ARIAD Announces 12-Month Data from Pivotal PACE Trial of Ponatinib in Heavily Pretreated Chronic-Phase CML Patients

  ARIAD Announces 12-Month Data from Pivotal PACE Trial of Ponatinib in
  Heavily Pretreated Chronic-Phase CML Patients

56 percent major cytogenetic response and 34 percent major molecular response

54th Annual Meeting of the American Society of Hematology

Business Wire

ATLANTA, & CAMBRIDGE, Mass. -- December 09, 2012

ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced twelve-month
follow-up data from the pivotal PACE trial of ponatinib, its investigational
BCR-ABL inhibitor, in heavily pretreated patients with resistant or refractory
chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute
lymphoblastic leukemia (Ph+ ALL). The study now shows that 56 percent of
chronic-phase CML patients in the trial, including 70 percent of patients with
a T315I mutation, achieved a major cytogenetic response (MCyR), the primary
end-point for chronic-phase CML patients.

The data are being featured today at 4:30 p.m. (ET) in an oral presentation at
the 54th Annual Meeting of the American Society of Hematology (ASH) being held
in Atlanta, Georgia. ARIAD filed for regulatory approval of ponatinib in the
third quarter of 2012 in the U.S. and in the E.U. based on clinical data from
the pivotal PACE trial.

“The 12-month results from the global PACE trial of ponatinib reinforce its
impressive anti-leukemic activity in heavily pretreated CML patients,
regardless of their mutation status or disease stage,” stated Jorge Cortes,
M.D., professor and deputy chair, Department of Leukemia, The University of
Texas M.D. Anderson Cancer Center, Houston, TX.

“Ponatinib demonstrated early responses in chronic-phase patients with
thirty-four percent of these patients achieving a major molecular response and
fifteen percent of those patients achieving a complete molecular response,” he
added. “Of particular importance, responses to ponatinib appear to be durable,
with 91 percent of chronic-phase CML patients projected to remain in major
cytogenetic response at one year.”

  *Trial Design

       *Efficacy data were reported at ASH on 444 treated patients in six
         pre-specified cohorts at 45 mg of ponatinib administered orally once
         daily, including 267 patients with chronic-phase CML. Findings were
         based on a minimum follow-up of 12 months in patients remaining on
       *Ninety-three percent of the patients in the trial had received at
         least two tyrosine kinase inhibitors prior to enrollment. Fifty-eight
         percent of the patients had received three or more tyrosine kinase
         inhibitors prior to enrollment.
       *Chronic-phase patients had bone marrow assessments approximately
         every three months for determination of cytogenetic response.

  *Chronic-phase CML patients evaluable for cytogenetic response (N=267)

       *Based on assessment of all evaluable chronic-phase patients in the
         trial, 56 percent (149 of 267) of patients achieved a MCyR, with 46
         percent achieving a complete cytogenetic response (CCyR). The median
         follow up of the chronic-phase CML patients is 15.3 months.
       *Of the 64 evaluable chronic-phase CML patients with the T315I
         mutation, 70 percent (45 of 64) of these patients achieved a MCyR,
         with 66 percent achieving a CCyR. The MCyR rate in evaluable
         chronic-phase patients without the T315I mutation was 51 percent (104
         of 203).
       *Thirty-four percent (91 of 267) of chronic-phase CML patients
         achieved a major molecular response (MMR).
       *Fifteen percent (39 of 267) of chronic-phase CML patients achieved a
         4.5-log reduction of BCR-ABL transcripts (MR4.5).

  *Responses in chronic-phase patients who had received only one prior TKI

       *There were 19 chronic-phase patients treated with ponatinib in the
         PACE trial who had previously received only one tyrosine kinase
         inhibitor (TKI).Thirteen of these patients had previously been
         treated with imatinib only, and six had previously received either
         dasatinib or nilotinib. Of these 19 patients, 84 percent (16 of 19)
         achieved a MCyR.

  *Safety profile (N=449)

       *The most common non-hematologic treatment-emergent adverse events in
         the PACE trial included rash (in 38% of patients), abdominal pain
         (38%), headache (35%), dry skin (35%), and constipation (34%), with
         the majority of these being grades 1 or 2 in severity.
       *The most common hematologic treatment-emergent adverse events were
         thrombocytopenia (42%), neutropenia (24%), and anemia (20%), which
         were primarily grades 3 or 4 in severity.
       *Pancreatitis and pneumonia were the most common non-hematologic
         treatment-emergent serious adverse events (5% each), followed by
         abdominal pain (4%), myocardial infarction (3%), congestive heart
         failure (3%), atrial fibrillation (3%), and pyrexia (3%). The most
         common hematologic serious adverse events were anemia, febrile
         neutropenia, and thrombocytopenia (3% each).

About Ponatinib

Internally discovered at ARIAD, ponatinib is an investigational BCR-ABL
inhibitor that also selectively inhibits certain other tyrosine kinases in
preclinical studies, including FLT3, RET, KIT, and the members of the FGFR,
PDGFR and VEGFR families of kinases.

The primary target for ponatinib is BCR-ABL, an abnormal tyrosine kinase that
is expressed in chronic myeloid leukemia (CML) and Philadelphia-chromosome
positive acute lymphoblastic leukemia (Ph+ ALL). Ponatinib was designed using
ARIAD’s computational and structure-based drug design platform to inhibit the
activity of BCR-ABL with high potency and broad specificity. Ponatinib targets
not only native BCR-ABL but also its isoforms that carry mutations that confer
resistance to treatment with existing tyrosine kinase inhibitors, including
the T315I mutation for which no effective therapy currently exists.

About CML and Ph+ ALL

CML is a cancer of the white blood cells that is diagnosed in approximately
5,000 patients each year in the United States. CML is a type of leukemia
characterized by the increased and unregulated growth of predominantly myeloid
cells in the bone marrow and the accumulation of these cells in the blood. The
genetic hallmark of CML is the Philadelphia chromosome, an abnormality
resulting in a fusion of the BCR and ABL genes. This is known as Philadelphia
chromosome-positive CML, or Ph+ CML.

Treatment of CML usually includes a targeted therapy, a tyrosine kinase
inhibitor (TKI) (e.g., imatinib, dasatinib or nilotinib), followed by
chemotherapy if the disease progresses. Ph+ ALL is a subtype of acute
lymphoblastic leukemia that carries the Ph+ chromosome that produces the fused
BCR-ABL gene. It is known to have a more aggressive course than CML and is
often treated with a combination of chemotherapy and TKIs. Because both of
these diseases express the BCR-ABL protein, this would render them potentially
susceptible to treatment with ponatinib.


ARIAD Pharmaceuticals, Inc. is an emerging global oncology company focused on
the discovery, development and commercialization of medicines to transform the
lives of cancer patients. ARIAD’s approach to structure-based drug design has
led to several internally discovered, molecularly targeted product candidates
for drug-resistant and difficult-to-treat cancers, including certain forms of
chronic myeloid leukemia and non-small cell lung cancer. For additional
information, visit

This press release contains “forward-looking statements” including, but not
limited to, statements relating to the updated clinical data for ponatinib,
the positive treatment effects of ponatinib over time and the timing of
regulatory filings for marketing approvals. 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.
For Investors
Kendra Adams, 617-503-7028
For Media
Liza Heapes, 617-621-2315
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