Shares of Ziopharm $ZIOP plunged this morning after Adam Feuerstein at TheStreet broke the story that one patient in its gene therapy study dubbed Ad-RTS-hIL-12 died due to an intracranial hemorrhage 15 days after starting treatment. The note on the death — one of several — was included in a slide presentation filed with the SEC. Notably, Feuerstein had the report even before FDA regulators were notified.
The gene therapy is designed to unleash an immune system attack on brain tumors. Ziopharm’s shares plummeted 22%, but recovered somewhat and were down 12% in mid-morning trading.
Here’s the company’s official response:
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. 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. Enrollment remains open in the study, and we will be discussing with our Safety Review Committee the appropriate course of action. For patients who have experienced multiple recurrences, as these patients have, prognoses are particularly poor. Median follow up in the first dose cohort from our study is now 8 months, in a population with an expected overall survival of 3 to 5 months for patients that have failed temozolomide and bevacizumab, or equivalent salvage chemotherapy. For the patients that remain in follow up in this Phase I study, we believe that preliminary overall survival remains encouraging. The Company expects to provide an update once a course of action has been determined.
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