ARIAD Announces Marketing Authorization for Iclusig® (ponatinib) in the European Union

  ARIAD Announces Marketing Authorization for Iclusig® (ponatinib) in the
  European Union

Business Wire

CAMBRIDGE, Mass. & LAUSANNE, Switzerland -- July 2, 2013

ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) today announced that the European
Commission (EC) has granted a marketing authorization for Iclusig^®
(ponatinib) as an orphan medicinal product for two indications:

  *The treatment of adult patients with chronic phase, accelerated phase or
    blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib
    or nilotinib; who are intolerant to dasatinib or nilotinib and for whom
    subsequent treatment with imatinib is not clinically appropriate; or who
    have the T315I mutation, and
  *The treatment of adult patients with Philadelphia-chromosome positive
    acute lymphoblastic leukaemia (Ph+ ALL) who are resistant to dasatinib;
    who are intolerant to dasatinib and for whom subsequent treatment with
    imatinib is not clinically appropriate; or who have the T315I mutation.

“We are delighted by the rapid approval of Iclusig in Europe and will now work
closely with the national health authorities to make Iclusig available to
Philadelphia-positive leukaemia patients as quickly as possible,” stated
Harvey J. Berger, M.D., chairman and chief executive officer of ARIAD. “The
clinical development of Iclusig involved many leukaemia experts throughout
Europe, and we want to recognize their critical roles in bringing Iclusig to
patients with resistant or intolerant CML and Ph+ ALL in the EU.”

The most common (>1%) serious adverse reactions for Iclusig were pancreatitis,
abdominal pain, pyrexia, anaemia, febrile neutropenia, decrease in platelet
count and neutrophil count, pancytopenia, myocardial infarction, diarrhea, and
increased lipase. The most common (≥20%) adverse reactions of any severity
were decrease in platelet count, rash, dry skin, and abdominal pain. There
were no new safety signals observed with Iclusig when compared to the other
approved products from the same class (BCR-ABL inhibitors).

ARIAD was granted accelerated assessment by the Committee for Medicinal
Products for Human Use for the Iclusig marketing authorization application.
The aim of accelerated assessment is to expedite the review process for new
medicines that address a major public-health interest. Accelerated assessment
is reserved for innovative products that respond to an unmet medical need and
that are expected to have a major impact on medical practice.

“Iclusig’s approval in Europe will offer CML patients, some of whom have run
out of other treatment options, a new opportunity to improve their clinical
outcome,” said Stephen G. O’Brien, M.D., Ph.D., Professor of Haematology at
the Northern Institute for Cancer Research at Newcastle University, United
Kingdom. “We have seen deep, durable responses from this once daily, oral
treatment, and it would appear that Iclusig is a very useful new medicine for
CML and Ph+ ALL patients who have become resistant to, or intolerant of, other
TKIs.”

The EC decision was based on results from the pivotal Phase 2 PACE (Ponatinib
Ph+ ALL and CML Evaluation) trial in patients with CML or Ph+ ALL who were
resistant or intolerant to prior tyrosine kinase inhibitor (TKI) therapy, or
who had the T315I mutation of BCR-ABL. Iclusig had robust anti-leukemic
activity achieving a major cytogenetic response (MCyR) in 54 percent of
chronic-phase CML patients and in 70 percent of patients with the T315I
mutation. MCyR was the primary endpoint of the PACE trial for chronic-phase
patients.

In patients with advanced disease, 58 percent of accelerated-phase CML
patients, 31 percent of blast-phase CML patients and 41 percent of Ph+ ALL
patients achieved a major hematologic response (MaHR) with Iclusig. MaHR was
the primary endpoint in the trial for patients with advanced disease.

CML is a cancer of the white blood cells that is diagnosed in approximately
7,000 patients each year in Europe^[1]. CML and Ph+ ALL patients treated with
TKIs can develop resistance or intolerance over time to these therapies.
Iclusig is a targeted cancer medicine discovered and developed at ARIAD. It
was designed by ARIAD scientists using ARIAD’s platform of computational
chemistry and structure-based drug design to inhibit BCR-ABL, including
drug-resistant mutants that arise during treatment. Iclusig is the only TKI
that has received an approval in Europe for an indication that includes CML
and Ph+ ALL patients with the T315I mutation.

“For European patients with CML and Philadelphia-positive ALL who have been
failed by prior therapy, the accelerated approval of Iclusig is very positive
news and underscores the need for new medicines,” said Sandy Craine, director
of The CML Support Group in the United Kingdom. “This approval is a
significant step in giving hope to patients coping with CML and
Philadelphia-positive ALL.”

To view the Iclusig Summary of Medicinal Product Characteristics, click here.

About CML and Ph+ ALL

CML is characterized by an excessive and unregulated production of white blood
cells by the bone marrow due to a genetic abnormality that produces the
BCR-ABL protein. After a chronic phase of production of too many white blood
cells, CML typically evolves to the more aggressive phases referred to as
accelerated phase and blast crisis. Ph+ ALL is a subtype of acute
lymphoblastic leukaemia that carries the Ph+ chromosome that produces BCR-ABL.
It has a more aggressive course than CML and is often treated with a
combination of chemotherapy and tyrosine kinase inhibitors. The BCR-ABL
protein is expressed in both of these diseases.

About Iclusig (ponatinib)

Iclusig is a kinase inhibitor. The primary target for Iclusig is BCR-ABL, an
abnormal tyrosine kinase that is expressed in chronic myeloid leukaemia (CML)
and Philadelphia-chromosome positive acute lymphoblastic leukaemia (Ph+ ALL).
Iclusig was designed using ARIAD’s computational and structure-based drug
design platform specifically to inhibit the activity of BCR-ABL. Iclusig
targets both native BCR-ABL and isoforms that carry mutations that confer
resistance to treatment, including the T315I mutation, which is a common
mutation among resistant patients.

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 leukaemia, 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 regulatory developments in Europe. 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, our ability to successfully launch, commercialize and generate
profits from sales of Iclusig; competition from alternative therapies, our
ability to obtain approval for Iclusig outside of the United States and the
European Union and/or in additional indications; our reliance on third-party
manufacturers, and on specialty pharmacies for the distribution of Iclusig;
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 and results of
preclinical and clinical studies of our product candidates; difficulties or
delays in obtaining regulatory approvals to market products or market access
in certain countries; the adequacy of our capital resources and the
availability of additional funding; patent protection and third-party
intellectual property claims; our failure to comply with extensive regulatory
requirements; the occurrence of serious adverse events in patients being
treated with Iclusig or our product candidates; risks related to key
employees, markets, economic conditions, health care reform, prices and
reimbursement rates; and other risk 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.

Reference:

1. Rohrbacher M, Hasford J. Epidemiology of chronic myeloid leukaemia (CML).
Best Pract Res Clin Haematol. 2009 Sep;22(3):295-302. Based on current
estimate of population of Europe (738,199,000 in 2010).

Contact:

For Investors
ARIAD Pharmaceuticals
Kendra Adams, 617-503-7028
Kendra.adams@ariad.com
or
For U.S. Media
ARIAD Pharmaceuticals
Liza Heapes, 617-621-2315
liza.heapes@ariad.com
or
For EU Media
Heather Grant, +44 (0) 207 632 1873
hgrant@biosector2.co.uk
 
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