- Confirmed overall response rate (ORR) of 21%
in the Phase 1b dose-escalation stage across all doses, with 1
complete response and 3 partial responses
- Confirmed ORR 33% at the highest and
recommended Phase 2 dose
- Durable and ongoing responses at the time of
data analysis, with progression-free survival exceeding 6 months in
all 4 responders and 16 months in 2 responders
- Shrinkage of injected as well as regional or
distant noninjected tumors, supportive of systemic (abscopal)
effects
Exicure, Inc. (NASDAQ: XCUR), the pioneer in gene regulatory and
immunotherapeutic drugs utilizing proprietary spherical nucleic
acid (SNA™) technology, today announced the presentation of updated
Phase 1b data from the ongoing Phase 1b/2 clinical trial evaluating
intratumoral cavrotolimod (AST-008), the Company’s SNA-enabled TLR9
agonist, in combination with the anti-PD-1 therapies pembrolizumab
(KEYTRUDA®) or cemiplimab (LIBTAYO®), in patients with Merkel cell
carcinoma, cutaneous squamous cell carcinoma, and other advanced
solid tumors (NCT03684785).
The Phase 1b dose-escalation stage was designed to assess the
safety, tolerability, pharmacokinetics, and pharmacodynamics of
cavrotolimod alone and in combination with pembrolizumab, and to
identify a recommended Phase 2 dose. Cavrotolimod was dosed
intratumorally once weekly for 8 weeks and subsequently once every
3 weeks. Following an initial cavrotolimod monotherapy period,
pembrolizumab was initiated at week 3 and dosed in combination with
cavrotolimod once every 3 weeks. The Phase 1b stage enrolled
patients with locally advanced or metastatic melanoma (n=10),
Merkel cell carcinoma (n=5), cutaneous squamous cell carcinoma
(n=2), head and neck squamous cell carcinoma (n=2), and
leiomyosarcoma (n=1).
Updated data from the Phase 1b stage demonstrated that the
combination of cavrotolimod and pembrolizumab continued to be well
tolerated, with a confirmed overall response rate (ORR) of 21%
(4/19 patients) according to RECIST v1.1 criteria. The combination
immunotherapy regimen induced durable and systemic anti-tumor
responses in patients with advanced solid tumors who previously
progressed on anti-PD-1 therapy. At the time of enrollment in the
clinical trial, 85% of patients were experiencing progressive
disease despite treatment with PD-1 blockade and 65% of patients
had been treated with 2 or more lines of systemic therapy.
Highlights from the Phase 1b Dose-Escalation Stage
- The RECIST-confirmed ORR was 21% (4/19 patients) overall in
the Phase 1b dose-escalation stage, reflecting 1 complete response
and 3 partial responses.
- In the highest cavrotolimod dose cohort (32 mg), which was
selected as the Phase 2 dose, the RECIST-confirmed ORR was 33% (2/6
patients).
- Responses were durable and ongoing at the time of data
analysis, with progression-free survival exceeding 6 months in all
4 responders and 16 months in 2 responders.
- 85% of patients overall (17/20 patients) and 75% of responders
(3/4) were progressing on anti-PD-1 therapy at the time of trial
enrollment.
- Systemic (abscopal) effects were observed, with shrinkage of
regional or distant noninjected tumors.
- The majority (98%) of treatment-related adverse events were
grade 1 or grade 2. No dose-limiting toxicities and no
treatment-related serious adverse events were reported in the Phase
1b stage. The most common adverse events were flu-like symptoms and
injection site reactions, consistent with other adverse events
experienced with local and systemic immune activation associated
with TLR9 agonism.
Details of the poster presentation are as follows:
Title: Safety and preliminary efficacy of intratumoral
cavrotolimod (AST-008), a spherical nucleic acid TLR9 agonist, in
combination with pembrolizumab in patients with advanced solid
tumors
Authors: Steven J. O’Day, Cesar A. Perez, Trisha M.
Wise-Draper, Glenn J. Hanna, Shailender Bhatia, Ciara M. Kelly,
Theresa M. Medina, Douglas E. Laux, Adil Daud, Sunandana Chandra,
Montaser Shaheen, Ling Gao, Melissa A. Burgess, Leonel
Hernandez-Aya, Emil M. deGoma, Weston L. Daniel, Douglas E.
Feltner, Laurel Sindelar, Robert E. Michel, Alice S. Bexon, Martin
Bexon, and Mohammed M. Milhem
Poster/Abstract Number: 423
The abstract and poster will be available on the SITC website
once the conference begins on Monday, November 9 at 8:00 a.m. EST.
The poster will also be made available on the Exicure website.
Live Q&A will take place Wednesday, November 11 from
5:15–5:45 p.m. EST and Friday, November 13 from 4:40–5:10 p.m.
EST.
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 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.
Exicure 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 ongoing Phase 1b/2
clinical trial of cavrotolimod (AST-008) . 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; 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 Quarterly Report on Form 10-Q for
the quarter ended June 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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version on businesswire.com: https://www.businesswire.com/news/home/20201109005342/en/
For Media: Karen Sharma MacDougall 781-235-3060
ksharma@macbiocom.com
For Investors: Thomas Hoffmann Solebury Trout
+1-646-378-2931 thoffmann@troutgroup.com
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