What: After reporting that the first patient has been dosed with its second generation CAR-T cancer killing therapy on Feb. 9 and releasing its fourth quarter financials on Feb. 24, Ziopharm Oncology (NASDAQ:ZIOP) sky-rocketed 49.4% in February, according to S&P Global Market Intelligence.
So what: The initiation of human trials for Ziopharm Oncology's second generation CAR-T therapy is big news because this next-generation approach includes the Sleeping Beauty technology that the company and its partner Intrexon (NASDAQ:PGEN) licensed from MD Anderson in January 2015.
Previously, T-cells were modified to find and destroy B-cell cancers by an expensive and time-consuming process that uses viruses to "teach" T-cells to recognize CD-19, a B cell specific protein.
Instead, Sleeping Beauty is a non-viral approach that relies on a DNA sequence that can change its location within the genome. After a chimeric antigen receptor is developed for each patient's specific cancer, the sequence is partnered up with Sleeping Beauty and introduced to patients via electroporation, which allows the DNA to enter the T-cells.
Ziopharm Oncology thinks that using Sleeping Beauty to deliver its CARs will reduce the cost and time associated with re-engineering a patient's immune system, in turn, making this second-generation approach more viable than the previous generation.
On Feb. 24, Ziopharm Oncology also updated investors on its financial picture, reporting that research and development and SG&A expenses resulted in the company losing $9.5 million and $120 million in the fourth quarter and in 2015, respectively.
Now what: Ziopharm's teaming up Sleeping Beauty with Intrexon's Rheoswitch technology, which allows T-cell activity to be turned on and off as necessary, could allow the company to leapfrog other CAR-T competitors, including Juno Therapeutics and Kite Pharma that are similarly developing B cell therapies.
However, Ziopharm Oncology's trials are in the early stages of human clinical testing and cancer drug development is complex and costly. As a result, investors might want to temper their optimism and keep a close eye on the company's clinical trial progress. Ziopharm Oncology should have additional data from brain cancer, breast cancer, and B-cell trials available in the coming year.
Investors should also keep tabs on the company's cash burn this year. Although the $140.7 million in cash on its books exiting December should give it the financial flexibility it needs for now, equity markets are less willing to provide funding to biotech stocks than they were a year ago, and that could make it a bit tougher to raise additional money to finance studies in the future.