GTx, Inc. (Nasdaq: GTXI) announced today that in a Phase IIb clinical study of enobosarm, there was significantly improved physical function after 16 weeks of treatment, compared to placebo, in both hypogonadal (low testosterone) and eugonadal (normal testosterone) subjects. The study met its primary endpoint of absolute change in total lean body mass (muscle) compared to placebo after 16 weeks of treatment. The data were reported by Adrian Dobs, M.D., M.H.S., professor of medicine and oncology, and Vice-Chair of the Department of Medicine, Faculty Development at The Johns Hopkins University Medical School, during the 2012 Annual Meeting of The Endocrine Society (ENDO 2012). Enobosarm is currently being evaluated in two pivotal Phase III clinical trials. In the presentation, “Prevalence and Impact of Hypogonadism in Cancer Patients with Muscle Wasting in a Phase IIb Enobosarm Trial,” Dr. Dobs reported that enobosarm, a selective androgen receptor modulator (SARM), may play an important role in the management of cancer-related muscle wasting. When correlating baseline testosterone levels with weight loss, hypogonadal male subjects in GTx’s Phase IIb clinical study of enobosarm demonstrated greater weight loss than eugonadal males. Dr. Dobs noted that baseline physical function (stair climb power) of subjects in the study was higher among eugonadal versus hypogonadal males. In the Phase IIb clinical study of enobosarm, 159 subjects were randomized to enobosarm or placebo for 16 weeks. The subjects were males greater than 45 years of age and postmenopausal females who had non-small cell lung cancer, colorectal cancer, non-Hodgkin’s lymphoma, chronic lymphocytic leukemia or breast cancer and experienced a 2% or greater weight loss during the 6 months prior to the study’s initiation. Of the 103 male subjects in the study, baseline testosterone levels were available for 93 men. 60% of the males were found to be hypogonadal at randomization, and the distribution of hypogonadism was similar across all forms of the cancers.
Trial DesignEnobosarm is a SARM which binds to the androgen receptor and is designed to deliver the beneficial effects of androgens while minimizing unwanted clinical side effects. GTx conducted its Phase IIb clinical study of enobosarm in 159 cancer patients (average age of 66 years) in 35 sites in the United States and Argentina. Participants were randomized to receive placebo, 1 mg or 3 mg oral tablet of enobosarm once daily for 16 weeks. Average reported weight loss prior to entry among all subjects was 8.8%, and subjects were allowed to have standard chemotherapy during the trial. The drop-out rate during the trial was 33%. GTx is conducting two pivotal Phase III clinical trials in the United States, Europe and South America, evaluating a once daily 3 mg dose of enobosarm for the prevention and treatment of muscle wasting in patients with non-small cell lung cancer. More information about these two clinical studies can be found at www.gtxclinicaltrials.com. About GTx GTx, Inc., headquartered in Memphis, Tenn., is a biopharmaceutical company dedicated to the discovery, development and commercialization of small molecules that selectively target hormone pathways for the treatment of cancer, cancer supportive care and other serious medical conditions. Forward-Looking Information is Subject to Risk and Uncertainty This press release contains forward-looking statements based upon GTx’s current expectations. Forward-looking statements involve risks and uncertainties, and include, but are not limited to, statements relating to its clinical trials. GTx’s actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, the risks (i) that GTx will not be able to commercialize its product candidates if clinical trials do not demonstrate safety and efficacy in humans; (ii) that GTx may not be able to obtain required regulatory approvals to commercialize its product candidates in a timely manner or at all; (iii) that clinical trials being conducted by GTx may not be completed on schedule, or at all, or may otherwise be suspended or terminated; or (iv) that GTx could utilize its available cash resources sooner than it currently expects and may be unable to raise capital when needed, which would force GTx to delay, reduce or eliminate its product candidate development programs or commercialization efforts. You should not place undue reliance on these forward-looking statements, which apply only as of the date of this press release. GTx’s annual report on Form 10-Q filed with the Securities and Exchange Commission on May 10, 2012 contains under the heading, “Risk Factors,” a more comprehensive description of these and other risks to which GTx is subject. GTx expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in its expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based.