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 Fast Track designations for its
clinical product candidate, cavrotolimod (AST-008), for two
development programs:
- cavrotolimod in combination with anti-programmed death-1 (PD-1)
therapy for the treatment of patients with locally advanced or
metastatic Merkel cell carcinoma (MCC) refractory to prior
anti-PD-1 blockade; and
- cavrotolimod in combination with anti-PD-1/anti-PD-ligand 1
(anti-PD-(L)1) therapy for the treatment of patients with locally
advanced or metastatic cutaneous squamous cell carcinoma (CSCC)
refractory to prior anti-PD-(L)1 blockade
Fast Track is a designation granted by the FDA that is intended
to facilitate development and expedite review of drugs to address
an unmet medical need in the treatment of a serious
life-threatening condition, and for which nonclinical or clinical
data has demonstrated the potential of the drug candidate to
address this medical need.
"There is an urgent need to investigate novel immunotherapeutic
agents such as cavrotolimod that can be given to enhance the
clinical efficacy of immunotherapy, particularly in patients with
refractory solid tumors," said Dr. Adil Daud, M.D., Clinical
Professor at UCSF Helen Diller Family Comprehensive Cancer Center
and principal investigator in the Phase 1b/2 clinical trial of
cavrotolimod.
Cavrotolimod is a spherical nucleic acid toll-like receptor 9
(TLR9) agonist designed to robustly activate the patient’s innate
and adaptive immune systems in order to potentially induce potent
anti-cancer immune responses. The Phase 1b dose-escalation stage of
the open-label, multi-center trial was designed to evaluate the
safety, tolerability, pharmacokinetics, pharmacodynamics and
preliminary efficacy of intratumoral cavrotolimod injections alone
and in combination with intravenous pembrolizumab in patients with
advanced solid tumors. The patients from the Phase 1b stage
included those with advanced or metastatic MCC, head and neck
squamous cell carcinoma, CSCC, melanoma and leiomyosarcoma. A
summary of the key highlights from the Phase 1b stage of the trial
include:
- No observed treatment-related serious
adverse events (“SAEs”) or dose limiting toxicity (DLT);
- Confirmed overall response rate (ORR) in
21% of evaluable patients (4/19 patients) in the Phase 1b dose
escalation stage across all doses, reflecting 1 complete response
and 3 partial responses;
- Confirmed ORR in 33% of evaluate patients
(2/6 patients) in the highest dose cohort (32 mg), which was
selected as the Phase 2 recommended dose;
- Overall responses occurred in two patients
with advanced MCC and two patients with melanoma;
- Three of four responders were progressing
on anti-PD-1 therapy at the time of enrollment in the trial;
- Durable and ongoing responses, with
progression-free survival exceeding six months in all four
responders and 16 months in two responders;
- In addition to the four overall responses,
target tumor shrinkage occurred in one CSCC patient and two
melanoma patients;
- Increases in leukocytes in injected tumors
after cavrotolimod (AST-008) alone and in combination with
pembrolizumab versus baseline. Uninjected tumors also showed
increased immune cell levels after patients received cavrotolimod
(AST-008) plus pembrolizumab;
- Dose-dependent activation of key immune
cells, including cytotoxic T cells and natural killer cells, as
well as increases in cytokine/chemokine levels in patient blood
after cavrotolimod (AST-008) treatment alone, and cavrotolimod
(AST-008) plus pembrolizumab treatment;
- Systemic (abscopal) effects were observed,
with regression in noninjected tumors distant from injected
lesions; and
- The cavrotolimod pharmacodynamic profile
corroborated the efficacy data, as increased serum
cytokines/chemokines, activated immune cells, and tumor
infiltration by immune cells were observed.
“I am encouraged by the Phase 1b dose-escalation results and
excited about the potential of cavrotolimod to address the
significant unmet need facing these patients,” said Dr. Michael
Wong, M.D., Ph.D., Professor at MD Anderson Cancer Center and
principal investigator in the Phase 1b/2 clinical trial of
cavrotolimod.
“These Fast Track designations underscore the pressing need to
develop new therapies to treat refractory non-melanoma skin cancers
as well as the promising preclinical and initial clinical results
of cavrotolimod in patients with locally advanced or metastatic
Merkel cell carcinoma and cutaneous squamous cell carcinoma,” said
Dr. Shailender Bhatia, M.D., Associate Professor at University of
Washington/Fred Hutchinson Cancer Research Center and principal
investigator in the Phase 1b/2 clinical trial of cavrotolimod.
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 ability to advance
cavrotolimod (AST-008) in a Phase 2 clinical trial and its
potential benefit as a treatment for of Merkel cell carcinoma (MCC)
and the treatment of cutaneous squamous cell carcinoma. 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 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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MacDougall Karen Sharma 781-235-3060 ksharma@macbiocom.com
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