Ziopharm Delays Phase III Glioblastoma Trial to Focus on Other IL-12 Studies


Shares of ZIOPHARM Oncology, Inc. are still sliding after the company announced it was pausing its planned Phase III trial for its lead gene therapy product designed to treat patients with recurrent glioblastoma (rGBM) as a monotherapy and in combination with an immune checkpoint inhibitor.

On Friday Boston-based Ziopharm said before the trial can continue it must resolve previously disclosed technical requirements related to Chemistry and Manufacturing Control (CMC) in order to make the asset ready for the Phase III trial in rGBM. This is the second delay the company has initiated for this particular study. Last year the program was pushed back several months and expected to start this year. Ziopharm did not provide a timeline for the Phase III trial to begin.

The company said it intends to advance Ad-RTS-hIL-12 plus veledimex as a treatment for rGBM, but before it does so, the company said it intends to “focus its resources on studies of Controlled IL-12 in additional tumor types to demonstrate the value of this platform technology.” That was the reason cited by the company for the delay in its first quarter financial report.

During a conference call with investors and analysts David Mauney, Ziopharm’s chief business development officer and interim Chief operating officer, said the company wants to focus on expanding its IL-12 program into new tumor types and with new combinations.

“…the idea here is to make immunologically cold tumors hot by calling in the bodies T cells via the tunable control of IL-12. Once these cold tumors are flush with cancer-fighting T cells, the gates are now open to turbo-charge the effects by adding in combination therapies like checkpoint inhibitors,” Mauney said during the call, according to transcripts. “Additionally, from a business perspective, we've seen several recent transactions that suggest having an ability to make cold tumors hot across multiple tumor types. Even with just Phase 1 data drives tremendous value relative to pursuing just one indication. We're now executing on a revised clinical plan for this technology, which is focused on delivering clinical data in multiple tumor types and in combination with checkpoint inhibitors as quickly and cash efficiently as possible.”

Mauney framed the decision to pause the glioblastoma trial as a business move that could potentially provide the company with a greater revenue stream. With Eli Lilly’s recent $1.6 billion acquisition of ARMO BioSciences and its cytokine programs, Ziopharm could be seeing the potential for lucrative partnerships.

“This effort greatly expands our addressable markets. It aligns us with what potential partners want to see in terms of clinical data, and have the additional benefit of tremendous cost savings in the near-term. We view this as a positive pivot point for this platform and executing on this plan will provide the information needed in the shortest time possible and for a fraction of the cost of a randomized controlled pivotal trial,” Mauney said in a statement.

While Ziopharm is indefinitely pausing the glioblastoma trial, the company said it is moving forward with its combination trial of Ad-RTS-hIL-12 and Opdivo, Bristol-Myers Squibb’s blockbuster checkpoint inhibitor. Ziopharm said it expects the first patient to be dosed in this trial in the second quarter. The company is also focused on its Sleeping Beauty program to speed up the manufacture of CAR-T cells. The company said it anticipates driving this program into the clinic in the second half of this year. The Sleeping Beauty program is designed to manufacture the CAR-T cells in two days, a move the company believes will address concerns of high cost to patients.

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