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ARIAD Announces Long-Term Molecular Response Data on Ponatinib



  ARIAD Announces Long-Term Molecular Response Data on Ponatinib

 51 percent of chronic-phase CML patients in Phase 1 trial and 34 percent in
             Phase 2 PACE trial achieve major molecular response

Business Wire

ATLANTA & CAMBRIDGE, Mass. -- December 10, 2012

ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced updated molecular
response data from its Phase 1 and pivotal Phase 2 trials of ponatinib, its
investigational BCR-ABL inhibitor, in heavily pretreated patients with
resistant or intolerant chronic myeloid leukemia (CML) or Philadelphia
chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The studies now
show that 51 percent of chronic-phase CML patients in the Phase 1 trial
achieved a major molecular response (MMR) with a median follow-up of 30
months, and 34 percent of chronic-phase patients achieved MMR in the PACE
trial with a median follow-up of 15 months.

These data are being featured today at 6:00 p.m. in two poster presentations
at the 54th Annual Meeting of the American Society of Hematology (ASH) being
held in Atlanta, Georgia.

Molecular response is a measurement of blood levels of the transcript product
of the BCR-ABL oncogene. MMR is defined as a value less than or equal to 0.1%
on the accepted International Scale. All patient samples were evaluated for
molecular response at a single central laboratory (Molecular MD) using a
standardized assay. MMR is a secondary efficacy endpoint for chronic-phase CML
patients in ARIAD’s Phase 1 and pivotal Phase 2 PACE trials of ponatinib.

Phase 1 Trial MMR Rates in Chronic-Phase CML Patients

The ongoing Phase 1 dose-escalation study of ponatinib enrolled 81 patients
with resistant or refractory hematologic cancers, including 43 patients with
chronic-phase CML. Sixty-one percent of the chronic-phase CML patients in this
study had failed at least three prior tyrosine kinase inhibitors (TKI).

  * With a median follow-up of 30 months, 51 percent (22 of 43) of patients
    with chronic-phase CML enrolled in the study achieved MMR, including 75
    percent (9 of 12) who had the T315I mutation, which is the most common
    mutation among resistant patients.

  * The median time to MMR was 5.6 months, and the median duration of MMR in
    chronic-phase CML has not yet been reached. At the time of analysis, 21 of
    22 patients who achieved MMR remained in the study and continued to
    receive ponatinib.
  * Molecular response rates increased over time with nine percent (4 of 43)
    of chronic-phase CML patients achieving MMR by 3 months and 51 percent (22
    of 43) achieving MMR overall. Patients continued to achieve MMRs after 12
    months of follow-up.
  * Thirty-three percent (14 of 43) of chronic-phase CML patients achieved MR4
    (4-log reduction in BCR-ABL transcripts).
  * The most common non-hematologic treatment-related adverse events in all
    patients in this trial included rash (42%), arthralgia (20%), increased
    lipase (20%), fatigue (19%) and dry skin (19%), with the majority of these
    being grades 1 or 2 in severity. The most common hematologic
    treatment-related adverse events included thrombocytopenia (34%),
    neutropenia (14%) and anemia (12%), with thrombocytopenia and neutropenia
    being primarily grades 3 or 4 in severity.

PACE Trial MMR Rates in Chronic-Phase CML Patients

The ongoing pivotal Phase 2 PACE trial enrolled 449 patients with chronic
myeloid leukemia (CML) or Philadelphia-positive acute lymphoblastic leukemia
(Ph+ ALL), who are resistant or intolerant to dasatinib or nilotinib or who
have the T315I mutation.

  * With a median follow up of 15 months, 34 percent (91 of 267) of
    chronic-phase CML patients achieved MMR; the reported prior MMR rate to
    their most recent TKI was three percent.
  * Fifteen percent (39 of 267) of patients achieved a 4.5-log reduction of
    BCR-ABL transcripts (MR4.5).
  * Fifty-three percent (10 of 19) of chronic-phase patients who failed only
    one prior approved TKI achieved MMR with ponatinib.
  * The median time to MMR among responders was 6 months. MMR was durable with
    81 percent of patients estimated to remain in MMR at 12 months (by
    Kaplan-Meier analysis). Median duration of MMR among chronic-phase CML
    patients has not yet been reached.

  * The most common non-hematologic treatment-emergent adverse events in all
    patients in the PACE trial included rash (38%), 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.

About ARIAD

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 http://www.ariad.com.

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.

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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