GTx Presents Results from Enobosarm POWER Trials for the Prevention and Treatment of Muscle Wasting in Patients with Non-Small

  GTx Presents Results from Enobosarm POWER Trials for the Prevention and
  Treatment of Muscle Wasting in Patients with Non-Small Cell Lung Cancer at
  15th World Conference on Lung Cancer

Enobosarm had a significant effect on Lean Body Mass (LBM) and stair climb
power (SCP) through Day 84 and 147 in POWER1 trial (platinum plus taxane)
using a longitudinal continuous variable analysis

Enobosarm had a significant effect on Lean Body Mass (LBM) through Day 84 and
147 in POWER2 trial (platinum plus non-taxane) using a longitudinal continuous
variable analysis

Patients who had a ≥ 1kg increase in LBM demonstrated a significant advantage
in Stair Climb Power (SCP)

Findings from both trials show that maintenance or improvement in LBM improves
survival by landmark analysis

Business Wire

MEMPHIS, Tenn. -- October 24, 2013

GTx, Inc. (Nasdaq: GTXI) announced today that a poster presentation, entitled
Results from two Phase 3 randomized trials of enobosarm, a selective androgen
receptor modulator (SARM), for the prevention and treatment of muscle wasting
in patients with non-small cell lung cancer (NSCLC) receiving chemotherapy,
will be given by Jeffrey Crawford, M.D., Chief, Division of Medical Oncology
at Duke University School of Medicine, and principal investigator for the
POWER1 (platinum plus taxane) and POWER2 (platinum plus non-taxane) trials,
which were chemotherapy add-on placebo controlled studies.

Top line results from the POWER trials showed that enobosarm 3 mg once daily
had a significant effect on LBM through Day 84 and 147 in both trials,
compared to placebo (taxane:p=0.0003 and <0.0001, respectively; non-taxane:
p=0.0227 and 0.0036, respectively, using a continuous variable analyses). By
the responder analysis, a larger proportion of patients receiving enobosarm
maintained or increased LBM at Day 84 and 147 in both clinical trials
(taxane:p=0.036 and 0.026;non-taxane:p=0.113 and 0.013), as compared to
placebo. Additionally, the POWER1 clinical study met the pre-specified
endpoints for the European regulatory agency, using the longitudinal
continuous variable analysis. As compared with placebo, enobosarm treated
patients in POWER1 achieved the primary endpoint in SCP through Day 84 (p=
0.0185) and the secondary endpoint of SCP through Day 147 (p=0.0486).

In a post-hoc analysis, regardless of treatment, patients with a ≥ 1kg
increase in LBM were more likely to demonstrate at least a 10% increase in SCP
compared to patients who did not have a ≥ 1kg increase in LBM (taxane: 43.7%
vs 29.3%, p=0.0250; and non-taxane: 40.5% vs 26.5%, p=0.0321). Importantly, a
larger proportion of enobosarm treated patients, with 1kg or greater increases
in LBM, demonstrated at least a 10% increase in SCP (taxane: p=0.0698, ≥1kg
46.4%, <1kg 29.6%; and non-taxane: p=0.0335, ≥1kg 39.6%, <1kg 20.4%), while
this same trend was not observed in placebo treated patients (taxane:
p=0.3149, ≥1kg 38.7%, <1kg 29.0%; and non-taxane: p=0.2852, ≥1kg 41.5%, <1kg
31.3%). This observation suggests that SCP improvements, in both trials, may
be related to enobosarm increases in LBM.

Post-hoc landmark survival analyses at Day 84 suggest improved survival in
patients who maintained or increased LBM in both clinical trials, regardless
of treatment. As required by the study protocols, the final survival analysis
will be completed after 450 deaths have been observed among patients in the
studies.

Enobosarm was very well tolerated in both clinical trials. Although only minor
differences in adverse events were observed between the groups treated with
enobosarm 3 mg and placebo in the POWER1 and POWER2 trials, there were notable
differences in the adverse event profile between studies with anemia and other
hematologic toxicities more prevalent in the POWER2 (platinum plus non-taxane)
clinical trial.

Abstract ID: 2266: Results from two phase 3 randomized trials of enobosarm,
selective androgen receptor modulator (SARM), for the prevention and treatment
of muscle wasting in NSCLC. NSCLC Novel Therapies Poster Session 3
(P3.11-026), Wednesday, October 30, 2013, from 9:30 AM -2:30 PM AEDT in the
Exhibit Hall, Ground Level at the 15^th World Conference on Lung Cancer being
held by the International Association for the Study of Lung Cancer in Sydney,
Australia.

About The POWER Trials

A 3 mg dose of enobosarm was studied in two Phase 3 clinical trials to prevent
and treat muscle wasting in patients with NSCLC. In each of these placebo
controlled, double blind, add-on clinical trials, approximately 325 patients
with stage III or IV NSCLC were randomized to oral daily doses of placebo or
enobosarm 3 mg at the time they began first-line standard platinum doublet
chemotherapy. The POWER trials were designed to assess the effect of enobosarm
versus placebo on maintenance or improvement of total LBM (muscle) assessed by
Dual-energy X-ray Absorptiometry (DXA) and improvement in physical function
measured by SCP. Durability of enobosarm treatment was assessed at five
months. Secondary endpoints included an assessment of whether
enobosarm-treated patients had an improved quality of life and reduced
healthcare resource utilization compared to placebo. Overall survival is being
assessed as an additional safety endpoint. GTx announced early this year that
the FDA has designated enobosarm for the prevention and treatment of muscle
wasting in patients with NSCLC as a Fast Track development program.

About Cancer-Induced Muscle Wasting

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 currently no drugs approved for the prevention and treatment
of muscle wasting in patients with cancer.

About Non-Small Cell Lung Cancer

The American Cancer Society estimates about 228,190 new cases of lung cancer
will be diagnosed in the United States in 2013, and approximately 85 to 90
percent of these are non-small cell lung cancer. Approximately 159,480
Americans are expected to die from lung cancer this year.

About GTx

GTx, Inc., headquartered in Memphis, Tenn., is a biopharmaceutical company
dedicated to the discovery, development, and commercialization of small
molecules 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
GTx's clinical trials for enobosarm (also known as Ostarine^® or GTx-024).
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 quarterly report on Form 10-Q filed
with the Securities and Exchange Commission on July 22, 2013 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.

Contact:

GTx, Inc.
Marc Hanover, 901-507-6915
President and Chief Operating Officer
mhanover@gtxinc.com
 
Press spacebar to pause and continue. Press esc to stop.