Exicure Announces Upcoming Neuroscience Pipeline Update at Virtual R&D Day
July 08 2021 - 7:00AM
Business Wire
Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory drugs
utilizing spherical nucleic acid (SNA™) technology, will host a
virtual R&D Day on Thursday, July 15, 2021 from 10:00 am to
11:00 am ET. The event will showcase Exicure’s neuroscience
pipeline, including its lead program for Friedreich’s Ataxia (FA),
XCUR-FXN, which is designed to address the underlying molecular
cause of FA.
Exicure will present new and previously unreleased preclinical
data and discuss progress with XCUR-FXN, which is on track for IND
filing in late Q4 2021. Additionally, Exicure will discuss its
perspective on XCUR-FXN’s competitive differentiation in FA and the
program’s path to clinical validation.
Also, Exicure will provide an update on its expanding pipeline
across several rare neurodegenerative diseases of high unmet need
and highlight progress with two preclinical programs targeting
SCN9A (Nav1.7) for neuropathic pain and CLN3 for Batten
Disease.
“We believe that our SNA platform is ideally suited to address
neurological disorders based on the demonstrated ability of SNAs in
nonclinical studies to achieve higher cellular uptake in all key
cell types and broad biodistribution in the CNS, including deep
brain regions,” says Dr. David Giljohann, Exicure’s CEO. “We are
excited to translate our platform’s capabilities to potential
benefits for patients as demonstrated by our bi-specific SNA,
XCUR-FXN for Friedreich’s Ataxia, anticipated to enter the clinic
in the first half of 2022.”
A live webcast will be available in the Events and Presentations
section of Exicure’s website on July 15, 2021 at 10:00 am ET. An
archived version will be available on the company website following
the event. Additional information can be found here:
https://event.on24.com/wcc/r/3305687/FA6C43097DA849A64659B1FB63A8C259
About Friedreich’s Ataxia
Friedreich’s Ataxia is the most commonly inherited ataxia, a
degenerative neuromuscular disease leading to progressive loss of
coordination and causing severe childhood disability and early
mortality, in most cases before age 40. It is a monogenic disorder
caused by mutations in the FXN gene resulting in reduced levels of
frataxin protein. FA affects about 13,500 people in the US, Europe,
Canada, and Australia combined. There are currently no approved
therapies for Friedreich’s Ataxia patients.
About SCN9A
SCN9A is the gene encoding Nav1.7, a trans-membrane sodium
channel, that plays a critical role in pain signal signaling.
Nav1.7 is strongly expressed in dorsal root ganglion (DRG) neurons,
which mediate transmission of peripheral pain signals to the brain.
Nav1.7’s critical role for pain signaling has been biologically
validated by human gain-of-function mutations leading to severe
pain conditions such as Inherited Erythromelalgia and Small Fiber
Neuropathy and human loss-of-function mutations lead to congenital
insensitivity to pain. Nav1.7-targeting therapies could provide a
novel treatment option for neuropathic pain conditions in which
currently available therapies are largely ineffective.
About CLN3 Batten Disease
CLN3 Batten Disease is a monogenic, autosomal recessive,
lysosomal storage disorder caused by mutations in the CLN3 gene
resulting in batten protein deficiency. Affected individuals
experience childhood blindness, pediatric dementia syndrome,
seizures, and early death between age 20 and 30. CLN3 Batten is
estimated to affect greater than 1,800 individuals in the United
States and Europe. There are currently no approved therapies for
these patients.
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 cavrotolimod (AST-008) is
currently in a Phase 2 clinical trial in patients with advanced
solid tumors. Exicure is in Chicago, IL and has an office 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 advancement of the Company’s
clinical programs and its expansion into neuroscience; its clinical
development of XCUR-FXN including developmental and regulatory
submission timelines and anticipated data read-outs; and the timing
and success of its preclinical programs including a program
targeting SCN9A for neuropathic pain and a program targeting CLN3
Batten Disease. 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 more severely than it has to date or more
severely than anticipated, which may have the effect of impacting
or delaying the company’s ongoing Phase 1b/2 clinical trial;
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 results of early clinical trials are
not always being predictive of future results; 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 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20210708005299/en/
For Media: Karen Sharma MacDougall 781-235-3060
ksharma@macbiocom.com
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