Full Press Release Details
Corporation Receives Recommendation from Independent Data Monitoring Committee to Consider Stopping the Phase III OPTIMA Study
Based on DMC's Finding Following the Second Pre-Planned Interim Analysis that the OPTIMA Study is Unlikely to Meet its Primary
Efficacy Endpoint of Overall Survival
to Conduct a Thorough Analysis of the Unblinded Study Data Before Making a Final Determination
Call to be Held Wednesday, July 15
N.J. (July 13, 2020) - Celsion Corporation (NASDAQ: CLSN), an oncology focused drug-development company, today
announced that it has received a recommendation from the independent Data Monitoring Committee (DMC) to consider stopping the
global Phase III OPTIMA Study of ThermoDox in combination with radiofrequency ablation (RFA) for the treatment of hepatocellular
carcinoma (HCC), or primary liver cancer.
was made following the second pre-planned interim safety and efficacy analysis by the DMC on July 9, 2020. The DMC analysis found
that the pre-specified boundary for stopping the trial for futility of 0.900 was crossed with an actual value of 0.903. However,
the 2-sided p-value of 0.524 for this analysis provides uncertainty, subsequently, the DMC has left the final decision
of whether or not to stop the OPTIMA Study to Celsion. There were no safety concerns noted during the interim analysis.
are surprised and disappointed that the OPTIMA Study results were not found to be more robust at this analysis. Nonetheless, we
intend to follow the advice of the DMC and will consider our options either to stop the study or continue to follow patients after
a thorough review of the data, and an evaluation of our probability of success. Timing for this decision is made less urgent by
the fact that the OPTIMA Study has been fully enrolled since August 2018 and that the vast majority of the trial expenses have
already been incurred," stated Michael
H. Tardugno, Celsion's chairman, president and chief executive officer.
of course, is inconclusive and difficult news for the medical community, HCC patients and our shareholders, and confirms the complexity
and challenge of treating primary liver cancer," he added. "The present development
had never been anticipated by the Company or our advisors based on both the first pre-planned efficacy analysis and on tracking
against the sub-group analysis of the Company's earlier HEAT Study upon which the OPTIMA Study is based. We will conduct
additional analyses of the unblinded data from the trial to better understand the results and to develop our plan going forward.
As always, we wish to acknowledge and thank all the patients and investigators for their participation in the trial."
OPTIMA Study is a global, randomized, double-blind, placebo-controlled clinical trial assessing the efficacy of ThermoDox
in combination with RFA, which was standardized to a minimum of 45 minutes for treating patients with a lesion 3-7 cm in size,
versus standardized RFA alone. The OPTIMA Study enrolled 554 patients at 65 clinical sites in North America, Europe, China and
Asia Pacific. In addition to the primary overall survival endpoint, progression-free survival, time to disease progression, and
safety are key secondary endpoints.
statistical plan for the OPTIMA Study included two interim efficacy analyses by the DMC. The first interim analysis was announced
in November 2019 following data lock in August 2019 after the prescribed minimum number of 128 patient events (deaths) was reached,
and the second interim analysis was conducted in July 2020 following data lock in April 2020 after the prescribed minimum number
of 158 events was reached.
will be holding a conference call and webcast on Wednesday, July 15 to discuss its current observations about the results of the
OPTIMA Study, the DMC's recommendations and the Company's next steps. Further information regarding the time of the
call and dial-in instructions will be provided separately.
most advanced program is a heat-mediated drug delivery technology that employs a novel heat-sensitive liposome engineered to address
a range of difficult-to-treat cancers. The first application of this platform is ThermoDox , a lyso-thermosensitive
liposomal doxorubicin (LTLD) whose novel mechanism of action delivers high concentrations of doxorubicin to a region targeted
with the application of localized heat at 40 C, just above body temperature. ThermoDox is positioned for use
with multiple heating technologies and has the potential to treat of a broad range of cancers including metastatic liver, recurrent
chest wall breast cancer and non-muscle invading bladder cancers.
LTLD technology leverages two mechanisms of tumor biology to deliver higher concentrations of drug directly to the tumor site.
In the first mechanism, rapidly growing tumors have leaky vasculature, which is permeable to liposomes and enables their accumulation
within tumors. Leaky vasculature influences a number of factors within the tumor, including the access of therapeutic agents to
tumor cells. Administered intravenously, ThermoDox is engineered with a half-life to allow significant accumulation
of liposomes at the tumor site as these liposomes recirculate in the blood stream.
the second mechanism, when an external heating device heats tumor tissue to a temperature of 40 C or greater, the heat-sensitive
liposome rapidly changes structure and the liposomal membrane selectively dissolves, creating openings that can release a chemotherapeutic
agent directly into the tumor and the surrounding vasculature. Drug concentration increases as a function of the accumulation
of liposomes at the tumor site, but only where the heat is present. This method damages only the tumor and the area subject to
tumor invasion, supporting more precise drug targeting.
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 development for other cancer indications. The Company's product pipeline also includes GEN-1, a DNA-based immunotherapy
for the localized treatment of ovarian cancer. Celsion has two platform technologies for the development of novel nucleic acid-based
immunotherapies and other anti-cancer DNA or RNA therapies.
statements in this news 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 or regulatory authorities; and other risks detailed from time
to time in the Celsion's periodic filings 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.
Vice President and CFO