- Friedreich’s ataxia chosen as first
neurological indication
- Reported Phase 1b data for lead
immuno-oncology candidate in solid tumors
-Development partnership with Allergan in hair
loss disorders
- Cash and short-term investments on Dec. 31,
2019 of $110.8 million
Exicure, Inc. (NASDAQ:XCUR), the pioneer in gene regulatory and
immunotherapeutic drugs utilizing spherical nucleic acid (SNA™)
technology, today reported full year financial results for year
ended December 31, 2019 and provided an update on corporate
progress.
“2019 was a pivotal year for Exicure as we joined strong
scientific and clinical progress with the capital resources and
distinguished investors necessary to expand our pipeline and build
our organization,” said Dr. David Giljohann, Chief Executive
Officer of Exicure. “Looking ahead, we intend to invest in growing
a pipeline of drug candidates targeting neurological indications,
as well as advance our Merkel cell and cutaneous squamous cell
carcinoma programs into Phase 2 trials. We believe our ongoing
clinical progress in immuno-oncology and dermatology has influenced
our ability to expand our SNA platform in additional therapeutic
areas through collaborative partnerships. Our collaboration with
Allergan in hair loss disorders is just one of what we hope will be
a number of such partnerships expanding our SNA platform into new
therapeutic areas,” concluded Dr. Giljohann.
Corporate Progress
Key achievements for Exicure during 2019 include:
- Established Friedreich’s ataxia as Exicure’s first
neurological indication, being developed in collaboration with the
Friedreich’s Ataxia Research Alliance
- Announced preliminary data in the ongoing Phase 1b/2 trial
of lead immuno-oncology candidate AST-008 in patients with solid
tumors
- Entered a collaboration agreement with Allergan for two
discovery programs in hair loss disorders with $25 million upfront
and up to $725 million in potential milestones
- Expanded scientific advisory board to include neurology
experts Dr. Susan Perlman and Dr. Hank Paulson
- Expanded Board of Directors with the addition of Jeffrey L.
Cleland of Orpheris, Bali Muralidhar of Abingworth LLP, Bosun Hau
of Tybourne Capital Management, and Tim Walbert of Horizon
Therapeutics
- Raised approximately $90.8 million in gross proceeds from
the sale of common stock in two public offerings and up-listed to
the NASDAQ Global Market
Pipeline Updates
Neurology
- In December of 2019, Exicure announced the development of
XCUR-FXN, an SNA–based therapeutic candidate, for the treatment of
Friedreich’s ataxia (FA). FA is driven by triplet repeats in the
frataxin gene which compromises the patient’s ability to generate
adequate levels of frataxin protein. Exicure believes its SNA
technology has the potential to address this genetic challenge and
that its therapeutic strategy may lead to increases in the frataxin
protein. Exicure will be developing XCUR-FXN with guidance from,
and in collaboration with, the Friedreich’s Ataxia Research
Alliance. Preclinical research is ongoing and IND-enabling studies
for XCUR-FXN are expected in late 2020.
- In 2020, Exicure expects to continue pre-clinical research on
the application of its SNA technology in neurological conditions,
building on its early proof-of-concept work with nusinersen and its
new therapeutic candidate, XCUR-FXN. Exicure is currently exploring
additional neurological conditions, including spinocerebellar
ataxia, Batten disease, amyotrophic lateral sclerosis (ALS), and
Huntington’s disease.
Immuno-oncology; AST-008
- In December of 2019, Exicure announced preliminary results from
the Phase 1b study of AST-008 in patients with solid tumors.
AST-008 is an investigational SNA consisting of toll-like receptor
9 (TLR9) agonists designed for immuno-oncology applications, and is
being evaluated in combination with pembrolizumab in patients with
solid tumors. At that time the study had enrolled fourteen patients
including five melanoma patients, four Merkel cell carcinoma (MCC)
patients, two cutaneous squamous cell carcinoma patients, two head
and neck squamous cell carcinoma patients, and one mucosal melanoma
patient. Prior to enrolling, most patients had progressive disease
on anti-PD-1 or anti-PD-L1 antibodies.
- Available data, as of December 11, 2019, showed that AST-008
administration, alone or in combination with pembrolizumab,
produced cytokine and chemokine expression and immune cell
activation in patient blood indicative of desired immune
activation. Of the 4 MCC patients, one patient, who had previously
progressed on anti-PD-1 antibody therapy, had confirmed stable
disease with decreased target lesion diameters for a period in
excess of twelve weeks, while a second MCC patient experienced a
target lesion complete response and a confirmed overall partial
response longer than 24 weeks. No treatment-related serious adverse
events or dose-limiting toxicities have been observed. The most
common reported adverse event was injection site reactions.
- Exicure is now completing the Phase 1b study and preparing to
begin a Phase 2 study in both Merkel cell carcinoma and in
cutaneous squamous cell carcinoma. Exicure currently has seven
trial sites open and seeks to expand to about fifteen sites.
Collaborations
Exicure entered into a collaboration with Allergan
Pharmaceuticals International Limited in late 2019 and is now
actively engaged in preclinical research and discovery in two
clinical programs related to the treatment of hair loss disorders.
Under the terms of the collaboration, Exicure received a $25
million upfront payment and is eligible to receive up to $725
million in potential milestones. In early 2019, Exicure also
entered into a collaboration agreement with Dermelix
Biotherapeutics under which Dermelix will develop a targeted
therapy for the treatment of Netherton Syndrome (NS).
2019 Financial Results and Financial Guidance
Cash Position: As of December 31, 2019, Exicure had cash and
cash equivalents of $48.5 million and short-term investments of
$62.3 million for a total of $110.8 million compared to $26.3
million of cash and cash equivalents and no short-term investments
as of December 31, 2018. In 2019, Exicure raised approximately
$90.8 million in gross proceeds from the sale of common stock and
received a $25 million upfront payment in connection with the
Collaboration Agreement with Allergan.
Research and Development Expense: Research and development
expense was $19.3 million for the year ended December 31, 2019
compared to $14.1 million for the year ended December 31, 2018. The
increase in research and development expense of $5.2 million was
primarily due to higher platform and discovery-related expenses of
$4.7 million, higher employee-related expense of $0.9 million and
higher facilities, depreciation, and other expenses of $0.2
million, partially offset by a net decrease of $0.6 million in
costs related to our clinical development programs. The increase in
platform and discovery-related expenses is mostly due to a license
fee of $3.8 million paid to Northwestern University in connection
with the $25.0 million upfront payment received from Allergan.
General and Administrative Expense: General and administrative
expense was $8.6 million for the year ended December 31, 2019 and
$7.8 million for the year ended December 31, 2018, an increase of
$0.8 million. This increase was primarily due to higher
compensation and related expenses, recruiting fees in connection
with adding two new board members, higher D&O insurance
premiums, Nasdaq listing costs and lease costs associated with our
Cambridge, MA office. These increased costs were partially offset
by reductions in legal and other transaction costs.
Net Loss: Net loss was $26.3 million for the year ended December
31, 2019, compared to net loss of $22.4 million for the year ended
December 31, 2018, an increase in loss of $3.9 million. Net loss
reflects the changes in expenses discussed above and is offset by
an increase in revenue of $1.2 million for the year ended December
31, 2019 compared to revenue for the year ended 2018. Revenue in
2019 was primarily related to the Dermelix Collaboration.
Cash Runway Guidance: Exicure believes that, based on its
current operating plans and as of the date of this press release,
its existing cash and cash equivalents as of December 31, 2019 is
sufficient to meet its anticipated cash requirements into early
2022.
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 no
FDA-approved treatments. An estimated 5,000 patients in the US and
15,000 patients worldwide are affected by FA.
About FARA
The Friedreich's Ataxia Research Alliance (FARA) is a 501(c)(3),
non-profit, charitable organization dedicated to accelerating
research leading to treatments and a cure for Friedreich's ataxia.
www.CureFA.org.
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 our
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 an
SNA–based therapeutic candidate, for the treatment of Friedreich’s
ataxia (FA). Exicure's drug candidate AST-008 is in a Phase 1b/2
trial in patients with advanced solid tumors. Exicure is based
outside of Chicago, IL and in Cambridge, MA.
For more information, visit Exicure’s website at
www.exicuretx.com.
Exicure Forward-Looking Statements
This press release contains forward-looking statements
(including within the meaning of Section 21E of the United States
Securities Exchange Act of 1934, as amended, and Section 27A of the
United States Securities Act of 1933, as amended) concerning the
Company, the Company’s technology, potential therapies and other
matters. Forward-looking statements generally include statements
that are predictive in nature and depend upon or refer to future
events or conditions, and include words such as “may,” “will,”
“should,” “would,” “expect,” “plan,” “believe,” “intend,” “look
forward,” and other similar expressions among others. Statements
that are not historical facts are forward-looking statements.
Forward-looking statements are based on 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:
unexpected costs, charges or expenses that reduce cash runway; that
Exicure’s pre-clinical 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; and the ability of Exicure to protect its
intellectual property rights. Furthermore, data from preclinical
studies often fails to be indicative of outcomes in human trials.
Risks facing the Company and its programs are set forth in the
Company’s filings with the SEC. Except as required by applicable
law, the Company undertakes no obligation to revise or update any
forward-looking statement (including without limitation its cash
runway guidance) or to make any other forward-looking statements,
whether as a result of new information, future events or
otherwise.
EXICURE, INC.
UNAUDITED CONSOLIDATED BALANCE
SHEETS
(in thousands, except share
and per share data)
December 31,
2019
2018
ASSETS
Current assets:
Cash and cash equivalents
$
48,460
$
26,268
Short-term investments
62,326
—
Accounts receivable
16
—
Unbilled revenue receivable
19
3
Prepaid expenses and other assets
1,955
1,392
Total current assets
112,776
27,663
Property and equipment, net
2,099
1,061
Other noncurrent assets
388
32
Total assets
$
115,263
$
28,756
LIABILITIES AND STOCKHOLDERS’
EQUITY
Current liabilities:
Current portion of long-term debt
$
4,965
$
—
Accounts payable
1,814
500
Accrued expenses and other current
liabilities
2,435
1,543
Current portion of deferred revenue
21,873
—
Total current liabilities
31,087
2,043
Long-term debt, net
—
4,925
Common stock warrant liability
414
797
Deferred revenue non-current
2,956
—
Other noncurrent liabilities
59
39
Total liabilities
$
34,516
$
7,804
Stockholders’ equity:
Common stock, $0.0001 par value per share;
200,000,000 shares authorized, 86,069,263 issued and outstanding,
December 31, 2019; 44,358,000 shares issued and outstanding,
December 31, 2018
9
4
Additional paid-in capital
162,062
75,942
Accumulated other comprehensive loss
(27
)
—
Accumulated deficit
(81,297
)
(54,994
)
Total stockholders' equity
80,747
20,952
Total liabilities and stockholders’
equity
$
115,263
$
28,756
EXICURE, INC.
UNAUDITED CONSOLIDATED
STATEMENTS OF OPERATIONS
(in thousands, except share
and per share data)
Year Ended December
31,
2019
2018
Revenue:
Collaboration revenue
$
1,296
$
118
Total revenue
1,296
118
Operating expenses:
Research and development expense
19,340
14,119
General and administrative expense
8,573
7,818
Total operating expenses
27,913
21,937
Operating loss
(26,617
)
(21,819
)
Other income (expense), net:
Dividend income
543
323
Interest income
178
4
Interest expense
(786
)
(672
)
Other income (loss), net
379
(249
)
Total other income (loss), net
314
(594
)
Net loss
$
(26,303
)
$
(22,413
)
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200303005295/en/
Media Contact: MacDougall Karen Sharma 781-235-3060
ksharma@macbiocom.com
Investor Contact: Stern IR, Inc. Kerry Conlin
212-362-1200 kerry.conlin@sternir.com
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