Exicure Granted Orphan Drug Designation by the U.S. Food and Drug Administration for Cavrotolimod
March 03 2021 - 7:00AM
Business Wire
Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and
immunotherapeutic drugs utilizing spherical nucleic acid (SNA™)
technology, today announced that the U.S. Food and Drug
Administration (FDA) has granted Orphan Drug Designation for its
clinical product candidate, cavrotolimod (AST-008), for the
treatment of patients with Merkel cell carcinoma (MCC).
“We are excited to have been granted Orphan Drug Designation by
the FDA for cavrotolimod for MCC,” said Dr. Douglas Feltner, Chief
Medical Officer of Exicure. “This designation marks significant
progress toward our goal of fulfilling the unmet medical need and
finding a treatment for Merkel cell carcinoma patients.”
Exicure previously announced that the FDA granted two Fast Track
designations for cavrotolimod (AST-008) for MCC and cutaneous
squamous cell carcinoma (CSCC), both in the advanced/metastatic
setting after progression on anti-PD-1/PD-L1 antibodies.
About Orphan Drug Designation
Orphan Drug Designation is a designation granted by the FDA
which provides orphan status to drugs or biologics which are
intended to treat rare diseases or disorders that affect fewer than
200,000 people in the United States. This designation provides the
sponsor of the drug certain incentives, including tax credits for
qualified clinical trials and fee waivers. Orphan Drug Designation
confers eligibility for seven years of market exclusivity to an
orphan drug post-approval, subject to a receipt by the FDA of
marketing approval.
About Cavrotolimod (AST-008)
Cavrotolimod (AST-008) is an SNA consisting of toll-like
receptor 9 agonists designed for immuno-oncology applications. In
December 2019, Exicure announced preliminary results from the Phase
1b stage of the clinical trial including potential signs of
anti-tumor activity with cavrotolimod in combination with
pembrolizumab in cancer patients, including those with MCC. To
date, 20 patients in the Phase 1b stage of the clinical trial have
been dosed, and no cavrotolimod-related serious adverse event or
dose-limiting toxicity has been reported. The most commonly
reported adverse events were injection site reactions and flu-like
symptoms. In the second quarter of 2020, Exicure initiated Phase 2
dose expansion cohorts of intratumoral cavrotolimod in combination
with approved checkpoint inhibitors to treat two cohorts of
patients with advanced or metastatic MCC or CSCC. Each cohort is
expected to enroll up to 29 patients whose tumors have progressed
on anti-PD-1/PD-L1, or programmed cell death protein 1/programmed
death-ligand 1, antibody monotherapy.
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 therapeutic candidate cavrotolimod 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 business strategy
regarding cavrotolimod (AST-008) including its potential
therapeutic benefits as a treatment for Merkel cell carcinoma (MCC)
and cutaneous squamous cell carcinoma. 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 of
cavrotolimod; 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
Quarterly Report on Form 10-Q for the quarter ended September 30,
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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Media:
MacDougall Karen Sharma 781-235-3060 ksharma@macbiocom.com
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