-- Enobosarm demonstrated clinical benefit and
met the primary efficacy endpoint for the 9 mg dose cohort in GTx’s
ongoing ER+/AR+ Phase 2 breast cancer trial --
-- Company expects to report preliminary
results from its Phase 2 clinical trial of enobosarm to treat
Stress Urinary Incontinence (SUI) in the third quarter of 2017
--
-- Company expects to begin an initial clinical
study of a Selective Androgen Receptor Degrader (SARD) in men with
castration-resistant prostate cancer in the second half of 2017
--
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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version on businesswire.com: http://www.businesswire.com/news/home/20170315005316/en/
GTx, Inc.Investors:Lauren Crosby,
901-271-8622lcrosby@gtxinc.comorMedia:Red House ConsultingDenise
Powell, 510-703-9491denise@redhousecomms.com
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