Investment Highlights for Celsion Corporation(CLSN-NASDAQ)
Celsion Corporation is a fully integrated biopharmaceutical company focused on developing cancer therapies for difficult-to-treat forms of cancers.
The Company aims to maximize the efficacy of proven therapeutic agents and minimize side effects common to cancer treatments through the application of its two distinct nanoparticle-based technology platforms—TheraPlas™, a nonviral vector delivery system for therapeutic plasmids and Lysolipid Thermally Sensitive Liposomes (LTSL), a heat-sensitive formulation that allows for the targeted delivery of known chemotherapies.
Celsion has develop a robust pipeline that includes two clinical programs: GEN-1, an immunotherapy for the localized treatment of ovarian cancer, and ThermoDox®, a heat-mediated delivery platform of chemotherapeutic agent for the treatment of hepatocellular Cancer (HCC)/primary liver cancer. The Company is also conducting preclinical studies to expand the application of its technology platforms in other cancer indications.
GEN-1, the first product designed via the TheraPlas™ platform technology, is a DNA-based immunotherapy in a Phase I/II study for the treatment of ovarian cancer (an indication for which it received Orphan Drug Designation in the U.S. and EU). GEN-1 is a non-viral delivery platform that facilitates the introduction of a plasmid payload into tumor cells, resulting in a durable localized secretion of interleukin-12 (IL-12)—an immune protein that plays a key role in generating an immune response against cancer.
GEN-1 consists of a human IL-12 plasmid formulated with a non-viral synthetic DNA delivery system. Following administration, GEN-1 nanoparticle profile enables cell transfection followed by durable local secretion of the IL-12 protein in the vicinity of the tumor, which may lead to prolonged infiltration of immune cells in the tumor, enhancing the local immune response against cancer.
In clinical trials, GEN-1 has resulted in a marked advantage in Progression Free Survival (PFS)—21 months, which was 75% higher than the estimate of 12 months for the control group. Results of its Phase I study OVATION 1 showed that administration of GEN-1 resulted in a dose dependent response, with higher doses of GEN-1 resulting in better objective tumor response (100% versus 60% for lower doses) and an increase in R-0 events (the surgical removal of all visible tumor), from 40% in lower doses to 88% at higher doses.
GEN-1 is currently is being evaluated in a Phase I/II study (OVATION 2), in combination with standard neoadjuvant chemotherapy for newly diagnosed ovarian cancer patients. The Phase II portion of the study is in line to complete enrollment in Q2 2021, with the Company expecting progression free survival (PFS) data reported by Q4 2022.
ThermoDox® uses the Company’s LTSL technology to produce heat sensitive liposomes that encapsulate doxorubicin, a widely used chemotherapeutic agent, to facilitate its targeted delivery at the tumor site. When heated, through the application of heat-based treatments (such as radiofrequency thermal ablation [RFA]), the heat-sensitive liposome changes structure, creating openings that release doxorubicin directly into the tumor.
The Company has demonstrated the ability of ThermoDox® to deliver a greater volume of doxorubicin to the tumor area—25x more versus doxorubicin IV infusion—leading to a better clinical outcome. In HCC patients with single lesions that underwent RFA for at least 45 minutes, the use of ThermoDox® resulted in a 2.1-year improvement in Overall Survival (OS).
ThermoDox® is currently being evaluated in a Phase III clinical trial for the treatment of HCC (the OPTIMA Study). Following completion of the second interim analysis by the independent Data Monitoring Committee (DMC), the DMC recommended to consider stopping the study.
Celsion is currently conducting independent analysis of its OPTIMA data, as the study crossed a futility boundary on its second scheduled interim analysis. Following preliminary evaluation of the unblinded data, Celsion decided to continue following patients, noting that the crossed futility boundary may be associated with a data maturity issue. Celsion expects to report findings from these independent statistical analyses by Q4 2020.
Celsion’s leadership team incorporates over 150 years of management experience within the healthcare industry, including clinical development program, R&D, pharmaceutical operations, and corporate finance.
The Company recently strengthened its balance sheet position, resulting in a minimal warrant overhang and cleaner capital structure, through four key financial activities during 2020: (1) a $10 million underwritten offering of common stock (June 2020), (2) the continued sale of New Jersey net operating losses (NOLs) (April 2020), (3) the repayment and restructure of its long-term debt with Horizon Technology Finance Corporation (September 2020), and (4) the Lincoln Park Capital Fund, LLC (LPC) financing (September 2020).
The Company ended Q3 2020 with $18.3 million in cash and cash equivalents. Coupled with future planned sales of its New Jersey NOL’s, the Company believes it has sufficient capital resources to fund its operations through the end of 2021.