Ziopharm reports death in Phase I trial of GBM drug candidate

Shares of US gene therapy developer Ziopharm Oncology (Nasdaq: ZIOP) dropped 22% to $4.44 in premarket trading on Friday, after it revealed that a Glioblastoma multiforme (GBM: brain cancer) patient died of cranial bleeding about two weeks after starting treatment with the company’s lead experimental drug, Ad-RTS-hIL-12.  The stock partially recovered, closed the day down 11.6% at $5.01.

The company's ongoing multicenter Phase I study is of Ad-RTS-hIL-12 + orally administered veledimex in recurrent or progressive glioblastoma (GBM) or grade III malignant glioma:

In a statement, the company explained: “This Phase I study is being conducted in late-stage, recurrent GBM, so these patients are all, unfortunately, medically fragile. The first two patient deaths, which occurred 6.7 months and 3.9 months after treatment, were unrelated to study drug. A third death has just been reported to us and we are collecting and analyzing information in order to properly and timely report it to the FDA [Food and Drug Administration]. The cause of death is intracranial hemorrhage, which occurred some time after the patient had been discharged from the treating center. This is an isolated case, and there have been no reported related instances of brain hemorrhage in any pervious cohort or prior studies with Ad-RTS-hIL-12 + veledimex.”