ZIOPHARM Oncology Inc. (NASDAQ:ZIOP), a biopharmaceutical company focused on new cancer immunotherapies, today announced that the Company is presenting initial results from an ongoing Phase 1 dose-escalation study of Ad-RTS-hIL-12 + orally administered veledimex in recurrent or progressive glioblastoma or grade III malignant glioma. The presentation, titled “Intratumoral Regulated Expression of IL-12 as a Gene Therapy Approach to Treatment of Glioma,” will be delivered at 5:15 pm CT, Saturday, November 21, 2015 at the 20th Annual Society for Neuro-Oncology (SNO) Annual Scientific Meeting in San Antonio, Texas. Ad-RTS-hIL-12 + the oral activator veledimex is a novel viral gene therapy candidate for the controlled expression of IL-12, a critical protein for stimulating an anti-cancer T-cell immune response.
“Immunotherapy is an attractive approach for the treatment of glioma, an aggressive cancer with few treatment options,” said Nino Chiocca, MD, PhD, Harvey W. Cushing Professor of Neurosurgery, Department of Surgery, Harvard Medical School, Surgical Director, Center for Neuro-oncology, Dana-Farber Cancer Institute, Chairman, Neurosurgery, Brigham And Women’s Hospital and Co-Director, Institute for the Neurosciences, Brigham And Women’s Hospital. “IL-12 is among the most potent anti-cancer immune cytokines, yet carries equally significant potential for immune-mediated toxicities. The ability to turn IL-12 expression on and off using an orally activated gene switch, particularly in the brain’s immune privileged environment, is a tremendous advancement in the potential of this therapeutic approach. We look forward to enrolling additional patients and follow up from this study to evaluate Ad-RTS-IL-12’s potential in this challenging, rapidly advancing and lethal disease.”
The ongoing multi-center Phase 1 trial of Ad-RTS-hIL-12 + veledimex examines a gene therapy strategy for recurrent high grade gliomas, with the goal of generating a localized anti-tumor 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 activated upon dosing with oral veledimex. Secondary objectives are to determine the Ad-RTS-hIL-12 + veledimex maximum tolerated dose, the immune responses elicited by Ad-RTS-hIL-12 + veledimex, and assessment of biologic response. The study is expected to enroll up to 72 subjects.
Preclinically, the effects of Ad-RTS-mIL-12 + veledimex were studied in orthotopic glioma animal models, demonstrating veledimex crossed the blood-brain-barrier. In a standard orthotopic glioma mouse model that evaluated dexamethasone, bevacizumab, temozolamide and a PD-1 inhibitor, Ad-RTS-mIL-12 + veledimex demonstrated a dramatic dose-related increase in survival, without significant adverse events, that was superior to all other treatments.
In the current, on-going Phase 1 study, five patients are available for initial assessment, two with recurrent grade III malignant glioma and three with grade IV. Results show IL-12 was detectable in peripheral blood along with downstream IFNg, indicating that veledimex crossed the blood brain barrier activating IL-12 expression from intra-tumorally administered Ad-RTS-hIL-12. Ad-RTS-hIL-12 + veledimex was well tolerated with minimal neurologic toxicity. The most common adverse events were headache, fever, hyponatremia and nausea/vomiting. Related serious adverse events were aseptic meningitis, neutropenia, thrombocytopenia, leukopenia, with all toxicity to date consistent with the “on-target” effects of immunotherapy.
“Observing that veledimex can cross the blood brain barrier and that IL-12 expression can be regulated in the brain, demonstrates a clear translation of results from the laboratory to the clinic,” said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM. “We look forward to follow-up of the current recipients and to further enrollment in this multi-center gene therapy study.” (Original Source)
Shares of Ziopharm Oncology are up 2.26% to $12.81 in pre-market trading. ZIOP has a 1-year high of $14.93 and a 1-year low of $3.42. The stock’s 50-day moving average is $11.62 and its 200-day moving average is $10.98.
On the ratings front, Ziopharm has been the subject of a number of recent research reports. In a report issued on October 30, J.P. Morgan analyst Cory Kasimov reiterated a Hold rating on ZIOP. Separately, on September 17, Griffin Securities’ Keith Markey reiterated a Buy rating on the stock and has a price target of $21.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Cory Kasimov and Keith Markey have a total average return of -0.1% and -20.4% respectively. Kasimov has a success rate of 51.1% and is ranked #2676 out of 3855 analysts, while Markey has a success rate of 26.3% and is ranked #3718.
ZIOPHARM Oncology Inc is a biopharmaceutical company. The Company is engaged in the acquiring, developing and commercializing portfolio of cancer therapies that can address unmet medical needs through synthetic biology.