Ad-RTS-hIL-12 is a novel gene therapy candidate for the controlled expression of IL-12, a critical protein for stimulating an anti-cancer T cell immune response.
The FDA’s Office of Orphan Products grants orphan drug status to support development of medicines for underserved patient populations or rare disorders affecting fewer than 200,000 people in the U.S. Orphan Drug Designation provides eligibility for a seven-year period of market exclusivity in the United States after product approval, an accelerated review process, accelerated approval where appropriate, grant funding, tax benefits and an exemption from user fees.
"Malignant glioma is an aggressive cancer with few treatment options," said Laurence J. N. Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM.
"This designation supports our effort to advance Ad-RTS-IL-12, a novel immuno-oncology therapy which has demonstrated promising preclinical efficacy in brain cancer, as a potential treatment for this indication. Enrollment in our Phase 1 study is progressing well, and we look forward to early results from this clinical trial later in the year."
Ad-RTS-hIL-12 + veledimex is currently being studied in a Phase 1 trial designed to examine a gene therapy treatment strategy for high grade gliomas, with the goal of generating an anti-tumor T cell immune response.
The primary objective of the study is to determine the safety and tolerability of a single intra-tumoral Ad-RTS-hIL-12 injection plus escalating oral veledimex doses.
Secondary objectives are to determine the veledimex maximum tolerated dose, the immune responses elicited by Ad-RTS-hIL-12 and veledimex, and investigator assessment of response, including the tumor objective response rate and progression-free survival, and determine overall survival, among other measures.
The study is expected to enroll up to 72 subjects at up to 12 leading treatment centers.