Exhibit 13.1
[GTX(TM) LOGO]
The Men's Health Biotech Company
SETTING THE PACE IN MEN'S HEALTH
[PICTURE OF MAN RUNNING]
2004 ANNUAL REPORT
EXCEEDING MILESTONES
Company Profile
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 and oncology. GTx's drug discovery and development
programs are focused on small molecules that selectively modulate the effects of
estrogens and androgens, two essential classes of hormones. GTx has a marketed
product and four clinical programs. The marketed product is FARESTON(R)
(toremifene citrate 60mg) tablets for the treatment of metastatic breast cancer.
The company is developing the same active compound, toremifene citrate, as
ACAPODENE(R) in two of its clinical programs in men: (1) a pivotal Phase III
clinical trial for the prevention of prostate cancer in high risk men and (2) a
pivotal Phase III clinical trial for the treatment of serious side effects of
androgen deprivation therapy for advanced prostate cancer. In its third clinical
program, GTx and its partner, Ortho Biotech Products, L.P., a subsidiary of
Johnson & Johnson, are developing andarine, a SARM, which they are working to
progress to Phase II clinical testing in the second half of this year. GTx is
developing its second SARM, ostarine, for andropause and other chronic
conditions related to aging, including sarcopenia. In addition, GTx has an
extensive preclinical pipeline generated from its own discovery program which
includes the specific product candidates, prostarine, a SARM for benign
prostatic hyperplasia (BPH), and andromustine, an anticancer drug, for hormone
refractory prostate cancer.
PASSION TO SUCCEED
To Our Shareholders, 2004 was indeed a defining year for GTx. Our company made
the important transition from a privately held business, incorporated in 1997,
to a publicly traded company listed on the NASDAQ national market under the
stock symbol, GTXI. Our initial public offering on February 3, 2004 raised
approximately $70 million which allowed us to advance our late stage lead
product candidate, ACAPODENE(R) (toremifene citrate), into two pivotal Phase III
programs for two separate large men's health indications and to fund the
development of our home grown selective androgen receptor modulator (SARM)
program. The lead SARM product candidate, andarine, completed four separate
Phase I clinical trials. We also advanced our second SARM compound, ostarine,
into Phase I clinical testing for andropause. With four clinical programs and a
deep pipeline, GTx has a strong future.
Achieving our milestones and positioning GTx for the future was the theme of
2004. Our lead product candidate ACAPODENE(R), a non-steroidal selective
estrogen receptor modulator (SERM) licensed from Orion Corporation, is being
developed for two large indications in men's health: 1) the prevention of
prostate cancer in high risk men and 2) the treatment of the serious side
effects of androgen deprivation therapy for advanced prostate cancer.
In June 2004, we announced positive results from our Phase IIb clinical trial
for the prevention of prostate cancer in high risk men. 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). This
large, prospective study of patients with high grade PIN confirmed that these
men are at high risk for developing prostate cancer, as over 31% of patients
taking a placebo were diagnosed with prostate cancer by one year. ACAPODENE(R)
20 mg reduced the prostate cancer incidence for all patients enrolled in the
studY receiving treatment by almost 22%, and those patients completing a full
year of therapy had a 46% reduction in the incidence of prostate cancer. This
study has been recognized by peer review scientific meetings that include the
4th International Prostate Cancer Congress July 2004, American Association for
Cancer Research (AACR) Frontiers in Cancer Prevention meeting October 2004,
American Society of Clinical Oncology (ASCO) May 2005, and the American
Urological Association (AUA) May 2005. In January 2005, we kicked off a
multi-center pivotal Phase III clinical trial for the prevention of prostate
cancer in these high risk men.
We also entered into collaboration agreements with three diagnostic companies,
Hybritech, Inc., a subsidiary of Beckman Coulter Inc., diaDexus, Inc., and
Tessera, Inc., to develop a blood or urine based test for high grade PIN. This
test should allow physicians to detect high grade PIN in men who may unknowingly
harbor these precancerous lesions of the prostate and expand the already growing
number of men who may benefit from ACAPODENE(R) in the future. We plan to
establish additional diagnostic relationships and continue to assist these
companies by providing clinical samples to develop and validate a potential high
grade PIN test.
STEADFAST IN OUR STRATEGY
Our second clinical program is testing ACAPODENE(R) 80mg in a multi-center,
pivotal Phase III clinical trial for another important indication, the treatment
of the serious side effects of androgen deprivation therapy for advanced
prostate cancer. This trial will enroll 1,200 patients in over 130 clinical
sites. We expect enrollment to be completed in the third quarter of 2005, and we
plan to announce the interim analysis of bone mineral density data in the first
200 patients during the second half of 2005.
Toremifene is approved by the FDA as a 60mg dose for the treatment of metastatic
breast cancer and is marketed under the tradename, FARESTON(R). FARESTON(R) has
the same active ingredient, toremifene citrate, AS ACAPODENE(R), but the dosage
form is different. Following the positive results from our proof of concept
trials for ACAPODENE(R) , GTx had the confidence to strategically acquire
FARESTON(R). In December 2004, WE signed an agreement with Orion Corporation to
acquire FARESTON(R) and all of the remaining rights to toremifene, making GTx
the sole licensee for toremifene for all indications in the United States and
for alL indications except breast cancer in all other countries. With this
agreement, we believe GTx is better positioned to maximize the commercial
potential of our toremifene-based product candidates.
Our GTx scientists were the first to create and clinically develop selective
androgen receptor modulators. SARMs are small molecules that selectively
modulate androgen receptors. In men, we believe SARMs may be able to mimic
testosterone's beneficial action in bone, muscle and brain while blocking
testosterone's harmful activity in the prostate. Because of these properties, we
believe SARMs can be developed to treat a range of medical conditions. In March
2004, we entered into a worldwide collaboration, license and co-promotion
agreement with Ortho Biotech, a subsidiary of Johnson & Johnson, for the
development of our lead SARM, andarine. This collaboration scientifically
validates our SARM technology and gives us access to the world class research
and marketing capabilities of Ortho Biotech, a leader in cancer supportive care.
Through the efforts of GTx and Ortho Biotech, andarine is poised to become the
first - in-class drug in cancer supportive care for the treatment of cancer
cachexia. We are planning for Phase II clinical trials for andarine in the
second half of 2005.
While our collaboration with Ortho Biotech provides favorable long term
financial value for GTx, we have retained full rights to the rest of our SARM
portfolio to allow our shareholders to maximally benefit from the future
potential of our extensive SARM product pipeline. For example, GTx initiated a
Phase I clinical trial for our second SARM product candidate, ostarine, in
February 2005. Ostarine is being developed for andropause as well as other
conditions associated with aging. We are especially excited about ostarine
because it continues to demonstrate our strength in drug discovery. We expect to
be in a Phase II clinical trial for ostarine by the end of 2005. GTx has
established itself as a leader in SARM technology.
ALL TO ENHANCE LIVES
The recent advent of drug therapies to treat common male diseases such as benign
prostatic hyperplasia (BPH) and erectile dysfunction has transformed the field
of urology from a predominantly surgical specialty into a medical specialty. A
number of effective drugs are now available to urologists to delay, and in some
instances, replace surgery as a preferred treatment. GTx is committed to being a
leader in this emerging focus on men's health. We have built an organization
that is unparalleled in the biotech industry. We have multiple product
candidates, including ACAPODENE(R) which is currently being tested in pivotal
Phase III trials for two separate indications, and our SARM portfolio is a
testament to our proven ability to bring product candidates from our discovery
laboratories to the clinic. We have an established track record of being
fiscally responsible by managing and executing our business strategy with less
capital than our peers. GTx is different. We are committed to excellence,
passionate about exceeding our goals and steadfast in building a fundamentally
strong business to create long term shareholder value.
[Picture of Mitchell S. Steiner, M.D., F.A.C.S.]
Mitchell S. Steiner, M.D., F.A.C.S.
Vice-Chairman and Chief Executive Officer
Product Candidate Pipeline
GTx, Inc., the men's health biotech company, is a biopharmaceutical company
dedicated to the discovery, development and commercialization of therapeutics
primarily related to the treatment of serious men's health conditions and
oncology.
Clinical
---------------------------
Phase Phase Phase
Program Indication Drug Class Preclinical I II III Marketed
- ------------ --------------------- ---------- ----------- ----- ----- ----- --------
Advanced breast
FARESTON(R) cancer SERM --------------------------------------------------->
Side effect of
ACAPODENE(R) androgen deprivation SERM ------------------------------------------>
therapy
Reduction in the
ACAPODENE(R) incidence of prostate SERM ------------------------------------------>
cancer in high risk
men
andarine Cancer cachexia SARM --------------------->
ostarine Andropause SARM --------------------->
Hormone refractory
andromustine prostate cancer Anticancer ---------->
[GTX(TM) LOGO]
The Men's Health Biotech Company
BOARD:
J. R. HYDE, III
Chairman
GTx, Inc.
MITCHELL S. STEINER, M.D., F.A.C.S.
Vice-Chairman and Chief Executive Officer
GTx, Inc.
MARC S. HANOVER
President and Chief Operating Officer
GTx, Inc.
ANDREW M. CLARKSON
Former Chairman of Finance Committee
AutoZone, Inc
J. KENNETH GLASS
Chairman of the Board, President and Chief Executive Officer
First Horizon National Corporation and First Tennessee Bank National Association
ROSEMARY MAZANET, M.D., PH.D.
Chief Executive Officer and President
Breakthrough Therapeutics, LLC
JOHN H. PONTIUS
President
Pittco Management, LLC
TIMOTHY R.G. SEAR
Chairman of the Board
Alcon, Inc.
Management:
MITCHELL S. STEINER, M.D., F.A.C.S.
Vice-Chairman and Chief Executive Officer
MARC S. HANOVER
President and Chief Operating Officer
HENRY P. DOGGRELL
Vice President, General Counsel and Secretary
MARK E. MOSTELLER
Vice President, Chief Financial Officer
K. GARY BARNETTE, PH.D.
Vice President, Clinical Research and Development
JAMES T. DALTON, PH.D.
Vice President, Preclinical Research and Development
GREGORY A. DEENER
Vice President, Marketing and Sales
TRANSFER AGENT CORPORATE COUNSEL
EquiServe, Inc. Bass, Berry & Sims, PLC
Blue Hills Office Park 100 Peabody Ave.
150 Royall Street Suite 950
Canton, MA 02021 Memphis, Tennessee 38103
PHONE: 781.575.2000 PHONE: 901.543.5900
FAX: 781.575.2500 FAX: 901.543.5999
CORPORATE WEBSITE: http://www.equiserve.com CORPORATE WEBSITE: www.bassberry.com
LEGAL ACCOUNTANTS
Cooley Godward LLP Ernst and Young
5 Palo Alto Square 6410 Poplar Avenue
3000 El Camino Real Suite 500
Palo Alto, CA 94306 Memphis, TN 38119
PHONE: 650.843.5000 PHONE: 901.526.1000
FAX: 650.849.7400 FAX: 901.543.5999
CORPORATE WEBSITE: http://www.cooley.com CORPORATE WEBSITE: http://www.ey.com
[GTX(TM) LOGO]
The Men's Health Biotech Company
GTx. Inc.
3 North Dunlap
Memphis, TN 38163
901.523.9700
901.523.9772 fax
www.gtxinc.com