FDA Grants Fast Track Designation for Amgen's AMG 531 and AMG 706 Experimental Therapies to Potentially Treat Life-Threatening Conditions
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Dec. 6, 2004--Amgen Inc.
(Nasdaq:AMGN), the world's largest biotechnology company, today
announced that the U.S. Food and Drug Administration (FDA) has granted
fast track designation for two of the company's experimental
therapies, AMG 531 and AMG 706. AMG 531 received orphan drug
designation in 2003.
"AMG 531 is Amgen's first peptibody and represents a new approach
to potentially treat immune thrombocytopenic purpura (ITP), an
autoimmune bleeding disorder. AMG 706, Amgen's first investigational
oral cancer therapy, may hold promise for various tumor types and is
currently in Phase 2 trials for the treatment of imatinib-resistant
gastrointestinal stromal tumors (GIST), a fatal cancer," said Beth
Seidenberg, M.D., chief medical officer and senior vice president of
global development at Amgen. "Fast track designation represents an
important step for both of these molecules and will help to streamline
development."
Under the FDA Modernization Act of 1997, fast track designation
allows the FDA to accept, on a rolling basis, portions of a marketing
application for review prior to the completion of the final
registrational package. Fast track designation may potentially
expedite the review of a drug that is intended for the treatment of a
serious life-threatening condition and demonstrates the potential to
address an unmet medical need for such a condition. The FDA orphan
drug designation provides marketing exclusivity incentives to
companies that develop drugs for diseases affecting less than 200,000
people in the United States.
The Use of AMG 531 in Immune Thrombocytopenia Purpura (ITP)
About Immune Thrombocytopenia Purpura (ITP)
Immune (idiopathic) thrombocytopenia purpura (ITP) is an
autoimmune bleeding disorder characterized by low levels of platelets
in the blood. ITP affects approximately 70,000 people in the U.S.
Current treatment of ITP involves reducing platelet destruction with
drugs (e.g., corticosteroids) that alter or suppress the immune system
or with surgical removal of the spleen (splenectomy), where the
antibody-tagged platelets are destroyed. Corticosteroids are
associated with side effects such as weight gain, rash and
exacerbation of diabetes and osteoporosis. Splenectomy and other
therapies used to treat ITP that suppress or modulate the immune
system can increase a patient's risk of infection. Since approximately
50 percent of patients do not respond to drug therapy and 40 percent
of patients do not respond to splenectomy, new therapies for ITP are
needed.
About AMG 531
As an investigational platelet growth factor, AMG 531 appears to
directly stimulate platelet production and may offer physicians a way
to shift the treatment focus from preventing platelet destruction to
boosting platelet production in patients with ITP. AMG 531 is an
engineered protein therapeutic developed by Amgen called a peptibody
that provides targeted action -- in this case, on the thrombopoietin
(TPO) receptor. Like TPO, AMG 531 binds to the TPO receptor and
stimulates precursor or "parent" cells of platelets, called
megakaryocytes, to mature into platelets.
In Phase 1 and 2 clinical studies recently reported at the 46th
Annual American Society of Hematology (ASH) meeting, AMG 531 appeared
to enhance platelet production in patients diagnosed with ITP. AMG 531
has been generally well tolerated. The most frequently reported
adverse events were bruising, nosebleeds, headache and mouth blisters.
The Use of AMG 706 in Imatinib-Resistant Gastrointestinal Stromal
Tumors (GIST)
About Gastrointestinal Stromal Tumors (GIST)
Gastrointestinal stromal tumors (GISTs) belong to a group of
cancers called soft tissue sarcomas. Sarcomas are a rare type of
cancer that can occur in connective tissues, bones, muscles, fat,
nerves, blood vessels and cartilage. GISTs start in the wall of the
stomach, small and large intestine and affect approximately 5,000 to
10,000 Americans annually. Since GIST are resistant to traditional
treatments such as chemotherapy and radiation, surgery is considered
the best way to initially treat GIST. However, many tumors cannot be
surgically removed because they are too large or have already spread
to other parts of the body before diagnosis. Since its approval in
2002, imatinib has been the mainstay of therapy for advanced or
metastatic GIST. However, no approved therapies exist for GIST
patients that no longer respond to imatinib.
About AMG 706
AMG 706 is an oral multi-kinase inhibitor (MKI) that works by
selectively targeting all known vascular endothelial growth factors
(VEGF), platelet derived growth factor (PDGF), Kit and Ret receptors.
Through the combined action of Kit and PDGF receptor inhibition,
coupled with potent VEGF receptor inhibition, AMG 706 potentially may
provide more than one mechanism of action in various cancers.
Activating mutations of Kit or PDGF receptors are critical to the
pathogenesis of more than 90 percent of GIST.
Early clinical data show signs of tumor regression with promising
preliminary safety data that may potentially allow for combination
therapy. AMG 706 is being evaluated as both a monotherapy and in
combination with other agents for the treatment of various cancers,
including imatinib-resistant GIST, non-small cell lung cancer and
colorectal cancer.
Patients and physicians can access www.amgentrials.com for more
information about ongoing AMG 531 and AMG 706 clinical trials.
About Amgen
Amgen is a global biotechnology company that discovers, develops,
manufactures and markets important human therapeutics based on
advances in cellular and molecular biology.
Forward-Looking Statement
This news release contains forward-looking statements that involve
significant risks and uncertainties, including those discussed below
and others that can be found in Amgen's Form 10-K for the year ended
December 31, 2003, and in Amgen's periodic reports on Form 10-Q and
Form 8-K. Amgen is providing this information as of the date of this
news release and does not undertake any obligation to update any
forward-looking statements contained in this document as a result of
new information, future events or otherwise.
No forward-looking statement can be guaranteed and actual results
may differ materially from those we project. Discovery or
identification of new product candidates or development of new
indications for existing products cannot be guaranteed and movement
from concept to product is uncertain; consequently, there can be no
guarantee that any particular product candidate or development of a
new indication for an existing product will be successful and become a
commercial product. Further, preclinical results do not guarantee safe
and effective performance of product candidates in humans. The
complexity of the human body cannot be perfectly, or sometimes, even
adequately modeled by computer or cell culture systems or animal
models. The length of time that it takes for us to complete clinical
trials and obtain regulatory approval for product marketing has in the
past varied and we expect similar variability in the future. We
develop product candidates internally and through licensing
collaborations, partnerships and joint ventures. Product candidates
that are derived from relationships may be subject to disputes between
the parties or may prove to be not as effective or as safe as we may
have believed at the time of entering into such relationship. Also, we
or others could identify side effects or manufacturing problems with
our products after they are on the market. In addition, sales of our
products are affected by the availability of reimbursement and the
reimbursement policies imposed by third party payors, including
governments, private insurance plans and managed care providers, and
may be affected by domestic and international trends toward managed
care and healthcare cost containment as well as possible U.S.
legislation affecting pharmaceutical pricing and reimbursement.
Government regulations and reimbursement policies may affect the
development, usage and pricing of our products.
In addition, we compete with other companies with respect to some
of our marketed products as well as for the discovery and development
of new products. We believe that some of our newer products, product
candidates or new indications for existing products, may face
competition when and as they are approved and marketed. Our products
may compete against products that have lower prices, established
reimbursement, superior performance, are easier to administer, or that
are otherwise competitive with our products. In addition, while we
routinely obtain patents for our products and technology, the
protection offered by our patents and patent applications may be
challenged, invalidated or circumvented by our competitors and there
can be no guarantee of our ability to obtain or maintain patent
protection for our products or product candidates. We cannot guarantee
that we will be able to produce commercially successful products or
maintain the commercial success of our existing products. Our stock
price may be affected by actual or perceived market opportunity,
competitive position, and success or failure of our products or
product candidates. Further, the discovery of significant problems
with a product similar to one of our products that implicate an entire
class of products could have a material adverse effect on sales of the
affected products and on our business and results of operations.
The scientific information discussed in this news release related
to our product candidates is preliminary and investigative. Such
product candidates are not approved by the U.S. Food and Drug
Administration (FDA), and no conclusions can or should be drawn
regarding the safety or effectiveness of the product candidates. Only
the FDA can determine whether the product candidates are safe and
effective for the use(s) being investigated. Further, the scientific
information discussed in this news release relating to new indications
for our products is preliminary and investigative and is not part of
the labeling approved by the U.S. Food and Drug Administration (FDA)
for the products. The products are not approved for the
investigational use(s) discussed in this news release, and no
conclusions can or should be drawn regarding the safety or
effectiveness of the products for these uses. Only the FDA can
determine whether the products are safe and effective for these uses.
Healthcare professionals should refer to and rely upon the
FDA-approved labeling for the products, and not the information
discussed in this news release.
EDITOR'S NOTE: An electronic version of this news release may be
accessed via our Web site at www.amgen.com. Journalists and media
representatives may sign up to receive all news releases
electronically at time of announcement by filling out a short form in
the Media section of the Web site.
CONTACT: Amgen Inc., Thousand Oaks
Trish Hawkins, 805-447-4587 (media)
Investor Relations, 805-447-1060
SOURCE: Amgen Inc.