Ponatinib: Additional Phase I data

Data from a subset of 43 patients with chronic-phase CML in an open-label, dose-escalation, U.S. Phase I trial showed that ponatinib produced a 98% complete hematologic response rate. Additionally, 72% of patients in the subgroup had a major cytogenetic response (MCyR) and 44% had a major molecular response (MMR). In 12 patients with chronic-phase CML with the T315l mutation of BCR-ABL tyrosine kinase, 100% had a complete hematologic response and 92% had a MCyR. In 13 patients with chronic-phase CML with no detectable mutations, 100% had a complete hematologic response and 62% had a MCyR. Furthermore, in 22 patients with accelerated-phase or blast-phase CML or Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL), 36% had a major hematologic response and 32% had a MCyR. The trial enrolled 81 patients with resistant hematologic cancers, including 60 with CML and 5 with Ph+ ALL. Patients received once-daily ponatinib at doses ranging from 2 to 60 mg. Data were published in the New England Journal of Medicine. ...