Oral Form of Anti-cancer Agent Shows Promise in Treating Sarcoma

The Institute for Drug Development, a division of The Cancer Therapy & Research Center (CTRC) at The University of Texas Health Science Center at San Antonio, today announced results of a Phase I dose-ranging study of oral deforolimus, a drug that was effective as an intravenous therapy in previous studies.

These data provide the rationale for selection of the oral dose and administration schedule in the ongoing Phase III SUCCEED trial of deforolimus for the treatment of advanced metastatic sarcomas to help improve patients’ quality of life.

The phase I study will be presented in full at the annual meeting of the American Society of Clinical Oncology (ASCO), to be held in Chicago from May 31 to June 3.

The data from this trial of 147 patients with solid tumors resistant to therapy will be presented in an oral session by clinical investigator and assistant professor Monica M. Mita, M.D., of the CTRC at the UT Health Science Center.

The presentation, titled “Deforolimus trial 106 – A Phase 1 trial evaluating 7 regimens of oral deforolimus (AP23573, MK-8669),” will be given by Dr. Mita at 1:15 pm (CDT) on Saturday, May 31, 2008, during the Clinical Science Symposium on PI-3 Kinase/mTOR Directed Agents.

“These results present us with important information about deforolimus' oral formulation, providing an alternative route of administration that has the potential to offer greater patient convenience and treatment flexibility than the intravenous form,” Dr. Mita said. “I also am very pleased to see that the anti-tumor activity and safety profile appear consistent with our previous clinical experience with the intravenous formulation of deforolimus.”

Oral deforolimus, an mTOR inhibitor that regulates normal cell growth and slows or stops the growth of cancer cells, was found to have a safety and anti-tumor activity consistent with the intravenous form.

This non-randomized, open-label, dose-escalation Phase I study was designed to measure tolerability and anti-tumor activity of oral deforolimus administered in seven different dosing regimens. The study found the dose of 40 mg QDx5 each week was an active and well-tolerated regimen. The daily and monthly exposure to oral deforolimus varied in each group, along with the duration of weekly “drug holidays.”

As in all previous clinical trials with the intravenous formulation, the only dose-limiting toxicity for all oral regimens was apthous ulcer-like mouth sores, which were reversible by dose reduction or symptomatic therapy. The selected optimal dose maximized exposure to oral deforolimus while maintaining a safety profile that was generally well tolerated by patients.

About the CTRC:

The Cancer Therapy & Research Center (CTRC) at The University of Texas Health Science Center at San Antonio is one of the nation’s leading academic research and treatment centers, serving more than 4.4 million people in the high-growth corridor of Central and South Texas including Austin, San Antonio, Laredo and the Rio Grande Valley. CTRC is one of a few elite cancer centers in the country to be named a National Cancer Institute (NCI) Designated Cancer Center, and is one of only three in Texas. CTRC handles more than 120,000 patient visits each year and is a world leader in developing new drugs to treat cancer. The CTRC Institute for Drug Development (IDD) is internationally recognized for conducting the largest oncology Phase I clinical drug trials program in the world, and participated in the clinical and/or preclinical development of many of the cancer drugs approved by the U.S. Food & Drug Administration.