-- Substudy promptly accrued 36 patients in less than six months, with majority of newly enrolled patients receiving low-dose steroids --
BOSTON, Feb. 11, 2019 (GLOBE NEWSWIRE) -- Ziopharm Oncology, Inc. (ZIOP) today announced that it rapidly completed enrollment and treated a total of 36 patients in less than six months in a substudy (Clinicaltrials.gov NCT03679754) to expand a Phase 1 trial evaluating its Controlled IL-12 platform as a monotherapy for the treatment of recurrent glioblastoma (rGBM). The trial was over enrolled with eleven more patients than the target goal of 25, which the Company attributes to enthusiasm stemming from the trial’s encouraging survival and tumor biopsy data.
Ziopharm is developing its Controlled IL-12 platform, or Ad-RTS-hIL-12 plus veledimex, as a drug to control the production of interleukin 12 (IL-12). In the setting for the treatment of rGBM, previously reported Phase 1 data demonstrated an increased survival benefit when using 20mg of veledimex, which is extended further when patients also received low-dose steroids versus higher doses. A majority of the newly enrolled patients in the substudy were treated with low-dose steroids.
“Glioblastoma is a difficult-to-treat brain cancer, with very few therapeutic options at recurrence and there is a significant need to develop new treatment options,” said Rimas Lukas, M.D., associate professor of Neurology at Feinberg School of Medicine, Northwestern University. “This investigational therapy designed to stimulate and control expression of interleukin 12, a very powerful immuno-stimulant, has shown promise in treating brain cancer, especially when minimizing the use of immune-suppressive steroids. This substudy accrued quite rapidly, and we look forward to the results from treating these additional patients with rGBM.”
The Company is now evaluating data from 51 patients with rGBM treated with 20mg veledimex dose and assessing the impact of systemic dosing of steroids. The safety profile in the substudy is consistent with the main, Phase 1, dose-escalation study (Clinicaltrials.gov NCT02026271) in which patients received varied systemic dosing of steroids, with all adverse reactions being manageable and reversible. Ziopharm anticipates reporting on preliminary data from this substudy at medical meetings this year.
About Ad-RTS-hIL-12 plus veledimex
At the 2018 annual meeting of the Society for Neuro-Oncology, Ziopharm presented data from its Phase 1 dose-escalation trial showing that Controlled IL-12 (Ad-RTS-hIL-12 plus veledimex) had a positive survival benefit, with 15 patients who received 20mg veledimex reaching 12.7 months median overall survival (mOS) at a mean follow up of 13.1 months. A subset of these patients (n=6) who received low-dose steroids (20mg or less of dexamethasone cumulatively over 15 days while receiving veledimex) had mOS of 17.8 months compared to 6.4 months mOS for patients (n=9) who received more than 20mg of dexamethasone during the same period. The survival data from patients who received the preferred dosing regimen of Controlled IL-12 with 20mg veledimex and low-dose steroids compare favorably to a benchmark mOS of 5 to 8 months for patients with rGBM that continues to serve as historical control.
The Company has treated more than 100 patients, including more than 75 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 effect. Biopsy data demonstrated that Controlled IL-12 turns immunologically-cold tumors hot based on sustained infiltration of killer T cells which is likely responsible for the preliminary improved survival observed with use of Ad-RTS-hIL-12 plus veledimex as monotherapy in patients with rGBM. Biopsy data also revealed upregulation of immune checkpoints providing a compelling rationale for combining Controlled IL-12 with PD-1 inhibitors.
Learn more about Controlled IL-12 online at https://ziopharm.com/controlled-il-12/.
About Ad-RTS-hIL-12 plus veledimex in combination with PD-1 inhibitors
The Company also is advancing Controlled IL-12 as a combination therapy with PD-1 inhibitors. Enrollment in a substudy of the ongoing Phase 1 trial to evaluate Controlled IL-12 in combination with the PD1 inhibitor OPDIVO® (nivolumab) is on track to be completed in the second quarter of this year (Clinicaltrials.gov NCT03636477). The Company is expected to begin a Phase 2 trial to evaluate Ad-RTS-hIL-12 plus veledimex in combination with Regeneron Pharmaceuticals’ PD-1 antibody Libtayo® (cemiplimab-rwlc) in the second quarter of 2019.
About Ziopharm Oncology, Inc.
Ziopharm Oncology is an immuno-oncology company focused on developing end-to-end cost-effective solutions using its non-viral Sleeping Beauty platform for TCR and CAR T-cell therapies and immune-stimulating gene therapy with Controlled Interleukin 12 (IL-12). The Sleeping Beauty platform genetically modifies T cells with DNA plasmids, instead of virus, to express T-cell receptors (TCRs) to target specific antigens in solid tumors, and chimeric antigen receptors (CARs) to target CD19 in blood cancers with membrane-bound interleukin 15 (mbIL15) and a kill switch for under 2-day manufacturing. The Sleeping Beauty platform is being advanced in collaboration with the National Cancer Institute, The University of Texas MD Anderson Cancer Center and Eden BioCell. The Company also is developing its Controlled IL-12 platform or Ad-RTS-hIL-12 plus veledimex as monotherapy and in combination with immune checkpoint inhibitors to treat brain cancer, including a collaboration with Regeneron Pharmaceuticals.
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