|ARIAD Begins Phase 1/2 Trial of Ponatinib in Japan|
Trial to evaluate ponatinib in Japanese patients with CML who are resistant or intolerant to prior therapy
“Many of the world’s foremost thought leaders on CML are in
The Phase 1 dose-escalation portion of the trial will have two dose cohorts: 30 mg and 45 mg administered orally once daily. At least six patients will be enrolled in each dose cohort. Once the recommended dose of ponatinib for Japanese patients is determined and safety evaluations have been completed, the Phase 2 portion of the trial will be initiated. The first patient in the trial has been dosed.
The single-arm, open-label Phase 2 component of the trial is expected to begin in the first quarter of 2013 and to enroll an additional 25 adult patients. ARIAD expects to fully enroll the study by mid-year 2013. In the Phase 2 portion of the trial, the primary efficacy endpoint for CML patients in chronic phase will be major cytogenetic response. For CML patients in accelerated phase or blast phase and for Ph+ ALL patients, the primary endpoint will be major hematologic response, defined as complete hematologic response or no evidence of leukemia. The study’s secondary endpoints for all patients will include major molecular response, time to response, duration of response, progression-free survival and overall survival.
For more information about the trial, patients and physicians should visit http://clinicaltrials.gov/ct2/show/NCT01667133?term=ponatinib&rank=1, call the U.S. toll-free number 1-877-621-2302 or the international number 1-617-621-2302, or e-mail inquiries to firstname.lastname@example.org.
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 and PDGFR families of kinases. A New Drug Application for ponatinib
was submitted to the
The primary target for ponatinib is BCR-ABL, an abnormal tyrosine kinase
that is expressed in chronic myeloid leukemia (CML) and
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 or 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 tyrosine kinase inhibitors. Because both of these diseases express the BCR-ABL protein, this would render them potentially susceptible to treatment with ponatinib.
This press release contains “forward-looking statements” including, but
not limited to, the timing of commencement of clinical trials for
ponatinib. 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