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VIDEOWhat's Next for Ziopharm and Its Cancer Drug?
The biotech faces new hurdles from the FDA and has to make a choice about how to proceed with its lead drug candidate, Zymafos.
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Whether it continues to have upward momentum will be determined in the next few months as Ziopharm management decides what to do about its lead drug candidate -- Zymafos, also known as palifosfamide.
Zymafos is a treatment for metastatic soft-tissue sarcoma. This is a cancer that begins in the soft tissues of the body -- fat, muscle, blood vessels, or nerves -- and usually affects the legs, arms, and abdomen. According to the National Cancer Institute, there are about 9,000 cases in the US annually.
Ziopharm garnered Orphan Drug status for the drug in 2007. This designation from the FDA allows the biotech to have seven years exclusivity on the drug if it reaches the market, grants it the option of accelerated approval, and gives it access to grant funding and tax benefits. The orphan designation is given to drugs that are meant to treat a rare disease area that affects less than 200,000 Americans.
Up until this week, the little biotech has been charging full steam ahead. The company recently garnered a prime place at the annual American Society of Clinical Oncology meeting where it presented updated data from its mid-stage study on Zymafos called Picasso. The study compared Zymafos plus a chemotherapy with patients only taking the chemotherapy. The arm of the study that had patients on both the Ziopharm drug and the chemotherapy showed 3.4 extra months of progression-free survival. This is a long time in cancer months for a patient to have no further signs of disease.
Ziopharm was all set to begin enrolling patients in its late-stage study of the drug until the FDA complicated the process. The biotech had applied for a special protocol assessment, which would have allowed the company to work with the FDA to determine a goal for the outcome of the clinical trial that may be different from what's normally expected of that sort of drug. This is often given to orphan drugs because any option is better than no option for most patients.
According to the company, the SPA was denied because the trial design included progression-free survival for patients as the primary goal of the study. It appears that the company would have to redesign the trial to have a primary endpoint of overall survival for it to get SPA status.
Why does this matter? Well, a redesign to the trial would push out the start time by several months, costing the company time and money. Beyond that, there's been little data presented on how much the drug improves overall survival in patients. A trial with that endpoint could show that it doesn’t work any better than the chemotherapy alone -- something that would be very bad for the company.
But Ziopharm has decided that it doesn’t need an SPA to move forward with the study. “FDA has indicated that the company could conduct the pivotal trial as designed without
SPA and that approvability would be determined by the data, balanced with risks and benefits,” said the biotech in a statement to the press.
Rodman & Renshaw analyst Simos Simeonidis sees the company’s confidence in moving forward with the trial as a good sign. “According to last night’s discussion with management, overall survival will remain the primary endpoint for full approval, but the company is currently planning on filing for accelerated approval based on the primary endpoint of progression-free survival, and regardless of the fact that the FDA did not grant an SPA with progression-free survival as the primary endpoint,” wrote Simeonidis in a note to investors.
So despite this bump in the regulatory road, Ziopharm still has plenty to bank on -- its lead compound showed superior safety and efficacy, the drug faces little competition in an unmet need disease area, and Zymafos has the potential to be used in other cancer settings.
If you aren’t convinced that it's time to buy, it’s easy to see that it's time to watch.Twitter: @biowriterchik
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