- Confirmed overall response rate (ORR) of 21% in the
dose-escalation stage across all doses, confirmed ORR 33% at the
highest and selected Phase 2 dose
- Target tumor shrinkage was observed in 37% of patients
- Preliminary data show activity in patients with melanoma,
Merkel cell carcinoma (MCC), and cutaneous squamous cell carcinoma
(CSCC)
- Phase 2 arms in both MCC and CSCC are currently recruiting
Exicure, Inc. (NASDAQ: XCUR), the pioneer in gene regulatory and
immunotherapeutic drugs utilizing proprietary spherical nucleic
acid (SNA™) technology, announced that it will host a virtual Key
Opinion Leader (KOL) event to present preliminary Phase 1b efficacy
and safety data for its intratumoral product candidate,
cavrotolimod, the company’s SNA-enabled TLR9 agonist being
developed for the treatment of solid tumors, in combination with
pembrolizumab. The event will take place today from 10:30 am –
12:00 pm ET.
The principal investigators of the Phase 1b portion of the
trial, Dr. Steven O’Day and Dr. Shailender Bhatia, will be
presenting and joining Exicure’s leadership team in discussing the
clinical data and trial progress to date.
"We are excited by the durable responses we have seen in
anti-PD-1 refractory patients, and look forward to further
exploring the efficacy of cavrotolimod in these highly treatment
refractory Merkel cell carcinoma and cutaneous squamous cell
carcinoma patients," said Dr. Douglas Feltner, Chief Medical
Officer of Exicure.
The event will be webcast live today, September 16th at 10:30 am
ET through a link on the Events and Presentations section of
Exicure’s website. An archived webcast will also be available on
Exicure’s website following the event. To RSVP for the event,
please use the link here
(https://troutaccess.com/investor.php/c/ExicureKOLDay2020) or email
rjohn@troutgroup.com.
Description of the Trial
The objectives of the Phase 1b dose-escalation stage of the
clinical trial were to evaluate 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 weekly for 8 weeks then every three
weeks thereafter. Pembrolizumab was added to the treatment regimen
starting in week three of the study. The first two weeks of the
trial, the period in which only cavrotolimod was dosed, allowed for
the assessment of cavrotolimod safety, tolerability,
pharmacokinetics and pharmacodynamics alone. Efficacy was assessed
every 12 weeks. Twenty patients have been enrolled and dosed in the
dose-escalation stage of the trial consisting of: ten (10) melanoma
patients, five (5) MCC patients, two (2) CSCC patients, two (2)
head and neck squamous cell carcinoma patients, and one (1)
leiomyosarcoma patient. At the time of enrollment, 85% of patients
were experiencing progressive disease while on anti-PD-1 antibody
therapy.
Highlights from the data
Highlights from the data update include:
- Confirmed ORR 21% (4/19 patients) overall
in the Phase 1b dose-escalation stage
- Confirmed ORR 33% (2/6 patients) in the
highest dose cohort (32 mg), which was selected as 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 addition to the four confirmed
responses, target tumor shrinkage occurred in one CSCC patient and
two melanoma patients. Systemic (abscopal) effects were observed,
with regression in noninjected tumors distant from injected
lesions.
- The cavrotolimod pharmacodynamic profile
corroborated the efficacy data, as increased serum
cytokines/chemokines, activated immune cells, and tumor
infiltration by immune cells were observed.
The median duration of response has not been reached as all four
confirmed responders have not progressed after a median follow-up
of 11 months. The longest response to date is 16 months from
initial dosing and is ongoing as of the data cut-off date.
Exicure continues to observe that cavrotolimod is well tolerated
with 98% of all treatment-emergent adverse events (AEs) assessed as
Grade 1 or 2 in severity. No treatment-related serious adverse
events were reported to date. The most common adverse events were
flu-like symptoms and injection site reactions, which are commonly
expected effects from a TLR9 mechanism of action.
Updated guidance
Exicure expects to provide interim ORR results from the MCC and
CSCC cohorts of the Phase 2 portion of the clinical trial in the
first half of 2021 and final ORR results by year end 2021.
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.
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) including the design,
clinical development, therapeutic potential and clinical results
and expectations as to the reporting of data;. 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/20200916005263/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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