OPTIMA Study Enrollment is Approaching 70%
Celsion Corporation (NASDAQ:CLSN), an oncology drug development
company, today provided a detailed update 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) which include recent investigators meetings in Bangkok,
Thailand and Shanghai, China. With the growing incidence of primary
liver cancer in China and Asia-Pacific, representing approximately
75% of the estimated 850,000 cases diagnosed annually, this region
represents a strategically important element of the Company’s
global registration and commercialization strategy for ThermoDox®.
The Company announced that enrollment in the
OPTIMA Study is now approaching 70% of the 550 patients necessary
to ensure that its primary end point, overall survival, can be
evaluated with statistical significance. The statistical plan for
the OPTIMA Study calls for two interim efficacy analyses by the
independent Data Monitoring Committee (DMC). The Company currently
projects full patient enrollment by mid-2018 and the first
pre-planned efficacy analysis after 118 overall survival events by
the first quarter of 2019.
“With independent confirmation by the NIH of the
relationship between RFA heating time and the significant impact
that it has on overall survival when combined with ThermoDox®,
OPTIMA Study investigators fully recognize the value of the
findings from the HEAT Study, reinforcing their interest and
support for our highly de-risked, ongoing global Phase III OPTIMA
Study," said Michael H. Tardugno, Celsion's chairman, president and
chief executive officer. "The previously announced unanimous
recommendation for study continuation by the independent Data
Monitoring Committee was based on their review of all available
clinical data from 275 patients, and is further evidence of
ThermoDox's recognized potential to provide a new and important
first line therapeutic option for patients with primary liver
cancer."
The design of the OPTIMA Study is supported by a
retrospective analysis of a large subgroup of 285 patients in the
Company's previous 701 patient HEAT Study in primary liver cancer.
This subgroup of patients who received ThermoDox® plus standardized
RFA demonstrated a statistically significant improvement in
survival of over two years compared to standardized RFA alone. The
median overall survival in the ThermoDox® plus standardized RFA arm
was approximately 80 months (6 ½ years), which is considered a
curative treatment for primary liver cancer.
On November 29, 2016, the Company announced
results from an independent retrospective analysis conducted by the
National Institutes of Health (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 consistent with
the HEAT Study in patients treated with ThermoDox® plus optimized
RFA over the optimized RFA only group.
In August 2017, the Company’s Data Monitoring
Committee, comprised of an independent group of medical and
scientific experts who are responsible for reviewing and evaluating
patient safety and efficacy data, completed a planned interim
analysis of the first 50% of patients randomized in the trial as of
April 2017 and unanimously recommended that the OPTIMA Study
continue as planned based on the risk to benefit analysis by the
Committee. The OPTIMA Study to date has accumulated data within
acceptable safety parameters.
"There is clear evidence that the duration of
the RFA regimen is critical when treating patients with ThermoDox®,
and the totality of the data presented to date demonstrate that
ThermoDox plus optimized RFA has a strong potential to serve as a
curative therapy for patients with liver cancer," said Professor
Won Young Tak, M.D., Ph.D., Division of Gastroenterology and
Hepatology, Department of Internal Medicine, School of Medicine,
Kyungpook National University, Daegu, Republic of Korea and lead
investigator in South Korea for the Company's HEAT and OPTIMA
studies. "The OPTIMA Study is designed to validate this approach in
an indication where there exists a strong unmet need for effective
treatment options."
Regulatory Strategy for
ThermoDox®. ThermoDox® has received U.S. FDA Fast
Track Designation and has been granted orphan drug designation for
primary liver cancer in both the U.S. and Europe. Further, the U.S.
FDA has provided ThermoDox® with a 505(b)(2) registration
pathway. Subject to a successful trial, the OPTIMA Study has
been designed to support registration in all key primary liver
cancer markets. Celsion fully expects to submit registrational
applications in the USA, Europe and China. The Company believes
that applications will be accepted in South Korea, Taiwan and
Vietnam, three other large and important markets for ThermoDox®
subject to approval in Europe, China or the USA.
“The Company’s optimism for Chinese registration
is based on our multiple interactions with the China Food and Drug
Administration,” said Nicholas Borys, MD, Celsion’s senior vice
president and chief medical officer. “During our December 2016
meeting with the Deputy Director of the Center for Drug Evaluation,
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 China only cohort was found to
be consistent with the overall HEAT Study findings. We were
informed 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
the European Union. 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,” noted Dr. Borys.
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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