GTx Announces Presentation of Preclinical Data Demonstrating the Ability of SARDs to Degrade and Inhibit the Androgen Receptor at the American Urological Association Annual Meeting

— Late breaking poster outlines first data from GTx SARD program;
results suggest these molecules are first-in-class androgen receptor
antagonists and degraders —

— Novel SARD compounds being developed initially as a potential
treatment for men with castration-resistant prostate cancer —

MEMPHIS, Tenn.–(BUSINESS WIRE)–GTx, Inc. (Nasdaq: GTXI) today announced the first public presentation
of preclinical data from the Company’s selective androgen receptor
degrader (SARD) program. The results demonstrate that the Company’s
highly potent SARDs selectively bind to the ligand binding domain (LBD)
and interact with the N-terminal domain (NTD) of the androgen receptor
(AR) and inhibit and degrade the AR at very low concentrations. These
preclinical results suggest that the Company’s SARDs may be the
first-in-class dual-interacting AR antagonists and degraders.

The preclinical data are being presented at the 2016 American Urological
Association (AUA) Annual Meeting taking place May 6-10, 2016, in San
Diego, California.

Poster:		Novel Dual-Binding Selective Degraders of Full Length and Splice Variant Androgen Receptors for the Treatment of Castration-Resistant Prostate Cancer
Presenter:		Ramesh Narayanan, Ph.D., Director, Center for Cancer Drug Discovery and Associate Professor, Department of Medicine, University of Tennessee and Consultant for GTx, Inc.
Date:		Saturday, May 7 at 10:30 am until Tuesday, May 10 at 1:00 pm

According to Dr. Narayanan, “One of the limitations of current prostate
cancer therapy is that some men with castration-resistant prostate
cancer do not respond or eventually develop resistance to the therapy.
These preclinical results suggest that novel SARD compounds may 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 androgen therapy.”

The Company’s lead SARD compounds are currently being evaluated in
preclinical studies in order to select the best SARD compounds for
continued development, with a goal of initiating first human clinical
trials in 2017.

About SARDs

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. GTx believes
that its novel SARD compounds will degrade multiple forms of the
androgen receptor, including AR splice variants, such as AR-V7. GTx
licensed the SARD technology from the University of Tennessee Research
Foundation in 2015 with the goal of expanding its portfolio of product
candidates targeting hormonal receptors.

About Prostate Cancer

Prostate cancer that is localized to the prostate can be effectively
treated with surgery, radiation, brachytherapy and other modalities in
an effort to eradicate all of the disease and cure the patient. In some
cases, the tumor advances locally or metastasizes; these are examples of
advanced prostate cancer. The goal of treatment for advanced prostate
cancer is to control the tumor and keep the patient alive for as long as
possible.

In advanced prostate cancer, a number of treatments with hormone
blocking therapies or chemotherapy are used to slow the spread of
metastases, shrink existing tumors, reduce symptoms and improve quality
of life. Although most men with advanced prostate cancer are not cured
of their disease, they can live a normal life for many years.

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 GTx’s ongoing preclinical development of its selective androgen
receptor degrader (SARD) technology, and its possible initiation of SARD
clinical trials in 2017. 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 the preclinical development
studies being conducted by GTx may not be completed on schedule, or at
all, or they may indicate that no SARD compound warrants further
development; (ii) that any additional preclinical or clinical
development of GTx’s SARD compounds beyond 2016 is contingent on GTx
entering into new collaborative arrangements with third parties for such
development or otherwise obtaining sufficient additional capital to
permit such development, which it may be unable to do; or (iii) that GTx
may not be able to obtain required regulatory approvals to initiate
clinical development of a SARD compound in a timely manner or at all. 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.

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