ARIAD Announces Submission of Marketing Authorization Application for Ponatinib to the European Medicines Agency
ARIAD Pharmaceuticals, Inc.Kendra Adams, 805-231-4070orLiza Heapes, 617-621-2315
(NASDAQ: ARIA) today announced the submission of a Marketing Authorization Application (MAA) for its investigational BCR-ABL inhibitor, ponatinib, to the European Medicines Agency (EMA). ARIAD is seeking marketing approval in the European Union of ponatinib in adult patients with resistant or intolerant chronic myeloid leukemia (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL). The Committee for Medicinal Products for Human Use (CHMP) has granted ARIAD’s request for accelerated assessment of the MAA.
“The accelerated assessment granted to our MAA further illustrates the major unmet medical need among patients with CML and Ph+ ALL who have become resistant or intolerant to prior tyrosine kinase inhibitor therapy,” stated , chairman and chief executive officer of ARIAD. “As we establish our European headquarters in Lausanne, Switzerland and develop our commercial infrastructure in the region, our regulatory team will continue to work closely with the EMA as it reviews our data and the submission, potentially along an accelerated timeline.”
Results from the ongoing PACE trial of ponatinib reported in June at this year’s annual meeting of the American Society of Clinical Oncology, and included in the MAA, showed that 54 percent of chronic-phase CML patients who were resistant or intolerant to prior tyrosine kinase inhibitor therapy in the trial, including 70 percent of patients who have a T315I mutation, achieved a major cytogenetic response (MCyR) – the primary endpoint of the trial. Thirty percent of these same patients achieved a major molecular response (MMR). MMR is the primary endpoint of ARIAD’s ongoing Phase 3 EPIC trial comparing ponatinib to imatinib in newly diagnosed chronic-phase CML patients.
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 U.S. Food and Drug Administration on July 30, 2012.
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 very 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.
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.
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 or difficult-to-treat cancers, including chronic myeloid leukemia and certain forms of non-small cell lung cancer. For additional information, visit or follow ARIAD on (@ARIADPharm).
This press release contains “forward-looking statements” including, but not limited to, statements relating to the clinical data for ponatinib, submission, review, and potential approval of the MAA, and timing of other 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.