Now here’s an idea everyone can get behind: cancer drugs that are effective and affordable. That’s the goal of an eight-year-old biotechnology company based in New York City, Ziopharm (Nasdaq:ZIOP). CEO Jonathon Lewis says that if successful in its quest, the company will usher in a new era of cancer drugs that won’t bankrupt patients requiring treatment.
Lewis, a former surgeon from Memorial Sloan-Kettering Cancer Center, has one deep-pocketed supporter who shares his optimism – and has demonstrated it by writing a huge check. Early this year, billionaire biotech investor Randal J. Kirk’s privately held Intrexon bought a 12.5% stake in Ziopharm for $11.6 million.
The stake could get much higher if Ziopharm brings any drug based on Intrexon’s technology into a second-stage trial. That technology uses synthetic biology to come up with new DNA-based drugs that trigger cells to produce powerful cancer-killing chemicals precisely where they are needed inside the body. This could have far fewer side effects than injecting cancer killing immune proteins directly into patients.
Ziopharm shares have enjoyed a nice growth spurt this year, gaining about $3 to $7.70 in early May before falling back below $7. It’s not known whether the bounce in Ziopharm shares was because investors felt more confident about the company’s prospects or were just playing “follow the leader.” After all, Kirk has racked up a nice track record with Halozyme Therapeutics (Nasdaq:HALO ) and made a fortune selling New River Pharmaceuticals to Shire (Nasdaq:SHPGY) in 2007 for $2.6 billion.
Despite his vast industry background, Kirk says the Inextron technology is head and shoulders above anything he’s ever seen in biotechnology and predicted in an interview that it will be “world-changing.”
With the synthetic biology technology, the new DNA drugs from Intrexon could be hundreds of times more potent than previous gene therapy or DNA vaccine approaches, claims Kirk. The lead treatment, now in Phase I trials, aims to produce a potent immune-boosting protein called IL-12 inside tumors, while avoiding healthy tissues. One possibility would be to use it as an alternative to surgery for difficult-to-operate-on tumors.
Under its partnership with Inextron, Ziopharm gets rights to Intrexon’s entire human in vivo effector platform within the field of oncology, which the company will use to develop and commercialize DNA-based therapeutics. The lead clinical candidate is in advanced Phase I study and a second will be the subject of an IND (Investigational New Drug) filing expected in the first half of this year.
Supposedly, Kirk didn’t even take a look at Ziopharm’s promising portfolio of drugs outside the partnership before making his investment. The lead candidate in that group is palifosfamide, a new version an existing chemo drug that is potent but too toxic to use on many people. Ziopharm’s focus here and on other candidates is to come up with less toxic versions of established cancer drugs. In an initial trial last year, palifosfamide delayed growth of sarcoma by about three months versus standard treatment. Final stages trials in sarcoma could yield results in 2012, while a small trial in lung cancer just started.
