GTx, Inc. GTXI today announced results of its two Phase 3
enobosarm clinical trials, the POWER trials, in patients with non-small cell
lung cancer (NSCLC) receiving chemotherapy. The Company announced that the
clinical trials failed to meet the overall criteria for the co-primary
responder endpoints of lean body mass and physical function as agreed upon
with the United States Food and Drug Administration (FDA); the responder
endpoints showed mixed results (for POWER1 and POWER2, p values at Day 84
for LBM were 0.036 and 0.113, respectively; p values at Day 84 for SCP were
0.315 and 0.289, respectively).
Initial exploratory quantitative (continuous variable) analysis demonstrated
that enobosarm had a consistent effect on LBM relative to placebo in both
studies at all assessment times (p values were 0.0003 and 0.0227 at Day 84
for POWER1 and POWER2, respectively). Corresponding analyses for SCP were
inconsistent between trials (p values were 0.0336 and 0.7923, respectively).
Missing data were well balanced between the arms in both trials for both
endpoints.
Across both clinical trials, enobosarm was generally well tolerated, with
the occurrence of serious adverse events and overall incidence of adverse
events similar across placebo and treatment groups. In POWER1, the four most
common adverse events reported (in decreasing order of incidence) were
nausea, alopecia, anemia and vomiting. In POWER2, the four most common
adverse events reported were anemia, nausea, neutropenia and vomiting. In
the safety analysis of survival, there was no evidence of a difference
between patients treated with enobosarm and placebo in either clinical
trial.
"While we are disappointed that both studies did not meet the pre-specified
responder analyses, we are encouraged by the unambiguous effect of enobosarm
on muscle and we are confident that it will translate to clinical benefit
and potentially increase survival in patients with non-small cell lung
cancer," said Mitchell Steiner, M.D., CEO of GTx. "We look forward to
sharing our clinical data from these and previous trials with FDA and
European authorities to discuss the path forward. I would like to personally
thank all the employees at GTx for their tremendous effort in conducting two
high quality Phase 3 clinical studies and the principal investigators and
their staff at over 80 clinical sites in 8 countries for their help
recruiting and managing these studies. Most of all, I want to thank the
patients with non-small cell lung cancer who participated in the POWER1 and
POWER2 clinical trials in order to make it possible for future patients to
potentially have access to important therapies."
Published observational data suggest that LBM is related to survival
outcome. This observational finding has been replicated based on exploratory
analysis of current survival data from the POWER studies using landmark
analysis and time-dependent covariate Cox regression modeling that includes
LBM response and arm as covariates. The effect size and direction were
similar in both trials.
GTx plans to initiate discussions with both the FDA and European regulatory
authorities to determine the path forward.
"Muscle wasting in patients with non-small cell lung cancer is devastating
and unfortunately it affects hundreds of thousands of patients worldwide,"
said Jeffrey Crawford, M.D., Chief, Division of Medical Oncology at Duke
University School of Medicine, and principal investigator for the POWER1 and
POWER2 trials. "While some of the pre-specified primary endpoints were not
met, I am encouraged by the substantial and consistent effect of enobosarm
on muscle in these patients with lung cancer receiving chemotherapy."
"Data from the POWER trials provide compelling evidence that enobosarm
maintains or increases muscle," said Carla Prado, Ph.D., Assistant
Professor, Nutrition, Food and Exercise Sciences at Florida State
University. "Loss of muscle, independent of weight loss, is a common and
often occult feature of cancer, and is acknowledged as a remarkable and
powerful prognostic indicator of shorter survival."
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 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 response rates of
enobosarm versus placebo for the co-primary endpoints at 3 months of
treatment on maintenance or improvement of total lean body mass (muscle)
assessed by Dual-energy X-ray Absorptiometry (DXA) and improvement in
physical function measured by the stair climb test (power). 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.
Conference Call Details
The GTx management team will host a conference call and webcast to discuss
this announcement. The event will be held today at 9:00 a.m. Eastern Time
(ET). The live event will be available from the GTx website at
http://www.gtxinc.com under the Investors section, or by calling (866)
700-5192 (domestic) or (617) 213-8833 (international). The access code is
35408233. A replay of the discussion will be available beginning at
approximately 11:00 a.m. ET today from the GTx website or by calling (888)
286-8010 (domestic) or (617) 801-6888 (international), using access code
18733881. The telephone and website replays will be available until 11:59
p.m. ET on September 2, 2013.
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(R) 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.
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