The US Food and Drug Administration (FDA) has accepted Ziopharm Oncology, Inc., a biopharmaceutical company with small molecule and synthetic biology approaches to new cancer therapies, investigational new drug (IND) application for the oral dosing of palifosfamide (Zymafos or ZIO-201), a form of administration offering several potential patient-oriented advantages and an outpatient treatment for expanded global commercial reach.
Palifosfamide is a novel DNA cross-linker in class with bendamustine, ifosfamide, and cyclophosphamide. In preclinical study it has been shown to bypass resistance mediated by aldehyde dehydrogenase (ALDH), in addition to conferring a favourable toxicity profile compared to other in-class agents. An intravenous (IV) form is currently being evaluated in a randomized, double-blinded, placebo-controlled phase 3 trial for the treatment of metastatic soft tissue sarcoma in the front-line setting (PICASSO 3) and completing a phase 1 study in solid tumours, including small cell lung cancer (SCLC). Palifosfamide is entering into an adaptive phase 3 trial in extensive SCLC expected to initiate in the second half of 2012. Clinical study with the oral form, tested preclinically in multiple tumour types, is expected to follow.
"An oral formulation of palifosfamide provides us with a broader range of development options," stated Jonathan Lewis, M.D., Ph.D., chief executive officer of Ziopharm. "Oral administration could be ideal for treating children with cancer and for allowing outpatient treatment, particularly in emerging markets where IV hospital administration is either not practical, cost-effective, or even possible."