Entered Exclusive Collaboration with Ipsen
Targeting Two Rare Neurodegenerative Disorders
Presented Progress in Three Promising
Neuroscience Programs including Friedreich’s Ataxia, Neuropathic
Pain and Batten Disease at R&D Day
Announced Interim Results for Phase 1b/2 trial
of Cavrotolimod (AST-008) and advancement of enrollment in MCC
cohort
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
June 30, 2021 and provided an update on corporate progress.
“We continue to drive towards a number of key value inflection
points across our platforms and programs,” commented Dr. David
Giljohann, Exicure’s Chief Executive Officer. “Notably, we believe
our momentum in neurology is particularly strong on the heels of
our recently announced collaboration with Ipsen for Huntington’s
disease and Angelman syndrome. Our team has also made impressive
progress in our preclinical neurology pipeline, with a planned IND
filing in Friedreich’s ataxia and work in pain and Batten disease
using our SNA technology.”
Pipeline Highlights & Updates
Neurology
Ipsen Collaboration
- On August 2, 2021, IPSEN BIOPHARM LIMITED (Ipsen) and Exicure
announced an exclusive collaboration agreement to research,
develop, and commercialize novel SNAs™ as potential investigational
treatments for Huntington’s disease (HD) and Angelman syndrome
(AS). Under the terms of the collaboration:
- Ipsen obtains two exclusive options to SNAs™ currently under
discovery evaluation for HD and AS;
- Exicure will be responsible for discovery and certain
preclinical development activities. In the event Ipsen exercises an
option with respect to the two collaboration programs, Ipsen will
be responsible for further development and worldwide
commercialization for the corresponding licensed product;
- Exicure received a $20 million upfront payment and is eligible
to receive up to $1 billion in option exercise fees and milestone
payments should Ipsen opt into both programs, as well as tiered
royalties.
XCUR-FXN – Friedreich’s Ataxia
- The Company hosted a virtual R&D Day on July 15, 2021 to
present new and previously unreleased preclinical data and discuss
progress with XCUR-FXN:
- Observed 2-3x fold change in measurable Frataxin protein in the
cerebellum and dorsal root ganglia (amongst other important brain
and spinal regions) in Pook800J mouse model indicating potential
for disease resolution;
- Showed no adverse, test-related histopathological findings in
repeat dose range finding rat study.
- The Company continues to expect to file for an IND for XCUR-FXN
in FA by the end of 2021 and to dose the first human patient in the
first half of 2022.
SCN9A – Neuropathic Pain
- Presented supporting data for targeting the SCN9A gene which
encodes for the NaV1.7 sodium channel, a sought after, but
difficult to drug target with highly selective SNA approach.
CLN3 – Batten Disease
- Generated in vivo Proof of Concept data of an SNA splice
switching mechanisms to correct mutations in the CLN3 gene and
upregulate CLN3 mRNA in the retinae of Batten disease mice.
Immuno-Oncology
Cavrotolimod (AST-008)
- 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 Merkel cell carcinoma (MCC) or cutaneous squamous cell
carcinoma (CSCC), is open and actively enrolling patients:
- As of August 4, 2021, total trial enrollment for the Phase 1b/2
trial including primary and exploratory cohorts was 51
patients.
- Interim results from the Phase 2 portion of the clinical trial
were reported on August 5, 2021 on 26 patients, 17 of whom were
evaluable.
- A complete response (CR) in one MCC patient met the
pre-specified Phase 2 stage threshold to continue advancing patient
enrollment in the MCC cohort;
- Injected and non-injected tumor lesions completely resolved in
the MCC patient with a CR, supporting systemic (abscopal)
effects;
- Eight evaluable patients were enrolled across either the CSCC
dose-expansion cohort, in which enrollment and data accrual is
continuing, or exploratory cohort;
- The confirmed overall response rate (ORR) in all evaluable MCC
patients enrolled in total in the Phase 1b/2 study was 21% (3 of
14) as of the July 1, 2021 data cutoff date, comprised of two CRs
and one partial response (PR);
- The Company expects to provide ORR results from the Phase 2
portion of the trial in the first half of 2022.
Corporate Updates
- Expanded the executive management team with the appointments of
Brian C. Bock as Chief Financial Officer and Elias D. Papadimas as
Chief Accounting Officer.
First Quarter Financial Results and Financial
Guidance
Cash Position: Cash, cash equivalents, short-term
investments, and restricted cash were $57.3 million as of June 30,
2021 compared to $68.6 million as of March 31, 2021.
Research and Development (R&D) Expenses: R&D
expenses were $10.8 million for the quarter ended June 30, 2021,
compared to $7.0 million for the quarter ended June 30, 2020. The
Company has increased full-time headcount in R&D from 41 at
June 30, 2020 to 66 at June 30, 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 $3.1 million for the quarter ended
June 30, 2021, compared to $2.2 million for the quarter ended June
30, 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 $14.3 million for the
quarter ended June 30, 2021 compared to net loss of $4.3 million
for the quarter ended June 30, 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 and higher G&A costs associated with an
increase in headcount.
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, including the $20 million upfront
payment received relating to the Company’s announced partnership
with Ipsen subsequent to June 30, 2021, will be sufficient to fund
its operations for at least the next 12 months.
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
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. For more information, visit Exicure’s
website at www.exicuretx.com.
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 could be
deemed forward looking including, but not limited to, statements
regarding the Company’s exclusive collaboration with Ipsen,
including the ability of the Company to realize contingent
milestone payments and royalties under the collaboration agreement
with Ipsen; statements regarding the design, timing and results of
the Company’s Phase 1b/2 clinical trial of cavrotolimod, including
patient enrollment expectations and opening of additional clinical
trial sites; the potential of cavrotolimod to provide therapeutic
benefit to patients with MCC and CSCC; the initiation, timing and
results of the Company’s other preclinical studies and clinical
trials, including XCUR-FXN; the ability of SNAs to potentially
enhance drug delivery to previously inaccessible target tissues and
other benefits of SNAs, including their ability to address the
genetic challenges posed by Friedreich’s ataxia and the potential
to provide therapeutic benefit to patients with Huntington’s
disease and Angelman syndrome; the advancement, timing and success
of the Company’s preclinical and clinical programs; 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 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; 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; risks that
preliminary results from preclinical studies and clinical trials
are not necessarily predictive of future results; the ability of
the Company to collaborate successfully with strategic partners;
regulatory developments; exposure to litigation, including patent
litigation, and/or regulatory actions; 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. All information in this press release is as of the date
of the release, and the Company undertakes no duty to update this
information, except as required by law.
EXICURE, INC.
UNAUDITED CONDENSED
CONSOLIDATED BALANCE SHEETS
(in thousands, except share
and per share data)
June 30, 2021
December 31,
2020
ASSETS
Current assets:
Cash and cash equivalents
$
42,587
$
33,262
Short-term investments
13,472
48,818
Accounts receivable
—
11
Prepaid expenses and other assets
4,588
4,231
Total current assets
60,647
86,322
Property and equipment, net
4,102
4,123
Right-of-use asset
8,281
8,606
Other noncurrent assets
1,437
1,393
Total assets
$
74,467
$
100,444
LIABILITIES AND STOCKHOLDERS’
EQUITY
Current liabilities:
Accounts payable
$
2,250
$
1,866
Accrued expenses and other current
liabilities
3,228
3,525
Deferred revenue, current
7,267
8,343
Total current liabilities
12,745
13,734
Long-term debt, net
16,730
16,589
Lease liability, noncurrent
7,696
7,959
Other noncurrent liabilities
656
656
Total liabilities
$
37,827
$
38,938
Stockholders’ equity:
Preferred stock, $0.0001 par value per
share; 10,000,000 shares authorized, no shares issued and
outstanding, June 30, 2021 and December 31, 2020
—
—
Common stock, $0.0001 par value per share;
200,000,000 shares authorized, 88,096,694 issued and outstanding,
June 30, 2021; 87,651,352 issued and outstanding, December 31,
2020
9
9
Additional paid-in capital
169,337
167,379
Accumulated other comprehensive income
5
83
Accumulated deficit
(132,711
)
(105,965
)
Total stockholders' equity
36,640
61,506
Total liabilities and stockholders’
equity
$
74,467
$
100,444
EXICURE, INC.
UNAUDITED CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share
and per share data)
Three Months Ended June
30,
Six Months Ended June
30,
2021
2020
2021
2020
Revenue:
Collaboration revenue
$
79
$
4,847
$
1,076
$
14,030
Total revenue
79
4,847
1,076
14,030
Operating expenses:
Research and development expense
10,843
7,008
21,105
13,083
General and administrative expense
3,098
2,229
5,990
4,803
Total operating expenses
13,941
9,237
27,095
17,886
Operating loss
(13,862
)
(4,390
)
(26,019
)
(3,856
)
Other (expense) income, net:
Dividend income
2
4
3
43
Interest income
43
267
131
627
Interest expense
(450
)
—
(859
)
(128
)
Other (expense) income, net
(2
)
(192
)
(2
)
153
Total other (expense) income, net
(407
)
79
(727
)
695
Net loss before provision for income
taxes
(14,269
)
(4,311
)
(26,746
)
(3,161
)
Provision for income taxes
—
—
—
—
Net loss
$
(14,269
)
$
(4,311
)
$
(26,746
)
$
(3,161
)
Basic and diluted loss per common
share
$
(0.16
)
$
(0.05
)
$
(0.30
)
$
(0.04
)
Weighted-average basic and diluted common
shares outstanding
88,043,447
87,174,533
87,948,440
87,126,847
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210812005197/en/
Media: Karen Sharma MacDougall 781-235-3060
ksharma@macbiocom.com
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