Ziopharm Oncology, Inc. announced that The Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA) adopted a positive opinion for darinaparsin (Zinapar or ZIO-101) designation as an orphan medicinal product for the treatment of Peripheral T-Cell Lymphoma (PTCL). A positive opinion by the COMP immediately precedes official designation of darinaparsin as an orphan drug by the European Commission (EC). Intravenous darinaparsin has demonstrated evidence of activity in lymphoma, in particular PTCL. Ziopharm expects to initiate a registration-directed study of darinaparsin in patients with PTCL by the end of 2011.

“Orphan designation recognizes the acute need for new therapies for unmet medical needs, in this case addressing PTCL, an aggressive form of lymphoma for which there remains such a need,” said Jonathan Lewis, MD, PhD, chief executive officer and chief medical officer of Ziopharm.

Orphan Drug Designation by the EC provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU). In addition to a 10-year period of marketing exclusivity in the EU after product approval, Orphan Drug Designation provides companies with scientific advice and regulatory assistance from the EMA during the product development phase, direct access to centralized marketing authorization, as well as reductions in certain fees associated with the application and approval process.

In September, Ziopharm announced that darinaparsin was granted Orphan Drug Designation by the US Food and Drug Administration (FDA) for the treatment of PTCL.

Peripheral T-Cell Lymphoma represents a subgroup of aggressive lymphomas that develop from T-cells in different stages of maturity. According to the Lymphoma Research Foundation, PTCL accounts for approximately 10-15% of the estimated 66,000 new cases of non-Hodgkin's lymphoma diagnosed each year in the United States (53,000 in the EU, according to the European Cancer Observatory). PTCL generally affects people of the age of 60 and is diagnosed in more men than women.

Ziopharm Oncology is a biopharmaceutical company engaged in the development and commercialization of a diverse portfolio of cancer drugs. The Company is currently focused on three clinical programmes.

Palifosfamide (ZymafosTM or ZIO-201) is a novel DNA cross-linker in class with bendamustine, ifosfamide, and cyclophosphamide. Ziopharm is currently enrolling patients in a randomized, double-blinded, placebo-controlled phase III trial with palifosfamide administered intravenously for the treatment of metastatic soft tissue sarcoma in the front-line setting. The Company is also currently conducting a phase I intravenous study of palifosfamide in combination with standard of care addressing small cell lung cancer and expects to initiate an additional study with drug in the oral form treating solid tumours.

Darinaparsin (ZinaparTM or ZIO-101) is a novel mitochondrial-targeted agent (organic arsenic) being developed intravenously for the treatment of peripheral T-cell lymphoma with a pivotal study expected to begin in late 2011. An oral form is in a Phase I trial in solid tumours.

Indibulin (ZybulinTM or ZIO-301) is a novel, oral tubulin binding agent that is expected to have several potential benefits including oral dosing, application in multi-drug resistant tumours, no neuropathy and minimal overall toxicity. It is currently being studied in Phase I/II in metastatic breast cancer.

Ziopharm is also pursuing the development of novel DNA-based therapeutics in the field of cancer pursuant to a partnering arrangement with Intrexon Corporation. The partnership includes two existing clinical-stage product candidates, the first of which is in a phase I b study and the second of which is the basis of an Investigational New Drug application that Ziopharm expects to submit during the first half of 2011.