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delivering a gene directly into patients. A pill is used to switch the gene into active mode, at which time it produces an immune-boosting molecule called IL-12 (interleukin 12).
The idea of using IL-12 as a therapy has been around for over a decade, but previous attempts produced toxic side effects. Ziopharm’s approach is to sidestep the side effects by getting the therapeutic DNA directly into patients’ cells and then turning it on for a limited amount of time. “It’s a biological switch,” Lewis says. “For the DNA to work, we have to switch it on with a pill. It’s a controllable way to give this drug.”
Other companies are working on different synthetic-biology approaches to making IL-12 palatable in oncology. San Diego-based OncoSec Medical, for example, is using electrical pulses to transmit IL-12 into skin tumors. And Lewis says he also expects some competition from genomics pioneer Craig Venter, who has a number of initiatives underway in synthetic biology, including some aimed at combating cancer. “The one that’s at the front in terms of human therapeutics, as far as we know, is Ziopharm,” Lewis says.
Lewis is driven by a personal need to improve the therapeutic choices for cancer patients. He is an oncologist who served as professor of surgery and medicine at Memorial Sloan-Kettering Cancer Center in New York, which has been involved in several of Ziopharm’s clinical trials. “I’ve experienced so many limitations as a treating physician,” he says. “We need better treatments.”
Ziopharm has a handful of other compounds in its pipeline, including the cancer drug that the company was founded on in 2004: darinaparsin, which is derived from arsenic. Darinaparsin is in early-stage trials in Japan and the Pacific Rim in partnership with the Japanese company Solasia Pharma.
Lewis says he has pursued a selective partnering strategy, and thus far the company has chosen to go it alone on its lead program. “There was a time when the financial markets were shut down and we were desperate to partner it,” he says. But with $110 million in cash, the company is now in a strong position to fund all its R&D work through next year, without the need to take on new partners, he says. Still, Ziopharm isn’t opposed to talking to potential partners, he says, and in fact, the company’s board members are well-versed in doing deals—especially Intrexon’s R.J. Kirk. His career is dotted with lucrative deals, including having served as the chairman of Fremont, CA-based Scios, which was acquired by Johnson & Johnson (NYSE: JNJ) for $2.69 billion in 2003, and as the chairman of Newton, MA-based Clinical Data, which was bought by Forest Laboratories (NYSE: FRX) in 2011 for $1.2 billion.
For now, Lewis remains focused on his immediate priorities: producing good data by year-end for palifosfamide and proving that Ziopharm’s foray into synthetic biology holds promise. And though he’s well aware Wall Street won’t be all that impressed with Ziopharm until there’s a product on the horizon, he’s convinced his company has more to look forward to in the long run. “I predict the future of medicine is going to change dramatically in the next 10 years,” Lewis says. Synthetic biology, he adds, “is going to change how we treat patients, how we interact with them. It’s just starting right now.”