Ziopharm says patient death unrelated to gene therapy
- Ziopharm Oncology said Tuesday a previously reported patient death in the company's early-stage study of a gene therapy candidate was unrelated to treatment.
- The patient had died of an intracranial hemorrhage 15 days after receiving the experimental gene therapy, sparking safety concerns.
- Ziopharm is testing the therapy among patients with recurrent or progressive glioblastoma, an aggressive type of brain tumor which annually affects about 74,000 people worldwide according to the company.
Ziopharm stock fell sharply on news of the patient death last week, which was the third death reported in clinical testing of the therapy.
Both other deaths had previously been ruled unrelated to treatment but it was unclear whether there was a link between the third death and the drug.
Additional information from Ziopharm and the study's safety review committee led to Tuesday's conclusion that the death was unrelated, the company said.
"Recurrent GBM is a devastating disease with an expected overall survival that remains far too short as this case illustrates," said Francois Lebel, chief medical officer at Ziopharm. "We will continue to work diligently toward understanding the full potential of Ad-RTS-hIL-12 + veledimex in this disease, with the goal of providing safe and effective treatment options to these patients."
Enrollment in the study will continue, with 48 participants expected according to federal database of clinical trials.
Ziopharm stock rebounded slightly in early trading Tuesday, but soon pared back those gains and turned negative by mid-afternoon.
Coming on the heels of the clinical hold placed on Juno Therapeutics CAR-T trial, the death in Ziopharm's study fed into concerns about the safety of gene therapies. The Food and Drug Administration halted Juno's trial after three patients died, although Juno was quickly given a green light to restart after modifying its treatment regimen.
While Juno and Ziopharm's treatments are unrelated and aimed at different diseases, both aim to boost the ability of the immune system to target and kill cancers by genetically engineering cells.
The FDA has not weighed in on Ziopharm's trial yet. The company said it plans to report the data to the regulator.
- Ziopharm Oncology Statement
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