Full Press Release Details
Corporation Reports Second Quarter 2020 Financial Results and Provides Business Update
Promising Phase II Study of GEN-1 in Advanced Ovarian Cancer
Continue Following Patients in Phase III OPTIMA Study for Overall Survival;
Call Begins Today at 11:00 a.m. Eastern Time
N.J. (August 14, 2020) - Celsion Corporation (NASDAQ: CLSN), an oncology drug development company, today announced
financial results for the three and six months ended June 30, 2020, and provided an update on clinical development programs with
GEN-1, its DNA-mediated IL-12 immunotherapy currently in Phase II development for the treatment of advanced stage ovarian cancer,
and ThermoDox , its proprietary heat-activated liposomal encapsulation of doxorubicin currently in Phase III development
for the treatment of hepatocellular carcinoma, or primary liver cancer.
our oncology-focused immunotherapy, continues to show encouraging results at the 100 mg/m² dose cohort in the OVATION 2 Study,
which is consistent with the results reported from our earlier Phase Ib trial (the OVATION 1 Study) in advanced-stage ovarian
cancer. In June 2020, the Data Safety Monitoring Board (DSMB) for the OVATION 2 Study recommended that the Phase II portion of
the OVATION Study proceed with the dose of 100 mg/m2," reported Michael H. Tardugno, Celsion's chairman,
president and chief executive officer. "These findings were reinforced by strong progression-free survival (PFS) when comparing
study patients to a statistically validated synthetic control arm (SCA) of matched patients from prior studies. In July 2020,
we announced the randomization of the first two patients in the Phase II OVATION 2 Study. This milestone was achieved approximately
five months ahead of our previously announced schedule. We have a very aggressive recruitment program and anticipate completing
enrollment of 105 patients in the second quarter of 2021. Importantly, as an open-label study, clinical updates will be provided
throughout the course of treatment including response rates and surgical resection scores," Mr. Tardugno added.
his comments, Mr. Tardugno noted, "In early July, Celsion received a wholly unexpected recommendation from the independent
Data Monitoring Committee (DMC) to consider stopping the global Phase III OPTIMA Study. This recommendation was made following
the DMC's second pre-planned interim safety and efficacy analysis of the OPTIMA Study on July 9, 2020. The DMC's 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 p-value of 0.524 for this analysis provides a high level of uncertainty as to the actual hazard ratio value, therefore,
the DMC left the final decision of whether to stop the OPTIMA Study to the Company.
Tardugno further stated, "This development had never been anticipated by the Company or our advisors, nor would it have
been forecasted by the first pre-planned efficacy analysis. Further, blinded data available to the Company appeared to be tracking
well against the sub-group analysis of the Company's earlier HEAT Study, upon which the OPTIMA Study is based."
early August, after conducting additional analyses of the unblinded data from the second pre-planned interim analysis, the Company
announced plans to continue following patients for overall survival (OS), noting that the unexpected and marginally crossed futility
boundary, suggested by the Kaplan-Meier analysis at the second interim analysis, may be associated with a data maturity issue.
Additionally, Celsion reported that it is sending all clinical trial data, including Chemistry, Manufacturing and Controls (CMC)
data, to the National Institutes of Health (NIH) for independent analysis, including computed tomography (CT) scans for NIH's
evaluation of PFS. Depending on the trends noted during the OS follow-up period, Celsion may choose to discontinue the Study at
any time. The Company also notes that the vast majority of expenses related to the OPTIMA Study already have been incurred.
of Phase II OVATION 2 Study in Advanced Ovarian Cancer. In July 2020, the Company announced the randomization of the first
two patients in the Phase II portion of the OVATION 2 Study with GEN-1 in advanced ovarian cancer. The Company anticipates completing
enrollment of up to 118 patients in the second quarter of 2021. Because this is an open-label study, clinical updates will be
provided throughout the course of treatment including response rates and surgical resection scores.
OVATION 2 Study combines GEN-1 with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV
ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy.
Following NACT, patients undergo interval debulking surgery, followed by three adjuvant cycles of chemotherapy and up to nine
additional weekly GEN-1 treatments, the goal of which is to delay progression and improve OS. The OVATION 2 Study is an open-label,
1-to-1 randomized trial, 80% powered to show the equivalent of a 33% improvement in PFS (HR=0.75), the primary endpoint, when
comparing the treatment arm (standard of care + GEN-1) with the control arm (standard of care alone).
Recommends GEN-1 to Proceed to Phase II of the OVATION 2 Study in Advanced Ovarian Cancer. In May 2020, the Company announced
the final recommendations of the DSMB following completion of the Phase I dose-finding and tolerance portion of the OVATION 2
Study with GEN-1 in advanced (Stage III/IV) ovarian cancer. Based on favorable safety data from 15 randomized patients, the DSMB
recommended that the Phase II portion of the OVATION Study proceed with the dose of 100 mg/m2. The DSMB also determined
that safety is satisfactory with an acceptable risk/benefit, and that patients tolerate up to 17 doses of GEN-1 during a course
of treatment that lasts up to six months. No dose limiting toxicities were reported.
March 2020, the Company announced the following clinical development achievements for GEN-1:
| Of the 15 patients treated in the Phase I portion of the OVATION 2 Study, nine were treated with GEN-1 at a dose of 100 mg/m² plus NACT and six were treated with NACT only. All 15 had successful resections of their tumors, with seven out of nine patients (78%) in the GEN-1 treatment arm having an R0 resection, which indicates a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed. Only three out of six patients (50%) in the NACT only treatment arm had an R0 resection | ||
| When combining these results with the surgical resection rates observed in the Company's prior Phase Ib dose-escalation trial (the OVATION 1 Study), a population of patients with inclusion criteria identical to the OVATION 2 Study, the data reflect the strong dose-dependent efficacy of adding GEN-1 to the current standard of care NACT: |
| % of Patients with | ||||||
| R0 Resections | ||||||
| 0, 36, 47 mg/m² of GEN-1 plus NACT | n=12 | 42 | % | |||
| 61, 79, 100 mg/m² of GEN-1 plus NACT | n=17 | 82 | % |
| GEN-1 Population | PFS Hazard Ratio (Confidence Interval) | |
| Intent-to-treat, n=15 | 0.53 (95% CI 0.16, 1.73); log-rank p = 0.29 | |
| Per-protocol, n=14 | 0.33 (95% CI 0.08, 1.37); log-rank p = 0.11 |
in Phase III OPTIMA Study Will Continue to be Followed for Overall Survival. In August 2020, the Company provided an update
on its ongoing review of unblinded data from the second pre-planned interim analysis of the global Phase III OPTIMA Study. The
Company announced it will continue following patients for OS, noting that the unexpected and marginally crossed futility boundary
suggested by the Kaplan-Meier analysis at the second interim analysis on July 9, 2020 may be associated with a data maturity issue.
The Company further notes that 26 consecutive patient deaths represented exclusively in the second analysis behave far differently
from the balance of the patients who have died as of that date. Removing the 26 consecutive patient deaths, which occurred between
September 2019 and March 2020, from the pre-planned interim analysis suggests that the OPTIMA Study OS pattern is similar to the
prospective HEAT Study subgroup upon which the OPTIMA Study is based, at the approximate comparable point in time. In addition,
subsequent to the second interim analysis there were eight patient deaths in a 3:1 ratio of control arm to treatment arm patients,
which further supports a concern for data maturity.
was further noted that OPTIMA Study sites in China and Vietnam, which enrolled over 37% of the subjects, joined the Study approximately
12 and 18 months, respectively, after the trial was initiated. The Kaplan-Meier curves for both geographies demonstrate a potential
data maturity issue when compared with the behavior of the HEAT Study subgroup and other OPTIMA Study testing site regions. The
China sites, in particular, show a negative Kaplan-Meier curve, yet with a 56% improvement in the treatment arm in the median
time to death. The Vietnam sites show a marginal Kaplan-Meier benefit, yet with a 45% improvement in the treatment arm in the
median time to death. The Company believes that this dichotomy must be reconciled, most probably with longer follow up, before
it can determine the Study's direction.
from the Independent DMC to Consider Stopping the Phase III OPTIMA Study of ThermoDox in Primary Liver Cancer.
In July 2020, the Company announced that it received a recommendation from the independent DMC to consider stopping the
global Phase III OPTIMA Study. The recommendation 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 p-value of 0.524 for this analysis provides uncertainty; subsequently,
the DMC 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.
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 on July 9, 2020 following data lock in April 2020 after the prescribed minimum number
of 158 events was reached.
Balance Sheet Through a $10 Million Underwritten Offering of Common Stock. In June 2020,
the Company entered into an underwriting agreement relating to the sale of 2,666,667 shares of its common stock at an offering
price of $3.75 per share. The net proceeds from the offering were $9.3 million, after deducting underwriting discounts and commissions,
but before expenses payable by the Company. The shares of common stock were sold to both existing and new institutional investors
of the Company. Oppenheimer & Co. Inc. acted as the sole underwriter for the offering.
$1.8 Million in Non-Dilutive Funding from the Sale of New Jersey State Net Operating Losses. In April 2020, the Company
announced it received $1.8 million of net cash proceeds from the sale of approximately $1.9 million of its unused New Jersey net
operating losses (NOLs). The NOL sales cover the tax years 2017 and 2018 and are administered through the New Jersey Economic
Development Authority's (NJEDA) Technology Business Tax Certificate Transfer (NOL) Program. An additional sale of $2.0 million
of unused New Jersey NOLs anticipated in the second half of 2020 will further increase Celsion's cash reserves on a non-dilutive
Quarter Financial Results
the quarter ended June 30, 2020, Celsion reported a net loss of $5.3 million ($0.18 per share), compared with $5.9 million ($0.29
per share) in the same period of 2019. Operating expenses were $4.9 million in the second quarter of 2020, which represented a
$0.8 million (14%) decrease from $5.7 million in the same period of 2019.
Company ended the second quarter of 2020 with $25.5 million in cash, investment securities and accrued interest receivable. With
$25.5 million in cash as of June 30, 2020 coupled with future sales of the Company's New Jersey NOL's, the Company
believes it has sufficient capital resources to fund its operations into the fourth quarter of 2021.