Celsion is committed to the development of efficient, effective, and targeted therapies for the treatment of cancer. Founded in 1985, the company has developed elegant therapeutic approaches that deliver high concentrations of widely used anti-cancer agents directly to the tumor site, maximizing efficacy while minimizing the side-effects of systemic treatments, with the goal of improving patient outcomes in underserved cancer indications.
Celsion Corporation, a publicly held biopharmaceutical company focused on the development of nucleic acid-based therapeutics for the treatment of cancer and other difficult-to-treat diseases. The acquisition established Celsion as a fully integrated oncology company, with programs on the leading edge of cancer research and with end-to-end R&D talent and competencies ranging from drug development to commercialization.
Today, Celsion has assets in directed chemotherapies, immunotherapies, and DNA- and RNA-based therapies. With clinical programs in Phases I, II, and III, and with an extensive pipeline of pre-clinical product candidates, Celsion is well positioned to deliver innovative new therapies to address areas of pressing unmet medical need in oncology.
Our lead product candidate, ThermoDox®, is a proprietary heat-activated liposomal encapsulation of doxorubicin, a frequently used and proven cancer drug. The ThermoDox program, driven by the lysolipid thermally sensitive liposome (LTSL) technology platform, offers a “pipeline within a product,” with the potential for ground-breaking advances in the treatment of multiple tumor types. In addition, by pairing our novel LTSL technology with a proven, approved drug, we have the opportunity to significantly improve upon existing treatment options without the level of risk inherent in the development of new chemical entities.
ThermoDox is a liposomal encapsulation of doxorubicin, an approved anthracycline chemotherapeutic that is frequently used in the treatment of a wide range of cancers. The LTSL technology allows for the delivery of high concentrations of doxorubicin in a region specifically targeted with the application of localized heat, such as in radiofrequency ablation (RFA). Localized heat at mild hyperthermia temperatures (greater than 39.5 degrees Celsius) releases the encapsulated doxorubicin from the liposome, enabling high concentrations of doxorubicin to be deposited preferentially in and around the targeted tumor.
Celsion recently received Food and Drug Administration (FDA) clearance to initiate the Phase III OPTIMA trial, which is designed to evaluate ThermoDox in combination with standardized RFA (sRFA 45) in primary liver cancer (hepatocellular carcinoma, or HCC). The two-arm, double-blinded, placebo-controlled, randomized study will enroll approximately 550 patients at up to 100 sites in North America, Europe, China, and the Asia-Pacific region. The primary endpoint is overall survival (OS), and the study is powered to demonstrate a 33% improvement in OS.
The Phase II DIGNITY study is evaluating ThermoDox plus local hyperthermia in patients with recurrent chest wall (RCW) breast cancer, a difficult-to-treat population of patients who have failed to respond to at least two lines of chemotherapyfollowing mastectomy, and have failed to respond to radiation before enrolling in the trial. Interim results show impressive and compelling clinical activity, with a local response rate of 50%, including three complete responses and two partial responses. Additionally, 60% of DIGNITY study participants have experienced disease stabilization. Those findings are consistent with the local response rate of 48.3% observed in 24 patients enrolled in two previous Phase I studies.
To generate growth over the long-term, we are supporting a broad range of studies using MRI-guided high-intensity focused ultrasound (HIFU) to provide ThermoDox activation in multiple indications. The most advanced program is recruiting patients with colorectal liver metastases in a Phase II study, in partnership with Oxford University in Britain. That program is followed closely by a Phase II study of ThermoDox + HIFU in patients with breast cancer in The Netherlands. Both of these programs are co-funded in Europe by government grants. Beyond those indications, ThermoDox holds promise in a wide variety of tumor types for which doxorubicin is widely used, including pancreatic cancer and glioblastoma, a type of brain cancer.
Our other current clinical-stage product candidate is GEN-1, a Phase II-ready, DNA-based immunotherapeutic for the localized treatment of ovarian and brain cancers. It is the first product produced under the versatile TheraPlas™ technology platform, which enables delivery of DNA and messenger RNA (mRNA) therapeutics via synthetic, non-viral carriers. TheraPlas is uniquely capable of providing cell transfection capability for double-stranded DNA plasmids and large therapeutic RNA segments such as mRNA.
GEN-1 is an interleukin-12 (IL-12) plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of a potent anti-cancer immunity response. It acts through the induction of T-lymphocyte and natural killer (NK) cell proliferation, and has also shown an anti-angiogenic effect and has demonstrated potential in a variety of cancers. As a recombinant protein, however, IL-12 has poor pharmacokinetics and historically has been associated with serious toxicities. Celsion believes these problems may be effectively addressed by GEN-1’s unique mechanism of action and method of administration.
Positive safety data and encouraging Phase I results with GEN-1 monotherapy in patients with peritoneally metastasized ovarian cancer strongly support a planned Phase II combination trial. Additionally, a Phase Ib trial of GEN-1 in combination with PEGylated doxorubicin in patients with platinum-resistant ovarian cancer is currently ongoing. GEN-1 has also demonstrated preclinical activity in glioblastoma multiforme (GBM), and Celsion plans to initiate a Phase I study in this indication.
GEN-2, an RNA therapeutic, is the first drug candidate based on our versatile TheraSilence™ technology platform, which focuses on synthetically generated small inhibitory RNAs (siRNAs), microRNAs, microRNA mimics, and related molecules that can regulate protein expression by exploiting endogenous cell mechanisms. EGEN has developed several classes of proprietary carriers that can efficiently deliver siRNAs to the cytoplasm of many types of cells both in vitro and in vivo. Currently in preclinical development, GEN-2 is a combination of two unique molecular targets for anticancer therapy, and has potential as a treatment for lung cancer. The concept uses a two-pronged approach: inhibiting tumor growth by anti-angiogenesis and promoting direct killing of the tumor with a microRNA.
Celsion is headquartered in Lawrenceville, New Jersey. Our discovery and preclinical operations, as well as our analytical services and laboratory functions, are based in Huntsville, Alabama.
Our strategy and focus are driven by our commitment to deliver innovative and effective treatments for cancer patients. With our expertise in oncology, experienced leadership team, network of world-class partners and infrastructure, we are building a fully integrated oncology company with a deep pipeline and multiple product platforms to address emerging needs in cancer treatment and improve patients’ lives.