BOSTON—Ziopharm Oncology Inc. recently announced that the first patient with diffuse intrinsic pontine glioma (DIPG) has been dosed in its Phase 1/2 study of Ad-RTS-hIL-12 with veledimex (Controlled IL-12) for the treatment of pediatric brain tumors.
“We are pleased to report that this young child has tolerated the dosing regimen well,” said Dr. Laurence Cooper, CEO of Ziopharm. “Working with our colleagues at Northwestern University and Lurie Children’s Hospital, we continue to monitor this patient’s progress and are evaluating additional patients for enrollment in Chicago and at the other trial sites. For Ziopharm, this trial represents an additional clinical path for of Controlled IL-12, beyond the ongoing studies in recurrent glioblastoma (rGBM).”
“One of the hallmark characteristics of DIPG is that immune cells cannot access the tumor, yet the microenvironment is not immunosuppressive,” added Dr. Stewart Goldman, division head of hematology-oncology, neuro-oncology and stem cell transplantation at Lurie Children's and an investigator. “Therefore, driving T cells into this tumor could change the outcome for children with this lethal disease. Ziopharm’s data evaluating Controlled IL-12 in rGBM demonstrates that there is a sustained infiltration of activated T cells, turning ‘cold’ tumors ‘hot’ for months after veledimex dosing is finished. Extensive experience, as well as encouraging survival data associated with treating rGBM in adults, underscores our desire to evaluate Controlled IL-12 in children with gliomas, who currently lack viable treatment options.”
The Phase 1/2 trial (NCT03330197) is designed to evaluate the safety and tolerability of a single intratumoral injection of Ad-RTS-hIL-12 given with up to 14 days of oral veledimex in children with gliomas. Up to 12 patients may be enrolled in phase 1 of the study, which is being conducted at leading pediatric cancer centers across the United States, including the Dana-Farber Cancer Institute in Boston and the University of California in San Francisco.
Ziopharm’s Controlled IL-12 platform is an investigational gene therapy designed to induce and control the production of human interleukin 12 (hIL-12), a master-regulator of the immune system. The company has treated more than 175 patients, including more than 125 patients with rGBM, with Ad-RTS-hIL-12 plus veledimex and administered more than 1,300 doses of veledimex across three types of solid tumors, building a significant safety profile, mechanistic dataset and evidence of anti-tumor effects.