Ziopharm Discloses Patient Death in Brain Cancer Trial, Shares Fall

Xconomy Boston — 

A patient died after receiving Ziopharm Oncology’s experimental treatment for an aggressive form of brain cancer, but the Boston-based biotech company describes the death as “an isolated case” and plans to continue the study.

Nonetheless, the news has spooked investors and Ziopharm’s (NASDAQ: ZIOP) stock price slipped more than 13 percent to $4.89 this morning.

Ziopharm’s (NASDAQ: ZIOP) treatment, known as Ad-RTS-hiL-12, is injected directly into a patient’s tumor. The company describes its treatment as a “gene switch” that controls the expression of a protein—interleukin-12, or IL-12—that can help drive an immune response against cancer. Ziopharm is studying Ad-RTS-hiL-12 in glioblastoma, breast cancer, and melanoma.

The patient death, first reported by TheStreet.com, occurred in a Phase 1 clinical trial in glioblastoma. Ziopharm disclosed the death in a slideshow for a Thursday presentation during an American Society of Hematology workshop on gene editing in Washington, DC. The company included the slideshow in a filing with securities regulators. In the document, the company said the patient died from bleeding in the brain 15 days after starting treatment with the Ziopharm drug. Two other patient deaths, occurring at 6.4 months and 3.8 months after treatment, were unrelated to the Ziopharm drug, the company said in the filing.

In a statement issued this morning, Ziopharm elaborated on the news, saying that the patient who experienced bleeding in the brain died after being discharged from the treatment center. The death had just been reported to the company, which is still collecting and analyzing information to report to the FDA.

Ziopharm has had an up and down history. Following the 2013 failure of a chemotherapy drug, Ziopharm refocused its efforts on synthetic biology, in part by forming deep ties with Intrexon Corp. (NYSE: XON). Ziopharm’s current cancer drugs, including Ad-RTS-hiL-12, were developed in partnership with Intrexon and the MD Anderson Cancer Center in Texas.

Ziopharm said today that it is continuing to enroll patients in the glioblastoma study and will also discuss the findings regarding the patient’s death with its safety review committee. In the meantime, the bad news stifled plans to raise additional cash. TheStreet.com reported that Ziopharm planned to raise as much as $50 million in a stock offering, but the deal was put on hold due to concerns about the patient death and the way that the company disclosed it.

Image of the brain courtesy of Flickr user Allan Ajifo under a Creative Commons license.