GTx Provides Corporate Update and Reports Third Quarter 2013 Financial Results

  GTx Provides Corporate Update and Reports Third Quarter 2013 Financial
  Results

  Further POWER Trial Analyses Add Support for Potential Regulatory Path for
                                  Enobosarm

Business Wire

MEMPHIS, Tenn. -- November 12, 2013

GTx, Inc. (Nasdaq: GTXI) today provided a Company update and reported
financial results for the third quarter of 2013. The Company summarized
additional analyses it has undertaken of the POWER1 and POWER2 clinical trials
evaluating enobosarm 3 mg for the prevention and treatment of muscle wasting
in patients with advanced non-small cell lung cancer (NSCLC). Using
longitudinal continuous variable analyses, which was the statistical analysis
pre-specified by the Company for submission of its data to the European
regulatory authorities, enobosarm 3 mg demonstrated significant improvement in
stair climb power, compared to placebo, in the POWER1 (platinum plus taxane)
trial. While the same analyses showed unequivocal improvements in lean body
mass, compared to placebo, in both POWER trials, enobosarm 3 mg did not show
statistically significant improvements in stair climb power in the POWER2
(platinum + non-taxane) trial. The Company is continuing to consider the
reasons that maintaining or improving lean body mass in the POWER2 trial did
not translate into similar improvements in stair climb power, compared to
placebo, but it believes it may be associated with the side effects of the
non-taxane chemotherapy received by the patients in the study, including
anemia in many of the patients.

“Since August, when we reported topline results from our two Phase 3 clinical
trials of enobosarm to prevent and treat muscle wasting in non-small cell lung
cancer patients, we have learned a good deal more from the POWER results and
remain encouraged by enobosarm’s potential,” said Mitchell S. Steiner, MD, CEO
of GTx. “We look forward to upcoming regulatory discussions and to potentially
clarifying a path to market approval for this important new drug product
candidate.”

The Company plans to meet with representatives from select member countries of
the European Medicines Agency (EMA) and with the United States Food and Drug
Administration (FDA) to review and discuss the results of the clinical trials
as well as a feasible regulatory path forward.

Clinical updates

Enobosarm (GTx-024) 3 mg, an oral selective androgen receptor modulator, being
developed for the prevention and treatment of muscle wasting in patients with
advanced non-small cell lung cancer: GTx recently announced that the POWER1
(platinum plus taxane) and POWER2 (platinum plus non-taxane) Phase 3 clinical
trials evaluating enobosarm 3 mg for the prevention and treatment of muscle
wasting in patients with advanced non-small cell lung cancer (NSCLC) failed to
meet the co-primary endpoints of lean body mass and physical function that
were assessed statistically using responder analyses. However, data from the
studies have demonstrated enobosarm’s consistent effect on maintaining or
improving lean body mass, compared to placebo. Pursuant to responder analyses,
a larger proportion of patients receiving enobosarm maintained or increased
lean body mass at both Day 84 and Day 147 in both clinical trials, compared to
placebo. Using longitudinal continuous variable statistical analyses, which is
the analysis the Company pre-specified in its statistical analysis plan for
data to be submitted to the European regulatory authorities, enobosarm 3 mg
had a significant effect on lean body mass through Day 84 and Day 147 in both
clinical trials, compared to placebo (POWER1: p=0.0002 and <0.0001,
respectively, and POWER2: p=0.0227 and 0.0036, respectively). Using this
analysis, the POWER1 (platinum plus taxane) clinical study met the
pre-specified primary endpoint of physical function for stair climb power
through Day 84 (p=0.0147) and the secondary endpoint of stair climb power
through Day 147 (p=0.0492).

Survival is being assessed as another safety endpoint to determine that
enobosarm treatment is not adversely affecting survival. As specified in the
Company’s statistical analysis plan, survival will be assessed after 450 of
the approximately 650 patients in the two studies have died, which currently
is expected to have occurred by March or April of 2014. To date, the Company
has seen no adverse effect on survival from enobosarm treatment from pooled
survival data. However, post-hoc landmark survival analyses of lean body mass
responders at Day 84 suggest improved survival in patients who maintained or
increased lean body mass in both clinical studies, regardless of treatment,
potentially reinforcing the importance of building lean body mass in this
patient population.

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

The POWER trials were designed to study the effectiveness and safety of a 3 mg
dose of enobosarm 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 effect of enobosarm versus placebo
on the maintenance or improvement of total lean body mass (muscle) was
assessed by Dual-energy X-ray Absorptiometry and patients’ improvement in
physical function was measured by stair climb 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.

Enobosarm 9 mg, being studied for the treatment of advanced breast cancer: GTx
has initiated a Phase 2, open label clinical study evaluating enobosarm 9 mg
for the treatment of estrogen receptor (ER) positive metastatic breast cancer
in women who have previously responded to hormonal therapy for the treatment
of their advanced breast cancer.This proof of concept study is enrolling
approximately twenty (20) postmenopausal women with ER positive metastatic
breast cancer who have previously responded to hormone therapy at
approximately six clinical sites in the United States. The women in the study
are receiving an oral 9 mg dose of enobosarm once a day until they show
evidence of clinical progression or have completed 336 days of treatment with
enobosarm. The primary endpoint of the study is clinical benefit response,
which will be assessed at six months, and is defined as either those women
receiving treatment who have demonstrated a complete response (disappearance
of all targeted lesions), a partial response (at least a 30 percent decrease
in the sum of the diameters of the targeted lesions) or stable disease (no
disease progression from baseline).

In preclinical and clinical studies, androgens suppress breast cancer growth.
Prior studies have shown that women with metastatic breast cancer who have
been previously treated with tamoxifen and whose cancer has progressed have
responded to non-selective androgens, with overall response rates ranging from
20 to 60 percent. Although these non-selective androgens have been used to
treat breast cancer, the unwanted virilizing side effects, including facial
and body hair, enlargement of voice box, acne, and edema have limited their
widespread clinical use. GTx believes that a selective androgen receptor
modulator, like enobosarm, by targeting the androgen receptor in metastatic
breast cancer, has the potential to provide clinical benefit to women with
advanced breast cancer by treating their disease while minimizing the unwanted
masculinizing side-effects associated with steroidal androgens. Furthermore,
unlike steroidal androgens, enobosarm cannot be converted to an estrogen that
could be detrimental in breast cancer.

GTx-758 (Capesaris^®), an oral nonsteroidal selective estrogen receptor alpha
agonist, being studied for secondary hormonal therapy in men with
castration-resistant prostate cancer and, potentially, as a secondary hormonal
treatment for advanced prostate cancer used in combination with ADT: GTx is
enrolling an open-label, Phase 2 clinical study of GTx-758 to treat men with
metastatic, castration-resistant prostate cancer (CRPC). GTx-758 has
previously demonstrated the ability to increase the production of a protein
called sex hormone binding globulin (SHBG) that binds testosterone and thereby
reduces free testosterone. The Phase 2 study is evaluating the safety and
effectiveness of two doses of GTx-758. The primary endpoint of the study is
the proportion of patients with a ≥ 50% decline from baseline in serum PSA by
Day 90. Other key endpoints include SHBG and total and free testosterone
levels, as well as prostate cancer progression, in the study subjects. In
addition, the clinical study is evaluating the ability of GTx-758 to treat
certain estrogen deficiency side-effects associated with LHRH agonists, such
as hot flashes, bone loss, and insulin resistance.

After reviewing data collected to date from the GTx-758 125 mg dosing arm
indicating the ability of the drug to substantially increase SHBG and lower
free testosterone without any unexpected side-effects occurring, the clinical
trial protocol was amended to eliminate the third dosing arm of 500 mg
originally designed for the study and to increase the number of subjects to be
enrolled in the 125 mg and 250 mg dosing arms to 38 patients per arm.
Enrollment in the 125 mg cohort is continuing with no reported venous
thromboembolic events (VTEs) or other significant adverse events to date.
Assuming there continues to be no unacceptable incidence of VTEs observed in
the patients being dosed at 125 mg of GTx-758, the Company will then determine
whether it will proceed with the dosing of the 250 mg cohort.

Upcoming Milestones

  *Meet with European and U.S. regulatory authorities in the coming months to
    clarify enobosarm regulatory requirements
  *Report survival results from enobosarm POWER trials (survival is being
    assessed as a safety endpoint and is expected mid way through the first
    half of 2014)
  *Complete enrollment this quarter of a Phase 2, open-label clinical study
    to evaluate enobosarm 9 mg for the potential treatment of metastatic
    breast cancer (primary endpoint assessment of clinical response is
    expected in the second quarter of 2014)
  *Complete enrollment this quarter of the expanded 125 mg dosing arm for the
    Phase 2, open-label clinical study to evaluate GTx-758 for the treatment
    of men with metastatic, castration-resistant prostate cancer

Financial highlights for the quarter ended September 30, 2013

The Company reported a net loss for the quarter ended September 30, 2013 of
$8.9 million compared to net income of $5.1 million for the same period in
2012. The net income for the quarter ended September 30, 2012 resulted from
the sale of the Company’s rights and certain assets related to FARESTON^®
(toremifene citrate) 60 mg tablets, approved for the treatment of metastatic
breast cancer in postmenopausal women in the United States, for which the
Company recognized a gain of $18.8 million. For the nine months ended
September 30, 2013, the Company reported a net loss of $34.3 million compared
to a net loss of $16.4 million for the same period of 2012.

Research and development expenses for the quarter ended September 30, 2013
were $6.5 million compared to $9.8 million for the same period of 2012.
General and administrative expenses for the quarter ended September 30, 2013
were $2.5 million compared to $3.0 million for the same period of 2012.

At September 30, 2013, GTx had cash and short-term investments of $21.0
million.

Conference call

There will be a conference call today at 9:00 a.m. Eastern Time. To listen to
the conference call, please dial 800-706-7745 from the United States or Canada
or 617-614-3472 from other international locations. The access code for the
call is 23964715. A playback of the call will be available from approximately
1:00 p.m. Eastern Time today through November 26, 2013 and may be accessed by
dialing 888-286-8010 from the United States or Canada or 617-801-6888 from
other international locations and referencing reservation number 68864622.
Additionally, you may access the live and subsequently archived webcast of the
conference call from the Investor Relations section of the Company’s website
at http://www.gtxinc.com.

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, including
prevention and treatment of cancer-related muscle wasting, 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 (GTx-024) and its clinical trial of
GTx-758 (Capesaris^®). 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 may not be able to obtain required regulatory approvals
to commercialize its product candidates in a timely manner or at all; (ii)
that clinical trials being conducted by GTx may not be completed on schedule,
or at all, or may otherwise be suspended or terminated; or (iii) 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. 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 being filed with the Securities and Exchange Commission later today
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.


GTx, Inc.
Condensed Balance Sheets
(in thousands, except share data)
                                                            
                                                September 30,     December 31,
                                                2013              2012
                                                (unaudited)
ASSETS
Current assets:
Cash and cash equivalents                       $  19,283         $ 48,044
Short-term investments                             1,675            8,045
Prepaid expenses and other current assets         830            726      
Total current assets                               21,788           56,815
Property and equipment, net                        217              507
Intangible and other assets, net                  487            452      
Total assets                                    $  22,492        $ 57,774   
LIABILITIES AND STOCKHOLDERS’ EQUITY
Current liabilities:
Accounts payable                                $  1,253          $ 1,707
Accrued expenses and other current                3,912          7,788    
liabilities
Total current liabilities                          5,165            9,495
Other long-term liabilities                        389              578
Commitments and contingencies
Stockholders’ equity:
Common stock, $0.001 par value:
120,000,000 shares authorized at both
September 30, 2013 and December 31, 2012;          63               63
63,185,389 and 62,818,424 shares issued
and outstanding at September 30, 2013 and
December 31, 2012, respectively
Additional paid-in capital                         464,445          460,887
Accumulated deficit                               (447,570 )      (413,249 )
Total stockholders’ equity                        16,938         47,701   
Total liabilities and stockholders’             $  22,492        $ 57,774   
equity
                                                                  


GTx, Inc.
Condensed Statements of Operations
(in thousands, except share and per share data)
(unaudited)
                                                     
                     Three Months Ended                    Nine Months Ended
                     September 30,                         September 30,
                     2013             2012               2013             2012
                                                                              
Expenses:
Research and
development          $ 6,477            $ 9,764            $ 26,230           $ 28,836
expenses
General and
administrative        2,483            2,999            8,190            7,987      
expenses
Total expenses        8,960            12,763           34,420           36,823     
Loss from              (8,960     )       (12,763    )       (34,420    )       (36,823    )
operations
Other income,         23               (47        )      99               14         
net
Loss from
operations             (8,937     )       (12,810    )       (34,321    )       (36,809    )
before income
taxes
Income tax            -                5,812            -                6,548      
benefit
Net loss from
continuing            (8,937     )      (6,998     )      (34,321    )      (30,261    )
operations
                                                                              
Income from
discontinued
operations             -                  20,214             -                  22,752
before income
taxes
Income tax            -                (8,115     )      -                (8,851     )
expense
Net income
from                  -                12,099           -                13,901     
discontinued
operations
                                                                              
Net income           $ (8,937     )     $ 5,101           $ (34,321    )     $ (16,360    )
(loss)
                                                                              
Net income
(loss) per
share - basic
and diluted:
Net loss from
continuing           $ (0.14      )     $ (0.11      )     $ (0.54      )     $ (0.48      )
operations
Net income
from                  -                0.19             -                0.22       
discontinued
operations
Net income
(loss) per           $ (0.14      )     $ 0.08            $ (0.54      )     $ (0.26      )
share
                                                                              
Weighted
average shares
outstanding:
Basic and             63,179,394       62,815,549       63,013,923       62,806,440 
diluted
                                                                              

Contact:

GTx, Inc.
Marc Hanover, 901-523-9700
President
 
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