On February 20, 2015 Celsion Corporation reported clinical and preclinical data demonstrating the safety, biological activity and clinical benefits of GEN-1, its DNA-based immunotherapy, as a single agent in advanced platinum-resistant and recurrent ovarian cancer patients, at the Molecular Medicine TRI-Conference in San Francisco (Press release Celsion, FEB 20, 2015, View Source [SID:1234501821]). These data provide support for advancing GEN-1 into clinical development in the front-line setting. The Company also announced that the U.S. Food and Drug Administration (FDA) has accepted, without comment, its planned Phase 1 dose-escalation clinical trial of GEN-1 in combination with the standard of care in neo-adjuvant ovarian cancer, which is expected to commence in mid-2015 at five to six U.S. clinical centers.

“These important findings significantly strengthen our established clinical and preclinical data providing additional evidence of GEN-1’s ability to effectively recruit a cellular immune system response, widely known for its anti-cancer activity. Evidence now in platinum-resistant and recurrent ovarian cancer patients, populations which historically have had little to no response to new investigational therapies, is highly encouraging,” stated Khursheed Anwer, Ph.D., Executive Vice President and Chief Scientific Officer of Celsion. “Based on the level of activity observed in these advanced patient populations, we believe that GEN-1 has the potential to produce a robust response in the neo-adjuvant setting, where patients typically have healthier immune systems and no prior treatment with immunosuppressive drugs.”

Today’s presentation at the Molecular Medicine TRI-Conference included data from the recently completed Phase 1b dose-escalation combination study of GEN-1, as well as a review of previously reported data. The Phase 1b study enrolled 16 patients with platinum-resistant ovarian cancer and evaluated the safety, tolerability and efficacy of GEN-1 in combination with pegylated doxorubicin. Patients received pegylated liposomal doxorubicin on day 1 and GEN-1 intra-peritoneally (IP) over days 1, 8, 15 and 22. This treatment course was repeated every 28 days in the absence of disease progression or unacceptable toxicity. The findings demonstrated that there were no overlapping toxicities between GEN-1 and pegylated doxorubicin. Biological activity and clinical efficacy results from this Phase 1b study including disease control rates, translational data and survival rates among the three doses evaluated have been submitted for presentation at the American Society of Clinical Oncologist (ASCO) (Free ASCO Whitepaper) Conference in the second quarter of 2015 and will be publicly available following ASCO (Free ASCO Whitepaper)’s normal publication schedule. Patients will be followed quarterly for up to one year following completion of study treatment.

The data from the Phase 1b study was consistent with previously reported data from two single-agent studies of GEN-1 in platinum-resistant recurrent ovarian cancer. In an earlier Phase 1 study, treatment with GEN-1 demonstrated a DCR of 31%, biological activity and median overall survival (OS) of 18 months. In the Phase 2 study, treatment with GEN-1 demonstrated a DCR of 45% and a median OS of 10 months. In both studies, GEN-1 was well tolerated and no maximum tolerated dose (MTD) was achieved.

“The data presented today highlights the potential value and promise of our IL-12 immunotherapy program and our expectations for the TheraPlas platform generally,” said Michael H. Tardugno, Celsion’s Chairman, President and Chief Executive Officer. With results that pave the way for GEN-1 to provide a promising new approach for treating ovarian cancer, we are now focused on launching our upcoming combination trial, which is designed to help identify a maximum tolerated dose of GEN-1 and will inform the design of our planned Phase 2 study in the front line setting, where new therapies are desperately needed. We will collect translational data to better understand the relationship between higher doses of GEN-1 and the effect on stimulation of the patients’ immune system.”

The combination trial is designed to enroll three to six patients per dose level until a safe, tolerable and potentially therapeutically active dose is identified. The study will evaluate safety and efficacy and attempt to define an optimal dose to carry forward into a Phase 2 trial.