ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA), today announced that
Health Canada has approved the use of Iclusig™ (as ponatinib
hydrochloride) in Canada for the treatment of adult patients with
all phases of chronic myeloid leukemia (CML) or Philadelphia
chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) for whom
other tyrosine kinase inhibitor (TKI) therapy is not appropriate,
including CML or Ph+ ALL that is T315I mutation positive, or where
there is prior TKI resistance or intolerance.
“Patients with CML or Ph+ ALL can become resistant to their
therapies over time,” said Professor Jeffrey Lipton, Ph.D., M.D.,
staff physician at The Princess Margaret Cancer Centre and one of
the PACE trial investigators. “Iclusig will be a valuable new
therapeutic option in Canada for patients with refractory CML and
Ph+ ALL, where we have a proven unmet medical need.”
Iclusig is approved under the Notice of Compliance with
Conditions (NOC/c) policy based on promising evidence of clinical
effectiveness following review by Health Canada. Products approved
under this policy are intended for the treatment, prevention or
diagnosis of a serious, life-threatening or severely debilitating
illness, and have demonstrated promising benefit, are of high
quality and possess an acceptable safety profile based on a
benefit/risk assessment. In addition, these products either respond
to a serious unmet medical need in Canada or have demonstrated a
significant improvement in the benefit/risk profile over existing
therapies.
“Throughout the development of Iclusig, we have worked closely
with leading academic centers and clinical investigators in Canada.
Iclusig is the only TKI that has been approved in Canada for
indications that include CML and Ph+ ALL patients with the T315I
mutation,” stated Harvey J. Berger, M.D., chairman and chief
executive officer of ARIAD. “We will strive to make Iclusig
available as quickly as possible to appropriate patients with Ph+
leukemias throughout Canada.”
Iclusig will be made available through a controlled distribution
program. Under this program, prescribers who have completed the
certification procedure are able to prescribe Iclusig. Trained
pharmacies will verify the prescriber’s certified status prior to
dispensing Iclusig to the patient.
“Iclusig provides a new treatment option for patients with
difficult-to-treat CML or Ph+ ALL who previously had limited
therapies available to them,” said Cheryl-Anne Simoneau, president
and chief executive officer at the Chronic Myelogenous Leukemia
Society of Canada. “For appropriate patients, this can be an
important new treatment option.”
The Health Canada decision was based on two-year data from the
pivotal Phase 2 PACE trial in patients with CML or Ph+ ALL who were
resistant or intolerant to prior TKI therapy, or who had the T315I
mutation of BCR-ABL. Iclusig demonstrated anti-leukemic activity,
achieving a major cytogenetic response (MCyR) in 56 percent of
chronic-phase CML patients and in 70 percent of patients with the
T315I mutation. MCyR within the first 12 months of treatment was
the primary endpoint of the PACE trial for chronic-phase
patients.
In patients with advanced disease, 57 percent of
accelerated-phase CML patients and 31 percent of blast-phase CML
patients achieved a major hematologic response (MaHR) with Iclusig.
MaHR within the first 6 months was the primary endpoint in the
trial for patients with advanced disease. In patients with Ph+ ALL,
41 percent achieved MaHR.
ARIAD’s upcoming dose-ranging trial to evaluate three starting
doses of Iclusig in patients with refractory, chronic-phase CML who
are resistant to at least two approved TKIs -- planned to begin in
mid-2015 -- will serve as the confirmatory trial for the Health
Canada approval.
About CML and Ph+ ALL
CML is a cancer of the white blood cells that according to the
Chronic Myelogenous Leukemia Society of Canada affects 1 in
100,000, with about 5,500 Canadians living with the disease. In
2010, more than 550 people in Canada were expected to be diagnosed
with CML, and 480 people were expected to be diagnosed with
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
leukemia 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 TKIs. The BCR-ABL protein is
expressed in both of these diseases.
About Iclusig™ (as ponatinib hydrochloride)
Iclusig is a kinase inhibitor. The primary target for Iclusig is
BCR-ABL, an abnormal tyrosine kinase that is expressed in chronic
myeloid leukemia (CML) and Philadelphia-chromosome positive acute
lymphoblastic leukemia (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
not only native BCR-ABL but also its isoforms that carry mutations
that confer resistance to treatment, including the T315I mutation,
which has been associated with resistance to other approved
TKIs.
Indications
ICLUSIG is indicated for the treatment of adult patients with
chronic phase, accelerated phase, or blast phase chronic myeloid
leukemia (CML) or Philadelphia chromosome positive acute
lymphoblastic leukemia (Ph+ ALL) for whom other tyrosine kinase
inhibitor (TKI) therapy is not appropriate, including CML or Ph+
ALL that is T315I mutation positive or where there is prior TKI
resistance or intolerance.
Marketing authorization with conditions is based on response
rate. There are no trials demonstrating increased survival or
improvement in symptoms with ICLUSIG. In the pivotal trial, the
majority of the hematological responses occurred within 1 month.
Consider discontinuing ICLUSIG if a hematological response has not
been achieved by 3 months (90 days).
ICLUSIG for this indication has been issued marketing
authorization with conditions, pending the results of studies to
verify its clinical benefit. Patients should be advised of the
conditional nature of the authorization.
Contraindications
- Do not use in patients who are
hypersensitive to ponatinib or to any ingredient in the formulation
or component of the container. For a complete listing, see the
DOSAGE FORMS, COMPOSITION AND PACKAGING section of the Product
Monograph.
- Do not use in patients who have
unmanaged cardiovascular risk factors, including uncontrolled
hypertension. Hypertension may contribute to the risk of arterial
thrombotic events. Blood pressure should be monitored and managed
to avoid hypertension.
- Do not use in patients who are not
adequately hydrated and with uncorrected high uric acid
levels.
Serious Warnings and Precautions
Iclusig has serious warnings and precautions for: vascular
occlusion, heart failure, hemorrhage, hepatotoxicity,
myelosuppression, and pancreatitis.
ICLUSIG should only be prescribed and monitored by a physician
who has completed the certification with the ICLUSIG Controlled
Distribution Program and who is experienced in the use of
antineoplastic therapy and in the treatment of CML or Ph+ ALL.
- Vascular Occlusion (arterial and venous
thrombosis and occlusions), occurred in 24% (129/530) of
ICLUSIG-treated patients with and without cardiovascular risk
factors (including patients less than 50 years old). In clinical
trials, serious treatment-emergent arterial thrombosis
(cardiovascular, cerebrovascular, and peripheral vascular) and
occlusions were seen in 14% of the ICLUSIG-treated patients
including fatal myocardial infarction, fatal cerebral infarction,
stroke, disseminated intravascular coagulation, and arterial
stenosis sometimes requiring urgent revascularization procedures.
Some of these events occurred within 2 weeks of starting treatment
with ICLUSIG. Monitor for evidence of thromboembolism and vascular
occlusion. Interrupt or consider discontinuation in patients who
develop arterial thrombotic events.
- Heart Failure (in some cases, fatal),
including left ventricular dysfunction and ejection fraction
decreases, occurred in 8% of ICLUSIG-treated patients, 5% of which
were serious.
- Hemorrhage events (some fatal)
including intracranial hemorrhage, hemorrhagic gastritis, (fatal),
hemorrhagic cerebral infarction (fatal). Most hemorrhagic events,
but not all, occurred in patients with grade 4
thrombocytopenia.
- Hepatotoxicity (including fatal acute
hepatic failure) has been reported. Monitor hepatic function prior
to and during treatment. Consider ICLUSIG dose interruption
followed by dose reduction or discontinuation in patients with
hepatotoxicity.
- Myelosuppression (thrombocytopenia,
neutropenia, and anemia).
- Pancreatitis (7%) and elevations in
amylase (2% grade 3 or greater) or lipase (12% grade 3 or greater)
have been reported.
- ICLUSIG has not been studied in
patients with renal impairment.
Most Common Adverse Reactions
Overall, the very common adverse reactions (≥ 10%) were platelet
count decreased, rash, dry skin, abdominal pain, neutrophil count
decreased, headache, lipase increased, fatigue, constipation,
myalgia, arthralgia, nausea, anemia, ALT increased, hypertension,
and AST increased.
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.com or follow ARIAD on Twitter
(@ARIADPharm).
Forward-Looking Statements
This press release contains forward-looking statements. Any
statements contained herein which do not describe historical facts,
including but not limited to, statements regarding: the therapeutic
potential of Iclusig and the timing of making Iclusig available in
Canada; the expected timing for commencing a confirmatory trial for
this approval in Canada; and our efforts related to new oncology
treatments, are forward-looking statements which are based on
management's good faith expectations and involve risks and
uncertainties that could cause actual results to differ materially
from those discussed in such forward-looking statements. These
factors, risks and uncertainties include, but are not limited to,
our ability to obtain pricing and reimbursement approval for
Iclusig in Canada and the level of any such pricing and
reimbursement, including reimbursement from third parties;
post-marketing regulatory requirements; the occurrence of adverse
safety events with our products; our ability to successfully
distribute, promote, market and sell Iclusig in Canada; additional
clinical trials of Iclusig, including our confirmatory trial for
this approval, may produce negative or inconclusive results or may
not be initiated or conducted in a timely manner; the costs
associated with ARIAD’s development and manufacturing, commercial
and other activities; our ability to maintain and enforce
intellectual property protection for Iclusig; the fact that drug
discovery and development is complex, time consuming, expensive and
fraught with a high risk of failure; technical difficulties,
excessive costs or other issues relating to the manufacture or
supply of Iclusig; the adequacy of 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. After the
date of this document, the Company does not intend to update any of
the forward-looking statements to conform to actual results or to
changes in the Company's expectations, except as required by
law.
Iclusig™ is a trademark of ARIAD Pharmaceuticals, Inc.
For ARIAD InvestorsKendra Adams,
617-503-7028Kendra.adams@ariad.comorFor ARIAD MediaLiza
Heapes, 617-621-2315Liza.heapes@ariad.com
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