GTx Provides Corporate Update and Reports First Quarter 2016 Financial Results

MEMPHIS, Tenn.–(BUSINESS WIRE)–GTx, Inc. (Nasdaq: GTXI) today reported financial results for the first
quarter ended March 31, 2016, and highlighted recent accomplishments and
upcoming milestones. The Company is currently enrolling patients in
three clinical trials: two trials evaluating enobosarm as a potential
treatment for women with advanced breast cancer and another assessing
enobosarm as a potential treatment for stress urinary incontinence in
postmenopausal women.

“During the first quarter, we executed on several ongoing initiatives
that we expect will make 2016 a year of considerable progress,” said Dr.
Robert J. Wills, Executive Chairman of GTx. “We have continued to make
progress in the enrollment of our two advanced breast cancer clinical
trials of enobosarm, with preliminary data from the first stage of each
study expected by the end of 2016. Also, we are excited to be
collaborating with doctors on a clinical trial that is the first
clinical study to use a SARM to treat stress urinary incontinence in
postmenopausal women and expect data from this study late in 2016.”

Corporate Highlights and Anticipated Milestones

Enobosarm in Breast Cancer: The Company’s lead product
candidate, 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 will be
clinical benefit, which is defined as a complete response, partial
response or stable disease.

  • ER+/AR+ breast cancer: We currently expect to complete
    enrollment in the first stage of the open-label, Phase 2 clinical
    trial of enobosarm in women with metastatic or locally advanced
    ER+/AR+ breast cancer in the second quarter of 2016, to allow us to
    determine during the fourth quarter of this year whether there is
    sufficient safety and efficacy data to warrant proceeding with the
    second stage of the clinical study. While the first stage of the trial
    will evaluate 18 patients for each of the two dosing arms, 9 mg and 18
    mg of enobosarm, the trial is designed to enroll up to 118 patients to
    obtain data from 44 evaluable patients in each study arm (a total of
    88 evaluable patients) to assess the primary efficacy objective of
    clinical benefit response following 24 weeks of treatment.
  • AR+ TNBC: We currently expect to complete enrollment in the
    first stage of the open-label, proof-of-concept Phase 2 clinical trial
    of 18 mg of enobosarm in women with advanced AR+ TNBC in the third
    quarter of 2016, to allow us to determine by the end of 2016 whether
    there is sufficient safety and efficacy data to warrant proceeding
    with the second stage of the clinical study. While the first stage
    will include 21 evaluable patients, the trial is designed to enroll up
    to 55 patients in total in order to obtain data from 41 evaluable
    patients to assess the primary efficacy objective of clinical benefit
    response following 16 weeks of treatment.

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

  • SUI: We are currently enrolling patients in a Phase 2
    proof-of-concept clinical trial of 3 mg of enobosarm to treat up to 35
    postmenopausal women with SUI, the first clinical trial to evaluate a
    SARM for SUI. Top-line data from the Phase 2 clinical trial is
    anticipated by the end of 2016.
  • DMD: The Company’s preclinical studies have continued to
    confirm beneficial effects from SARMs in mice genetically altered to
    simulate DMD, compared to control groups. The Company continues to
    advance its preclinical initiatives while pursuing a strategic
    collaboration with potential biopharma partners experienced in orphan
    drug development.

SARDs in Prostate Cancer: Our 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.

  • CRPC: Several lead SARD compounds are currently being evaluated
    in preclinical studies to select the best SARD compounds for continued
    development, as well as to develop data necessary to initiate first in
    human clinical trials in 2017.

First Quarter 2016 Financial Results

  • As of March 31, 2016, cash and short-term investments were $24.3
    million compared to $29.3 million at December 31, 2015.
  • Research and development expenses for the quarter ended March 31, 2016
    were $4.0 million compared to $2.9 million for the same period of 2015.
  • General and administrative expenses were $2.1 million for both the
    quarter ended March 31, 2016 and March 31, 2015.
  • The Company recognized a non-cash gain of $8.2 million and $2.6
    million for the quarter ended March 31, 2016 and 2015, respectively,
    due to the change in fair value of the Company’s warrant liability.
    During the first quarter of 2016, the Company recorded a non-cash
    reclassification of this warrant liability to stockholders’ equity due
    to the modification of these warrants. No adjustments to the fair
    value of these warrants will be made in the future.
  • Net income for the quarter ended March 31, 2016 was $2.1 million
    compared to a net loss of $2.4 million for the same period in 2015.
    Net income for the quarter ended March 31, 2016 included the non-cash
    gain of $8.2 million related the revaluation of our warrant liability.
    The net loss for the quarter ended March 31, 2015 included a non-cash
    gain of $2.6 million related to the change in the fair value of the
    Company’s warrant liability.
  • GTx had approximately 141.7 million shares of common stock outstanding
    as of March 31, 2016. Additionally, there remain warrants outstanding
    to purchase approximately 64.3 million shares of GTx common stock at
    an exercise price of $0.85 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 anticipated identification of clinical
SARD candidates and the potential evaluation thereof in clinical
studies; 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 the 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 annual
report on Form 10-K for the year ended December 31, 2015 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)
 
March 31, December 31,
2016 2015
(unaudited)
ASSETS
Current assets:
Cash and cash equivalents $ 12,063 $ 14,056
Short-term investments 12,200 15,200
Prepaid expenses and other current assets   2,295     2,633  
Total current assets 26,558 31,889
Property and equipment, net 12 5
Intangible assets, net   134     137  
Total assets $ 26,704   $ 32,031  
LIABILITIES AND STOCKHOLDERS’ EQUITY
Current liabilities:
Accounts payable $ 903 $ 382
Warrant liability 27,349
Accrued expenses and other current liabilities   2,116     2,441  
Total current liabilities 3,019 30,172
Commitments and contingencies
Stockholders’ equity:

Common stock, $0.001 par value: 400,000,000 shares authorized at
March 31, 2016 and December 31, 2015; 141,749,150 and 140,374,112
shares issued and outstanding at March 31, 2016 and December 31,
2015, respectively

142 141
Additional paid-in capital 534,911 515,192
Accumulated deficit   (511,368 )   (513,474 )
Total stockholders’ equity   23,685     1,859  
Total liabilities and stockholders’ equity $ 26,704   $ 32,031  
 
   
GTx, Inc.
Condensed Statements of Operations
(in thousands, except share and per share data)
(unaudited)
 
Three Months Ended
March 31,
2016     2015
 
Expenses:
Research and development expenses $ 3,971 $ 2,948
General and administrative expenses   2,114     2,111  
Total expenses   6,085     5,059  
Loss from operations (6,085 ) (5,059 )
Other income, net 28 27
Gain on change in fair value of warrant liability   8,163     2,648  
Net income (loss) $ 2,106   $ (2,384 )
 
Net income (loss) per share — basic and diluted $ 0.01   $ (0.02 )
 
Weighted average shares outstanding:
Basic   141,522,043     140,335,875  
Diluted   143,448,168     140,335,875  

Contacts

Investors:
GTx, Inc.
Lauren Crosby, 901-271-8622
lcrosby@gtxinc.com
or
Media:
Red
House Consulting
Denise Powell, 510-703-9491
denise@redhousecomms.com