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Sep 15, 2004

ZIOPHARM Licenses A New Class Of Arsenic Drugs From M. D. Anderson Cancer Center Smart New Chemistry for Cancer Treatment

ZIOPHARM Licenses A New Class Of Arsenic Drugs From M. D. Anderson Cancer Center Smart New Chemistry for Cancer Treatment

NEW HAVEN, CT SEPT 15 - Privately-held ZIOPHARM, Inc. announced it completed a worldwide licensing agreement with The University of Texas M. D. Anderson Cancer Center and Texas A&M University for a new class of organic arsenicals, and said it plans to develop them for a wide range of cancers. The original synthesis of this class of organic arsenicals was conducted by Ralph A. Zingaro, PhD, Professor of Chemistry at Texas A&M University.

The development program, announced by ZIOPHARM Chief Executive Officer Jonathan J. Lewis, MD, PhD, opens the potential of expanding the modern-day version of arsenic, currently used to treat APL (acute promyelocytic leukemia), to the much more common hematologic and solid cancers.

Phase I studies in adults with blood cancers and lymphomas are expected to begin in 1Q of 2005. Parallel Phase I studies in solid cancers are planned for late 2Q-2005. Once the maximum tolerated dose is determined, Phase-II studies are planned in both hematologic and solid cancers.

In the new drug class, an arsenic atom is complexed to organic molecules. As a result, ZIO-101 is substantially safer than inorganic arsenics like arsenic trioxide. For example, animal studies show the ability to give 5- to 10-fold more ZIO-101 than arsenic trioxide without damaging the heart. Other dose-limiting toxicities of inorganic arsenics, including damage to the liver, bone marrow and skin, are much less prominent with ZIO-101.

A large dose increase, possible with ZIO-101, is expected to allow cancer physicians to take advantage of arsenic's ability to kill cancer cells specifically by causing cell cycle arrest and cell death rather than cell differentiation, as is the case with inorganic arsenic.

"ZIOPHARM is excited to be working on a potentially widely effective anti-cancer drug with a favorable safety profile," said Dr. Lewis, a surgeon and molecular biologist who has 15 years of combined experience at Yale-New Haven Medical Center, Memorial Sloan-Kettering Cancer Center and Antigenics, Inc. "ZIOPHARM's senior management team has a unique ability to move this molecule from IND through clinical trials in an efficient, focused fashion to ensure value creation for cancer patients, oncologists and our investors."

Srdan Verstovsek, MD, PhD, a faculty member at M. D. Anderson Cancer Center, also commented on the potential benefits of ZIO-101: "The key obstacle to using arsenic to treat solid cancers is the inability to give enough to kill cancer cells without causing serious side effects. Preclinical data on ZIO-101 suggests an ability to increase the dose 5- to 10-fold over traditional arsenic while increasing efficacy with minimal toxicity. Prospects for clinical trials in solid cancers are very promising."

About ZIOPHARM, Inc.

ZIOPHARM, Inc. is a privately held company. It was founded in January 2004 to develop and commercialize a diverse, risk-sensitive portfolio of in-licensed cancer therapies. All products are focused on addressing unmet medical needs, with the potential for expedited approval and broad usage.

ZIOPHARM's first in-licensed product, ZIO-101, is a small molecule from The University of Texas M. D. Anderson Cancer Center and is planned for Phase I study initiating late in 1Q-2005. The company anticipates licensing of ZIO-201 by 4Q-2004.

ZIOPHARM is actively evaluating and negotiating for additional product candidates with the objective of a balanced portfolio of at least three clinical-stage compounds and at least one late preclinical candidate by the end of 2005. Each product candidate undergoes a tightly managed evaluation process leveraging the company's management team's combined 100+ years of oncology experience in clinical development, regulatory strategy, business development and product commercialization.

Both ZIOPHARM's structure and mission set it apart from other cancer drug companies. The company's rigorously disciplined approach to screening product candidates is designed to accelerate clinical programs while reducing the expense and risk typically incurred in researching, developing and launching new cancer products. With an intimate understanding of the regulatory approval process, the company expects to break new ground as regards to speed and efficiency as it brings new cancer compounds to market.