Exhibit 99.1
Contacts:
GTx, Inc. John Nugent (investors)
Carney Duntsch Kathy Jones-Nugent, Ph.D. (media)
Director, Corporate Communications Burns McClellan
901-523-9700 212-213-0006
GTx, Inc. Announces Positive Phase IIb Data for ACAPODENE(TM)
for the Prevention of Prostate Cancer in High Risk Men
MEMPHIS, TENN. - JUNE 2, 2004 -- GTx, Inc. (Nasdaq: GTXI), a biopharmaceutical
company dedicated to the discovery, development and commercialization of
therapeutics for serious men's health conditions announced today positive Phase
IIb results for its lead product ACAPODENE(TM) (toremifene citrate) tablets. The
data demonstrated that ACAPODENE(TM) was well tolerated and reduced the
incidence of prostate cancer in men who are at high risk for prostate cancer.
Patients were classified as being at high risk because they had biopsy proven
precancerous lesions of the prostate called high grade prostatic intraepithelial
neoplasia (PIN). GTx plans to initiate a Phase III clinical trial in 2004
following discussions with the Food and Drug Administration (FDA).
ACAPODENE(TM), GTx's lead product candidate, is a nonsteroidal selective
estrogen receptor modulator (SERM).
STUDY RESULTS
The ACAPODENE(TM) Phase IIb study was double-blind, placebo-controlled, one year
clinical trial in 514 men at high risk for prostate cancer and high grade PIN.
The primary endpoint was the incidence of prostate cancer. This is the largest
prospective study of the natural history of patients with high grade PIN. This
well controlled study confirmed that men who have high grade PIN are at high
risk as 31% of placebo patients were diagnosed with prostate cancer by 1 year.
The intent-to-treat analysis, defined as any patient who had at least one
on-study biopsy, showed that ACAPODENE(TM) 20 mg had a 20% reduction in prostate
cancer incidence. The reduction of prostate cancer incidence improved in men who
received ACAPODENE(TM) 20mg for one year with a 46% reduction in this high risk
population compared to the placebo group, which is consistent with the interim
analysis GTx conducted last year. For men who developed prostate cancer, those
treated with ACAPODENE(TM) had similar tumor grades to those of placebo
patients. ACAPODENE(TM) was well tolerated as the number of adverse events were
similar between those patients receiving ACAPODENE(TM) compared to placebo.
"This study confirms that men who have high grade PIN are indeed high-risk
patients for prostate cancer with 31% of the men receiving placebo being
diagnosed with cancer within one year. Developing a successful treatment option
to prevent prostate cancer in high risk patients will have a positive clinical
impact" said Mitchell Steiner MD, FACS, Vice-Chairman and CEO of GTx. "ACAPODENE
20mg showed a significant reduction in prostate cancer incidence in men who had
taken ACAPODENE for 12 months compared to placebo. ACAPODENE at all doses tested
was well tolerated. While we need to discuss the results of this study with the
FDA and continue analyzing the data, we are excited about these results and plan
to begin the Phase III trial later this year" said Dr. Steiner.
ABOUT THE STUDY
The Phase IIb clinical study was a 4-arm, double blind, placebo controlled, one
year treatment study at 64 clinical sites in the United States involving 514
patients. The four arms included in this study were 20mg, 40mg and 60mg of
ACAPODENE(TM) and placebo given orally once a day. There were approximately 125
patients per arm. The primary entry criterion for the study was men with biopsy
proven and confirmed high grade PIN. All patients were rebiopsied at 6 and 12
months from randomization.
ABOUT PIN
High grade PIN has been established as a premalignant lesion that has strong
potential to progress to prostate cancer. In the United States, approximately
1,300,000 prostate biopsies are performed annually to detect 230,000 new cases
of prostate cancer. There are approximately 115,000 new cases of high grade PIN
diagnosed each year, representing an estimated 9% of prostate biopsies.
Currently, patients diagnosed with high grade PIN have to be followed closely by
their urologist and are subjected to repeat prostate biopsies.
ABOUT ACAPODENE(TM)
ACAPODENE(TM) 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(TM)
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.
CONFERENCE CALL
There will be a conference call today at 10:00 a.m. Eastern Time to discuss
GTx's Phase II data for ACAPODENE(TM) for the prevention of prostate cancer in
high risk men. If you would like to participate in the call, please dial
800-915-4836 from the United States or Canada or 973-317-5319 from outside North
America. A playback of the call will be available today from approximately 12:00
p.m. Eastern Time through June 9, 2004 and may be accessed by dialing
800-428-6051 from the United States or Canada or 973-709-2089 from outside North
America. The rebroadcast code is 359201
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(TM), 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 STATEMENTS
This press release contains forward-looking statements, including, without
limitation, statements related to GTx's current and anticipated clinical trials
of ACAPODENE(TM) and its other 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 collaboration
partners will not 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. You should not place undue reliance on
these forward looking statements, which apply only as of the date of the press
release. 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.
###