EXHIBIT 99.1
ARIAD Announces Agreement with
FDA on Special Protocol Assessment for Phase 3 Clinical Trial of Oral
Deforolimus
Progression-Free Survival is the Primary Endpoint of Phase 3 Trial
in Metastatic Soft-tissue and Bone Sarcomas
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sept. 10, 2007--ARIAD
Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced that it has
reached agreement on a Special Protocol Assessment (SPA) with the U.S.
Food and Drug Administration (FDA) for its global Phase 3 trial of
oral deforolimus in patients with metastatic sarcomas. Based on the
SPA, progression-free survival (PFS) will be the primary endpoint of
the Phase 3 trial, and overall survival will be a secondary endpoint.
The Company expects to begin patient enrollment in the trial later
this month.
The SPA is a written agreement between the trial's sponsor and the
FDA regarding the design, endpoints, and planned conduct and analysis
of a trial to be used in support of regulatory approval. The European
Medicines Agency (EMEA) has provided protocol advice consistent with
that of the FDA regarding the Phase 3 trial design as part of its
Protocol Assistance program. ARIAD and Merck & Co., Inc. have a global
collaboration to jointly develop and commercialize deforolimus for use
in cancer.
"We are extremely pleased with the outcome of our positive and
collaborative discussions with the FDA review staff regarding the
design of our Phase 3 trial for oral deforolimus in metastatic
sarcomas. Working with leading sarcoma experts and our clinical
investigators, we were able to successfully resolve the open issues
regarding the trial's endpoints and reach agreement with the Agency on
PFS as the primary endpoint," said Camille Bedrosian, M.D., chief
medical officer of ARIAD.
ARIAD and Merck plan to conduct a Phase 3 trial of oral
deforolimus in patients with metastatic soft-tissue and bone sarcomas
following a favorable response to chemotherapy - a period when
continued treatment with traditional chemotherapeutic agents has not
been established to provide additional clinical benefit. Thus, absent
new alternatives, patients would generally not receive other cancer
therapies.
This double-blind trial is designed to evaluate approximately 650
patients who will be randomized (1:1) to oral deforolimus or placebo
at approximately 125 sites. The trial is 90% powered to detect a 33%
increase in median PFS (corresponding to a hazard ratio of 0.75)
comparing the oral deforolimus arm with the placebo arm. Two interim
analyses are included. Complete patient enrollment and the second
interim analysis are expected to take place within approximately two
years of the first patient being enrolled.
Pierre F. Dodion, M.D., senior vice president, oncology of ARIAD,
added, "FDA agreement on our overall Phase 3 trial design, patient
population and endpoints, as well as our newly established partnership
with Merck represent important achievements for the global development
of deforolimus."
About Sarcoma
Sarcomas are a group of aggressive cancers of connective tissue of
the body for which there are currently limited treatment options.
Sarcomas can arise anywhere in the body and are divided into two main
groups - bone tumors and soft-tissue sarcomas. They are further
subdivided based on the type of cell or tissue from which the tumor
developed. There are approximately 12,000 new cases of sarcoma
diagnosed each year in the United States and approximately 100,000
sarcoma patients overall in the United States. More information about
sarcomas is available at http://www.curesarcoma.org and at
http://www.sarcoma.net/facts/htm.
About Deforolimus
ARIAD's lead product candidate, deforolimus, is a novel
small-molecule inhibitor of the protein mTOR, a "master switch" in
cancer cells. Blocking mTOR creates a starvation-like effect in cancer
cells by interfering with cell growth, division, metabolism, and
angiogenesis. Patient enrollment has been completed in multiple Phase
1 and 2 clinical trials of deforolimus in patients with solid tumors
and hematologic cancers. The global Phase 3 trial of oral deforolimus
in metastatic soft-tissue and bone sarcomas is the subject of a
Special Protocol Assessment with the U.S. Food and Drug Administration
(FDA). Deforolimus has been designated both as a fast-track product
and an orphan drug by the FDA and as an orphan drug by the European
Medicines Agency for the treatment of sarcomas. ARIAD has a global
partnership with Merck & Co., Inc. to develop and commercialize
deforolimus in multiple cancer indications. ARIAD also is
collaborating with Medinol Ltd. to develop stents and other medical
devices that deliver deforolimus to prevent reblockage at sites of
vascular injury following stent-assisted angioplasty.
About ARIAD
ARIAD is engaged in the discovery and development of breakthrough
medicines to treat cancer by regulating cell signaling with small
molecules. ARIAD is developing a comprehensive approach to patients
with cancer that addresses the greatest medical need - aggressive and
advanced-stage cancers for which current treatments are inadequate.
ARIAD has a global partnership with Merck & Co., Inc. to develop and
commercialize deforolimus, ARIAD's lead cancer product candidate.
Medinol Ltd. is also developing stents and other medical devices that
deliver deforolimus to prevent reblockage at sites of vascular injury
following stent-assisted angioplasty. ARIAD has an exclusive license
to pioneering technology and patents related to certain NF-(kappa)B
treatment methods, and the discovery and development of drugs to
regulate NF-(kappa)B cell-signaling activity, which may be useful in
treating certain diseases. Additional information about ARIAD can be
found on the web at http://www.ariad.com.
This press release contains "forward-looking statements,"
including statements related to the design, conduct and timing of the
Phase 3 clinical trial of deforolimus in metastatic sarcoma.
Forward-looking statements are based on management's expectations and
are subject to certain factors, risks and uncertainties that may cause
actual results, outcome of events, timing and performance to differ
materially from those expressed or implied by such statements. These
risks and uncertainties include, but are not limited to, the costs
associated with our research, development, manufacturing and other
activities, the conduct and results of pre-clinical and clinical
studies of our product candidates, difficulties or delays in obtaining
regulatory approvals to market products resulting from our development
efforts, our reliance on partners, including Medinol and Merck, and
other key parties for the successful development, manufacturing and
commercialization of products, the adequacy of our capital resources
and the availability of additional funding, patent protection and
third-party intellectual property claims relating to our and any
partner's product candidates, the timing, scope, cost and outcome of
legal and patent office proceedings concerning our NF-(kappa)B patent
portfolio, the potential acquisition of or other strategic transaction
regarding the minority stockholders' interests in our 80%-owned
subsidiary, ARIAD Gene Therapeutics, Inc., future capital needs, key
employees, markets, economic conditions, prices, reimbursement rates,
competition and other factors detailed in the Company's public filings
with the U.S. Securities and Exchange Commission. The information
contained in this document is believed to be current as of the date of
original issue. The Company does not intend to update any of the
forward-looking statements after the date of this document to conform
these statements to actual results or to changes in the Company's
expectations, except as required by law.
CONTACT: ARIAD Pharmaceuticals, Inc.
Edward M. Fitzgerald, 617-621-2345
or
Andrea L. Johnston, 910-681-1088