ARIAD announced the initiation of a Phase 2 clinical trial of its investigational pan-BCR-ABL inhibitor, ponatinib (previously known as AP24534), in patients with resistant or intolerant chronic myeloid leukemia (CML) and Philadelphia positive acute lymphoblastic leukemia (Ph+ ALL). The PACE (Ponatinib Ph+ ALL and CML Evaluation) trial is designed to provide definitive clinical data for regulatory approval of ponatinib in this setting. Ponatinib has been granted orphan drug status in the United States for the treatment of CML and Ph+ ALL. The PACE trial is a global, single-arm clinical study of oral ponatinib in 320 patients with chronic phase, accelerated phase, or blast phase CML, as well as Ph+ ALL. Patients resistant or intolerant to Sprycel (dasatinib) or Tasigna (nilotinib), or with T315I mutation of BCR-ABL, will be enrolled in the trial. Patients will be grouped into one of six separate cohorts based on their phase of CML (i.e., chronic, accelerated or blast) and BCR-ABL mutation status (i.e., with or without the T315I mutation); Ph+ ALL patients will be grouped with blast phase CML. A total of 160 patients with chronic phase CML will be included in over 60 sites. The primary endpoints are major cytogenetic response rate for chronic phase patients and major hematologic response rate for accelerated or blast phase CML patients and Ph+ ALL patients. Patients will receive ponatinib in tablet form once daily at a dose of 45 mg.

Ponatinib is an oral multi-targeted kinase inhibitor being developed for the treatment of hematological cancers. 

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