Positive trial updates from Ziopharm’s glioma gene therapy at ASCO

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A gene therapy highlight from the latest American Society of Clinical Oncology (ASCO) annual meeting was an update from Boston based biopharm company Ziopharm. The latest results from the company’s Phase 1 trial of its combinatorial therapy Ad-RTS-hIL-12 + veledimex in advanced glioblastoma patients is beginning to present a clear correlation between the treatment and survival, as well as a positive safety profile.

Ad-RTS-hIL-12 comprises of an adeno associated virus (AAV) vector engineered to express hIL-12 (INXN-2001). IL-12 is a protein that may improve the body’s natural response to disease by enhancing the ability of the immune system to kill tumor cells and interfering with blood flow to the tumor. In Ziopharm’s study this gene therapy is being investigated in conjunction with oral veledimex, an activator ligand which mediates the expression of IL-12.

Results were reported from 25 patients with recurrent or progressive Grade III or IV glioma enrolled in three veledimex dosing cohorts. Median survival from this cohort currently stands at 12.5 months, with the trial ongoing until December this year. A strong, dose-dependent correlation between veledimex dose, veledimex blood brain barrier penetration, IL-12 and IFN-gamma production was observed. The ratio of CD8+/FOXP3+ (effector/suppressor) T cells measured in peripheral blood 14 to 28 days after viral injection correlated with immune activation by the therapy.

On these latest results, Harvard Medical School Neurosurgery professor Antonio Chicoca commented “These data suggest that intra-tumor expression of IL-12 is well tolerated by patients with recurrent glioblastoma. There are also highly encouraging observations that activation of the immune system in the patients may result in anti-tumor effects. I look forward to understanding Ad-RTS-IL-12 + veledimex’s full potential in this challenging disease in a larger study.”

Source: ZIOPHARM Oncology Announces Positive Updated Results of Ad-RTS-hIL-12 + Veledimex in Recurrent Glioblastoma at the 2017 ASCO Annual Meeting Press Release