GTx Provides Corporate Update and Reports Third Quarter 2017 Financial Results

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— Positive results achieved in all patients after 12-week treatment
in the enobosarm Phase 2 proof-of-concept clinical trial in women with
stress urinary incontinence (SUI) —

— Enobosarm Phase 2 proof-of-concept clinical trial for SUI
continues to show sustained durability of response —

— Initiated ASTRID study, a Phase 2 placebo-controlled clinical
trial of orally-administered enobosarm for the treatment of SUI —

— Closed private placement with $45.6 million in net proceeds —

GTx, Inc. (NASDAQ:GTXI) today reported financial results for the third
quarter of 2017 and highlighted recent accomplishments and upcoming
milestones.

“During the quarter, we achieved a key milestone for the company when we
reported positive results from our first clinical trial in stress
urinary incontinence,” said Robert J. Wills, Ph.D., Executive Chairman
of GTx. “Remarkably, 18 out of 18 women who received enobosarm for 12
weeks responded. These responses also appear to be durable, lasting
months after dosing. These exciting results provided the basis for our
recently initiated, placebo-controlled clinical trial of enobosarm for
the treatment of SUI.”

“Recent data suggests 50 percent of women report SUI, for which there
are no FDA-approved pharmaceutical therapies. An orally-available
therapy would offer numerous advantages over existing treatments
including surgery, and would provide a significant new option that many
women would choose in order to address this medical condition,” said
Kenneth M. Peters, M.D., Chairman of Urology, Oakland University William
Beaumont School of Medicine and the principal investigator in the trial.

Third Quarter 2017 Clinical Highlights and Anticipated Milestones

Stress Urinary Incontinence (SUI):

Enobosarm, a Selective Androgen Receptor Modulator (SARM), is being
evaluated in Phase 2 clinical development for SUI, the Company’s lead
indication. Recent important milestones are summarized as follows:

  • Reported positive results from the Phase 2 proof-of-concept (POC)
    clinical trial of enobosarm 3 mg administered orally in
    post-menopausal women with SUI. With the inclusion of the final
    patient completing treatment in the POC clinical trial, data from the
    18 evaluable patients completing the required 12 weeks of daily
    treatment showed a clinically meaningful reduction (50 percent or
    greater) in stress leaks per day, compared to baseline. The mean
    decrease in stress leaks per day was 81 percent overall (5.17 mean
    leaks/day at baseline to 1.0 mean leaks/day at 12 weeks).
  • Patients are being followed for an additional 28 weeks post-treatment
    to assess the durability of treatment effect. Durability of response
    for patients who completed the 28-week observation phase has resulted
    in a 41 to 100 percent reduction in stress leaks/day from baseline
    (N=6). For those patients who have not completed the 28-week
    observation phase, the durability of response, measured beginning 4
    weeks post dosing, continues to be sustained.
  • Highlighted the Phase 2 POC results at the International Continence
    Society (ICS) annual meeting in a poster entitled, “Kegels in a
    Bottle: Preliminary Results of a Selective Androgen Receptor Modulator
    (GTx-024) for the Treatment of Stress Urinary Incontinence in
    Post-Menopausal Women”, which subsequently was voted best poster for
    the conference.
  • Initiated a second clinical trial, Assessing Enobosarm for Stress
    Urinary Incontinence Disorder (ASTRID): a randomized,
    double-blinded, placebo-controlled, Phase 2 trial to assess the
    efficacy and safety of two doses of enobosarm (1 mg and 3 mg)
    administered orally in post-menopausal woman with SUI compared to
    placebo. The primary endpoint of the trial is the percentage of
    patients with at least a 50 percent reduction in mean leaks/day,
    compared to baseline. This trial is expected to enroll approximately
    400 patients across 70 clinical sites in the U.S. Top-line results are
    expected to be available by the end of 2018.

Breast Cancer:

Enobosarm is also being evaluated as a hormonal therapy for women with
estrogen receptor positive (ER+) and androgen receptor positive (AR+)
breast cancer in a Phase 2 clinical trial for this advanced breast
cancer population. As reported earlier for the 9 mg cohort, the Phase 2
trial pre-specified threshold for success, clinical benefit response
(CBR), was attained and therefore met the primary efficacy endpoint. In
addition, the 18 mg cohort has also met the primary efficacy endpoint.
The trial has now completed enrollment of the predefined number of
evaluable patients in both dosage arms with at least 44 patients in each
of two cohorts receiving 9 mg or 18 mg daily doses of enobosarm.

  • In the 9 mg cohort, following 24 weeks of treatment, a total of 14
    patients achieved a CBR out of 49 evaluable patients confirmed as AR
    positive (28.6%), with two patients achieving a partial response and
    12 reporting stable disease. Currently, four patients in this cohort
    remain on study. In the 18 mg cohort, with 48 evaluable patients, 12
    patients achieved a CBR (25%) at 24 weeks with one patient
    demonstrating a partial response and 11 patients reporting stable
    disease. Three patients remain on study in the 18 mg cohort. Both
    doses of enobosarm appear to be safe and generally well tolerated. A
    complete summary of the study results will be submitted for
    presentation or publication in 2018.
  • Although both the 9 mg and 18 mg cohorts met the primary efficacy
    endpoint in the Phase 2 clinical trial, after evaluating the drug
    development environment for breast cancer, where treatment paradigms
    are shifting to immunotherapies and/or combination therapies, the
    Company has decided that the time and cost of conducting the necessary
    clinical trials for approval in this indication do not warrant further
    development of enobosarm in this indication at this time.

Duchenne Muscular Dystrophy (DMD):

SARMs have also been evaluated in preclinical models of DMD, in which
GTx SARMs have increased lean muscle mass and physical function. 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.

Prostate Cancer:

The Company has a Selective Androgen Receptor Degrader (SARD)
preclinical program to evaluate its novel SARD technology in
castration-resistant prostate cancer (CRPC). The Company has ongoing
mechanistic preclinical studies designed to select the most appropriate
compound to advance into a first-in-human clinical trial.

Third Quarter 2017 Corporate Highlights and Financial Results

  • During the quarter, GTx raised net proceeds of $45.6 million in a
    private placement of its common stock and warrants to purchase its
    common stock. GTx sold 5,483,320 immediately separable units,
    comprised of an aggregate of 5,483,320 newly-issued shares of common
    stock and warrants to purchase up to 3,289,988 additional shares of
    common stock. Both the common stock and warrants have been registered
    for resale with the Securities and Exchange Commission.
  • As of September 30, 2017, cash and short-term investments were $53.6
    million compared to $21.9 million at December 31, 2016.
  • Research and development expenses for the quarter ended September 30,
    2017 were $5.9 million compared to $4.6 million for the same period of
    2016.
  • General and administrative expenses for the quarter ended September
    30, 2017 were $2.6 million compared to $2.3 million for the same
    period of 2016.
  • Net loss for the three months ended September 30, 2017 was $8.5
    million compared to a net loss of $6.9 million for the same period in
    2016.
  • Net loss for the nine months ended September 30, 2017 was $21.2
    million compared to a net loss of $10.9 million for the same period in
    2016. The nine months ended September 30, 2016 included a non-cash
    gain of $8.2 million due to the change in 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.
  • GTx had approximately 21.5 million shares of common stock outstanding
    as of September 30, 2017. Additionally, there are warrants outstanding
    to purchase approximately 6.4 million shares of GTx common stock at an
    exercise price of $8.50 per share and approximately 3.3 million shares
    of GTx common stock at an exercise price of $9.02.

About GTx

GTx is a biopharmaceutical company dedicated to the discovery,
development and commercialization of medicines to treat serious medical
conditions, including stress urinary incontinence and prostate cancer.

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
placebo-controlled clinical trial of enobosarm (GTx-024) in
post-menopausal women with stress urinary incontinence (SUI), as well as
GTx’s plans for its ongoing preclinical research and potential future
development of GTx’s licensed selective androgen receptor degrader
(SARD) technology, as well as the development of selective androgen
receptor modulators (SARMs) for the treatment of Duchenne muscular
dystrophy (DMD) and the timing thereof; 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 SUI or if GTx does determine to move forward with
development of its SARD program or a SARM, 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 to advance into clinical studies or the clinical
product candidate may fail such clinical studies; (iv) that the Phase 2
placebo-controlled clinical trial of enobosarm to treat 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 quarter
ending September 30, 2017, which is being filed subsequent to this
release, 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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