ARIAD Presents New Preclinical Data Showing Ponatinib Overcomes Resistant Mutations in KIT, an Oncogenic Driver of

  ARIAD Presents New Preclinical Data Showing Ponatinib Overcomes Resistant
  Mutations in KIT, an Oncogenic Driver of Gastrointestinal Stromal Tumors

AACR Annual Meeting 2013

Business Wire

CAMBRIDGE, Mass. & WASHINGTON -- April 09, 2013

ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced the presentation of
preclinical data on ponatinib (Iclusig™) at the American Association for
Cancer Research (AACR) Annual Meeting 2013, in Washington. The study,
“Ponatinib, a pan-BCR-ABL inhibitor, potently inhibits key activating and
drug-resistant KIT mutants found in GIST,” shows that ponatinib inhibits
activated and mutated forms of KIT, a clinically proven oncogenic driver found
in approximately 80 percent of patients with gastrointestinal stromal tumors
(GIST).

The structure of Iclusig(TM) (ponatinib) bound to KIT, an oncogenic driver
found in approximately 80 ...

The structure of Iclusig(TM) (ponatinib) bound to KIT, an oncogenic driver
found in approximately 80 percent of patients with gastrointestinal stromal
tumors (GIST). (Photo: Business Wire)

According to the American Cancer Society, approximately 4,000 to 5,000 people
develop 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 mutations. ARIAD plans to begin a
Phase 2 trial of Iclusig in patients with GIST in the second quarter of 2013.

“ARIAD’s development program for Iclusig focuses on its continued evaluation
in patients with CML and exploration of its activity against other clinically
validated targets. For the first time we were able to solve the x-ray crystal
structure of ponatinib bound to KIT, rationalizing its activity in advance of
the Phase 2 trial,” said Timothy P. Clackson, Ph.D., president of research and
development and chief scientific officer at ARIAD. “These data provide
important support for the use of Iclusig in patients with GIST who have become
resistant to prior tyrosine kinase inhibitor therapy.”

The research conducted by ARIAD scientists used a broad panel of cell lines
harboring mutant forms of KIT. Ponatinib showed potent activity against
activated forms of KIT and against a range of secondary mutations that confer
resistance to imatinib and/or sunitinib. These include mutations at the T670I
gatekeeper residue and mutations in the activation loop (A-loop), common
mutations found in patients who have failed currently available therapies.

About ARIAD

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.comor follow ARIAD on
Twitter (@ARIADPharm).

This press release contains “forward-looking statements” including, but not
limited to, updates on preclinical developments for our product candidates.
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.

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Contact:

ARIAD Pharmaceuticals, Inc.
For Investors
Kendra Adams, 617-503-7028
Kendra.adams@ariad.com
or
For Media
Liza Heapes, 617-621-2315
Liza.heapes@ariad.com
 
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