Celsion’s (CLSN) stock has been on a tear since mid-April, rising more than 50% in anticipation of the company’s expected September interim results for its pivotal Phase III HEAT study of ThermoDox in primary liver cancer.
At a June 10 presentation the company’s slides (pdf) provided details of the study and some updates. Celsion said that there is a Special Protocol Assessment in place for the trial. The product candidate has been granted the Fast Track designation as well as a Priority review. The rolling NDA is expected in 2011, with the interim analysts of the trial expected in September. The product candidate is also 505(b)(2) eligible, meaning that the pre-clinical studies are sufficient to support NDA submission. On June 10, approximately 95% of the patients were enrolled for the trial.
For some background, ThermoDox is a proprietary heat-activated liposomal encapsulation of doxorubicin, an approved and frequently used oncology drug for the treatment of a wide range of cancers. In the HEAT Study, ThermoDox is administered intravenously in combination with radiofrequency ablation (RFA). Localized mild hyperthermia (39.5 - 42 degrees Celsius) created by the RFA releases the entrapped doxorubicin from the liposome. This delivery technology enables high concentrations of doxorubicin to be deposited preferentially in a targeted tumor.
For primary liver cancer, ThermoDox is being evaluated in a 600-patient global Phase III study. The study is designed to evaluate the efficacy of ThermoDox in combination with RFA when compared with patients who receive RFA alone as the control. The primary endpoint for the study is progression-free survival (PFS) with a secondary confirmatory endpoint of overall survival. A pre-planned, unblinded interim efficacy analysis will be performed by the independent Data Monitoring Committee when enrollment in the HEAT Study is complete and 190 PFS events are realized in the study population.
Primary liver cancer is one of the most deadly forms of cancer and ranks as the fifth most common solid tumor cancer. The incidence of primary liver cancer is approximately 20,000 cases per year in the United States, approximately 40,000 cases per year in Europe and is rapidly growing worldwide at approximately 700,000 cases per year, due to the high prevalence of Hepatitis B and C in developing countries. The standard first-line treatment for liver cancer is surgical resection of the tumor; however, 90% of patients are ineligible for surgery. RFA has increasingly become the standard of care for non-resectable liver tumors, but the treatment becomes less effective for larger tumors. There are few non-surgical therapeutic treatment options available as radiation therapy and chemotherapy are largely ineffective in the treatment of primary liver cancer.
The stock has pulled back recently, after reaching as high as $4.37, so this may be a good opportunity to jump in for a trading opportunity before the run-up resumes as we get closer and closer to interim results in September.