Spherical Nucleic Acid Technology Shows Promising Results in Phase 0 Trial in Patients With Glioblastoma at Northwestern Univ...
March 11 2021 - 10:03AM
Business Wire
Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and
immunotherapeutic drugs utilizing spherical nucleic acid (SNA™)
technology, is pleased to share that researchers at Northwestern
University have utilized Exicure’s licensed first generation
gold-nanoparticle SNA technology in an investigator-initiated trial
for the treatment of glioblastoma (GBM), a deadly form of brain
cancer.
The results were published in an article in Science
Translational Medicine.
https://stm.sciencemag.org/content/13/584/eabb3945
DOI:10.1126/scitranslmed.abb3945
The researchers that led the study, Drs. Priya Kumthekar and
Alexander Stegh, conducted a single-arm, open-label phase 0
first-in-human clinical trial (NCT03020017) to determine safety,
pharmacokinetics, intratumoral accumulation and gene-suppressive
activity of systemically administered SNAs comprised of siRNA
specific for the GBM oncogene Bcl2L12. Patients with recurrent GBM
were treated with intravenous administration of siBcl2L12-SNAs
(NU-0129).
The paper reports first-in-human evidence that siRNA-based SNAs
can be administered intravenously, cross the blood-brain-barrier in
glioblastoma patients, accumulate in GBM tumor cells, and engage
with their target gene.
“We congratulate Drs. Kumthekar and Stegh on these results,”
said Dr. David Giljohann, the CEO of Exicure. He added, “We are
excited about the impact to patients and its implications in
treating other diseases, as Exicure continues to build and grow our
pipeline in neurological disorders.”
Exicure anticipates filing an IND for its lead program in
Friedreich’s ataxia by the end of 2021.
About Glioblastoma
Glioblastoma (GBM) is the most common and most aggressive type
of primary malignant tumor of the central nervous system. The
global incidence of glioblastoma is less than 10 per 100,000
people. Standard treatment for patients with newly diagnosed
glioblastoma can include surgery followed by radiation and
chemotherapy, but treatment options are limited. The last
investigational medicine to improve survival for patients with
newly diagnosed glioblastoma was approved by the U.S. Food and Drug
Administration in 2005. The five-year survival rate of patients
with GBM is less than five percent.
About Friedreich’s Ataxia
Friedreich’s ataxia (FA) 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 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 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.
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, the initiation, timing and results of the Company’s
preclinical studies and clinical trials, including its lead program
in Friedreich’s ataxia; and 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
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 coronavirus disease 2019, or
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; 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, which is expected to be filed on or
approximately on the same date hereof, 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. In addition, the COVID-19 pandemic and
the associated containment efforts have had and continue to have a
serious adverse impact on the economy, the severity and duration of
which are uncertain. Government stabilization efforts have only
partially mitigated the consequences. The extent and duration of
the impact on the Company’s business and operations is highly
uncertain, and that impact includes effects on its clinical trial
operations, timelines and supply chain. Factors that will influence
the impact on the Company’s business and operations include the
duration and extent of the pandemic, the extent of imposed or
recommended containment and mitigation measures, and the general
economic consequences of the pandemic. The COVID-19 pandemic or its
impact or effects could have a material adverse impact on the
Company’s business, operations and financial results for an
extended period of time.
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version on businesswire.com: https://www.businesswire.com/news/home/20210311005676/en/
Karen Sharma MacDougall 781-235-3060 ksharma@macbiocom.com
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