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Positive Phase 2 Data Announced for Toremifene, Treatment for the Prevention of Prostate Cancer

MEMPHIS, Tenn., June 2 /PRNewswire-FirstCall/ -- GTx, Inc. (Nasdaq: GTXI - News), a biopharmaceutical company dedicated to the discovery, development and commercialization of therapeutics for serious men's health conditions announced today positive Phase IIb results for its lead product ACAPODENE(TM) (toremifene citrate) tablets. The data demonstrated that ACAPODENE(TM) was well tolerated and reduced the incidence of prostate cancer in men who are at high risk for prostate cancer. Patients were classified as being at high risk because they had biopsy proven precancerous lesions of the prostate called high grade prostatic intraepithelial neoplasia (PIN). GTx plans to initiate a Phase III clinical trial in 2004 following discussions with the Food and Drug Administration (FDA). ACAPODENE(TM), GTx's lead product candidate, is a nonsteroidal selective estrogen receptor modulator (SERM).

Study Results
The ACAPODENE(TM) Phase IIb study was double-blind, placebo-controlled, one year clinical trial in 514 men at high risk for prostate cancer and high grade PIN. The primary endpoint was the incidence of prostate cancer. This is the largest prospective study of the natural history of patients with high grade PIN. This well controlled study confirmed that men who have high grade PIN are at high risk as 31% of placebo patients were diagnosed with prostate cancer by 1 year. The intent-to-treat analysis, defined as any patient who had at least one on-study biopsy, showed that ACAPODENE(TM) 20 mg had a 20% reduction in prostate cancer incidence. The reduction of prostate cancer incidence improved in men who received ACAPODENE(TM) 20mg for one year with a 46% reduction in this high risk population compared to the placebo group, which is consistent with the interim analysis GTx conducted last year. For men who developed prostate cancer, those treated with ACAPODENE(TM) had similar tumor grades to those of placebo patients. ACAPODENE(TM) was well tolerated as the number of adverse events were similar between those patients receiving ACAPODENE(TM) compared to placebo.

"This study confirms that men who have high grade PIN are indeed high-risk patients for prostate cancer with 31% of the men receiving placebo being diagnosed with cancer within one year. Developing a successful treatment option to prevent prostate cancer in high risk patients will have a positive clinical impact," said Mitchell Steiner MD, FACS, Vice-Chairman and CEO of GTx. "ACAPODENE 20mg showed a significant reduction in prostate cancer incidence in men who had taken ACAPODENE for 12 months compared to placebo. ACAPODENE at all doses tested was well tolerated. While we need to discuss the results of this study with the FDA and continue analyzing the data, we are excited about these results and plan to begin the Phase III trial later this year," said Dr. Steiner.

About the Study
The Phase IIb clinical study was a 4-arm, double blind, placebo controlled, one year treatment study at 64 clinical sites in the United States involving 514 patients. The four arms included in this study were 20mg, 40mg and 60mg of ACAPODENE(TM) and placebo given orally once a day. There were approximately 125 patients per arm. The primary entry criterion for the study was men with biopsy proven and confirmed high grade PIN. All patients were rebiopsied at 6 and 12 months from randomization.

About PIN
High grade PIN has been established as a premalignant lesion that has strong potential to progress to prostate cancer. In the United States, approximately 1,300,000 prostate biopsies are performed annually to detect 230,000 new cases of prostate cancer. There are approximately 115,000 new cases of high grade PIN diagnosed each year, representing an estimated 9% of prostate biopsies. Currently, patients diagnosed with high grade PIN have to be followed closely by their urologist and are subjected to repeat prostate biopsies.

ACAPODENE(TM) is a nonsteroidal SERM, a small molecule that binds and selectively modulates the estrogen receptor. SERMs have been shown to block estrogen receptors in the prostate. GTx has licensed the right to develop, market and distribute toremifene citrate, the active ingredient of ACAPODENE(TM) tablets, worldwide in the field of prevention and treatment of prostate cancer from Orion Corporation, Finland.

About Prostate Cancer
In the United States, there is estimated to be over 230,000 new prostate cancer cases and 30,000 prostate cancer deaths this year. This makes prostate cancer the most commonly diagnosed cancer and the second leading cause of cancer-related deaths in men in the United States.

Source: GTx, Inc.

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