Data Monitoring Committee Completes Mid-Study Review of Celsion’s Phase III ThermoDox® OPTIMA Study in Primary Liver Cance...
August 07 2017 - 8:00AM
OPTIMA Study at Over 60% Patient Enrollment, DMC
Provides Unanimous Recommendation to Continue
Celsion Corporation (NASDAQ:CLSN), an oncology drug development
company, today announced that the independent Data Monitoring
Committee (DMC) for the Company's 550 patient, multinational,
double-blind, placebo-controlled, pivotal Phase III clinical study
of ThermoDox® in combination with radiofrequency ablation (RFA) for
primary liver cancer (the OPTIMA study), has completed a planned
interim analysis of the first 50% of patients randomized in the
trial as of April 2017 for safety and efficacy and unanimously
recommended that the study continue according to protocol to its
final data readout based on the risk to benefit analysis by the
Committee. The OPTIMA study to date has accumulated data within
acceptable safety parameters.
The DMC is comprised of an independent group of
medical and scientific experts and is responsible for reviewing and
evaluating patient safety and efficacy data for the Company's Phase
III OPTIMA Study. The DMC reviews study data at regular intervals
in order to ensure the safety of all patients enrolled in the trial
and to monitor the quality and overall conduct of the trial
including each site’s compliance with the minimum RFA heating time
of 45 minutes specified in the study protocol. The Company also
announced that enrollment in the OPTIMA Study is now over 60% of
the 550 patients necessary to ensure that its primary end point,
overall survival, can be achieved with statistical
significance.
"Following independent confirmation of our
hypothesis by the National Institutes of Health (NIH) in November
2016 that ThermoDox® in combination with optimized RFA can be a
treatment with curative intent for primary liver cancer, we are
very pleased that the DMC has recommended continuation of the
OPTIMA Study without modification. Based on their review of all the
available study data from 275 patients enrolled as of April 2017,
the DMC has concluded that ThermoDox® is safe for newly diagnosed,
intermediate stage patients and that the study is being conducted
according to the highest of clinical research standards," said
Nicholas Borys, MD, Celsion's senior vice president and chief
medical officer. "A key component of the OPTIMA Study protocol is
the investigators’ adherence to the recommended RFA heating time
for tumors greater than 3 cm. We are pleased to report that
there has been a greater than 99% compliance rate with the study
protocol."
On November 29, 2016, the Company announced
results from an independent retrospective analysis conducted by the
NIH on the intent-to-treat population of the 701 patient HEAT Study
of ThermoDox® plus optimized RFA for the treatment of primary liver
cancer. The NIH analysis, which sought to evaluate the
correlation between RFA burn time per tumor volume (min/ml) and
clinical outcome, concluded that increased RFA “burn time” per
tumor volume significantly improved overall survival (OS) in
patients with solitary lesions treated with RFA + ThermoDox®
compared to patients treated with RFA alone. The NIH analysis
included 437 patients with a single lesion from the Company’s HEAT
Study, the same patient population being treated in the Company’s
ongoing Phase III OPTIMA study.
The NIH findings are consistent with Celsion’s
own analysis of the HEAT Study data, which demonstrated that over a
3.5 year period, there was a statistically significant survival
benefit of approximately 2 years for patients treated with
ThermoDox® plus optimized RFA over the optimized RFA only
group.
“We are very pleased that the DMC has
unanimously recommended continuation of the OPTIMA study based on
their review of all available clinical data, both safety and
efficacy, in over 275 patients," stated Mr. Michael H. Tardugno,
Celsion's chairman, president and chief executive officer. "The
DMC's affirmative review is further evidence of ThermoDox's
potential to provide a new and important first line therapeutic
option for patients with primary liver cancer."
Regulatory Strategy for
ThermoDox®. ThermoDox® has received FDA Fast Track
Designation and in the prior HEAT Study had been designated as a
Priority Trial for primary liver cancer by the National Institutes
of Health. ThermoDox® has been granted orphan drug designation for
primary liver cancer in both the U.S. and Europe. Based on prior
discussions and subject to a successful trial, Celsion fully
expects that the U.S. FDA and European EMA enrollment objectives
will be met, and that the OPTIMA Study will also enrolled a
sufficient number of patients to support registrational filings in
China, South Korea, Taiwan and Vietnam, four other large and
important markets for ThermoDox®.
In December 2016, the Company met with the China
Food and Drug Administration (CFDA) to discuss the ongoing Phase
III OPTIMA program and regulatory pathway for ThermoDox in China.
During the meeting, Celsion presented the final overall survival
data from the Chinese patient cohort from the prior HEAT study,
which demonstrated a survival benefit in patients treated with
ThermoDox plus optimized RFA versus optimized RFA alone. The CFDA
informed Celsion that if the ongoing Phase III OPTIMA trial is
successful, the trial could serve as the basis for a direct
regulatory filing in China without the need to file for prior
approval in the U.S. or European Union which is currently required
for foreign company application. This would allow the Company to
accelerate its plans for a regulatory filing in China and, if
approved, provide for a significantly earlier launch date in China
than originally expected.
The OPTIMA study's design and statistical plan
incorporates two pre-planned interim efficacy analysis by the DMC
(after patient enrollment is complete) with the intent of
evaluating safety, efficacy and futility to determine if there is
overwhelming evidence of clinical benefit or a low probability of
treatment success to continue, modify or terminate the study. The
results from the first interim efficacy analysis are expected to be
made public following DMC review in Q1 of 2019.
About the OPTIMA Study
The Phase III OPTIMA Study is expected to enroll
up to 550 patients in up to 70 clinical sites in the United States,
Europe, China and Asia Pacific, and will evaluate ThermoDox® in
combination with optimized RFA, which will be standardized to a
minimum of 45 minutes across all investigators and clinical sites
for treating lesions three to seven centimeters, versus optimized
RFA alone. The primary endpoint for the trial is Overall Survival,
which is supported by post-hoc analysis of data from the Company's
701 patient HEAT Study, where optimized RFA has demonstrated the
potential to significantly improve survival when combined with
ThermoDox®. The statistical plan calls for two interim efficacy
analyses by an independent Data Monitoring Committee.
About Celsion
Corporation
Celsion is a fully-integrated oncology company
focused on developing a portfolio of innovative cancer treatments,
including directed chemotherapies, immunotherapies and RNA- or
DNA-based therapies. The Company's lead program is ThermoDox®, a
proprietary heat-activated liposomal encapsulation of doxorubicin,
currently in Phase III development for the treatment of primary
liver cancer and in Phase II development for the treatment of
recurrent chest wall breast cancer. The pipeline also
includes GEN-1, a DNA-based immunotherapy for the localized
treatment of ovarian and brain cancers. Celsion has two
platform technologies for the development of novel nucleic
acid-based immunotherapies and other anti-cancer DNA or RNA
therapies. For more information on Celsion, visit our
website: http://www.celsion.com. (CLSN-LTSL/ThermoDox® CLSN-Optima
Study/HCC)
Celsion wishes to inform readers that
forward-looking statements in this release are made pursuant to the
"safe harbor" provisions of the Private Securities Litigation
Reform Act of 1995. Readers are cautioned that such
forward-looking statements involve risks and uncertainties
including, without limitation, unforeseen changes in the course of
research and development activities and in clinical trials; the
uncertainties of and difficulties in analyzing interim clinical
data, particularly in small subgroups that are not statistically
significant; FDA and regulatory uncertainties and risks; the
significant expense, time, and risk of failure of conducting
clinical trials; the need for Celsion to evaluate its future
development plans; possible acquisitions or licenses of other
technologies, assets or businesses; possible actions by customers,
suppliers, competitors, regulatory authorities; and other risks
detailed from time to time in the Celsion's periodic reports and
prospectuses filed with the Securities and Exchange
Commission. Celsion assumes no obligation to update or
supplement forward-looking statements that become untrue because of
subsequent events, new information or otherwise.
Celsion Investor Contact
Jeffrey W. Church
Sr. Vice President and CFO
609-482-2455
jchurch@celsion.com
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