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GTx Provides Corporate Update and Reports Fourth Quarter and Year-End 2016 Financial Results

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GTx, Inc. (NASDAQ:GTXI) today reported financial results for the fourth
quarter and year ended December 31, 2016, and highlighted recent
accomplishments and upcoming milestones. The Company has two ongoing
clinical trials of enobosarm (GTx-024) in women with advanced breast
cancer and one ongoing trial with enobosarm as a potential treatment for
stress urinary incontinence (SUI) in postmenopausal women. The Company
has identified lead compounds from its selective androgen receptor
degrader (SARD) portfolio and has preclinical studies underway that are
required prior to initiating a clinical trial in men with
castration-resistant prostate cancer (CRPC) during the second half of
2017.

"In 2016, we broadened our clinical development efforts for enobosarm
beyond breast cancer with the initiation of our clinical trial in SUI in
postmenopausal women. Early results in this study are very promising,
and we have added additional clinical sites and expect to receive
top-line results from this trial in the third quarter. Later this year,
we also expect to initiate a first in human clinical trial of a SARD in
men with advanced prostate cancer," said Robert J. Wills, Ph.D.,
Executive Chairman of GTx. "There is a great deal of interest in our
SARD program based on data we have generated that demonstrate our SARD
compounds degrade and inhibit multiple forms of the androgen receptor,
including AR splice variants, and may therefore potentially treat CRPC
in men who are non-responsive to current androgen therapy."

Corporate Highlights and Anticipated Milestones

Enobosarm in Breast Cancer: The Company's lead product
candidate, enobosarm, a selective androgen receptor modulator (SARM), is
being developed as a targeted treatment for two advanced breast cancer
indications: (i) estrogen receptor positive (ER+) and androgen receptor
positive (AR+) breast cancer, and (ii) AR+ triple negative breast cancer
(TNBC). For both clinical trials, the primary efficacy endpoint is a
determination of clinical benefit (CB), which is defined as a complete
response, partial response or stable disease.

ER+/AR+ Breast Cancer: The Company has an ongoing open-label,
multi-center Phase 2 clinical trial of enobosarm in women with advanced,
ER+, AR+ breast cancer. Patients receive orally-administered enobosarm
(9 mg or 18 mg) daily for up to 24 months. The first stage of evaluation
was assessed among the first 18 evaluable patients for each cohort. At
least 3 of 18 patients per cohort achieved CB at week 24, and the trial
has proceeded to the second stage of enrollment. In Stage 2, if at least
9 of 44 evaluable patients achieve CB at week 24, the trial will have
successfully demonstrated its primary endpoint, and those patients
achieving CB at 24 weeks will be able to continue treatment for a total
of up to 24 months. The two dose cohorts in the trial are being treated
independently for the purpose of assessing efficacy. To date, in this
ongoing clinical trial:

  • CB has been demonstrated in both the 9 mg and 18 mg dose cohorts in
    Stage 1 of the trial, allowing both cohorts to advance to Stage 2 of
    the trial.
  • A sufficient number of evaluable patients have already demonstrated CB
    at week 24 in the 9 mg dose group for the study to achieve the
    pre-specified primary efficacy endpoint in this ongoing clinical
    trial. Of the 40 patients in the 9 mg dose cohort whose AR status has
    been confirmed AR+, 10 patients have demonstrated CB at week 24, 23
    patients have discontinued either at or prior to week 24, and 7
    patients remain on study and have not yet reached week 24. There are
    another 5 patients who have been enrolled to the 9 mg cohort whose AR
    status has not yet been confirmed. Of the 10 evaluable patients
    achieving CB, 2 had a partial response and 8 had stable disease. The
    majority of adverse events are grade 1 and 2, and the most common
    adverse events reported (occurring in ≥10% of patients) include nausea
    (31%), fatigue (18%), and arthralgias (13%). Elevations in
    transaminases (ALT and AST) during enobosarm treatment were mild with
    the majority being grade 1 or 2.

An abstract submitted by Dr. Beth Overmoyer, the lead principal
investigator for the clinical trial and a medical oncologist with the
Dana-Farber Cancer Center Institute, detailing data from Stage 1 of the
9 mg cohort, has been accepted for the "poster walk" at the European
Society for Medical Oncology IMPAKT Breast Cancer Conference, which is
being held May 4-6, 2017, in Brussels, Belgium.

Enobosarm appears to be safe and generally well tolerated. The
independent Safety Monitoring Committee met in December 2016, and
recommended that the clinical trial continue as planned. The trial will
continue with a daily dose of either enobosarm 9 mg or 18 mg until 44
evaluable patients in each cohort have completed treatment in order to
better characterize the CB response. The Company expects to report
top-line results from this study in the third quarter of 2017.

AR+ TNBC: The Company also has an ongoing open-label,
multi-center Phase 2 clinical trial to evaluate the efficacy and safety
of orally-administered enobosarm in up to 55 women with advanced, AR+
TNBC. Patients receive 18 mg of enobosarm once daily for up to 12
months. Stage 1 of the trial is being assessed among the first 21
evaluable patients, and if at least 2 of 21 patients achieve CB at week
16, then the trial can proceed to Stage 2 of enrollment of up to a total
of 41 evaluable patients. The primary efficacy objective of the trial is
CB response following 16 weeks of treatment in 41 evaluable patients.

  • The Company expects to have sufficient data from Stage 1 of the trial
    by the second quarter of 2017 to determine if patient enrollment
    should continue into Stage 2 of the trial.

SARMs in Non-Oncologic Indications: The Company also is
developing SARMs as potential treatments for both stress urinary
incontinence (SUI) in postmenopausal women and Duchenne muscular
dystrophy (DMD), a rare disease characterized by progressive muscle
degeneration and weakness.

Stress Urinary Incontinence: Enrollment continues in the
Company's ongoing Phase 2 proof-of-concept clinical trial of 3 mg of
enobosarm in postmenopausal women with SUI. The Company expects to have
top-line results from the trial in the third quarter of 2017.

Duchenne Muscular Dystrophy: Utilizing data developed from its
preclinical development efforts, the Company is pursuing a potential
strategic collaboration with biopharma companies experienced in orphan
drug development to continue the development of a SARM for the treatment
of DMD.

SARDs in Prostate Cancer: the Company's selective androgen
receptor degrader (SARD) technology is being evaluated as a potentially
novel treatment for men with castration-resistant prostate cancer
(CRPC), including those who do not respond or are resistant to currently
approved therapies. The Company believes that its SARD compounds will
degrade multiple forms of the androgen receptor, including AR splice
variants, such as AR-V7, along with mutant versions of the receptor.

Castration-Resistant Prostate Cancer: The Company has screened
dozens of compounds from its extensive patented SARD portfolio and has
now selected lead compounds that are undergoing further preclinical
studies required for a first in human clinical trial, including
toxicology studies. It is anticipated that a first in human clinical
trial of a SARD will be initiated during the second half of 2017.

The preclinical studies supporting the SARD program have been accepted
for presentation and received recognition of merit at several upcoming
international meetings, including the Endocrine Society's annual
meeting, ENDO 2017, from April 1-4, 2017, and the annual meeting of the
European Association of Urology being held from March 24-28, 2017.

Fourth Quarter and Year-End 2016 Financial Results

  • As of December 31, 2016, cash and short-term investments were $21.9
    million compared to $29.3 million at December 31, 2015.
  • Research and development expenses for the quarter ended December 31,
    2016 were $4.6 million compared to $3.9 million for the same period of
    2015. Research and development expenses for the year ended December
    31, 2016 were $17.2 million compared to $13.6 million for the year
    ended December 31, 2015.
  • General and administrative expenses for the quarter ended December 31,
    2016 were $2.3 million compared to $2.1 million for the same period of
    2015. General and administrative expenses for the year ended December
    31, 2016 were $8.7 million compared to $8.2 million for the year ended
    December 31, 2015.
  • The net loss for the quarter ended December 31, 2016 was $6.9 million
    compared to a net loss of $3.2 million for the same period in 2015.
    The net loss for the quarter ended December 31, 2015 included a
    non-cash gain of $2.7 million related to the change in the fair value
    of the Company's warrant liability. During the first quarter of 2016,
    the Company modified its outstanding warrants with no further
    adjustment to the fair value of these warrants being required
    subsequent to the first quarter of 2016.
  • The net loss for the year ended December 31, 2016 was $17.7 million
    compared to a net loss of $18.7 million for the year ended December
    31, 2015. The net loss for the years ended December 31, 2016 and
    December 31, 2015 included a non-cash gain of $8.2 million and $3.1
    million, respectively, related to the change in the fair value of the
    Company's warrant liability.
  • GTx had approximately 15.9 million shares of common stock outstanding
    as of December 31, 2016. Additionally, there remain warrants
    outstanding to purchase approximately 6.4 million shares of GTx common
    stock at an exercise price of $8.50 per share.

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, including treatments for
breast and prostate cancer, 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 the enrollment and conduct of GTx's ongoing Phase 2 proof-of-concept
clinical trial of enobosarm (GTx-024) to treat stress urinary
incontinence (SUI) and its Phase 2 clinical trials of enobosarm for the
treatment of advanced breast cancer, as well as the potential
preclinical and other future development of GTx's licensed SARD
technology and the development of selective androgen receptor modulators
(SARMs) for the treatment of Duchenne muscular dystrophy (DMD) and the
timing thereof, including the identification of lead SARD clinical
candidates and the potential evaluation thereof for the initiation of a
first-in-man clinical study; and the potential therapeutic applications
for, and potential benefits of SARM (including enobosarm) and SARD
technology. 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's evaluation of its licensed SARD
technology or a SARM for the treatment of DMD are at very early stages
and it is possible that GTx may determine not to move forward with any
meaningful development of one or both programs; (ii) that if GTx
determines to move forward with additional development of enobosarm for
the treatment of advanced breast cancer or for the treatment of SUI or
if GTx does determine to move forward with meaningful development of its
SARD program or a SARM for the treatment of DMD, GTx will require
additional funding, which it may be unable to raise, in which case, GTx
may fail to realize the anticipated benefits from its SARM and/or SARD
technology; (iii) that GTx may not be successful in developing a
clinical SARD product candidate or a SARM for the treatment of DMD to
advance into clinical studies or the clinical product candidate may fail
such clinical studies; (iv) that the clinical trials of enobosarm to
treat advanced breast cancer or SUI being conducted by GTx may not be
completed on schedule, or at all, or may otherwise be suspended or
terminated; (v) related to the difficulty and uncertainty of
pharmaceutical product development, including the time and expense
required to conduct preclinical and clinical trials and analyze data,
and the uncertainty of preclinical and clinical success; and (vi)
related to issues arising during the uncertain and time-consuming
regulatory process, including the risk that GTx may not receive any
approvals to advance the clinical development of one or more potential
clinical SARM or SARD candidates. In addition, GTx will continue to need
additional funding and may be unable to raise capital when needed, which
would force GTx to delay, reduce or eliminate its product candidate
development programs and potentially cease operations. 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. 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 for the period ending
September 30, 2016, 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)

 
 
      December 31,
2016     2015
(unaudited)
ASSETS
Current assets:
Cash and cash equivalents $ 8,910 $ 14,056
Short-term investments 12,959 15,200
Prepaid expenses and other current assets   2,429     2,633  
Total current assets 24,298 31,889
Property and equipment, net 81 5
Intangible assets, net   123     137  
Total assets $ 24,502   $ 32,031  
LIABILITIES AND STOCKHOLDERS' EQUITY
Current liabilities:
Accounts payable $ 1,220 $ 382
Warrant liability - 27,349
Accrued expenses and other current liabilities   3,391     2,441  
Total current liabilities 4,611 30,172
Commitments and contingencies
Stockholders' equity:
Common stock, $0.001 par value: 60,000,000 and 400,000,000 shares
authorized at December 31, 2016 and December 31, 2015, respectively;
15,919,572 and 14,037,411 shares issued and outstanding at December
31, 2016 and December 31, 2015, respectively
16 14
Additional paid-in capital 551,073 515,319
Accumulated deficit   (531,198 )   (513,474 )
Total stockholders' equity   19,891     1,859  
Total liabilities and stockholders' equity $ 24,502   $ 32,031  
 
 
GTx, Inc.
Condensed Statements of Operations
(in thousands, except share and per share data)
(unaudited)
 
 
      Three Months Ended     Year Ended
December 31, December 31,
2016     2015 2016     2015
 
Expenses:
Research and development expenses $ 4,585 $ 3,879 $ 17,228 $ 13,607
General and administrative expenses   2,279     2,079     8,705     8,234  
Total expenses   6,864     5,958     25,933     21,841  
Loss from operations (6,864 ) (5,958 ) (25,933 ) (21,841 )
Other (expense) income, net - (4 ) 46 57

Gain on change in fair value of warrant liability

  -     2,729     8,163     3,081  
Net loss $ (6,864 ) $ (3,233 ) $ (17,724 ) $ (18,703 )
 
Net loss per share:
Basic $ (0.44 ) $ (0.23 ) $ (1.22 ) $ (1.33 )
Diluted $ (0.44 ) $ (0.40 ) $ (1.22 ) $ (1.47 )
 
Weighted average shares outstanding:
Basic   15,713,210     14,037,411     14,559,541     14,036,468  
Diluted   15,713,210     14,952,920     14,559,541     14,777,404  
 

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