Exicure Announces Agenda for Upcoming Neuroscience Pipeline Update at Virtual R&D Day
January 04 2021 - 7:00AM
Business Wire
Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene
regulatory and immunotherapeutic drugs utilizing spherical nucleic
acid (SNA™) technology, will host a virtual R&D Day on
Thursday, January 7, 2021 from 09:00 am to 10:30 am ET to discuss
Exicure’s neuroscience pipeline, including its lead program for
Friedreich’s Ataxia (FA).
Exicure will discuss its progress with XCUR-FXN, which advanced
into IND-enabling studies in Q4 2020, and provide its perspective
on the path for the product candidate to clinical validation for
the treatment of Friedreich’s Ataxia. In addition, Exicure will
provide an update on its clinical pipeline across a number of rare
neurodegenerative diseases of high unmet need, and highlight the
advancement of two preclinical programs: one targeting SCN9A for
neuropathic pain and the other targeting CLN3 Batten Disease.
Underscoring the differentiating nature of Exicure’s SNA
platform for discovery of oligonucleotide-based therapeutics for
neurodegenerative diseases, Exicure will present results of CNS
biodistribution studies of SNA therapeutics in both rodent and
non-human primates.
Exicure’s Scientific Advisory Board member Dr. Susan Perlman and
the CEO of the Friedreich’s Ataxia Research Alliance (FARA)
Jennifer Farmer, will join Exicure’s leadership team in discussing
Exicure’s progress in the treatment of Friedreich’s Ataxia as well
as discuss its neuroscience pipeline.
Susan L. Perlman, MD, is a Clinical Professor in the Department
of Neurology at the David Geffen School of Medicine at UCLA, where
she is also the Director of Clinical Trials and Director, Ataxia
Clinic for UCLA’s Neurogenetics program. She is a member of the
Medical Research Advisory Board for the National Ataxia Foundation
and has been a primary investigator for several Friedreich’s Ataxia
trials.
Jennifer Farmer, MS is the CEO of the Friedreich’s Ataxia
Research Alliance (FARA). Since joining FARA in 2006, she has led
FARA’s efforts to establish clinical research infrastructure and
clinical trial readiness, grown the research grant program, and led
efforts to engage bio-pharmaceutical companies in drug discovery
and development for Friedreich’s Ataxia.
A live webcast will be available in the Events and Presentations
section of Exicure’s website on January 7, 2021 at 09:00 am ET. An
archived version will be available on the company website following
the event. Additional information can be found here:
https://troutaccess.com/index.php/c/ExicureResearchDay2021
About Friedreich’s Ataxia
Friedreich’s Ataxia is the most commonly inherited ataxia, a
degenerative neuromuscular disease leading to progressive loss of
coordination, 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 battenin 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 about 700 individuals in the United States.
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 AST-008 is currently in a
Phase 1b/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 timelines and anticipated data
read-outs; and 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, 2019, 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210104005186/en/
For Media: Karen Sharma MacDougall 781-235-3060
ksharma@macbiocom.com
Exicure (NASDAQ:XCUR)
Historical Stock Chart
From Jun 2024 to Jul 2024
Exicure (NASDAQ:XCUR)
Historical Stock Chart
From Jul 2023 to Jul 2024