
diminished vision, hearing and speech, scoliosis, increased risk of
diabetes, and a life-threatening heart condition. There are
currently no FDA-approved treatments. The Company estimates that
approximately 13,000 patients across the United States, Europe,
Canada and Australia are affected by FA.
About Exicure, Inc.
Exicure, Inc. is a clinical-stage biotechnology company
developing therapeutics for neurology, immuno-oncology,
inflammatory diseases and other genetic disorders based on its
proprietary Spherical Nucleic Acid, or SNA technology. Exicure
believes that its proprietary SNA architecture has distinct
chemical and biological properties that may provide advantages over
other nucleic acid therapeutics and may have therapeutic potential
to target diseases not typically addressed with other nucleic acid
therapeutics. Exicure is in preclinical development of XCUR-FXN a
lipid-nanoparticle SNA–based therapeutic candidate, for the
intrathecal treatment of Friedreich’s ataxia (FA). Exicure's
therapeutic candidate cavrotolimod (AST-008) is in a Phase 1b/2
clinical trial in patients with advanced solid tumors. Exicure is
based in Chicago, IL and in Cambridge, MA.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements in this press release other than statements of
historical fact should be deemed forward looking including, but not
limited to, statements regarding the design, timing and results of
the Company’s Phase 1b/2 clinical trial of cavrotolimod (AST-008)
including patient enrollment expectations and opening of additional
clinical trial sites; the initiation, timing and results of its
other preclinical studies and clinical trials, including XCUR-FXN;
the potential of the Company’s SNA technology to provide
therapeutic benefit to target diseases, including its ability to
address the genetic challenges posed by Friedreich’s ataxia and
other neurological conditions; the potential of the Company’s
collaborations and R&D efforts; the Company’s ability to
advance its clinical and preclinical pipeline to benefit patients
with unmet medical need; the Company’s expectations with respect to
its continued growth; and the Company’s anticipated cash runway.
The forward-looking statements in this press release speak only as
of the date of this press release, and the Company undertakes no
obligation to update these forward-looking statements.
Forward-looking statements are based on management’s current
beliefs and assumptions that are subject to risks and uncertainties
and are not guarantees of future performance. Actual results could
differ materially from those contained in any forward-looking
statement as a result of various factors, including, without
limitation: the risks that the ongoing COVID-19 pandemic or its
impact or effects may disrupt the Company’s business and/or the
global healthcare system (including its supply chain) more severely
than it has to date or more severely than anticipated, which may
have the effect of further impacting or delaying the Company’s
ongoing Phase 1b/2 clinical trial of cavrotolimod (AST-008);
unexpected costs, charges or expenses that reduce the Company’s
capital resources; the Company’s preclinical or clinical programs
do not advance or result in approved products on a timely or cost
effective basis or at all; the cost, timing and results of clinical
trials; that many drug candidates do not become approved drugs on a
timely or cost effective basis or at all; the ability to enroll
patients in clinical trials; possible safety and efficacy concerns;
regulatory developments; risks that preliminary results from
clinical trials are not necessarily predictive of future clinical
trial results; and the ability of the Company to protect its
intellectual property rights. For a discussion of other risks and
uncertainties, and other important factors, any of which could
cause the Company’s actual results to differ from those contained
in the forward-looking statements, see the section titled “Risk
Factors” in the Company’s Annual Report on Form 10-K for the year
ended December 31, 2020, as updated by the Company’s subsequent
filings with the Securities and Exchange Commission.
EXICURE, INC. UNAUDITED
CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands, except share
and per share data)
March 31, 2021
December 31,
2020
ASSETS
Current assets:
Cash and cash
equivalents...........................................................................................
$
34,296
$
33,262
Short-term
investments................................................................................................
33,140
48,818
Accounts
receivable.....................................................................................................
—
11
Prepaid expenses and other
assets..............................................................................
6,551
4,231
Total current
assets.........................................................................................................
73,987
86,322
Property and equipment,
net..........................................................................................
4,092
4,123
Right-of-use
asset...........................................................................................................
8,443
8,606
Other noncurrent
assets..................................................................................................
1,426
1,393
Total
assets........................................................................................
$
87,948
$
100,444
LIABILITIES AND STOCKHOLDERS’
EQUITY
Current liabilities:
Accounts
payable.........................................................................................................
$
2,976
$
1,866
Accrued expenses and other current
liabilities..........................................................
2,441
3,525
Deferred revenue,
current............................................................................................
7,346
8,343
Total current
liabilities...................................................................................................
12,763
13,734
Long-term debt,
net........................................................................................................
16,659
16,589
Lease liability, noncurrent
7,829
7,959
Other noncurrent
liabilities............................................................................................
656
656
Total
liabilities................................................................................................................
$
37,907
$
38,938
Stockholders’ equity:
Preferred stock, $0.0001 par value per
share; 10,000,000 shares authorized, no shares issued and
outstanding, March 31, 2021 and December 31, 2020...
—
—
Common stock, $0.0001 par value per share;
200,000,000 shares authorized, 87,993,598 issued and outstanding,
March 31, 2021; 87,651,352 issued and outstanding, December 31,
2020.............................................................
9
9
Additional paid-in
capital..............................................................................................
168,442
167,379
Accumulated other comprehensive income
(loss)........................................................
32
83
Accumulated
deficit.......................................................................................................
(118,442
)
(105,965
)
Total stockholders'
equity..............................................................................................
50,041
61,506
Total liabilities and stockholders’
equity
$
87,948
$
100,444
EXICURE, INC. UNAUDITED
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands,
except share and per share data)
Three Months Ended March
31,
2021
2020
Revenue:
Collaboration
revenue......................................................................................................................
$
997
$
9,183
Total
revenue......................................................................................................................
997
9,183
Operating expenses:
Research and development
expense......................................................................................................................
10,262
6,075
General and administrative
expense......................................................................................................................
2,892
2,574
Total operating
expenses......................................................................................................................
13,154
8,649
Operating (loss)
income......................................................................................................................
(12,157
)
534
Other (expense) income, net:
Dividend
income......................................................................................................................
1
39
Interest
income......................................................................................................................
88
360
Interest
expense......................................................................................................................
(409
)
(128
)
Other income,
net......................................................................................................................
—
345
Total other (expense) income,
net......................................................................................................................
(320
)
616
Net (loss) income before provision for
income
taxes......................................................................................................................
(12,477
)
1,150
Provision for income
taxes......................................................................................................................
—
—
Net (loss)
income......................................................................................................................
$
(12,477
)
$
1,150
Basic (loss) earnings per common
share......................................................................................................................
$
(0.14
)
$
0.01
Diluted (loss) earnings per common
share......................................................................................................................
$
(0.14
)
$
0.01
Weighted-average basic common shares
outstanding......................................................................................................................
87,852,378
87,079,160
Weighted-average diluted common shares
outstanding......................................................................................................................
87,852,378
88,244,632
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210512005906/en/
Exicure Contact: Rebecca Peterson VP, Head of Investor
Relations & Corporate Communications 617-678-5711
rpeterson@exicuretx.com
Media Contact: Karen Sharma MacDougall 781-235-3060
ksharma@macbiocom.com
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