ARIAD Announces Initiation of Phase 2 Trial of Ponatinib in Patients with
Gastrointestinal Stromal Tumors
CAMBRIDGE, Mass. -- June 11, 2013
ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced the initiation of
the Phase 2 trial of Iclusig^® (ponatinib) in adult patients with metastatic
and/or unresectable gastrointestinal stromal tumors (GIST). The trial is
designed to provide initial clinical data evaluating the efficacy and safety
of ponatinib in patients with GIST following failure of prior tyrosine kinase
inhibitor (TKI) therapy.
“This trial represents an important step in ponatinib’s expanded clinical
development beyond Philadelphia-positive leukemias and into solid tumors. KIT
is a clinically validated oncogenic target for GIST and is known to drive
approximately 85 percent of these cancers. In preclinical studies ponatinib
was highly potent against activated KIT and most of its resistance mutants,”
stated Timothy P. Clackson, Ph.D., president of research and development and
chief scientific officer of ARIAD. “Patients with GIST have limited treatment
options following development of resistance to TKI therapy, and we are pleased
to work with leading clinical research sites to investigate ponatinib as a new
treatment for these patients.”
The Phase 2 trial is an open-label, multicenter trial to evaluate the efficacy
and safety of ponatinib in patients with metastatic and/or unresectable GIST
after prior failure of at least one TKI. Patients whose tumors have an
activating mutation in exon 11 of cellular KIT, the primary driver for
patients with GIST and KIT mutations, will be enrolled in Cohort A. Patients
whose tumors have other activating mutations will be enrolled into Cohort B.
The trial will be conducted at three investigational sites in the United
States: Dana Farber Cancer Institute, Fox Chase Cancer Center and Oregon
Health Science University.
A total of approximately 45 patients will be enrolled in the trial, with at
least 30 patients in Cohort A. Patients will receive the same dose of
ponatinib, 45 mg administered orally once daily, as used in patients with
The Clinical Benefit Rate (CBR), measuring disease control and defined as the
composite of complete response, partial response, and stable disease lasting
16 weeks or more, is the primary endpoint for the trial and will be analyzed
in Cohort A. Secondary endpoints will include progression-free survival,
objective response rate, overall survival, safety and pharmacokinetic
For more information about this trial, patients and physicians should call the
U.S. toll-free number 1-877-621-2302 or the international number
1-617-621-2302, or e-mail inquiries to Clinicaltrials@ariad.com.
According to the American Cancer Society, approximately 4,000 to 5,000 people
develop gastrointestinal stromal tumors (GIST) each year in the United States.
Approved agents for the treatment of patients with GIST include imatinib for
newly diagnosed patients, sunitinib for patients in whom imatinib has failed,
and regorafenib for patients who have failed imatinib and sunitinib. Patients
can develop resistance to any of these therapies by acquiring secondary KIT
About Iclusig^® (ponatinib) tablets
Iclusig is a kinase inhibitor indicated for the treatment of adult patients
with chronic phase, accelerated phase, or blast phase chronic myeloid leukemia
(CML) that is resistant or intolerant to prior tyrosine kinase inhibitor
therapy or Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+
ALL) that is resistant or intolerant to prior tyrosine kinase inhibitor
therapy. This indication is based upon response rate. There are no trials
verifying an improvement in disease-related symptoms or increased survival
Important Safety Information for Iclusig^® (ponatinib)
WARNING: ARTERIAL THROMBOSIS and HEPATOTOXICITY
Arterial Thrombosis: Cardiovascular, cerebrovascular, and peripheral vascular
thrombosis, including fatal myocardial infarction and stroke have occurred in
Iclusig-treated patients. In clinical trials, serious arterial thrombosis
occurred in eight percent of Iclusig-treated patients. Interrupt and consider
discontinuation of Iclusig in patients who develop arterial thrombotic events.
Hepatotoxicity: Hepatotoxicity, liver failure and death have occurred in
Iclusig-treated patients. Monitor hepatic function prior to and during
treatment. Interrupt and then reduce or discontinue Iclusig for
Please visit www.iclusig.com for full prescribing information, including boxed
ARIAD Pharmaceuticals, Inc., headquartered in Cambridge, Massachusetts and
Lausanne, Switzerland, is an integrated global oncology company focused on
transforming the lives of cancer patients with breakthrough medicines. ARIAD
is working on new medicines to advance the treatment of various forms of
chronic and acute leukemia, lung cancer and other difficult-to-treat cancers.
ARIAD utilizes computational and structural approaches to design
small-molecule drugs that overcome resistance to existing cancer medicines.
For additional information, visit http://www.ariad.com or follow ARIAD on
This press release contains “forward-looking statements” including, but not
limited to, statements relating to the potential that ponatinib is an
effective tyrosine kinase inhibitor against KIT in patients with
gastrointestinal stromal tumors. 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,
preclinical data and early-stage clinical data that may not be replicated in
later-stage clinical studies, the costs associated with our research,
development, manufacturing and other activities, the conduct, timing and
results of pre-clinical and clinical studies of our product candidates, the
adequacy of our capital resources and the availability of additional funding,
and other factors detailed in the Company's public filings with the U.S.
Securities and Exchange Commission. The information contained in this press
release 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.
ARIAD Pharmaceuticals, Inc.
Kendra Adams, 617-503-7028
Liza Heapes, 617-621-2315
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