Results from GTx's Phase 2 clinical trial of enobosarm published online

GTx, Inc. (Nasdaq: GTXI) today announced that the journal, The Lancet Oncology, has published online ahead of its April print edition (www.thelancet.com/journals/lanonc/onlinefirst) the results from GTx's randomized, double-blind, placebo-controlled Phase 2 clinical trial of enobosarm to assess its effects on muscle wasting and physical function in patients with cancer. Enobosarm (GTx-024) is a selective androgen receptor modulator (SARM), a new class of non-steroidal, tissue-specific anabolic agents that has the potential to increase muscle mass and improve physical function without the unwanted side effects on the prostate, skin or hair that are commonly associated with testosterone or non-selective, synthetic anabolic steroids. The lead author was Dr. Adrian S. Dobs, Professor of Medicine at the Division of Endocrinology and Metabolism, Johns Hopkins University School of Medicine in Baltimore, Maryland.

Cancer-induced muscle wasting begins early in the disease process, resulting in decreased physical function and other detrimental consequences, such as fatigue and weight loss, which can contribute to disability, reduced quality of life and shorter overall survival, compared with patients without muscle loss. There are no drugs approved for the prevention and treatment of muscle wasting in patients with cancer. The Phase 2 trial, which enrolled patients with non-small cell lung cancer (NSCLC), colorectal cancer (CRC), non-Hodgkin lymphoma (NHL), chronic lymphocytic leukemia (CLL) or breast cancer, demonstrated significant increases in lean body mass (primary endpoint) and physical function (one of the secondary endpoints) in patients treated with enobosarm 1 mg or 3 mg oral daily doses. Patients receiving placebo during the same period did not show significant increases in total lean body mass or in physical function.

The lead author, Dr. Adrian Dobs, an investigator in the Phase 2 trial, stated, "Muscle wasting is a common and devastating cancer-related symptom for which there are no effective therapies today. This is the first well controlled clinical trial to show that a selective anabolic agent, enobosarm, has the ability to not only increase muscle mass, but also improve physical function in a clinically relevant stair climb activity across a broad number of cancer types. After seeing the results of this Phase 2 clinical trial, I am looking forward to the results from the two Phase 3 clinical studies, POWER 1 and POWER 2, to confirm enobosarm's ability to prevent and treat muscle wasting in patients with advanced non-small cell lung cancer."