Nine-month-old Ziopharm Inc. completed a worldwide licensing agreement with two Texas universities for a new class of organic arsenicals that show promise in attacking cancer cells without causing dose-limiting side effects.

Founded in January, the privately held company just relocated its headquarters to New Haven, Conn., and secured the rights to its first compound from The University of Texas M.D. Anderson Cancer Center and Texas A&M University.

The product, ZIO-101, is expected to move into Phase I studies in blood cancers and lymphomas in the first quarter of 2005, and then into solid cancers later in the second quarter. Once researchers determine the maximum tolerated dose, Ziopharm will move the initial product into Phase II studies in both hematologic and solid cancers.

The goal is to expand arsenic beyond its use as a therapy for acute promyelocytic leukemia patients.

"Arsenic is actually a very, very effective molecule in treating a variety of cancers," said Jonathan Lewis, CEO of Ziopharm. "The big issue with it, though, is that it's toxic, and we just really haven't learned how to use it."

Professor Ralph Zingaro, of Texas A&M, conducted the original synthesis of the new class of arsenicals in which an arsenic atom was complexed to organic molecules. That makes ZIO-101 safer than inorganic arsenics such as arsenic trioxide, Lewis said. Animal studies have shown that physicians should have the ability to dose five- to 10-fold more ZIO-101 than arsenic trioxide without damaging the heart. ZIO-101 also has a better safety profile than inorganic arsenics when looking at other dose-limiting toxicities, such as damage to the liver, bone marrow and skin.

If oncologists can increase the dose of arsenic with ZIO-101, the compound might offer an advantage for killing cancer cells by causing cell-cycle arrest and cell death rather than cell differentiation.

With about 12 employees, Ziopharm was formed by Lewis and some unnamed seed investors to in-license cancer therapies that have the potential for expedited approval and broad use. The company plans to broaden its pipeline beyond organic arsenics. Lewis expects Ziopharm to do another financing round in the fourth quarter, around the same time the company in-licenses its second product, which will be called ZIO-201.

"We don't want to go into details right now as to what it is or where it is going to come from," Lewis told BioWorld Today. "But we have several leads, and we're negotiating."

The company has a goal to have three in-licensed products in the clinic and one in the preclinical setting in 2005. While it plans to partner its products overseas, Ziopharm intends to carry them through development and onto the U.S. market, building its own sales force.

"In general, in the world of oncology, we do not believe you need a large sales force at all," Lewis said. "You've got to have the right way of doing it."

He believes the expertise at the company will provide Ziopharm the wherewithal to be successful. Lewis, himself, is a surgeon and molecular biologist who has 15 years of combined experience at Yale-New Haven Medical Center, Memorial Sloan-Kettering Cancer Center and New York-based Antigenics Inc.

Earlier this week, the company announced the formation of its medical advisory board and its board of directors. The nine members joining the medical advisory board are cancer experts, four of whom are past presidents of the American Society of Clinical Oncology or the American Association of Cancer Research. They are James Armitage, Joseph Bertino, George Demetri, Lawrence Einhorn, Robert Peter Gale, Alan Houghton, Alberto Pappo, David Spriggs and Mark Thornton. In addition to Lewis, who will serve as chair, Ziopharm's board will consist of its president and chief operating officer Richard Bagley, former U.S. Senator Wyche Fowler, and Murray Brennan, James Cannon, Gary Fragin, David Tanen and Michael Weiser.

Lewis said the company is in the process of hiring several people. As part of its strategy moving forward, Ziopharm will contract out some of its research, development and manufacturing work.

"We will go to the best experts in the world in each area," Lewis said, "and we believe that we will be far more cost-efficient and we will get it done by the best people."

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