-- 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.
GTx, Inc.Condensed Balance
Sheets(in thousands, except share data)
September 30,
December 31, 2017 2016 (unaudited)
ASSETS Current assets: Cash and cash equivalents $ 53,431 $
8,910 Short-term investments 200 12,959 Prepaid expenses and other
current assets 2,049 2,429 Total
current assets 55,680 24,298 Property and equipment, net 57 81
Intangible assets, net 112 123 Total
assets $ 55,849 $ 24,502
LIABILITIES AND
STOCKHOLDERS’ EQUITY Current liabilities: Accounts payable $
2,451 $ 1,220 Accrued expenses and other current liabilities
6,828 3,391 Total current liabilities 9,279
4,611 Commitments and contingencies Stockholders’ equity: Common
stock, $0.001 par value: 60,000,000 shares authorized at September
30, 2017 and December 31, 2016; 21,541,909 and 15,919,572 shares
issued and outstanding at September 30, 2017 and December 31, 2016,
respectively 22 16 Additional paid-in capital 598,908 551,073
Accumulated deficit (552,360 ) (531,198 ) Total
stockholders’ equity 46,570 19,891
Total liabilities and stockholders’ equity $ 55,849 $ 24,502
GTx, Inc.Condensed Statements of
Operations(in thousands, except share and per share
data)(unaudited)
Three Months Ended
Nine Months Ended September 30, September 30,
2017 2016 2017
2016 Expenses: Research and development expenses $
5,914 $ 4,614 $ 14,555 $ 12,643 General and administrative expenses
2,617 2,313 6,701
6,426 Total expenses 8,531 6,927
21,256 19,069 Loss from operations
(8,531 ) (6,927 ) (21,256 ) (19,069 ) Other income, net 27 13 94 46
Gain on change in fair value of warrant liability -
- - 8,163 Net loss $
(8,504 ) $ (6,914 ) $ (21,162 ) $ (10,860 ) Net loss
per share -- basic and diluted $ (0.53 ) $ (0.49 ) $ (1.32 ) $
(0.77 ) Weighted average shares outstanding: Basic and
diluted 16,115,835 14,189,226
16,059,383 14,172,177
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version on businesswire.com: http://www.businesswire.com/news/home/20171114006551/en/
Investors:Argot PartnersKimberly Minarovich or Sam
Martin, 212-600-1902orMedia:Red House ConsultingDenise
Powell, 510-703-9491denise@redhousecomms.com
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