Ponatinib Helps Refractory Leukemia in Nearly Half of Patients

December 13, 2011

By Deena Beasley

SAN DIEGO (Reuters) Dec 12 - Early results from a phase III trial of Ariad Pharmaceuticals Inc's leukemia drug ponatinib show it is effective in nearly half of patients who had stopped responding to currently available drugs.

An interim look at the study, presented here at a meeting of the American Society of Hematology, shows that 47% of patients with chronic myeloid leukemia (CML) in chronic phase had a major response to ponatinib, meaning that at least two-thirds of their bone marrow was normal.

Thirty-nine percent of those patients achieved complete remission of their leukemia.

The trial involves 449 patients -- the interim results are from 392 subjects -- who had stopped responding to treatment with Sprycel (dasatinib) and Tasigna (nilotinib), both of which are tyrosine kinase inhibitors.

Ariad's drug is designed to target an abnormal tyrosine kinase that is closely associated with CML and Philadelphia chromosome positive acute lymphoblastic leukemia.

Side effects seen in the trial included rash (in 32% of patients), thrombocytopenia (in 31%), dry skin (24%), abdominal pain (19%), and headache (17%).

Ariad said 6% of patients developed pancreatitis but were able to remain in the trial.

Researchers said the deaths of four patients, all of whom had advanced leukemia and other serious medical conditions, may have been related to treatment with ponatinib.

"The side effect profile is every bit as good as other drugs in this class," said Ariad Chief Executive Officer Harvey Berger.

Ariad plans to file for U.S. regulatory approval of ponatinib in mid-2012.

Berger also said next year Ariad will launch a trial of ponatinib in newly-diagnosed patients.


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