ACAPODENE
for the Treatment of Side Effects of Androgen Deprivation Therapy
MEMPHIS, Tenn., June 7, 2004—GTx, Inc.,
(Nasdaq: GTXI) a biopharmaceutical company dedicated to the discovery,
development and commercialization of therapeutics primarily related
to the treatment of serious men's health conditions, announced today
at the American Society of Clinical Oncology in New Orleans positive
data from a preclinical study designed to demonstrate the ability
of ACAPODENE (toremifene citrate) tablets to prevent bone loss as
a result of androgen deprivation therapy (ADT) and positive data
from its Phase II clinical trial of ACAPODENE for the treatment
of side effects associated with ADT. ACAPODENE, GTxŐs lead product
candidate, is a nonsteroidal selective estrogen receptor modulator
(SERM) currently in a large scale Phase III trial designed to assess
ACAPODENEŐs ability to reduce skeletal fractures in men on ADT.
Preclinical Results
This study showed that ACAPODENE reduced bone turnover, prevented
bone mineral density (BMD) loss and protected bone microarchitecture
in animal models. "In orchiectomized rats treated with toremifene
via subcutaneous pumps, we observed that toremifene treatment reversed
the orchiectomy- induced loss in BMD, significantly reduced the
orchiechtomy-induced increase in bone biomarker levels and protected
bone microarchitecture as evidenced by increased trabecular bone
volume and trabecular number in only 6 weeks" said Sharan Raghow
Ph.D., senior scientist at GTx, Inc.
Phase II Trial Results
In a Phase II study consenting patients who had been on ADT for
a least 12 months were randomized to 20 mg, 40 mg, or 60 mg of ACAPODENE
or placebo administered orally once daily for six months. ACAPODENE
was well tolerated at all doses studied. As a result, there was
an increase in bone mineral density after six months of therapy
compared to a decrease in the placebo group (p<0.05), as well as
beneficial effects on other side effects of ADT.
The two abstracts presented at ASCO were:
Abstract 1594
Toremifene Citrate versus Placebo for Treatment of Bone Loss and
Other Complications of Androgen Deprivation Therapy in Patients
with Prostate Cancer
Presenter: M. Steiner
Abstract 8108
Can Toremifene Prevent Bone Loss In the Orchiectomized Rat?
Presenter: S. Raghow
"The positive results on bone markers and bone
mineral density from the Phase II study gave GTx the confidence
to initiate a Phase III trial for ACAPODENE. After receiving approval
for a special protocol assessment from the FDA, we launched the
Pivotal Phase III trial with 1200 patients last November", stated
Mitchell Steiner, M.D., CEO GTx. "We believe that ACAPODENE has
great promise as a potential new therapeutic to prevent bone loss
and treat serious side effects of androgen deprivation therapy which
will improve the quality of life in patients who are suffering from
prostate cancer."
About Androgen Deprivation Therapy
Androgen deprivation therapy is the standard medical treatment for
patients who have prostate cancer that is advanced, recurrent, or
metastatic. ADT is accomplished either surgically, by removal of
the testes, or chemically, by treatment with luteinizing hormone
releasing hormone agonists (LHRH agonists), and results in stopping
production of testosterone by the testes. An increasing number of
prostate cancer patients are being treated by ADT because studies
have shown that survival of prostate cancer patients is prolonged
when on the therapy and serum PSA testing detects prostate cancer
recurrences earlier than in the past. As a result of the increasing
number of prostate cancer patients on ADT, the serious side effects
now contribute significantly to their quality of life. Availability
of a single drug to address multiple side effects of ADT may become
a mainstay treatment for patients on ADT. PatientŐs undergoing ADT
have 50% to 90% reduction in estradiol. This reduction in estradiol
leads to a bone loss rate of 3% to 5% per year and a progressive
increase in the risk of fractures. Up to 50% of men on ADT suffer
vertebral fractures. Skeletal fractures are a significant clinical
problem because prostate cancer patients who develop skeletal fractures
have shorter survival rates compared to patients who do not develop
skeletal fractures, with the median survival time shortened by 39
months.
About ACAPODENE
ACAPODENE is a nonsteroidal SERM, a small molecule that binds and
selectively modulates the estrogen receptor. SERMs have been shown
to block estrogen receptors in the prostate. GTx has licensed the
right to develop, market and distribute toremifene citrate, the
active ingredient of ACAPODENE tablets, worldwide in the field of
prevention and treatment of prostate cancer from Orion Corporation,
Finland.
About Prostate Cancer
In the United States, there is estimated to be over 230,000 new
prostate cancer cases and 30,000 prostate cancer deaths this year.
This makes prostate cancer the most commonly diagnosed cancer and
the second leading cause of cancer-related deaths in men in the
United States.
Goldman Sachs Healthcare Conference
GTx will present at 9:50am Pacific Time on Tuesday, June 8th at
the Goldman Sachs Annual Healthcare Conference in Laguna Nigel,
CA. A webcast of the presentation will be available starting June
8, 2004 at 10am Pacific Time until June 30, 2004 by visiting www.gtxinc.com
and clicking on the Events tab under Investor Relations.
About GTx
GTx is a biopharmaceutical company dedicated to the discovery, development
and commercialization of therapeutics primarily related to the treatment
of serious men's health conditions. GTx's drug discovery and development
programs are focused on small molecules that selectively modulate
the effects of estrogens and androgens. GTx currently has two product
candidates that are in human clinical trials. The company is developing
ACAPODENE, its most advanced product candidate, through clinical
trials for two separate indications: (1) its now completed Phase
IIb clinical trial for the reduction in the incidence of prostate
cancer in high risk men with precancerous prostate lesions and (2)
a pivotal Phase III clinical trial for the treatment of serious
side effects of advanced prostate cancer therapy. GTx is developing
its second product candidate, andarine, and other specified backup
compounds, with its partner, Ortho Biotech Products, L.P., a subsidiary
of Johnson & Johnson. Andarine will be entering a planned Phase
II clinical trial this year. GTx retains all rights to the discovery,
development, and commercialization of the rest of its SARM program
including its other specific product candidates ostarine, prostarine
and andromustine.
Forward Looking Statement
This press release contains forward-looking statements, including,
without limitation, statements related to GTx's current and anticipated
clinical trials and research and development programs. These forward-looking
statements are based upon GTx's current expectations. Forward-looking
statements involve risks and uncertainties. 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, risks that neither
GTx nor its partner will be able to commercialize its product candidates
if preclinical studies do not produce successful results or clinical
trials do not demonstrate safety and efficacy in humans; if third
parties do not manufacture the Company's product candidates in sufficient
quantities and at an acceptable cost, clinical development and commercialization
of its product candidates would be delayed; use of third-party manufacturers
may increase the risk that the Company will not have adequate supplies
of its product candidates; if third parties on whom the Company
relies do not perform as contractually required or expected, the
Company may not be able to obtain regulatory approval for or commercialize
its product candidates; the Company is dependent upon collaborative
arrangements to complete the development and commercialization of
some of its product candidates, and these collaborative arrangements
may place the development of its product candidates outside its
control, may require it to relinquish important rights or may otherwise
be on terms unfavorable to the Company; and if the Company is not
able to obtain required regulatory approvals, the Company will not
be able to commercialize its product candidates. The annual report
filed on Form 10-K with the U.S. Securities and Exchange Commission
on March 26, 2004 contains under the heading "Additional Factors
That Might Affect Future Results" 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.
