Ziopharm sharpens budget ax after cancer drug palifosfamide flops in PhIII

Shares of Ziopharm Oncology ($ZIOP) went down in flames this morning when the biotech put out the news that it is dumping its cancer drug palifosfamide after the drug failed a late-stage study for metastatic soft tissue sarcoma. The company didn't spell out the data initially, but it made clear that the drug failed to hit the primary endpoint on progression-free survival--prompting executives to call for the budget ax. 

The failure triggered an immediate 60% plunge in Ziopharm's share price, which had inflated rapidly in the lead-up to the release. Small developers have a notoriously bad track record when it comes to late-stage cancer drug development. They also have a habit of becoming an attraction for biotech investors looking for a big payday--even though the odds can be terrible.

TheStreet's Adam Feuerstein, who had recently begun to doubt his own projected win for Ziopharm, reported that the control arm had an unexpectedly high PFS rate of 5.2 months, with the drug arm doing only slightly better, falling behind expectations.

Ziopharm noted that the independent monitoring board had recommended that the company follow up on the patients to track overall survival rates. But the biotech shrugged that suggestion off and quickly scrambled to circle the wagons around its partnership with Intrexon, owned by billionaire RJ Kirk. Ziopharm added that it is working on a restructuring plan as it concentrates its resources on a "highly focused team" for the synthetic biology work with Intrexon.

Ziopharm's next chapter will center on the new lead therapeutic candidate, Ad-RTS IL-12, a DNA therapeutic "to enable controlled delivery of therapeutic interleukin-12 (IL-12), a protein important for an immune response to cancer. This is achieved by placing IL-12 under the control of Intrexon's proprietary biological 'switch' (the RheoSwitch Therapeutic System, RTS) to turn on/off the therapeutic protein expression. Ad-RTS IL-12 is currently being tested in two Phase II studies, the first for the treatment of advanced melanoma, and the second in combination with palifosfamide for the treatment of non-resectable recurrent or metastatic breast cancer."

"We are disappointed that the PICASSO 3 study did not meet its primary endpoint of progression-free survival," said Ziopharm CEO Jonathan Lewis. "We sincerely thank the trial investigators, clinical sites and the ZIOPHARM team for conducting a highly rigorous study, and are deeply appreciative to the cancer patients and their families for their participation in this trial."

Dr. Lewis added: "It is imperative that the Company rapidly focus its resources and efforts on our highly promising synthetic biology programs, employing therapeutic motifs that represent the next-generation in biotechnology."

- here's the press release
- read the report from TheStreet

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