The Company ended the year with $73.3 million in cash and cash equivalents, which is currently expected to support operations into the second half of 2013.
Progress and Milestones within Lead Programs
Palifosfamide (Zymafos ® or ZIO-201):
ZIOPHARM's palifosfamide program has made significant progress, including in its two pivotal Phase 3 studies and the initiation of a Phase 2 study.The Company announced in February 2013 that the Phase 3 trial of palifosfamide in first-line metastatic soft tissue sarcoma (PICASSO 3) reached its target number of progression-free survival (PFS) events. PICASSO 3 is an international, randomized, double-blind, placebo-controlled trial with a primary endpoint of PFS and a secondary endpoint of overall survival (OS). The trial is powered for both PFS and OS, with PFS determined by independent, blinded radiologic review. The Company expects to announce results from this trial, including topline PFS data and an interim OS futility analysis, during the last week of March 2013. The trial is expected to remain blinded for OS. The study is also being submitted as a late-breaking abstract for the annual meeting of the American Society of Clinical Oncology (ASCO). In June 2012, ZIOPHARM announced the initiation of an international, multi-center, open-label, adaptive, randomized, pivotal Phase 3 trial (MATISSE) of palifosfamide in combination with carboplatin and etoposide (PaCE) chemotherapy compared to carboplatin and etoposide alone in up to 548 chemotherapy naïve patients with metastatic small cell lung cancer. After 20 patients received at least two chemotherapy cycles, the study's Independent Data Monitoring Committee conducted an analysis of safety data and recommended that the trial proceed as planned. Enrollment is ongoing and remains ahead of projections. ZIOPHARM also recently announced the initiation of a multicenter, single arm Phase 2 investigator-sponsored study of palifosfamide in patients with recurrent metastatic germ cell (testicular and ovarian) tumors who have relapsed on initial platinum-based therapy and high dose chemotherapy, or patients who are not eligible for high dose chemotherapy. The study is being led by Lawrence Einhorn, M.D., Distinguished Professor of the Department of Medicine, Division of Hematology/Oncology at the Indiana University School of Medicine, who is also the lead investigator for the MATISSE trial.
ZIOPHARM continues to make significant strides with its synthetic biology platform. The Company recently initiated a randomized, open label study of Ad-RTS IL-12, ZIOPHARM's lead, DNA-based therapeutic for the expression of interleukin-12 (IL-12), a protein important for an immune response to cancer, in combination with palifosfamide in patients with non-resectable recurrent or metastatic breast cancer. This study is based on recent research positively correlating survival with an immune response in breast cancers targeted with non-immune treatments, together with preclinical data demonstrating powerful synergy.