Celsion Corporation (Nasdaq: CLSN), a leading oncology drug development company, announced that after reviewing data from 401 patients enrolled in its pivotal Phase III ThermoDox ® clinical study (the HEAT study) for primary liver cancer, the Data Monitoring Committee (DMC) has unanimously recommended that the trial continue to enroll patients with the goal of reaching the 600 patients required to complete the study. The Company previously announced that the U.S. Food and Drug Administration (FDA) designated the HEAT study of its investigational drug, ThermoDox®, in combination with radiofrequency ablation (RFA), as a Fast Track Development Program. ThermoDox, a proprietary heat-activated liposomal encapsulation of doxorubicin, is currently being evaluated under a Special Protocol Assessment (SPA) agreement with the FDA in this global Phase III trial in patients with non-resectable hepatocellular carcinoma (HCC), commonly referred to as primary liver cancer. With nearly 75% of patients enrolled in the trial, Celsion is targeting to complete patient enrollment over the next 4 months. As a part of its commitment to Japan's PMDA, the DMC will continue to independently assess safety in patients randomized at Japanese sites. The goal is to provide data from this cohort sufficient to support immediate registration in Japan following a successful study.
The DMC for the HEAT study is comprised of an independent group of medical and scientific experts with the responsibility for reviewing and evaluating patient safety and efficacy data from the Company's Phase III ThermoDox HEAT study. The DMC reviews study data at regular intervals with their primary responsibilities to ensure the safety of all patients enrolled in the trial, the quality of the data collected, and the continued scientific validity of the trial design. The trial design and statistical plan for the HEAT study also incorporates a pre-planned interim efficacy analysis by the DMC (after patient enrollment is complete and 190 progression-free survival events are realized in the study population) with the intent of evaluating safety and efficacy results to determine if there is overwhelming evidence of clinical benefit or a low probability of treatment success (a futility analysis) to continue, modify or terminate the trial.
"We are very pleased that the DMC has unanimously recommended continuation of the HEAT study based on its review of all available clinical data, both safety and efficacy, in over 400 patients," stated Mr. Michael H. Tardugno, Celsion's President and Chief Executive Officer. "The DMC's affirmative review is further evidence of ThermoDox's potential to provide a first line of chemotherapy for the treatment of primary liver cancer."
The National Institutes of Health and regulatory agencies around the world as well as the liver cancer medical community have all recognized the potential of ThermoDox for the treatment of this unmet medical need as evidenced by the following:
- HEAT study is being conducted under a Special Protocol Assessment with the FDA;
- HEAT study has been granted a Fast Track Designation by the FDA; and
- The National Cancer Institute's recent designation of the HEAT study as a Priority Clinical Trial at its recent Clinical Trial Planning Meeting for HCC.
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