ZIOPHARM Oncology Inc. (ZIOP) Completes Enrollment in Second Patient Cohort and Initiates Enrollment in Third Cohort in Phase 1 Study of Gene Therapy Candidate Ad-RTS-hIL-12 in Brain Cancer

ZIOPHARM Oncology Inc. (NASDAQ:ZIOP), a biopharmaceutical company focused on new cancer immunotherapies, today announced the successful completion of enrollment in the first and second dosing cohorts as well as the initiation of enrollment in a third cohort in the Company’s ongoing multi-center Phase 1 study of Ad-RTS-hIL-12 + orally administered veledimex to treat recurrent or progressive glioblastoma (GBM) or grade III malignant glioma. Ad-RTS-hIL-12 + veledimex is a novel viral gene therapy candidate for the controlled expression of interleukin 12  (IL-12), a critical protein for stimulating an anti-cancer immune response.

The primary objective of the study is to determine the safety and tolerability of a single intratumoral Ad-RTS-hIL-12 injection activated upon dosing with oral veledimex. Secondary objectives are to determine the maximum tolerated dose, the immune responses elicited, and assessment of biologic response. The first cohort of seven patients received 20 mg doses of veledimex, the second cohort of six patients received 40 mg doses of veledimex, and the third cohort will receive 30 mg doses of veledimex to refine the effect of activating the immune response within the tumor. The resultant immunologic activity that follows IL-12 expression in the brain suggests that no further dose escalation will be necessary and the optimal dosing may be reached sooner than initially anticipated.

Francois Lebel, M.D., Executive Vice President, Research and Development, Chief Medical Officer at ZIOPHARM, commented: “With the RheoSwitch® (RTS®) technology, the only switch currently in the clinic that operates on gene transcription, we have demonstrated the ability for veledimex to cross the human blood brain barrier and activate production of IL-12 in GBM tumors in a dose-dependent manner, giving us the potential to precisely tune the balance between activity and tolerability.”

Data from 11 patients with recurrent high-grade gliomas were recently presented at the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting. All of these patients failed at least two prior lines of therapy and underwent partial resection leaving residual tumors, in certain cases with significant tumor burden. Ad-RTS-hIL-12 was administered through direct injection into the brain tumor and veledimex was taken orally to activate the production of IL-12 from the tumor site and stimulate an immune response.

As of May 18th, the date of data collection for the ASCO presentation, overall median follow up was 6.2 months, with 10 of 11 recipients alive. IL-12 in the bloodstream was measured and was found to be proportional to the amount of veledimex administered, demonstrating that this orally-delivered activator crossed the blood brain barrier to turn on the RheoSwitch® technology in a dose-dependent manner.

To date, toxicities in both dose cohorts were consistent with those previously reported, with a higher incidence of grade 3 or greater adverse events in the 40 mg dose group.  All related side effects were reversed upon cessation of veledimex.  No subsequent deaths have been reported.

The Company expects to present updated results from the study at a scientific meeting later in the year.

“Overall survival remains the gold standard of therapeutic success in glioblastoma, particularly in high-grade, recurrent disease, where survival is too often measured by just a few months,” said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM. “We remain encouraged by the outcomes of Ad-RTS-hIL-12 as a single agent tuning the immune system in this GBM study. We believe that these early results also have positive implications for our combination approach utilizing this novel gene therapy with immune check point inhibitors. We look forward to additional follow up as we work to fine-tune dosing levels using the RheoSwitch® technology.”

Ad-RTS-hIL-12 + veledimex has been granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with malignant glioma. (Original Source)

Shares of Ziopharm closed today at $5.31, down $0.33 or -5.85%. ZIOP has a 1-year high of $14.93 and a 1-year low of $4.56. The stock’s 50-day moving average is $6.86 and its 200-day moving average is $7.25.

On the ratings front, Ziopharm has been the subject of a number of recent research reports. In a report issued on June 6, Wells Fargo analyst Jim Birchenough maintained a Sell rating on ZIOP. Separately, on June 2, Raymond James’ Reni Benjamin initiated coverage with a Hold rating on the stock .

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Jim Birchenough and Reni Benjamin have a total average return of 17.4% and -13.9% respectively. Birchenough has a success rate of 42.9% and is ranked #102 out of 3886 analysts, while Benjamin has a success rate of 28.1% and is ranked #3830.

ZIOPHARM Oncology, Inc. is a biopharmaceutical company currently in developmental stage that seeks to acquire, develop and commercialize, on its own or with commercial partners, a diverse portfolio of cancer therapies. It is focused on two clinical stage product candidates: Ad-RTS-IL-12 + veledimex and DC-RTS-IL-12 + veledimex. 


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